Sarepta Therapeutics, Inc. (SRPT) Q3 2023 Earnings Call Transcript

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Q3 2023 Earnings Convention Name November 1, 2023 4:30 PM ET

Firm Individuals

Francesca Nolan – IR Director

Douglas Ingram – President and CEO

Louise Rodino-Klapac – EEVP, Chief Scientific Officer and Head of Analysis & Improvement

Dallan Murray – EVP & Chief Buyer Officer

Ian Estepan – EVP & CFO

Convention Name Individuals

Anupam Rama – JPMorgan

Tazeen Ahmad – Financial institution of America Securities

Gena Wang – Barclays

Colin Bristow – UBS

Brian Skorney – Baird

Salveen Richter – Goldman Sachs

Uy Ear – Mizuho

Danielle Brill – Raymond James

Ritu Baral – TD Cowen

Mike Ulz – Morgan Stanley

Tim Lugo – William Blair

Gil Blum – Needham & Firm

Brian Abrahams – RBC Capital

Jason Bouvier – Cantor Fitzgerald

Joseph Schwartz – Leerink Companions

Operator

Good afternoon and welcome to the Sarepta Therapeutics Third Quarter 2023 Earnings Name. Right now, all contributors are in listen-only mode. After the audio system’ presentation, there shall be a question-and-answer session. [Operator Instructions] Please be suggested that in the present day’s convention is being recorded.

Right now. I will flip the decision over to Francesca Nolan, Govt Director, Investor Relations and Company Communications. Please go forward.

Francesca Nolan

Thanks, Jonathan. And thanks all for becoming a member of in the present day’s name. Earlier this afternoon, we launched our monetary outcomes for the third quarter 2023. The press launch is out there on our web site at sarepta.com and our 10-Q was filed with the Securities and Change Fee this afternoon.

Becoming a member of us on the decision in the present day, are Doug Ingram; Ian Estepan; Dallan Murray and Dr. Louise Rodino-Klapac. After our additional remarks, we’ll open the decision for Q&A.

I would prefer to know that in this name, we shall be making quite a lot of forward-looking statements. Please take a second to evaluation our slides on the webcast which comprises our forward-looking statements. These forward-looking statements contain dangers and uncertainties lots of that are past Sarepta’s management. Precise outcomes might materially differ from these forward-looking statements and any such dangers can materially adversely have an effect on the enterprise, the outcomes of operations and buying and selling costs for Sarepta’s widespread inventory.

For an in depth description of relevant dangers and uncertainties, we encourage you to evaluation the corporate’s most up-to-date quarterly report on Type 10-Q filed with the SEC, in addition to the corporate’s different SEC filings. The Firm doesn’t undertake any obligation to publicly replace its forward-looking assertion, together with any monetary projections supplied in the present day primarily based on subsequent occasions or circumstances.

And now we’ll flip the decision over to our President and CEO, Doug Ingram, who will present an summary of our current product progress. Doug?

Douglas Ingram

Thanks, Fran. Good afternoon, and thanks for becoming a member of Sarepta Therapeutics’ third quarter 2023 monetary outcomes convention name. It was solely two days in the past that we introduced the outcomes of our double-blind placebo-controlled trial EMBARK. Subsequently, I can’t linger on the outcomes right here, however I’ll start by summarizing Sarepta’s perspective.

First, taken as an entire, the outcomes of EMBARK verify that ELEVIDYS stabilizes muscle tissue, slows or totally arrests decline, does so throughout the ages, and does so with a laudable security profile not shared by different applications for Duchenne.

Second, the EMBARK outcomes haven’t solely happy the confirmatory necessities for our June approval, however have proven that ELEVIDYS advantages sufferers throughout age teams in step with its mechanism of motion. Therefore, we are going to quickly be submitting a BLA complement to broaden the ELEVIDYS label to take away age and ambulation restrictions.

And eventually, we now have already engaged in productive and inspiring discussions with FDA, they usually have confirmed that they’re dedicated to reviewing an utility to broaden the label and are dedicated to doing so quickly.

Now let me touch upon quarterly efficiency. The third quarter was a defining second for Sarepta. We launched our fourth remedy and the primary gene remedy for boys with Duchenne Muscular Dystrophy. We continued to drive nice efficiency with our three PMOs, and importantly, on a non-GAAP foundation, we achieved profitability, putting us in ever extra rarefied biotech territory.

As you should have seen in our launch, led by an distinctive launch of ELEVIDYS and persevering with efficiency of our three accepted PMOs, EXONDYS, VYONDYS and AMONDYS. Third quarter complete income got here in at $332 million, and complete web product income stands at $309.32 million, rising 49% over the identical quarter final yr reflecting the crew’s potential to execute and serve Duchenne sufferers. ELEVIDYS web product income got here in at $69.11 million, practically tripled imply exterior consensus.

Likewise, our PMOs achieved $240.21 million in web product income, rising 16% over the identical quarter prior yr, and non-GAAP earnings stood at $38 million within the quarter, a serious milestone for Sarepta as we transitioned to a worthwhile and within the close to time period money circulation optimistic group.

The crew has achieved an amazing job, working with households, physicians, and payers within the quarter and it reveals in these outcomes. Dallan Murray, our Chief Buyer Officer will stroll you thru what has been nothing in need of a exceptional launch of ELEVIDYS.

And trying to the close to future, we are going to take our confirmed monitor document of execution and transfer ahead quickly to broaden the label of ELEVIDYS so this crew can make use of that stage of execution to make ELEVIDYS out there to the vast majority of Duchenne sufferers in the US.

Following Dallan’s dialogue, Dr. Rodino-Klapac, who will present an replace on our pipeline progress? Now, as you’d count on, we aren’t offering up to date steering this early within the launch, but in addition clearly throughout our 4 accepted therapies, we’re going to considerably exceed $1 billion this yr, one other vital milestone to make sure.

And with that, let me flip the decision to Dallan for a industrial replace. Dallan?

Dallan Murray

Thanks, Doug, and good afternoon. The launch of ELEVIDYS within the third quarter was Sarepta’s fourth Duchenne launch. It was by far probably the most advanced and difficult thus far. And I am proud to say the crew was prepared on day one they usually have knocked it out of the park so far.

As Doug has talked about, we generated simply over $69 million in web product revenues within the third quarter for ELEVIDYS. Notably, the crew exceeded our personal lofty website readiness expectations with practically 70 websites able to dose in the present day. This helps us help the sufferers prone to growing older out in the present day and likewise units us up for long run success going ahead.

We approached this launch by constructing organically upon what was already best-in-class Duchenne industrial and medical groups. We put further duty on everybody throughout the board quite than constructing out separate gene remedy groups. And so they have all stepped up magnificently to fulfill this vital second. Our early success was achieved in 3 ways.

First, flawless execution with our exterior stakeholders, that’s the neuromuscular KOLs, gene remedy websites of care, and the eligible sufferers they serve. Secondly, proactive payer engagement to expedite entry for these sufferers who’re eligible primarily based on the label. And third, establishing a well-functioning, versatile distribution mannequin supported by the Sarepta SiPs crew to get every affected person’s custom-made equipment to the positioning of care on the proper time, simply in time for the infusion.

I want to take a second now to acknowledge the Duchenne neighborhood and the way they got here collectively to expedite entry for sufferers who’re eligible for ELEVIDYS. Led by our docs, nurses, and different HCPs, all stakeholders had been prepared for this vital second, and it was humbling to see the entire neighborhood come collectively to help eligible sufferers of their remedy journey. This launch additionally demonstrated the progress our groups and the specialists have made previously a number of years, educating the payers about Duchenne. We had been gratified by the urgency of payers and expediting insurance policies that allowed entry for eligible sufferers.

Moreover, and importantly, the payers performed a key position in supporting sufferers who’re prone to growing older out. Saying all of that we nonetheless have rather more work to do with among the payers to realize our objective of securing entry and remedy for all eligible sufferers throughout the nation, no matter plan.

The crew is working diligently as we communicate, educating the payers on the robustness of the newly out there EMBARK knowledge. We’re assured that this knowledge units the stage properly for entry to align with our label in the present day, in addition to after we acquire a broader label. The quick begin in Q3 was a operate of the crew’s efforts within the quarter itself, and simply as importantly, their efforts over the previous seven years constructing the mannequin which we now have established to help the entire Duchenne sufferers eligible for our therapies.

Had this been Sarepta’s first launch, our trajectory within the third quarter would have been very completely different and far slower. We have gained deep information and experience via three PMO launches, and I am glad to say we had been capable of apply these learnings to the launch of ELEVIDYS.

So to summarize ELEVIDYS, it was an awesome first quarter for the launch as a result of our crew and our key stakeholders had been ready they usually executed flawlessly to help the sufferers we serve. Pushed largely by the strong ELEVIDYS income within the third quarter, we grew total web product income by roughly 30% over the prior quarter. Web product income in Q3 of 2023 was $309.3 million.

Importantly, as Doug mentioned, along with our success with ELEVIDYS, we additionally had our most profitable quarter ever, serving sufferers with our established PMO franchise. We see continued alternatives within the U.S. and globally for our PMO enterprise despite the truth that we additionally count on cannibalization from ELEVIDYS over time. For the reason that 4-5 [Ph] inhabitants represents far lower than 10% of the PMO enterprise within the U.S., this cannibalization won’t have a cloth influence on our 2023 web product income. I would prefer to take a second right here to thank all of those that are relentlessly supporting our PMO sufferers.

So because of the entire crew’s effort, the online product income from our PMO enterprise in Q3 was $240 million, representing a roughly 16% enhance over the identical quarter in 2022. Trying now at every of our PMOs individually, web product income for EXONDYS 51 was $142.3 million in Q3, which was over 16% above the identical quarter in 2022.

VYONDYS 53 web product income was $31.7 million, 3.4% above Q3 of 2022. And AMONDYS 45generated web product income of $66.3 million in Q3 of 2023. This represents roughly 21% development over Q3 of 2022.

As we talked about in earlier calls, we’re within the mature section of the market now for all three of our accepted PMOs. Because of this, whereas we count on the U.S. development to proceed to flatten and the ex-U.S. revenues whereas nonetheless within the development section to stay lumpy from quarter-to-quarter and thus troublesome to venture on a quarterly foundation.

Taken collectively, we are able to reiterate our annual steering of larger than $925 million in web product income for our PMO enterprise in 2023. I will finish by saying that I have been frequently amazed and impressed by the resilience, dedication, and execution of our Sarepta groups over time. And whereas the success over these years has been spectacular, what the groups have achieved within the third quarter of 2023 stands above and past something I’ve seen in my 10 years working to serve the Duchenne neighborhood. The longer term is vivid for Sarepta and for the Duchenne neighborhood who’ve been ready for and really a lot deserve this progress. Phrases cannot adequately specific simply how proud I’m of our complete crew. The person tales from throughout the nation are too quite a few to say right here, nor can we as a crew put into phrases the enjoyment we really feel after we rejoice every affected person who positive aspects entry to any of our Dystrophin restoration therapies.

And so with that, I will flip the decision over to Dr. Louise Rodino-Klapac. Louise?

Louise Rodino-Klapac

Thanks, Dallan. Our dedication to the science stays steadfast and our objective to vary the lives of sufferers with uncommon illness is unwavering. Our alternative to do good is limitless for these dwelling with Duchenne, limb girdle, and lots of different ailments for which therapies are both insufficient or non-existent.

As Doug has already detailed the EMBARK outcomes, I will focus my feedback on the progress of our gene remedy and RNA applications. First, limb girdle muscular dystrophy or LGMD. We stay dedicated to advancing our LGMD portfolio throughout quite a lot of subtypes and stay up for offering steady updates on these vital applications within the months forward.

We offered on our LGMD pipeline this previous weekend on the Communicate Basis’s 2023 Worldwide LGMD Convention and shared our urgency with the neighborhood to deliver forth genetic medicines for LGMD. To start, we made wonderful progress for Voyagene, our Section 1 research evaluating SRP-9003 for the remedy of limb girdle muscular dystrophy Sort 2E, in ambulant grownup sufferers and non-ambulant sufferers utilizing scientific course of SRP-9003 materials. We’re happy to report that we accomplished enrollment in Voyagene and we stay on monitor to provoke our section 3 research utilizing commercially consultant course of materials later this yr.

Mixed with optimistic expression and practical knowledge shared from our preliminary research, SRP 9003-101, we imagine the information from Voyagene will give us insights right into a broader affected person inhabitants. We’re additionally excited to report that we accomplished dosing in our systemic pilot research, NAVIGENE for SRP-6004 twin vector RH74 mediated gene remedy, to deal with people with LGMD2B. LGMD2B is characterised by the absence of the protein Dysferlin. The revolutionary twin vector technique permits us to ship the complete size of Dysferlin, the only reason behind LGMD2B. We stay up for reporting outcomes from this research within the first half of 2024.

As talked about final quarter, our LGMD pure historical past research of the Sarcoglycanopathy, LGMD2E, 2C and 2D, referred to as JOURNEY, has been totally enrolled and we have adopted sufferers for 36 months. We proceed to make progress in scalable manufacturing for all of our LGMD candidates in our pipeline and stay up for initiating scientific research as quickly as doable.

Turning now to the progress we have made with our RNA platform. We had been happy to finish enrollment within the first quarter of 2023 for a momentum research for SRP-5051. And we’re focusing on readout of the research in 2023. Concerning our post-marketing research for the PMOs, as talked about final quarter, we accomplished enrollment within the ESSANCE trial, or publish advertising and marketing necessities for golodirsen and casimersen. As a reminder, ESSANCE is a two-year research and is because of learn out in early 2026.

Lastly, we had been happy to have accomplished enrollment in our MIS51ON research, our dose ranging post-marketing dedication for EXONDYS.

MIS51ON is a randomized double-blind security and efficacy dose discovering research evaluating the accepted dosage of Eteplirsen, a 30 mg per kg weekly, to a dosage that gives considerably larger publicity, as much as 200 mg/per kg weekly.

MIS51ON is a two-part section 3 research that was totally enrolled in October, 2023 with 160 sufferers enrolled. We stay dedicated to quickly and diligently advancing MIS51ON and sharing knowledge as quickly because it turns into out there. We stay up for reporting continued progress with our RNA applications within the coming months.

On a private notice, my ardour for science and its promise to assist others started early in life. These a few years later, as I mirror on my profession and the place we’re in the present day in realizing the promise of genetic medication, I am grateful and I am humbled. And but we now have a lot extra to do. We transfer ahead from right here in the present day towards a extra promising future for people around the globe dwelling with uncommon illness.

In closing, I would prefer to take a second to thank the Sarepta crew for his or her continued dedication and keenness to sufferers, the science and our mission.

I will now flip the decision over to Ian for an replace on our monetary outcomes. Ian?

Ian Estepan

Effectively mentioned, Louise. Good afternoon everybody. This afternoon’s monetary outcomes press launch supplied particulars for the third quarter of 2023 on a non-GAAP foundation in addition to a GAAP foundation. Please consult with our press launch out there on Sarepta’s web site for full reconciliation of GAAP to non-GAAP monetary outcomes.

We’re clearly fairly happy with the monetary outcomes for this quarter on the again of an amazing begin for the EXONDYS launch. We really achieved non-GAAP profitability and assuming an enlargement to the label to the broader Duchenne inhabitants, we must always obtain sustained profitability.

We’re fairly thrilled to realize this milestone simply within the first quarter of the launch. For the three months ended September thirtieth, 2023, the corporate recorded complete revenues of $331.8 million, which consists of web product revenues and collaboration revenues in comparison with revenues of $230.3 million for a similar interval of 2022, a rise of $101.5 million.

Web product income for the third quarter of 2023 from ELEVIDYS was $69.1 million. Web product income for a similar interval from our exon skipping franchise was $240.2 million in comparison with $207.8 million for a similar interval of 2022.

For the quarter, particular person web product gross sales had been $142.3 million for EXONDYS 51, $66.3 million for AMONDYS 45, and $31.7 million for VYONDYS 53. The rise in web product income primarily displays growing demand for our PMO merchandise, in addition to web product income related to the gross sales of ELEVIDYS.

In every of the quarters ended September thirtieth, 2023, and 2022, we acknowledged $22.5 million of collaboration income, which pertains to our collaboration association with Roche. The reimbursement of co-development prices underneath the Roche settlement totaled $34.9 million for the third quarter of 2023 in comparison with $22 million for a similar interval of 2022.

On a GAAP foundation, we reported a web lack of $40.9 million, or $0.46 per primary and diluted share, and $257.7 million, or $2.94 per primary and diluted share, for the third quarter of 2023 and 2022, respectively. We reported a non-GAAP web revenue of $37.7 million, or $0.37 per diluted share within the third quarter of 2023, in comparison with a non-GAAP web lack of $70 million, or $0.80 per diluted share within the third quarter of 2022.

Within the third quarter of 2023, we recorded roughly $37 million in the price of gross sales in comparison with $40 million for a similar interval of 2022. The lower in price of sale primarily displays write-off for sure batches of our PMO merchandise not assembly our high quality specs within the three months ended September thirtieth, 2022, with no comparable exercise for a similar interval of 2023, partially offset by growing demand for our PMO merchandise.

On a GAAP foundation, we recorded $194.3 million and $216.7 million in R&D bills for the third quarter of 2023 and 2022, respectively, a year-over-year lower of $22.4 million. The lower is primarily attributable to a lower in our manufacturing bills, partially offset by will increase in scientific trial bills. On a non-GAAP foundation, R&D bills had been $163.9 million for the third quarter of 2023, in comparison with $193.7 million for a similar interval of 2022, a lower of $29.8 million.

Now turning to SG&A, on a GAAP foundation, we recorded roughly $120.9 million and $104.8 million of bills for the third quarter of 2023 and 2022, respectively, a rise of $16.1 million. The rise was pushed primarily by a rise in skilled companies and compensation and different personnel bills, partially offset by a lower in stock-based compensation.

On a non-GAAP foundation, the SG&A bills had been $92.8 million for the third quarter of 2023, in comparison with $66.8 million for a similar interval of 2022, a rise of $26 million. On a GAAP foundation, we recorded $12.3 million in different expense web for the third quarter of 2023, in comparison with $400,000 in different revenue web for a similar interval of 2022. The change is primarily as a result of impairment of our funding and loss on contingent consideration, that partially offset by will increase in enhance of an funding low cost web and curiosity revenue as a result of funding mixture of our funding portfolio, in addition to reductions of curiosity expense incurred because of the compensation of our December 2019 time period mortgage throughout 2022.

We had roughly $1.8 billion in money, money equivalents, investments, and long-term restricted money as of September 30, 2023. We’re clearly happy with the quantity of capital on our steadiness sheet, however in turbulent markets, we all know money turns into much more beneficial. We frequently consider our bills. That mentioned, primarily based on the EMBARK outcomes and the knowledge we now have in the present day, there is not any higher use of our money than to construct stock to serve these with DMD.

With that, I will flip the decision over to Doug to start out Q&A. Doug?

Douglas Ingram

Thanks very a lot, Ian. Michelle, let’s open the decision for questions.

Query-and-Reply Session

Operator

Thanks. [Operator Instructions] The primary query comes from Anupam Rama with JPMorgan. Your line is open.

Anupam Rama

Hey, guys. Thanks a lot for taking the query. Simply going again to the EMBARK outcomes, we have gotten this query. In the event you guys regulate for multiplicity in your key practical secondary endpoints, would you continue to have statistically important outcomes on these key measures? Thanks a lot.

Douglas Ingram

Sure, thanks for that query, Anupam. I’ll flip this name over to Louise.

Louise Rodino-Klapac

Thanks. We really carried out a world statistical take a look at, and we did this to do exactly that, to check for multiplicity and present that the secondaries didn’t hit important simply by likelihood. So, we basically examined NSAA mixed with the secondaries and confirmed that they had been, in actual fact, statistically important. So this can be a quantitative technique to take a look at the totality of proof in respect. So, in case you consider the forest pot that we confirmed on our name, that is basically a statistical take a look at to point out that collectively we see that these are statistically important.

Operator

Please stand by for the subsequent query. The following query comes from Tazeen Ahmad with Financial institution of America Securities. Your line is open.

Tazeen Ahmad

Hello, guys. Good afternoon. Thanks a lot for taking my query. For me, I feel some of the common inbounds I have been getting within the final two days is simply making an attempt to triangulate timing. So, Doug, to the extent which you could present us coloration with what steps are concerned subsequent in an effort to full your submitting, and is there any sort of precedent for a submitting like this on the time it might take the company to evaluation an utility? May or not it’s this calendar yr that this might all be full, or wouldn’t it be one thing extra conventional like a six-month evaluation, for instance? Thanks.

Douglas Ingram

Tazeen, thanks in your query, and let me preface my query by saying I am in all probability going to frustrate you by not giving arduous and quick timelines, apart from we’ll transfer quickly and we now have a dedication from the division to maneuver quickly as effectively. Our objective is to file a BLA complement. I imagine it is an efficacy complement, and we’ll try this very quickly. The crew is engaged on it proper now. I do not need to decide to the precise date, however very, very quickly that shall be submitted. I feel historically the company could have six months to evaluation. I don’t imagine. I imagine the company is dedicated to shifting as quick as in all fairness doable to evaluation this, and there’s precedent for this. You see this in different areas like oncology on a regular basis the place you will get for one thing like this very quick turnaround. And naturally keep in mind this is not a BLA however a BLA complement so the inquiry whereas terribly vital is targeted.

And that focus is on the elemental query does the totality of the proof justify the conclusion {that a} ELEVIDYS is bringing a greater life to those sufferers and naturally we imagine that it does. The usual for that is fairly clear. It is substantial proof wanting on the totality of proof. The statute on that is fairly clear. I apologize I do not know what that music is. I promise I am not taking part in a guitar proper now. The statute says it is very clear. Can one pretty and responsibly conclude that the remedy may have the impact it purports to have and the laws are additionally notably clear that for life-threatening and severely debilitating diseases one’s life will be shed particularly the place no passable different remedy exists.

The FDA has decided that it’s acceptable to train the broadest flexibility in making use of the statutory requirements. And as Louise simply identified to all people not solely are the proof on complete very compelling that ELEVIDYS is arresting the decline in these sufferers however in case you do the precise statistics and look throughout the first and our practical secondaries you possibly can see statistically adjusting for any danger of a false optimistic, adjusting for multiplicities that’s powerfully statistically important.

So all of which is to say winding again to the unique query Tazeen that we’ll submit a BLA complement very quickly. The company is dedicated to working with us very quickly and whereas I will not give an actual timeline, I’m assured that we’ll transfer shortly to evaluation this and if profitable broaden this label.

Tazeen Ahmad

Okay, thanks Doug.

Operator

Please stand by for the subsequent query. The following query comes from Gena Wang with Barclays. Your line is open.

Gena Wang

Thanks for taking my questions. Perhaps simply observe this remark. Doug, will you announce when BLA efficacy complement was accepted and within the knowledge package deal are you able to lay out what sort of knowledge shall be included along with what you shared with us? Would that even be like say the protein correlation of the protein stage versus operate? Will these knowledge even be included within the package deal?

Douglas Ingram

There shall be definitely extra within the package deal after which the highest line we’re nonetheless evaluating different data together with as an example protein and different issues, CK and the like. However clearly the main focus of the evaluation goes to be in the beginning the efficacy and the security for this remedy. After which in fact it is all going to be evaluated in relation to our request to broaden this label by eradicating age limitations and ambulation limitations on that. I do not suppose we have made any closing choices about what we’re asserting throughout this course of however clearly typically talking we are typically fairly clear with of us.

Gena Wang

Thanks.

Operator

Please stand by for the subsequent query. The following query comes from Colin Bristow with UBS. Your line is open.

Colin Bristow

Hey, good afternoon and congrats on the spectacular ELEVIDYS gross sales. Perhaps one other one on the EMBARK knowledge. Are you able to communicate to the interpatient variability you noticed in ELEVIDYS handled sufferers? A form of query we have been getting is had been the optimistic outcomes pushed by a small group of excessive responders? And perhaps in case you might additionally touch upon how this variability in comparison with the section 2 expertise that will be useful. Thanks.

Douglas Ingram

Positive. I’ll flip this over to Louise to reply particularly. However I might typically notice that the optimistic outcomes, the P worth on these optimistic outcomes, they are not shut. On the time to rise it is .002. On the ten meter stroll run it is .004. On the worldwide statistical evaluation when one seems on the major and all of the secondaries collectively it is .004. So it is very, very highly effective. However Louise, maybe you need to reply extra particularly on among the variability points.

Louise Rodino-Klapac

Sure. Usually we didn’t see variability, excessive variability amongst the sufferers. The usual deviation was both at or beneath what we anticipated from our earlier research that we used to energy and EMBARK. So there we didn’t see any excessive variability.

Colin Bristow

Thanks. That is useful.

Operator

Please stand by for the subsequent query. The following query comes from Reed Forsyth [Ph] with Guggenheim. Your line is open.

Unidentified Analyst

Hello, that is Rye on for Debjit. Did the highest line knowledge supplied to the FDA from EMBARK embrace data on…?

Douglas Ingram

I’m very sorry, however I used to be unable to listen to that reply, that query. Are you able to ask it once more, maybe?

Unidentified Analyst

Sure. That is Rye on Debjit. Did the highest line knowledge supplied to the FDA on EMBARK embrace data on…?

Douglas Ingram

Goodness, I’m so sorry, Rye. I heard the start, however I do not hear the query itself. I am very apologetic.

Unidentified Firm Consultant

I feel he requested if it included knowledge on microdystrophin expression.

Douglas Ingram

Oh, effectively, I think about that we’ll have that knowledge out there throughout the evaluation course of with the FDA. As Louise mentioned, the expression we’re seeing is within the hunted vary of what we would usually seen. So there’s nothing uncommon about it. In truth, the P worth on it’s .0, many zeros. It’s extremely strongly, robustly, as you’d count on, the restrict is robustly combined. That is the query, although.

Unidentified Analyst

That helps. Thanks.

Douglas Ingram

Thanks a lot, Rye. Apologies, I could not hear you. I am sorry about that.

Operator

Please stand by for the subsequent query. The following query comes from Brian Skorney with Baird. Your line is open.

Brian Skorney

Good afternoon. Thanks for taking the query. I suppose it wasn’t one thing we checked out very carefully earlier than, however I am beginning to inform, I feel, time to rise had a non-significant distinction in favor and remedy, however not that completely different by way of magnitude. I feel perhaps just below half a second distinction. You possibly can inform me if that is proper or not. So I do know you have talked extensively in regards to the baseline imbalance concern right here, and it appears notably acute within the case of baseline time to rise, the place the energetic arm was 5.1 at baseline and placebo was 3.6.

So I used to be simply questioning in case you’ve gone again and checked out making changes for baseline imbalances to guage the time to rise variations in research 102, and specifically, if the 4 to five-year-old subgroup seems the identical in 102 because it did in EMBARK.

Douglas Ingram

Sure, thanks for that query, Brian. I will flip this to Louise to reply.

Louise Rodino-Klapac

I’ll communicate typically, as a result of what we did was take the inclusion standards that we have generated for EMBARK and utilized it to our earlier knowledge after we in contrast it to the exterior management. And what we discovered there’s a distinction if you exclude these sufferers that will have been excluded by that standards in 102, the place you had these speedy decliners. So in that case, we noticed a extra important distinction, however the particular numbers are escaping again proper now. However we did try this evaluation the place we sort of utilized the identical exclusion standards and did see a distinction.

Brian Skorney

Nice. Thanks.

Douglas Ingram

Sure. And perhaps only one factor so as to add in that evaluation to Louise’s level, we noticed a very good consistency between what we noticed in 102 and what we noticed in 301.

Operator

Please stand by for the subsequent query. The following query comes from Salveen Richter with Goldman Sachs. Your line is open.

Salveen Richter

Good afternoon. Thanks for taking my query. With regard to the regulatory submission, are there formal or regulatory procedures concerned with revisiting an accelerated approval after the first endpoint fails and in a confirmatory trial right here? After which simply any preliminary suggestions in your knowledge from payers and the way that may influence the prevailing label or from physicians with regard to how they give thought to use in sufferers right here?

Douglas Ingram

Once more, on the primary query, I am not a 100% assured I perceive the query. Let me be very clear, the usual for affirmation of an accelerated approval is wanting on the totality of proof and figuring out whether or not the advantages of that remedy have been confirmed by the whole knowledge set, not simply the confirmatory knowledge, however the entire surrounding proof that will exist, together with different research.

And I might strongly argue that not solely EMBARK, however the entire supporting proof as effectively, has strongly confirmed the advantages of this remedy. So I feel we’re in superb form there. The main target of our evaluation with this division goes to be on the breadth of the enlargement of this label. That, I’m fairly assured, goes to be the evaluation focus. And as pertains to that, as you understand, our robust view is that having confirmed these outcomes, having confirmed them throughout sufferers and looking out on the totality of those proof and looking out on the forest plot for instance, and looking out on the statistical evaluation of the forest plot adjusted for multiplicities, it’s fairly clear that this remedy is arresting the decline in these sufferers and deserves to be made out there to the sufferers with out limitation to age or synthetic restrictions round angulation.

Because it pertains to payers, that is further proof in our armamentarium with payers. Issues have gone very effectively. Dallan and his crew, Medical Affairs, our industrial, our subject power, entry to reimbursement and the like have simply achieved a superb job supporting the launch of the ELEVIDYS. And I hope everybody will agree with me that it reveals in our efficiency this quarter and this bolsters the dialogue that the crew can have.

Now we can provide for instance. We now have a very highly effective metric that it is compelling on the velocity with which one you set a equipment on remedy. In order I’ll remind you on the time to rise, not solely is the P worth, I feel, 0.002, if I’m not mistaken — however in time to rise is the only best prognosticator of lack of ambulation and an increase time above 5 seconds, as we have talked about typically and is within the literature robustly is the only best predictor of early lack of ambulation. And EMBARK has proven that utilizing ELEVIDYS reduces the chances of that occurring in a 52-week interval by over 90%.

So this gives an extra compelling level with payers who, frankly up to now have achieved a very good job of offering entry. This gives further proof that it truly is vital to get children on this remedy as quickly as doable. And I might argue wanting ahead to label enlargement and is a compelling argument for why this remedy ought to be — this label ought to be expanded as quickly as doable as effectively. So everybody has entry to it as effectively.

Operator

Please standby for the subsequent query. The following query comes from Uy Ear with Mizuho. Your line is open.

Uy Ear

Hey guys thanks for taking the query. Congrats on the good quarter for ELEVIDYS. So I suppose my first query is, had been these — the sufferers with dose had been they primarily those that had been just about anticipated the approval the accelerated approval, they usually get them extra just like the bolus. And like what number of would you be capable of share what number of sufferers are ready to be dosed within the coming quarter? And simply proceed on this theme. Now that the EMBARK knowledge has learn out, do you’ve gotten any sense of any shift in sufferers receptivity to the product in any respect? I do know its early days. Thanks.

Douglas Ingram

Sure. Let me reply these questions shortly. First, I do not suppose there shall be any shift within the want for this remedy. I feel apart from sufferers that aren’t within the dad and mom of sufferers that aren’t within the 4 to five year-old vary I feel are in all probability much more compelled to need this remedy and never going to attend impatiently as they need to be in affected person to have this label broadened.

We do not share affected person numbers. We’ll use as our metric for fulfillment and the measure of our success on web product income. As we mentioned, on the difficulty of form of bolus or warehousing of sufferers, there have been definitely some variety of sufferers that physicians had been getting collectively and gathering to make sure that they may preferentially get dosed earlier than they aged out as a result of the label, clearly, as you understand, restricts the dosing to six and beneath 6% and 5%.

However we have had a gradual stream of recent begin types for the reason that approval. And so wanting ahead, we now have a gradual stream of begin types which might be working via the method. And one different factor individuals have been requested, however I will say it achieved the crew, Dallan and others have achieved a superb job on website activation. I feel our objective, as you understand, our objective and aspirationally been to have 50 websites able to infuse at launch. After which very aspirationally, we thought maybe by the top of the yr, someday subsequent yr, we are able to get all the way in which to 70 websites, however we’re at 70 in the present day.

So the crew has simply achieved a superb soar making — getting websites prepared and up and operating and there’s a regular stream of begin types to work via.

Uy Ear

Thanks.

Operator

Please standby for the subsequent query. The following query comes from Danielle Brill with Raymond James. Your line is open.

Danielle Brill

Hello guys, good afternoon. Thanks a lot for the query. Doug, so we spent a variety of time discussing EMBARK efficacy at knowledge I would like to change over to security. I recall myocardis occasions being mentioned on the AdCom, together with one occasion that had occurred in EMBARK on the time. Simply curious if there are any further security occasions of this nature that occurred within the research or every other SAEs resulting in hospitalization? Thanks.

Douglas Ingram

Thanks in your query, Danielle. I’ll flip the decision over to Louise to reply.

Louise Rodino-Klapac

Sure, we didn’t see any variations within the forms of SAEs or the frequency of the SAEs that was some of the reassuring issues EMBARK was the continued security profile and now taken collectively the entire earlier trials, we now have a big security knowledge base that is constant on these trials.

Operator

Please standby for the subsequent query. The following query comes from Ritu Baral with TD Cowen. Your line is open.

Ritu Baral

Good afternoon everybody. Thanks for taking the query. Doug, might you — and perhaps, Dallan, stroll us via the way you see affected person numbers for the completely different situations which might be regulatory specialists counsel might are in play basically for label enlargement. You talked about 10% of your PMO gross sales are for 45 — these are coated — however how would you phase it by perhaps 6 to 7 year-olds if FDA needs to go down that route once more? And the way that quantity would change you do acquired the complete ambulatory inhabitants, which per my calls now extends to love 12 years of age.

Douglas Ingram

For ambulatory?

Ritu Baral

Sure. Like that is the typical age of lack of ambulation now 12 13.

Douglas Ingram

That could be appropriate. I’ll have Dallan verify that in a second. Broadly talking, I imply, look, to start with, I need to be very clear, we’re not in search of a label enlargement to go from 4.5 to 4 to 7, and we do not suppose there’s any motive scientifically one could be restricted to 4 to 7, given the information.

And we have by no means seen that earlier than. As soon as — you have by no means seen in every other label for Duchenne remedy, these types of age-related restrictions. However answering a broadest query, the all ambulatory versus all ambulatory and non-ambulatory is about 50%. So there’s — so the ambulatory inhabitants is about half of all sufferers and the non-ambulatory the opposite half, in fact, — and in order that, to our thoughts, is the massive minimize. However Dallan, is there anything you’d prefer to say on this subject.

Dallan Murray

No, I feel you coated it, Doug. Our objective is to focus on all of the sufferers that we are able to get in the whole inhabitants, learn you to your query about common age of lack of ambulation. I feel you are in the correct vary. However the KOLs have been speaking quite a bit in regards to the definition of lack of ambulation too as a result of, as you understand, there’s heterogeneity. So you’ve gotten some sufferers strolling at a lot older ages, and we now have an entire cohort of sufferers who’ve been handled with the PMOs for years now which might be going to, we imagine, have older ages of lack of ambulation as effectively.

So it will not be — we imagine the ambulatory inhabitants will not be outlined by age. However as Doug mentioned, our aspiration is a broad label and focusing on the entire sufferers who shall be eligible.

Ritu Baral

And is that fifty% of your PMO gross sales too?

Douglas Ingram

Sure. The ambulatory versus non-ambulatory cut up, I feel it is in that vary of fifty%. I feel the entry is harder within the older sufferers. So on common, we have got the next penetration within the youthful given inhabitants.

Ritu Baral

Nice. Thanks.

Operator

Please standby for the subsequent query. The following query comes from Mike Ulz with Morgan Stanley. Your line is open.

Mike Ulz

Good afternoon and thanks for taking the query. You talked about now that you’ve about 70 websites are energetic. Are you able to give us a way of what proportion of these websites have really prescribed ELEVIDYS up to now? Thanks.

Douglas Ingram

We’re going to — apologies I’ll frustrate you. We’re going to withstand that stage of element both on numbers of web sites are infused or in all probability extra particularly variety of sufferers infused at anyone time. As we now have achieved for the final seven years, and I feel we have achieved typically over time to good success with of us. We use web product income is the marker for fulfillment and uptake and the like. So apologies for that, Mike.

Operator

Please standby for the subsequent query. The following query comes from Tim Lugo with William Blair. Your line is open.

Tim Lugo

Thanks for the query. You talked about earlier within the week that you just did not count on an extra advisory committee for broadening the label. However we are able to all keep in mind after we did count on one for the accelerated approval dialogue. I suppose why not request an AdCom? It looks like listening to the AdCom, the contributors had been rather more amenable to non-age-restricted approval than the company was.

Douglas Ingram

Sure. Okay. So it is a good query, and I am not stunned by the remark. There are those who would possibly say that I’ve a poor historic monitor document of predicting AdComs. However the issue the final time I mentioned we had been going to have AdCom, I did have that in writing. The rationale that we do not imagine we’ll have an AdCom is that we do not imagine that we are going to want one. And I feel that — I imagine, as we sit right here in the present day, that, that could be a view that will be shared by us and FDA management.

As we have mentioned earlier than, — we had a really productive and inspiring dialogue with the FDA management on the information and on the potential for submitting it for a broad label. And I might additionally notice that the company has modified the division, specifically, has gone via some fairly important adjustments over the course of this yr. There’s been a reorganization simply to remind you the place OTAT has been changed by the tremendous workplace OTP.

And never too way back, a brand new chief, Dr. Nicole Verdun, took the helm because the Head of OTP. So I might say had been to not do not forget that the division is evolving. Clearly, within the occasion that there was an advisory committee, we’d be effectively ready for it. And I imagine we’d carry out exceptionally effectively there. I feel Dr. Rodino-Klapac and crew simply did a superb job representing us. And as one could recall, we did finally win that AdCom. However as we sit right here in the present day, we really feel fairly assured that we are able to get a little bit enlargement with out an advisory committee.

Tim Lugo

Alright. Thanks.

Douglas Ingram

Thanks very a lot.

Operator

Please standby for the subsequent query. The following query comes from Gil Blum with Needham & Firm. Your line is open.

Gil Blum

Good afternoon and congratulations on all of the progress. Going again to a query, a follow-up on Alan’s [Ph] earlier questions on any questions in the past. So if you’re on Research 102. Is there — any likelihood that there could be some follow-up, particularly on the sufferers that had been older and had been crossed over partly 2 of Research 102 concerning time to rise. It is going to be fascinating to see how that knowledge seems compared to the EMBARK knowledge. Thanks.

Douglas Ingram

Positive. Louise, any ideas on that?

Louise Rodino-Klapac

Okay. Though one or two sufferers are continued to observe for as much as 5 years. In order that’s definitely one thing that we are able to take a look at over time. We do not have that knowledge at hand, so we are able to take a look at that.

Operator

Please standby for the subsequent query. The following query comes from Brian Abrahams with RBC Capital. Your line is open.

Brian Abrahams

Hello there, congrats on the robust first full quarter of the ELEVIDYS launch. Thanks for taking my query. Are you able to remind us of the protocols in place in EMBARK to guard in opposition to practical unblinding — was this a subject that ever got here up with the FDA in your current discussions? And why you count on any variations within the impact practical and blinding, if there was any, it might need on time assessments versus on NSAA? Thanks.

Douglas Ingram

Sure. I can enable Louise to debate the protocol points of the blinding course of, which was exceptionally rigorous. We will typically assume that if that these are very goal time take a look at could be much less topic to any sort of affect within the occasion there wasn’t unblinding. However I might say additionally that I feel the protocol was superb in regards to the blinding course of and the research itself, one ought to keep in mind, was really very effectively run. I need to be clear about that. Louise any ideas on the blinding course of.

Louise Rodino-Klapac

Simply particularly so the sufferers and caregivers clearly blinded the PIs in addition to the bodily therapist doing the practical assessments are all fully blinded. So the research is preserve blinded, research employees disrupt is blinded. It is maintained by a 3rd celebration. So there’s a rigorous course of in place to guarantee that the blinded stays intact.

Brian Abrahams

That’s useful. Thanks.

Operator

Please standby for the subsequent query. The following query comes from Kristen Kluska with Cantor Fitzgerald. Your line is open.

Jason Bouvier

Hello, good afternoon. That is Jason Bouvier on for Kristen Kluska. Thanks for taking my query and congrats on the robust quarter for ELEVIDYS. One query from us, the cadence treating sufferers goes sooner than the unique time traces you laid out. So we’re simply questioning what the most important drivers are there? And the way would possibly this additionally influence the doubtless broader launch subsequent yr? Thanks.

Douglas Ingram

Effectively, I’ll take the query despite the fact that Dallan needs to be, as a result of I need to brag about our crew. I imply I feel there’s two important explanation why the cadence of this launch goes exceptionally effectively and why this launch is it, from my perspective, an unprecedented success in gene remedy, the primary of which, in fact, is the remedy itself.

ELEVIDYS is an awfully wanted remedy that sufferers who’ve been on it and households that share their expertise with strongly of the assumption that they want this remedy, and these children have been stabilizing or doing issues age-specific that untreated children have not been capable of do.

After which in case you do not thoughts me bragging a little bit bit in regards to the crew. I imply that is an instance of exceptionally nice execution by the Sarepta of us led by Dallan — Buyer Officer, by going past that, that is our manufacturing and distribution of us as effectively, simply a big crew effort to execute on this. And this is not new for us. I need to remind everybody now that we now have now 4 therapies that we now have launched. Each a type of therapies and their launches have gone exceptionally effectively.

I imply if we take a look at the P&L, it is simply digress for a second. I imply we are actually from our first PMO that was accepted in late 2016. We’re nonetheless rising. We grew at 16% quarter over the identical quarter final yr, at the same time as we’re launching ELEVIDYS is doing brilliantly there. So I feel there is a mixture each of the good remedy as all of our 4 therapies, I imagine, have been and distinctive centered, granular, well-informed execution.

And what does this imply for the longer term? It signifies that we all know how one can serve the Duchenne neighborhood and one of many issues that excites us a few broader label is we’ll be capable of deliver ELEVIDYS to the vast majority of kids and younger males in the US which might be dwelling with and producing irreparably from this ferocious illness. And so I am actually excited in regards to the alternative to deliver this remedy to extra sufferers at the same time as we’re doing very well with the launch proper now. Thanks very a lot in your query. Thanks very a lot in your query.

Operator

Please standby for the subsequent query. The final query comes from Joseph Schwartz with Leerink Companions. Your line is open.

Joseph Schwartz

Nice. Thanks very a lot for becoming me in. I used to be questioning how are you fascinated about upcoming scientific knowledge for a special gene remedy candidate, which may have an interim look quickly. Is there something that you will be centered on specifically? And the way do you see the trade-off between security and efficacy. If it had been capable of produce a larger influence on FAA, how would that influence your relative worth proposition for ELEVIDYS?

Douglas Ingram

Thanks for the query. Look, as I’ve mentioned many occasions earlier than, we now have in entrance of us an exceptionally ferocious competitor and that Duchenne muscular dystrophy. And all of our focus and all of our vitality is in beating the rattling illness. And I feel this crew is doing a superb job of that. We’re exceptionally happy with the efficiency of ELEVIDYS. There have been some — a few years in the past, individuals made choices about constructs and capsids and the like. And with the advantage of a few years of expertise, we’re exceptionally happy with.

And albeit, nothing lower than thrilled with the actual capsid and assemble that we now have. It is proven not solely, nevertheless it is ready to intervene shield these muscle tissue of those kids and arrested decline nevertheless it couldn’t try this with a very laudable security profile given the quantity of remedy required right here and the truth that it is full physique infusions. RH74 has been a standout. And we’re fairly assured that anybody who’s rational who had a chance to decide in the present day about what capsid they might use and what assemble they might develop. I am certain they might do their greatest to attempt to copy us. So we’re — we’re not centered on any competitor moreover Duchenne muscle dystrophy, and we’re ferociously dedicated to beating this illness. In order that’s our focus proper now. However thanks very a lot in your questions.

Operator

I might now like to show the decision again over to Doug for closing remarks.

Douglas Ingram

Effectively, thanks very a lot, Michelle. And thanks, everybody, for attending tonight, and thanks in your very considerate questions. Let me summarize. This quarter has been an awfully vital one. With our fourth approval, we launched ELEVIDYS and in my view, we launched it brilliantly. We have continued to serve the neighborhood with our PMOs, which proceed strong development even within the face of an ELEVIDYS launch. And on a non-GAAP foundation, we are actually worthwhile, and we’re marching towards a money circulation optimistic within the very close to future. We now have constructed a powerful enduring group that’s centered on two main issues. The primary is serving our affected person neighborhood via good science and the second is executing and getting issues achieved. And that’s exactly what we shall be doing over the approaching months. We intend to proceed our robust efficiency and dedication to serving this neighborhood. We intend to maneuver with velocity to submit our efficacy complement and conclude the evaluation on the broadening of the ELEVIDYS label.

And when our label has been up to date to take away age and ambulation restrictions, we intend to deliver this remedy to the vast majority of sufferers dwelling with Duchenne in the US. And with that, I stay up for updating all of you on our progress alongside the way in which and have a stunning night. Thanks.

Operator

This concludes in the present day’s convention name. Thanks for collaborating. It’s possible you’ll now disconnect.