Sarepta Therapeutics, Inc.

Good afternoon, and welcome to the Startup to Therapeutics first quarter 2023 earnings call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1-1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1-1 again. 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, Shannon, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the first quarter of 2023. The press release is available on our website at Sarepta.com, and our 10-key was filed with the Securities and Exchange Commission this afternoon. Joining us on the call today are Doug Ingram, Ian Estefan, Dallin Murray, and Dr. Louise Rodino-Playback. After our formal remarks, we'll open the call for Q&A. I'd like to note that during this call, we'll be making a number of forward-looking statements. Please take a moment to review our slide on the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, many of which are beyond SRAPTA'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 SRAPTA's common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent quarterly report on Form 1022, 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, to provide an overview of our recent progress. Doug.

Thank you, Mary. Good afternoon, and thank you for joining Sarepta Therapeutics' first quarter 2023 financial results conference call. Perhaps you will see this as a break from the past, but I intend to keep my remarks this evening brief. As you know, in a mere 10 days from now, we will be attending and presenting at the FDA Advisory Panel for SRP 9001, our gene therapy intended for the treatment of Duchenne muscular dystrophy. We believe that the primary areas of discussion at the advisory committee will be these, whether the totality of evidence supports the conclusion But SRP9001 dystrophin protein at the levels expressed by this therapy is reasonably likely to predict clinical benefit. The totality of the evidence will include natural history, the preclinical data, biomarker results, and the functional results from our clinical trials. The panel will also address the risk-benefit analysis associated with the administration of SRP9001 for the treatment of ambulatory patients with BMD, in the context of accelerated approval. And finally, an assessment of the ability to bring to conclusion, embark, or study 301, the proposed post-marketing confirmatory trial to support the accelerated approval of SRP 9001 in the event that accelerated approval is granted. The team is well-prepared and excited to share with the advisory committee the wealth of compelling evidence supporting the conclusion that SRP9001-distributed protein and the amounts expressed by that therapy is reasonably likely to predict clinical death. So to set expectations for this call, we intend to discuss first-quarter performance, and Dr. Luis Rodino-Klapek will provide a pipeline update. But to respect the process and in light of how soon the meeting will take place. We will not discuss or entertain questions on regulatory matters pertaining to SRP 9001 or the upcoming advisory committee meeting until it's concluded on May 12th. Moving now to the quarter, I am very pleased to announce another strong quarter of performance. First quarter total revenue came in at $253.5 million. Net product revenue came in at $231.5 million. That's a 23% increase over the same quarter of the prior year and exceeding analyst consensus. It is this very patient-oriented execution that we intend to apply to SRP 9001 if approved. We continue to progress our important post-marketing commitments for our three approved TMO therapies. We have already completed 11 of our post-marketing commitments. With respect to Essence, which is our two-year blinded placebo-controlled study for Vionis and Imondas, we were fully enrolled last year, and that trial is proceeding. With respect to Mission, which is our dose-ranging post-marketing commitment for Exondas, part two of that study is substantially enrolled and progressing. As relates to SRP 9001, in addition to preparing for the May 12 advisory committee and prosecuting the BLA with the May 29 PDUFA date in mind, we are ensuring that we will be prepared to successfully launch 9001 and serve the community if and when approved. By now, we have successfully concluded all FDA inspections. That includes three GMP inspections and two GCP inspections. With our partner, Catalan, we are producing material to successfully launch 9001 upon approval, and we are finalizing our launch readiness work. And we are finalizing our plans to commence the studies necessary to expand the 9001 label to the broadest population supported by the science, including our commencement of our non-ambulatory study in vision, or study 303, and our multiple studies to explore the removal of neutralizing antibodies to RH74. Beyond that, we've been advancing our broader pipeline, and Dr. Rodino-Clayfat will provide an update on our pipeline momentarily. Now, the coming weeks and months are monumentally important to the patients that we serve. As I have said many times by now, we stand at a bellwether moment. with the greatest evidence-based hope yet in history to bring a better life to families today living with and unfortunately today dying from Duchenne muscular dystrophy. And we are also well aware that this BLA stands as a bellwether test for gene therapy itself and for the ability to effectively lean in and use the tools available to us to translate groundbreaking genetic science to medicine that can extend and improve patients' lives now, not merely at some theoretical point in the future. We feel an enormous obligation to the patients that we serve, and our every decision and action is taken and done with that obligation front of mind. And with that, let me turn the call over to our head of R&D and chief scientific officer, Dr. Luis Rodino-Kleipach. Luis.