enGene Therapeutics Inc. Common Stock Wells Fargo 21st Annual Healthcare Conference

Well, real pleasure to be here.

Yeah, sure. So, enGene is a public company. We are focused on non-viral gene therapies, so very unique platform that we have. It is a company that is well advanced. Our lead asset, detalimogene voraplasmid, is a non-viral gene therapy that we are developing for a form of non-muscle invasive bladder cancer, those patients that are resistant to BCG, who have carcinoma in situ. Our clinical development program is called the LEGEND program. It has multiple cohorts. The pivotal cohort is one of the largest in this space of 125 patients. And we are at a very exciting period for the company, where we expect to have that data maturing near the end of this year. We expect to meet with the FDA later this year, and we plan to file by the end of this year and expect an approval next year. This morning, we announced our quarterly.

We have $266 million of cash, and that gets us through all of those things. We are a company that is well capitalized and that is poised for a real growth period.

Yeah, sure. We are a public company, we are also a Canadian company. Our disclosure requirements are different. The LEGEND cohort 1 study is an open label study, and we provided a data update in May, which from our perspective, the market grossly overreacted to, and I will tell you why. First off, when you think about these products, the primary endpoint is complete response rate at any time. We displayed a 54% CR at any time, which is in line with all the other approved products. That is the primary endpoint. Secondary endpoint is of those 54% of patients, what percentage are durable at the 12-month time point? If you look at the products that are approved, it is a pretty tight range, about 40%-50%, so let us call it half of the patients. We had 21 patients that were complete responders that had not progressed to that point.

It is a little bit early for us. Another endpoint that is a Wells Fargo focus is 12-month CR landmark number, and at that time, we had, again, immature data, but projected to have around 25%. That is the efficacy part of it. That is always just half of the story, right? The tolerability part of our medicine is really important. 80% of the patients are in community practices, and community doctors really want to have products that are efficacious and tolerable and easy to use. Our tolerability continues to hold even with more exposures. For example, our treatment interruption and discontinuation rate is very low, 2.4%, where many other products are in double digits or high double digits. I think we shared what I consider to be a pretty solid data update, where our CR at any time, the primary endpoint, is in line with other products.

Tolerability, in line with other products, but probably best in class from that perspective. An immature durability data set, which we will hear more about in the fourth quarter of this year.

I think there are a couple things that we are looking for in the fourth quarter. First of all, CR at any time was almost finalized by that point. That will be finalized, right? The durability, the percentage of those patients that are durable 12 months and beyond, we will have some more data on that, but probably not all of that data. The 12-month landmark number, we will have the majority of those patients, but we will have that information. Hopefully around that time as well, we would have had an opportunity to meet with the FDA, and we will have some insights from our pre-BLA meeting.

Yeah. Our market research suggests that detalimogene will have a very strong position within the NMIBC high-risk space. Why is that? First of all, when you look at the marketplace, let's call it 20% are in the academic space, 80% are in the community space. The needs of each of these groups is different. In the academic space, they're getting patients that have been through multiple lines of therapy, been sequenced, and have landed up probably more serious disease. The academics, in general, would prefer to go to radical cystectomy because removal of the bladder has a 100% CR rate, right? It's easily accessible to them. But most patients don't want to have organs removed, right? Radical cystectomy for an average patient who's 75 years of age, it's a pretty heavy surgery. It has high mortality and has high morbidity.