enGene Therapeutics Inc. Common Stock Morgan Stanley 24th Annual Global Healthcare Conference

Great. Well, first of all, Judah, thanks for the invitation to be here. Great to see you. It is a pretty exciting year for enGene. What is enGene? enGene is a company that looked at the challenges of viral gene therapies and said, "I think we can solve some of these challenges." Those challenges being packet size, redosability, and the manufacturing challenge. enGene was discovered to build non-viral gene therapies, and then through the discovery process, translated a product called detalimogene, which is the first non-viral gene therapy that we are developing for non-muscle invasive bladder cancer. Detalimogene has been studied in the LEGEND program in cohort 1, which is a pivotal program, 125 patients, which is one of the larger programs. We're heading 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 detalimogene in the U.S., and expect a potential approval in 2027. Sitting with $266 million in cash, we're all capitalized to achieve all of those things.

I was shocked when this opportunity presented myself. The treatment in bladder cancer, actually, particularly non-muscle invasive bladder cancer, is pretty poor. The definitive treatment is removal of your bladder.

The radical cystectomy, and then when I learned more about radical cystectomy, 4 to 6-hour surgery, multi-organ, 5%-15% mortality, and horrific morbidity as well, individuals landing with an ostomy, loss of sexual function. So treatment's not very good. When you think about the epi, there are about 90,000 in the U.S., in the instant population, about 90,000 individuals have bladder cancer, 80% of those are non-muscle invasive bladder cancer. And then within that, it's estimated somewhere between 20,000 to 40,000 individuals each year have non-muscle invasive 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.

Again, I think we're at the early stages of these new agents being available. Part of the challenge is when you look at the market overall, let's call it 20%-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 don't slide easily into a urology practice.

So 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 urologists right now, what do they have? They have BCG, if they can get it.

Right. Then they have gemcitabine, which they can use, but there's some questions about efficacy, and it's not really financially viable.

Then of the new agents- gemcitabine on the pretzel may or may not be viable.

There's just not a lot of choices for these community urologists, and that's where detalimogene can really make a big difference.