enGene drops preclinical cystic fibrosis program to strengthen bladder cancer strategy

EnGene is halting work on a cystic fibrosis program to focus resources on moving its lead candidate further into bladder cancer.

The Canadian company is already testing the nonviral, intravesical therapy, known as detalimogene voraplasmid or EG-70, in the phase 2 LEGEND study for patients with non-muscle invasive bladder cancer (NMIBC) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG). EnGene’s current goal is to submit EG-70 to the FDA for approval in 2026.

In the meantime, the biotech is considering additional applications of EG-70 within the bladder. This would involve expanding the LEGEND trial to include a third cohort of patients with high-risk BCG-unresponsive papillary-only NMIBC and modifying the second cohort, which previously featured BCG-patients, to include BCG-exposed patients, the company said in its second-quarter earnings report.

Enrollment of the third cohort is due to begin in the fourth quarter of 2024, while the planned changes to the second cohort will see enrollment in that group temporarily paused, enGene explained.

“As a result of the prioritization of these potential new indications in bladder cancer, the company has deprioritized preclinical development of EG-i08 for cystic fibrosis and has paused further activities on that program,” enGene said.

CEO Jason Hanson said BCG-unresponsive papillary-only NMIBC has a "persistent unmet need for which EG-70 may be well-suited,” in a statement from this morning’s release. Interim data from the LEGEND BCG-unresponsive cohort are expected by the end of September 2024, the CEO added.

EnGene, which became a publicly listed company in November 2023 when it merged with a special purpose acquisition company set up by VC firm Forbion, ended April with $264.8 million in cash and equivalents. The biotech estimates this pot of funds, which was topped up by a $200 million private placement in February, will see it through to 2027.

EG-70 was created using enGene’s dually derivatized chitosan platform that is built to allow nonviral gene therapies to penetrate mucus barriers. The company has previously set out hopes to extend the reach of gene therapies by producing safe, locally deliverable, non-viral therapies that don’t require physicians to handle procedures like many viral-based gene therapies currently do.