5/6/2026

speaker
Conference Operator

Good afternoon, and welcome to Sarepta's first quarter 2026 earnings results call. As a reminder, today's program is being recorded. I would now like to turn the call over to Tam Thornton, Sarepta's Director of Investor Relations. Please go ahead.

speaker
Tam Thornton
Director of Investor Relations

Thank you, Antoine, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the first quarter of 2026. The press release, along with our presentation slides and supplementary information, will be available on the investor section of our company website. We plan to file our Form 10-Q for the quarter today with the SEC. Joining me on the call today are Doug Ingram, our CEO, Dr. Louise Rodino-Claypack, President of Research and Development in Technical Operations, Patrick Moss, our Chief Commercial Officer, and Ryan Wong, our chief financial officer. Additionally, joining us in the Q&A portion of the call are Ian Estepan, president and chief operations officer, and Dr. James Richardson, our chief medical officer. Before we begin the formal remarks, I would like to note that during this call, we will be making a number of forward-looking statements. Please refer to slide two of our presentation to view the formal text of these safe harbor statements. These statements involve varying risks and uncertainties, many of which are beyond Syrepta's control. Actual results could materially differ from these forward-looking statements, and such risks can adversely affect our business, our results of operations, and the trading crisis throughout this common stock. We strongly encourage all listeners to review the company's most recent SEC filings for a detailed description of these applicable risks. The replica explicitly states that it does not undertake any obligation to publicly update or revise its forward-looking statements or financial projections based on subsequent events. Furthermore, please note that we will discuss non-GAAP financial measures during today's webcast. Complete descriptions and reconciliations of our GAAP to non-GAAP financial measures are included in today's press release and the accompanying slide presentation available to investors on our website. With that, I will now turn the call over to our CEO, Doug Ingram. Doug?

speaker
Doug Ingram
Chief Executive Officer

Thank you, Tam. Good afternoon, everyone, and thank you for joining Sarepta Therapeutics' first quarter 2026 financial results conference call. We entered 2026 with a clear set of priorities. Stabilize the business, restore confidence and growth, maintain financial strength, and continue advancing a pipeline with the potential to define Sarepta's next era. In the first quarter, we made meaningful progress against each of these priorities. First, our marketed product performance has stabilized following the disruption and uncertainty of 2025. With expanded field reach, increased physician engagement, and a growing body of compelling evidence supporting the disease-modifying impact of Alevitis, we believe that therapy is positioned to return to growth. Second, Sarepta remains in a strong financial position. We ended the quarter with approximately $748 million in cash and investments, delivered positive GAAP and non-GAAP earnings, generated positive cash flow excluding Arrowhead-related payments, and remain on track for positive cash flow even under our base case assumptions. The financial strength matters. It allows us to fund our pipeline without relying on the equity markets. We are fully funding our programs in DM1, FSHD, Huntington's disease, the spinocerebellar ataxias, and our preclinical and research portfolio. And third, we are making progress in what we believe is a potentially best-in-class portfolio of siRNA therapies, as Dr. Luis Rodino-Klapak will discuss shortly. We are especially encouraged by the early data from SRP1001 and FSHD. and SRP1003 and DM1. And we look forward to additional readouts in the second half of this year. Turning to first quarter performance, total net product revenue was $331 million. That included $229 million from our PMO therapies, Exondus, Myondus, Amondus, and $102 million from Malevitus. Starting with the PMO franchise, we were pleased that the FDA agreed We can submit our clinical data and real-world evidence for Vyondis and Amondis. We have submitted SMDAs seeking to transition those therapies from accelerated approval to traditional approval. That is an important step for patients, physicians, and, of course, the durability of this franchise. Turning to Alevitis, Patrick will provide more detail of the commercial initiatives underway and the early signs of progress we are seeing. But I want to underscore the central point. Our confidence in Alevitis is grounded in evidence, not in mere aspiration. To date, more than 1,300 patients have been treated with Alevitis across clinical trials and commercial use around the world. The totality of the evidence continues to strengthen. The data show that Alevitis is protecting muscle and changing the trajectory of Duchenne. It is giving boys and young men an opportunity that, until now, this disease has never afforded them. We saw this in EMBARQ, the only robust, double-blinded, placebo-controlled trial demonstrating the benefit of a gene therapy in Duchenne. We saw meaningful benefits at year one. We saw those benefits grow at year two. And yet again, at year three, boys continued to diverge on every measure from the expected course of untreated disease. That matters because Duchenne does not wait. Muscle damage is progressive, irreversible, and cumulative. Every month of delay risks further loss. The evidence increasingly supports a simple but urgent message. Physicians and families should evaluate a levitus now before additional irreversible damage occurs. We have also seen supportive muscle MRI evidence showing that a levitus dystrophin helps protect muscle from damage, resulting in less muscle loss and less replacement by fat and fibrotic tissue. This is exactly the kind of biological evidence one would hope to see from a therapy intended to alter the course of Duchenne. We are confident that as physicians and families better understand the disease-modifying potential level evidence and the urgency of intervening before further decline, more patients will move towards treatment. That said, we are still in the middle of our commercial and educational initiatives, and the path from consideration to infusion takes time. For that reason, we are reiterating our full-year guidance of $1.2 to $1.4 billion and would counsel prudence in raising estimates prematurely. Now, staying with the Levitas, we are also advancing our Endeavor Cohort 8 study, which uses serolimus as a pretreatment. More than 25% of sites are already using serolimus on their own initiative. In our interim analysis from the long-term real-world evidence study that we have ongoing, Patients pretreated with serolimus have shown no evidence of elevated liver enzyme to date. That gives us further confidence in cohort 8 and in the potential, with the success of that study and the concurrence of the FDA, to resume offering Alevitas to non-ambulatory patients. Turning to the pipeline, our focus is execution. We are rapidly advancing our siRNA portfolio, including our DM1 and FSHD programs. The first data readout earlier this year showed encouraging signals that these may be differentiated, potentially best-in-class approaches for two very challenging diseases. We look forward to providing additional data later this year. We have initiated and are on track to dose our first patients in our Huntington's disease program, and we are progressing clinical trials in SCA2 and idiopathic pulmonary fibrosis. So to summarize, the business is stabilized. Elevitus is supported by an increasingly compelling body of evidence. Our financial position is strong, and our high-value SIRNA pipeline is advancing across multiple programs. We believe SREP is well-positioned in 2026 and beyond. And with that, let me turn the call over to Louise, who will provide an update on our SIRNA portfolio and broader development progress. Louise?

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