Full Transcript: Regenxbio Q2 2026 Earnings Call

Regenxbio (NASDAQ: RGNX ) released second-quarter financial results and hosted an earnings call on Thursday. Read the complete transcript below. This transcript is brought to you APIs. For real-time access to our entire catalog, please visit for a consultation.

View the webcast at Summary Regenxbio reported strong progress in its gene therapy pipeline, notably completing enrollment in the confirmatory study for RGX202 and achieving a $100 million milestone payment from AbbVie. S. commercial launch preparation for RGX202. Key program updates include advancing RGX202 for Duchenne muscular dystrophy, with plans to submit the BLA to the FDA in Q3 2026, and continuing development of Surivec in collaboration with AbbVie for retinal diseases.

Management expressed confidence in the company's strategy and financial position, highlighting the potential for multiple product launches and transformative therapies in the coming 18 months. S. regulatory submissions and global trials for its therapies. Full Transcript Elaine, Operator Welcome, everyone, to the second quarter 2026 Regenxbio earnings conference call.

My name is Elaine, and I will be your conference operator today. All lines have been placed on mute to prevent any background noise, and after the speakers' remarks there will be a question-and-answer session. If you would like to ask a question during this time, simply press star then 1 on your telephone keypad. To withdraw your question, press star-1 again.

At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of Regenxbio. Please go ahead. Patrick Christmas, Chief Legal Officer 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 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 Factors sections of Regenxbio'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, 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.

Curran Simpson, Chief Executive Officer and President 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 RGX202, reached alignment with FDA on the path to resubmit the BLA for RGX121, and dosed the first patient in the NAVIGATE trial of Surivec 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 RGX202, and brings us closer to our goal of delivering 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 RGX202, our wholly owned, potential best-in-class therapy for Duchenne muscular dystrophy. We believe the top-line pivotal data shared in May further establish RGX202 as a differentiated gene therapy candidate. RGX202 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 RGX202 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 RGX202 remains strong. S.

commercial launch of RGX202. 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.

S. S. approval in the second half of 2027. Moving to RGX121, 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, and 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 2b/3 NAVIGATE 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 Surivec. With the NAVIGATE study now underway in diabetic retinopathy, near term, focus of the partnership has shifted to the upcoming pivotal readout for Surivec 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. Steve Pakola, Chief Medical Officer Thank you, Curran.

I'll start with the RGX202 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, RGX202 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. S. 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 Surivec as a differentiated, potential one-time gene therapy for retinal disease.

Last month at ASRS, we presented multiple data sets that further reinforced 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 1/2 study. Results demonstrated Surivec 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. 5-year follow-up data from the ALTITUDE study. These results demonstrated Surivec 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 show that there is 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. Finally, this summer we've had the privilege of joining both the Duchenne and Hunter syndrome communities at family and advocacy conferences. These families and advocates inspire us and power our mission every day. Every interaction we have at these events underscores how urgently families are waiting for new treatment options that can meaningfully change the course of these diseases.

We're deeply grateful for the community's partnership, support, and enthusiasm for our programs. With that, I'll turn the call over to Mitch to review our financial results. Mitch Chan (Chief Financial Officer) Mitch, thank you Steve, and good morning everyone. Regenxbio ended the second quarter of 2026 with cash, cash equivalents and marketable securities of $106 million.

Research and development and general and administrative expenses are generally consistent with the same period in 2025, reflecting the continued advancement of our late-stage clinical programs and our operational capabilities to support our planned transition to a commercial-stage organization. Subsequent to the quarter, we strengthened our financial position through two important financing events. First, we received the $100 million milestone payment from AbbVie following our first patient dose in the Phase 2b/3 NAVIGATE study for DR, reflecting the continued progress within our strategic retinal collaboration.

In addition, we successfully completed a follow-on public offering of approximately $108 million in net proceeds. We end the quarter pro forma with more than $310 million. S. RCT study to support future regulatory opportunities outside the United States.

Including these proceeds, Regenxbio's cash runway is into the fourth quarter of 2027, which enables us to complete multiple milestones, including the top-line data in wet AMD and expected PDUFA date for RGX-202. This cash runway guidance does not include any potential receipt of proceeds associated with additional potential non-dilutive sources of funding, including healthcare royalty agreement, milestone payment associated with our partner programs, or any proceeds from the potential sale of RGX-121 PRV. We find ourselves well positioned to leverage these and other funding options as we advance towards multiple product launches.

With that, I turn the call back to Curran to provide final thoughts. Curran Simpson, Chief Executive Officer and President Thank you, Mitch. We leave today's call with confidence in our strategy, our execution and our financial position. With the capital to deliver against multiple catalysts, we remain sharply focused on executing and advancing potentially transformative gene therapies to patients.

We believe the next 18 months will be a defining period for Regenxbio and we look forward to sharing our progress. With that, I'll turn over the call for questions. Elaine, Operator Thank you. We will now begin the question-and-answer session.

If you have dialed in and would like to ask a question, please press star one on your telephone keypad to raise your hand and enter the queue. If you would like to withdraw your question, simply press star one again. We will pause for just a moment to compile the Q&A roster. Your question comes from the line of Judah Frommer.

Your line is now open. Judah Frommer Good morning, guys. Congrats on the progress and thanks for taking the questions. Two from us.

Maybe first, can you just give us a little more color on enrollment for the Affinity confirmatory study? What demand looked like from patients and investigators there? It does seem like that enrolled relatively quickly, and then maybe just feedback from ASRS, kind of general excitement for gene therapy versus alternative modalities that extend treatment. Any particular feedback on subretinal and suprachoroidal delivery versus intravitreal gene therapies, and relative unmet need in wet AMD versus DR for gene therapy?

Thanks. Curran Simpson, Chief Executive Officer and President And just to clarify, Judah, the Affinity confirmatory is speaking to AffinityRise, the new program, or a different one? Judah Frommer The confirmatory that I believe will, I think it's set up as the phase three portion of Affinity Duchenne. Is that right?

Curran Simpson, Chief Executive Officer and President Okay. Yeah, that completed in June. The enrollment, that completed in June. Yeah.

I think when we think forward to the global study that we just announced today, we certainly think enrollment, just at a 100,000-foot level, will be very positive. We enrolled the 30 patients in our confirmatory study post the pivotal ahead of schedule. S. So I think we feel that enrollment in that study is positive.

I'll turn it to Steve for thoughts. Steve Pakola, Chief Medical Officer Sure. So, Curran, you gave the 100,000-foot view. I can sort of give the ground-level view.

And as you mentioned, Judah, enrollment was really good, and I think the more data we gathered the more enthusiasm there has been and, you know, as sites would treat a patient they would get more excited as well. So I think a lot of that is obviously the differentiation of 202 that we've talked about in terms of the product itself and the safety profile, as well as the encouraging functional results that we're already seeing. So yeah, I think that really gives us a lot of momentum going into AffinityRise. The other question you had was on RGX-314 and ASRS last week, and I think the one word key message, or what was absorbed, was durability.

So in both wet AMD and DR we presented longer-term follow-up — so five-year results from wet AMD and two-and-a-half-year results from DR — and we're seeing great durability and excellent safety with longer-term follow-up. And I think to the follow-up, or the additional question on that as far as unmet need that we're seeing compared to some of the other nice advances that have happened in the space in terms of durability, one of the nice things with the greater durability is that we're seeing more and more interest in the one-time option. So big advance for unmet need if you can have that and sustained anti-VEGF.

And in DR the unmet need and the differentiation is even clearer because in that disease, with an asymptomatic nature, you really need a one-time treatment, not repeated injections over time. Thanks. Elaine, Operator Your next question comes from Lily from Leerink. Your line is now open.