Sarepta Therapeutics, Inc.

Good afternoon and welcome to the Sarepta Therapeutics fourth quarter and full year 2022 earnings call. At this time, all participants are on a listen-only mode. After the speaker's presentations, there will be a question and answer session. To ask a question at that time, please press star 11 on your telephone. As a reminder, today's conference call is being recorded. At this time, I will turn the call over to Mary Jenkins, Associate Director, Investor Relations. Please go ahead.

Thank you, Valerie, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the fourth quarter and full year 2022. The press release is available on our website at Sarepta.com, and our 10-K 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-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 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 Seraptus 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 Seraptus 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-K, 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. I'll now 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, everybody, and thank you for joining Sarepta Therapeutics for our fourth quarter and full year 2022 financial results conference call. 2023 may be the most eventful year in Sarepta's event-filled history. Our biologics license application or BLA for our gene therapy SRP 9001 for Duchenne muscular dystrophy will be a significant bellwether moment. First, for the Duchenne families who are waiting without the luxury of patience for a therapy that might arrest the brutal decline associated with this disease. And next, for the promise of gene therapy as a class to deliver meaningful improvements in the lives of patients with rare degenerative diseases, not at some distant vanishing point in the future, but in time to do good now. And given the keen interest in the 9001 BLA submission, I'm going to comment on the BLA review before I move to quarterly and yearly performance. As you will recall, in the fall of last year, we submitted our BLA for SRP 9001 to treat ambulant Duchenne patients, and in the fourth quarter, the FDA accepted the BLA for filing, granted 9001 priority review, and set May 29, 2023 as our action date. We have been diligently prosecuting the BLA. We have by now completed a very productive mid-cycle review with the divisions. At the mid-cycle, the division requested additional CMC information, and we have by now provided answers to all of those questions. The division formally informed us as well at the mid-cycle meeting that they see no significant safety issues that have been identified. And the division has determined that there is no need for an advisory committee meeting for SRP 9001BLA. As you may have read, FDA has announced that the Office of Tissues and Advanced Therapies, or OTAT, is being reorganized and a new super office entitled the Office of Therapeutic Products, or OTP, is being established. The goal is to improve alignment, increase resources and capacity, and enhance expertise. A couple of thoughts. First, Sarepta is delighted by the establishment of OTP. We believe it will enhance reviews and serve Dr. Peter Marks' publicly stated vision to lean in and accelerate the transformative potential of cell and gene therapy. Second, FDA has stated that the reorganization and establishment of OTP will not impact any timelines. And we can confirm that we have not experienced any delay or disruption of any kind as a result of this reorganization. Indeed, we see the establishment of OTP as unequivocally positive and a potentially great benefit to patients. So going forward, we're going to focus on the following. First, answering any remaining questions the FDA may have on the file. Second, preparing for and managing pre-approval inspections. The FDA has already scheduled three pre-approval manufacturing inspections, and along with our manufacturing partner, Catalent, we are preparing to make those inspections a success. Third, building inventory for launch. And fourth, of course, completing our launch readiness. Now, I've provided an update on the mid-cycle today, as there has been a significant amount of interest in whether the division would see the need for an advisory committee. However, from here, we will be focusing on prosecuting the BLA and will provide an update on or after the 9001 action date. Moving now to performance. As one ponders how Sarepta may execute the launch of 9001, if given that opportunity, I would ask you to consider the team's consistent performance quarter over quarter and year over year, serving the Duchenne community with our three approved therapies, Exondys, Vyondys, and Amondys. From an in-market perspective, 2022 was yet another year where our cross-functional team, including commercials, medical affairs, patient services, access and reimbursement, and manufacturing and supply chain, to name a few, executed together and delivered for our patients. Fourth quarter total revenue stood at $258.4 million, while net product revenue came in at $235.9 million. That is a 32% increase over the same quarter of the prior year. Full year total revenue came in at $933 million, and net product revenue for the year came in at $843.8 million, representing a 38% year-over-year increase. For the last five years, we have grown at a consistent 40% compounded annual growth rate, all of which performance comes from serving the Duchenne community and none of which comes from price increases. Now, as we announced at the JPMorgan conference in January, for 2023, our net product revenue guidance for our three currently approved PMO therapies, excluding the impact of a 9001 approval, is $925 million or greater. Moving back to SRP 9001 for a moment, we are commencing studies this year to ensure that we have the broadest label for SRP 9001 as is possible, consistent with the science. We have already commenced our study to limit mutation-related exclusions. Further, in the coming months, we will be starting a study in the non-ambulant population called Envision, or Study 303. And we will commence two separate studies with alternative approaches to removing pre-existing antibodies to make SRP9001 available to R874 NAB-positive patients as well. Additionally, we are making significant progress with our limb-girdle pipeline, as Dr. Luis Rodino-Klapak will discuss in a moment as she provides updates across our research and development activities. On the RNA platform, we will complete enrollment of the Momentum study this quarter. Momentum, as you know, is our study for our first next-generation peptide-conjugated PMO, also known as a PPMO. and that's SRP 5051 designed to treat Duchenne patients who are Exon 51 amenable. Momentum will read out later this year, and if successful, we will discuss a filing for SRP 5051 with the neurology division this year. I am proud of the progress that we have made these last six years, and yet it pales in comparison for the good that we can do in the coming years together. We have the potential to improve the lives of countless patients and to greatly reward those who have been committed to and invested in this mission. Indeed, the opportunity in front of us is breathtaking. To realize that opportunity, we will need laser-focused and tenacious execution, but as Sarepta has proven time and again, this is a team that knows how to execute. And with that, let me turn the call over to our Head of R&D and Chief Scientific Officer, Dr. Luis Rodino-Klapak.