Q1 2021 Sarepta Therapeutics Inc Earnings Call

Good afternoon, ladies and gentlemen, and welcome to the <unk> Therapeutics first quarter 2021 earnings call. At this time all participants are in listen only mode. After the speaker's presentation. There will be a question and answer session to ask a question. During this session and you'll need to press star one on your telephone as a reminder, today's program is being recorded.

Operator: and gentlemen, and welcome to Therapeutics' first quarter 2021 earnings call. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you'll need to press star 1 on your telephone. As a reminder, today's program is being recorded. I'd now like to introduce your host for today's program, Mary Jenkins, Senior Vice President, Investor Relations. Please go ahead.

And I'd like to introduce your host for today's program.

<unk> Senior management Investor Relations. Please go ahead.

Thank you Jonathan and thank you all for joining today's call earlier today, we released our financial results for the first quarter 2020, one the precedents and available and I sit on our website and through the Dot Com and our 10-Q was filed with the Securities and Exchange Commission earlier this afternoon and joining us on the call today are Doug Ingram and extra time Alan Murray.

Mary Jenkins: Thank you, Jonathan, and thank you all for joining today's call. Earlier today, we released our financial results for the first quarter of 2021. The press list is available on our website at surrepta.com, and our 10Q was filed with the Securities and Exchange Commission earlier this afternoon. Joining us on the call today are Doug Engram, Annetteette, Dallan, Dr. Gilmore O'Neill, and Dr. Louise Rodina

And Dr. Gilmore O'neill and Dr. Luisa go back after our formal remarks, we'll open the call for Q&A.

Mary Jenkins: After our formal remarks, we'll open the call for Q&A. I'd like to note that during 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 Surreptus' control. Actual results could materially differ from these four booking statements, and any such risks can materially and adversely affect the business, the results of operations, and the trading prices for Surruptus Common Stock.

I'd like to note that during this call we were making a number of forward looking statements. Please take a moment to review our side and the webcast, which contains our forward looking statements. These forward looking statements involve risks and uncertainties many of which are beyond <unk> control actual results could materially differ.

From these forward looking statements and any such risks can materially and adversely affected.

Lots of operations and trading prices for swap with Comstock for a detailed description of applicable risks and uncertainties. We encourage you to review the company's most recent quarters and report on form 10-Q filed with the SEC as well as the company's other SEC filings. The company does not undertake any obligation to publicly update and forward looking statements, including any financial projections.

Mary Jenkins: For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report on Form 10Q 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 shifting cases. And now I'll turn the call over to our CEO, Doug Ingram. We'll provide an overview of our recent progress.

As I did today based on subsequent events or circumstances.

And now I'll turn the call over to our CEO, Doug Ingram, who will provide an overview of our recent progress.

Douglas S. Ingram: Thank you, Mary. Good afternoon, everyone, and thank you for joining us throughout the Therapeutics first quarter, 2021 investor conference call. I am pleased to share with you this afternoon the exceptional performance we achieved this quarter with respect to our three approved therapies and the significant progress we have made in advancing our multi-platform portfolio. Commencing with our quarterly performance, I'm very proud of Surreteam's commercial effectiveness. As we are a fully integrated commercial stage biotech with a focus on execution, we have a well-honed ability to address the often very sophisticated challenges of delivering to and serving patients with a weekly infused therapy for rare diseases.

Thank you Mary good afternoon, everyone and thank you for joining us throughout the therapeutics first quarter, 2020, one Investor Conference call.

I am pleased to share with you this afternoon and the exceptional performance we've achieved this quarter with respect to our three approved therapies.

And the significant progress we have made advancing our multi platform portfolio.

Commensurate with our quarterly performance I'm very proud of this throughout the team's commercial effectiveness and we are a fully integrated commercial stage biotech with a focus on execution.

Well, how and ability to address the off and very sophisticated challenges of delivering to and serving patients with the weekly and th therapy for rare disease for my first launch in late 2016, we have enjoyed compounded growth over the last for years of over 19% and.

Douglas S. Ingram: From our first launch in late 2016, we have enjoyed compounded growth over the last four years of over 19%. And as we have never taken a price increase and priced all our therapies at parity, this performance comes from our ability to serve the patient community. I'm very pleased to report that in the first quarter, our execution continued, and we achieved product revenue of approximately $125 million, and that represents a nearly 25% growth over the same quarter last year.

And as we have never taken a price increase and twice all other therapies at parity.

This performance comes from our ability to serve the patient community and.

And very pleased to report in the first quarter, our execution continued and we achieved product revenue of approximately $125 million and that represents a nearly 25% growth over the same quarter last year. Our performance comes from our three currently approved therapies as of February of this year, joining exon 51.

Douglas S. Ingram: Our performance comes from our three currently approved therapies. As of February of this year, joining Exondis 51 and Viandis 53, the FDA approved Amandis 45 to treat those 8% or more of Duchenne children with an Exxon 45 amenable mutation. Having prepared well, we were able to launch on day one, including labeling and shipping drugs to the warehouse to enable our first patient to be dosed within a week of our approval. With our third approval, we can now offer therapy to nearly 30% of children with Duchenne in the United States.

And my honest and 53, the FDA approved demand is 45 to treat those 8% or more duchenne children with an exon 45 amenable mutation.

And prepared well, we were able to launch on day, one, including labeling and shipping drug through the warehouse what warehouse to enable our first patient to be dosed within a week of our approval day.

With our third approval, we can now offer therapy to nearly 30% of children with Duchenne in the United States and from there we can theoretically build constructs to offer therapy to as many 80%, 80% or more of the Duchenne community.

Douglas S. Ingram: And from there, we can theoretically build constructs to offer therapy to as many 80% or more of the Duchenne community. We also have the opportunity and, and frankly, the mission-driven obligation to bring our U.S. and future RNA therapies to children outside of the United States. So as much as three internally designed, developed, and approved therapies place us in a small cadre of biotech companies. We have still barely begun to realize the opportunity available in our RNA. Moving now to our pipeline progress. I will stay with our RNA plat.

We also have the opportunity and frankly, the mission driven obligation to bring our U S approved and future RNA therapies to children and outside of the United States. So as much as three internally designed and developed and approved therapies waste.

Place us in a small cadre of biotech companies, we are still barely begun to realize the opportunity available in our RNA franchise.

Moving now to our pipeline progress I will stay with our RNA platform.

Douglas S. Ingram: As we announced just two days ago, we announced what we believe to be profoundly important results from Part A of the Momentum trial. This is our multi-ascending dose study for our peptide conjugated PMO, SRP 5051, our next generation construct. To remind you, our three approved PMO therapies for Duchen have been among the most significant advancements in the treatment of Duchen since the DMG gene was discovered in 1986. And they are currently the standard of care in the United States for children with the confirmed mutations that our therapies address. Still,

Just two days ago, we announced what we believe to be profoundly important results from part a of the momentum trial and so our multi ascending dose study for our peptide conjugated PMO SRP per seats that would be one of our next generation construct to.

To remind you our three approved PMO therapy for Duchenne and have been among the most significant advancements in the treatment of Duchenne since the DMG gene was discovered in 1986 and they are currently the standard of care and the United States for children with the confirmed mutations that our therapy.

Address still.

Douglas S. Ingram: Our goal is to continually improve in an effort to extend and improve the quality of life of those living with Dushan. And to that end, the hypothesis that we tested, Part A of our momentum trial, was that if we conjugated our proprietary, positively charged peptide to our PMO construct, we could achieve much greater tissue exposure, greater exon skipping, and therefore, greater distipine production. And if we could confirm that high popper, we would have a significant possibility of a greatly improved next generation of Shen Therapy that we could take not only for patients in the United States but outside the United States as well.

Our goal is to continually improve and an effort to extend and improve the quality of life of those living with duchenne and to that and the hypothesis that we tested and part a of our momentum trial.

Is that if we conjugated our proprietary positively charged peptide.

And our PMO current strip, we could achieve much greater tissue exposure greater exon skipping and therefore greater dystrophin production.

And if we can confirm that hypothesis, we would have a significant possibility of a greatly improved and next generation Duchenne therapy that we could take not only the patients and the United States.

But outside the United States as well.

Part a of our momentum trial resoundingly confirmed our hypothesis that our peptide could improve PK and PD.

Douglas S. Ingram: Part A of our momentum trial resoundingly confirmed our hypothesis that our peptide could improve PPD and induce significantly more distrave. At monthly versus weekly dosing of our 30-mig-k cohort, SRP 50-51, exhibited 18 times greater Exxon Skim and more than 400% greater district versus Exotus 51. And they did this with only 12% of the dose exposure. And it's just about half, we are able to safely confirm this level of dystrophin in the next phase of our trial. The PPMO will have transformed our RNA platform, not only in the United States, but internationally as well, multiplying the potential Duchenne population who will have access to our therapies. And beyond Duchenne, we can evaluate other disease areas where a more potent steric blocking could provide benefits, and have achieved a profound improvement in tissue exposure and distance.

And then do significantly more dystrophin.

And monthly versus weekly dosing of our 30 Meg per kg cohort.

P 50, 51 exhibited 18 times, greater exon skipping and more than 400% greater dystrophin versus exon 51, and they did this with only 12% the dose exposure.

And and just about half the time.

If we are able to safely confirm this level of dystrophin and the next phase of our trial.

The P. P M and will have transformed our RNA platform.

Not only in the United States, but internationally as well multiplying and a potential duchenne population, who will have access to our therapy and beyond Duchenne.

We can evaluate other disease areas and we're even more potent steric logging technology could provide benefit.

Having reached a profound improvement and tissue exposure and district.

Douglas S. Ingram: Our dose exploration of Part A is complete. This is an important moment for our program, for Serepta, and, most importantly, for the patient community. We will share these results with the FDA, and then we will discuss with the FDA the path for the commencement of Part B of momentum. Subject to further discussions with the FDA, our goal is to make Part B our pivotal trial and to seek accelerated approval in the United States if Part B is successful. Separately, we will engage with Ministries of Health, ex-us, about the clinical path for our PMO therapies outside of the United States, moving now to our GED therapy platform.

Are those exploration and part a is complete and.

This is an important moment for our program for Raptor and most importantly for the patient community. We will share. These results with the FDA and then we will discuss with the FDA the path for the commencement of part B of momentum subs.

Subject to further discussions with the FDA. Our goal is to make part b, our pivotal trial and to seek accelerated approval in the United States. If part B is successful separately, we will engage with ministries of health ex U S about the clinical path for our P. P. M O therapies outside of the United States.

Moving now to our gene therapy platform.

Douglas S. Ingram: We have made significant progress, and we have important milestones coming over the coming months and quarters to share with you. With respect to SRP 901, we have gained significant additional insight from part one of study 102, confirming our confidence that this therapy has the potential to be a significant advancement in the treatment of Duchenne and gaining insight that has helped us hone our protocol for our next clinical trial. Study 301, and we believe with our insight, we have enhanced the probability of success for 301.

We have made significant progress and we have important milestones coming over the coming months and quarters to share with you with respect to SRP 901, we have gained significant additional insight from part one of study 102, confirming our confidence that this therapy has the potential to be a significant advance.

And the treatment of Duchenne and gaining insight that has helped us owner a protocol for.

Our next clinical trial.

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And we believe with our insight we've enhanced the probability of success for three of what we have completed our analysis and refined our protocol and we will share this with the community. Once we have met with the division obtained their concurrence and have come.

Douglas S. Ingram: We have completed our analysis, they have refined our protocol, and we will share this with the community once we have met with the division, obtained their concurrence, and have commenced our next trial. Now, we have a significant number of milestones for SRP 9-001 over the coming months and quarters, and let me list them here for you.

Next our next trial and we have a significant number of milestones restaurant benign 001.

Over the coming months and quarters, and let me cataloged and here for you first this quarter, we will have the expression and safety results from our first cohort of study one O. Three that's our trial to confirm the characteristics of our commercially representative SRP 901 material.

Douglas S. Ingram: First, this quarter, we will have the expression and safety results from our first cohort of study 103. That's our trial to confirm the characteristics of our commercially representative SRP-9-001 material. All patients in study 103 have been dosed using the Surretha manufacturing process intended for all future clinical trials, and it has been approved for commercial supply as well. It is difficult to overstate the importance of this readout. As you will recall, in part one of study 102, the tittering method used by our clinical supply manufacturer created some variability and resulted in about 60% of patients receiving lower than Target Doe.

All patients in study one and three of the dose using the throughout the manufacturing process and <unk>.

And for all future clinical trials, and if approved for commercial supply as well.

It is difficult to overstate the importance of this readout as you'll recall important one and study 102.

And the tighter and method used by our clinical supply manufacturer and created some variability and resulted in about 60 per cent of patients.

Seeding and lower than target dose.

Douglas S. Ingram: Our microdisturban levels in study 102, 1 were nevertheless 28.1%. These are very impressive and statistically significant results, even with the variability from titering. We have developed a much tighter manufacturing process and a much improved tittering method for our intended commercial process.

Our micro dystrophin levels and study 102 part one would nevertheless, 28, 1%. These are very impressive and statistically significant result, even with the variability from tighter and we have developed a much tighter manufacturing process and a much improved tighter and method for our intended commercial process, hence with our new process.

Douglas S. Ingram: Hence, with our new process, we believe we will achieve expression levels even higher than the 28.1% seen in part one. Study 103 will also characterize our therapy. We strongly believe that based on all of our prior work and the significant number of patients that we have treated with SRP901, we have a greatly differentiated and enhanced gene therapy in terms of safety, expression, and ultimately benefit. If study 103 is successful, it will go a long way toward confirming that belief using the therapy and the process that we will launch with if we are successful.

We believe we will achieve expression levels, even higher than the 28, 1% seen and part one.

Study one O three will also characterize our therapy, we strongly believe that based on all of our prior work and a significant number of patients that we've treated with SRP 9001 that we have a greatly differentiated and enhanced <unk>.

Gene therapy in terms of safety expression.

And ultimately benefit the study 103 is successful it will go a long way toward confirming that belief using the therapy and the process that we will launch with if we are successful.

Douglas S. Ingram: And beyond even that, study 103 results will represent the culmination, an enormous investment, and a journey to become one of the few real world leaders in gene therapy manufacturing. And again, we will have and report those results this quarter.

And beyond even that study one O three results will represent the culmination of an enormous investment and a journey.

Become one of the few real world leaders and gene therapy manufacturer and again, we will have and report those results this quarter.

Douglas S. Ingram: Second, with study 103 data and FDA approval, we will seek a meeting with the FDA as a predicate to commencing our next trial. We are targeting that meeting around the middle of this year, and we're going to commence that next trial as soon thereafter as possible. And finally, the last patient, and last visit in part two of study 102 will occur in December of this year, after which we will unblind that study. That means that by early next year, we will have a wealth of additional information on the performance of our therapy, vastly more than any other company.

Second with studying one or three data in hand, we will seek a meeting with the FDA as a predicate to commencing our next trial, we are targeting that meeting around the middle of this year and we're gonna commenced that next trial as soon thereafter.

And as is possible.

And finally, the last patient last visit and part two of study one or two will occur in December of this year, after which we will and blind that study.

That means that by early next year, we will have a wealth of additional information on the performance of our therapy vastly more than any other company.

Douglas S. Ingram: We will have data from this double-blind placebo-controlled trial for 41 patients, 20 of whom will have been on treatment for two years, and 21 of whom will have been tracked for a year without therapy, and then we'll have been tracked for a year after the intervention of our care.

We will have data for them this double blind placebo controlled trial for 41 patients.

Many of whom will have better and treatment for two years.

And 21 of whom who have been tracked for a year without therapy and then we will have been tracked for a year after.

The interventional therapy.

Douglas S. Ingram: So we have a lot to do with SRP 9001 and a lot of readouts this year into early next year before moving on to our next advanced gene therapy program in the first quarter of this year. Dr. Luis Rodino Clayback presented the two-year data from our high and low-dose cohorts for SRP903, our gene therapy, for the treatment of LGMD2E. As Dr. Redino-Pak reported, SRP903 with a picture and a promoter identical. SRP-901 for Microdistropin resulted in very robust expression of the native protein beta sarcoglycan, the lack of which causes LGMD2E disease, and it was associated with a restoration of the distropin-associated protein common. I mean, it exhibited a very good safety profile, essentially identical to what we've been seeing with SRP 901.

And so we've a lot to do with SRP nine years zero, one and a lot of Readouts. This year into early next year.

Moving onto our next and advanced gene therapy program and the first quarter of this year.

Roger Louise Rodino Claypan presented the two year data from our high and low dose cohorts for SRP 9003, our gene therapy for the treatment of L. G M D to eat.

As Doctor and redeemed unclaimed back reported SRP nine zero and zero three with a picture and a promote or identical to SRP nine zero and zero one for micro dystrophin resulted and very robust expression of the native protein and beta asarco black and the lack of which causes L. G. M. D. TUI disease that it was associated with the restoration of the <unk>.

Strict and associated protein complex and then it exhibited a very good safety profile essentially identical to what we've been seeing with SRP 9001.

Douglas S. Ingram: Importantly, SOP9003 showed excellent durability to the two-year mark, and SOP 9003 resulted in substantial improvements in NSAD and timed function tests, and these children and young adults were much improved versus age-matched natural history. So, REST 50903 and the rest of our six programs, LGND portfolio, we intend to work with a division to devise an efficient and executable path to approval. We will prioritize that meeting behind SRP901 and that meeting, so it will occur in the second half of this year.

Importantly, SRP 9003 showed excellent durability to the two year Mark and Ed.

So it'd be 9003 resulted in substantial improvements and and as J D and <unk>.

Jim to function tests, and these children and young adults were much improved versus age matched natural history.

For SRP 9003, and the rest of our sixth program L. G. N V portfolio, we intend to work with the division to devise and efficient and executable path to approval, we will prioritize that meeting behind SRP 901, and that meeting so it will occur and the second half of this year. Our goal is to commence what we hope to be.

Douglas S. Ingram: Our goal is to commend, but we hope to be a pivotal trial by the end of this year. Now, in the interest of time, I will limit my remarks to these near-term programs that I've just discussed. But know this, that our work continues on our very deep, multi-platform pipeline of additional therapies and new approaches to the treatment of rare diseases, and that includes new and enhanced delivery modalities, our efforts to solve the gene therapy redosing issue, and dosing patients with pre-existing neutralizing antibodies. And additionally, our progress continues in our gene editing innovation center as well. And with that, let me turn the call over to Ian Estepan, who will provide an update on the financials.

Pivotal trial.

By the end of this year now.

And now in the interest of time I will limit my remarks to these near term programs and I've just discussed but know this and our work continues and our very deep multiplatform pipeline of additional therapies and and new approaches for the treatment of rare disease and that includes new and enhanced delivery modalities, our efforts to solve the gene therapy re dosing.

Issue and dosing patients with pre existing neutralizing antibodies and Additionally, our progress continues and our gene editing innovation center as well.

And with that let me turn the call to Ian asked the Pan our CFO, who will provide an update on the financials Ian.

Ian M. Estepan: Thanks, good afternoon. This afternoon's press release provided details for the first quarter of 2021 on a non-gap basis as well as on a gap basis. The press release is available on Thruptive's website; please refer to our press release for a full reconciliation of gas to non-gap financial results.

And good afternoon, everyone.

Backing and press release provided details for the first quarter of 2021 on a non-GAAP basis as well as the GAAP basis.

Press release is available on scrap and website.

Refer to our press release for a full reconciliation of GAAP to non-GAAP financial results.

Ian M. Estepan: Our total net product revenue for the first quarter of 2021 from our PMO, the Exxpinschipping franchise, was $124.9 million, compared to $100.4 million for the same period of 2020. For the first quarter of 2021, individual net product sales were $107. $107.5 million for Bionnda, $5 million for Bionda 53, and $2 million for the recently launched Amand is 45. So, increasing sales primarily reflect higher demand for our stock. We are reiterating our 2021 sales guidance of $537 to $547 million for our RNA franchise. We have gained good experience over the past five years launching these therapies, and we have now been operating for over a year in the COVID-19 environment. These factors serve as a foundation for our guidance.

Our total net product revenue for the first quarter 2021 from our PMO exon skipping franchise was $124 $9 million compared to $104 million for the same period of 2020.

First quarter of 2021 individual and net product sales for 107 $2 million for exon 50, $117 $5 million for biology.

And.

And point $2 million for the recently launched a modest 45.

The increase in sales primarily reflects higher demand for our products.

We are reiterating our 2021 sales guidance of 537 for $547 million for our RNA franchise.

We have gained good experience over the past five years and launching these therapy and we've now executed for over a year and a COVID-19 environment. These factors served as the foundation for our guidance.

And the quarter ended March 30 for 2021, and you recognized $22 million of collaboration revenue compared to $13 $2 million recognized and the same period of 2020, which relates to our collaboration arrangement with Roche and <unk>.

Ian M. Estepan: In the quarter ended March 31, 2021, we recognized $22 million of collaboration revenue compared to $13.2 million recognized in the same period of 2020, which relates to our collaboration arrangement with Roarov. The reimbursable co-development cost under the Roche Agreement totaled $13. For the first quarter,

Reimbursable co development costs under the Roche agreement totaled $13 4 million for the first quarter.

Ian M. Estepan: On a gap basis, we reported a net loss of $167.3 million and $17. $2.10 and $23 per basic and diluted share for the first quarter of 2021 and 2020, respectively. We have reported a non-gap net loss of $122.5 million, or $1.54 per basic and diluted share, in the first quarter of 2021 compared to a non-gap net loss of $79.8 million, or $1.4 per basic and diluted share, in the first quarter of 2020.

On a GAAP basis, we reported a net loss of $167 $3 million and $17 $5 million or $2 and Tencent and 23 cents per basic and diluted share for the first quarter of 2021 and 2020, respectively.

We have reported and non-GAAP net loss of $122 5 million or $1.54 per basic and diluted share in the first quarter 2020, one compared to a non-GAAP net loss of $79 $8 million or $1.04 per basic and diluted share and the first quarter 2020.

Jim.

Ian M. Estepan: In the first quarter of 2021, we recorded approximately $22.3 million in cost of sales compared to $12.6 million in the same period of 2020. The increase in the cost of sales is primarily due to increasing demand for the company's products and the write-off of certain batches of Exxon to 51 not meeting the company's quality specifications in the first quarter of 2021, with no similar activities in the same period of 2020.

And the first quarter 2021 2021 and we recorded approximately $22 $3 million and cost of sales compared to $12 $6 million and the same period of 2020 the.

The increase and the cost of sales, primarily due to increasing demand for the company's products and the write off of certain batches and exon 51, and not meeting the company quality specification and the first quarter point and 21 with no similar activity and the same.

Period end point and time.

On a GAAP basis, and recorded $195 $1 million.

Ian M. Estepan: On a gap basis, we recorded $195.1 million and $136. in R&D expenses for the first quarter of 2021 and 2020, respectively, a year over year increase of $59 million. This increase is primarily due to a continuing ramp-up of the company's gene therapy program.

$136 $1 million and R&D expenses for the first quarter of 2021, and 2020, respectively, a year over year increase of $59 million.

This increase is primarily due to a continuing ramp up of the company gene therapy program.

Ian M. Estepan: On a GAAP basis, the expenses were $173.5 million for the first quarter of 2021, compared to $11.14.2 million for the same period of 2020, an increase of $59.3 million. Now turning to SGNA on a GAAP basis, we recorded approximately $71.1 million and $82.8 million for expenses for the first quarter of 2021 and 2020, respectively, a decrease of $11.7 million. The year-over-year decrease was driven by a decrease in professional services.

On a GAAP basis, R&D expenses were $103 $5 million for the first quarter of 'twenty, 'twenty, one compared to $114 $2 million for the.

Same period of 2020 and increase of $59 $3 million.

Now turning to SG&A on a GAAP basis, we recorded approximately $71 $1 million and $82 $8 million.

And as for the first quarter 2021, and 2020, respectively, a decrease of $11 $7 million for year over year decrease was driven by decrease and professional services.

Ian M. Estepan: On a non-gap basis, SESGNA expenses were $51 million for the first quarter of 2021, compared to $54.5 million for the same period of 2020, a decrease of $3 million. On a gap basis, we recorded $15.5 million in other expenses net for the first quarter of 2021 compared to $7.4 million in other expenses net for the same period of 2020. The increase primarily reflects an increase in interest expense incurred on the company's loan debt facilities through an increase in the outstanding balance partially offset by a reduction of interest expense incurred on the company's convertible debt related to the adoption of ASU 2020-06.

On a non-GAAP basis, SG&A expenses were $51 $5 million for the first quarter, 2020, one compared to $54 $5 million for the same period of 2020, a decrease of $3 million.

On a GAAP basis, and we recorded $15 $5 million and other expense net for the first quarter of 2021 compared to $7 $4 million and other expenses net for the same period of 2020.

The increase primarily reflect an increase in interest expense incurred on the company.

Loan debt facilities due to an increase and the outstanding balance partially offset by a reduction of interest expense incurred on the company's convertible debt related to the adoption of ASU 2000 and dash.

We had approximately $1 $7 billion and cash cash equivalents and investments as of March 31, and 2021 as a reminder, and sold the priority review voucher for $102 million. This transaction closed in mid April and the sale will be recognized and our second quarter 2021 financials with that I'll turn.

Ian M. Estepan: We had approximately $1.7 billion in cash, cash equivalence, and investment as of March 31, 2021. As a reminder, we sold the priority review voucher for $102 million. This transaction closed in mid-April, and the sale will be recognized in our second quarter of 2021.

The call over to down for an update on our commercial activity Alan.

Douglas S. Ingram: Thank you, Ian, and good afternoon, everyone. With the approval in late February launch of Amondas 45, our third PMO-based exon skipping medicine, the team exceeded expectations in the first quarter of 2021. As mentioned, total revenue reached approximately 125 million, representing our 18th consecutive quarter of growth since launching in Q4 of 2016. This kind of consistent growth in the face of a pandemic is only possible due to flawless execution by our field teams and home office teams and the determination and commitment of our patients in the larger DMD community.

Thank you Ian and good afternoon, everyone with.

With the approval in late February launch of a modest 45, our third PMO based exon skipping medicine.

<unk> exceeded expectations and the first quarter 2021.

As mentioned total revenue reached approximately $125 million, representing our 18th consecutive quarter of growth since launching in Q4 for 2016.

This kind of consistent growth and the face of a pandemic is only possible due to flawless execution by our field teams and home office teams and the determination and commitment of our patients and the larger DMD community.

The February 25th approval of him and his 45 pessimism has served as a catalyst to accelerate our momentum even further.

Douglas S. Ingram: The February 25th approval of Amondas 45, Tasmercen, has served as a catalyst to accelerate our momentum even further. While launching during a pandemic can present many challenges, Surrepta has benefited from almost five years of building relationships and trust within the Duchenne community. The power of this experience and these relationships is best illustrated by the fact that within a week of Amondas 45 approval, we had attained access, shipped product to the site of care, and dosed the first commercial patient in Florida.

While launching during a pandemic can present many challenges for rep that has benefited from almost five years and building relationships and trust within the Duchenne community.

The power of this experience and these relationships are best illustrated by the fact that within a week of the a modest 45 approval, we and obtained access shipped product to the site of care and dosed, the first commercial patient and Florida.

Douglas S. Ingram: It's this type of urgency and commitment that goes into driving access for each and every patient. In the first quarter of 2021, March was our third approval, where we were launch ready within 24 hours with all facets of our commercial, manufacturing, and medical organizations ready for the Amandas 45 launch on day one. We will continue exploring options for patients to safely initiate therapy with Amondas 45 in a way that expedites access for medical patients.

It's this type of urgency and commitment that goes into driving access for each and every patient.

The first quarter of 2021 March our third approval, where we were launch ready within 24 hours with all facets of our commercial manufacturing and medical organizations ready for the a modest 45 launch on day one.

We will continue exploring options for patients to safely initiate therapy with a modest 45 and <unk>.

And that Expedites access for our medical patients, we anticipate that the demographics for a modest 45 will be similar to the other two populations exon 51 and bond is 53.

Douglas S. Ingram: We anticipate that the demographics for Amandas 45 will be similar to the other two populations, Exondis 51 and Biondas 53, with regard to the average age of patients on therapy and the payer. We are pleased with the demand for starter forms, and the launch is proceeding on pace. As we saw with Fiondis 53, the pandemic impacts the speed with which patients get on drugs. The primary reasons are the desire to see patients in the center and institutional restrictions.

With regards to the average age of patients on therapy and.

And the payer mix.

We are pleased with the demand for start forms and the launch is proceeding on pace.

And as we saw with 553 depend emmick impacts the speed with which patients get on drug.

Primary reasons are the desire to see patients in the center and institutional restrictions. This is why rare disease companies have seen a softness for a slowness with respect to the rate of new patient starts compared to normal times.

Douglas S. Ingram: This is why rare disease companies have seen a softness or a slowness with respect to the rate of new patient starts compared to normal times. At Serepta, we expect to see this same dynamic play out with Amondas 45. Now transitioning to our broader PMO franchise as a whole and performance in the first quarter, many biotech companies often face headwinds related to typical health plan enrollment cycles that impact revenues in the first quarter of each calendar year. This is especially so with companies like Sarepta that focus on rare diseases.

And so wrapped up we expect to see the same dynamic at play with a modest 45.

Now transitioning to our broader PMO franchise, as a whole and performance and the first quarter.

Many biotech companies often face headwinds related to typical health plan enrollment cycles that impact revenues and the first quarter of each calendar year is especially so with companies like <unk>, who focus on rare diseases.

Douglas S. Ingram: I'm particularly proud that the team has navigated these issues.

I'm, particularly proud that the team has navigated these issues and Q1 more successfully than what we've seen in previous years for our existing patients on exon 51, and by and it's 53.

Douglas S. Ingram: in Q1 more successfully than what we've seen in previous years for our existing patients on exondis 51 and viandum 53. The launch of Viondis-con continues on pace with minimal competitive impact on the growth trajectory of new patients starting. We also continue to see growing revenue from Exonda 51.

The launch of my honest and 53 and continues on pace with minimal competitive impact on the growth trajectory of new patient starts.

We also continue to see growing exhausted and 51 revenue.

Douglas S. Ingram: As in previous quarters, the impact of COVID-19 pandemic has been evident and tracks closely with the U.S. national rates of new infections. That said, any impact within the U.S. has been primarily due to misdoses because of COVID infections within the immediate family or the direct care team. From a drug supply standpoint, we are working continuously with our manufacturers, distributors, and specialty pharmacy partners to ensure uninterrupted drug supply. As such, there have been no disruptions in supplying Exondis 51, Biontus 53, or Amondis 45 to patients in the face of the unique challenges resulting from the ongoing pandemic.

As in previous quarters, the impact of COVID-19, pandemic has been evident and tracked closely with the U S national rate of new infections.

That said any impact within the U S has been primarily due to missed doses because of COVID-19 infections within the immediate family for the direct care team.

From a drug supply standpoint, we are working continuously with our manufacturers distributors and specialty pharmacy partners to ensure uninterrupted drug supply as such there have been no disruptions and supplying exon just 51 by 153.

For a modest 45 the patient.

In the face of the unique challenges, resulting from the ongoing pandemic.

Douglas S. Ingram: We continue to monitor the situation closely, stand ready to adapt and respond, and, most importantly, we'll do everything in our power to mitigate the risks and potential impact of COVID-19 on the Dushan patients we serve. Patient safety is our top priority, and we will continue assessing any and all efforts to ensure patients feel safe and supported during this unusual time. Now I'll turn the call over to Gilmore for our research, for an update on our research and development activities, Gilmore. Thank you, darling, and good afternoon, everyone. Our commitment to and success in serving the Duchenne Muscular District

We continue to monitor the situation closely stand ready to adapt and respond and most importantly, we will do all and our power to mitigate the risks and potential impact of COVID-19 on the Duchenne patients we serve patient.

Patient safety is our top priority and we will continue assessing any and all efforts to ensure patients feel safe and supported during this unusual time.

Now I'll turn the call over to Gilmore for our research for an update on our research and development activities Kilmer.

And thank you Alan and good afternoon, everyone.

Our commitment to and success in serving the Duchenne muscular dystrophy community has been greatly strengthened by the achievement of a number of critical milestones over the past several months.

Douglas S. Ingram: The community has been greatly strengthened by the achievement of a number of critical milestones over the past several months. Just two days ago, on Monday, we were thrilled to announce positive clinical data from the 30 mink-per-k-k-arm of the Momentum study, evaluating

Just two days ago on Monday may 3rd we were thrilled to announce positive clinical data from the 30 Meg per kg arm of the momentum study evaluating our lead P. PMO based candidate SRP 50 51.

Douglas S. Ingram: evaluating our lead PPMO-based candidate SRP 5051. The data we shared included safety, measurements, and change from Bayside at week 12 for exon skipping as measured by digital drop polymerase chain reaction or DDPCR and dystrophin expression in muscle by Western blood. There were four patients biopsied in the 30-a-k cohort of momentum.

The day should we shared included safety.

Measurements and change from baseline at week 12 for exon skipping as measured by digital dropped polymerase chain reaction or D D PCR and dystrophin expression and muscle by Western blot.

They were for patients biopsy, and the 13 Meg per kg cohort and momentum.

Douglas S. Ingram: Both ambulant and non-ambulant.

Both ambulatory and non ambulant three of the patients were in there and 18th and one patient for seven years old at the time of treatment of.

Douglas S. Ingram: Three of the patients were in their late teens, and one patient was seven years old at the time.

Douglas S. Ingram: note and due to COVID-imposed delays, patient one received five doses of SRP 50-51 before he was biopsied, while the other three patients received their biopsy after three doses. The 30-8 per K cohort showed a significant dose-dependent increase in exon skipping. SRP 5051, when dosed once per month, at 30migs per kig, achieved approximately 11% mean exon skipping at week 12.

Note.

And due to COVID-19 imposed delays patient one and receive five doses of SRP 50, 51 before he was biopsied while the other three patients received their biopsy after three doses.

The 30 day Cookie cohort showed a significant dose dependent increase in exon skipping and Sop.

P 50, 51, and dosed once per month.

Turkey mix per kg achieved approximately 11% mean exon skipping at week 12.

Douglas S. Ingram: Compared to the PPMO 20-PK dose, we observed a great

Compared to the P. P. M O 20, Meg per kg dose, we observed and greater than four folds dose dependent increase and exon skipping after only a 50% increase in dose, indicating that we have hit the steep part of the dose response curve predicted by our preclinical models.

Douglas S. Ingram: than fourfold dose-dependent increase in exon skipping after only a 50% increase in dose, indicating that we have hit this deep part of the dose response curve predicted by our preclinical model. When compared to the current standard of care at Teperson,

When compared to the current standard of care at 10%, we observed and 18 fold increase and exon skipping.

Douglas S. Ingram: We observed an 18-fold increase in Exxon Skips. Now, in terms of expression, the 30-Mig dose of SRP 5051 resulted in more than 6.5% distrofen protein expression as measured by Western blood, representing a greater than 100% increase in expression versus the 20-Mik per Kig cohort at Week 12. Now, it is important to note that these results were not driven by a single person; all the patients responded well to

Now in terms of expression the 30 Meg per kg dose of SRP 50, 51 resulted in more than six 5% dystrophin protein expression as measured by western blot, representing a greater than 100 percentage increase and expression versus the 20 Meg per kg cohort at week 12.

Now and it's important to note that these results were not driven by a single patient all of the patients responded well to therapy.

Douglas S. Ingram: Indeed, if we remove patient one, who, as you will recall, received five doses and had the highest expression at 12% from the analysis, the mean expression for the three other patients was approximately 5%, a clinically meaningful and robust number. Furthermore, based on our predictive modeling, patient one also provides strong support and validation of that model, which shows we should comfortably achieve greater than 10% distrofen with once-per-month dosing over time.

If we remove patient one who as you will recall receive five doses and had the highest expression of 12% from the analysis. The mean expression for the three other patients was approximately 5% a clinically meaningful and robust number.

Furthermore, based on our predictive modeling patient. One also provides strong support and validation of that model, which shows we should comfortably achieved greater than 10% dystrophin with once per month dosing overtime.

Douglas S. Ingram: It is also important to highlight that baseline dystrophin levels are not a predictor of post-treatment expression. In fact, we observed that two patients with the lowest baselines had the highest level of post-treatment expression. With regard to safety, we observed three serious treatment-related adverse events in two patients, two cases of hypomagnoemia and one of hypomagnosis.

It is also important to highlight that baseline dystrophin levels are not a predictor of post treatment expression in.

In fact, we observed it to patients with the lowest baseline had the highest level post treatment expression.

With regards to safety, we observed three serious treatment related adverse events in two patient.

And two cases of Hypomagnesaemia, and one up hypokalemia or low potassium.

Douglas S. Ingram: hypokalemia or low potassium. Events were not symptomatic and have all resolved with treatment.

The events were not symptomatic and have all resolved with magnesium supplementation.

Douglas S. Ingram: with magnesium supplementation. Our primary working hypothesis is that as an infusion, the PPMO may be competing for magnesium transporter proteins, causing a decrease in magnesium after infusion.

Our primary working hypothesis is that other infusion the P. P. M O maybe competing for magnesium transporter proteins, causing a decrease and magnesium after infusion.

Douglas S. Ingram: Our analysis of clinical and non-clinical kidney biobarcard data

Our analysis of the clinical and non clinical kidney biomarker data Sherman urinary.

Douglas S. Ingram: lead us to conclude that hypomagnesemia appears reversible, not correlated with changes in renal function, and is manageable by magnesium supplementation. Importantly, we do not believe there is a cumulative effect, as we've seen no evidence that the hypomagnesemia worsens over time upon receiving multiple doses. We and our external experts believe that serum monitoring of magnesium at oral supplementation with magnesium is a feasible approach to enable early detection and management going forward. In summary, SRP 5051, dosed monthly at 30 milligrams per kig, delivers profound and clinically meaningful levels of dystrophen that represent a significant improvement in potency over our marketed first-generation POMOM Medicine.

Lead us to conclude that Hypomagnesaemia appears reversible not correlation for changes in renal function and is manageable by magnesium supplementation.

Accordingly, we do not believe there is a cumulative effect as we've seen no evidence that the hypomagnesaemia worsens over time upon receiving multiple doses.

We and our external experts believe that serial monitoring of magnesium and oral supplementation with magnesium is a feasible approach to enable early detection and management going forward and.

Summary, SRP.

And sorry, 50, 51 dose monthly 30, Meg per kg delivers profound and clinically meaningful levels of dystrophin that represents a significant improvement and potency over our marketed first generation Puma medicines and SRP 50, 51 achieves that increased potency with more convenient and less burdensome.

Douglas S. Ingram: SRP 5051 achieves that increased

Douglas S. Ingram: with more convenient and less burdensome once-per-month dosing with a manageable safety profile. We are delighted with these results. Now, shifting to our gene therapy program, In March, our chief scientific officer, Dr. Louise Regina Claypack, will present results from our SRP-903 gene therapy compound in development to treat limb girdle muscular dystrophy type 2E at the 2021 Muscular Distributee Association annual meeting.

And once per month dosing with a manageable safety profile.

Delighted with these results now.

Shifting to our gene therapy programs in March our Chief Scientific Officer, Dr. Louise Rodino <unk> presented results from our SRP nine years, Europe, III gene therapy compound in development to treat limb girdle muscular dystrophy type TUI.

For 2021, muscular Dystrophy Association annual clinical and scientific conference.

Douglas S. Ingram: Association, annual, and scientific conference

We believe.

Douglas S. Ingram: that durability data will be a key differentiating factor for gene therapies, so we were quite pleased to observe that protein expression in muscle was sustained for two years following treatment in cohort 1, the low-dose cohort, with mean beta-starcic glycan expression of 54% at 24 months, compared to 36% at day 60, as measured by Western blot of muscle biopal. In functional outcome assessments measured by the North Star assessment for dysferrenopsis, or NSAT, In cohort 1, a mean NSAD score improvement of 5.7 points from baseline was sustained through 24 months, and in cohort 2, the high-dose cohort, a mean NSAD score improvement of four points from baseline at one year was observed.

But durability data will be a key differentiation factor for gene therapy. So we're quite pleased to observe that protein expression in muscle with sustained for two years following treatment in cohort one the low dose cohort with mean Baker chocolate like an expression of 54% at 20 for me pretty for months compared to 32.

6% at day 60.

And as measured by Western block of muscle biopsies.

In functional outcome assessments measured by the North Star assessment for just for on and off these are and sad.

In cohorts, one and mean and sat score improvements of five seven points for baseline with sustained through 24 months and and cohort to the high dose cohort and mean and sat score improvement of four points from baseline at one year was observed.

Douglas S. Ingram: Results in both cohorts continue to reinforce the safety and tolerability profile of SRP 9003 and the RH74 gene therapy platform. As Doug already alluded to, we look forward to releasing the results for the first 11 patients in our microdiscrofen study 901-103, when we will report on vector genome copy number and dystrophen expression measured by both Western blot and immunohistochemistry. I would also reiterate that in study 103, we utilized our linear QPCR titering assay and can confirm all patients received the target dose of 1.33 to 14 VG per kilogram.

Results in both cohorts continue to reinforce the safety and Tolerability profile of SRP nine years, there were three and the RH 70 for gene therapy platform.

As Doug already alluded to we look forward to releasing the results for the first 11 patients and our micro dystrophin study nine user one one O. Three when we will report on vector genome copy number and just broke and expression measured by both western blot and immunohistochemistry.

Also reiterate and in study 103, we utilized our linear Q PCR tightening assay and can confirm all patients received the target dose of $1 33 to the 14th.

G per kilogram.

In conclusion.

Douglas S. Ingram: I would like to take this opportunity to thank our R&D team, investigators, study participants, and their families for their relentless dedication.

I would like to take this opportunity to thank our R&D team investigators study participants and their families whose relentless dedication. Despite the prolonged impact for the pandemic have made all of these achievements for patients possible.

Douglas S. Ingram: Despite the prolonged impact of the pandemic, we have made all of these

Douglas S. Ingram: made all of these achievements for patients possible. And with that, I'll pass back to Doug for Q&A.

And with that I'll pass.

Back to Doug for Q&A.

Doug.

Thank you very much larger O'neill, Jonathan let's open up the lines for questions now.

Douglas S. Ingram: Thank you very much, Dr. O'Neill. Jonathan, let's open up the line for questions now.

Operator: Certainly, once again, if you have a question, please press star and then one on your touch-tone telephone. If you wish to remove yourself from the queue, please press the Pound Key. Our first question comes from the line of Gina Wang from Barclays. Your question, please?

Certainly once again, if you have a question. Please press Star then one and you touched on telephone if you wish to remove yourself from the queue. Please press the pound key our first question comes from the line of Gena Wang from Barclays. Your question. Please.

I think thank you for taking our questions. This is shouting no for Gina.

Sheldon Nel: Thank you for taking our question. This is Sheldon, Nel, for Gina.

Well the question young.

Douglas S. Ingram: So I have one question on the potency essay for study 301. I think you mentioned in the past that FDA had agreed with your approach to the potency assays, but we recently got many questions on this after Pfizer's setback. Could you confirm that all the potency essay matrix has been cleared, and you don't need to do any additional work before those in patients in study 301? Yeah, thank you for that question.

<unk> for.

For study 301, I think I've mentioned in the past that FDA has agreed to Peter appeal shifts.

Yes.

But if we recently got many questions on days after Pfizer's setback.

Could you confirm that all of the potency assay matrix has been cleared and you don't need to for any additional work before it goes and patient in Israel one.

Yes, thank you for that question.

Douglas S. Ingram: For those who I'm sure everyone saw it, but Pfizer noted today, they're still in discussions with the FTA regarding their potency assay for the release of their therapy and for their trial, so they can start dosing in the United States. So let me be clear about where we are with this. The number one issue is that we have to have a meeting with the division as a predicate to commencing 301. One will recall that in September of last year, in connection with the commencement of what we call study 103.

For those who I am sure everyone saw the advisor noted today.

We're still in discussions with the FDA regarding their potency assay for the release of their therapy and.

For their trial, so they can start dosing and the United States.

So, let's let me be clear about where we are with the number one issue that we have to have and meeting with the division as a predicate for commencing 301, one will recall that in September of last year and connection with the commencement of what we call study one out of three.

Douglas S. Ingram: We had discussions with the agency. We were hung up on potency assays at that time as well. I'm very proud of the team. Actually, within a short two weeks or so after that initial meeting, we were able to get a live meeting with the division, have a very productive discussion, understand better what they were looking for, and devise an approach to a potency assay that they would agree with that would allow us to start 103. We've done additional work. on potency assays even since then.

We had discussions with the agency we were hung up on potency assays at that time as well I'm very proud of the team actually within a short two weeks or so after that initial meeting we were able to get a live meeting with the division have a very productive discussion.

Understand better what they were looking for and devised and approach to a potency assay that they would agree with that would allow us to start one O. Three we've done additional work on and potency assays.

And since then so we continue to evolve that.

Douglas S. Ingram: So we continue to evolve. Now, the most significant thing we have to do is actually take all of our work, CMC, and our protocol for our next trial and then have a discussion with the agency, and we're going to target that through the middle of this year. We feel very good about where we are. We feel very good about our potency assay. We feel very good about the data that supports our potency and all of the rest of our CMC. This is one of the many things that make up the character of a product.

Most significant thing we have to do is actually take all of our work CMC and our protocol for our next trial and then have a discussion with the agency and we're going to target that for the middle of this year, we feel very good about where we are and we feel very good about our potency assay and we feel very good about the data that supports our potency assay.

And all of the rest of our CMT and this was one of the many things that make up the characterization of the product, but so were clear and we need to actually compile all of that and have.

Douglas S. Ingram: But so we're clear, we need to actually compile all of that and have an active discussion and a dialogue with the division. And we're going to try to do that as soon as we have the 103 data, which will be right around the middle of this year. And then our goal is to start our next trial, you know, as soon thereafter as we are permitted to do so. Now, with that said, Dr. Radino-Clapec, have I missed anything? There's some nuance, but I've forgotten to mention it. No,

And active discussion and dialogue with the division and we're going to try to do that.

As soon as we have the 103 data and when and so that'll be right around the middle of this year and then our goal is to start on ex trial.

Soon thereafter, as we are permitted to do now with that said, Dr or really and I play it back, thereby missed anything or is there any nuances for non dimension.

No I think.

Douglas S. Ingram: No, I think you put it perfectly. Just a follow up on 301, what is the remaining gaining factor for the initiation? The big, the two big ones are we got to get the 103 data. So just to remind everybody, this is a very important trial for us. That is the trial where we're going to look at the performance of the therapy, both expression and safety, in patients with Dushchevistricti using our commercially representative material, the actual process that we'll be doing all of our future trials on, the next one, 301, as well as the commercial material, both in the United States and outside of the United States.

And I wouldn't add any cash.

Yes.

Yeah, just a follow up.

Phil one.

And what are the remaining gating factor for the initiation.

The big two big ones are going and get the one O three data so just to remind everybody.

Study 103 is a very important trial for us that as the trial, where we're going to look at the performance of the therapy both expression.

And safety in patients with Duchenne muscular dystrophy, using our commercially representative material. The actual process that we'll be doing all of our future future trials on the next one and 301 as well as the commercial material both in United States and outside of the United States. So number one is we have to get that data and of course.

Douglas S. Ingram: So number one is we have to get that data. And of course, you've heard in my opening remarks, we'll have that data this quarter, and once we have that data, and it's been too seen, we'll share it with the investment community and with the rest of the world. And that'll happen this quarter.

And you've heard in my opening remarks, we will have that data this quarter and once we have that data and it's been you've seen and we'll share it with the with the investment community and with the.

The rest of the world and that'll happen this quarter. So that's been one big issue and asset growth and then the next and then the second one just say no but it is basically done is that we needed to gain as much insight as we could from part one of study one O. Two that's the study that we reported out in January to really hone and the protocol.

Douglas S. Ingram: And then the next issue, and then the second one, just saying, no, but it is basically done, is that we needed to gain as much insight as we could from part one of study 102, that's the study that we reported out in January, to really hone the protocol for our next study, study 301, and we have done that. And we've made enormous progress and really believe in the insight that we've gained.

<unk> for our next studies study 301, and we have done that and.

And we've made an enormous progress and and really believe that the insight a new game for part one of study 102 has been invaluable and should increase the probability of success and that study that's been done and then with all of that and all of our CMC materials as well and there's a lot of CMC data, we need to meet with the agency, we will do that around the middle of this year and as.

Douglas S. Ingram: gained from part one of study 102 has been invaluable and should increase the probability of success in that study. And then, with all of that and all of our CMC materials as well, there's a lot of CMC data. We need to meet with the agency. We'll do that around the middle of this year, and as soon as we've done that, we'll get the approval from the division. We'll commence our next study, and as everyone knows, we'll move as fast as we can on that.

Soon as we've done that and we get the concurrence from the division will commence our next study and as everyone knows we're moving as fast as we can thereafter.

Operator: Thank you. And ladies and gentlemen, we ask that you please limit yourself to one question.

Thank you and ladies and gentlemen.

Ask that you. Please limit yourself to one question you may get back and the in the queue. As time allows our next question comes from the line of Ami <unk> from Bank of America. Your question. Please.

Operator: You may get back in the queue as time allows. Our next question comes from Tizin Ahmed from Bank of America. Can I ask you a question, please?

Hi, good afternoon, and thanks for taking my question. So does my one question for you is about timelines for study three one.

Tazeen Ahmad: Hi, good afternoon. Thanks for taking my question. So, Doug, my one question to you is about timelines for study 301. If you are able to start the study, you know, sometime this summer, is it your expectation that you would be able to enroll by year end? Just given the calculations that Pfizer provided on its call, it does seem that you could significantly narrow the gap between completion of your study and theirs.

And if you are able to start the study sometime this summer is it your expectation.

And you would be able to enroll by year and just given the calculation that Pfizer provided on its call. It does seem that you could significantly narrowed the gap between completion of your city and theirs and just wanted to clarify your thoughts on that thanks.

Douglas S. Ingram: I just wanted to clarify your thoughts on that. Thanks. Yeah.

Douglas S. Ingram: Yeah, thank you for that, Zee. First, of course, no one will be surprised to hear us say we want to move as fast as possible. So we want to get this meeting with the agency as soon as we can. We have to have the study 103 data in hand for that meeting. I think that meeting will occur around the middle of this year.

Yes. Thank you for that disease, So U verse and of course, no one will be surprised to hear us say, we want to move as fast as possible and so we want to get this meeting with the agency as soon as we can you have to have the study one or three data.

And hand to have that for you I think that meeting will occur around the middle of this year and then we want to start or soon thereafter.

Douglas S. Ingram: And then we want to start as soon thereafter as we're permitted to do. The timing of full enrollment will depend in part on the end of the study, of course, and we're still refining that. I'm not going to provide a ton of detail on that because that will go into some of the work that we've done on the protocol. But we want to move as fast as possible. I would know that Pfizer. I don't remember the number of sites that Fiver has XUS, but we'll have significantly more sites on board.

And as we're permitted to do that the timing of.

Full enrollment will depend in part on the end of the study of course, and we're still and refining that and I'm not going on for quite a ton of detail on that because that that will go into some of the work that we've done and the protocol, but we want to move as fast as possible I would note that.

Pfizer I don't remember the number of sites that fiber has ex U S. But we will have significantly more sites onboard. So we shouldn't be able to rapidly enroll that that therapy, our goal would be to be able to do it by the end of this year or.

Douglas S. Ingram: So we should be able to rapidly enroll that therapy. Our goal would be to do it by the end of this year or, you know, as soon into next year as we can from a, you know, pure operational perspective. But I think there are a number of things that we need to consider. One, consider that we will have a significant number of sites up and running. over the course of this year, if we can commence that study as soon as possible.

Soon into next year as we can from a from a pure operational perspective, but I think a number of things that we do.

We need to consider one considered that we will have a significant number of sites.

Up and running over the course of this year and if we can commence that study as stable as possible and the next big thing to also understand is and I think theres going to be an enormous amount of demand for this trial. So I don't think theres going to be a patient demand issue, that's going to slow us down. So it's really incumbent upon us first to get the concurrence from the agency to commence that trial and.

Douglas S. Ingram: And the next big thing to also understand is that I think there's going to be an enormous amount of demand for this trial. So I don't think there's going to be a patient demand issue that's going to slow us down. So it's really incumbent upon us, first to get the consent from the agency to commence that trial, and then to really operate with a significant amount of operational excellence to get it enrolled.

And then to really operate with a significant amount of operational excellence to get and enrolled and I think I do think that we'll be able.

Douglas S. Ingram: And I think, you know, I do think that we'll be able to move very fast with that. I do want to take one issue with some people that don't have me that sort of say we need to close a gap.

And to move very fast with that I do want to take one issue and with some people.

Johnson.

And that sort of say, we need to close a GAAP I do want to be clear, we have dose and then significantly more and more patients than others. We have a significantly greater amount of information about the performance of our therapy and the way. These kids are working with this therapy. We are the only ones that have had it read out on the placebo control.

Douglas S. Ingram: I do want to be clear. We've dosed significantly more patients than others. We have a significantly greater amount of information about the performance of our therapy and the way these kids are working with this therapy. We're the only ones that have had a readout on the placebo control trial. I will note that while we had an imbalance in the six and seven-year-olds, and we wish we hadn't, but in the places where we didn't have an imbalance, the 45-year-olds.

While I will note that while we had an imbalance in the six and seven year olds and we wish we had and the places where we didn't have an imbalance.

For the five year old we saw a very significant statistically significant benefit and this therapy and I will say that we will we alone not to be and being a little bit defensive here, but what surprised anybody that might be a little defensive.

Douglas S. Ingram: We saw a very significant, statistically significant benefit from this therapy. And I will say that we alone, not to be, I'm being a little bit defensive here, but it won't surprise anybody that doesn't have to be a little defensive.

Douglas S. Ingram: We will, early next year, have a readout on part two of this study, and that's going to be just an enormous amount of information. We'll have 41 patients, all who have been treated with our therapy. We'll have a cohort, you know, about half of those kids will have something really really going on in therapy for two years, and we'll be able to look at that when we unblind. And another group will be really interesting as well.

We will and early next year and have a readout on part two of this study and that's going to be just an enormous amount of information. We will have 41 patients all who have been treated with our therapy will have a cohort of about half of those kids will have something really really are a therapy for two years and we'll be able to look at that when we and.

And another group will be really interesting as well will have been able to follow those kids for a year without a therapy and then watch the interventions and therapy and the impact that that intervention and therapy might have with these children against.

Douglas S. Ingram: We'd have been able to follow those kids for a year without therapy and then watch the intervention of the therapy and the impact that that intervention of therapy might have on these children against, you know, an age-matched natural history cohort as an example. And that's all blinded right now. So we, you know, I think we're doing very, very well. And I think if we can move fast, if we can get the agency by around the middle of this year, we can get the next study started shortly thereafter, then we should, I believe, be able to enroll this study with rapidity because there's going to be an enormous amount of demand, both from investigators and from families with Duchenne Musco.

Ah matched and age matched natural history cohort as an example, and that's all blinded right now.

I think we're doing very very well and I think if we can we can move fast if we can get the meeting with the agency by around the middle of this year, we can get the next studies started.

Shortly thereafter, then we should I believe be able to enroll this study with repeatedly because theres going to be an enormous amount of demand both from investigators and for families with duchenne muscular dystrophy.

Operator: Thank you. Our next question comes from Joseph Schwartz from SVB Lerink. Can I ask you a question, please?

Thank you. Our next question comes from the line of Joseph Schwartz from SBB Leerink. Your question. Please.

Kelly Gerskis: Great, this is Kelly Gerskis on for Joe. On your close back in December for the 20 Meg Per Kig SRP, 50-one group. You mentioned there was a slight delay in muscle biopsies due to COVID-19. Could you give us a bit of color on the average time from last dose to biopsy in a 30-mig per-k group? And could this difference in biopsy timing confound the dose response comparison we might be looking at? Thanks.

Great. Thanks. This is Kelly Chris gets on for Joe.

And back in December for two.

20, Meg per kg SRP.

P. 50, 51 group you had mentioned there was a slight delay and muscle biopsies due to COVID-19 and.

Could you give a better color on the average time from last dose to biopsy and a 30.

And make per K grip and could this difference and biopsy timing confounded dose response comparison, and we might be looking at.

Douglas S. Ingram: Great, I don't think we had a significant issue with the biopsy times for the 30 mix, but I will turn this over to Dr. O'Neill and come. Thank you, Doug. In fact, there were some delays in those

Yeah.

Great.

And don't think we have a significant issue on the <unk>.

<unk> times for the third and Nathan, but I will turn this over to Dr O'neill and could.

Comment on that.

Thank you and in fact, there were some delays actually in those muscle biopsies.

Dr. O'Neill: muscle biopsies. However, because of their duration, we do not believe that they confound the comparison anyway. In fact, if they were to do that, they would have confounded against the 30 mix or kicks. I think that we are very confident in our conclusion that we're seeing a definite dose-dependent effect and that we are actually seeing a robust response. And, very importantly, that we are on the steep part of the dose-dependent

And because of their duration, we do not believe that they confound the comparison anyway and fat.

If they were to do that they would've compounded against the therapy makes for kicks and I think that we are very confident.

And our conclusion that we are seeing a definite dose dependent effect and that we are actually seeing a robust.

And with pump and very importantly that we are on the steep part of the dose response curve has predicted by our preclinical market. So in summary, and we are very confident.

Dr. O'Neill: We are actually seeing a robust response, and very importantly, we are on the steep part of the dose-response curve as predicted by our pretty critical model. So in summary, we are very confident that our results, and from the conclusion for results, we asked the robustness of the expression effect in.

And that our results.

And from the conclusion for our results.

But the robustness of the expression of effect.

Douglas S. Ingram: Thank you. And one final thing, I wouldn't note. For those who wonder, I mean, I really, remember the following.

Thank you and one final thing I would note for those who wonder I mean, I really remember the following.

Douglas S. Ingram: The thesis is pretty straightforward. Tissue exposure leads to exon skipping, leads to dyserfin production, and leads to functional benefit. Look at the exon skipping. For those who wonder, look at the significant multiple increase in exon skipping that occurs at the 30 mix per kig. That should be very predictive of a significant benefit over time. And then look at it versus exonis.

Thesis is pretty straightforward tissue exposure leads to exon skipping leaves the dystrophin production leads to functional benefit look at the exon skipping and for those who wonder and look at these significant multiple increase and exon skipping that occurs at the dirty and mix per kg that should be very predictive of a significant benefit over time.

And then look at it versus exon dish, if you went and one wonders why.

Douglas S. Ingram: If you know, one wonders why we feel very good about the results that we have and about the potential that we have to confirm in future studies in our Part B, the potential for a profoundly improved therapy versus Exondis. Simply look at the fact that we're seeing 18 full, literally 1,800% more Exxon skipping over Exondis. And we're seeing that at 12% of the dose exposure and in half, just about half the time. So, you know, we do feel very good.

We feel very good about the results that we have and about the potential at least that we have to confirm and future studies and are part D and the potential for a profoundly improve therapy versus exon there's simply looked at the fact that we're seeing 18 for literally 1800 person.

Sent more exon skipping over a scientist and we're seeing that at 12% of the dose exposure and and have just about half the time.

So we didn't feel we feel very good and then again Doctor O'neil mentioned and in his opening remarks, and apologies for belaboring, it but all of the kids and other small cohort is only for children and I get that but all of the kids and the cohort that we looked at and partner and were significant responders Theres no. One responder that drove anything here and we did see one significant respond to that.

Douglas S. Ingram: And, again, Dr. O'Neill mentioned it in his opening remarks, and apologies for belaboring it, but all of the kids, it's a small cohort, it's only four children, I get that. But all of the kids in the cohort that we looked at in Part A were significant responders. There was no one responder that drove anything here.

Douglas S. Ingram: We did see one significant responder, and that was the 12% kid, but all the other kids responded as well. And the only difference that we can find between the 12% kid and the other kids is that the 12% kid had two more doses than the others. So when we look to the future and predict the potential of this therapy, I would keep that in mind because that is a pretty important signal for what we think we're going to see.

It was the 12% kit, but all the other kids responded as well and the only difference that we can find between the 12% Jared and the other kids is that 12% Jay to add to our doses and the other so when we look to the future and we predict the potential of this therapy I would keep that in mind, because that is a pretty important and signaled for what we think we're going to see it.

Douglas S. Ingram: If we look at this therapy over, let's say, six months or even over 12 months, what might we see in the way of district and production and then, of course, ultimately benefit from this therapy. Thank you. Our next question comes from the line of Brian Abrahams, from RBC.B.

Look at this therapy over let's say six months or even over 12 months and what we might see and the way a district and production and then of course ultimately benefit from this therapy.

Thank you. Our next question comes from the line of Brian Abrahams from RBC capital markets. Your question. Please.

Operator: Thank you. The next question comes from the line of Brian Abraham from RBC Capital Markets. Your question, please.

Okay.

Hi, This is Steve on for Brian. Thanks for taking our question. So just as a clarification, where any functional data collected a baseline from the patients enrolled in study one and a three and do you plan to assess and report.

Brian Corey Abrahams: The kids, I'm going to say this and then you're going to, Dr. O'Neill or Dr. Real Clibble will correct me. We do take functional measures with the kids in 103, but there won't be any report on that for a host of reasons. It's an open-label study, so there won't be any reports on it immediately. I'd say when we announce the results to you this quarter, we'll be providing results on expression and safety.

And all changes for Ms patients.

The kids I'm going to say this and then youre going to Dr. O'neill, Our Doctor reactor will correct me, we do take functional measures with the kids and one out of three but there won't be any report on that for a host of reasons. It's an open label study and won't be and reports on it.

Mediately I'm, saying when we when we announce to you the results and this quarter, we will be providing results on.

Expression and safety and I wouldn't envision and results number one it's an open label study, but more important number two it's a very early time point as we're looking at kids. After just a couple of months on therapy, but perhaps I missed.

Brian Corey Abrahams: I wouldn't anticipate any sort of result. Number one, it's an open-label study, but more importantly, number two, it's a very early time point. We're looking at kids after just a couple of months on therapy. But perhaps I'm misstating things, so I will ask Dr. O'Neill to confirm or deny my statement in that regard.

Misstating thing so I will I will ask Dr O'neill to confirm or deny my statement and that was.

Douglas S. Ingram: No, I think you've summarized it nicely.

Got it.

No I think you summarized it nicely.

Operator: Thank you. Our next question comes from the line of Aletia Young from Cancer Fitzgerald. Your question, please?

Okay.

Thank you. Our next question comes from the line of Alicia Yap from Cantor Fitzgerald. Your question. Please.

Aletia Young: Hi, this is Emily speaking on behalf of Alethea. Thanks for taking our question. I was wondering how you are thinking about running studies for some of the other limb girdles beyond 2E. Is there a way to potentially do a basket trial, or are they two different things?

Hi, This is Emily on purely it there and thanks for taking our question and I was wondering how are you thinking about running studies for some of the other limb girdles beyond two E is there a way to potentially do a basket trial or day to day.

Douglas S. Ingram: So, yeah, it was the short answer, probably not the most satisfying answer. The short answer is we're looking at a lot of different approaches right now to the, and there are a lot of ideas we have and potentially some basket, basket studies around some of, for instance, the Sarks or other very similar therapies or the kinds of things we're pondering, but the predicate for all of it has to be a discussion with the agency about the pathway for limb girdle type 2E, because that So we have a number of different ideas. I don't want to get too far out on my skis around them until we've actually met with the division. We will do that again this year.

Yeah.

So yes, there were.

The short answer probably not the most satisfying answer and short answer is we're looking at a lot of different approaches right now too.

And I know Theres a lot of ideas, we have and potentially some basket basket studies around summer for instance for SRP or other very similar.

And therapies are the kinds of things, we're pondering, but the predicate for all of it has to be a discussion with the agency about the pathway for limb girdle type two we because that will really set the framework within which we can have discussions about the efficient pathway for all of these therapies. So we have a number of different ideas and I don't want to get too far out of my skis.

And until we've actually met with the division we will do that this year as I said in my opening remarks, we really want to prioritize that right behind 9001 simply because we don't want to overburden the division and our own team as we're dealing with the various things we're dealing with so we will first focus on non 001 will have a meeting on non 003 hope.

Douglas S. Ingram: As I said in my opening remarks, we really want to prioritize that right behind 9001, simply because we don't want to overburden the division in our own team as we're dealing with the various things we're dealing with. So we'll first focus on 9701. We'll have a meeting on 9703, hopefully, very shortly.

Very shortly thereafter, and our goal is to find a very efficient pathway.

Douglas S. Ingram: after, and our goal is to find a very efficient pathway and hopefully to get started on what we like to believe will be a pivotal trial this year, but we'll have to do that with the concurrence input from the division. And there are a lot of reasons why we think we should be able to move quickly. Just to remind everybody with respect to SRP 9003, and this is similar to most of the other limb girdles, if not all of them. We're dealing with the native protein. These are all monogenic diseases characterized by the absence of a protein, usually a structural protein, and the absence of that structural protein causes degeneration leading to death.

And hopefully they got started on what we like to believe will be a pivotal trial this year, but and we'll have to do that with the concurrence and put some division and Theres a lot of reasons why we think we shouldn't be able to move quickly. This just to remind everybody with respect to SRP 9003, and this is similar to most of the other limb girdles and.

Not all of them were dealing with the native approach and these are all monogenic diseases characterized by the absence of a protein and usually a structural protein and the Apple and structural Burger King is causing degeneration and death.

Douglas S. Ingram: These therapies, let's go, let's start with SRP 9003, which is a gene therapy that codes for the actual native protein. So we're able to deliver it to these children who are dying from the absence of beta-sarkaliccan. The native beta-clican protein is properly localized at the muscle membrane, acting as the structural protein it must for these kids to benefit from it. We do that really robustly. So far, the data on safety looks very good. The kids are doing, though it is open label. The kids appear to be doing very well compared to natural history, and the therapy is very durable.

These therapies, let's go let's start with SRP 9003, as the lead here nine zero and zero three is a gene therapy that codes for the actual native protein and so we're able to deliver to these children, who are dying from the absence of day to start to look like and the native data. So I don't know I can't throw gene properly.

Localized at the muscle membrane and acting as the structural approaches must for these kids to benefit from it and we do that really robustly. So far the data on safety looks very good for kids are doing open label, though it is the kids appear to be doing very well versus natural history and the therapy is very durable. So we think we.

Douglas S. Ingram: So, you know, we think we have at least the opportunity to have a good, robust discussion with the agency about, in the context of what we're seeing in the context of this very rare disease and in the context of this native protein, coming up with a development program that is fit for purpose for that disease state. We'll have that discussion. Then we'll report out on that and what our goals are with that.

And have the at least the opportunity to have a good robust discussion with the agency about in the context of what we're seeing and the context of this very rare disease and in the context of this native protein developing coming with a development program that is.

Fit for purpose for that disease state and we'll have that discussion then we'll and we'll report out on that.

And our goals are with that and that will inform the rest of our limb girdles and we'll be able to provide some additional information on that over time, but ideas like basket studies and the kinds of things. We're pondering we'd have to find clever ways to address these very rare diseases, particularly rare diseases that have significant therapeutic commonalities.

Douglas S. Ingram: And that will inform the rest of our limb girdles, and we'll be able to provide some additional information on that over time. But, you know, ideas like basket studies are the kinds of things we're pondering. We have to find clever ways to address these very rare diseases, particularly rare diseases that have significant therapeutic commonality.

Thank you. Our next question comes from the line of Gil Blum from Needham and company. Your question. Please.

Operator: Thank you. Our next question comes from the line of Gil Bloom from Needham and Company. Your question, please?

Gil Blum: Hello everyone, and thank you for taking our questions and congratulations on your progress on the pipeline. So we've discussed the potential for accelerated approval in the US for SRP 5051. Now, maybe going on the global aspect of this,

Hello, everyone and thank you for taking our questions and congratulations on your progress on pipeline.

So we've discussed the potential for accelerated approval and the U S for SRP 50 51.

Maybe going on the global aspect of this.

Douglas S. Ingram: Would there be challenges in enrolling a randomized study for SRP?

Would there be challenges and and rolling a randomized study for SRP 50, 51, assuming a placebo control for example.

Douglas S. Ingram: SRP 5051, assuming a placebo control, for example.

And I apologize I missed that I missed a piece of and it cut out can you repeat that for the last part of the question and I apologize.

Operator: I apologize, I missed the start, I missed a piece of it that was cut out. Can you repeat the last part of the question? Absolutely. Absolutely.

Yeah, absolutely so when thinking of a global study for SRP 50, 51 could there be potential challenges and enrollment if theres a placebo arm.

Douglas S. Ingram: Could there be potential challenges in enrollment if there's a placebo arm? Well, you know, I will say, I don't think there are going to be significant challenges in enrollment simply because, you know, Deshaumasphysrophy is a devastating, 100% fatal disease. I will also say, and let's not kid ourselves. In this rare disease, it is a very painful thing for patients to, you know, at times, accept the concept of placebo-controlled trials. They're willing to do it. We've seen this over and over again.

Well I will say I don't think there is going to be significant challenges enrollment simply because.

And the share muscular dystrophy is a devastating and 100% stable disease and I will also say and let's not let's not kid ourselves and this rare disease. It is a very painful thing for patients to the.

Time to accept the concept of placebo controlled trials they are willing to do it and we've seen this over and over again, we've done it with essence, which is a very significantly longer placebo controlled trial. We've done it with study 102 will deal with the upcoming 301, So I'd say we are.

Douglas S. Ingram: We've done it with essence, which is a very significantly, you know, longer placebo-controlled trial. We've done it with study 102. And we'll do it with the upcoming 301. So I'd say, you know, we've, we've, you know, we've.

We've got to be very mindful about these issues and think of the patient first on things like placebo controlled trials I do think if the ultimate answer is that we need a placebo controlled trial I do think we can enroll and and I think patients well, we'll do that particularly if its a thoughtful program I think the good thing about 50 51 is that we make dystrophin very <unk>.

Douglas S. Ingram: We've got to be very mindful about these issues and think of the patient first on things like placebo-controlled trials. I do think if the ultimate answer is that we need a placebo-controlled trial, I do think we can enroll it. I think patients will do that, particularly if it's a thoughtful program. I think, you know, the good thing about 50-51 is that we make districts very rapidly, so perhaps that means we can have a more moderated approach to this length of the study.

And so perhaps that means we can have a more moderated.

Our approach to this length of the study now as long as we're talking about ex U S. I think it's important to really focus on the value to the patient community from SRP 900, net I'm, sorry, 50, 51 with respect to ex U S.

We look back historically and our PMO exon does my honest and remind us those have all been approved for the accelerated approval process and the United States on.

Douglas S. Ingram: Now, as long as you're talking about XU-1, I think it's important to really focus on the value to the patient community from SRP 9-0-50-1 with respect to X-U-S. If we look back historically at our PMOs, Xondis, Vy, and Vyondis, those have all been approved through the accelerated approval process in the United States, on the basis of a biomarker that reasonably predicts clinical benefit, and then we've got studies thereafter on that. That mechanism doesn't exist outside of the United States in really any significant way.

On the basis of a biomarker earlier that reasonably.

Predicted clinical benefit and then we've got studies thereafter on that that mechanism doesn't exist outside of the United States and really any significant way and so that limits our ability to take that approach ex U S and bring people. The PMO forwards. So we do get ex U S sales and we have we.

Our managed access program ex U S and it has benefit and kids around the world, but generally speaking and this has been a largely U S.

U S. A service with our PMO and the P. P M and this could really change that.

And we're talking about a therapy that if we can confirm it and the next.

Douglas S. Ingram: And so that limits our ability to take that approach, XUS, and bring the PMO forward. So we do get XUS sales, and we have a managed access program, XUS, that has benefited kids around the world. But generally speaking, this has been a big U.S. U.S. service with our PMOs.

Clinical trial and part B this could induce dystrophin rapidly and it could reduce dystrophin that could be significantly over 10%, which I think most people would say is this is just a profound homerun amount of dystrophin and that very likely opens for us the opportunity to bring.

This therapy and a very lean way ex U S around the world, including Europe, We've already had in the past some discussions with EMA around these issues, but obviously, there's actually and we need to do separate from our discussions with the FDA.

Douglas S. Ingram: The PBMOs could really change. You know, we're talking about a therapy that, if we can confirm it in the next clinical trial in Part B, could induce, you know, dystrophin rapidly. And it could induce dystrophin that could be, you know, significantly over 10%, which I think most people would say is just a profound home run amount of dystrophin. And that very likely opens up for us the opportunity to bring this therapy in a very lean way, XUS, around the world, including Europe. We've already had some

And some discussions with some ministries of health around the world to ensure that we're building a critical development program, that's fit for their purposes as well as for the FDA purposes as well. So we will do all of that but it's important to us and important to the patient community of course, you can't be a U S centric organization with our therapies are kids around the world hundreds of thousands of kids.

Around the world and with Duchenne muscular dystrophy, who need treatment. So we've got to find an avenue for them and I think the P. BMO 50 could be warm and its progeny could be that avenue potentially and of course down the roads.

The next couple of years, so could <unk>.

P. Nine here as Youre one of course, we're significantly focused on the rest of the world with respect to other gene therapy as well.

Operator: Thank you. Our next question comes from the line of Colin Brist from UBS. Can I ask you a question, please?

Thank you. Our next question comes from the line of Cowen and Bristol from UBS. Your question. Please.

Colin Nigel Bristow: Good evening, and congrats on the quarter. So on 901001,

Good evening and congrats on the quarter, so on and there's a one you mentioned that the linear Kaye P. C. A touchscreen and method it's achieving.

Douglas S. Ingram: and you mentioned that the linear QPCR titering method

Douglas S. Ingram: achieving expression levels greater than you saw in part one of study 101. Looking at my notes, I think you said that you saw no correlation between discipline expression and function in part one. So can you confirm whether you saw a threshold relationship?

Expression levels, which are great for the MISO and part one study want to achieve.

Looking at my notes I think you said that you saw no correlation between distribute expression and function and part one. So can you confirm whether you saw a threshold relationship with expression levels and could you maybe put some levels around that and then just another quick one could you is there any interest on your part and pursuing a filing.

Douglas S. Ingram: with expression levels, and could you maybe put some levels around that, and then just

Douglas S. Ingram: Another quick one: is there any interest on your part in pursuing a filing strategy?

And even with the FDA based on the four to five year olds.

Douglas S. Ingram: with the FDA based on four to five-year-olds. Obviously, a healing endeavor looks promising. Thanks.

Obviously, a shooting and Devon looks okay. Thanks.

Douglas S. Ingram: Okay, thanks. Yeah, so on the first one, I want to make sure we did this right. I'm not suggesting there's no correlation between expression and function. They're unquestionably it, and that's why really maximizing expressions is extraordinarily important to us. I think what we were saying before, just to put a fine point on it, is when we look at part one of study 102, when we look at the six to seven-year-olds, which is really where you look, and you ask, why did we miss the primary endpoint? It was the six-to-seven-year-olds, and it was the baseline characteristics. And it wasn't the fact that we had variability in t Actually, we had variability in titering for the four to five-year-olds, and we knocked it out of the park.

Yes, so on the first one I want to make sure we did I'm not suggesting there is not a correlation between expression and function they're unquestionably yes.

And that's why we're really maximizing expression is extraordinarily important to us I think what we were saying before just to put a fine point on it is when we look at the.

The part one study one O two and we look at the six to seven year olds or is this really where you look.

And you you asked why did we missed the primary endpoint was the six to seven year old and it was the baseline characteristics and it wasn't the fact that we had variability and tightening in fact, we had variability and tighter and for the four to five year olds and we knocked it out and the park. So just so we're very clear about that and we do.

Douglas S. Ingram: So just so we're very clear about that, you know, we do think that more is better with respect to distristen, but the 28.1% we had in part one of 102, we believe, had we had the baseline characteristics right, would have correlated to significant functional benefits, and we would have hit our primary endpoint. So that's really what we're saying there. We don't have further data on the upcoming cohort, but we certainly don't have data on. We certainly don't have data on that.

And things that more is better with respect to dystrophin, but the 28, 1% we had in part one of 102, we believe.

How do we add the baseline characteristics right would have correlated to significant functional benefits and we would have hit our primary endpoint and so that's really what we're saying there we don't have further data on the.

The <unk>.

Coming cohort, we certainly don't have that on my three yet.

Douglas S. Ingram: 1-103 yet; we'll be getting that data in the very near term from an expression perspective as well. And then on the pathway forward with the division, let me say that we are not without. We want to be creative to find avenues to bring this therapy as rapidly as possible to patients suffering from Duchenne muscular dystrophy. And so we will have, you know, we'll have wide-ranging discussions with the agency over time.

Getting that data and the very near term from and expression perspective as well.

And then on the pathway forward with the Division, let me say that we are not without.

We wanted to be creative to find avenues to bring this therapy as rapidly as possible to patients suffering from duchenne muscular dystrophy and.

So we will have we will have wide ranging discussions with the agency over time I think for planning purposes.

Douglas S. Ingram: For planning purposes, I think it would be wise of people for planning purposes to presume that it is study 301 that will be our pathway to approval in the United States. And that's really, I think, the most probable approach that we're going to take. It isn't to suggest that we don't have other ideas and we're not thinking about things, and we won't have discussions with the division.

And it would be wise of people for planning purposes to presume that it is study 301 that will be our pathway to approval.

And the United States as our base case assumption and.

And that's really I think the most probable approach that we're going to take isn't I suggest that we don't have other ideas and what I'm thinking about things and it won't have discussions with the division, but I think if we're going to if we're going to plan and I think people should plan for 301 being the mechanism and that means we need to get to the division, we need to show them, our CMC and protocol and get their concurrence.

Douglas S. Ingram: But I think, you know, if we're going to plan, I think people should plan for 301 being the mechanism. And that means we need to get to the division. We need to show them our CMC and protocol and get their approval. We need to start 301 as soon as we can this year. We need to get that enrolled as fast as possible, and we need to get a read out of that as soon thereafter as possible.

And we need to start 301 as soon as we can this year, we need to get that enrolled as fast as possible, we need to get a read out of that as soon thereafter as possible. It will be a 52 week study and we can be and a good place to file a BLA. If we were successful and the United States and really not very far into the future. If we can just get going.

Douglas S. Ingram: It'll be a 52-week study. And we could be in a good place to file a BLA if we were successful in the United States and, you know, really not very far into the future if we can just get going this year. So that's, that I think, has to be our base case assumption for all of

This year. So that's that I think has to be our base case assumption all of us.

Operator: Thank you. Our next question comes from the line of Danielle Brill from Raymond James. Your question, please?

Thank you. Our next question comes from the line of Danielle Brill from Raymond James Your question. Please.

Danielle Brill: Yes, hi, good afternoon. This is Danielle on behalf of Danielle.

Hi, Good afternoon. This day to Neil on for Danielle and thank you for taking the question.

Douglas S. Ingram: Thank you for taking the question. Just very quickly, you mentioned previously that your plans are to enroll additional 4 to 5-year-olds in Study 103. Just wanted to ask if those things have been completed in those patients, and do you need to wait for those data before meeting with the agency to discuss plans for Phase 3?

And just very quickly you mentioned previously that you plan to enroll additional for the five year olds and.

Study 103, just wanted to ask you.

And he has been completed and those patients and do you need to wait for those data before meeting for agents.

And just to discuss plans for.

Douglas S. Ingram: So we're going to answer the last question first. We're not going to wait.

And for Phase III. Thank you.

So those are for the last question first we're not we're not going away. So the meeting with the agency is gonna be founded on the original the first 11 patients where sort of our initial cohort of patients. We did update the protocol as you may recall, so that we could add some additional patients to the protocol, we did that for purposes.

Douglas S. Ingram: So the meeting with the agency is going to be founded on the original, the first 11 patients, so our initial cohort of patients. We did update the protocol, as you may recall, so that we could add some additional patients to the protocol. We did that for purposes of sort of creatively thinking of what kind of data sets we need if we were going to have far more extensive discussions with the agency about faster pathways in the United States.

And sort of creatively thinking of.

What kind of data sets, we need if we were going to have far more mains and discussions with the agency about faster pathways and the United States toward that and we've dosed a significant number of them I don't know if we've dosed I suspect we haven't dosed all patients yet, but we've dosed a significant number of Peter.

Douglas S. Ingram: Toward that end, we've dosed a significant number of them. I don't know if we've dosed enough. I suspect we haven't dosed all patients yet, but we've dosed a significant number of people. Well, actually, I think, let me open the line to Dr. Louis Sardina Claypec. I can't tell if she's agreeing with me or disagreeing with me. Yeah, all the papers, yeah.

I actually I think let me, let me open the lines and Dr. Louise Rodino credit like I cant tell if she is agreeing with me or disagree with me.

Dr. Louis Sardina Claypec: Apologies. We're better than I thought, just all of those patients. But in any event, we don't, we are not going to wait, we don't wait for the outcome of those patients to be evaluated by the FDA. We, we are going to, our meeting with the FDA is predicated on our first cohort of patients. And that will occur, I've said before, around the middle of this year once we get the 103 data. Apology for misstating.

And apologies for better than I thought I apologize for that.

And just all of those places, but any of that and we don't we are not going away. We don't wait for the outcome of those patients for the FDA, we are going to our meeting with the FDA is predicated on a for scheduling of patients and.

And that'll work for as I've said before around the middle of this year and once we get the one offs readout at hand.

And apologies for misstating.

Our next question comes from the line of Brian's Koning from Baird. Your question. Please.

Operator: Our next question comes from the line of Brian Scorney from Baird. Your question, please.

Luke: Hi, this is Luke on for Brian. On 901 and 103 again, I guess, what can we expect to see in terms of individual patient levels of expression? Well,

Hi, This is Luke on for Brian on 90 day zero, one and one of three again I guess, what can we expect to see in terms of individual.

Patient level of expression.

Well I don't know if we've made all the decisions on the exact.

Douglas S. Ingram: Well, I don't know if we haven't made all the decisions on the exact presentation yet, but obviously, we haven't seen the data yet. I think, broadly speaking, what you should look for is the expression data, which will be both Western BOD as well as IF and the safety data.

<unk> and obviously, we haven't seen the data yet I think broadly speaking what you should look for is the expression data, which will be both western blot as well as I am and the safety data. That's that's the information and that's going to tell us about it I think what other things back.

Douglas S. Ingram: That's the information that's going to tell us about it. I think, you know, one of the things that That's the primary information. That'll tell us about the performance of the therapy. That'll tell us about what therapy we have for purposes of our clinical trials, and that'll give us a view of what this commercial process material is going to look like when we get approved. And then the nuanced details of that we haven't worked out, obviously.

And by the net.

The primary information that will tell us about the performance of the therapy that will tell us about what therapy rehab for purposes of our clinical trials and metal.

Give us a view of what this commercial process material and it looked like when we got approved and other than new.

New Washington, and tells US that we haven't worked out obviously.

Operator: Thank you. Our next question comes from the line. Salveen Richter from Goldman Sachs. Your question, please?

Thank you. Our next question comes from the line of <unk> Richter from Goldman Sachs. Your question. Please.

Sonia: Hi, this is Sonia from Unfer Salvine. Thank you so much for taking our question.

This is sonya on for solving and thank you so much for taking our question and the other.

Douglas S. Ingram: Beyond DMD, what other indications can the PPMO platform be applied to? And would you be able to do that in parallel?

D M D. What other indications Ken the P. P. M O platform be applied to and would you be able to do that and parallel.

Douglas S. Ingram: Yeah, I'm laughing because we were having this discussion just today, and I was saying, I don't want to get out of my skis and promise the particular disease days. There are a number of diseases that may very, that are very serious diseases that may benefit from this steric blocking technology.

Yeah, that's a great and.

Laughing because we were having this discussion just today and I was saying I don't Wanna get out over my skis and promise that.

Particular disease day, there are a number of disease.

Diseases that may vary.

They are very serious diseases that may benefit from this steric blocking technology, and we've talked about them and the past and you'll see them and we actually have some IP on some of them, but the.

Douglas S. Ingram: And we've talked about them in the past, and you'll see them, and we actually have some IP on some of them. But the discussion changes even more. significantly when you have not only a really precise therapy like our PMO, but you have a very much more potent version of that with our peptide conjugated PMO. So I guess what I would say is I don't want to discuss additional diseases because I feel like I'll be committing myself to things that we haven't yet decided as an organization.

The discussion changes even more significantly when you have not only a really precise.

Therapy like our PMO, but you have a very a much more potent version of that with our peptide conjugated PMO. So I guess, what I would say is I don't want to discuss additional diseases and I feel like I'll be committing myself the things that we haven't yet decided as an organization, but there is a cascade of opportunity here. So let's think about the near term.

Douglas S. Ingram: But there is a cascade of opportunity here. So let's think about the near term and the immediate and then right after the immediate. Mediate is, get through the agency, get their concurrence on the approach, and with their concurrence, start Part B for 50-51 itself.

And the immediate and then right after the immediate immediate is <unk>.

Get through the agencies.

Get their concurrence on the approach and with their concurrence start part B for 50 51 itself. The next great. The next big opportunity for us.

Douglas S. Ingram: The next big opportunity for us is the other Exxons. Remember, we're not an Exxon 51 company; we're a DMB company, and so the wonderful thing about our technology and the wonderful thing about Exxon skipping is there are very few mutations that wouldn't benefit from Exxon skipping and where this wouldn't work. So we could, at least theoretically, get to, overreactually, 80%. I think the estimates are up to almost 85% of kids with Duchenne where we could build constructs to serve that.

Is the other exon remember, we're not and exon 51 company worthy and the company and so the wonderful thing about our technology and the wonderful thing about exon skipping is there are very few mutations that wouldn't benefit from exon skipping and where this wouldn't work. So we could at least theoretically we could get to over 80%.

And the estimates are up to almost 85 per cent of kids with Duchenne, where we could build contracts to serve that that's the next big opportunity. The next big opportunity and this is a massive opportunity and my view is expanding the regions that we can go to so think about where this can take US right now we're doing very well.

Douglas S. Ingram: That's the next big opportunity. The next big opportunity, and this is a massive opportunity in my view, is expanding the regions that we can go to. So think about where this can take us. Right now, we're doing very well. We have been, Dallin Murray and his team have done a brilliant job.

It was Dallas Maria and as Jim has done a brilliant job we've been growing at 19% compounded growth and we.

Douglas S. Ingram: We've been growing at 19% compounded growth, and we've served, we've grown 25% this quarter. And, you know, that's just fantastic work. And by the way, I will note, no price increase on that, no change in pricing, parity pricing. So that's all serving the community. And that's tremendous work. But imagine what we could do if we could actually treat not just the 10,000 children in the U.S. but some XU.S. sales, but actually get a pretty good profit.

Served we've grown 25% this quarter.

And and that's just fantastic work and by the way I will note no price increase and that no change and pricing parity pricing. So that's all serving the community and that is tremendous work.

But imagine what we could do if we could actually treat not for 10000 children and the U S and some ex U S sales, but actually get approvals around the world I mean that would just multiply the population of patients that could benefit from our RNA splicing technology and then the next one there and I'm not suggesting this is this is chronic chronological.

Douglas S. Ingram: around the world. I mean, that would just multiply the population of patients that could benefit from our RNA splicing technology. And then the next one there, and I'm not suggesting this is chronic chronological because we're going to be looking at all of these, you know, very soon, is, okay, now that we have a very potent new version of our PMO and we're getting confident about it, where do we take this? What's the next disease state or two disease states that we take this to?

Because we're going to be looking at all of these you know very soon and is okay. Now that we have a very potent new version of our PMO.

And we're getting confident about it where do we take this was what's the next disease state or two disease states that we take this and that's something we're going to be looking at over the course of the rest of this year, but I don't want to commit ourselves to it yet because we've got a lot of work to do to really decide what the what the smartest next move is for the P. BMO post 50, 51 and and the other.

Douglas S. Ingram: And that's something we're going to be looking at over the course of the rest of this year. But I don't want to commit ourselves to it yet because we've got a lot of work to do to really decide what the smartest next move is for the PPMO post 50-50 Lyman and then the other end.

Operator: Thank you. Our next question comes from the line. Tim Lugo from William Blair. Your question, please?

And excellence.

Thank you. Our next question comes from the line of Tim Lugo from William Blair. Your question. Please.

Thanks for taking the question.

Tim Lugo: So as you start to think about XUS and the PPMO platform, you signed a deal for XUS for microdistrophine gene therapy. Can you maybe discuss why you would take PPM to Europe on your own while you still have a partner for the gene therapy products and your kind of thinking around going alone versus partnership with PPMO? But we'll have to have some discussions with our partner Roche regarding that. Roche has an option for the RNA technology XUS, so we'll have discussions with them about that. But either way, I mean, let me be very clear about this.

So as we start to think about ex U S and the P. P. M. O platform you signed a deal for ex U S for micro Dystrophin gene therapy can you maybe discuss why you would take TTM out of Europe on your own while you still have a partner for the gene therapy product and kind of your thinking around <unk>.

Alone versus partnership with P. P M.

But we'll have to have some discussions with our partner Roche regarding that but the Roche has an option to the RNA technology ex U S and will have discussions with them about that but.

But either way I mean, and then it would be very clear about this our first and foremost goal is to develop therapies to benefit.

Douglas S. Ingram: Our first and foremost goal is to develop therapies to benefit children around the world, including children with Dushanastal dystrophy. And, you know, either directly, ourselves or through our partner, Roche, or through someone else, we're going to serve that community. So your point is good one. The approach that we take to getting the therapy to other places around the world may vary depending on, you know, discussions that we have with others, including our partner Roche, that we're here with with SRP901. But we'll get this. If the therapy bears out as successful, we'll do everything we can to make sure that kids around the world get access to this therapy.

Benefit children around the world, including children, with Duchenne muscular dystrophy, and either directly ourselves or through our partner Roche or through someone else sort of net community. So your points and good one and I mean, that's the approach that we take to getting the therapy to other places around the world.

It may vary depending on discussions that we have with others, including our partner Roche net.

And here there with SRP nine years here of one <unk>.

But we'll get there.

The therapy, there was out and successful worthy everything we can and make sure that kids around the world and get access to this therapy.

Operator: Thank you. Our next question comes from the line of Envita Gupta from Cowan. Your question, please.

Thank you. Our next question comes from the line event and and Vita Gupta from Cowen Your question. Please.

Anvita Gupta: Hi, this is Anwithan for Rithu. Thanks for taking our questions and congratulations on the recent progress.

Hi, guys. This is Andrew on for Ritu, Thanks for taking our questions and congrats on the recent progress.

Douglas S. Ingram: So based on your insights gained from study 1 or 2 Part A and acknowledging that this is an ongoing process, but what might study 3 or 1 size inclusion and exclusion criteria look like? And would it have an interim analysis built in? Like perhaps also looking at muscle fat content via MRIs at an earlier time point. So any color there would be really used. Yes.

So based on your insights gained from study one and two pints and I'm kind of thing that this is an ongoing process, but for my study to be a one size and inclusion exclusion criteria and look like and who didn't have an interim analysis and like perhaps I'll say looking at myself back content via MRI and <unk>.

I'm point for any color there would be really useful. Thank you, yes. So.

Douglas S. Ingram: Yes. But let me be very clear.

Douglas S. Ingram: Those are great questions, and I apologize if I frustrated you and told you that I'm not going to answer. Then I apologize. We have done, and they're all the right questions, you know, what's the right end, what's the, how we're looking at endpoints, what's the exclusion and inclusion criteria, are we refining things, you know, are we looking at other aspects of the trial? So we build the protocols around a lot of the issues that you raise.

Now let me be very clear those are all those are great questions and I apologize to frustrate you and tell you that I'm not going to answer that and I apologize.

And there are all the right questions, what's the right and what's that.

Looking at endpoints, what's exclusion and inclusion or we were finding things and you know we're looking at other other aspects and the trial. So we built a protocols around a lot of the issues that you raised.

Douglas S. Ingram: I will, there are a couple of reasons why I want to wait to discuss the protocols with the outside world. First, of course, is that the protocol, in a very real sense, is tentative until we have discussions with the division. and get their input and get their views. And the second one, of course, is that we have a lot of, from our perspective, a lot of insight and proprietary insight around the thoughtful way to build the next trial in a way that will maximize the probability of success and speed.

Will.

A couple of reasons why I want to wait to discuss the protocols with the outside World first of course is that the protocol and a very real sense is tentative until we have discussions with the division and how to get their input and their views and the second one of course is that we have a lot of.

From our perspective, a lot of insight and proprietary insight around the thoughtful way to build the next trial and.

And a way that will maximize the probability of success and speed and so we want to meet with the agency to get their input and then I promise you. We will provide all of that information, but it'll be post the meeting with the agency.

Douglas S. Ingram: And so we want to meet with the agency, get their input, and then, I promise you, we will provide all of that information, but it'll be after the meeting with the agency at or right around the time that that trial commences, and we'll share it.

And at or right around the time that we'll be we'll commence that trial and we'll share it with the world.

Operator: Thank you. Our next question comes from the line of Defei Yang from Mizzell. Your question, please?

Thank you. Our next question comes from the line of day pay Yang from Mizuho. Your question. Please.

Alex: Hi, good afternoon; this is Alex from DFA. Thanks for taking the question. So just one on the LGMD gene therapy program and the recent presentation at the MDM meeting. In cohort one, we saw increasing district expression over time, but DCN and protein intensity were

Hi, Good afternoon, everyone and this is Alex on for a day say thanks for taking the question. So just one on the LG and <unk> gene therapy program and the recent presentation at the MGM meeting and.

Cohort, one we saw increasing dystrophin expression over time, the bcm and and protein intensity was a bit lower.

Douglas S. Ingram: a bit lower, but it was going down over time. I'm wondering if you could give us some details on that. How should we think about that? Are we reading too much into the VCN data here?

And that's going down over time and I'm wondering if you could.

Give us some details on that how are we how should we think about that and we.

You're reading too much into the the bcm data here and or.

Louise R. Rodino: and data here, or was there any changing methodology for these measurements? Thank you. I think Louisa is going to say you're probably, we're probably reading too much into the BCN, but I'll turn it over to Dr. Radino-Caq so she can respond. Yeah, just

Was there and changing methodologies for these measurements. Thank you.

And we said is going to say, you're probably we'd probably be reading too much into the V C and but I'll turn it over to dark for really don't play back to respond.

Yeah, just to so we try and beta sacrifice and expression day lockdown measures by Western as you said and also by them and you have for asking for a number of positive fibers and tested and the back of copying and for all within the range that we saw within the first cohort and we didn't see anything significant in terms of and <unk>.

Louise R. Rodino: Yeah, so we started beta-circlicin expression by multiple measures by Western, as you said, and also by immunophoresis, both by a number of positive fibers, intensity, and a vector copy, and those were all within the range that we saw within the first cohort, so we didn't see anything significant in terms of drop-it. We were very pleased with the durability that we saw over that course of time and also the increased expression of the dystrofen associated protein. We were quite pleased with the results we saw two years in the Lodos cohort and continued in the high dose as well.

And we're very pleased that the durability of that we saw over that for some time and and also the increase.

And expression and dystrophin associated protein complex now.

And quite pleased with the results, we thought 10 years and download that cohort and continued and the high dose as well.

Operator: Thank you. Our next question comes from the line of Anupamra from JP Morgan. Your question, please?

Great. Thank you.

Thank you. Our next question comes from the line of nuclear and Remo from J P. Morgan Your question. Please.

Tessa: Hi, this is Tessa on for Anupon this evening. Thanks so much for taking our question. Just one question from us. Bridging from the Pfizer news earlier this week, a question that we've been thinking about is if every D&D gene therapy sponsor could run into some of the similar issues with respect to the potency assay that you have overcome. What are your thoughts here and what advantage could sort? up to have if you guys are the only ones who can dose in the U.S. for a gene therapy trial? Thanks so much.

Hey, guys. This is tessa.

For this evening. Thanks, so much for taking my question and.

Just one question for Mike.

And the bridging from the Pfizer News earlier this week a question that we've been thinking about and if.

And every DMD gene therapy on credit.

Right in Q and this is Jim.

And she is with respect to the potency assay and that you have over time.

What are your thoughts here and what advantage, Chris threat to have and.

You guys are the only ones you can dose and the U S.

For our gene therapy trial, thanks, so much.

Douglas S. Ingram: And thanks. But I, you know, obviously, if we were the only ones that had the ability to start the trial in the U.S., the advantage to that would be astronomical. I don't want to get out over my skis and say that.

Yeah. Thanks Bruce.

Obviously, we were the only ones that have the ability to start the trial for U S advantage to that would be astronomical.

I don't want to get out over my skis and say that I wanted to let me, let me start with an enormous amount of humility and we've got to meet with the division ourselves now I do think.

Douglas S. Ingram: I want to, let me, let me start with an enormous amount of humility. We've got to deal with the division ourselves. Now, I do think that we have benefited from the fact that we had an opportunity to dialogue around potency assays and expectations with the division last year in the sort of September into October timeframe. And I'd like to believe that that's really benefited us and really provided us with the kind of direction.

But we have benefited from the fact that we had an opportunity to dialogue around potency assays and expectations.

And with the division last year, and the sort of September and.

The October timeframe and.

I like to believe that that's really benefited us and really provided us with the kind of direction and we've built our potency assays and even evolved our potency assays based on those discussions so that as we prepare for our meeting with the division around the middle of this year.

Douglas S. Ingram: And we've built our potency assays and even evolved our potency assays based on those discussions so that as we prepare for our meeting with the division around the middle of this kind of direction this year, I can at least say that we are confident in our data package. I think we have a very good data package. I think we have a, I think, a very thoughtful approach to potency assays.

I can I can at least and say that we are confident and our data package I think we have a very good data package I think we have I think a very.

Fossil approach to potency assays I think be the data that we have on things and things like and we'll just focus just on potency, but things like on the potency assay.

Douglas S. Ingram: I think the data that we have on things like, I don't just focus just on potency, but things like the potency assay are, you know, very, very good, very positive data, and we think we have a very strong package. I still want to be very clear.

Is.

Very very good very positive data and we think we have a very strong package I still want to be very clear, we've got and meet with the agency ourselves and we got to talk CMC and talk to the protocol and get the agency's blessing and then we'll commence our next trial I think guessing and United States is a very important issue of course and.

Douglas S. Ingram: We've got to meet with the agency ourselves. We've got to discuss CMC and discuss the protocol and get the agency's blessing, and then we'll commence our next trial. I think dosing in the United States is a very important issue, of course. And then do you even need me to say that?

And I think anyone even leads me to say Hey, Matt.

Douglas S. Ingram: And so it's a big focus of ours to have a successful meeting with the agency. I think we're going to be in great shape with it. Let us assume that the study one or three data looks good as well. We'll have that data shortly, and then, armed with all of our CMC data, with our 103 data, with the data that we've seen in part one and 102 and our protocol, I think we'll have a very productive discussion with the division and then, hopefully, if all goes well, be able to start that dosing in the United States and around the world very shortly thereafter this year.

And so it's a big focus of ours to have a successful meeting with the agency and I think we're gonna be in great shape with it let us assume that the study one or three data looks good as well, we'll have that data shortly and then armed and all of our CMC data with our one O three data with the data that we've seen and part one and one or two.

And our protocol I think we'll have a very productive discussion with the division and then hopefully if all goes well and be able to start that dosing and the United States and around the world very shortly thereafter this year.

Operator: Thank you. This does conclude the question and answer session for today's program. I'd like to hand the program back to Doug In Group for any further remarks.

Thank you. This does conclude the question and answer session of today's program I'd like to hand, the program back to Doug Ingram for any further remarks.

Douglas S. Ingram: Okay, thank you very much. Jonathan, thank everyone for joining us, and thanks for your very thoughtful questions this evening. I think it's pretty obvious from our remarks and from our answers to the questions, but we have a lot to do this year. We have a very focused team. We'll have a number of very significant readouts and milestones over the course of 2021 into 2022. I've got, in my view, a fantastic team, probably the best team we've ever had, very focused and energetic about getting things done.

Okay. Thank you very much Jonathan and thanks, everyone for joining us and thanks for your very thoughtful questions. This evening I think it's pretty obvious from our remarks and for my answers to the questions that we have a lot to do this year. It was very focused team will have a number of very significant readouts and milestones over the course.

2021 into 2022, I've got and my view, a fantastic team and probably the best Jim we've ever had a very focused and energetic about getting things done and advancing these therapies and certainly as we all know we have a mission that matters and we're getting up every day fighting like Mad to advance.

Douglas S. Ingram: done and advancing these therapies. And certainly, as we all know, we have a mission that matters. So we're getting up every day fighting like mad to advance these therapies and improve the lives of patients with rare diseases, including but not limited to the children that Duchenne Muscle District in Dyshire and all of the children and adults who have the very slim girdles on which we're developing therapies. And that is only the tip of the iceberg of the work that our team, including our research team, or during to advance our programs. So thank you all very much, and I look forward to additional updates across the course of 2021 as we read them out.

These therapies and improve the lives of patients with rare disease, including but not limited to the children, who have duchenne muscular dystrophy, and all of the children and adults who have for various limb girdles on which we're developing therapies and that is only that is only the tip of the iceberg of the work that our team, including our research team are doing to advance our <unk>.

Programs. So thank you all very much and I look forward to additional updates across the course of 2021 as we read them out.

Operator: Thank you, ladies and gentlemen, for your participation in today's conference. This does not include the program. You may now disconnect. Good day, and and

Thank you, ladies and gentlemen for your participation in today's conference. This does conclude the program you may now disconnect good day.

[music].