Regenxbio at RBC Conference: Strategic Moves and Clinical Progress

On Tuesday, 20 May 2025, Regenxbio Inc. (NASDAQ:RGNX) presented at the RBC Capital Markets Global Healthcare Conference 2025, outlining its strategic financial maneuvers and clinical advancements. The company highlighted a non-dilutive financing deal aimed at preparing for commercial launches, alongside updates on pivotal clinical programs. Despite challenges in the competitive landscape, Regenxbio remains optimistic about its differentiated product offerings and manufacturing capabilities.

Key Takeaways

• Regenxbio secured a non-dilutive financing deal to accelerate pre-commercial preparations for RGX-202 and RGX-314.
• The company is progressing with pivotal studies in its wet AMD program with AbbVie and anticipates top-line data next year.
• Regenxbio’s RGX-202 program for Duchenne Muscular Dystrophy shows promise with a favorable safety profile and ongoing patient enrollment.
• The company emphasizes its in-house manufacturing capacity as a key differentiator for high-dose AAV therapies.
• Regenxbio expects regulatory approval for its Hunter program in 2027 following a 2026 filing.

Financial Results

• Regenxbio announced a strategic non-dilutive financing deal to advance commercial launch preparations, particularly for RGX-202 and RGX-314.
• The financing, triggered by intrathecal data, involves limited recourse, meaning the company is not obligated to repay if sales underperform.
• The company retains 100% ownership of its Priority Review Voucher, which has exceeded $150 million in recent data points.
• Regenxbio anticipates meaningful double-digit royalty streams from product sales, contributing to bond repayment.

Operational Updates

• The Hunter program’s Biologics License Application has been accepted, with ongoing interactions with the FDA.
• Regenxbio is preparing for a potential Advisory Committee but considers its likelihood low, based on past precedents.
• The wet AMD program is advancing with pivotal studies, aiming for a 50% reduction in injection burden for patients.
• The RGX-202 program continues to enroll patients, unaffected by Sarepta’s recent challenges, with top-line data expected next year.

Future Outlook

• Regenxbio is confident in its in-house manufacturing capabilities, producing 2,500 doses per year for high-dose AAV indications.
• The company is positioning itself for potential commercial launches while monitoring the regulatory and competitive landscapes.
• With an improved cash runway, Regenxbio remains focused on strategic financial management and clinical progress.

Q&A Highlights

• Regenxbio reported ongoing, routine interactions with the FDA regarding the Hunter program, with no concerns affecting the overall strategy.
• The company addressed community reactions to Sarepta’s safety issues, emphasizing the safety profile of RGX-202.
• Regenxbio expects the market for its therapies to grow, with potential for a second-generation therapy offering an improved benefit-risk ratio.

In conclusion, Regenxbio’s presentation at the RBC Capital Markets Global Healthcare Conference 2025 provided insights into its strategic financial initiatives and clinical progress. For a detailed account, refer to the full transcript below.

Full transcript - RBC Capital Markets Global Healthcare Conference 2025:

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: Luc Aysi, senior biotech analyst here at RBC Capital Market. And today is our great privilege to have REGENXBIO with us, as part of our twenty twenty five health care conference here at RBC. Representing the company, have Curran Simpson, chief executive officer Steve Pakola, chief medical officer and, obviously, Michi Chen, chief financial officer. So, guys, thanks so much for taking your time. Looking forward to the conversation here.

We do have a long list of questions here. But maybe before we go into those questions, given this is fresh off the press, maybe if you don’t mind, I’ll start with you. Mitch, just walk us through the deal that you have announced, yesterday, what’s the rationale behind it and why you’re excited about it?

Michi Chen, Chief Financial Officer, REGENXBIO: Yes. I think the deal yesterday was a very opportunistic non dilutive financing. It really allows us to pull forward some of the midterm royalty stream and bring it forward to today so we can get ready for precommercial launches, such as getting ready for 02/2002 as well as RGX-three 14. As you probably recall, we are expecting top line data early next year for both two zero two as well as 03/14. But this capital will get us well beyond just the top line into commercial launch.

And this financing deal was really opportunistic, as I kind of described, when the primarily for Zolgensma and intrathecal data came to light, we actually got multiple bids, and this was the best offer that we received. Given that was limited recourse, in other words, if for whatever reason, the sales performance of these products do not perform well, the company is under no obligation to repay it. Having said that, if it becomes a blockbuster, the royalty stream remains with us. So the tail end of residual value of these royalty streams remains with the company. So it is there’s good debt and there’s bad debt.

There’s clearly a good debt to have.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: Super helpful, especially in this environment. Companies probably need to be a little creative with how you extend the runway. Maybe I want to jump into the FDA. Obviously, some changes, with, Doctor. Prasad at CBER.

He’s been historically pretty outspoken against the approval Sarepta. I think he has been, you know, a little critical also on kind of using surrogate biomarker more broadly, even in the his category, if you will, with MRD or multiple myeloma, which, again, not necessarily relate to you guys, but the broader it’s in the broader themes of, surrogate endpoint and approval. Maybe current, what’s your take? What was your reaction when you saw Doctor. Prasad being nominated?

And how are thinking about, implications for REGENX?

Curran Simpson, Chief Executive Officer, REGENXBIO: Yeah. I think it’s something we, are keeping a really close eye on. Obviously, we’re in the middle of a VLA review for our Hunter program. And so we have sort of day to day or week to week interaction with FDA at the review level. And to date, we see this sort of operating as business usual, the type of information requests that we’re getting.

As you know, Hunter is also an accelerated approval pathway submission. Our pretty ordinary information request, meaning, you know, details of CMC, but not anything that would make us question the overall strategy or accelerated approval. I think the Reagan Udell meeting as an example was one where industry and FDA and others, patient advocacy groups came together, I think that those discussions, I think, still make sense and still are in the interest of bringing new therapies to patients as soon as possible. So I think we’ll we’ll we’ll stay tuned on on developments and, continue to push hard on accelerated approval. What really I think matters the most and what mattered when we reported our first Duchenne data last November is, can you associate consistent microdystrophin levels with functional benefit?

And if the answer is yes to that, then I think we have a really good discussion coming with FDA, assuming that our pivotal dataset reflects that initial data.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: That’s super helpful. Maybe, Steve, if I can pivot to you quickly. Obviously, the tragic deaths of the patients in the Sarepta commercial settings, like what has been the broader reaction from the DMD community? Are now docs being a lot more cautious in prescribing this in non ambulatory patients? And is that informing in any capacity your strategy going forward here for your DMD?

Steve Pakola, Chief Medical Officer, REGENXBIO: Sure. So I and a lot of our team were on the ground at MVA. Quite a coincidence when that news came out, but it did give us an opportunity to speak to our investigators and a lot of the thought leaders in in the space to get really an immediate reaction. And, you know, I think there was the understandable, how to describe this, initial instinctive caution or somewhat reminiscent of what happened with the Pfizer. They gave you don’t know if, like, a a.

And I think at a macro level, there’s there was that initial caution. But the more we we spoke to the leaders in in the field, the more it also became clear that you have to look at the context. So this was an older boy, a heavier boy, non ambulatory boy, where there always has been that the risk may be greater there, both because of a higher viral load because per kilogram based dosing and also the comorbidities that this in older boys. So I I think there’s more caution when you think of that end of the spectrum. But it it was really interesting, Luca.

There there was for us, at a our micro level, if you look at different programs, this really shined the spotlight on state, of course, within there’s always a safe person. So it really reminds the field of that. And that’s where we actually had investigators coming to us saying how comforted they are with what we’ve seen from a safety standpoint, where we’ve seen zero liver AESIs in our Phase III study ever since special interest, including none related to the liver. And it’s always been known that forty percent or so of the levodates have LFT elevation. So that is creating supporting the differentiation that we see with our program and also supporting our original decision to have a very robust immune modulating regimen that’s relatively short, but gives you the investigator and the patient families comfort to really mitigate the risk of type of fortunate events.

So how does that relate to enrollment? We’re seeing no change in our patient funnel. That’s why we continue to be excited about pivotal enrollment, this year.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: Got it. Got it. Super helpful. Maybe just, one more on kind of Sarepta. Obviously, they also lowered the guide, the financial guide for the year.

So is that a good thing or a bad thing for you guys? On one side, you can argue the good thing because maybe by the time you launch, you know, prevalence pool, a larger proportion of the prevalence pool will be up for grabs from you guys. On the other side, you can maybe take the other other argument and say that maybe the demand is not, you know, as robust as some of us who are kind of originally gaining. So good thing or bad thing?

Curran Simpson, Chief Executive Officer, REGENXBIO: I think it you know, if you look at the launch in totality, you know, it’s it’s a successful launch for an AAV based program and we’re certainly rooting for for more of those. But I do think your point, it provides more opportunity. If we follow our path through a filing in 2026, We would expect an approval in ’27 and mentioned that prevalent market would be even greater than we were estimating. We were thinking at least half of it being available at that point, perhaps more. I hope that, you know, if there is hesitancy in Duchenne, some of that hesitancy is potentially waiting for a second generation program to come along that has differentiation in terms of benefit risk ratio.

And so this is the opportunity that I think we have. We’re at all the meetings this week here at Duchenne. We’ll be at PPMD talking with investigators and getting a better feel for for sentiment in general, but we we really like the profile of our program and think that it does have a fit and will have a demand associated with what we’re seeing in our clinical setting, but down the road in commercial.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: That’s super helpful. Maybe one more in the competitive landscape. What are maybe, Steve, for you, going back to you, what’s your views on the skippers? It feels like the death yield is making some meaningful progress when you look at like somebody conjugated, whether we didn’t want to flow anybody or an antibody fragment, no high level of expression that we have historically have seen from the naked constructs with. So like, appreciate this is not necessarily like, we can debate if it’s complementary or competitive, but like, what’s your view on how the two technologies will put the market, one stuff, hopefully, all of them come reality?

Steve Pakola, Chief Medical Officer, REGENXBIO: Yeah. First, I’d say we’re we’re excited in all of the areas we’re working, particularly treating these patients and helping families. So it’s exciting to see different modalities advance in some of the encouraging results that we’re seeing. We definitely see it as complementary and that there’s always going to be a need for improved therapies and to have a one time gene therapy where you can pass the activity. And that can be mutation agnostic, I think, is the other aspect.

So there’s always going to be that need beyond the traditional high prevalent exon mutation. So excited to see the field move forward and feel excited to potentially play a prominent role in that differentiated that we so far see with our product.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: Sure. Sure. Super helpful. And, yeah, there’s an argument. We’ll see how the payers respond, but not inconceivable that if, you know, you have a family member that has it, you’ll they’re both.

Right? Maybe they’re the person who’s skipper down the line. But, that is very helpful. Maybe let’s talk about, maybe one more on kind of competitive, solid bios out there also with some pretty interesting data. Maybe what’s your big picture view on their data first?

And then second, how are you thinking about the cardiac biomarkers and how you plan to capture cardiac benefits there?

Steve Pakola, Chief Medical Officer, REGENXBIO: Sure. So I think a couple of key factors in looking at data is how long out do you have the data and what kind of clinical studies you have. So it’s still early days. I think there is the microdystrophin depression levels, I think, still to be seen how that plays in terms of functional data. And also, what you might expect to see in an older patient, which is where we in the field are very excited with the data that we’ve seen.

Quite surprising for the field high consistent levels, even above age range, has not been approved other other therapies. Cardiac function for us and the the field of investigators, others that we speak to feel it it makes sense that that really is the safety biomarker in the short term standpoint. So when you’re you’re looking at three, six months a you’re really looking at safety. And it really takes longer to really evaluate on the efficacy side of the coin. And that’s even more the case if you’re looking at younger boys that you haven’t yet seen actual dysfunction.

But I think that’s really how we look at it. So I think time will tell. We certainly look at ejection fraction MRI. So we’ll be looking at those things. But realistically, it’s going to take longer than you’re looking at that other than at the table.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: That is super helpful. Maybe, Karin, going back to you. There’s a data update, obviously, coming up Just give us a little bit of a preview on what we should focused on and maybe what’s the extent of the functional data that you’re going to be able to share with us? And how are you going to help us contextualize this data versus natural history or baseline characteristics or or any of the other components there?

Any thoughts?

Curran Simpson, Chief Executive Officer, REGENXBIO: Yeah. If you go back to our release last November, you’re gonna see a lot of consistency with the approach we took there. So we’ll primarily focus the the update on dose level two, which is the same dose we have in our pivotal studies, and we’ll expand. At that time, we reported nine month data on two patients. We’re pointing towards, although it’s still in draft form, nine months data on five patients and roughly twelve or twelve month data on roughly four patients is is what we’re pointing towards.

And I I think that the comparisons will be made, you know, versus their baseline incoming functional capability. And then we’ll also do the external match controls that we showed before to compare where those patients would have been based on natural history and do the comparisons and try to equate that to what we consider a clinically different. So very similar approach to what we did in November, but the main goal here is for those that said, well, it’s only an n of two. Now we’re trying to enlarge that dataset. And then I would expect second half of this year more focused on our pivotal study enrollment updates, and then that’ll point us towards first half of next year.

We’ll be really excited to report out our top line data from the pivotal study.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: That is very, very helpful. Maybe in the interest of time, let’s pivot to Hunter. Maybe just what’s the latest thinking on that one? What’s the likelihood of an AdCom at this point? How should we think about the PRV and around the PRV and the timing?

And then maybe, Mitch, if you don’t mind us, maybe remind us the economics that you have still left in that program. So we’re

Curran Simpson, Chief Executive Officer, REGENXBIO: we’re well underway with the review of the BLA submission was filed. And not too long ago, we reported that we had gotten acceptance in the BLA, which is, I think, an important derisking element to to a filing for accelerated approval. We see a steady stream of information requests coming in, which is a good sign of active review ongoing and feel really good about that. Now in terms of the AdCom, we don’t have a final view yet from FDA, but we are, number one, preparing as if we need one so that if if one is required, we’re ready to go. But we think the precedent, there’s a program in front of us for MPS III that they’ve been public about not reading or not requiring an adcom from FDA.

We think that if you equate that to a similar program in MPS II, that the likelihood is fairly low. But we’ll be ready either way. I think all of this, in general, the information request, the acceptance of the BLA really does derisk PRV, which we will be eligible for upon approval. And Mitch will talk a little bit about the recent market, if you will, if we please monetize.

Michi Chen, Chief Financial Officer, REGENXBIO: Yeah. So I think the PRV, last five data point has gone north of $150,000,000. We still own 100% of the PRV. So we, you know, could actually afford to see how the market evolves as we get closer to approval timing. Regarding the economics of the deal that you just disclosed yesterday, for the product sales, we do get royalty streams.

We basically said meaningful double digits. That would obviously contribute to the bond at this stage. Once the bond is repaid off, that royalty stream, any proceeds will come back to us at that stage. And any development milestones, the vast majority we actually retain, that remains an impact for the company.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: That’s helpful. Let’s maybe pivot to ocular here. Maybe, Steve, back to you. Just walk us through kind of what your big picture strategy. And then maybe you know, I I feel like the whole space of gene therapy for what the MD or Doctor kind of pulled off recently.

There’s a little bit of a perception that maybe the standard of care has made so much progress, especially on the what the MD side that and some of the novel novel approaches. Like, do we even need some care for you? What what’s your what’s your take?

Steve Pakola, Chief Medical Officer, REGENXBIO: I can’t help but take the the latter question first.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: And you’re going to do that. Yeah.

Steve Pakola, Chief Medical Officer, REGENXBIO: You know, and and you can speak to the outside in part, but having worked in the retina space for a lot of years and seen the nice evolution in VEGF treatment option. And there has been for a while a recognition of the big unmet need, less frequent burden with patients because they’re simply not getting VEGF And we’ve seen incremental advances, first EYLEA, Babizmo, and EYLEA high dose, where these are blockbuster potentials that are being proven. And really, though, this is truly incremental. We’re talking about extending the probability of needing every eight weeks to twelve week or in some patients, twelve to fifteen weeks. And even that relatively small incremental benefit is leading to block.

We actually view this as validating us as opposed to worrying about any anyone getting our our lunch because this is not this is a a paradigm shift in terms of being able to tell a patient not that, okay, you’re still getting injections for the rest of your life, but it may be a little less frequent, which they value and the clinicians value. But here, we’re talking about dramatic reduction over 50% as a target and even a sizable proportion of patients that may not need any infection or that you need treatment for the rest of the patient’s life. So for us and AbbVie, this is why we in big pharma who understand ophthalmology and global potential here, are excited about our strategy. So pivoting to the first part of your question, we and AbbVie continue to advance on our initial subretinal program for treatment of wet AMD with our pivotal studies atmosphere and stent, enrolling 1,200 patients, the largest gene therapy program ever executed. And this is on a global basis, It’s very important to us, but also AbbVie.

And we’re going to complete enrollment this year to allow for top line pivotal results, the one year endpoint next year. So there’s a lot of excitement there as we advance toward that. Me and AbbVie, also super excited about in office suprachoroidal delivery where we announced, along with AbbVie, plans to advance into pivotal development for diabetic retinopathy later this year. And to your question of potential with the anti VEGF given some of the increased duration products, this space is completely available just to a one time patient’s at that stage where you want to prevent the vision threatening complication, they’re still asymptomatic at that stage. So we’re seeing that those patients are not signing up to get no matter how infrequent they are.

So that’s one of the reasons we have the year or excited about it.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: Is super helpful. Maybe one last one on subretinal for wet AMD. Obviously, the pivotal data is coming in the foreseeable future, the new trial that you just articulated. But it feels to me that the primary endpoint is pretty high POS, right, given just the way the trial is designed. But how should I think about a secondary endpoint?

Like, what’s what’s the bogey here for actual clinical adoption? Do you need to show at least fifty percent of patients that are injections for you one year? Should the number be higher? Like, it could probably require require an operating room. And so what’s the bogey in the second right point?

Steve Pakola, Chief Medical Officer, REGENXBIO: Yeah. It’s an interesting, Luca. The response from the clinician over the years has been pretty consistent. It’s really been around about fifty percent. And that’s always been our target profile.

And it continues to be because, as you can imagine, are constantly keeping my finger on the pulse. And really, again, it comes back to that value proposition, where if you can bring this for that much, including patients who don’t that is real and getting a lot of traction.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: Got it. That’s helpful. Supracoroidal, quickly, what’s your latest thinking on safety? You know, you guys have obviously optimized, some of the, some of the regimen here to minimize the risk of inflammation. But, like, what’s your latest thinking on on that potential risk?

It’s key, especially in indications where, you know, the bar is pretty high given the

Steve Pakola, Chief Medical Officer, REGENXBIO: Yeah.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: Current standard of care is actually safe.

Steve Pakola, Chief Medical Officer, REGENXBIO: And historically, in this space, a lot

Michi Chen, Chief Financial Officer, REGENXBIO: of Yeah. Right.

Steve Pakola, Chief Medical Officer, REGENXBIO: Sensitivity to inflammation, particularly. And with gene therapy, particularly with intravitreal injection, where the issue is the non localized, non localized delivery where you get off target. And that’s the reason right from the beginning. For our follow on to subretinal in an attempt to get in office, a requirement was that it’d be around a good duration that preserved the really nice aspects of the retinal, which is being as close to the target. And that’s what we have in suprachoroidal.

And we believe that’s a big component of why we’ve seen such good safety, where we only apply a relatively short topical prophylactic steroid regimen. So we need a lot shorter than what has been needed for other programs, particularly for pivotal, which is very important to the client. So I think we’re in a really good position, and we’ve already hit our target product profile on the efficacy side from our Phase II study. So I think we’re really set up well for the pivotal.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: Super helpful. Maybe, Kuran, last question for you. Big picture. What do you think is the most underappreciated aspects of the REGENX story that, we haven’t asked you or any kind of closing remarks or any thoughts before we wrap the conversation.

Curran Simpson, Chief Executive Officer, REGENXBIO: I think if you look at what we heard today, the cash runway been improved with the recent deal that with HCR. And then pointing to next year’s top line data on repivotal programs, I think we’re in terrific shape. One thing I would add to the mix is from a gene therapy aspect, we’re the only one with in house manufacturing scale that we have. So these high dose AAV indications like Duchenne, where you have in house manufacturing able to make 2,500 doses per year at a great cost of goods, that’s a differentiator for us. But maybe people are missing that now.

But as we get closer to launch, everything will come back.

Luc Aysi, Senior Biotech Analyst, RBC Capital Market: Got it. Super close. I have 300 more questions, but no more time. So thanks again for joining us. Thanks, everyone, for joining us for this conversation.

And, yeah, we’ll we’ll talk soon. Thanks for having us. Thanks, Kent. Absolutely. Thank you.

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