REGENXBIO Inc.

Thank you for standing by, and welcome to Regenexx BIO's fourth quarter and year-end 2025 earnings conference call. At this time, all participants are in a listen-only mode. After the speaker presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 11 on your telephone. To remove yourself from the queue, you may press star 11 again. I would now like to hand the call over to Patrick Christmas, Chief Legal Officer of Regenexx Bio. Please go ahead.

Good morning, and thank you for joining us today. Earlier this morning, Regenexx Bio released financial and operating results for the fourth quarter and year ending December 31, 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 and the Management's Discussion and Analysis section of Regenexx BIO's annual report on Form 10-K for the full year ended December 31, 2025, and Comparable Risk Factors section of Regenexx BIO's quarterly reports on Form 10-Q, which will be 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 5, 2026. 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 in 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 Regenexx Bio. Curran?

Thank you, Patrick. Good morning everyone and thank you for joining our call today. 2026 is set to be a pivotal year for Regenexx Bio. With great focus on advancing our late-stage pipeline in 2025, we enter the year with near-term top-line Phase III readouts and ongoing commercial readiness activities in Duchenne muscular dystrophy and wet AMD. Last but not least, we are also entering the pivotal Phase 2B-3 program named Navigate for diabetic retinopathy being developed in collaboration with our eye care partner, AbbVie. Our clear focus on execution in 2025 has led to a robust set of important catalysts to transform Regenexx Bio from a late-stage development organization to a commercial entity. Today, I am joined by Dr. Steve Piccola, our Chief Medical Officer, who will walk through the ongoing clinical advancement of our programs, and Mitch Chan, our Chief Financial Officer, to provide our financial updates. With that, let me start with RGX202, our late-stage Duchenne program. I'm pleased to report that momentum for RGX202, our potential best-in-class gene therapy for Duchenne, remains strong. We completed dosing in our pivotal study last fall and are seeing robust enrollment in our confirmatory trial, reflecting continued enthusiasm from the Duchenne community. Our growing clinical data set, including the 18-month Phase 1-2 NSAA results shared in January, demonstrate meaningful differentiation from the known natural history of Duchenne across multiple validated measures. Combined with a favorable safety and biomarker profile to date, we believe RGX202 has the potential to deliver durable, clinically meaningful benefit. With less than 1% of the global Duchenne population having received an approved gene therapy and no approved option for patients ages 1 to 3, the unmet need remains significant. We look forward to sharing additional Phase 1-2 data next week at the Muscular Dystrophy Association's annual meeting and top line data from our pivotal study early in the second quarter as we advance towards a BLA submission. We look forward to engaging with FDA midyear to discuss our planned BLA submission using the accelerated approval pathway. We have FDA engagement planned ahead of the pre-BLA meeting and intend to support our primary micro dystrophin endpoint with significant functional data, both in the pre-BLA meeting and at the time of submission. By the fall of 2026, we will have 12-month functional data on the majority of pivotal trial patients. In addition, with the enrollment momentum in our confirmatory study, our safety database continues to expand. In our retinal disease programs, our partnership with AbbVie continues to progress. Top line data for subretinal Cirovec in wet AMD are expected in Q4 this year. Atmosphere and ascent represent the largest global gene therapy program in this indication, and if approved, Cirovec would be the first gene therapy for wet AMD. I'm also pleased to share that site activation plans are also underway in the Navigate Pivotal Study in Diabetic Retinopathy with first patient dosing expected next quarter, triggering a $100 million milestone for MADVI. Finally, turning to our MPS programs, since receiving the CRL for RGX121 two weeks ago, We have also received the clinical hold letters for both RGX-111 and RGX-121. We believe that the requirements to remove the holds are addressable and, in fact, had already been in the process of addressing them. We continue to work on the CRL response and working towards a Type A meeting with the goal of resubmitting the BLA. We remain committed to the NPS community and the Hunter Syndrome patients waiting for a treatment for this devastating disease. As we enter 2026, our focus is clear, execute against our key milestones, and bring the hope for transformative gene therapies closer to patients in need. With that, I'll turn it over to Steve.