REGENXBIO Inc. 2026 Global Healthcare Conference
Review the key takeaways and the transcript of this earnings call.
Transcript
Preview the first fifteen paragraphs, organized by speaker.
Baird Global Healthcare Conference. I'm Bryan Skorney. For those of you who don't know me, I'm one of Baird's senior biotech analysts. I have with us for my first fireside chat of the session, REGENXBIO. This is a company that I do cover, big fan of. They're working on a number of gene therapies. I would say they're more or less the preeminent gene therapy company out there as far as AAV gene therapy goes. We're going to walk through the story. They have a number of clinical programs, very late-stage at this point. A couple of potential approvals over the next 2 years, more than a couple. Maybe just to get things kicked off, it'd be great if you could just give us a very high-level overview of what's REGENXBIO, what do you guys do, what's the core competency, and what's the value of your AAV platform?
Yeah. Glad to be here, and thanks for the invite. We've been in business for roughly 15 years, working on AAV, primarily AAV8 and AAV9, as the base capsids that we've been innovating on. The company is really divided into 2 different areas. One is the retina programs. We've got Steve here, who's expert in retina, that can speak to that. And then the other is the rare disease programs, which is primarily near-term, the Duchenne program, which we started a few years back. And then behind that, we have the Hunter program that we're still working on an active BLA. So we have a broad set of indications, broad set of ways to deliver AAV, and probably, I think, the lead on how to deliver safely, which has always been a concern around AAV over the years.
I think we've really leveraged all of the institutional knowledge gained over those years and really applied it to our programs.
Great. So maybe we'll start with the Duchenne muscular dystrophy to kick things off. This has been a hot and cold area for the entire time I've been covering it, which I think goes back almost 15 years at this point. There's been a number of therapies approved. There's been a number of disappointments in terms of clinical trial data. Talk to us about RGX-202, your DMD gene therapy. When you compare and contrast it to the other gene therapy on the market, ELEVIDYS, what do you see as the key advantages here and in terms of the design of the dystrophin itself, in terms of the vector and the C-Terminal domain, and what that could potentially mean in terms of functional benefits?
Yeah. I think there are several pillars in the design that differentiate our product. One is the core design of the construct that includes the C-terminus, which is naturally present in full-length dystrophin. It has been incorporated into our microdystrophin. Early on in preclinical studies, we could see it behaving differently and able to affect actually repair the muscle as opposed to just enabling the muscle to show force. That is a huge differentiator. I think also the purity level of the product. We have been working on AAV manufacturing for over 10 years. We are probably the only company to be able to make high-dose AAV at 80% full capsid, which I think is extremely appealing to FDA.
Particular to Duchenne, Steve's team really looked at other, I think, technologies that evolved in transplant, for example, where we used a modified immune suppression regimen to enable us to deliver safely this high-dose AAV and give patients the best possible chance at functional benefit. Early on in the program, we could see right out of the gate that our SAE rates were much lower than the currently approved program. Then over time, as we have accrued additional functional data, we have started to see this really large magnitude of effect from patients versus their baseline levels that really is differentiating on function as well. Alongside of that, more at the biomarker level, very high levels of microdystrophin, very high vector copy numbers per cell, which we hope will translate to long-term durability of effect as well. We love the data that has been created.
The top-line data we think was really compelling. Everyone that we have shown it to, investigators, patient groups, all really are supportive of it. Our sole focus now is to move that towards the BLA submission and potential approval next year.
Great. One of the things that we have been very impressed about on the dystrophin expression side of things is not just the depth of microdystrophin expression that you are getting, but just the consistency compared to ELEVIDYS. I know in the FDA review, they had slides showing a fairly large percentage of patients are at very low levels of expression. You guys have shown some interesting data. Can you talk a little bit about how you have presented the expression correlation with functional benefit out of the patients they have treated so far?
Yeah, absolutely. Maybe I'll let Steve take that one.
Sure. I think that's one of the numerous differentiators that we see from our data package. I think one of the key aspects is, just to presage that, is all of those safety benefits are what allow us to get to an optimal dose. We believe that's key for differentiation, not just on safety, but also the efficacy. It may be where it actually makes sense, and if you only have a modest efficacy benefit, you may not be able to show correlation. For us the consistency of the microdystrophin, and also translation of that into consistency on functional benefit has been a key story, and related to that, particularly for accelerated approval, that translating into actual correlation. That's always been in our statistical analysis plan, and we've actually shown that.
Based on looking at NSAA correlation to microdystrophin, we've seen not only a high statistical significant correlation, but what's important is it's a very strong correlation. If you look at the correlation coefficient, it's roughly 90%, or 0.9 I should say. That's much stronger than what's been seen previously. With some cuts with ELEVIDYS, there's a very small correlation.
The Peter Marks cut. Yeah.
Peter Marks showed that, and it shows there's an interest in that from a regulatory standpoint, but that wasn't viewed as compelling because of how weak that correlation was, and that's why in the label, it's very clear that clearly stated that there's not a correlation. The other thing is we see that consistently across the different ways of looking at NSAA, whether it's propensity score weighting, which is the key primary, but also, external control matching or cTAP approach. That's based on the top line cut that we did, that we presented earlier this year, and we look forward to having that data with a larger dataset, as we advance and have the full dataset for the pivotal studies.
Great. We just touched on Peter Marks, but the DMD space has been one of the hot button topics in regulatory overall. We've seen a lot of leadership interventions, in DMD reviews, over the last 10 years. We have sort of new leadership in CBER right now, and you guys are in the process of submitting or planning to submit in the very near term, a BLA for RGX-202. So maybe just walk through us where regulatory discussions are and what stage you're at in terms of planning for BLA submission and what those next steps are and relative level of confidence and how the regulators are looking at your package.
Yeah, and this is where I think, excuse me, the Hunter program is a little bit intertwined with RGX-202 in that we just submitted a BLA for Hunter. So from an organizational standpoint, we know how to do that again for Duchenne. We have the non-clinical section of the BLA already complete, and ready to go. CMC and clinical are going as planned, so we'll expect to do a Q1 submission. What's a little bit new is we've had back-and-forth discussions with leadership at FDA. They want to see the data, and so we're very eager to bring the data to them. So we'll do a pre-BLA meeting late this year with FDA to go through it. And if you think about it, the last time FDA saw our data was in the end-of-Phase 2 meeting. We have about 10 times that amount of data.
FULL TRANSCRIPT
Continue the full translated transcript in StockNow.
Access every statement, the English original, and speaker-by-speaker history with StockNow Pro.
View the full transcript with ProCall participants
4 people spoke on this call — only 2 are shown here.
PARTICIPANT LIST
View participant details in StockNow.
Log in to see executives and analysts, their roles, and complete speaking history.
Log in to view all participantsKeep exploring