Sarepta Therapeutics, Inc.

Good afternoon, and welcome to the Sarepta Therapeutics First Quarter 2025 Financial Results Conference Call. As a reminder, today's program is being recorded. At this time, I'll turn the call over to Mary Jenkins, Associate Director, Investor Relations. Please go ahead.

Thank you, Marvin, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the first quarter of 2025. The press release slides and supplementary information are available on the investor section of our website at Sarepta.com, and our 10-Q was filed with the SEC this afternoon. Joining us on the call today are Doug Ingram, Ian Estepan, Dallin Murray, and Dr. Louise Rodino-Claypack. After our formal remarks, we'll open the call for Q&A. I'd like to note that during this call, we were making a number of forward-looking statements. Please refer to slide 2 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 Sarepta's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent 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. As noted on slide three, we will discuss non-GAAP financial measures on this webcast. Descriptions of these non-GAAP financial measures and reconciliations of GAAP to non-GAAP financial measures are included in today's press release and the slide presentation available in the investor section of our website. 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' first quarter 2025 financial results conference call. Across biotech and indeed the market more generally, the first quarter of 2025 has been a challenging one. Likewise, for Sarepta, we have faced challenges that have motivated us to take a more measured view for the remainder of 2025. Fortunately, Sarepta is in a better position than most of biotech today. as we have a significant number of approved therapies and significant revenue, a strong P&L and balance sheet, and the ability to continue to independently drive our portfolio of gene therapies and siRNA programs. We are well positioned to weather this chaotic period. In the first quarter of 2025, we achieved $612 million in total net product revenue, representing 70% growth over the same quarter last year. Our PMO franchise grew 5%, achieving $237 million for the quarter, and Alevitis achieved sales of $375 million in the quarter, representing a 180% increase over the same quarter last year. While our Alevitis first quarter growth still represents the most successful in vivo gene therapy launch yet in history, in fact, in Q1, we treated more patients with gene therapy than ever before in the US in a single quarter, we nevertheless fell short of expectations. In a moment, Dallin will explain some of those quarterly factors, including administrative issues, the severe flu season that resulted in delays, and the effect of the recently reported safety event that motivated some families with scheduled infusions to pause for additional information. Looking forward, we are changing our net product revenue guidance for the year to $2.3 billion to $2.6 billion across our four approved therapies. This change is driven by three factors. First, as you know, we recently reported that a boy infused with Alevitas suffered an acute liver failure and passed away. This was an unimaginably tragic event for that family, who with Alevitas finally had reason to hope for a brighter future for their son. At Sarepta, we are deeply committed to the patients we serve, so this event so markedly different than all other experience with Alevitus, was heartbreaking for all of us. While it is of no comfort to the family involved, it's important to remember that more than 800 patients have been infused with Alevitus. The outcome of this event is inconsistent with all other experience with Alevitus, and we are continuing to explore what was uniquely different about this case than every of the other hundreds and hundreds of infusions that have occurred to date. What we do know is that while this young man's outcome was surprised, this did not represent a new safety signal for AAV-mediated gene therapy generally, or Alevitis more specifically, nor did it change the positive risk benefit of Alevitis, which has had a stable and laudable safety profile in the context of AAV-mediated gene therapy since first being infused back in January of 2018. And, of course, to support optimal outcomes, we have always prioritized patient safety. We have proactively placed significant monitoring requirements in our label. We require all sites to complete training before being permitted to treat, and we have a never-before-seen or executed program, one called Sarepta Exchange, that provides physicians with near real-time access to the most experienced and leading experts around the world so that outcomes are consistent across the country. We also provide education for our patient community and we are committed to providing timely information about this life-enhancing therapy. What we have observed is that top thought leaders who understand the data have not changed their treatment approach and informed families understand the positive risk benefit of Alevitas. But there will be an impact on infusions as we roll out education in the broader physician community and families gain better insight into the profile of this disease-modifying therapy and have their questions answered. Second, as we have discussed, the administrative process from start form to infusion is particularly complicated for gene therapy. It includes, for instance, more appointments, more screenings, and steps like single case agreements far more than other therapies. As we have launched the broader label and expanded the actual cadence from start form to infusion, is taking about one month to six weeks longer than our original estimates, informing our forward forecasts. For the avoidance of doubt, this is not about ultimate access, but the administrative steps to get infused. As you know, we are quite proficient in working with sites to gain access. In fact, for our PMOs, our ultimate rate for gaining payer access has been well over 90%, and for Elevitus, it currently remains at 100%. We are taking actions to shorten that turnaround time. There are some reasons to believe that our first quarter experience, which includes illness-related delays and the typical first quarter insurance changes that can cause delays, may have been longer than steady state. But for purposes of setting guidance for the year, we are assuming our first quarter experience on turnaround times is representative of the year as a whole. This will have a significant timing impact on infusion and hence a significant impact on revenue in 2025. Finally, three, the success we have seen with top sites has caused an imbalance that we need to address across this year. Sixty percent of our current revenue comes from top sites and top thought leaders. Their experience and enthusiasm for Elevitus has resulted in many of them being fully booked out to 12 months. We need to direct much of our attention going forward to sites with more capacity. And we do not take lightly our decision to change guidance. But given the environment and some of the constraints we are working to address, we do believe it prudent. What we do know is this. Alevitus is the first and only available disease-modifying therapy that offers the possibility of arresting further muscle damage in 80% or more of patients being daily damaged by Duchenne muscular dystrophy. It is a disease-modifying restorer of significant amounts of a form of muscle-protecting dystrophin, and as observed over multiple studies, over eight years of clinical experience and clinical evidence, and in hundreds of patients of data, it is the only gene therapy proven to preserve function in boys dying from Duchenne. Without intervention, every day, muscle of boys with Duchenne is destroyed and permanently replaced by fat and fibrotic tissue. With the FDA following the positive expert advisory panel, and approving Alevitis broadly for boys with Duchenne, families finally now have a chance to choose a different journey. Now that a disease-modifying therapy is finally available to them, if families are delayed in getting a therapy that can finally protect them from Duchenne's relentless damage, and they are delayed either because of administrative issues or other delays or lack of information or even, at times, circulating misinformation, then we will have failed in our mission, and I hope we have proven repeatedly over these last eight years that we do not intend to fail in our mission. After our chief customer officer, Dallin Murray, provides an overview, we will hear from our head of R&D and chief scientific officer, Dr. Louise Rodino-Klapek. She will speak to you about the continuing evolution of data for the impressive efficacy of Alevitus and our plans to expand even further the population of patients who will have access to Alevitus. Finally, Dr. Rodino-Clayback will provide an overview of the great progress we are making on our LGMD portfolio, and we'll discuss our progress with our SIRNA platform, including our plans to share our proof of biology and proof of concept data from both our FSHD and DM1 programs in the second half of this year. And with that, let me turn the call over to our Chief Customer Officer, Dallin Murray. Dallin? Thank you, Doug, and good afternoon.