REGENXBIO Inc.

Welcome everyone to the fourth quarter and year-end 2024 Regenexx BioEarnings conference call. At this time, all participants are in a 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 1 1 on your telephone. You will then hear an automated message advising your hand is raised. To withdraw your question, please press star 1 1 again. Please be advised that today's conference is being 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 fourth quarter and year ended December 31, 2024. 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 and the management discussion and analysis section of Regenexx BIOS annual report on Form 10-K for the full year ended December 31st, 2024, and comparable risk factor sections of Regenexx BIOS 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, March 13th, 2025. and we undertake no obligation 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 to any unaudited or pro forma financial information, that may be provided as preliminary and does not report 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 Regenexx

Thank you, Patrick, and thank you, everyone, for joining us today. 2025 is a transformational year for Regenexx Bio, and we're off to an exciting start. We've submitted our first BLA and expect our first FDA approval in the fourth quarter for RGX-121, which is our treatment for Boy's with Hunter's Syndrome. We will build on this momentum by advancing our diabetic retinopathy program into pivotal stage this year, then head into 2026 and 2027 with potential BLA filings for large opportunities, including RGX202 for Duchenne muscular dystrophy and ABBD RGX314 for wet AMD. We are in a strong position as we prepare to launch multiple first or best in class gene therapies and have robust commercial capabilities and global partners. This is all with a view to sustainable profitability and is the result of 15 plus years of gene therapy leadership. On today's call, I will review our recent business highlights and outline a vision for what we believe will be catalyst-rich years ahead. Then, our Chief Medical Officer, Dr. Steve Kola, will summarize our clinical progress before handing it over to Mitch Chan, Chief Financial Officer. Mitch will provide an overview of our financial results and share the many potential non-dilutive capital sources ahead. I'll then make some closing remarks before we open for Q&A. Starting with our recent exciting news in MPS, we are thrilled to have completed the submission of the BLA for RGX-121 or Chlamyd-Sagin-Lamparbovac under the Accelerated Approval Pathway and partnered with Nippon Shin'yaku for both of our MPS programs. This partnership marries our collective strengths, Regenexx Bio's development and manufacturing expertise with Nippon's experience in successfully commercializing rare disease products. Our teams are planning for potential approval of RGX121 for MPS2 in Q4 2025, and are working diligently to prepare for the commercial launch. Along with enabling access to these important medicines for patients in the U.S. and Asia, this partnership is strategically significant to Regenexx Bio. As Mitch will share, this agreement provides meaningful potential milestones and revenue for us. The potential approval of RGX 121 also provides strategic value for the rest of our pipeline, as we'd receive commercial licensure of our manufacturing facility prior to launching in our larger opportunities, including RGX 202 for Duchenne. Moving to RGX 202, I am pleased to report that our pivotal study is advancing rapidly and that our unique second to market or fast follower opportunity is on track for a mid-2026 BLA filing. Recent updates have indicated tremendous interest in the patient community for new treatments with RGX202 as a valued option. Input from our growing investigator community supports our belief that RGX202 has the potential to be a preferred and differentiated treatment option. I'll remind you that RGX202 is the only investigational next-generation DMD gene therapy in pivotal study and with both robust microdystrophin and functional data available. The pivotal trial of RGX202 is rapidly enrolling approximately 30 ambulatory patients aged one and over. I'm pleased to share that this pivotal trial is nearly half enrolled, and we expect to complete enrollment this year. We are also on track to submit a BLA under the accelerated approval pathway by mid-2026. We expect more than half of the prevalent population to remain untreated through the next few years, And this large population will need more than one treatment to serve all Duchenne patients. That's why we're confident in the rapid progress we've made thus far and our path to delivering a potentially preferred gene therapy option. Also, let me remind you that we are commercial ready when it comes to manufacturing 202. Our in-house state-of-the-art suspension-based bioreactor process is currently producing 202 for our pivotal study, with industry-leading purity levels of more than 80% full capsules. Our Manufacturing Innovation Center can produce 2,500 doses of RGF202 per year. As we continue to aggressively accelerate BLA-enabling activities and commercial planning, we will share more meaningful updates. Specifically, we plan to share additional positive Phase I-II biomarker data later this month at MDA, as well as updated Phase I-II functional data in the first half of this year. With positive biomarker and functional data in hand, encouraging interactions with FDA and commercial-ready manufacturing, we believe our second-to-market position in Duchenne remains strong. Lastly, I will highlight a few updates on ABBV-RGX314, or SUROP gene lamparvovac. This is our global AbbVie-partnered retinal franchise that is advancing in late-stage studies. As announced with AbbVie in January, data from the pivotal studies evaluating subretinal 314 in patients with wet AMD are expected in 2026. We expect to complete enrollment of both of these pivotal studies this year. In our diabetic retinopathy program, evaluating in-office superchoroidal delivery of 314, we held a successful end-of-phase two meeting with the FDA in the fourth quarter of 2024. We are now planning a pivotal program with AbbVie to support future global regulatory filings. As we have said before, wet AMD and DR are very large commercial opportunities, and 314 represents a potential alternative for the patients losing vision with today's standard of care. Importantly, we will be entitled to additional milestone payments that are part of this $1.8 billion collaboration with AbbVie. In summary, we are excited for and well-positioned to deliver on the opportunities ahead of us to drive values, for patients and shareholders. With that, I would now like to turn the call over to Steve for an update on our clinical programs. Steve?