enGene Announces FDA Clearance of IND Application for EG-70, an intravesical dual-immune modulator for the treatment of non-muscle invasive bladder cancer

Phase 1/2 clinical study expected to begin in early 2021

EG-70 is enGene's first clinical-stage product candidate

BOSTON and MONTRÉAL, Nov. 2, 2020 /PRNewswire/ - enGene Inc., a biotechnology company developing non-viral gene therapies for local administration into mucosal tissues enabled by its proprietary DDX platform, announced today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for EG-70, a first-in-class non-viral gene therapy for the expression of innate and adaptive immune system activators circumscribed to the bladder. Under this IND, enGene intends to initiate its Phase 1/2, multi-center study in patients with Bacille Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) in early 2021.

"The FDA's clearance of our IND for EG-70 is a significant moment in the field of gene therapy, and importantly, a major milestone for patients, as we are now one step closer to a treatment that is so critically needed," said Jason Hanson, President and Chief Executive Officer of enGene. "This milestone is a remarkably exciting one for us as we embark on our next chapter as a clinical stage company, harnessing the power of our non-viral gene therapy platform to impact the lives of patients around the globe."

"Our preclinical data suggest that EG-70 has the potential to produce meaningful and durable responses in patients with cancer," said Jose Lora, enGene's Chief Scientific Officer. "EG-70 brings our technology to BCG-unresponsive NMIBC patients, a population with very limited therapeutic options. We believe EG-70 can change that."

"EG-70 entering the clinic is the culmination of a number of technological refinements to our platform and our ability to manufacture our proprietary DDX gene therapies at clinic-ready scale and quality," commented Anthony Cheung, Co-founder and Chief Technology Officer at enGene. "Our formulation work has resulted in a stability profile that enables storage and use of EG-70 in the same way that community urologists care for NMIBC patients today - a significant advantage over other gene therapy modalities."

The study design consists of an open label dose escalation Phase 1 study to evaluate the safety and tolerability of EG-70 and to determine the recommended Phase 2 dose, followed by a Phase 2 portion to evaluate efficacy and safety.

Jose Lora added "Based on our findings to date, we expect to achieve a crucial therapeutic advantage: locally harnessing the power of IL-12 and RIG-I activation without paying the price of systemic toxicity.  Establishing this paradigm would provide a foundation for EG-70 to be examined across multiple tumor types with high unmet medical need."

About enGene Inc.

enGene Inc. is a biotechnology company developing a proprietary non-viral gene therapy platform for localized delivery of nucleic acid payloads to mucosal tissues. The dually derived chitosan (DDX) platform has a high-degree of payload flexibility including DNA and various forms of RNA with broad tissue and disease applications. In addition to developing EG-70 for BCG-unresponsive NMIBC, enGene is developing unique genetic medicines for respiratory indications and the company is evolving its technology to enable applications in multiple mucosal tissues with areas of high unmet medical need. http://www.engene.com 

Note regarding forward-looking statements

This press release contains certain "forward-looking statements" that reflect the Company's beliefs and assumptions based on currently available data and information. These forward-looking statements fall within the meaning of the "safe harbor" provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "target," "believe," "expect," "will," "may," "anticipate," "estimate," "would," "positioned," "future," and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on enGene's current beliefs, expectations, and assumptions that by definition involve risks, uncertainties, that are difficult to predict and are subject to factors outside of management's control and that could cause actual results to differ substantially from statements made including but not limited to: risks associated with the success of preclinical studies, clinical trials, research and development programs, as well as regulatory approval processes. Actual results and outcomes may differ materially from those indicated in the forward-looking statements. enGene has no approved drugs available for sale marketing at this time and may never have an approved drug. You are cautioned not to rely on enGene's forward looking statements, which are only made as of the date hereof. The Company is under no obligation to update these statements.

SOURCE enGene

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