enGene Shifts Focus from Cystic Fibrosis to Bladder Cancer

EnGene, a Canadian biotechnology company, became a publicly listed entity in November 2023 following a merger with a special purpose acquisition company established by the venture capital firm Forbion. The company has recently announced a shift in its research focus, halting its cystic fibrosis program to concentrate on advancing its primary candidate in bladder cancer treatment.

EnGene is actively developing a nonviral, intravesical therapy known as detalimogene voraplasmid, or EG-70, which is currently being tested in a phase 2 study named LEGEND. This study targets patients with non-muscle invasive bladder cancer (NMIBC) who either do not respond to Bacillus Calmette-Guérin (BCG) treatment or have not been treated with BCG before. The company's objective is to submit EG-70 for FDA approval by 2026.

In parallel, EnGene is evaluating additional applications for EG-70 within the treatment of bladder cancer. The expansion plans include adding a third cohort to the LEGEND study, focusing on patients with high-risk BCG-unresponsive papillary-only NMIBC. Additionally, they plan to modify the second cohort to incorporate BCG-exposed patients, a change from its previous design which featured BCG-naïve patients. This adjustment will temporarily pause the enrollment in the second cohort, while the third cohort enrollment is expected to commence in the fourth quarter of 2024.

This strategic shift has led EnGene to deprioritize the preclinical development of another candidate, EG-i08, intended for cystic fibrosis. The company has decided to pause all activities related to this program to focus resources on EG-70's potential new indications in bladder cancer. CEO Jason Hanson noted that there is a persistent unmet need for treatments in BCG-unresponsive papillary-only NMIBC, and expressed optimism that EG-70 could be well-suited for this role. Interim data from the LEGEND study's BCG-unresponsive cohort is anticipated by the end of September 2024.

Financially, EnGene reported having $264.8 million in cash and equivalents at the end of April, bolstered by a $200 million private placement in February. The company estimates that these funds will sustain its operations through to 2027.

EG-70 leverages EnGene’s proprietary dually derivatized chitosan platform, which facilitates the delivery of nonviral gene therapies by enabling them to penetrate mucus barriers. This platform aligns with EnGene's broader vision of extending the reach of gene therapies, aiming to create safe, locally deliverable treatments that do not necessitate complex handling procedures often associated with viral-based gene therapies.

In summary, EnGene is realigning its strategic priorities to maximize the potential of its leading candidate, EG-70, within the realm of bladder cancer treatment. By halting cystic fibrosis research and focusing on new indications for EG-70, the company aims to address significant unmet medical needs while leveraging its innovative gene therapy platform.