Q2 2024 Sarepta Therapeutics Inc Earnings Call

August 7, 2024

Okay.

Operator: Good day, and thank you for standing by, welcome to the Sarepta Therapeutics second quarter 2024 financial results conference call. At this time, all participants are in a listen-only mode, after the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 11 on your telephone, you will then hear an automated message advising you to raise your hand. To withdraw your question, please press star 11 again. Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker for today, Mary Jenkins, Associate Director, Investments Relations and Corporate Communications. Please go ahead.

Speaker Change: Good day, and thank you for standing by welcome to the soft up there.

Speaker Change: Therapeutics second quarter 2024 financial results conference call.

Operator: At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 11 on your telephone. You will then hear an automated message advising you to raise your hand. To withdraw your question, please press star 11 again. Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker for today, Mary Jenkins, Associate Director, Investments Relations and Corporate Communications. Please go ahead.

Speaker Change: At this time all participants are in a listen only mode.

Speaker Change: After the Speakers' presentation there'll be a question answer session.

Speaker Change: To ask a question during the session you will need to press star one on your telephone.

Speaker Change: You will then hear an automated message and a bias in your hand, just raced to withdraw your question. Please press star one again please.

Speaker Change: Please be advised that today's conference is being recorded I would now like to turn the conference over to your speaker for today Mary Jenkins.

Mary Jenkins: Associate Director Investor Relations and corporate Communications. Please go ahead.

Mary Jenkins: Thank you, Lisa, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the second quarter of 2024, the press release is available on our website at Sarepta.com, and our 10-Q was filed with the Securities and Exchange Commission this afternoon. Joining us on the call today are Doug Ingram, Ian Estepan, Dallan Murray, and Dr. Louise Rodino-Klapack. After our formal remarks, we'll open the call for Q&A. I'd like to note that during the call, we'll be making a number of forward-looking statements, please take a moment to review our slide on the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements, and any such risks can materially and adversely affect the business, the results of operations, and trading prices for Sarepta's common stock.

Mary Jenkins: Thank you Lisa and thank you all for joining today's call earlier. This afternoon, we released our.

Speaker Change: Financial results for the second quarter 'twenty 'twenty four the press release is available on our website a threat to that Com and our 10-Q was filed with the Securities and Exchange Commission. This afternoon Tony.

Speaker Change: Joining us on the call today are that demand and at the parent boundary and Dr. Louise I can't quite back after our formal remarks, we'll open the call for Q&A.

Mary Jenkins: After our formal remarks, we'll open the call for Q&A. I'd like to note that during the call, we'll be making a number of forward-looking statements. Please take a moment to review our slide on the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements, and any such risks can materially and adversely affect the business, the results of operations, and trading prices for Sarepta's common stock.

Speaker Change: Note that during the call, we'll be making a number of forward looking statements. Please take a moment to review our slide on the webcast, which contains our forward looking statements. These forward looking statements involve risks and uncertainties many of which are beyond throughout this control actual results could materially differ from these forward looking statements and any such risks and can materially and adversely affect the does.

Mary Jenkins: These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements, and any such risks can materially and adversely affect the business, the results of operations, and trading prices for Sarepta's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual report on Form 10-Q filed with the SEC, as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today, based on subsequent events or circumstances. And now I'll turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress, Doug.

Speaker Change: That's the result of operations and trading prices for scrap this common stock.

Mary Jenkins: For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual report on Form 10-Q filed with the SEC, as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today, based on subsequent events or circumstances. And now I'll turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress.

Speaker Change: For a detailed description of applicable risks and uncertainties. We encourage you to review the company's most recent annual report on Form 10-Q filed with the SEC as well as the company's other SEC filings. The company does not undertake any obligation to publicly update as far as looking statements, including any financial projections provided today based on subsequent events or circumstances and now I'll turn the call over.

Mary Jenkins: The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today, based on subsequent events or circumstances. And now I'll turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress, Doug.

Speaker Change: Our president and CEO, Doug Ingram, who will provide an overview of our recent progress.

Doug Ingram: Thank you Mary.

Douglas S. Ingram: Thank you, Mary, good afternoon, everyone, and thank you for joining Sarepta Therapeutics' second quarter 2024 Financial Results Conference call. I joined Sarepta a little over seven years ago for one reason, I believe that Sarepta has the greatest hope of developing medicines that could improve the lives of a majority of Duchenne patients, and I wanted to be part of that effort. We've had many accomplishments over the last seven years, successfully launching four life-changing therapies and, through an obsessive attention to detail, making all of those launches very successful. We have grown revenue at about 150% CAGR from the beginning of 2017 through the end of 2023. We've built a deep pipeline.

Doug Ingram: Good afternoon, everyone and thank you for joining through up at Therapeutics second quarter 2024 financial results Conference call.

Douglas S. Ingram: We have grown revenue at about 150% CAGR from the beginning of 2017 through the end of 2023. We've built a deep pipeline, we've established ourselves as the leaders in RNA and gene therapy for rare diseases, and we have become profitable, and as of this quarter, we are cashflow positive. But it was only in the second quarter of this year that we can finally say that we have reached that objective that brought me and so many of my compatriots to Sarepta. Because it was in June of 2024 that we finally obtained broad approval to make ELEVIDYS available to over 80% of patients living with, and dying from, Duchenne Muscular Dystrophy in the United States.

Douglas Ingram: We've established ourselves as the leaders in RNA and gene therapy for rare diseases. And we have become profitable, and as of this quarter, we are cashflow positive. But it was only in the second quarter of this year that we can finally say that we have reached that objective that has brought me and so many of my compatriots to Sarepta. Because it was in June of 2024 that we finally obtained broad approval to make Levitas available to over 80% of patients living with and dying from Duchenne muscular dystrophy in the United States.

Doug Ingram: I joist were up a little over seven years ago for one reason I believe that's where up to have the greatest hope.

Speaker Change: Developing medicines that could improve the lives of the majority of Duchenne patients and I wanted to be part of that effort.

Speaker Change: We had many accomplishments over the last seven years successfully launching four life changing therapies and through an obsessive attention to detail, making all of those launches very successful we have grown revenue at about 150% CAGR from the beginning of 2017 through 23, we built a deep pipeline.

Speaker Change: Established ourselves as the leaders in RNA and gene therapy for rare disease, and we have become profitable and as of this quarter. We are cash flow positive, but it was only in the second quarter of this year that we can finally say that we have reached that objected. They brought me and so many of my compatriots to stir up.

Douglas S. Ingram: Because it was in June of 2024 that we finally obtained broad approval to make ELEVIDYS available to over 80% of patients living with, and dying from, Duchenne Muscular Dystrophy in the United States. As you are aware, in June of this year, the FDA granted traditional approval for all ambulant patients four and older, and consistent with every other Duchenne approval in history, also granted approval for the treatment of non-ambulatory patients on an accelerated basis. Also in June, our partner Roche announced that the European Medicines Agency had accepted the ELEVIDYS submission for review. Roche also has obtained six ex-US approvals so far, and is making good progress serving those communities. So here we are, launching the largest gene therapy yet approved, with the first opportunity yet in history to bring a dystrophin restorative therapy to the vast majority of Duchenne. Anyone who has been watching over the last seven plus years will realize that this is exactly what we are particularly good at.

Speaker Change: Because it was in June of 2024 that we finally obtained a broad approval to make elaborate its available to over 80% of patients living with a dying from duchenne muscular dystrophy.

Douglas Ingram: As you are aware, in June of this year, the FDA granted traditional approval for all ambulant patients four and older, and consistent with every other Duchenne approval in history, also granted approval for the treatment of non-ambulatory patients on an accelerated basis.

Speaker Change: In the United States as you are aware in June June of this year. The FDA granted traditional approval for all ambulant patients for an older and consistent with every other day shut approval in history also granted approval for the treatment of non ambulatory patients on an accelerated basis also in June our.

Douglas Ingram: Also in June, our partner Roche announced that the European Medicines Agency had accepted the Elevitist submission for review. Roche also has obtained six SXUS approvals so far and is making good progress with those. So here we are, launching the largest gene therapy yet approved, with the first opportunity yet in history to bring a dystrophin restorative therapy to the vast majority of Duchenne. Anyone who has been watching over the last seven plus years will realize that this is exactly what we are particularly good at.

Speaker Change: Partner Roche announced that the European Medicines agency had accepted the elaborate submission for review Roche also has obtained six ex U S approvals, so far and it is making good progress serving those communities.

Douglas S. Ingram: So here we are, launching the largest gene therapy yet approved, with the first opportunity yet in history to bring a dystrophin restorative therapy to the vast majority of Duchenne. Anyone who has been watching over the last seven plus years will realize that this is exactly what we are particularly good at. Certainly, we are great at developing therapies for rare diseases, and we are great at managing the process to get them approved, and we have become exceptional at managing complex manufacturing and distribution. But perhaps, above all else, we are second to no one in the world at launching Duchenne Therapies, working with payers, and ensuring actions. To that point, in the second quarter, we achieved net product revenue of approximately $361 million. That's a 51% increase over the same quarter last year. Elevitas came in at $121.7 million in the second quarter, in line with our earlier forecast.

Speaker Change: So here, we are launching the largest gene therapy yet approved.

Speaker Change: With the first opportunity yet in history to bring a district in the restorative therapy to the vast majority of Duchenne patients.

Speaker Change: Any one who has been watching over the last seven plus years, we'll realize.

Speaker Change: But this is exactly what we are particularly good at certainly we are great at developing therapies for rare disease, and we are great at managing the process to get them approved and we have become exceptional at managing complex manufacturing and distribution.

Douglas Ingram: Certainly, we are great at developing therapies for rare diseases, and we are great at managing the process to get them approved, and we have become exceptional at managing complex manufacturing and distribution. But perhaps, above all else, we are second to no one in the world at launching Duchenne Therapies, working with payers, and ensuring action. To that point, in the second quarter, we achieved net product revenue of approximately $361 million. That's a 51% increase over the same quarter last year. Elevitas came in at $121.7 million in the second quarter, in line with our earlier forecast.

Douglas S. Ingram: But perhaps, above all else, we are second to no one in the world at launching Duchenne Therapies, working with payers, and ensuring actions. To that point, in the second quarter, we achieved net product revenue of approximately $361 million dollars, that's a 51% increase over the same quarter last year, ELEVIDYS came in at $121.7 million dollars in the second quarter, in line with our earlier forecast. Even with an exceptionally narrow and restrictive label, in the first full 12 months of launch, we achieved approximately $456 million dollars, more than doubling all other gene therapies launched in the last few years combined. As we have noted previously with the broader label granted in June of this year, the opportunity to serve patients and, in so doing, reward committed investors will be enormous. Our early launch has exceeded even our optimistic expectations, all signals are currently positive, from physician and patient demand to enrollment forms to assay kit ordering to positive payer interaction.

Speaker Change: Perhaps above all else we are second to no one in the world.

Speaker Change: Launching duchenne therapies, working with payers and ensuring access to that point in the second quarter. We achieved net product revenue of approximately $361 million, that's a 51% increase over the same quarter last year.

Speaker Change: <unk> came in at $121 $7 million in the second quarter in line with our earlier forecast, even with an exceptionally narrow and restrictive label in the first full 12 months of launch we achieved approximately $456 million more than doubling.

Douglas Ingram: Even with an exceptionally narrow and restrictive label, in the first full 12 months of launch, we achieved approximately $456 million, more than doubling the amount of all other gene therapies launched in the last few years combined. As we have noted previously with the broader label granted in June of this year, the opportunity to serve patients and, in so doing, reward committed investors will be enormous. Our early launch has exceeded even our optimistic expectations. All signals are currently positive, from physician and patient demand to enrollment forms to assay kit ordering to positive payer interaction.

Speaker Change: All other gene therapies launched in the last few years combined.

Douglas S. Ingram: As we have noted previously with the broader label granted in June of this year, the opportunity to serve patients and, in so doing, reward committed investors will be enormous. Our early launch has exceeded even our optimistic expectations, all signals are currently positive, from physician and patient demand, to enrollment forms, to assay kit ordering, to positive payer interaction. To manage near-term expectations, let me note again that the process from enrollment form to infusion will typically take between three and five months, so we will see moderate revenue growth in the third quarter, but we anticipate strong growth, commencing in the fourth quarter of this year. And then exiting this year, 2024, will be a very strong year of growth, we currently anticipate that our 2025 net product revenue across our four approved therapies will come in between $2.9 billion dollars, and $3.1 billion dollars, and that is still years away from peak year sales.

Speaker Change: As we have noted previously with the broader label granted in June of this year the opportunity to serve patients and in so doing reward committed investors will be enormous our early launch has exceeded even our optimistic expectations. All signals are currently positive from physician and patient demand to enroll.

Speaker Change: It forms to assay kit ordering to positive payor interactions.

Douglas Ingram: To manage near-term expectations, let me note again that the process from enrollment form to infusion will typically take between three and five months. So we will see moderate revenue growth in the third quarter, but we anticipate strong growth beginning in the fourth quarter of this year, and then exiting this year, 2024, will be a very strong year of growth. We currently anticipate that our 2025 net product revenue across our four approved therapies will come in between $2.9 billion and $3.1 billion, and that is still years away from peak year sales.

Speaker Change: To manage near term expectations, let me note again that the protests from enrollment form to infusion will typically take.

Speaker Change: <unk> three and five months, so we will see moderate revenue growth in the third quarter, but we anticipate strong growth commencing in the fourth quarter of this year and then exiting this year 2024 will be a very strong year of growth.

Douglas S. Ingram: And then exiting this year, 2024, will be a very strong year of growth, we currently anticipate that our 2025 net product revenue across our four approved therapies will come in between $2.9 billion dollars, and $3.1 billion dollars, and that is still years away from peak year sales. Indeed, we will be treating the prevalent population over the course of this entire decade before moving to the incident population in 2030 to 2031, Dallan Murray, our Chief Customer Officer, will provide more color on the launch in just a moment. Our Head of R&D and Chief Scientific Officer, Dr. Louise Rodino-Klapak, will then discuss our R&D progress from last quarter, including our plans for presenting multiple data sets from across the SRP-9001 clinical program at the World Muscle Society Congress later this year, and she will also provide updates on the advancement of our LGMD programs. I look forward to updating you across the year on our launch performance, and the advancement of our pipeline, and with that I will turn the call to Dallan Murray for more details on commercial performance and plans. Dallan.

Speaker Change: Currently anticipate that our 2025 net product revenue across our four approved therapies will come in between $2 9 billion and $3 1 billion and that is still years away from the peak year sales. Indeed, we will be treating the prevalent population over the.

Douglas Ingram: Indeed, we will be treating the prevalent population over the course of this entire decade before moving to the incident population in 2030 to 2031. Dallan Murray, our Chief Customer Officer, will provide more color on the launch in just a moment. Our Head of R&D and Chief Scientific Officer, Dr. Louise Rodino-Klapak, will then discuss our R&D progress from last quarter, including our plans for presenting multiple data sets from across the SRP 9001 clinical program at the World Muscle Society Congress later this year, and she will also provide updates on the advancement of our LGMD program.

Speaker Change: Of course of the entire decade before moving to the incident population.

Speaker Change: $30 to 2031, Alan Murray, our Chief customer Officer, who will provide more color on the launch in just a moment, our head of R&D and Chief Scientific Officer, Dr. Louise Rodino quite back will then discuss our R&D progress from last quarter, including our plans for presenting multiple data sets from across the SRP nine zero.

Douglas S. Ingram: Our Head of R&D and Chief Scientific Officer, Dr. Louise Rodino-Klapak, will then discuss our R&D progress from last quarter, including our plans for presenting multiple data sets from across the SRP-9001 clinical program at the World Muscle Society Congress later this year, and she will also provide updates on the advancement of our LGMD programs. I look forward to updating you across the year on our launch performance, and the advancement of our pipeline, and with that I will turn the call to Dallan Murray for more details on commercial performance and plans. Dallan.

Speaker Change: One clinical program at the World Muscle Society Congress later this year.

Speaker Change: He will also provide updates on the advancement of our L. G. M. D programs I look forward to updating you across the year on our launch performance and the advancement of our pipeline and with that I will turn the call to Dallas Murray for more details on commercial performance and plans.

Speaker Change: Alan.

Dallan: Thank you, Doug, and good afternoon, as Doug outlined, we generated roughly $361 million in net product revenues from our Duchenne franchise in the second quarter of 2024. Q2 was the last quarter in which we would have executed on the limited 45 year old label for ELEVIDYS, given that we received a broad label on June 20th. As we had expected the Net Product Revenue for ELEVIDYS was essentially flat when compared to each of the two prior quarters. Off note, a few patients rescheduled doses at the end of the second quarter which resulted in dose infusion being completed fully in the third quarter. Net product revenue for the PMO franchise in Q2 was roughly $239 million, which represented modest growth over Q1. Going forward, the team remains committed to supporting those existing PMO patients who have been remarkably compliant with their weekly PMO regimen for years now. We're confident in their ability to manage this, given the fact that these are the same centers who navigated all of the recent Duchenne and SMA launches, including Zildjian's. Your uptake assumptions should reflect the patient's journey to obtain an infused gene therapy. We're only several weeks into this new launch.

Dallan Murray: Thank you, Doug, and good afternoon, as Doug outlined, we generated roughly $361 million in net product revenues from our Duchenne franchise in the second quarter of 2024. Q2 was the last quarter in which we would have executed on the limited 45 year old label for ELEVIDYS, given that we received a broad label on June 20th. As we had expected the Net Product Revenue for ELEVIDYS was essentially flat when compared to each of the two prior quarters. Off note, a few patients rescheduled doses at the end of the second quarter which resulted in dose infusion being completed fully in the third quarter. Net product revenue for ELEVIDYS was nearly $122 millions for the quarter.

Dallas Murray: Thank you Doug and good afternoon.

Speaker Change: Doug outlined we generated roughly 361 million of net product revenues from our Duchenne franchise in the second quarter of 2024.

Speaker Change: Q2 is the last quarter in which we will have executed on the limited four to five year old label for <unk>.

Speaker Change: Given that we received a broad label on June 20th.

Speaker Change: As we had expected the net product revenue for <unk> was essentially flat when compared to each of the two prior quarters.

Speaker Change: Of note a few patients rescheduled doses at the end of the second quarter.

Speaker Change: Which resulted in those infusions being completed early in the third quarter.

Speaker Change: Net product revenues for our loved us for nearly $122 million for the quarter.

Speaker Change: Net product revenue for the PMO franchise in Q2 was roughly $239 million, which represented modest growth over Q1.

Speaker Change: Going forward the team remains committed to supporting those existing PMO patients who've been remarkably compliant with their weekly PMO regimen for years now.

Speaker Change: And while we expect some cannibalization by <unk> in the U S.

Dallan Murray: Net product revenue for the PMO franchise in Q2 was roughly $239 million, which represented modest growth over Q1. Going forward, the team remains committed to supporting those existing PMO patients who have been remarkably compliant with their weekly PMO regimen for years now. And while we expect some cannibalization by ELEVIDYS in the US in the coming quarters, ex-US [inaudible] growth will help to mitigate this cannibalization. Now turning to the ELEVIDYS launch, we¡re pleased with the launch progress to date, and are on track to realize the opportunity in front of us. To put the current situation into perspective, almost the entire Duchenne population became eligible for ELEVIDYS essentially over night, what we're seeing right now is the key neuromuscular centers reacting to unprecedented demand from the entirety of their Duchenne patient populations. The treating sites are rapidly working through with prioritizing patient demand, we're confident in their ability to manage this, given the fact that these are the same centers who navigated all of the recent Duchenne and SMA launches, including Zolgensma, your uptake assumptions should reflect the patient's journey to obtain an infused gene therapy. We're only several weeks into this new launch.

Speaker Change: In the coming quarters ex U S. PLO growth will help to mitigate this cannibalization.

Speaker Change: Now turning to the 11th this launch we're pleased with the launch progress to date.

Speaker Change: And are on track to realize the opportunity in front of us.

Speaker Change: But the current situation into perspective.

Speaker Change: The entire Duchenne population became eligible for <unk> essentially overnight.

Speaker Change: What we're seeing right now is the key neuromuscular centers reacting to unprecedented demand from the entirety of their duchenne patient populations. The treating sites are rapidly working through with prioritizing patient demand.

Dallan Murray: To put the current situation into perspective, almost the entire Duchenne population became eligible for ELEVIDYS essentially over night, what we're seeing right now is the key neuromuscular centers reacting to unprecedented demand from the entirety of their Duchenne patient populations. The treating sites are rapidly working through with prioritizing patient demand, we're confident in their ability to manage this, given the fact that these are the same centers who navigated all of the recent Duchenne and SMA launches, including Zolgensma, your uptake assumptions should reflect the patient's journey to obtain an infused gene therapy. We're only several weeks into this new launch.

Speaker Change: We're confident in their ability to manage this given the fact that these are the same centers, who navigated all of the recent duchenne.

Speaker Change: And SMA launches, including <unk>.

Speaker Change: [laughter] your uptick assumptions should reflect the patient journey.

Speaker Change: To obtain a fused gene therapy.

Dallan Murray: We're only several weeks into this new launch, however, we have some exciting successes to report, which highlight the progress the team has made in the short time we've had with the ELEVIDYS label expansion. To begin, one of the first steps in the patient's journey, and therefore an important leading indicator of demand, is screening for pre-existing antibodies. In the initial hours of the label expansion, we were nearly overwhelmed with requests for assay screening kits, despite our careful preparation, forecasting, and market insight. Our team rapidly adapted and shipped well over a thousand assay kits in the first weeks of launch.

Dallan Murray: However, we have some exciting successes to report, which highlight the progress the team has made in the short time we've had with the Levitas label expansion. To begin, one of the first steps in the patient's journey, and therefore an important leading indicator of demand, is screening for pre-existing antibodies. In the initial hours of the label expansion, we were nearly overwhelmed with requests for assay screening kits, despite our careful preparation, forecasting, and market insight. Our team rapidly adapted and shipped well over a thousand assay kits in the first weeks of launch.

Speaker Change: We're only several weeks into this new launch however, we have some exciting successes to report.

Speaker Change: Which highlight the progress the team has made in the short time, we've had with the <unk> label expansion.

Speaker Change: But again one of the first steps in the patient journey and therefore, an important leading indicator of demand is screening for pre existing antibodies.

Speaker Change: In the initial hours at the label expansion, we were nearly overwhelmed with requests for assay screening kits, despite our careful preparation forecasting and market insight.

Speaker Change: Our team rapidly adapted and shipped well over a thousand assay kits in the first weeks of launch.

Dallan Murray: As you would expect from assay kit demand, all of our other indicators show that patient demand is very robust and growing. Our market research indicates that the vast majority of Duchenne patients are already interested in seeking therapy, and we only expect this to grow with time. This demand manifested itself in the early days of the launch with a flood of patients reaching out to our key treatment sites, this has forced each center to determine the best approach to handling the unprecedented demand, and plan for how to support their patients and navigating the treatment journey. As is we're hearing form the community that some patients are presently being advised of long wait times. enrollment forms have exceeded our own [inaudible] expectations, just a few weeks into the launch, HCPs have submitted enrollment forms for patients ranging from 4 to 38 years of age, with an average age right now just over 10. It's also critical to highlight that we have the support of the broader physician community who have gained experience with ELEVIDYS over the last year in the 4 to 5 years old population. Since our initial 2023 approval, we've received enrollment forms for more than 125 HCPs form 46 states, including from 97% of our sites. And we can see early on that the physicians community are approaching label expansion, as they have our previous DMD launches, prioritizing ambulatory patients first, followed closely by the non-ambulatory, and nearly diagnosed patient populations. Off note, the early days of this gene therapy launch are following the same pattern of all of our previous Exon-Skipping launches. One of the critical success factors in any launch is that the key treatment centers have had hands-on experience with the drug, to that end, over 75% of our sites have dosed at least one patient, and we do expect this to grow now that we have a broad label. And turning to the critical issue of patient access, we've already started to see encouraging signs on the payer policy front, to date our teams have had productive and engaged meetings with payer's representing greater that 225 million patient lives, including the top commercial plans, and state medicate agencies. We anticipate it will takeI'm also pleased to highlight that as of this call, there are both commercial and Medicaid payers who have established policies covering levitate therapy for the expanded Medically Accepted Indication.

Speaker Change: As you would expect from assay kit demand all of our other indicators show that patient demand is very robust and growing our market research indicates that the vast majority of duchenne patients are already interested in seeking therapy.

Speaker Change: And we only expect this to grow with time.

Speaker Change: Yeah.

Speaker Change: This demand manifested itself in the early days of the launch with a flood of patients reaching out to our key treatment sites.

Speaker Change: This has forced each center to determine the best approach to handling the unprecedented demand and plan for how to support their patients and navigating the treatment journey.

Speaker Change: As such we are hearing from the community that some patients are presently being advised of long wait times.

Speaker Change: Enrollment forms have exceeded our own lofty expectations, just a few weeks into the launch.

Dallan Murray: As is we're hearing form the community that some patients are presently being advised of long wait times. enrollment forms have exceeded our own [inaudible] expectations, just a few weeks into the launch, HCPs have submitted enrollment forms for patients ranging from 4 to 38 years of age, with an average age right now just over 10. It's also critical to highlight that we have the support of the broader physician community who have gained experience with ELEVIDYS over the last year in the 4 to 5 years old population. Since our initial 2023 approval, we've received enrollment forms for more than 125 HCPs form 46 states, including from 97% of our sites. And we can see early on that the physicians community are approaching label expansion, as they have our previous DMD launches, prioritizing ambulatory patients first, followed closely by the non-ambulatory, and nearly diagnosed patient populations. Off note, the early days of this gene therapy launch are following the same pattern of all of our previous Exon-Skipping launches. One of the critical success factors in any launch is that the key treatment centers have had hands-on experience with the drug, to that end, over 75% of our sites have dosed at least one patient, and we do expect this to grow now that we have a broad label. And turning to the critical issue of patient access, we've already started to see encouraging signs on the payer policy front, to date our teams have had productive and engaged meetings with payer's representing greater that 225 million patient lives, including the top commercial plans, and state medicate agencies. We anticipate it will takeI'm also pleased to highlight that as of this call, there are both commercial and Medicaid payers who have established policies covering levitate therapy for the expanded Medically Accepted Indication.

Dallan Murray: As is we're hearing form the community that some patients are presently being advised of long wait times. enrollment forms have exceeded our own lofty expectations, just a few weeks into the launch, HCPs have submitted enrollment forms for patients ranging from 4 to 38 years of age, with an average age right now just over 10. It's also critical to highlight that we have the support of the broader physician community who have gained experience with ELEVIDYS over the last year in the 4 to 5 years old population.

Speaker Change: <unk> submitted enrollment forms for patients ranging from four to 38 years of age with an average age right now just over 10.

Speaker Change: It is also critical to highlight that we have the support of the broader physician community who have gained experience with 11 of us over the last year in the four to five year old population.

Dallan Murray: Since our initial 2023 approval, we've received enrollment forms for more than 125 HCPs. One of the critical success factors in any launch is that the key treatment centers have had hands-on experience with the drug. And I'm also pleased to highlight that as of this call, there are both commercial and Medicaid payers who have established policies covering levitate therapy for the expanded Medically Accepted Indication.

Speaker Change: Since our initial 2023 approval, we have received enrollment forms for more than 125 hcp's.

Speaker Change: 46 states, including from 97% of our sites.

Speaker Change: And we can see early on that the physician community are approaching label expansion as they have our previous DMD launches.

Dallan Murray: Since our initial 2023 approval, we've received enrollment forms for more than 125 HCPs form 46 states, including from 97% of our sites. And we can see early on that the physicians community are approaching label expansion, as they have our previous DMD launches, prioritizing ambulatory patients first, followed closely by the non-ambulatory, and nearly diagnosed patient populations. Off note, the early days of this gene therapy launch are following the same pattern of all of our previous Exon-Skipping launches. One of the critical success factors in any launch is that the key treatment centers have had hands-on experience with the drug, to that end, over 75% of our sites have dosed at least one patient, and we do expect this to grow now that we have a broad label. And turning to the critical issue of patient access, we've already started to see encouraging signs on the payer policy front, to date our teams have had productive and engaged meetings with payer's representing greater that 225 million patient lives, including the top commercial plans, and state medicate agencies. We anticipate it will takeI'm also pleased to highlight that as of this call, there are both commercial and Medicaid payers who have established policies covering levitate therapy for the expanded Medically Accepted Indication.

Speaker Change: Advertising declining ambulatory patients first.

Speaker Change: Followed closely by the non ambulatory.

Speaker Change: And newly diagnosed patient populations.

Speaker Change: Of note. The early days of this gene therapy launch are following the same pattern of all of our previous exon skipping launches.

Speaker Change: One of the critical success factors in any launch is that the key treatment centers have had hands on experience with the drug.

Dallan Murray: Off note, the early days of this gene therapy launch are following the same pattern of all of our previous Exon-Skipping launches. One of the critical success factors in any launch is that the key treatment centers have had hands-on experience with the drug, to that end, over 75% of our sites have dosed at least one patient, and we do expect this to grow now that we have a broad label. And turning to the critical issue of patient access, we've already started to see encouraging signs on the payer policy front, to date our teams have had productive and engaged meetings with payer's representing greater that 225 million patient lives, including the top commercial plans, and state medicate agencies. We anticipate it will takeI'm also pleased to highlight that as of this call, there are both commercial and Medicaid payers who have established policies covering levitate therapy for the expanded Medically Accepted Indication.

Speaker Change: To that end over 75% of our sites have dosed at least one patient and we do expect this to grow now that we have a broad label.

Dallan Murray: One of the critical success factors in any launch is that the key treatment centers have had hands-on experience with the drug, to that end, over 75% of our sites have dosed at least one patient, and we do expect this to grow now that we have a broad label. And turning to the critical issue of patient access, we've already started to see encouraging signs on the payer policy front, to date our teams have had productive and engaged meetings with payer's representing greater that 225 million patient lives, including the top commercial plans, and state medicate agencies. We anticipate it will take three to six months for most to establish new medical policies, in the mean time we are having success with payers reviewing each patient request on a case by case basis. And to that end we already have authorization approvals for both ambulatory and non-ambulatory patients from both commercial and Medicaid payers for patients in the expanded population, with ages raging from four to 21 years of age. I'm also pleased to highlight that as of this call, there are both commercial and Medicaid payers who have established policies covering ELEVIDYS to the expanded Medically Accepted Indication.

Speaker Change: And turning to the critical issue of patient access we have already started to see encouraging signs on the payer policy firm date, our teams have had productive and engaged meetings with payers representing greater than 225 million patient lives.

Speaker Change: Including the top commercial plans and state Medicaid agencies.

Speaker Change: We anticipated it will take three to six months for both to establish new medical policies in the meantime.

Dallan Murray: And turning to the critical issue of patient access, we've already started to see encouraging signs on the payer policy front, to date our teams have had productive and engaged meetings with payer's representing greater that 225 million patient lives, including the top commercial plans, and state medicate agencies. We anticipate it will takeI'm also pleased to highlight that as of this call, there are both commercial and Medicaid payers who have established policies covering levitate therapy for the expanded Medically Accepted Indication.

Speaker Change: We are having success with payers reviewing each patient requests on a case by case basis.

Dallan Murray: We anticipate it will take three to six months for most to establish new medical policies, in the mean time we are having success with payers reviewing each patient request on a case by case basis. And to that end we already have authorization approvals for both ambulatory and non-ambulatory patients from both commercial and Medicaid payers for patients in the expanded population, with ages raging from four to 21 years of age. I'm also pleased to highlight that as of this call, there are both commercial and Medicaid payers who have established policies covering ELEVIDYS to the expanded Medically Accepted Indication. And finally, with multiple authorizations in hand, we expect to see growth accelerate modestly in the third quarter. What you'll see is a jump in quarterly over quarterly elultous revenues of nearly one third from Q2 to Q3 of this year, followed by a doubling of the elultous revenue. Our 2025 Net Product Revenue Guidance of $2.9 to $3.1 billion for the Duchenne franchise should show you where we are heading with our revenue growth and our confidence around this market potential.

Speaker Change: And to that end, we already have authorization approvals for both ambulatory and non ambulatory patients from both commercial and Medicaid payers for patients. It expanded population with AIDS rates ages, ranging from four to 21 years of age.

Speaker Change: And I'm also pleased to highlight that as of this call. There are both commercial and Medicaid payers, who have established policies covering all evidence to the expanded.

Speaker Change: Medically accepted indication.

Dallan Murray: And finally, with multiple authorizations in hand, we expect to see growth accelerate modestly in the third quarter. What you'll see is a jump in quarterly over quarterly elultous revenues of nearly one third from Q2 to Q3 of this year, followed by a doubling of the elultous revenue. Our 2025 Net Product Revenue Guidance of $2.9 to $3.1 billion for the Duchenne franchise should show you where we are heading with our revenue growth and our confidence around this market potential.

Speaker Change: And then finally with multiple authorizations in hand.

Speaker Change: We already have patients from the expanded population scheduled for dosing and we expect the first of these to be dosed. This week.

Speaker Change: As a result of all of this and as Doug indicated.

Doug Ingram: We expect to see growth accelerating modestly in the third quarter, what you'll see is a jump in quarter over quarter 11 as revenues of nearly one third from Q2 to Q3 of this year.

Dallan Murray: And then finally, with multiple authorizations in hand, we already have patients from the expanded population scheduled for dosing, and we expect the first of these to be dosed this week. As a result of all this, and as Doug indicated, we expect to see growth accelerating modestly in the third quarter, what you'll see is a jump in quarter over quarter ELEVIDYS revenues of nearly one third from Q2 to Q3 of this year, followed by a doubling of the ELEVIDYS revenue from Q3 to Q4. For the remainder of 2024 we'll only [inaudible] to what we'll see in 2025 and beyond, when the sites have had the opportunity to fully adapt themselves to a broad label. Our 2025 Net Product Revenue Guidance of $2.9 to $3.1 billion for the Duchenne franchise should show you where we are heading with our revenue growth and our confidence around this market potential. And that market potential is absolutely massive, let me spend a moment painting the picture of how we expect this launch to play out. There are three things which illustrate just how exciting this opportunity truly is from a commercial perspective. First and foremost, with no near-term gene therapy competition, we expect to treat the eligible prevalent Duchenne population in collaboration with the key centers over the remainder of this decade. Secondly, our track record over the past eight years should provide you all reassurance to our ability to secure access for Duchenne patients in a broad label setting. While it may take time for some patients we will ultimately secure access to the vast majority of Duchenne patients who are eligible. Lastly and very importantly, the ELEVIDYS one time dose reach patient is entirely upfront, and along with that, so is the revenue. I don't think this fact has been fully appreciated outside of Sarepta, and this is what I was describing earlier, after that early adjustment period in the back half of this year, we expect the revenue we expect the revenue to take off robustly. We have been planning for this opportunity for many years. We've anticipated the challenges. And what I'm most proud of is that patient safety is at the center of everything we do. We are taking a thoughtful approach to integrating education and supportproactively into all of our issues. Enabling a fully informed discussion between doctor and patient is both the right thing to do and sets everyone up for long-term success.

Speaker Change: All led by a doubling of the <unk> revenue from Q3 to Q4.

Doug Ingram: But the remainder of 2024 will only be a prelude to what we'll see in.

Speaker Change: In 2025 and beyond.

Speaker Change: When the sites have had the opportunity to fully adapt themselves to a broad label.

Speaker Change: Our 2025 net product revenue guidance of $2 nine to $3 1 billion for the Duchenne franchise should show you, where we are headed with our revenue growth.

Speaker Change: And our confidence around this market potential.

Dallan Murray: And that market potential is absolutely massive. Let me spend a moment painting the picture of how we expect this launch to play out. There are three things which illustrate just how exciting this opportunity truly is from a commercial perspective. First and foremost, with no near-term gene therapy competition, we expect to treat the eligible prevalent Duchenne population in collaboration with the key centers over the remainder of this decade. And along with that, so is the revenue. But I don't think this fact has been fully appreciated outside of Sarepta.

Speaker Change: And that market potential is absolutely massive let me spend a moment painting the picture for how we expect this launch to play out.

Dallan Murray: Our 2025 Net Product Revenue Guidance of $2.9 to $3.1 billion for the Duchenne franchise should show you where we are heading with our revenue growth and our confidence around this market potential. And that market potential is absolutely massive, let me spend a moment painting the picture of how we expect this launch to play out. There are three things which illustrate just how exciting this opportunity truly is from a commercial perspective. First and foremost, with no near-term gene therapy competition, we expect to treat the eligible prevalent Duchenne population in collaboration with the key centers over the remainder of this decade. Secondly, our track record over the past eight years should provide you all reassurance to our ability to secure access for Duchenne patients in a broad label setting. While it may take time for some patients we will ultimately secure access to the vast majority of Duchenne patients who are eligible. Lastly and very importantly, the ELEVIDYS one time dose reach patient is entirely upfront, and along with that, so is the revenue. I don't think this fact has been fully appreciated outside of Sarepta, and this is what I was describing earlier, after that early adjustment period in the back half of this year, we expect the revenue we expect the revenue to take off robustly.

Speaker Change: There are three things, which illustrate just how exciting this opportunity truly is from a commercial perspective.

Speaker Change: First and foremost with no near term gene therapy competition.

Speaker Change: We expect to treat the eligible prevalent duchenne population in collaboration with the key centers over the remainder of this decade.

Speaker Change: Secondly, our track record over the past eight years should provide you all reassurance of our ability to secure access for duchenne patients.

Speaker Change: Rod label setting.

Speaker Change: Yes.

Speaker Change: While it may take time for some patients we will ultimately secure access to the vast majority of duchenne patients who are eligible.

Dallan Murray: Secondly, our track record over the past eight years should provide you all reassurance to our ability to secure access for Duchenne patients in a broad label setting. While it may take time for some patients we will ultimately secure access to the vast majority of Duchenne patients who are eligible. Lastly and very importantly, the ELEVIDYS one time dose reach patient is entirely upfront, and along with that, so is the revenue. I don't think this fact has been fully appreciated outside of Sarepta, and this is what I was describing earlier, after that early adjustment period in the back half of this year, we expect the revenue to take off robustly.

Speaker Change: Lastly, and very importantly, the <unk> onetime dose for each patient is entirely upfront.

Speaker Change: And along with that so as the revenue.

Speaker Change: I don't think this factor has been fully appreciated outside of swept up.

Speaker Change: And this is what I was describing earlier after that early adjustment period in the back half of this year.

Dallan Murray: We expect the revenue to take off robustly. We have been planning for this opportunity for many years. We've anticipated the challenges. And what I'm most proud of is that patient safety is at the center of everything we do. We are taking a thoughtful approach to integrating education and supportproactively into all of our issues. Enabling a fully informed discussion between doctor and patient is both the right thing to do and sets everyone up for long-term success.

Speaker Change: We expect the revenue to takeoff robustly.

Speaker Change: We have been planning for this opportunity for many years, we've anticipated the challenges.

Speaker Change: And have now had a full year to learn and evolve with the team that was originally built for the full duchenne population.

Dallan Murray: We have been planning for this opportunity for many years, we've anticipated the challenges, and have now had a full year to learn and evolve with a team that was originally built for the whole Duchenne population. What I'm most proud of is that patient safety is at the center of everything we do, we are taking a thoughtful approach to integrating education and support proactively into all of our issues. Enabling a fully informed discussion between doctor and patient is both the right thing to do and sets everyone up for long-term success. As with any launch we'll see our share have ups and downs, it will take time for us in the centers to work through the demand that we're seeing today, and there may be some frustration as we do our best to support these deserving patients. As we demonstrated in our previous launches, there is no team in the rare disease, and I would even argue, in the entire biopharma landscape, who could execute better on such an incredible opportunity as the one we have in front of us right now.

Speaker Change: What I'm most proud of is that patient safety is at the center of everything we do.

Speaker Change: We're taking a thoughtful approach in integrating education and support.

Speaker Change: Proactively into all of our initiatives, enabling a fully informed discussion between doctor and patient.

Speaker Change: It's both the right thing to do.

Speaker Change: Everyone up for long term success.

Speaker Change: As with any launch, we'll see our share of ups and downs.

Dallan Murray: As with any launch we'll see our share have ups and downs, it will take time for us in the centers to work through the demand that we're seeing today, and there may be some frustration as we do our best to support these deserving patients. As we demonstrated in our previous launches, there is no team in the rare disease, and I would even argue, in the entire biopharma landscape, who could execute better on such an incredible opportunity as the one we have in front of us right now. So, in summary, the interest and demand from all stakeholders is strong, as we had anticipated, we expect this demand will only increase with time. Obviously, all of the patients who are seeking treatment right now cannot be accommodated by the system immediately, we and the Physicians community are working as hard as we can to support all of the patient fit for treatment, but we will support them and, in so doing, realize perhaps the biggest near-term revenue opportunity in all of biotech. I'd like to end by thanking everyone at Sarepta and all of our stakeholders for their role in getting us to this important moment, as we sit here today, the majority of Duchenne patients are eligible for dystrophin restoration therapy, yet the majority, as of today, have not yet received dystrophin restoration therapy. We aim to change that, and we continue to execute with the realization that every minute matters for these patients and their families. Now, with that, I'll hand it over to our head of R&D, Dr. Louise Rodino-Klapac. Louise.

Dallan Murray: It will take time for us in the centers to work through the demand that we're seeing today, and there may be some frustration as we do our best to support these deserving patients. As we demonstrated in our previous launches, there is no team in rare disease, and I would even argue, in the entire biopharma landscape, who could execute better on such an incredible opportunity as the one we have in front of us right now.

Speaker Change: It will take time for us in the centers to work through the demand that we're seeing today and there may be some frustration as we do our best to support these deserving patients.

Speaker Change: Is that as we demonstrated in our previous launches there is no team in the rare disease and I would even argue in the entire biopharma landscape, who could execute better on such an incredible opportunity.

Speaker Change: We have in front of us right now.

Dallan Murray: So, in summary, the interest and demand from all stakeholders is strong, as we had anticipated. We expect this demand will only increase with time. Obviously, all of the patients who are seeking treatment right now cannot be accommodated by the system immediately.

Speaker Change: So in summary, the interest and demand from all stakeholders as strong as we had anticipated.

Dallan Murray: So, in summary, the interest and demand from all stakeholders is strong, as we had anticipated, we expect this demand will only increase with time. Obviously, all of the patients who are seeking treatment right now cannot be accommodated by the system immediately, we and the Physicians community are working as hard as we can to support all of the patient fit for treatment, but we will support them and, in so doing, realize perhaps the biggest near-term revenue opportunity in all of biotech. I'd like to end by thanking everyone at Sarepta and all of our stakeholders for their role in getting us to this important moment, as we sit here today, the majority of Duchenne patients are eligible for dystrophin restoration therapy, yet the majority, as of today, have not yet received dystrophin restoration therapy. We aim to change that, and we continue to execute with the realization that every minute matters for these patients and their families. Now, with that, I'll hand it over to our head of R&D, Dr. Louise Rodino-Klapac. Louise.

Speaker Change: We expect this demand will only increase with time.

Speaker Change: Obviously all of the patients who are seeking treatment right now cannot be accommodated by the system immediately.

Speaker Change: We in the physician community are working as hard as we can to support all of the patients seeking treatment.

Dallan Murray: But we will support them and, in so doing, realize perhaps the biggest near-term revenue opportunity in all of biotech. I'd like to end by thanking everyone at Sarepta and all of our stakeholders for their role in getting us to this important moment. As we sit here today, the majority of Duchenne patients are eligible for dystrophin restoration therapy, yet the majority, as of today, have not yet received dystrophin restoration therapy. We aim to change that. And we continue to execute with the realization that every minute matters for these patients and their families. Now, with that, I'll hand it over to our head of R&D, Dr. Louise Rodino-Clepak.

Speaker Change: But we will support them and in so doing realized perhaps the biggest near term revenue opportunity in all of biotech.

Speaker Change: I'd like to end by thanking everyone at Raptor and all of our stakeholders for their role in getting us to this important moment.

Dallan Murray: I'd like to end by thanking everyone at Sarepta and all of our stakeholders for their role in getting us to this important moment, as we sit here today, the majority of Duchenne patients are eligible for dystrophin restoration therapy, yet the majority, as of today, have not yet received dystrophin restoration therapy. We aim to change that, and we continue to execute with the realization that every minute matters for these patients and their families. Now, with that, I'll hand it over to our head of R&D, Dr. Louise Rodino-Klapac. Louise.

Speaker Change: As we sit here today the majority of Duchenne patients are eligible for dystrophin restoration therapy.

Speaker Change: The majority as of today have not yet received dystrophin restoration therapy.

Speaker Change: We aim to change that and we continue to execute with the realization that every minute matters for these patients and their families.

Speaker Change: Now with that I'll hand, it over to our head of R&D, Dr. Louise Rodino <unk> Suisse.

Louise Rodino-Klapac: Thanks, Dallan, and good afternoon, I'll begin my remarks with the approval of the ELEVIDYS label expansion and then provide an update on our pipeline. Following decades of research and development, and after a thorough scientific review that critically considered all of the available evidence across multiple studies, the Office of Therapeutic Products at the FDA approved an expansion of the ELEVIDYS label based on the following. The clinical benefits of ELEVIDYS in ambulatory patients were confirmed by our double-blind, placebo-controlled study in BARC and related studies, and based on these data, traditional approval was granted to all ambulatory Duchenne patients ages 4 and older. The mechanism of action of ELEVIDYS is universal, regardless of disease state, as long as muscle is present.

Louise Rodino-Klapac: Thanks, Dallan, and good afternoon, I'll begin my remarks with the approval of the ELEVIDYS label expansion and then provide an update on our pipeline. Following decades of research and development, and after a thorough scientific review that critically considered all of the available evidence across multiple studies, the Office of Therapeutic Products at the FDA approved an expansion of the ELEVIDYS label based on the following. The clinical benefits of ELEVIDYS in ambulatory patients were confirmed by our double-blind, placebo-controlled study EMBARK and related studies, and based on these data, traditional approval was granted to all ambulatory Duchenne patients ages 4 and older.

Speaker Change: Thanks, John and good afternoon.

Speaker Change: I'll begin my remarks that the approval of the <unk> label expansion and then provide an update on our pipeline.

Speaker Change: Following decades of research and development and after a thorough scientific review that critically considered all of the available evidence across multiple studies.

Speaker Change: Office of therapeutic products that the FDA approved expansion of the a lot of it is label based on the following.

Speaker Change: The clinical benefits of <unk> and ambulatory patients with confirmed by our double blind placebo controlled study embark and related study and based on these data traditional approval is granted to all ambulatory Duchenne patients ages four and older.

Speaker Change: The mechanism of action of <unk> is the universal regardless of disease state as long as muscle is prevalent.

Speaker Change: As a result.

Speaker Change: This drove an express buyer therapy and non ambulatory patients is reasonably likely to confirm clinical benefit in this population.

Louise Rodino-Klapac: The mechanism of action of ELEVIDYS is universal, regardless of disease state, as long as muscle is present, as a result the ELEVIDYS [inaudible] therapy in non-ambulatory patients is relatively likely to confirm clinical benefit in this population. A a result, accelerated approval or AA has been granted for the treatment of non-inventory patients ages four and older. The accelerated approval for non-ambulatory patients includes a post-marketing commitment to confirm clinical benefits, which we addressed via our Non-Ambulatory and Late Ambulatory Study 303, also known as ENVISION. As a reminder, ENVISION is a global, randomized, double-blind, placebo-controlled two part study, evaluating the safety and efficacy of SRP-9001 gene therapy in non-ambulatory and older ambulatory individuals for Duchenne. ENVISION is progressing well, with the US enrollment complete, and the remaining recruitment accruing ex-US. Enrollment is expected to be completed in 2025, with our last patient, last visit expected in 2027, following an 18-month placebo-controlled period. We also continue to advance clinical studies that [inaudible] a long-term follow-up with ELEVIDYS. Our long-term follow-up studies include ENDURE and EXPEDITION. ENDURE is a Phase IV observational study that will follow individuals treated with ELEVIDYS for up to 10 years. In addition, EXPEDITION is a Phase III study enrolling approximately 400 patients that were previously enrolled in a Levitas clinical trial and followed for consistent safety and efficacy measures for up to five years. Study 101 Five-Year Longitudinal Data

Louise Rodino-Klapac: The mechanism of action of ELEVIDYS is universal, regardless of disease state, as long as muscle is present. As a result the ELEVIDYS dystrophin expressed by our therapy in non-ambulatory patients is relatively likely to confirm clinical benefit in this population. A a result, accelerated approval or AA has been granted for the treatment of non-inventory patients ages four and older. The accelerated approval for non-ambulatory patients includes a post-marketing commitment to confirm clinical benefits, which we addressed via our Non-Ambulatory and Late Ambulatory Study 303, also known as ENVISION.

Speaker Change: As a result accelerated approval or a E has been granted for the treatment of non ambulatory patients ages four and older.

Louise Rodino: The accelerated approval for non-ambulatory patients includes a post-marketing commitment to confirm clinical benefits, which will be addressed via our Non-Ambulatory and Late Ambulatory Study 303, also known as NVISION. Enrollment is expected to be completed in 2025, with our last patient and last visit expected in 2027, following an 18-month placebo-controlled period. In addition, EXPEDITION is a Phase III study enrolling approximately 400 patients that were previously enrolled in a Levitas clinical trial and followed for consistent safety and efficacy measures for up to five years. Study 101 Five-Year Longitudinal Data

Speaker Change: The accelerated approval for non ambulatory patients include the post marketing commitment to confirm clinical benefit which will be addressed via our non ambulatory and late ambulatory study 303.

Speaker Change: Known as ambitious.

Speaker Change: As a reminder, envision is a global randomized double blind placebo controlled two part study evaluating the safety and efficacy of SRP 9001 gene therapy, not ambulatory and older ambulatory individuals petition.

Speaker Change: Envision is progressing well with U S enrollment complete and the remaining recruitment occurring ex U S.

Speaker Change: Enrollment is expected to be completed in 2025 with our last patient last visit expected in 2027, following an 18 month placebo controlled period.

Speaker Change: We also continue to advance clinical studies that monitor long term follow up with a limited.

Speaker Change: Our long term follow up studies include endure an expedition and Terra is a phase four observational study that will follow individuals' stringent with a lot of it is for up to 10 years.

Louise Rodino-Klapac: As a reminder, ENVISION is a global, randomized, double-blind, placebo-controlled two part study, evaluating the safety and efficacy of SRP-9001 gene therapy in non-ambulatory and older ambulatory individuals for Duchenne. ENVISION is progressing well, with the US enrollment complete, and the remaining recruitment accruing ex-US. Enrollment is expected to be completed in 2025, with our last patient, last visit expected in 2027, following an 18-month placebo-controlled period. We also continue to advance clinical studies that [inaudible] a long-term follow-up with ELEVIDYS. Our long-term follow-up studies include ENDURE and EXPEDITION. ENDURE is a Phase IV observational study that will follow individuals treated with ELEVIDYS for up to 10 years. In addition, EXPEDITION is a Phase III study enrolling approximately 400 patients that were previously enrolled in a Levitas clinical trial and followed for consistent safety and efficacy measures for up to five years. Study 101 Five-Year Longitudinal Data

Speaker Change: One third of the sites have been activated and up to 500 patients will be involved.

Speaker Change: In addition expedition is a phase III study enrolling approximately 400 patients that were previously involved in a love it as clinical trials and followed for consistent safety and efficacy measures for up to five years.

Louise Rodino-Klapac: ENVISION is progressing well, with the US enrollment complete, and the remaining recruitment accruing ex-US. Enrollment is expected to be completed in 2025, with our last patient, last visit expected in 2027, following an 18-month placebo-controlled period. We also continue to advance clinical studies that [inaudible] a long-term follow-up with ELEVIDYS. Our long-term follow-up studies include ENDURE and EXPEDITION. ENDURE is a Phase IV observational study that will follow individuals treated with ELEVIDYS for up to 10 years. In addition, EXPEDITION is a Phase III study enrolling approximately 400 patients that were previously enrolled in a Levitas clinical trial and followed for consistent safety and efficacy measures for up to five years. Study 101 Five-Year Longitudinal Data

Speaker Change: Regarding patient currently ineligible to receive allowed under the expanded label we're conducting multiple studies.

Speaker Change: So the approximately 15% of patients who are screened out for preexisting anti AAV RH 74 antibodies. We have commenced a study to cleave and inactivate antibodies with simplicity and will shortly begin a separate study to remove antibodies with plasmapheresis.

Speaker Change: In addition for patients under the age of four we have treated patients as young as two and our study 103 and together with our partner Roche, where executing study 302 to gain experience I think patients under or and as young as three months.

Louise Rodino-Klapac: Our long-term follow-up studies include ENDURE and EXPEDITION. ENDURE is a Phase IV observational study that will follow individuals treated with ELEVIDYS for up to 10 years. One third of sites has been activated, and up to 500 patients will be enrolled. In addition, EXPEDITION is a Phase III study enrolling approximately 400 patients that were previously enrolled in ELEVIDYS clinical trial and followed for consistent safety and efficacy measures for up to five years. Regarding patients currently ineligible to receive ELEVIDYS under the expanded label, we're conducting multiple studies.

Speaker Change: We will continue to transparently communicate the range of trial experience in patients treated with the Libya from those that are under four to those with more advanced disease.

Louise Rodino-Klapac: ENDURE is a Phase IV observational study that will follow individuals treated with ELEVIDYS for up to 10 years, 1/3 of it's size has been activated, and up to 500 patients will be enrolled. In addition, EXPEDITION is a Phase III study enrolling approximately 400 patients that were previously enrolled in ELEVIDYS clinical trial and followed for consistent safety and efficacy measures for up to five years. Regarding patients currently ineligible to receive ELEVIDYS under the expanded label, we're conducting multiple studies. For the approximately 15% of patients who are strained up for preexisting anti-AAV rh74 antibodies, we've commenced the study to [inaudible] and inactivate antibodies within [inaudible] and will shortly begin a separate study to remove antibodies with plasmapheresis. In addition, for patients under the age of 4, we have treated patients who are young as two in our study 103, and together with our partner Roche, we're executing study 302 to gain experience dosing patients under four and as young as three months. We will continue to transparently communicate the range of trial experience in patients treated with ELEVIDYS, from those that are under four, to those with more advanced disease. As of the end of June 2024, we have dosed over 60 late ambulatory, and non-ambulatory patients within our clinical program. We have multiple scientific disclosures planned throughout 2024, we look forward to releasing multiple data sets from across the ELEVIDYS slinical program at the next available forum, which is the World Muscle Society Congress in early October. Among the presentations accepted are additional EMBARK data, including muscle MRI and cardiac MRI. ENDEAVOR or Study 103, safety and expression data in late ambulatory and non-ambulatory patients and Study 101, Five-Year Longitudinal Data. Multiple manuscript submissions are in process or planned, as our plan to release numerous abstracts from additional congresses, including the International Congress on Neuromuscular Diseases, the Academy of Managed Care Pharmacy, and the American Association of Neuromuscular and Electrodiagnostic Medicine. Finally we're pleased to report that the primary EMBARK naive script has been accepted for publication in a high-impact journal, and will be available online in the coming months. To summarize ELEVIDYS, we're extremely happy for the Duchenne community on the expansion of our label, and the expanded opportunity for those living with Duchenne that wish to receive ELEVIDYS. I'd like to offer a very special thank you o the courageous families who chose to participate in our studies that support this approval. We will continue to diligently serve the community through execution of our ongoing clinical studies, in communication of safety and efficacy data on an ongoing basis as it becomes available. Moving now to out programs on Limb-Girdle Muscular Dystrophies or LGMDs, starting with SRP-9003. We are encouraged by the agency's willingness to create a viable pathway for SRP 9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Louise Rodino-Klapac: ENDURE is a Phase IV observational study that will follow individuals treated with ELEVIDYS for up to 10 years. In addition, EXPEDITION is a Phase III study enrolling approximately 400 patients that were previously enrolled in a Levitas clinical trial and followed for consistent safety and efficacy measures for up to five years. Study 101 Five-Year Longitudinal Data

Louise Rodino-Klapac: ENDURE is a Phase IV observational study that will follow individuals treated with ELEVIDYS for up to 10 years.

Speaker Change: As of the end of June 2024, we have dosed over 60 late ambulatory and non ambulatory patients within our clinical program.

Speaker Change: We have multiple scientific disclosures plan throughout 2024, we look forward to releasing multiple datasets from the cross sell of its clinical program at the next available for them, which is the world Muscle Society Congress in early October.

Louise Rodino-Klapac: In addition, EXPEDITION is a Phase III study enrolling approximately 400 patients that were previously enrolled in a Levitas clinical trial and followed for consistent safety and efficacy measures for up to five years. Study 101 Five-Year Longitudinal Data Multiple manuscript submissions are in process or planned, as we plan to release numerous abstracts from additional congresses, including the International Congress on Neuromuscular Diseases, the Academy of Managed Care Pharmacy, and the American Association of Neuromuscular and Electrodiagnostic Medicine. We are encouraged by the agency's willingness to create a viable pathway for SRP 9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: Among the presentations accepted our additional embarked data, including muscle MRI and cardiac MRI.

Speaker Change: Endeavour study 103 safety and expression data in late ambulatory and non ambulatory patients and.

Speaker Change: In study 101, five year longitudinal data.

Louise Rodino: Multiple manuscript submissions are in process or planned, as we plan to release numerous abstracts from additional congresses, including the International Congress on Neuromuscular Diseases, the Academy of Managed Care Pharmacy, and the American Association of Neuromuscular and Electrodiagnostic Medicine. We are encouraged by the agency's willingness to create a viable pathway for SRP 9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: Multiple manuscript submissions are in process or planned.

Speaker Change: Our plans to release numerous aftershocks additional congresses, including the international Congress on neuromuscular diseases. The Academy of managed care pharmacy, and the American Association of neuromuscular and electro diagnostic medicine.

Louise Rodino-Klapac: Regarding patients currently ineligible to receive ELEVIDYS under the expanded label, we're conducting multiple studies. For the approximately 15% of patients who are strained up for preexisting anti-AAV rh74 antibodies, we've commenced the study to cleave and inactivate antibodies with Imlifidase and will shortly begin a separate study to remove antibodies with plasmapheresis. In addition, for patients under the age of 4, we have treated patients who are young as two in our study 103, and together with our partner Roche, we're executing study 302 to gain experience dosing patients under four and as young as three months. We will continue to transparently communicate the range of trial experience in patients treated with ELEVIDYS, from those that are under four, to those with more advanced disease.

Speaker Change: Finally, we are pleased to report that the primary embarked manuscript has been accepted for publication in a high impact journal and will be available online in the coming months.

Speaker Change: To summarize the Lumpiness, we are extremely happy for the Duchenne community on the expansion of our label and the expanded opportunity for those living with Duchenne that wish to receive a lot of that.

Louise Rodino-Klapac: For the approximately 15% of patients who are strained up for preexisting anti-AAV rh74 antibodies, we've commenced the study to [inaudible] and inactivate antibodies within [inaudible] and will shortly begin a separate study to remove antibodies with plasmapheresis. In addition, for patients under the age of 4, we have treated patients who are young as two in our study 103, and together with our partner Roche, we're executing study 302 to gain experience dosing patients under four and as young as three months. We will continue to transparently communicate the range of trial experience in patients treated with ELEVIDYS, from those that are under four, to those with more advanced disease. As of the end of June 2024, we have dosed over 60 late ambulatory, and non-ambulatory patients within our clinical program. We have multiple scientific disclosures planned throughout 2024, we look forward to releasing multiple data sets from across the ELEVIDYS slinical program at the next available forum, which is the World Muscle Society Congress in early October. Among the presentations accepted are additional EMBARK data, including muscle MRI and cardiac MRI. ENDEAVOR or Study 103, safety and expression data in late ambulatory and non-ambulatory patients and Study 101, Five-Year Longitudinal Data. Multiple manuscript submissions are in process or planned, as our plan to release numerous abstracts from additional congresses, including the International Congress on Neuromuscular Diseases, the Academy of Managed Care Pharmacy, and the American Association of Neuromuscular and Electrodiagnostic Medicine. Finally we're pleased to report that the primary EMBARK naive script has been accepted for publication in a high-impact journal, and will be available online in the coming months. To summarize ELEVIDYS, we're extremely happy for the Duchenne community on the expansion of our label, and the expanded opportunity for those living with Duchenne that wish to receive ELEVIDYS. I'd like to offer a very special thank you o the courageous families who chose to participate in our studies that support this approval. We will continue to diligently serve the community through execution of our ongoing clinical studies, in communication of safety and efficacy data on an ongoing basis as it becomes available. Moving now to out programs on Limb-Girdle Muscular Dystrophies or LGMDs, starting with SRP-9003. We are encouraged by the agency's willingness to create a viable pathway for SRP 9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: I'd like to offer a very special thank you to the courageous families who chose to participate in our studies that support this approval.

Speaker Change: We'll continue to diligently serve the community through execution of our ongoing clinical studies and communication of safety and efficacy data on an ongoing basis as it becomes available.

Speaker Change: Moving now to our programs to the limb girdle muscular dystrophy or <unk>.

Speaker Change: Starting with SRP 9003.

Speaker Change: As we mentioned on the first quarter call dosing is underway in study FRP nicer zero three 301.

Speaker Change: Also known as averaging our phase III multinational open label clinical trial of SRP 9003 for the treatment of lung girdle muscular dystrophy type to E Bay, a cyclical I cannot foresee.

Speaker Change: The agreed primary endpoint of Enbridge, Inc. Is expression of beta circle glad can the absence of which is the sole causes the disease.

Speaker Change: We are encouraged by the agencies a willingness to create a viable pathway for SRP 9003, what's changed what's changed in ultra rare genetic condition that is progressively developing result in loss of ambulation and leads to early mortality.

Speaker Change: Assuming a positive pre BLA meeting, we would anticipate BLA filing in 2025.

Louise Rodino-Klapac: We will continue to transparently communicate the range of trial experience in patients treated with ELEVIDYS, from those that are under four, to those with more advanced disease. As of the end of June 2024, we have dosed over 60 late ambulatory, and non-ambulatory patients within our clinical program. We have multiple scientific disclosures planned throughout 2024, we look forward to releasing multiple data sets from across the ELEVIDYS slinical program at the next available forum, which is the World Muscle Society Congress in early October. Among the presentations accepted are additional EMBARK data, including muscle MRI and cardiac MRI. ENDEAVOR or Study 103, safety and expression data in late ambulatory and non-ambulatory patients and Study 101, Five-Year Longitudinal Data. Multiple manuscript submissions are in process or planned, as our plan to release numerous abstracts from additional congresses, including the International Congress on Neuromuscular Diseases, the Academy of Managed Care Pharmacy, and the American Association of Neuromuscular and Electrodiagnostic Medicine. Finally we're pleased to report that the primary EMBARK naive script has been accepted for publication in a high-impact journal, and will be available online in the coming months. To summarize ELEVIDYS, we're extremely happy for the Duchenne community on the expansion of our label, and the expanded opportunity for those living with Duchenne that wish to receive ELEVIDYS. I'd like to offer a very special thank you o the courageous families who chose to participate in our studies that support this approval. We will continue to diligently serve the community through execution of our ongoing clinical studies, in communication of safety and efficacy data on an ongoing basis as it becomes available. Moving now to out programs on Limb-Girdle Muscular Dystrophies or LGMDs, starting with SRP-9003. We are encouraged by the agency's willingness to create a viable pathway for SRP 9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: We are also very pleased to announce that we received fast track designation for SRP 9003.

Speaker Change: Fast track designation is a process designed to facilitate the development and expedited review drugs, but treat serious conditions and fill unmet medical needs in.

Speaker Change: In addition to fast track SRP 9003 has also been previously granted rare pediatric disease designation.

Speaker Change: And orphan drug designation from FDA as well as orphan drug designation from the European Medicines Agency.

Louise Rodino-Klapac: As of the end of June 2024, we have dosed over 60 late ambulatory, and non-ambulatory patients within our clinical program. We have multiple scientific disclosures planned throughout 2024, we look forward to releasing multiple data sets from across the ELEVIDYS slinical program at the next available forum, which is the World Muscle Society Congress in early October. Among the presentations accepted are additional EMBARK data, including muscle MRI and cardiac MRI. ENDEAVOR or Study 103, safety and expression data in late ambulatory and non-ambulatory patients and Study 101, Five-Year Longitudinal Data. Multiple manuscript submissions are in process or planned, as our plan to release numerous abstracts from additional congresses, including the International Congress on Neuromuscular Diseases, the Academy of Managed Care Pharmacy, and the American Association of Neuromuscular and Electrodiagnostic Medicine. Finally we're pleased to report that the primary EMBARK naive script has been accepted for publication in a high-impact journal, and will be available online in the coming months. To summarize ELEVIDYS, we're extremely happy for the Duchenne community on the expansion of our label, and the expanded opportunity for those living with Duchenne that wish to receive ELEVIDYS. I'd like to offer a very special thank you o the courageous families who chose to participate in our studies that support this approval. We will continue to diligently serve the community through execution of our ongoing clinical studies, in communication of safety and efficacy data on an ongoing basis as it becomes available. Moving now to out programs on Limb-Girdle Muscular Dystrophies or LGMDs, starting with SRP-9003. We are encouraged by the agency's willingness to create a viable pathway for SRP 9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: The ability to progress our small and F 15, biomarker study together with our ability to demonstrate delivery of our functional data sorry for glycol protein is extremely important not just for this program, but for the other cyclical I can offer these in our pipeline, including LG M. D. D. L. G M D to C. Both of which are progressing through the <unk>.

Speaker Change: Mike.

Speaker Change: Having successfully advanced SRP, neither Israel free we will submit our SRP nine zero, therefore, R&D update reflecting our suspension process. This year with phase one initiation expected to start by year's end.

Speaker Change: As a reminder, SRP 9000, <unk> four is designed for the treatment of limb girdle muscular dystrophy type two D for alpha circles like an off the thing.

Speaker Change: Finally, we are also rapidly progressing our program for SRP 9005 for the treatment of limb girdle muscular dystrophy type two C. Organosol quite compensate the plan to engage with FDA in Q4 of this year with plans to initiate a clinical study in Q1 2025 pending the outcome of that meeting.

Speaker Change: To summarize we are very pleased with the progress of our <unk> portfolio and expect to have three of our L. G. M D as in the clinic and less than nine months.

Speaker Change: We are maximizing the synergies across this platform sunblock on R&D and manufacturing perspective, and our sights firmly set on accelerating the remainder of the <unk> assets to the clinic.

Speaker Change: The subject matter expertise that we've built positions us well could rapidly advanced our current and future pipeline.

Louise Rodino-Klapac: Multiple manuscript submissions are in process or planned, as our plan to release numerous abstracts from additional congresses, including the International Congress on Neuromuscular Diseases, the Academy of Managed Care Pharmacy, and the American Association of Neuromuscular and Electrodiagnostic Medicine. Finally we're pleased to report that the primary EMBARK naive script has been accepted for publication in a high-impact journal, and will be available online in the coming months. To summarize ELEVIDYS, we're extremely happy for the Duchenne community on the expansion of our label, and the expanded opportunity for those living with Duchenne that wish to receive ELEVIDYS. I'd like to offer a very special thank you o the courageous families who chose to participate in our studies that support this approval. We will continue to diligently serve the community through execution of our ongoing clinical studies, in communication of safety and efficacy data on an ongoing basis as it becomes available. Moving now to out programs on Limb-Girdle Muscular Dystrophies or LGMDs, starting with SRP-9003. We are encouraged by the agency's willingness to create a viable pathway for SRP 9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: Continuing with our RNA platform and beginning with our P&L.

Speaker Change: The essence trial, our post marketing requirements for Cologuard and cast in person as well as mission our post marketing commitment for Exxon to us are both fully enrolled and remain on track.

Speaker Change: As a reminder, <unk> is a two year two to one randomized placebo controlled study of both gold or spin and CASM person and is due to read out in early 2026.

Speaker Change: This study was fully enrolled with 228 patients.

Speaker Change: Michigan is a randomized double blind safety efficacy dose finding study comparing the approved dosage of accomplishment 30 mix per kg lately.

Speaker Change: Dosage that provide significantly higher exposure up to 200 Meg per kg Mitch.

Speaker Change: <unk> is a two part phase III study it was fully enrolled with 160 patients.

Speaker Change: <unk> committed to rapidly intelligently advancing mission and sharing data as soon as it becomes available.

Speaker Change: Turning to P. P M L.

Speaker Change: Early in the first quarter, we announced positive results from part B of a medicine study or study SRP 5051 201.

Louise Rodino-Klapac: To summarize ELEVIDYS, we're extremely happy for the Duchenne community on the expansion of our label, and the expanded opportunity for those living with Duchenne that wish to receive ELEVIDYS. I'd like to offer a very special thank you o the courageous families who chose to participate in our studies that support this approval. We will continue to diligently serve the community through execution of our ongoing clinical studies, in communication of safety and efficacy data on an ongoing basis as it becomes available. Moving now to out programs on Limb-Girdle Muscular Dystrophies or LGMDs, starting with SRP-9003. We are encouraged by the agency's willingness to create a viable pathway for SRP 9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: Momentum is an ongoing study of SRP 50, 51, our investigational peptide conjugated PMO or P. P. M L.

Speaker Change: We are actively engaging with Peter regarding the phase III study that could serve as a post marketing requirement our PMA study for a potential accelerated approval.

Speaker Change: <unk> on the phase III <unk> study is a prerequisite for an accelerated approval filing.

Speaker Change: Our intent is to gain agreement for a study that considers the current landscape can be completed in a reasonable timeframe and reflects patients reluctance to enroll in a placebo controlled trial.

Louise Rodino-Klapac: I'd like to offer a very special thank you o the courageous families who chose to participate in our studies that support this approval, we will continue to diligently serve the community through execution of our ongoing clinical studies, in communication of safety and efficacy data on an ongoing basis as it becomes available. Moving now to out programs on Limb-Girdle Muscular Dystrophies or LGMDs, starting with SRP-9003, as we've mentioned in the first quarter call, dosing is under way. In study SRP-9003-301, also known as EMERGENE, our Phase III, multinational, open-label clinical trial SRP-9003 for the treatment of Limb-Girdle Muscular Dystrophies type 2E or beta sarcoglycanopathy. The agreed primary endpoint of EMERGENE is expression of beta-sarcoglycan, the absence of which is the sole cause of the disease. We are encouraged by the agency's willingness to create a viable pathway for SRP-9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of ambulation and leads to early mortality. Assuming a positive [inaudible] meeting, we would anticipate [inaudible] in 2025, we are also very pleased to announce that we've received fast-track designation for SRP-9003. A fast-track designation is a process designed to facilitate the development, and expedite a reviewed drug which treat serious conditions and fill unmet medical needs. In addition to fast-track SRP-9003 has also been previously granted rare pediatric disease designation, and orphan drug designation from FDA, as well as orphan drug designation from the European Medicines Agency. The ability to progress a small NS-15 biomarker study, together with our ability to demonstrate delivery of a functional beta-sarcoglycan protein is extremely important, not just for this program but for the other sarcoglycanopathies in our pipeline, including LGMD2D and LGMD2C, those [inaudible] progressing to the clinic. [inaudible] successfully advance SRP-9003, we will submit our SRP-9004 IND update, reflecting our suspension process this year, with Phase I initiation expected to start by year's end. As a reminder SRP-9004 is designed for the treatment of Limb-Girdle Muscular Dystrophy type 2D for alpha-sarcoglycanopathy. Finally we're also rapidly progressing our program for SRP-9005 for the treatment of Limb-Girdle Muscular Dystrophies type 2C, or gamma-sarcoglycanopathy. We plan to engage with FDA in Q4c of this year, but plan to initiate the clinical study in Q1 2025, pending the outcome of that meeting. To summarize The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: Our hope is that an agreement can be reached on the trial design this year.

Speaker Change: In summary, our team looks forward to a bright future ahead as we work diligently to advance our mission with the goal of serving patients around the world living with rare disease.

Louise Rodino-Klapac: We will continue to diligently serve the community through execution of our ongoing clinical studies, in communication of safety and efficacy data on an ongoing basis as it becomes available. Moving now to out programs on Limb-Girdle Muscular Dystrophies or LGMDs, starting with SRP-9003. We are encouraged by the agency's willingness to create a viable pathway for SRP 9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: I'd like to take a moment to thank my R&D colleagues for their unwavering devotion to advancing our entirely our entire portfolio.

Louise Rodino-Klapac: In study SRP-9003-301, also known as EMERGENE, our Phase III, multinational, open-label clinical trial SRP-9003 for the treatment of Limb-Girdle Muscular Dystrophies type 2E or beta sarcoglycanopathy. The agreed primary endpoint of EMERGENE is expression of beta-sarcoglycan, the absence of which is the sole cause of the disease. We are encouraged by the agency's willingness to create a viable pathway for SRP-9003, which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of ambulation and leads to early mortality. Assuming a positive [inaudible] meeting, we would anticipate [inaudible] in 2025, we are also very pleased to announce that we've received fast-track designation for SRP-9003. A fast-track designation is a process designed to facilitate the development, and expedite a reviewed drug which treat serious conditions and fill unmet medical needs. In addition to fast-track SRP-9003 has also been previously granted rare pediatric disease designation, and orphan drug designation from FDA, as well as orphan drug designation from the European Medicines Agency. The ability to progress a small NS-15 biomarker study, together with our ability to demonstrate delivery of a functional beta-sarcoglycan protein is extremely important, not just for this program but for the other sarcoglycanopathies in our pipeline, including LGMD2D and LGMD2C, those [inaudible] progressing to the clinic. [inaudible] successfully advance SRP-9003, we will submit our SRP-9004 IND update, reflecting our suspension process this year, with Phase I initiation expected to start by year's end. As a reminder SRP-9004 is designed for the treatment of Limb-Girdle Muscular Dystrophy type 2D for alpha-sarcoglycanopathy. Finally we're also rapidly progressing our program for SRP-9005 for the treatment of Limb-Girdle Muscular Dystrophies type 2C, or gamma-sarcoglycanopathy. We plan to engage with FDA in Q4c of this year, but plan to initiate the clinical study in Q1 2025, pending the outcome of that meeting. To summarize The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: They have demonstrated admirable dedication scientific integrity, and a passion for helping patients, but I'm extremely proud of.

Speaker Change: I'll now turn the call over to Ian <unk> for an update on our financial results Ian.

Ian: Thanks, so much good afternoon everybody.

Ian: Before I review, our financial results I wanted to discuss quickly three topics first I just want to add some color on our approach to revenue guidance.

Louise Rodino-Klapac: Assuming a positive [inaudible] meeting, we would anticipate [inaudible] in 2025, we are also very pleased to announce that we've received fast-track designation for SRP-9003. A fast-track designation is a process designed to facilitate the development, and expedite a reviewed drug which treat serious conditions and fill unmet medical needs. In addition to fast-track SRP-9003 has also been previously granted rare pediatric disease designation, and orphan drug designation from FDA, as well as orphan drug designation from the European Medicines Agency. The ability to progress a small NS-15 biomarker study, together with our ability to demonstrate delivery of a functional beta-sarcoglycan protein is extremely important, not just for this program but for the other sarcoglycanopathies in our pipeline, including LGMD2D and LGMD2C, those [inaudible] progressing to the clinic. [inaudible] successfully advance SRP-9003, we will submit our SRP-9004 IND update, reflecting our suspension process this year, with Phase I initiation expected to start by year's end. As a reminder SRP-9004 is designed for the treatment of Limb-Girdle Muscular Dystrophy type 2D for alpha-sarcoglycanopathy. Finally we're also rapidly progressing our program for SRP-9005 for the treatment of Limb-Girdle Muscular Dystrophies type 2C, or gamma-sarcoglycanopathy. We plan to engage with FDA in Q4c of this year, but plan to initiate the clinical study in Q1 2025, pending the outcome of that meeting. To summarize The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Louise Rodino-Klapac: Assuming a positive pre-BLA meeting, we would anticipate BLA filing in 2025. We are also very pleased to announce that we've received fast-track designation for SRP-9003. A fast-track designation is a process designed to facilitate the development, and expedite a reviewed drug which treat serious conditions and fill unmet medical needs. In addition to fast-track SRP-9003 has also been previously granted rare pediatric disease designation, and orphan drug designation from FDA, as well as orphan drug designation from the European Medicines Agency.

Speaker Change: We plan to refine the guidance as we get further into the launch, but I think what we provided today will help everyone model.

Speaker Change: Elaborate if launch curve more appropriately.

Speaker Change: Got it.

Speaker Change: Likely isn't surprising to anyone that the trajectory is consistent with our previous camera launches and the guidance also provide some visibility of our expectations and how we expect.

Louise Rodino-Klapac: The ability to progress a small NS-15 biomarker study, together with our ability to demonstrate delivery of a functional beta-sarcoglycan protein is extremely important, not just for this program but for the other sarcoglycanopathies in our pipeline, including LGMD2D and LGMD2C, those [inaudible] progressing to the clinic. [inaudible] successfully advance SRP-9003, we will submit our SRP-9004 IND update, reflecting our suspension process this year, with Phase I initiation expected to start by year's end. As a reminder SRP-9004 is designed for the treatment of Limb-Girdle Muscular Dystrophy type 2D for alpha-sarcoglycanopathy. Finally we're also rapidly progressing our program for SRP-9005 for the treatment of Limb-Girdle Muscular Dystrophies type 2C, or gamma-sarcoglycanopathy. We plan to engage with FDA in Q4c of this year, but plan to initiate the clinical study in Q1 2025, pending the outcome of that meeting. To summarize The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Louise Rodino-Klapac: The ability to progress a small NS-15 biomarker study, together with our ability to demonstrate delivery of a functional beta-sarcoglycan protein is extremely important, not just for this program but for the other sarcoglycanopathies in our pipeline, including LGMD2D and LGMD2C, both of which are progressing to the clinic. Having successfully advance SRP-9003, we will submit our SRP-9004 IND update, reflecting our suspension process this year, with Phase I initiation expected to start by year's end. As a reminder SRP-9004 is designed for the treatment of Limb-Girdle Muscular Dystrophy type 2D, or alpha-sarcoglycanopathy. Finally we're also rapidly progressing our program for SRP-9005 for the treatment of Limb-Girdle Muscular Dystrophies type 2C, or gamma-sarcoglycanopathy. We plan to engage with FDA in Q4 of this year, but plan to initiate the clinical study in Q1 2025, pending the outcome of that meeting.

Speaker Change: This will impact our P&L franchise.

Doug Ingram: Apparent that we expect modest modest cannibalization of the PMO as over the upcoming year. So all in all the broad takeaway from our guidance is that the <unk> launch will put us in a very very strong financial condition. In fact, as Doug mentioned, we are actually cash flow positive for the first time in our history, we expect to achieve sustainable cash flow positivity in them.

Louise Rodino-Klapac: As a reminder SRP-9004 is designed for the treatment of Limb-Girdle Muscular Dystrophy type 2D for alpha-sarcoglycanopathy. Finally we're also rapidly progressing our program for SRP-9005 for the treatment of Limb-Girdle Muscular Dystrophies type 2C, or gamma-sarcoglycanopathy. We plan to engage with FDA in Q4c of this year, but plan to initiate the clinical study in Q1 2025, pending the outcome of that meeting. To summarize The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Speaker Change: All of next year, but with a strong revenue growth, reaching almost through the end of the decade. We ran several questions on how do we plan to utilize our cash. So I wanted to briefly talk about how we're thinking about deploying capital in our BD strategy and before I launch into it I.

Louise Rodino-Klapac: Finally we're also rapidly progressing our program for SRP-9005 for the treatment of Limb-Girdle Muscular Dystrophies type 2C, or gamma-sarcoglycanopathy. We plan to engage with FDA in Q4c of this year, but plan to initiate the clinical study in Q1 2025, pending the outcome of that meeting. To summarize we're very pleased with the progress of our LGMD portfolio, and expect to have three of our LGMD's in the clinic in less that 9 months. We're maximizing the synergies across these platforms, from both an R&D and manufacturing perspective, and [inaudible] are firmly set on accelerating the remainder of the LGMD assets to the clinic, the subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline. Continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Louise Rodino-Klapac: Finally we're also rapidly progressing our program for SRP-9005 for the treatment of Limb-Girdle Muscular Dystrophies type 2C, or gamma-sarcoglycanopathy. We plan to engage with FDA in Q4 of this year, but plan to initiate the clinical study in Q1 2025, pending the outcome of that meeting. To summarize we're very pleased with the progress of our LGMD portfolio, and expect to have three of our LGMD's in the clinic in less that 9 months. We're maximizing the synergies across these platforms, from both an R&D and manufacturing perspective, and [inaudible] are firmly set on accelerating the remainder of the LGMD assets to the clinic, the subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline.

Louise Rodino-Klapac: We plan to engage with FDA in Q4c of this year, but plan to initiate the clinical study in Q1 2025, pending the outcome of that meeting. To summarize The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline, continuing with our RNA platform and beginning with our PMO. The study was fully enrolled with 228 patients.

Louise Rodino-Klapac: To summarize we're very pleased with the progress of our LGMD portfolio, and expect to have three of our LGMD's in the clinic in less that 9 months. We're maximizing the synergies across these platforms, from both an R&D and manufacturing perspective, and our sights are firmly set on accelerating the remainder of the LGMD assets to the clinic, the subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline.

Speaker Change: I think it often gets overlooked that we have a proven track record of successful business development. In fact, we wouldn't be here today and we had identified 11 is a differentiated approach to treating DMD and acquired it.

Louise Rodino-Klapac: Continuing with our RNA platform and beginning with our PMO. The ESSENCE trial, our post marketing requirements for GOLODIRSEN and CASIMERSEN, as well as MIS51ON, our post-marketing commitment for EXONDYS are both fully enrolled an remain on track. As a reminder, ESSENCE is a two year, two to one randomized placebo-controlled study ob both GOLODIRSEN and CASIMERSEN, and is due to read out in early 2026, this study was fully enrolled with 228 patients. MIS51ON is a randomized, double-blinded safety and efficacy, dose-binding study comparing the approved dosage of ETEPLIRSEN, 30mg/kg weekly, to a dosage that provide a significantly higher exposure, up to 200mg/kg weekly. MIS51ON is a two part Phase III study, it was fully enrolled with 160 patients, we remain committed to rapidly and diligently advancing MIS51ON and sharing data as soon as it becomes available. Turning to PPMO, early in the first quarter we announced positive results from

Louise Rodino-Klapac: Continuing with our RNA platform and beginning with our PMO. The ESSENCE trial, our post marketing requirements for GOLODIRSEN and CASIMERSEN, as well as MIS51ON, our post-marketing commitment for EXONDYS are both fully enrolled an remain on track. As a reminder, ESSENCE is a two year, two to one randomized placebo-controlled study ob both GOLODIRSEN and CASIMERSEN, and is due to read out in early 2026, this study was fully enrolled with 228 patients. MIS51ON is a randomized, double-blinded safety and efficacy, dose-binding study comparing the approved dosage of ETEPLIRSEN, 30mg/kg weekly, to a dosage that provide a significantly higher exposure, up to 200mg/kg weekly. MIS51ON is a two part Phase III study, it was fully enrolled with 160 patients. We remain committed to rapidly and diligently advancing MIS51ON and sharing data as soon as it becomes available.

Louise Rodino-Klapac: MIS51ON is a randomized, double-blinded safety and efficacy, dose-binding study comparing the approved dosage of ETEPLIRSEN, 30mg/kg weekly, to a dosage that provide a significantly higher exposure, up to 200mg/kg weekly. MIS51ON is a two part Phase III study, it was fully enrolled with 160 patients, we remain committed to rapidly and diligently advancing MIS51ON and sharing data as soon as it becomes available. Turning to PPMO, early in the first quarter we announced positive results from

Louise Rodino-Klapac: MIS51ON is a two part Phase III study, it was fully enrolled with 160 patients, we remain committed to rapidly and diligently advancing MIS51ON and sharing data as soon as it becomes available. Turning to PPMO, early in the first quarter we announced positive results from part b of our MOMENTUM study, or study SRP-5051-01, MOMENTUM is an ongoing study of SRP-5051, our investigational peptide conjugated PMO, or PPMO. We're actively engaging with Peter, regarding the [inaudible] study that could serve as a Post margin requirement or PMR study for a potential accelerated approval, agreement on the Phase III PMR study is a prerequisite for an accelerated approval filing. Our intent is to get [inaudible] study that considers the current landscape, can be completed in a reasonable timeframe, and reflects patient's reluctance to enroll in a placebo-controlled trial. Our hope is an agreement can be reached on a trial design this year. In summary, our team looks forward to a bright future ahead as we work diligently to advance our mission with the goal of serving patients around the world living with rare disease. I'd like to take a moment to thank my R&D colleagues for their unwavering devotion to advancing our entire portfolio, they've demonstrated admirable dedication, scientific integrity and a passion for helping patients that I'm extremely proud of. I'll now turn the call over to Ian Estepan for an update on our financial result. Ian.

Louise Rodino-Klapac: MIS51ON is a two part Phase III study, it was fully enrolled with 160 patients. We remain committed to rapidly and diligently advancing MIS51ON and sharing data as soon as it becomes available. Turning to PPMO, early in the first quarter we announced positive results from part b of our MOMENTUM study, or study SRP-5051-01, MOMENTUM is an ongoing study of SRP-5051, our investigational peptide conjugated PMO, or PPMO. We're actively engaging with Peter, regarding the [inaudible] study that could serve as a Post margin requirement or PMR study for a potential accelerated approval, agreement on the Phase III PMR study is a prerequisite for an accelerated approval filing.

Louise Rodino-Klapac: Turning to PPMO, early in the first quarter we announced positive results from part b of our MOMENTUM study, or study SRP-5051-01, MOMENTUM is an ongoing study of SRP-5051, our investigational peptide conjugated PMO, or PPMO. We're actively engaging with [inaudible] regarding the Phase III study that could serve as a post-margin requirement or PMR study for a potential accelerated approval. Agreement on the Phase III PMR study is a prerequisite for an accelerated approval filing.

Louise Rodino-Klapac: We're actively engaging with Peter, regarding the [inaudible] study that could serve as a Post margin requirement or PMR study for a potential accelerated approval, agreement on the Phase III PMR study is a prerequisite for an accelerated approval filing. Our intent is to get [inaudible] study that considers the current landscape, can be completed in a reasonable timeframe, and reflects patient's reluctance to enroll in a placebo-controlled trial. Our hope is an agreement can be reached on a trial design this year. In summary, our team looks forward to a bright future ahead as we work diligently to advance our mission with the goal of serving patients around the world living with rare disease. I'd like to take a moment to thank my R&D colleagues for their unwavering devotion to advancing our entire portfolio, they've demonstrated admirable dedication, scientific integrity and a passion for helping patients that I'm extremely proud of. I'll now turn the call over to Ian Estepan for an update on our financial result. Ian.

Louise Rodino-Klapac: Our intent is to get [inaudible] study that considers the current landscape, can be completed in a reasonable timeframe, and reflects patient's reluctance to enroll in a placebo-controlled trial. Our hope is an agreement can be reached on a trial design this year. In summary, our team looks forward to a bright future ahead as we work diligently to advance our mission with the goal of serving patients around the world living with rare disease. I'd like to take a moment to thank my R&D colleagues for their unwavering devotion to advancing our entire portfolio, they've demonstrated admirable dedication, scientific integrity and a passion for helping patients that I'm extremely proud of. I'll now turn the call over to Ian Estepan for an update on our financial result. Ian.

Louise Rodino-Klapac: Our intent is to gain agreement for a study that considers the current landscape, can be completed in a reasonable timeframe, and reflects patient's reluctance to enroll in a placebo-controlled trial. Our hope is an agreement can be reached on a trial design this year. In summary, our team looks forward to a bright future ahead as we work diligently to advance our mission with the goal of serving patients around the world living with rare disease. I'd like to take a moment to thank my R&D colleagues for their unwavering devotion to advancing our entire portfolio, they've demonstrated admirable dedication, scientific integrity and a passion for helping patients that I'm extremely proud of. I'll now turn the call over to Ian Estepan for an update on our financial result. Ian.

Louise Rodino-Klapac: I'd like to take a moment to thank my R&D colleagues for their unwavering devotion to advancing our entire portfolio, they've demonstrated admirable dedication, scientific integrity and a passion for helping patients that I'm extremely proud of. I'll now turn the call over to Ian Estepan for an update on our financial result. Ian. Thanks so much Louise, good afternoon everyone, before I review our financial results I want to discuss quickly just three topics. First, I just want to add some color on our approach to revenue guidance, we plan to refine the guidance as we get further into the launch, but I think what we've provided today will help everyone model the ELEVIDYS launch curve more appropriately. The guidance likely hasn't been surprising anyone as the trajectory is consistent with our previous PMO launches, and the guidance also provides some visibility of our expectations and how we expect ELEVIDYS will impact our PMO franchise, is apparent we would expect modest cannibalization of the PMOs over the upcoming year. So all and all, the broad takeaway from our guidance is that the ELEVIDYS launch will put us in a very very strong financial position, in fact as Doug mentioned, we're actually cash flow positive for the first time in our history, we expect to achieve sustainable cash flow positivity in the middle of next year. So with strong revenue growth reaching almost to the end of the decade, we're having several questions on how we plan to utilize our cash. So I want to briefly talk about how we're thinking about deploying capital on our BD strategy, and before I [inaudible] into it, I think it often gets overlooked that we have a proven track record of succesfull business development, in fact we wouldn't be here today if we hadn't identified ELEVIDYS as a differentiated approach to treating DMD and acquired it. We'll continue to leverage our capabilities in both the neuromuscular and rare disease space, and this will remain a core area of focus for us. We have the ability to use propensity-matched controls to better evaluate efficacy early in studies, which can provide valuable insight in determining the probability of success of a program.

Louise Rodino: We'll continue to leverage our capabilities in both the neuromuscular and rare disease space, and this will remain a core area of focus for us. We have the ability to use propensity-matched controls to better evaluate efficacy early in studies, which can provide valuable insight into determining the probability of success of a program.

Speaker Change: We will continue to lever our leverage.

Louise Rodino-Klapac: I'll now turn the call over to Ian Estepan for an update on our financial result. Ian.

Louise Rodino-Klapac: Thanks so much Louise, good afternoon everyone, before I review our financial results I want to discuss quickly just three topics. First, I just want to add some color on our approach to revenue guidance, we plan to refine the guidance as we get further into the launch, but I think what we've provided today will help everyone model the ELEVIDYS launch curve more appropriately. The guidance likely hasn't been surprising anyone as the trajectory is consistent with our previous PMO launches, and the guidance also provides some visibility of our expectations and how we expect ELEVIDYS will impact our PMO franchise, is apparent we would expect modest cannibalization of the PMOs over the upcoming year. So all and all, the broad takeaway from our guidance is that the ELEVIDYS launch will put us in a very very strong financial position, in fact as Doug mentioned, we're actually cash flow positive for the first time in our history, we expect to achieve sustainable cash flow positivity in the middle of next year. So with strong revenue growth reaching almost to the end of the decade, we're having several questions on how we plan to utilize our cash. So I want to briefly talk about how we're thinking about deploying capital on our BD strategy, and before I [inaudible] into it, I think it often gets overlooked that we have a proven track record of succesfull business development, in fact we wouldn't be here today if we hadn't identified ELEVIDYS as a differentiated approach to treating DMD and acquired it. We'll continue to leverage our capabilities in both the neuromuscular and rare disease space, and this will remain a core area of focus for us. We have the ability to use propensity-matched controls to better evaluate efficacy early in studies, which can provide valuable insight in determining the probability of success of a program.

Ian Estepan: Thanks so much Louise, good afternoon everyone, before I review our financial results I want to discuss quickly just three topics. First, I just want to add some color on our approach to revenue guidance, we plan to refine the guidance as we get further into the launch, but I think what we've provided today will help everyone model the ELEVIDYS launch curve more appropriately. The guidance likely hasn't been surprising anyone as the trajectory is consistent with our previous PMO launches, and the guidance also provides some visibility of our expectations and how we expect ELEVIDYS will impact our PMO franchise, is apparent we would expect modest cannibalization of the PMOs over the upcoming year.

Speaker Change: Leverage our capabilities and bulk neuromuscular rare disease space.

Louise Rodino-Klapac: The guidance likely hasn't been surprising anyone as the trajectory is consistent with our previous PMO launches, and the guidance also provides some visibility of our expectations and how we expect ELEVIDYS will impact our PMO franchise, is apparent we would expect modest cannibalization of the PMOs over the upcoming year. So all and all, the broad takeaway from our guidance is that the ELEVIDYS launch will put us in a very very strong financial position, in fact as Doug mentioned, we're actually cash flow positive for the first time in our history, we expect to achieve sustainable cash flow positivity in the middle of next year. So with strong revenue growth reaching almost to the end of the decade, we're having several questions on how we plan to utilize our cash. So I want to briefly talk about how we're thinking about deploying capital on our BD strategy, and before I [inaudible] into it, I think it often gets overlooked that we have a proven track record of succesfull business development, in fact we wouldn't be here today if we hadn't identified ELEVIDYS as a differentiated approach to treating DMD and acquired it. We'll continue to leverage our capabilities in both the neuromuscular and rare disease space, and this will remain a core area of focus for us. We have the ability to use propensity-matched controls to better evaluate efficacy early in studies, which can provide valuable insight in determining the probability of success of a program.

Speaker Change: It will remain a core area of focus of ours.

Louise Rodino-Klapac: So all and all, the broad takeaway from our guidance is that the ELEVIDYS launch will put us in a very very strong financial position, in fact as Doug mentioned, we're actually cash flow positive for the first time in our history, we expect to achieve sustainable cash flow positivity in the middle of next year. So with strong revenue growth reaching almost to the end of the decade, we're having several questions on how we plan to utilize our cash. So I want to briefly talk about how we're thinking about deploying capital on our BD strategy, and before I [inaudible] into it, I think it often gets overlooked that we have a proven track record of succesfull business development, in fact we wouldn't be here today if we hadn't identified ELEVIDYS as a differentiated approach to treating DMD and acquired it. We'll continue to leverage our capabilities in both the neuromuscular and rare disease space, and this will remain a core area of focus for us. We have the ability to use propensity-matched controls to better evaluate efficacy early in studies, which can provide valuable insight in determining the probability of success of a program.

Ian Estepan: So all and all, the broad takeaway from our guidance is that the ELEVIDYS launch will put us in a very very strong financial position, in fact as Doug mentioned, we're actually cash flow positive for the first time in our history, we expect to achieve sustainable cash flow positivity in the middle of next year. So with strong revenue growth reaching almost to the end of the decade, we're having several questions on how we plan to utilize our cash. So I want to briefly talk about how we're thinking about deploying capital on our BD strategy, and before I [inaudible] into it, I think it often gets overlooked that we have a proven track record of succesfull business development, in fact we wouldn't be here today if we hadn't identified ELEVIDYS as a differentiated approach to treating DMD and acquired it. We'll continue to leverage our capabilities in both the neuromuscular and rare disease space, and this will remain a core area of focus for us.

Doug Ingram: The ability to use propensity matched controls to better evaluate efficacy early in studies, which can provide valuable insight and determining the probability of success of the program. Doug also rightly noted that our commercial capability.

Louise Rodino-Klapac: So I want to briefly talk about how we're thinking about deploying capital on our BD strategy, and before I [inaudible] into it, I think it often gets overlooked that we have a proven track record of succesfull business development, in fact we wouldn't be here today if we hadn't identified ELEVIDYS as a differentiated approach to treating DMD and acquired it. We'll continue to leverage our capabilities in both the neuromuscular and rare disease space, and this will remain a core area of focus for us. We have the ability to use propensity-matched controls to better evaluate efficacy early in studies, which can provide valuable insight in determining the probability of success of a program.

Ian Estepan: So I want to briefly talk about how we're thinking about deploying capital on our BD strategy, and before I [inaudible] into it, I think it often gets overlooked that we have a proven track record of succesfull business development, in fact we wouldn't be here today if we hadn't identified ELEVIDYS as a differentiated approach to treating DMD and acquired it. We'll continue to leverage our capabilities in both the neuromuscular and rare disease space, and this will remain a core area of focus for us. We have the ability to use propensity-matched controls to better evaluate efficacy early in studies, which can provide valuable insight in determining the probability of success of a program. Doug also rightly noted that our commercial capabilities are second to none in rare disease, so we'll evaluate opportunities to see if we're best positioned to deliver transformative therapy for patients in need. We'll also look at areas that are tangental to [inaudible] muscular expertise, such as [inaudible] or cardiovascular, and continue to look to expand our capabilities.

Ian Estepan: So I want to briefly talk about how we're thinking about deploying capital on our BD strategy, and before I launch into it, I think it often gets overlooked that we have a proven track record of succesfull business development, in fact we wouldn't be here today if we hadn't identified ELEVIDYS as a differentiated approach to treating DMD and acquired it. We'll continue to leverage our capabilities in both the neuromuscular and rare disease space, and this will remain a core area of focus for us.

Louise Rodino-Klapac: We have the ability to use propensity-matched controls to better evaluate efficacy early in studies, which can provide valuable insight in determining the probability of success of a program. Doug also rightly noted that our commercial capabilities are second to none in rare disease, so we'll evaluate opportunities to see if we're best positioned to deliver transformative therapy for patients in need. We'll also look at areas that are tangental to [inaudible] muscular expertise, such as [inaudible] or cardiovascular, and continue to look to expand our capabilities. Now with all of this being said, and with the growth that we have in front of us, we're well positioned to transact, but we have no need to do anything immediately, we have the luxury to being able to find assets that squarely fit into our strategy. We'll have substantially more resources to deploy, but we'll continue to use the same financial discipline that has gotten us here today. As we've witnessed over the past week, market dynamics can change quite quickly, our strong fundamentals should somewhat insulate us from the macro environment and allow us to strategically deploy capital where appropriate.

Ian Estepan: Doug also rightly noted that our commercial capabilities are second to none in rare disease. We'll have substantially more resources to deploy, but we'll continue to use the same financial discipline that has gotten us here today. As we've witnessed over the past week, market dynamics can change quite quickly. Our strong fundamentals should somewhat insulate us from the macro environment and allow us to strategically deploy capital where appropriate.

Doug Ingram: And rare disease. So we will evaluate opportunities to see if we're best positioned to deliver transformative therapy of patients in need.

Ian Estepan: We have the ability to use propensity-matched controls to better evaluate efficacy early in studies, which can provide valuable insight in determining the probability of success of a program. Doug also rightly noted that our commercial capabilities are second to none in rare disease, so we'll evaluate opportunities to see if we're best positioned to deliver transformative therapy for patients in need. We'll also look at areas that are tangental to [inaudible] muscular expertise, such as [inaudible] or cardiovascular, and continue to look to expand our capabilities. Now with all of this being said, and with the growth that we have in front of us, we're well positioned to transact, but we have no need to do anything immediately, we have the luxury to being able to find assets that squarely fit into our strategy. We'll have substantially more resources to deploy, but we'll continue to use the same financial discipline that has gotten us here today. As we've witnessed over the past week, market dynamics can change quite quickly, our strong fundamentals should somewhat insulate us from the macro environment and allow us to strategically deploy capital where appropriate.

Speaker Change: Also look at areas that are tangential to our.

Speaker Change: Muscular expertise such as CNS cardiovascular and continue to look to expand our capabilities now with all this being said and with the growth that we have in front of US we are well positioned to transact, but we have no need to do anything immediately we have the luxury of being able to find assets that squarely fit into our strategy.

Louise Rodino-Klapac: Now with all of this being said, and with the growth that we have in front of us, we're well positioned to transact, but we have no need to do anything immediately, we have the luxury to being able to find assets that squarely fit into our strategy. We'll have substantially more resources to deploy, but we'll continue to use the same financial discipline that has gotten us here today. As we've witnessed over the past week, market dynamics can change quite quickly, our strong fundamentals should somewhat insulate us from the macro environment and allow us to strategically deploy capital where appropriate.

Ian Estepan: Now with all of this being said, and with the growth that we have in front of us, we're well positioned to transact, but we have no need to do anything immediately, we have the luxury to being able to find assets that squarely fit into our strategy. We'll have substantially more resources to deploy, but we'll continue to use the same financial discipline that has gotten us here today. As we've witnessed over the past week, market dynamics can change quite quickly, our strong fundamentals should somewhat insulate us from the macro environment and allow us to strategically deploy capital where appropriate.

Speaker Change: We will have substantially more resources to deploy but we will continue to use the same financial discipline that has gotten us here today.

Speaker Change: We've witnessed over the past week market dynamics can change quite quickly.

Speaker Change: Our strong fundamentals should somewhat insulate us from the macro environment and allow us to strategically deploy capital when appropriate.

Ian Estepan: So, finally, in the continued spirit of helping the street model our financials, I wanted to highlight that we've issued a notice to our second supplier to terminate our development and supply agreement related to our adherent manufacturing process for one of our gene therapy programs. As Doug mentioned, we're in a good place from a supply perspective, and based on several factors, including the progress we've made with our suspension manufacturing process, we do not believe that it makes strategic or financial sense to continue to invest in the adherent process for this program. The termination will be effective as of August 21, and we're currently performing analysis of the financial impact of the termination. As of today, we expect to record approximately 55 to 65 million in additional research and development expenses as a result of the termination for the three and nine months ended September 30th, 2024. This range is the net impact of any expected termination expenses right off in the reduction to operating expenses for applicable reimbursement costs under the Rocha agreement. We're still analyzing any further impact on the termination. That will be recorded.

Speaker Change: So finally, and the continued spirit of helping the street model our financials I wanted to highlight that we issued a notice of.

Speaker Change: <unk>.

Speaker Change: So our second supplier to terminate our development and supply agreement related to our manufacturing process for our Jeep for one of our gene therapy programs.

Speaker Change: Doug mentioned, we're in a good place from a supply perspective and based on several factors, including the progress we've made with our suspension manufacturing process. We do not believe that it makes strategic or financial sense to continue to invest in the year.

Speaker Change: For this program the termination will be effective as of August 21, and we're currently performing analysis of the financial impact of the termination.

Speaker Change: As of today, we expect to record approximately 55% to $65 million in additional research and development expenses as a result, the determination for the three and nine months ended September 32020 for this range as a net impact of any.

Speaker Change: <unk> termination expenses write offs and a reduction to operating expenses for applicable reimbursement costs under the Roche agreement.

Speaker Change: We're still analyzing any further impact from the termination that'll be recorded three months ended and nine months ended September 32024.

Speaker Change: Further reiterate that they've had no impact on our suspension based manufacturing agreements.

Speaker Change: Now moving to our financials. This afternoon financial results press release provided details for the second quarter of 2024 on a GAAP basis as well as a non-GAAP basis. Please refer to our press release available on <unk> website for a full reconciliation of GAAP to non-GAAP financial results.

Speaker Change: In the fourth quarter of 2023 amortization of license rights and income tax expense are no longer excluded from the non-GAAP results.

Ian Estepan: The termination will be effective as of August 21, and we're currently performing analysis of the financial impact of the termination. As of today, we expect to record approximately $55 to $65 million in additional research and development expenses as a result of the termination for the three and nine months, ended September 30th, 2024. This range is the net impact of any expected termination expenses right off in the reduction to operating expenses for applicable reimbursement costs under the Roche agreement. We're still analyzing any further impact on the termination, that will be recorded three months ended, and nine months ended September 30th, 2024. I want to further reiterate that this has no impact on our expansion [inaudible] manufacturing agreement, now moving to our financials, this afternoon's financial results press release provided details for the second quarter of 2024 on a GAAP basis as well as a non-GAAP basis, please refer to our press release available on Sarepta's website for a full reconciliation of GAAP and non-GAAP financial results. Ending in the 4th Quarter of 2023, amortization of [inaudible] and income tax expense are no longer excluded for non-GAAP results, the [inaudible] is added to income tax-effective adjustments, which represents the estimated income tax [inaudible] each pre-tax, non-GAAP adjustment based o the applicable effect of income tax rate. Non-GAAP financial results for the secon quarter of 2023 have been updated to reflect this change for comparability purposes. For the three months end at June 30th 2024, the company recorded total revenues of $362.9 million dollars, which consisted in net product revenues, and collaboration revenues, and other revenues compared to revenues of $261.2 million dollars for the same period of 2023, an increase of $101.7 million dollars. Net product revenue for the second quarter of 2024 from ELEVIDYS, was $121.7 million dollars, net product revenue for the second quarter of 2024 from our PMO Exon skipping franchise was $238.8 million dollars, compared to $239 million dollars for the same period of 2023. For the second quarter of 20204

Speaker Change: The company has added the income tax effect of adjustments, which represent the estimated income tax impact of each pretax non-GAAP adjustments based on the applicable effective income tax rate non-GAAP financial results for the second quarter of 2023 have been updated to reflect this change for comparability purposes.

Speaker Change: For the three months ended June 32024, the company recorded total revenues of $362 9 million.

Ian Estepan: We're still analyzing any further impact on the termination, that will be recorded three months ended, and nine months ended September 30th, 2024. I want to further reiterate that this has no impact on our expansion [inaudible] manufacturing agreement, now moving to our financials, this afternoon's financial results press release provided details for the second quarter of 2024 on a GAAP basis as well as a non-GAAP basis, please refer to our press release available on Sarepta's website for a full reconciliation of GAAP and non-GAAP financial results. Ending in the 4th Quarter of 2023, amortization of [inaudible] and income tax expense are no longer excluded for non-GAAP results, the [inaudible] is added to income tax-effective adjustments, which represents the estimated income tax [inaudible] each pre-tax, non-GAAP adjustment based o the applicable effect of income tax rate. Non-GAAP financial results for the secon quarter of 2023 have been updated to reflect this change for comparability purposes. For the three months end at June 30th 2024, the company recorded total revenues of $362.9 million dollars, which consisted in net product revenues, and collaboration revenues, and other revenues compared to revenues of $261.2 million dollars for the same period of 2023, an increase of $101.7 million dollars. Net product revenue for the second quarter of 2024 from ELEVIDYS, was $121.7 million dollars, net product revenue for the second quarter of 2024 from our PMO Exon skipping franchise was $238.8 million dollars, compared to $239 million dollars for the same period of 2023. For the second quarter of 20204

Ian Estepan: We're still analyzing any further impact on the termination, that will be recorded three months ended, and nine months ended September 30th,

Speaker Change: Which consisted of net product revenue and collaboration revenue and other revenues compared to revenues of $261 $2 million for the same period of 2023, an increase of $101 $7 million net.

Ian Estepan: 2024. I want to further reiterate that this has no impact on our expansion [inaudible] manufacturing agreement, now moving to our financials, this afternoon's financial results press release provided details for the second quarter of 2024 on a GAAP basis as well as a non-GAAP basis, please refer to our press release available on Sarepta's website for a full reconciliation of GAAP and non-GAAP financial results. Ending in the 4th Quarter of 2023, amortization of [inaudible] and income tax expense are no longer excluded for non-GAAP results, the [inaudible] is added to income tax-effective adjustments, which represents the estimated income tax [inaudible] each pre-tax, non-GAAP adjustment based o the applicable effect of income tax rate. Non-GAAP financial results for the secon quarter of 2023 have been updated to reflect this change for comparability purposes. For the three months end at June 30th 2024, the company recorded total revenues of $362.9 million dollars, which consisted in net product revenues, and collaboration revenues, and other revenues compared to revenues of $261.2 million dollars for the same period of 2023, an increase of $101.7 million dollars. Net product revenue for the second quarter of 2024 from ELEVIDYS, was $121.7 million dollars, net product revenue for the second quarter of 2024 from our PMO Exon skipping franchise was $238.8 million dollars, compared to $239 million dollars for the same period of 2023. For the second quarter of 20204

Ian Estepan: I want to further reiterate that this has no impact on our [inaudible] based manufacturing agreements. Now moving to our financials, this afternoon's financial results press release provided details for the second quarter of 2024 on a GAAP basis as well as a non-GAAP basis, please refer to our press release available on Sarepta's website for a full reconciliation of GAAP and non-GAAP financial results. Ending in the 4th Quarter of 2023, amortization of [inaudible] and income tax expense are no longer excluded for non-GAAP results, the [inaudible] is added to income tax-effective adjustments, which represents the estimated income tax [inaudible] each pre-tax, non-GAAP adjustment based o the applicable effect of income tax rate.

Ian Estepan: I want to further reiterate that this has no impact on our [inaudible] based manufacturing agreements.

Ian Estepan: Now moving to our financials, this afternoon's financial results press release provided details for the second quarter of 2024 on a GAAP basis as well as a non-GAAP basis, please refer to our press release available on Sarepta's website for a full reconciliation of GAAP and non-GAAP financial results. Ending in the fourth quarter of 2023, amortization of [inaudible] and income tax expense are no longer excluded from non-GAAP results. The company has added the income tax-effective adjustments, which represents the estimated income tax impact of each pre-tax, non-GAAP adjustment based on the applicable effect of income tax rate.

Speaker Change: Net product revenue for the second quarter of 2024 from <unk> with $121 $7 million.

Speaker Change: Net product revenue for the second quarter of 2024 from our PMO exon skipping franchise was $238 $8 million.

Ian Estepan: Ending in the 4th Quarter of 2023, amortization of [inaudible] and income tax expense are no longer excluded for non-GAAP results, the [inaudible] is added to income tax-effective adjustments, which represents the estimated income tax [inaudible] each pre-tax, non-GAAP adjustment based o the applicable effect of income tax rate. Non-GAAP financial results for the secon quarter of 2023 have been updated to reflect this change for comparability purposes. For the three months end at June 30th 2024, the company recorded total revenues of $362.9 million dollars, which consisted in net product revenues, and collaboration revenues, and other revenues compared to revenues of $261.2 million dollars for the same period of 2023, an increase of $101.7 million dollars. Net product revenue for the second quarter of 2024 from ELEVIDYS, was $121.7 million dollars, net product revenue for the second quarter of 2024 from our PMO Exon skipping franchise was $238.8 million dollars, compared to $239 million dollars for the same period of 2023. For the second quarter of 20204

Speaker Change: <unk> hundred $39 million for the same period of 2023.

Speaker Change: For the second quarter of 2024 individual and net product sales were $129 $8 million for exon 50, $177 4 million for a minus 45, and $31 6 million or <unk> 53, the increase in net product revenue primarily reflects the net product revenue associated with sales of <unk>.

Ian Estepan: Non-GAAP financial results for the secon quarter of 2023 have been updated to reflect this change for comparability purposes. For the three months end at June 30th 2024, the company recorded total revenues of $362.9 million dollars, which consisted in net product revenues, and collaboration revenues, and other revenues compared to revenues of $261.2 million dollars for the same period of 2023, an increase of $101.7 million dollars. Net product revenue for the second quarter of 2024 from ELEVIDYS, was $121.7 million dollars, net product revenue for the second quarter of 2024 from our PMO Exon skipping franchise was $238.8 million dollars, compared to $239 million dollars for the same period of 2023. For the second quarter of 20204

Speaker Change: For the three months ended June 32024, the company recognized $2 $4 million of collaboration other revenues, which primarily relates to royalty revenue from Roche compared to collaboration revenues of $22 3 million for the same period of 2023, a decrease of $19 $9 million.

Ian Estepan: Three months ended, and nine months ended September 30th, 2020. The reimbursable co-development costs under the Roche Agreement totaled $17.9 million for the second quarter of 2024, compared to $28.2 million for the same period of 2023. On a gap basis, we reported a net income of $6.5 million, or $0.07 per basic and diluted share, and a net loss of $23.9 million, or $0.27 per basic and diluted share, for the second quarters of 2024 and 2023, respectively.

Three months ended, and nine months ended September 30th, 2020.

Speaker Change: The reimbursement co development across the Roche agreement totaled $17 $9 million for the second quarter of 2024 compared to $28 2 million for the same period of 2023.

Ian Estepan: For the second quarter of 2024 individual net product sales where $129.8 million dollars for EXONDYS 51, $77.4 million dollars for AMONDYS 45 and $31.6 million dollars for VYONDYS 53, an increase in Net product revenue primarily reflects the Net product revenue associates with sales of ELEVIDYS. For the three months ending June 30th 2024 the company recognized $2.4 million dollars of collaboration and other revenues which primarily relates to royalty revenues from Roche, compared to collaboration revenues of $22.3 million dollars for the same period of 2023, a decrease of $19.9 million dollars. The reimbursable co-development costs under the Roche Agreement totaled $17.9 million dollars for the second quarter of 2024, compared to $28.2 million dollars for the same period of 2023. On a gap basis, we reported a net income of $6.5 million dollars, or $0.07 per basic and diluted share, and a net loss of $23.9 million dollars, or $0.27 per basic and diluted share, for the second quarters of 2024 and 2023, respectively. We reported non-GAAP net income of $46.7 million dollars or $0.44 per share in the second quarter of 2024, compared to a non-GAAP net loss of $89.9 million dollars or $1.01 per diluted share in the second quarter of 2024.

The reimbursable co-development costs under the Roche Agreement totaled $17.9 million for the second quarter of 2024, compared to $28.2 million for the same period of 2023. On a gap basis, we reported a net income of $6.5 million, or $0.07 per basic and diluted share, and a net loss of $23.9 million, or $0.27 per basic and diluted share, for the second quarters of 2024 and 2023, respectively.

Speaker Change: On a GAAP basis, we reported net income of $6 5 million or seven cents per basic and diluted share and a net loss of $23 9 million or 27 cents per basic and diluted share for the second quarter of 2024 and 2023, respectively.

Ian Estepan: The reimbursable co-development costs under the Roche Agreement totaled $17.9 million dollars for the second quarter of 2024, compared to $28.2 million dollars for the same period of 2023. On a gap basis, we reported a net income of $6.5 million dollars, or $0.07 per basic and diluted share, and a net loss of $23.9 million dollars, or $0.27 per basic and diluted share, for the second quarters of 2024 and 2023, respectively. We reported non-GAAP net income of $46.7 million dollars or $0.44 per share in the second quarter of 2024, compared to a non-GAAP net loss of $89.9 million dollars or $1.01 per diluted share in the second quarter of 2024.

Speaker Change: We reported non-GAAP net income of $46 7 million or <unk> 44 per share in the second quarter of 2024 compared to a non-GAAP net loss of $89 9 million or $1 <unk> 10 per base per diluted share in the second quarter of 2023.

Ian Estepan: In the second quarter of 2024, we recorded approximately $44.5 million dollars in cost of sales compared to $34.1 million dollars in the same period of 2023. The increase in cost of sales primarily reflects the cost of sales related to ELEVIDYS during the three months ended June 30th, 2024, following it's FDA approval and launch in June of 2023, with no similar activity for the same period of 2023. On a gap basis, we recorded $179.7 million dollars and $241.99 million dollars in R&D expenses for the second quarter of 2024 and 2023, respectively, a year over year decrease of $62.2 million dollars. The decrease is primarily driven due to the capitalization of commercial batches of ELEVIDYS manufactured after it's approval in June of 2023. On a non-GAAP basis, R&D expenses were $153.9 million dollars for the second quarter of 2024, compared to $212.2 million dollars for the same period of 2023, a decrease of $58.3 million dollars. Now turning to SG&A, on a GAAP basis we recorded approximately $138.8 million dollars and $118.6 million dollars in expenses for the second quarter of 2024 and 2023 respectively, an increase of $20.2 million dollars. The increase was primarily driven by an increase in professional services due to the launch of ELEVIDYS and ongoing litigation matters, the timing of charitable contributions, as well as the achievement of performance conditions related to certain performance stock units.

Speaker Change: In the second quarter of 2024, we recorded approximately $44 5 million in cost of sales compared to $34 $2 million in the same period of time three the increase in cost of sales primarily reflects the cost of sales related to <unk>. During the three months ended June 32024, following its FDA approval and launch in June.

Speaker Change: And in 2023 with no similar activity for the same period of 2023.

Speaker Change: On a GAAP basis, we recorded $179 $7 million and 240 to $1 $99 million in R&D expenses for the second quarter of 2024, and 2023, respectively, a year over year decrease of $62 $2 million.

Ian Estepan: The decrease is primarily driven due to the capitalization of commercial batches of ELEVIDYS manufactured after it's approval in June of 2023. On a non-GAAP basis, R&D expenses were $153.9 million dollars for the second quarter of 2024, compared to $212.2 million dollars for the same period of 2023, a decrease of $58.3 million dollars. Now turning to SG&A, on a GAAP basis we recorded approximately $138.8 million dollars and $118.6 million dollars in expenses for the second quarter of 2024 and 2023 respectively, an increase of $20.2 million dollars. The increase was primarily driven by an increase in professional services due to the launch of ELEVIDYS and ongoing litigation matters, the timing of charitable contributions, as well as the achievement of performance conditions related to certain performance stock units.

Speaker Change: The decrease was primarily due to <unk>.

Speaker Change: Capitalization of <unk> commercial batches of <unk> manufactured after the approval in June of 2023.

Speaker Change: non-GAAP basis, R&D expenses were $153 9 million for the second quarter of 2024 compared to $12 $2 million for the same period of 2023, a decrease of $58 3 million now turning to SG&A on a GAAP basis, we recorded approximately $138 eight.

Speaker Change: $8 million and $118 $6 million of expenses for the second quarter of 2024, and 2023, respectively. An increase of $22 million. The increase was primarily driven by an increase in professional services use of the launch of <unk> and ongoing litigation matters, the timing charitable contribution as well.

Ian Estepan: The increase was primarily driven by an increase in professional services due to the launch of ELEVIDYS and ongoing litigation matters, the timing of charitable contributions, as well as the achievement of performance conditions related to certain performance stock units. On a non-GAAP basis, the SG&A expenses were $106 million dollars for the second quarter of 2024, compared to $90.3 million for the same period of 2023, an increase of $15.7 million dollars. On a GAAP basis, we recorded $14.3 million dollars in other income net for the second quarter of 2024, compared to $16.9 million dollars for the same period of 2023. The change is primarily due to a decrease in the accretion of our investment discount net due to the investment mix of our investment portfolio. We have approximately $1.5 billion in cash, cash equivalents, and investments in long-term restricted cash as of June 30, 2024. And with that, I'll turn the call back over to Doug to start the Q&A. Doug.

Speaker Change: The achievement of performance conditions related to certain performance stock units.

Ian Estepan: On a non-GAAP basis, SG&A expenses were $106 million for the second quarter of 2024 compared to $90.3 million for the same period of 2023, an increase of $15.7 million. On a GAAP basis, we recorded $14.3 million in other income net for the second quarter of 2024, compared to $16.9 million for the same period of 2023. The change is primarily due to a decrease in the accretion of our investment discount net due to the investment mix of our investment portfolio. We have approximately $1.5 billion in cash, cash equivalents, and investments in long-term restricted cash as of June 30, 2024. And with that, I'll turn the call back over to Doug to start the Q&A. Okay?

Speaker Change: On a non-GAAP basis, the SG&A expenses were $106 million for the <unk>.

Speaker Change: Second quarter of 2024 compared to $93 million for the same period of 2023, an increase of $15 7 million.

Speaker Change: On a GAAP basis, we recorded a $14 $3 million and other income net for the second quarter of 2024 to.

Speaker Change: $16 9 million for the same period of 2023. The change is primarily due to decrease in the accretion of our investment discount due to the investment mix of our investment portfolio.

Ian Estepan: The change is primarily due to a decrease in the accretion of our investment discount net due to the investment mix of our investment portfolio. We have approximately $1.5 billion in cash, cash equivalents, and investments in long-term restricted cash as of June 30, 2024. And with that, I'll turn the call back over to Doug to start the Q&A. Doug.

Doug Ingram: We had approximately $1 $5 billion in cash cash equivalents and investments in long term restricted cash as of June 32024, and with that I'll turn the call back over to Doug to start the Q&A.

Douglas Ingram: Thank you very much. Ian, Lisa, let's open the lines for Q&A. Thank you, as a reminder if you'd like to ask a question please press star 11 on your telephone, we also ask that you wait for your name and the company to be announced before proceeding with your question, please keep your question down to one question. One moment and we'll be ready for the first question. And our first question today will be coming from Tazeen Ahmad of Bank of America securities, your line is open.

Douglas S. Ingram: Thank you very much. Ian, Lisa, let's open the lines for Q&A.

Doug Ingram: Thank you very much Lisa let's open the lines for Q&A.

Speaker Change: Thank you.

Operator: Thank you, as a reminder if you'd like to ask a question please press star 11 on your telephone, we also ask that you wait for your name and the company to be announced before proceeding with your question, please keep your question down to one question. One moment and we'll be ready for the first question. And our first question today will be coming from Tazeen Ahmad of Bank of America securities, your line is open.

Doug Ingram: As a reminder, if you would like to ask a question. Please press star one on your telephone.

Doug Ingram: Also ask that you wait for your name and company should be announced before proceeding with your question. Please keep your questions down to one question, one moment and we'll be ready for the first question.

Speaker Change: Yes.

Speaker Change: And our first question today will be coming from <unk> <unk> of Bank of America Securities. Your line is open.

Speaker Change: Hi, guys. Good afternoon, Thanks for taking my question.

Speaker Change: Maybe I just wanted to get an understanding of what the actual bottleneck here is because based on everything that you said and based on work that seemingly a lot of folks have done demand seems to be quite high from patients and physicians.

unknown: Hi guys, good afternoon, thanks for taking my questions. maybe I just want to get an understanding of what the actual bottleneck here is? Because based on everything that you said, and based on work that seemingly a lot of folks have done, demand seems to be quite high from patients and physicians. You talked about start forms, and you know you're pleased with the number of start forms, but what is the issue with the timing of getting the patient journey from prescription to actual drug in hand? Has that changed? You know, from the time that you got approved for the four to five-year-olds, when did it start lengthening to what you're saying, what is it, three to six months? That is going to take. Three to five about three to five months. When did that start?

Tazeen Ahmad: Hi guys, good afternoon, thanks for taking my questions. maybe I just want to get an understanding of what the actual bottleneck here is? Because based on everything that you said, and based on work that seemingly a lot of folks have done, demand seems to be quite high from patients and physicians. You talked about start forms, and you know you're pleased with the number of start forms, but what is the issue with the timing of getting the patient journey from prescription to actual drug in hand? Has that changed? You know, from the time that you got approved for the four to five-year-olds, when did it start lengthening to what you're saying, what is it, three to six months? That is going to take.

Speaker Change: You talked about start forms in.

Tazeen Ahmad: You know, from the time that you got approved for the four to five-year-olds, when did it start lengthening to what you're saying, what is it, three to six months? That is going to take.

Speaker Change: We're pleased with the number of start forms.

Three to five about three to five months. When did that start?

Douglas S. Ingram: Three to five.

Tazeen Ahmad: About three to five months, when did that started? Is that the key bottleneck here? Thanks.

unknown: Is that the key bottleneck here? Thanks.

Speaker Change: But what is the issue with the timing of getting the patient journey from from prescription to actual drug in hand has that changed from.

Speaker Change: For the time that you've got approved for four to five year olds. When did it start lengthening so what youre, saying what is it three to six months that its going to agreed upon.

Speaker Change: Five months.

Speaker Change: When did that start it is that the key bottleneck here. Thanks.

Douglas S. Ingram: Thank you very much for your questions, the short answer is there really is no bottleneck at all. Now, as I think we said in the last earnings call, it's clearly the case that with the four to five-year-olds, we were all in, and I mean all, not just us, physicians, families, and the payers, we're all in kind of a crisis mode, prioritizing kids that were about to age out of the label, and we're able to do it more rapidly than is normal. But the normal process is about three to five months, and I mean, normal, that's not atypical for these sorts of therapies, but it's very typical for EXONDYS, VYONDYS, AMONDYS , and now, ELEVIDYS. And ELEVIDYS has some additional requirements, including, for instance, the requirement that one test for it is negative for neutralizing antibodies.

Speaker Change: Thank you very much for your question. The short answer is there really is no bottleneck at all now it is as we said in the last earnings call.

Douglas Ingram: Now, as I think we said in the last earnings call, it's clearly the case that with the four to five-year-olds, we were all in, and I mean all, not just us, physicians, families, and the payers, we're all in kind of a crisis mode, prioritizing kids that were about to age out of the label, and we're able to do it more rapidly than is normal, but the normal process is And I mean, normal. That's not atypical for these sorts of therapies, but it's very typical for exonus, myonus, imundus, and now, elevitus. And Eleutherius has some additional requirements, including, for instance, the requirement that one test for it is negative for neutralizing antibodies.

Speaker Change: Clearly the case that with the four to five year olds, we will all in.

Speaker Change: All not just us physicians families and the Payors were all in kind of a crisis mode prioritizing kids that were about to age out of the label and we were able to do it more rapidly than is normal but the normal process is about three to five months and I mean normal.

Douglas S. Ingram: But the normal process is about three to five months, and I mean, normal, that's not atypical for these sorts of therapies, but it's very typical for EXONDYS, VYONDYS, AMONDYS , and now, ELEVIDYS. And ELEVIDYS has some additional requirements, including, for instance, the requirement that one test for it is negative for neutralizing antibodies. So to be very clear, there is no bottleneck, we're doing brilliantly, the start forms are great, patient and physician demand is great, manufacturing is great, everything is going very, very well. Payer interaction are really, really encouraging, I think Dallan mentioned we've been having conversations for quite a long time, but just recently, we had conversations with payers that cover some 225, almost a quarter of a billion lives in the United States, and that's enormous, And they've been very productive, and we've already seen pulled through good policies, now, some of these policies may take some time. So, just so we're very clear, it takes about three to five months from start form to infusion, we get an enormous number of start forms, we're getting a crazy number of assay kit requirements. We had to literally go into our own mini-crisis mode to make sure we could satisfy all of the assays that were necessary, [inaudible] getting approval.

Speaker Change: Not atypical for these sorts of therapies, but it's very typical for Exxon as violent as a modest and now <unk> had some additional requirements, including for instance, the requirement.

Speaker Change: One test for it is negative for neutralizing antibodies so to be very clear there is no bottleneck here, we're doing brilliantly.

Douglas Ingram: So to be very clear, there is no bottle of We're doing brilliantly. The start forms are great. Patient and physician demand is great, and manufacturing is also great. Everything is going very, very well. Payer interaction is really, really encouraging. I think Dallan mentioned we've been having conversations for quite a long time, but just recently, we had conversations with payers that cover some 225, almost a quarter of a billion lives in the United States. And that's enormous.

Speaker Change: Start forms are great.

Speaker Change: <unk> and physician demand is great manufacturing is great everything is going very very well payer interactions are really really encouraging I think Darren mentioned, we've been having conversations for a quite a long time, but just recently we've had conversations with.

Douglas S. Ingram: Payer interaction are really, really encouraging, I think Dallan mentioned we've been having conversations for quite a long time, but just recently, we had conversations with payers that cover some 225, almost a quarter of a billion lives in the United States, and that's enormous, And they've been very productive, and we've already seen pulled through good policies, now, some of these policies may take some time. So, just so we're very clear, it takes about three to five months from start form to infusion, we get an enormous number of start forms, we're getting a crazy number of assay kit requirements. We had to literally go into our own mini-crisis mode to make sure we could satisfy all of the assays that were necessary, [inaudible] getting approval, and so this just is going to take that amount of time. We're doing brilliantly, the last quarter, Q2, of course, was all predicated on the prior label, and we did really well there, you'll notice that we were basically flat, we would have been perfectly flat, but you know, a couple of kids that were scheduled toward the back half of the quarter actually had a viral infection and then were pushed into this quarter, and they'll be dosed this quarter, so we're not losing them. And then it's going to take, you know, three to five months, that means that we're going to have nice growth in Q3, but it'll be moderated, and then Q4 will be very strong growth, as we've mentioned, more than double the growth in Q4 of this year, and then, as we model right now, based on everything we're seeing. on this planet.

Darren: Payers that cover some 225, almost a quarter of a 1 billion lives in the United States, that's enormous and they've been very productive and we've already seen pulled through good policy now somebody's policy that takes some time. So just we're very clear it takes about three to five months.

Douglas Ingram: And they've been very productive, and we've already seen good policies implemented. Now, some of these policies may take some time. So, just so we're very clear, it takes about three to five months from start form to infusion. We get an enormous number of start forms, we get a crazy number of assay kit requirements. We had to literally go into our own mini-crisis mode to make sure we could satisfy all of the assays that were necessary, predicated on getting approval.

Darren: From start form to infusion, we're getting an enormous number of start forms were getting.

Speaker Change: Crazy number of assay kit requirements, we had to literally go into our own mini crisis mode to make sure we could satisfy all of the assays that were necessary.

Speaker Change: Predicate to getting approval and so this is just it's going to take that amount of time, we're doing brilliantly.

Douglas Ingram: And so this just is going to take that amount of time. We're doing brilliantly. The last quarter, Q2, of course, was all predicated on the prior label, and we did really well there. You'll notice that we were basically flat; we would have been perfectly flat, but you know, a couple of kids that were scheduled toward the back half of the quarter actually had a viral infection and then were pushed into this quarter, and they'll be dosed this quarter, so we're not losing them.

Darren: The last quarter Q2 of course was all predicated on the private label and we did really well there you'll notice that we were basically flat we would have been perfectly flat, but a couple of kids that were.

Douglas S. Ingram: We had to literally go into our own mini-crisis mode to make sure we could satisfy all of the assays that were necessary, [inaudible] getting approval, and so this just is going to take that amount of time. We're doing brilliantly, the last quarter, Q2, of course, was all predicated on the prior label, and we did really well there, you'll notice that we were basically flat, we would have been perfectly flat, but you know, a couple of kids that were scheduled toward the back half of the quarter actually had a viral infection and then were pushed into this quarter, and they'll be dosed this quarter, so we're not losing them. And then it's going to take, you know, three to five months, that means that we're going to have nice growth in Q3, but it'll be moderated, and then Q4 will be very strong growth, as we've mentioned, more than double the growth in Q4 of this year, and then, as we model right now, based on everything we're seeing. on this planet.

Douglas S. Ingram: We had to literally go into our own mini-crisis mode to make sure we could satisfy all of the assays that were necessary, predicate to getting approval, and so this just is going to take that amount of time. We're doing brilliantly, the last quarter, Q2, of course, was all predicated on the prior label, and we did really well there, you'll notice that we were basically flat, we would have been perfectly flat. But you know, a couple of kids that were scheduled toward the back half of the quarter actually had a viral infection and then were pushed into this quarter, and they'll be dosed this quarter, so we're not losing them.

Speaker Change: Scheduled toward the back half of the quarter actually had a viral infection and they've been pushed into this quarter and they will be dosed. This quarter. So we're not losing them and then it's going to take three to five months that means that we're going to have nice growth in Q3, but it'll be moderated and then Q4 will be very strong growth as we've mentioned with more than <unk>.

Douglas Ingram: And then it's going to take, you know, three to five months. That means that we're going to have nice growth in Q3, but it'll be moderated, and then Q4 will be very strong growth, as we've mentioned, more than double the growth in Q4 of this year. And then, as we model right now, based on everything we're seeing on this planet.

Darren: Double the growth in Q4 of this year and then as we model right now based on everything we're seeing.

Darren: We're going to do between $2 9 billion and $3 $1 billion in revenue across the board therapy next year, which speaks to the success that we believe is happening with <unk> and it speaks to the continuing success of our PMO and the fact that we're seeing fairly modest.

John: Cannibalization that we imagine will see fairly modest cannibalization into next year. So the short answer is things are going very well there is no fundamental bottleneck. It's just a process that one has to go through we've been dealing with it for very long John maybe this is what Sean just as likely whereby all of this is like if you were a modest lag.

John: And just I know I'm getting a bit of a ramp here, but hopefully I'll answer everybody's questions that Fred just so we're very clear.

Douglas S. Ingram: And then it's going to take, you know, three to five months, that means that we're going to have nice growth in Q3, but it'll be moderated, and then Q4 will be very strong growth, as we've mentioned, more than double the growth in Q4 of this year, and then, as we model right now, based on everything we're seeing. We're going to do between $2.9 billion and $3.1 billion dollars in revenue across [inaudible] therapy next year, which speaks to the success we believe is happening with ELEVIDYS, and speaks to the continuing success of our PMOs and the fact that we're seeing fairly modest cannibalization, and we imagine we'll see fairly modest cannibalization into next year. So the short answer is, things are going very well, there is no fundamental bottleneck, it's just a process that one has to go through, we've been dealing with it for a very long time, this is what EXONDYS was like, what VYONDYS was like, what AMONDYS is like. And just I would, I know I'm getting on a bit of a rant here, but I'm going to answer everybody's questions up front just so we're very clear, I mean we're seeing exactly the sort of ramp one would have anticipated, in fact a little bit more optimistic than even we were and as you know, we're optimistic folks, and is very consistent with the launch curves that we've seen with our other three approved therapies. So I will end this soliloquy with the statement I made in the earnings script itself, which is, you can say a lot of things about us but no one should dispute that there is no organization on this planet.

Speaker Change: We are seeing is exactly the sort of ramp one would have anticipated in fact, a little bit more optimistic than we were and as you know we are optimistic folks and it's very consistent with the launch curves that we've seen with our other three approved therapies. So I will end. This this soliloquy with the statement I made.

Douglas S. Ingram: And speaks to the continuing success of our PMOs and the fact that we're seeing fairly modest cannibalization, and we imagine we'll see fairly modest cannibalization into next year. So the short answer is, things are going very well, there is no fundamental bottleneck, it's just a process that one has to go through, we've been dealing with it for a very long time, this is what EXONDYS was like, what VYONDYS was like, what AMONDYS is like. And just I would, I know I'm getting on a bit of a rant here, but I'm going to answer everybody's questions up front just so we're very clear.

Douglas S. Ingram: So the short answer is, things are going very well, there is no fundamental bottleneck, it's just a process that one has to go through, we've been dealing with it for a very long time, this is what EXONDYS was like, what VYONDYS was like, what AMONDYS is like. And just I would, I know I'm getting on a bit of a rant here, but I'm going to answer everybody's questions up front just so we're very clear, I mean we're seeing exactly the sort of ramp one would have anticipated, in fact a little bit more optimistic than even we were and as you know, we're optimistic folks, and is very consistent with the launch curves that we've seen with our other three approved therapies. So I will end this soliloquy with the statement I made in the earnings script itself, which is, you can say a lot of things about us but no one should dispute that there is no organization on this planet.

Douglas S. Ingram: So the short answer is, things are going very well, there is no fundamental bottleneck, it's just a process that one has to go through, we've been dealing with it for a very long time, this is what EXONDYS was like, what VYONDYS was like, what AMONDYS is like. And just I would, I know I'm getting on a bit of a rant here, but I'm going to answer everybody's questions up front just so we're very clear. I mean we're seeing exactly the sort of ramp one would have anticipated, in fact a little bit more optimistic than even we were and as you know, we're optimistic folks, and is very consistent with the launch curves that we've seen with our other three approved therapies.

Speaker Change: The earnings script itself, which as.

Douglas S. Ingram: I mean we're seeing exactly the sort of ramp one would have anticipated, in fact a little bit more optimistic than even we were and as you know, we're optimistic folks, and is very consistent with the launch curves that we've seen with our other three approved therapies. So I will end this soliloquy with the statement I made in the earnings script itself, which is, you can say a lot of things about us but no one should dispute that there is no organization on this planet with more expertise, more ability and a better track record in launching Duchenne Therapy, serving this community, and making these launches a success. We're in really good shape right now, but thank you very much for your question, Tazeen. I appreciate it. Thank you. One moment for the next question. Then our next question will be coming from Anupam. Rama of J&P ULINAS. Hey guys, thanks for taking the questions.

Douglas S. Ingram: I mean we're seeing exactly the sort of ramp one would have anticipated, in fact a little bit more optimistic than even we were and as you know, we're optimistic folks, and is very consistent with the launch curves that we've seen with our other three approved therapies. So I will end this soliloquy with the statement I made in the earnings script itself, which is, you can say a lot of things about us but no one should dispute that there is no organization on this planet with more expertise. More ability and a better track record in launching Duchenne Therapy, serving this community, and making these launches a success. We're in really good shape right now, but thank you very much for your question, Tazeen. I appreciate it.

Darren: You can say a lot of things about us, but no one should should dispute that there is no organization.

Douglas S. Ingram: So I will end this soliloquy with the statement I made in the earnings script itself, which is, you can say a lot of things about us but no one should dispute that there is no organization on this planet.

Darren: On this planet.

Darren: With more expertise more ability and a better track record in launching Duchenne therapies, serving this community and making these launches as access we're in really good shape, but thank you very much for your questions I appreciate it.

Douglas Ingram: More ability and a better track record in launching Duchenne Therapy, serving this community, and making these launches a success. We're in really good shape right now, but thank you very much for your question, Tazeen. I appreciate it. Thank you. One moment for the next question. Then our next question will be coming from Anupam. Rama of J&P ULINAS. Hey guys, thanks for taking the questions.

Douglas S. Ingram: More ability and a better track record in launching Duchenne Therapy, serving this community, and making these launches a success. We're in really good shape right now, but thank you very much for your question, Tazeen. I appreciate it.

Operator: Thank you, one moment for the next question. And our next question will be coming from Anupam Rama of J.P. Your line is open. Hey guys, thanks for taking the questions.

Operator: Thank you, one moment for the next question. And our next question will be coming from Anupam Rama of J.P. Your line is open.

Douglas S. Ingram: Hey guys, thanks for taking the question, also it's JPM, thanks for providing the 2025 net product guidance and I think you said multiple times, addressing the prevalent population, kind of into the 2030-2031 time frame. Should we be thinking about more linear growth over that time or how should we be thinking about say like the time to peak scenario over the course of the [inaudible]? Yeah I mean we've given, thank you very much for your question Anupam, we've given obviously more granular, longer term guidance than the typical of us, but we wanted to really reflect accurately what we're seeing in this launch. I'm not going to get into a time where granularity on the long-term forecast other than to say peakier sales will ocurr in the back half of this, significantly in the back half of this decade. And as I said before in the script, we'll be treating the prevalent population through the entire decade, and we'll be building to engine in population in 2030, thank you though.

Anupam Rama: Hey guys, thanks for taking the question, also it's JPM, thanks for providing the 2025 net product guidance and I think you said multiple times, addressing the prevalent population, kind of into the 2030-2031 time frame. Should we be thinking about more linear growth over that time or how should we be thinking about say like a time to peak scenario over the course of the next--?

Douglas S. Ingram: Yeah I mean we've given, thank you very much for your question Anupam, we've given obviously more granular, longer term guidance than the typical of us, but we wanted to really reflect accurately what we're seeing in this launch. I'm not going to get into a time where granularity on the long-term forecast other than to say peakier sales will ocurr in the back half of this, significantly in the back half of this decade. And as I said before in the script, we'll be treating the prevalent population through the entire decade, and we'll be building to engine in population in 2030, thank you though.

Operator: Thank you. One moment for the next question. Then our next question will be coming from Anupam. Rama of GMP. Your line is open.

Speaker Change: Thank you one moment for the next question.

Speaker Change: And our next question will be coming from Amazon.

Speaker Change: Grandma of JMP Your line is open.

Speaker Change: Hey, guys. Thanks for.

Speaker Change: The question.

Speaker Change: Also at two P M.

Speaker Change: The.

Speaker Change: Thanks for providing that 2025 net product guidance and I think you've said multiple times addressing the prevalent prevalent population.

Speaker Change: Into the 2030 2031 timeframe.

Speaker Change: Is that should we be thinking about more linear growth over that time or how should we be thinking about say like a time to peak.

Speaker Change: Scenario.

Speaker Change: Over the course of the day.

Speaker Change: Yes, we've given obvious thank you very much for your question on upon we'd given obviously.

Speaker Change: More granular longer term guidance that is typical of us, but we wanted to really reflect accurately what we're seeing in this launch I'm not going to get into a ton more.

Speaker Change: Granularity on the long term forecast other than to say peak year sales will occur in the back half of this so giving me in the back half of this decade and as I said before in the script, we will be treating the prevalent population through the.

Speaker Change: The entire decade and will be moving to internet population in the 2000 Thirty's.

Bill: Thank you Bill.

Operator: Thank you. And our next question will be coming from Gena Wang of Barclays.

Gena Wang: Thank you, I will also ask one more question regarding the revenue. So, did I hear you correctly? 3Q, you still expect modest growth, even, you know, despite you will take 3 to 5 months for [inaudible] to infusion? So that was the first question just to confirm, and then the guidance, sorry I will ask again in guidance $2.9 to $3.1 billion, when we look at that's in line first order analytics of a $3 billion 2025 consensus, and they do have the numbers. The sell-side consensus number for ELEVIDYS for 2025 is $2 billion, so do you think that number is in line with your assumptions? And what are the early launch data to support your assumption?.

Operator: Thank you, and our next question will be coming from Gena Wang of Barclays. Your line is open. Thank you. I will also ask one more question regarding revenue. So, did I hear you correctly? For 3Q, you still expect modest growth, even, you know, despite the fact that you will take 3 to 5.

Operator: Thank you, and our next question will be coming from Gena Wang of Barclays. Your line is open.

Speaker Change: Thank you and our next question will be coming from Gena Wang of Barclays.

Speaker Change: Your line is open.

Gena Wang: Thank you I'll also ask a one more question regarding the revenue. So Doug did I hear you correctly <unk> you still expect modest growth. Even despite you will take three to five months for stuff walk to infusion. So that was the first question just to confirm and then the guidance sorry, I'll ask it again.

Speaker Change: <unk> guidance $2 90 to $3 1 billion.

Speaker Change: But when we look at that is inline with first order analytics over 3 billion in 2025 consensus and they do have numbers.

Speaker Change: Slide consensus number for <unk> for $2025 2 billion.

Speaker Change: Do you think that that number is in line with your assumption of what other early launch data to support your assumption.

Douglas S. Ingram: Yeah, thank you very much for your questions, Gina. So first on Q3, relative to what I would characterize as the explosive growth that we're going to have as the start forms turn into infusions. In the third quarter, I've said we'll have modest growth, but that's, you know, still 30% quarter, over the immediate preceding quarter, so it'll be very nice, robust growth. But remember, we just got approved at the end of Q2, and then it does take about three to five months, so it'll be really kind of back-end loaded in the quarter, but we'll see, broadly speaking, about 30% growth, and we'll double it in Q4. And then, in 2025, I think we are largely in line with where the external world is, although that wasn't our goal, I think it's a happy accident that we happen to be lined up. And to the question of, you know, sort of what are we seeing? It is early days, so what are we seeing in these early days that gives us confidence? There's a lot, just so we're very clear. Hi all, this is Will on behalf of Joe. Thanks for taking our question and congratulations on the progress this quarter.

Douglas S. Ingram: Yeah, thank you very much for your questions, Gina. So first on Q3, relative to what I would characterize as the explosive growth that we're going to have as the start forms turn into infusions. In the third quarter, I've said we'll have modest growth, but that's, you know, still 30% quarter, over the immediate preceding quarter, so it'll be very nice, robust growth. But remember, we just got approved at the end of Q2, and then it does take about three to five months, so it'll be really kind of back-end loaded in the quarter, but we'll see, broadly speaking, about 30% growth, and we'll double it in Q4. And then, in 2025, I think we are largely in line with where the external world is, although that wasn't our goal, I think it's a happy accident that we happen to be lined up. And to the question of, you know, sort of what are we seeing? It is early days, so what are we seeing in these early days that gives us confidence? There's a lot, just so we're very clear, again, no one has more experience in launching Duchenne therapies than Sarepta

Douglas S. Ingram: Yeah, thank you very much for your questions, Gena. So first on Q3, relative to what I would characterize as the explosive growth that we're going to have as the start forms turn into infusions. In the third quarter, I've said we'll have modest growth, but that's, you know, still 30% quarter, over the immediate preceding quarter, so it'll be very nice, robust growth. But remember, we just got approved at the end of Q2, and then it does take about three to five months, so it'll be really kind of back-end loaded in the quarter, but we'll see, broadly speaking, about 30% growth, and we'll double it in Q4.

Speaker Change: Yes. Thank you very much for your questions. So first of all in Q3 relative to what I would characterize as the explosive growth that we're going to have.

Speaker Change: As we start forms turn into infusions in the third quarter I have said, we will have modest growth, but thats still 30% quarter over the immediate preceding quarter. So it will be very nice robust growth, but remember we just got approved at the end of Q2 and then it does take about three to five months. So.

Douglas Ingram: So it'll be really kind of back-end loaded in the quarter. But we'll see, broadly speaking, about 30% growth, and we'll double it in Q4. And then, in 2025, I think we are largely in line with where the external world is, although that wasn't our goal. I think it's a happy accident that we happen to be lined up.

Speaker Change: That will be really kind of backend loaded in the quarter, but we will see broadly speaking about 30% growth and we will double it in Q4.

Speaker Change: And then.

Speaker Change: <unk> 2025, I think we are largely in line with where the external world is although that wasn't our goal I think its a happy accident that we happen to be.

Speaker Change: Lined up and to the question of sort of what are we seeing it is early days. So what are we seeing early days that gives us confidence there is a lot just so we're very clear.

Sohrab: Again, no one has more experience in launching Duchenne therapies, then sohrab. This is our first therapy and some weird ways. It's our fifth therapy, because we launched this with very narrow label evidenced in that we're launching with a broader label. So couple of things, we know extraordinarily well.

Speaker Change: We know it would be down to the state level. So we're very confident on the abbvie here.

Douglas S. Ingram: And then, in 2025, I think we are largely in line with where the external world is, although that wasn't our goal, I think it's a happy accident that we happen to be lined up. And to the question of, you know, sort of what are we seeing? It is early days, so what are we seeing in these early days that gives us confidence? There's a lot, just so we're very clear, again, no one has more experience in launching Duchenne therapies than Sarepta, this is our fourth therapy, in some weird ways this is our fifth therapy because we launched this with a very narrow label of ELEVIDYS and now we're launching with a broader label. So a couple of things, we know [inaudible] extraordinarily well, we know [inaudible] down to the state level, so we're very confident on the [inaudible] here. We have a lot of confidence around the launch curve itself, what we're envisioning from the launch curve right now is exactly what we envision we would see in all of our modeling and it is consistent with, again, the launch of EXONDYS, AMONDYS and VYONDYS. And we have a lot of granular data, just remember we have downed the patient-level data on star forms and the like, we know all on the sites, we just have an enormous amount of visibility. It's still early days, so to Ian's very good point, we will update our views on things as we track to the end of the year into early next year. But you know obviously early days we felt a pretty significant amount of confidence to provide people with this overall shape of the launch and discuss 2025, which for us is a bit unusual, we wouldn't provide this kind of level of information this early other than we wanted to really map out what we're seeing right now. We feel like we're being transparent with folks, so we feel very good about this, long-term. Thank you, our next question today will be coming from Joseph Schwartz of Leerink, your line is open.

Speaker Change: We have a lot of confidence around the launch curves itself and what we're envisioning from the launch curve right now is exactly what we envisioned we would see in all of our modeling and it is consistent with again the launch of axon to show modest and by on this and we have a lot of granular data you should remember we have down to patient.

Douglas S. Ingram: So a couple of things, we know [inaudible] extraordinarily well, we know [inaudible] down to the state level, so we're very confident on the [inaudible] here. We have a lot of confidence around the launch curve itself, what we're envisioning from the launch curve right now is exactly what we envision we would see in all of our modeling and it is consistent with, again, the launch of EXONDYS, AMONDYS and VYONDYS. And we have a lot of granular data, just remember we have downed the patient-level data on star forms and the like, we know all on the sites, we just have an enormous amount of visibility. It's still early days, so to Ian's very good point, we will update our views on things as we track to the end of the year into early next year. But you know obviously early days we felt a pretty significant amount of confidence to provide people with this overall shape of the launch and discuss 2025, which for us is a bit unusual, we wouldn't provide this kind of level of information this early other than we wanted to really map out what we're seeing right now. We feel like we're being transparent with folks, so we feel very good about this, long-term. Thank you, our next question today will be coming from Joseph Schwartz of Leerink, your line is open.

Speaker Change: Level data on start forms and the like we know all of the sites. We we just have an enormous amount of visibility its still early days. So it's very good point, we will update our views on things as we track to the end of the year into early next year, but obviously early.

Douglas S. Ingram: It's still early days, so to Ian's very good point, we will update our views on things as we track to the end of the year into early next year. But you know obviously early days we felt a pretty significant amount of confidence to provide people with this overall shape of the launch and discuss 2025, which for us is a bit unusual, we wouldn't provide this kind of level of information this early other than we wanted to really map out what we're seeing right now. We feel like we're being transparent with folks, so we feel very good about this, long-term.

Speaker Change: Days, we felt a pretty significant amount of confidence to provide people with this overall shape of the launch and discuss 2025, which for US is a bit unusual we wouldn't provide this kind of level of information. This early other than wanting to really map out what we're seeing right now and feel like we're being.

Operator: Thank you, our next question today will be coming from Joseph Schwartz of Leerink, your line is open.

Joseph Patrick Schwartz: Hi all, this is Will on behalf of Joe, thanks for taking our question and congrats on the progress this quarter, so one from us. For many years we've seen the company successfully execute on a mission to help boys with DMD, however this is something that may not be fully appreciated by some in the community. As it appears that the company will continue to invest in next generation therapies and support these patients, why do you think there is this disconnect with some of the community and what are you doing to actively address it, especially with competition, or potential competition on the horizon? Thank you.

Speaker Change: Transparent with folks so we feel very good about this launch right now.

Speaker Change: Thank you.

Speaker Change: Next question today will be coming from Joseph Schwartz of Leerink. Your line is open.

Douglas Ingram: And to the question of, you know, sort of what are we seeing? It is early days. So what are we seeing in these early days that gives us confidence? There's a lot, just so we're very clear. Hi all, this is Will on behalf of Joe. Thanks for taking our question and congratulations on the progress this quarter.

unknown: Hi all, this is-

Joseph Schwartz: Hi, all this is will on for Joe. Thanks for taking my question and congrats on the progress this quarter.

Speaker Change: I'm from us for many years, we've seen the company successfully execute on our mission to help with the Dnb. However, this is something that may not be fully appreciated by some of the community as it appears that the company will continue to invest in next generation therapies and support. These patients why do you think there is a disconnect with some of the community and what are you doing to actively address it especially.

Speaker Change: With competition or potential competition on the horizon. Thank you.

Douglas S. Ingram: Well, I think anybody who's been watching us carefully over the last seven to eight years will know that we have been wholly committed to bringing a better life to the Duchenne community. And we've, you know, at the risk of being a bit modest, been pretty brilliant in our ability to do that. Four therapies later, all very successful launches, and I believe very transformative, meaningful, district, and restorative therapies, which we now can reach, you know, well over 80% of the Duchenne population. And we're working on actually making it even greater, we're successful with our attempts to knock down pre-existing neutralizing antibodies, and we have a lot of conviction that we will be. We're already dosing in one of those two studies; the other one will come very soon.

Speaker Change: I think anybody who's been watching those carefully over the last seven to eight years will know that we have.

Then wholly committed to bringing a better aligned to the Duchenne community and we.

Speaker Change: Yeah, the risk of being a bit of a modest we've been pretty brilliant and our ability to do that it's worth therapies. Later all of very successful launches and I believe very transformative meaningful district been restored therapies, which we now can.

Douglas Ingram: Four therapies later, all very successful launches, and I believe very transformative, meaningful, district, and restorative therapies, which we now can reach, you know, well over 80% of the Duchenne population, and we're working on actually making it even greater. We have been successful with our attempts to knock down pre-existing neutralizing antibodies, and we have a lot of conviction that we will. We're already dosing in one of those two studies; the other one will come very soon.

Can reach well over 80% of the Duchenne population and we're working on actually making it even greater if were successful with.

Douglas S. Ingram: And we're working on actually making it even greater, we're successful with our attempts to knock down pre-existing neutralizing antibodies, and we have a lot of conviction that we will be. We're already dosing in one of those two studies, the other one will come very soon, you know, we'll be getting to much higher, even 80% of the Duchenne community. And the good news is that I think that those who are informed and have been watching us get this. I think to be honest, the great bulk of the patient community fully understands and is cheering us along. I can tell you I, we all stay very close with the community, and the community understands that, and that's why, in addition to cheering us along, people have been watching the data and are very excited and hopeful they can get on the therapy as soon as possible. Same answer with the physician community, I would say, you know, to those who are curious, just talk to physicians who have actually dosed this therapy across the spectrum of Duchenne and ask them what their experiences have been, and I think you'll hear that they're very excited about that. So I think, you know, the right-minded people are getting it, and I think that, you know, there's a lot of enthusiasm for this therapy as well as there should be. And it creates for us an enormous responsibility to get this therapy out to these kids.

Speaker Change: Our attempts to knockdown.

Pre existing neutralizing antibody and we have a lot of conviction that we will be we're already dosing.

Joseph Schwartz: One of those two studies and the other one will come very soon we will be getting to much higher even than 80% of the duchenne community and the good news is that I think that those who are informed and have been watching I forget this.

Douglas Ingram: You know, we'll be getting to much higher, even 80% of the Duchenne community. And the good news is that I think that those who are informed and have been watching us get this. I think to be honest, the great bulk of the patient community fully understands and is cheering us along. I can tell you that I, we all stay very close with the community, and the community understands that. And that's why, in addition to cheering us along, people have been watching the data and are very excited and hopeful they can get on the therapy as soon as possible. Same answer with the physician community. I would say, you know, to those who are curious, just talk to physicians who have actually dosed this therapy across the spectrum of Duchenne.

I think to be honest.

Great bulk of the patient community fully understands and is cheering us along I can tell you is we.

Douglas S. Ingram: I can tell you I, we all stay very close with the community, and the community understands that, and that's why, in addition to cheering us along, people have been watching the data and are very excited and hopeful they can get on the therapy as soon as possible. Same answer with the physician community, I would say, you know, to those who are curious, just talk to physicians who have actually dosed this therapy across the spectrum of Duchenne and ask them what their experiences have been, and I think you'll hear that they're very excited about that. So I think, you know, the right-minded people are getting it, and I think that, you know, there's a lot of enthusiasm for this therapy as well as there should be. And it creates for us an enormous responsibility to get this therapy out to these kids.

I'll stay very close with the community and the community understands that and Thats why.

Speaker Change: In addition to cheering us along people have been watching the data and are very.

I'm excited and.

Hopefully they can get on the therapy as soon as possible same answer with the physician community.

I would say that those who are curious just talked to physicians.

Who have actually dose this therapy across the spectrum of Duchenne and asked them what their experiences have been I think you'll hear that they are very excited about that so.

Douglas S. Ingram: So I think, you know, the right-minded people are getting it, and I think that, you know, there's a lot of enthusiasm for this therapy as well as there should be. And it creates for us an enormous responsibility to get this therapy out to these kids, and this team is doing, you know, everything they can to do that, and the great news is that there's no team better than this team to do it. So I don't want to give Dallan and his team too big a head, but the fact is that this team is brilliant at serving this community, and it will not change, with ELEVIDYS we're off to a great start.

Douglas Ingram: Ask them what their experiences have been, and I think you'll hear that they're very excited about that. So I think, you know, the right-minded people are getting it. And I think that, you know, there's a lot of enthusiasm for this therapy as well as there should be. And it creates for us an enormous responsibility to get this therapy out to these kids.

I think I think the right minded people are getting and then I think that.

Theres a lot of enthusiasm for this therapy as well there should be.

Douglas Ingram: And this team is doing, you know, everything they can to do that. And the great news is that there's no team better than this team to do it. So I don't want to give Dallin and his team too much credit. But the fact is that this team is brilliant at serving this community, and it will not change with the Levitas.

Speaker Change: And it creates for US an enormous responsibility to get this therapy out to these kids and his team is doing.

Everything they can to do that and the great News is that there is no team better than this team to do it so.

I don't want to give gallon and his team to big ahead.

But the fact is that this team is brilliant at serving this community and it will not change with the <unk>, we're off to a great start.

Operator: We're off to a great start. Thank you. Our next question will be coming from Brian Abrahams of RBC Capital. Hey, good afternoon, guys. Thanks for taking my question and I really appreciate all the granularity here.

We're off to a great start.

Thank you. Our next question will be coming from Brian Abrahams of RBC Capital. Hey, good afternoon, guys. Thanks for taking my question and I really appreciate all the granularity here.

Operator: Thank you, our next question will be coming from Brian Abrahams of RBC Capital.

Brian Abrahams: Hey, good afternoon, guys, thanks for taking my question and I really appreciate all the granularity here on the launch dynamics. You did mentioned that there was some wait times at centers as they navigate demand so it sound like capacity, you know, is somewhat of a gating factor here, can you give us a sense of what some of the limitations here are at the center level? Any additional infrastructure or proceses that these sites need to put into place to administer ELEVIDYS and monitor patients, and where do you think that could expect over time? What's embedded in your guidance in terms of capacity expansion? Thanks.

Operator: Thank you. Our next question will be from Brian Abrahams of RBC Capital.

Thank you. Our next question will be coming from Brian Abrahams.

RBC capitals.

Hey, good afternoon, guys. Thanks for taking my question and really appreciate.

All the granularity here on the launch dynamics you did mention that there were some wait times at centers as they navigate demand so it sounds like.

Capacity is somewhat of a gating factor here can you give us a sense of what some of the limitations here are at the center level any additional infrastructure our processes at the sites need to put into place to administer <unk> and monitor patients and where you think that could expand over time, what's embedded in your guidance in terms of capacity.

Thanks.

Douglas S. Ingram: Okay, I'm going to turn this question over to Dallan, but let me say a few things in advance. First, you know, we don't have a fundamental capacity issue, I want to be very clear about that, we have about 75 sites in the US, 75% of which have already dosed patients. The number one, probably the number one metric to the success of therapy is the experience with it and how well it's working, but with that said, the experience with the actual infusion is really important. So having such a significant number of sites, I believe more than any other gene therapy I've ever launched with, and having 75% of them already having those patients is significant. And I think one of the things we're seeing in the early days is there are definitely some sites that, in the absence of more information, are just worried about the onslaught and trying to manage. The amount of demand that's coming in, because there are, you know, some 12,000 or so patients in the United States with Duchenne Muscular Dystrophy. You know 80% or more of them can be treated with this therapy, they've got to prioritize and think that issue through, and they're pondering it. But with that, Dallan, if you want to provide more granularity than I have on this topic [inaudible].

Okay I'm going to turn this question over to down, but let me say a few things in advance first we don't have a fundamental capacity issue.

Douglas Ingram: We have about 75 sites in the U.S., 75% of which have already dosed patients. The number one, probably the number one metric to measure the success of therapy is the experience with it and how well it's working. But with that said, the experience with the actual infusion is really important.

Wanted to be very clear about that we have about 75 sites in the U S, 75% of which have already dosed patients.

Number one probably the number one metric to their successes therapy.

Is the experience with it and how well it's working but it would that would that said then the experience with the actual infusions is really important so.

Douglas Ingram: So having such a significant number of sites, I believe more than any other gene therapy I've ever launched with, and having 75 percent of them already have those patients is significant. And I think one of the things we're seeing in the early days is there are definitely some sites that, in the absence of more information, are just worried about the onslaught and trying to manage. The amount of demand that's coming in, because there are, you know, some 12,000 or so patients in the United States with Duchenne Muscular Dystrophy, and 80% or more of them can be treated with this therapy. They've got to prioritize and think that issue through, and they're pondering it. But with that, Dallan, if you want to provide more granularity than I have on this topic, yeah, thanks, Doug. And

So having such a significant number of sites, I believe more than any other gene therapy I've ever launched with, and having 75 percent of them already have those patients is significant. And I think one of the things we're seeing in the early days is there are definitely some sites that, in the absence of more information, are just worried about the onslaught and trying to manage. The amount of demand that's coming in, because there are, you know, some 12,000 or so patients in the United States with Duchenne Muscular Dystrophy, and 80% or more of them can be treated with this therapy. They've got to prioritize and think that issue through, and they're pondering it. But with that, Dallan, if you want to provide more granularity than I have on this topic,

Having such a significant number of sites I believe more than any other gene therapy has ever launched with and having 75% of them already having those patients is significant and I think one of the things you're seeing early days.

Douglas S. Ingram: And I think one of the things we're seeing in the early days is there are definitely some sites that, in the absence of more information, are just worried about the onslaught and trying to manage. The amount of demand that's coming in, because there are, you know, some 12,000 or so patients in the United States with Duchenne Muscular Dystrophy. You know 80% or more of them can be treated with this therapy, they've got to prioritize and think that issue through, and they're pondering it. But with that, Dallan, if you want to provide more granularity than I have on this topic, please.

There are definitely some sites that in the absence of more information or just worried about the onslaught and trying to manage that.

The amount of demand that's coming in because there are some 12000 or so patients in the United States with Duchenne muscular dystrophy.

80% or more of them can be treated with this therapy, they've got prioritize that issue through partnering but with that down if you want to provide more granularity than I have on this topic. Please yes.

Dallan Murray: Yeah, thanks, Doug, and Brian, thanks for the question, and as Doug said, you know, we've got more than enough capacity, we've got more than enough capacity today to accommodate even the peak years of sales, Brian, but really, what is happening out there is, as I said in my remarks that essentially overnight, the vast majority of patients became eligible. So each individual center just has to work through all of their patients, they've got all of their patients reaching out and figuring out how they're going to approach, who they're going to prioritize, who they're going to dose first. And, you know, this is to be expected, and everything is really exactly as we have forecast, anticipated, and expected.

Dallan Murray: Yeah, thanks, Doug. And Brian, thanks for the question.

Yes, Thanks, Doug.

Brian Thanks for the question.

Dallan Murray: And as Doug said, you know, we've got more than enough capacity; we've got more than enough capacity today to accommodate even the peak years of sales, Brian, but really, what is happening out there is, as I said in my remarks, Essentially overnight, the vast majority of patients became eligible. So each individual center just has to work through all of their patients. They've got all of their patients reaching out and figuring out how they're going to approach who they're going to prioritize, who they're going to dose first. And, you know, this is to be expected. And everything is really exactly as we have forecast, anticipated, and expected.

And as Doug said.

We've got more than enough capacity, we've got more than enough capacity today to accommodate even the peak years.

Of sales, Brian, but really what what is happening out there is as I said.

In my remarks that essentially overnight the vast majority of patients became eligible so each individual center. It just have to work through all of their patients they've got all of their patients reaching out and figure out how they're going to approach, who theyre going to prioritize that theyre going to dose first and.

Dallan Murray: And, you know, this is to be expected, and everything is really exactly as we have forecast, anticipated, and expected, and we're doing everything we can to support these centers. What makes it, you know, what should provide a lot of confidence is these are centers that are at the forefront of precision genetic medicine. They were the ones that figured out SPINRAZA, they figured out ZOLGENSMA ,they figured out our previous narrow label, and they're just in the process of figuring this out. And these early stages are just an adjustment period as they ramp up their ability to serve the whole patient population.

This is this is to be expected.

And everything is really exactly as we have forecast anticipated and expected and we're doing everything we can to support these centers what makes it.

Dallan Murray: And we're doing everything we can to support these centers. What makes it, you know, what should provide a lot of confidence is that these are centers that are at the forefront of precision genetic medicine. They were the ones that figured out Spinraza and Zolgensma. They figured out our previous narrow label, and they're just in the process of figuring this out. And these early stages are just an adjustment period as they ramp up their ability to serve the whole patient population.

What should provide a lot of confidence as these are the centers that are at the forefront of precision genetic medicine. They were the ones that figured out spin rather they figured out such as well.

They figure it out.

Our previous narrow label and they are just in the process of figuring this out and this are these early stages or just an adjustment period as they as they ramp up their ability to serve the whole patient population.

Yeah.

Okay.

Operator: Thank you. Our next question today will be coming from Salveen Richter of Goldman Sachs. Your line is open.

Salveen Richter: Good afternoon, thanks for taking my question, just going back here to this guidance. You know, it seems like you have the infusion centers but there's some kind of bottleneck or lever using with regard to how you're determining 2025 guidance. so is the gating factor here, as you've given the demand is it the number of patients that can be treated in an infusion center over a time period or is it the manufacturing supply in the context of the demand. And then help us understand how you're thinking about cannibalization of the PMO side of the business in your '25 guidance. Thank you.

Operator: Thank you. Our next question today will be coming from Salveen Richter, of Goldman Sachs. Your line is open. Good afternoon. Thanks for taking my question. Just going back here to this guidance. You know, it seems like you have the Thank you.

Operator: Thank you. Our next question today will be coming from Salveen Richter, of Goldman Sachs. Your line is open.

Thank you. Our next question today will be coming from solving victors.

Of Goldman Sachs. Your line is open.

Good afternoon. Thanks for taking my question just going back here to this guidance.

It seems like you have the infusion centers, but there is some kind of bottleneck or lever you're using with regard to kind of how youre determining 2025 guidance. So.

Is is.

Is the gating factor here as you've given the demand is there.

The number of patients that can be treated in an infusion center at a over a time period or is it manufacturing supply in the context of the demand and then help us understand.

How you're thinking about cannibalization of the PMO side of the business in your 'twenty guidance. Thank you.

Douglas S. Ingram: So, again, I would say that, first of all, thank you for your question, Salveen, I would say I think it would be a mischaracterization to imply that there is some significant bottleneck. The shape of the curve, you know, is related to a number of things, it's not related to demand; patient demand, physician demand are going to be very, very significant. But then there's process, the process of getting through the infusions, payer interaction, and the like, all defining the shape of the revenue curve over time, and we're very pleased with where we are. And we don't have a problem to solve, we will if all goes well, and it is going exactly that well, we're going to do $2.9 billion to $3.1 billion dollars next year, which means a very significant number of patients are going to significantly benefit from our therapies. And I would remind everyone that this is years away from peak year sales, and we'll be treating the prevalent population over the entire course of this decade into the early 2030s.

So again I would say that first of all thank you for your questions I'll being I would say I think it would be a mischaracterization to envision that there is some significant bottleneck.

<unk>.

The curve.

As related to a number of things it is not related to demand patient demand physician demand is going to be.

Very very significant but then theres process the process of getting through the infusions payer interaction and alike. All defines the shape of the revenue.

Curve over time, and we're very pleased with where we are in there. We don't have a problem to solve we will if all goes well and it is going exactly that well means that we're going to do $2 9 billion to $3 $1 billion next year, which means a very significant number of patients are going to cigna.

Douglas Ingram: And we're very pleased with where we are. And we don't have a problem to solve. If all goes well, and it is going exactly that well, we're going to do $2.9 billion to $3.1 billion next year, which means a very significant number of patients are going to significantly benefit from our therapies. And I would remind everyone that this is years away from peak year sales, and we'll be treating the prevalent population over the entire course of this decade into the early 2030s.

Douglas S. Ingram: And we don't have a problem to solve, we will if all goes well, and it is going exactly that well, we're going to do $2.9 billion to $3.1 billion dollars next year, which means a very significant number of patients are going to significantly benefit from our therapies. And I would remind everyone that this is years away from peak year sales, and we'll be treating the prevalent population over the entire course of this decade into the early 2030s. And then on cannibalization, there will be cannibalization if for no other reason that, you know, there is a kind of competition for start forms, for patients as well. But this year and into next year, we see that cannibalization is quite modest. Thank you again for your question.

Inefficiently benefit from our therapies and I would remind everyone that is years away from peak year sales and we'll be treating the prevalent population over the entire course of this decade into the early 2000, <unk> and then on cannibalization there will be cannibalization.

Douglas Ingram: And then on cannibalization, there will be cannibalization if for no other reason that, you know, there is a kind of competition for start forms, for patients as well. But this year and into next year, we see that cannibalization is quite modest. Thank you again for your question.

Douglas S. Ingram: And then on cannibalization, there will be cannibalization if for no other reason that, you know, there is a kind of competition for start forms, for patients as well. But this year and into next year, we see that cannibalization is quite modest. Thank you again for your question.

If for no. Other reason that there is a kind of a competition for start forms from patients as well, but in this year and into next year.

Douglas S. Ingram: But this year and into next year, we see that cannibalization is quite modest. Thank you again for your question.

See that cannibalization is quite modest but thank you again for your questions.

Operator: Thank you, and our next question will be coming from Ritu Baral of TD Cowen, your line is open.

Thank you.

And our next question will be coming from Virtu morale of PD Cowen Your line is open.

Ritu Baral: Good afternoon, guys, thanks for taking the question. Doug, you've given different sort of aspects of guidance going forward, including the fact that you guys think that peak is going to be towards the end of the decade. You had previously given us a rough figure of about 4 billion peak for ELEVIDYS, and this was a few years ago, and the centers were all not all up and running and stuff like that. But do you still see that as the general peak in your internal assumptions? And I wanted to also ask about the sort of levers between the top of your guidance and the bottom of your guidance, it's a really, really tight guidance, so I'm assuming you have start forms that will track all the way out through some element of 2025. Can you talk to the volume of start forms and whether, what the levers are for the top and the bottom and how far out those start forms will map your revenue, in handstand forms?

Good afternoon.

Guys. Thanks for thanks for taking the question.

Doug.

Given.

Different sort of aspects of guidance going forward, including the fact that you guys think that it can be towards the end of the decade.

Previously given us a rough figure of about 4 billion peak for <unk>, evidenced with a few years ago in centers, where.

Not all up and running and stuff like that but do you still see that as the general peak.

In your internal assumptions and I wanted to also ask about the.

Ritu Baral: And I wanted to also ask about the sort of levers between the top of your guidance and the bottom of your guidance, it's a really, really tight guidance, so I'm assuming you have start forms that will track all the way out through some element of 2025. Can you talk to the volume of start forms and whether, what the levers are for the top and the bottom and how far out those start forms will map your revenue, in handstand forms?

Douglas Ingram: And I wanted to also ask about the sort of levers between the top of your guidance and the bottom of your guidance. It's a really, really tight guide. So I'm assuming you have start forms that will track all the way out through some element of 2025. Can you talk about the volume of start forms and whether or not the levers are for the top and the bottom and how far out those start forms are? will map your revenue, in handstand form.

Sort of levers between the top of your guidance on the bottom of your guidance, it's really really tight guidance. So I must have start for them that will track all the way out through some element of 2025 can you talk to the volume of start forms and whether.

What the levers are for the top and the bottom and how far out those start forms.

Well Matthew or revenues.

The enhanced arguments.

Douglas S. Ingram: So, a couple of thoughts, on your first question, yes, some years ago, I provided long-term guidance, there is, we have no basis for amending that guidance at all, we feel very comfortable about the prior guidance that we provided, so there's no need to update that, it does appear to still be very accurate. And on the number one thing, we have a lot of starter forms, we don't have all the starter forms yet, of course, that's not the way it works, but remember, we have, more than anything else, great experience. We know what level of start forms correlate to what, we know what the launch curve should look like, we have a ton of algorithmic ways to look at this launch and map our expectations versus the current launch. And the short answer is, based on all of our experience and knowledge in algos, we are doing very well, as I've said, the early signals exceed our optimistic view on this launch.

Douglas Ingram: So a couple of thoughts on your first question. Yes, some years ago, I provided long-term guidance. But we have no basis for amending that guidance at all.

So couple of thoughts on your first question, yes, some years ago I provided long term guidance.

There is we have no basis for amending that guidance at all we feel very comfortable about the prior guidance that we provided.

Douglas Ingram: We feel very comfortable about the prior guidance that we provided. So there's no need to update that. It does appear to still be very accurate. And on the number one thing, we have a lot of starter forms. We don't have all the starter forms yet, of course. That's not the way it works.

There is no need to update that it's it is does appear still to be very accurate.

And on the number one thing we have a lot of the Starplex you don't have all the start forms yet of course, that's not the way it works, but remember we have more than anything else great experience like we know what level of start forms correlate to what we know what the launch curve should look like we are we have a ton of algorithmic ways to look at this launch and.

Douglas Ingram: But remember, we have, more than anything else, great experience. We know what level of start forms correlate to what. We know what the launch curve should look like. We have a ton of algorithmic ways to look at this launch and map our expectations versus the current launch. And the short answer is, based on all of our experience and knowledge in algos, we are doing very well. As I've said, the early signals exceed our optimistic view of this launch.

Douglas S. Ingram: We know what level of start forms correlate to what, we know what the launch curve should look like, we have a ton of algorithmic ways to look at this launch and map our expectations versus the current launch. And the short answer is, based on all of our experience and knowledge in algos, we are doing very well, as I've said, the early signals exceed our optimistic view on this launch. I'll just say again, we are comfortable with the guidance that we're providing right now. And to your point, we are getting to be a relatively big company, so while it's tight, I mean, it's still $100 million dollars on each side, but to your point, it's getting to be pretty tight. But we feel very comfortable about providing that level of guidance. Thank you. And our next question will be coming from Mike Ulz of Morgan Stanley. Your line is open.

Matt.

Our expectations versus the current launch and the short answer is based on all of our experience and knowledge in Alagoas.

We are doing very well as I've said the early signals exceed our optimistic view on this launch so.

Douglas Ingram: I'll just say again, we are comfortable with the guidance that we're providing right now. And to your point, we are getting to be a relatively big company. So while it's tight, I mean, it's still $100 million on each side. But to your point, it's getting to be pretty tight. But we feel very comfortable about providing that level of guidance. Thank you. And our next question will be coming from Mike Ulz of Morgan Stanley. Your line is open.

I'll just say again, we are comfortable with.

The guidance that we're providing right now and to your point it's.

We're getting to be a relatively big company so while.

It's tight I mean, there's still $100 million on each side, but if you are to your point, it's getting to be pretty tight, but we feel very comfortable about providing that level of guidance.

Douglas S. Ingram: And to your point, we are getting to be a relatively big company, so while it's tight, I mean, it's still $100 million dollars on each side, but to your point, it's getting to be pretty tight. But we feel very comfortable about providing that level of guidance.

Thank you.

And our next question will come from Mike <unk> of Morgan Stanley. Your line is open.

Hey, guys. Thanks for taking the question, maybe just a quick follow up on the PMO cannibalization.

Youre expecting modest cannibalization in 2025, but just curious how youre thinking about that longer term should we still be thinking.

Operator: Thank you, and our next question will be coming from Mike Ulz of Morgan Stanley. Your line is open.

Some modest impact.

Or should that ramp up as sales start to ramp up.

Douglas Ingram: Hey guys thanks for taking my question, maybe just a few follow up on the PMO cannibalization. You're expecting modest cannibalization in 2025, I'm just curious how you're thinking about that longer term, should we still be thinking some modest impact or should that ramp up as sales start to ramp up? Well, it's a very, first of all, thank you for your question, Mike. It's an interesting issue. We've always modeled some robust, eventually some robust cannibalization of the PMO franchise. What we've seen so far has been fairly, and very modest. And what we're modeling into next year is modest, you know, might be more significant in years beyond that. But you know, there's also some reason to believe that our views have been a little bit conservative on, you know, how much cannibalization there will be, or I should say, aggressive on the amount of cannibalization.

Mike Ulz: Hey guys thanks for taking my question, maybe just a few follow up on the PMO cannibalization. You're expecting modest cannibalization in 2025, I'm just curious how you're thinking about that longer term, should we still be thinking some modest impact or should that ramp up as sales start to ramp up?

Thanks.

So first of all thank you for your question, Mike. It's an interesting issue we've always modeled some robust eventually some robust cannibalization of the PMO franchise, what we've seen so far has been thoroughly.

Douglas S. Ingram: Well, it's a very, first of all, thank you for your question, Mike. It's an interesting issue, we've always modeled some robust, eventually some robust cannibalization of the PMO franchise. What we've seen so far has been barely and very modest, and what we're modeling the next year is modest, you know, might be more significant in years beyond that. But you know, there's also some reason to believe that our views have been a little bit conservative on, you know, how much cannibalization there will be, or I should say, aggressive on the amount of cannibalization. We may have been a bit, you know, our own internal models may be a bit aggressive on the amount or cannibalization. But what I would say right now is that our models suggest that the cannibalization is very modest now, it'll remain pretty modest next year, and it might be more significant in the years after that. And our next question will be from Brian Skorney of Beard. Your line is open. Hey, good afternoon, guys. Yeah, my question is on the cannibalization of PMO as well. Trying to contextualize when you say fairly modest cannibalization, why isn't it just a straight-up equation based on the

Very modest and what we're modeling into next year is modest.

It might be more significant in years beyond that but there's also some reason to believe that our views have been a little bit conservative on.

How much cannibalization there will be I should say.

Douglas S. Ingram: We may have been a bit, you know, our own internal models may be a bit aggressive on the amount or cannibalization. But what I would say right now is that our models suggest that the cannibalization is very modest now, it'll remain pretty modest next year, and it might be more significant in the years after that.

Aggressive on the amount of cannibalization, we may have been a bit.

Our own internal models, maybe a bit aggressive on the amount of cannibalization, but what I would say right. Now is our model suggests that the cannibalization is very modest now it'll be it remains pretty modest next year and it might be more significant in the years after that.

Douglas Ingram: We may have been a bit, you know, our own internal models may be a bit aggressive on the amount. But what I would say right now is that our models suggest that the cannibalization is very modest now, it'll remain pretty modest next year, and it might be more significant in the years after that. And our next question will be from Brian Skorney of Beard. Your line is open. Hey, good afternoon, guys. Yeah, my question is on the cannibalization of PMO as well. Trying to contextualize when you say fairly modest cannibalization, why isn't it just a straight-up equation based on the

Douglas S. Ingram: Thank you, and our next question will be from Brian Skorney of Baird. Your line is open. Hey, good afternoon, guys. Yeah, my question is on the cannibalization of PMO as well. Trying to contextualize when you say fairly modest cannibalization, why isn't it just a straight-up equation based on the

Operator: Thank you, and our next question will be from Brian Skorney of Baird. Your line is open.

Brian Peter Skorney: Hey, good afternoon, guys, my question is on the cannibalization of PMO as well. I'm just trying to contextualize when you say fairly modest cannibalization, why isn't it just a straight-up equation based on the prevalence of the dual exon amenable patients and then penetration that you have into PMO. I would sort of calculate the, somewhere between 8 and 10 ELEVIDYS stations should result in one PMO patient cannibalization, assuming that person would have to be forced by their insurance company to choose to take ELEVIDYS and come off the PMO. Is that not the case? Are you seeing patients who are getting commercially treated with ELEVIDYS and maintaining their PMO? Post ELEVIDYS.

Operator: Thank you. And our next question will be coming from Brian Skorney of Beard. Your line is open.

Thank you and our next question will be coming from Brian <unk>.

Baird Your line is open.

Hey, Good afternoon, guys. My question is on the cannibalization.

The PMO as well and just.

Trying to contextualize when you say fairly modest cannibalization why isn't it just a straight up equation based on the prevalence of the draw.

Brian Peter Skorney: I would sort of calculate the, somewhere between 8 and 10 ELEVIDYS stations should result in one PMO patient cannibalization, assuming that person would have to be forced by their insurance company to choose to take ELEVIDYS and come off the PMO. Is that not the case? Are you seeing patients who are getting commercially treated with ELEVIDYS and maintaining their PMO? Post ELEVIDYS.

Exxon amenable patients and the penetration that you have into PMO like I would sort of calculate that somewhere between eight and 10 a lot of those patients should result in one PMO patient cannibalization assuming.

That person would have to.

But be forced by the insurance somebody to choose to take a lot at us and come off the PMO is that not the case are you seeing patients who are getting commercially treated with <unk> and maintaining their PMO.

Post a lot of just yeah.

Douglas S. Ingram: Yeah, Dallan, do you want to touch on this?

Alan do you want to touch on this.

Dallan Murray: Yeah, it's a good question, you know, one of the reasons, it's not a, you can't map it out that way is that, right now there's a significant percentage of our PMO business that doesn't come from the US. In terms of commercially right now, are there patients that have been dosed with gene therapy that have gotten PMOs? We haven't seen that to date, but we've seen that in the SMA space, and I think we're aware of some patients that are trying to get access that had dosed with ELEVIDY before, but we haven't seen that to date. So, noting that. Noting that that's the problem, Dallan touched on one of the confounders to your analysis, Brian, which is that XUS sales exist and have been relatively robust. So your math assumes a closed system, and we don't have a closed system; we actually have an open system with XUS PMO sales.

Yes, it's a good question you know one of the reasons.

It's not a <unk>.

Can't map it out that way is that is that.

Right now Theres, a significant percentage of our pls business that doesn't come from the U S.

In terms of of commercially right. Now are there are patients that have been dosed with gene therapy that that have gotten PMO.

We haven't seen that to date, but we've seen that in the SMA space and.

And I think we're aware of some patients that are that are trying to get access.

Just to elaborate as before but we havent I havent seen that today.

Douglas Ingram: Noting that that's the problem, Dallan touched on one of the confounders to your analysis, Brian, which is that XUS sales exist and have been relatively robust. So your math assumes a closed system, and we don't have a closed system; we actually have an open system with XUS PMO sales.

Yeah, so noting that thats the problem.

Douglas S. Ingram: So, noting that that's the problem, Dallan touched on one of the confounders to your analysis, Brian, which is that ex-US sales exist and have been relatively robust. So your math assumes a closed system, and we don't have a closed system, we actually have an open system with ex-US PMO sales.

Don touched on one of the contenders to your analysis.

Brian which is that ex U S sales exist and have been relatively robust so.

Your math, you're about to see what the closed system and we don't have a close system. We actually have an open system with ex U S PMA sales.

Operator: Thank you. One moment for our next question. And our next question is coming from Uy Ear of Mitsu. Your line is open. Guys, thanks for taking our question. So, I guess maybe just going back to the start form, you know, you said that sent the approval in 2023, and you've been receiving forms from all these sites and physicians, Just wondering if there were any forms that failed on patients that aged out before they can get treatment? And could you maybe also sort of define what you mean by patients who are declining, are these older patients or the ages doesn't matter at all? thanks.

Operator: Thank you. One moment for our next question, and our next question is coming from Uy Ear of Mizuho. Your line is open.

Thank you.

One moment for our next question.

Operator: Thank you, one moment for our next question, and our next question is coming from Uy Ear of Mizuho. Your line is open.

And our next question is coming from we ear of Mitsui.

Your line is open.

Guys. Thanks for taking our question so.

Uy Ear: Guys, thanks for taking our question. So, I guess maybe just going back to the start form, you know, you said that sent the approval in 2023, and you've been receiving forms from all these sites and physicians, Just wondering if there were any forms that failed on patients that aged out before they can get treatment? And could you maybe also sort of define what you mean by patients who are declining, are these older patients or the ages doesn't matter at all? thanks.

I guess, maybe just going back to the start form you know as you said.

Since the approval in 2023, you've been receiving forms from.

All of these sites and physicians just wondering if there were any storms that we feel are in patients who.

Aged out before they can get treatment.

And could you maybe also sort of define what you mean by.

Patients who are declining are these older patients who are there.

Does it.

Doesn't matter at all thanks.

Dallan Murray: Guys thanks for taking our question. So I guess, maybe just going back to the start form, you said that sent the approval in 2023, and you've been receiving forms from all these sites and physicians, Just wondering if there were any forms that failed on patients that aged out before they can get treatment? And could you maybe also sort of define what you mean by patients who are declining, are these older patients or the ages doesn't matter at all? thanks. I'm going to apologize, Dallan, if you understand, I didn't quite understand the question. I apologize. Yeah, no, I think I understood.

Uy Ear: Guys thanks for taking our question. So I guess, maybe just going back to the start form, you said that sent the approval in 2023, and you've been receiving forms from all these sites and physicians, Just wondering if there were any forms that failed on patients that aged out before they can get treatment? And could you maybe also sort of define what you mean by patients who are declining, are these older patients or the ages doesn't matter at all? thanks.

I'm going to apologize.

If you understand I didn't quite understand yes, I apologize, yes, no I think I understood. It. Please let me know if I did in terms of the last part of your question to the.

I'm going to apologize, Dallan, if you understand, I didn't quite understand the question. I apologize. Yeah, no, I think I understood.

Douglas S. Ingram: I'm going to apologize, Dallan, if you understand, I didn't quite understand the question. I apologize.

Yeah, no, I think I understood it, and please let me know if I didn't. In terms of the last part of your question, the decline, it was where the doctors seem to be prioritizing, no two sites are doing it exactly the same way. But they, as we've seen with our PMO launches, patients who are in the declining ambulatory phase, so that's kind of in the nine to 11 year old age ranges, it, they, those patients were predominantly prioritized in the previous three broad label exon skipping launches, and we've seen the same thing here. And what was the first part of your question again? I think he may have been asking if we, any patients that would have otherwise aged out?

Dallan Murray: Yeah, no, I think I understood it, and please let me know if I didn't. In terms of the last part of your question, the decline, it was where the doctors seem to be prioritizing, no two sites are doing it exactly the same way. But they, as we've seen with our PMO launches, patients who are in the declining ambulatory phase, so that's kind of in the nine to 11 year old age ranges, it, they, those patients were predominantly prioritized in the previous three broad label exon skipping launches, and we've seen the same thing here. And what was the first part of your question again?

Dallan Murray: Yeah, no, I think I understand it. And please let me know if I don't.

Dallan Murray: In terms of the last part of your question, the decline, it was where the doctors seem to be prioritizing; no two sites are doing it exactly the same way. But they, as we've seen with our PMO launches, patients who are in the declining ambulatory phase, so that's kind of in the nine to 11 year old age ranges, it, they, those patients were predominantly prioritized in the previous three broad label exon skipping launches, and we've seen the same thing here. And what was the first part of your question again?

But where the where the doctors.

Doctors seem to be prioritizing no two sites are doing it exactly the same way, but the.

As we've seen with our PMO launches patients who are in the declining ambulatory phase. So that's kind of in the nine to 11.

Our old age ranges.

It.

Those patients were predominantly prioritizing.

The previous three broad label exon skipping launches and we've seen the same thing here and what was the first part of your question again.

Douglas S. Ingram: I think he may have been asking if we, any patients that would have otherwise aged out?

I think you may have been asking.

Any patients that would have otherwise.

Dallan Murray: Aged out. Oh, aged out. Yeah, the aged out. Yeah, I was excited about that one because the team did such a great job in execution.

Aged out.

Dallan Murray: Oh, aged out, aged out, yeah, the aged out, yeah, I was excited about that one because the team did such a great job in execution, there were very few patients that did age out with the, in the initial narrow label. There were a few, and of course, the team never gives up on any patient, and we will, you know, those patients that aged out in the prior label will now be eligible again. So we will do everything we can to support anybody who did age out and comes back into the system. And then finally, what Dallan is suggesting on prioritizing the late declining is simply that it's just a mathematical fact. You're seeing patients, start forms from as early as four years old into the 30s. And then, you know, you see kind of a peak right now in the, you know, right in that kind of 10, 11, 9 age range. And that's what we've seen with the PMOs as well. But the good news is we're, you know, we're seeing a robust start across that entire group, which gives us a lot of confidence in where we're heading as an organization. Thank you. Our next question today will be coming from Robert Finke.

In Asia, The Asia, Yeah, I was excited about that one because the team did such a great job on execution. There were very few patients that did age out with in the initial narrow label.

There were a few and of course the team never gives up on any patient we will.

Both patients that have aged out in the private label will now be eligible again. So we will do everything we can to support anybody who did age out.

Dallan Murray: There were very few patients that did age out with the initial narrow label. There were a few, and of course, the team never gives up on any patient, and we will, you know, those patients that aged out in the prior label will now be eligible again. So we will do everything we can to support anybody who did age out and comes back into the system.

And comes back into the system.

Douglas Ingram: And then finally, what Dallan is suggesting on prioritizing the late declining is simply that it's just a mathematical fact. You're seeing patients, start forms from as early as four years old into the 30s. And then, you know, you see kind of a peak right now in the, you know, right in that kind of 10, 11, 9 age range. And that's what we've seen with the PMOs as well. But the good news is we're, you know, we're seeing a robust start across that entire group, which gives us a lot of confidence in where we're heading as an organization. Thank you. Our next question today will be coming from Robert Finke.

And so and then finally, what Dallas is suggesting on prioritizing delayed decline is simply Thats, just a mathematical fact youre seeing patients.

Dallan Murray: And [inaudible] what Dallan is suggesting on prioritizing the late declining, is simply that it's just, it is a mathematical fact, you're seeing patients, start forms from as early as four years old into the 30's, and then, you know, you see kind of a peak right now in the, you know, right in that kind of 10, 11, 9 age range, and that's what we've seen with the PMOs as well. But the good news is we're, you know, we're seeing a robust start forms across that entire group, which gives us a lot of confidence in where we're heading as an organization. Thank you. Our next question today will be coming from Robert Finke.

Douglas S. Ingram: And then sorry what Dallan is suggesting on prioritizing the late declining, is simply that it's just, it is a mathematical fact, you're seeing patients, start forms from as early as four years old into the 30's, and then, you know, you see kind of a peak right now in the, you know, right in that kind of 10, 11, 9 age range, and that's what we've seen with the PMOs as well. But the good news is we're, you know, we're seeing a robust start forms across that entire group, which gives us a lot of confidence in where we're heading as an organization.

Start forms from as early as four years old into the Thirty's and then you see kind of a peak right now.

Right and that kind of 10 11, nine age range and that's what we've seen with the PMO OS as well, but the good news is we're seeing a robust start forms across that entire.

Group, which gives us a lot of confidence in where we're heading as an organization.

Dallan Murray: Thank you. Our next question today will be coming from Robert Finke.

Operator: Thank you, our next question today will be from Robert Finke of Guggenheim. Your line is open.

Thank you.

Our next questioner today will be coming from Robert <unk> of Guggenheim. Your line is open.

Hey, Thanks for taking our question this is Robert Entre dubbed yet.

From our side a couple of questions here.

Do you anticipate manufacturing capacity, you will be getting factor.

Between now and peak sales.

Operator: Hey thank you for taking our question, this is Robert on for Debjit. From our side, a couple of questions here, do you anticipate manufacturing capacity will be getting factored at any point between nos and peak sales? Thank you very much. So the short answer is that we don't think manufacturing is going to be the bottleneck for the next few years. And by the time that we would see it as being a rate limiter, we'll hopefully be in suspension by that point, and then we shouldn't see any limitation. So we're very good where we are right now. Thank you. The next question is from David. Hougang City Group, your line is open. Hi there. Thanks for all the great updates and taking my...

Robert Finke: Hey thank you for taking our question, this is Robert on for Debjit. From our side, a couple of questions here, do you anticipate manufacturing capacity will be getting factored at any point between nos and peak sales?

Okay.

That's the question.

So the short answer is.

We don't envision that manufacturing is going to be the bottleneck for the next few years and by the time that we would see it as being a rate limiter will.

[inaudible] So the short answer is that we don't envision that manufacturing is going to be the bottleneck for the next few years, and by the time that we would see it as being a rate limiter, we'll hopefully be in suspension by that point, and then we shouldn't see any limitation. So we're very good where we are right now. Thank you. The next question is from David. Hougang City Group, your line is open. Hi there. Thanks for all the great updates and taking my...

Douglas S. Ingram: [inaudible] So the short answer is that we don't envision that manufacturing is going to be the bottleneck for the next few years, and by the time that we would see it as being a rate limiter, we'll hopefully be in suspension by that point, and then we shouldn't see any limitation. So we're very good where we are right now.

We'll be hopefully in suspension by that point and then we shouldnt see any limitation. So we feel very good where we are right now.

Thank you. The next question is from David. Hougang City Group, your line is open. Hi there. Thanks for all the great updates and taking my...

Operator: Thank you. Our next question is from David Hoang of Citigroup, your line is open. Hi there.

Operator: Thank you. Our next question is from David Hoang of Citigroup, your line is open.

Thank you. Our next question is from David.

Oh gosh.

Citigroup Your line is open.

Hi, there thanks for all the great updates and taking my question.

Just wanted to ask if you've got any feedback from your centers about post administration monitoring of these patients and there's obviously a number of labs that needs to be followed for a period of time for safety and I just wanted to ask whether.

That is a consideration and sort of.

The speed of how fast patient can be dose and then ultimately overall.

David Hoang: Hi there, thanks for all the great update there and taking my question. I just wanted to ask if you've gotten any feedback from your centers about post administration monitoring of these patients, there's always been a number of labs that need to be followed for a period of time for safety. And I just wanted to ask whether that is a consideration in sort of the speed of how fast patients can be dosed? And then ultimately overall this shape of the launch curve?

Because of the launch curve.

Douglas S. Ingram: Yeah, thank you very much for that question, and it's very insightful to, you know a lot of times we think about the infusion at the last moment, and that's sort of the cadence of infusions describes the amount of patients that can be dosed. But of course, if one is going to do this in a thoughtful way, it's important that there's a lot of very good follow-up, and there is, so the amount of infusions you can do relates not just to the amount of infusion room, infusion appointments you can have but a good follow-up, and we're very, very supportive of great follow-up. So that is a that is a part of the entire process, and to the best of my knowledge, we're seeing a very stable safety profile as well based on that monitoring, but Louise or [inaudible] in particular, if I'm getting any of that wrong, please let me know.

Yes. Thank you very much for that question and it's very insightful.

Other times, if you think about the infusion at the last moment.

And that that sort of the cadence of infusions describes the amount of patients that can be dosed, but of course, if one is going to do this in a thoughtful way. It's important that there is a lot of very good follow up and there is no debt amount of infusions you can do relate not just the amount of infusion or an infusion.

Our appointment you can have a good follow up and we're very very supportive.

Douglas Ingram: And we're very, very supportive of great follow-up. So that is a that is a part of the entire process. And to the best of my knowledge, we're seeing a very stable safety profile as well based on that monitor. Louise or Louise in particular. If I'm getting any of that wrong, please let me know.

Follow up so that is a.

That is a part of the entire process and that's what my knowledge, we're seeing a very stable safety profile as well based on that monitoring, but luis or.

Douglas S. Ingram: So that is a that is a part of the entire process, and to the best of my knowledge, we're seeing a very stable safety profile as well based on that monitoring, but Louise or [inaudible] in particular, if I'm getting any of that wrong, please let me know.

I think we showed particular.

Getting any of that wrong. Please let me know.

Operator: Thank you. [inaudible]

Operator: Thank you.

Thank you.

Louise Rodino-Klapac: [inaudible]

Operator: I'm sorry.

Oh I'm sorry.

Louise Rodino: Go ahead, Louise. Yeah, no, exactly right. Sorry.

Douglas S. Ingram: Go ahead, Louise.

Go ahead. Please go ahead.

Louise Rodino-Klapac: Yeah, no, exactly right. Sorry, that's all

Alright, sorry about that.

Operator: I didn't want your absence to be a negative. Our next question will be coming from Kristen Kluska of Conter Fitzgerald. Your line is open. Hi, everyone, and all the best with the expanded launch here.

Douglas S. Ingram: I didn't want your answer to be a negative a negative one.

Operator: Our next question will be coming from Kristen Kluska of Cantor Fitzgerald. Your line is open. Hi, everyone, and all the best with the expanded launch here.

Operator: Our next question will be coming from Kristen Kluska of Cantor Fitzgerald. Your line is open.

I didn't know I didn't want you or actions to be.

Negative.

Operator: Our next question will be coming from Kristen Kluska of Conter Fitzgerald. Your line is open. Hi everyone.

Our next question will be coming from Chris and consequent of Cantor Fitzgerald. Your line is open.

Hi, everyone and all the best with the expanded launch here wanted to ask if your 2025 numbers assume anything related to a European approval is that baked in here and then understand that this process takes a few months time, but what's the key driver behind reaching peak sales in a few years versus.

Kristen Kluska: Hi, everyone, and all the best with the expanded launch here. I wanted to ask if your 2025 numbers assume anything related to a European approval, is that baked in here? And then I understand that this process takes a few months time but what's the key driver behind reaching peak sales in a few years versus say, the 2026-2027 time frame? Thank you.

Say the 2026 2000 2007 timeframe. Thank you.

Douglas S. Ingram: So our guidance does not assume, first of all I want to be very clear, we'll leave it to Rose to talk about their ex-US approvals, and as everybody knows, EMA has already accepted their submission for review, and that should result in an action in 2025. But the numbers that we're giving here right now don't presume that issue either way, this is really relating to our US sales. And the short answer on the revenue curve is, of course, there is a revenue curve that occurs, and it is a function of everything from the process to the site monitoring, and site infusions, and payer interactions, and the like. And so, as I've said, we believe right now that we're going to do $2.9 to $3.1 billion dollars next year across all our therapies, we will continue to treat the prevalent population over the course of this entire decade. Peak year sales will be some years after 2025, and we feel very good about where we are, and the good work we're doing for patients. Thank you. Our next question will be coming from Biren Amin of Piper Sandler. Your line is open.

Yeah, So our guidance does not.

I assume.

I want to be very clear, we will leave it to Roche to discredit to talk about there ex U S approvals and as everybody knows <unk> already accepted our submission for review and that that should.

The result in the action.

That action in 2025, but the numbers that we're giving here right now don't presume that.

Issue either way this is really relating to our U S sales and the short answer on the revenue curve is of course, there is the revenue curve that occurs and it is a function of everything from the process too.

Douglas S. Ingram: And so, as I've said, we believe right now that we're going to do $2.9 to $3.1 billion dollars next year across all our therapies, we will continue to treat the prevalent population over the course of this entire decade. Peak year sales will be some years after 2025, and we feel very good about where we are, and the good work we're doing for patients.

The site monitoring.

<unk> infusions and payer interactions and the like and so as I've said.

I believe right now that we're going to do $2 90 to $3 $1 billion next year across all of our therapies. We will continue to treat the prevalent population over the course of this entire decade peak year sales will be some years. After 2025, and we feel very good about where we are and the good work we're doing for patients.

Douglas Ingram: We will continue to treat the prevalent population over the course of this entire decade. Peak year sales will be some years after 2025, and we feel very good about where we are and the good work we're doing for patients. Thank you. Our next question will be coming from Biren Amin of Piper Sandler. Your line is open.

Douglas S. Ingram: Thank you. Our next question will be coming from Biren Amin of Piper Sandler. Your line is open.

Operator: Thank you. Our next question will be coming from Biren Amin of Piper Sandler. Your line is open.

Thank you.

Our next question will be coming from <unk>.

Barry Amman.

Piper Sandler your line is open.

Yes. Thanks for taking my question guys you comment on the number of infusing sites over the last few quarters with a number of infusing sites, increasing in Q2 from Q1 by about 13% to 15 sites. According to our calculations. So how do we think about patient.

Treated per site and the consistency of the volume of patients treated per site.

Douglas Ingram: Yeah thanks for taking my questions guys, you commented on the number of infusion sites increasing in q2 from q1 by about 13 to 15 sites according to our calculation, so how do we think about patient studied per site and the consietency of the volume of patients treated per site. Well, you know, so let's first turn this over to Dallan. He can chat a bit about that. But just so we're very clear, we're very comfortable with the number of sites that we see. Now, what you may see over time is a general increase in some sites. That is not part of some grand proactive strategy but really relates more than anything else to being responsive to requests from sites. So if an appropriate site asked to be a site and wanted to go through the education process and be validated to be a site, we would certainly consider that.

Biren Amin: Yeah thanks for taking my questions guys, you commented on the number of infusing sites over the last few quarters, with the number of infusing sites increasing in Q2 from Q1 by about 13 to 15 sites according to our calculation. So how do we think about patients treated per site? And the consistency of the volume of patients treated per site?

Well.

So let's first of all I'll turn this over to Dallas, you can chat a bit about that but just so we're very clear we are very comfortable with the number of sites that we see now what you may see over time is a general increase in some sites that is not part of some grand proactive strategy, but really really.

Douglas S. Ingram: Well, you know, so let's first, I'll turn this over to Dallan, he can chat a bit about that, but just so we're very clear, we're very comfortable with the number of sites that we see. Now, what you may see over time is a general increase in some sites, that is not part of some grand proactive strategy but really relates more than anything else to being responsive to requests from sites. So if an appropriate site asked to be a site, and wanted to go through the educational process and be validated to be a site, we would certainly consider that, but as it sits here right now, we are very comfortable. As I will remind everyone, in advance of this launch, we had an aspiration to be 50 sites, with a goal of maybe even someday getting to 70 sites, all of which would be much higher than any other launch of a gene therapy. And so sitting at 75 sites, we feel very, very good about it, but if you want to provide any other metrics, Dallan, on kind of productivity by site, or the like [inaudible] Yeah, no, thank you.

<unk> more than anything else to being responsive to requests from side. So if appropriate site asked via site. When they go through the educational process to be validated a site, we would certainly consider that but as I sit here right. Now we are very comfortable as I will remind everyone in advance of this launch we had an.

Douglas Ingram: But as it sits here right now, we are very comfortable. As I will remind everyone, in advance of this launch, we had an aspiration to be 50 sites, with a goal of maybe even someday getting to 70 sites, all of which would be much higher than any other launch of a gene therapy. And so sitting at 75 sites, we feel very, very good about it. But if you want to provide any other metrics, Dallan, on kind of productivity by side of the like, Yeah, no, thank you.

<unk> to be 50 sites with a goal of maybe even someday getting to 70 sites all of which would be much higher than any other.

Launch of a gene therapy, and so sitting at 75 sites, we feel very very good about it but if you want to provide any other metrics dial in on kind of.

<unk> productivity by side of the light rail freight.

Dallan Murray: Yeah, no, thank you for the question, and I don't know where those numbers came from, because our site, the number of sites has been very consistent, we have a right-sized model, and, you know, we've been hovering right around 75. Now, that said, just as Doug said, our model is flexible, so we can bring new sites on, get them up to speed, and bring them on very rapidly. And, you know, one of the things with this broad label is the possibility that we'll be bringing on some adult neuromuscular sites to accommodate older patients as well. So, as Doug alluded to, we have the, we have more than enough capacity today. But importantly, to serve patients, we have built flexibility into our model so that, as needed to support patients, we can bring on new sites very, very rapidly. The team is very responsive. Thank you. And our next question will be coming from Gavin Clark, Gardner of Evercore. Your line is open. Hey guys, thanks for taking the question. I fully appreciate the longer conversion cycle as we're

Dallan Murray: Yeah, no, thank you for the question, and I don't know where those numbers came from, because our site, the number of sites has been very consistent, we have a right-sized model, and, you know, we've been hovering right around 75. Now, that said, just as Doug said, our model is flexible, so we can bring new sites on, get them up to speed, and bring them on very rapidly. And, you know, one of the things with this broad label is the possibility that we'll be bringing on some adult neuromuscular sites to accommodate older patients as well. So, as Doug alluded to, we have the, we have more than enough capacity today, but importantly, to serve patients, we have built flexibility into our model so that, as needed to support patients, we can bring on new sites very, very rapidly, the team is very responsive.

Dallan Murray: Yeah, no, thank you for the question. And I don't know where those numbers came from, because our site, the number of sites has been very consistent; we have a right-sized model. And, and, you know, we've been hovering right around 75. Now, that said, just as Doug said, our model is flexible. So we can bring new sites on, get them up to speed, and bring them on very rapidly. And, you know, one of the things with this broad label is the possibility that we'll be bringing on some adult neuromuscular sites to accommodate older patients as well.

Yeah no. Thank you for the question and I don't know where those numbers came from because our site. The number of sites has been very consistent.

<unk> sized our model.

We've been hovering right around 75, now that said just as Doug said our model is flexible. So we can bring new sites on get them up to speed and bring them on very rapidly and one of the things with this broad label is the possibility that we will be bringing on some idle adult neuromuscular sites to accommodate.

Dallan Murray: So, as Doug alluded to, we have the, we have more than enough capacity today, but importantly, to serve patients, we have built flexibility into our model so that, as needed to support patients, we can bring on new sites very, very rapidly, the team is very responsive. Thank you. And our next question will be coming from Gavin Clark, Gardner of Evercore. Your line is open. Hey guys, thanks for taking the question. I fully appreciate the longer conversion cycle as we're

Older patients as well so as Doug alluded.

Dallan Murray: So, as Doug alluded to, we have the, we have more than enough capacity today. But importantly, to serve patients, we have built flexibility into our model so that, as needed to support patients, we can bring on new sites very, very rapidly. The team is very responsive. Thank you. And our next question will be coming from Gavin Clark, Gardner of Evercore. Your line is open. Hey guys, thanks for taking the question. I fully appreciate the longer conversion cycle as we're

We have more than enough capacity a day, but importantly to serve patients we have built flexibility into our model so that.

Dallan Murray: Thank you. And our next question will be coming from Gavin Clark-Gartner of Evercore. Your line is open. Hey guys, thanks for taking the question. I fully appreciate the longer conversion cycle as we're

Operator: Thank you. And our next question will be coming from Gavin Clark-Gartner of Evercore. Your line is open.

As needed to support patients we can bring on new sites very very rapidly the team is very responsive.

Operator: Thank you. And our next question will be coming from Gavin Clark, CEO of Evercore. Your line is open.

Thank you and our next question will be coming from Gavin Clark Gartner of Evercore. Your line is open.

Hey, guys. Thanks for taking the question.

So I fully appreciate the longer conversion cycle as we're looking at the back half of 'twenty four and into 2025.

Gavin Clark-Gartner: Hey guys, thanks for taking the question. I fully appreciate the longer conversion cycle as we're looking at the back half of '24 and into 2025, but you've also referenced your approved PMOs as analogs for adoption. Those themselves have the largest ramps within the first one or two years after approval when we're talking about new patient adds, and that's basically how all other rare disease launches go with very high unmet need. So I guess like even with a few months of the light process, why would peak sales not be at some point in 2026?

But you've also referenced your approved PMO OS as analog for adoption.

Themselves are the largest ramps within the first one or two years after approval when we're talking about new patient adds and Thats basically how all other rare disease launches go with very high unmet need so I guess like even with a few months of a lag process why would peak sales not be at some point in 2026.

Because it won't be.

Douglas S. Ingram: Because it won't be. We have good modeling, we know exactly, you know, we have a very good view of where we're heading with, you know, the capacities and the process and the like, and we're confident that it won't be peaky or so in 2026. So another thing you have to remember, and by the way, and I think that people are trying to solve for whether there is some problem. There's not. We're very pleased with this approach. One has to remember that there's a one-time therapy that we have to be, you know, we have to prioritize, being responsible, and we want to make sure that all of these sites are in a good place, where they're not prioritizing getting as many patient's as possible dosed without regard to safety and follow-up in the like. And we've done a very good job at that, the sites are doing a very good job at that, all of which means We're going to be very successful this year. We're going to have good, modest growth in Q3. We're going to have very, very robust growth in Q4, double in Q4. We're going to do $2.9, $3.1 billion across our therapies in 2025. And then, as it relates to peak year sales, it'll be in the back half of and Doug, can I ask you a question?

We have good modeling we know exactly we have a very good view of where we're heading.

Is the capacities in the process and so on.

And we're confident that it won't be a peak year sales in 2022 and the other thing you have to remember and by the way and I think that people are trying to solve for whether there is some bulk there's not we're very pleased with this approach one has to remember.

Douglas Ingram: We're very pleased with this approach. One has to remember that there's a one-time therapy that we have to be, you know, we have to prioritize. We're going to be very successful this year. We're going to have good, modest growth in Q3. We're going to have very, very robust growth in Q4, double in Q4. We're going to do $2.9, $3.1 billion across our therapies in 2025. And then, as it relates to peak year sales, it'll be in the back half of and Doug, can I ask you a question?

Douglas S. Ingram: One has to remember that there's a one-time therapy that we have to be, you know, we have to prioritize, being responsible, and we want to make sure that all of these sites are in a good place, where they're not prioritizing getting as many patient's as possible dosed without regard to safety and follow-up in the like. And we've done a very good job at that, the sites are doing a very good job at that, all of which means we're going to be very successful this year. We're going to have good, modest growth in the Q3, we're going to have very, very robust growth in Q4, doubling in Q4, we're going to do $2.9, $3.1 billion dollars across our therapies in 2025. And then, as it relates to peak year sales, it'll be in the back half of this decade. and Doug, can I ask you a question?

Matt.

This is a onetime therapy that we have to be.

We have to prioritize being responsible.

And we want to make sure that all of these sites are in a good place where theyre not prioritizing getting as many patients as possible dosed without regard to safety and follow up and alike.

We've done a really good job of that the sites are doing a very good job of that all of which means we're going to be very successful. This year, we're going to have good modest growth in Q3, and we have very very robust growth in Q4, doubling in Q4, we're going to do $2 $93 $1 billion across our therapies in 2025, and then as it relates to.

Douglas S. Ingram: We're going to have good, modest growth in the Q3, we're going to have very, very robust growth in Q4, doubling in Q4, we're going to do $2.9, $3.1 billion dollars across our therapies in 2025. And then, as it relates to peak year sales, it'll be in the back half of this decade.

Dallan Murray: And Doug, can I just jump in? I think you've been alluding to this, but Gavin, the question's a good one, and I think Doug's been alluding to the fact. We have visibility at these sites at a very granular level, we have it mapped out by payer coverage, by patient, we know exactly who's lined up, and how they're going to be lined up. We map this out in very, very granular detail, and this is why we have a very high level of confidence in our forecast assumptions. So I hope that added level of detail helps a bit.

Peak year sales it'll be in the back half of this decade.

Dallan Murray: And Doug, can I just jump in? I think you've been alluding to this, but Gavin, the question's a good one, and I think Doug's been alluding to the fact that we have visibility at these sites at a very granular level. We have it mapped out by payer coverage, by patient. We know exactly who's lined up, and how they're going to be lined up. We map this out in very, very granular detail, and this is why we have a very high level of confidence in our forecast assumptions. So I hope that added level of detail helps a bit.

And Doug can I just jump in I think you've been alluding to this but gavin but your question's a good one I think doug's been alluding to the fact, we have visibility at these sites at a very granular level, we haven't mapped out by payer coverage by patients we know exactly who's lined up how theyre going to be lined up.

We mapped this out in a very very granular detail and this is why we have a very high level of confidence in our forecast assumptions.

I hope that added level of detail helps a bit.

Operator: Thank you, Dallan. Thank you. And our next question will be coming from Kostas Biliouris, of BMO Capital Markets. Your line is open. Hi, this is Dale on behalf of Kostas. Thank you for taking our question. So our question is about the LGMD gene therapy policy.

Douglas S. Ingram: Thank you, Dallan.

Thank you. And our next question will be coming from Kostas Biliouris, of BMO Capital Markets. Your line is open. Hi, this is Dale on behalf of Kostas. Thank you for taking our question. So our question is about the LGMD gene therapy policy.

Thank you Don.

Operator: Thank you. And our next question will be coming from Kostas Biliouris of BMO Capital Markets. Your line is open. Hi, this is Dale on behalf of Kostas. Thank you for taking our question. So our question is about the LGMD gene therapy policy.

Operator: Thank you. And our next question will be coming from Kostas Biliouris of BMO Capital Markets. Your line is open.

Thank you and our next question will be coming from Cortez malaria.

<unk> B M O capital markets. Your line is open.

Hi does this forecast.

Forecast us. Thank you for taking my question. So our question is on <unk>.

<unk> sorry for your portfolio given that half of your LNG MD gene therapy portfolio uses the same factor.

Operator: Hi, this is Dale on for Kostas, thank you for taking our question. So our question is on the LGMD gene therapy portfolio. Given that half of your LGMD genes therapy portfolio uses the same vector and promoter, and actually all of them are using the same vector as ELEVIDYS. So how are you thinking about potentially leveraging FDA platform, designation platform, to accelerate these developments off to LGMD gene therapies and other early pipeline assets. And also if you could comment on the potential impact from the inclusion of ELEVIDYS effect on the time function test in the label, on patients and [inaudible] uptake payers negotiations in the potential future competitions? thank you. I'll turn the LGMD question over to Louise and our approach to accelerating LGMD development.

Kostas Biliouris: Hi, this is Dale on for Kostas, thank you for taking our question. So our question is on the LGMD gene therapy portfolio. Given that half of your LGMD genes therapy portfolio uses the same vector and promoter, and actually all of them are using the same vector as ELEVIDYS. So how are you thinking about potentially leveraging FDA platform, designation platform, to accelerate these developments off to LGMD gene therapies and other early pipeline assets. And also if you could comment on the potential impact from the inclusion of ELEVIDYS effect on the time function test in the label, on patients and [inaudible] uptake payers negotiations in the potential future competitions? Thank you.

Our motor and actually all of them are using the same factor as <unk>. So how are you thinking about.

Leveraging FDA platform designation platform Turkcell.

To accelerate the development of the MGMT gene therapies and other early pipeline assets and also if you can comment on the potential impact from the inclusion of this effect on the time function test in the label on patients and physician uptake payer negotiations and a potential future competition. Thank you.

Louise Rodino: I'll turn the LGMD question over to Louise and our approach to accelerating LGMD development.

Eric.

<unk>.

<unk> question over to Luis and sort of our approach to accelerating the LG MD development.

Douglas S. Ingram: I'll turn the LGMD question over to Louise and of our approach to accelerating the LGMD development.

Louise Rodino-Klapac: Yeah, thanks for the question, I think you're exactly right in terms of using the ELEVIDYS experience to help accelerate the LGMD platform. We've been using the same RH-74 vector for these programs, just to cite the sarcoglycanopaties similarities between them. We've received fast track designation, as I mentioned in my remarks, and pulling on every lever possible with FDA to make sure that we're moving in the fastest speed possible. We've been very happy with the interaction so far, and that's our 9003 pivotal trial, as noted, is our open-label trial with, in terms of looking at this ultra-rare population and being thoughtful about the way that we develop these with LGMD 2D and 2C coming along. So certainly, we're leveraging everything possible, and our safety experience is ELEVIDYS for the LGMD portfolio.

Yeah. Thanks for the question I think you're exactly right in terms of using the <unk> experience to help accelerate the LGD platform.

And then using the same <unk> 74, a vector for these programs and just to to cite the cyclical I cannot with any similarities between them.

Received fast track designation as I mentioned in my remarks, and pulling on every lever possible with FDA to make sure that we're on.

Moving and are in the fastest.

Possible.

Louise Rodino: And that's our 9003 pivotal trial, as noted, is our open-label trial with, In terms of looking at this ultra-rare population and being thoughtful about the way that we develop these with LGMD 2D and 2C coming along. So certainly, we're leveraging everything possible, and our safety experience is levitous for the LGMD portfolio.

We've been very happy with the interaction so far and that's our ninth or is there are three pivotal trial as is noted as our open label trial with that.

Louise Rodino-Klapac: We've been very happy with the interaction so far, and that's our 9003 pivotal trial, as noted, is our open-label trial with, in terms of looking at this ultra-rare population and being thoughtful about the way that we develop these with LGMD 2D and 2C coming along. So certainly, we're leveraging everything possible, and our safety experience is ELEVIDYS for the LGMD portfolio.

In terms of looking at this ultra rare population and being thoughtful about the way that we develop this.

With LG, Indeed, QD and can't see coming along so certainly we're leveraging everything possible and our safety experience with <unk> for the outstanding portfolio.

Okay.

Douglas S. Ingram: Hold on, before we move on, Dallan, did you have something else to add?

Alright.

Thanks.

Before we move on down to just something else to add.

I think bill asked about the time function test the data that's in our label and how those may be impacting the payer conversations and Dale. It's a great really really great question because that is really what the team is focused on with the Payors is that incredible new data that's in our label and there has been as Doug outlined in his comments I did.

A little bit in mind the engagement the interest from the payers to understand that data and so that's a big part of that engagement with the payers right now is going through that new data that supports a broader label for allowed us.

Dallan Murray: Just to remind everyone that not only did we hit all of those anomalies statistically significantly, but the importance of that can't be overstated. I would remind people that time to rise, the benefit we've seen on time to rise is correlated with over 90% um decrease in the risk of early loss of ambulation, so these are not only important metrics, but they correlate importantly with the very things that matter a lot to patients. Thank you. And our next question will be coming from Tim Lugo of World Health.

Douglas S. Ingram: Just to remind everyone that not only did we hit all of those, the anomalies statistically significantly, the importance of that can't be overstated. I would remind people that on time to rise, the benefit we've seen on time to rise is correlated with over a 90% decrease in the risk of early loss of ambulation. So I mean, these are not only important metrics, but they correlate importantly with the very things that matter a lot to patients.

Dallan Murray: Yeah, I think Dale asked about the time function test, the data that's in our label and how those may b e impacting the payer conversation? And Dale that's a great, really, really a great question because that is really what the team is focused on with the payers, is that incredible [inaudible] data that's in our label, and there has been. As Doug outlined in his coments, I did a little bit in mine, the engagement, the interest from the payers to understand that data . And so that's a big part of that engagement with the payers, right know, going through that new data that supports a broader label for ELEVIDYS. Just a reminder:

Dallan Murray: Yeah, I think Dale asked about the time function test, the data that's in our label and how those may be impacting the payer conversation? And Dale that's a great, really, really a great question because that is really what the team is focused on with the payers, is that incredible new data that's in our label, and there has been, as Doug outlined in his comments, I did a little bit in mine, the engagement, the interest from the payers to understand that data. And so that's a big part of that engagement with the payers, right know, going through that new data that supports a broader label for ELEVIDYS.

Just to remind everyone not only did we hit all of those.

Statistically significantly the importance of that can't be overstated, I would remind people that.

Time to rise the benefit we've seen on time to rise, it's correlated with over 90%.

Decrease in the risk of early loss of ambulation. So I mean these are.

Not only important metrics, but they correlate importantly to vary.

Things that matter a lot to patients.

Operator: Thank you, and our next question will be coming from Tim Lugo of William Blair. Your line is open. Hey team, this is John on for Tim, thanks so much for taking our question, maybe a follow-up on the last one. Beyond the internal pipeline with your healthy balance sheet and your expanded launch in front of you, just wondering if you could give us any updated thoughts on how you're thinking about potentially in-licensing new programs?

Operator: Thank you, and our next question will be coming from Tim Lugo of William Blair. Your line is open.

Thank you and our next question will be coming from Tim Lugo of William Blair. Your line is open.

Tim Lugo: Hey team, this is John on for Tim, thanks so much for taking our question, maybe a follow-up on the last one. Beyond the internal pipeline with your healthy balance sheet and your expanded launch in front of you, just wondering if you could give us any updated thoughts on how you're thinking about potentially in-licensing new programs?

Hey, Tim This is John on for Tim. Thanks, So much for taking my question, maybe a follow up on the last one beyond the internal pipeline with your healthy balance sheet and your expanded launch in front of you I'm. Just wondering if you can give us any updated thoughts on how youre thinking about potentially in licensing new program.

Douglas S. Ingram: Sure, I'll turn this to Ian, Ian.

Sure.

This two Ian Ian.

Ian Estepan: Hi, thanks for the question, and yeah, I mean, you know, this is what I discussed in our prepared remarks, I mean, you know, as an organization that's been developing therapies and successfully manufacturing them, and all the way to commercialization, it gives us, you know, a wide amount of substrates to evaluate. And so, you know, we're going to be just looking at ones that fit very well with our existing capabilities, and being able to leverage that, and being able to deliver it to patients quickly. So, you know, we've been really fiscally responsible as the transactions we've done in the past, like I said in the prepared remarks, we're going to have a lot more resources to deploy, but we're still going to use that same approach. You know, there are a lot of interesting therapies that are being developed, but, you know, before maybe this week, the valuations were incredibly high, and we're not going to, you know, we're not going to chase opportunities. We're going to look for ways to build value from a development perspective, you know, for patients but also for investors, so, you know, we're very keen on looking at valuation and making sure that it makes sense, you know, based on the market opportunities.

Hey, Thanks for the question.

This is what I discussed in our prepared remarks I mean.

As a as an organization, that's developing therapies and successfully manufacturing them in all the way the commercialization. It gives us you know.

A wide amount of substrate to evaluate.

And so we're going to be just looking at ones that fit very well with our existing capabilities and being able to leverage that.

Ian Estepan: So, you know, we've been really fiscally responsible for the transactions we've done in the past. Like I said in the prepared remarks, we're going to have a lot more resources to deploy, but we're still going to use that same approach. You know, there are a lot of interesting therapies that are being developed, but, you know, before maybe this week, the valuations were incredibly high, and we're not going to, you know, chase opportunities.

And being able to deliver to patients quickly so.

Ian Estepan: So, you know, we've been really fiscally responsible as the transactions we've done in the past, like I said in the prepared remarks, we're going to have a lot more resources to deploy, but we're still going to use that same approach. You know, there are a lot of interesting therapies that are being developed, but, you know, before maybe this week, the valuations were incredibly high, and we're not going to, you know, we're not going to chase opportunities. We're going to look for ways to build value from a development perspective, you know, for patients but also for investors, so, you know, we're very keen on looking at valuation and making sure that it makes sense, you know, based on the market opportunities.

We've been really fiscally responsible.

The transactions we've done in the past like I said in the prepared remarks, we're going to have a lot more resources.

To deploy but we're still going to use that same approach.

There are a lot of interesting therapies that are being developed but.

Ian Estepan: You know, there are a lot of interesting therapies that are being developed, but, you know, before maybe this week, the valuations were incredibly high, and we're not going to, you know, we're not going to chase opportunities. We're going to look for ways to build value from a development perspective, you know, for patients but also for investors, so, you know, we're very keen on looking at valuation and making sure that it makes sense, you know, based on the market opportunities.

Before maybe this week the valuations were incredibly high and we're not going to win.

Not going to chase opportunities, we're going to look for ways to build value.

Ian Estepan: We're going to look for ways to build value from a development perspective, you know, for patients but also for investors, so, you know, we're very keen on looking at valuation and making sure that it makes sense, you know, based on the market opportunities.

From a development perspective for patients, but also for investors. So we're very keen on.

Ian Estepan: We're going to look for ways to build value from a development perspective, you know, for patients but also for investors. So, you know, we're very keen on looking at valuation and making sure that it makes sense, you know, based on the market opportunities.

Looking at valuation and making sure that it makes sense.

On the market opportunities.

Yeah.

Operator: Thank you. And our next question will be coming from Gil Blum of Needham & Company. Your line is open.

Thank you and our next question will be coming from GE Bloom.

Of Needham and company your line is open.

Gil Blum: Hi, everyone, thanks for squeezing us in, maybe a last question, I'm trying to understand the potential bottlenecks here, but doesn't it really just boil down to how many treating physicians you have at each center? And if you can provide any thoughts on how that metric works. Thanks.

Hi, everyone. Thanks for squeezing us in.

Maybe a last question I'm trying to understand the potential bottlenecks here, but it doesn't it really just boil down to how many treating physicians you have with each center and if you can provide any any thoughts on how that metric works. Thanks.

Douglas S. Ingram: So again, I'm going to frustrate you by answering this question again the same way, which is that we don't have bottlenecks, we have a launch curve, we're doing very, very well. We have a significant number of sites, a significant number of treating physicians at sites, a lot of enthusiasm, we have more than enough sites, more than enough physicians, extraordinary demand from patients and as physicians, clean interactions with payers. And a very strong manufacturing approach right now, I feel very good about that from a capacity and supply perspective, and a great distribution channel. As a result of that, we're going to have a really successful back half of this year, and we're going to have 2025 that will mean that we're going to do revenue across our four approved therapies of some $2.9 billion dollars to $3.1 billion dollars. We are profitable today, we were cash flow positive this quarter, we will be in the next couple of quarters very consistently cash flow positive on a go-forward basis. We're in a very different place than the vast majority of Biotechs Today. We have a very strong, ver sustainable business all of which is focused first and foremost in bringing better life for these patients, we're going to bring a better life to a lot of patients over the next, you know, many years this decade. And secondarily, but also importantly, rewarding those investors who have stuck with us and committed themselves to this mission, so we feel like we're in great shape. And not to be overly defensive, but we don't have a bottleneck that we need to solve for, we feel very good about where we are.

So again.

Frustrate me by answering this question again, the same way, which is we don't have bottlenecks we.

We have a launch curve.

Very very well, we have a significant number of sites a significant number of treating physicians at sites a lot of enthusiasm.

We have more than enough sites more than enough positions extraordinary demand from patients and physicians, great interactions with payers a very strong manner.

Douglas Ingram: I feel very good about that from a capacity and supply perspective and a great distribution channel. As a result of that, we're going to have a really successful back half of this year, and we're going to have 2025 that will mean that we have revenue across our approved therapies of some $2.9 billion to $3.1 billion. Over the next, you know, many years this decade, and secondarily, but also importantly, rewarding those investors who have stuck with us and committed themselves to this mission. So we feel like we're in great shape. And not to be overly defensive, but we don't have a bottleneck that we need to solve for. We feel very good about it.

A manufacturing approach right now.

Very good about that from a capacity and supply perspective, and a great distribution channel as a result of that we're going to have a really successful back half of this year and we're going to have a 2025 that will.

Douglas S. Ingram: As a result of that, we're going to have a really successful back half of this year, and we're going to have 2025 that will mean that we're going to do revenue across our four approved therapies of some $2.9 billion dollars to $3.1 billion dollars. We are profitable today, we were cash flow positive this quarter, we will be in the next couple of quarters very consistently cash flow positive on a go-forward basis. We're in a very different place than the vast majority of Biotechs Today. We have a very strong, ver sustainable business all of which is focused first and foremost in bringing better life for these patients, we're going to bring a better life to a lot of patients over the next, you know, many years this decade. And secondarily, but also importantly, rewarding those investors who have stuck with us and committed themselves to this mission, so we feel like we're in great shape. And not to be overly defensive, but we don't have a bottleneck that we need to solve for, we feel very good about where we are.

<unk> mean that were going to do revenue across our vital across our four approved therapies of some $2 9 billion to $3 1 billion.

We are profitable today, we were cash flow positive this quarter.

We'll be in the next couple of quarters very consistently cash flow positive on a go forward basis, we're in.

Douglas S. Ingram: We have a very strong, ver sustainable business all of which is focused first and foremost in bringing better life for these patients, we're going to bring a better life to a lot of patients over the next, you know, many years this decade. And secondarily, but also importantly, rewarding those investors who have stuck with us and committed themselves to this mission, so we feel like we're in great shape. And not to be overly defensive, but we don't have a bottleneck that we need to solve for, we feel very good about where we are.

Very different place than the vast majority of biotech today, we have a very strong very sustainable.

All of which is focused first and foremost and bring a better life for these patients we're going to bring a better life through a lot of patients.

Over the next many years this decade and secondarily, but also importantly, rewarding those investors who have stuck with us and have committed themselves to this mission. So we feel like we're in great shape and not to be overly defensive but we don't have a bottleneck that we need to solve and we feel very good about.

Douglas S. Ingram: And secondarily, but also importantly, rewarding those investors who have stuck with us and committed themselves to this mission, so we feel like we're in great shape. And not to be overly defensive, but we don't have a bottleneck that we need to solve for, we feel very good about where we are.

Wherever you are.

Operator: Thank you, this concludes today's Q&A session and I would like to turn the call over to Doug for closing remarks. Please go ahead.

Thank you. This concludes today's Q&A session I would like to turn the call over to Doug for closing remarks. Please go ahead.

Douglas S. Ingram: Thank you very much, thank you all for your questions, your insightful questions this evening, and thank you to my team for those great answers. I'm very excited about where we are going for the rest of this year, we have a lot of work to do this year. Our launch is going well, our continuing service of our PMOs are going very well, we understand the enormous responsibility we have to these patients to ensure that the greatest number of these patients can benefit from our therapies, and we're going to make the ELEVIDYS launch brilliant.

Thank you very much. Thank you all for your questions. Your insightful questions. This evening and thank you to my team for.

Those great answers.

We're excited about where we're going for the rest of this year, we have a lot of work to do this year. Our launch is going well, our continuing service of our PMO are going very well.

We understand the enormous responsibility we have to these patients to ensure that the greatest number of these patients.

Douglas Ingram: And we're going to make the ELEVIDYS launch brilliant. I look forward to updating you throughout this year on the launch, performance, and with the team to update you on our pipeline progress as well. And with that, have a lovely evening, and I look forward to talking to you soon. Thanks.

And we're going to make the ELEVIDYS launch brilliant.

Can benefit from our therapies and we're going to make the elaborate us launch brilliant I look forward to updating you across this year on launch performance and with the team to update you on our pipeline advancement as well.

Douglas S. Ingram: I look forward to updating you across this year on the launch, performance, and with the team to update you on our pipeline advancement as well. And with that, have a lovely evening, and I look forward to talking to you soon. Thanks.

Have a lovely evening and look forward to talking to you soon thanks.

Operator: This does conclude today's conference call. You may all disconnect.

This does conclude today's conference call you may all disconnect.

Okay.

[music].

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