Great, thank you for your patience. Welcome back to another session here at Oppenheimer's 36th Annual Healthcare Life Sciences Conference. I'm Leland Gershell, one of the analysts with the biotech research team here at OpCo, and really delighted to have with us as our next presenting company, enGene. enGene is a public company. The ticker is ENGN. We've been covering enGene, and like the story, we have an outperform rating on the stock. On behalf of the company, CEO Ron Cooper will be joining us to run through a slide presentation, and if we have some time at the end, we'll ask some questions. Please feel free to post those through the portal if you want us to weave them in. Without further ado, I'll turn the microphone over to Ron.
Well, great! Well, Leland Gershell, first of all, great to see you, and thank you for this opportunity. Hello, everybody, and for those of you that are not familiar with enGene, boy, this is an exciting time, you know, to learn more about our company. I'd call 2026 the penultimate year. First thing you should know, though, about enGene is that our focus is in a very unique area, and that is non-viral genetic medicines, and I'll talk more about that in a few moments. As you expect, I'm gonna make a few forward-looking statements during today's discussion. As first as an overview, you know, what is enGene about? Well, it's really about our lead product called detalimogene voraplasmid that we're developing for NMIBC, or non-muscle invasive bladder cancer.
We believe this is an exciting new advance in the space. First of all, the space is exciting. We believe actually that this is a market that's going to explode. A market that's gonna explode because the, you know, proliferation of new agents are gonna make it a lot better for patients and a lot better for the practicing physicians. We think detalimogene being a novel class of gene therapy that has, you know, an effect in two different axes of activity can make a big difference. We also believe that what clinicians are looking for are products that are efficacious, well-tolerated, and just slide into their practices, and that's the profile of detalimogene. As I said before, this is the penultimate year for the company and for detalimogene.
You know, this year we plan to provide a data update in the second half of the year on our pivotal trial. We plan to file the BLA by the end of the year, and we expect a potential launch in 2027, so an exciting time, you know, for detalimogene and for enGene. I talked about this non-viral gene therapy, what's great about non-viral gene therapies? When you think about gene therapies, what is the problem with it? Small packet size. You only put so much genetic material. We're not limited in the amount of genetic material, so we have a large cargo. Usually, gene therapies are one and done because they're immunogenic. We are not. We redose detalimogene. The third thing, is gene therapies are often very expensive to make, and they're very difficult to handle.
In this case, detalimogene being non-viral, and using simple ingredients that are readily available in the market, no special handling, you know, we should have a very competitive cost of goods, you know, for detalimogene. A little bit about NMIBC. Bladder cancer is a pretty significant disease in the U.S. As you see here, you know, over 740,000 patients. If you look at NMIBC, 27,500. Yeah, NMI, high-risk NMIBC. NMIBC is the majority of patients. How does it present? You see here on the right, where you have what are called, you know, CIS or carcinoma in situ.
These are sort of like plaque-like lesions that are all around the bladder. You can also have growths, and growths can be Ta growth or T1 growth. T1 growths are actually the most serious. The combination of CIS plus T1 can be actually very, very serious. What happens to these patients now, in general? They are diagnosed at the community urologist. They're given a product called BCG, that many of you are aware has been, you know, in back order for over a decade now. It's a product that if you give more of it becomes a bit more toxic.
The options are either to go to newer therapies, and some of those have been clunky to use, or you go to IV chemo, and again, you know, this is a product that has, you know, limited data and limited efficacy. Ultimately, patients are having their bladders removed, and that has a high level of mortality and morbidity. You can see here, 5%-15% mortality. It's a very big surgery, long surgery, but individuals, you know, land up with an ostomy, land up removal of their prostate. This is really tough on patients, both from a mortality and morbidity perspective. NMIBC, you know, I think one of the things you need to think about is it's a little bit different than other cancers. First of all, it's a slow cancer, right? It's recurrent.
It happens, continues to recur, but doesn't progress that quickly. About 20% progression from NMIBC to muscle-invasive over the 10-year timeframe. It's a big cancer. It's one of the top 10, and it's a very expensive cancer, you know, to manage. You know, because of that, the FDA has put forward special guidance to say if you do about 100 patients open label, you will get an approval and a full approval, and you see there's been a number of agents that have been approved by this pathway. If you think about what a customer is really looking for? First of all, there's a misnomer that these patients are in academic centers. Majority of the patients are in the community.
As you see here, our data suggests that over 80% of the patients are in the community, and what are those community doctors looking for? It's not just one thing. It's a combination of tolerability, of efficacy, and all of the things that allow it to slide into their practice. When you look at detalimogene, that's just a terrific fit for that. The other thing that these practices are really struggling with are pressures from staff, shortages, revenue. For them, there's a real opportunity, you know, with buy and bill, generating more revenue for the practices. Those practices, you know, those products that require less expenses, fewer clinical resources are really attractive in a buy and bill circumstance. Getting back to detalimogene itself, you know, what exactly is detalimogene?
I said activation across two major axes before. You know, it stimulates both the innate and the adaptive pathways. You think about detalimogene, we create a nanoparticle. That nanoparticle has two RNA agonists, two RIG-I agonists that stimulate the innate system. We have IL-12, a well-known anticancer agent, you know, probably one that has a lot of side effects, but intravesically, we minimize that, and that stimulates the adaptive system you see on the right. Effectively, we put down detalimogene mixed in water, and it just bathes the bladder. When you think about detalimogene, you know, what do community urologists look for? They're looking for a great combination of efficacy, great tolerability, and safety.
I think right now, detalimogene fulfills their needs, and I'm gonna show you some data that really supports that. You know, starting off with, you know, can we make this product? I said, I'm pretty excited about manufacturing and the competitive advantage that we have. You know, as I said, simple, available ingredients. These ingredients are readily sourced. We're already manufacturing at scale, and in fact, you know, we're almost on our FDA validation batches. The FDA has also provided us a designation, CDRP. CDRP effectively is given to nine companies, one in nine companies, a pilot program, and it's to help us through the manufacturing process, so it de-risks the potential of a complete response letter.
It also helps us ensure that we're manufacturing at the commercial level. For us, manufacturing is a real competitive advantage. Now, clinically, where are we studying detalimogene? I would say, first off, you know, the key cohort for us is this pivotal cohort, which has been fully enrolled, in fact, over-enrolled, with 125 patients versus a target of 100 patients. We have a few other cohorts that we are enrolling, and these cohorts are more to provide information into providing information to the prescribing physician. Cohort 2A is BCG-naive patients, 2B are unexposed, then we have a cohort just for patients who have papillary disease. Of course, we continue to do some good work within our platform.
Let me tell you a little bit more about our pivotal study and share a little bit of data there. I think what we're excited about is it's a global study. It's fully enrolled, in fact, as I said, over-enrolled. Excuse me, popped ahead. Patients receive detalimogene. They receive detalimogene at weeks one and two, weeks five and six, rinse, repeat three more times that year, and then the following year, they go into maintenance phase, weeks one and two. That is, you know, that is a very low burden on the patients, a real competitive advantage for us. We're aligned with the FDA, primary endpoint, which they've used across all of these agents, so that at any time, Complete Response rate. As I said, we have enrolled 125 patients.
94 of those patients have come after some pretty significant protocol changes. 31 were pre-amendment. The analysis that we have, the final analysis, will be heavily driven by the patients in this post-protocol amendment group, and I'm gonna share an update that we provided late last year of 62 of those 94 patients. Baseline characteristics, pretty similar to what you would expect for this population, you know, mostly male, mostly older gentlemen. I think the big difference, though, is that, you know, this is probably a higher-risk patient population than some of the other datasets. A high % of patients have had another therapy other than BCG, and a very high % of the patients not only had CIS, but they also had papillary disease, Ta and T1.
When you look at these results, you say, "Wow, these are interesting results, particularly in the background of a highly experienced patient population." What we were delighted to share with the marketplace last year is that we achieved about a 62% six-month CR rate to 63% CR rate at any time. Both of these numbers are pretty similar to what some of the other agents have demonstrated. We're also excited that some of the patients that didn't respond to the three-month standpoint, four of those patients responded to six months, so this speaks to the immunotherapy. While we don't have much long-term data as yet, because the dataset has not been mature, but we're really encouraged that the first five patients that could be evaluated at the nine-month timeframe, all five were responders.
That was pretty exciting for us. If you compare this to the patients before we made protocol changes, you see here that that's a material change in Complete Response rate at the six-month timeframe. If you look at that in the context of some of the other products, you know, that have been approved or are in development, if you look at the CR rate, anytime, you know, most of these agents are around two-thirds, right? Again, we are in this range. Remember that these are all relatively small data sets. Ours would be one of the largest, about 125, but many of these data sets are 70 or 80 patients. If you then, if I point you to the six-month CR rate with these post-amendment patients at 62%, that compares very favorably to the other agents.
What we are seeing here is an emerging competitive efficacy profile. Now, what about tolerability? Again, most of the AEs are Grade one and Grade one and two, mostly associated with catheterization, a pretty low rate overall. I think what we're pretty excited about is do patients actually stay on this medicine? You see here, dose interruptions are very low. You see here, you know, less than 2%. Dose continuations are again, very low. If you compare this to some of the other approved agents or agents that are being studied, see the NE rate, CR rate is very competitive. I would draw your attention in particular to dose interruptions, dose discontinuations, where you see the dose interruptions, you know, can be, you know, as high, you know, as in the 40s, right?
Whereas ours are, you know, low, low single digits. Dose discontinuation, you know, not even 1% in other agents. You know, that occurs much more frequently. Emerging competitive efficacy profile, and really right now looking like a best-in-class tolerability profile. The second part is how does this fit into both the patient's life and the practice itself, right? From this slide here, you can see from a patient perspective, you know, even compared to BCG, right? Whereas BCG, if you've got to be reinduced, it's, you know, it's 12 weeks, and BCG is fewer doses. The chair time right now is an hour. That's in the clinical trial. We anticipate actually that patients will be given detalimogene. That'll take about five minutes, and they can void it at home.
There's no special bleaching or precautions. That allows them to avoid it at home. From a practice perspective, storage is gonna be terrific with detalimogene. We expect that in the freezer, it'll be in a regular freezer, it could stay there for years, and in a regular fridge, it's gonna be for many months. Doesn't require special rooms, doesn't require special nurses to deal with it, and there's no extra cleanup and decontamination. If you compare that again to the agents that are on the market and to come, we're the only agent that does not have complex shipping and storage, the only agent that doesn't have, you know, pre-wash, you know, that has. Sorry, this is the only agent that has this combination of ease of use, right? No complex shipping and storage. We don't have any pre-washes, right?
It's easy to administer. You don't have to do any decontamination of the room. When the patient leaves, there's no special precautions, and you don't need any special staff. If you look at the, what I've described here is an emerging competitive efficacy profile, an emerging best-in-class tolerability profile, and probably a profile that is, you know, best-in-class from a convenience and ease of use for the practice itself. How do we see the world changing? I think that when you look at these new agents, all these new agents are gonna be used. On the top right, you kind of see what I shared with you earlier, you know, that, you know, the practice was really one of being diagnosed, BCG, chemo, and some other agents, and having your bladder removed. We see a new world for those patients.
We see a world where there's a community sequencing and an academic sequencing. You know, that's occurring now, we think BCG will continue to be the mainstay of therapy. Something like detalimogene, a non-viral approach, and a chemo are well within the abilities of the community urologist, where most of the patients are. When it gets more complicated, it makes more sense to go to more complicated medicines like viral gene therapies or checkpoint inhibitors like KEYTRUDA or removal of the bladder. If you look at the incentives for both the physicians and for the patients, most of these patients would like to stay in the community. If you look at all of these medicines, the 12-month CR rate, you know, is around 20%-40%.
That means recurrence is 60%-80%. Patients are going to need other major medicines, right? This is going to be great for patients, but also this will be an interesting economic boom for these community practices. They'll have happier patients, and their practice will thrive with the income from bill and buy and from these patients. It's an exciting time, I think, for enGene, it's an exciting time for patients, and it's an exciting time, you know, for urologists. Our team is executing well. You see here we have a team that's actually developed multiple products, have had multiple products approved, and have successfully launched multiple products. I believe that we have the right group of individuals to ensure that, you know, we fulfill our commitments.
Just to close up here, I wanna leave a few moments for any questions, you know, that we, that we might have. Why am I excited, why are we excited at enGene? First of all, we're in a place where this market is going to change, all of the incentives for both the patients, and the physicians suggest is this market is gonna be one of sequencing, where multiple agents are gonna be used, market growth will go from where it is now, around $1 billion, to over $20 billion. The second thing is that when you think about new products, right? You think about the adoption of these new products, particularly in community practices, so think about these practices where three to four doctors are together with a minimum amount of staff.
Our non-viral approach just slides in so nicely for the practice. Similarly, it is just easier on the patients, right? Fewer visits to the doctor, should have a low side effect profile, and just be easier for these patients. We're, as I said, an exciting time for the organization in that, you know, we expect to provide data in the second half of this year, file this year and get an approval in 2027. Wrap it around the fact that we have enough cash to get us past all these important inflection points, cash in the, in the second half of 2028. I think it's an exciting time for enGene and pretty exciting time, you know, for detalimogene. With that, I'm happy to take any comments or questions.
Great. Thank you so much, Ron. That was a terrific update. Yeah, clearly a very dynamic area, lots going on, huge market opportunity with lots of growth potential in MIBC. Not only BCG, you know, unresponsive or refractory, but naive, and people have been talking more about this. You guys have a cohort there. People have been focused on BCG, you know, kind of post-BCG, would you share more about your thoughts on what the opportunity could be ahead of using BCG? Maybe as part of that, do you think there's an opportunity for combination therapy, given the clean safety profile of detalimogene, would it make sense at some point for you to look at combination therapies in the BCG naive setting? Thanks.
Well, let me, let me take the second part of that question first. You know, the nice thing about detalimogene being a non-viral approach and having low cost of goods, it lends itself to being able to be combined with many different agents. Right now, these agents are being developed individually, but as I said, you know, these 12-month CR rates are somewhere between, you know, 20%-40%. They're not 80%-90%. Perhaps the combinations you could do that, and the combination of detalimogene, because of the technology, allows us to be combined with all of these agents, right? There's a lot of logic to that. I think when the agents start to, you know, get approved, you're gonna see more and more of the combinations.
Specifically for the naive population, I think our belief is that, you know, BCG is heavily ensconced there just because of habit and because of cost. That being said, BCG is a product that's hard to get in certain places, right? Doctors need other need other agents. While BCG, you know, has been used and there's a good habit behind it, you know, it's still a pretty heavy drug to use, right? You know, increasingly, there's restrictions on it and precautions that are needed as people update, 'cause it's an older product. As you give more to patients, you know, there is more toxicity associated with it, and a product like detalimogene could solve a lot of those issues.
Yeah. All right, good. We'll look forward to further data out of LEGEND later this year. Presumably, we'll get a, at least a first look at 12-month, response data. Is that right?
Yeah, I think our intent is the second half of the year. you know, the LEGEND patients, you know, the majority of the patients, you know, would have reached the 12-month period, so that's the point where we'd like to share a good update, with all of you.
Good. I guess, just lastly, maybe a little bit early, but just pricing. Obviously, ANKTIVA came to market with a pretty robust price point. You know, we'll see how that launch pans out as we hear from J&J, but just any thoughts at this point that you might wanna share?
I think that's a competitive advantage for us, right? Having low cost of goods that allows us to go up and down on the scale, but I think also, you know, sitting behind some of these other agents, we're gonna see what happens from a pricing and reimbursement. We'll be able to maximize the opportunity, you know, with detalimogene. I think it's a real opportunity, you know, for us and for the other agents.
Excellent. Good. Well, I think we're just at our time here, so we'll wrap there. Thank you so much, Ron, for joining us, and thank you all for Zooming in, and enjoy the rest conference.
Pleasure to be here, Leland. Appreciate the opportunity.
My pleasure.