Sarepta Therapeutics, Inc.

Good afternoon and welcome to this Repta Therapeutics second quarter 2023 earnings call. At this time, all participants are in listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you'll need to press star 11 on your telephone. If your question has been answered and you'd like to remove yourself from the queue, simply press star 1-1 again. As a reminder, today's program is being recorded. At this time, I'll turn the call over to Francesca Nolan, Executive Director, Investor Relations and Corporate Communications. Please go ahead.

Thank you, Jonathan, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the second quarter of 2023. The press release is available on our website at Sarepta.com. And our 10Q 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. 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 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 Sarepta's control. Actual results can materially differ from these forward-looking statements, and any such risk can materially and adversely affect the business, the results of operations, and trading prices for Sarepta'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 obligation to publicly updated forward-looking statements, including any financial projections provided today based on subsequent events or circumstances. And now we'll turn the call over to our President and CEO, Doug Ingram, who will provide an overview of our recent progress. Doug?

Thank you, Fran. Good afternoon, and thank you for joining our second quarter 2023 financial results conference call. The second quarter of this year was perhaps the most consequential period in the long consequential history of Sarepta. For families living with Duchenne, it was far more than that still. Buddy Cassidy, living with Duchenne and an Elevitus Advisory Committee member, shared the collective view of the Duchenne community on the June approval in words far more eloquent than I could possibly muster. As Buddy said, and I quote, there is more to do. But now let us pause and bask in the glow of our achievement. Let us bask in gratitude. Let us pause in celebration. Let us watch as dawn rises and brings in the day." In May of this year, the FDA held an advisory committee meeting on our gene therapy SRP9001, now called Alevitus. With a majority vote, the advisory committee recommended the approval of Alevitus for the treatment of ambulatory patients with Duchenne muscular dystrophy. On June 22, the FDA granted an accelerated approval for a levitis to treat Duchenne muscular dystrophy, currently labeled for four- and five-year-old patients. At the time of the approval, FDA leadership informed Sarepta and the patient community that if our confirmatory trial in BARC meets its objectives, the FDA will, in the words of Dr. Peter Marks, move with maximum speed following submission of the data to the agency, minimizing any impediments to review results rapidly, and broadening the label by removing any age restrictions. So we have our path. Our confirmatory trial is well designed and powered to show a statistically significant benefit in the studied population. For the avoidance of doubt, we have powered this study to show a benefit in the studied population, that's four to seven years old, And with that, we will have confirmed the mechanism of action in Duchenne patients applicable to all age ranges. We will have the top line for Embark in the fourth quarter of this year. We will announce top line results as we submit them to the division, followed shortly by a label supplement. If successful, we should be able to expand our label in the first half of 2024 to include the majority of Duchenne patients. Additionally, we have already commenced our non-ambulatory trial in vision or study 303, and we intend to commence two trials with alternative approaches to cleaving or clearing pre-existing antibodies. Our goal is to expand our label for Alevitus to cover as much as 95% of the Duchenne patients. In a moment, our Chief Customer Officer, Dallin Murray, will provide details on the early days of the Alevitus launch. Elevitus is our fourth approved Duchenne therapy, and we have been very successful with all of our prior launches. Consistent with our track record, the Elevitus launch is going well. A significant number of sites are now initiated and ready to infuse. Payer discussions and negotiations have been more positive than our prior successful therapies. And there is a significant amount of enthusiasm from physicians and families as we have a substantial number of start forms and more coming weekly. In fact, I am pleased to announce that the first reimbursed Alevitus infusion occurred earlier today. That is faster than we projected and speaks to the team's ability to execute. Now, as positive as this is, please bear in mind that the significant rampant infusions will begin later this year. This is primarily due to payer logistics and to the release protocol for Alevitis, which requires that both we and the FDA release each lot. Shortly, our head of research and development and chief scientific officer, Dr. Luis Rodino-Clayback, will provide an overview of our development activities, including progress with our next generation PPMO SRP 5051 and on the gene therapy side with our LGMD portfolio. To remind you, in addition to Alevitis, we have a deep pipeline and are advancing a number of therapies in 2023. In fact, we have 24 ongoing human clinical trials by the end of this year. And we continue to advance our next generation viral capsid, myoAAV, which in early animal models looks to be as much as tenfold more tropic than current AAVs. From a manufacturing perspective, our technical operations professionals, more than 400 in all, are focused with our partner, Cavalent, on producing and releasing lots to support our launch. Looking to the midterm, we will also continue to work to establish comparability at our Thermo Fisher plant for Elevitus. And then looking to the future, we are advancing our approach to manufacturing to support our entire pipeline. In fact, we have made great progress in next generation suspension technology which we will use across our gene therapy portfolio, including the LGMD programs, and even potentially for Alevitus. In fact, we have largely completed our suspension-based process development for Alevitus, and we have made three runs at 250 liters with a fourth run to occur later this year with the goal of scaling to 1,000 liters in the near term. We have seen yields that are multiples greater than the current standards. As the leader in gene therapy for rare diseases, we will continue to focus on improving the science and the delivery of our therapies. Moving finally to quarterly performance, we have had another strong quarter with our three approved PMOs, Extondis, Amondis, and Vyondis. I am pleased to announce that second quarter total revenue came in at $261.2 million, and net product revenue from our PMOs came in at $239 million, exceeding internal estimates and analyst consensus. In the second quarter, we saw no negative impact on the approved PMOs from any warehousing related to the elevitous approval and do not anticipate substantial impact over the course of the year. We remain comfortable with our full-year guidance of $925 million for total PMO, net product revenue, with a current bias to modestly exceeding that guidance. It is too early in the launch to provide an accurate guidance for levitis, but we will continue to evaluate that. At Sarepta, we have a distinct culture. It is easy to say one is patient-focused, but our words are backed by action. We put the welfare of our patients above all other considerations, and when necessary, we fight for them. And the tools we use for that fight include our commitment to scientific excellence, and the courage to represent the patients who depend upon us. Our fight may not always be easy, but it is paying off. We have four approved therapies and a wealth of potentially life-enhancing programs in our pipeline. We will achieve $925 million on the first three of our four approved therapies this year and far more next year when our newest approval is considered. We have a near-term high probability of success plan to broaden the label of the first and only gene therapy for Duchenne. And if our plans are successful, we could be profitable in the next few quarters. We have done a lot already, but with our plans, our pipeline, and our commitment to dogged execution, we have only just begun. And with that, I will turn the call over to Dr. Rodino Clayback, who will provide an update on our research and development progress. Luis?