Regenxbio at TD Cowen Conference: Gene Therapy Advancements

On Wednesday, 05 March 2025, Regenxbio (NASDAQ: RGNX) took the stage at the TD Cowen 45th Annual Healthcare Conference to discuss its strategic roadmap. CEO Karen Simpson highlighted both the company’s progress and challenges, focusing on its gene therapy pipeline aimed at treating debilitating diseases. While optimistic about upcoming milestones, the presentation acknowledged the competitive landscape and the need for successful regulatory approvals.

Key Takeaways

• Regenxbio plans to file a Biologics License Application (BLA) for RGX-121, targeting Hunter Syndrome, potentially the first gene therapy for this condition.
• The Duchenne program (RGX-202) has advanced to a pivotal stage, with a market opportunity of $7 billion.
• The retinal franchise, in collaboration with AbbVie, targets the growing wet AMD market, expected to reach $30 billion.
• The company emphasizes its in-house manufacturing capabilities, crucial for high-dose therapies.
• Potential commercial launches and pivotal data are expected in 2025 and 2026.

Financial Results

• RGX-121 (Hunter Syndrome):

- Market opportunity: up to $1 billion

- Partnership with NS Pharma includes a $110 million upfront payment and up to $700 million in sales milestones

• RGX-202 (Duchenne):

- Market opportunity: potentially $7 billion

• Retinal Franchise (partnered with AbbVie):

- Wet AMD market: currently $18 billion, expected to grow to $30 billion

Operational Updates

• RGX-121 (Hunter Syndrome):

- Imminent filing of the final BLA module

- Partnership with NS Pharma finalized

• RGX-202 (Duchenne):

- Entered pivotal stage with a strong safety profile; no serious adverse events reported

- High levels of microdystrophin expression observed

- Pivotal study enrollment expected to complete this year

• Retinal Franchise (partnered with AbbVie):

- Two administration modes: subretinal and suprachoroidal

- Pivotal studies for subretinal treatments nearing completion

- Suprachoroidal study for diabetic retinopathy to begin dosing this year

• Manufacturing:

- In-house capabilities allow production of up to 2,500 doses annually

Future Outlook

• RGX-121 (Hunter Syndrome):

- Potential commercial launch in 2026, subject to approval

• RGX-202 (Duchenne):

- Top-line data anticipated in 2026, with potential approval in 2027

• Retinal Franchise (partnered with AbbVie):

- Top-line data for subretinal program expected next year

- BLA filing by AbbVie planned

- Ongoing suprachoroidal studies in phase one/two for wet AMD and diabetic macular edema

For a detailed overview of Regenxbio’s presentation, refer to the full transcript below.

Full transcript - TD Cowen 45th Annual Healthcare Conference:

Cabral Happy, TD Cowan: Good afternoon, everyone. Hope you’re all doing well. My name is Cabral Happy and I’m here with TD Cowan. Thank you so much for joining us today. We have, a presentation that’s gonna be led by, CEO of Regenexx, Karen Simpson.

So without further ado, I’d like to welcome him over, and thank you again for your time.

Karen Simpson, CEO, REGENXBIO: Thank you for the nice intro and thank you for the invite and thanks for coming over right after lunch. Much appreciated. Really thrilled to talk to you a little bit about REGENXBIO. I will be making forward looking statements. There will be a quiz after this as to what was on line 12 for those that want to take a quick peek.

But let me just talk a little bit about the company. We are one of the founding gene therapy companies, have been going for fifteen years. We originally started out as a licensing organization, Zolgensma, which is a huge product for Novartis, came from our original IP. But over time, we decided we’d rather internalize development, develop our new products internally and do less licensing, and that’s how we’ve pursued the last five to seven years. Our vision, is a world where debilitating diseases can be treated with a one time therapy, and our mission is to seek in how to improve lives through the curative potential of gene therapy.

The areas that we’re pursuing are significant. Our lead program in terms of near term commercialization is RGX 121, which is for Hunter syndrome, represents a market opportunity up to $1,000,000,000 We have a Duchenne program that I’ll talk about, that represents a market opportunity of potentially $7,000,000,000 And then the wet AMD programs, which are partnered with AbbVie, is a huge market, 18,000,000,000 and growing every year. So large significant opportunities within our portfolio. As I look at our pipeline, I’d like to mention as I mentioned the RGX 121 program, we’re in the process now of imminently filing our last module of the BLA for that program. Our first BLA is a company.

And yesterday, we were pleased to announce the closure of the deal with NS Pharma, which got through Hart Scott Rodino review, and that’s a significant partnership for us as we move towards commercialization. And I’ll talk a little bit about that partnership later, but we’re thrilled to to announce that closing of the deal. We have a major program in Duchenne, which is just, last fall entered pivotal stage. We’ve released really interesting data over the course of last year in terms of safety, biomarker data, and then later in the year, functional data. And we’re really excited to be recruiting patients now in a pivotal phase.

And then we have this large retinal franchise that’s partnered with AbbVie, two modes of administration being explored. One is subretinal, which is an OR based procedure, which for wet AMD. We’re getting close to closing enrollment on two large studies, almost 1,200 patients across the two. And then, we’re looking forward with AbbVie this year to moving into a new mode of administration, suprachoroidal, which is an in office procedure, for diabetic retinopathy, with phase one, two studies still going on for suprachoroidal administration, in wet AMD and DME as well. So for a three forty five person gene therapy company, we’ve got a lot of really valuable late stage assets that we’re excited to continue to develop.

What’s in store for ’25 and ’26? These are really, really important years for us. That’s why we titled it. I think we stole that from somebody, but the future is now. And I’ve been with the company for over ten years.

And every year we talked about getting closer to commercialization. And, well, here we are. We’re getting very close. So as I mentioned, imminent, filing of the last module of our BLA for RGX one twenty one, which is being filed as part of an accelerated approval potential for Hunter Syndrome and as I mentioned now partnered with NS Pharma. We also this year will report additional phase one, two functional data for our Duchenne program.

We gave an update last November and will further that update, during this, this year. And we also intend to complete enrollment of our pivotal study for the program this year. So we started that, late November of FDA feedback and are doing really well with enrollment, excited about the potential there. And then, as we’ve disclosed earlier in the year, we intend to complete two large pivotal studies for subretinal with AbbVie. One’s called ATMOSPHERE and the other one’s called Ascent.

Ascent is interesting in that, over time, we’ve globalized the program with AbbVie and they’re recruiting in Europe as well. So really excited about the potential there. And for diabetic retinopathy, we’re planning a pivotal program to dose the first patient in this year. In ’twenty six, even more exciting, commercial launch, we hope, of 01/21 pending a successful review and approval, top line pivotal data from our Duchenne program, top line pivotal data for our subretinal program. So a lot of catalysts and milestones over the next two years for very large opportunities.

One thing that differentiates Regenexx Bio from others is that we have our own in house manufacturing. And so that was a decision we took a couple of years ago, realizing that, all the way from Peter Marks criticizing gene therapy manufacturing as immature and needing development, we’ve invested over the last seven years on what we feel is really a top line commercial ready manufacturing process that particularly can support the Duchenne program, which is a high dose indication. And we have control of our own internal manufacturing, including fill finish capability. So it’s a really significant differentiator for us as we think about commercialization. I’ll talk briefly about RGX one hundred and twenty one.

This would be the first gene therapy potentially approved for Hunter syndrome. There are no, effective cures or treatments available at this point, so the unmet need is significant. RGX one hundred and twenty one has the potential to treat up to seventy percent of MPS II patients. We’ve already shown our pivotal data, about a year ago, which was successful in reducing a biomarker called d two s six, close to normalized levels. And so we feel we, and we presented that data to FDA in our pre BLA meeting, and got very positive feedback.

So we feel very positive about our eligibility for accelerated approval. And as we mentioned, our partnership with Nippon, NS Pharma, also helps us on cash runway. So there’s a hundred and $10,000,000 upfront payment that was made or will be made as part of the deal closing. But even more importantly, partnering with NS Pharma allows us to bring, this product to patients faster. They have an existing commercial infrastructure, and we will manage this commercial supply chain for the program.

So really exciting developments here. The prevalent population for MPS two is significant. There’s almost five hundred patients in The US that have MPS two, and the incident population is roughly fifty patients per year. So significant opportunity that we look forward to developing with NS Pharma in terms of commercial outlook. NS Pharma, as you know, has experience deep experience in rare disease commercialization.

And as well, the deal structure allows us to receive up to $700,000,000 in sales milestones as part of the launch and sales performance. So really not just adding short term benefit to REGENXBIO in terms of our cash position and ability to commercialize, but long term value being recognized as the program progresses. So, this is something that we’re launching actively now. Given that we’re roughly nine months from launch, you can imagine the planning and activities that are are occurring on the commercial side, which is where we wanted to be. I won’t go into too much detail here, but we this is our first BLA as a company.

That’s always a significant element. And I think there’s two elements of it. One is obviously we’re excited to submit the final module of the BLA for Hunter. We’re really proud of the data that’s been generated. The patient community is anxiously awaiting new therapies.

But more importantly, from a strategic standpoint, this sets the stage for our Duchenne program as well. So writing your first BLA, you develop all the templates, all the ways of doing it. You have the review process in hand, and we basically are looking forward to repeating that entire process next year, as we prepare our BLA for RGX two zero two. And speaking of which, so RTX two is on track to be the next gene therapy for DMD. We think we have the potential to be second to market, and that’s significant because when we started this program two and a half years ago, we thought we might be fourth to market.

So, we’ve made up a lot of time, and we’ve had really important results over the last year, starting first with safety, and I’ll cover in detail the the safety and efficacy data that we have today, but also, extremely encouraging interim data, especially in older patients where we’re seeing, high levels of microdystrophin expressed in those patients. And what differentiates us coming second to market from the approved product is the inclusion of what’s called the C terminus in the construct, which we feel and actually, if you go all the way back to the adcom for Levitus was mentioned that it makes our microdystrophin more like full length natural dystrophin, and has an important element in repairing muscle once the muscle has exerted force. So we feel like not only are we potentially second to market in a large indication, but providing potential for additional functional outcomes that would be positive based on the differentiated design. So this is a program that is top of mind for us. Our main goal this year is to enroll the study as quickly as we possibly can, and beat the timelines that we’ve set out publicly.

I think the market for Duchenne is being defined as we speak. We see really good uptake commercially. Zolgensma led the way for gene therapy, but I think people were looking for what’s the next Zolgensma. And I think Alevitus is following a similar path in terms of sales, but I feel like, there will be a significant amount of the market available presuming a potential approval in 2027. We feel that the prevalent market will still be quite wide open in terms of entries for new products, and, we can manufacture, again, back to our differentiator in manufacturing, twenty five hundred doses per year internally, and that’s a significant level.

If you think about patients treated for Levitus this year, estimates are something in the range of a thousand, maybe a two thousand to three thousand next year on consensus estimates. So we can make that in one year in our own manufacturing capability, and we intend to begin commercial build next year to be ready for that. This is just a pictorial of constructs that are either approved or in the clinic, and just I wanted to point out the comparison for our program. You see RGX two zero two, and at the end of that, you can see the CT domain that differentiates it. One of the reasons that some of the early constructs may not have had the CT domain included is the cassette size for AAV is limited.

You can only package so much within AAV. And there was a constraint that if you included the CT domain, you couldn’t actually produce AAV vector to produce this transgene. So we’ve worked out ways with our proprietary manufacturing process to do that and to produce a product, which interestingly is at 80% full capsid, with a really high degree of genomic integrity. So this is a technological advance we’ve made in manufacturing to enable this differentiated construct to be made. So we’re real excited to implement that.

This is just a pictorial. I’ll just have your eye train to the orange bar. The blue bar is microdystrophin devoid of the CT domain. It’s not identical to anyone else’s in either marketed product or in the clinic, but it is a, quote, standard microdystrophin. The orange bar is that same microdystrophin with the CT domain included.

And this study measures a muscle flexed 20 times to look at the initial force that the muscle can withstand, and you can see a clear difference preclinically in the ability of the c terminus to affect function and resilience of the muscle to repair itself. And we think this is underpinning a clinical benefit that we’re hoping to see as we start dosing patients and look at long term outcomes that on a functional basis, those patients might have more opportunity to benefit. So what have we accomplished in the last year that’s gotten us to a pivotal study this year? So far, we have a really exceptional safety profile for this program. No SAEs to date in 11 patients treated, and no AEs of special interest, which is really significant.

We have a proprietary immune suppression regimen that we employ that we think helps with this, and definitely reduces the burden of monitoring for patients post treatment as a result of it. So we’re really pleased with that. And, I’ll show some data on the next couple of slides. The biomarker data that we’ve produced to date also shows very high levels of microdystrophin, particularly many of the patients we treated early were eight and older. And in those patients, other studies have shown fairly low levels of transduction and expression of microdystrophin.

We’re seeing much higher levels, which gives those children a better chance to benefit. And we’re seeing that in some of our early functional data, which is really exciting. So I like this table to be as boring as possible. As you can see, a lot of zeros in terms of these are, AEs that have been noted in other studies, common AEs that are found are elevation liver enzymes and complement activation. And we’re just not seeing that in our study, and I think that’s highly encouraging and I think highly encouraging also from an enrollment standpoint.

You know, people thinking about whether or not they wanna go on to a clinical study. This is the first step is to review safety, and, I think we’re being sort of favorably reviewed at the investigator level when they when they see this. But also as importantly, we did a dose escalation study last year. We started with dose level one, knowing that we wanted to ultimately get to dose level two because our preclinical data showed that we could restore function in the MDX mouse model roughly equivalent to wild type at the higher dose. And so we were always heading towards dose level two if we could show safety at dose level one and then dose escalate, which we did.

So at dose level one, though, you can see some of the different outcomes, but we saw microdystrophin levels in the four to seven year olds around sixty percent, very high percent positive fibers generated. And interestingly for our programs, very high vector copy number results, which indicates good biodistribution, in the program. Why is that important? For two reasons. One, biodistribution is great, even better if it’s localized where it needs to be on the fiber, which you can see from some of these slides.

Second is it could lead could, lead to longer durability of effect and because of that. So this is something that that we’re really hoping in our two year data will be born out and beyond. Now you can look at the dose level two data and you see, obviously, the vector copy numbers going up, higher dose, double the dose. A lot of vector ends up, in the liver at lower doses, and so it doesn’t get necessarily to the in the liver at lower doses, and so it doesn’t get necessarily to the muscle where you want it. But as you go up in dose, more of it gets to the periphery where you do want it, and you can see that in these numbers.

55, vector copy numbers per nucleus is really exceptional. And as exceptional as that is the microdystrophin data at seventy seven percent in four year olds and then forty percent, in the eight to eleven year olds. And and that’s an n of five in the eight to to eleven, so it’s getting to be a more significant dataset as we proceed. Comparatively, other programs are showing much lower levels of microdystrophin in these older children. So that’s one of the differentiators we’re seeing, with the Duchenne program.

And then this is data we put out in November for some of the timed function tests. And this is ultimately what matters to patients and to the community is how are the children doing, how are they progressing. And so these are data against matched external controls. So we have a database of roughly 400 patients that we pull from, and we match these patients to those natural history outcomes. And you can see in the blue bars, this is the NSAA development at dose level one for those patients at 12.

And you can see what would have happened if in the natural history setting they weren’t treated. And these are really, really significant differences in function. And I keep saying it, hopefully it comes true. If we can just reproduce this in our pivotal study with 30 patients, we have a great story for FDA, and I really feel like we’re onto something big here. Now turning to dose level two, little less data available at this point.

These patients were treated in the latter part of last year. But, just as impressive results, I will admit with a N of two patients, one of the patients is doing incredibly well. 10 and SAA improvement, I don’t think has been reported in any other gene therapy study. So we’ll need to widen that dataset out with additional patients, but I’ll take it, and I’m sure the patient will take it. We have a video of that child riding a bicycle through Manhattan.

They’d never been able to ride a bike before. So it’s pretty moving, to see it. But we really, really, enjoy seeing this data. Where is FDA looking? NSAA, if you think about Duchenne, if you followed it, was sort of discounted as an indice because it didn’t read out as positive in other studies.

Our data looks fantastic on NSAA so far, but also time to rise, is or time to climb as well. Those are other indices that are important. So we’re measuring all of these, and the full dataset is ultimately what we’re looking forward to reporting out through the course of this year as we get more data, but off to a great start. So we also monitor caregiver reported function as well. And again, these are things that children were able to do post treatment that some of them weren’t able to do before.

There’s the video of the child riding the bike, and running, participating in recreation, and walking in the community. So things that aren’t sort of sterile clinical data, if you will, that are really, really important to the parents of these children, we’ve been pleased to to be helping and improving lives in that way. So stay tuned. We’re going as fast as we can. And we’ve got a lot of enthusiasm in the patient community for enrollment.

So we feel like enrolling the full study this year is well within our reach, and that will enable, next year us to report top line data and file our BLA. Switching completely across to a different topic, as I mentioned, we have a large program in our retina franchise partnered with AbbVie, and this is something that we’ve been working on since Jim Wilson’s lab, nine years ago, in the ocular space. The subretinal studies were started initially, independently by us. And then sort of midway through those studies, we signed up the partnership with AbbVie who has, you know, an awesome commercial infrastructure to help us maximize value. We have two routes of administration, in the clinic, subretinal and suprachoroidal, so getting to the outside of the eye and to the inside.

Both are in studies. In, the SR study, I’ll show is in late stage pivotal studies will complete enrollment this year, top line data next year. Suprachoroidal studies will start a pivotal study in diabetic retinopathy this year, and, the wet AMD and DME studies are ongoing in phase onetwo still. Huge unmet need in terms of retinal centers being overwhelmed. Gene therapy may provide an opportunity to take patients, particularly patients who are, as as some of the docs call them, frequent flyers, who are needing 12 injections a year on average.

And so, we have data on the subretinal program, which I’ll show, that takes us out to two to three years now of durability, being shown stable vision and minimal, supplemental injections. It’s really exciting. The wet AMD market is expected to grow substantially over the next ten years, probably, up in the 30,000,000,000 range is is what we’re hearing and what people are estimating. So we have, to be transparent, people say subretinal, it’s an OR based procedure. Who would use it?

The feedback we get is that 10% to 15% of the market would potentially use it, especially those with severe disease. Ten percent to fifteen percent of three bill 30,000,000,000 is, well worth pursuing in gene therapy. So I’m really excited about the potential for both subretinal and then following behind it, suprachoroidal, which is in office procedure, could unlock even further value. So as I mentioned, these are large studies. Atmosphere is roughly five forty patients and Ascent at six sixty, so very large global studies.

And as I mentioned, we’re recruiting in Europe now, ascent as well because the intention for AbbVie is to file these BLAs, and they have responsibility for that globally. And then we are continuing for phase one, two studies in suprachoroidal for DME and for wet AMD. Those are still dose in the dose rate ranging studies. And then very interestingly on, diabetic retinopathy, I’ll show a little bit of data with the time left. We we see really interesting results on so Lucentis was approved for diabetic retinopathy, but the use is very low because most patients who have diabetic retinopathy don’t wanna sign up for a monthly injection.

So gene therapy is a perfect fit, for that because of the one time administration is much more attractive. But we’re delivering the same transgene as we are in subretinal. So this is just showing you for subretinal. Again, here are patients that are three or four years out with stable vision, minimal supplemental injections along the way. So long term durability and very importantly for gene therapy, ocular is very good safety profile, so very little intraocular inflammation being shown.

And again, we’re pointing towards top line readout of this data next year and and subsequent to that, filing of the BLA. And for diabetic retinopathy, when we think about the only administration that would be feasible if the if the market is not willing to sign up for monthly injections. They’re also not probably willing to sign up for subretinal administration. So we developed this program from scratch using the suprachoroidal administration. It’s called the ClearSide device that’s been approved in other types of programs, so it’s a commercial device.

I’ll just show you some of the data here. So on the left, you can see the control group over the course of, I think this is the twelve month data. Yes, one year. And you can see a certain number of the patients worsening, some with no change, but very few improving. And then you can see the control arm of the study I’m sorry, the treatment arm of the study showing no one worsening and either stable disease or improvement.

So the concept in diabetic retinopathy is upon diagnosis, treat these patients early and minimize the progression of Doctor, which will eventually become will worsen. And, we’re really excited from this data to show that, and there are two different regulatory endpoints available for that two step worsening or two step improvement that we’re following. And this is also showing vision threatening events, huge reduction, 37 in the control arm versus four percent in the treatment arm. So really nice improvement using the suprachoroidal administration. So to summarize, really excited about the potential with Retina, with Duchenne, near term with Hunter.

I don’t know of another gene therapy company positioned with such a broad portfolio with these late stage assets that it’s in the same situation that we are. But we’re really, really excited that, as I said earlier, the future is now for the company, and we’re really excited that we’re on the cusp of delivering these these therapies to patients. So I thank you for your time and appreciate you coming in and hearing our story.

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