Sarepta Therapeutics, Inc.

and full year 2024 financial results conference call. As a reminder, today's program is being recorded. At this time, I'd like to turn the call over to Mary Jenkins, Associate Director, Investor Relations and Corporate Communications. Please go ahead.

Thank you, Lisa, and thank you all for joining today's call. Earlier this afternoon, we released our financial results for the fourth quarter and full year 2024. The press release, slides, and supplementary information are available on the investor section of our website at Sarepta.com. Our 10-K will be filed with the SEC later this week. 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 were making a number of forward-looking statements. Please refer to slide two on the webcast, which contains our forward-looking statements. These forward-looking statements involve risks and uncertainties, many of which are beyond surreptitious 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 surreptitious common stock. For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent 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. As noted on slide three, we will discuss non-GAAP financial measures on this webcast. Descriptions of these non-GAAP financial measures and reconciliations of GAAP to non-GAAP are included in today's press release and the slide presentation available on the investor section of our website. 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 for joining Threat to Therapeutics' fourth quarter and full year 2024 financial results conference call. As you may know, back in late 2017, we created an expansive strategic plan that we called Project Moonshot, one that would require that we raise billions of dollars, chart a course through the unknown, and overcome multiple unforeseen obstacles, obstacles that would have deterred a less committed team, but also a strategic plan, if achieved, which would ensure a better life for thousands of Duchenne patients and cement the durable future of Sarepta. After years of work and reams of new insight into the treatment of Duchenne in 2024, we achieved all of the goals laid out in Project Moonshot. We obtained approval for the broadest possible label for Alevitus, achieving our goal of a transformative therapy that could serve the vast majority of Duchenne patients. We worked with the FDA to reframe the approach to the development of rare and ultra-rare diseases so that therapies previously rendered unviable by FDA requirements now have the potential to help patients. And as a result, our LGMD franchise is moving with renewed speed towards three potential approvals over the next three years. We broadly launched 11 in 2024 and achieved results greater than all other in vivo gene therapies combined. At the same time, we continue to serve the community with our PMOs, which grew even in the face of a robust gene therapy launch. We became sustainably profitable and cash flow positive in 2024. Indeed, by the end of 2030, we will do over $16 billion cumulatively in operating income and over $13 billion in free cash flow, and this is after we have closed and made our initial investment in the Arrowhead transaction. we have secured a durable and even more diversified future through our Arrowhead collaboration, pursuant to which we now add to our gene therapy pipeline a broad platform of siRNA, including four clinical programs, three preclinical programs, and six research programs across neuromuscular, CNS, cardiomyopathy, and pulmonary. In addition to multiple gene therapy launches in the coming few years, we will have multiple potential blockbuster siRNA launches before the end of this decade. Now speaking more specifically of performance, we had an exceptionally strong fourth quarter and full year performance with our four approved therapies. For the fourth quarter, our net product revenues stood at $638 million, growing at 75% over the same quarter prior year. This represents more than $60 million in over performance to our prior guidance. Our full year net product revenues stood at $1.8 billion, representing year-over-year growth of 56%, and this overperformed to our guidance by over $100 million. Turning to Alevitas, in 2024, we had by a wide margin the most successful launch of a gene therapy yet in history. For the fourth quarter, Alevitas sales stood at $384 million, representing a 112% increase over the prior sequential quarter. And while we have already achieved over $1 billion in sales since our initial approval in 2023, this represents less than 5% of the on-label addressable opportunity, so clearly this is just the beginning. Our three PMOs, Exondus 51, Amondus 45, and Biondus 53, achieved $254 million in the fourth quarter and $967 million for the full year. We were profitable on a gap and a non-gap basis in the fourth quarter, and we were cash flow positive. Dallin Murray, our chief customer officer, will provide more color on commercial performance in a moment. And then to round out the call, our CFO, Ian Estepan, will discuss our financials in more detail. Looking forward, in 2025, we will capitalize on the successes of 2024. As you know, our net product revenue guidance for 2025, excluding royalties from Roche, is between $2.9 billion and $3.1 billion, approximately one-third of which will come from the PMOs and a bit over two-thirds of which will come from Alevitus. This represents year-over-year growth of 70% for total net product revenue, and for Alevitus, this represents over 160% year-over-year growth. On the R&D and tech ops sides, we have more upcoming milestones this year than any other time in our history. As you know, We already met our important Alevitis milestone in late January. We reported the two-year and one-year crossover results for Alevitis from our pivotal trial in bark and in all pre-specified measures. That includes all functional measures, muscle health, and biomarkers. Those on Alevitis did strongly statistically significantly better than untreated natural history would have predicted. We have passed 600 patients now on therapy across a broad range of ages and weights. These data are further proof of the transformative potential of Alevitis to change the future course of this disease for patients. To continue to serve the global market and improve COGS, we are planning for a move to suspension manufacturing. And after completing our GMP runs, we intend to commence our suspension manufacturing bridging study this year. As noted, we are now moving rapidly with our LGMD platform. We are continuing to dose our study for SRP9004 to treat LGMD type 2D, and we have initiated our registration study for SRP9005 to treat LGMD type 2C. We have completed dosing in our pivotal trial for SRP9003 to treat LGMD type 2E, and we will be submitting our VLA for approval later this year. We also intend to file our IND for SRP 9010 to treat LGMD type 2A later this year. If successful, we will launch SRP 9003 next year. We will launch SRP 9004 the following year, and SRP 9005 will be launched the year after that. As you know, we have closed our transaction with Arrowhead this month, and already there will be several very significant milestones in 2025. This year, we will have our initial readout for cohorts for arrow Dux4 to treat FSHD type 1. Again, this could be a very significant proof of biology. We will be looking at muscle concentration, downstream gene correction, and safety. We're also very excited about the potential to directly measure knockdown of the aberrant protein of interest Dux4. No other program to date has been able to achieve this, and this would be a very powerful result if successful. Likewise, we will have a readout of our initial cohorts for arrow DM1 to treat DM1, where we could obtain proof of biology looking at muscle concentration, knockdown, gene splicing, and safety. If successful, we could launch our therapy for DM1 in 2029. Our clinical programs for MMP7 and SCA2 are progressing well as well. We intend to initiate our first in-human clinical study before the end of this year for AeroHTT, which is intended to treat Huntington's disease. And finally, we have a significant number of siRNA and gene therapy pipeline programs underway, and so we intend to schedule an R&D day later this year to discuss all of them in more detail. Our head of R&D and chief scientific officer, Dr. Luis Rodino-Klapack, will discuss our pipeline progress in a moment. With grit and operational excellence, we achieved all of our multi-year objectives in 2024. In 2025, we will continue to execute. We will build on our foundation as one of the very few fully integrated, profitable, and cash flow positive biotechs in the world today, and we will achieve our next ambition, which is to become the next globally relevant big biotech focused on improving the lives of those among us with life-limiting rare disease. And with that, I will turn the call to Dallin Murray, for more detail on commercial performance and plans. Dallin?