REGENXBIO Inc.

Welcome everyone to the second quarter 2025 RegeneX Bio earnings conference call. If you would like to ask a question during this time, simply press star followed by the number one on your telephone keypad. If you would like to withdraw your question, again press the star one. Please note, slide reference during this call are available in the webcast and at the events page of RegeneX Bio's website. At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of RegeneX Bio. Please go ahead.

Good morning and thank you for joining us today. Earlier this morning, RegeneX Bio released financial and operating results for the second quarter and the June 30th, 2025. The press release is available on our website at .regenxbio.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's discussion and analysis section of RegeneX Bio's annual report on Form 10-K for the full year ended December 31, 2024, and comparable risk factor sections of RegeneX 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, August 7, 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 RegeneX Bio. Curran?

Thank you, Patrick, and thank you everyone for joining us. I hope you're having a great summer. Today I'm pleased to be joined by Dr. Steve Picola, our Chief Medical Officer, and Mitch Chan, our Chief Financial Officer, to review the exceptional progress being made by the RegeneX Bio team as we prepare for our first commercial approval this year and multiple potential near-term product launches. Before we begin, I'd like to acknowledge the Duchenne community and the recent events they have faced. We understand these have been incredibly challenging and uncertain times and express our deepest sympathies to the families experiencing loss. These recent events have only reinforced our strong commitment to deliver RGX202 as a potential -in-class gene therapy for Duchenne. We believe that RGX202 has the potential to safely provide strong functional benefit and durability, offering hope for better lives. With positive and growing interest from the patient community and physicians, I am very pleased to share that we are accelerating our guidance for RGX202. We now expect to complete enrollment in the ongoing pivotal study this October. Our ability to move this timeline up from end of year further solidifies RGX202's position as the potential next gene therapy to market for Duchenne. The community is clearly in need of additional options. We continue to actively enroll and open new sites in the Affinity Duchenne Pivotal Trial with plans to roll directly into a confirmatory study to support our accelerated approval. Additionally, we are in the unique position of having drug supply in hand for our pivotal and confirmatory trials. We remain on track to report top-line data in early 2026, submit a BLA in mid-2026, and potentially be on the market by 2027 when the vast majority of the DMD population is expected to remain untreated. From the beginning of this program, we implemented a differentiated therapeutic approach. We pioneered a proactive immune suppression regimen to drive improved safety outcomes and reduce the potential risk of known liver issues found in other programs. Our Phase 1-2 data to date supports that our approach is effective. RGX202 also has consistent industry-leading purity levels in Duchenne with over 80% full capsid content in our product. This is important because higher purity leads to less total vector load delivered to patients and may contribute to the positive safety profile we've seen to date. We believe our in-house manufacturing capabilities are a highly strategic asset and gives us a unique ability to serve the large Duchenne market available at launch. We are pleased to announce we are initiating commercial manufacturing this fall at our Manufacturing Innovation Center here in Rockville, Maryland. This GMP facility can produce up to 2,500 doses of RGX202 per year. With our strong momentum and it's the only investigational gene therapy enrolling in a Phase 3 study in North America, RGX202 is well positioned to be next to market. And a potential -in-class gene therapy for Duchenne. Let's turn our focus to our retinal disease franchise. In partnership with AbbVie, we continue to advance ADDV-RGX314, also known as Serafgene Lomparvovac or ShuraVac, as the potential one-time gene therapy for chronic retinal conditions. I am very pleased that earlier this morning we announced an update to our agreement with AbbVie to advance ShuraVac into pivotal phase for diabetic retinopathy. Based on positive two-year data from the Phase 2 Altitude Trial, we will initiate a Phase 2b-3 trial. The cost of the study will be covered by the accompanying milestones, including the $100 million we receive upon the first patient dosed in the Phase 2b portion of the trial. Stephen Mitchell will share more about the data and financials shortly, but let me reiterate our excitement about this program, which gives us another pivotal program in chronic eye care with our partner AbbVie. Both wetAMD and DR represent large, multibillion-dollar commercial opportunities, and we believe ShuraVac has the potential to preserve vision, prevent disease progression, and serve as a meaningful alternative to today's standard of care. Last but not least, the FDA accepted our BLA for RGX121, now known as Chlamyzzogene Lamparvovac, for the treatment of MPS2 or Hunter syndrome. This BLA was accepted under the Accelerated Approval Pathway with a target PDUFA date of November 9. Pre-BLA activities are progressing well. We have completed the mid-cycle review meeting and our first PLI and BMO inspections with the FDA successfully with no observations. These are exciting achievements and a testament to the exceptional quality of our people, process, and science. If approved, RGX121 will be Regenexx Bio's first approved gene therapy. Commercial preparations with our partner Nippon Shinjaku are progressing well. Products intended for launch has already been produced, and we are committed with our partner to bringing this potentially transformative treatment to patients in early 2026. These accomplishments demonstrate our seamless and focused execution against our strategy to bring potentially transformative gene therapies to patients. We remain excited for and well positioned to deliver on opportunities ahead of us. With that, I would like to now turn the call over to Steve for an update on our clinical programs. Steve?