enGene Therapeutics Inc. Common Stock H.C. Wainwright 28th Annual Global Investment Conference

Ron, welcome. Well, great. Thank you very much for the opportunity to be here.

This is a particularly special time for enGene 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. 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. First of all, our lead clinical asset, detalimogene, 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.

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 detalimogene 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. With a tolerability profile that's trending towards best-in-class, we think that detalimogene is going to be an important part of sequencing and managing NMIBC patients. An attribute that's really important and often overlooked when it comes to community urologists is how products fit into their practice flow. The beauty of detalimogene 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 any time, as we said, we're in the range of the approved products. But we've always, at enGene, looked to see how could we make detalimogene 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 detalimogene with a surfactant bladder rinse. 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. As I said at the very beginning, you're catching us at really an exciting time.

We're sitting with $266 million in cash. 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. As I said before, you're catching us at a particularly special time for enGene. Why are we excited about detalimogene, 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 redose the product, and because we use four readily available ingredients, the cost of goods for our product is actually one of the lowest.

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. NMIBC represents the largest majority of bladder cancer diagnosis. Bladder cancer is the sixth biggest cancer in the U.S., and you see when you get down to the funnel, the actual incident population's about 20,000, some say as high as 40,000. NMIBC is characterized by CIS, carcinoma in situ, which is all over the bladder, so you can't cut those out. You need to use intravesical therapy to get to that. Patients may or may not have growth tumors, papillary tumors, which you see here. In general, these patients present with blood in their urine.

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 timeframe. The treatment consideration is different. Also when you consider the average patient age is around mid-70s. What usually occurs is these patients are started on BCG. Many of you are aware that BCG has had a chronic shortage, so it's sometimes difficult to get BCG, but it is an effective therapy. However, after a couple of years, 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 I'll talk about shortly. The ultimate treatment for bladder NMIBC is actually, when I say ultimate, 100% CR is removal of the bladder.

Sounds simple, but this is a very long surgery with very high mortality, very high morbidity. Patients in general want to avoid this. Where are these patients? When you look across the U.S., the majority of the patients are in the community, about 75%-80% of that. The treatment considerations in the community are different from academic centers. 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 detalimogene is uniquely differentiated. What is happening in these practices? They have limited resources. They have workflow challenges. They have economic challenges. The unique aspects of detalimogene, which I'll talk about shortly, fit in very nicely. Let's give a little background. What is detalimogene? Detalimogene, 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 detalimogene, though, is that it has both an impact on the innate and adaptive immune systems. So it's a one-two punch. As I was talking to you earlier about our components in manufacturing, how is detalimogene 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, instill with patients.

I'm delighted that we've already completed our FDA validation batches. We're manufacturing at scale. Since we have the CDRP program with the FDA, we have a lot of dialogue, and we feel pretty confident about our filing from that perspective. What is the design of detalimogene? 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 1 and 2, week 5 and 6. Very little time in the office. Then there's nothing that occurs afterwards in terms of extra precautions like urine bleaching or the like. For the practice, as I said, most practices have a fridge or a freezer. Many of them don't have an ultra-cold chain storage freezer. It can be administered by anyone, any qualified medical professional.

As I said, there's no decontamination needed within the practice, no hoods required or the like. If you summarize this, detalimogene is the only product that does not have complex shipping or storage, that has a short administration timeframe, that does not have any bacterial or viral decontamination, that doesn't require any post-treatment action, and can be administered by anybody in the practice itself. So where are we studying detalimogene? Our clinical trial is the LEGEND program. Cohort 1 is with BCG unresponsive patients with CIS. 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 1, which is detalimogene without a surfactant. Again, it's BCG high-risk patients, a global study that we've put 125 patients on. Primary endpoint, CR anytime. If you look at the baseline characteristics, they are pretty typical for these patients, mostly male, average age in the 70s.

Please note a couple of things here. We actually have a patient population that is highly experienced. Almost 40% of the patients have CIS plus a papillary disease. Even though our study is designed for BCG and detalimogene, we have a high percentage of patients, 25% of the patients that have had a prior treatment. At the recent American Urological Association's meeting, we shared preliminary data. What you see here is a 54% complete response time, again in line with other products. We provided some updates on where we were from a 12-month landmark perspective. 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.