REGENXBIO Inc.

Welcome, everyone, to the third quarter 2025 Regenexx Bio earnings 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 this session, please press star 11 on your telephone and wait for your name to be announced. Please limit yourself to one question and one follow-up, And if you have additional questions, please return to the queue. And to withdraw your question, please press star 11 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 morning, and thank you for joining us today. Earlier this morning, Regenexx Bio released financial and operating results for the third quarter ended September 30th, 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 RegenicBio's annual report on Form 10-K for the full year ended December 31st, 2024. and comparable risk factor 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, November 6, 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, 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, and thank you, everyone, for joining us today. I am pleased to share the strong momentum across our late-stage pipeline of gene therapies with the potential to deliver the best outcomes in devastating diseases. Our progress is anchored in our leading end-to-end capabilities, including in-house commercial-ready manufacturing and innovative science, all driven by our commitment to bring new medicines to patients in need. Today, I am joined by Dr. Steve Licola, our Chief Medical Officer, to discuss the exciting clinical development across our rare and retinal franchises, and Mitch Chan, our Chief Financial Officer, to provide financial updates. Let's dive in and start with RGX202, our wholly owned program for Duchenne muscular dystrophy. We are very encouraged by our progress in this program, and are moving rapidly to deliver a much needed differentiated treatment to Duchenne patients. Our comprehensive therapeutic approach behind RGX202 includes a novel construct. RGX202 is the only micro dystrophin that includes the CT domain, making it closest to naturally occurring dystrophin. A novel immune suppression regimen designed to enable high dose delivery and proactively counter issues seen in other AAV programs. And commercial-ready manufacturing that is delivering the highest purity levels in Duchenne gene therapies. Boys with Duchenne have one chance at gene therapy. That's why we specifically designed RGX202 with these elements to maximize opportunity for functional benefit. We are very pleased to see that this approach is resulting in the favorable safety and efficacy profile seen in Phase 1-2. We are also very excited to announce that we completed enrollment in the Affinity Duchenne Pivotal Trial, putting us on track to share top-line pivotal data in early Q2 2026 and submit a BLA using the accelerated approval pathway in mid-2026. To meet demand from the patient community and support accelerated approval plans, the confirmatory trial is open and continues to enroll patients. As a reminder, we were able to complete enrollment ahead of our original year-end guidance, underscoring the community's enthusiasm for RGX202 and strong need for new treatment options. We have more than enough supply of RGX202 ready and available for the entire confirmatory study. Delivering on the commercial readiness plans we shared last quarter, we have produced the first batches of RGX202 intended for commercial supply at our Manufacturing Innovation Center here in Rockville and expect to imminently complete our PPQ or Process Performance Qualification campaign. There is a significant commercial opportunity for RGX202 due to our novel construct, immune suppression regimen, and our manufacturing capabilities, including our ability to produce 2,500 doses per year, which make us uniquely prepared for clinical and commercial success. We continue to invest in preparations for a commercial launch in 2027, when the vast majority of the prevalent population will remain available. Now to RGX 121, also known as Chlamydia Gene Lamparvovac, the potential first gene therapy and one-time treatment for MPS II. Our recent FDA interactions regarding the ongoing BLA have been highly productive, and we remain confident in RGX 121 approval by early next year. We delivered positive 12-month data to FDA, which Steve will discuss in more detail. And the FDA completed inspections of our clinical sites and in-house manufacturing facility with no observations, a rare and significant achievement. These developments have further strengthened our confidence in the ongoing BLA review. With our commercial MPS partner, Nippon Shinnyaku, we look forward to the February 8th PDUFA date and delivering the first commercial doses of RGX121 by early next year. Let's turn our focus to our retinal disease franchise and our ongoing partnership with AbbVie to develop Surabgene Lamparvovac, or SuraVac, for wet AMD and diabetic retinopathy. These programs have the strength of AbbVie's leading clinical and commercial global eye care infrastructure behind them. In subretinal wet AMD, we recently announced that the last patient was enrolled in our two global phase three studies. This is a tremendous accomplishment. Together, Atmosphere and Ascent represent the largest global gene therapy program ever conducted, with over 1,200 patients enrolled across 200 sites. If approved, subretinal CERVEC would not only be the first gene therapy for wet AMD, a disease that impacts millions worldwide, but also the first gene therapy for a non-rare indication. We look forward to sharing top line data in the fourth quarter of 2026. As a reminder, we manufacture all clinical and future commercial Cirovec at our facility here in Rockville. Like WET-AMD, our diabetic retinopathy program is designed to deliver Cirovec to patients that rely on frequent lifelong injections to halt degeneration and vision loss. And we look forward to initiating the phase 2B slash 3 pivotal program. Across our pipeline, these achievements reflect the strength of focused execution and a differentiated approach to developing and delivering best-in-class therapeutics. With that, I would like to now turn the call over to Steve for more in-depth updates on our clinical programs.