enGene Holdings Inc. (Nasdaq: ENGN) has announced promising preliminary data from its LEGEND study evaluating detalimogene voraplasmid in patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS). The company also provided a business update, highlighting strategic advancements and financial results for the full year ended October 31, 2024.
Detalimogene Demonstrates High Complete Response Rate
Preliminary data from the LEGEND study's pivotal cohort showed a 71% anytime complete response rate in BCG-unresponsive NMIBC patients. Specifically, the complete response rate at three months was 67%, and at six months, it was 47%. Detalimogene was generally well-tolerated, with no patients discontinuing treatment due to related adverse events.
LEGEND Study Protocol Amendment and Enrollment Expansion
enGene has implemented protocol changes in the LEGEND study to better align with current treatment standards and real-world practice. These changes affect disease management throughout the trial and criteria for study discontinuation. Furthermore, recruitment has expanded across all four cohorts of NMIBC patients, including those who are BCG-naïve, those who have been exposed to but not adequately treated with BCG, and those with papillary-only disease.
The LEGEND study now includes maintenance dosing, allowing patients in complete response at 12 months to continue receiving detalimogene on a dose-reduced schedule for up to three years. This maintenance treatment consists of two instillations of detalimogene per three-month cycle, potentially providing further evidence of long-term benefit and reducing the burden of frequent urology visits.
Strategic Corporate Updates and Financial Position
The LEGEND study has been included in the Society of Urologic Oncology’s Clinical Trial Consortium (SUO CTC), enhancing trial enrollment and conduct across over 250 sites in the U.S. and Canada. enGene also announced key executive hires and management appointments, including Joan Connolly as Chief Technology Officer and Anthony Cheung, Ph.D., as Chief Scientific Officer.
As of October 31, 2024, enGene reported cash, cash equivalents, and marketable securities of $297.9 million, expected to fund operations into 2027. Total operating expenses for the year were $62.3 million, compared to $26.1 million in 2023, with increased research and development expenses primarily due to the LEGEND study. The net loss attributable to common shareholders was approximately $55.1 million, or $1.46 per share, compared to $104.7 million, or $151.22 per share, for the full year 2023.
Future Outlook
With the BLA filing for detalimogene in BCG-unresponsive NMIBC with CIS on track for mid-2026, enGene is poised to potentially address a significant unmet need in bladder cancer treatment. Additional preliminary data from the pivotal cohort is anticipated in the second half of 2025, offering further insights into the efficacy and safety of detalimogene.
