Sarepta Therapeutics, Inc.

Good afternoon and welcome to Sarepta's fourth quarter 2025 earnings results call. As a reminder, today's program is being recorded. At this time, I'll turn the call over to Tam Thornton, Sarepta's Director of Investor Relations. Please go ahead.

Thank you, Operator, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the fourth quarter of 2025. The press release and slides are available on the investor section of our website at Sarepta.com, and our 10-K will be filed with the Securities and Exchange Commission next Monday. Joining us on the call today are Doug Ingram, Dr. Louise Rodino-Claypack, Patrick Moss, 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 Surreptice 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 Surreptice 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 in the slide presentation available on the investor section of our website. And I'll now turn the call over to our CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?

Thank you, Kim. Good afternoon, and thank you for joining us throughout the Therapeutics Fourth Quarter and Full Year 2025 Financial Results Conference Call. As you will hear today, we've entered 2026 on a strong footing. There are three pillars to our strength. Our first pillar is our financial position. As our CFO, Ryan Wong, will discuss in more detail because of the actions we took in 2025, we entered 2026 in a solid financial position with a large and growing cash balance, significant revenue, positive operating cash flow, and removal of any near-term debt overhang. We exited 2025 with $954 million in cash and investments, growing $89 million in the fourth quarter. We anticipate, under all reasonable scenarios, to be cash flow positive and profitable on a non-GAAP basis this year, even as we fully invest in our pipeline and our marketed therapies. Our second pillar is our four durable life-improving therapies. As you will have seen, our three approved PMOs have remained stable, even in the face of a levitis cannibalization. We read out the results of our Avondis and Byondis conformatory study, Essence, late last year, and we have scheduled a meeting with the FDA for later this quarter to discuss the potential to transition those two therapies to a traditional approval. When one considers the PMOs, it is important to recall several things. The PMOs have been serving the Duchenne community well for many years now, in some cases, exceeding a decade. Over these many years they have been on the market, there has accumulated an exceptional body of published real-world evidence consistently showing the significant disease-moderating benefits of these therapies across measures and across organ groups. And Essence is supportive of the risk-benefit of these therapies. Patients and their physicians see significant value in the PMOs as represented by year-after-year compliance rates consistently over 90%. Indeed, families are fiercely supportive of these therapies. And importantly, the safety profile of the PMOs over the last decade is stellar. For these reasons, the evidence supports, and we think the time has come, to transition among us and beyond us to a traditional approval, although we do stand ready to provide additional reasonable prospective real-world evidence if ultimately required. Moving on to elevatives. The unanticipated, in two instances, tragic events of 2025 created uncertainty around Alevitus in the middle of last year. But much of that uncertainty has been cleared by now. While short-lived FDA actions caused questions about the future availability of Alevitus, that is now resolved with an updated label, updated precautions and monitoring, and a traditional approval for all ambulatory patients for and over. The FDA has approved our serolimus pretreatment study for non-ambulatory patients, which is an important step on the potential pathway back to treating non-ambulatory patients assuming a successful outcome. That trial is already enrolling patients, and we expect results by the end of this year. Once we have the data in hand and assuming its success, we will discuss with the FDA the fastest possible pathway forward to resume commercial dosing in the non-ambulatory population. Those results will also be an important element of moving forward with our BLA for SRP9003 to treat LGMD2E. Over 1,200 patients have already been treated with Alevitis, and yet the majority of patients remain to be treated. That is an enormous opportunity. As we address the issues of 2025, we were left with little time to provide balanced information on the safety and efficacy of this simply remarkable therapy. And that information deficit has resulted in some patient and physician hesitation that must be addressed to ensure the greatest number of Duchenne patients benefit from the Levitas. We have well-designed plans to address information deficits so that families and treating physicians are fully armed with accurate and balanced information to inform the decision to treat with the Levitas. In a moment, Patrick Moss, our Chief Commercial Officer, will walk you through our plans. Equally important, the overwhelming evidence that Alevitus significantly alters the course of this disease has only grown over the last year with updated three-year embarked data that was universally positive in showing that the gap between Alevitus-treated Duchenne boys and those who unfortunately have not yet been treated grows greater and greater with every passing year. These plans will take some time to execute given both the timing of the plans and the long cycle nature of start, form to infusion. But based on the market research and advisory boards we have conducted, we are confident about our plans. We previously chose not to provide guidance on total net product revenue for 2026 due primarily to the uncertainty on the timing of our Levitas initiatives. However, we will provide broad guidance today to help investors model the year. Now, while it is challenging to perfectly model the exact shape of the revenue curve of a one-time therapy like Alevitus. We believe there is a significant opportunity among the prevalent and incident population over the next three-year horizon. A delay in impact of our initiatives may affect near-term sales, but they should not impact the ultimate opportunity as we expect to pick up those sales as our initiatives result in better understanding of the disease-modifying benefits of this therapy and the compelling need to treat as soon as possible. But that is not to suggest that we will be satisfied before all patients and physicians have the information they need to make informed decisions about this therapy. Regardless of our long-term sales, every day that goes by without treatment, a boy or young man loses muscle that he can never get back. Patrick will discuss this in more detail, but given the timing of the initiatives and the long cycle time for Levitas, we do not expect that we will begin to see the impact of our educational efforts until at least well into the second half of this year. Looking at the first quarter itself, we should achieve revenue that's about flat to perhaps down 15% from the prior quarter. For total net product revenue for the entire year, we are providing a guidance range of $1.2 billion to $1.4 billion for our free therapies. To be clear on our expectations, without our educational efforts, we would expect to track to $1.2 billion in net product revenue for the year. Depending on the timing of the initiatives, that would be as high as $1.4 billion if the initiatives more immediately showed impact. Given the long cycle times I just outlined and the resulting quarter-to-quarter variability, we believe that it is prudent to model toward the low end of the range, at least for the time being. While we are being disciplined in our near-term projections, our confidence in the ultimate demand for Levitas and the transformational nature of this therapy remains exceptionally strong, and we believe our initiatives will support this opportunity. Now, the third pillar of our company is the strength of our pipeline. We have an exciting siRNA pipeline that is advancing. We have now five clinical stage programs, including our neuromuscular programs for DM1 and FSHT, our pulmonary program for IPS, and our CNS programs for SCA2, and now for Huntington's disease. Given the unique approach we are taking with the Arrowhead Trim platform, these programs hold the potential to be best in class. They also hold the potential to bring a better life to more than 160,000 patients in the United States and multiples of that number internationally. We have two additional programs completing preclinical work for deadly CNS diseases, that's SCA1 and SCA3, and we have additional discovery programs with Arrowhead for high-value targets across neuromuscular and cardiac and CNS diseases. We are making good progress with our SIR&A programs, and we will have some important updates this year, as Dr. Rodino-Playback will discuss later in this call. And with that, let me turn the call to our Chief Commercial Officer, Patrick Moss. Patrick?