enGene Announces First-in-Human Dosing of EG-70 for the Treatment of Non-Muscle Invasive Bladder Cancer in Phase 1/2 Clinical Trial

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May 27, 2021, 10:57 ET

- EG-70 is a first-in-class intravesical non-viral genetic medicine encoding RIG-I activators and IL-12 as a monotherapy

- First clinical candidate from company's novel proprietary platform engineered to activate both innate and adaptive immune systems

BOSTON and MONTRÉAL, May 27, 2021 /PRNewswire/ - enGene Inc., a clinical-stage biotechnology company developing non-viral gene therapies for local administration into mucosal tissues enabled by its proprietary DDX platform, today announced the dosing of the first patient in the LEGEND study, a first-in-human Phase 1/2 clinical trial of EG-70 in patients with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC).

Based on enGene's DDX platform, EG-70 is a non-viral gene therapy encoding two RIG-I agonists to stimulate the innate immune system, and IL-12 to stimulate the adaptive immune system. By stimulating both arms of the immune system, intravesically administered EG-70 yields remarkable tumor regression in preclinical models of bladder cancer, with induction of effective immunological memory and minimal signs of toxicity.

"Initiating our first clinical study of EG-70 is an important milestone for enGene, validating the potential of our platform to develop novel therapeutics towards clinically meaningful outcomes," said Jason Hanson, Chief Executive Officer at enGene. "This is the first time a non-viral gene therapy directly activating both innate and adaptive immunity is being evaluated as an anti-tumor agent in humans, and we are excited about the outcome of this trial towards positively impacting the lives of patients battling difficult-to-treat cancers."

"Patients with BCG-unresponsive high risk NMIBC have limited treatment options other than radical cystectomy which is associated with significant morbidity and quality of life alterations. Based on our understanding of high grade bladder cancer, the mechanism of BCG resistance/relapse, and the EG-70 mechanism of action, I strongly believe that this novel intravesical therapy has the potential, with few side effects, to make a meaningful difference for patients," said Dr. Gary Steinberg, Professor and Director at the Perlmutter Cancer Center Goldstein Urology Bladder Cancer Program of NYU Langone Health, and lead Principal Investigator for the EG-70 Phase 1/2 study.

The LEGEND study, both first-in-human and first-in-class, is an open-label, monotherapy, multi-center, dose-escalation trial evaluating safety and tolerability, pharmacokinetics, pharmacodynamics, and efficacy of EG-70 administered by intravesical instillation. To learn more about the first-in-human clinical trial of EG-70 in BCG-unresponsive NMIBC, please visit ClinicalTrials.gov.

About enGene Inc.
enGene Inc. is a clinical-stage biotechnology company developing non-viral gene therapies based on 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 preclinical models, enGene's DDX technology has been demonstrated to effectively induce expression of therapeutic genes following delivery to the lung, gastrointestinal tract and urinary bladder. Engene has developed scalable GMP-compliant manufacturing of DDX products. 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