REGENXBIO Q2 2026 Earnings Report

REGENXBIO EPS Results

• Actual EPS: $0.43
• Consensus EPS: $0.08
• Beat/Miss: Beat by +$0.35
• One Year Ago EPS: N/A

REGENXBIO Revenue Results

• Actual Revenue: $108.02 million
• Expected Revenue: $97.63 million
• Beat/Miss: Beat by +$10.39 million
• YoY Revenue Growth: N/A

REGENXBIO Announcement Details

• Quarter: Q2 2026
• Date: 8/6/2026
• Time: Before Market Opens
• Conference Call Date: Thursday, August 6, 2026
• Conference Call Time: 8:00AM ET

REGENXBIO (NASDAQ:RGNX) is a clinical‐stage biotechnology company specializing in the development of gene therapies using its proprietary NAV® AAV (adeno‐associated virus) platform. The company engineers next‐generation AAV vectors designed to deliver functional genes to targeted cells, aiming to address a range of rare genetic diseases and ocular, metabolic and neurologic disorders. REGENXBIO’s pipeline features several product candidates in various stages of preclinical and clinical development, including RGX-314 for wet age‐related macular degeneration, RGX-121 for mucopolysaccharidosis II (Hunter syndrome) and RGX-121 for other rare lysosomal storage diseases.

In addition to its internally funded programs, REGENXBIO has established partnerships with major biopharmaceutical companies to advance its NAV technology. These collaborations leverage the company’s vector engineering expertise to create bespoke gene therapies across multiple therapeutic areas. Through such alliances, REGENXBIO gains access to complementary development resources, while partners benefit from its deep knowledge of AAV vector design and scalable manufacturing processes.

Founded in 2012 and headquartered in Rockville, Maryland, REGENXBIO operates a state‐of‐the‐art manufacturing facility to support both clinical and commercial production of AAV vectors. The company’s leadership team comprises industry veterans with extensive experience in gene therapy research, development and regulatory affairs. REGENXBIO’s scientific advisory board includes academic and clinical experts who guide vector selection, preclinical study design and clinical trial strategy.

REGENXBIO continues to expand its global footprint through clinical trial sites in North America, Europe and Asia, reflecting its commitment to bringing novel gene therapies to patients worldwide. By harnessing the NAV AAV platform’s versatility and safety profile, the company seeks to deliver one‐time treatments that have the potential to transform care for individuals living with severe genetic and ocular diseases.

REGENXBIO Q2 2026 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: REGENXBIO ended the quarter with more than $310 million pro forma, following a $100 million AbbVie milestone and a $108 million follow-on offering. Management expects this to fund operations into Q4 2027, including the anticipated RGX-202 PDUFA date.
• Positive Sentiment: RGX-202 enrollment and dosing in the confirmatory Duchenne muscular dystrophy study were completed ahead of schedule, with more than 60 patients across pivotal and confirmatory studies. The company plans to submit the first BLA module in Q3 2026 and initiate the global AFFINITY RISE trial in the first half of 2027.
• Positive Sentiment: The FDA agreed that RGX-121’s existing data are sufficient for accelerated-approval review and said no additional studies, including a randomized controlled trial, are required for BLA resubmission. REGENXBIO plans to resubmit the application in Q3 2026 with longer-term efficacy, safety, imaging, and neurocognitive data.
• Positive Sentiment: REGENXBIO and AbbVie began the Phase IIb/III NAAVIGATE study of sura-vec in diabetic retinopathy, triggering the $100 million milestone payment. The partners expect fourth-quarter top-line results from the ATMOSPHERE and ASCENT wet AMD studies, while longer-term follow-up showed durable effects and favorable safety through five years in wet AMD and 2.5 years in diabetic retinopathy.
• Negative Sentiment: The company acknowledged that its U.S. patent covering Zolgensma has expired, although royalties will continue in roughly 20 countries outside the United States. Management highlighted Evrysdi coverage and potential future royalties from Itvisma as offsets, but did not provide specific royalty-rate guidance.

Presentation

[Operator]

Welcome everyone to the second quarter of 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. After the speaker's remarks, there will be a question-and-answer session. If you would like to ask a question during this time, simply press star then number one on your telephone keypad. To withdraw your question, press star one 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 at REGENXBIO]

Good morning and thank you for joining us today. Earlier this morning, REGENXBIO released financial and operating results for the second quarter ended June 30th, 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.

[Patrick Christmas, Chief Legal Officer at REGENXBIO]

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 31st, 2025.

[Patrick Christmas, Chief Legal Officer at REGENXBIO]

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 6th, 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.

[Patrick Christmas, Chief Legal Officer at REGENXBIO]

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, President and CEO at 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.

[Curran Simpson, President and CEO at REGENXBIO]

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.

[Curran Simpson, President and CEO at REGENXBIO]

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.

[Curran Simpson, President and CEO at REGENXBIO]

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.

[Curran Simpson, President and CEO at REGENXBIO]

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.

[Curran Simpson, President and CEO at REGENXBIO]

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.

[Curran Simpson, President and CEO at REGENXBIO]

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.

[Curran Simpson, President and CEO at REGENXBIO]

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.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

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.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

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.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

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.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

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.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

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 2.5 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.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

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. 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.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Every interaction we have at these events underscore 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?

[Mitch Chan, CFO at REGENXBIO]

Thank you, Steve, and good morning, everyone. REGENXBIO ended the second quarter of 2026 with cash equivalent, and marketable securities of $106 million. Research and development and general 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.

[Mitch Chan, CFO at REGENXBIO]

First, we received the $100 million milestone payment from AbbVie following our first patient dose in the phase II-B/III NAAVIGATE 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. These additional resources support both near and long-term strategic priorities, including advancing commercial readiness activity across our late-stage programs.

[Mitch Chan, CFO at REGENXBIO]

Specific near-term investment to prepare for the potential RGX-202 commercial launch in the United States, and planned initiation of RGX-202 ex-U.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.

[Mitch Chan, CFO at REGENXBIO]

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, President and CEO at REGENXBIO]

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. We look forward to sharing our progress. With that, I'll turn over the call for questions. Operator?

[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 from MS. Your line is now open.

[Judah Frommer, Analyst at Morgan Stanley]

Good morning, guys. Congrats on the progress and thanks for taking my 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. Then maybe just feedback from ASRS, kind of general excitement for gene therapy versus alternative modalities that extend treatment.

[Judah Frommer, Analyst at Morgan Stanley]

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.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Just to clarify, Judah, the AFFINITY confirmatory, you're speaking to AFFINITY RISE, the new program, or a different one?

[Judah Frommer, Analyst at Morgan Stanley]

The confirmatory that I believe will I think it's set up as the phase III portion of AFFINITY DUCHENNE. Is that right?

[Curran Simpson, President and CEO at REGENXBIO]

Okay. Yeah. I think.

[Judah Frommer, Analyst at Morgan Stanley]

That completed in June. The enrollment of that completed in June.

[Curran Simpson, President and CEO at REGENXBIO]

Yeah. I think when we think forward to the global study that we just announced today, we certainly think enrollment, just at 100,000 ft level, will be very positive. We enrolled the 30 patients in our confirmatory study post the pivotal ahead of schedule. I think that's an indicator of overall patient demand. Globally as well, you see significant uptake of gene therapy ex-U.S. I think we feel that enrollment in that study is positive. I'll turn it to Steve for thoughts.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Sure. Curran, you gave the 100,000 ft view. I can sort of give the ground-level view. As you mentioned, Judah, enrollment was really good. I think the more data we gathered, the more enthusiasm there has been. As sites would treat a patient, they would get more excited as well. 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.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Yeah, I think that really gives us a lot of momentum going into AFFINITY RISE. 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. In both wet AMD and DR, we presented longer-term follow-ups. five-year results from wet AMD and 2.5-year results from DR. We're seeing great durability and excellent safety with longer-term follow-up.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

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 on the one-time option. Big advance or unmet need if you can have that in sustained anti-VEGF. 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.

[Judah Frommer, Analyst at Morgan Stanley]

Thanks.

[Operator]

Your next question comes from Lili Nsongo from Leerink. Your line is now open.

[Lili Nsongo, Analyst at Leerink]

Hi, good morning. Thank you for the update. Maybe just a question regarding the patent portfolio. How should we think about the potential impact of patent expiry on Zolgensma royalty stream in the coming years? How should we think about potential revenue stream or royalty rate for Itvisma compared to Zolgensma?

[Patrick Christmas, Chief Legal Officer at REGENXBIO]

Sure. This is Patrick Christmas. I can start. As we've announced, our U.S. patent on Zolgensma has expired, but we do have coverage in about 20 countries outside the United States where we'll continue to see revenue and royalties on Zolgensma. In addition, we have coverage on Evrysdi both in the U.S. and worldwide on that product. I think Novartis has announced that they could reach up to $2 billion in sales. We expect to see continued significant royalties there as well.

[Lili Nsongo, Analyst at Leerink]

Thank you.

[Operator]

Your next question comes from the line of Annabel Samimy from Stifel. Your line is now open.

[Annabel Samimy, Analyst at Stifel]

Hi. Thanks for taking my question. A couple here. For wet AMD, as you approach data, I know most of these physicians are retinal surgeons and perform vitrectomies, but how many physicians would you say have already been trained on your procedure specifically? Can you remind us how many patients have opted for bilateral treatment? Secondly, on DMD, I guess for the timing of the RCT, I think it's going to be in full swing as you are going through review. Given that you could potentially file in H1 2027.

[Annabel Samimy, Analyst at Stifel]

I guess suggest that you'll have some of the data and any expectations that FDA is going to wait for the biomarker outcome of that study to make any decisions on your own program. Thanks. Will require the data. Thank you.

[Curran Simpson, President and CEO at REGENXBIO]

I'll take the second one, and then we'll have Steve speak to the first question. I think on the RCT and the timing, the goal here is obviously to have it up and in active enrollment at the time of review. We think that's important in terms of the overall regulatory strategy. I don't think that there would be an expectation of data being available or specific data from that study playing onto the active review that would be for accelerated approval. I think one of the reasons for that is that we'll have over 60 patients dosed at the time of filing.

[Curran Simpson, President and CEO at REGENXBIO]

So our safety database will be pretty extensive from the pivotal study and the confirmatory study that we completed enrollment on. We also, in terms of the timing, will also have roughly half the patients through 12 months of their functional assessment. We think we'll have a very strong data package underpinned by the really positive data we showed with our top-line release earlier this year. I think one thing that we are seeing, though, that I think is really important to our global study is.

[Curran Simpson, President and CEO at REGENXBIO]

I believe in terms of obtaining accelerated approval, having a reasonable timeline for completion of a confirmatory study is an element of that. I just don't think it has to be in proximity to the approval of an AA pathway. Steve, I'll let you talk through the.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Sure.

[Curran Simpson, President and CEO at REGENXBIO]

Right now.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Hi, Annabel. Thanks for the question. On wet AMD, we've trained quite a lot of surgeons globally now. Over 500 surgeons have actually been trained, and I think this speaks the actual evidence of what we've been discussing over time. The scalability of this is really straightforward, not surprising given these are retina specialists who are used to doing much more complicated procedures. We're really excited about that as far as moving forward. We and AbbVie, of course. The bilateral question. We're seeing great interest.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

One issue is technically, to enroll in the bilateral study, you have to meet all of the study requirements. That already will cut out a certain proportion of patients. Although it's a bilateral disease, exactly when patients will meet the criteria in terms of disease activity will change over time. Given those aspects, we're really encouraged. We hear a lot of anecdotal cases from surgeons of asking, can they have their fellow eye treated if they are already getting repeated injections in both eyes before they saw whatever response they had in the 314 sura-vec-treated eye.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

We haven't given any specific numbers on this. We are at AbbVie, but certainly we're encouraged.

[Annabel Samimy, Analyst at Stifel]

Great. Thank you.

[Operator]

Your next question comes from the line of Brian Skorney from Baird. Your line is now open.

[Brian Skorney, Analyst at Baird]

Hey, good morning, guys. Thanks for taking the question. My question's on AFFINITY RISE, actually. I was hoping you could go over some more details around the study design. I sort of heard the highlights of it at the beginning of the call. How long is the placebo-controlled period, and can you just review the primary and key secondary endpoints and enrollment criteria in terms of the ages of patients? What ex-U.S. regions do you anticipate enrolling in this study?

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Yeah, thanks, Brian. What we've provided that you see in the press release are the high-level aspects of the design. For ex-U.S. requirements, including Europe, not surprising, placebo-controlled. Importantly, we've chosen a two to one randomization with active, we think that's a nice positive for families and their children as far as odds of getting treatment. As far as the other questions that you asked, we haven't disclosed those in terms of the regions that we're going to go.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Also some of the other aspects that you've mentioned, we certainly look forward to getting into more details about the design as we get closer to the initiation of the trial in the first half.

[Operator]

Your next question comes from Alec Stranahan from Bank of America. Your line is now open.

[Alec Stranahan, Analyst at Bank of America]

Hey, guys. Thanks for taking my questions. I guess one on MPS II and then one follow-up on AFFINITY RISE. On the resubmission MPS II, I guess what specific question from the FDA is the longer term follow-up in imaging answering? Do you expect this will meaningfully shift the known clinical profile for one to one in this setting? On AFFINITY RISE, I guess how should we think about crossover from placebo in the study? I guess just thinking about the ethics of keeping patients on placebo for a progressive disease like DMD.

[Alec Stranahan, Analyst at Bank of America]

Just to clarify, will this study enroll any U.S. patients? Thank you.

[Curran Simpson, President and CEO at REGENXBIO]

Sure. I'll take the first question, and Steve can walk through the second. On MPS II, if you think about sort of where we started with the program, the initial data provided was six-month data for biomarker. Over the course of the ongoing review, we've now expanded that to two-year biomarker data and two-year neurocognitive data as we've updated the filing. What was requested in the Type A was really just a consolidation of that and the safety update that would go along with that in terms of extending out the time.

[Curran Simpson, President and CEO at REGENXBIO]

I think the really positive, if you focus back to the CRL, there were a couple of components of the CRL that was issued in February that were really challenging to overcome, which were really the trial design and the inclusion of a control arm. The Type A meeting, we feel, reset that to now basically an ongoing review of the data that we've provided. We were not asked to provide additional patients being dosed or additional patient data beyond the two-year horizon.

[Curran Simpson, President and CEO at REGENXBIO]

Now I believe we'll see something closer to an evaluation of the benefit risk at that point, with all of that data consolidated into one resubmission. I'll let Steve talk through AFFINITY RISE.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Yeah, Alec, great question in terms of crossover. One aspect is even how we want to get access for these patients in need and certainly patients who commit to getting into a clinical trial. That's one of the reasons we wanted the 2-to-1 randomization. But that still leaves the other third of patients that you're referring to. I think it's reasonable to expect that we would give those boys a chance in a crossover, and then we also get to learn more with more exposures in the trial.

[Operator]

Your next question comes from Luca Issi from RBC Capital Markets. Your line is now open.

[Analyst at RBC Capital Markets]

Great. Hi, team. This is Shelby on for Luca, thanks for taking our question. On DMD, I believe you said the BLA will include a combined safety data set for over 60 patients, 12 months functional data for at least half of the pivotal cohort. Can you quantify how many patients will have that 12-month functional data at the time of BLA initiation this quarter versus at completion in the first quarter of 2027? Does the FDA require a pre-specified minimum for accelerated approval? Any color there much appreciated.

[Curran Simpson, President and CEO at REGENXBIO]

Sure. Yeah, I can take the last part of the last question there. There's no pre-specified level of functional data that's required for submission. To your question around the number of patients, if you roll back to our top-line data, we presented data for nine patients at 12 months. By the time the clinical module is submitted, the critical path is really defined by the CMC module, as we've talked about before, but that does give us the advantage to add additional patients to the clinical module.

[Curran Simpson, President and CEO at REGENXBIO]

We're estimating out of the 30 that were treated in the pivotal portion of the study, roughly half of those would be through 12 months. We're not super precise whether it's 14 or 15, that depends a lot on visits and assessment and QC of the data. We obviously feel that we want to present the strongest package at that time, and that's one element of this.

[Operator]

Your next question comes from the line of Ellie Merle from Barclays. Please go ahead.

[Analyst at Barclays]

Hi, this is Joseph on for Ellie, thank you for taking our questions. For RGX-314, what is your latest perspective on the commercial outlook in the evolving landscape? In terms of launch preparation, could you characterize your site qualification efforts so far and provide any color on which commercial sites you plan to qualify initially? Thank you.

[Curran Simpson, President and CEO at REGENXBIO]

I'm sorry, just to clarify, that's for subretinal wet AMD?

[Analyst at Barclays]

That's correct.

[Curran Simpson, President and CEO at REGENXBIO]

I think one aspect, just to step back a bit, is ultimately on the commercialization effort for subretinal, AbbVie will have the primary leadership in that role. It will not be surprising that many of our clinical sites, which Steve, correct me if I'm wrong, were well above 100 sites used for enrollment in the U.S., some of those sites would be obvious for also commercialization of the product. We're just starting now joint development of the commercial plan with AbbVie, given that we expect a 12-month review cycle on the subretinal BLA filing.

[Curran Simpson, President and CEO at REGENXBIO]

That work is just beginning now. It's a bit early to be specific about it, but we have no doubt that the strength of AbbVie's commercial team, and their familiarity now with the program over the last 3.5 years, will get a running start on commercialization. On the prospects of the product, we look at wet AMD, we look forward to a launch date in which the wet AMD market could be in the range of $10 billion. I think historically, we think subretinal has a meaningful place in that market.

[Curran Simpson, President and CEO at REGENXBIO]

As Steve mentioned with treatment of the fellow eye, that raises the potential commercial prospects even further. I would suggest that whenever we speak to doctors who are very experienced with this program, they can easily identify out of 10 patients, two or three that are obvious candidates for the subretinal administration because of various factors, availability for monthly injections, disease burden, etc. Again, we feel like there's a very meaningful market here, potentially one of the largest opportunities in gene therapy.

[Operator]

Your next question comes from Sean McCutcheon from Raymond James. Your line is now open.

[Sean McCutcheon, Analyst at Raymond James]

Hi, guys. Thanks for the question, kind of speaking to the wet AMD readouts in the fourth quarter. Can you speak to the expected non-inferiority margin in ATMOSPHERE and ASCENT? Would you anticipate it to be significantly narrower than the standard4.5 letters due to the partially masked nature of the studies? Any commentary on the powering of the studies for that would be helpful. Thanks.

[Curran Simpson, President and CEO at REGENXBIO]

Great. I'll defer that one to Steve.

[Steve Pakola, Chief Medical Officer at REGENXBIO]

Sure. Sean, you named the non-inferiority margin 4.5 letters, and that has been standard in recent history, and the FDA has reiterated that. We've never had that challenged by the FDA based on the design, and that masking approach has been in there throughout the study. Working with AbbVie, we have very large sample sizes. These are the largest gene therapy programs ever executed, so we have 90% power in these studies.

[Operator]

Your next question comes from the line of Paul Choi from Goldman Sachs. Please go ahead.

[Paul Choi, Analyst at Goldman Sachs]

Hi. Good morning, thanks for taking our questions, and congrats on all the progress. I have two questions. First, on 202, can you update us on what the potential cadence of additional data updates might be as you proceed to your BLA filing? Just what additional longer-term updates you plan to provide from the pivotal portion there. My second question is on 121, with the recent approval and launch of Denali's AVLAYAH, can you maybe just update us on what you're hearing in the field in terms of receptivity to new therapies here for hunters.

[Paul Choi, Analyst at Goldman Sachs]

How the market might potentially be primed for your and your partner's launch in the future? Thank you.

[Curran Simpson, President and CEO at REGENXBIO]

Yeah. I'll comment first on the 121 question, I don't have any specific information regarding Denali's launch progress. I think from our frequent interactions with the patient advocacy groups, we see a very high level of interest in their program. I think it's well over 20 years before a new therapy has been available to patients. I think there's excitement. I think thinking about RGX-121, our goal is to provide potential options for patients to choose their therapy beyond what's already available.

[Curran Simpson, President and CEO at REGENXBIO]

I do think indications of strong uptake, which I think is positive for the field, indications that patients still want choices in the therapy that they choose. I think the obvious benefit of gene therapy in this case being the one-time treatment potential. In terms of additional data updates on 202, we haven't disclosed any specific updates and probably won't until the BLA filing is complete in Q1 of next year. I think around that time, once the data has been submitted, we'll obviously consider an update at that point.

[Curran Simpson, President and CEO at REGENXBIO]

In the meantime, right now, we don't have a specific update planned other than certainly notifying the field and the market regarding submission of the modules and timing around that to confirm that we're on track.

[Paul Choi, Analyst at Goldman Sachs]

Okay, great. Thank you.

[Curran Simpson, President and CEO at REGENXBIO]

Thanks.

[Operator]

Your next question comes from the line of Yi Chen from H.C. Wainwright. Your line is now open.

[Analyst at H.C. Wainwright]

Hey, good morning. This is Katie on for Yi. Just a couple quick clarification questions. For ATMOSPHERE and ASCENT, looks like you're looking for top line in the fourth quarter of this year. Will you disclose both trials together with the full non-inferiority margin and injection burden data, or is that something that's mostly AbbVie's call? Is there a milestone tied to the top line versus the 2027 submissions?

[Curran Simpson, President and CEO at REGENXBIO]

Yeah, I can cover that. The studies will be disclosed together. They're pretty much right on top of each other in terms of timing. We haven't given specific guidance regarding the data that will be disclosed at that point. That is a joint effort between AbbVie and ourselves regarding the ultimate release. Certainly, you would expect to see primary endpoint disclosure for both studies, and we'll be more specific as we get closer around secondaries as well. There is not a milestone associated with top-line data.

[Curran Simpson, President and CEO at REGENXBIO]

There are milestones that we haven't disclosed the specific amounts around for BLA acceptance and then BLA approval. Those are elements that Mitch mentioned in his update, additional non-dilutive financing options that can move our cash runway even further out.

[Analyst at H.C. Wainwright]

Great. Thank you, guys.

[Operator]

Ladies and gentlemen, that concludes our Q&A session and today's call. Thank you all for joining. You may now disconnect.

Participants

Executives

• Patrick Christmas, Chief Legal Officer
• Curran Simpson, President and CEO
• Steve Pakola, Chief Medical Officer
• Mitch Chan, CFO

Analysts

• Judah Frommer, Analyst at Morgan Stanley
• Lili Nsongo, Analyst at Leerink
• Annabel Samimy, Analyst at Stifel
• Brian Skorney, Analyst at Baird
• Alec Stranahan, Analyst at Bank of America
• Analyst at RBC Capital Markets
• Analyst at Barclays
• Sean McCutcheon, Analyst at Raymond James
• Paul Choi, Analyst at Goldman Sachs
• Analyst at H.C. Wainwright