REGENXBIO Inc. 2026 Q2 Earnings Call

Please go ahead. Good morning and thank you for joining us today.

Earlier this morning, REGENXBIO released financial and operating results for the second quarter ended June 30, 2026. The press release is available on our website at www.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 and the Management's Discussion and Analysis sections of REGENXBIO's annual report on Form 10-K for the full year ended December 31, 2025, and comparable Risk Factor sections of REGENXBIO's quarterly reports on Form 10-Q, which will be on file with the Securities and Exchange Commission 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 6, 2026, 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 REGENXBIO.

Thank you, Patrick, and good morning everyone. Thank you for joining us today. The second quarter was another period of positive momentum for REGENXBIO, achieving key milestones across our late-stage pipeline of gene therapies. During the quarter, we announced that we completed enrollment in the confirmatory study for RGX-202, reached alignment with FDA on the path to resubmit the BLA for RGX-121, and dosed the first patient in the NAAVIGATE trial of sura-vec in diabetic retinopathy, achieving a $100 million milestone payment from AbbVie. We have substantially strengthened our financial position through receipt of over $200 million total, inclusive of the AbbVie milestone payment and new capital, ending the quarter pro forma with more than $310 million.

This extends our runway into Q4 2027, which includes the expected PDUFA date for RGX-202 and brings us closer to our goal of delivering new needed medicines to patients and generating our first product revenues. Before I turn the call over to Dr. Steve Pakola, our Chief Medical Officer, and Mitch Chan, our Chief Financial Officer, to provide updates on our clinical and financial progress, respectively, I'd like to highlight a few key program updates. Let's start with RGX-202, our wholly-owned potential best-in-class therapy for Duchenne muscular dystrophy. We believe the top-line pivotal data shared in May further establish RGX-202 as a differentiated gene therapy candidate. RGX-202 has uniquely demonstrated a large magnitude of effect relative to baseline and strong correlation between microdystrophin expression and functional improvement at one year. This is a comprehensive body of evidence that we believe will support potential accelerated approval.

We recently reported that we had fully enrolled and completed dosing in the confirmatory study of RGX-202 ahead of schedule. We have enrolled over 60 patients in the pivotal and confirmatory trials to support a robust safety data set in the planned BLA filing. Momentum for RGX-202 remains strong. Our strengthened cash position enables continued investment in our strategic priorities, including preparing for the U.S. commercial launch of RGX-202. We will soon initiate a randomized placebo-controlled study in Duchenne named AFFINITY RISE outside the U.S. to support future global regulatory submissions. We also continue to manufacture intended commercial supply at our FDA-inspected commercial-ready manufacturing facility located in Rockville, Maryland.

We remain committed to bringing RGX-202 to patients as soon as possible through multiple key milestones, including initiating the ex-U.S. RCT in the first half of 2027 and submitting the first module of the BLA to FDA in Q3 2026 with potential U.S. approval in the second half of 2027. Moving to RGX-121, following our collaborative discussion with FDA in June, where we aligned on a path forward, we have since held a productive Type A meeting with the agency in July. During the meeting, the FDA confirmed that our available data is sufficient for review under the accelerated approval pathway, and no additional studies, including an RCT, will be required for BLA resubmission. The resubmission will include longer-term efficacy and safety data, including participant imaging that we have submitted and continue to compile and analyze as part of our ongoing monitoring requirements.

We are working to resubmit the BLA in Q3. Beyond our rare programs, we continue to make meaningful progress across our retinal franchise through our strategic collaboration with AbbVie. Along with dosing the first patient in the Phase II-B/III NAAVIGATE study for diabetic retinopathy, we and AbbVie recently presented long-term data in both wet AMD and diabetic retinopathy. These studies highlight the durable safety and efficacy profile that underscores our confidence in the potential commercial success of sura-vec. With the NAAVIGATE study now underway in diabetic retinopathy, near-term focus of the partnership has shifted to the upcoming pivotal readout for sura-vec in subretinal wet AMD, a milestone that is highly anticipated in the field. We are pleased with the continued momentum across the collaboration and look forward to sharing top-line data from the ATMOSPHERE and ASCENT studies in the fourth quarter.

Our focus is clear for the remainder of 2026. Execute against our key milestones and bring hope for transformative gene therapies closer to patients in need. With that, I'll turn it over to Steve.

Thank you, Curran. I'll start with the RGX-202 program for the treatment of Duchenne. As Curran referenced, we are incredibly excited by the continued momentum we have seen in the AFFINITY DUCHENNE clinical program and look forward to initiating BLA submission this quarter. As a reminder, RGX-202 is the most advanced clinical-stage gene therapy program in Duchenne, and both the pivotal and confirmatory studies enrolled ambulatory patients aged one and older. As reported in May, top-line results from our pivotal study demonstrated a highly compelling combination of robust microdystrophin expression, encouraging functional improvement, including in older boys, and a favorable safety profile. These positive results, together with the statistically significant strong correlation observed between microdystrophin expression and NSAA improvement, will serve as a key component of our upcoming BLA submission.

As Curran shared, we are also progressing plans to expand the clinical development of 202 to support future regulatory submissions outside the U.S. through the ex-U.S. AFFINITY RISE study. This global, double-masked, placebo-controlled randomized trial is designed to enroll approximately 100 patients with a 2-to-1 active to placebo randomization. With enrollment now complete in the confirmatory study, we are excited to initiate this study in the first half of next year. We look forward to sharing more as we progress. Turning now to our retina franchise, we continue to be encouraged by the growing body of evidence supporting sura-vec as a differentiated potential one-time gene therapy for retinal disease. Last month at ASRS, we presented multiple data sets that further reinforce the durability, efficacy, and safety profile of the program across both wet AMD and DR. In subretinal wet AMD, we reported long-term follow-up data from our Phase I/II study.

Results demonstrated sura-vec maintained or improved visual acuity with a meaningful reduction in treatment burden through five years. These results are even more impressive as they reflect a wet AMD population that faced a high burden of chronic anti-VEGF injections prior to receiving one-time gene therapy. We're very encouraged by these results as we approach top-line data later this year. In DR, we presented new two-and-a-half-year follow-up data from the ALTITUDE study. These results demonstrated sura-vec maintained durable improvements in disease severity, continued prevention of vision-threatening complications, and a favorable long-term safety profile following a single administration. While chronic anti-VEGF injections are approved for DR, real-world data shows that there is a staggeringly low use due to the high treatment burden. We believe the potential to prevent vision-threatening complications with a one-time in-office administration represents an important option for these patients.