Sarepta Therapeutics, Inc.

Good day, and thank you for standing by. Welcome to the SOSEPTA Therapeutics Second Quarter 2024 Financial Results Conference Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask the question during the session, you will need to press star 11 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, Please press star 11 again. Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker for today, Mary Jenkins, Associate Director, Investments Relations and Corporate Communications. Please go ahead.

Thank you, Lisa, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the second quarter of 2024. The press release is available on our website at Sarepta.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, Dallin Murray, and Dr. Louise Regina-Claypack. After our formal remarks, we'll open the call for Q&A. I'd like to note that during the call, we've been 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, and any such risks can materially and adversely affect the business, the results of operations, and trading prices for disruptive common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual report on Form 10-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? Thank you, Mary. Good afternoon, everyone, and thank you for joining Sarepta Therapeutics' second quarter 2024 financial results conference call. I joined Sarepta a little over seven years ago for one reason. I believe that Sarepta had the greatest hope of developing medicines that could improve the lives of a majority of Duchenne patients And I wanted to be part of that effort. We've had many accomplishments over the last seven years, successfully launching four life-changing therapies and through an obsessive attention to detail, making all of those launches very successful. We have grown revenue at about 150% TAGR from the beginning of 2017 through the end of 2023. We've built a deep pipeline. We've established ourselves as the leaders in RNA and gene therapy for rare disease. And we have become profitable, and as of this quarter, we are cash flow positive. But it was only in the second quarter of this year that we could finally say that we have reached that objective that brought me and so many of my compatriots to Sarepta. Because it was in June of 2024 that we finally obtained a broad approval to make Alevitus available to over 80% of patients living with and dying from Duchenne muscular dystrophy in the United States. As you are aware, in June of this year, the FDA granted traditional approval for all ambulant patients, four and older, and consistent with every other Duchenne approval in history, also granted approval for the treatment of non-ambulatory patients on an accelerated basis. Also in June, our partner, Roche, announced that the European Medicines Agency had accepted the 11th submission for review. Roche also has obtained six XUS approvals so far and is making good progress serving those communities. So here we are, launching the largest gene therapy yet approved with the first opportunity yet in history to bring a dystrophin restorative therapy to the vast majority of Duchenne patients. Anyone who has been watching over the last seven-plus years will realize that this is exactly what we are particularly good at. Certainly, we are great at developing therapies for rare disease, and we are great at managing the process to get them approved, and we have become exceptional at managing complex manufacturing and distribution. But perhaps above all else, we are second to no one in the world at launching Duchenne therapies, working with payers, and ensuring access. To that point, in the second quarter, we achieved net product revenue of approximately $300 and $61 million. That's a 51% increase over the same quarter last year. Alevitis came in at $121.7 million in the second quarter, in line with our earlier forecast. Even with an exceptionally narrow and restrictive label, in the first full 12 months of launch, we achieved approximately $456 million, more than doubling all other gene therapies launched in the last few years combined. As we have noted previously with the broader label granted in June of this year, the opportunity to serve patients and in so doing reward committed investors will be enormous. Our early launch has exceeded even our optimistic expectations. All signals are currently positive from physician and patient demand to enrollment forms to assay kit ordering to positive payer interaction. To manage near term expectations, let me note again, that the process from enrollment form to infusion will typically take between three and five months. So, we will see moderate revenue growth in the third quarter. But we anticipate strong growth commencing in the fourth quarter of this year. And then exiting this year, 2024 will be a very strong year of growth. We currently anticipate that our 2025 net product revenue across our four approved therapies will come in between $2.9 billion and $3.1 billion, and that is still years away from peak-year sales. Indeed, we will be treating the prevalent population over the course of this entire decade before moving to the incident population in 2030 to 2031. Dallin Murray, our Chief Customer Officer, will provide more color on the launch in just a moment. Our Head of R&D and Chief Scientific Officer, Dr. Luis Rodino-Clayfax, will then discuss our R&D progress from last quarter, including our plans for presenting multiple data sets from across the SRP 9001 clinical program at the World Muscle Society Congress later this year. And she will also provide updates on the advancement of our LGMD programs. I look forward to updating you across the year on our launch performance and the advancement of our pipeline. And with that, I will turn the call to Dallin Murray for more details on commercial performance and plans. Dallin?

Thank you, Doug, and good afternoon. As Doug outlined, we generated roughly $361 million in net product revenues from our Duchenne franchise in the second quarter of 2024. Q2 is the last quarter in which we will have executed on the limited four- to five-year-old label for Levitas, given that we received a broad label on June 20th. As we had expected, the net product revenue for Levitas was essentially flat when compared to each of the two prior quarters. Of note, a few patients rescheduled doses at the end of the second quarter, which resulted in those infusions being completed early in the third quarter. Net product revenues for Levitas were nearly 122 million for the quarter. Net product revenue for the PMO franchise in Q2 was roughly 239 million, which represented Modest growth over Q1. Going forward, the team remains committed to supporting those existing PMO patients who have been remarkably compliant with their weekly PMO regimen for years now. And while we expect some cannibalization by Alevitis in the U.S. in the coming quarters, ex-U.S. PMO growth will help to mitigate this cannibalization. Now turning to the Alevitis launch. We're pleased with the launch progress to date and are on track to realize the opportunity in front of us. To put the current situation into perspective, almost the entire Duchenne population became eligible for Levitas essentially overnight. What we're seeing right now is the key neuromuscular centers reacting to unprecedented demand from the entirety of their Duchenne patient populations. The treating sites are rapidly working through and prioritizing patient demand. We're confident in their ability to manage this, given the fact that these are the same centers who navigated all of the recent Duchenne and SMA launches, including Zolgensma. Your uptake assumptions should reflect the patient's journey to obtain an infused gene therapy. We're only several weeks into this new launch. However, we have some exciting successes to report, which highlight the progress the team has made in the short time we've had with the Levitas label expansion. To begin, one of the first steps in the patient journey, and therefore an important leading indicator of demand, is screening for preexisting antibodies. In the initial hours of the label expansion, we were nearly overwhelmed with requests. assay screening kits, despite our careful preparation, forecasting, and market insights. Our team rapidly adapted and shipped well over 1,000 assay kits in the first weeks of launch. As you would expect from assay kit demand, all of our other indicators show that patient demand is very robust and growing. Our market research indicates that the vast majority of Duchenne patients are already interested in seeking therapy, And we only expect this to grow with time. This demand manifested itself in the early days of the launch with a flood of patients reaching out to our key treatment sites. This has forced each center to determine the best approach to handling the unprecedented demand and plan for how to support their patients in navigating the treatment journey. As such, we're hearing from the community that some patients are presently being advised of long wait times. Enrollment forms have exceeded our own lofty expectations just a few weeks into the launch. ACTs have submitted enrollment forms for patients ranging from 4 to 38 years of age, with an average age right now just over 10. It's also critical to highlight that we have the support of the broader physician community who have gained experience with the levitas over the last year in the four to five-year-old population. Since our initial 2023 approval, we've received enrollment forms for more than 125 HCPs from 46 states, including from 97% of our sites. And we can see early on that the physician community are approaching label expansion as they have our previous DMV launches, prioritizing declining ambulatory patients first, followed closely by the non-ambulatory and newly diagnosed patient populations. Of note, the early days of this gene therapy launch are following the same pattern of all of our previous exon skipping launches. One of the critical success factors in any launch is that the key treatment centers have had hands-on experience with the drug. To that end, over 75% of our sites have dosed at least one patient, and we do expect this to grow now that we have a broad label. And turning to the critical issue of patient access, we've already started to see encouraging signs on the payer policy front. To date, our teams have had productive and engaged meetings with payers representing greater than 225 million patient lives, including the top commercial plans and state Medicaid agencies. We anticipated it will take three to six months for most to establish new medical policies. In the meantime, we are having success with payers reviewing each patient request on a case-by-case basis. And to that end, we already have authorization approvals for both ambulatory and non-ambulatory patients from both commercial and Medicaid payers for patients in the expanded population with ages ranging from 4 to 21 years of age. And I'm also pleased to highlight that as of this call, there are both commercial and Medicaid payers who have established policies covering 11 to the expanded medically accepted indication. And then finally, with multiple authorizations in hand, we already have patients from the expanded population scheduled for dosing, and we expect the first of these to be dosed this week. As a result of all this, and as Doug indicated, we expect to see growth accelerating modestly in the third quarter. What you'll see is a jump in quarter-over-quarter, elevitous revenues of nearly one-third from Q2 to Q3 of this year, followed by a doubling of the 11th revenue from Q3 to Q4. But the remainder of 2024 will only be a prelude to what we'll see in 2025 and beyond when the sites have had the opportunity to fully adapt themselves to a broad label. Our 2025 net product revenue guidance of 2.9 The 3.1 billion for the Duchamp franchise should show you where we are heading with our revenue growth and our confidence around this market potential. And that market potential is absolutely massive. Let me spend a moment painting the picture for how we expect this launch to play out. There are three things which illustrate just how exciting this opportunity truly is from a commercial perspective. First and foremost, with no near-term gene therapy competition, we expect to treat the eligible prevalent Duchenne population in collaboration with the key centers over the remainder of this decade. Secondly, our track record over the past eight years should provide you all reassurance of our ability to secure access for Duchenne patients in a broad label setting. While it may take time for some patients We will ultimately secure access to the vast majority of Duchenne patients who are eligible. Lastly, and very importantly, the 11th one-time dose for each patient is entirely upfront. And along with that, so is the revenue. I don't think this fact has been fully appreciated outside of Sarepta. And this is what I was describing earlier. after that early adjustment period in the back half of this year, we expect the revenue to take off robustly. We've been planning for this opportunity for many years. We've anticipated the challenges and have now had a full year to learn and evolve with a team that was originally built for the full Duchenne population. What I'm most proud of is that patient safety is at the center of everything we do. We are taking a thoughtful approach and integrating education and support proactively into all of our initiatives, enabling a fully informed discussion between doctor and patient is both the right thing to do and sets everyone up for long-term success. As with any launch, we'll see our share of ups and downs. It will take time for us in the centers to work through the demand that we're seeing today and there may be some frustration as we do our best to support these deserving patients. As we demonstrated in our previous launches, there is no team in the rare disease, and I would even argue in the entire biopharma landscape who could execute better on such an incredible opportunity is the one we have in front of us right now. So, in summary, the interest and demand from all stakeholders is strong as we had anticipated. We expect this demand will only increase with time. Obviously, all of the patients who are seeking treatment right now cannot be accommodated by the system immediately. We in the physician community are working as hard as we can to support all of the patients seeking treatment. But we will support them, and in so doing, realize perhaps the biggest near-term revenue opportunity in all of biotech. I'd like to end by thanking everyone at Sarepta and all of our stakeholders for their role in getting us to this important moment. As we sit here today, the majority of Duchenne patients are eligible for dystrophin restoration therapy, yet the majority, as of today, have not yet received dystrophin restoration therapy. We aim to change that, and we continue to execute with the realization that every minute matters for these patients and their families. Now, with that, I'll hand it over to our head of R&D, Dr. Louise Rodino-Slaypack. Louise.

Thanks, Alan, and good afternoon. I'll begin with my remarks with the approval of the Elevitus label expansion and then provide an update on our pipeline. Following decades of research and development and after a thorough scientific review that critically considered all of the available evidence across multiple studies, the Office of Therapeutic Products of the FDA approved expansion of the Levitas label based on the following. The clinical benefits of Levitas in ambulatory patients was confirmed by our double-blind, placebo-controlled study in BARC and related studies. And based on these data, traditional approval was granted to all ambulatory D-stranding patients ages 4 and older. The mechanism of action of Alevitus is universal, regardless of disease state, as long as muscle is present. As a result, the Alevitus dystrophin expressed by our therapy in non-ambulatory patients is reasonably likely to confirm clinical benefit in this population. As a result, accelerated approval, or AA, has been granted for the treatment of non-ambulatory patients ages 4 and older. The accelerated approval for non-ambulatory patients includes a post-marketing commitment to confirm clinical benefits, which will be addressed via our non-ambulatory and late ambulatory study 303, also known as Envision. As a reminder, Envision is a global, randomized, double-blind, placebo-controlled, two-part study evaluating the safety and efficacy of SRP9001 gene therapy in non-ambulatory and older ambulatory individuals with Duchenne. Envision is progressing well with U.S. enrollment complete and the remaining recruitment occurring ex-U.S. Enrollment is expected to be completed in 2025, with our last patient last visit expected in 2027, following an 18-month placebo-controlled period. We also continue to advance clinical studies that monitor long-term follow-ups with the Levitas. Our long-term follow-up studies include Endure and Expedition. Endure is a Phase 4 observational study that will follow individuals treated with elevatives for up to 10 years. One-third of sites have been activated, and up to 500 patients will be enrolled. In addition, Expedition is a Phase 3 study enrolling approximately 400 patients that were previously enrolled in elevatives clinical trials and followed for consistent safety and efficacy measures for up to five years. Regarding patients currently ineligible to receive Alevitis under the expanded label, we are conducting multiple studies. For the approximately 15% of patients who are screened out for preexisting anti-AEV RH74 antibodies, we've commenced the study to cleave and inactivate antibodies with amlipidase, and we'll shortly begin a separate study to remove antibodies with plasmapheresis. In addition, for patients under the age of four, we have treated patients as young as two in our study 103, and together with our partner, Roche, we are executing study 302 to gain experience nursing patients under four and as young as three months. We will continue to transparently communicate the range of trial experience in patients treated with the Levitis from those that are under four to those with more advanced disease. As of the end of June 2024, We have dosed over 60 late ambulatory and non-ambulatory patients within our clinical program. We have multiple scientific disclosures planned throughout 2024. We look forward to releasing multiple data sets from the Crossbow Levitis Clinical Program at the next available forum, which is the World Muscle Society Congress in early October. Among the presentations accepted are additional embarked data, including muscle MRI and cardiac MRI. Endeavor or study 103, safety and expression data in late ambulatory and non-ambulatory patients, and study 101, five-year longitudinal data. Multiple manuscript submissions are in process or planned, as are plans to release numerous abstracts of additional congresses, including the International Congress on Neuromuscular Diseases, the Academy of Managed Care Pharmacy, and the American Association of Neuromuscular and Electrodiagnostic Medicines. Finally, we are pleased to report that the primary Embark manuscript has been accepted for publication in a high-impact journal and will be available online in the coming months. To summarize the Levitas, we are extremely happy for the Duchenne community on the expansion of our label and the expanded opportunity for those living with Duchenne that wish to receive a Levitas. I'd like to offer a very special thank you to the courageous families who chose to participate in our studies that support this approval. We will continue to diligently serve the community through execution of our ongoing clinical studies and communication of safety and efficacy data on an ongoing basis as it becomes available. Moving now to our programs for the limb girdle muscular dystrophies, or LGMDs, starting with SRP9003. As we mentioned on the first quarter call, dosing is underway in study SRP9003-301. also known as Emergene, are Phase III multinational open-label clinical trial of SRP9003 for the treatment of limb girdle muscular dystrophy type 2E or beta-sarcoglycanopathy. The agreed primary endpoint of Emergene is expression of beta-sarcoglycan, the absence of which is the sole cause of the disease. We are encouraged by the agency's willingness to create a viable pathway for SRP9003 which treats an ultra-rare genetic condition that is progressively debilitating, results in loss of angulation, and leads to early mortality. Assuming a positive pre-BLA meeting, we would anticipate BLA filing in 2025. We are also very pleased to announce that we received fast-track designation for SRP 9003. The fast-track designation is a process designed to facilitate the development and expedited review of drugs that treat serious conditions and fill unmet medical needs. In addition to fast-track, SRP 9003 has also been previously granted rare pediatric disease designation and orphan drug designation from FDA, as well as orphan drug designation from the European Medicines Agency. The ability to progress a small NF15 biomarker study together with our ability to demonstrate delivery of a functional beta-cercaglycan protein is extremely important, not just for this program, but for the other cercaglycanopathies in our pipeline, including LGMG2D and LGMG2C, both of which are progressing to the clinic. Having successfully advanced SRP9003, we will submit our SRP9004 IND update reflecting our suspension process this year, with Phase I initiation expected to start by year's end. As a reminder, SRP 9004 is designed for the treatment of limb girdle muscular dystrophy type 2D, or alpha-circle glycanopathy. Finally, we are also rapidly progressing our program for SRP 9005 for the treatment of limb girdle muscular dystrophy type 2C, or gamma-circle glycanopathy. We plan to engage with FDA in Q4 of this year with plans to initiate a clinical study in Q1 2025, pending the outcome of that meeting. To summarize, we are very pleased with the progress of our LGMD portfolio and expect to have three of our LGMDs in the clinic in less than nine months. We are maximizing the synergies across this platform from both an R&D and manufacturing perspective, and our sights are firmly set on accelerating the remainder of the LGMD assets to the clinic. The subject matter expertise that we've built positions us well to rapidly advance our current and future pipeline. Continuing with our RNA platform and beginning with our PMO, the ESSENCE trial, our post-marketing requirements for Golodursin and Casimersin, as well as mission, our post-marketing commitment for Exondus, are both fully enrolled and remain on track. As a reminder, ESSENCE is a two-year, two-to-one randomized placebo-controlled study of both Golodursin and Casimersin. and is due to read out in early 2026. The study was fully enrolled with 228 patients. MISSION is a randomized double-blind safety and efficacy dose-finding study comparing the approved dosage of teplisin 30 mg per kg weekly to a dosage that provides significantly higher exposure, up to 200 mg per kg weekly. MISSION is a two-part Phase III study. It was fully enrolled with 160 patients. We remain committed to rapidly and diligently advancing missions and sharing data as soon as it becomes available. Turning to PPML. Early in the first quarter, we announced positive results from Part B of our Momentum study, or study SRP 5051-201. Momentum is an ongoing study of SRP 5051, our investigational peptide conjugated PMO, or PPML. We are actively engaging with CDER, regarding the Phase 3 study that could serve as a post-marketing requirement or PMR study for a potential accelerated approval. Agreement on the Phase 3 PMR study is a prerequisite for an accelerated approval filing. Our intent is to gain agreement for a study that considers the current landscape, can be completed in a reasonable timeframe, and reflects patient's reluctance to enroll in a placebo-controlled trial. Our hope is that an agreement can be reached on the trial design this year. In summary, our team looks forward to a bright future ahead as we work diligently to advance our mission with the goal of serving patients around the world living with rare disease. I'd like to take a moment to thank my R&D colleagues for their unwavering devotion to advancing our entire portfolio. They have demonstrated admirable dedication, scientific integrity, and a passion for helping patients that I'm extremely proud of. I'll now turn the call over to Ian Estepan for an update on our financial results. Ian?

Thanks so much, Luis. Good afternoon, everyone. Before I review our financial results, I want to discuss quickly just three topics. First, I just want to add some color on our approach to revenue guidance. We plan to refine the guidance as we get further into the launch, but I think what we've provided today will help everyone model the Levitas launch curve more appropriately. The guidance likely isn't surprising to anyone if the trajectory is consistent with our previous CMO launches. And the guidance also provides some visibility of our expectations and how we expect Elevitus will impact our PMO franchise. It's apparent that we expect modest cannibalization of the PMOs over the upcoming year. So, all in all, the broad takeaway from our guidance is that the Elevitus launch will put us in a very, very strong financial position. In fact, as Doug mentioned, we were actually cash flow positive for the first time in our history. We expect to achieve sustainable cash flow positivity in the middle of next year. So with strong revenue growth reaching almost to the end of the decade, we've had several questions on how we plan to utilize our cash. So I want to briefly talk about how we're thinking about deploying capital in our BD strategy. And before I launch into it, I think it often gets overlooked that we have a proven track record of successful business development. In fact, we wouldn't be here today if we hadn't identified 11 as a differentiated approach to treating BNP and acquired it. We'll continue to leverage our capabilities in both neuromuscular and rare disease space, and this will remain a core area of focus of ours. We have the ability to use propensity match controls to better evaluate efficacy early in studies, which can provide valuable insight in determining the probability of success of the program. Doug also rightly noted that our commercial capabilities are not in rare disease, So, we'll evaluate opportunities to see if we're best positioned to deliver transformative therapies to patients in need. We'll also look at areas that are tangential to record for muscular expertise, such as BNS or cardiovascular, and continue to look to expand our capabilities. Now, with all this being said, and with the growth that we have in front of us, we're well-positioned to transact, but we have no need to do anything imminently. We have the luxury of being able to find assets that squarely fit into our strategy. We'll have substantially more resources to deploy, but we'll continue to use the same financial discipline that has gotten us here today. As we've witnessed over the past week, market dynamics can change quite quickly. Our strong fundamentals should somewhat insulate us from the macro environment and allow us to strategically deploy capital where appropriate. So, finally, in the continued spirit of helping the street model our financials, I wanted to highlight that we've issued a notice to our second supplier to terminate our development and supply agreement related to our adherent manufacturing process for one of our gene therapy programs. As Doug mentioned, we're in a good place from a supply perspective, and based on several factors, including the progress we've made with our suspension manufacturing process, we do not believe that it makes strategic or financial sense to continue to invest in the adherent process for this program. The termination will be effective as of August 21st, and we're currently performing analysis of the financial impact of the termination. As of today, we expect to record approximately $55 to $65 million in additional research and development expenses as a result of the termination for the three and nine months ended September 30th, 2024. This range is net impact of any expected termination expenses, write-offs, and the reduction to operating expenses for applicable reimbursement costs under the Roche Agreement. We're still analyzing any further impact on the termination. That will be recorded three months ended and nine months ended September 30th, 2024. I want to further reiterate that this has no impact on our suspension-based manufacturing agreements. Now, moving to our financials, this afternoon's financial results press release provided details for the second quarter of 2024 on a GAAP basis as well as a non-GAAP basis. Please refer to our press release available on CERECTI's website for full reconciliation of GAAP and non-GAAP financial results. Beginning in the fourth quarter of 2023, amortization of in-life rights and income tax expense are no longer excluded from non-GAAP results. The company has added income tax effective adjustments, which represent the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable effective income tax rate. Non-GAAP financial results for the second quarter of 2023 have been updated to reflect this change for comparability purposes. For the three months ended June 30, 2024, the company recorded total revenues of $362.9 million, which consists of net product revenues and collaboration revenues and other revenues, compared to revenues of $261.2 million for the same period of 2023, an increase of $101.7 million. Net product revenue for the second quarter of 2024 from the Levitas was $121.7 million. Net product revenue for the second quarter of 2024 from our PMO Exxon Skipping franchise was $238.8 million compared to $239 million for the same period of 2023. For the second quarter of 2024, individual net product sales were $129.8 million for Exxon to 51. $77.4 million for Armand is 45, and $31.6 million for Vianda is 53. The increase in net product revenue primarily reflects the net product revenue associated with sales of 11. For the three-month end of June 30, 2024, the company recognized $2.4 million of collaboration and other revenues, which primarily relates to royalty revenue from Roche compared to collaboration revenues of $22.3 million for the same period of 2023-2024. a decrease of $19.9 million. The reimbursable co-development cost of the Roche agreement totaled $17.9 million for the second quarter of 2024, compared to $28.2 million for the same period of 2023. On a GAAP basis, we reported a net income of $6.5 million, or $0.07 per basic and diluted share, and a net loss of $23.9 million, or $0.27 per basic and diluted share, for the second quarter of 2024 and 2023, respectively. We recorded a non-GAAP net income of $46.7 million, or 44 cents per share, in the second quarter of 2024, compared to a non-GAAP net loss of $89.9 million, or $1.01 per diluted share in the second quarter of 2023. In the second quarter of 2024, we recorded approximately $44.5 million in cost of sales, compared to $34.2 million in the same period of 2023, The increase in cost of sales primarily reflects the cost of sales related to Levis during the three-month end of June 30th, 2024, following its FDA approval and launch in June of 2023, with no similar activity for the same period of 2023. On a GAAP basis, we recorded $179.7 million and $241.9 million in R&D expenses for the second quarter of 2024 and 2023, respectively. a year-over-year decrease of $62.2 million. The decrease is primarily due to the capitalization of commercial batches of 11 manufactured actors approval in June of 2023. On a non-GAAP basis, R&D expenses were $153.9 million for the second quarter of 2024 compared to $212.2 million for the same period of 2023, a decrease of $58.3 million. Now, turning to SG&A on a GAAP basis, we recorded approximately $138.8 million and $118.6 million of expenses for the second quarter of 2024 and 2023, respectively, an increase of $20.2 million. The increase was primarily driven by an increase in professional services used for the launch of a levity and ongoing litigation matters, the timing of charitable contributions, as well as the achievement of performance conditions related to certain performance stock units. On a non-GAAP basis, the SG&A expenses were $106 million for the second quarter of 2024 compared to $90.3 million for the same period of 2023, an increase of $15.7 million. On a GAAP basis, we recorded $14.3 million in other income net for the second quarter of 2024, 16.9 million for the same period of 2023. The change is primarily due to a decrease in the accretion of our investment discount due to the investment of our investment portfolio. We have approximately $1.5 billion in cash, cash equivalents, and investments in long-term restricted cash as of June 30th, 2024. And with that, I'll turn the call back over to Doug to start the Q&A. Doug?

Thank you very much. And Lisa, let's open the lines for Q&A.

Thank you. As a reminder, if you would like to ask a question, please press star 11 on your telephone. We also ask that you wait for your name and company to be announced before proceeding with your question. Please keep your questions down to one question. One moment, and we'll be ready for the first question. And our first question today will be coming from Tazine Hamad of Bank of America Securities. Your line is open.

Hi, guys. Good afternoon. Thanks for taking my question. Maybe I just want to get an understanding of what the actual bottleneck here is, because based on everything that you said and based on work that seemingly a lot of folks have done, demand seems to be quite high from patients and physicians. You talked about SART forms and, you know, you're pleased with the number of SART forms, but what is the issue with the timing of getting the patient journey from prescription to actual drug in hand. Has that changed, you know, from the time that you got approved for the four- to five-year roles? When did it start lengthening to what you're saying, what is it, three to six months that it's going to take? Three to five. Three to five months. When did that start? Is that the key bottleneck here? Thanks.

Thank you very much for your question, Susie. The short answer is there really is no bottleneck at all. Now, as I think we said in the last earnings call, it's clearly the case that with the four- to five-year-olds, we were all in – I mean all, not just us, physicians, families, and the payers. We're all in kind of a crisis mode, prioritizing kids that were about to age out of the label, and we're able to do it more rapidly than is normal. But the normal process is about three to five months, and I mean normal – That's not atypical for these sorts of therapies, but it's very typical for exonus, myonus, amonus, and now alevinus. And alevinus has some additional requirements, including, for instance, the requirement that one tests for and is negative for neutralizing antibodies. So to be very clear, there is no bottleneck here. We're doing brilliantly. The start forms are great. Patient and physician demand is great. Manufacturing is great. Everything is going very, very well. Payer interactions are really, really encouraging. I think Dallin mentioned we've been having conversations for quite a long time, but just recently we've had conversations with payers that cover some 225, almost a quarter of a billion lives in the United States, and that's enormous. And they've been very productive, and we've already seen pulled through Good policy. Now, somebody's policy might take some time. So, just we're very clear, it takes about three to five months from start form to infusion. We're getting an enormous number of start forms. We're getting a crazy number of assay kit requirements. We had to literally go into our own mini crisis mode to make sure we could satisfy all of the assays that were necessary. predicate to getting approval. And so this is going to take that amount of time. We're doing brilliantly. The last quarter, Q2, of course, was all predicated on the prior level, and we did really well there. You'll notice that we were basically flat. We would have been perfectly flat, but, you know, a couple of kids that were scheduled toward the back half of the quarter actually had a viral infection and had been pushed into this quarter, and they'll be dosed this quarter, so we're not losing them. And then it's going to take, you know, three to five months. That means that we're going to have nice growth in Q3, but it'll be moderated. And then Q4 will be very strong growth, as we've mentioned. We'll more than double the growth in Q4 of this year. And then as we model right now, based on everything we're seeing, we're going to do between $2.9 billion and $3.1 billion in revenue across the four therapies. next year, which speaks to the success that we believe is happening with Elevitus, and it speaks to the continuing success of our PMOs and the fact that we're seeing fairly modest cannibalization, and we imagine we'll see fairly modest cannibalization in the next year. So the short answer is things are going very well. There is no fundamental bottleneck. It's just a process that one has to go through. We've been dealing with it. for, you know, a very long time. Like, this is what a saundus is like. This is what a biondus is like. This is what a mondus is like. And just, I know I'm getting on a bit of a rant here, but I hope I'll answer all of these questions up front. Just so we're very clear. I mean, we are seeing exactly the sort of rant one would have anticipated. In fact, a little bit more optimistic than even we were. And as you know, we are optimistic folks. And it's very consistent with the launch curves that we've seen with our other three approved therapies. So I will end this soliloquy with the statement I made in the earnings script itself, which is you can say a lot of things about us, but no one should dispute that there is no organization on this planet with more expertise, more ability, and a better track record in launching Duchenne Therapy, serving this community, and making these launches a success. We're in really good shape right now. But thank you very much for your question. I appreciate it.

Thank you. One moment for the next question. And our next question will be coming from . Your line is open.

Hey, guys. Thanks for taking the question. Also, it's KPM. Thanks for providing that 2025 net product guidance, and I think you said multiple times addressing the prevalent population kind of into the 2030-2031 timeframe. Should we be thinking about more linear growth over that time, or how should we be thinking about, say, like a time to peak scenario over the course of the next year?

Yeah, and we've given obvious – thank you very much for your question, Anupam. We've given, obviously, more granular, longer-term guidance than is typical of us, but we wanted to really reflect accurately what we're seeing in this launch. I'm not going to get into a ton more granularity on the long-term forecast other than to say peak-year sales will occur in the back half of this – you know, significantly in the back half of this decade. And as I said before in the script, We'll be treating the prevalent population through the entire decade, and we'll be moving the incident population in the 2030s. Thank you, Bill.

Thank you. And our next question will be coming from Gina Wong of Barclays. Your line is open.

Thank you. I will also ask one more question regarding the revenue. So, did I hear you correctly? 3Q, you still expect modest growth, even, you know, despite you will take three to five months for soft-warm to infusion. So that was the first question, just to confirm. And then the guidance, sorry, I will ask again, guidance 2.9 to 3.1 billion. But when we look at that's in line with first-order analytics, there's a 3 billion 2025 consensus, and they do have the numbers. The sell-side consensus number for 11 days for 2025 is 2 billion. So, do you think that that number is in line with your assumption, and what are the early launch data to support your assumption?

Yeah, thank you very much for your questions, Gina. So, first on Q3, you know, relative to what, you know, I would characterize as the explosive growth that we're going to have as these start forms turn into infusions. In the third quarter, I've said we'll have modest growth, but that's, you know, still 30% quarter over the quarter. immediate preceding quarter. So it'll be very nice, robust growth. But remember, we just got approved the end of Q2. And then it does take about three to five months. So it'll be really kind of back end loaded in the quarter. But we'll see broadly speaking about 30% growth and we'll double it in Q4. And then on 2025, I think we are largely in line with where the external world is, although That wasn't our goal. I think it's a happy accident that we happen to be lined up. And to the question of, you know, sort of what are we seeing? It is early days. So what are we seeing early days that gives us confidence? There's a lot. Just so we're very clear, again, no one has more experience in launching Duchenne therapies than Sarepta. This is our force therapy. In some weird ways, it's our fit therapy because we launched this with a very narrow label of Levitas, and now we're launching it with a broader label of So, a couple things. We know epi extraordinarily well. We know epi down to the state level, so we're very confident on the epi here. We have a lot of confidence around the launch curves itself, and what we're envisioning from the launch curve right now is exactly what we envisioned we would see in all of our modeling, and it is consistent with, again, the launch of Exondus, Amandus, and Biondus. And we have a lot of granular data. Just remember, we have down to patient level data on start forms and the like. We know all of the sites. We just have an enormous amount of visibility. It's still early days. So to Ian's very good point, we will update our views on things as we track to the end of the year into early next year. But, you know, obviously, you know, early days we felt a pretty significant amount of confidence. to provide people with this overall shape of the launch and discuss 2025, which, you know, for us is a bit unusual. We wouldn't provide this kind of level of information this early other than, you know, we wanted to really map out what we're seeing right now and feel like we're being transparent with folks. So, we feel very good about this launch right now.

Thank you. Our next question today will be coming from Joseph Schwartz of LeRinc. Your line is open.

Hi, all. This is Will on for Joe. Thanks for taking our question, and congrats on the progress this quarter. So one from us. For many years, we've seen the company successfully execute on a mission to help boys with DMD. However, this is something that may not be fully appreciated by some in the community. As it appears that the company will continue to invest in next generation therapies and support these patients, why do you think there is a disconnect with some of the community, and what are you doing to actively address it, especially with competition or potential competition on the horizon? Thank you.

Well, I think anybody who's been watching us carefully over the last seven to eight years will know that we've been wholly committed to bringing a better life to the Duchenne community. And we've, you know, at the risk of being a bit modest, we've been pretty brilliant in our ability to do that. Four therapies later, all of them very successful launches, and I believe very transformative, meaningful dystrophin restorative therapies, which we now can – can reach, you know, well over 80% of the Duchenne population. And we're working on actually making it even greater. We are successful with our attempts to knock down pre-existing neutralizing antibody. We have a lot of conviction that we will be. We're already dosing in one of those two studies. The other one will come very soon. You know, we'll be getting to much higher even than 80% of the Duchenne community. And the good news is that I think that those who are informed and have been watching us get this. I think, to be honest, the great bulk of the patient community fully understands and is cheering us along. I can tell you we all stay very close with the community, and the community understands that, and that's why, in addition to cheering us along, people have been watching the data and are very excited and hopeful that they can get on the therapy as soon as possible. Same answer with the physician community. I would say, you know, to those who are curious, just talk to physicians who have actually dosed this therapy across the spectrum of Duchenne and ask them what their experiences have been. I think you'll hear that they're very excited about that. So I think, you know, I think the right-minded people are getting it. And I think that, you know, there's a lot of enthusiasm for this therapy as well there should be. And it creates for us an enormous responsibility to get this therapy out to these kids. and this team is doing everything they can to do that, and the great news is that there's no team better than this team to do it. So I don't want to give Dallin and his team too big a head, but the fact is that this team is brilliant at serving this community, and it will not change with the Levitas. We're off to a great start.

Thank you. Our next question will be coming from Brian Abrams of RBC Capitalist.

Hey, good afternoon, guys. Thanks for taking my question and really appreciate all the granularity here on the launch dynamics. You did mention that there were some wait times at centers as they navigate demand, and so it sounds like capacity, you know, is somewhat of a gating factor here. Can you give us a sense of, you know, what some of the limitations here are at the center level any additional infrastructure or processes that these sites need to put into place to administer elevitis and monitor patients, and where you think that could expand over time? What's embedded in your guidance in terms of capacity expansion? Thanks.

Okay. I'm going to turn this question over to Dallin, but let me say a few things in advance. First, you know, we don't have a fundamental capacity issue. I want to be very clear about that. We have about 75 sites in the U.S., 75% of which have already dosed patients. The number one probably – well, the number one metric to the success of therapy is the experience with it and how well it's working. But with that said, then the experience of the actual infusion is really important. So having such a significant number of sites, I believe more than any other gene therapy we've ever launched with, and having 75% of them already having those patients is significant. And I think one of the things you're seeing early days is there are definitely some sites that – in the absence of more information, are just worried about the onslaught and trying to manage the amount of demand that's coming in. Because there are, you know, some 12,000 or so patients in the United States with Duchenne muscular dystrophy. You know, 80% or more of them can be treated with this therapy. They've got to prioritize and think that issue through, and they're pondering it. But with that, Dallin, if you want to provide more granularity than I have on this topic, please do.

Yeah, thanks, Doug. And, Brian, thanks for the questions. And as Doug said, you know, we've got more than enough capacity. We've got more than enough capacity today to accommodate even the peak years of sales, Brian. But really what is happening out there is, as I said in my remarks, that essentially overnight the vast majority of patients became eligible. So each individual center just has to work through all of their patients. They've got all of their patients reaching out. and figure out how they're going to approach who they're going to prioritize, who they're going to dose first. And, you know, this is to be expected, and everything is really exactly as we have forecast, anticipated, and expected. And we're doing everything we can to support these centers. What makes it, you know, what should provide a lot of confidence is These are the centers that are at the forefront of precision genetic medicine. They were the ones that figured out Spinraza. They figured out Zolgensma. They figured out our previous narrow label, and they're just in the process of figuring this out. And these early stages are just an adjustment period as they ramp up their ability to serve the whole patient population.

Thank you. Our next question today will be coming from Salveen Richter of Goldman Sachs. Your line is open.

Good afternoon. Thanks for taking my question. Just going back here to this guidance, you know, it seems like you have the infusion centers, but there's some kind of bottleneck or lever you're using with regard to kind of how you're determining 2025 guidance. So is – Is the gating factor here, as given the demand, is it the number of patients that can be treated at an infusion center over a time period, or is it the manufacturing supply in the context of the demand? And then help us understand how you're thinking about cannibalization of the PMO side of the business in your 25 guidance. Thank you.

So, again, I would say that, first of all, thank you for your question, Salvi. I would say I think it would be a mischaracterization to envision that there is some significant bottleneck. The shape of the curve, you know, is related to a number of things. It's not related to demand, patient demand, physician demand is going to be very, very significant. But then there's this process, the process of getting through the infusions, payer interaction, and the like all defines the shape of the revenue curve over time, and we're very pleased with where we are, and we don't have a problem to solve. We will if all goes well, and it is going exactly that well. It means we're going to do $2.9 billion to $3.1 billion next year, which means a very significant number of patients are going to significantly benefit from our therapies, And I would remind everyone that is years away from peak year sales and we'll be treating the prevalent population over the entire course of this decade into the early 2030s. And then on cannibalization, there will be cannibalization if for no other reason that, you know, there is a kind of a competition for start forms for patients as well. But in this year and into next year, we see that cannibalization is quite modest. Thank you again for your question.

Thank you. And our next question will be coming from Ritu Burrell of PDCAL. Your line is open.

Good afternoon, guys. Thanks for taking the question. Doug, you've given different sort of aspects of guidance going forward, including the fact that you guys think that PEAT is going to be towards the end of the decade, You had previously given us a rough figure of about 4 billion peak for Alevitas, and this was a few years ago, and, you know, the centers were all not all up and running and stuff like that. But do you still see that as the general peak in your internal assumptions? And I wanted to also ask about the sort of levers between the top of your guidance and the bottom of your guidance. It's really, really tight guidance. So I'm assuming you have start forms that will track all the way out through some element of 2025. Can you talk to the volume of start forms and what the levers are for the top and the bottom and how far out those start forms will map your revenues in the enhanced start forms?

So a couple thoughts on your first question. Yes, some years ago I provided long-term guidance. We have no basis for amending that guidance at all. We feel very comfortable about the prior guidance that we provided. So there's no need to update that. It does appear still to be very accurate. And on the number one thing, we have a lot of the start forms. We don't have all the start forms yet, of course. That's not the way it works. But remember, we have more than anything else great experience. Like we know what level of start forms correlate to what. We know what the launch curve should look like. We have a ton of algorithmic ways to look at this launch and map our expectations versus the current launch. And the short answer is based on all of our experience and knowledge in algos, we are doing very well. As I've said, the early signals exceed our expectations. optimistic view on this launch. So we're, I'll just say again, we are comfortable with the guidance that we're providing right now. And, you know, to your point, it's, you know, it is, we're getting to be a relatively big company. So while, you know, it's tight, I mean, it's still $100 million on each side, but to your point, it's getting to be pretty tight, but we feel very comfortable about providing that level of guidance.

Thank you. And our next question will come from Mike Alts of Morgan Stanley. Your line is open. Hey, guys.

Thanks for taking the question. Maybe just a quick follow-up on the PMO cannibalization. You're expecting modest cannibalization in 2025, but just curious how you're thinking about that longer term. Should we still be thinking some modest impact, or should that ramp up as sales start to ramp up?

Well, first of all, thank you for your question, Mike. It's an interesting issue. We have always modeled some robust, eventually some robust cannibalization of the PMO franchise. What we've seen so far has been fairly, been very modest. And what we're modeling into next year is modest. It might be more significant in years beyond that, but there's also some reason to believe that our views have been a little bit conservative on, you know, how much cannibalization there will be. I should say aggressive on the amount of cannibalization. We may have been a bit, you know, our own internal models may be a bit aggressive on the amount of cannibalization. But what I would say right now is our models suggest that the cannibalization is very modest now. It will remain pretty modest next year, and it might be more significant in the years after that.

Thank you. And our next question will be coming from Brian Scorney of Beard. Your line is open.

Hey, good afternoon, guys. My question is on the cannibalization of PMO as well, and just trying to contextualize when you say fairly modest cannibalization. Why isn't it just a straight-up equation based on the prevalence of the exon amenable patients and the penetration that you have into PMO? I would sort of calculate that you know, somewhere between 8 and 10 Levitas patients should result in one PMO patient cannibalization, assuming that person would have to be forced by their insurance company to choose to take a Levitas and come off the PMO. Is that not the case? Are you seeing patients who are getting commercially treated with a Levitas and maintaining their PMO post-Levitas? Yeah.

Dylan, do you want to touch on this?

Yeah, it's a good question. You know, one of the reasons it's not a, you can't map it out that way is that right now there's a significant percentage of our PMO business that doesn't come from the U.S. In terms of commercially right now, are there patients that have been dosed with gene therapy that have gotten PMOs? We haven't seen that to date, but we've seen that in the SMA space, and I think we're aware of some patients that are trying to get access that have dosed with the Levitas before, but we haven't seen that to date.

So, noting that, that's the problem. Dallin touched on one of the confounders to your analysis, Brian, which is that, you know, XUS sales exist and have been, you know, relatively robust. So, Your math assumes a closed system, and we don't have a closed system. We actually have an open system.

Thank you. One moment for our next question. And our next question is coming from of . Your line is open.

Guys, thanks for taking our question. I guess maybe just going back to the start form, you know, you said that since the approval in 2023, you've been receiving forms from all these sites and physicians. Just wondering if there were any forms that would be filled in patients who aged out before they can get treatment. And could you maybe also sort of define what you mean by you know, patients who are declining? Are these older patients or does it, or ages? It doesn't matter at all. Thanks.

I'm an apologist. If you understand, I didn't quite understand the question. Apologist.

Yeah, no, I think I understood it, and please let me know if I didn't. In terms of the last part of your question, the decline, where the doctors seem to be prioritizing. No two sites are doing it exactly the same way. But as we've seen with our PMO launches, patients who are in the declining ambulatory phase, so that's kind of in the 9 to 11-year-old age ranges, those patients were predominantly prioritizing the previous three broad-label exon skipping launches, and we've seen the same thing here. And what was the first part of your question again?

I think you may have been asking if any patients that would have otherwise aged out.

Oh, aged out, aged out. Yeah, the aged out. Yeah, I was excited about that one because the team did such a great job in execution. There were very few patients that did age out in the initial narrow label. There were a few, and, of course, the team never gives up on any patient, and we will, you know, those patients that aged out in the prior label. will now be eligible again. So we will do everything we can to support anybody who did age out and comes back into the system.

And then finally, what Dallin is suggesting on the prioritizing the late declining, it's simply that's just a mathematical fact. You're seeing patients start formation as early as four years old into the 30s. And then, you know, you see kind of a peak right now in the, you know, right in that kind of 10, 11, 9 age range. And that's what we've seen with the PMOs as well. But the good news is, you know, we're seeing a robust start forms across that entire group, which gives us a lot of confidence in where we're heading as an organization.

Thank you. Our next question today will be coming from Robert Finke. of Guggenheim, your line is open.

Hey, thank you for taking our question. This is Robert . From our side, a couple questions here. Do you anticipate manufacturing capacity will be a gating factor at any point between now and peak sales?

That's a good question. So the short answer is we don't envision that manufacturing is going to be the bottleneck for the next few years, and by the time that we would see it as being a rate limiter, we'll be hopefully in suspension by that point, and then we shouldn't see any limitations. So, we're very good where we are right now.

Thank you. Our next question is from David Hogan of Citigroup. Your line is open.

Hi there. Thanks for all the great updates and taking my question. I just wanted to ask if you've got any feedback from your centers about post-administration monitoring of these patients. There's obviously a number of labs that need to be followed for a period of time for safety, and I just wanted to ask whether, you know, that is a consideration and sort of the speed of how fast patients can be dosed and then ultimately overall the shape of the launch curve.

Yeah, thank you very much for that question, and it's very insightful. You know, a lot of times you think about the infusion at the last moment and that that's sort of the cadence of infusion describes the amount of patients that can be dosed. But, of course, if one's going to do this in a thoughtful way, it's important that there's a lot of very good follow-up, and there is. So that amount of infusions you can do relate not just to the amount of infusion rooms, appointments you can have, but good follow-up, and we're very, very supportive of great follow-up. So, that is a part of the entire process, and to the best of my knowledge, we're seeing a very stable safety profile as well based on that monitoring, but Luis or Luis in particular, if I'm getting any of that wrong, please let me know.

Thank you. Yep.

I'm sorry.

No, I was just going to say, yeah, no, exactly right. Sorry.

That's it. Okay. I didn't want your absence to be, you know, a negative.

Our next question will be coming from Kristen Kuska of Contour Fitzgerald. Your line is open. Okay.

Hi, everyone, and all the best with the expanded launch here. Wanted to ask if your 2025 numbers assume anything related to a European approval. Is that baked in here? And then understand that this process takes a few months' time, but what's the key driver behind reaching peak sales in a few years versus, say, the 2026, 2027 timeframe? Thank you.

So, our guidance does not assume – first, I want to be very clear. We'll leave it to Roche to talk about their ex-U.S. approvals, and as everybody knows, EMA has already accepted their submission for review, and that should result in an action in 2025. But the numbers that we're giving here right now Don't presume that issue either way. This is really relating to our U.S. sales. And the short answer on the revenue curve is, of course, there is a revenue curve that occurs, and it is a function of everything from the process to the site monitoring and site infusions and payer interactions and the like. And so, as I said, you know, we believe right now that we're going to do $2.9 to $3.1 billion next year across all our therapies. We will continue to treat the prevalent population over the course of this entire decade. Peak year sales will be some years after 2025, and we feel very good about where we are and the good work we're doing for patients.

Thank you. Our next question will be coming from . Your line is open.

Yeah, thanks for taking my question, guys. You commented on the number of infusing sites over the last few quarters with the number of infusing sites increasing in Q2 from Q1 by about 13 to 15 sites, according to our calculations. So how do we think about patients treated per site and the consistency of the volume of patients treated per site?

Well, you know, so let's first, I'll turn this over to Dallin. He can chat a bit about that. But just so we're very clear, we're very comfortable with the number of sites that we see. Now, what you may see over time is a general increase in some sites. That is not part of some grand proactive strategy. but really relates more than anything else to being responsive to requests from sites. So if an appropriate site asked to be a site and wanted to go through the educational process and to be validated to be a site, we would certainly consider that. But as it sits here right now, we are very comfortable. As I will remind everyone in advance of this launch, we had an aspiration to be 50 sites with a goal of maybe even someday getting to 70 sites, all of which would be much higher than than any other launch of a gene therapy. And so, sitting at 75 sites, we feel very, very good about it. But if you want to provide any other metrics now and on kind of, you know, productivity by site of the life, feel free.

Yeah, no, thank you for the question. And I don't know where those numbers came from, because our site, the number of sites has been very consistent. We have a right-sized model and You know, we've been hovering right around 75. Now, that said, just as Doug said, our model is flexible. So, we can bring new sites on, get them up to speed, and bring them on very rapidly. And, you know, one of the things with this broad label is the possibility that we'll be bringing on some adult neuromuscular sites to accommodate older patients as well. So, as Doug alluded, we have more than enough capacity today But importantly, to serve patients, we have built flexibility into our model so that as needed to support patients, we can bring on new sites very, very rapidly. The team is very responsive.

Thank you. And our next question will be coming from Gavin Clark, Gartner of Evercore. Your line is open.

Hey, guys. Thank you for taking the question. So I fully appreciate the longer conversion cycle as we're looking at the back half of 24 and into 2025. But you've also referenced your approved PMOs as analogs for adoption. Those themselves are the largest ramps within the first one to two years after approval when we're talking about new patient ads. And that's basically how all other rare disease launches go with very high on that need. So I guess like even with a few months of the lag process, why would peak sales not be at some point in 2026?

because it won't be. We have good modeling. We know exactly, you know, we have a very good view of where we're heading with, you know, the capacities and the process and the like, and we're confident that it won't be TQ or sales in 2020. So, another thing you have to remember, and by the way, and I think that people are trying to solve for whether there is some problem. There's not. We're very pleased with this approach. One has to remember that this is a one-time therapy that we have to be, you know, we have to prioritize being responsible and, you know, we want to make sure that all of these sites are in a good place where they're not prioritizing getting as many patients as possible dose without regard to safety and follow-up and the like. And we've done a really good job of that. The sites are doing a very good job of that, all of which means We're going to be very successful this year. We're going to have good, modest growth in the Q3. We're going to have very, very robust growth in Q4, doubling in Q4. We're going to do $2.9, $3.1 billion across our therapies in 2025. And then as it relates to teacher sales, it'll be in the back half of this decade.

And, Doug, can I just jump in? I think you've been alluding to this, but, Gavin, your question's a good one. And I think Doug's been alluding to the fact that We have visibility at these sites at a very granular level. We have it mapped out by payer coverage, by patient. We know exactly who's lined up, how they're going to be lined up. We map this out in very, very granular detail, and this is why we have a very high level of confidence in our forecast assumptions. So I hope that added level of detail helps a bit. Thank you, Dom.

Thank you. And our next question will be coming from Kotas Valerius of BMO Capital Markets. Your line is open.

Hi. This is Dale on Focuses. Thank you for taking our question. So our question is on the LGMD gene therapy portfolio. Given that half of your LGMD gene therapy portfolio uses the same vector and promoter, and actually all of them are using the same vector as Elividus, So how are you thinking about potentially leveraging FDA platform designation platform to accelerate the development of the LGM-DGN therapies and other early pipeline assets? And also, if you can comment on the potential impact from the inclusion of a limitless effect on the time function test in the label on patients and physician-pair negotiations and the potential future competitions. Thank you.

I'll turn the LGMD question over to Luis and our approach to accelerating the LGMD development.

Yeah, thanks for the question. I think you're exactly right in terms of using the Alevitis experience to help accelerate the LGMD platform. We've been using the same RH74 vector for these programs and just to cite the cyclic glycanopathies, the similarities between them. We've received fast-track designation, as I mentioned in my remarks, and pulling on every lever possible with FDA to make sure that we're moving in the fastest speed possible. We've been very happy with the interaction so far, and that's our 9003 pivotal trial, as noted, is our open-label trial in terms of looking at this ultra-rare population and being thoughtful about the way that we develop these with LGMD 2D and 2C coming along. So, certainly, we're leveraging everything possible and our safety experience is levered for the LGMD portfolio.

Thank you.

Hold on. Before we move on, Dallin, did you have something else to add?

I think Dale asked about the time function test, the data that's in our label and how those may be impacting the payer conversations. And, Dale, it's a great, really, really great question because that is really what the team is focused on with the payers is that incredible new data that's in our label. And there has been, as Doug outlined in his comments, I did a little bit in mine, the engagement, the interest from the payers to understand that data. And so that's a big part of that engagement with the payers right now is going through that new data that supports a broader label for Levitas.

Just to remind everyone that not only did we hit all of those, the anomaly statistically significantly, the importance of that can't be overstated. I would remind people that time to rise, the benefit we've seen on time to rise is correlated with over 90% decrease in the risk of early loss of ambulation. So, I mean, these are not only important metrics, but they, you know, correlate importantly to very things that matter a lot to patients.

Thank you. And our next question will be coming from Tim Lugo of William Blair. Your line is open.

Hey, team. This is John on for Tim. Thanks so much for taking our question. Maybe a follow-up on the last one. Beyond the internal pipeline, with your unhealthy balance sheet and your expanded launch in front of you, just wondering if you can give us any updated thoughts on how you're thinking about potentially in-licensing new programs.

Sure. I'll turn this to Ian. Ian?

Hey, thanks for the question. This is what I discussed in our prepared remarks. As an organization that's been developing therapies and successfully manufacturing them and all the way to commercialization, it gives us a wide amount of substrate to evaluate. We're going to be just looking at ones that fit very well with our existing capabilities and being able to leverage that and being able to deliver it to patients quickly. So we've been really fiscally responsible at the transactions we've done in the past. Like I said in the prepared remarks, we're going to have a lot more resources to deploy, but we're still going to use that same approach. There are a lot of interesting therapies that are being developed, but before maybe this week, the valuations were incredibly high, and we're not going to You know, we're not going to chase opportunities. We're going to look for ways to build value from a development perspective, you know, for patients but also for investors. So, you know, we're very keen on looking at valuation and making sure that it makes sense, you know, based on the market opportunities.

Thank you. And our next question will be coming from G. Bloom. of Neal and Company. Your line is open.

Hi, everyone, and thanks for squeezing us in. I think maybe a last question, I'm trying to understand a potential bottleneck here, but doesn't it really just boil down to how many treating physicians you have at each center, and if you can provide any thoughts on how that metric works.

Thanks. So, again, I'm going to frustrate you by answering this question again the same way, which is we don't have bottlenecks. We have a launch course. We're doing very, very well. We have a significant number of sites, a significant number of treating physicians at sites, a lot of enthusiasm. We have more than enough sites, more than enough physicians, extraordinary demand from patients and those physicians, great interactions with payers, a very strong, manufacturing approach right now. I feel very good about that from a capacity and supply perspective and a great distribution channel. As a result of that, we're going to have a really successful back half of this year, and we're going to have a 2025 that will mean that we're going to do revenue across our four approved therapies of some $2.9 billion to $3.1 billion. We are profitable today. We were cash flow positive this quarter. We will be in the next couple of quarters very consistently cash flow positive on a go-forward basis. We're in a very different place than the vast majority of biotechs today. We have a very strong, very sustainable business, all of which is focused first and foremost on bringing a better life to these patients. We're going to bring a better life to a lot of patients. over the next, you know, many years this decade, and secondarily, but also importantly, rewarding those investors who have stuck with us and have committed themselves to this mission. So we feel like we're in great shape. And, you know, not to be overly defensive, but we don't have a bottleneck that we need to solve for. We feel very good about where we are.

Thank you. This concludes today's Q&A session. I would like to turn the call over to Doug for closing remarks. Please go ahead.

Thank you very much. Thank you all for your questions, your insightful questions this evening, and thank you to my team for those great answers. I'm very excited about where we're going for the rest of this year. We have a lot of work to do this year. Our launch is going well. Our continuing service of our PMOs are going very well. We understand the enormous responsibility we have to these patients to ensure that the greatest number of these patients can benefit from our therapies, and we're going to make the Elevitus launch brilliant. I look forward to updating you across this year on launch performance and with the team to update you on our pipeline advancement as well. And with that, have a lovely evening, and I look forward to talking to you soon. Thanks.

This does conclude today's conference call. You may all disconnect.