Regenxbio at Guggenheim Conference: Strategic Milestones Ahead

On Monday, 10 November 2025, Regenxbio Inc (NASDAQ:RGNX) outlined its strategic roadmap at the Guggenheim Securities 2nd Annual Healthcare Innovation Conference. The company highlighted its progress towards significant milestones, including a potential FDA approval and pivotal data releases. While the outlook is promising, challenges remain in achieving regulatory success and market penetration.

Key Takeaways

• Regenxbio is anticipating a PDUFA date in February for its Hunter syndrome program, which currently lacks effective therapies.
• The Duchenne program has completed pivotal cohort enrollment, with top-line data expected in Q2 2026.
• For the wet AMD program, partnered with AbbVie, top-line results are anticipated by the end of 2026.
• The company is focused on maintaining a strong relationship with the FDA and is seeking a commissioner’s voucher for its DMD program.
• Investment in manufacturing aims to align costs with biologics, enhancing the benefit-to-risk ratio.

Operational Updates

Regenxbio is advancing several late-stage assets, each poised for critical developments:

• The Hunter syndrome program is awaiting a PDUFA date in February. This could mark a breakthrough for a disease with limited treatment options.
• The Duchenne muscular dystrophy (DMD) program has completed pivotal cohort enrollment. Key differentiators include a construct mimicking natural dystrophin and an immunosuppression regimen that has shown no liver injury in patients.
• For the wet AMD program, a collaboration with AbbVie, the company expects to report top-line results by the end of 2026. This program’s subretinal administration could address compliance issues seen with current treatments.

Future Outlook

Regenxbio’s strategic focus is on securing regulatory approvals and optimizing its manufacturing capabilities:

• The company is in ongoing discussions with the FDA regarding functional endpoints and external controls for its Duchenne program.
• Regenxbio aims to secure a commissioner’s voucher for the DMD program, which could expedite the approval process.
• Investment in manufacturing is expected to keep the cost of goods in line with biologics, potentially improving the benefit-to-risk ratio.

Partnership and Market Potential

The collaboration with AbbVie on the wet AMD program features a 50/50 profit share, highlighting the commercial potential:

• The one-time administration of RGX-314 could capture significant market share by addressing compliance issues associated with frequent injections.
• AbbVie is leading commercial efforts, leveraging its market presence to maximize the program’s reach.

Conclusion

For more detailed insights, readers are encouraged to refer to the full transcript of the conference call.

Full transcript - Guggenheim Securities 2nd Annual Healthcare Innovation Conference:

Devjit, Therapeutic Analyst, Guggenheim: Good morning, and thank you for joining Guggenheim’s second annual Healthcare Innovations Conference. I am Devjit, one of the therapeutic analysts here, and our next presenting company is REGENXBIO. Joining us from REGENXBIO is CEO Curran Simpson and CMO Steve Pakola. Thank you for your time.

Curran Simpson, CEO, REGENXBIO: Thank you.

Devjit, Therapeutic Analyst, Guggenheim: Given that this is your first exposure to Guggenheim’s conference, a very quick overview to start off with before we get into the Q&A?

Curran Simpson, CEO, REGENXBIO: Sure. Thanks for having us. It’s a really, really great city to do these types of meetings. Yeah, it’s a very pivotal year for us. We’ve been working on AAV delivery for over 15 years. We were one of the original NAV technology developers, which ultimately resulted in products like Zolgensma. Next year, we’re pointing towards a really significant year for our late-stage assets. Sort of in chronological order, that would be the ongoing BLA review for our Hunter program, which is for MPS II disease. There’s really no therapy beyond steroids available to patients, and we think this has an important place in their treatment paradigm. That PDUFA date is in February.

For our Duchenne program, just a week or two ago, we announced that we fully enrolled our pivotal cohort and now can point to top-line data in Q2 next year, which is really exciting for us, huge opportunity. We have also off-enrollment of two very large non-rare disease indications for wet AMD in a program that’s partnered with AbbVie. We expect to read out top-line data on a program called RGX-314, which is also exciting towards the end of 2026.

Devjit, Therapeutic Analyst, Guggenheim: Thank you for that. Given that 314 does not, for the lack of a better phrase, see much light of the day because everybody is more focused on the DMD side, let’s start on that. As you mentioned, data in the fourth quarter, what do you expect to cover when you read out that data? Steve, you want to take it?

Steve Pakola, CMO, REGENXBIO: Sure, I can take that. Thanks again for having us. It is interesting, all the attention on some of the other programs that have certain milestones coming up. I think we in AbbVie continue to be super excited about the 314 program. You mentioned the subretinal program, which is one of the two routes of delivery that we’re looking at, and it’s going to be a big milestone for us. Q4, we, as Curran mentioned, completed enrollment. The only added thing I’ll say is these are the two largest gene therapy clinical trials ever conducted. I think with our 15 years’ experience, we’ve really come a long way. Top-line results in wet AMD, which is this indication, fortunately is greatly de-risked. It’s basically BCVA, or what’s called best-corrected visual acuity at one year. That’s in a non-inferiority design against on-label anti-VEGF treatments.

It is really great because we’re going to learn a lot about what’s done in clinical practice or even really something more standard in a controlled clinical trial. These patients are getting very frequent injections on label with Lucentis in one trial and Eylea in the other trial. This is sort of a unique situation where if you can show non-inferiority in that type of setting, that translates into superiority in the real world because we know patients in the real world do not get anything close to the frequency of injection because it is just not sustainable to keep getting these injections because of the tremendous burden for the patient and the caregivers.

So it really sets us and AbbVie up globally for a really great value proposition of decreasing treatment burden, but also leading to better vision outcomes and less blindness in the long run in the real world.

Devjit, Therapeutic Analyst, Guggenheim: Got it. Maybe a couple of, if you could lay out how this program is different from the other approaches that have been tried with gene therapy.

Steve Pakola, CMO, REGENXBIO: Sure. I think one of the aspects is right from the beginning for our first route of administration, we went with subretinal. The key there is we know that’s an immune privilege space. It’s really the most clinically validated way to deliver gene therapy for retinal indications, basically all the anti-VEGF indications. Other routes that people have tried include intravitreal injection. The issue there is that’s not a compartmentalized space. You wind up getting transduction at other tissues that you don’t want transduction, including anterior eye structures. You get a lot more inflammation and you have to treat patients with steroids. There have been even worse outcomes that have occurred. There have been other subretinal programs that include the first-ever approved gene therapy, Luxturna, for a certain blinding monogenic disorder.

The advance here is, and this is another case where we’ve been leaders, is in this case, we’re using gene therapy to treat a common non-monogenic disorder by delivering a therapeutic protein. So a lot of groundbreaking aspects that come with this program.

Devjit, Therapeutic Analyst, Guggenheim: Got it. Given that AbbVie is your partner here, what are your commercial roles when you’re assuming you hit non-inferiority and it’s approved and launched? Where do you fit in?

Curran Simpson, CEO, REGENXBIO: Yeah, certainly AbbVie will lead the majority of the commercial aspects. They have 7,000 MSLs, et cetera, very large commercial engine. That was actually the original strategy behind the partnership in general was there would be no way for us to want to build a large sales force for retina. Partnering with AbbVie made a ton of sense there. We will certainly have input. We already have had input to the target product profile. Certainly Steve’s team has led the development. In terms of commercialization, AbbVie will be the lead for it. The economics of that are it’s a 50/50 profit share.

Devjit, Therapeutic Analyst, Guggenheim: Got it. And Hamid, REGENXBIO does not intend to have own sales force?

Steve Pakola, CMO, REGENXBIO: No.

Devjit, Therapeutic Analyst, Guggenheim: Got it. What is the problem that this product actually will address solving? And depending upon the outcome, what do you and AbbVie think could be the real market share here?

Curran Simpson, CEO, REGENXBIO: I think it’s a variety of things. Steve can elaborate, I think, on the disease conditions we see potential prescribers talking about being prime candidates for our therapy. But I think one of the main problems we’re trying to solve is compliance. So a lot of patients, even those that go back and forth from Boston to Florida every year, you lose them to treatment six months out of every year. So one of the benefits of gene therapy is simply the one-and-done nature of the administration. We’ve got patients now out four to five years post-treatment where they’re not getting supplemental injections. And so it’s really just solving for one of the main drivers of loss of vision is lack of compliance. And I don’t know if you want to talk specifically about patients.

Steve Pakola, CMO, REGENXBIO: Yeah, sure. We’re seeing this translate in the real world. So it’s not just a theoretical concept because we know for years that the treatment burden is the main unmet need, and this is translated into dollar impact that you can actually see that these patients want less frequent injections. And we know this, for example, given the evolution of different anti-VEGFs that have tried to solve this in incremental steps. So for example, when we went from Lucentis to Eylea, we went from needing more frequent injections to allowing patients to go every eight weeks or every 12 weeks in clinical practice, and that had a blockbuster impact. Then we have gone very recently from every other month dosing to now with Fovista in high-dose Eylea to every three months and potentially in some patients every four months. Again, blockbuster indications that have played out in reality.

So this shows you even with incremental decrease in injection burden, there is a major unmet need. And we have TKIs advancing. So these are all great advances. But at the end of the day, patients still need ongoing injections at a certain frequency basically for the rest of their lives. So this is more of a transformational change to actually go with one-time injection, whereas Curran mentioned some of these patients are not needing any injections. And we’re seeing that with our clinical trial sites, the enthusiasm that they’re getting from their patients.

Devjit, Therapeutic Analyst, Guggenheim: Got it. And as a company, you guys have sort of focused on manufacturing a lot. Given the relatively small volumes of AAV vector required here, where do you think the COGS will line up? Because in the end, it’s a 50/50 profit share.

Curran Simpson, CEO, REGENXBIO: Yeah, we’ve invested heavily in manufacturing. It particularly applies to the Duchenne program where cost of goods is definitely a question in the high vector loading. But even in retina, there’s 100,000 patients out there. So significant numbers of batches are still required. But we have very, very good yields in terms of our suspension bioreactor process. And I think our cost of goods will be, if you think about it, more in line with what you see in biologics rather than what you’ve seen historically in gene therapy.

Devjit, Therapeutic Analyst, Guggenheim: Got it. So let’s transition over to the DMD program because this will have data as well next year. How is your construct different than what’s been or what’s approved or what’s going to be in the clinic as well?

Curran Simpson, CEO, REGENXBIO: Cover the C terminus?

Steve Pakola, CMO, REGENXBIO: Oh, sure. So again, 15 years of experience, we’ve gotten to learn a lot about this area. And our CSO, Olivier Danos, has worked for many decades on DMD and also gene therapy. So way back before starting clinical trials and even preclinical studies, our goal was to come up with a construct that was closest to naturally occurring dystrophin to really replicate what you see with natural dystrophin. And the most critical part is the C terminal domain that includes a lot of the functional elements that allows dystrophin to do what it does. And importantly, we’ve shown this preclinically where a construct that has the C terminal domain has better efficacy in recognized models for DMD than a construct that does not have the C terminal domain. And we also believe there’s, and we’ve seen a longer half-life in in vivo setting.

And when you think about a product, it’s not just the construct and some of the other optimization that we’ve been able to make that others who started with constructs a while ago before some of these understandings were available is also the purity of the product. So it’s not only the molecule, it’s also how pure the product is. So we can actually get more construct into a patient without the overall viral load because we have industry-leading vector full capsid over 80% compared to what’s on the market, which is more in the 50s-ish. And that’s allowed us to go to a higher dose also with some of the safety advantages that we have. So all of this is translated into what we’re now seeing in the clinic.

Devjit, Therapeutic Analyst, Guggenheim: Got it. The immunosuppression regimen, you guys took the lead in this a while back. How did you work this out? Because this has been another challenge, especially on the DMD side.

Steve Pakola, CMO, REGENXBIO: Yeah, here our view was we got to learn from other programs, both in DMD and non-DMD. So all the programs use a higher dose of steroid, which you can think of as a crude immune modulation. It’s important, but you still get with other programs different side effects, some quite serious, including fatal, unfortunately. But our view was let’s target those areas and really go after those. So how do we do that? So one area is complement activation that leads to thrombocytopenia, low platelets, and also more serious side effects like microangiopathy and actual thrombosis. So we use eculizumab. And it’s just a couple of weeks of treatment. And we’ve seen in the clinic zero thrombocytopenia, which is different than other gene therapies in Duchenne.

The other big issue, of course, that’s been in the news this year is fatal injury, which with existing therapy, you see 40% of patients that have liver injury. And again, unfortunately, sometimes fatal. And that’s really created a big unmet need for safer as well as more efficacious therapies. So we’ve added serolimus where, again, it’s for a confined period, two months with an additional month taper. And long story short, all of this has led to better safety so far from what we’ve seen. 13 patients treated, no liver injury already. That’s a clear differentiation than 40% with existing therapy.

Devjit, Therapeutic Analyst, Guggenheim: Obviously, lots of changes within the FDA. How close or how often or when was your last interaction with the FDA? And are you planning to meet with them before the data?

Curran Simpson, CEO, REGENXBIO: Yeah, we’ve had really consistent interaction with FDA. Now we have the ongoing Hunter program, VLA that’s under review, and all of the customary early, mid, and late cycle meetings that go along with that. And also we’ve had, as part of our end of phase two meeting on Duchenne, a protocol review of the protocol we just executed and completed enrollment in. And in that protocol, we spelled out prospectively the functional endpoints and the external controls that we would use to compare. And that’s similar to what we’ll do in the confirmatory study. So the next formal interaction with FDA will be sometime in the first half of next year as we do the pre-VLA meeting and as we bring out our top line data in Q2.

Devjit, Therapeutic Analyst, Guggenheim: Got it. And the NSA data to date has been pretty stellar. Is that purely a function of the differentiated construct or there is something else that goes into it?

Curran Simpson, CEO, REGENXBIO: It’s a multivariate. So if you think about it, the construct being different, which includes the C terminus, and that’s borne out through preclinical data that that leads to a differentiated result. What we see that definitely stands out is the higher number of vector copy number that we’re seeing, which is pointing towards really good biodistribution. So we’re getting good biodistribution, good transgene expression. The microdystrophin results that we’ve gotten in older patients in particular have been much higher than what was published previously. Add those all together. Yeah, I think going into the program, we would have been pleased to see the patients, let’s say, that are seven and older being stabilized in terms of their function. But as Steve could say, we’re seeing improvement in those patients. That is somewhat unexpected.

And improvement in NSAA, which was really people were stepping away from that functional assessment because of lack of statistical significance in some of the other studies.

Steve Pakola, CMO, REGENXBIO: One aspect here, we talked about the immune regimen and safety. And this is a case where that translates into an important aspect of efficacy. So we’re the only ones able to go to what we think is an optimal dose from what we’ve seen preclinically, which is 2E14 vector genome per kg. And that’s critical. So because we plan to have the most safe product given the purity and also our immune regimen, we’re able to get to that dose. And fortunately, we’re seeing that translate into something that’s differentiated based on at least one aspect, what Curran mentioned, these unprecedented results that we’re seeing in those older boys.

Devjit, Therapeutic Analyst, Guggenheim: There have been obviously major label changes to Elevidus. Clearly, one part of the market is not getting served. What would be your expectation or what should be the expectations for the label from you?

Curran Simpson, CEO, REGENXBIO: Yeah, right now we’re pursuing only the ambulatory population, so not the non-ambulatory. That’s something that we’ll consider as the program matures, as our safety data set increases. And we’re pursuing a wide label. So we’re treating patients one and older. So it could be that during discussions, there’s a desire to see some of the patients that were very young when they’re treated. You may have to wait longer to see them diverge from the control. And that’s expected. But our going-in assumption is with the safety profile that we’ve seen and with the functional profile we’ve seen, we feel like we have a distinct advantage in benefit to risk ratio that is one of the tenets of accelerated approval.

Devjit, Therapeutic Analyst, Guggenheim: Got it. So in the last few minutes, I want to touch on the Hunter Syndrome program. The FDA requested some additional information. Where are you in that process right now? And do you think that’s going to be a major amendment?

Curran Simpson, CEO, REGENXBIO: Already has been. So we were asked in our mid-cycle meeting sort of in August timeframe of last year to provide we had provided six-month data on the patients for neurocog development. And by the time we had completed the rolling VLA, I think FDA looked at the dosing dates and knew we would have one year’s worth of data. So we supplied that off our mid-cycle meeting. And that did trigger a major amendment. So that moved us the PDUFA date from November to early February. We did, though, have a meeting post-submission of that data with FDA to confirm that there would be no additional request for a data set beyond maybe single patient questions or queries. And they confirmed that that was true. So we feel like the data set they have is adequate for their review.

And we think that teases up well because Steve can speak to it that we really like the data. It correlates beautifully with the biomarker and shows exactly what we had hoped to show on neurocog development.

Steve Pakola, CMO, REGENXBIO: And the nice thing is having the one-year data only helps further. We got to present that at ICIEM pretty recently. Had a very good response. And the correlation holds up beautifully. So that’s what we need to show.

Devjit, Therapeutic Analyst, Guggenheim: Got it. And so at the February 8th PDUFA, there are no real risks that we should be wary of?

Curran Simpson, CEO, REGENXBIO: No. I think we see normal information requests coming through, some of which now in an encouraging way are around post-commercial commitments, things like that. So our expectation is that and I also think this is just being optimistic that there’s an opportunity here for a traditional approval, not just accelerated because think about it, we’ve provided the clinical data that would normally go with a traditional approval. So I think that you saw that with Precision as an example. So I think we’re hoping to be in that category.

Steve Pakola, CMO, REGENXBIO: Another really nice aspect is all the other parts of review, like manufacturing and CMC. So we’ve already completed a product license inspection, clean as a whistle, no observations, as well as the typical clinical inspections at both of our facility and also clinical sites. Again, no observations. So it’s really a testament to the program and the company. And that’s going to help us with Duchenne. So we’ve really de-risked a lot of the key components from a regulatory standpoint.

Devjit, Therapeutic Analyst, Guggenheim: Got it. Is this program eligible for a PRV?

Curran Simpson, CEO, REGENXBIO: Yes, it is. So if we’re approved before September of 2026, we would receive a PRV, which we would have the option to monetize.

Devjit, Therapeutic Analyst, Guggenheim: Got it. So you own it, not your partner?

Curran Simpson, CEO, REGENXBIO: We do, yes.

Devjit, Therapeutic Analyst, Guggenheim: Got it. And for the DMD program, if the data matures like you think it should, would you pursue the commissioner’s voucher?

Curran Simpson, CEO, REGENXBIO: I think it’s definitely on the list of things that we’re trying to do to accelerate approval. You see, I think the type of products so far that have received that voucher are a little bit different than a Duchenne gene therapy, but given that our manufacturing is all US and that we have a mature data set coming on the clinical side, I think that’s very much something we’ll pursue. I’ve heard that there’s thousands of applications, so trying to be modest with expectations.

Devjit, Therapeutic Analyst, Guggenheim: Got it. Well, appreciate the time today. Thank you so much. And looking forward to a pretty amazing 26th.

Curran Simpson, CEO, REGENXBIO: Thank you.

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