REGENXBIO Inc.

Welcome, everyone, to the first quarter 2025 Regenexx Bio Earnings Conference call. At this time, all participants are in listen-only mode. After the speaker's prepared remarks, we will conduct a question and answer session. To ask a question, please press star 1-1. To remove yourself from the queue, please press star 1-1 again. As a reminder, this call may be recorded. At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of Regenexx Bio. Please go ahead.

Good afternoon, and thank you for joining us today. Earlier this afternoon, Regenexx Bio released financial and operating results for the first quarter ended March 31st, 2025. The press release is available on our website at www.regenexxbio.com. Today's conference call will include forward-looking statements regarding our financial outlook in addition to regulatory and product development plans. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted and can be identified by words such as expect, plan, will, may, anticipate, believe, should, intend, and other words of similar meaning. Any such forward-looking statements are not guarantees of future performance and involve certain risks and uncertainties. These risks are described in the risk factors in the management discussion and analysis section of Regenexx BIO's annual report on Form 10-K for the full year ended December 31st, 2020, and comparable factors and sections of Regenexx BIO's quarterly reports on Form 10-Q, which are on file with the Securities and Exchange Commission and available on the SEC's website. Any information we provide on this conference call is provided only as of the date of this call, May 12th, 2025, and we undertake no obligations to update any forward-looking statements we may make on this call on account of new information, future events, or otherwise. Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially. I'll now turn the call to Curran Simpson, President and CEO of RegentsBio. Curran?

Thank you, Patrick, and thank you everyone for joining us today. We've had an impressive start to the year at Regenexx Bio and are in store for an exciting remainder of 2025. Today, you will hear about our strong late-stage clinical progress, which sets us up to potentially bring multiple first or best-in-class gene therapies to patients over the coming years. We will also discuss other key elements that we believe position us to successfully transition to commercial stage including our U.S.-based in-house commercial-ready manufacturing and broad potential to secure non-dilutive funds. With me on today's call are Dr. Steve Picola, our Chief Medical Officer, and Mitch Chan, our Chief Financial Officer. I'll start with a review of recent business highlights, then turn it over to Steve and Mitch for clinical and financial updates. Then I'll have a few closing remarks before opening the call for Q&A. Starting with our most advanced program, RGX121, or chlamydzogene lenparvovac, the potential first gene therapy and one-time treatment for MPS II or Hunter syndrome, a devastating disease that affects approximately 2,000 patients worldwide. Any day now, we expect the FDA acceptance of the BLA, which we submitted under the accelerated approval pathway in March 2025. RGX 121 remains on track for potential FDA approval in the second half of this year. Earlier this year, we established a key strategic partnership with Nippon Shinnyaku to commercialize our neurodegenerative franchise, including RGX 121 and RGX111 for severe MPS1. And commercial preparations are progressing well. Together with Nippon, our goal is to deliver RGX121 to patients beginning in the first half of 2026. Accelerating quickly, right behind RGX121 is RGX202, our next generation candidate for Duchenne muscular dystrophy. I am pleased to report that our pivotal study continues advancing rapidly. We have surpassed 50% enrollment for our pivotal data set. We're seeing increased interest and enthusiasm from the patient community about RGX202 and its differentiated profile, and we remain on track to submit a BLA in mid-2026 and seize our unique second-to-market or fast-follower opportunity in Duchenne. Positive input from our growing investigator community supports our belief that RGX2 has the potential to be a preferred and differentiated treatment option. It's important to keep in mind that, one, RGX202 is the only investigational next-generation DMD gene therapy in pivotal study and the only investigational therapy with both robust microdystrophin and functional data available. RGX202 has the potential to be approved for patients aged 1 to 3 who currently have no access to gene therapy. And last, DMD represents a large addressable market, with over half of the prevalent DMD population projected to remain untreated as of 2027, the expected year of RGX202's commercial launch. Given our strong and rapid clinical progress, conviction in our differentiated profile, and in recognition of the ongoing unmet need, we will begin producing RGX202 commercial supply at our Manufacturing Innovation Center here in Rockville, Maryland in the third quarter of this year. With full clinical and planned confirmatory supply already in hand, we will build commercial inventory to be prepared for a smooth launch and meet patient needs immediately upon the potential approval of RGX202. As a reminder, our Manufacturing Innovation Center is a state-of-the-art integrated GMP facility that can produce up to 2,500 doses of RGX202 annually, enough to treat approximately one-fifth of the estimated North American DMD population, all while delivering industry-leading purity levels in Duchenne with over 80% full capsules. We look forward to sharing additional Phase I-II functional data for RGX202 in the first half of this year as we aggressively advance towards commercialization. Moving to our retinal programs, we continue to work closely with our partner, Abbey, to progress ABBV-RGX314, or Serabgene Lamparvovac. 314 is advancing in two pivotal studies for subretinal wet AMD, one phase two study for supracoroidal wet AMD, and preparations for a pivotal program are underway in diabetic retinopathy, or DR, using supracoroidal delivery. We remain on track to be the first gene therapy on the market for wet AMD, with a product that has demonstrated compelling durability and strong patient interest. Both wet AMD and DR represent large, multibillion-dollar commercial opportunities, and we believe 314 has the potential to preserve vision and serve as a meaningful alternative to today's standard of care. In summary, we remain excited for and are well-positioned to deliver on the opportunities ahead of us. With that, I would like to now turn the call over to Steve for an update on our clinical program. Steve?