Sarepta Therapeutics, Inc.

The press release and slides are available on the investor section of our website at serepta.com, and our 10Q will be filed with the Securities and Exchange Commission on Thursday after market. Joining us on the call today are Doug Ingram, Dr. Louise Rodino-Claypack, Patrick Moss, Ian Estepan, and Ryan Wong. After our formal remarks, we'll open the call for Q&A. I'd like to note that during this call, we will be making a number of forward-looking statements. Please refer to slide two on the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, many of which are beyond disruptive control. Actual results can 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 SEC filings. The company does not undertake any obligations 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 on the investor section of our website. And now I'll turn the call over to our CEO, Doug Ingram, who will provide an overview of our recent progress.

Doug?

Thank you, Kim. Good afternoon, everyone. Thank you for joining us for our third quarter 2025 financial results conference call. Next slide, please. We have much to discuss this evening, but let's begin by reviewing the completion of our confirmatory study for our two ultra-rare disease TMOs, Lyondis and Amondis, before coming back to our quarterly update. Next slide. I'm going to turn the call over to Dr. Reno-Klaypak very shortly, but let me first give some broad conclusions. First, we are very proud to have completed our primary confirmatory obligations. Both Amondis and Lyondis serve ultra-rare populations with total prevalence numbering for each no more than about 500 to 800 patients in the United States and an incident rate per year of maybe a few dozen patients. This disease is also heterogeneous and degenerates not over months or years, but literally over decades. These together make the powering and conduct of a placebo-controlled trial particularly challenging. I want to give a huge thanks to our investigators and then very importantly to the brave families who have had the courage to enroll and risk a placebo arm for 22 months. Without this special community that we serve, we would not have completed this unusually onerous study. Second, when reviewing the evidence to support transition from accelerated to traditional approval of a therapy, one of course looks to the totality of the evidence. Here the division recognized the challenges associated with this ultra rare disease trial and set out a very specific standard for continuing marketing authorizations. As referenced in our Vyondis approval material, the FDA gave us a very specific written language about voluntarily withdrawing marketing authorization, which would only occur if, quote, no relevant analyses find sufficient evidence of a clinical benefit. As you will hear from Dr. Rodino-Claypack, we believe that we have met that standard and that we have sufficient evidence to discuss with the agency transitioning from accelerated to traditional approval. Consider, as you will have seen in our press release, the study missed its statistical significance. However, the data demonstrated a consistent and clinically favorable trend across the trial population. Importantly, a portion of this study was conducted over the COVID pandemic period, and as with many studies over that period, the study results were impacted for a variety of reasons. During the pandemic, the rate of missed doses was unusually high, with nearly all patients missing doses and approaching half of whom missed substantial consecutive doses. Participants in the study were also largely shut in and suffered deep conditioning and loss of mobility. All of this appears to have confounded the results. When one excludes the COVID participants, we see a meaningful treatment benefit, slowing disease progression by about 30%, which Dr. Rodino-Klapak will further explain. Likewise, to enroll this study, we were required to admit a broad population from as young as six years old to as old as 13 years old, including those who would clearly have confounding ceiling and floor effects. In a subgroup analysis of those likely to progress, there was a strong statistically significant benefit, and this was across not only the primary but the other endpoints as well. There is also a wealth of published real-world evidence for the PMOs. As just a few examples of the multiple real-world studies across our PMOs, We see that when tracking viandus over six years, there's an 88% reduction in risk of loss of ambulation with a Kaplan-Meier analysis of delay of about three years. Likewise, with both viandus and amandus, we see over time a significant attenuation in pulmonary decline and a significant delay in time to cough assist and ventilation. We see the same trend in our own study essence. As part of the benefits they are seeing, patients are required to be infused weekly, a fairly onerous protocol, and yet their compliance rate has been well over 90% commercially year over year over year. When one considers all of the evidence for benefit and then ways a favorable, stable safety profile is tracked over many years, we believe the risk benefit remains positive. We not only anticipate continuing marketing authorization, but we believe we have a good argument for traditional approval. Our plan is to schedule a meeting with the division to review the totality of the evidence. Dr. Rudina-Fayback will now discuss those results in more detail. Louise?