Sarepta Therapeutics, Inc.

Therapeutics First Quarter 2024 Earnings Call. At this time, all participants are in a listen-only mode. 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 and Corporate Communications. Please go ahead.

Thank you, Victor, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the first quarter 2024. The press release is available on our website at SORUPTA.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 Rodino-Claypack. I'd like to note that during this call, we will be making a number of forward-looking statements. Please take a moment to review our slide in the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, many of which are beyond SORUPTA's 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 of 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?

Well, thank you, Mary. Good afternoon, everyone, and thank you for joining Sarepta Therapeutics' first quarter 2024 financial results conference call. Before I begin, let me provide you with an update. As you know, we have submitted a BLA supplement to expand the labeled population for our Duchenne gene therapy Levitas. We were recently informed by the FDA that they will provide us with a draft Levitas label imminently and sooner than previously indicated. As we move into the late stage of our review, we will be going into a quiet period, and as we have done in similar situations, will not be taking questions on the call today so with that this afternoon we announced another strong quarter of performance with total revenue of 413 million dollars in net product revenue from our four approved therapies of approximately 360 million dollars growing at over 55 percent versus the same quarter last year in particular Elevitus net product revenue was nearly $134 million, modestly above fourth quarter revenue. As we signaled in our last earnings call, Elevitus sales will flatten as we plan for label expansion. This is not at all surprising, as our current Elevitus label is very narrow. Indeed, the labeled four- and five-year-old age range is quite narrow on its face, but the addressable patient population is actually more narrow still than that. Let me explain. About half of Duchenne patients in this age group have not yet been diagnosed and are not yet available for treatment. And for those diagnosed, they are dissimilar to any other age group, as they will have just learned of this devastating and life-altering disease. And they must get educated, find the right physician, even before submitting a start form and beginning the process leading to treatment. Despite these limitations, we have already posted over $334 million since our approval last June, far exceeding all other gene therapies approved in the last few years combined. This says much about the opportunity in front of us. Physician and patient demand are significant. We are working well with public and private payers to facilitate access. And our multi-year obsessive preparation in site readiness, manufacturing, distribution, access, and support is all paying off. Having served this community with four therapies over seven years in every state in this country, we have a keen understanding of the prevalence of Duchenne in the United States. And therefore, we know quite well the opportunity that will come from a broadened label, both for the patient community and for the investment community that has bet on bringing a better life to patients. Moving to our BLA supplement, as you know, in December of last year, we submitted an application with the FDA to expand availability of Alevitus by removing age and ambulation restrictions in the label and also transitioning that approval from accelerated to traditional. As we are in the midst of this review, we cannot yet comment substantively on the outcomes. But I can say this. As we all know, Dr. Peter Marks, the FDA CBER Center Director, has publicly and repeatedly expressed a vision for the future of gene and cell therapy, one where the division is more collaborative and engaged, and one that leans in and uses its regulatory flexibility and the tools available to it, like the accelerated approval mechanism, to advance therapies for serious life-limiting rare disease. In service of that mission, and vision, Dr. Marks created a new super office of therapeutic products, or OTP, and brought in new aligned leadership under Dr. Nicole Verdun, the division director. Since Dr. Verdun has arrived, she has publicly echoed much of Dr. Marks' vision. And more than just words, we have seen an improved level of engagement. But we have also seen tangible evidence of progress toward that vision. For instance, Since Dr. Verdun's arrival, we were able to quickly align on a viable trial for SRP9003, our gene therapy for the ultra-rare disease LGMD type 2E. Our pivotal trial, Emergene, is a single-arm study with an appropriately sized N of 15 patients and uses a biomarker, the missing beta-sarcopylican protein, as the primary endpoint. As we announced in our last earnings call, we have already commenced dosing in Emergene. This meaningful evolution gives us renewed optimism that we can move forward more swiftly with the rest of our LGMD portfolio. Now, in a moment, our head of R&D and chief scientific officer, Dr. Luis Rodino-Clayback, will discuss our R&D progress from last quarter, including the expanding body of real-world evidence for our PMOs, our LGMD progress, and the results and plans for our advanced peptide-conjugated PMO, SRT 5051. 2024 will be a pivotal year for Sarepta and for the patients that we serve. Indeed, it promises to be the most important year of progress yet in the fight against Duchenne muscular dystrophy. But we are also aware that it stands for more than that. By necessity, we are the leaders in gene therapy today, and this year's outcome could set the direction for cell and gene therapy for years to come. Seven years ago, there was an enormous excitement for the transformative potential of gene therapy. That excitement flagged over the last years, not because the science failed us, but because many began to question whether this fledgling technology would be properly nurtured and supported. If we can achieve our goals this year, it will stand as a strong signal of renewed energy and excitement for a technology which after decades of brilliant progress has the potential of saving countless lives otherwise stolen by rare genetic disease. I look forward to updating you, and hopefully celebrating our progress with you, and more important still, with the Duchenne community. I will now turn the call to our Chief Customer Officer, Dallin Murray. Dallin?