Q4 2023 Sarepta Therapeutics Inc Earnings Call

February 28, 2024

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Yes.

Operator: Good afternoon, and welcome to the Sarepta Therapeutics Q4 and Full Year 2023 Earnings 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 your hand is raised. To withdraw your question, please press star 11 again. As a reminder, today's program is being recorded. At this time, I'll turn the call over to Francesca Nolan, Executive Director, Investor Relations at Corporate Communications. Please go ahead.

Operator: Good afternoon, and welcome to the Sarepta Therapeutics 4th quarter and Full Year 2023 Earnings 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 this session, you will need to press star 1-1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1-1 again. As a reminder, today's program is being recorded.

Speaker Change: The speaker's presentation, there will be a question and answer session to ask a question. During this session you will need to press star one one of your telephone you will then hear an automated message advising your hand is raised.

Speaker Change: Your question. Please press star one again.

Speaker Change: As a reminder, today's program is being recorded.

Speaker Change: At this time I'll turn the call over to Francesca Nolan, Executive Director of Investor Relations and Corporate Communications. Please go ahead.

Francesca Nolan: Thank you, Shannon, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the Q4 and full year 2023. The press release is available on our website at sarepta.com, and our 10-K 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-Klapac. After our formal remarks, we'll open the call for Q&A. I'd like to note that during this call, we will 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.

Francesca Nolan: Thank you, Shannon and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the 4th quarter and full year 2023. The press release is available on our website at sarepta.com and our 10-K 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-Klapac. After our formal remarks, we'll open the call for Q&A.

Francesca Nolan: This afternoon, we released our financial results for the fourth quarter and full year 2023.

Francesca Nolan: The press release is available on our website at <unk> Dot Com and our 10-K was filed with the Securities and Exchange Commission. This afternoon.

Francesca Nolan: Joining us on the call today are talking room, you can act upon Alan Murray and Dr. Louise Rodino quite pack.

Speaker Change: After our formal remarks, we'll open the call for Q&A. I'd like to note that during this call, we will 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 our control. Actual results could materially differ from these forward-looking statements, 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-K filed with the SEC as well as the company's other SEC filings. The company does not undertake any obligation to publicly update forward-looking statements including any financial projections provided today, based on subsequent events or circumstances.

After our formal remarks, we'll open the call for Q&A.

Speaker Change: Like to note that during this call we will 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.

I'd like to note that during this call, we will 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 our control. Actual results could materially differ from these forward-looking statements, 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-K filed with the SEC as well as the company's other SEC filings. The company does not undertake any obligation to publicly update forward-looking statements including any financial projections provided today, based on subsequent events or circumstances.

Speaker Change: These forward looking statements involve risks and uncertainties, many of which would be honest erupted control.

Francesca Nolan: Actual results can 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-K 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. 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: Actual results could materially differ from these forward looking statements any such risks can materially and adversely affect the business.

Speaker Change: The results of operations and trading prices for <unk> common stock.

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-K filed with the SEC as well as the company's other SEC filings. The company does not undertake any obligation to publicly update forward looking statements. Any financial projections provided today based on subsequent events or circumstances.

Speaker Change: The company does not undertake any obligation to publicly update forward looking statements.

Speaker Change: Any financial projections provided today based on subsequent events or circumstances.

Speaker Change: And now, I will turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?

Douglas Ingram: Thank you, Fran. By the way, everyone, it was Fran's birthday yesterday, so happy birthday, Fran. Good afternoon, everyone, and thank you for joining Sarepta Therapeutics' fourth quarter 2023 financial results conference call. Led by an exceptional launch of ELEVIDYS and continuing performance of our approved PMOs, EXONDYS 51, VYONDYS 53, and AMONDYS 45, we announced this afternoon another strong quarter of full-year growth and quarterly growth as we serve the patient community. As we pre-announced in January at the J.P. Morgan conference, fourth quarter total net product revenue came in at $365.1 million, growing some 55% over the same quarter prior year. Full-year net product revenue achieved $1.14 billion, growing 36% over the prior year. In addition to continuing strong performance among our three approved therapies, ELEVIDYS' performance was particularly impressive and reflects first-in-class launch excellence.

Douglas S. Ingram: Thank you, Fran. And by the way, everyone, it was Fran's birthday yesterday so, happy birthday Fran. And good afternoon, everyone. Thank you for joining Sarepta Therapeutics 4th quarter 2023 Financial Results Conference Call. Led by an exceptional launch of ELEVIDYS and continuing performance of our approved PMOs, EXONDYS, VYONDYS and AMONDYS.

Douglas S. Ingram: And good afternoon, everyone and thank you for joining throughout the Therapeutics fourth quarter 2023 financial results Conference call.

Douglas S. Ingram: Led by an exceptional launch of <unk> and continuing performance of our approved T. F. L X.

Led by an exceptional launch of ELEVIDYS and continuing performance of our approved PMOs, EXONDYS, VYONDYS and AMONDYS, we announced this afternoon another strong quarter of full year growth and quarterly growth, as we serve the patient community. As we pre-announced in January at the JP Morgan Conference, 4th quarter total net product revenue came in at $365.1 million, growing some 55% over the same quarter, prior year and full year net product revenue achieved $1.14 billion, growing 36% over the prior year. In addition to continuing strong performance among our three approved therapies, ELEVIDYS' performance was particularly impressive and reflects first-in-class launch excellence. Notwithstanding a label limited to four and five-year olds, representing only about 3% or so of the total Duchenne population, ELEVIDYS' net product revenue was $131.2 million for the quarter and over $200 million for the full year. I'm exceptionally proud of the team's performance here, which speaks to our level of preparation and attention to detail, expert understanding of all aspects of launching innovative rare disease therapies and, of course, our passion for bringing a better life to those living with Duchenne.

Douglas S. Ingram: John This is my honest with them on this.

Douglas S. Ingram: We announced this afternoon, another strong quarter of full year growth. And quarterly growth as we serve the patient community as we pre announced in January at the JP Morgan Congress Conference fourth quarter total net product revenue came in at $365 1 million growing 55% over the same quarter prior year. Full year net product revenue achieved 114 billion growing 36% over the prior year. In addition to continuing strong performance among our three approved therapies. However, this performance was particularly impressive. And reflects first in class launch excellence, notwithstanding a label limited to four and five year olds, representing only about 3% or so of the total duchenne population allowed us net product revenue was $131 $2 million for the quarter and over $200 million for the full year.

Douglas S. Ingram: And quarterly growth as we serve the patient community as we pre announced in January at the JP Morgan Congress Conference fourth quarter total net product revenue came in at $365 1 million growing 55% over the same quarter prior year.

Douglas S. Ingram: Full year net product revenue achieved 114 billion growing 36% over the prior year. In addition to continuing strong performance among our three approved therapies. However, this performance was particularly impressive.

In addition to continuing strong performance among our three approved therapies, ELEVIDYS' performance was particularly impressive and reflects first-in-class launch excellence. Notwithstanding a label limited to four and five-year olds, representing only about 3% or so of the total Duchenne population, ELEVIDYS' net product revenue was $131.2 million for the quarter and over $200 million for the full year. I'm exceptionally proud of the team's performance here, which speaks to our level of preparation and attention to detail, expert understanding of all aspects of launching innovative rare disease therapies and, of course, our passion for bringing a better life to those living with Duchenne. Dallan Murray, our Chief Customer Officer, will speak to this in his remarks shortly. We also continue to exercise the discipline of a fully-integrated commercial stage biotech organization.

In addition to continuing strong performance among our three approved therapies, ELEVIDYS' performance was particularly impressive and reflects first-in-class launch excellence. Notwithstanding, a label limited to 4 and 5-year olds, representing only about 3% or so of the total Duchenne population, ELEVIDYS' net product revenue was $131.2 million for the quarter and over $200 million for the full year. I'm exceptionally proud of the team's performance here, which speaks to our level of preparation and attention to detail, expert understanding of all aspects of launching innovative rare disease therapies and, of course, our passion for bringing a better life to those living with Duchenne. Dallan Murray, our Chief Customer Officer, will speak to this in his remarks shortly.

In addition to continuing strong performance among our three approved therapies, ELEVIDYS' performance was particularly impressive and reflects first-in-class launch excellence.

Notwithstanding a label limited to four and five-year olds, representing only about 3% or so of the total Duchenne population, ELEVIDYS' net product revenue was $131.2 million for the quarter and over $200 million for the full year. I'm exceptionally proud of the team's performance here, which speaks to our level of preparation and attention to detail, expert understanding of all aspects of launching innovative rare disease therapies and, of course, our passion for bringing a better life to those living with Duchenne. Dallan Murray, our Chief Customer Officer, will speak to this in his remarks shortly. We also continue to exercise the discipline of a fully-integrated commercial stage biotech organization.

Douglas S. Ingram: And reflects first in class launch excellence, notwithstanding a label limited to four and five year olds, representing only about 3% or so of the total duchenne population allowed us net product revenue was $131 $2 million for the quarter and over $200 million for the full year.

Douglas Ingram: Notwithstanding a label limited to 4- and 5-year-olds representing only about 3% or so of the total Duchenne population, ELEVIDYS' net product revenue was $131.2 million for the quarter and over $200 million for the full year. I'm exceptionally proud of the team's performance here, which speaks to our level of preparation and attention to detail, expert understanding of all aspects of launching innovative rare disease therapies, and of course, our passion for bringing a better life to those living with Duchenne. Dallan Murray, our Chief Customer Officer, will speak to this in his remarks shortly. We also continue to exercise the discipline of a fully integrated commercial-stage biotech organization. We were profitable on a GAAP basis in Q4, having achieved non-GAAP profitability in Q3 2023.

I'm exceptionally proud of the team's performance here, which speaks to our level of preparation and attention to detail, expert understanding of all aspects of launching innovative rare disease therapies and, of course, our passion for bringing a better life to those living with Duchenne. Dallan Murray, our Chief Customer Officer, will speak to this in his remarks shortly.

Douglas S. Ingram: I'm exceptionally proud of the team's performance here, which speaks to our level of preparation and attention to detail. Expert understanding of all aspects of lodging integrated rare disease therapies and of course, our passion for bringing a better life to those living with Duchenne gallon.

Douglas S. Ingram: Expert understanding of all aspects of lodging integrated rare disease therapies and of course, our passion for bringing a better life to those living with Duchenne gallon.

Douglas S. Ingram: Dallan Murray, our Chief Customer Officer, will speak to this in his remarks shortly. We also continue to exercise the discipline of a fully integrated commercial stage biotech organization, we were profitable on a GAAP basis on the floor in the fourth quarter, having achieved non-GAAP profitability in the third quarter of 2023, and we exited 2023 with approximately one $7 billion of cash cash. <unk> restricted cash and investments.

Dallan Murray, our Chief Customer Officer, will speak to this in his remarks shortly.

Dallan Murray: We also continue to exercise the discipline of a fully integrated commercial stage biotech organization, we were profitable on a GAAP basis on the floor in the fourth quarter, having achieved non-GAAP profitability in the third quarter of 2023, and we exited 2023 with approximately one $7 billion of cash cash.

We also continue to exercise the discipline of a fully-integrated commercial stage biotech organization. We were profitable on a GAAP basis in the 4th quarter, having achieved non-GAAP profitability in the 3rd quarter of 2023. And we exited 2023 with approximately $1.7 billion of cash, cash equivalent, restricted cash and investments on our balance sheet. Our CFO, Ian Estepan, will provide more color on financial performance shortly. We also advanced our pipeline in the 4th quarter. In the 4th quarter, we submitted a BLA supplement for ELEVIDYS with the goal of both expanding the label by removing agent ambulation restriction and transitioning our approval from accelerated to traditional. And in February of this year, the FDA accepted our BLA supplement for review and set June 21 as our target review completion date.

We also continue to exercise the discipline of a fully-integrated commercial stage biotech organization.

We were profitable on a GAAP basis in the 4th quarter, having achieved non-GAAP profitability in the 3rd quarter of 2023. And we exited 2023 with approximately $1.7 billion of cash, cash equivalent, restricted cash and investments on our balance sheet. Our CFO, Ian Estepan, will provide more color on financial performance shortly. We also advanced our pipeline in the 4th quarter. In the 4th quarter, we submitted a BLA supplement for ELEVIDYS with the goal of both expanding the label by removing agent ambulation restriction and transitioning our approval from accelerated to traditional. And in February of this year, the FDA accepted our BLA supplement for review and set June 21 as our target review completion date.

Douglas Ingram: We exited 2023 with approximately $1.7 billion of cash, cash equivalents, restricted cash, and investments on our balance sheet. Our CFO, Ian Estepan, will provide more color on financial performance shortly. We also advanced our pipeline in the fourth quarter. In the fourth quarter, we submitted a BLA supplement for ELEVIDYS with the goal of both expanding the label by removing age and ambulation restrictions and transitioning our approval from accelerated to traditional. In February of this year, the FDA accepted our BLA supplement for review and set 21 June as our target review completion date. In the fourth quarter, we also commenced EMERGENE, our trial for SRP-9003 to treat LGMD type 2E.

Dallan Murray: <unk> restricted cash and investments.

Dallan Murray: Our balance sheet.

Dallan Murray: Our CFO Ian asked the pad and we'll provide more color on financial performance shortly.

We also advanced our pipeline in the 4th quarter. In the 4th quarter, we submitted a BLA supplement for ELEVIDYS with the goal of both expanding the label by removing agent ambulation restriction and transitioning our approval from accelerated to traditional. And in February of this year, the FDA accepted our BLA supplement for review and set June 21 as our target review completion date.

Dallan Murray: We also advanced our pipeline in the fourth quarter and the fourth quarter, we submitted a BLA supplement for <unk> with the goal of both expanding the label by removing agent ambulation restriction and transitioning our approval from accelerated to traditional and in February of this year. The FDA accepted our BLA supplement.

And in February of this year, the FDA accepted our BLA supplement for review and set June 21 as our target review completion date. In the fourth quarter, we also commenced EMERGENE, our trial for SRP-9003 to treat LGMD Type 2E. Also in January of this year, we announced the positive results of our trial MOMENTUM Part B, investigating the use of our next generation peptide conjugated PMO, SRP-5051, to treat Duchenne patients that are exon 51 amenable. Dr. Louise Rodino-Klapac, our Head of Research and Development, will provide more color on our pipeline progress shortly.

And in February of this year, the FDA accepted our BLA supplement for review and set June 21 as our target review completion date.

Speaker Change: I'll review.

Speaker Change: June 'twenty, one as our target review completion date.

In the 4th quarter, we also commenced EMERGENE, our trial for SRP-9003 to treat LGMD Type 2E. Also in January of this year, we announced the positive results of our trial MOMENTUM Part B, investigating the use of our next generation peptide conjugated PMO, SRP-5051, to treat Duchenne patients that are exon 51 amenable. Dr. Louise Rodino-Klapac, our Head of Research and Development, will provide more color on our pipeline progress shortly.

Speaker Change: In the fourth quarter, we also commenced EMERGENE, our trial for SRP-9003 to treat LGMD Type 2E. Also in January of this year, we announced the positive results of our trial MOMENTUM Part B, investigating the use of our next generation peptide conjugated PMO, SRP-5051, to treat Duchenne patients that are exon 51 amenable. Dr. Louise Rodino-Klapac, our Head of Research and Development, will provide more color on our pipeline progress shortly. You know, in 2017, starting with one approved therapy, $5 million in sales, a modest pipeline, a short cash runway and little more than ambition and grit -- we set out to build a sustainable, mature biotech organization, improve the lives of the greatest possible number of Duchenne patients along the way and to become the leaders in the use of RNA and gene therapy to treat rare genetic disease. If we are successful in our plans this year, we will have achieved that vision. From there, we can and we will expand our ambition and rely on our scientific and financial strength; we intend to advance our internal pipeline but also to bring in external innovation to grow from here, not incrementally, but in great multiples. In short, 2024 is going to be a very important year

In the fourth quarter, we also commenced EMERGENE, our trial for SRP-9003 to treat LGMD Type 2E. Also in January of this year, we announced the positive results of our trial MOMENTUM Part B, investigating the use of our next generation peptide conjugated PMO, SRP-5051, to treat Duchenne patients that are exon 51 amenable. Dr. Louise Rodino-Klapac, our Head of Research and Development, will provide more color on our pipeline progress shortly.

Douglas Ingram: Also in January of this year, we announced the positive results of our trial MOMENTUM Part B, investigating the use of our next-generation peptide conjugated PMO, SRP-5051, to treat Duchenne patients that are exon 51 amenable. Dr. Louise Rodino-Klapac, our Head of Research and Development, will provide more color on our pipeline progress shortly. You know, in 2017, starting with one approved therapy, $5 million in sales, a modest pipeline, a short cash runway, and little more than ambition and grit, we set out to build a sustainable, mature biotech organization, improve the lives of the greatest possible number of Duchenne patients along the way, and to become the leaders in the use of RNA and gene therapy to treat rare genetic disease. If we are successful in our plans this year, we will have achieved that vision.

Speaker Change: SRP 50, 51 to treat duchenne patients that are exon 51 amenable Dr. Louise Rodino quite back our head of research and development will provide more color on our pipeline progress shortly.

You know, in 2017, starting with one approved therapy, $5 million in sales, a modest pipeline, a short cash runway and little more than ambition and grit -- we set out to build a sustainable, mature biotech organization, improve the lives of the greatest possible number of Duchenne patients along the way and to become the leaders in the use of RNA and gene therapy to treat rare genetic disease. If we are successful in our plans this year, we will have achieved that vision. From there, we can and we will expand our ambition and rely on our scientific and financial strength; we intend to advance our internal pipeline but also to bring in external innovation to grow from here, not incrementally, but in great multiples. In short, 2024 is going to be a very important year.

Speaker Change: You know in 2017, starting with one approved therapy $5 million.

Speaker Change: Sales, a modest pipeline of short cash runway and little more than ambition and grit, we set out to build a sustainable mature biotech organization improve the lives of the greatest possible number of Duchenne patients along the way as it become the leaders in the use of RNA and gene therapy.

Speaker Change: To treat rare genetic diseases. If we are successful in our plan for this year, we will have achieved that vision from there we can and we will expand our ambition and rely on our scientific and financial strength, we intend to advance our internal pipeline, but also to bring in external.

If we are successful in our plans this year, we will have achieved that vision. From there, we can and we will expand our ambition and rely on our scientific and financial strength; we intend to advance our internal pipeline but also to bring in external innovation to grow from here, not incrementally, but in great multiples. In short, 2024 is going to be a very important year.

Douglas Ingram: From there, we can and we will expand our ambition, and relying on our scientific and financial strength, we intend to advance our internal pipeline, but also to bring in external innovation to grow from here, not incrementally, but in great multiples. In short, 2024 is going to be a very important year. With that, I will turn the call to our Chief Customer Officer, Dallan Murray. Dallan?

Speaker Change: Base to grow from here not incrementally, but in great multiples in short 2024 is going to be.

Speaker Change: A very important year and with that I will turn the call to our chief customer officer.

A very important year

And with that, I will turn the call to our Chief Customer Officer, Dallan Murray. Dallan?

Dallan Murray: Alan Murray.

Dallan Murray: Thank you, Doug, and good afternoon. Q4 2023 represented a strong finish to an already impressive year as the team generated over $1 billion in net product revenue, a milestone for Sarepta. As previously noted, net product revenue for 2023 totaled $1.14 billion, consisting of roughly $945 million from our PMO franchise and $200 million from the launch of ELEVIDYS, the first gene therapy approved for patients with Duchenne muscular dystrophy. Each of these accomplishments stands on their own merits, and the performance of both surpassed our internal projections and external consensus. In the seventh year of our PMO franchise, we again grew net product revenue by double digits from the nearly $844 million in net product revenue from 2022. As with previous years, we delivered this growth organically without taking price increases on any of our approved PMO products.

Dallan Murray: Sure. Thank you, Doug and good afternoon. The 4th quarter of 2023 represented a strong finish to an already impressive year, as the team generated over $1 billion in net product revenue.

Dallan Murray: Thank you, Doug and good afternoon.

Dallan Murray: Thank you Doug and good afternoon, the fourth quarter of 2023 represented a strong finish to an already impressive year as the team generated over 1 billion in net product revenue.

Dallan Murray: The 4th quarter of 2023 represented a strong finish to an already impressive year, as the team generated over $1 billion in net product revenue. A milestone for Sarepta. As previously noted, net product revenue for 2023 totaled $1.14 billion, consisting of roughly $945 million from our PMO franchise and $200 million from the launch of ELEVIDYS, the first gene therapy approved for patients with Duchenne muscular dystrophy. Each of these accomplishments stands on their own merits and the performance of both surpassed our internal projections and external consensus.

Dallan Murray: A milestone for us Raptor.

Dallan Murray: As previously noted net product revenue for 2023 totaled 114 billion consisting of roughly $945 million from our PMO franchise.

Dallan Murray: And $200 million from the launch of <unk>. The first gene therapy approved for patients with Duchenne muscular dystrophy.

Dallan Murray: Each of these accomplishments stands on.

Dallan Murray: On their own merits and the performance of both surpassed our internal projections.

Dallan Murray: And external consensus.

Dallan Murray: In the 7th year of our PMO franchise, we again grew net product revenue by double digits from the nearly $844 million in net product revenue from 2022. As with previous years, we delivered this growth organically without taking price increases on any of our approved PMO products. As such, this growth represents an increase in the number of patients we are serving, reflecting our commitment to the Duchenne community.

Dallan Murray: As with previous years, we delivered this growth organically without taking price increases on any of our approved PMA products.

Dallan Murray: As such, this growth represents an increase in the number of patients we are serving, reflecting our commitment to the Duchenne community. Turning to ELEVIDYS, we're extremely pleased with the launch execution, exceeding our own lofty expectations. In fact, the $200 million in net product revenue surpassed the combined 2023 revenue of the other five gene therapy launches from the past 18 months, remarkable given the ELEVIDYS approval occurred just this past summer. The success of ELEVIDYS shows that gene therapy can be commercially viable, providing hope for those patients with Duchenne and for all those with genetic conditions with unmet need. While revenue is how we quantify the success of this launch externally, we measure ourselves on how we support patients. Our preparation was deliberate and intense, and our process was put to the test with a narrow label.

Dallan Murray: As such this growth represents an increase in the number of patients we are serving.

Dallan Murray: Collecting our commitment to the Duchenne community.

Turning to ELEVIDYS, we're extremely pleased with the launch execution, exceeding our own lofty expectations. In fact, the $200 million in net product revenue surpassed the combined 2023 revenue of the other five gene therapy launches from the past 18 months. Remarkable, given the ELEVIDYS approval occurred just this past summer. The success of ELEVIDYS shows that gene therapy can be commercially viable, providing hope for those patients with Duchenne and for all those with genetic conditions with unmet need. While revenue was how we quantify the success of this launch externally, we measure ourselves on how we support patients. Our preparation was deliberate and intense and our process was put to the test with a narrow label.

Dallan Murray: Seeding our own lofty expectations.

Dallan Murray: Factor $200 million in net product revenue surpassed the combined 2023 revenue of the other five gene therapy launches from the past 18 months.

Dallan Murray: Remarkable given the <unk> approval occurred just this past summer.

Dallan Murray: The success of <unk> shows that gene therapy can be commercially viable providing hope for those patients with duchenne and for all those with genetic conditions with unmet need.

While revenue was how we quantify the success of this launch externally, we measure ourselves on how we support patients. Our preparation was deliberate and intense and our process was put to the test with a narrow label. The team responded to that challenge with an incredible commitment to supporting all eligible 4 to 5-year old patients. This was our 4th Duchenne launch and our knowledge and experience played a significant role in how the team rallied, worked together and rapidly supported those patients who are approaching their 6th birthday and who were at risk of becoming ineligible for therapy. Ensuring no eligible patients are left behind is what motivates us.

Dallan Murray: While revenue was how we quantify the success of this launch externally we measure ourselves on how we support patients.

Dallan Murray: Our preparation was deliberate and intense and our process was put to the test with a narrow label.

Dallan Murray: The team responded to that challenge with an incredible commitment to supporting all eligible 4- to 5-year-old patients. This was our fourth Duchenne launch, and our knowledge and experience played a significant role in how the team rallied, worked together, and rapidly supported those patients who were approaching their sixth birthday and who were at risk of becoming ineligible for therapy. Ensuring no eligible patients are left behind is what motivates us. Let's now review the results from the fourth quarter, starting with ELEVIDYS. Net product revenue for the quarter was roughly $131 million. This represented a nearly 90% increase over Q3. We are pleased with our penetration into this very small and narrow segment of the Duchenne population. This 4- to 5-year-old label has presented a number of unique executional challenges that are relevant moving forward.

Dallan Murray: The team responded to that challenge with an incredible commitment to supporting all eligible for a five year old patients. This was our fourth Duchenne launch and our knowledge and experience played a significant role in how the team rallied worked together and rapidly supported those patients who are approaching their sixth birthday, and who were at risk of becoming ineligible for therapy. Ensuring no eligible patients are left behind it is what motivates us.

This was our 4th Duchenne launch and our knowledge and experience played a significant role in how the team rallied, worked together and rapidly supported those patients who are approaching their 6th birthday and who were at risk of becoming ineligible for therapy. Ensuring no eligible patients are left behind is what motivates us.

Dallan Murray: This was our fourth Duchenne launch and our knowledge and experience played a significant role in how the team rallied worked together and rapidly supported those patients who are approaching their sixth birthday, and who were at risk of becoming ineligible for therapy.

Dallan Murray: Ensuring no eligible patients are left behind it is what motivates us.

Dallan Murray: Let's now review the results from the 4th quarter, starting with ELEVIDYS. Net product revenue for the quarter was roughly $131 million. This represented a nearly 90% increase over Q3. We are pleased with our penetration into the very small and narrow segment of the Duchenne population. This four to five-year old label has presented a number of unique executional challenges that are relevant moving forward. Firstly, a significant proportion of the patients in this age group are not yet diagnosed, given that the average age of diagnosis is around five in the United States. Secondly, those who are diagnosed have not had a lot of time to be fully educated about Duchenne, which means the patients and families need to understand that diagnosis, become aware of therapy options, go through a more involved and longer pre-treatment process than the PMOs -- one example of this being antibody testing. And on top of this, the patients must also secure access to ELEVIDYS in this very short time window. Our team has found themselves in a race against time to help these patients. And finally, the relatively small number of diagnosed four to five-year old patients results in a situation in which we will be working through this prevalent population quickly within the first half of the year.

Let's now review the results from the 4th quarter, starting with ELEVIDYS. Net product revenue for the quarter was roughly $131 million. This represented a nearly 90% increase over Q3. We are pleased with our penetration into the very small and narrow segment of the Duchenne population. This 4 to 5-year old label has presented a number of unique executional challenges that are relevant moving forward. Firstly, a significant proportion of the patients in this age group are not yet diagnosed, given that the average age of diagnosis is around 5 in the United States.

Dallan Murray: Net product revenue for the quarter was roughly $131 million. This.

Dallan Murray: Rented and nearly 90% increase over Q3.

Dallan Murray: We are pleased with our penetration into the very small and narrow segment of the Duchenne population.

Dallan Murray: This four to five year old label has presented a number of unique execution challenges that are relevant moving forward.

Dallan Murray: Firstly, a significant proportion of the patients in this age group are not yet diagnosed, given that the average age of diagnosis is around five in the United States. Secondly, those who are diagnosed have not had a lot of time to be fully educated about Duchenne, which means the patients and families need to understand that diagnosis, become aware of therapy options, go through a more involved and longer pretreatment process than the PMOs, one example of this being antibody testing. And on top of this, the patients must also secure access to ELEVIDYS in this very short time window. Our team has found themselves in a race against time to help these patients. And finally, the relatively small number of diagnosed four- to five-year-old patients results in a situation in which we will be working through this prevalent population quickly within the first half of the year.

Dallan Murray: Firstly, a significant proportion of the patients in this age group are not yet diagnosed.

Dallan Murray: Given that the average age of diagnosis is around five in the United States.

Secondly, those who are diagnosed have not had a lot of time to be fully educated about Duchenne, which means the patients and families need to understand that diagnosis, become aware of therapy options, go through a more involved and longer pre-treatment process than the PMOs -- one example of this being antibody testing. And on top of this, the patients must also secure access to ELEVIDYS in this very short time window. Our team has found themselves in a race against time to help these patients. And finally, the relatively small number of diagnosed 4 to 5-year old patients results in a situation in which we will be working through this prevalent population quickly within the first half of the year.

Dallan Murray: Secondly, those who are diagnosed have not had a lot of time to be fully educated about duchenne, which means the patients and families need to understand that diagnosis become aware of therapy options.

Dallan Murray: Go through a more involved in longer pre treatment process in the PMO is one example of this being antibody testing.

And on top of this, the patients must also secure access to ELEVIDYS in this very short time window. Our team has found themselves in a race against time to help these patients. And finally, the relatively small number of diagnosed 4 to 5-year old patients results in a situation in which we will be working through this prevalent population quickly within the first half of the year.

Dallan Murray: And on top of this the patients must also secure access to <unk> in this very short time window.

Dallan Murray: Our team has found themselves in a race against time to help these patients.

Dallan Murray: And finally, the relatively small number of diagnosed four to five year old patients results in a situation in which we will be working through that as prevalent population quickly within the first half of the year. Yeah.

Dallan Murray: On the flip side, many of these dynamics are reversed once patients have transitioned over to the decline phase of the disease. This is illustrated by our real-world experience with the PMOs, where we see a larger proportion treated in the older age groups, which is on top of a larger diagnosed patient pool. Because of all this, we do not expect to see significant additional growth within the existing population through the first half of this year. Notably, however, by the time of label expansion, we expect to have cleared the way for those older patients to get dosed as rapidly as possible upon eligibility. I would caution analysts, therefore, to not use this current younger population as a frame of reference for our market potential in the overall population.

Dallan Murray: Yeah.

Dallan Murray: On the flip side, many of these dynamics are reversed once patients have transitioned over to the decline phase of the disease. This is illustrated by our real world experience with the PMOs, where we see a larger proportion treated in the older age groups. This is on top of a larger diagnosed patient pool. Because of all this, we do not expect to see significant additional growth within the existing population through the first half of this year. Notably, however, by the time of label expansion, we expect to have cleared the way for those older patients to get dosed as rapidly as possible upon eligibility. I would caution analysts, therefore, to not use this current younger population as a frame of reference for our market potential in the overall population. The team is preparing as we speak, for a broad label with a focus on building upon the successful launch execution to date. And we have the access and capacity in place to execute successfully on any broader label scenario.

On the flip side, many of these dynamics are reversed once patients have transitioned over to the decline phase of the disease. This is illustrated by our real world experience with the PMOs, where we see a larger proportion treated in the older age groups. This is on top of a larger diagnosed patient pool. Because of all this, we do not expect to see significant additional growth within the existing population through the first half of this year. Notably, however, by the time of label expansion, we expect to have cleared the way for those older patients to get dosed as rapidly as possible upon eligibility.

Dallan Murray: This is illustrated by our real world experience with the panels, where we see a larger proportion treated in the older age groups.

This is on top of a larger diagnosed patient pool.

Dallan Murray: Because of all this we do not expect to see significant additional growth within the existing population through the first half of this year.

Dallan Murray: Notably however by the time of label expansion, we expect to have cleared the way for those older patients they get dosed as rapidly as possible upon eligibility.

Notably, however, by the time of label expansion, we expect to have cleared the way for those older patients to get dosed as rapidly as possible upon eligibility. I would caution analysts, therefore, to not use this current younger population as a frame of reference for our market potential in the overall population. The team is preparing as we speak, for a broad label with a focus on building upon the successful launch execution to date. And we have the access and capacity in place to execute successfully on any broader label scenario.

Dallan Murray: I would caution analyst airport to not use this current younger population as a frame of reference for our market potential in the overall population.

I would caution analysts, therefore, to not use this current younger population as a frame of reference for our market potential in the overall population. The team is preparing as we speak, for a broad label with a focus on building upon the successful launch execution to date. And we have the access and capacity in place to execute successfully on any broader label scenario.

Dallan Murray: The team is preparing, as we speak, for a broad label with a focus on building upon the successful launch execution to date. We have the access and capacity in place to execute successfully on any broader label scenario. Let's now take a look at the PMO franchise as a whole. As previously mentioned, 2023 net product revenue of $945 million exceeded our full-year guidance of $925 million. This performance represents solid revenue growth across all three brands. In fact, both VYONDYS 53 and AMONDYS 45 continued their double-digit growth trajectory. Looking now at the fourth quarter of 2023, the team delivered roughly $234 million in net product revenue. This was flat versus Q4 of 2022, net product revenue of roughly $236 million.

Dallan Murray: The team is preparing as we speak for a broad label with a focus on building upon the successful launch execution to date and we have the access and capacity in place to execute successfully on any broader label scenario.

Dallan Murray: Let's now take a look at the PMO franchise as a whole. As previously mentioned, 2023 net product revenue of $945 million exceeded our full year guidance of $925 million. This performance represents solid revenue growth across all three brands. In fact, both by VYONDYS 53, and AMONDYS 45 continued their double digit growth trajectory. Looking now at the 4th quarter of 2023, the team delivered roughly $234 million in net product revenue. This was flat versus Q4 of 2022, net product revenue of roughly $236 million. As you may recall from our Q4 2022 earnings call, we cautioned around keeping the guidance of $925 million for the year due to an increase in the quarter-to-quarter lumpiness that we were observing at that time. Looking now in retrospect, it's clear, the PMOs performed exactly as we expected and guided.

Let's now take a look at the PMO franchise as a whole. As previously mentioned, 2023 net product revenue of $945 million exceeded our full year guidance of $925 million. This performance represents solid revenue growth across all three brands. In fact, both by VYONDYS 53, and AMONDYS 45 continued their double digit growth trajectory. Looking now at the 4th quarter of 2023, the team delivered roughly $234 million in net product revenue.

Dallan Murray: This performance represents solid revenue growth across all three brands in.

Dallan Murray: In fact, both by honest 53, and a modest 45 continued their double digit growth trajectory.

Dallan Murray: Looking now at the fourth quarter of 2023, the team delivered roughly $234 million and net product revenue.

This was flat versus Q4 of 2022, net product revenue of roughly $236 million. As you may recall from our Q4 2022 earnings call, we cautioned around keeping the guidance of $925 million for the year due to an increase in the quarter-to-quarter lumpiness that we were observing at that time. Looking now in retrospect, it's clear, the PMOs performed exactly as we expected and guided.

Dallan Murray: This was flat versus Q4 of 2022 net product revenue of roughly $236 million.

Dallan Murray: As you may recall from our Q4 2022 earnings call, we cautioned around keeping the guidance of $925 million for the year due to an increase in the quarter-to-quarter lumpiness that we were observing at that time. Looking now in retrospect, it's clear the PMOs performed exactly as we expected and guided. And finally, the performance we just discussed in our PMO business was achieved despite the incredible effort on the ELEVIDYS launch. Notably, we saw minimal impact on our PMO business from ELEVIDYS cannibalization in 2023, given the narrow age range. In closing in 2023, Sarepta set a new standard for gene therapy launches with ELEVIDYS. We beat external expectations and delivered roughly $200 million. Equally important, we continued to increase the number of patients we support with our PMOs globally. I continue to be immensely proud of our mission-driven team.

As you May recall from our Q4 2022 earnings call.

Dallan Murray: We cautioned around keeping the guidance of $925 million for the year due to an increase in the quarter to quarter Lumpiness that we were observing at that time.

Dallan Murray: Looking now in retrospect, it's clear the PMO has performed exactly as we expected and guided.

Dallan Murray: And finally, the performance we just discussed in our PMO business was achieved despite the incredible effort on the ELEVIDYS launch. Notably, we saw minimal impact on our PMO business from ELEVIDYS cannibalization in 2023, given the narrow age range. In closing, in 2023, Sarepta set a new standard for gene therapy launches with ELEVIDYS. We beat external expectations and delivered roughly $200 million. Equally important, we continued to increase the number of patients we support with our PMOs globally. I continue to be immensely proud of our mission-driven team. Combining our PMO and gene therapy businesses, we exceeded $1 billion in net product revenue for the first time and we have entered 2024 with momentum.

And finally, the performance we just discussed in our PMO business was achieved despite the incredible effort on the ELEVIDYS launch. Notably, we saw minimal impact on our PMO business from ELEVIDYS cannibalization in 2023, given the narrow age range. In closing, in 2023, Sarepta set a new standard for gene therapy launches with ELEVIDYS. We beat external expectations and delivered roughly $200 million.

Dallan Murray: Notably we saw minimal impact on our PMO business from 11 as cannibalization in 2023, given the narrow age range.

Dallan Murray: In closing in 2023, so raptor set a new standard for gene therapy launches with <unk>.

Dallan Murray: We beat external expectations and delivered roughly $200 million.

Equally important, we continued to increase the number of patients we support with our PMOs globally. I continue to be immensely proud of our mission-driven team. Combining our PMO and gene therapy businesses, we exceeded $1 billion in net product revenue for the first time and we have entered 2024 with momentum.

Dallan Murray: Equally important we continued to increase the number of patients we support with our PMO globally.

Dallan Murray: I continue to be immensely proud of our mission driven team.

Dallan Murray: Combining our PMO and gene therapy businesses, we exceeded $1 billion in net product revenue for the first time, and we have entered 2024 with momentum. With that, let me turn the call over to our Head of R&D and Chief Scientific Officer, Dr. Louise Rodino-Klapac. Louise.

Dallan Murray: Combining our PMO and gene therapy businesses, we exceeded 1 billion in net product revenue for the first time.

Dallan Murray: And we have entered 2024 with momentum.

Speaker Change: And with that let me turn the call over to our Head of R&D and Chief Scientific Officer, Dr. Louise Rodino-Klapac. Louise?

Louise Rodino-Klapac (Sarepta Therapeu: Thanks, Dallan. 2023 was a year of great accomplishment for Sarepta, for the advancement of science, and for the health and well-being of patients living with rare disease. 2023 will also be remembered as being a defining moment in genetic medicine. In June 2023, the FDA granted accelerated approval to ELEVIDYS, first gene therapy to treat Duchenne muscular dystrophy. Since that time, we've been successfully treating ambulatory pediatric patients aged four through five years with Duchenne who have a confirmed mutation in the DMD gene. And then, just about two weeks ago, and as Doug mentioned, we were thrilled to announce that the FDA accepted and filed our efficacy supplement for ELEVIDYS, whereby they will now evaluate broadening the approved indication of ELEVIDYS by removing age and ambulation restrictions and converting the ELEVIDYS accelerated approval to a traditional approval.

Louise Rodino-Klapac: Thanks, Dallan. 2023 was a year of great accomplishment for Sarepta, for the advancement of science, and for the health and well-being of patients living with rare disease. 2023 will also be remembered as being a defining moment in genetic medicine. In June 2023, the FDA granted accelerated approval to ELEVIDYS, first gene therapy to treat Duchenne muscular dystrophy. Since that time, we've been successfully treating ambulatory pediatric patients aged four through five years with Duchenne who have a confirmed mutation in the DMD gene. And then, just about two weeks ago, and as Doug mentioned, we were thrilled to announce that the FDA accepted and filed our efficacy supplement for ELEVIDYS, whereby they will now evaluate broadening the approved indication of ELEVIDYS by removing age and ambulation restrictions and converting the ELEVIDYS accelerated approval to a traditional approval.

Louise R. Rodino: Thanks, Dallan. 2023 was a year of great accomplishment for Sarepta, for the advancement of science and for the health and well-being of patients living with rare disease. 2023 will also be remembered as being a defining moment in genetic medicine. In June 2023, the FDA granted accelerated approval to ELEVIDYS, first gene therapy to treat Duchenne muscular dystrophy. Since that time, we've been successfully treating ambulatory pediatric patients aged four through five years with Duchenne, who have a confirmed mutation in the DMD gene. And then just about two weeks ago, and as Doug mentioned, we were thrilled to announce that the FDA accepted and filed our efficacy supplement for ELEVIDYS, whereby they will now evaluate broadening the approved indication of ELEVIDYS by removing age and ambulation restrictions and converting the ELEVIDYS accelerated approval to a traditional approval. Should we receive accelerated approval for ELEVIDYS IN the non-ambulant population in the United States, our ENVISION study, also called SRP-9001-303, will serve as our confirmatory study for this population.

Louise Rodino: Thanks, Dallan. 2023 was a year of great accomplishment for Sarepta, for the advancement of science and for the health and well-being of patients living with rare disease. 2023 will also be remembered as being a defining moment in genetic medicine. In June 2023, the FDA granted accelerated approval to ELEVIDYS, the first gene therapy to treat Duchenne muscular dystrophy. Since that time, we've been successfully treating ambulatory pediatric patients aged 4 through 5 years with Duchenne, who have a confirmed mutation in the DMD gene.

Louise R. Rodino: 23, with a year of great accomplishment for Raptor. <unk> spine and for the health and well being of patients living with rare disease. Funny 'twenty three will also be remembered as being a defining moment in genetic medicine. In June 2023, the FDA granted accelerated approval to eliminate first gene therapy to treat this and muscular dystrophy. At that time, we have been successfully trading ambulatory pediatric patients aged four or five years with duchenne. Firmest mutation in the D M D G. And then just about two weeks ago and as Doug mentioned, we were thrilled to announce that the FDA accepted and filed our efficacy supplement for a loving it. Whereby they will now evaluate broadening the approved indication of <unk>. Removing age and ambulation restrictions. Neil that's accelerated approval to a traditional approval. Should we receive accelerated approval for a limited and the non <unk> population in the United States. Our envision study also called SRP nine zero. There are one three or three plus surfers our confirmatory study for this population.

Louise R. Rodino: <unk> spine and for the health and well being of patients living with rare disease.

Speaker Change: Funny 'twenty three will also be remembered as being a defining moment in genetic medicine.

Speaker Change: In June 2023, the FDA granted accelerated approval to eliminate first gene therapy to treat this and muscular dystrophy.

Speaker Change: At that time, we have been successfully trading ambulatory pediatric patients aged four or five years with duchenne.

Speaker Change: Firmest mutation in the D M D G.

And then just about two weeks ago, and as Doug mentioned, we were thrilled to announce that the FDA accepted and filed our efficacy supplement for ELEVIDYS; whereby they will now evaluate broadening the approved indication of ELEVIDYS by removing age and ambulation restrictions and converting the ELEVIDYS accelerated approval to a traditional approval. Should we receive accelerated approval for ELEVIDYS IN the non-ambulant population in the United States, our ENVISION study, also called SRP-9001-303, will serve as our confirmatory study for this population.

Speaker Change: And then just about two weeks ago and as Doug mentioned, we were thrilled to announce that the FDA accepted and filed our efficacy supplement for a loving it.

Speaker Change: Whereby they will now evaluate broadening the approved indication of <unk>.

Speaker Change: Removing age and ambulation restrictions.

Speaker Change: Neil that's accelerated approval to a traditional approval.

Louise Rodino-Klapac (Sarepta Therapeu: Should we receive accelerated approval for ELEVIDYS in the non-ambulatory population in the United States, our ENVISION study, also called SRP-9001-303, will serve as our confirmatory study for this population. ENVISION is a global, randomized, double-blind, placebo-controlled, two-part study evaluating the safety and efficacy of delandistrogene moxeparvovec gene therapy in non-ambulatory, and older ambulatory individuals with Duchenne. This study is ongoing, with all remaining patients being enrolled outside of the United States. With U.S. enrollment completed and the remaining 85% of recruitment occurring ex-U.S., we are confident in our ability to complete this trial. Moving now to our limb-girdle muscular dystrophy, or LGMD, programs. On 16 January, we announced that screening was underway in study SRP-9003-301, also known as the EMERGENE study. We are pleased to now share that the first patient has been successfully dosed in that study.

Louise Rodino-Klapac: Should we receive accelerated approval for ELEVIDYS in the non-ambulatory population in the United States, our ENVISION study, also called SRP-9001-303, will serve as our confirmatory study for this population. ENVISION is a global, randomized, double-blind, placebo-controlled, two-part study evaluating the safety and efficacy of delandistrogene moxeparvovec gene therapy in non-ambulatory, and older ambulatory individuals with Duchenne. This study is ongoing, with all remaining patients being enrolled outside of the United States. With U.S. enrollment completed and the remaining 85% of recruitment occurring ex-U.S., we are confident in our ability to complete this trial. Moving now to our limb-girdle muscular dystrophy, or LGMD, programs. On 16 January, we announced that screening was underway in study SRP-9003-301, also known as the EMERGENE study. We are pleased to now share that the first patient has been successfully dosed in that study.

Speaker Change: Should we receive accelerated approval for a limited and the non <unk> population in the United States. Our envision study also called SRP nine zero. There are one three or three plus surfers our confirmatory study for this population.

Speaker Change: ENVISION is a global, randomized, double-blind, placebo-controlled, two-part study evaluating the safety and efficacy of delandistrogene moxeparvovec-rokl gene therapy in non-ambulatory and older ambulatory individuals with Duchenne. This study is ongoing with all remaining patients being enrolled outside of the United States. With the U.S. enrollment completed and the remaining 85% of recruitment occurring ex-U.S., we are confident in our ability to complete this trial.

This study is ongoing with all remaining patient being enrolled outside of the United States.

Speaker Change: With the U S enrollment completed and the remaining 85% of recruitment occurring ex U S.

Speaker Change: We're confident in our ability to complete this trial.

Moving now to our limb-girdle muscular dystrophy or LGMD program. On January 16th, we announced that screening was underway in Study SRP-9003-301, also known as the EMERGENE study. We're pleased to now share that the first patient has been successfully dosed in that study. To remind you, EMERGENE is a Phase III, multinational, open-label clinical trial of SRP-9003 for the treatment of limb-girdle muscular dystrophy type 2E or beta-sarcoglycanopathy. The primary endpoint of EMERGENE is expression of beta-sarcoglycan, which is an extremely important endpoint for this program, for the other sarcoglycanopathies, including LGMD2D and LGMD2C, and for the field of gene therapy. I'll explain why.

Speaker Change: On January 16th we announced that screening was underway and steady SRP 900 grade 301 also known as the emerging study were placed in Austria that the first patient has been successfully dose in that study.

Louise Rodino-Klapac (Sarepta Therapeu: To remind you, EMERGENE is a phase 3 multinational open-label clinical trial of SRP-9003 for the treatment of limb-girdle muscular dystrophy type 2E, or beta-sarcoglycanopathy. The primary endpoint of EMERGENE is expression of beta-sarcoglycan, which is an extremely important endpoint for this program, for the other sarcoglycanopathies, including LGMD2D and LGMD2C, and for the field of gene therapy. I'll explain why. Beta-sarcoglycanopathy is characterized by a mutation of the beta-sarcoglycan gene, which sits in a complex of the membrane called the sarcoglycan complex and is important for function and for preventing muscle damage during contraction. The sarcoglycan complex is a subcomplex of the dystrophin-associated protein complex, or DAPC. A defective sarcoglycan protein results in loss or reduced expression of the other sarcoglycans, as well as other proteins in the complex, such as dystrophin.

Louise Rodino-Klapac: To remind you, EMERGENE is a phase 3 multinational open-label clinical trial of SRP-9003 for the treatment of limb-girdle muscular dystrophy type 2E, or beta-sarcoglycanopathy. The primary endpoint of EMERGENE is expression of beta-sarcoglycan, which is an extremely important endpoint for this program, for the other sarcoglycanopathies, including LGMD2D and LGMD2C, and for the field of gene therapy. I'll explain why. Beta-sarcoglycanopathy is characterized by a mutation of the beta-sarcoglycan gene, which sits in a complex of the membrane called the sarcoglycan complex and is important for function and for preventing muscle damage during contraction. The sarcoglycan complex is a subcomplex of the dystrophin-associated protein complex, or DAPC. A defective sarcoglycan protein results in loss or reduced expression of the other sarcoglycans, as well as other proteins in the complex, such as dystrophin.

Speaker Change: To remind you emerging as a phase III multinational open label clinical trial of SRP 9000 growth rate for the treatment of limb girdle muscular dystrophy type two E or beta cyclical iconography.

Speaker Change: The primary endpoint of emerging expression of beta stuck like N, which is an extremely important end point for this program.

Speaker Change: For the other sarcoglycanopathies, including LGMD2D and LGMD2C and for the field of gene therapy. I'll explain why. Data circuits like an afterthought as characterized by mutation of the beta start with like Engie, which sits in a complex at the membrane called the cyclical I can complex. And it's important for function and for preventing muscle damage during contraction. The second question complex as a sub complex of the dystrophin associated protein complex printouts fee. Factors circled back and protein results in la a reduced expression of the other cyclical like them as well as other proteins in the complex such as disruptive.

For the other sarcoglycanopathies, including LGMD2D and LGMD2C and for the field of gene therapy. I'll explain why.

Beta-sarcoglycanopathy is characterized by a mutation of the beta-sarcoglycan gene, which sits in a complex at the membrane called the sarcoglycan complex and is important for function and for preventing muscle damage during contraction. The sarcoglycan complex as a sub-complex of the dystrophin-associated protein complex or DAPC. A defective sarcoglycan protein results in loss or reduced expression of the other sarcoglycan, as well as other proteins in the complex, such as dystrophin. Therefore, by restoring the missing proteins, such as beta-sarcoglycan, we were able to restore that functional complex at the membrane and thereby restore function to the muscle. Further, earlier this month, I had the opportunity to participate in the Speak Foundation's LGMD Scientific Workshop, which also featured officials from FDA, including Drs. Marks and Verdun as well as patients, caregivers and clinicians, among others. The key takeaways from the workshop included the perspective that traditional trial designs are not suitable for certain types of LGMD and that to ensure these therapies has the best chance of success, the totality of evidence must be considered.

Speaker Change: Explain why.

Speaker Change: Data circuits like an afterthought as characterized by mutation of the beta start with like Engie, which sits in a complex at the membrane called the cyclical I can complex.

Speaker Change: And it's important for function and for preventing muscle damage during contraction.

Speaker Change: The second question complex as a sub complex of the dystrophin associated protein complex printouts fee.

A defective sarcoglycan protein results in loss or reduced expression of the other sarcoglycan, as well as other proteins in the complex, such as dystrophin. Therefore, by restoring the missing proteins, such as beta-sarcoglycan, we were able to restore that functional complex at the membrane and thereby restore function to the muscle.

Speaker Change: Factors circled back and protein results in la a reduced expression of the other cyclical like them as well as other proteins in the complex such as disruptive.

Louise Rodino-Klapac (Sarepta Therapeu: Therefore, by restoring the missing protein, such as beta-sarcoglycan, you are able to restore that functional complex at the membrane and thereby restore function to the muscle. Further, earlier this month, I had the opportunity to participate in the SPEAK Foundation's LGMD scientific workshop, which also featured officials from FDA, including Dr. Marks and Verdun, as well as patients, caregivers, and clinicians, among others. The key takeaways from the workshop included the perspective that traditional trial designs are not suitable for certain types of LGMD, and that to ensure these therapies have the best chance of success, the totality of evidence must be considered. Dr. Marks and Verdun also expressed their strong support for regulatory flexibility and a higher tolerance for uncertainty for rare diseases such as LGMD when you're replacing the native protein, as well as support for surrogate endpoints for gene therapies.

Louise Rodino-Klapac: Therefore, by restoring the missing protein, such as beta-sarcoglycan, you are able to restore that functional complex at the membrane and thereby restore function to the muscle. Further, earlier this month, I had the opportunity to participate in the SPEAK Foundation's LGMD scientific workshop, which also featured officials from FDA, including Dr. Marks and Verdun, as well as patients, caregivers, and clinicians, among others. The key takeaways from the workshop included the perspective that traditional trial designs are not suitable for certain types of LGMD, and that to ensure these therapies have the best chance of success, the totality of evidence must be considered. Dr. Marks and Verdun also expressed their strong support for regulatory flexibility and a higher tolerance for uncertainty for rare diseases such as LGMD when you're replacing the native protein, as well as support for surrogate endpoints for gene therapies.

Speaker Change: Therefore by restoring the missing proteins, such as beta cyclical I can they were able to restore that functional complex at the membrane and thereby restoring function to the muscle. Further earlier this month I had the opportunity to participate and speak Foundation L. G. M. D. Scientific workshop, which also featured officials from SBA, including doctors marks and for done well patients caregivers and clinicians among others. The key takeaways from the workshop and put into perspective that traditional trial designs are not suitable for certain types of LTE M D. And that's to ensure these therapies has the best chance of success the totality of evidence must be considered.

Further, earlier this month, I had the opportunity to participate in the Speak Foundation's LGMD Scientific Workshop, which also featured officials from FDA, including Drs. Marks and Verdun as well as patients, caregivers and clinicians, among others. The key takeaways from the workshop included the perspective that traditional trial designs are not suitable for certain types of LGMD and that to ensure these therapies has the best chance of success, the totality of evidence must be considered. Drs. Marks and Verdun also expressed their strong support for regulatory flexibility and a higher tolerance for uncertainty for rare diseases, such as LGMD, when you're replacing the native protein as well as support for surrogate endpoints for gene therapies. Currently, no treatments exist to effectively treat LGMD2E or the other LGMDs. The EMERGENE study -- which will enroll 15 participants who are ambulatory and non-ambulatory, ages four and older -- not only holds great promise for individuals suffering from LGMD2E but will lay the foundation for other LGMD programs, as well as provide a viable regulatory pathway that supports the development of future gene therapies for rare and ultra-rare diseases.

Speaker Change: Further earlier this month I had the opportunity to participate and speak Foundation L. G. M. D. Scientific workshop, which also featured officials from SBA, including doctors marks and for done well patients caregivers and clinicians among others.

Speaker Change: The key takeaways from the workshop and put into perspective that traditional trial designs are not suitable for certain types of LTE M D.

Speaker Change: And that's to ensure these therapies has the best chance of success the totality of evidence must be considered.

Speaker Change: That goes market's overdone also expressed their strong support for regulatory flexibility and a higher tolerance for uncertainty for rare diseases, such as LG M D. When you're replacing the native protein as well as support for surrogate endpoints for gene therapies. Currently no treatments exist to effectively treat L. J M D to E or the other L. G M D. The emerging study, which will enroll 15 participants who are ambulatory and nonambulatory. For an older not only holds great promise for individuals suffering from LG M D to eat but will lay the foundation for other LTE M. D programs as well as provide a viable regulatory pathway that supports the development of future gene therapies for rare and ultra rare diseases.

Louise Rodino-Klapac (Sarepta Therapeu: Currently, no treatments exist to effectively treat LGMD2E or the other LGMDs. The EMERGENE study, which will enroll 15 participants who are ambulatory and non-ambulatory, ages four and older, not only holds great promise for individuals suffering from LGMD2E, but will lay the foundation for our other LGMD programs, as well as provide a viable regulatory pathway that supports the development of future gene therapies for rare and ultra-rare diseases. These data, combined with positive expression and functional data shared from our initial LGMD2E study, SRP-9003-101, which was also published in Nature Medicine earlier this year, and our VOYAGENE study, SRP-9003-102, which establishes safety experience across a broader patient population, serve together as totality of evidence.

Louise Rodino-Klapac: Currently, no treatments exist to effectively treat LGMD2E or the other LGMDs. The EMERGENE study, which will enroll 15 participants who are ambulatory and non-ambulatory, ages four and older, not only holds great promise for individuals suffering from LGMD2E, but will lay the foundation for our other LGMD programs, as well as provide a viable regulatory pathway that supports the development of future gene therapies for rare and ultra-rare diseases. These data, combined with positive expression and functional data shared from our initial LGMD2E study, SRP-9003-101, which was also published in Nature Medicine earlier this year, and our VOYAGENE study, SRP-9003-102, which establishes safety experience across a broader patient population, serve together as totality of evidence.

Currently, no treatments exist to effectively treat LGMD2E or the other LGMDs. The EMERGENE study -- which will enroll 15 participants who are ambulatory and non-ambulatory, ages four and older -- not only holds great promise for individuals suffering from LGMD2E but will lay the foundation for other LGMD programs, as well as provide a viable regulatory pathway that supports the development of future gene therapies for rare and ultra-rare diseases. These data, combined with positive expression and functional data shared from our initial LGMD2E Study SRP-9003-101 -- which is also published in Nature Medicine earlier this year -- and our VOYAGENE Study SRP-9003-102, which establishes safety experience across a broader patient population, paired together is totality of evidence. As a reminder, VOYGENE is a Phase I study evaluating SRP-9003 for the treatment of LGMD2E in patients ages 18 and older in the ambulant population and ages 4 to 50 in the non-ambulant population. The primary endpoints are safety and change in beta-sarcoglycan expression. We expect to have the clinical results this year.

Currently no treatments exist to effectively treat L. J M D to E or the other L. G M D.

Speaker Change: The emerging study, which will enroll 15 participants who are ambulatory and nonambulatory.

Speaker Change: For an older not only holds great promise for individuals suffering from LG M D to eat but will lay the foundation for other LTE M. D programs as well as provide a viable regulatory pathway that supports the development of future gene therapies for rare and ultra rare diseases.

Speaker Change: These data combined with positive expression and functional data shared from our initial L. G. M D to E study. <unk> 003, 101, which is also published in nature Medicine earlier this year. Our voyage study SRP 90031 or two. Establish a safety experience across a broader patient population. Sir together is totality of evidence.

These data, combined with positive expression and functional data shared from our initial LGMD2E Study SRP-9003-101 -- which is also published in Nature Medicine earlier this year -- and our VOYAGENE Study SRP-9003-102, which establishes safety experience across a broader patient population, paired together is totality of evidence. As a reminder, VOYGENE is a Phase I study evaluating SRP-9003 for the treatment of LGMD2E in patients ages 18 and older in the ambulant population and ages 4 to 50 in the non-ambulant population. The primary endpoints are safety and change in beta-sarcoglycan expression. We expect to have the clinical results this year.

<unk> 003, 101, which is also published in nature Medicine earlier this year.

Speaker Change: Our voyage study SRP 90031 or two.

Speaker Change: Establish a safety experience across a broader patient population.

Speaker Change: Sir together is totality of evidence.

Louise Rodino-Klapac (Sarepta Therapeu: As a reminder, VOYAGENE is a phase 1 study evaluating SRP-9003 for the treatment of LGMD2E in patients ages 18 and older in the ambulant population and ages 4 to 50 in the non-ambulant population. The primary endpoints are safety and change in beta-sarcoglycan expression. We expect to have the clinical results this year. Moving now to our RNA platform. We were also pleased to recently announce positive results from Part B of our MOMENTUM study, study SRP-5051-201. Based on the data we've generated to date, we believe SRP-5051 represents a best-in-class therapy from an efficacy perspective. MOMENTUM is a global multi-ascending dose clinical trial of SRP-5051. Our next-generation peptide phosphorodiamidate morpholino oligomer treatment for patients with Duchenne who are amenable to exon 51 skipping.

Louise Rodino-Klapac: As a reminder, VOYAGENE is a phase 1 study evaluating SRP-9003 for the treatment of LGMD2E in patients ages 18 and older in the ambulant population and ages 4 to 50 in the non-ambulant population. The primary endpoints are safety and change in beta-sarcoglycan expression. We expect to have the clinical results this year. Moving now to our RNA platform. We were also pleased to recently announce positive results from Part B of our MOMENTUM study, study SRP-5051-201. Based on the data we've generated to date, we believe SRP-5051 represents a best-in-class therapy from an efficacy perspective. MOMENTUM is a global multi-ascending dose clinical trial of SRP-5051. Our next-generation peptide phosphorodiamidate morpholino oligomer treatment for patients with Duchenne who are amenable to exon 51 skipping.

Speaker Change: As a reminder, Voyager is a phase one study evaluating <unk> 9003 for the treatment of LTE M D to E and patients ages 18, and older and the ambulant population and it is four to 50 and the non ambulant population. The primary endpoints are safety and change in beta cycles like an expression, we expect to have the clinical results. This year.

Speaker Change: The primary endpoints are safety and change in beta cycles like an expression, we expect to have the clinical results. This year.

Speaker Change: Moving now to our RNA platform. We were also pleased to recently announce positive results from Part B of our MOMENTUM study, the SRP-5051-201. Based on the data we've generated to date, we believe SRP-5051 represents a best-in-class therapy from an efficacy perspective. MOMENTUM is a global, multi-ascending dose clinical trial of SRP-5051, our next-generation peptide phosphorodiamidate morpholino oligomer treatment for patients with Duchenne who are amenable to exon 51 skipping. As previously discussed, and based on these results, we believe we have a path for it to an NDA and are planning a meeting with FDA to discuss an accelerated approval. We anticipate that this meeting will occur in the 3rd quarter of 2024. Regarding our post-marketing studies for the PMOs, as mentioned, we completed enrollment in the ESSENCE trial, our post-marketing requirement for GOLODIRSEN and CASIMERSEN.

Moving now to our RNA platform. We were also pleased to recently announce positive results from Part B of our MOMENTUM study, the SRP-5051-201. Based on the data we've generated to date, we believe SRP-5051 represents a best-in-class therapy from an efficacy perspective. MOMENTUM is a global, multi-ascending dose clinical trial of SRP-5051, our next-generation peptide phosphorodiamidate morpholino oligomer treatment for patients with Duchenne who are amenable to exon 51 skipping.

Speaker Change: We were also pleased to recently announced positive results from part B of a momentum of study.

Speaker Change: The SRP 50 51 201.

Speaker Change: Based on the data we've generated to date, we believe SRP 50, 51 represents a best in class therapy from an efficacy perspective.

Speaker Change: Momentum is a global multi ascending dose clinical trial of SRP 50 51.

Speaker Change: Next generation peptide Phosphorothioate marshalling all all of them are treatment for patients with Duchenne, we are amenable to exon 51 skipping.

As previously discussed, and based on these results, we believe we have a path for it to an NDA and are planning a meeting with FDA to discuss an accelerated approval. We anticipate that this meeting will occur in the 3rd quarter of 2024. Regarding our post-marketing studies for the PMOs, as mentioned, we completed enrollment in the ESSENCE trial, our post-marketing requirement for GOLODIRSEN and CASIMERSEN.

Louise Rodino-Klapac (Sarepta Therapeu: As previously discussed, and based on these results, we believe we have a path forward to an NDA and are planning a meeting with FDA to discuss an accelerated approval. We anticipate that this meeting will occur in Q3 2024. Regarding our post-marketing studies for the PMOs, as mentioned, we completed enrollment in the ESSENCE trial, our post-marketing requirement for golodirsen and casimersen. As a reminder, ESSENCE is a 2-year study and is due to read out in early 2026. In addition, we are pleased to have completed enrollment in our MISSION study, our dose-ranging post-marketing commitment for EXONDYS 51. MISSION is a randomized double-blind safety and efficacy dose-finding study comparing the approved dosage of eteplirsen, 30 mg/kg weekly, to a dosage that provides significantly higher exposure, up to 200 mg/kg weekly. MISSION is a 2-part Phase 3 study.

Louise Rodino-Klapac: As previously discussed, and based on these results, we believe we have a path forward to an NDA and are planning a meeting with FDA to discuss an accelerated approval. We anticipate that this meeting will occur in Q3 2024. Regarding our post-marketing studies for the PMOs, as mentioned, we completed enrollment in the ESSENCE trial, our post-marketing requirement for golodirsen and casimersen. As a reminder, ESSENCE is a 2-year study and is due to read out in early 2026. In addition, we are pleased to have completed enrollment in our MISSION study, our dose-ranging post-marketing commitment for EXONDYS 51. MISSION is a randomized double-blind safety and efficacy dose-finding study comparing the approved dosage of eteplirsen, 30 mg/kg weekly, to a dosage that provides significantly higher exposure, up to 200 mg/kg weekly. MISSION is a 2-part Phase 3 study.

Speaker Change: As previously discussed and based on these results. We believe we have a path for it to an NDA.

Speaker Change: Planning a meeting with FDA to discuss an accelerated approval.

Speaker Change: Anticipate that this meeting will occur in the third quarter of 2024.

Speaker Change: Regarding our post marketing studies for the PMO as mentioned, we completed enrolment in the essence trial, our post marketing requirement for Golar garrison and CASM Carsten <unk>.

Speaker Change: As a reminder, ESSENCE is a two-year study and is due to read out in early 2026. In addition, we are pleased to complete the enrollment in our MIS51ON study, our dose-ranging, post-marketing commitment for EXONDYS. MIS51ON is a randomized, double-blind, safety and efficacy, dose-finding study comparing the approved dosage of ETEPLIRSEN, 30mg/kg weekly, to a dosage that provides significantly higher exposure, up to 200mg/kg weekly. MIS51ON is a two-part, Phase III study. It was fully enrolled in October 2023 with 160 patients. We remain committed to rapidly and diligently advancing MIS51ON and sharing data as soon as it becomes available. In conclusion, the months ahead are filled with great promise to advance our mission and serve patients around the world living with rare disease.

As a reminder, ESSENCE is a two-year study and is due to read out in early 2026. In addition, we are pleased to complete the enrollment in our MIS51ON study, our dose-ranging, post-marketing commitment for EXONDYS. MIS51ON is a randomized, double-blind, safety and efficacy, dose-finding study comparing the approved dosage of ETEPLIRSEN, 30mg/kg weekly, to a dosage that provides significantly higher exposure, up to 200mg/kg weekly.

Speaker Change: In addition, airplanes just completed enrollment in our mission study our dose ranging post marketing commitment for example.

Speaker Change: Michigan is a randomized double blind safety and efficacy dose finding study comparing the approved dosage of a couple of cents 13th extra can't quickly.

Speaker Change: Dosage that provides significantly higher exposure up to 200, Meg, particularly clear.

MIS51ON is a two-part, Phase III study. It was fully enrolled in October 2023 with 160 patients. We remain committed to rapidly and diligently advancing MIS51ON and sharing data as soon as it becomes available. In conclusion, the months ahead are filled with great promise to advance our mission and serve patients around the world living with rare disease.

Speaker Change: It's a two part phase III study it was fully enrolled in October 2023, with 160 patients.

Louise Rodino-Klapac (Sarepta Therapeu: It was fully enrolled in October 2023 with 160 patients. We remain committed to rapidly and diligently advancing Mission and sharing data as soon as it becomes available. In conclusion, the months ahead are filled with great promise to advance our mission and serve patients around the world living with rare disease. I will now turn the call over to Ian Estepan for an update on our financial results. Ian.

Louise Rodino-Klapac: It was fully enrolled in October 2023 with 160 patients. We remain committed to rapidly and diligently advancing Mission and sharing data as soon as it becomes available. In conclusion, the months ahead are filled with great promise to advance our mission and serve patients around the world living with rare disease. I will now turn the call over to Ian Estepan for an update on our financial results. Ian.

Speaker Change: We remain committed to rapidly and diligently advancing mission and Sharon data as soon as it becomes available.

Speaker Change: In conclusion. The months ahead are filled with great promise to advance our mission and serve patients around the world living with rare disease. I will now turn the call over to E&S, Japan for an update on our financial results Yeah. Thanks Al Okay. Good afternoon, everyone. This afternoon's financial result press release provided details for the fourth quarter of 2023 on.

In conclusion, the months ahead are filled with great promise to advance our mission and serve patients around the world living with rare disease. I will now turn the call over to Ian Estepan for an update on our financial results

In conclusion, the months ahead are filled with great promise to advance our mission and serve patients around the world living with rare disease.

Speaker Change: I will now turn the call over to E&S, Japan for an update on our financial results Yeah. Thanks Al Okay. Good afternoon, everyone. This afternoon's financial result press release provided details for the fourth quarter of 2023 on.

I will now turn the call over to Ian Estepan for an update on our financial results.

Ian Estepan: Thanks, Alor Kay. Good afternoon, everyone. This afternoon's financial result press release provided details for the fourth quarter of 2023 on a non-GAAP basis as well as a GAAP basis. Please refer to our press release available on Sarepta's website for a full reconciliation of GAAP to non-GAAP financial results. Before we get to the results, I just wanted to flag that beginning in the fourth quarter of 2023, amortization of in-license rights and income tax expense or benefit are no longer excluded from non-GAAP results. The company has added income tax effective adjustments, which represents the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the fourth quarter and full year of 2022 have been updated to reflect this change for comparability purposes.

E&S: Thanks Al Okay. Good afternoon, everyone. This afternoon's financial result press release provided details for the fourth quarter of 2023 on.

Yeah. Thanks Al Okay. Good afternoon, everyone. This afternoon's financial result press release provided details for the fourth quarter of 2023 on.

Thanks, LRK. Good afternoon, everyone. This afternoon's financial result press release provided details for the 4th quarter of 2023 on a non-GAAP basis as well as a GAAP basis. Please refer to our press release available on Sarepta's website for a full reconciliation of GAAP to non-GAAP financial results. Before we get to the results, I just wanted to flag that beginning in the 4th quarter of 2023, amortization of in-license rights and income tax expense or benefit are no longer excluded from non-GAAP results. The company has added income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax, non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the 4th quarter and full year 2022 have been updated to reflect this change for comparability purposes.

Ian M. Estepan: Thanks, LRK. Good afternoon, everyone.

This afternoon's financial result press release provided details for the 4th quarter of 2023 on a non-GAAP basis as well as a GAAP basis. Please refer to our press release available on Sarepta's website for a full reconciliation of GAAP to non-GAAP financial results. Before we get to the results, I just wanted to flag that beginning in the 4th quarter of 2023, amortization of in-license rights and income tax expense or benefit are no longer excluded from non-GAAP results. The company has added income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax, non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the 4th quarter and full year 2022 have been updated to reflect this change for comparability purposes.

This afternoon's financial result press release provided details for the fourth quarter of 2023 on.

E&S: On a non-GAAP basis as well as the GAAP basis. Please refer to our press release available on <unk> website for a full reconciliation of GAAP to non-GAAP financial results before we get to the results I just wanted to flag that beginning in the fourth quarter of 2023 amortization of in license rights and income tax expense or benefit or no longer.

E&S: Excluded from non-GAAP results. The company has added income tax effect of adjustments, which represents the estimated income tax impact of each pretax non-GAAP adjustment based on the applicable effective income tax rate non-GAAP financial results for the fourth quarter and full year 2022 have been updated to reflect this change for <unk>.

The company has added income tax effect of adjustments, which represents the estimated income tax impact of each pre-tax, non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the 4th quarter and full year 2022 have been updated to reflect this change for comparability purposes.

Ian Estepan: So for the three months ended 31 December 2023, the company recorded total revenues of $396.8 million, which consists of net product revenues, collaboration and other revenues compared to revenues of $258.4 million for the same period of 2022, an increase of $138.4 million. Net product revenue for the fourth quarter of 2023 from ELEVIDYS was $131.2 million. Net product revenue for the fourth quarter of 2023 from our PMO exon skipping franchise was $233.8 million compared to $235.9 million for the same period of 2022. For the fourth quarter of 2023, individual net product sales were $131 million for EXONDYS 51, $69.9 million for AMONDYS 45, and $32.9 million for VYONDYS 53. The increase in net product revenue primarily reflects increasing demand for our PMO products as well as net product revenue associated with sales of ELEVIDYS.

E&S: Our ability purposes.

E&S: So, for the three months ended December 31, 2023, the company recorded total revenues of $396.8 million, which consists of net product revenues and collaboration and other revenues, compared to revenues of $258.4 million for the same period of 2022, an increase of $138.4 million. Net product revenue for the 4th quarter of 2023 from ELEVYDIS was $131.2 million. Net product revenue for the 4th quarter of 2023 from our PMO exon skipping franchise was $233.8 million compared to $235.9 million for the same period of '22. For the 4th quarter 2023 individual net product sales were $131 million for EXONDYS 51, $69.9 million from AMONDYS 45 and $32.9 million for VYONDYS 53. The increase in net product revenue primarily reflecting increasing demand for our PMO products, as well as net product revenue associated with sales of ELEVIDYS.

So, for the three months ended December 31, 2023, the company recorded total revenues of $396.8 million, which consists of net product revenues and collaboration and other revenues, compared to revenues of $258.4 million for the same period of 2022, an increase of $138.4 million. Net product revenue for the 4th quarter of 2023 from ELEVYDIS was $131.2 million.

E&S: Net product revenue for the fourth quarter of 2023 from <unk> was $131 $2 million.

Net product revenue for the 4th quarter of 2023 from our PMO exon skipping franchise was $233.8 million compared to $235.9 million for the same period of '22. For the 4th quarter 2023 individual net product sales were $131 million for EXONDYS 51, $69.9 million from AMONDYS 45 and $32.9 million for VYONDYS 53. The increase in net product revenue primarily reflecting increasing demand for our PMO products, as well as net product revenue associated with sales of ELEVIDYS.

E&S: Net product revenue for the fourth quarter of 2023 from our PMO exon skipping franchise was $233 $8 million compared to $235 $9 million for the same period of 'twenty two.

E&S: Fourth quarter 2023 individuals net product sales were $131 million for exon 50, $169 $9 million from a minus 45 and $32 9 million for <unk> 53.

E&S: The increase in net product revenue, primarily reflecting increasing demand for our PMO products as well as net product revenue associated with sales of <unk>.

Ian Estepan: In the quarter ended December 31, 2023, we recognized $31.7 million of collaboration and other revenues compared to $22.5 million for the same period of 2022. This revenue primarily relates to our collaboration arrangement with Roche. For the quarter ended December 31, 2023, the company recognized $9.2 million of contract manufacturing collaboration revenue associated with multiple batches of commercial ELEVIDYS supply delivered to Roche, with no similar activity ended December 31, 2022. The reimbursable co-development costs under the Roche agreement totaled $23.5 million for the fourth quarter of 2023 compared to $51.7 million for the same period of 2022. On a GAAP basis, we reported a net income of $45.7 million, or $0.49 per basic and $0.47 per diluted share, and a net loss of $109.2 million, or $1.24 per basic and diluted share for the fourth quarter of 2023 and 2022, respectively.

E&S: In the quarter ended December 31, 2023, we've recognized $31.7 million of collaboration and other revenues, compared to $22.5 million for the same period of 2022. This revenue primarily relates to our collaboration arrangement with Roche. For the quarter ended December 31, 2023, the company recognized $9.2 million of contract manufacturing collaboration revenue associated with multiple batches of commercial ELEVIDYS supply delivered to Roche, with no similar activity ended December 31, 2022. The reimbursement co-development costs under the Roche agreement totaled $23.5 million for the 4th quarter of 2023, compared to $51.7 million for the same period of 2022.

E&S: This revenue primarily relates to our collaboration arrangement with Roche.

E&S: For the quarter ended December 31, 2023, the company recognized $9 $2 million of contract manufacturing collaboration revenue associated with multiple batches of commercial laboratory supply delivered to Roche with no similar activity ended December 30, 31 2022.

E&S: The reimbursement co development costs under the Roche agreement totaled $23 $5 million for the fourth quarter of 2023 compared to $51 $7 million for the same period of 2022.

E&S: On a GAAP basis, we reported net income of $45.7 million or $0.49 per basic and $0.47 per diluted share and a net loss of $109.2 million or $1.24 per basic and diluted share for the 4th quarter of 2023 and 2022, respectively. We reported non-GAAP net income of $86.6 million or $0.82 per diluted share in the 4th quarter of 2023, compared to a non-GAAP net loss of $53.6 million or $0.61 per diluted share in the 4th quarter of 2022. In the 4th quarter of 2023, we recorded approximately $44.2 million in cost of sales compared to $30.8 million for the same period of 2022. The increase in cost of sales primarily reflect increasing demand for our PMO products and increase in royalty payments due to ELEVIDYS sales in 2023, with no similar activity in 2022 and write-off of certain batches of our products not meeting our quality specifications in the three months ended December 31, 2023, with no similar activity for the same period of 2022. On a GAAP basis, we recorded a $195.5 million and $213.8 million in R&D expenses for the 4th quarter of 2023 and 2022, respectively -- a year-over-year decrease of $18.3 million. The decrease is primarily due to a decrease in manufacturing expenses and upfront milestone and other expenses. On a non-GAAP basis, R&D expenses were $165.1 million for the 4th quarter of 2023, compared to $186.8 million for the same period of 2022, a decrease of $21.7 million.

On a GAAP basis, we reported net income of $45.7 million or $0.49 per basic and $0.47 per diluted share and a net loss of $109.2 million or $1.24 per basic and diluted share for the 4th quarter of 2023 and 2022, respectively. We reported non-GAAP net income of $86.6 million or $0.82 per diluted share in the 4th quarter of 2023, compared to a non-GAAP net loss of $53.6 million or $0.61 per diluted share in the 4th quarter of 2022. In the 4th quarter of 2023, we recorded approximately $44.2 million in cost of sales compared to $30.8 million for the same period of 2022. The increase in cost of sales primarily reflect increasing demand for our PMO products and increase in royalty payments due to ELEVIDYS sales in 2023, with no similar activity in 2022 and write-off of certain batches of our products not meeting our quality specifications in the three months ended December 31, 2023, with no similar activity for the same period of 2022. On

Ian Estepan: We reported a non-GAAP net income of $86.6 million, or $0.82 per diluted share in the fourth quarter of 2023 compared to a non-GAAP net loss of $53.6 million, or $0.61 per diluted share in the fourth quarter of 2022. In the fourth quarter of 2023, we recorded approximately $44.2 million in cost of sales compared to $30.8 million for the same period of 2022. The increase in cost of sales primarily reflects increasing demand for our PMO products, an increase in royalty payments due to ELEVIDYS sales in 2023 with no similar activity in 2022, and write-off of certain batches of our products not meeting our quality specifications in the three months ended 31 December 2023, with no similar activity for the same period of 2022.

E&S: We reported non-GAAP net income of $86 6 million or <unk> 82 per diluted share in the fourth quarter of 2023 compared to a non-GAAP net loss of $53 $6 million or <unk> 61 per diluted share in the fourth quarter of 2022.

In the 4th quarter of 2023, we recorded approximately $44.2 million in cost of sales compared to $30.8 million for the same period of 2022. The increase in cost of sales primarily reflect increasing demand for our PMO products and increase in royalty payments due to ELEVIDYS sales in 2023, with no similar activity in 2022 and write-off of certain batches of our products not meeting our quality specifications in the three months ended December 31, 2023, with no similar activity for the same period of 2022.

E&S: In the fourth quarter of 2023, we recorded approximately $44 $2 million and cost of sales compared to $38 million for the same period of 2022. The increase in cost of sales primarily reflects increasing demand for our PMO products, an increase in royalty payments due to elaborate of sales in 2023 with no similar active.

E&S: <unk> in 2022 and write off of certain batches of our products not meeting our quality specifications in the three months ended December 31, 2023 with no similar activity for the same period of 2022.

Ian Estepan: On a GAAP basis, we recorded $195.5 million and $213.8 million in R&D expenses for the fourth quarter of 2023 and 2022, respectively, a year-over-year decrease of $18.3 million. The decrease is primarily due to a decrease in manufacturing expenses and upfront milestone and other expenses. On a non-GAAP basis, R&D expenses were $165.1 million for the fourth quarter of 2023 compared to $186.8 million for the same period of 2022, a decrease of $21.7 million. Now, turning to SG&A, on a GAAP basis, we recorded approximately $131.7 million and $120.5 million of expenses for the fourth quarter of 2023 and 2022, respectively, an increase of $11.2 million. The increase was driven primarily by an increase in professional services, compensation, and other personnel expenses, partially offset by a decrease in stock-based compensation.

On a GAAP basis, we recorded a $195.5 million and $213.8 million in R&D expenses for the 4th quarter of 2023 and 2022, respectively -- a year-over-year decrease of $18.3 million. The decrease is primarily due to a decrease in manufacturing expenses and upfront milestone. Other expenses on. On a non-GAAP basis, R&D expenses were $165 1 million for the fourth quarter of 2023 compared to $186 $8 million for the same period of 2022, a decrease of $21 $7 million.

On a GAAP basis, we recorded a $195.5 million and $213.8 million in R&D expenses for the 4th quarter of 2023 and 2022, respectively -- a year-over-year decrease of $18.3 million. The decrease is primarily due to a decrease in manufacturing expenses and upfront milestone and other expenses. On a non-GAAP basis, R&D expenses were $165.1 million for the 4th quarter of 2023, compared to $186.8 million for the same period of 2022, a decrease of $21.7 million.

a GAAP basis, we recorded a $195.5 million and $213.8 million in R&D expenses for the 4th quarter of 2023 and 2022, respectively -- a year-over-year decrease of $18.3 million. The decrease is primarily due to a decrease in manufacturing expenses and upfront milestone and other expenses. On a non-GAAP basis, R&D expenses were $165.1 million for the 4th quarter of 2023, compared to $186.8 million for the same period of 2022, a decrease of $21.7 million.

E&S: Other expenses on.

E&S: On a non-GAAP basis, R&D expenses were $165 1 million for the fourth quarter of 2023 compared to $186 $8 million for the same period of 2022, a decrease of $21 $7 million.

E&S: Now turning to SG&A on a GAAP basis. We recorded approximately $131.7 million and $120.5 million of expenses for the 4th quarter of 2023 and 2022, respectively, an increase of $11.2 million. The increase was driven primarily by an increase in professional services and compensation and other personnel expenses, partially offset by a decrease in stock-based compensation. On a non-GAAP basis, the SG&A expenses were $105.7 million for the 4th quarter of 2023 compared to $86.6 million for the same period of 2022, an increase of $19.1 million. On a GAAP basis, we recorded a $15.7 million in other income net for the 4th quarter of 2023 compared to $5.5 million for the same period of 2022. The change was primarily due to an increase in the accretion investment discount net due to an increase in interest rates. We had approximately $1.7 billion in cash, cash equivalents and investments in long-term restricted cash as of December 31, 2023. So, to conclude, we're incredibly pleased to achieve our first quarter profitability from a GAAP perspective.

Now turning to SG&A on a GAAP basis. We recorded approximately $131.7 million and $120.5 million of expenses for the 4th quarter of 2023 and 2022, respectively, an increase of $11.2 million. The increase was driven primarily by an increase in professional services and compensation and other personnel expenses, partially offset by a decrease in stock-based compensation. On a non-GAAP basis, the SG&A expenses were $105.7 million for the 4th quarter of 2023 compared to $86.6 million for the same period of 2022, an increase of $19.1 million. On a GAAP basis, we recorded a $15.7 million in other income net for the 4th quarter of 2023 compared to $5.5 million for the same period of 2022. The change was primarily due to an increase in the accretion investment discount net due to an increase in interest rates. We had approximately $1.7 billion in cash, cash equivalents and investments in long-term restricted cash as of December 31, 2023.

E&S: <unk> expenses for the fourth quarter of 2023, and 2022, respectively, an increase of $11 $2 million.

E&S: The increase was driven primarily by an increase in professional services and compensation and other personnel expenses, partially offset by a decrease in stock based compensation.

Ian Estepan: On a non-GAAP basis, the SG&A expenses were $105.7 million for the fourth quarter of 2023 compared to $86.6 million for the same period of 2022, an increase of $19.1 million. On a GAAP basis, we recorded $15.7 million in other income net for the fourth quarter of 2023 compared to $5.5 million for the same period of 2022. The change was primarily due to an increase in the accretion investment discount net due to an increase in interest rates. We had approximately $1.7 billion in cash, cash equivalents in investments, and long-term restricted cash as of 31 December 2023. So to conclude, we're incredibly pleased to achieve our first quarter of profitability from a GAAP perspective. It is impressive to reach this milestone in just the second quarter of the ELEVIDYS launch and highlights the leverage in our business.

E&S: On a non-GAAP basis, the SG&A expenses were $105 $7 million for the fourth quarter of 2023 compares to $86 6 million for the same period of 2022, an increase of $19 $1 million.

On a GAAP basis, we recorded a $15.7 million in other income net for the 4th quarter of 2023 compared to $5.5 million for the same period of 2022. The change was primarily due to an increase in the accretion investment discount net due to an increase in interest rates. We had approximately $1.7 billion in cash, cash equivalents and investments in long-term restricted cash as of December 31, 2023.

E&S: On a GAAP basis, we recorded a $15 $7 million and other income net for the fourth quarter of 2023 compared to $5 $5 million for the same period of 2022. The change was primarily due to an increase in the accretion investment discount net due to an increase in interest rates we.

E&S: We had approximately $1 $7 billion in cash cash equivalents and investments in long term restricted cash as of December 31, 2023, setting conclude we're incredibly pleased to achieve our first quarter profitability from a GAAP perspective.

So, to conclude, we're incredibly pleased to achieve our first quarter profitability from a GAAP perspective. It is impressive to reach this milestone in just the 2nd quarter of the ELEVIDYS launch and highlights the leverage in our business. For the reasons Dallan outlined, there may be some lumpiness on the bottom line in the first half. That said, with an expansion of the label, we expect to be highly profitable for the full year. Also, as I previously noted, in preparation to serve the community in the event of a label expansion, we are continuing to ramp our manufacturing of ELEVIDYS. This continued investment will be apparent in the 1st quarter financials and the remainder of the year. We are thrilled to have the resources on hand to continue to build the inventory needed to deliver ELEVIDYS to the Duchenne community.

It is impressive to reach this milestone in just the second quarter of the 11 inch launch it and highlights the leverage in our business. And the reason is down and outline there may be some lumpiness on the bottom line in the first half that said with an expansion of the label, we expect to be highly profitable for the full year also as I previously noted in preparation to serve the community in the event of a label expansion, we are continuing to ramp our manufacturing of <unk>. This continued investment will be apparent in the first quarter financials and the remainder of the year. We're thrilled to have the resources on hand to continue to build the inventory needed to deliver allowed us to the duchenne community and with that I'll turn the call back over to Doug to start the Q&A.

Ian Estepan: For the reasons down and outlined, there may be some lumpiness on the bottom line in the first half. That said, with an expansion of the label, we expect to be highly profitable for the full year. Also, as I previously noted, in preparation to serve the community in the event of a label expansion, we're continuing to ramp our manufacturing of ELEVIDYS. This continued investment will be apparent in the Q1 financials and the remainder of the year. We are thrilled to have the resources on hand to continue to build the inventory needed to deliver ELEVIDYS to the Duchenne community. With that, I'll turn the call back over to Doug to start the Q&A. Doug.

E&S: And the reason is down and outline there may be some lumpiness on the bottom line in the first half that said with an expansion of the label, we expect to be highly profitable for the full year also as I previously noted in preparation to serve the community in the event of a label expansion, we are continuing to ramp our manufacturing of <unk>.

Also, as I previously noted, in preparation to serve the community in the event of a label expansion, we are continuing to ramp our manufacturing of ELEVIDYS. This continued investment will be apparent in the 1st quarter financials and the remainder of the year. We are thrilled to have the resources on hand to continue to build the inventory needed to deliver ELEVIDYS to the Duchenne community.

E&S: This continued investment will be apparent in the first quarter financials and the remainder of the year.

E&S: We're thrilled to have the resources on hand to continue to build the inventory needed to deliver allowed us to the duchenne community and with that I'll turn the call back over to Doug to start the Q&A.

And with that, I'll turn the call back over to Doug to start the Q&A. Doug?

Doug Ingram: Thank you very much, Ian and Shannon. Let's open the call for questions.

Douglas S. Ingram: Thank you very much, Ian. Shannon, let's open the call for questions.

Operator: Thank you. As a reminder, to ask a question, please press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again. We ask that you please limit yourself to one question. Our first question comes from the line of Gena Wang with Barclays. Your line is now open.

Operator: Thank you. As a reminder, to ask a question, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. We ask that you please limit yourself to one question.

Douglas S. Ingram: We expect please limit yourself to one question.

Douglas S. Ingram: Our first question comes from the line of Gena Wang with Barclays. Your line is now open.

Gena Wang: Thank you. I think, Operator, your voice was cut off, so I don't know how many questions I can ask. I just asked two very quick questions. So one is ELEVIDYS. So for the proposed label, was that based on FDA feedback, or was it purely from Sarepta's end? And a second, regarding the EMERGENE phase 3 study in Limb-girdle 2E, was it confirmed with the FDA that 15 patients would be sufficient, and what protein level would be approvable?

Gena Wang: Thank you. I think, operator, your voice was cut off so I don't know how many questions I can ask. I just have two very quick questions, one is ELEVIDYS. So, for the proposed label, was that based on FDA feedback or was purely from Sarepta's end? And a second regarding the EMERGENE Phase III study in limb-girdle 2E, was it confirmed with the FDA that 15 patient would be sufficient and what protein level will be approvable?

Gena Wang: One is it.

Speaker Change: I love it.

Gena Wang: So for the proposed label was that based on FDA feedback always purely from Raptor fan and a second regarding that.

Gena Wang: Emerging phase III study in Laguardia E was it confirm was the D. Eight FTE that 15 patient would be sufficient and what protein level will it be approvable.

Doug Ingram: Yeah. Thank you very much for your questions, Gena. Just for the future, it is one question per analyst, but I appreciate your questions. Let me answer them. So the basis for our request to expand the label by removing both the age and ambulation restrictions, that is driven by the science. I want to be very clear about that. We're in the midst of a review. We're very pleased with the progress of the review so far, but it's too early right now to give you a substantive view on the division's perspective on that. But our view is that the science and the totality of the evidence supports the label expansion that we've asked for, and it's the right answer from our perspective. It's the right answer from the science. It's the right answer from a policy perspective.

Speaker Change: Thank you very much for your questions, Gena. For the future, it is one question per analyst. But I appreciate your questions, let me answer them. So, the basis for our request to expand the label by removing both the age and ambulation restriction, that is driven by the science. I want to be very clear about that. We're in the midst of a review, we're pleased with the progress of the review so far but it's too early right now to give you a substantive view on the division's perspective on that. But our view is that the science and the totality of the evidence supports the label expansion that we've asked for and it's the right answer from our perspective, it's the right answer from the science, it's the right answer from a policy perspective and most of all, it's the right answer for patients living with Duchenne muscular dystrophy, which I hope will be all of our north stars in this regard.

Douglas S. Ingram: Thank you very much for your questions, Gena. For the future, it is one question per analyst. But I appreciate your questions, let me answer them. So, the basis for our request to expand the label by removing both the age and ambulation restriction, that is driven by the science. I want to be very clear about that. We're in the midst of a review, we're pleased with the progress of the review so far but it's too early right now to give you a substantive view on the division's perspective on that.

Speaker Change: For the future. It has one question for Alex.

Speaker Change: Appreciate your questions, let me answer them.

Speaker Change: So the basis for our request to expand the label by removing both the age and ambulation restriction that is driven by the science.

Speaker Change: Clear about that.

The review were very well.

Speaker Change: We're pleased with the progress of the room.

Speaker Change: So far but it's too early right now to give you a subsequent with you on that.

Speaker Change: Division's perspective on that but our view is that.

But our view is that the science and the totality of the evidence supports the label expansion that we've asked for and it's the right answer from our perspective, it's the right answer from the science, it's the right answer from a policy perspective and most of all, it's the right answer for patients living with Duchenne muscular dystrophy, which I hope will be all of our north stars in this regard.

Speaker Change: The science and the totality of the evidence supports the label expansion that we've asked for and it's it's the right answer from our perspective, it's the right answer from the science, it's the right answer for us.

Speaker Change: From a policy perspective, and most of all it's the right answer for patients living with Duchenne muscular dystrophy, which I hope will be all of our north stars.

Doug Ingram: And most of all, it's the right answer for patients living with Duchenne muscular dystrophy, which I hope will be all of our North Stars in this regard. With respect to EMERGENE, I should say, the protocol for that has been shared with, and reviewed by, the FDA, and they have, on the basis of those discussions, including the end of the study, given us their blessing to commence that study and will provide additional information on the level of expression likely to predict the clinical benefit at a later date. I would note, in our prior studies, we've had two cohorts. We've seen very robust expression of the missing beta-sarcoglycan protein, the absence of which is the sole and exclusive cause of the degeneration in patients who have LGMD type 2E. Thank you.

Speaker Change: In this regard.

Speaker Change: With respect to EMERGENE -- Or EMERGENE, I should say, the protocol for that has been shared with and reviewed by the FDA and they have, on the basis of those discussions including the end of the study, given us their blessing to commence that study. We will provide additional information on the level of expression likely to predict clinical benefit at a later date. I would note in our prior studies, which had two cohorts, we've seen very robust expression of the [inaudible] beta-sarcoglycan protein, the absence of which is the sole and exclusive cause of the degeneration in patients who have LGMD type 2E. Thank you.

Speaker Change: Or emerging I should say.

Speaker Change: Yes.

Speaker Change: The protocol for that has been shared with and reviewed with Baidu.

Speaker Change: By the FDA and they have on the basis of those discussions including the end of the study.

Speaker Change: Given us their blessing.

Speaker Change: Study.

Speaker Change: We will provide additional information on the level of expression.

Speaker Change: Likely to predict clinical benefit at a later date.

Speaker Change: I would note in our prior studies, which had two cohorts we've seen very robust expression.

Speaker Change: The mystery beta <unk> protein in the absence of which is the sole and exclusive cause of the deterioration in patients who have <unk>.

Speaker Change: Thank you.

Operator: Thank you. Our next question comes from the line of Tazeen Ahmad with Bank of America. Your line is now open.

Tazeen Ahmad: Hi. Good evening, and thanks for taking my question. I just wanted to get a sense as you look for the expansion of label on how to model COGS, especially as more patients get on therapy over time and the weight of the patients presumably will go higher as you get to older patients. What's a reasonable range of COGS that we should be assuming? Thank you.

Speaker Change: Hi, good evening and thanks for taking my question. I just wanted to get a sense as you look for the expansion of label and how to model COGS, especially as more patients get on therapy over time and the weight of the patients presumably will go higher as you get to older patients. what's a reasonable range of COGS that we should be assuming? Thank you. Yes, yes. This two year.

Tazeen Ahmad: Hi, good evening and thanks for taking my question. I just wanted to get a sense as you look for the expansion of label and how to model COGS, especially as more patients get on therapy over time and the weight of the patients presumably will go higher as you get to older patients. what's a reasonable range of COGS that we should be assuming? Thank you.

Speaker Change: Especially as more patients getting on therapy over time and the weight of the patients presumably will go higher as you get older patients, what's a reasonable range of costs that we should be assuming.

Doug Ingram: Yeah. Ian, I'll turn this to you.

Speaker Change: Yes, yes.

Yeah. Ian, I'll turn this to you. Sure so from a cost perspective, we've been pretty clear. That we're targeting 80, obviously from our financials that we reported today you see it's much higher than that. But over time as heavier patients getting on therapy, and obviously, our pre expense inventory is exhausted youll see. The Cogs, obviously start to increase in our margin to start to decline more to that 80% range, which we've been guiding obviously, we can't give. Complete clarity around that because we have a. Suspension, which could be coming online. With as much higher yielding process in time for some of the older heavier patients to be coming on. Which would offset you know their weight so. As we said over time, you would see some erosion of the margin from where we currently are. But modeling and around 80% is probably reasonable.

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

Ian Estepan: Sure. So from a COGS perspective, we've been pretty clear that we're targeting 80, obviously, from our financials that we reported today. You see it's much higher than that. But over time, as heavier patients get on the therapy and, obviously, our pre-expense inventory is exhausted, you'll see the COGS obviously start to increase and our margin to start to decline more to that 80% range, which we've been guiding. Obviously, we can't give complete clarity around that because we have an expansion, which could be coming online, which is a much higher-yielding process, and time for some of the older, heavier patients to be coming on, which would offset their weight. So as we said, over time, you'd see some erosion of the margin from where we currently are, but modeling it around 80% is probably reasonable.

This two year.

Speaker Change: Sure so from a cost perspective, we've been pretty clear. That we're targeting 80, obviously from our financials that we reported today you see it's much higher than that. But over time as heavier patients getting on therapy, and obviously, our pre expense inventory is exhausted youll see. The Cogs, obviously start to increase in our margin to start to decline more to that 80% range, which we've been guiding obviously, we can't give. Complete clarity around that because we have a. Suspension, which could be coming online. With as much higher yielding process in time for some of the older heavier patients to be coming on. Which would offset you know their weight so. As we said over time, you would see some erosion of the margin from where we currently are. But modeling and around 80% is probably reasonable.

Ian M. Estepan: Sure. So, from a COGS perspective, we've been pretty clear that we're targeting 80. Obviously, from our financials that we reported today, you see it's much higher than that. But over time as heavier patients get on the therapy and, obviously, our pre-expense inventory is exhausted, you'll see the COGS rapidly start to increase and our margin to start to decline more to that 80% range, which we've been guiding. We can't give complete clarity around that because we have a suspension which could be coming online with as much higher yielding process, in time for some of the older heavier patients to be coming on, which would offset their weight. As we said over time, you'd see some erosion of the margin from where we currently are but modeling it around 80% is probably reasonable.

Speaker Change: That we're targeting 80, obviously from our financials that we reported today you see it's much higher than that.

Speaker Change: But over time as heavier patients getting on therapy, and obviously, our pre expense inventory is exhausted youll see.

Speaker Change: The Cogs, obviously start to increase in our margin to start to decline more to that 80% range, which we've been guiding obviously, we can't give.

Ian M. Estepan: We can't give complete clarity around that because we have a suspension which could be coming online with as much higher yielding process, in time for some of the older heavier patients to be coming on, which would offset their weight. As we said over time, you'd see some erosion of the margin from where we currently are but modeling it around 80% is probably reasonable.

Speaker Change: Complete clarity around that because we have a.

Speaker Change: Suspension, which could be coming online.

Speaker Change: With as much higher yielding process in time for some of the older heavier patients to be coming on.

Speaker Change: Which would offset you know their weight so.

Speaker Change: As we said over time, you would see some erosion of the margin from where we currently are.

Speaker Change: But modeling and around 80% is probably reasonable.

Operator: Thank you. Our next question comes from the line of Joseph Schwartz with Leerink Partners. Your line is now open.

Speaker Change: Our next question comes from the line of Joseph Schwartz with Leerink Partners. Your line is now open.

Dallan Murray: Hi all. Thanks for taking our questions. This is Will on for Joe. Congrats on the great quarter here. So one question for us on manufacturing. With the recent acquisition of Catalent by Novo and the expected expiration of your agreement with Catalent at the end of this year, how are you guys thinking about manufacturing moving forward? And then, in terms of capacity, the potential for an expanded label this year, are there any potential for supply constraints in the near term? Thank you.

Joseph Schwartz: Hi all. Thanks for taking our questions. This is Will on for Joe. Congrats on the great quarter here. So one question for us on manufacturing. With the recent acquisition of Catalent by Novo and the expected expiration of your agreement with Catalent at the end of this year, how are you guys thinking about manufacturing moving forward? And then, in terms of capacity, the potential for an expanded label this year, are there any potential for supply constraints in the near term? Thank you.

Unknown: Hi all, thanks for taking our questions. This is Will on for Joe. Congrats on the great quarter here. So, one question for us in manufacturing. With the recent acquisition of Catalent by Novo and the expected expiration of your agreement with Catalent at the end of this year, are you guys thinking about manufacturing moving forward? And then in terms of capacity, the potential for an expanded label this year, are there any potential for supply constraints in the near-term? Thank you.

Speaker Change: Potential for an expanded label. This year are there any potential for supply constraints in the near term. Thank you.

Doug Ingram: Yeah. Thank you for your questions, Will. First of all, I think there might be a misunderstanding of some sort. Let me be very clear. Our agreement with Catalent does not end at the end of this year. We have a long-term relationship with Catalent. They've been very good partners of ours. So there will be, from our perspective, no impact from the acquisition of Catalent by Novo Holdings. As we understand it, Novo Holdings would be acquiring the entity as a whole. The GLP-1-related manufacturing, I think, is going to be sold down to Novo Nordisk, and then Novo Holdings will hold the rest of Catalent, and it'll be run the way it's run today. And I have had direct discussions with the senior management at Catalent, and I'm quite confident that it is business as usual with them. So we're feeling very good about that.

Speaker Change: Yeah. So, essentially for your questions -- well, first of all, I think there might be misunderstanding of some sort. Let me be very clear, our agreement with Catalent does not end at the end of this year. We have a long-term relationship with Catalent, they've been very good partners of ours. There will be, from our perspective, no impact from the acquisition of Catalent by Novo Holdings. As we understand it, Novo Holdings will be acquiring the entity as a whole; the GLP-1-related manufacturing, I think it's going to be sold down to Novo Nordisk and then Novo Holdings will hold the rest of Catalent and it'll be run the way it's run today. And we've had direct discussions with the senior management at Catalent and quite confident that it is business as usual with them. So, we're feeling very good about that. And generally, from a supply perspective, I will say what we have done together over the last -- 2018, at the beginning of this journey, from a manufacturing and CMC perspective, would be nothing other than miraculous if it wasn't for the fact that its science and hard work-driven. And as we sit here today, we're doing a really brilliant job of serving the community and we'll be continuing to do a brilliant job of serving the community over the course of this year with our partner, Catalent. So, we're feeling very good about where we are.

Douglas S. Ingram: Yeah. So, essentially for your questions -- well, first of all, I think there might be misunderstanding of some sort. Let me be very clear, our agreement with Catalent does not end at the end of this year. We have a long-term relationship with Catalent, they've been very good partners of ours. There will be, from our perspective, no impact from the acquisition of Catalent by Novo Holdings. As we understand it, Novo Holdings will be acquiring the entity as a whole; the GLP-1-related manufacturing, I think it's going to be sold down to Novo Nordisk and then Novo Holdings will hold the rest of Catalent and it'll be run the way it's run today.

Speaker Change: Misunderstanding of some sort let me be very clear our agreement with catalyst does not end at the end of this year, we have a long term relationship with Cadillac there they've been very good partners of ours. So.

Speaker Change: There will be from our perspective, no impact from the Av acquisition.

Speaker Change: <unk> catalyst by Novo holdings.

Speaker Change: We understand that Novo holdings would be acquiring the entity as a whole the DLP one related manufacturing I think there's going to be sold down to Novo Nordisk, and then Novo holdings as well.

Speaker Change: Hold the rest of Cadillac and it'll be run the way it's run today and we've had.

And we've had direct discussions with the senior management at Catalent and quite confident that it is business as usual with them. So, we're feeling very good about that. And generally, from a supply perspective, I will say what we have done together over the last -- 2018, at the beginning of this journey, from a manufacturing and CMC perspective, would be nothing other than miraculous if it wasn't for the fact that its science and hard work-driven. And as we sit here today, we're doing a really brilliant job of serving the community and we'll be continuing to do a brilliant job of serving the community over the course of this year with our partner, Catalent. So, we're feeling very good about where we are.

Speaker Change: Direct discussions with the.

Speaker Change: The senior management of Cowen <unk> co.

Speaker Change: Quite confident that it is business as usual with them. So.

Speaker Change: We're feeling very good about that generally from a supply perspective.

Doug Ingram: And generally, from a supply perspective, I will say what we have done together over the last since 2018 at the beginning of this journey from a manufacturing and CMC perspective would be nothing other than miraculous if it wasn't for the fact that it's science and hard work-driven. And as we sit here today, we're doing really a brilliant job of serving the community, and we'll be continuing to do a brilliant job of serving the community over the course of this year with our partner, Catalent. So we're feeling very good about where we are.

Speaker Change: I will say, what we have done together over the last. 2018 at the beginning of this journey from a manufacturing and CMC perspective, yes would be nothing. Other than miraculous if it wasn't for the fact that its science and hard work driven. And as we sit here today, we're doing a really a brilliant job of serving the community and will be continuing. Job of serving the community over the course of this year with our partner catalyst. So we're feeling very good about where we are.

Speaker Change: 2018 at the beginning of this journey from a manufacturing and CMC perspective, yes would be nothing.

Speaker Change: Other than miraculous if it wasn't for the fact that its science and hard work driven.

Speaker Change: And as we sit here today, we're doing a really a brilliant job of serving the community and will be continuing.

Speaker Change: Job of serving the community over the course of this year with our partner catalyst. So we're feeling very good about where we are.

Operator: Thank you. Our next question comes from the line of Gil Blum with Needham & Company. Your line is now open.

Operator: Thank you. Our next question comes from the line of Gil Blum with Needham and Company. Your line is now open.

Ian Estepan: Hi everyone. Good afternoon, and thanks for taking our question. So I know this may be knowable, but can you remind us what the population divide is between ambulatory and non-ambulatory patients, and what is kind of the median age for loss of ambulation? Thanks.

Gil Blum: Hi everyone. Good afternoon, and thanks for taking our question. So I know this may be knowable, but can you remind us what the population divide is between ambulatory and non-ambulatory patients, and what is kind of the median age for loss of ambulation? Thanks.

Gil Blum: Hi, everyone. Good afternoon and thanks for taking our question. So, I know this may be knowable but can you remind us what the population divide is between ambulatory and non-ambulatory patients and what it is kind of the median age for loss of ambulation? Thanks.

Gil Joseph Blum: Can you remind us what the population divided between ambulatory and non ambulatory patients, but it's kind of the median age for loss of ambulation.

Doug Ingram: So I can give you the broad strokes. It is, epidemiologically, about 50/50 between ambulatory and non-ambulatory patients. Dallan, do you have the recent estimates of the loss of ambulation?

Gil Joseph Blum: So I can give you the broad strokes. It is about Epidemiologically about 50 50 between ambulatory and non ambulatory patients. Recent estimates of the. Yes of ambulation. You cut out there for a second did you say the diagnosed patients. No I said what is the average age of loss of ambulation.

So, I can give you the broad strokes. It is about, epidemiologically, about 50/50 between ambulatory and non-ambulatory patients. And Dallan, you have the recent estimates of the [inaudible] of ambulation? You cut out there for a second, did you say the diagnosed patients?

Douglas S. Ingram: So, I can give you the broad strokes. It is about, epidemiologically, about 50/50 between ambulatory and non-ambulatory patients. And Dallan, you have the recent estimates of the [inaudible] of ambulation?

Gil Joseph Blum: Recent estimates of the.

Gil Joseph Blum: Yes of ambulation.

Dallan Murray: You cut out there for a second, did you say the diagnosed patients?

Dallan Murray: You cut out there for a second. Did you say the diagnosed patients?

Gil Blum: You cut out there for a second. Did you say the diagnosed patients?

Gil Joseph Blum: You cut out there for a second did you say the diagnosed patients.

Doug Ingram: No, I said, what is the average age of loss of ambulation?

Douglas S. Ingram: No, I said what is the average age of loss of ambulation.

Speaker Change: No I said what is the average age of loss of ambulation.

Dallan Murray: Oh, average age of loss of ambulation. I think Louise would probably have better data than me, but it would be depending on different publications in the 11 to 12 range. Louise, is that right from what?

Gil Blum: Oh, average age of loss of ambulation. I think Louise would probably have better data than me, but it would be depending on different publications in the 11 to 12 range. Louise, is that right from what?

Dallan Murray: Oh, average age of loss of ambulation. I think Louise would probably have better data than me but, it would be depending on different publications, in the 11 to 12 range. Louise, does that sound right?

Speaker Change: Depending on different publications in the 11% to 12 range.

Luis: Louise does that sound right.

Louise Rodino-Klapac (Sarepta Therapeu: Yeah. Some are slightly earlier. To your point, there are some differences. Yeah.

Louise Rodino-Klapac: Yeah. Some are slightly earlier. To your point, there are some differences. Yeah.

Louise Rodino: Yes, some are slightly earlier. To your point, there are some differences.

Luis: Okay.

Speaker Change: Yeah.

Doug Ingram: Thank you, Bill.

Gil Blum: Thank you, Bill.

Gil Blum: Thank you [inaudible].

Operator: Thank you. Our next question comes from the line of Brian Abrahams with RBC Capital Markets. Your line is now open.

Speaker Change: Our next question comes from the line of Brian Abrahams with RBC capital markets. Your line is now open.

Joseph Schwartz: Hi. This is Leonid Timashev for Brian. So maybe I'll ask 1.5 questions, if that's okay, not quite 2. So I guess can you clarify the application process with respect to ELEVIDYS? I guess, is the conversion to a full approval and the label expansion two separate questions before the FDA that have potentially separate timelines, reviews, discussions around them, and can they be separated? And then I guess just related to that, you mentioned in the prepared remarks you're thinking through broad label scenarios. And you mentioned if you get an accelerated approval in the non-ambulatory setting. I guess, is that part of those scenarios? Is that something you've heard from the FDA, or is this just preparing for all the possibilities? Thanks.

Brian Abrahams: Hi. This is Leonid Timashev for Brian. So maybe I'll ask 1.5 questions, if that's okay, not quite 2. So I guess can you clarify the application process with respect to ELEVIDYS? I guess, is the conversion to a full approval and the label expansion two separate questions before the FDA that have potentially separate timelines, reviews, discussions around them, and can they be separated? And then I guess just related to that, you mentioned in the prepared remarks you're thinking through broad label scenarios. And you mentioned if you get an accelerated approval in the non-ambulatory setting. I guess, is that part of those scenarios? Is that something you've heard from the FDA, or is this just preparing for all the possibilities? Thanks.

Lean: Hi, this is Leonard on for Brian. So, maybe I'll ask 1.5 questions if that's okay, not quite 2. I guess, can you clarify the application process with respect to ELEVIDYS? Is the conversion to a full approval and the label expansion, two separate questions before the FDA that have potentially separate timelines, reviews, discussions around them and can they be separated? And then, just related to that. You mentioned in the prepared remarks, you're thinking through broad label scenarios and you mentioned, if you get an accelerated approval in the non-ambulatory setting -- is that part of those scenarios? Is that something you've heard from the FDA or is this just preparing for all of the possibilities? Thanks.

Unknown: Hi, this is Leonard on for Brian. So, maybe I'll ask 1.5 questions if that's okay, not quite 2. I guess, can you clarify the application process with respect to ELEVIDYS? Is the conversion to a full approval and the label expansion, two separate questions before the FDA that have potentially separate timelines, reviews, discussions around them and can they be separated?

Lean: So I guess can you clarify the application process with respect to <unk> I guess is the conversion to a full approval and the label expansion two separate questions before the FDA that have potentially separate timelines reviews discussions around them and can they be separated and then I guess just related to that.

And then, just related to that. You mentioned in the prepared remarks, you're thinking through broad label scenarios and you mentioned, if you get an accelerated approval in the non-ambulatory setting -- is that part of those scenarios? Is that something you've heard from the FDA or is this just preparing for all of the possibilities? Thanks.

Lean: You mentioned in the prepared remarks.

Lean: We're thinking through broad label scenarios.

Speaker Change: So and you mentioned, if you get an accelerated approval in the non ambulatory setting I guess is that part of those scenarios is that something you've heard from the FDA or is this just preparing for all of the possibilities.

Doug Ingram: So a couple of things. Yes, there are essentially two issues at the agency at the same time. One is modifying the label to expand the label and to provide access to a much broader population of patients. And as we said, and it bears repeating, our BLA supplements requested the removal of all age and ambulation-related restrictions from the label because we believe that's founded on science. And of course, the second question then is the view that we have satisfied the requirements for our confirmatory trial and that the entire approval should be translated from accelerated to a traditional approval. There are many different scenarios there.

Douglas S. Ingram: So, a couple of things. Yes, there are essentially two issues at the agency, at the same time. One is modifying the label to expand the label and to provide access to a much broader population of patients. As we said, and it bears repeating, our BLA supplement requesting the removal of all age and ambulation-related restrictions from a label because we believe that's founded on science. And of course, the second question then is this -- the view that we have satisfied the requirements for a confirmatory trial and that the entire approval should be translated from accelerated to a traditional approval.

Speaker Change: Issues at.

Speaker Change: At the agency at the same time one ish.

Speaker Change: <unk> the label to expand the label.

Speaker Change: And to provide access to a much broader population of patients as we said in the bears repeating we are BLA supplements requested the removal of all age and emulation related restrictions from a label because we believe that's founded on sites and of course. The second question. Then is this the view that we have.

Speaker Change: Satisfied.

Speaker Change: The requirements for a confirmatory trial and that the entire.

Speaker Change:

Speaker Change: Approval should be.

Speaker Change: Translated from accelerated to a traditional approval.

Speaker Change: There are many different scenarios there. There's obviously going to be dialogue about the breadth of the label and then, of course, there'll be this dialogue about what portions of that are confirmed for traditional approval and what portions of that are confirmed for -- will remain an accelerated approval. So, there are many iterations. We have a strong view on what we think the science justifies. First remark, administrative perspective. The timelines are identical so, we have a June 21 target completion date -- that is for both of those issues. We'll address both of those issues with June 21 and we're very pleased with June 21, as you know, because we had been guiding people to end of August from that perspective. And the substantive issues or what's going to happen in the review, that's going to be subject to review and we're in the early days. Again, I'd say, we're very pleased at least with the engagement of the divisions thus far but we have more work to do and more discussions to be had and I don't want to overpromise before the end of this. I will also say, there are a lot of different iterations from our perspective -- priority number one is the broadest possible label. So, as between the two issues that we're discussing, we are first and foremost focused on the broadest possible label, giving the potential of access and, hopefully, a better life to the greatest number of patients. And then, of course, we also -- it's important to us but the secondary issue is translating this problem.

There are many different scenarios there. There's obviously going to be dialogue about the breadth of the label and then, of course, there'll be this dialogue about what portions of that are confirmed for traditional approval and what portions of that are confirmed for -- will remain an accelerated approval. So, there are many iterations. We have a strong view on what we think the science justifies. First remark, administrative perspective. The timelines are identical so, we have a June 21 target completion date -- that is for both of those issues.

Doug Ingram: There's obviously going to be dialogue about the breadth of the label, and then, of course, there'll be this dialogue about what portions of that are confirmed for traditional approval and what portions of that are confirmed for an or will remain an accelerated approval. So there are many iterations. We have a strong view on what we think the science justifies. So first, from an administrative perspective, the timelines are identical. So we have a 21 June target completion date. That is for both of those issues. So we'll address both of those issues with 21 June. And we're very pleased with 21 June, as you know, because we had been guiding people to end of August from that perspective. On the substantive issues or what's going to happen in the review, that's going to be subject to the review, and we're in the early days.

Speaker Change: Or it will remain an accelerated approval. So there are many iterations. So we have a strong view on what we think the science justified.

Speaker Change: So first remark administrative perspective, the timelines are identical so we have a June 21. Target. <unk> date that is where both of those issues. So we will address both of those issues with June 'twenty, one and we're very pleased with June 'twenty. One as you know because we had been guiding people to end of August. Sure.

We'll address both of those issues with June 21 and we're very pleased with June 21, as you know, because we had been guiding people to end of August from that perspective. And the substantive issues or what's going to happen in the review, that's going to be subject to review and we're in the early days. Again, I'd say, we're very pleased at least with the engagement of the divisions thus far but we have more work to do and more discussions to be had and I don't want to overpromise before the end of this. I will also say, there are a lot of different iterations from our perspective -- priority number one is the broadest possible label. So, as between the two issues that we're discussing, we are first and foremost focused on the broadest possible label, giving the potential of access and, hopefully, a better life to the greatest number of patients. And then, of course, we also -- it's important to us but the secondary issue is translating this problem.

Speaker Change: Target.

Speaker Change: <unk> date that is where both of those issues. So we will address both of those issues with June 'twenty, one and we're very pleased with June 'twenty. One as you know because we had been guiding people to end of August.

Speaker Change: Sure.

Speaker Change: Our perspective on the substantive issues or what what's going to happen in the review that's going to be subject to review and we're in the early days.

Doug Ingram: Again, I'd say we're very pleased, at least with the engagement of the divisions thus far, but we have more work to do and more discussions to be had, and I don't want to overpromise before the end of this. I will also say there are a lot of different iterations. From our perspective, priority number one is the broadest possible label. So as between the two issues that we're discussing, we are first and foremost focused on the broadest possible label, giving the potential of access and hopefully a better life to the greatest number of patients. And then, of course, we also, it's important to us, but the secondary issue is translating this from accelerated to traditional, and the breadth of that will be a separate discussion.

Speaker Change: And I'd say, we're very pleased at least with the engagement of the divisions, thus far but we have more work to do and more discussions to be had and I don't want to over promise before the end of this I will also say there are a lot of different iterations from our perspective priority number one is the broadest possible label so as between the two issues.

I will also say, there are a lot of different iterations from our perspective -- priority number one is the broadest possible label. So, as between the two issues that we're discussing, we are first and foremost focused on the broadest possible label, giving the potential of access and, hopefully, a better life to the greatest number of patients. And then, of course, we also -- it's important to us but the secondary issue is translating this from accelerated to traditional and the breadth of that will be a separate discussion. But we have more work to do and more discussions and work to do with the agency and June 21 will be our return date on all of that.

Speaker Change: Is that we are discussing we are first and foremost.

Speaker Change: Focus on the broadest possible label given the potential of access in a hopefully a better lives to the greatest number of patients and then of course, we also its important to us but the secondary issue is trash.

Speaker Change: Translating this problem.

Speaker Change: Accelerated to a traditional and the breadth of that will be a separate discussion, but we have more work to do and more.

Doug Ingram: But we have more work to do and more discussions and work to do with the agency, and 21 June will be our return date on all of that.

Speaker Change: Discussions and work to do with the agency in June 'twenty, one will be our return data on all of that.

Operator: Thank you. Our next question comes from the line of Kostas Biliouris with BMO Capital Markets. Your line is now open.

Operator: Thank you. Our next question comes from the line of Kostas Biliouris with BMO capital markets. Your line is now open.

Costas: Our next question comes from the line of cost Us <unk> <unk> with BMO capital markets. Your line is now open.

Kostas Biliouris: Thanks for taking our question and congrats on the impressive progress. So one quick question from us. If we assume that your label will restrict ELEVIDYS use to ambulatory patients only for all ages, how straightforward would it be for physicians and, importantly, payers to clearly determine whether someone is ambulatory or not, given that patients are losing ambulation progressively? So there may be a stage where the ambulation status is unclear. So would it be clear for physicians and payers to determine if someone is eligible or not? Thank you.

Speaker Change: Okay.

Kostas Biliouris: Thanks for taking our question and congrats on the impressive progress. So, one quick question from us -- if we assume that your label will restrict ELEVIDYS use to ambulatory patients only for all ages, how straightforward would it be for physicians and, importantly, payers to clearly determine whether someone is ambulatory or not, given that patients are losing ambulation progressively so, there may be a stage where the ambulation status is unclear. Would it be clear for physicians and payers to determine if someone is eligible or not? Thank you.

Cost Us: Levy these used to ambulatory patients only for all agencies, how straightforward with you would be for <unk>, and importantly, payers to clearly determine where that someone needs ambulatory or not given that patients are losing copulation progressively so that may be a stage where the ambulation.

Cost Us: In start dose is unclear so would it be clear for physicians and payers to determine if somebody's eligible or not thank you.

Doug Ingram: Yeah. Okay. Thank you very much for your question. First, let me be clear. I resist the assumption that we may only get ambulatory alone, of course, because we are, at this point, seeking the removal of the ambulatory restrictions. But to your point, ambulation is in the label today. And the question then is, how do physicians determine what ambulation means? The short answer is that that is left to the medical judgment of physicians, but there is good guidance in medical practice for that. It is based on functional assessments and functional tests. There are well-defined approaches. Probably the most common, perhaps the most gold standard approach, is the definition from synergy, which is that there is a patient-reported continuous use of a wheelchair without ambulation, and then that is verified by the physician by the inability of the patient to walk 10 meters unassisted.

Douglas S. Ingram: Yeah. Okay, thank you very much for your question. First, let me be clear. I resist the assumption that we may only get ambulatory alone, of course, because we are, at this point, seeking a removal of the ambulatory restrictions. B ut to your point, ambulation is in the label today and the question then is, how did physicians determine what ambulation means. The short answer is that, that is left to the medical judgment of physicians but there is good guidance in medical practice for that. It is based on functional assessments and functional test. There are well-defined approaches, probably the most common, perhaps the most gold standard approach is the definition from synergy, which is that there is a patient-reported, continuous use of a wheelchair without ambulation and then that is verified by the physician by the inability of the patient to walk 10 meters unassisted. So, that would be a typical functional assessment that a physician would use to determine ambulatory status. This isn't an enormously difficult functional assessment and conclusion to make by physicians.

Speaker Change: I resist the.

Speaker Change: Assumption that we'd really helped me get ambulatory alone of course.

Speaker Change: Because we are we are at this point.

Speaker Change: Seeking a removal of the ambulatory restrictions, but to your point ambulation is in the label today and the question. Then is what is how did acquisitions determine what formulation needs.

Speaker Change: The short answer is that is left to the medical judgment of physicians, but there is good guidance and medical practice with that it is based on functional assessments and functional test there are well defined approach is probably the most.

Speaker Change: Comment, perhaps the most gold standard approach is.

Douglas S. Ingram: There are well-defined approaches, probably the most common, perhaps the most gold standard approach is the definition from synergy, which is that there is a patient-reported, continuous use of a wheelchair without ambulation and then that is verified by the physician by the inability of the patient to walk 10 meters unassisted. So, that would be a typical functional assessment that a physician would use to determine ambulatory status. This isn't an enormously difficult functional assessment and conclusion to make by physicians.

Speaker Change: The definition from synergy, which is that there is a patient reported.

Speaker Change: Continuous use a wheelchair without ambulation and then that is verified by the physician.

Speaker Change: By the inability of the patient to work 10 meters honest guess goods. So that would be a typical functional assessment that a physician would use to determine ambulatory status. This is.

Doug Ingram: So that would be a typical functional assessment that a physician would use to determine ambulatory status. So this isn't an enormously difficult functional assessment and conclusion to make by physicians.

Speaker Change: This isn't an enormously difficult.

Speaker Change: Functional assessment in conclusion to make by physicians.

Operator: Thank you. Our next question comes from the line of Neena Bitritto-Garg with Deutsche Bank. Your line is now open.

Speaker Change: Our next question comes from the line of Neena <unk> Garg with Deutsche Bank. Your line is now open.

Louise Rodino-Klapac (Sarepta Therapeu: Hey, guys. Thanks for taking my question. Just to follow up on the last question, could you just remind us what data was included in the filing on patients that are non-ambulatory, if any, just to kind of clarify that? Thanks.

Neena Bitritto-Garg: Hey, guys. Thanks for taking my question. Just to follow up on the last question, could you just remind us what data was included in the filing on patients that are non-ambulatory, if any, just to kind of clarify that? Thanks.

Neena Bitritto: Hey, guys. Thanks for taking my question. Just to follow up on the last question, could you just remind us what data was included in the filing on patients that are non-ambulatory if any, just to kind of clarify that? Thanks.

Doug Ingram: Yeah. We have data from children up to 20 years old from a separate study that we call Study 103 that went in with it. We obviously also have an ongoing study, ENVISION, which is a study for non-ambulatory patients. Now, that is an ongoing placebo-controlled trial, but the safety from that study is obviously made available to the FDA on a continuous basis, and all of that supports our BLA supplements.

Douglas S. Ingram: Yeah. We have data from children up to 20 years old from a separate study that we call Study 103 that went in with it. We, obviously, also have an ongoing study, ENVISION, which is this study for non-ambulatory patients. Now, that is an ongoing placebo-controlled trial but the safety from that study is, obviously, made available to the FDA on a continuous basis. All of that supports our BLA supplements.

Speaker Change: After 20 years, all from a separate study that we call study 103.

Speaker Change: That went in with it we obviously also have an ongoing study.

Speaker Change: Envision which is this study for non ambulatory patients now that is an ongoing placebo controlled trial, but the safety from that study is obviously.

Speaker Change: Made available to the FDA on a continuous basis.

Speaker Change: All of that.

Speaker Change: Supports our BLA supplements.

Operator: Thank you. Our next question comes from the line of Tim Lugo with William Blair. Your line is now open.

Speaker Change: Our next question comes from the line of Tim Lugo with William Blair. Your line is now open.

Tim Lugo: Thanks for the question. Can you discuss the level of warehousing currently occurring in the DMD community and maybe any subpopulations ahead of this potential expansion this summer, which we should be aware of? I know we all focus on ambulatory and age-related. Is there anything else, though, that we should be also looking at, maybe lung function or other subpopulations?

Tim Lugo: Thanks for the question. Can you discuss the level of warehousing currently occurring in the DMD community and maybe any subpopulations that, ahead of this potential expansion this summer, which we should be aware of? I know we all focus on ambulatory, age-related -- is there anything else though that we should be also looking at, maybe lung function or other subpopulations?

Timothy Francis Lugo: Thanks for the question can you discuss the level of warehousing currently occurring in the DMD community and maybe any subpopulation that ahead of this potential expansion this summer, which we should be aware of.

Timothy Francis Lugo: I'll focus on ambulatory AIDS related is there anything else there that we should be also looking at the function.

Timothy Francis Lugo: Or other subpopulations.

Doug Ingram: Well, I'm not 100% sure what we mean by warehousing, to be honest. There is going to be an exceptional amount of demand for this therapy when the label is expanded, both from the physician community but from families with Duchenne muscular dystrophy. And we stand prepared to build the material and have lots available to fully serve that demand over the course of 2024. But Dallan, do you have other thoughts on the warehousing question itself?

Douglas S. Ingram: I don't -- I'm not 100% sure what you mean by warehousing, to be honest. There is going to be an exceptional amount of demand for this therapy when the label is expanded, both from the physician community but some families with Duchenne muscular dystrophy. And we stand prepared to build the material and have lots available to fully serve that demand over the course of 2024. But, Dallan, you have other thoughts on that warehousing question itself?

Speaker Change: Im not 100% sure what we mean by warehousing to be honest, there is going to be an exceptional amount of demand for this therapy.

Speaker Change: When the label is expanded both from the physician community by some families with Duchenne muscular dystrophy.

Speaker Change: And we stand prepared to build the material and have lots available to fully serve that demand over the course of 2020 for the Dallas you have other thoughts on that.

Speaker Change: Warehousing question itself.

Dallan Murray: Yeah. I think it's related to the PMOs and warehousing with the PMOs in anticipation of the gene therapy. We were worried about that leading up to the initial launch, and the team did an exceptional job of making sure that that did not happen. And we don't see widespread warehousing where patients are getting delayed or really any warehousing where people are foregoing PMO in anticipation of the gene therapy. So I think, as I mentioned in the script, one of the things we're particularly pleased with this year is the continued, incredibly excellent execution of the PMO team for patients who were not eligible for the gene therapy for ELEVIDYS.

Dallan Murray: Yeah. I think to relate it to the PMOs and warehousing with the PMOs in anticipation of the gene therapy, we were worried about that leading up to the initial launch. And the team did an exceptional job of making sure that that did not happen and we don't see widespread warehousing where patients are getting delayed -- or really, at any warehousing, where people are sort of going PMO in anticipation of the gene therapy. So you know, I think as I mentioned in the script, one of the things we're particularly pleased with this year is the continued incredibly excellent execution of the PMO team for patients who were not eligible for the gene therapy, for ELEVIDYS.

Speaker Change: Or really at any warehousing, where people are sort of going PMO in anticipation of the gene therapy. So so you know.

Speaker Change: I think as I mentioned in the script, we were one of the things, we're particularly pleased with this year is the continued.

Speaker Change: Credibly excellent execution of the PMO team for patients who were not eligible for for the gene therapy for <unk>.

Operator: Thank you. Our next question comes from the line of Uy Ear with Mizuho. Your line is now open.

Mizuho: Our next question comes from the line of point here with Mizuho. Your line is now open.

Kostas Biliouris: Hey, guys. Thanks for taking my question. So, just going back to the exon-skipping products. Could you? I'm not asking for full guidance or anything to that effect. Maybe just help us understand the first half of the year. In the prior quarter, year-over-year growth was relatively strong in the first half. Just wondering how we should sort of think about the first half of 2024 for those three products. Thanks.

Uy Ear: Hey, guys. Thanks for taking my question. So, just going back to the exon-skipping products. Could you? I'm not asking for full guidance or anything to that effect. Maybe just help us understand the first half of the year. In the prior quarter, year-over-year growth was relatively strong in the first half. Just wondering how we should sort of think about the first half of 2024 for those three products. Thanks.

Uy Ear: Hey, guys. Thanks for taking my question. So, just going back to the exon skipping products, could you -- and I'm not asking for guidance or anything but maybe just help us understand, the first half of the year and the prior quarter, year-over-year growth was relatively strong in the first half. Just wondering how we should sort of think about the first half of 2024 for those three products. Thanks.

Point: Could you.

Point: And I'm not asking for guidance or anything, but I think maybe just help us understand that.

Point: Half of the year.

Point: In the prior quarter growth year over year growth was relatively strong in the first half just wondering how we should sort of think about the first half of 2024 for those three products. Thanks.

Doug Ingram: We haven't provided guidance on the PMOs for this year, but we're doing very well. I think you saw very good performance of all three of the PMOs last year. In fact, AMONDYS 45 and VYONDYS 53 were still growing at double digits. And one of the things that's interesting is that we are not seeing right now and probably won't see a significant amount of cannibalization from ELEVIDYS over the course of 2024 that will have a significant impact on the PMO. So we feel very good about where we are with our three RNA-based therapies in 2024.

Douglas S. Ingram: We haven't provided guidance on the PMOs for this year but we're doing very well. I think you saw very good performance of all three of the PMOs last year. In fact, AMONDYS and VYONDYS were still growing double digits. And one of the things that's interesting is that we are not seeing right now -- and probably won't see a significant amount of cannibalization from ELEVIDYS over the course of 2024 that will have a significant impact on the PMO. So, we feel very good about where we are with our three RNA-based therapies in 2024.

Speaker Change: All three of the PMO as last year in fact demand is in by August we're still growing double digits.

Speaker Change: And one of the things that's interesting is that we are not seeing right now.

Speaker Change: And probably won't see a significant amount.

Speaker Change: Cannibalization from <unk> over the course of 2024 that will have a <unk>.

Speaker Change: Significant impact on the GMO. So we feel very good about where we are with our three RNA based therapies in 2024.

Operator: Thank you. Our next question comes from the line of Salveen Richter with Goldman Sachs. Your line is now open.

Speaker Change: Okay.

Operator: Thank you. Our next question comes from the line of Salveen Richter with Goldman Sachs. Your line is now open.

Speaker Change: Our next question comes from the line of solving Richter with Goldman Sachs. Your line is now open.

Salveen Richter: Hi. This is Lydia on for Salveen. Thanks so much for taking our question. So if the label for ELEVIDYS is expanded and the broader population opens up, how do you think that physicians might prioritize which patients to treat first in the context of site constraints? Thank you so much.

Unknown: Hi, this is Lydia, on for Salveen. Thanks so much for taking our question. So, if the label for ELEVIDYS is expanded and the broader population opens up, how do you think that physicians might prioritize which patients to treat first in the context of [inaudible] constraints? Thank you so much.

Doug Ingram: Yeah. That will be an issue that physicians are going to have to grapple with. There are a number of constraints. One of the things that you have to recall or remember is that we need to ensure that we are always prioritizing great outcomes and safety over, for instance, short-term revenue. So we need to make sure that physicians and physicians will know this, and they're going to certainly consider this. They need to make sure that they can not only infuse patients but follow up appropriately. And so that will likely require them to prioritize patients as they consider this therapy. The short answer is that we can't invade the practice of medicine and decide for them what to do. There's compelling arguments on both across the spectrum.

Douglas S. Ingram: That will be an issue that physicians are going to have to grapple with. There are a number of constraints, one of the things that you have to recall or remember is that we need to ensure that we're always prioritizing great outcomes and safety over, for instance, short-term revenue. So, we need to make sure that physicians -- and physicians will know this and they're going to certainly consider this -- they need to make sure that they can, not only infuse patients but follow up appropriately and so, that will likely require them to prioritize cases as they consider this therapy.

Speaker Change: We'll be in.

Speaker Change: And issue that physicians are going to have to grapple with.

Speaker Change: There are a number of constraints one of the things that you have to recall or remember is that we need to ensure that we're always prioritizing great outcomes and safety over for instance short term.

Speaker Change: Revenue, so we need to make sure that physicians and physicians will know this.

Speaker Change: Certainly considered there's no need to make sure that they can not only a huge patient follow up.

Speaker Change: Appropriate Lee and so that will likely require them to prioritize cases as they consider this therapy.

Speaker Change: The short answer is that we can't invade the practice of medicine and decide for them what to do. There's compelling arguments on both across the spectrum. On the one hand, there may very well be physicians who want to prioritize very young children to get in and intervene before damage is done. On the other hand, for the non-ambulatory patients and later patients, there is a race against time for those patients. And so, getting to those patients and stopping damage before it's too late is extraordinarily important. So, these are going to be difficult decisions physicians are going to have to work that out and I'm sure there'll be no white papers and discussions among thought leaders on that topic. The issue today is a slightly different one. The prioritization today, given the label that we have, is to ensure that we get kids on the label -- and this really comes from the physician more than from us -- to get kids on the label before they age out and the therapy won't be available to them. If you look at the bias here, the bias tends to be in the tight age range of 4-5, to be in the later ages, because they're really trying to get to those kids to get through ADOC, administrative process will age out to 6 years old. The good news is that, if we can expand this label, that issue goes to the to decide when will you have to deal with the other issue. It is about prioritization and the like but it ultimately will be a physician decision. A decision on the issue of the practice of medicine for physicians.

The short answer is that we can't invade the practice of medicine and decide for them what to do. There's compelling arguments on both across the spectrum. On the one hand, there may very well be physicians who want to prioritize very young children to get in and intervene before damage is done. On the other hand, for the non-ambulatory patients and later patients, there is a race against time for those patients. And so, getting to those patients and stopping damage before it's too late is extraordinarily important.

Speaker Change: Aside for them what to do there is a compelling arguments on both.

Doug Ingram: On the one hand, there may very well be physicians who want to prioritize very young children to get in and intervene before damage is done. On the other hand, for the non-ambulatory patients and later patients, there is a race against time for those patients. And so getting to those patients and stopping damage before it's too late is extraordinarily important. So these are going to be difficult decisions. Physicians are going to have to work that out, and I'm sure there'll be white papers and discussions among thought leaders on that topic. The issue today is a slightly different one. The prioritization today, given the label that we have, is to ensure that we get kids on the label. And this really comes from the physician more than from us, to get kids on the label before they age out and the therapy won't be available to them.

Speaker Change: Loss of spectrum on the one hand, there may very well be physicians, who want to prioritize very young children.

Speaker Change: And in energy and before damage is done on the other hand for the non ambulatory patients and later patients. There is a race against time for those patients and so getting to those patients.

So, these are going to be difficult decisions physicians are going to have to work that out and I'm sure there'll be no white papers and discussions among thought leaders on that topic. The issue today is a slightly different one. The prioritization today, given the label that we have, is to ensure that we get kids on the label -- and this really comes from the physician more than from us -- to get kids on the label before they age out and the therapy won't be available to them. If you look at the bias here, the bias tends to be in the tight age range of 4-5, to be in the later ages, because they're really trying to get to those kids to [inaudible], get through the administrative process, will age out to 6 years old. The good news is, that if we can expand this label, that issue goes to the side then we'll have to deal with the other issues about prioritization and the like but it, ultimately, will be a physician decision and the issue of the practice of medicine for physicians.

Speaker Change: And stopping damage before it's too late is extraordinarily important. So these are going to be difficult decisions physicians are going to have there.

Speaker Change: To work that out and I'm sure there'll be no white papers and discussions among thought leaders on that topic with the issue today is slightly different one the prioritization today. Given the label that we have is to ensure that we get kids on the label and this really comes from a physician.

Speaker Change: Given the label that we have is to ensure that we get kids on the label and this really comes from a physician.

Speaker Change: More than from us to get kids on the label or the age out and the therapy won't be available to them. So if you looked at the bias here the bias is tends to be to the.

If you look at the bias here, the bias tends to be in the tight age range of 4-5, to be in the later ages, because they're really trying to get to those kids to [inaudible], get through the administrative process, will age out to 6 years old. The good news is, that if we can expand this label, that issue goes to the side then we'll have to deal with the other issues about prioritization and the like but it, ultimately, will be a physician decision and the issue of the practice of medicine for physicians.

Doug Ingram: So if you looked at the bias here, the bias tends to be to the in this tight age range of 4 and 5 to be in the later ages because they're really trying to get to those kids who, if they don't get through the administrative process, will age out to 6 years old. The good news is that if we can expand this label, that issue goes to the side, and then we'll have to deal with the other issues about prioritization and the like. But it ultimately will be a physician decision and the issue of the practice of medicine for physicians.

Speaker Change: Tight age range of 45 to be in the later ages, because they're really trying to get to those kids to get through the administrative process will age up to six years old. The good news is that if we can expand this label that issue goes to the to decide when will you have to deal with the other issue.

Speaker Change: It is about prioritization and the like but it ultimately will be a physician decision.

Speaker Change: A decision on the issue of the practice of medicine for physicians.

Operator: Thank you. Our next question comes from the line of Ritu Baral with TD Cowen. Your line is now open.

Ritu Baral: Hi, guys. Thanks for taking the question. I want to just follow up on the sort of guidance given, your comment on how not to expect additional growth in ELEVIDYS through the first half of the year, at least until the PDUFA. Is that a comment or a reflection on just the fact that you've reached sort of a run rate on 4- to 5-year-olds, or is that a comment on sort of capacity of the system's throughput? Are you getting through as many patients as possible? And can you comment on what that sort of throughput capacity of the centers is at this point for when, hopefully, the label expands? Thanks.

Ritu Baral: Hi, guys. Thanks for taking the question. I want to just follow up on the, sort of, guidance given your comment on how not to expect additional growth in ELEVIDYS through the first half of the year, at least until the PDUFA. Is that a comment or a reflection on just the fact that you've reached a run rate on 4 to 5-year olds? Or is that a comment on, sort of, capacity of the systems throughput? Are you getting through as many patients as possible? And can you comment on what that throughput capacity of the centers is, at this point, for when hopefully, the label expands? Thanks.

Richard V. Miller: Is that a comment or a reflection on just the fact that you've reached sort of a run rate on four to five year old or is that a comment on sort of capacity of the systems that we put.

Richard V. Miller: Are you getting through as many patients as possible and can you comment on what that sort of.

Richard V. Miller: Throughput capacity up like the center is at this point for when hopefully the legal expense.

Doug Ingram: Yeah. This really isn't a capacity issue right now. So I think, as Dallan commented in his opening remarks, the label that we have today in this population, it's really a fairly unique population. First, as we all know, it's a very narrow age range, four to five years old. But that alone doesn't really describe the unique nature of this population because, beyond that, there's a significant percentage of those patients that are not yet diagnosed. And so that makes it unusual. And then when they are diagnosed, remember that they're not all getting diagnosed on four years and one day. Some of these kids are getting diagnosed deep into their fives. And so what does that mean?

Speaker Change: Yeah. This really isn't a capacity issue right now. I think it was Dallen's comment in his opening remarks, the label that we have today in this population is -- it's really a fairly unique population. First, as we all know, it's a very narrow age range, 4 to 5 years old. But that alone doesn't really describe the unique nature of this population because beyond that, there is a significant percentage of those patients that are not yet diagnosed. And so, that makes it unusual. And then, when they are diagnosed -- remember, they're not all getting diagnosed on 4 years and 1 day, some of these kids are getting diagnosed deep into their 5s. And so, what does that mean? It means that a family is faced with, what is very likely, the most devastating piece of news so far in their life, has to come to grips with that news. And I understand it well enough. And then go through the administrative process that exists to get approval, to get antibody test, to get an infusion date that they get infused. So, this is a very unique population in light of that.

Yeah. This really isn't a capacity issue right now. I think it was Dallen's comment in his opening remarks, the label that we have today in this population is -- it's really a fairly unique population. First, as we all know, it's a very narrow age range, 4 to 5 years old. But that alone doesn't really describe the unique nature of this population because beyond that, there is a significant percentage of those patients that are not yet diagnosed. And so, that makes it unusual. And then, when they are diagnosed -- remember, they're not all getting diagnosed on 4 years and 1 day, some of these kids are getting diagnosed deep into their 5s. And

Douglas S. Ingram: Yeah. This really isn't a capacity issue right now. I think it was Dallen's comment in his opening remarks, the label that we have today in this population is -- it's really a fairly unique population. First, as we all know, it's a very narrow age range, 4 to 5 years old. But that alone doesn't really describe the unique nature of this population because beyond that, there is a significant percentage of those patients that are not yet diagnosed. And so, that makes it unusual. And then, when they are diagnosed -- remember, they're not all getting diagnosed on 4 years and 1 day, some of these kids are getting diagnosed deep into their 5s.

Speaker Change: Five years old, but that alone doesn't really describe the unique nature of this.

Speaker Change: Population because beyond that.

Speaker Change: A significant percentage.

Speaker Change: Those patients that are not yet diagnosed and so that.

Speaker Change: It makes it unusual and then when they are diagnosed remember they're not all getting diagnosed on four years and one day you know some of these guys are getting diagnosed deep into their fives and so what does that mean it means that a family is faced with what is very likely the most devastating piece of news.

And so, what does that mean? It means that a family is faced with, what is very likely, the most devastating piece of news so far in their life, has to come to grips with that news. And I understand it well enough. And then go through the administrative process that exists to get approval, to get antibody test, to get an infusion date that they get infused. So, this is a very unique population in light of that. Just to give a shout out to the team, it is particularly impressive when one considers that we did $200 million in that product revenue this year. So, what Dallen's really talking about when we're talking about this, is just simply that, as we work through the prevalent population, this is a really unique population and much narrower than you might imagine a 4-5. And so, by the middle of this year, assuming no label expansion, you would get to a place where you'd begin to be treating the incident population as opposed to a bolus of prevalent population in this group. Now, the good news is that we have a June 21 date for our request for label expansion so, hopefully, by July we'll have a broader label and will be able to serve a much greater percentage of the population.

so, what does that mean? It means that a family is faced with, what is very likely, the most devastating piece of news so far in their life, has to come to grips with that news. And I understand it well enough. And then go through the administrative process that exists to get approval, to get antibody test, to get an infusion date that they get infused. So, this is a very unique population in light of that.

Doug Ingram: It means that a family faced with what is very likely the most devastating piece of news so far in their life has to come to grips with that news, then understand it well enough, and then go through the administrative process that exists to get approval, to get antibody tests, to get an infusion date, and to get infused. So this is a fairly unique population. In light of that, just to give a shout-out to the team, it is particularly impressive when one considers that we did $200 million in that product revenue this year. So what Dallan's really talking about when we talk about this is just simply that, as we work through the prevalent population, this is a really unique population and much narrower than you might imagine the 4 to 5.

Speaker Change: So far in the Hawaii has to come to grips with that news.

Richard V. Miller: And I understand it well enough and then go through the administrative process that exists.

Richard V. Miller: To get approval to get antibody test to get an infusion days they get into years. So this is this is a very unique.

Richard V. Miller: Population in light of that.

Richard V. Miller: Just to give a shout out to the team. It is particularly impressive when one considers that we did $200 million in that product revenue. This year. So what Dan was really talking about when we're talking about this is just simply that whereas we worked through the prevalent population. This is a really huge.

So, what Dallen's really talking about when we're talking about this, is just simply that, as we work through the prevalent population, this is a really unique population and much narrower than you might imagine a 4-5. And so, by the middle of this year, assuming no label expansion, you would get to a place where you'd begin to be treating the incident population as opposed to a bolus of prevalent population in this group. Now, the good news is that we have a June 21 date for our request for label expansion so, hopefully, by July we'll have a broader label and will be able to serve a much greater percentage of the population.

Richard V. Miller: <unk> population.

Richard V. Miller: And then much narrower than you might imagine the four to five and so by the middle of this year, assuming no label expansion you would get to a place where you would begin to be treating the incident population as opposed to a bolus of <unk>.

Doug Ingram: So by the middle of this year, assuming no label expansion, you would get to a place where you'd begin to be treating the incident population as opposed to a bolus of prevalent population in this group. Now, the good news is that we have a 21 June date for our request for label expansion. So hopefully, by July, we'll have a broader label, and we'll be able to serve a much greater percentage of the population.

Richard V. Miller: <unk> population in this group.

Richard V. Miller: Good news is that we have a June 21 day. For our request for label expansion. So hopefully by July we'll have a broader label and won't be able to serve. Greater percentage of the population.

Richard V. Miller: For our request for label expansion. So hopefully by July we'll have a broader label and won't be able to serve.

Richard V. Miller: Greater percentage of the population.

Operator: Thank you. Our next question comes from the line of David Hong with Citi. Your line is now open.

Operator: Thank you. Our next question comes from the line of David Hoang with Citigroup. Your line is now open.

Speaker Change: Our next question comes from the line of David Hong with Citigroup. Your line is now open.

Yigal Nochomovitz: Hi. Thanks for taking the question. I just want to ask a little bit more about the suspension culture manufacturing you have in the works. How fast could something like that come online? And once that is operationalized, how might that improve your margins?

David Hong: Hi. Thanks for taking the question. I just want to ask a little bit more about the suspension culture manufacturing you have in the works. How fast could something like that come online? And once that is operationalized, how might that improve your margins?

David Hoang: Right, thanks for taking the question. I just want to ask a little bit more about the suspension culture manufacturing you have in the works. How fast does something like that come online and once that is operational, how might that improve your margins?

David Timothy Hoang: It's something like that come online and once that is operation like how might that improve your margins.

Doug Ingram: So we're very excited about where we are from a suspension perspective. There's still a ton of more work to do, but a couple of things to think about. We are very advanced, actually, in the suspension work that we're doing, not just with 9001, but we're actually getting great results from a suspension perspective with some of our limb-girdle programs. A couple of our sarcoglycan programs actually transitioned to suspension, and we're making great progress there. When we focus specifically on ELEVIDYS, we are in engineering runs right now on suspension. We've done 500-liter. We're doing another 500-liter. We'll start in the next month or so, 2,000-liter. We're so far getting not only really tremendous enhancement on yields but really good product qualities. If all goes well, our goal is to have this suspension process available commercially by 2026. Now, that's a moonshot goal on our part.

Richard V. Miller: So, we're very excited about where we are from a suspension perspective. There's still a ton of more work to do but -- a couple of things to think about. We are very advanced, actually, in the suspension work that we're doing. Not just in 9001 but we're actually getting great results, from a suspension perspective, with some of our limb-girdle programs. A couple of sarcoglycan programs actually transition to suspension and we're making great progress there. When we focus specifically on ELEVIDYS, we are on engineering runs right now on suspension. We've done 500 liter, we're doing another 500 liter; we'll start in the next month or so, 2000 liter. We're, so far, getting not only really tremendous enhancement on yield but really good product qualities. If all goes well, our goal is to have this suspension process available commercially by 2026. Now, that's a moonshot goal on our part with keeping the patients front and center, we're going to try to move as fast as possible. But, hopefully, we have the history to prove that we can come up with audacious plans and serve them. So, we're really excited about that. As far as what that might do, both from a capacity perspective as we go around the world with our partner Roche and a COGS perspective, that's going to require more work and more clarification as we get these runs done, but it won't be modest. It'll be multiples. And we are seeing, you know, multiple times greater yields with suspension than we get with our iCELLis process, which Ian mentioned still drives, right now, an 80% margin. So, I mean, you probably hear in my voice a certain amount of enthusiasm for our manufacturing approach and our suspension approach. It is still early days and we're just in engineering runs right now but we're very, very excited about what this could mean for our ability to bring this therapy across the world and at a lower cost of goods as we go across the world.

Douglas S. Ingram: So, we're very excited about where we are from a suspension perspective. There's still a ton of more work to do but -- a couple of things to think about. We are very advanced, actually, in the suspension work that we're doing. Not just in 9001 but we're actually getting great results, from a suspension perspective, with some of our limb-girdle programs. A couple of sarcoglycan programs actually transition to suspension and we're making great progress there. When we focus specifically on ELEVIDYS, we are on engineering runs right now on suspension. We've done 500 liter, we're doing another 500 liter; we'll start in the next month or so, 2000 liter.

David Timothy Hoang: We're very excited about where we are from from a suspension perspective, there's still a ton of more work to do but.

David Timothy Hoang: A couple of things to think about where we are.

David Timothy Hoang: Advanced actually and the suspension of work that we're doing not just your bank tier one, but we're actually.

David Timothy Hoang: Getting great results from our suspension perspective, with some of our limb girdle programs a couple of our cycle glide Cam programs actually transition to suspension and we're making great progress there when we focused specifically on <unk> we are.

David Timothy Hoang: In engineering runs right now on suspension, we've done 500 leader we're doing another 500 leader will do we will start in the next month or so 2000 leader.

We're, so far, getting not only really tremendous enhancement on yield but really good product qualities. If all goes well, our goal is to have this suspension process available commercially by 2026. Now, that's a moonshot goal on our part with keeping the patients front and center, we're going to try to move as fast as possible. But, hopefully, we have the history to prove that we can come up with audacious plans and serve them. So, we're really excited about that. As far as what that might do, both from a capacity perspective as we go around the world with our partner Roche and a COGS perspective, that's going to require more work and more clarification as we get these runs done, but it won't be modest. It'll be multiples. And we are seeing, you know, multiple times greater yields with suspension than we get with our iCELLis process, which Ian mentioned still drives, right now, an 80% margin. So, I mean, you probably hear in my voice a certain amount of enthusiasm for our manufacturing approach and our suspension approach. It is still early days and we're just in engineering runs right now but we're very, very excited about what this could mean for our ability to bring this therapy across the world and at a lower cost of goods as we go across the world.

David Timothy Hoang: So far getting.

David Timothy Hoang: Not only.

David Timothy Hoang: Really tremendous tremendous enhancement on yields, but really good product qualities if all goes.

David Timothy Hoang: Well our goal is to have this suspension process available commercially by 2026.

David Timothy Hoang: Moonshot goal on our part.

Doug Ingram: And with keeping the patients front and center, we're going to try to move as fast as possible. But hopefully, we have the history to prove that we can come up with audacious plans and serve them. So we're really excited about that. As far as what that might do, both from a capacity perspective as we go around the world with our partner, Roche, and a COGS perspective, that's going to require more work and more clarification as we get these runs done. But it won't be modest. It'll be multiples. I mean, we are seeing multiple times greater yields with suspension than we get with our iCELLis process, which, as Ian mentioned, still derives right now an 80% margin. So I mean, you're probably hearing my voice, a certain amount of enthusiasm for our manufacturing approach and our suspension approach.

David Timothy Hoang: Keeping the patients Rotten center, we're going to try to move as fast as possible.

David Timothy Hoang: But hopefully we have the history to prove that we can make we can come up with plans and serve them. So we're really excited about that as far as what that might do both from a capacity perspective as we go around the world with our partner Roche and a Cogs perspective.

As far as what that might do, both from a capacity perspective as we go around the world with our partner Roche and a COGS perspective, that's going to require more work and more clarification as we get these runs done, but it won't be modest. It'll be multiples. And we are seeing, you know, multiple times greater yields with suspension than we get with our iCELLis process, which Ian mentioned still drives, right now, an 80% margin. So, I mean, you probably hear in my voice a certain amount of enthusiasm for our manufacturing approach and our suspension approach. It is still early days and we're just in engineering runs right now but we're very, very excited about what this could mean for our ability to bring this therapy across the world and at a lower cost of goods as we go across the world.

David Timothy Hoang: They require more.

David Timothy Hoang: Work and more clarification as we get these runs done but it won't be modest it'll be multiples and we are seeing you know.

David Timothy Hoang: Multiple times Gray.

David Timothy Hoang: Greater yields with suspension than we get with.

David Timothy Hoang: With our <unk> process, which as you know.

David Timothy Hoang: You mentioned still drives right now an 80% margin so.

David Timothy Hoang: I mean, you probably hear in my voice, a certain amount of enthusiasm for our manufacturing approach and our suspension approach.

Doug Ingram: It is still early days, and we're just in engineering runs right now. We're very, very excited about what this could mean for our ability to bring this therapy across the world and at a lower cost of goods as we go across the world.

David Timothy Hoang: It is still early days and we're just in engineering runs right now, but we're very very excited about what this could mean for our ability to bring this therapy.

David Timothy Hoang: Across the world and at a lower cost of goods as we go across the world.

Operator: Thank you. Our next question comes from the line of Michael Ulz with Morgan Stanley. Your line is now open.

Unknown: Thank you. Our next question comes from the line of Mike Ulz with Morgan Stanley. Your line is now open.

Matthew Harrison: Hey, guys. Thanks for taking the question. Maybe just a quick one on timing. Since the PDUFA date was granted two months ahead of expectations, just curious if the thinking now is you'll probably get a decision close to the PDUFA date, or is the FDA still really committed to rapidly reviewing this, and we might get an answer sooner? Thanks.

Michael Ulz: Hey, guys. Thanks for taking the question. Maybe just a quick one on timing. Since the PDUFA date was granted two months ahead of expectations, just curious if the thinking now is you'll probably get a decision close to the PDUFA date, or is the FDA still really committed to rapidly reviewing this, and we might get an answer sooner? Thanks.

Mike Ulz: Hey, guys. Thanks for taking the question. Maybe just a quick one on timing -- since the PDUFA date was granted two months ahead of expectations, just curious if the thinking now is, you'll probably get a decision close to the PDUFA date or is the FDA still really committed to rapidly reviewing this and we might get an answer sooner. Thanks.

Michael: I'm just curious if the thinking now is you'll probably get a decision close to the Paducah date or is the FDA still really committed to rapidly reviewing this and make it an answer sooner.

Doug Ingram: Well, I have no reason to believe that the FDA isn't committed to rapidly reviewing this. With that said, there's still a lot of work to be done, and I think it would be prudent for all of us to assume that our target completion date of 21 June is the date on which we're going to get the answer on this. So we're planning for 21 June, even though we are, I don't think anyone thinks we fiddle around. We try to move as fast as possible. But we're assuming 21 June is when we're going to get our label expansion request answered.

Douglas S. Ingram: Well, I have no reason to believe that the FDA isn't committed to rapidly reviewing this. With that said, there's still a lot of work to be done and I think it would be prudent for all of us to assume that our target completion date of June 21 is the date on which we're going to get the answer on this. We're planning for June 21 even though we are -- I don't think anyone thinks we settled down, we try to move as fast as possible but we're assuming June 21 is when we're going to get our label expansion request answered.

Speaker Change: Committed to rapidly reviewing this with that said.

Speaker Change: There's still a lot of work to be done and I think it would be prudent for all of us to assume that.

David Timothy Hoang: Our.

David Timothy Hoang: The target completion date of June 21 is the date on which we're going to get the answer on this.

David Timothy Hoang: For June 'twenty one.

David Timothy Hoang: Even though we are you know I don't think anyone thinks we settle around we try to move as fast as possible, but we're assuming June 'twenty, one that's when we're going to get on it.

David Timothy Hoang: Label expansion request answers.

Operator: Thank you. Our next question comes from the line of Danielle Brill with Raymond James. Your line is now open.

Speaker Change: Our next question comes from the line of Danielle Brill with Raymond James Your line is now open.

Danielle Brill: Hey, guys. This is Alex on for Danielle. Thanks for taking the question. I know the PMO confirmatory trials are fully enrolled. Could you just remind us whether we should expect data from the MISSION this year?

David Timothy Hoang: Hey, guys. This is Alex on for Danielle Thanks for taking the question. No. The PMO confirmatory trials are fully enrolled could you just remind us whether. We should expect data for submission this year. No we shouldnt it doesn't read out until 2020 steps.

Unknown: Hey, guys. This is Alex, on for Danielle. Thanks for taking the question. Now the PMO confirmatory trials are fully enrolled, could you just remind us whether we should expect data for the MIS51ON this year?

Alex: No. The PMO confirmatory trials are fully enrolled could you just remind us whether.

Alex: We should expect data for submission this year.

Doug Ingram: No, we shouldn't. It doesn't read out till 2026.

Douglas S. Ingram: No, we shouldn't -- it doesn't read out until 2026.

Speaker Change: No we shouldnt it doesn't read out until 2020 steps.

Operator: Thank you. Our next question comes from the line of Brian Skorney with Baird. Your line is now open. Brian Skorney with Baird. Your line is now open.

Operator: Thank you. Our next question comes from the line of Brian Skorney with Baird. Your line is now open.

Alex: Our next question comes from the line of Brian <unk> with Baird. Your line is now open.

Alex: Brian <unk> with Baird. Your line is now open.

Charlie Moore: Hi. This is Charlie Moore on for Brian. Thanks for taking our question. So we were just wondering if you've heard anything from Roche regarding timelines with the EMA, as well as if the European opportunity were to come online, would there be any supply limitations resulting from that? I know you're very comfortable with the expansion of the label, but bringing on a whole nother continent, do you think you'd have to do any prioritization between regions? Thank you.

Unknown: Hi, this is Charlie, on for Brian. Thanks for taking our question. So, we were just wondering if you've heard anything from Roche regarding timelines with the EMA, as well as, if the European opportunity were to come online, would there be any supply limitations resulting from that? I know you're very comfortable with the expansion of the label but bringing on a whole another continent, do you think you'd have to do any prioritization between regions. Thank you.

Charlie: If you've heard anything from Roche regarding timelines with the EMEA as well as.

Charlie: If the European opportunity were to come on line would there be any supply limitations, resulting from that I know, you're very comfortable with the expansion of the label, but bringing on a whole. Another constant do you think you'd have to do any prioritization between regions. Thank you.

Doug Ingram: Roche is publicly saying that their goal is to submit in 2024. And then as relates to supply, our supply plans include our partner, Roche.

Douglas S. Ingram: Roche is publicly saying that their goal is to submit in 2024. And then, as it relates to supply, our supply plans include our partner, Roche.

Charlie: In 2024.

Charlie: And then as it relates to supply our supply plans include our partner Roche.

Operator: Thank you.

Doug Ingram: Just one quick follow-up just to the previous question around MISSION and the timing. Just one thing to remind everyone is that this is a dose compared to doses of the drug. And so the data which we'll read out will be comparative from the 30 mg per kg compared to either the 100 mg or 200 mg per kg, and there's no risk of the drug being pulled off the market.

Speaker Change: Thank you and just one quick follow up to that. The previous question around mission and the timing just one thing to remind everyone that this is a dose. Compares the doses of the drug and so. The data, which we'll read out will be comparative from the 30 Meg per kg compare it to either 100, Meg or 200, Meg per kg and there's no risk of the drug. Being pulled off the market.

Operator: Thank you.

Dallan Murray: And just one quick follow up to the previous question around MIS51ON and the timing. Just one thing to remind everyone that this is a dose - it compares the doses of the drug and so, the data which we'll read out will be comparative from the 30mg/kg compared to either 100mg or 200mg/kg and there's no risk of the drug being pulled off the market.

Speaker Change: The previous question around mission and the timing just one thing to remind everyone that this is a dose.

Charlie: Compares the doses of the drug and so.

Charlie: The data, which we'll read out will be comparative from the 30 Meg per kg compare it to either 100, Meg or 200, Meg per kg and there's no risk of the drug.

Charlie: Being pulled off the market.

Operator: Thank you. Our next question comes from the line of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Operator: Thank you. Next question comes from the line of Kristen Kluska with Cantor Fitzgerald. Your line is now open.

Charlie: Next question comes from the line of Kristen <unk> with Cantor Fitzgerald. Your line is now open.

Rick Miller: Hi. This is Rick Miller on for Kristen. Thanks for taking our question. Since launching, are you able to talk about if and how the profiles of DMD patients that are looking to get on ELEVIDYS have changed? Or to put it another way, I imagine you had highly engaged caregivers who were ready for ELEVIDYS on day one. So are you seeing more patients coming forward now after a few months of commercial experience or caregivers to get their child in line for ELEVIDYS? Thanks.

Rich Miller: Hi. This is Rich Miller, on for Kristen. Thanks for taking our question. Since launching, are you able to talk about if and how the profiles of DMD patients that are looking to get on ELEVIDYS have changed? Or to put it another way, I imagine you had highly engaged caregivers who are ready for ELEVIDYS on day one. So, are you seeing more patients coming forward now after a few months of commercial experience or caregivers to get their child in line for ELEVIDYS? Thanks.

Richard V. Miller: Since launching are you able to talk about if and how the profiles of DMD patients that youre looking to get on <unk> have changed or to put it another way I imagine you had highly engaged caregivers who are ready on for 11. If on day. One. So are you seeing more patients coming forward now after a few months of commercial experience.

Charlie: For us to get their child in line for <unk>. Thanks.

Doug Ingram: Yeah. And I would say in the broadest of strokes, there is going to be substantial demand for this therapy. I don't think that's changed. I think it's what we anticipated before launch. I don't think it's changed after launch. Duchenne muscular dystrophy is a devastating disease, and the opportunity that's offered by ELEVIDYS is an important one. I think physicians understand that. Families with Duchenne muscular dystrophy, boys understand that as well. But Dallan, if you have any additional color on this or views, please share them.

Douglas S. Ingram: Yeah. And I would say, in the broadest of strokes, there is going to be substantial demand for this therapy. I don't think that's changed, I think it's what we anticipated before launch. I don't think it's changed after launch. Duchenne muscular dystrophy is a devastating disease and the opportunity that's offered by ELEVIDYS is an important one. And I think physicians understand that families with Duchenne muscular dystrophy will always understand that as well. But, Dallan, if you have any additional color on this or views, please share them.

Speaker Change: I don't think Thats changed I think it's what we anticipated.

Speaker Change: Before launch I don't think its changed after launch.

Speaker Change: Duchenne muscular dystrophy is a devastating disease and.

Speaker Change: The opportunity that's offered by our limit is.

Speaker Change: <unk> is an important one and I think physicians understand that families with duchenne muscular dystrophy.

Speaker Change: Boy, if I understand that as well, but Alan if you have any additional color on this or your views please share them.

Dallan Murray: No, I think broadly speaking, you're exactly right, Doug. We're not seeing a real major change in terms of the patients coming in. It is, as we set up front, a small population. And so as soon as they're getting diagnosed and have had their discussions with the docs, they're coming in through the enrollment forms. And so we're just working through those patients as fast as we can prior to them aging out. But no major differences, for example, there wasn't a big kind of bolus of patients that initially came in, and now that's different. It's been a steady clip, essentially.

Dallan Murray: No. I think broadly speaking here, exactly right, Doug. We're not seeing a real major change in terms of the patients coming in, it is as we set upfront a small population and so, soon as they're getting diagnosed and they've had their discussions with the docs, they're coming in through the enrollment forms. And so we're just working through those patients as fast as they can -- as we can prior to them aging out. But no major differences in, for example, there wasn't a big kind of bolus of patients that initially came in and now that's different. It's just that, it's been a steady clip, essentially.

Dallan Murray: We're not seeing we're not seeing a real a real major change in terms of the patients coming in it as we set upfront a small population and so.

Dallan Murray: As soon as they're getting diagnosed and they've had their their discussions with the docs, they're coming in through the enrollment forms and so we're just working through those patients in as fast as they can as we can.

Dallan Murray: Prior to them aging out, but no major differences in.

Dallan Murray: For example, there wasn't a big kind of bolus of patients that initially came in and now that's different it's just that.

Dallan Murray: It's been a steady clip essentially.

Operator: Thank you. Our next question comes from the line of Anupam Rama with J.P. Morgan. Your line is now open.

Operator: Thank you. Our next question comes from the line of Anupam Rama with JP Morgan. Your line is now open.

Dallan Murray: Our next question comes from the line of <unk> Rama with Jpmorgan. Your line is now open.

Anupam Rama: Hey, guys. Thanks so much for taking the question. At the conference in January, you talked about 70 sites being active, and about half of those sites had dosed patients. Can you give us a sense of where you are kind of now in the first couple of months of the year? Thanks so much.

Anupam Rama: Hey, guys. Thanks so much for taking the question. At the conference in January, you talked about 70 sites being active and about half of those sites have dosed patients. Can you give us a sense of where you are now, in the first couple months of the year? Thanks so much.

Rama: At the conference in January you talked about 70 sites being active in about half of those sites and those patients can you give us a sense of where you are now in the first couple months of the year. Thanks, So much.

Doug Ingram: Yeah. I mean, I believe that that remains the accurate answer. But Dallan, you can give us an update if there's any change in that.

Speaker Change: Yeah. I believe that that remains the accurate answer but, Dallan, you could give us an update if there is any change in that. No on upon that it really hasn't hasn't changed all that much. There's obviously more than there was then. There was a higher proportion of sites that have dosed. But. But. We're actively looking at this on a on a daily basis and we have this flexible model, where we can bring on new sites get them trained and up and ready as needed. So so we've got more than enough. <unk> up and running and we. Throughout we've got great geographic coverage, but the team is also ready to support and serve as needed out there. I really do want to given. Kudos to this team for for where we are. I think everybody knows the goal was to try to get to 50 matches 50 sites in 2024, three by the end of 'twenty, three which would have been. Would've been record smashing for a gene therapy launch and that there was going to be this aspiration, but maybe someday we can get to 70 sites that would be trained and up and running and this team got as you know over 70 sites in 2023, but also the way. They got there is something that <unk>. As you me an enormous amount. Pride, which is as I said earlier in this call. We don't we don't prioritize revenue over patient safety and great outcomes. Far more important for us to ensure great outcomes and this team has done a ton in that direction 70 sites that are activated or activating because they've all been well trained very very good shape to be able to safely. Infuse this therapy and get good outcomes and consistent outcomes, which is important we have a very laudable safety profile for a full body infusion gene therapy, and we want to ensure that remains the case over the long term not the short term and then of course, there's a lot of other programs around that the ability to access experts. So that every physician has an opportunity to get the right kinds of information to inform the way they not only infuse that monitor patients and react in something that has been. That is unique I'm not sure anyone else has ever done the kind of work we've done. So we're in great shape right now to serve this community and serve it responsibly side I'm really proud of where the changes. And frankly it also explains our. Our revenue in 2023.

Douglas S. Ingram: Yeah. I believe that that remains the accurate answer but, Dallan, you could give us an update if there is any change in that.

Dallan Murray: No, Anupam, that really hasn't changed all that much. There's obviously more than there was then, and there's a higher proportion of sites that have dosed. But we're actively looking at this on a daily basis, and we have this flexible model where we can bring on new sites, get them trained enough and ready as needed. So we've got more than enough sites up and running, and throughout, we've got great geographic coverage, but the team is also ready to support and serve as needed out there.

No, Anupam, it really hasn't changed all that much. There's, obviously, more than there was then and there's a higher proportion of sites that have dosed but we're actively looking at this on a daily basis and we have this flexible model where we can bring on new sites, get them trained and up and ready as needed. So, we've got more than enough sites up and running and throughout, we've got great geographic coverage but the team is also ready to support and serve as needed out there. I really do want to given. Kudos to this team for for where we are. I think everybody knows the goal was to try to get to 50 matches 50 sites in 2024, three by the end of 'twenty, three which would have been. Would've been record smashing for a gene therapy launch and that there was going to be this aspiration, but maybe someday we can get to 70 sites that would be trained and up and running and this team got as you know over 70 sites in 2023, but also the way. They got there is something that <unk>. As you me an enormous amount. Pride, which is as I said earlier in this call. We don't we don't prioritize revenue over patient safety and great outcomes. Far more important for us to ensure great outcomes and this team has done a ton in that direction 70 sites that are activated or activating because they've all been well trained very very good shape to be able to safely. Infuse this therapy and get good outcomes and consistent outcomes, which is important we have a very laudable safety profile for a full body infusion gene therapy, and we want to ensure that remains the case over the long term not the short term and then of course, there's a lot of other programs around that the ability to access experts. So that every physician has an opportunity to get the right kinds of information to inform the way they not only infuse that monitor patients and react in something that has been. That is unique I'm not sure anyone else has ever done the kind of work we've done. So we're in great shape right now to serve this community and serve it responsibly side I'm really proud of where the changes. And frankly it also explains our. Our revenue in 2023.

Dallan Murray: No, Anupam, it really hasn't changed all that much. There's, obviously, more than there was then and there's a higher proportion of sites that have dosed but we're actively looking at this on a daily basis and we have this flexible model where we can bring on new sites, get them trained and up and ready as needed. So, we've got more than enough sites up and running and throughout, we've got great geographic coverage but the team is also ready to support and serve as needed out there.

Gallon: No on upon that it really hasn't hasn't changed all that much. There's obviously more than there was then.

Speaker Change: There was a higher proportion of sites that have dosed.

Speaker Change: But.

Gallon: We're actively looking at this on a on a daily basis and we have this flexible model, where we can bring on new sites get them trained and up and ready as needed. So so we've got more than enough.

Dallan Murray: <unk> up and running and we.

Dallan Murray: Throughout we've got great geographic coverage, but the team is also ready to support and serve as needed out there.

I really do want to give kudos to this team for where we are. I think everybody knows the goal was to try to get to as much as 50 sites in 2024, by the end of 2023, which would have been record-smashing for a gene therapy launch. And that there was going to be this aspiration that maybe someday we can get to 70 sites that would be trained and up and running. And this team got, as you know, over 70 sites in 2023. But also the way they got there is something that gives me an enormous amount of pride, which is -- as I said earlier in this call, we don't prioritize revenue over patient safety and great outcomes. Far more important for us to ensure great outcomes and this team has done a ton in that direction. These 70 sites that are activated or activating because they've all been well-trained, very good shape to be able to safely infuse this therapy and get good outcomes and consistent outcomes, which is important. We have a very laudable safety profile for a full body infusion gene therapy and we want to ensure that remains the case over the long-term, not the short-term. And then, of course, there's a lot of other programs around that, the ability to access experts so that every physician has an opportunity to get the right kinds of information, to inform the way they not only infuse but monitor patients and react in something that has been -- that is unique. I'm not sure anyone else has ever done the kind of work we've done. So, we're in great shape right now to serve this community and serve it responsibly. I'm really proud of where the team is right now. And, frankly, it also explains our revenue in 2023.

Douglas S. Ingram: I really do want to give kudos to this team for where we are. I think everybody knows the goal was to try to get to as much as 50 sites in 2024, by the end of 2023, which would have been record-smashing for a gene therapy launch. And that there was going to be this aspiration that maybe someday we can get to 70 sites that would be trained and up and running. And this team got, as you know, over 70 sites in 2023. But also the way they got there is something that gives me an enormous amount of pride, which is -- as I said earlier in this call, we don't prioritize revenue over patient safety and great outcomes.

Doug Ingram: I really do want to give kudos to this team for where we are. I think everybody knows the goal was to try to get to as much as 50 sites in 2023, by the end of 2023, which would have been record-smashing for a gene therapy launch, and that there was going to be this aspiration that maybe someday we could get to 70 sites that would be trained and up and running. And this team got, as you know, over 70 sites in 2023. But also the way they got there is something that gives me an enormous amount of pride, which is, as I said earlier in this call, we don't prioritize revenue over patient safety and great outcomes. Far more important for us to ensure great outcomes. And this team has done a ton in that direction.

Dallan Murray: I really do want to given.

Dallan Murray: Kudos to this team for for where we are.

Dallan Murray: I think everybody knows the goal was to try to get to 50 matches 50 sites in 2024, three by the end of 'twenty, three which would have been.

Dallan Murray: Would've been record smashing for a gene therapy launch and that there was going to be this aspiration, but maybe someday we can get to 70 sites that would be trained and up and running and this team got as you know over 70 sites in 2023, but also the way. They got there is something that <unk>.

But also the way they got there is something that gives me an enormous amount of pride, which is -- as I said earlier in this call, we don't prioritize revenue over patient safety and great outcomes. Far more important for us to ensure great outcomes and this team has done a ton in that direction. These 70 sites that are activated or activating because they've all been well-trained, very good shape to be able to safely infuse this therapy and get good outcomes and consistent outcomes, which is important. We have a very laudable safety profile for a full body infusion gene therapy and we want to ensure that remains the case over the long-term, not the short-term. And then, of course, there's a lot of other programs around that, the ability to access experts so that every physician has an opportunity to get the right kinds of information, to inform the way they not only infuse but monitor patients and react in something that has been -- that is unique. I'm not sure anyone else has ever done the kind of work we've done. So, we're in great shape right now to serve this community and serve it responsibly. I'm really proud of where the team is right now. And, frankly, it also explains our revenue in 2023.

But also the way they got there is something that gives me an enormous amount of pride, which is -- as I said earlier in this call, we don't prioritize revenue over patient safety and great outcomes.

Dallan Murray: As you me an enormous amount.

Dallan Murray: Pride, which is as I said earlier in this call. We don't we don't prioritize revenue over patient safety and great outcomes.

Far more important for us to ensure great outcomes and this team has done a ton in that direction. These 70 sites that are activated or activating because they've all been well-trained, very good shape to be able to safely infuse this therapy and get good outcomes and consistent outcomes, which is important. We have a very laudable safety profile for a full body infusion gene therapy and we want to ensure that remains the case over the long-term, not the short-term. And then, of course, there's a lot of other programs around that, the ability to access experts so that every physician has an opportunity to get the right kinds of information, to inform the way they not only infuse but monitor patients and react in something that has been -- that is unique. I'm not sure anyone else has ever done the kind of work we've done. So, we're in great shape right now to serve this community and serve it responsibly. I'm really proud of where the team is right now. And, frankly, it also explains our revenue in 2023.

Dallan Murray: Far more important for us to ensure great outcomes and this team has done a ton in that direction 70 sites that are activated or activating because they've all been well trained very very good shape to be able to safely.

Doug Ingram: These 70 sites that are activated are activated because they've all been well-trained. They're in good shape to be able to safely infuse this therapy and get good outcomes and consistent outcomes, which is important. We have a very laudable safety profile for a full-body infusion gene therapy, and we want to ensure that remains the case over the long term, not the short term. And then, of course, there's a lot of other programs around that, the ability to access experts so that every physician has an opportunity to get the right kinds of information to inform the way they not only infuse but monitor patients and react, is something that is unique. I'm not sure anyone else has ever done the kind of work we've done. So we're in great shape right now to serve this community and serve it responsibly.

We have a very laudable safety profile for a full body infusion gene therapy and we want to ensure that remains the case over the long-term, not the short-term. And then, of course, there's a lot of other programs around that, the ability to access experts so that every physician has an opportunity to get the right kinds of information, to inform the way they not only infuse but monitor patients and react in something that has been -- that is unique. I'm not sure anyone else has ever done the kind of work we've done. So, we're in great shape right now to serve this community and serve it responsibly. I'm really proud of where the team is right now. And, frankly, it also explains our revenue in 2023.

Dallan Murray: Infuse this therapy and get good outcomes and consistent outcomes, which is important we have a very laudable safety profile for a full body infusion gene therapy, and we want to ensure that remains the case over the long term not the short term and then of course, there's a lot of other programs around that the ability to access experts.

And then, of course, there's a lot of other programs around that, the ability to access experts so that every physician has an opportunity to get the right kinds of information, to inform the way they not only infuse but monitor patients and react in something that has been -- that is unique. I'm not sure anyone else has ever done the kind of work we've done. So, we're in great shape right now to serve this community and serve it responsibly. I'm really proud of where the team is right now. And, frankly, it also explains our revenue in 2023.

Dallan Murray: So that every physician has an opportunity to get the right kinds of information to inform the way they not only infuse that monitor patients and react in something that has been.

Dallan Murray: That is unique I'm not sure anyone else has ever done the kind of work we've done. So we're in great shape right now to serve this community and serve it responsibly side I'm really proud of where the changes.

Doug Ingram: So I'm really proud of where the team is right now. And frankly, it also explains our revenue in 2023.

Dallan Murray: And frankly it also explains our.

Dallan Murray: Our revenue in 2023.

Dallan Murray: Thanks, Doug. As you were speaking, that team did get us a direct answer to Anupam's question. We're closing in on 60% of the sites have dosed. Exactly as you said, it's just increasing as we go.

Dallan Murray: Thanks, Doug. And as you were speaking, that team did get us a direct answer to Anupam's question and we're closing in on 60% of the sites have dosed. So, exactly as you said, it's just increasing as we go.

Speaker Change: As a direct answer to honour Pons question and we're closing in on 60% of the of the sites have dose. So it just exactly as you said, it's just increasing as we go.

Doug Ingram: All right. Thank you.

Operator: Thank you. Now I'd like to turn the call back over to Doug Ingram for closing remarks.

Anupam Rama: Alright, thank you.

Operator: Thank you. I'd now like to turn the call back over to Doug Ingram for closing remarks.

Doug Ingram: Thank you, Shannon. Thank you all for joining us today, and thank you for your insightful questions. We appreciate it. At the risk of repeating myself, I think 2023 was an extraordinarily important year for Sarepta. A lot of folks on this team, in concert with the patient community, investigators, and physicians, frankly, moved heaven and earth to get us where we are today, which is, I think, a fast-maturing, fully integrated biotech organization committed to bringing a better life to patients with Duchenne muscular dystrophy, limb-girdle, and other serious, rare diseases. 2024 is actually going to be even more significant if we're successful. That's going to put us in a position where we can do a lot of good for a lot of rare disease patients and where we can reward those who have stuck with us and invested in a better life for these patients.

Douglas S. Ingram: Thank you, Shannon. Thank you all for joining us today, thank you for your insightful questions. We appreciate it.

Douglas S. Ingram: At the risk of repeating myself, I think 2023 was an extraordinarily important year for Sarepta. A lot of folks on this team, in concert with the patient community and investigators and physicians, frankly, moved heaven and Earth to get us where we are today. Which is, I think, fast-maturing, fully integrated biotech organization committed to bringing a better life to patients with Duchenne muscular dystrophy, limb-girdles and other serious, rare diseases. 2024 is actually going to be even more significant, if we are successful. And that is going to put us in a position where we can do a lot of good for a lot of rare disease patients and where we can reward those who have stuck with us and invested in a better life for these patients. I will remind you, if we are successful this year and we broaden the label for ELEVIDYS, we can meet our goals this year. We are going to leverage our acumen and our financial strength and the talent of this team and through both advancing our internal pipeline but looking to external innovation, we will have the goal of growing even after ELEVIDYS. Not incrementally, but in multiples. So, we're going to build for ourselves a very big ambition if we are successful in 2024. With that said, we need to focus on 2024 and be successful and the team is prepared to do that and I look forward to updating you along the way, as well. So, thank you and have a lovely evening.

At the risk of repeating myself, I think 2023 was an extraordinarily important year for Sarepta. A lot of folks on this team, in concert with the patient community and investigators and physicians, frankly, moved heaven and Earth to get us where we are today. Which is, I think, fast-maturing, fully integrated biotech organization committed to bringing a better life to patients with Duchenne muscular dystrophy, limb-girdles and other serious, rare diseases. 2024 is actually going to be even more significant, if we are successful. And that is going to put us in a position where we can do a lot of good for a lot of rare disease patients and where we can reward those who have stuck with us and invested in a better life for these patients.

Douglas S. Ingram: A lot of folks on this team in concert with the patient community and <unk>.

Speaker Change: Investigators and physicians, frankly moved heaven and Earth to get US, where we are today, which is I think fine.

Speaker Change: Fast maturing fully integrated biotech organization committed to bringing a better life.

Speaker Change: For patients with Duchenne muscular dystrophy limb girdles and other serious.

Speaker Change: Rare diseases.

Speaker Change: 2021.

Speaker Change: Actually going to be even more significant if we are successful and that is going to put us in a position where we can do a lot of good for a lot of.

Speaker Change: Rare disease patients and where we can reward those who have stuck with us and invested in a better life.

I will remind you, if we are successful this year and we broaden the label for ELEVIDYS, we can meet our goals this year. We are going to leverage our acumen and our financial strength and the talent of this team and through both advancing our internal pipeline but looking to external innovation, we will have the goal of growing even after ELEVIDYS. Not incrementally, but in multiples. So, we're going to build for ourselves a very big ambition if we are successful in 2024. With that said, we need to focus on 2024 and be successful and the team is prepared to do that and I look forward to updating you along the way, as well. So, thank you and have a lovely evening.

Doug Ingram: So I will remind you, if we are successful this year and we broaden the label for ELEVIDYS and we can meet our goals this year, we are going to leverage our acumen and our financial strength and the talent of this team. And through both advancing our internal pipeline but looking to external innovation, we will have the goal of growing even after ELEVIDYS, not incrementally, but in multiples. So we're going to build for ourselves a very big ambition if we are successful in 2024. With that said, we need to focus on 2024 and be successful, and the team is prepared to do that. And I look forward to updating you along the way as well. So thank you, and have a lovely evening.

Speaker Change: Or are these patients so I will remind you with the if we are successful this year and we broaden the label for <unk> 11. So we can meet our goals. This year, we are going to leverage our acumen and our financial strength and the talent of this team and through both advancing our internal pipeline, but looking to external innovation.

Speaker Change: We will have the goal of growing.

Speaker Change: Even after <unk>.

Speaker Change: Not incrementally, but in multiples so we're going to build for ourselves a very big ambition. If we are successful in 2024 with that said, we need to focus on 2024 and be successful and the team is prepared to do that and I look forward to updating you along the way as well. So thank you and have a lovely evening.

Operator: This concludes today's conference call. Thank you for your participation. You may now disconnect.

Speaker Change: Okay. [music]. Okay. [music].

Speaker Change: [music].

Speaker Change: Okay.

Speaker Change: [music].

Q4 2023 Sarepta Therapeutics Inc Earnings Call

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