Sarepta Therapeutics Q3 2023 Earnings Call Transcript

Quartr
Provided by Quartr
November 1, 2023

There are 16 speakers on the call.

Operator

Good afternoon, and welcome to the Sarepta Therapeutics Third Quarter 2023 Earnings Call. At this time, all participants are in a listen only mode. After the speakers' presentation, there will be a question and answer 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.

Speaker 1

Thank you, Michelle, and thank you all for joining today's call. Earlier this We released our financial results for the Q3 of 2023. The press release is available on our website at sirepta.com and our 10 Q was filed with the Securities and Exchange Commission this afternoon. Joining us on the call today are Doug Ingram, Ian Estepan, Dalen Murray and Doctor. Louise Rodino Klapac.

Speaker 1

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

Speaker 1

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'll turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?

Speaker 2

Thank you, Fran. Good afternoon, and thank you for joining Sarepta Therapeutics' 3rd quarter 2023 financial results conference call. It was only 2 days ago that we announced the results of our double blind placebo CONTROL trial, EMBARK, therefore, I will not linger on the results here, but I will begin by summarizing Sarepta's perspective. First, taken as a whole, The results of Embark confirm that Alevitus stabilizes muscles, slows or entirely arrests decline, does so across the ages and does so with a laudable safety profile not shared by other programs for Duchenne. 2nd, the Embark results have not only satisfied The confirmatory requirements for our June approval, but have shown that levinist benefits patients across Age is consistent with its mechanism of action.

Speaker 2

Hence, we will soon be submitting a BLA supplement to broaden the Alevitus label to remove age and 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 doing so rapidly. Now let me comment on quarterly performance. The 3rd quarter was a defining moment for Sarepta. We launched our 4th therapy and the first Gene therapy for boys with Duchenne muscular dystrophy.

Speaker 2

We continued to drive great performance with our 3 PMOs and importantly on a non GAAP 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 Alevitus and continuing performance of our 3 approved PMOs, Exandis, Mayandis and Amandis, 3rd quarter total revenue came in at $332,000,000 and total net Product revenue stands at $309,320,000 growing 49% over the same quarter last year, Reflecting the team's ability to execute and serve Duchenne patients, Alevitus net product revenue came in at 69 point $11,000,000 nearly tripled mean external consensus. Likewise, Our PMOs achieved $240,210,000 in net product revenue growing 16% over the same quarter prior year. And non GAAP earnings stood at $38,000,000 in the quarter, a major milestone for Sarepta 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.

Speaker 2

Dalen Murray, our Chief Customer Officer, will walk you through what has been nothing short of a remarkable launch of Alevitus. And looking to the near future, we will take our proven track record of execution and move forward rapidly to expand the label of Alevitus, So this team can employ that level of execution to make Alevedis available to the majority of Duchenne patients in the United States. Following the balance discussion, Doctor. Rodino Klapac 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 4 approved therapies, We are going to substantially exceed $1,000,000,000 this year, another important milestone to be sure.

Speaker 2

And with that, let me turn the call to Dalen for a commercial update. Dalen?

Speaker 3

Thank you, Doug, and good afternoon. The launch of Alevitus in the Q3 was Sarepta's 4th Duchenne launch. It was by far the most complex and challenging to date. And I'm proud to say the team was ready on day 1 and they have knocked it out of the park thus far. As Doug has mentioned, We generated just over $69,000,000 in net product revenues in the Q3 for Alevitus.

Speaker 3

Notably, the team seated 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 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 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. Our early success was achieved in 3 ways.

Speaker 3

1st, flawless 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. And third, establishing a well functioning flexible distribution model supported by the Sarepta Sips team to get each patient's customized kit to the site of care at the right time just in time for the infusion. 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 lebitis, Led by our doctors, nurses and other HCPs, 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.

Speaker 3

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

Speaker 3

The fast start in Q3 was a function of the team's efforts in the quarter itself. And just as importantly, their efforts over the past 7 years building the model, which we have established to support all of the Duchenne patients eligible for our therapies. Had this been Sarepta's first launch, our trajectory in the Q3 would have been very different and much slower. We've gained deep knowledge and expertise through 3 PMO launches, and I'm glad to say we were able to apply these learnings to the launch of alevitus. So to summarize alevitus, it was a great Q1 for the launch because our team and our key stakeholders were prepared And they executed flawlessly to support the patients we serve.

Speaker 3

Driven in large part by the robust Alevitus 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 $309,300,000 Importantly, as Doug said, in addition to our success with Alevitus, 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 olebitis over time.

Speaker 3

Since the 4% to 5% 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 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 $240,000,000 representing roughly 16% increase over the same quarter in 2022. Looking now at each of our PMOs individually, Net product revenue for EXONDYS fifty one was $142,300,000 in Q3, which was over 16% above the same quarter in 2022.

Speaker 3

Viannis 53 net product revenue was 31,700,000 3.4% above Q3 of 2022 and AMONDYS 45 generated net product revenue of 66 $300,000 in Q3 of 2023. This represents roughly 21% growth over Q3 of 2022. 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 U. S.

Speaker 3

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 annual guidance of greater than $925,000,000 in net product revenue for our PMO business in 2023. I'll end by saying that I've been continually amazed and impressed by the resilience, Commitment and execution of our Sarepta teams over the years.

Speaker 3

And while the success over those years has been impressive, What the teams have achieved in the Q3 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 Sarepta 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. The individual stories from across the country are too numerous to mention here. Nor can we as a team put into words the joy we feel where we celebrate each and every patient who gains access to any of our dystrophin restoration therapies.

Speaker 3

And so with that, I'll turn the call over to Doctor. Louise Rodino Klapac. Louise?

Speaker 1

Thanks, Dalen. 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 Duchenne, limb girdle and many other diseases for which therapies are either inadequate were non existent. As Doug has already detailed the embark results, I'll focus my comments on the progress of our gene therapy and RNA programs. First, Limb girdle muscular dystrophy or LGMD.

Speaker 1

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 at the Speak Foundation's 2023 International LGMD Conference and shared our urgency with the community to bring forth genetic medicines for LGMD. To begin, we made excellent progress for VOYAGINE, our Phase 1 study evaluating SRP-nine thousand and three for the treatment of limb girdle muscular dystrophy type 2E in ambulant adult patients and non ambulant patients using clinical process SRP-nine thousand and material. We are pleased to report that we completed enrollment in Voyaging and we remain on track to initiate our Phase 3 study using commercially representative process material later this year. Combined with positive expression and functional data shared from our initial study, SRP9003-1 hundred and one.

Speaker 1

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 SRP-six thousand and four Dual vector rh74 mediated gene therapy to treat individuals with LGMD2b. LGMD2b is characterized by the absence of the protein Disferlin. The innovative dual vector strategy allows us to deliver the full length dysferlin gene, the sole cause of LGMD2b. We look forward to reporting results from the study in the first half of twenty twenty four.

Speaker 1

As mentioned last quarter, our LGMD natural history study of the sarcoglycanopathies, LGMD IIe, IIc and IId, called Journee, has been fully enrolled and we will follow patients for 36 months. We continue to make progress in scalable manufacturing for all of our LGMD 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 Q1 of 2023 for a MOMENTUM study for SRP-five thousand and fifty one, and we're targeting readout of the study in 2023. Regarding our post marketing studies for the PMOs, as mentioned last quarter, We completed enrollment in the ESSENCE trial, a post marketing requirement for golodirsen and casimersen.

Speaker 1

As a reminder, Essence is a 2 year study and is due to readout in early 2026. Finally, we are pleased to have completed enrollment in our mission study, Our dosed ranging post marketing commitment for EXONDYS. Mission is a randomized double blind safety and efficacy dose finding study Comparing the approved dosage of ateplirsen, a 30 mgs per kg weekly to a dosage that provides significantly higher exposure up to 200 mgs per kg weekly. MISSION is a 2 part Phase 3 study that was fully enrolled in October 2023 with 160 patients enrolled. We remain committed to rapidly and diligently advancing mission and sharing data as soon as it becomes available.

Speaker 1

We look forward to reporting continued progress with our RNA in the coming months. On a personal note, my passion for science and its promise to help others began early in life. These many years later, as I reflect on my career and where we are today in realizing the promise of genetic medicine, I'm grateful and I'm humbled. And yet, we have so much more to do. We move forward from here today toward a more promising future for individuals around the world living with rare disease.

Speaker 1

In closing, I'd like to take a moment to thank the Sarepta team for their continued dedication and passion to patients, the science and our mission. I'll now turn the call over to Ian for an update on our financial results. Ian?

Speaker 4

Well said, Luis. Good afternoon, everyone. This afternoon's financial results press release provided details for the Q3 of 2023 on a non GAAP basis as well as a GAAP basis. Please refer to our press release available on Sarepta's website for a full reconciliation of GAAP to non GAAP financial results. We're obviously quite pleased with the financial results for this quarter.

Speaker 4

On the back of a tremendous start for the EXONDYS launch, we actually achieved non GAAP profitability. And assuming an expansion to the label to the broader Duchenne population, we should achieve sustained profitability. We're quite thrilled to achieve this milestone just in the Q1 of the launch. For the 3 months ended September 30, 2023, the company recorded Total revenues of $331,800,000 which consists of net product revenues and collaboration revenues compared to revenues of $230,300,000 for the same period of 2022, an increase of $101,500,000 Net product revenue for the Q3 of 2023 from OLEVIUS was $69,100,000 Net product revenue for the same period from our Exxon's skipping franchise was $240,200,000 compared to $207,800,000 for the same period of 2022. For the quarter, individual net product sales were $142,300,000 for EXONDYS 51, dollars 66,300,000 for AMONDYS 45 and $31,700,000 for VYONDYS 53.

Speaker 4

The increase in net product revenue primarily reflects increasing demand for our PMO products as well as net product revenue associated with the sales of ELEVITIVE. In each of the quarters ended September 30, 2023, 2022, We recognized $22,500,000 of collaboration revenue, which relates to our collaboration arrangement with Roche. The reimbursement of co development costs under the Roche agreement totaled $34,900,000 for the Q3 of 2023 compared to $22,000,000 for the same period of 2022. On a GAAP basis, we reported a net loss of $40,900,000 or 0.46 dollars per basic and diluted share and $257,700,000 or $2.94 per basic and diluted share For the Q3 of 2023 2022, respectively, we reported a non GAAP net income of $37,700,000 or $0.37 per diluted share in the Q3 of 2023 compared to a non GAAP net loss of $70,000,000 or $0.80 per diluted share in the Q3 of 2022. In the Q3 of 2023, we recorded approximately $37,000,000 in the cost of sales compared to $40,000,000 for the same period of 2022.

Speaker 4

The decrease in cost of sale primarily reflects write off for certain batches of our PMO products not meeting our quality specifications in the 3 months ended September 30, 2022, with no similar activity for the same period of 2023, partially offset by increasing demand for our PMO products. On a GAAP basis, we recorded $194,300,000 $216,700,000 in R and D expenses For the Q3 of 2023 2022, respectively, a year over year decrease of $22,400,000 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 and D expenses were $163,900,000 for the Q3 of 2023 compared to $193,700,000 for the same period of 2022, a decrease of $29,800,000 Now turning to SG and A. On a GAAP basis, we recorded approximately $120,900,000 and $104,800,000 of expenses for the Q3 of 2023 2022, respectively, an increase of $16,100,000 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 and A expenses were $92,800,000 for the Q3 of 2023 compared to $66,800,000 the same period of 2022, an increase of $26,000,000 On a GAAP basis, we recorded $12,300,000 in other expense Net for the Q3 of 2023 compared to $400,000 in other income net for the same period of 2022.

Speaker 4

The change is primarily due to an impairment of our investment and loss on contingent consideration, net partially offset by increases in increases of 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 $1,800,000,000 in cash, cash Investments in long term restricted cash as of September 30, 2023. We're obviously pleased with the amount of capital on our Balance sheet in turbulent 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 our cash than to build inventory to serve those with DMD.

Speaker 4

With that, I'll turn the call over to Doug to start Q and A. Doug?

Speaker 2

Thank you very much, Ian. Michelle, let's open the call for questions.

Operator

Thank Please standby while we compile the Q and A roster. The first question comes from Anupam Rama with JPMorgan. Your line is open.

Speaker 5

Hey, guys. Just going back to the Embark results, we've gotten this question. If you guys adjust for multiplicity on your key functional secondary endpoints. Would you still have statistically significant outcomes on these key measures? Thanks so much.

Speaker 2

Yes. Thank you for that question, Anupam. I will turn this call over to Louise.

Speaker 1

Thank you. We actually performed a global statistical test and we did this to do just that, the test for multiplicity and show that the secondary is do not hit significant just by chance. So we essentially tested NSAA combined with the secondary and show that they were in fact statistically significant. So this is a quantitative way to test the totality of evidence In respect, so if you think of the 4 spot that we showed on our call, that's essentially a statistical test to show that together, we see that these are statistically significant.

Operator

The next question comes from Tazeen Ahmad with Bank of America Securities. Your line is open.

Speaker 6

Hi, guys. Good afternoon. Thanks so much for taking my question. For me, I think one of the most popular inbounds I've been getting in the last 2 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?

Speaker 6

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

Speaker 2

Tazeen, thank you for your question. And then let me preface my question by saying, I'm probably going to frustrate you By not giving hard and fast 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.

Speaker 2

I don't want to commit to the exact date, but very, very soon That will be submitted. I think traditionally the agency may have 6 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 Prejudice for this. You see this in other areas like oncology all the time where you can get For something like this, very fast turnarounds.

Speaker 2

And of course, remember, this isn't a BLA, but a BLA supplement. So The inquiry, while extraordinarily important, is focused and that focus is on The fundamental question, does the totality of the evidence justify the conclusion that Alevitus is bringing a better life to these patients? And of course, we believe that it does. The standard for this is quite clear. It's Substantial evidence, looking at the totality of evidence, the statute on this is quite clear.

Speaker 2

I apologize. I don't know what that means because I promise I'm not playing a guitar right now. The statute says, it's very clear, I want to fairly and responsibly conclude that the therapy will have the effect it reports to have and the regulations are also particularly clear That's for life threatening and severely debilitating illnesses, one's like Duchenne, especially where no satisfactory alternative therapy exists, The FDA has determined that it is appropriate to exercise the broadest flexibility in applying the statutory standards. And as Louise just pointed out to everybody, not only are the evidence on whole Very compelling that Alevitus 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 multiplicities that is powerfully Statistically significant.

Speaker 2

So all of which is to say, winding back to the original question to Zine, 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 broadness label.

Speaker 1

Okay. Thanks Doug. Thank you very much.

Operator

Please standby for the next question. The next question comes from Gena Wang with Barclays. Your line is open.

Speaker 7

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 shared with us? Would that also be like say the protein correlation of the protein level versus function, will These data also will be included in the package?

Speaker 2

There will be certainly more in the package than the top line. 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, 1st and foremost, The efficacy and the 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 age limitations and ambulation limitations on that. I don't think we've made any final decisions about what we're announcing during this process.

Speaker 2

But obviously, generally speaking, we tend to be pretty transparent with

Operator

The next question comes from Collyn Bristow with UBS. Your line is open.

Speaker 8

Hey, good afternoon and congrats on the impressive Enevedis sales. Maybe another one on the EMBARK data. Can you speak to the inter patient variability you saw in ELEVADIS treated patients? A sort of a question we've been getting is, You were the positive results driven by a small group of high responders. And maybe if you could also comment on how this variability compared to the Phase 2 That would be helpful.

Speaker 8

Thanks.

Speaker 2

Sure. I'm going to flip this over to Luis to answer specifically. But I would generally note The positive results, the P value on these positive results are they're not close. On the time to rise, It's 0.002 on the 10 meter walk around, it's 0.004. On the global statistical analysis, when one looks primary and all the secondaries together, it's 0.004.

Speaker 2

So it's very, very powerful. But Louise, perhaps you want to answer more specifically on some of the variability issues.

Speaker 1

Yes. And generally, we did not see variability high variability amongst the patients. The standard deviation was either 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.

Speaker 8

Thank you. That's helpful.

Operator

Please standby for the next question. The next question comes from re Forsyth with Guggenheim. Your line is open.

Speaker 3

Hi, this is Rai on for Debjit. Does the top line data provided to the FDA from

Speaker 2

I am very sorry, but I was unable to hear that answer that question. Can you ask it again perhaps?

Speaker 3

Yes. This is Rai on for Debjit. Did the top line data provided to the FDA on Embark

Speaker 2

Goodness, I am so sorry, right. I heard the beginning, but I don't hear the question itself.

Speaker 3

I think he asked if it included data on microdystrophin expression.

Speaker 2

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 There's nothing unusual about it. In fact, the P value on it is 0.0, many zeros. It's very strongly, Robustly, as you wouldn't expect, 11 is robustly makes dystrophin.

Speaker 2

That's the question, Pavel.

Speaker 3

That helps. Thank you.

Speaker 2

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

Operator

Please standby for the next question. The next question comes from Brian Skorney with Baird. Your line is open.

Speaker 9

Hey, good afternoon. Thanks for taking the question. I guess it wasn't something to look at very closely Before, Bob, I just want to tell, I think Contarize had a non significant difference in favor in treatment, but not that different in terms of magnitude, I think maybe just under half a second differences. Can you tell me if 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 to rise The active arm was 5.1 at baseline and placebo was 3.6.

Speaker 9

So I was just wondering if you've gone back and looked at making adjustments for baseline imbalances to evaluate And to rise differences in Study 102 and in particular if the 4 to 5 year old subgroup looks the same in 102 as a dead end of mark?

Speaker 2

Yes. Thank you for that question, Brian. I'll turn this to Louise to respond.

Speaker 1

I'm going to speak generally 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 difference when you exclude those patients that would have been excluded by that criteria in 102 where you had those rapid decliners. So in that case, we saw a more significant difference, but Specific numbers are escaping that right now, but we did do that analysis where we kind of applied the same exclusion criteria and did see a difference.

Speaker 3

Great. Thank you.

Speaker 4

Yes. Maybe just one thing to add in that analysis to Luis' point. We saw a very we saw a really good consistency between, what we observed in 102 and what we have observed in 301.

Operator

Please stand by for the next question. The next question comes from Salveen Richter with Goldman Sachs. Your line is open.

Speaker 10

Good afternoon. Thanks for taking my questions.

Speaker 1

With regard to the

Speaker 10

regulatory submission, are there Formal or regulatory procedures involved with revisiting an accelerated approval after, the primary endpoint fails and in a confirmatory 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?

Speaker 2

Again, on the first question, I'm not 100% confident I understand the question. Let me be very clear. The standard for confirmation of an accelerated 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 confirmatory data, But all of the surrounding evidence that would exist, including other studies. And I would strongly argue that Not only Embark, but all of the supporting evidence as well has strongly confirmed the benefits of this therapy. So I think we're in very good shape there.

Speaker 2

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 it relates to that, as you know, our strong view is that having confirmed these results, having confirmed them across Patients and looking at the totality of this evidence and looking at the forest blot as an example and looking at the statistical analysis of the forest blot, Adjusted for multiplicities, it is quite clear that this therapy is arresting the decline in these patients and deserves to be Made available to patients without limitation to age or artificial restrictions around ambulation. As it relates to payers, this is additional evidence in our armamentarium with payers. Things have gone Very well. Dalen and his team, medical affairs, our commercial, our field force, access to reimbursement and the like I've just done a fabulous job supporting the launch of levetis, and I hope everyone will agree with me that it shows in our performance This quarter and this bolsters the discussions that the team can have.

Speaker 2

Now we can As an example, we now have a really powerful metric that is compelling on the speed with which one should put a kid On therapy. So as well, I will remind you on the time to rise, not only is the p value, I think 0.002, if I am not mistaken. But in time to rise is the single greatest prognosticator Loss of ambulation and a rise time above 5 seconds, as we've talked about often and in the literature, robustly, Is the single greatest predictor of early loss of ambulation and And Mark has shown that using a Levitus reduces the odds of that occurring in a 52 week period by over 90%. So this provides an additional compelling point with payers who frankly so far have done a really good job of providing access. This provides additional evidence that it really is important to get kids on this therapy as soon as possible.

Speaker 2

And I would argue looking forward to label expansion and it's a compelling argument for why this therapy should be this label should be expanded as soon as possible as So everyone has access to it.

Operator

Please standby for the next question. The next question comes from Yair with Mizuho. Your line is open.

Speaker 11

Hey, guys. Thanks for taking my question. Congrats on the great quarter for Elavides. So I guess my first question is, were those the patients who were dosed, were those primarily those who We're pretty much anticipated, the approval, the accelerated approval and they I guess, more like the bolus. And like how many would you be able to share how many patients are waiting to be dosed in the coming quarter?

Speaker 11

And just continuing this theme, now that the AMBARK 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.

Speaker 2

Yes, let me answer those questions quickly. First, I don't think there will be any shift in the Desire for this therapy, I think, except for patients that are not in the parents or patients that are not in the 4 to 5 year old range, I I think are probably even more compelled 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 metric for success and the measure of our success Net product revenue, as we've said, on the issue of sort of bolus or warehousing of patients, there were certainly some number of patients That physicians were getting together and gathering to ensure that they could preferentially get dosed before they Aged out because the label obviously as you know restricts the dosing to 6 and below 6 and 5. But we've had a steady stream of new start forms since the approval.

Speaker 2

And so looking forward, we have a steady Stream of start forms that are working through the process. And one other thing people have been asked, but I'll say it, Dennis, the team, Dell and others has done a brilliant job on-site activation. I think our goal as you know, our Goal had aspirationally been to have 50 sites ready to infuse at launch. And then very aspirationally, we thought perhaps by the end of the year or 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 through.

Speaker 11

Thank you.

Operator

Please stand by for the next question. The next question comes from Danielle Brill with Raymond James. Your line is Fin?

Speaker 1

Hi, guys. Good afternoon. Thanks so much for the question. Doug, so we spent a lot of time discussing embark efficacy data. I'd like to switch over to safety.

Speaker 1

I recall myocarditis events being discussed at the AdCom, including one event that had occurred in Embark at the time. Just curious if there are any additional safety events of this nature that occurred in the study or any other SAEs leading to

Speaker 2

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

Speaker 1

Yes. We did not see any differences in the types of SAEs or the frequency of those SAEs. That was One of the most, reassuring things about Embark was the continued safety profile and now taken together all of the previous trials, we have a large safety data base

Operator

The next question comes from Ritu Baral with TD Cohen, your line is open.

Speaker 12

Good afternoon, everyone. Thanks for taking the question. Doug, could you, and maybe Dalen walk us through how you see patient numbers For the different scenarios that our regulatory experts suggest could are in play essentially for label expansion, You mentioned 10% of your PMO sales are for 4 to 5, those are covered. But how would you segment it by Maybe 6 to 7 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 per my calls now extends to like 12 years of age?

Speaker 2

For ambulatory?

Speaker 12

Yes. Like that's the average age of loss of ambulation now, 12, 13?

Speaker 2

That may be correct. I'll let Alan 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 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, Given the data, you've never seen that before. Once the you've never seen in any other Label for Duchenne therapy, these sorts of age related restrictions.

Speaker 2

But answering the broadest Question, the all ambulatory versus all ambulatory and non ambulatory It's 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 to our mind is the big cut. But Dalen, is there anything else you'd like to say on this topic?

Speaker 3

No, I think you covered it, Doug. Our goal is to target all the patients that we can get in the entire population. Ritu, to your question about average age of loss of ambulation, I think you're in the right range. But the KOLs have been talking a lot about the definition of loss So, Van Galatian too, because as you know, there's heterogeneity. So 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 ambulation as well.

Speaker 3

So it won't be we believe the ambulatory population won't be defined by age. But as Doug said, our aspiration is A broad label and targeting all of the patients who will be eligible.

Speaker 12

And is that 50% of your PMO sales too?

Speaker 2

On the

Speaker 3

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 The higher penetration in the younger angiopulmonary population.

Speaker 12

Great. Thank you.

Operator

Please standby for the next question. The next question comes from Mike Ulz with Morgan Stanley. Your line is open.

Speaker 13

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 Aleveitis so far? Thanks.

Speaker 2

We are going to apologies, 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 7 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, Mike.

Operator

Please standby for the next question. The next question comes from Tim Lugo with William Blair. Your line is open.

Speaker 5

Thanks for the question. You mentioned earlier in the week that you didn't expect an additional advisory committee for broadening the label, But we can all remember when we didn't expect 1 for the accelerated approval discussion. I guess Why not request an AdCom? It seems like listening to the AdCom, the participants were much more amenable to a non age restricted approval Than the agency was?

Speaker 2

Yes. 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 adcoms. Notwithstanding the fact that the last time I said we weren't going to have an adcom, I did have that in writing.

Speaker 2

The reason that we don't believe we're We're going to have an adcom is that we don't believe that we will need 1. And I think that I believe as we sit here today That is a view that would be shared by us and FDA leadership. As 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 The agency has changed the division, in particular, has gone through some pretty significant changes over the course of this year. There's been a reorganization, just to remind you, where OTAT, has been replaced by this super office OTP. And not too long ago, a new leader, Doctor.

Speaker 2

Nicole Verdun, took the helm as the Head of OTP. So I would say just that we're not to remember that the division is evolving. 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 Doctor.

Speaker 2

Rodino Klapac and team just did a fabulous job representing us. And As one may recall, we did ultimately win that AdCom. But as we sit here today, we feel pretty confident that We can get a label expansion without an advisory committee. All right. Thank you.

Speaker 2

Thank you very much.

Operator

Please standby for the next question. The next question comes from Gil Blum with Needham and Company. Your line is open.

Speaker 3

Good afternoon and congratulations on all the progress. Going back to a question follow-up on Colin's earlier question about many questions ago. 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 and Part 2 of Study 102 regarding time to rise, it would be interesting to see how that data looks in comparison to the embark data. Thanks.

Speaker 5

Sure. Louise, your thoughts on that?

Speaker 1

Yes. All the 102 patients are continued to fall for up to 5 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.

Operator

The next question comes from Brian Abrahams with RBC Capital, your line is open.

Speaker 14

Hi there. Congrats on the strong first full quarter of the Elevides launch. Thanks for taking my question. Can you remind us of the protocols in place in Embark to protect against functional unblinding? Was this a topic that ever came up with the FDA in your recent discussions?

Speaker 14

And But you expect any differences in the effect functional and blinding, if there was any, might have on time tests versus on NSAA? Thanks.

Speaker 2

Yes. I can allow Luis to discuss the protocol aspects of the blinding process, which was exceptionally Rigorous. We can generally assume that The very objective time test would be less subject to any kind of influence in the event there was an unblinding. But I would say also that I think the protocol was very good about the blinding process and the study itself, one should remember was actually very well run. I want to be clear about that.

Speaker 2

But Louise, any thoughts on the blinding process?

Speaker 1

Yes. And just specifically for the patients and caregivers obviously blinded the PIs as well as The physical therapists doing the functional tests are all completely blinded. So the studies maintain blinded, The staff that's rep is blinded, it's maintained by a third party. So there's a rigorous process in place to make sure that the blind remains intact.

Speaker 14

That's helpful. Thank you.

Operator

Please stand by for the next question. The next question comes from Kristen Kluszko with Cantor Fitzgerald. Your line is open.

Speaker 15

Hi, good afternoon. This is Jason Bouvier on for Kristen Kluska. Thank you for taking our question and congrats on the strong quarter for Alevitus. One question, from us, the cadence of treating patients is going, faster than the original timelines you laid So we're just wondering what the biggest drivers are there and how might this also impact the potentially broader launch next year?

Speaker 2

Thank you. Well, I'm going to take the question even though Dylan wants to because I want to brag about our team. I mean, I think there's 2 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 Levitas is extraordinarily needed therapy that patients who have been on it and families that share their experience with it are Strongly of the belief that they need this therapy and these kids have been stabilizing or doing things, age specific that untreated kids haven't I've been able to do.

Speaker 2

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 Sarepta folks led by Dallin and their Chief Commercial Customer Officer. By going beyond that, this is our manufacturing and distribution folks, as well, just A large team effort to execute on this. And this isn't new for us. I want to remind everyone now that we have now 4 therapies that we have launched. Every one of those therapies and their launches have gone exceptionally well.

Speaker 2

I mean, if we look at the PMO, just 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 Alevitus and doing brilliantly there. So I think there's a combination both of a great therapy as all of our 4 therapies I believe have been and exceptional Focused, granular, well informed execution.

Speaker 2

Now what does this mean for the future? It means that we're going we know how to Serve the Duchenne community. And one of the things that excites us about a broader label is we'll be able to bring Alevitus To the majority of children and men, young men in the United States that are living with and degenerating Irreparably from this ferocious disease. And so, I'm really excited about the opportunity to bring this therapy to more patients, Even as we're doing really well with the launch right now. Thank you very much for your question.

Operator

Please stand by for the next question. The last question comes from Joseph Schwartz with Deleerink Partners. Your line is open.

Speaker 5

Great. Thanks very much for fitting me in.

Speaker 4

I was wondering How are you thinking about upcoming clinical data for

Speaker 5

a different gene therapy candidate, which we'll have an interim 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 it were able to produce a greater impact on an SAA? How would that impact Your relative value proposition for Levitus.

Speaker 2

Thank you for the question. Look, as I've said many times before, We have in front of us an exceptionally ferocious competitor and that's Duchenne muscular dystrophy. 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 Levitus.

Speaker 2

There were some many years ago, People made decisions about constructs and capsids and the like. And with the benefit of many years of experience, we are exceptionally Proud of and frankly nothing less than thrilled with the particular capsidin construct that we have. It's shown Not only that it is able to intervene, protect these muscles of these children and arrest the decline, That 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. Rh74 has been a standout and we're 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.

Speaker 2

So we're not focused on any competitor besides Duchenne muscular dystrophy, And we are ferociously committed to beating this disease. So that's our focus right now. Thank you very much for your questions.

Operator

I would now like to turn the call back over to Doug for closing remarks.

Speaker 2

Well, thank you very much, Michelle, and thanks everyone for attending tonight and thank you for your very thoughtful questions. Let me summarize. This quarter has been an extraordinarily important one. With our 4th approval, we launched Alevitus, 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 Alevitus launch and on a non GAAP basis, we are now profitable and we are marching toward a cash positive cash flow positive in the very near future.

Speaker 2

We have built a strong enduring organization that is focused on 2 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 of the Alevitus label.

Speaker 2

And when our label has been updated to remove age and ambulation restrictions, we intend to bring this therapy to the majority of patients living with Duchenne in the United States. And with that, I look forward to updating all of you on our progress along the way and have a lovely evening. Thank you.

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Earnings Conference Call
Sarepta Therapeutics Q3 2023
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