enGene Therapeutics Inc. (ENGN) September 2026 Conference Transcript

All right, good morning, everyone, and welcome to the 2026 Morgan Stanley Global Health Care Conference. I'm Judah Fromer, one of the mid-camp biotech analysts here. We're very excited to have Ron Cooper from NGene kicking off the conference. Let me just read a quick research disclosure before we get started. Please visit www.morgansfamily.com forward slash research disclosures. It's an important year for NGene, Ron. Before we dive in, can you give the audience a quick intro to the company and a bit of the background on your decision to pursue non-viral gene therapy? Great.

Well, first of all, Judah, thanks for your invitation to be here. Great to see you. It is a pretty exciting year for NGene. So what is NGene? NGene is a company that looked at the challenges of viral gene therapies and said, I think we can solve some of these challenges. And those challenges being packet size, redosability, and the manufacturing challenge. So NGENE was discovered to build non-viral gene therapies. And then through the discovery process, translated a product called Dettolimaging, which is the first non-viral gene therapy that we are developing for non-muscle invasive bladder cancer. Dental imaging has been studied in the LEGEND program in Cohort 1, which is a pivotal program, 125 patients, which is one of the larger programs. And so we're having a pretty exciting time in the company. In this next quarter, we'll have some data that will mature, some durability data that will mature. We plan to meet with the FDA. We plan to file dental imaging in the U.S. and expect the potential approval in 2027. And sitting with $266 million in cash, we'll capitalize to achieve all of those things.

Okay, great. So like you said, you're developing dental imaging. And the indication is BCG-unresponsive, non-muscle invasive bladder cancer with cysts. So maybe can you tell us a little bit about the burden of this disease, a little bit on the epidemiology, how many patients are diagnosed, how they're managed, and really what the unmet need is that you're looking to address?

I was shocked when this opportunity presented myself. The treatment in bladder cancer, actually, particularly non-muscle-based bladder cancer, is pretty poor. The definitive treatment was removal of your bladder. So the radical susectomy. And then when I learned more about radical susectomy, four to six-hour surgery, multi-organ, five to 15% mortality, and horrific morbidity as well. You know, individuals landing with an ostomy, loss of sexual function. So treatment's not very good, right? Now, when you think about the epi, there are about 90,000 in the U.S. You know, the instant population, about 90,000 individuals have bladder cancer. 80% of those are non-muscle invasive bladder cancer. And then within that, you know, it's estimated somewhere between 20,000 to 40,000 individuals each year have non-muscle and basal bladder cancer that is resistant to BCG. So it's a pretty significant patient population. This is the number six cancer in the United States. So it is a relevant cancer. And the FDA put out guidance to, say, if you did a study, about 100 patients or so open label that, in fact, you can get an approval. So we're actually at the dawn of something very special and exciting in the management of bladder cancer.

Okay, great. So in recent years, you know, we've gone from having very few therapeutic options to a handful, spanning different mechanisms, and practice is still evolving. But what we've seen so far as to how urologists are adopting these therapies, you know, how are they adopting kind of these newer options that are being made available to them?

Again, I think we're at the early stages of these new agents being available. And part of the challenge is when you look at the market overall, let's call it 20% to 25% of the patients are in academic institutions. And let's call it 75%, 80% are in the community. The newer agents, while very good, come with some challenges. And they come with challenges in that they require multiple pre-washes, the intensity is very high, it requires post-treatment activity, it requires infrastructure, and they just don't slide easily into a urology practice. Those practices have a lot of resource. Academics have adopted them quite significantly. I would say that the community area is almost barren. So if you think about community or all this right now, what do they have? They have BCG, if they can get it, right? And then they have gemcitabine, which they can use, but there's some questions about efficacy, and it's not really financially viable. And then of the new agents, gemcitabine on the pretzel may or may not be viable. So there's just not a lot of choices for these community urologists, and that's where dental imaging can really make a big difference.

So with these dynamics in mind and clearly kind of different dynamics between academic centers and community urologists, is there an estimate of how big you think the non-muscle invasive bladder cancer market could be? I think in the past you pointed to the multiple myeloma market. That's maybe a relevant precedent. but what does history in that therapeutic area kind of tell you where NMIBC might be able to go?

I think it's a fantastic analogy, and I think it's something that Wall Street needs to really start to pay attention to. With the advent of new agents, what you get is patients avoiding that horrible removal of their blood. Nobody wants to have an organ removed, right? And they will take, my market research says, right now, they'll take three or four lines of therapies at least, right? That's a big change. So what does that do? That starts to boost the prevalent population. So let's go back to the analogy of multiple myeloma. When Revlimid was launched, multiple myeloma is a billion-dollar market. Right now, if you look at multiple myeloma with over a dozen new agents, it's a $20 billion market. and we're now working to a point of where it's almost a functional cure. What an advance for these patients. Well, I'm hoping we'd have the same sort of advance for patients here in bladder cancer. Right now, the agents that we have are useful, but some of them are difficult to use. But every single one of these agents, if you look at the durability of the products, they range between 40% to 50%. So what does that mean? At the end of a year, half the patients need another medicine. So medicines like dental imaging can make a really big difference. Okay, great.

Okay, diving a little deeper into dental imaging, can you tell us a little bit more about the asset, the construct and delivery vehicle, and can you touch a little bit more on your DDX platform and how it's factored into dental imaging?

So dental imaging is quite simple but quite elegant, how it's put together, because actually it starts off with the simple generic plasmid, We have two RIG-I genes and IL-12. Then we take, and this is where the platform comes from, the secret sauce is our proprietary sugar, DDX. They are mixed together in an in-line manner, and then a proprietary pegylation occurs to create these nanoparticles. What's fascinating about dental imaging is that all of the ingredients that I just described to you are readily available or inexpensive, and we're able to put them together in a proprietary manner. The result is we have a product that we've already gone through our FD validation matches. We're manufacturing at scale. So this is a product that is easy to handle, can be stored in a regular fridge or a regular freezer, but we should have the lowest cost of goods of all the immunotherapies.

Okay, great. And you've reported a good amount of data from your pivotal cohort in the Legend study thus far. Maybe just from a high level, what have been key takeaways from the interim data that we've seen so far that you've highlighted?

Yeah, I think we're excited to show that Datal Imaging is an active and useful product. The primary endpoint for approval in this category is complete response rate at any time. Ours is 54%, which is in the range of approvable products. The next thing the FDA looks at is safety and tolerability. So being a non-biotic gene product, quite safe. It's described sitting in your refrigerator. But from a tolerability standpoint, we seem to have one of the lowest levels of AEs. And in particular, if I draw your attention to treatment interruptions or treatment discontinuation, a real measure of our people taking the medicine, ours is 2.4%, very low. So we're trending towards best-in-class handling, best-in-class tolerability. Also below that, what was interesting is that over 90% of the patients responded within the first three months, so they responded very quickly. If they didn't respond, less than 3% progressed, so very little risk of using the product early, and you'll know very quickly if it works. And what we're waiting for is our data to mature from a durability standpoint. We have very early 12-month data, 12-month landmark data, and we have very early durability, the percentage of patients that are durable 12 months and beyond, and that's what we'll get later this year.

Okay, great. So speaking of that, you've got it to that 12-month data later this year, and it sounds like the L.A. submission could come after that. So what are you hoping to see in that update? What profile do you think would support regulatory approval in that update?

Well, you think about what does the FDA look at? The FDA first looks at the risk benefits in any category, right? So what's the risk? For us, first of all, as I said to you, trending towards best-in-class tolerability, trending towards best-in-class safety and handling, and the risk is this is a new approach, a new platform. The FDA likes to have, they've approved four different agents, types of agents. This would be totally different. So that's on the risk part, which I would say relatively low. Then the primary endpoint is CR anytime. That's heavily weighted. Ours is 54% in line with the other products.

Now what the FDA is looking for is some durability data.

So, you know, because if you have a 54% CRN time and you have three months of durability, it's not very useful. But if you have something, you know, like the majority of the patients approach a year, you know, that gets competitive. So we'll have that data, 12-month landmark data in the second half of the year. Not as much of the 12-month durability data. That package will go in together, and that will form our planned BLA initiation.

Okay. And I think you've said you have a planned FDA interaction later this year, so ahead of the BLA. So is there any insight you can give us into maybe key points you'll be looking to discuss with the agency in that meeting? And then, again, I guess just how much of this 12-month data do you think you'll have or do you think would be helpful to have for the meeting?

Yeah, so the meeting itself, you know, you covered two topics. You covered manufacturing and clinical. So we, the FDA has given us CDRP, which is a special program for manufacturing, so we're always in dialogue with them. But we will go and ask them a couple of questions about are manufacturing is sufficient. And then on the clinical stuff, we will, it's pretty simple. So does the FDA believe we have sufficient enough data to initiate the filing? And I think at that time we feel pretty confident that we'll have a sufficient amount of data to be able to do that.

And I think you've talked about alignment with FDA on the SAP to potentially exclude some patients for the efficacy analysis. So can you remind us of the context for that discussion and give us a sense of the range of potential outcomes on exclusions? Are there certain patients you're fairly confident could be excluded?

Where does that discussion stand? Yeah, so with any pivotal program, you have a discussion with the FDA on the statistical announcements, quite normal. If you see within some of the other products, they have a publication number with X, right, and in their label, why? This is the process that we're under. We've made a proposal to the FDA. The FDA has come back to us. So we're in that back and forth period. I think our upcoming pre-BLA meeting will help clarify that further, but we would expect that our final label will have less than the 125 patients that we've enrolled. And, again, the 125 patients makes it one of the largest programs. So to lose some patients is not going to have much of an impact.

That's helpful. And then maybe just one more on the regulatory front, and we've discussed this before, but with the evolving treatment landscape here and organizational changes at FDA, there's more and more talk about single-arm trials, and maybe the sentiment is that FDA is more amenable to those these days versus maybe a few months ago. But I guess just any thoughts on kind of trial design and how, you know, the evolving nature of staffing at the agency could impact receptivity.

Yeah, I actually, our interaction with the FDA has been nothing short of phenomenal, right? You know, so what do I look at? It's not that long ago when Lexo was improved, right? So the path is still there. The second thing is if you look at other companies that are coming into the space, they've been very clear with them that their studies are not second-line studies. They're third-line. So ours is BCG, then our product. Any new product is BCG, a new product, and a third product. So they've been pretty clear with that. I guess then the third thing is I look at our level of engagement, and the FDA does not hand out very easily things like RMAT designations, CDRP. We've had the same project managers. if we've had the exact same clinical reviewer, I feel pretty confident that if we deliver the package that we're planning on delivering, we're in very good shape. Okay, great.

And maybe just transitioning to the potential commercial launch for Dental Imogene. You and the team have done a lot of interesting work to understand the market and the unmet need here. So if we think about Dental Imogene's unique profile, how is it positioned relative to other treatments, and how does that factor into your launch strategy?

Well, I think you have to step back at the market, first of all, right? So the market is, let's call it 25% academic, 75% community. Dental imaging was designed for community urologists. And, in fact, when you survey, do market research for community urologists, what do they say they want? They say, well, we want something that's efficacious, that our patients will take, that's tolerable, And that's easy to slide into our practice. That is the dental imaging profile. With a 54% CRA at any time, it shows great efficacy. With a very low treatment interruption and discontinuation rate, patients will stay on the medicine. And, in fact, dental imaging is probably even easier than BCG to integrate into the practice, given that it'll sit in the freezer for what we expect to be years and in a regular fridge for many months. It's not going to require much resource from a staffing perspective because with deadalimaging on its own, there are no pre-washes. You don't need to have urologists administer it. It needs to be a medical professional, reach it in the freezer, mix it, and still send the patient home. So that, to me, is pretty a compelling proposition for those community urologists who have very few options.

Okay, great. So maybe just diving a little bit deeper into that, Is there further segmentation you'd highlight between kind of academic and community practices? Are there different levels of receptivity to dental imaging versus, you know, maybe a more onerous treatment in the work that you've done? Yes.

So our market research shows that there's a unique position for dental imaging. So if you, you know, it's easy to characterize all urologists to say, right. Then you can say, okay, let's split them by academics and community. And that still is still fairly simple as well. Within the community, we can go down even further to find segments that are particularly attracted to dental imaging. So there are practices. So if you look at it from a numbers perspective, that's 25%, 20% to 25% academic, 75% to 80% community. And within that, we've identified over 40% of the practices have unique attributes that dental imaging would actually solve a big problem with us. And these are resource-constrained practice, economically-focused practices, where the profile of dental imaging differentially.

That's helpful. And then just thinking about efficacy as these urologists are considering their profile here, we've broadly talked about CR rate at any time and durability. You know, how are those data points weighed in different practice settings so that urologist thinks differently about those endpoints?

Yeah, marker research is pretty clear. CR anytime is in the land of community urologists. Does this product work or not? And with no disrespect to any community urologist, the 12-month number for them, they likely will not be focused on. Because remember, they're treating a wide range of patients, right? Does the product work or not? CR any time. For the academics, they're much more focused in on patients that are very experienced. They, in general, would almost want to prefer to remove the bladder. The patient doesn't want to remove the bladder. They're very focused in on durability and the highest efficacy possible because they also have the benefit of resources. So where we see dental imaging being used with the community is very early in the treatment because, as I indicated before, it works fast. If it doesn't work, there's very little damage, right? You can continue on to something else, and quite frankly, our early science, so pretty good efficacy and pretty good durability. Where we talk to the academics, their use would be more focused on the fact that it's non-viral. So they're juggling different types of chemo, immunotherapy, having a non-viral approach, you know, for a patient. So we'd probably receive more third- or fourth-line usage there.

And then one more just on kind of the breakdown of the population here. So it sounds like about 40% of those community practices would potentially be, you know, rapid or excited adopters of dental imaging. Can you give us an idea of how many patients are seen in that subset of practices? Is it kind of one-for-one in terms of practice?

Yeah, that is the patient number. So if you think about it, 75% of the practice are community. Within that, dental imaging could be attracted to all 75%. Where 40% of the patients are, there are differential attributes of dental imaging that unlock that practice that other products just cannot do. So that is a real differentiating point where something special like dental imaging can make a big difference.

And you touched on it. It sounds like dependent on practice setting and the patient, obviously, you know, where dental imaging could slot in terms of treatment sequencing might be different, but I guess is there an overall profile you're thinking about? Will this also depend on where the patient's treated?

Well, I think, like in multiplying myeloma, these doctors now have the opportunity to have a real dialogue with their patients, and it'll be very practice-specific and patient-specific. So the combination will be something along the lines of, you know, I'm sorry, Judah, your BCG is not working. I've got two or three. Here are the pros and cons of these agents, right? You have to come in every six weeks, and if it doesn't work, it's another six weeks. intense therapy. I'm going to insert a device into your bladder. It's here every three weeks. You have to drink a liter and a half every day. You're going to feel that. Or I can start you with something like that, a limo gene. We're going to know within three months whether it's working. You come in week one and two, week five and six. It's very well tolerated and I can start you right now. So I think that's where the benefit of dental imaging really differentiates itself.

Do you get the sense, I guess, kind of beyond burden on the patient, which it sounds like is a large consideration here, are there certain urologists considering mechanism and therapeutic mechanism when making these sequencing decisions? And what is that thought process that's evolving now?

I think that's more in the realm of the academics. Yes, so the academics say, because remember, let's step back a little bit. This is a disease, it's cancer, it's a very serious cancer. However, it progresses at about 20% over 10 years. So it's a slow cancer. It's also a cancer that's affecting the average age is around 70, 75 years old. So these are people that are getting a little older, right, and they have comorbidities, generally they're smokers, right? So the treatment choices and objectives are different, right? How can we control this bladder cancer so the patient may pass from something else, right? And so for the academics, I think they like to go immunotherapy, chemo, immunotherapy, chemo. And if one of these products is not working, then I think the mechanism makes a big difference. Non-viral gene therapy is something different. It's less important for the community. The community is more driven about what can I get my hands on? what will my practice administrator use, what fits into the flow. And in both cases, dental imaging can be a useful product.

And you mentioned, you think, kind of two, three, maybe more lines of sequencing before radical susectomy. I guess in your payer conversations, you know, what's the reaction on that side of the table in terms of potentially limiting the degree of sequencing? You know, how are you thinking about how many lines payers are kind of willing to accept?

Yeah, market research says probably more than three or four lines of therapy. given that a radical susceptibility is such a severe outcome. And in our payer research, if you have NCCN guidelines, in general you're going to be reimbursed. So this is not a large barrier to update for dental imaging.

And I guess speaking of the competitive dynamic here in the sequencing, what are your latest thoughts on pricing? Can you remind us of kind of the relevant benchmarks that are going to be valid for dental imaging. And in those payer conversations, do you have any insight into where pricing could head?

So the range in pricing for these products is $220,000 to about $700,000 a year. As we've had dialogue with payers, our payer research goes back to what I said before. It's cancer, it's serious. it's on the NCCN guidelines it's generally going to get reimbursed and what's interesting that it kind of changes the picture a little bit because if the new benchmark pricing $700,000 that's 2,000 patients that you need to hit a billion dollars which when I talk to you about incidents of 20,000 to 40,000 patients per year there's lots of room for lots of agents that makes sense And as you think about a potential launch, can you give us a sense of the level of investment required to build out commercial infrastructure?

What could a sales footprint look like? What are the particular call points that would maybe be first to tackle as well?

Well, what's great about this is it's quite viable for a company like ours to launch the product. We'll need about 40 to 60 sales reps as a proxy. You know, when you think about these community practices, these lug pus, say there's 30 doctors, 30 urologists, three or four are focusing on bladder cancer. So we're able to target those individuals, you know, with that. So very much well within the abilities of a company like this.

Okay, great. And then I just wanted to touch on the surfactant bladder rents cohort that you recently initiated. So what was the impetus behind that, and how might it enhance dental imaging's profile? And, you know, I guess at what point in the, you know, clinical or commercial kind of launch of the drug would this factor in?

So when we designed dental imaging, we designed it for community urologists. Unlike other products, no pre-washes, very few installations, and no after-treatment activities needed for high-risk NMI-VC. We'd always thought of bringing forth a surfactant bladder rinse for other indications as well. When you look at the other gene therapies in their preclinical data, they get very little transfection without a bladder rinse. We actually, with that limogen, without a bladder rinse, we do get a pretty significant amount of transfection. You see that relates into pretty good clinical efficacy. In our preclinical models, we see IL-12 expression going up as much as 10 times with a pretty mild surfactant called polydocanol. So we selected polydocanol. It's a proof drug. It's got a lot of safety information. It's actually used at up to 1% in a lot of shampoos. And what we've done is to still, again, we're always trying to make our drug more efficacious and simpler. What we've done is we've reduced the dwell time from an hour to half an hour. Now, remember, these are generally incontinent gentlemen, right, with bladders that are beaten up. Hard to hold it in. It'll be easier to hold it in. And very simply, what occurs is you put polydokinol in, let it sit for five minutes with the catheter, take it out, put dental imaging in, and you tell the patient to keep it in for half an hour. So the net of that is the patient actually stays 25 minutes. So we expect to have better convenience for the patients, better experience for them, and we expect to have better durability, efficacy and durability given what the preclinical data is. We're already up and going with the cohort. We've just gotten through the safety run-in, so we will have data very soon, and this has been an important part of the urologist armamentarium.

Okay, great. We'll look forward to that. In the last couple of minutes, you may remember from last year, we do kind of a mini-survey that we asked all of our companies, He's touching on kind of a few topical areas that are of interest to biotech investors these days. So first, with China's rise in biotech innovation, how are you thinking about your competitive position here? And could this influence your R&D or business development strategy?

Well, where has China gone? China used to be a place to go for manufacturing things very quickly and inexpensively. And now they've just moved up the curve on innovation. I think for us, you know, specifically, as it relates to dental imaging, it has relatively little impact for us because we're so far along the line for commercialization. But as we consider business development and building energy that's available in China, it becomes very interesting. So, you know, I really, you know, at the end of the day, we're here to get new medicines to help patients. Eastern China is really good here in the United States and around the world.

Would you say NGIN is leveraging AI in a particular way or thinking about the potential for AI to disrupt the space that you're in?

I would say in the early part of it. When you think about AI, AI in our industry really started it in the discovery part, right, in identifying compounds. We've already developed a platform. We've already translated the platform. So now the AI is probably more about how do we commercialize. And, again, there's a lot of interesting agents to help our commercialization. And particularly a small company like ours, AI actually allows us to probably punch a little higher than previously. So I'm kind of excited about some of the work the team's been doing. Okay, great.

And, Les, we touched on it, but on the regulatory side, It sounds like FDA has been relatively stable in your interactions. But anything else, whether it's at the agency, on pricing, maybe MFN, tariffs, anything that you're thinking about on the regulatory side that kind of keeps you up at night?

I would say, first of all, on the FDA, you're always wondering because things can change. But I would say even in the stormiest times, our stuff has been absolutely boring, which is fantastic. Boring is good. So tariffs are, again, something to think about. But, again, as a company that's not commercialized, if not, it's still around the edges, right? Unfortunately, as we commercialize, it's something we have to think about. And MFN puts, you know, that's the one part where you think about a product like that, a limogene, really is an attractive drug for the globe, easy to handle, right? So MFN is something that we have to keep one eye open and just see how that evolves over time.

Okay, great. With that, we're out of time, and thank you again for the time today, Ron. Good, great to see you.

I appreciate it.