Regenxbio - Earnings Call - Q1 2025

Executive Summary

• Q1 2025 revenue was $89.012M and GAAP diluted EPS was $0.12; both missed Wall Street consensus ($105.350M revenue, $0.34 EPS). Revenue strength came from $71.8M recognized under the Nippon Shinyaku collaboration, while EPS benefitted from higher investment income and restrained R&D, but fell short of estimates.
• Cash, cash equivalents and marketable securities rose to $272.7M, with management reiterating cash runway into H2 2026; shortly after quarter-end, FDA accepted RGX‑121 (Priority Review; PDUFA Nov 9, 2025), and non‑dilutive financing further extended runway (early 2027).
• Strategic catalysts: RGX‑121 Priority Review in MPS II, RGX‑202 pivotal enrollment >50% with planned BLA mid‑2026, and AbbVie-partnered RGX‑314 (wet AMD/DR) progressing toward pivotal/2026 data; commercial supply manufacturing for RGX‑202 begins in Q3 2025.
• Stock-relevant narrative: near-term regulatory de‑risking (RGX‑121 acceptance), robust pipeline momentum, and reiterated cash runway, offset by the revenue/EPS miss vs consensus and continued operating/investment expense headwinds.*

What Went Well and What Went Wrong

What Went Well

• RGX‑121 BLA accepted with Priority Review; management highlighted potential first-in-class gene therapy for MPS II and PRV monetization optionality: “Acceptance of the RGX‑121 BLA marks an exciting milestone...” (PDUFA 11/9/2025).
• RGX‑202 pivotal study “beyond 50% enrolled”; commercial supply manufacturing starts in Q3 2025; in-house capacity up to 2,500 doses/year with >80% full capsid purity, supporting fast-follower positioning in Duchenne.
• Strong balance sheet and non-dilutive financing levers: CFO reiterated runway into H2 2026 and outlined additional options (milestones, PRV sale, Zolgensma royalty reversion) to extend well beyond 2026.

What Went Wrong

• Revenue/EPS both missed consensus despite collaboration revenue recognition; revenue $89.012M vs $105.350M est., EPS $0.12 vs $0.34 est.* The delta appears tied to timing/mix of license and service revenue and higher interest expense ($8.57M).*
• Operating expense/investment headwinds persist: total operating expenses were $76.885M; interest expense rose to $8.57M, tempering bottom-line leverage.
• Regulatory/safety scrutiny in DMD heightened post-Elevidys event; management emphasized immunomodulation and high product purity, but investors remain sensitive to FDA expectations around accelerated approval and functional data requirements.

Transcript

Operator (participant)

Welcome, everyone, to the first quarter 2025 REGENXBIO earnings conference call. At this time, all participants are in listen-only mode. After the speaker's prepared remarks, we will conduct a question-and-answer session. To ask a question, please press star one one. To remove yourself from the queue, please press star one one again. As a reminder, this call may be recorded. At this time, I'd like to turn the conference over to Patrick Christmas, Chief Legal Officer of REGENXBIO. Please go ahead.

Patrick Christmas (CLO)

Good afternoon, and thank you for joining us today. Earlier this afternoon, REGENXBIO released financial and operating results for the first quarter ended March 31, 2025. 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, in 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 discussion and analysis section of REGENXBIO's annual report on Form 10-K for the full year ended December 31st, 2024, and comparable risk factors sections of REGENX'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, May 12, 2025, and we undertake no obligations to update any forward-looking statements we may make on this call on account of new information and future events 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.

Curran Simpson (President and CEO)

Thank you, Patrick, and thank you, everyone, for joining us today. We've had an impressive start to the year at REGENXBIO and are in store for an exciting remainder of 2025. Today, you will hear about our strong late-stage clinical progress, which sets us up to potentially bring multiple first or best-in-class gene therapies to patients over the coming years. We will also discuss other key elements that we believe position us to successfully transition to commercial stage, including our U.S.-based in-house commercial-ready manufacturing and broad potential to secure non-dilutive funds. With me on today's call are Dr. Steve Pakola, our Chief Medical Officer, and Mitch Chan, our Chief Financial Officer. I'll start with a review of recent business highlights, then turn it over to Steve and Mitch for clinical and financial updates. Then I'll have a few closing remarks before opening the call for Q&A.

Starting with our most advanced program, RGX-121, or clemidzagene lemparvovec, the potential first gene therapy and one-time treatment for MPS II or Hunter syndrome, a devastating disease that affects approximately 2,000 patients worldwide. Any day now, we expect the FDA acceptance of the BLA, which we submitted under the accelerated approval pathway in March 2025. RGX-121 remains on track for potential FDA approval in the second half of this year. Earlier this year, we established a key strategic partnership with Nippon Shinyaku to commercialize our neurodegenerative franchise, including RGX-121 and RGX-111 for severe MPS I, and commercial preparations are progressing well. Together with Nippon, our goal is to deliver RGX-121 to patients beginning in the first half of 2026. Accelerating quickly, right behind RGX-121, is RGX-202, our next-generation candidate for Duchenne muscular dystrophy. I am pleased to report that our pivotal study continues advancing rapidly.

We have surpassed 50% enrollment for our pivotal dataset. We're seeing increased interest and enthusiasm from the patient community about RGX-202 and its differentiated profile, and we remain on track to submit a BLA in mid-2026 and seize our unique second-to-market or fast-follower opportunity in Duchenne. Positive input from our growing investigator community supports our belief that RGX-202 has the potential to be a preferred and differentiated treatment option. It's important to keep in mind that, one, RGX-202 is the only investigational next-generation DMD gene therapy in pivotal study and the only investigational therapy with both robust microdystrophin and functional data available. Two, RGX-202 has the potential to be approved for patients aged one to three who currently have no access to gene therapy.

DMD represents a large addressable market, with over half of the prevalent DMD population projected to remain untreated as of 2027, the expected year of RGX-202's commercial launch. Given our strong and rapid clinical progress, conviction in our differentiated profile, and in recognition of the ongoing unmet need, we will begin producing RGX-202 commercial supply at our Manufacturing Innovation Center here in Rockville, Maryland, in the third quarter of this year. With full clinical and planned confirmatory supply already in hand, we will build commercial inventory to be prepared for a smooth launch and meet patient needs immediately upon the potential approval of RGX-202.

As a reminder, our Manufacturing Innovation Center is a state-of-the-art integrated GMP facility that can produce up to 2,500 doses of RGX-202 annually, enough to treat approximately one-fifth of the estimated North American DMD population, all while delivering industry-leading purity levels in Duchenne with over 80% full capsules. We look forward to sharing additional phase I-to-functional data for RGX-202 in the first half of this year as we aggressively advance towards commercialization. Moving to our retinal programs, we continue to work closely with our partner AbbVie to progress ABBV-RGX-314, or cerabgene lemparvovec. 314 is advancing in two pivotal studies for subretinal wet AMD, one phase II study for suprachoroidal wet AMD, and preparations for a pivotal program are underway in diabetic retinopathy, or DR, using suprachoroidal delivery.

We remain on track to be the first gene therapy on the market for wet AMD with a product that has demonstrated compelling durability and strong patient interest. Both wet AMD and DR represent large multi-billion-dollar commercial opportunities, and we believe 314 has the potential to preserve vision and serve as a meaningful alternative to today's standard of care. In summary, we remain excited for and are well-positioned to deliver on the 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?

Steve Pakola (CMO)

Thank you, Curran. I'll start with RGX-202, a potential one-time gene therapy for the treatment of Duchenne. RGX-202 uses the NAV AAV8 vector and is the only microdystrophin construct to include the C-terminal domain, a key element of naturally occurring dystrophin critical to protecting muscle from contraction-induced damage. This novel construct, combined with the highest purity levels in the field, makes 202 a potential best-in-class gene therapy for Duchenne. As Curran mentioned, I'm pleased to share that today we announced the pivotal phase of the Affinity Duchenne trial is beyond 50% enrolled. Our accumulating phase one-to-results are showing impressive evidence of differentiation on safety, biomarker, and functional outcomes. This is driving increased excitement throughout the Duchenne community, giving us further confidence as we move toward our target of completing pivotal enrollment in the second half of this year.

This trial is enrolling ambulatory patients aged one and above, generating data where limited results exist for Duchenne gene therapies. Based on the clinical profile and in partnership with our investigators, we expanded the inclusion criteria to a broader range of exon mutations. Additional trial sites continue opening in the U.S. and Canada, and we are making great strides in our pivotal strategy to enroll across a wider age range with an aim to secure a broad label supported by a clear and robust product profile. In November, we reported positive safety and efficacy data from the phase one-to-two study, including positive functional outcomes from the first five participants at nine and twelve months. These results presented last November showed 202 recipients exceeded external natural history controls and established benchmarks for clinical outcomes.

Specifically, we observed functional improvements in all five evaluated patients, including those at dose level two, consistent robust expression, transduction, and localization of our differentiated 202 microdystrophin in muscle with all participants above 10% expression. We have also seen a favorable safety profile with no serious adverse events or AEs of special interest. Building on this data, in March at the 2025 MDA conference, we presented new biomarker data from two patients, including the first data from our cohort of patients under four years of age. This patient, age three at dosing, had a microdystrophin expression level of 122% of control. Patients age one to three represent a significant portion of the prevalent Duchenne population, yet this group has no access to approved gene therapy.

Overall, the phase one-to-two data show consistent microdystrophin expression in all 12 patients spanning all age groups, functional improvements, and evidence of altering the trajectory of disease and a favorable safety profile. As the program advances, we expect an increased focus on the dose level two cohorts as these patients receive the commercial dose level. We look forward to sharing additional data from the phase one-to-two study in the first half of this year. Now on to our retina franchise, 314, which is being developed in collaboration with AbbVie to treat wet AMD and diabetic retinopathy, or DR. I'll start with 314 for DR, being evaluated in the phase two ALTITUDE trial using in-office suprachoroidal delivery. Like wet AMD, DR is a progressive disease that causes vision loss and ultimately blindness if not treated appropriately.

As we've shared, we completed our end-to-phase two meeting with the FDA in the fourth quarter of last year and are actively working with AbbVie on plans for our phase three clinical program that would support global regulatory filings. We look forward to sharing more on that program as preparations progress. In wet AMD, we are evaluating 314 via two different delivery forms, subretinal and suprachoroidal. Within subretinal, we have two ongoing pivotal trials, Atmosphere and Ascent, in the U.S., Europe, and Japan. These trials continue to progress well. As we've announced in January, enrollment of both pivotal trials is expected to complete this year, and we expect to share top-line data in 2026.

314 is on track to be the first gene therapy on the market for wet AMD and is poised to deliver a compelling product profile based on the impressive safety and durability seen in our phase 1/2a trial. In this trial, 314 achieved meaningful reduction in treatment burden and sustained treatment effect through four years. Overall, we remain encouraged by the ongoing progress with 314. I'd like to particularly highlight the differentiated safety profile observed in our in-office suprachoroidal program. This is particularly notable in the setting of short-course seven-week prophylactic steroid eye drops, a significantly shorter regimen than those used in other gene therapy trials. This continues to support the potential of 314 as a meaningful treatment option for patients and physicians.

Finally, turning to our MPS programs, it's an incredibly exciting time for REGENXBIO in the Hunter syndrome community with the recent submission of our BLA for RGX-121. This filing is supported by data from the Campsite trial, which met its primary pivotal endpoint with high statistical significance. In addition, we previously reported that 80% of pivotal dose patients either discontinued enzyme replacement therapy or remained treatment naive, along with evidence of neurodevelopment improvement sustained through four years post-dosing. 121 represents a potential significant advancement, not only improving patient outcomes but also improving the daily lives of patients and families. As a one-time gene therapy, 121 has the potential to achieve these benefits while also reducing the treatment burden and the significant amount of time families spend getting weekly enzyme replacement therapy. We look forward to an anticipated FDA approval decision in the second half of 2025.

To conclude, we are making significant progress with data updates and trial progression across all programs in our pipeline. I'd like to thank all of the patients, families, clinicians, and patient advocacy representatives who have been involved in and supported all of these trials. With that, I'll turn the call over to Mitch to review our financial guidance. Mitch?

Mitch Chan (CFO)

Thank you, Steve. REGENXBIO ended the quarter on March 31st, 2025, with cash, cash equivalent, and marketable securities of $272 million, compared to $245 million as of December 31st, 2024. The increase was primarily driven by the $110 million upfront payment received under the Nippon Shinyaku collaboration and was partially offset by cash used to fund operating activities during the first quarter of 2025. R&D expenses were $53 million for the quarter ended March 31, 2025, compared to $54.8 million for the quarter ended March 31st, 2024. The decrease was primarily due to clinical trial expenses for ABBV-RGX-314 and RGX-202. We expect the balance in cash, cash equivalent, and marketable securities of $272 million as of March 31st, 2025, to fund our operations into the second half of 2026.

This cash runway guidance is based on the company's current operational plans and excludes the impact of any future potential royalty income associated with RGX-121. If we include additional non-dilutive financing, we expect the cash runway to potentially extend well beyond 2026. For instance, some examples of our non-dilutive financing options include development and sales milestones, reversion of our royalty income, and the potential sale of our priority review voucher for RGX-121, which has recently sold for at least $150 million. Collectively, we have many non-dilutive financing optionalities that could extend our cash runway well beyond the second half of 2026. With that, I turn the call back to Curran to provide final thoughts.

Curran Simpson (President and CEO)

Thanks, Mitch. As you heard today, there's tremendous momentum and strong execution across our pipeline to advance our programs towards key milestones. To recap, we have submitted our first BLA, and our partnership with Nippon Shinyaku for our MPS programs is off to a strong start. We look forward to a potential FDA approval of RGX-121 this year. The pivotal study of RGX-202 is moving rapidly, and we believe it remains well-positioned to potentially serve as the next and preferred gene therapy in Duchenne. We are planning for success and will initiate commercial supply manufacturing in the coming months in anticipation of a 2027 commercial launch. We also plan to share updated functional data in the first half of this year. With enrollment more than halfway through, we expect to complete enrollment of the pivotal study this year and share top-line data in the first half of 2026.

Our partnership with AbbVie is advancing towards multiple large global commercial opportunities. We expect to complete enrollment of two global pivotal trials for subretinal wet AMD and are working with AbbVie on a pivotal study in diabetic retinopathy in 2025. As we lead the way in AAV Gene therapy, our team is looking forward to presenting at ASGCT this week. Our presentations in New Orleans highlight our in-house end-to-end capabilities and deep translational expertise. Presentations will include preclinical research supporting the malleable construct of RGX-202, including the C-terminal domain, capsid discovery research, and the RGX-202 manufacturing process development, enabling industry-leading purity levels in Duchenne gene therapy. We are in a very unique position in our industry.

Each of our assets represent one-time treatments with the potential to transform the trajectory and management of disease for patients in need of new and better options and are demonstrating beneficial differentiation against standard of care and available treatments. We have a balance sheet that enables us to better navigate the current macro environment and execute against our near-term and exciting milestones. With that, thanks everyone for your time today. I'll turn the call over for questions. Operator?

Operator (participant)

Thank you. As a reminder, to ask a question, please press star one one. If your question has been answered and you'd like to remove yourself from the queue, please press star one one again. Our first question comes from Mani Foroohar with Leerink Partners. Your line is open.

Mani Foroohar (Senior Managing Director of Genetic Medicines)

Hey, guys. Thanks for taking the call. I've got two quick ones. I think first is around timing for the Hunter BLA you mentioned this month. I had a couple of people who've reached out around timing, and I admit to doing this math myself. With 60 days marched out from March 13th, it should be right around now. Is that math too close, or are we thinking about it wrong? I think there's just a little bit of sensitivity around any hypothetical FDA delay, or is this just we're not taking into account federal holidays? I have a follow-up.

Curran Simpson (President and CEO)

Hi, Mani. Thanks for the call and the question. Yeah, we're certainly pointing towards imminent in terms of timing around the BLA acceptance. I think we feel really good about where the review stands. We've been getting regular interactions in a few areas, the information requests that normally come in as part of the review. Also, we do orientation meetings early on regarding the submission and the BLA contents. All of that is pretty much business normal. We haven't gotten any questions that would lead us to believe otherwise. I think you'll expect to hear from us soon about, hopefully, an acceptance.

Mani Foroohar (Senior Managing Director of Genetic Medicines)

Great. Moving on to something a little more substantive. Obviously, you and others have watched closely as your competitor, Sarepta, has had some stumbles both commercially and from a regulatory perspective, and there have been changes at the helm of CBER, which have driven a lot of debate around the space. I'm not the first or the second or 1,000th to ask that question. How do you think about the bar for approvability in DMD on an accelerated basis? Expectations around biomarker/biomarker data versus functional data. Where is that conversation, and how has it evolved with the additional input of what we've seen from Sarepta, as well as any potential changes at the helm of CBER?

Curran Simpson (President and CEO)

Yeah, thanks. I think from the very beginning on the program, we've pointed towards an accelerated approval pathway. Those were discussions we had with the FDA in our end-of-phase two meeting, which were very positive and productive. Our intention from the beginning has been to provide functional corollary, if you will, to microdystrophin as part of the filing and review process. We feel really good about our data, the strength of the data in terms of safety profile and initial functional data that we provided. We really look forward to additional functional updates the first half of this year to continue that story. I think we've seen nothing but continued and maybe increased opportunity for us, given the benefit-to-risk ratio we think we're developing and the existence of what we think will be a very sizable prevalent market upon potential approval in 2027.

I think our conviction and our opportunity only continues to increase as the program evolves. I think in terms of how FDA would view that, I think certainly Dr. Makary provided good context on rare disease development and the support for programs like ours where I think we can provide benefit to patients.

Mani Foroohar (Senior Managing Director of Genetic Medicines)

Great. Thanks for that clarity, guys.

Curran Simpson (President and CEO)

Thanks.

Operator (participant)

Thank you. Our next question comes from Gena Wang with Barclays. Your line is open.

Gena Wang (Managing Director and Biotech Equity Research)

Thank you for taking my questions. I will follow Mani's questions. I will also ask a few more. Giving recent elevator safety event, I will ask a very specific question, like, do you expect any changes from the FDA regarding requirement of safety profiles? Also, for your pivotal study, any changes in terms of enrollment speed and the patient type? My second question, lastly, after recent appointment of a new CBAR director, do you expect any changes on your accelerated approval path that was previously aligned with the FDA?

Curran Simpson (President and CEO)

I think I'll take the last one. I think it's very early to project any potential changes in accelerated approval, but we certainly don't see any signs of that to date. Our program, in terms of enrollment, is absolutely on track with how we projected it. We feel highly confident that we will enroll the study this year, the pivotal study of N equals 30. We'll continue to do enrollment throughout the remainder of the year as well to support our confirmatory study as well. I think from the beginning of the program, I've been pointing towards during the time of submission and review, having substantial functional data to accompany that. I don't see anything emerging in some of the dialogue that we've heard from FDA that's contrary to that approach. I think our plan is unaltered at this point regarding that.

I do think that the strong functional data we reported in November, coupled with the safety profile to date that we've been able to portray in our earnings and press releases, really points towards potential improvement in benefit-to-risk for Duchenne patients. We think that will be something that will be interesting to the review team as we complete our filing next year. I do think that this points towards our immune suppression regimen, which is, I think, innovative in the sense of proactively addressing known SAE types for Duchenne patients treated with high-dose AAV. I think looking at that approach today is directly in line with potentially better outcomes for patients, is what we've seen so far in terms of not just the experience during treatment, but then the post-treatment monitoring. Maybe I'll let Steve elaborate a bit on that from the clinical perspective.

Steve Pakola (CMO)

Hi, Gena. Thanks for the question. It is certainly an area of high interest in the Duchenne community given the recent death, unfortunately, with the Elevatus program. I'd say the overarching aspect is that I think you've heard and seen, and we certainly have seen this both at MDA and in our subsequent one-on-one discussions, is this just brightens the spotlight on safety. I think even before this event, safety is king. I think that's why a lot of the points that Curran mentioned are only giving greater comfort to our investigators and the patient families that they speak to about the differentiated aspects of our program. I think this is why we designed the construct that we did.

This is why over the years we've advanced, and we're very proud of our high purity level with the highest full-to-empty capsid ratio in the field, and also the robust immune modulation regimen that Curran mentioned. I think those factors are giving us and the community comfort, and that's why we're absolutely on track with our recruitment.

Gena Wang (Managing Director and Biotech Equity Research)

Thank you.

Curran Simpson (President and CEO)

I think I'd also point to the imminent review and outcome of the hopeful acceptance of the 121 program. I think that'll be sort of an early data point on accelerated approval. As you know, the Hunter program is following an accelerated approval pathway. In that case, we had a pre-BLA meeting that was also supportive for that program. Assuming that we conclude with an acceptance of that BLA in the near term, I think that'll be a first data point to support that that pathway is still viable and an active process in the FDA.

Operator (participant)

Thank you. Our next question comes from Juddah Frommer with Morgan Stanley. Your line is open.

Juddah Frommer (Executive Director and Senior Equity Research Analyst)

Yeah. Hi, guys. Congrats on the progress. Thanks for taking the questions. First, I was hoping we could get a little incremental color on the planning process along with AbbVie for the diabetic retinopathy phase three trial, any updated thinking on timing or potential impacts, the probability of recognizing that milestone. Then separately, just more broadly on interactions with FDA, given how many programs you have that you are interacting with them on, any changes recently you'd point to, or is it sort of business as usual and contact with the same individuals as you'd anticipate? Thank you.

Curran Simpson (President and CEO)

Thanks. Yeah, I think I can probably comment on the second question first, which is we are actually having a pretty significant number of interactions with FDA, both on submissions that have already been filed and information requests that are being provided. The only way I can really characterize it right now is that it is very much business normal. Without getting into detail on the type or the details of information requests, none of them are really questioning the broader strategy of the program or the clinical data that was provided. It is more customary questions about the CMC process, etc. I think I just would characterize it as business normal. The review teams seem to be quite intact in terms of good continuity with the teams that we met with last year.

Like I said, with the Hunter program, we're going to have a very near-term outcome in terms of BLA acceptance, we hope, which will confirm that we're on track for a late-fall potential approval. On DR, we are continuing to collect the final feedback that, as we pointed to early in the year, AbbVie and we have obtained feedback from U.S., EU, Japan regulatory agencies. As you know, in DR, there's choices to be made in terms of endpoints that have already been established, two steps worsening, two steps improvement. All of those are being considered as part of developing a final pivotal protocol. Once that's complete, we'll begin site activation, etc. We're still pointing towards first patient dose this year, and we'll be more specific on timing as we get near that event.

Operator (participant)

Thank you. Our next question comes from Ellie Merle with UBS. Your line is open.

Hi, guys. This is Tejas on for Ellie. I guess just some of the incremental updates we can expect this year in DMD. You mentioned some additional functional data in 1H. Am I correct in assuming that's not at ASGCT? Is there an arena you'd expect to present that at, or should we expect a press release of that data? You also mentioned starting commercial supply manufacturing later this year. You meant it's supply, but is it the same process that you're using in your current trials? Will you be able to provide any color on the CMC process with the FDA or any other meetings you might have on the statistical plan throughout the year? Thank you.

Curran Simpson (President and CEO)

Sure. Let me start with the CMC process. Yes, we did point to initiation of commercial production this year. That is a combined effort. Number one, the batches that are produced as part of our first commercial production are also batches that will be submitted as part of the planned mid-2026 BLA. The process that will be validated as part of that exercise is the exact same process that is in the clinic today. No changes anticipated to what patients are being dosed currently in our pivotal program as we move to our commercial process. As we have pointed out, we have a quite mature CMC approach off our platform, NAVXpress process, which we think FDA will really regard highly. In fact, it is the same process that they have reviewed in other programs and actually toured on-site as part of FDA training in the last couple of years.

We feel really strongly that we have a very low-risk approach to CMC with a process that produces very high purity capsid, which FDA has been clear as an expectation, and not any significant changes required to get to commercial, in which we can produce 2,500 doses per year. Back to the first question. In terms of data, there's only, I think, six weeks left in first half 2025. I think you could expect likely a press release around the additional data. The focus of the data will really be on expanding the dose-level 2 patients that were treated in the phase one-two study out to 12 months. A larger number of patients out that far and showing all the same functional outcomes that we did in November.

Just trying to enlarge the data set there and as well update on any biomarker data that may have come in recently. It really will be focusing on the pivotal level dose and how those patients are doing. I think just in general, the feedback we're getting from sites and from investigators is really positive right now.

Thanks. And just quickly, those pivotal dose patients in the phase I/II will be included in the pivotal data set for filing in mid-2026, correct?

Yeah. All patients that qualify for the inclusion criteria of the pivotal program would be included in the pivotal data set.

Thanks.

Operator (participant)

Thank you. Our next question comes from Annabel Samimy with Stifel. Your line is open.

Annabel Samimy (Managing Director and Senior Analyst in Healthcare)

Hi. Thanks for taking my question. Just to go back to regulatory bodies and changes there, just given the FDA's focus on safety with gene therapy, I guess we've been talking a lot about the rare conditions and how the carrier is very focused on the rare diseases and life-threatening conditions. Does that make it potentially harder for gene therapy and non-life-threatening conditions like retinal disease, for example? Are there any issues related to that for the DR indication, any delays there? One last question regarding regulatory bodies. RFK Jr. just asked the HHS Committee on Genetic Screening. Can you tell us how this might affect not MPS, but rather DMD in trying to get into the younger population and whether there are any complications there? Thanks.

Curran Simpson (President and CEO)

Sure. I can take the second one, but I think maybe first I'll let Steve comment on whether or not we're seeing any change in FDA stance regarding retinal programs. I think what I would convey before Steve speaks is I think we know that safety in non-rare is an absolute focus. I think that's probably the incredible promise of the subretinal program. The safety profile in the data that we've seen today looks excellent. Maybe I'll let Steve elaborate on that a little bit.

Steve Pakola (CMO)

Sure. Hi, Annabel. On retina, I think another broad aspect is that's not an accelerated approval pathway. There is not a request for greater flexibility. Fortunately, there we know the regulatory route, and we're already executing on that, for example, in subretinal. We have a plan to do the same in suprachoroidal. On the safety side, just as Curran mentioned, we feel very good about the safety package that we'll have for subretinal and suprachoroidal. That's because we've intentionally selected routes of administration for 314 that are compartmentalized and that decrease the risk of the types of safety findings, specifically inflammation, that have really hounded other routes of administration like intravitreal. We feel very well set up not only in our rare programs, but also in the common retina indications that we're looking at when it comes to safety.

Curran Simpson (President and CEO)

Annabel, to come back to the question on genetic screening, newborn screening, we're working closely with the MPS Society and with the Duchenne Advocacy Groups on newborn screening. Their approach largely is at a state level to advance and increase the number of states that provide that. I think it would be ideal if all entities were working together in concert on that. That does not mean that if one group is not supporting it, the others will not be able to fill that gap and move forward. We see that as a priority in rare disease, and it is something we will work with the advocacy groups to support.

Annabel Samimy (Managing Director and Senior Analyst in Healthcare)

Okay. If I can just ask one quick follow-up to be clear on the last question about ASGCT, you will not be presenting functional data there, correct? I understood it to be a PR within the next six weeks. Is that understandable?

Curran Simpson (President and CEO)

Yeah. Yeah. It will not be at ASGCT, so no need to book your flight. We'll definitely likely provide that as a press release in the near term.

Annabel Samimy (Managing Director and Senior Analyst in Healthcare)

Okay. Great. Thank you.

Operator (participant)

Thank you. Our next question comes from Alec Shanahan with Bank of America. Your line is open.

Matthew Harrison (Managing Director and Senior Equity Research Analyst)

Hey, guys. Thanks for taking our questions. This is Matthew on for Alec. Maybe just double-clicking on a previous point. Has there been any change in baseline characteristics, patient age, etc., that you've seen for the DMD trial? Then maybe second, what's the percentage purity for your preps that you're comfortable with or looking for sort of as you're moving forward with manufacturing?

Curran Simpson (President and CEO)

Yeah. That is something that will be shown at ASGCT. We have a presentation on the CMC process that I think will give quite a nice level of detail for people to see. We're very proud of it. The purity level for the product is 80% or greater. I think just as important, consistency between batches, which we've heard others struggle with, is also highly consistent between batches. That is what we would point to. In terms of, I think your question was around enrollment of the pivotal study, we're quite pleased so far with not just the pace of enrollment, but the breadth of patient ages that are enrolling in the study to date.

As we look at it today and as we look out towards the remainder of the year, we feel like we will have a very balanced number of patients across the ages to support a broad label when we file.

Operator (participant)

Thank you. Our next question comes from Luca Issi with RBC Capital Markets. Your line is open.

Oh, great. Hi, team. This is Shelby on for Luca. Thanks for taking the question. Maybe on wet AMD subretinal, you have shown recently that the fellow eye can be treated safely, confirming that the eye is somewhat immune privileged. Are you planning on filing that data? Is your initial label going to reflect bilateral dosing? Any color there? Much appreciated. Thanks.

Curran Simpson (President and CEO)

Hi, Shelby. Nice to see you. Steve is part of the joint development committee that plans sort of the strategy around the filings. Maybe he can comment on that question.

Steve Pakola (CMO)

Sure. Hi, Shelby. Great question. This is actually something that we prospectively addressed even as early as our end-of-phase II meeting because it's very relevant in wet AMD where most patients do have bilateral disease. It would be very advantageous if both eyes ultimately could be treated. We, again, always saw that our safer routes of administration, where you would expect less immune response, would be more amenable to being able to treat both eyes. That is why we prospectively addressed this explicitly with the FDA. That is what led to our fellow eye study. As you mentioned, we're seeing that we're in a good position as far as fellow eye. Yes, I confirm that that's certainly our intent, that this is certainly part of any package that would go in that would inform clinicians on being able to treat both eyes.

Operator (participant)

Thank you. Our next question comes from Paul Choi with Goldman Sachs. Your line is open.

Paul Choi (Biotechnology Analyst)

Hi. Thank you. Good afternoon. Thanks for taking our questions. I want to ask first on RGX-121 at Hunter's with the expectations of an imminent acceptance of your BLA. I just want to see if you're also potentially assuming an adcom, given that this could potentially be the first approved gene therapy for the condition. Second, regarding the RGX-202 Duchenne phase one-two functional data update that you referenced earlier, can you maybe just comment on, again, how many patients we'd expect follow-up for and how current the data cut might potentially be for that functional update that's coming up soon? Thank you.

Curran Simpson (President and CEO)

Sure. Yeah. I can comment on the first question around adcom. We are planning as if we need an adcom. From an internal perspective, we have a team working on being ready for that should it be requested. We do not have a final answer from FDA as to whether one is required yet. Initial discussions were hinting towards no, but we are waiting to get confirmation one way or another through the review process. We will be prepared if one is needed ahead of time. For 202, in terms of just coming back to—oh, I am sorry. Yeah. About the next upcoming functional release. We are planning on enlarging the data set to four, possibly five patients dosed at DL2 as part of the functional update. Not all of them may be out to 12 months, but minimally, they would be out to 9 months.

That's still something that we're pulling together, QCing the data, and looking forward to getting out before the first half of the year.

Paul Choi (Biotechnology Analyst)

Okay. Great. Thank you for that.

Operator (participant)

Thank you. Our next question comes from Brian Scorney with Baird. Your line is open.

This is Luke on for Brian. Thanks for the question. Sorry if this was already asked, but on the 202 commercial build-out, has the team set a goal for the quantity of product that you expect to be available by the time of a potential launch?

Curran Simpson (President and CEO)

It's a great question. We're actually going through that process of review now as we evaluate our commercial strategy in total. I would just remind you that in the course of one year, we can produce 2,500 doses. We will come to a point as we get closer to review of the BLA to talk a little bit more about how many doses we would have at launch. We would certainly want to be in a position to quickly address the prevalent market with a significant number of doses. We would estimate the prevalent market could be in the ambulatory side of the market, something in the order of 5,000-7,000 patients. We would want to be prepared to quickly be able to address that market.

We'll get more specific on the number of doses available at launch, but we're in a unique position to be able to build a significant amount of inventory ahead of 2027.

Great. Thanks. And just one more quick one. On your 314 wet AMD studies, has the availability of Pavlu impacted enrollment pace at all, or are these generally unique groups of patients an indication?

Thanks for that one. I think I'll ask Steve. He's a little closer to that particular question.

Steve Pakola (CMO)

Yeah. Across different agents that are out there, either on the market or investigational, we over time haven't seen any of that impact our recruitment. We're at a good clip. That's why we keep reiterating our guidance. We're excited to finish enrollment this year. Excited to have top-line results next year.

Awesome. Thank you.

Operator (participant)

Thank you. Our next question comes from Sean McCutcheon with Raymond James. Your line is open.

Sean McCutcheon (VP of Biotechnology Equity Research)

Hey, guys. Thanks for taking the question. Just to build on wet AMD, can you provide some detail on how you're thinking about the suprachoroidal opportunity? Obviously, we're looking forward to the start of the NPDR phase three, but I think we're all curious on the dose necessary to move forward in wet AMD and how you're thinking about that value proposition on the heels of subretinal. Thanks.

Curran Simpson (President and CEO)

Hi. I think I'll let Steve answer that question.

Steve Pakola (CMO)

Sure. This is obviously a massive market. If you look at the retina space driven by the anti-VEGF target mechanism and treatments thereof, we are at $18 billion and continuing to grow. I think there is a lot of opportunity across the VEGF-driven retinopathies. We in AbbVie are advancing, as we were just saying, on the subretinal global program for wet AMD. We are obviously excited about subretinal delivery for that indication. We do see increased optionality and opportunities with the one-time in-office suprachoroidal, and that is why we both are advancing in that space.

I think DR in particular is compelling when you consider the reality that patients with non-proliferative diabetic retinopathy who have not developed the sight-threatening complications like DME and like proliferative disease, that these patients really are going to need an in-office one-time treatment option that even if you have greater durability agents, if you're going to need repeat injections indefinitely for the rest of your life, that's not going to be a very compelling opportunity. That is why we in AbbVie are so excited about the opportunity of a one-time in-office treatment to really address that unmet need of DR.

Sean McCutcheon (VP of Biotechnology Equity Research)

Thank you.

Operator (participant)

Thank you. As a reminder, to ask a question, please press star one one. Our next question comes from Yi Chen with HC Wainwright & Company. Your line is open.

Eduardo Martinez-Montes (Research Analyst)

Hi there. Good afternoon. This is Eduardo on for Yi. Just a quick question if you had any thoughts on the recent announcement about this morning, I think, or last night about potential pricing on branded drugs. Do you feel like that's going to have a significant impact on your ability to price your gene therapies across these indications?

Curran Simpson (President and CEO)

Yeah. I think it's early to tell. I think if you look at some of the commentary by John Crowley from Bio or the Alliance for Regenerative Medicine, I think that initially, the impact to cell and gene therapy in general might be less than could be for broader branded therapies. It's too early to tell. I think from our perspective, there's no immediate impact to anything that we're doing, but certainly something we have to pay attention to in sort of the macro environment as we get closer to an approval.

Eduardo Martinez-Montes (Research Analyst)

Got it. Thanks. That's really helpful.

Operator (participant)

Thank you. I'm showing no further questions at this time. This does conclude the question-and-answer session, and you may now disconnect. Everyone, enjoy the rest of your day.