FerGene has published positive Phase III data from the US clinical trial analysing investigational gene therapy, nadofaragene firadenovec (rAd-IFN / Syn3), for treating patients with high-grade, Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC).

Nadofaragene firadenovec is a non-replicating adenovirus vector-based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder (intravesical) once every three months.

It is believed to target the patient’s bladder wall cells to boost the body’s natural defences to fight cancer.

The trial, which enrolled 157 patients from 33 sites in the US, met its primary endpoint with about 53.4% of CIS ± Ta / T1 patients (carcinoma in situ; with or without concomitant high-grade Ta or T1 disease) achieving a complete response (CR) in three months.

In addition, 45.5% of those who achieved a CR continued to remain free of high-grade recurrence at 12 months.

The long-term follow-up phase of the study is progressing with patients being monitored in the four-year study.

Instillation site discharge, fatigue, bladder spasm, micturition urgency and hematuria were the most common adverse events (AEs) occurred in patients in order of decreasing frequency during the trial.

FerGene Medical Affairs vice-president Vijay Kasturi said: “We believe the important clinical findings highlighted in The Lancet Oncology with this novel gene therapy may fulfil a significant unmet need for patients and have the potential to be practice-changing.

“We are extremely grateful to the investigators, the patients who participated in the study, FKD Therapies and the SUO-CTC for the important findings highlighted in this publication.”

The company also submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for nadofaragene firadenovec.