enGene Therapeutics Inc. Common Stock KOL event

Good morning, everyone, and thank you for joining. With me on today's call are Ron Cooper, CEO, Anthony Cheung, enGene's Co-founder, and we're pleased to welcome Doctors Suzanne Merrill and David Cahn of Colorado Urology. Before we begin, I would like to remind you that during our prepared remarks and Q&A session to follow, we may make forward-looking statements for purposes of U.S. and Canadian securities laws.

These statements include, but are not limited to, our current expectations regarding the potential benefits of detalimogene voraplasmid or detalimogene, the efficacy, safety, and product profile of detalimogene, potential impacts of incorporating a surfactant bladder rinse prior to treatment with detalimogene, timing of clinical data, timing of regulatory submission, prospects for regulatory approval of detalimogene, projections of market opportunity and market share, anticipated market acceptance of detalimogene, if approved, our current views and market research on NMIBC bladder cancer implications or takeaways from our market research, estimates, projections, or forecasts of payer coverage of therapies, expectations regarding how detalimogene may be used by healthcare providers or sequencing of products. They involve risk, uncertainties and assumptions that are difficult to predict and may not prove to be accurate.

These statements should be considered only in conjunction with the information in our filings made with Canadian and American securities regulators, including our Risk Factors section of our annual report on Form 10-K. At this time, I'll turn the call over to Ron Cooper, enGene's President and CEO.

Ron? Great. Thanks, Lauren. Thank you all for joining us this morning.

You see here on slide 3 is an overview of this morning's agenda. Before we begin the formal presentation, I'll start by reiterating that we believe the NMIBC market is fundamentally changing as next generation medicines meet the different needs of community urologists, and that detalimogene is uniquely suited to address the combined efficacy, tolerability, and logistical challenges of community urologists. First, we believe that the NMIBC market is still immature and that the advent of more options for patients will drive an increase in the prevalent population through increased sequencing of therapies to grow the overall market and create room for multiple therapies. This is not going to be a winner-take-all market. Second, we believe that detalimogene's clinical profile to date is compelling.

Our most recent interim data update for the LEGEND pivotal cohort, which has studied detalimogene without a surfactant, was in May of this year. While our long-term durability data was still immature, with a median follow-up of just 5.5 months for all patients, we were encouraged that our study's primary endpoint, complete response at any time, was 54%, within the range of products already approved for this indication. Even more encouraging was detalimogene's tolerability profile, which we believe is trending towards best in class. Rates of treatment-related adverse events leading to dose interruption or discontinuation were only 2.4%, respectively. Third, detalimogene was designed with our non-viral DDX technology, which uses low-cost, readily available components supporting both scalable manufacturing and simplified storage and handling, which makes it viable for all practices and should allow for broad adoption in any practice setting.

Fourth, we're already thinking about lifecycle management through the incorporation of a surfactant bladder rinse. Our pre-clinical data suggests that this approach may improve both the efficacy of detalimogene and reduce dwell time, another potential advantage to patients and providers. Importantly, we remain well-funded to accomplish all our upcoming milestones and through potential approval in 2027. Now, before handing it over to our community urology experts, I'd like to review highlights from market research that give insight into the new dynamics impacting the NMIBC market. First, with next gen therapies, we believe the community urology market will begin to unlock, and the prevalent market should grow with increased sequencing. We also outlined the evolving pricing dynamics over the course of several years, with the most recent group products launching in the range of $500K-$700K per year and with little payer pushback.

Importantly, 40% of patients are being treated in community practices that are either resource-limited, workflow-constrained, or economics-focused. These are practices looking to balance efficacy, safety, resources, and economics when making treatment recommendations. Given these constraints, there's a continued need for new bladder-sparing treatments, and detalimogene as a next gen product has the potential to unlock these segments as a workflow-friendly option. I've shared with you market research highlights, but there's nothing better than hearing from physicians that are on the front line treating these patients. With us today to shed light on these aspects are two leading community urologists from Colorado Urology, Dr. David Cahn and Dr. Suzanne Merrill. With that, I'll turn it over to Doctors Merrill and Cahn to introduce themselves, their practice, and review illustrative patient cases Thank you, Ron.

I am Dr. Suzanne Merrill, and it is a pleasure to be with you today. Following my fellowship in urologic oncology at the Mayo Clinic, I spent 6 years in academic medicine as an associate professor of urologic oncology and residency program director at Penn State Hershey Medical Center. I then transitioned to private practice at Colorado Urology just over 5 years ago. I would like to take this opportunity to tell you a little bit more about my practice before diving into a case study discussing the real-world management of non-muscle invasive bladder cancer. With my background, I have unique insights into the differences in academic and community urology. Colorado Urology is a large group practice in Denver. We are part of a larger national group, commonly called LUGPA, that has multiple practice sites across our nation and employs over 220 providers.

At Colorado Urology, we have over 30 urologists and 20 advanced practice providers serving 18 locations through the greater Denver and Boulder metropolitan areas, which collectively serve close to 130,000 patients. Our practice is structured similarly to other LUGPAs across the nation. However, there are still many smaller, independent community practices that are different from our organization. Many such large practices have provider champions for specific urologic disease states, and I lead our Colorado Bladder Cancer program, where we look to offer patients access to the most cutting-edge bladder cancer treatments as well as clinical trials. I will let Dr. Cahn, our director of clinical research, tell you a bit more about that.

David? Thanks, Suzanne. I completed my residency in urology at Montefiore Medical Center and a fellowship at University of Colorado, and I have been in private practice since 1998.

In 2000, I founded Foothills Urology, which became Colorado Urology. I have always had a passion for clinical research, and I have the pleasure of leading the practice's clinical trial program. At present, we have 38 active trials ongoing, and I have served as the PI on more than 70 clinical trials. What we look forward to speaking to is the often long and burdensome patient journey and the continued need for additional therapies in the BCG unresponsive space. With that, I would like to turn to our patient case studies to shed some light on the various treatments and lifestyle considerations impacting treatment decision. Let me turn it back over to you, Suzanne.

Our first case involves an 82-year-old male named Walter, a retired veteran who moved into an apartment down the street from his daughter and 3 grandchildren after his wife passed. Walter is a long-term smoker with a history of peripheral vascular disease, coronary artery disease, COPD, and atrial fibrillation, who experiences urinary frequency and urgency, which are currently managed with Myrbetriq. His cardiologist has prescribed him Eliquis. Walter's initial presentation starts in January of 2022, where he develops gross hematuria or blood in the urine, and his urine cytology was found to be positive with a normal CT scan. Using cystoscopy, we saw diffuse erythema, which means severe redness within his bladder, and thus we performed a TURBT, which pathology confirmed multifocal carcinoma in situ. As a standard for NMIBC with CIS, Walter began BCG induction.