Q3 2023 Sarepta Therapeutics Inc Earnings Call

Okay.

Good afternoon and welcome.

The therapeutics third quarter 2023 earnings call at this time, all participants are in a listen only mode.

The speaker's presentation, there will be a question and answer session to ask a question. During this session you will need to press star one one on your telephone.

You will then hear an automated message advising you're hanging just raised to work.

Draw. Your question. Please press Star one one again please be advised that today's conference is being recorded at this time I'll turn the call over to Francesca Nolan Executive Director Investor Relations and corporate Communications. Please go ahead.

Thank you Michelle and thank you all for joining today's call earlier. This afternoon, we released our financial results for the third quarter of 2023.

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

Joining us on the call today are Doug Ingram, E&S coupon Dallin, Maury and Dr. Louise Rodino Clay pack after our formal remarks, well open the call for Q&A.

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 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 affect the business the results of operations and trading prices for <unk> common stock.

For a detailed description of applicable risks and uncertainties. We encourage you to review the company's most recent quarterly report on Form 10-Q filed with the SEC as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward looking statements.

Including any financial projections provided today based on subsequent events or circumstances.

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

Thank you Fred good afternoon, and thank you for joining US directed Therapeutics third quarter 2023 financial results Conference call.

It was only two days ago, we announced the results of our double blind placebo control controlled trial embarked therefore, I will not linger on the results here, but I will begin by summarizing strep this perspective.

First taken as a whole the results of embark confirm that elaborative stabilizer muscles slows or entirely arrest decline doesn't show across the ages and does so with a laudable safety profile not shared by other programs for Duchenne.

I can be embark results of not only satisfy the confirmatory requirements for our June approval, but have shown little evidenced benefits patients across age is consistent with its mechanism of action.

Hence we will soon be submitting a BLA couple of back to broaden the elaborate its label to remove agent ambulation restrictions and finally, we have already engaged in productive and encouraging discussions with FDA and they have confirmed that they are committed to reviewing an application to broaden the label and are committed to do.

And so rapidly now.

Now, let me comment on quarterly performance in the third quarter was a defining moment for strep, we launched out for therapy and the first gene therapy for boys with Duchenne muscular dystrophy, we continued to drive great performance with our three P. M O's and importantly on a non-GAAP basis, we achieved profitability.

Racing us an ever more rarefied biotech territory.

As you will have seen in our release led by an exceptional launch of <unk> and continuing performance of our three approved PMO exon based by August and our mothers.

Third quarter total revenue came in at $332 million and total net product revenue stands at 393 $2 million growing 49% over the same quarter last year.

Collecting the team's ability to execute and serve.

<unk> patients <unk> net product revenue came in at 69 point $11 million nearly tripled mean external consensus.

Likewise, our PMO has achieved 240 point to $1 million in net product revenue growing 16% over the same quarter prior year and non-GAAP earnings stood at $38 million in the quarter, a major milestone for <unk> as we transition to a <unk>.

<unk> and in the near term cash flow positive organization.

Uh huh.

The team has done a tremendous job working with families physicians and payers in the quarter and it shows.

These results.

Alan Murray, our Chief customer Officer will walk you through what has been nothing short of a remarkable launch of elaborate as I'm looking to the near future. We will take our proven track record of execution and move forward rapidly to expand the label of elaborate as so this team can employ that level of execution.

Can make a lot of it is available to the majority of Duchenne patients in the United States. Following balanced discussion Dr. Regina payback will provide an update on our pipeline progress.

Now as you would expect we are not providing updated guidance. This early in the launch but also obviously across our four approved therapies.

We're going to substantially exceed $1 billion. This year another important milestone to be sure.

And with that let me turn the call to Darling for a commercial update Alan.

Thank you Doug and good afternoon.

Launch of <unk> in the third quarter was <unk> for Duchenne March it was by far the most complex and challenging to date.

I am proud to say the team was ready on day, one and they have knocked it out of the park thus far.

As Douglas mentioned, we generated just over 69 million of net product revenues in the third quarter for <unk>.

Notably the team exceeded our own lofty site readiness expectations.

With nearly 70 sites ready to dose today.

This helps us support the patients at risk of aging out today.

It also sets us up for longer term success going forward.

We approached this launch by building organically upon what was already best in class Duchenne commercial and medical teams.

We put additional responsibility on everyone across the board rather than building out separate gene therapy teams and do they have all stepped up magnificently to meet this important moment.

Our early success was achieved in three ways first.

While our execution with our external stakeholders that is the neuromuscular kols gene therapy sites of care and the eligible patients they serve.

Secondly, proactive payer engagement to expedite access for those patients who are eligible based on the label.

Third establishing a well functioning flexible distribution model supported by the <unk> team to get each patient customized kit to the site of care at the right time, just as high for the operation.

I would like to take a moment now to recognize the Duchenne community and how they came together to expedite access for patients who are eligible for <unk>.

Led by our doctors nurses and other HCP also.

All stakeholders, we're ready for this important moment and it was humbling to see the whole community come together to support eligible patients in their treatment journey.

This launch also demonstrated the progress our teams and the experts have made in the past several years educating the payers about duchenne.

We were gratified by the urgency of payers that expediting policies that allowed access for eligible patients.

Additionally, and importantly, the payers played a key role in supporting patients who are at risk of aging out.

Saying all of that we still have much more work to do with some of the payers to achieve our goal of <unk>.

Securing access and treatment for all eligible patients across the country regardless of plan.

The team is working diligently as we speak educating payers on the robustness of the newly available embarked data. We're confident that this data set the stage nicely for access to align with our label today as well as whenever he gave me a broader label.

This all started in Q3 was a function of the team's efforts in the quarter itself.

And just as importantly, their efforts over the past seven years building the model, which we have established to support all of the duchenne patients eligible prior therapies.

Had this been throughout this first launch our trajectory in the third quarter would have been very different and much slower.

We gained deep knowledge and expertise through three PMO launches.

And I'm glad to say, we were able to apply these learnings to the launch of <unk>.

So to summarize all of it is.

It was a great first quarter for the launch because our team and our key stakeholders, we're prepared and they executed flawlessly to support the patients we serve.

Driven in large part by the robust 11 its revenue in the third quarter. We grew overall net product revenue by roughly 30% over the prior quarter net product revenue in Q3 of 2023 was $309 3 million.

Importantly, as Doug said in addition to our success with <unk>. We also had our most successful quarter ever serving patients with our established PMO franchise.

We see continued opportunities in the U S and globally for our PMO business. In spite of the fact that we also expect cannibalization from our loved us overtime.

Is the four to five population represents far less than 10% of the PMO business in the U S. This cannibalization will not have a material impact on our 2023 net product revenue.

I'd like to take a moment here to thank all of those who are relentlessly supported our PMO patients.

So as a result of the whole team's effort.

The net product revenue from our PMO business in Q3 was $240 million, representing roughly 16% increase over the same quarter in 2022 looking now at each of our PMO individually.

Product revenue for Exalt is 51 was $142 3 million in Q3, which was over 16% above the same quarter in 2022.

This 53 net product revenue was $31 7 million.

3.4% above Q3 of 2022.

And a modest 45 generated net product revenue of $66 3 million in Q3 of 2023. This represents roughly 21% growth over Q3.

2022.

As we've mentioned in previous calls we are in the mature phase of the market now for all three of our approved Pmo's as a result, while we expect the U S growth to continue to flatten.

And the ex U S revenues, while still in the growth phase to remain lumpy from quarter to quarter, and thus difficult to project on a quarterly basis.

Taken together, we can reiterate our guidance of greater than 925 million in net product revenue for our PMO business in 2023.

Okay.

I'll end by saying that I've been continually amazed and impressed by the resilience commitment and execution of our psoroptic teams over the years.

And while the success over those years has been impressive what the teams have achieved in the third quarter of 2023 stands above and beyond anything I've seen in my 10 years working to serve the Duchenne community.

The future is bright for <unk> and for the Duchenne community, who have been waiting for and very much deserved this progress.

Words can't adequately express just how proud I am of our whole team the individual stories from across the country are too numerous to mention here.

Nor can we as the team put into words, the joy, we feel when we celebrate each and every patient who gains access to any of our dystrophin restoration therapies.

And so with that I'll turn the call over to Dr. Louise Rodino <unk>.

Thanks, Don.

Our commitment to the science remains steadfast in our goal to change the lives of patients with rare diseases unwavering.

Our opportunity to do good and deliberate left for those living with Duchenne limb girdle and many other diseases for which therapies are either an adequate or nonexistent.

As Doug has already detailed the Embarq results I'll focus my comments on the progress of our gene therapy and RNA programs.

First the limb girdle muscular dystrophy or L. G M D wave.

We remain committed to advancing our L. G M D portfolio across a variety of subtypes and look forward to providing continuous updates on these important programs in the months ahead.

We presented on our <unk> pipeline. This past weekend at the speak Foundation 2023 International L. G. M D Conference.

As shared our urgency with the community to bring forth genetic medicines for ALS J&J.

Unknown Executive: Good afternoon, and welcome to the Sarepta Therapeutics Third Quarter 2021 earnings call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1-1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1-1 again. Please be advised that today's conference is being recorded.

To begin we made excellent progress for voyaging, our phase one study evaluating SRP 9003 for the treatment of limb girdle muscular dystrophy type to eat in ambulant adult patients and non ambulant patients isn't clinical process SRP 9003 material.

We are pleased to report that we completed enrollment in Voyager and we remain on track to initiate our phase III study using commercially representative processed material later this year.

Combined with positive expression and functional data shared from our initial study SRP nine carroll's or all three 101, we believe the data from voyaging will give us insights into a broader patient population.

Francesca Nolan: At this time, I'll turn the call over to Francesca Nolan, Executive Director, Ambassador Relations and Corporate Communications. Please go ahead. Thank you, Michelle, and thank you all for joining today's call.

We are also excited to report that we completed dosing in our systemic pilot study now the gene for SRP 6004, dual vector alright, 74 mediated gene therapy to treat individuals with LG M D to b.

Francesca Nolan: Earlier this afternoon, we released our financial results to the third quarter of 2023. The press release is available on our website at surrupted.com, and our 10-2 was followed with the Securities and Exchange Commission this afternoon. Joining us on the call today are Doug Ingram, A&S Tepon, Dow & Murray, and Dr. Louisa Roudino Claypac. 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.

L D M D to B is characterized by the absence of the protein gets Charlotte.

The innovative tool vector strategy allows us to deliver the full month just throw in gene the sole cause of L. G. M D C.

We look forward to reporting results from this study in the first half of 2024.

As mentioned last quarter, our L. G M D natural history study of the circles like an off the base.

Francesca Nolan: Please take a moment to review our slide on the webcast, which contains forward-looking statements. These forward-looking statements involve risks and certainties many of which are beyond surrupted control. Actual results could materialy differ from these forward-looking statements, any such risks can materialy and adversely affect the business, the results of operations and trading prices for surrupted common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent poorly report on Form 10Q, followed with the SEC, as well as the company's other SEC filings. The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today, based on subsequent events or circumstances.

M D to E two C and T D.

<unk> journey has been fully enrolled and we will follow patients for 36 months.

We continue to make progress and scalable manufacturing for all of our LGD candidates in our pipeline and look forward to initiating clinical studies as rapidly as possible.

Turning now to the progress we've made with our RNA platform.

We were pleased to complete enrollment in the first quarter of 2023 for a momentous study for SRP 50, 51, and we're targeting readout of the study in 2023.

Regarding our post marketing studies for the P&L as mentioned last quarter, we completed enrollment in the essence trial post marketing requirement for Golden person in CASM. MRSA is a reminder, ISS has a two year study and is due to read out in early 2026.

Doug Ingram: And now we'll turn the call over to our President and CEO Doug Ingram, who will provide an overview of our recent progress. Thank you, Fran.

Finally, we are pleased to have completed enrollment in our mission study our dose ranging post marketing commitment for example.

Doug Ingram: Good afternoon, and thank you for joining us to wrap up the therapeutic third quarter 2021 financial results conference call. It was only two days ago that we announced the results of our double-blind, placebo-controlled trial and bark, therefore I will not linger on the results here, but I will begin by summarizing surrupted as perspective. First, taking as a whole, the results of the bark confirm that a levittal stabilizes muscles, slows or entirely arrests decline, does so across to ages, and does so with a lot of will save the profile not shared by other programs for new shifts.

Mission is a randomized double blind safety.

Doug Ingram: Second, the embark results have not only satisfied the confirmatory requirements for our June approval, but has shown that a levittal benefit patient across ages consistent with its mechanism of action. Hence, we will soon be submitting a BLA supplement to broad and the 11th label to remove age and abulation restrictions. And finally, we have already engaged in productive and encouraging discussions with FDA and they have confirmed that they are committed to reviewing an application to broaden the label and are committed to doing so rapidly.

Doug Ingram: Now, let me comment on quarterly performance. The third quarter was a defining moment for Sarepta. We launched our fourth therapy and the first gene therapy for boys with Dushan Muscoa District. We continued to drive great performance with our three PMOs and importantly on a non-gap basis we achieved profitability, placing us in ever more rarefied biotech territory. As you have seen in our release led by an exceptional launch of 11th and continuing performance of our three approved PMOs, Exandes, Mayandes, and Amandes.

Well said Louise good afternoon, everyone.

Afternoon financial results press release provided details for the third quarter of 2023 on a non-GAAP basis as well as the gap basis. Please refer to our press release available on strapped. His website for full reconciliation of GAAP to non-GAAP financial results were obviously quite pleased with the financial results for this quarter on the back of a tremendous start for.

The goddess launch, we actually achieve non-GAAP profitability and assuming an expansion to the label to the broader <unk> population, we should achieve sustained profitability.

Doug Ingram: Third quarter total revenue came in at $302 million. And total net product revenue stands at $309.32 million growing 49% over the same quarter last year. Reflecting the team's ability to execute and serve to shen patients. 11th is net product revenue came in at $69.11 million nearly tripled mean external consensus. Likewise, our PMOs achieved $240.21 million in net product revenue growing 16% over the same quarter prior year. And non-gap earnings stood at $38 million in the quarter.

We're quite thrill to achieve this milestone just in the first quarter of the launch.

For the three months ended September 30th 2023. The company recorded total revenues of $331.8 million, which consists of net product revenues in collaboration revenues compared to revenues of $230.3 million for the same period of 2022, an increase of $101.5 million.

Net product revenue for the third quarter of 2023 from <unk> with $69.1 million <unk>.

And that product revenue for the same period for.

Exxon skipping franchise with $240.2 million compared to $207.8 million for the same period of 2022.

For the quarter individual in that product sales or $142.3 million. For example is 50 $166.3 million for a mind, it's 45 and $31.7 million for Bion has 53, increasing that product revenue primarily reflects increasing demand for our PMO products as well as in that product revenue.

Doug Ingram: A major milestone for us as we transition to a profitable and in the near term cash flow positive organization. The team has done a tremendous job working with families, physicians, and payers in the quarter and it shows in these results.

Dallan Murray: Dalai Murray, our chief customer officer, will walk you through what has been nothing short of a remarkable launch of a lebedess. And looking to the near future, we will take our proven track record of execution and move forward rapidly to expand the label of a lebedess.

She hated with the sales of elaborated.

And each of the quarters and it's September 30th 2023, and 20 twenty-two we recognized 22 and a half million dollars with collaboration revenue, which relates to our collaboration arrangement with Roche.

Reimburse Milco development costs under the grocery agreement totaled $34.9 million for the third quarter of 2023 compared to $22 million for the same period of 2022.

Dallan Murray: So this team can employ that level of execution to make a lebedess available to the majority of duchin patients in the United States.

Doug Ingram: Following Dalai's discussion, Dr. Regino Clayback will provide an update on our pipeline progress. Now as you would expect, we are not providing updated guidance this early in the launch, but also obviously across our four approved therapies. We are going to substantially exceed $1 billion this year, another important milestone to be sure.

On a gap basis, we reported a net loss of $40.9 million or 46.

Per basic and diluted share and $257.7 million or $2.94 per basic and diluted chair for the third quarter of 2023 and 2022, respectively.

Report it non-GAAP net income of $37.7 million or 37 cents per per diluted share in the third quarter of 2023 compared to a non-GAAP net loss of $70 million or 80 cents per diluted share in the third quarter of 2022.

Dallan Murray: And with that, let me turn the call to Dalai for a commercial update. Dalai. Thank you, Doug, and good afternoon.

Dallan Murray: The launch of a lebedess in the third quarter was Sireptus Force Gushan launch. It was by far the most complex and challenging to date. And I'm proud to say the team was ready on day one, and they have knocked it out of the park thus far. As Doug has mentioned, we generated just over $69 million in that product revenues in the third quarter for 11. Joseph. Notably, the team exceeded our own lofty site readiness expectations with nearly 70 sites ready to those today.

In the third quarter of 20th 23, we recorded approximately $37 million and the cost of sale compared to $40 million for the same period of 2022.

The decrease in cost of sale, primarily reflects right off a certain bashes a R. P. M O products not meeting our quality specifications for the three months ended September 30th 2022 with no similar activity for the same period of 2023, partially offset by increasing demand for R. P M will products.

On a gap basis, we recorded $194.3 million and $216.7 million in R&D expenses for the third quarter of 2023, and 2022, respectively, a year over year decrease of $22.4 million.

Dallan Murray: This helps us support the patients at risk of aging out today, and also sets us up for longer term success going forward. We approached this launch by building organically upon what was already best in class Duchenne commercial and medical teams. We put additional resources on responsibility on everyone across the board, rather than building out separate gene therapy teams, and they have all stepped up magnificently to meet this important moment.

The decrease is primarily due to a decrease in our manufacturing expenses, partially offset by increases in clinical trial expenses.

On a non-GAAP basis, R&D expenses for $163.9 million for the third quarter of 2023 compared to $193.7 million for the same period 2022, a decrease of $29.8 million.

Dallan Murray: Our early success was achieved in three ways. First, flawless execution with our external stakeholders, that is the neuromuscular KOL gene therapy sites of care, and the eligible patients they serve. Secondly, proactive payer engagement to expedite access for those patients who are eligible based on the label. And third, establishing a well-functioning, flexible distribution model, supported by the Sarepta Therapeutics team, to get each patient customized kit to the site of care at the right time, just in time for the infusion.

Now turning to SG&A on a gap basis, we recorded approximately $120.9 million and $104.8 million of expenses for the third quarter of 2023, and 2022, respectively, an increase of $16.1 million.

The increase was driven primarily by an increase in professional services and compensation and other personnel expenses, partially offset by a decrease in stock based compensation on a non-GAAP basis. The SG&A expenses were $92.8 million for the third quarter of 2023 compared to $66.8 million for the same period of 2022 and.

Dallan Murray: I would like to take a moment now to recognize the Duchenne community and how they came together to expedite access for patients who are eligible for a levitous led by our doctors, nurses, and other HP's all stakeholders were ready for this important moment, and it was humbling to see the whole community come together to support eligible patients in their treatment journey. This launch also demonstrated the progress our teams and the experts have made in the past several years educating the payers about Duchenne. We were gratified by the urgency of payers and expediting policies that allowed access for eligible patients. Additionally, and importantly, the payers played a key role in supporting patients who were at risk of aging out.

The increase of $26 million.

On a gap basis, we recorded $12.3 million in other expense net for the third quarter of 2023 compared to $400000 and other income net for the same period of 2022, the changes primarily due to the impairment of our investment in Boston contingent consideration that partially offset by inquiry.

He says and increase your I'm, an investment discount net and increases in interest income due to the investment mix of our investment portfolio as well as reductions of interest expense incurred as a result of the repayment of our December 2019 term loan during 2022.

We had approximately $1.8 billion in cash cash equivalent to investments in longterm restricted cash as of September 30th 2023.

Really pleased with the amount of capital on our balance sheet been turbulent market, we know cash becomes even more valuable we continually evaluate our expenses that said based on the embark resolved in the information we have today, there's no better use of our cash them to build inventory to serve those with the M D.

Dallan Murray: Saying all of that, we still have much more work to do with some of the payers to achieve our goal of securing access and treatment for all eligible patients across the country regardless of plans. The team is working diligently as we speak, educating the payers on the robustness of the newly available embark data. We're confident that this data sets the stage nicely for access to align with our label today, as well as when we gain a broader label.

That I'll turn the call over to Doug to start Q&A Doug.

[noise]. Thank you very much and Michelle let's open the call for questions. Thank you as a reminder to ask a question. Please press star one one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one one again and please limit.

Dallan Murray: The fast starting Q3 was a function of the team's efforts in the quarter itself. And just as importantly, their efforts over the past seven years, building the model, which we have established to support all of the patient's eligible for our therapies. Had this been syrup this first launch, our trajectory in the third quarter would have been very different and much slower. We've gained deep knowledge and expertise through three PMO launches. And I'm glad to say we were able to apply these learnings to the launch of a levidus.

One question.

Please standby, we compiled the Q&A roster.

The first question comes from a new Pan Rahmat with J P. Morgan Your line is open.

Hey, guys. Thanks, so much for taking my question just going back to the embark results we.

We've gotten this question. If you guys are just for multiplicity multiplicity on your teeth functional secondary and points would you still have statistically significant outcomes on these T measures. Thanks, so much.

Dallan Murray: So the summarized a levidus, it was a great first quarter for the launch because our team and our key stakeholders were prepared and they executed flawlessly to support the patients we serve. Driven in large part by the robust 11th revenue in the 3rd quarter, we grew overall net product revenue by roughly 30% over the prior quarter. Net product revenue in Q3 of 2023 was 309.3 million. Importantly, as Doug said, in addition to our success with 11th, we also had our most successful quarter ever serving patients with our established PMO franchise.

Yeah. Thank you for that question not a problem I will turn this call over to Luis.

Thank you.

We actually performs a global statistical task can we get this to to do just that detachment multiplicity and show that the secondary cannot insignificant just by chance that we essentially tested NFA a combined with a secondary.

Sure that they were in fact statistically significant. So this is a quantitative way to test the totality of evidence and respect. So if you think of the forest part that we shut on our call. That's essentially a statistical test to show that together, we see that these are statistically significant.

Dallan Murray: We see continued opportunities in the US and globally for our PMO business in spite of the fact that we also expect cannibalization from 11th over time. Since the 4 to 5 population represents far less than 10% of the PMO business in the US, this cannibalization will not have a material impact on our 2023 net product revenue.

Okay.

Please stand by for the next question.

Dallan Murray: I'd like to take a moment here to thank all of those who are remotely supporting our PMO patients. So as a result of the whole team's effort, the net product revenue from our PMO business in Q3 was 240 million dollars, representing roughly 16% increase over the same quarter in 2022. Looking now at each of our PMO individually, that product revenue for Xandas 51 was 142.3 million in Q3, which was over 16% above the same quarter in 2022.

The next question comes from <unk> with Bank of America Securities. Your line is open.

Hi, guys. Good afternoon. Thanks, so much for taking my question you know for me I think one of the most popular and then nothing.

Getting the last two days is just trying to triangulate timing so to the extent that you can provide us color with what steps are involved in order to complete your filing and is there any kind of precedent for a filing like this all the time it would take the agency to review an application.

Dallan Murray: But on this 53 net product revenue was 31.7 million, 3.4% above Q3 of 2022. And a modest 45 generated net product revenue of 66.3 million in Q3 of 2023. This represents roughly 21% growth over Q3 of 2022.

B. This calendar here that this could all be complete or would it be something more traditional like a six month review for example, thanks.

<unk>. Thank you for your question and then let me preface my question by saying I'm, probably gonna frustrate you by not giving card in vast timelines. Other then we're going to move rapidly and we have a commitment from the division to move rapidly as well our goal is to file a b L. A supplement I believe it's an advocate.

Dallan Murray: As we mentioned in previous calls, we are in the mature phase of the market now for all three of our approved PMOs. As a result, while we expect the US growth to continue to flatten and the XUS revenues while still in the growth phase to remain lumpy from quarter to quarter and thus difficult to project on a quarterly basis.

Supplement and we're going to do that very soon the team is working on it right now I don't want to commit to the exact date, but but very very sure that will be submitted I think traditionally the agency may have six months to review I I do not believe I believe.

Dallan Murray: Taken together, we can reiterate our annual guidance of greater than 925 million in net product revenue for our PMO business in 2023.

The agency is committed to moving as fast as as reasonably possible to review this and there is precedent for this you see this in other areas like oncology.

Dallan Murray: I'll end by saying that I've been continually amazed and impressed by the resilience, commitment and execution of our surreptive teams over the years. And while the success over those years has been impressive, what the teams have achieved in the third quarter of 2023 stands above and beyond anything I've seen in my 10 years working to serve the Duchenne community. The future is bright for surrepta and for the Duchenne community who has been waiting for and very much deserve this progress. Words can't adequately express just how proud I am of our whole team.

All the time, where you can get for something like this very fast turnarounds and of course remember this isn't a BLA BLA supplement so the the inquiry wallet extraordinarily important.

Is focused in that focus is on the fundamental question does the totality of the evidence justifies. The conclusion that 11 is just bringing your better like patience and.

Of course, we believe that that is that it does the standard for this is quite clear it's substantial evidence like near the Joe Jollity of evidence statue statute on this is quite clear.

Dallan Murray: The individual stories from across the country are too numerous to mention here. Nor can we as the team put into words the joy we feel when we celebrate each and every patient who gains access to any of our distant frustration therapy.

I apologize I don't know what that music is I promise I'm not playing a guitar right now the statute says it is very clear can one fairly and responsibly conclude the therapy will have the effect. It reports they have in the regulations are also particularly clear that for life threatening and severely debilitating illness.

Louise Rodino: and so with that, I'll turn the call over to Dr. Louise Rodino Clay-Pak, Louise. Thanks down. Our commitment to the science remains steadfast and our goal to change the lives of patients with rare diseases unwavering. Our opportunity to do good is limitless for those living with dushen, limb girdle, and many other diseases for which therapies are either inadequate or non-existent. As Doug has already detailed the embark result, I'll focus my comments on the progress of our gene therapy and RNA programs.

Is ones like do shed, especially warehouse it satisfactory alternative therapy exist.

The F. D. A has determined that it is appropriate to exercise without broadest flexibility in and applying the statutory standards. It is Louise just pointed it out to everybody not only are the the the evidence on hold very compelling that <unk>.

Louise Rodino: First, limb girdle muscular dystrophy or LGMD. We remain committed to advancing our LGMD portfolio across a variety of subtypes and look forward to providing continuous updates on these important programs in the months ahead. We presented on our LGMD pipeline this past weekend as a Speak Foundation's 2023 International LGMD conference and shared our urgency with the community to bring forth genetic measurements for LGMD. To begin, we made excellent progress for voyaging. Our phase one study evaluating SRP-9003 for the treatment of limb girdle muscular dystrophy type IIE in ambulance adult patients and non-ambulant patients, using clinical process SRP-9003 material.

Is arresting the decline in these patients, but if you do the actual statistics.

And look across the primary and are functional secondaries, you can see statistically.

Adjusting for any risk of a false positive adjusting for most of multiplicity that is powerfully statistically significant <unk> all of which is to say whining back to the original question <unk> that we're gonna submit a b L. A supplement very soon the agency is is committed.

Working with us very rapidly and while I won't give an exact timeline I am confident that we're going to move quickly to review this and you're successful abroad. This label.

Okay. Thanks [noise].

Please.

The next question.

Louise Rodino: We are pleased to report that we completed enrollment and voyaging and we will remain on track to initiate our phase three study using commercially representative process material later this year. Combined with positive expression and functional data shared from our initial study SRP-9003101, we believe the data from voyaging will give us insights into a broader patient population. We are also excited to report that we completed dosing in our systemic pilot study, Navigene, for our SRP-6004 dual vector RH-74 media gene therapy to treat individuals with LGMD2B.

[noise]. The next question comes from Genoa, Wang What's Barclays. Your line is open.

Thank you for taking my questions. Maybe just follow this comment a duck. We you know went PLE efficacy supplements was accepted and then in the data package can you lay out what kind of data will be included in addition to what you share with us would that also be.

E like see the protein correlation of the protein level fishes function will be state I also will be included in your package.

Louise Rodino: LGMD2B is characterized by the absence of the protein distrolin. The innovative dual vector strategy allows us to deliver the full length distrolin gene, the sole cause of LGMD2B. We look forward to reporting results from the study in the first half of 2024. As mentioned last quarter, our LGMD natural history study of the LGMD2E2C and 2D called journey has been fully enrolled and we follow patients for 36 months. We continue my progress in scalable manufacturing for all of our LGMD candidates in our pipeline and look forward to initiating clinical studies as rapidly as possible.

There will be certainly more than the package and then the top like we're still evaluating other information, including for instance protein and another drink she K and the like but but obviously the focus of the the review is going to be.

Yeah, first and foremost the efficacy and safety for this therapy and then of course, the all gonna be evaluated in relation to our request abroad and this label by removing age limitations and an ambulance simulation limitations on that you know we I don't think we've made any final decisions about.

You know what we're announcing during this process, but obviously generally speaking we tend to be pretty transparent with folks.

Louise Rodino: Turning now to the progress we've made with our RNA platform, we replaced a complete enrollment in the first quarter of 2023 for a momentum study for SRP-5051 and we're targeting readout of the study in 2023. Regarding our post-marketing studies for the PMO's as mentioned last quarter, we completed enrollment in the essence trial for post-marketing requirements for Golderson and Kazemarza. As a reminder, essence is a two-year study and is due to read out in early 2026.

Thank you.

Police Samfie during the next question.

The next question comes from calling Bristow, which UBS your line is open.

And you get off the name and and congrats on the the impressive and evidence. So maybe another one on the <unk> data can you speak to the into patient vary a bit of T. V. So in the 11th is treated patients.

Instead of a question we've been getting is the positive results driven by a small group of high respond as it may be for you to say a comment on how this variability compared to the face to experience that would be helpful. Thanks.

Sure I'm Gonna Uhm flip this over to Luis to answer specifically, but I would generally know the positive results of the P value on these positive results or they're not close you know on the time to rise.

0.002 on the Pet me to walk around with a 0.004 on the global Statistical analysis, when one looks at the primary and all the secondaries together.

0.004, so it's very very powerful, but Louise perhaps you wanted an answer more specifically on some of the.

Variability issues.

Yeah, Yeah, and generally we did not see uhm variability high variability amongst the patience of Saturday vacation with either.

At or below what we anticipated from our previous that is that we used to to power and embark on there and did not see any high variability.

Ikea is helpful.

Mhm.

Please stand by for the next question.

The next question comes from re for sex with Guggenheim. Your line is open.

Hi, This is ray.

Data provided to the F D a.

The information.

I am very sorry, but I I was unable to hear.

That that answer that question can you can you ask him again, perhaps.

Yes. This is ray on forget.

The top line data.

To the F D. A on Embarq include information on my Christian.

Cause I'm, so sorry, I heard the beginning but I don't hear the question itself.

I think yeah.

Thank you activate included data on micro dystrophin expression.

Well it with the I imagine that will have that data available during the review process with the <unk>, There's as Louisa said the expression, we're seeing as in as in the hundred range of what we normally see them. So it's nothing nothing unusual about it in fact the P value on it is.

Point zero as many zeroes.

It's very strongly.

Robustly mm mm.

As as you would expect a limited to robustly next district.

That's the question.

That that help thank you.

Thank you so much I apologize I couldn't hear you I'm sorry about that.

Please stand by for the next question.

The next question comes from Brian's Corny with your line is open.

Yeah. Good afternoon. Thanks for taking my question I guess it wasn't people you looked at very closely before but on Sunday when I <unk> I think <unk> at a nonsignificant differences in favor and treatment, but not that different in terms of magnitude I think maybe just under half a second differences.

You tell me, if that's right or not so I know you've talked extensively about based on an imbalance issue here and it seems particularly acute in the case of baseline time for us with the active on was 5.1 at baseline and placebo was 3.6 I was just wondering if you've gone back and looked at making adjustments rebaselining balances to evaluate upon to rise differences and studied 102 and <unk>.

Pickler, if the 45 year old subgroup. It looks the same in 102 isn't that anymore.

Yeah. Thank you for that question Bryan I'll tell Mister Luis to respond.

Mmm.

Uhm I'm gonna have to be Catholic because what we did was take the inclusion criteria that we've generated for embark and applied it to our previous data when we compared it to the external control and what we found there is a a difference when you exclude those patients that would've been excluded by that criterion 102.

Louise Rodino: On a gap basis, we recorded $194.3 million dollars and $216.7 million dollars in R&D expenses for the third quarter of 2023 and 2022 respectively, a year over year decrease of $22.4 million dollars. The decreases primarily due to decrease in our manufacturing expenses, partially offset by increases in clinical trial expenses. On a non-gap basis, R&D expenses were $163.9 million dollars for the third quarter of 2023, compared to $193.7 million dollars for the same period of 2022, a decrease of $29.8 million dollars.

You had those rapid decliners so in that case, we saw.

A significant difference but.

Specific numbers are escaping that right now, but we can't do that analysis, where we kind of apply the same exclusion criteria in good standing.

Great. Thank you yeah.

Just one thing to add in that analysis to leave this point, we saw very <unk>.

Louise Rodino: Now turning to SGNA on a gap basis, we recorded approximately $120.9 million dollars and $144.8 million dollars of expenses for the third quarter of 2023 and 2022 respectively, an increase of $16.1 million dollars. The increase was driven primarily by an increase in professional services and compensation and other personnel expenses, partially offset by a decrease in stock-based compensation. On a non-gap basis, the SGNA expenses were $92.8 million dollars for the third quarter of 2023, compared to $66.8 million dollars for the same period of 2022 and increased of $26 million dollars.

Really good consistency between what we observed in 102, and what we have observed and 301.

Please stand by for the next question.

[noise]. The next question comes from South been Rector with Goldman Sachs. Your line is open.

Good afternoon. Thanks for taking my question with regard to the regulatory submission are there formal or regulatory procedures involved with revisiting an accelerated approval after.

Louise Rodino: On a gap basis, we recorded $12.3 million dollars in other expense, and that for the third quarter of 2023, compared to $400,000 dollars in other income, and that for the same period of 2022. The change is primarily due to the impairment of our investment and lawful and contingent consideration. That partially offset by increase is an increase of an investment discount and an increase in income due to the investment mix of our investment portfolio as well as reductions of interest expense incurred as a result of the repayment of our December 2019 term loan during 2022.

The primary endpoint fails in a confirmatory trial here and then just any preliminary feedback on your data from Paris, and how that might impact the existing label or uhm from physicians with regard to how they think about Houston patience here.

Yeah.

Again on the first question I'm not 100 per cent.

And I understand the question, let me be very clear.

The standard for confirmation of an accelerated approval is looking at the totality of evidence in determining whether the benefits of that therapy have been confirmed by the entire data set not just to confirm it confirmatory data, but all of the surrounding evidence that would exist including other studies.

Louise Rodino: We had approximately $1.8 billion in cash cash equivalence investments in long-term restricted cash as is the 10 or 30 of 2023. Obviously, please, with the amount of capital on our balance sheet, the interventment markets, we know cash becomes even more valuable. We continually evaluate our expenses. That said, based on the embark results and the information we have today, there's no better use of a cash into building inventory to serve those with D&D.

And I would strongly argue that not only embarked but all of the supporting them.

Evidence as well as strongly confirm the benefits of this therapy. So I I think we're in very good shape. There the focus of our review with this Uhm division is going to be on the breath of the expansion of this label that I am quite confident is going to be the review folk.

Doug Ingram: With that, I'll turn the call over to Doug to start Q&A. Doug. Thank you very much, Ian.

Unknown Executive: Michelle, let's open the call for questions. Thank you. As a reminder to ask a question, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. And please limit to one question. Please stand by while we compile the Q&A roster.

And as it relates to that as you know are strong.

A strong view is that having confirm these results and confirm them across.

<unk> I'm looking at the totality of this evidence and looking at the Forest Lord as an example of looking at the statistical analysis of the forest, what uhm adjusted for multiplicity easy. It is quite clear that this therapy is arresting the decline of these patients and deserves to be.

Anupam Rama: The first question comes from a new panorama with JP Morgan, your line is open. Hey, guys. Thanks so much for taking the question. Just going back to the embark results. We've gotten this question. If you guys are just for multiplicity on your key functional secondary importance, would you still have statistically significant outcomes on these key measures? Thanks so much. Thank you for that question, Anupam. I will turn this call over to Louise.

Made available to go to.

Patience, uhm without limitation to age or or or artificial restrictions around ambulation.

As it relates to payers. This is additional evidence in our armamentarium repairs things have gone very well <unk> and his team medical affairs are commercial of our field force access to reimbursement them alike have just done a fabulous job supporting the law.

Anupam Rama: Thank you. We actually performed a global statistical test, and we did this to do just that, the test from local society, and show that the secondary is do not hit significant just by chance, so we essentially tested NSAA combined with the secondarys and show that they were in fact statistically significant. So this is a quantitative way to cast the totality of evidence in respect, so if you think of the forest plot that we showed on our call, that's essentially a statistical test to show that together, we see for these are statistically significant.

Lunch of <unk> and I hope everyone will agree with me that it shows in our performance this quarter and Uhm. This bolsters the discussion that the team can have now it gives me a good example, we now have a really powerful metric that is compelling on the speed with which ones.

Unknown Executive: Please stand by for the next question.

Put a kid on on therapy, so as well I will remind you.

The time to rise not only is the P value.

I think 0.002, if I am not mistaken, but and categorize as the single greatest prognosticater of loss of ambulation and arise time about five seconds as we can.

Talked about often in the literature robustly is the single greatest predictor of early loss of Ambulation and embark has shown that using <unk> reduces the odds of that occurring in a 50 G period by over 90%.

Tazeen Ahmad: The next question comes from Tazine Amaz with Think of America because security here line is open. Hi guys, good afternoon. Thanks so much for taking my question. You know, for me, I think one of the most popular events I've been getting the last two days is just trying to triangulate timing. So Doug, to the extent that you can provide us color with what steps are involved next in order to complete your filing.

This provides an additional compelling point with payers, who frankly, so far I've done a really good job in providing access to this provides additional evidence that it really is important to get kids on this therapy as soon as possible and I would.

Tazeen Ahmad: And is there any kind of precedent for a filing like this on the time it would take the agency to review an application. Could it be this calendar year that this could all be complete, or would it be something more traditional like a six month review. For example, thanks.

Are you looking forward to label expansion and there's a compelling argument for why this therapy should be this label should be expanded as soon as possible as well so everyone has access to it.

Doug Ingram: Tazine, thank you for your question and I'm going to prep this my question by saying, probably going to frustrate you by not giving hard and vast timelines other than we're going to move rapidly and we have a commitment from the division to move rapidly as well. Our goal is to file a BLA supplement. I believe it's an efficacy supplement and we're going to do that. Very soon the team is working on it right now.

Please stand by for the next question.

The next question comes from my ear with the <unk>. Your line is open.

Hey, guys. Thanks for taking a question yeah. Congrats on the the great quarter for <unk>. So I guess my first question is <unk>.

Doug Ingram: I don't want to commit to the exact date, but but very very soon that will be submitted. I think traditionally the agency may have six months to review. I do not believe. I believe the agency is committed to moving as fast as is reasonably possible to review this and there is precedent for this. You see this in other areas like oncology all the time where you can get for something like this very fast turnaround and of course remember.

You know the patients who would does what they was primarily those who were pretty much anticipated the approval the accelerated approval M. They I guess I'm you know more like the the bullets and like how many would you be able to show how many patients are waiting to be dose and they come in.

<unk>.

And just continue on this theme.

Now that the embolic data is read out do you have any sense of any ship in patients receptivity to the product at all I know it's early days. Thanks.

Doug Ingram: This isn't to be led but a BLA supplement. So the inquiry while extraordinarily important is focused and that focused is on the fundamental question does the totality of the evidence justify the conclusion that a love it is just bringing a better life to these patients and of course we believe that. That is that it does the standards for this is quite clear it's substantial evidence looking at the totality of evidence the statute statute on this is quite clear you know.

Mmm.

Yeah, I was looking to answer those questions quickly first I don't think it will be a shift in the desire for this therapy I think.

Except for patients that are not in the parents are patients that are not in that four to five year old range I think are probably even more compelled.

And I don't want this therapy, and they're gonna wait impatiently as they should be impatient to have this label abroad.

We don't share patient numbers were going to use as I'm metric for success in the measure of our success Medtronic revenue as we as we said on the issue of sort of bolus or warehousing of patients. There were certainly some number of patients that physicians work getting together and gathering to ensure that they could.

Doug Ingram: I apologize I don't know what that means because I promise I'm not playing a guitar right now. The statute says you know is very clear can one fairly and responsibly conclude that the therapy will have the effective reports to have and the regulations are also particularly clear that for life threatening and severely debilitating illnesses one why can't be shed especially where no satisfactory alternative therapy exist. The FDA is determined that it is appropriate to exercise the broadest flexibility in applying the statutory standards and as the least just pointed out to everybody not only are the the evidence on whole.

Preferably leg preferentially get dose before they aged out because the label obviously as you know uhm restricts the dosing to fix and below six five but we've had a steady stream of a new start for hours since the approval and so you know.

Looking forward, we have a steady.

Stream of of start four of us that are working through the process.

Doug Ingram: Very compelling that a Levittus is arresting the decline in these patients but if you do the actual statistics and look across the primary and our functional secondaries you can see statistically. Adjusting for any risk of a false positive adjusting for most multiplicities that is powerfully. Statistically significant so all of which is to say winding back to the original question to seen. We're going to submit a BLA supplement very soon the agency is committed to working with us very rapidly and while I won't give an exact timeline I am confident that we're going to move quickly to review this and this successful broaden this label. Okay, thanks, Doug. Please hand by for the next question.

And one other thing people have been asked what I'll say say it Dennis the team <unk> done a brilliant job on site activation you know I think our goal is you know or go ahead aspiration really been to have 50 sites ready to to infuse at long.

<unk> and then very aspiration Lee we thought perhaps by the end of the year. Some time next year, we could get all the way to 70 size.

But we're at 70 today. So the team has just done a brilliant job.

Making getting sites ready and up and running and there is a steady stream of start forms to work for.

Thank you.

Please stand by for the next question.

The next question comes from Danielle Brill with Raymond James Your line is open.

Gena Wang: The next question comes from Gena Wang with Barclays. Your line is open. Thank you for taking my questions. Maybe just follow this comment. Doug, will you announce when BLA efficacy supplement was accepted? And in the data package, can you lay out what kind of data will be included in addition to what you share with us? Would that also be the protein correlation of the protein level versus function? Will these data also be included in the package?

Yeah. Good afternoon. Thanks, so much for that question done. So we spend a lot of time discussing embark apathy data I'd like to switch over to safety I recall myocarditis events being discussed at the AD com, including one event that had a curtain embark at the time just curious if there are any additional safety events of this.

Nature that occurred in the study or any other at a leading to hospitalization. Thank you.

Thank you for your question Danielle I will turn the call over to Luis to respond.

Gena Wang: There will be certainly more in the package than the top line we're still evaluating other information, including for instance, protein and other things, CK and the like. But obviously, the focus of the review is going to be, you know, first and foremost, the efficacy and safety for this therapy. And then of course, it's all going to be evaluated in relation to our request to broaden this label by removing AIDS limitations and ambulance and relation limitations on that.

Yeah, we did not see any differences in that types of <unk> and the frequency of that is F. A is that with.

One of the most reassuring things about embark was the continued safety profile and now taken together that's all of the previous trials and we have a large safety data base, that's consistent among those trial.

Please stand by for the next question.

[noise]. The next question comes from Reto Boral with T V. Cowan Your line is open.

Gena Wang: You know, we I don't think we've made any final decisions about, you know, what we're announcing during this process, but obviously generally speaking, we tend to be pretty transparent with folks. Thank you. Please stand by for the next question.

Good afternoon, everyone. Thanks for taking my question.

Could you maybe gallon walk us through.

See patient numbers for the different scenarios that our regulatory experts.

<unk> <unk> are in play essentially for local expansion.

Colin Bristow: The next question comes from Colin Bristow with UBS. Your line is open. Thank you.

And 10% of your piano shelter.

That is a covered but how would you segmented five maybe six to seven year olds S. F. D. A wants to go down that route again, and how that number will change. It can do got purple ambulatory population, which her my call <unk> July 12th.

Brian Scorney: Good afternoon and congrats on the impressive and evidence sales. Maybe another one on the embarked data. Can you speak to the the interpatient variability you saw in elevated treated patients? You know, a sort of a question we've been getting is, were the positive results driven by a small group of high responders? And maybe if you don't think of it on how this variability compared to the phase two experience, that would be helpful.

Here's an H.

Mmm.

For ambulatory.

Yeah like that that's that's.

That's the average age of lots of Annulation now 12 13.

That may be correct <unk> confirm that in a second broadly speaking I mean look first of all I want to be very clear, we're not looking for a label expansion.

Brian Scorney: Thanks. Sure. I'm going to split this over to Louise to answer specifically, but I would generally note that the positive results, the p-value on these positive results are they're not close. You know, on the time to rise, it's 0.002 on the 10 meter walk run at 0.004. On the global statistical analysis, when one looks at the primary and all the secondaries together, it's 0.004. So it's very, very powerful. But we, you know, perhaps you want to answer more specifically on some of the the variability issues.

To go from four to five to four to seven and we don't think.

There's any reason scientifically one would be limited to afford a sevens given the data and he'd never seen that before once you know you've never seen in any other label for a duchenne therapy. These sorts of age related restrictions, but the answering the broadest question.

The you know the at all ambulatory versus all ambulatory and non ambulatory is about 50 per cent. So theirs. So the ambulatory populations about half of all patients in the non ambulatory as the other half of course.

Brian Scorney: Yeah. Generally, we did not see variability. High variability amongst patients with standard deviation was other at or below what we anticipated from our previous studies that we used to power and embark. So there, we did not see any high variability. Thank you. Please stand by for the next question.

And so that <unk>.

<unk> <unk> the balance is there anything else you'd like to stay on the subject.

No I think you've covered it dug our goal is to you know target all the patients that we can get in the entire population.

<unk> question about average age of loss of Ambulation, I think you're in the right range, but the Ky also been talking a lot about you know the definition of loss of installation too because there's you know there's heterogeneity. So you'll you'll have some patience walking at much older Ages, and we have a whole cohort of patients who.

Unknown Executive: The next question comes from me for set with Googanheim, your line is open. This is right on the subject. Is the top line data provided to the FDA from embarking to the information on micro-discision?

Been treated with the Pmo's for years now that are gonna, we believe <unk> older ages of loss of installation as well. So it won't be we believe you know the ambulatory population won't be defined by age, but as Doug said, our aspiration was abroad label.

Unknown Executive: I'm very sorry, but I was unable to hear that answer that question. Can you ask again, perhaps? Yes, this is right on the subject. Can the top line data provided to the FDA on embarking to include information on micro-discision? Good.

Targeting all of the patients who who will be eligible.

And does that 50 per cent in your piano sounds too.

Mmm.

On.

Yeah.

Ambulatory versus non ambulatory split.

I think it's in in that range of of 50 per cent I think the access is more difficult than the older patients. So so an outrage we've got a higher penetration to younger population.

Unknown Executive: I'm so sorry, Ryan. I heard the beginning, but I don't hear the question itself. I think he asked if it included data on micro-discision expression. Well, I imagine that we'll have that data available during the review process with the FDA. As Luis has said, the expression we're seeing is in the hunted range of what we've normally seen, so it's nothing unusual about it. In fact, the p-value on it is 0.0. It's very strongly robustly. It would be expected to limit it to robustly next year.

Great. Thank you.

Please stand by for the next question.

The next question comes from my calls with Morgan Stanley. Your line is open.

Good afternoon, and thanks for taking the question.

You mentioned now that you have about 70 sites are active can you give us a sense of what percentage of those sites of actually prescribed a limited so far thanks.

Unknown Executive: That's the question, folks. That helps. Thank you. Thank you so much.

Now, we're going to apologize I am Gonna Frustrate you were gonna resist that level.

Unknown Executive: I'm apologies. I couldn't hear you.

Level of detail either on numbers of sites are accused or you know I'm, probably more specifically number of patients infused at any one time as we have done for the last seven years and I think we've done generally overtime to good success with folks we use.

Unknown Executive: I'm sorry about that.

Unknown Executive: Please stand by for the next question.

Brian Scorney: The next question comes from Brian Scourney, which they are. Your line is open. They get afternoon. Thanks for taking the question. I guess it wasn't something we looked at very closely before, but I don't want to tell you when I tell you, I think, kind of rise. I had a non-significant difference in favor of treatment, but not that different in terms of magnitude. I think maybe just under half a second differences.

Ned product revenue is the marker for success in uptake in my life.

So apologies for that my.

Police standby for the next question.

Brian Scorney: You can tell me that's right or not. So I know you've talked extensively about the baseline imbalance issue here. And it seems particularly acute in the case of baseline time for rise. But the act of arm was 5.1 at baseline, and the table was 3.6. So it's just wondering if you've gone back and looked at making adjustments for baseline imbalances to evaluate the pond to rise differences in study 102. And in particular, if the four to five year old subgroup looks the same in 102 as a dead end of bark.

The next question comes from Kim Lugo with William Blair. Your line is open.

Thanks for the question.

You mentioned earlier in the week that you didn't expect an additional advisory committee for broadening the label.

But you know we can all remember where we didn't expect one for the accelerated approval discussion [laughter] I guess why not requesting <unk> seemed like listening to the <unk>. The participants were much more amenable to a non age restricted approval when the agency was.

Louise Rodino: Thank you for that question, Brian. I'll turn this to Louise to respond. I'm going to speak generally because what we did was take the inclusion criteria that we've generated for embarking and applied it for our previous data when we compared it to the external control. And what we found there is a difference when you exclude those patients that would have been excluded by that criteria and 102 where you had those rapid declineers.

Yeah.

Okay. So that's a good question and I.

I'm not surprised by the comment I. There are those that might say that I have I have a poor historical track record of predicting adcoms.

Notwithstanding the fact that the last time I saw that we were going to have it out and kind of I didn't have that in writing.

The reason that we don't believe we're we're going to have an outcome is that we don't believe that we will need one and I think that.

Louise Rodino: So in that case, we saw a more significant difference. But the specific numbers are escaping that right now, but we did do that enough. This is where we kind of applied the same exclusion criteria and did see it there.

Believe as we sit here today that is a view that would be shared by us and F. D. A leadership, we've said before we had a very productive and encouraging.

Doug Ingram: Thank you. Yeah, that may be just one thing to add in that analysis, to Louise's point, we saw a very, we saw a really good consistency between what we observed in 102 and what we observed in 301.

Discussion with the F D. A leadership on the data in on the possibility of submitting it for [noise] abroad label and I would also note that you know the the agency has.

Salveen Richter: Please stand by for the next question. The next question comes from Salveen Richter with Goldman Sachs, your line is open. Good afternoon, thanks for taking my question.

Change the division in particular has gone through some pretty significant changes over the course of this year.

Reorganization, just to remind you where old cat.

Has been replaced by the Super Office O T T and did not too long ago, a new leader Doctor Nicole Verdun.

Doug Ingram: With regard to the regulatory submission, are there formal or regulatory procedures involved with revisiting and accelerated approval after the primary endpoint fails and in a conformatory trial here? And then just any preliminary feedback on your data from payers and how that might impact the existing label or from physicians with regard to how they think about use in patients here. Again, I'm on the first question, I'm not 100% confident I understand the question.

Took the helm asthma head of O T. T. So I would say you know just to.

Remember that the the division is is evolving obviously in the event that there wasn't advisory committee, we would be well prepared for it and I believe we would perform a strike exceptionally well there I think activating a quaint back in team just did a fabulous job representing awesome.

As one may recall, we did ultimately win that outcome.

Doug Ingram: Let me be very clear. The standard for confirmation of an accelerator approval is looking at the totality of evidence and determining whether the benefits of that therapy have been confirmed by the entire data set, not just the conformatory data, but all of this surrounding evidence that would exist including other studies. And I would strongly argue that not only embarked, but all of the supporting evidence as well as strongly confirmed the benefits of this therapy.

We sit here today, we feel pretty confident that we can get a label expansion without an advisory committee.

Alright, thank you.

Thank you very much please.

Please stand by for the next question.

[noise]. The next question comes from Gil Bluhm with Needham and company. Your line is open.

Good afternoon, and congratulations on all the progress.

I'm going back to a question.

Doug Ingram: So I think we're in very good shape there. The focus of our review with this division is going to be on the breadth of the expansion of this label. That I am quite confident is going to be the review focus and as relates to that as you know our strong view is that having confirmed these results and confirm them across patients and looking at the totality of these evidence and looking at the forest plot as an example of looking at the physical analysis of the forest plot.

No up on columns and any other questions about any questions to go.

So 50, Orange 30 102.

Any chance that there would be some follow up especially on the patients that were older and were crossed over and park to study 102 regarding time to rise it just be interesting to see how that.

Data it looks in comparison to the embarked data.

Sure Luis.

Thoughts on that.

Yeah, although 102 patients that continued to fall for up to five years. So that's certainly something that we can look at over time.

Doug Ingram: Adjusted for multiplicities it is quite clear that the therapy is arresting the decline and the patients and deserves to be made available to the patients when out limitation to age or artificial restrictions around the regulation. As relates to payers, this is additional evidence in our armamentarian with payers things have gone very well. Dalton and his team medical affairs are commercial field floors access reimbursement of the like have just done a fabulous job supporting the launch of the Lebanese and I hope everyone will agree with me that it shows in our performance.

We don't have that data in house that we can kind of look at that.

Please stand by for the next question.

The next question comes from Brian Abrahams with our B C capital Your line is open.

Hi, there congrats on the strong first full quarter of the <unk>. Thanks for taking my question can.

Can remind us of the protocols in place and embark to protect against functional on blinding was this the topic that ever came up with the F. D. A in your recent discussions about you expect any differences in the effects functional and blinding. If there wasn't any might have on time tests versus an N S. I a thanks.

Doug Ingram: This quarter and this bolsters the discussions that the team can have now we can in an example we now have a really powerful metric that it's compelling on the speed with which once you put a kid on on therapy so as well I will remind you on the time to rise not only is the key value. I think 0.002 if I am not mistaken but in tenderizing the single greatest prognosticator of loss of amulation and a rise time above five seconds as we've talked about often in the literature.

Yeah, I can allow louie used to discuss the protocol aspects of the blinding process, which was exceptionally rigorous.

<unk>, we can we can generally assume that is you know.

That would be a very objective time test would be less subject to you know like any kind of influence in the event there wasn't a blinding, but I would say all Saturday and I think the the protocol was very good about the blinding process in the study itself wants you to remember was actually very well run I wanted to be clear about that.

Doug Ingram: Robossely is the single greatest predictor of early loss of amulation and in Marcus shown that using a levittist reduces the odds of that occurring in a 52 degree period by over 90% so this provides an additional compelling. Point with payers who you know frankly so far have done a really good job of providing access this provide additional evidence that it really is important to get kids on this therapy as soon as possible.

Luis any thoughts on.

Blending process.

Yeah, I'm, just specifically survey patients and caregivers, obviously bonded D P I as as well as the.

Uhm.

The <unk>.

Physical therapist, showing that functional tests are all completely blinded so the 30th maintain winded.

The threat is blinded to maintain by a third party. So there is a rigorous process in place to make sure that they're blind remains intact.

Doug Ingram: And I would argue looking forward to labor expansion and to take compelling argument for why this therapy should be this label should be expanded as soon as possible as well so everyone has that.

That's helpful. Thank you.

Please stand by for the next question.

Uy Ear: Please stand by for the next question. The next question comes from Y Ear with Mizzouro, your line is open. Hey guys, thanks for taking a question. You can grab on the great quarter for 11s. So I guess my first question is, were those, you know, the patients who were those, were they primarily those who were pretty much anticipated the approval, the accelerator approval, and they, I guess I'm, you know, more like the bolus.

The next question comes from Kristin Classico with Cantor Fitzgerald. Your line is open.

Hi, Good afternoon. This is Jason Bouvier on for Christian Costco. Thank you for taking my question and congrats on a strong quarter for 11 is.

One question from us the cadence.

Treating patients is going faster than the original time lines you laid out. So we're just wondering what the biggest drivers are there.

And how might this also impact the potentially broader launch next year. Thank you.

Mmm.

Well I'm I'm going to take the question even know dialing once you because I want to brag about our gene I mean, I think there too.

Uy Ear: And like how many, would you be able to share how many patients are waiting to be dose in the coming quarter, and just continue in this theme. Now that the embark data is read out, do you have any sense of any shift in patients receptivity to the product at all I know it's early days. Thanks.

Significant reasons why the cadence of this launch is going.

<unk> well and why this launches from my perspective, and unprecedented success in gene therapy. The first of which of course is the therapy itself.

11 is is extraordinary Lee needed therapy that patients who have been on it and families to share their experience with it <unk>.

Doug Ingram: Yeah, let me answer those questions quickly. First, I don't be able to be a shift in the desire for this therapy, I think, except for patients that are not in the parents of patients that are not in the four to five-year-old range, I think are probably even more compelled to, you know, to want this therapy and are going to wait impatiently as they should be impatient to have this label broadened. We don't share patient numbers we're going to use as our metrics for success and the measure of our success on that product revenue, as we, as we've said, on the issue of sort of bolus or warehousing and patients, there were certainly some number of patients that physicians were getting together and gathering to ensure that they could prefer, preferentially get dose before they aged out, because the label, obviously, as you know, restricts the dosing to six and below six and five.

Strongly of the belief that they need this therapy and these kids have been stabilising are doing things.

Age specific that untreated kids haven't been able to do and then if you don't mind me bragging a little bit about the team I mean this is an example of exceptionally great execution.

By the <unk> by down I'm Gonna take a commercial customer officer by going beyond that this is a manufacturing and distribution folks as well you know just.

Large team effort to execute on this and this is a new for us I want to remind everyone. Now that we have now for therapies that we have launched every one of those therapies and their launches have gone exceptionally well I mean, if you look at the piano assistant.

Digress for a moment and then we are now from our first PMO that was approved in late 2016, we're still growing we grew it's 16% quarter over the same quarter last year, even as we're launching 11th.

Doug Ingram: But we've had a steady stream of new start forms since the approval. And so, you know, looking forward, we have a steady stream of start forms that are working through the process. And, you know, one other thing, people have been asked what I'll say, say it, you know, the team, Val, I've never just done a brilliant job on site activation. You know, I think our goal, as you know, our goal had aspirationally been to have 50 sites ready to infuse at launch.

I'll be there so I think there's a combination of both a great therapy as all of our four therapies I believe have been and exceptional focused granular Ah well informed execution now what does this mean for the future. It means that we're <unk>, we know how to serve the dish.

<unk> community.

And and and you know.

One of the thing that excites us about a broader label is will be able to bring a <unk> to the majority of children and men and young men in the United States that are living with and the generating irreparably from this ferocious disease and so I'm really excited.

Doug Ingram: And then very, very aspirationally, we thought perhaps by the end of the year, sometime next year, we could get all the way to 70 sites, but we're at 70 today. So the team has just done a brilliant job making getting sites ready and up and running and there is a steady stream of start forms to work for. Thank you.

The opportunity to bring this there would be more patience, even as we're doing really well with a watch right now. Thank you very much for your question.

Danielle Brill: Please stand by for the next question. The next question comes from Danielle Brill with Raymond James, your line is open. Hi guys, good afternoon, thanks so much for the question. Doug, so we spent a lot of time discussing Embark at the C-Data.

Please stand by for the next question.

Louise Rodino: I'd like to switch over to safety. I recall myocardis events being discussed at the adcom, including one event that had occurred in Embark at the time. Just curious if there were any additional safety events of this nature that occurred in the study, or any other essay leading to hospitalization. Thank you. Thank you for your question. Danielle, I will turn the call over to Louise to respond. Yeah, we did not see any differences in the types of essays and the frequency of those essays.

[noise]. The last question comes from Joseph Schwartz Leerink Partners. Your line is open.

Great. Thanks, very much for putting me in.

I was wondering how are you thinking about upcoming clinical data for a different gene therapy candidate, which will have an Arab look soon is there anything that you'll be focused on in particular and how do you see the tradeoff between safety and efficacy if if we're able to produce a greater impact on innocent.

That impact your royalty value proposition for all evidence.

Oh.

Thank you for the question.

As I've said many times before we have in front of us and exceptionally ferocious competitor and <unk> muscular dystrophy, and all of our focus in all of our energy and then beating this damn disease and I think this team is doing a brilliant job of that.

Louise Rodino: That was one of the most reassuring things about Embark was the continued safety profile and now taking together all of the previous trials. We have a large safety data space that's consistent among those trials. Please stand by for the next question.

We are exceptionally pleased with the performance of a <unk> you know there were some you know many years ago people made decisions about <unk> and the like and with the benefit of many years of experience we are exceptionally proud of and.

Ritu Baral: The next question comes from Ritu Borrell with T.D. Cohen. Your line is open. Good afternoon, everyone. Thanks for taking the question.

Ritu Baral: Doug, could you maybe dial and walk us through how you see patient numbers for the different scenarios that are regulatory experts. Suggest could are in play essentially for labor expansion. You mentioned 10% of your PMO shells are for 4.5. There's a cover. But how would you segment it by maybe six to seven year olds if FDA wants to go down that route again. And how that number would change if you do got the full ambulatory population, which her mic calls now extends to like 12 years of age. For ambulatory. Yeah, like that's that's the average age of a lot of amulation now 1213. That may be correct. I'm down confirm that in a second.

Frankly, nothing less than thrilled with the particular Thompson and construct that we have it's shown not only then it is able to intervene protect these muscles of these these children and arrest. The decline then it can do that with a particularly laudable safety profile.

Given the amount of therapy required here and the fact that it's full body infusions are 874 has been a stand out and we are quite confident that anyone who is rational who had an opportunity to make a decision today about what captured they would use.

And what can't strike they would develop.

I'm sure they would do their best to try to copy yeah. So we're we're not focused on any competitor besides duchenne muscular dystrophy and we are for.

Doug Ingram: Broadly speaking, I mean, look, first of all, one of the very clear, we're not looking for a label expansion to go from 4 to 5 to 4 to 7. And we don't think there's any reason scientifically one would be limited to 4 to 7. And we've never seen that before. Once the, you know, you've never seen in any other label for Duchenne therapy, these sorts of age related restrictions. But, you know, answering the broadest question.

Ferociously committed to beating this disease. So that's our that's our our focus right now, but thank you very much for your questions.

I would now like to turn the call back over to jobs before closing remarks.

Well, thank you very much Michelle and thanks, everyone for attending the night and thank you for your very thoughtful questions.

Let me summarize this quarter has been an extraordinarily important one with our fourth approve approval. We launched 11th this in my opinion, we launched it brilliantly we've continued to serve the community with our P. M O's, which continue robust growth even in the face of an 11 his lunch and on a non-GAAP basis, we are now.

Doug Ingram: And the, you know, the all ambulatory versus all ambulatory and non ambulatory is about 50%. So there's so the ambulatory population is about half of all patients and then non ambulatory is the other half, of course. And so that's our mind is the big cut, but down is anything else you'd like to stay on the subject. No, I think you covered it. Our goal is to, you know, target all the patients that we can get in the entire population.

Profitable and we are marching toward a cash positive cash flow positive.

In the very near future.

We have built a strong enduring organization that is focused on two major things. The first is serving our patient community through brilliant science and the second is executing and getting things done and that is precisely what we will be doing over the coming months, we intend to continue our strong performance and <unk>.

Dallan Murray: Retute to your question about average age of loss of amulation. I think you're in the right range, but the K wells have been talking a lot about, you know, the definition of loss of amulation to because as you know, there's heterogeneity. So you'll, you have some patients walking at much older ages and we have a whole cohort of patients who've been treated with the PMOs for years now that are going to we believe have older ages of loss of amulation as well.

<unk> disturbing this community, we intend to move with speed to submit our efficacy supplement and conclude the review on the broad on the broadening of the 11th this label and when our label has been updated to remove agent ambulation restrictions, we intend to bring this therapy to the majority of patients living with Shannon the United States.

Dallan Murray: So it won't be, we believe, you know, the ambulatory population won't be defined by age, but as Doug said, our aspiration is a broad label and targeting all the patients who will be eligible. And is that 50% of your PMO sales, too? Yeah, the ambulatory versus non-ambulatory split. I think it's in that range of 50%, I think the access is more difficult in the older patients, so on average, we've got a higher penetration in the younger, in the younger population.

And with that I look forward to updating all of you on our progress along the way and have a lovely evening. Thank you.

This concludes today's conference call. Thank you for participating you may now disconnect.

Unknown Executive: Great, thank you. Please stand by for the next question.

Mmm.

[music].

Mike Ulz: The next question comes from Mike Ulz with Morgan Stanley, your line is open. Good afternoon and thanks for taking the question. You mentioned now that you have about 70 sites are active. Can you give us a sense of what percentage of those sites have actually prescribed 11 is so far? Thanks.

Doug Ingram: Now, we're going to apologies, and I'm going to frustrate you. We're going to resist that level of detail, either on numbers of sites are infused or probably more specifically, number of patients infused at any one time. As we have done for the last seven years, and I think we've done generally over time to good success with folks. We use net product revenue as the marker for success and uptake and the like. So apologies for that.

Unknown Executive: Please stand by for the next question.

Tim Lugo: The next question comes from Tim Lugo with William Blair, your line is open. Thanks for the question.

Doug Ingram: You mentioned earlier in the week, but you didn't expect an additional advisor committee for broadening the label, but, you know, we can all remember when we didn't expect one for the accelerator approval discussion. I guess why not request an outcome. It seemed like listening to the outcome. The participants were much more amenable to a non-adjusted approval than the agency was.

Mmm.

[music].

Doug Ingram: Okay, so it's a good question, and I'm not surprised by the comment. There are those that might say that I have a poor historical track record of predicting outcomes. Notwithstanding the fact that the last time I said that we were going to have an outcome, I did have that in writing. The reason that we don't believe we're going to have an outcome is that we don't believe that we will need one.

Doug Ingram: And I think that, you know, I believe as we said it today, that that is a view that would be shared by us and FDA leadership that we've said before. We had a very productive and encouraging discussion with the FDA leadership on the data and on the possibility of submitting it for a broad label. And I would also note that, you know, the agency has changed the division, a particular has gone through some pretty significant changes over the course of this year.

Doug Ingram: There's been a reorganization just to remind you where OTAG has been replaced by this super office OTP and not too long ago. A new leader, Dr. Nicole Verdun, took the helm as the head of OTP. So I would say, you know, just that we're not a remember that the division is evolving.

Doug Ingram: Obviously in the event that there was an advisory committee, we would be well prepared for it. And I believe we would perform exceptionally well there. I think Dr. Medina Clayback and team just did a fabulous job representing us. And as one may recall, we did ultimately win that outcome. But as we sit here today, we feel pretty confident that we can get a label extension without an advisory. Thank you, Debbie.

Unknown Executive: All right, thank you.

Unknown Executive: Thank you very much. Please stand by for the next question.

Gil Blum: The next question comes from Gil Blum with Needleman Company. Your line is open. Definitely congratulations on all the progress.

Louise Rodino: I'm going back to a question follow up on Colin's earlier questions about any questions we got. So, TTR study 102, is there any chance that there would be some follow up especially on the patients that were older and were crossed over in part two of study 102 regarding time to rise? It would be interesting to see how that data looks in comparison to the embark data. Thanks. Yeah, all the 102 patients are continued to follow for up to five years, so that's certainly something that we can look at over time. We don't have that data in hand, but we can look at that.

Brian Abrahams: Please stand by for the next question.

Brian Abrahams: The next question comes from Brian Abrahams with RBC Capitol. Your line is open.

Doug Ingram: Hi there, congrats on the strong first full quarter of the elevator. Thanks for taking my question. Can you remind us of the protocols in place in embark to protect against functional on blinding? Was this a topic that ever came up with the FDA in your recent discussions and might you expect any differences in the effect functional on blinding if there was any might have on time tests versus on NSA. Thanks. Yeah, I can allow Lisa to discuss the protocol aspects of the blinding process, which was exceptionally rigorous.

Doug Ingram: We can, you know, we can generally assume that it's, you know, that a very objective time test would be less subject to, you know, any kind of influence in the event there wasn't on blinding, but I would say also that I think the protocol was very good about the blinding process in the study itself. One should remember was actually very well run. I'm going to be clear about that. But Louise and thoughts on the blinding process.

Doug Ingram: Yeah, I'm just specifically so the patients and caregivers obviously blinded the PIs as well as the, the physical therapists doing the functional test are all completely blinded. So the studies maintained blinded study staff that threat is blinded maintained by a third party. So there's rigorous process in place to make sure that the blind remains intact. That's helpful. Thank you.

Louise Rodino: Please stand by for the next question.

Kristen Kluska: The next question comes from Kristen Kluska with Canter Fitzgerald. Your line is open. Hi, good afternoon. This is Jason Boothe on for Kristen Kluska.

Unknown Executive: Thank you for taking our question and congrats on the strong quarter for 11th. One question from us, the cadence of treating patients is going faster than the original timelines you laid out. So we're just wondering what the biggest drivers are there and how might this also impact the potentially broader launch next year. Thank you.

Doug Ingram: Well, I'm going to take the question even though Dallan wants to because I want to brag about our team. I mean, I think there's two significant reasons why the cadence of this launch is going exceptionally well and why this launch is from my perspective, an unprecedented success in gene therapy. The first of which, of course, is the therapy itself. This 11th is a extraordinarily needed therapy that, you know, patient to have been on it and families that share their experience with it strongly of the belief that they need this therapy and these kids have been stabilized and are doing things age specific that untreated kids haven't been able to do.

Doug Ingram: And then, you know, to do, but you don't mind me bragging a little bit about the team. I mean, this is an example of exceptionally great execution by the Sarepta folks led by Dallan and they should commercial customer officer. By going beyond that, this is our manufacturing and distribution folks as well, you know, just a large team effort to execute on this. And this isn't new for us.

Doug Ingram: I want to remind everyone now that we have now four therapies that we have launched every one of those therapies and their launches have gone exceptionally well. I mean, if you look at the piano system to digress for a moment, I mean, we are now from our first PMO that was approved in late 2016. We're still growing. We grew at 16% quarter over the same quarter last year, even as we're launching 11th, it's enjoying brilliantly there. So I think there's a combination, both of the great therapy as all of our fourth therapies, I believe, have been an exceptional focus, granular, well informed execution.

Doug Ingram: And what does this mean for the future? It means that we're going, we know how to serve the Duchenne community. And you know, one of the things that excites us about a broader label is we'll be able to bring a levitus to the majority of children and men, young men in the United States that are living with and degenerating irreparably from this ferocious disease. So I'm really excited about the opportunity to bring this therapy to more patients, even as we're doing really well with a launch right now.

Unknown Executive: Thank you very much for your question.

Joseph Schwartz: Please stand by for the next question. The last question comes from Joseph Schwartz with Delaring Partners. Your line is open. Great. Thanks very much for fitting me in. I was wondering, how are you thinking about upcoming clinical data for a different gene therapy candidate? What should have an error look soon? Is there anything that you'll be focused on in particular? And how do you see the trade off between safety and efficacy? If we're able to produce a greater impact on NSAA, how would that impact your relative value proposition for a levitus?

Doug Ingram: Oh, thank you for the question. Look, you know, as I've said many times before, we have in front of us an exceptionally ferocious competitor. And that's Duchenne, muscular. And all of our focus and all of our energy is in beating this damn disease. And I think this team is doing a brilliant job of that. We're exceptionally pleased with the performance of 11 is, you know, there were some, you know, many years ago, people made decisions about constructs and capsids and the like.

Doug Ingram: And with the benefit of many years of experience, we are exceptionally proud of. And frankly, nothing less than thrilled with the particular capsidant construct that we have. It's shown not only that it is able to, you know, intervene, protect these muscles of these children and arrest the decline. But it can do that with a particularly lot of all safety profile given the amount of therapy required here in the fact that it's full body infusions are at 74 has been a standout.

Doug Ingram: And we're, you know, quite confident that anyone who was rational, who had an opportunity to make a decision today about what capsid they would use and what construct they would develop, I'm sure they would do their best to try to copy us. So we're, we're not focused on any competitor besides Michelle must redistribute and we are for, for roses, we committed to beating this disease. So that's our, that's our focus right now.

Doug Ingram: But thank you very much for your questions.

Doug Ingram: I would now like to turn the call back over to Doug for closing remarks. Well, thank you very much, Michelle, and thanks everyone for attending tonight and thank you for your very thoughtful questions.

Doug Ingram: Let me summarize this quarter has been an extraordinarily important with our fourth approved approval. We launched a levitas in my opinion we launched it brilliantly. We have continued to serve the community with our PMOs, which continue robust growth, even in the face of an 11th launch. And on a non-gap basis, we are now profitable and we are marching toward a cash positive cash flow positive in the very near future.

Doug Ingram: We have built a strong enduring organization that is focused on two major things. The first is serving our patient community through brilliant science and the second is executing and getting things done. And that is precisely what we will be doing over the coming months. We intend to continue our strong performance and commitment to serving this community. We intend to move with speed to submit our efficacy supplement and conclude the review on the broadening on the broadening of the 11th is labeled. And when our label has been updated to remove age and regulation restrictions, we intend to bring this therapy to the majority of patients living with the Shannon United States.

Doug Ingram: And with that, I look forward to updating all of you on our progress along the way and have a lovely evening.

Unknown Executive: Thank you.

Unknown Executive: This concludes today's conference call. Thank you for participating.

Unknown Executive: You may now disconnect. Thank you very much. .