enGene Therapeutics Inc. (ENGN) September 2026 Conference Transcript

Welcome to H.C. Wainwright's 20th Annual Global Investment Conference. I am Ananda Ghosh, Senior Biotech Analyst at Wainwright. Today we are very pleased to host Ron Cooper, CEO of NG Therapeutics for our company presentation series. Ron, welcome.

Well, thank you very much for the opportunity to be here. This is a particularly special time for NG Therapeutics. you're catching us at a moment of where the company's going to transition very quickly. So I'm looking forward to sharing that information with you today. Now, as you'd expect, I'm going to be making some forward-looking statements during today's presentation. Let me begin with just a large overview. So first of all, our lead clinical asset, dental imaging, we consider this to be a next-gen therapy for NMIBC. There's a couple points I'd like to make, first of all, before going into the presentation. The first point is I think it's underappreciated the unmet medical need, particularly with community urologists, and that the advent of new agents is not going to be something where one agent is going to win. But in fact, as we talk to urologists, Sequencing is going to become even more prevalent. And that population, the TAM, is going to increase. Therefore, the market is going to get larger. What we're excited about with Dettolimagine is we have a clinical profile that is emerging quite nicely. With a 54% complete response rate at any time, this is in line with already approved products. And with a tolerability profile that's trending towards best in class, we think that dental imaging can be an important part of sequencing and managing NMIBC patients. Now, an attribute that's really important and often overlooked when it comes to community urologists is how products fit into their practice flow. And the beauty of Dettolimagine is that it is stored in either a regular freezer or refrigerator. It is easily transported, and it fits so nicely into the practice setting. Now, with a 54% CR anytime, as we said, we're in the range of the approved products. But we've always, at NGene, looked to see how could we make dental imaging even better and even more convenient for patients. So we're delighted that we just recently have completed the safety run-in of a new cohort of patients combining dental imaging with a surfactant bladder rinse. and that has the potential to not only improve efficacy and durability, but reduce dwell time from 60 minutes to 30 minutes. So the net would be a better efficacious product, but also a product that's more convenient for patients. And as I said at the very beginning, you're catching us at really an exciting time. We're sitting with $266 million in cash, but in the next quarter, we plan an update on our pivotal data set where we'll share some more of our data that's maturing, particularly from a durability standpoint. We've said that we have a meeting with the FDA, a pre-BLA meeting, and we plan to initiate the filing by the end of this year. So as I said before, you're catching us at a particularly special time for NG. Now, why are we excited about a dead-of-limmon gene and what's exciting about this non-viral approach. It actually addresses some of the shortcomings of the viral gene therapies in that we can deliver, in this case, three big genes. We can re-dose the product. And because we use four readily available ingredients, the cost of goods for our product is actually one of the lowest. And being non-viral, It also reduces the cost of handling and the like. Let me spend a few minutes talking a little bit about NMIBC. First of all, non-muscle invasive bladder cancer, right? So NMIBC represents the largest majority of bladder cancer diagnosis. Bladder cancer is the sixth biggest cancer in the United States, and you see when you get down to the funnel, the actual incident population is about 20,000. and some say as high as 40,000. NMIBC is characterized by cysts, carcinoma in situ, which is all over the bladder, so you can't cut those out. You need to use intravascular therapy to get to that. And patients may or may not have growth tumors, papillary tumors, which you see here. Now, in general, these patients present with urine in their, their, sorry, blood in their urine, blood in their urine. And the one thing, while this is cancer, and it's a very serious cancer, it is a slow progressing cancer, about a 20% progression over a 10-year time frame. So the treatment consideration is different. And also when you consider the average patient age is around the mid-70s, right? So what usually occurs is these patients are started on BCG. Many of you are aware that BCG has had a chronic shortage. So sometimes difficult to get BCG, but it is an effective therapy. However, after a couple of years, you know, there's about a 50% recurrence. Standard of care right now is to use some of the new therapies or IV chemo. And there's some limitations with all of the agents that are there we'll talk about shortly. The ultimate treatment for bladder NMIBC is actually, when I say ultimate, 100% CR is removal of the bladder. It sounds simple, but this is a very long surgery with very high mortality, very high morbidity.

So patients in general want to avoid this.

So then you say, well, where are these patients? So when you look at across the U.S., the majority of the patients are in the community, about 75 to 80% of that. And the treatment considerations in the community are different from academic centers. Where you see here from this market research, what are these community urologists looking for? They're looking for the perfect balance of tolerability, efficacy, and ease of use, how the product moves into their practice flow. And now what's interesting as well, when you look across this community group, there's even a subgroup of the community where I would say about 40% of them through our market research, where they have a characteristic of where the profile of dental imaging is uniquely differentiated. So what is happening in these practices? They have limited resources. They have workflow challenges. They have economic challenges. And the unique aspects of dental imaging, which I'll talk about shortly, fit in very, very, very nicely. So let's give a little background. What is dental imaging? So dental imaging, as I said, is a non-viral gene therapy. What we do is we take a simple generic plasmid. We put two RIG-I genes and IL-12 in that. That is the lyophilized powder, which is mixed with water and instilled in the bladder, which you see here in the last graph. What's beautiful about dental imaging, though, is that it has both an impact on the innate and adaptive immune systems. So it's a one-two punch. And as I was talking to you earlier about our components in manufacturing, how is dental imaging made? We take that plasmid. The secret sauce is our proprietary sugar, the DDX. In line, mix them, throw a little pegylation on top of that to create nanoparticles. Now, why is this good? Well, first of all, this is a product that is very stable and easy to store and transport. It will probably sit, once we complete our discussions with the FDA, for years in just a regular freezer, the same one you have at home, and for many months in the fridge. So a clinician or someone in their practice can just reach into the fridge, mix with water, and instill with patients. And I'm delighted that we've already completed our FDA validation batches we're manufacturing at scale. And since we have the CDRP program with the FDA, we have a lot of dialogue, and we feel pretty confident about our following from that perspective. So what is the design of dental imaging? It's designed to be easy for patients and easy for the practice. Easy for patients from what perspective? Very few doses. We give the drug week one and two, week five and six. Very little time in the office. and then there's nothing that occurs afterwards in terms of extra precautions like urine bleaching or or the like for the practice as i said most practice have a fridge or a freezer they you know many of them don't have a ultra cold chain storage uh freezer it can be administered by anyone any qualified medical professional and as i said there's no decontamination needed within the practice no hoods required you know or or the like so if you summarize this dental limiting is the only product right that does not have complex shipping or storage that has a short administration time frame that does not have any bacterial or viral decontamination that doesn't require any post-treatment action and can be administered by by anybody in in the practice itself. So where are we studying dental imaging? Our clinical trial is the LEGEND program. Cohort one is with BCG, unresponsive patients with cysts, one of the bigger programs, 125 patients. And we have some other cohorts as well. But let me go a little bit deeper into cohort one, which is dental imaging without a surfactant. Again, it's BCG high-risk patients, a global study that we put 125 patients on primary endpoint CR anytime. If you look at the baseline characteristics, they're pretty typical for these patients, mostly male, you know, average age in the 70s. But please note a couple of things here. We actually have a patient population that's highly experienced. You know, almost 40% of the patients have cyst plus a papillary disease. And even though our our study is designed for bcg and then dental imaging we have a high percentage of patients 25 of the patients that have had a uh a prior treatment now at the recent american uh urological associations uh meeting we shared preliminary data and what you see here is a 54 complete response time again in line with other with other with other products and we provided some updates on on where we were from a 12-month landmark perspective. And you can see here at 13.3%, that seems to be relatively low. However, you see in the bottom there, that has the whole denominator. It does not have all the numerator. And we have 19 patients that are complete responders and potentially two more that are progressing. And so if you look at the Kaplan-Meier curve, we anticipate a 12-month landmark CR of about 24, 25, 25%. So when you think about your approvability, though, and if you think about what the FDA is looking at, first of all, they're looking at the primary endpoint. Currently, we're at 54%, which is in the range of the other products, where there's some confusion on the screen is what is the key secondary endpoint for durability and what it is is a 54 of those patients what percentage of those patients are still responding 12 months and beyond and you can see here that's roughly 40 to 50 percent about half the patients so we're waiting for our data to mature and you know we would expect to be within fda requirements for that. One number that the market is quite focused on is this 12-month CRA, which I will remind the individual is not a number that is an endpoint. It's not a promotable number. But later this year, we will be providing you more information in regards to that. And as I said, right now, our Kaplan-Meier curve has us looking very much like an stellardry, you know, type profile for that parameter. Tolerability is absolutely excellent. You see here a pretty low risk of treatment-related AEs, 55%. Put it in context of the approved products and the products in development, that is really competitive. I would draw your attention, though, to the real measure of how well-tolerated the product is. Dose interruptions and dose discontinuations are very low, 2.4%, and that appears to be trending towards best-in-class. So we would consider ourselves treading towards best in class tolerability. So the package that we say for dental imaging is efficacy that is in class, potential best in class tolerability, best in class ease of use and handling. But we said to ourselves, we've always wanted to make sure that we could do everything we can to make dental imaging the drug for community urologists how could we make it even more effective and even more convenient and we've considered using a a surfactant bladder rinse for dental imaging so as i said before dental imaging on its own has really a a compelling profile if we add a surfactant in what would that look like from a tolerability perspective, and can we improve efficacy? Now, if you look at the other viral gene therapies that you see in this slide, in the top, you see the usage without a surfactant. What you see is either no transfection or very little transfection. You add a surfactant, and you see in green there or in the staining that that really does boost transfection. What does that look like from dental imaging. Well, you see in the top left that dental imaging, you actually see pretty good expression indicated by the colors there. But if you add a surfactant, you see an increase in both depth and breadth. And if you look to the right there, that's a 10 times increase in IL-12 expression. Now that is in the mouse model. You said, well, any other models? Well, here in the nhp what you see again a nine times increase in expression but also an improvement in distribution so we believe that this could make a material impact on the efficacy of of of the product so then the next question is tolerability and from this chart here you see that there is very little systemic exposure with the surfactant that we're using we've selected a surfactant polydocanol. The reason we've selected that is it's widely studied. It's an approved product, has a big safety database, and it's actually used in shampoos at 1%. We're using a quarter of the dose. So as I said, we're up and going with another cohort of patients. And the way that it is administered is the patient is catheterized. You put polydocanol in for five minutes, take polydocal, I'll put that a lima gene in, and instead of having a dual time of an hour, it's a dual time of half an hour. Net saving of 25 minutes, you know, approximately for the patient or for the practice. So that's a material convenience saving. And as I said, the data would suggest an improvement in IL-12 expression, which should translate into, you know, greater efficacy, you know, over time. And as I said, we've just finished the safety run-in for these patients. We're actively enrolling sites, and we look forward to updating the market as we have more data. So let's close off by talking about how we see this NMIBC market evolve. It's a very exciting time, you know, for NMIBC patients and for the providers, because Finally, there are going to be options. And I think the way that we think about this is our market research first says that patients will take three to four, if not more, lines of therapy, sequencing products. And when you survey patients, you know, the vast majority, 90% of them, do not want to have an organ removal, a radical cystectomy. And so what we see is that right now, many of the newer products are being used in the academic centers because they're a little bit more difficult to use, as I described before. Whereas if you look at the community where 70% to 80% of the patients are, right now they get BCG. We see dental imaging being used right after BCG because A, efficacy, well-tolerated, slides into the practice. But also, you will know usually within three months, 90% of the patients respond at that time. And if you don't respond, only 3% of the patients progress. So it is really ideal at that pace. And we anticipate three or four lines of therapy at the community and then going on to the academics. In the bottom right, you see an illustration of what we mean about what's going to happen with building this prevalent population. So, you know, it's not one drug that's going to win. Multiple drugs are going to win. So if you think in year one there's 10,000 second-line patients after BCG, about half of those from the durability data that I talked about before will recur, right? And then they'll need a second line, they'll need another second line patient. And then some of those patients will pop in at year two, but you can see how you're creating a compounding effect of more patients in the prevalence pool, therefore, you know, a bigger market, a bigger market overall. And I think it's pretty exciting what's happening within the marketplace. And I do believe that payers are recognizing that these new technologies can make a really big, big difference, right? And from our market research, payers have said to us that, look, if you have NCCN guidelines, you're generally going to be, you know, covered. Because it's, seriously, it's cancer. You do see, though, that over time, the pricing benchmark has changed from roughly $250,000 per patient a year to closer to $700,000. And just doing simple math, to achieve a billion dollars of sales, you need roughly about 2,000 patients. And so getting back to the treatment available patient population, you see here in the high risk BCG on responsive and exposed patient, that's going to be around 40,000 to 75,000 patients, naive patients, incremental on that, intermediate a little bit bigger, but this total patient population is pretty significant. And as I've indicated earlier, with these new agents and with sequencing, this market is going to get bigger. So let me close off by saying, first of all, we have an incredible team here at Edgy, and this team is really dedicated to making sure that new medicines get to the marketplace. And they've done it multiple times before and have commercialized products before. So we're pretty confident this team can get the job done. And then, you know, just to close up here, what I'd like you to walk away with is that, first of all, this NMIBC market is going to look a lot different in the next five years. And the analogy I like to use is the multiple myeloma market. When Revlimid was launched, that was a $1 billion market at that point. You add over a dozen agents, a $20 billion market, because patients are receiving multiple lines of therapy and sequencing so this is a large market now it's only going to get bigger with these with these new agents not one product is going to win what's exciting about dental imaging is what community urologists tell us that they want is they want the right balance of efficacy tolerability and ease of use and dental imaging is unique it is the only product that balances all three highly differentiated product and then on top of that with the surfactant like other people have used we have the potential to improve efficacy and make it even more convenient so a good data limiting perhaps could get even better with the use of surfactant and then closing you know this next couple of quarters are pretty exciting quarters for the company we will provide an update in the on the data uh in q4 of this year we have a pre-bla meeting with the fda and we would like to report on that our intention is to initiate our filing by the end of the year we expect an approval uh for dental imaging on platform designation in 2027 you're wrapping around we're sitting with 266 of cash enough cash to get us through all those important milestones So this is really an exciting time to be part of the NG journey. And we're at a point of where a lot is going to happen with dental imaging. So I thank you all for your attention. I'm happy to take any questions if there's a bit of time.