November 30, 2020 -- FerGene has published data from a phase III clinical trial evaluating an investigational gene therapy, nadofaragene firadenovec (rAd-IFN/Syn3), for the treatment of patients with bacillus Calmette-Guérin (BCG)-unresponsive nonmuscle invasive bladder cancer (NMIBC).
The data, published in the Lancet Oncology, indicates that the study met its primary end point with more than half of the patients achieving complete responses. Of these patients, 45.5% continued to remain free of high-grade recurrence at 12 months. In the study, nadofaragene firadenovec was administered directly into the patient's bladder once every three months.
Bladder cancer is the sixth most common cancer in the U.S., with NMIBC representing approximately 75% of all new bladder cancer cases.
Nadofaragene firadenovec (rAd-IFN/Syn3) is an investigational nonreplicating adenovirus vector-based gene therapy containing the gene interferon alfa-2b, a naturally occurring anti-cancer protein. The vector enters bladder wall cells and releases internal DNA machinery. The DNA sequence gets translated, resulting in secretion of high quantities of the alfa-2b protein. In this way, the novel gene therapy turns the patient's own bladder wall cells into interferon "microfactories."