Sarepta Therapeutics, Inc.

Today, ladies and gentlemen, thank you for standing by and welcome to CERTA Territic's second quarter 2020 earnings conference call. At this time, all participants are on a listen-only mode. After the speakers prepare remarks, there will be a question and answer session. To ask a question during this session, you will need to press star 1-1. Please be advised that today's conference may be recorded. I would now like to end the conference with your speaker host today, Mary Jenkins, Senior Manager of Investor Relations.

Thank you, Livia, and thank you all for joining today's call. Earlier today, we released our financial results for the second quarter 2022. The press release is available on our website at Sarepta.com, and our 10Q was filed with the Securities and Exchange Commission earlier this afternoon. Joining us on the call today are Doug Ingram, Ian Esipan, Dallin Murray, and Dr. Louise Rodino-Klapak. 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 take a moment to read the 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 10-Q, filed with the SEC, as well as the company's other 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. 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?

Thank you, Mary. Good afternoon, everyone, and thank you all for joining Threat for Therapeutics second quarter 2022 Financial Results Conference Call. I will discuss our outstanding quarterly performance in a moment, but given it's important to the patients that we serve, to Sarepta, and in my view, to the entire field of gene therapy, I will begin by focusing on our progress this quarter with the largest near-term gene therapy opportunity in biopharma, and that's SRP 9001, our gene therapy for the treatment of Duchenne muscular dystrophy. As you will recall, we previously disclosed that we were engaging with the U.S. FDA about the possibility of submitting a biologics license application, or BLI, for the accelerated approval of SRP 9001 to treat Duchenne muscular dystrophy. We also cautioned numerous times that we would not change our base case assumption on the timing of approval unless we had strong conviction on the receptivity to an accelerated approval BLI by the FDA. As we announced last week, our discussions are now complete and our base case assumption has indeed changed. Over the course of the second quarter, we engaged with FDA in an in-depth review with the agency of the wealth of evidence that supports the safety and efficacy of SRP 9001 and the functional benefits associated with the robust expression of shortened but functional dystrophin when treated with SRP 9001. This included the safety and tolerability data unique to SRP9001, the preclinical of animal models supporting its benefits, the various function-related biomarkers associated with the nearly 90 patients dosed with SRP9001 across studies 101, 102, and 103, and the impressive and consistent functional results, which is, of course, NSAA and multiple time tests across those studies. I would like to thank the FDA for its time, its commitment, and its input as the review spanned multiple meetings and included input and guidance across the FDA, including CBER leadership, the Office of Tissues and Advanced Therapies, as well as the Center for Drug Evaluation and Research, including its Office of Neuroscience and Office of New Drugs. Based on that review and the written guidance provided to Sarepta last week, we announced that we intend to submit a BLA for accelerated approval of SRP 9001 to treat ambulatory patients that have Duchenne muscular dystrophy. We intend to compile and submit our BLA this fall. We will hear whether an advisory committee is requested by the FDA after our BLA is filed. If one is requested by the FDA, we would anticipate it in the spring of 2023, and we are planning and preparing for just that. Assuming a successful review, we anticipate approval and launch in mid-2023. To that end, in addition to completing and submitting our BLA, we are immediately readying ourselves for what will be the largest gene therapy launch in the United States. That will include augmenting our commercial and medical affairs organizations, site readiness, and importantly, building sufficient inventory to serve the community at launch without delay. As we prosecute our BLA, we continue to execute EMBARQ. That's our 120-patient pivotal trial for SRP 9001. Demand has been intense for EMBARQ, and site initiation and enrollment ramped enormously in the second quarter. Based on our current screen rate, we should be fully screened and enrolled in the next few weeks. One of the concepts that has been discussed both within the FDA and within Congress is is encouraging companies to have their confirmatory trial underway at the time of an accelerated approval. We anticipate Embark to act as our confirmatory trial for an accelerated approval, and Embark will be fully enrolled by the time our DLA is filed. Furthermore, we anticipate dosing an additional cohort in Study 103 to study what our research informs us are low-risk mutations in the currently excluded 1-17 range, with the goal to narrow the exclusions to a low single-digit percentage by the accelerated approval time we also intend to initiate a separate placebo-controlled non-non-ambulatory study later this year that study is designated study 303 or in vision on the rna platform encouraged by seeing 18 times greater exon skipping and eight times greater dystrophin production in half the time and one-fifth the dose of current standard of care, we initiated Momentum Part B, a pivotal trial for exon 5051, our next-generation RNA-based PPMO for the treatment of Duchenne patients amenable to skipping exon 51. While dosing continues globally, we announced in the quarter that FDA has placed a hold on dosing in the United States while we provide additional information and context around cases of hypomagnesemia. We have provided the requested information and we continue to guide to completion of enrollment for Momentum Part B in the second half of 2022. If confirmed in Momentum Part B, SLP5051 could be a profound improvement over current standard of care. So at the same time, we are progressing the preclinical work for additional PPMOs to treat a greater percentage of the Duchenne population. Our head of R&D and chief scientific officer, Dr. Luis Rodino-Claypack, will provide further color on the performance of our R&D pipeline. Now to quarterly performance. This afternoon, we announced another exceptional quarter of execution and performance as we continue to serve the Duchenne patient community with our approved, three approved therapies, Exondys 51, Myondys 53, and Demondys 45. Total revenue for the quarter was approximately $233.5 million. Net product revenue was $211.2 million, exceeding consensus and growing almost 50% versus the same quarter last year. Given our overperformance, we are now raising our full-year net product revenue guidance to between $825 million and $840 million. Summarizing. I could hardly be prouder of the Sarepta team for their tenacious execution this quarter. Even as we served the community with our currently approved therapies, we made what could be a monumental leap in the advancement of SRP 9001 to the broader patient community. My colleagues and I are confident that this work will translate into a better life for individuals and families living with Duchenne in the United States and around the world. I want to thank my Sarepta team. I want to thank our committed investigators and the patients who are participating in our studies. And I want to give a special thanks to the FDA for its time and guidance as we progress. All of these cohorts are necessary for the success of SRP 9001. And with that, let me turn the call over to our head of R&D and chief scientific officer, Dr. Luis Rodino-Clayfax.