REGENXBIO Q3 2025 Earnings Call Transcript

Quartr
Provided by Quartr
November 6, 2025

Key Takeaways

  • Positive Sentiment: RGX-202 (Duchenne) — Enrollment in the Affinity pivotal trial is complete, with top-line pivotal data expected in early Q2 2026, a mid‑2026 BLA planned via the accelerated pathway, and in‑house commercial manufacturing (PPQ imminent) supporting a 2027 launch.
  • Positive Sentiment: RGX-121 (MPS II) — The BLA is under review with a PDUFA date of February 8, 2026, FDA inspections of clinical sites and the Rockville facility completed with no observations, and positive 12‑month data supporting approval and early‑2026 commercial dosing plans with partner Nippon Shinyaku.
  • Positive Sentiment: ABBV‑RGX‑314 (Seravec) — retinal franchise — AbbVie/REGENX completed enrollment in two global phase 3 subretinal wet AMD studies (>1,200 patients) with top‑line readout expected Q4 2026, and a suprachoroidal DR pivotal program is advancing toward phase 2b/3 initiation.
  • Neutral Sentiment: Financial position and runway — Cash and equivalents were $302 million at 9/30/25, which management says funds operations into early 2027, with potential non‑dilutive opportunities (PRV sale, milestone payments, royalty monetization) that could extend runway further but are not guaranteed.
AI Generated. May Contain Errors.
Earnings Conference Call
REGENXBIO Q3 2025
00:00 / 00:00

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Operator

Welcome, everyone, to the third quarter 2025 REGENXBIO earnings conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during this session, please press star one one on your telephone and wait for your name to be announced. Please limit yourself to one question and one follow-up, and if you have additional questions, please return to the queue. To withdraw your question, please press star one one again. Please be advised that today's conference is being 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
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 third quarter ended September 30th, 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, 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 section of REGENXBIO's annual report on Form 10-K for the full year ended December 31st, 2024.

Patrick Christmas
Patrick Christmas
Chief Legal Officer at REGENXBIO

Comparable risk factor sections of REGENXBIO'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, November 6th, 2025. We undertake no obligation to update any forward-looking statements we may make on this call on account of new information, future events, or otherwise. Please be advised that today's call is being recorded and webcast. In addition, any unaudited or pro forma financial information that may be provided is preliminary and does not purport to project financial positions or operating results of the company. Actual results may differ materially. I'll now turn the call to Curran Simpson, President and CEO of REGENXBIO. Curran.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Thank you, Patrick, and thank you, everyone, for joining us today. I am pleased to share the strong momentum across our late-stage pipeline of gene therapies with the potential to deliver the best outcomes in devastating diseases. Our progress is anchored in our leading end-to-end capabilities, including in-house, commercial-ready manufacturing, and innovative science, all driven by our commitment to bring new medicines to patients in need. Today, I am joined by Dr. Steve Pakola, our Chief Medical Officer, to discuss the exciting clinical development across our rare and retinal franchises, and Mitch Chan, our Chief Financial Officer, to provide financial updates. Let's dive in and start with our RGX-202, our wholly-owned program for Duchenne Muscular Dystrophy. We are very encouraged by our progress in this program and are moving rapidly to deliver a much-needed differentiated treatment to Duchenne patients. Our comprehensive therapeutic approach behind RGX-202 includes a novel construct.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

RGX-202 is the only microdystrophin that includes the CT domain, making it closest to naturally occurring dystrophin. A novel immune suppression regimen designed to enable high-dose delivery and proactively counter issues seen in other AAV programs. Commercial-ready manufacturing is delivering the highest purity levels in Duchenne gene therapies. Boys with Duchenne have one chance at gene therapy. That is why we specifically designed RGX-202 with these elements to maximize opportunity for functional benefit. We are very pleased to see that this approach is resulting in the favorable safety and efficacy profile seen in phase I/II. We are also very excited to announce that we completed enrollment in the AFFINITY DUCHENNE Pivotal Trial, putting us on track to share top-line pivotal data in early Q2 2026. We will submit a BLA using the accelerated approval pathway in mid-2026.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

To meet demand from the patient community and support accelerated approval plans, the confirmatory trial is open and continues to enroll patients. As a reminder, we were able to complete enrollment ahead of our original year-end guidance, underscoring the community's enthusiasm for RGX-202 and strong need for new treatment options. We have more than enough supply of RGX-202 ready and available for the entire confirmatory study. Delivering on the commercial readiness plans we shared last quarter, we have produced the first batches of RGX-202 intended for commercial supply at our Manufacturing Innovation Center here in Rockville and expect to imminently complete our PPQ, or Process Performance Qualification, campaign. There is a significant commercial opportunity for RGX-202 due to our novel construct, immune suppression regimen, and our manufacturing capabilities, including our ability to produce 2,500 doses per year, which make us uniquely prepared for clinical and commercial success.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

We continue to invest in preparations for a commercial launch in 2027, when the vast majority of the prevalent population will remain available. Now to RGX-121, also known as clemidsogene lanparvovec, the potential first gene therapy and one-time treatment for MPS II. Our recent FDA interactions regarding the ongoing BLA have been highly productive, and we remain confident in RGX-121 approval by early next year. We delivered positive 12-month data to FDA, which Steve will discuss in more detail. The FDA completed inspections of our clinical sites and in-house manufacturing facility with no observations, a rare and significant achievement. These developments have further strengthened our confidence in the ongoing BLA review. With our commercial MPS partner, Nippon Shinyaku, we look forward to the February 8 PDUFA date and delivering the first commercial doses of RGX-121 by early next year.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Let's turn our focus to our retinal disease franchise and our ongoing partnership with AbbVie to surabgene lomparvovec, or sura-vec, for wet AMD and diabetic retinopathy. These programs have the strength of AbbVie's leading clinical and commercial global eye care infrastructure behind them. In subretinal wet AMD, we recently announced that the last patient was enrolled in our two global phase III studies. This is a tremendous accomplishment. Together, ATMOSPHERE and ASCENT represent the largest global gene therapy program ever conducted, with over 1,200 patients enrolled across 200 sites. If approved, subretinal sura-vec would not only be the first gene therapy for wet AMD, a disease that impacts millions worldwide, but also the first gene therapy for a non-rare indication. We look forward to sharing top-line data in the fourth quarter of 2026. As a reminder, we manufacture all clinical and future commercial sura-vec at our facility here in Rockville.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Like wet AMD, our diabetic retinopathy program is designed to deliver sura-vec to patients that rely on frequent lifelong injections to halt degeneration and vision loss, and we look forward to initiating the phase II-B/III pivotal program. Across our pipeline, these achievements reflect the strength of focused execution and a differentiated approach to developing and delivering best-in-class therapeutics. With that, I would like to now turn the call over to Steve for more in-depth updates on our clinical programs. Steve?

Steve Pakola
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 mentioned, we are incredibly excited that enrollment has completed in the AFFINITY DUCHENNE Pivotal Trial. As a reminder, this study is designed to enroll ambulatory patients aged one and older and is the most advanced clinical stage gene therapy program for Duchenne. RGX-202 has demonstrated a highly differentiated safety and efficacy profile with consistent, robust microdystrophin expression in the phase I/II study. Last month, at the International Congress of the World Muscle Society, we presented individual and SAA data on the first four patients who received the Pivotal dose. All four patients, one year after dosing, exceeded expected disease trajectory across multiple methods of assessment. Specifically, each patient exceeded their expected functional outcomes when compared to matched external controls and the well-established CTAP disease progression model.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

These data, combined with the June 2025 data showing all patients demonstrated improvement on timed function tests, reinforce our belief that 202 is driving meaningful functional benefits for patients with this degenerative disease. It's important to note the majority of these patients were eight years and older at dosing, an age when functional decline is expected, making these results particularly impressive. The Duchenne patient and physician communities continue to recognize the excellent safety profile 202 has demonstrated to date. As reported in the phase I/II study, we have seen no SAEs or adverse events of special interest, including no thrombocytopenia or liver injury. We attribute this to our proactive immune suppression regimen, our novel construct using the NAV AAV8 vector, and our field-leading product purity with more than 80% full capsids.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

We are very pleased with how these differentiated elements enable us to deliver 202 at the 2E14 vector genome per kilogram dose. We believe this dose gives patients and families the best shot at efficacy without compromising safety. In the phase I/II study, this approach has translated into a favorable safety and efficacy profile for patients. With this momentum in our pivotal study and results to date in phase I/II, we intend to expand the RGX-202 program outside of the U.S. and are actively exploring opportunities to do so, starting in Europe. Shifting focus to RGX-121, the positive 12-month pivotal data delivered to the FDA and presented at ICIM in September were consistent with the previous findings and demonstrated the long-term potential of RGX-121 to change the course of MPS II. Further, we saw a continued favorable safety profile and a strong correlation between biomarker level and neurodevelopmental improvement.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

If approved, RGX-121 would become the first and only gene therapy for MPS II and potentially the only one-time treatment option to address the neurodevelopmental decline for this devastating disease. The Hunter and MPS communities have been fierce advocates for the need to deliver new treatment options to their children as quickly as possible. We look forward to continuing to advance our BLA and potentially bringing this much-needed therapy to boys living with Hunter syndrome in the coming months. Turning to our retina sura-vec franchise for wet AMD and diabetic retinopathy, or DR. Completing enrollment in our pivotal wet AMD studies is a major milestone in our and AbbVie's continued effort to serve the millions of patients worldwide suffering retinal diseases. The data from our subretinal wet AMD program have been excellent, with durable outcomes reported through four years.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Additionally, in the fellow eye study, sura-vec has demonstrated comparable safety and efficacy when dosed in the second eye. These results, along with patient enthusiasm to return and receive treatment in their second eye, underscore the robust interest we're seeing among patients and physicians. Finally, I'll speak to sura-vec for DR using in-office suprachoroidal delivery. We continue to make progress towards initiating a global pivotal program. Site selection is in progress for the phase II-B/III double-masked sham injection control trial for patients with non-proliferative DR, or NPDR. In the phase II Altitude trial, a single in-office injection of sura-vec was well tolerated. In patients with NPDR, sura-vec demonstrated durable long-term efficacy, including meaningful DRSS improvement and an over 70% reduction in the risk of vision-threatening events.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Finally, I'd like to express my sincere gratitude to all the patients, families, clinicians, site staff, and patient advocacy representatives who have supported all these trials. With that, I'll turn the call over to Mitch to review our financial guidance. Mitch?

Mitch Chan
Mitch Chan
CFO at REGENXBIO

Thank you, Steve, and good morning, everyone. REGENXBIO ended the quarter on September 30th, 2025, with cash, cash equivalents, and marketable securities of $302 million, compared to $245 million as of December 31st, 2024. The increase was primarily driven by the $110 million upfront payment from Nippon Shinyaku in the first quarter of 2025 and $145 million in net proceeds received from the royalty monetization with Healthcare Royalty Partners in the second quarter of 2025, and was partially offset by the cash used to fund operating activities in the first three quarters of 2025. Revenues were $30 million for the quarter ended September 30th, 2025, compared to $24 million for the quarter ended September 30th, 2024. The increase was primarily due to the development service revenue under the Nippon Shinyaku partnership in the third quarter of 2025.

Mitch Chan
Mitch Chan
CFO at REGENXBIO

We expect the September 30th cash balance reported today to fund our operations into early 2027. Note, this cash runway guidance does not include multiple non-dilutive financing opportunities that could further extend our cash runway. These include the sale of our anticipated priority review voucher for RGX-121, development or sales milestone for our MPS programs, development milestone associated with our diabetic retinopathy program per the AbbVie collaboration, and potential additional funds from the May 2025 Healthcare Royalty Agreement. Together, these non-dilutive opportunities could further extend our cash runway well beyond 2027. In all, we find ourselves in a strong financial position as we advance towards multiple product launches. With that, I turn the call back to Curran to provide final thoughts.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Thank you, Mitch. As you've heard today, our strong execution throughout 2025 has positioned us for an exciting and transformational year ahead, including the anticipated approval of RGX-121 by February and top-line readouts in Duchenne and wet AMD, both large indications and large commercial opportunities. Each of these potential best-in-class gene therapies address a significant unmet need for patients and are built upon our 15-year history of pioneering AAV gene therapy with a commitment to scientific excellence and disciplined execution. I want to thank our REGENXBIO team, partners, and the patients and families who participate in our trials. Your partnership is key to advancing our mission to improve lives through gene therapy. With that, I'll turn the call over for questions. Operator?

Operator

Thank you. As a reminder, to ask a question, please press star one one on your telephone and wait for your name to be announced. To withdraw your question, please press star one one again. As a reminder, please limit yourself to one question and one follow-up. If you have additional questions, please return to the queue. Our first question is going to come from Judah Frommer with Morgan Stanley. Your line is open.

Judah Frommer
Judah Frommer
Executive Director and Senior Equity Research Analyst at Morgan Stanley

Hi, good morning, guys. Thanks for taking the questions and congrats on the progress here. Maybe first just on 202. Can you help us with when next interactions with FDA will be? Can we get your thoughts on, clearly, news coming out of FDA that impacts the DMD community with arguably higher unmet need in DMD, but also just on external controls and other gene therapy programs? How are you thinking about the potential for the accelerated pathway for 202 at this point? Thanks.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Thanks, Judah. Yeah, I mean, if you think about the progress next year, we'll have top-line data that we pointed to in early Q2, and then we've also guided to a mid-2026 BLA submission. Absolutely, we'll have potentially several FDA interactions. Of course, the key one is the pre-BLA meeting. Based on that timing, we haven't got a specific date set for it, but it would happen somewhere around the vicinity of top-line data and preparing to file the BLA. I think in general we are watching carefully other programs going through FDA and the debate around external controls. We've continued to update our access to some of the same databases, for example, that were used in the Elevate as approval. I think that when we look at it, one of the things that's striking to me.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Is, especially in our older patients, we're not looking at a marginal stabilization of patient functional outcomes. We're looking at a really significant difference from natural history in which we're matching that patient to, say, 12 up to 20 patients. I don't think we're going to be going into FDA with sort of a modest benefit approach. I think what we're seeing in our data is a significant benefit and even improvement in patients that you would expect maybe the best case at their age would be stabilization. I think that's the strength at which we think we're eligible for accelerated approval. I won't leave out, to date, the safety profile of the program. You think about benefit to risk ratio, and I think we can provide a significant advantage over the product on the market today.

Judah Frommer
Judah Frommer
Executive Director and Senior Equity Research Analyst at Morgan Stanley

Great. Thanks. Maybe just a quick one for Mitch on cash runway. I guess, kind of when you internally probability weight potential for these non-dilutive financings that you listed, can you give us a sense of where those could potentially get you through without kind of bringing in external capital or how you're thinking about the potential for a cash runway if we do layer in some of those non-dilutive options? Thank you.

Mitch Chan
Mitch Chan
CFO at REGENXBIO

Yeah, absolutely. If you factor in the non-dilutive financing options, inclusive of the PRV as an example, and for modeling purposes, if you use the market price, as you're probably well aware of what the prices are for these PRVs, it could significantly get us well into 2027, if not even early parts of 2028. Again, this is all contingent on what the market price of some of these items can go for. Other non-dilutive financings, for example, the milestone associated with the DR, first patient dose, that we already disclosed is $100 million. That in itself, you can factor into the cash runway.

Judah Frommer
Judah Frommer
Executive Director and Senior Equity Research Analyst at Morgan Stanley

Great. Thanks.

Operator

The next question will come from Mani Foroohar with Leerink. Your line is open.

Lili Nsongo
Lili Nsongo
VP of Equity Research and Biotechnology at Leerink

Hi, good morning. This is Lily Nsongo on for Mani. Thanks for taking our question. Just a quick question regarding the confirmatory trial for DMD. Maybe could you provide an update in terms of the tempo of enrollment and where do you see enrollment be at the time of filing?

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Good question. We've begun enrolling the confirmatory study, actually right at the end of October when we completed the pivotal enrollment. It's hard to predict at this point where we'll be, let's say, mid-2026 at the point of filing, but I would expect us to be substantially through enrollment of the confirmatory study, given that that's pre-specified in our protocol as an additional 30 patients. We'll have to monitor that as we go, and we'll definitely give updates throughout the first half of next year regarding progress. Steve, maybe you just want to comment quickly on high-level design for confirmatory?

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure. The basic concept is very similar to what we've already done. We're looking for a broad patient population of ambulatory patients, one-year-old and above. Very broad. This is really based on the data that we've seen to date, where, as Curran mentioned, we're seeing very good differentiation, not just on safety, but also in terms of functional benefit for these boys. That's particularly striking in the older boys. We're seeing results that have never been seen by any program when you look at function in those eight and older boys. No placebo control, so that's stayed consistent. We're looking to enroll an additional 30 patients. It's key what Curran mentioned. We're just continuing to roll along with all the sites that we've got up and going that we're enrolling in the pivotal. They're energized.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

One of the things we're seeing is with each new update that we give that's showing more and more differentiation, we're getting more and more enthusiasm from the investigators and the patient families that they see. We are very excited about the pace of enrollment.

Lili Nsongo
Lili Nsongo
VP of Equity Research and Biotechnology at Leerink

Thank you.

Operator

The next question will come from Gena Wang with Barclays. Your line is open.

Gena Wang
Gena Wang
Managing Director and Biotech Equity Research Analyst at Barclays

Thank you for taking my questions. I would just ask one regarding the regulatory part. I know you have two programs like DMD and MPS II. You need to talk to the FDA. Just wondering, for the DMD, when will you have a pre-BLA meeting with FDA? Also regarding the MPS II, I know PDUFA is February 8. Any additional meetings set up before approval? Maybe any update color you can have. In terms of interacting with the FDA, especially after Nicole Vedant's departure, any concern regarding PRI agreed upon the accelerated approval path?

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Sure. Hi, Gena. I'll take the second question first, then circle back to Duchenne. For the Hunter program, we're far along with the BLA review. As you know, the PDUFA date was moved to February 8 from the initial date. We expect between now and that decision point to have a late cycle meeting with FDA, and we're getting messages from the project leader to schedule that. That's moving along as what I would call business as usual. I think it's important to point out on the Hunter program we're already past our facility inspection. No observations. Our clinical sites have been inspected as well with no observations as well. Some of the things that have held up other programs were de-risked, if you will, in that process.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

The other, I think, area that we interact as we undergo this review is in the information requests that we get from FDA. I think I would characterize those as well as typical requests, some of which in an encouraging way are questions related to commercial aspects of a program, specifications, pharmacovigilance. The color of the interactions we're having is positive, and we feel positive about the data we provided for that program. Therefore, our confidence is high that this program, and we think patients support that, has a really, really important place in therapies for Duchenne. The pre-BLA meeting will happen between our top-line data, which we pointed to early Q2 of 2026, and the filing of the BLA. We haven't got a specific date for it yet, but somewhere in that timeframe is when we would expect to have it.

Operator

Our next question will come from Luca Issi with RBC Capital Markets. Your line is open.

Operator

Good morning, team. Thanks for taking our question. This is Cassio for Luca. Congrats on the progress this quarter. Maybe a question on your manufacturing capacity, if you can talk about your in-house capacity. The Rockville site is obviously up and running and already produced first commercial batch. I'm wondering, what is the scale of production volume that the site is designed to deliver today? What percentage of domestic patients are you planning on capturing with that site? Maybe for both the MPS II and DMD, if you could speak to that. Thank you so much.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Sure. Yeah, the manufacturing facility contains a 2,000-liter bioreactor. It's the largest bioreactor that we know of utilized in gene therapy, and we've already scaled the program up to that level. We've been public about being able to produce up to 2,500 doses of our RGX-202 per year. I think keeping in mind that just is what we can do on an ongoing basis. We can certainly inventory, which we're doing presently, more quantities than that to have a significant number of doses available at launch. The Hunter program, being an ultra-rare program, I would characterize it uses less than 5% of our overall capacity. It's not. We have good yields, good expression, and we have our own internal fill-finish capability. We can run very efficiently, but it doesn't take up a significant amount of our overall capacity.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Thanks so much.

Operator

Our next question will come from Annabel Samimy with Stifel. Your line is open.

Annabel Samimy
Annabel Samimy
Managing Director at Stifel

Hi. Thanks for taking my question and great progress on all the programs. Just on the issue of using FDA natural history as a control in DMD. I guess it's clear that you have both propensity match comparisons and now the CTAP disease progression model. Is that something designed into Affinity prospectively, and could that lend further support? Is it a measurement that's accepted not only in the DMD community but also considered as a valid metric from FDA? That's the first question.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Okay. Great. Hi, Annabel. I think that's a great one for Steve to address.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure. Hi, Annabel. Great question. I think right off the bat, it's important to recognize each program and indication is different. Starting with DMD. External natural history matching can be done in different ways, and you mentioned a couple of them. That's why we're excited about our data. We're seeing clear response across the dose range that's very consistent when we look at a traditional external natural history matching by baseline characteristics and also the CTAP model. There is also the propensity score matching approach that's been accepted by the FDA, and that's actually prospectively specified in our protocol as an accepted approach for 202. On the 121 program. Importantly, we've had numerous interactions with the FDA, of course, and there hasn't been an issue of how we've prospectively looked at our individual data.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

The only thing that's been asked for from an efficacy standpoint was what we provided in terms of the encouraging one-year data. Since then, we discussed with the FDA, was there any other data that would be needed? The answer was no, that this would allow them to complete their review. I think this reality of what type of feedback we've gotten from the current regime puts us in a good place for both these indications.

Annabel Samimy
Annabel Samimy
Managing Director at Stifel

Okay. Great. I'm going to be different and ask a wet AMD question. I guess, can you opine on the recent M&A and licensing activity for gene therapy and wet AMD? Obviously, we had Lilly, Fredberm, and for DMT licensing and, of course, your licensing. Does the street have it wrong on the level of interest from retina specialists? Where does your program rank as far as level of engagement with these retinal specialists?

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

It might be good, Steve, to reflect on what we just heard out of AAO regarding gene therapy in general and specifically our program.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure. I think there's tremendous excitement about one-time treatment, and the way to do that is gene therapy. One strong piece of evidence is what we're hearing actually from retina specialists. This is coming from the Pat ASRS survey of close to 1,000 retina specialists, where every year a number of questions are asked of these experts in the field, the actual treating clinicians. An important one is exactly what you're asking. What pipeline approaches are you most interested in as retina specialists across all the different approaches? Half the respondents say gene therapy because of the one-time potential here that isn't the case for any of the other treatment approaches like TKIs or any other durability approaches, because no matter how long you extend that durability, you're still going to need injections indefinitely for any of the indications that we're looking at.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

I think we're seeing that in terms of interest in the field and some of the licensing deals. M&A deals that you mentioned, Annabel. Of course, we have a major global partnership with AbbVie, who has quite a lot of experience globally, including quite a reputation when it comes to ophthalmology and experience there. Their continued advancement with our program, including paying two of every $3 for the advancements, both in diabetic retinopathy and other areas, I think really speaks to the validation of not just gene therapy, but specifically our program with the muscle of AbbVie behind it globally.

Annabel Samimy
Annabel Samimy
Managing Director at Stifel

Okay. That's great color. Thank you.

Operator

The next question will come from Alec Stranahan with Bank of America. Your line is open.

Operator

Hey, guys. This is Matthew on for Alec. Appreciate you taking our questions. Given a recent expectation for a box warning for a competitor product and the removal of the non-ambulatory indication, just curious whether your expectation is for something similar on your label or other AAV gene therapy labels and how you're thinking about long-term development in the non-ambulatory population in general. Thanks.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Thanks for the question. I think in terms of expectations, I mean, I think our track record in phase one two on safety has been exemplary. I wouldn't expect black box warning of that nature, given that we've shown clearly the incidence rate of any sort of liver injury for us is virtually nonexistent in terms of the data, whereas we're seeing on the label for Levadis or in their filing that they have upwards of 40% liver injury in the approved product. I think we don't expect that. In fact, we expect to leverage safety as part of the accelerated approval pathway that we're pursuing. I think accompanying that is strong conviction that the differentiation of the product with the immune suppression regimen we utilize and the construct and manufacturing purity, put those all together, I think we have something very different.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Very exciting to the patient community today.

Operator

Our next question will come from Brian Skorney with Baird. Your line is open.

Brian Skorney
Brian Skorney
Senior Research Analyst of Biotechnology at Baird

Hey, good morning, everyone. I'd actually like to tack two regulatory questions on the EMA side of things. I know EMA's granted one, two on the ATMP designation, but I was just wondering the status of any plans for it at the EMA. Also, any thoughts on the potential sufficiency of the AFFINITY DUCHENNE study for EMA review, or what else you think you need to do there? Is your FDA confirmatory sufficient for EMA filing? Thanks.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Yeah. We've had some interactions with EMA regarding RGX-121, and I think that what we're typically seeing there is the requirement for a control arm, placebo control arm, still seems to be the predominant feedback that we get. Having said that, there's a significant opportunity in name patient sales that would potentially exist off an accelerated approval. I think that's something that we are carefully evaluating and could be part of our commercialization approach. Steve, do you want to address the Duchenne question?

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure. Hi, Brian. Based on all the discussions we've had stateside, so to speak, we're very confident on our approach here. Not having had the discussions outside the U.S., we don't want to project what will be the case. Certainly, historically, there has been a need for a placebo control there. We recognize the problems of that as well as with the patient advocacy community. There is increased data that keeps growing with different ways of assessing function that are becoming accepted in different regulatory regions like the SV95C, just to give one example. It's something that's an evolving field, but it's really going to take actual discussion. We keep coming back to differentiation because it matters. The functional data, if we continue to see what we're seeing, including in the older boys, not just stabilization but improvement, we think we're in a stronger position.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

For being able to come up with an approach that would be acceptable ex-U.S.

Brian Skorney
Brian Skorney
Senior Research Analyst of Biotechnology at Baird

Great. Thank you.

Operator

The next question will come from Sean McCutcheon with Raymond James. Your line is open.

Sean McCutcheon
Sean McCutcheon
VP of Biotechnology Equity Research at Raymond James

Hi, guys. Thanks for the question. Just one for me on diabetic retinopathy. A competitor was able to get an agreement with FDA for an ordinal two-step DRSS change, primary endpoint for their pivotal studies in NPDR. Can you speak to how that's informing your discussions with AbbVie on your pivotal NPDR program and ability to adjust that study plan to potentially improve probability of success relative to a two-step DRSS improvement at one year? Thanks.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure. I can take that.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

That's a great question. Steve, go ahead.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure. Yeah. Thanks for the question. Yeah. I think one item off the bat is that the ordinal approach is something people have thought about over time. The prior lead or director of the ophthalmic division had always required a meaningful change in DRSS with meaningful defined as either two-step improvement change or in either direction of worse or improvement. The advance of being able to get more information out of each patient's DRSS change by looking in an ordinal way now with the new regime has come on the table. It is definitely something we've been looking at, both us and AbbVie in close collaboration. I think it gives us more options. Fortunately, what we've seen is we look good in both respects. Two-step improvement, a greater proportion of patients achieving that, and also two-step worsening, a greater proportion of patients.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Showing that in a placebo control setting, as well as what you expect without treatment in the real world and other controlled negative control arms. We think we're really well positioned to really take advantage of what looks best in our discussions with the FDA.

Sean McCutcheon
Sean McCutcheon
VP of Biotechnology Equity Research at Raymond James

Thank you.

Operator

The next question will come from Daniil Gataulin with Chardan. Your line is open.

Daniil Gataulin
Daniil Gataulin
Senior Equity Research Analyst of Biotechnology at Chardan

Yes. Hi. Good morning, guys. Thank you for taking my question. Quick one on super choroidal with AMD. First, can you remind us how many patients you're looking to enroll at dose level four for that study? Now with the subretinal program fully enrolled, do you expect the speed of enrollment for super choroidal program to pick up a little bit now with the shifted focus to that program? Thank you.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Steve, go ahead.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Thanks for the question, Daniil. SCS with AMD is advancing. We're looking to enroll 20 patients in that arm. As you mentioned, we reached the quite significant milestone of completing enrollment in the SR with AMD pivotal studies, the largest gene therapy program ever conducted across any indication. It's a great point that now with that trial completed enrollment, a lot of the overlapping sites can now focus on the suprachoroidal delivery route. Yes, indeed, we're seeing a pickup in enrollment in that study.

Daniil Gataulin
Daniil Gataulin
Senior Equity Research Analyst of Biotechnology at Chardan

Got it. A quick follow-up for subretinal program. Do you expect AbbVie to file in the U.S. and EU roughly at about the same time?

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Yeah. I think that's a safe assumption. We haven't got a specific timeline for the filing mapped out. We're primarily focused on top-line data next year, but we would expect a global filing. We'll be more specific as we get closer about the interval between U.S. and ex-U.S. filings. They're recruiting. Not all the basis of the ASCENT study being recruited in Europe was to enable that to be a smooth process.

Daniil Gataulin
Daniil Gataulin
Senior Equity Research Analyst of Biotechnology at Chardan

Okay. Got it. Thank you very much, and congrats on all the progress.

Operator

The next question will come from Bill Maughan with Clear Street. Your line's open.

Bill Maughan
Managing Director and Equity Research of Biotechnology at Clear Street

Good morning and thanks. I just wanted to look again at the functional endpoints on 202 and just ask about the powering around those and whether or not you believe there's a certain threshold or, I guess, outcome that you need to see as a bar for success to really kind of put together a definitive answer in front of the FDA that kind of can't be denied.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Steve, do you want to cover that?

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure. Thanks for the question. We are seeing a nice response, both in terms of change from baseline with improvement, even in the older boys where you might expect just stabilization or worsening, and with improvement, just stabilization. I think we're in a good position where if we continue to see what we're seeing and what we report on in early Q2 next year, with not only the primary endpoint for accelerated approval but also the microdystrophin, I think we're going to be very well set up. That's based on, to your question, looking at the traditional endpoints. Certainly NSAA, where marginal effect, if any, depending on the age of the patients, has been seen previously, and also the time function tests. The traditional time to stand, 10 m walk run, time to climb.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

As the traditional ones where there's a lot of data out there and we have the opportunity to look with propensity matching. How good of a data do we have to see? Again, if we continue to see what we're seeing, we're going to pass that bar because of the differentiation that we're seeing. Some bars you can look at are not only against natural history, but also there's data and published interpretations of the data on minimally important clinical difference that we're surpassing. We can also look by age and by baseline status what's been seen with approved therapy. That allows us to feel that we're in a good position to have power based on the data that we're seeing. Again, if we continue to see what we're seeing, the overall package is important as well.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

The benefit-risk relative to that functional improvement, then we go to the safety where, again, we're seeing very good differentiation.

Bill Maughan
Managing Director and Equity Research of Biotechnology at Clear Street

On your suprachoroidal program, where are you on your understanding of how closely that can recapitulate the amount of vector delivery of your subretinal delivery?

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

So on.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Are you asking if the protein levels match in terms of transgene expression between the two?

Bill Maughan
Managing Director and Equity Research of Biotechnology at Clear Street

Yeah.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Yeah. I think because Steve can comment further, but because they're delivered to different compartments of the eye, it's very difficult to do direct comparison. Ultimately, what we look at is sustained vision and safety. Steve, maybe you want to comment further on the differences we see.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Sure. Geographically, they're different spaces. They're both close to the target tissue, and they're both in compartmentalized spaces. That's why we selected them, so that they greatly limit off-target tissue potential side effects. In terms of immunogenicity, that also changes, as Curran mentioned, the reality that you really can't compare the apples and oranges. Fortunately, what really matters is what do you see clinically, which shows that you have the transgene product at the target tissue where you need it. That's where the diabetic retinopathy data is so compelling for us in AbbVie, that you're seeing what you want to see. That's why we're advancing with suprachoroidal already and actually accelerated development so that we and AbbVie can actually start phase 2B3 next year.

Bill Maughan
Managing Director and Equity Research of Biotechnology at Clear Street

Got it. Thank you.

Operator

Our next question will come from Yi Chen with H.C. Wainwright. Your line is open.

Yi Chen
Yi Chen
Managing Director and Equity Research Analyst at H.C. Wainwright

Good morning. Thank you for taking my questions. Could you comment on your current thinking regarding the pricing strategy for RGX-121 versus RGX-202? Also, how large a sales team do you think you need for RGX-121 versus RGX-202? Thank you.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Yeah. If you recall, earlier in the year, we signed an agreement with NS Pharma for commercialization in the U.S. The pricing decisions will be made by NS Pharma as we move through the approval process. Mitch can comment. We certainly will enjoy a nice royalty from sales, but the actual price determination will be made by them. We would eventually make the price decisions since we wholly own the Duchenne asset. I'd say at this point, it's very preliminary for us to state any sort of thinking on pricing until we get closer to commercialization.

Steve Pakola
Steve Pakola
Chief Medical Officer at REGENXBIO

Yeah. As you recall from the NS Pharma partnership, we are able to receive meaningful double digits in sales royalty. As Curran kind of mentioned, pricing decisions will be made by NS Pharma at a later time.

Yi Chen
Yi Chen
Managing Director and Equity Research Analyst at H.C. Wainwright

Thank you.

Operator

The next question will come from Paul Choi with Goldman Sachs. Your line is open.

Paul Choi
Paul Choi
Biotechnology Analyst at Goldman Sachs

Hi. Thank you. Good morning, everyone. Thanks for taking the questions. With regard to 202, could you please comment on where you think you might see the potentially greatest demand initially as you start to commercialize in 2027? Do you think about it in particular exon skipper experience patients or just thinking primarily about newly diagnosed patients? Any color there would be helpful. Then second, with regard to your collaboration with AbbVie and 314, how are you thinking about sort of rough timelines for potential Medicare coverage of 314 post-approval? Just sort of what are the block steps there involved for gene therapy coverage in this particular Medicare population? Thank you very much.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Okay. Yeah. I'll take the first one. I think, obviously, if you look at the inclusion criteria for the study, we're dosing patients one and older. So we'll have data at the time of review for potentially a broad label. One thing that we see based upon the public announcements on sales is that the prevalent market really isn't changing in terms of Duchenne. By 2027, the prevalent market will be something like 14,000 patients. If you think about eligible for gene therapy, it's probably closer to 3,000 patients when you subtract out non-ambulatory. At the time of launch, our aim is to have a broad label and really be able to address patients of all ages. To your point, we do expect that over time, as the prevalent market diminishes, which could be past 2030.

Curran Simpson
Curran Simpson
President and CEO at REGENXBIO

Is that at that point, we would be in a great position to address the incident market. Most KOLs think early treatment is ultimately the answer for gene therapy. And we'll have the data in hand to support. That age group. Based on the fact that we're already dosing kids. Right after diagnosis in some cases. I think on the Medicare coverage for 314, that's a question that I'll defer out a bit. We haven't had those discussions with AbbVie. AbbVie will obviously be leading the significant element of the commercialization. But we do believe broad access for gene therapy is fairly easily achieved. It's just too early in terms of our conversations with AbbVie to be specific there.

Paul Choi
Paul Choi
Biotechnology Analyst at Goldman Sachs

Okay. Thank you.

Operator

This does conclude today's question and answer session and conference call. Thank you for participating, and you may now disconnect. Have a good day.

Executives
Analysts
    • Daniil Gataulin
      Senior Equity Research Analyst of Biotechnology at Chardan
    • Lili Nsongo
      VP of Equity Research and Biotechnology at Leerink
    • Annabel Samimy
      Managing Director at Stifel
    • Sean McCutcheon
      VP of Biotechnology Equity Research at Raymond James
    • Yi Chen
      Managing Director and Equity Research Analyst at H.C. Wainwright
    • Paul Choi
      Biotechnology Analyst at Goldman Sachs
    • Bill Maughan
      Managing Director and Equity Research of Biotechnology at Clear Street
    • Analyst at Bank of America
    • Gena Wang
      Managing Director and Biotech Equity Research Analyst at Barclays
    • Judah Frommer
      Executive Director and Senior Equity Research Analyst at Morgan Stanley
    • Analyst at RBC Capital Markets
    • Brian Skorney
      Senior Research Analyst of Biotechnology at Baird
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