FerGene’s Bladder Cancer Phase III Trial Hits Its Mark

Hit the mark

FerGene, a gene therapy company formed by Ferring Pharmaceuticals and Blackstone Life Sciences, reported that its Phase III trial of nadofaragene firadenovec (rAd-IFN/Syn3) met its primary endpoint. The trial is evaluating the gene therapy for the treatment of high-grade, Bacillus Calmette-Guerin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC).

FKD Therapies Oy (FKD) is a specialist gene therapy company located in Kuopio, Finland. The company was originally founded to develop adenovirus-mediated interferon alfa-2b. It has led the overall development of nadofaragene firadenovec via manufacturing at FinVector Oy, late-stage clinical trials, and the current Biologics License Application (BLA).

FerGene was created to possibly commercialize the therapy in the U.S. and to advance global clinical development. New York-based investment firm Blackstone Life Sciences is investing $400 million in the company and Ferring, a Swiss company, is investing up to $170 million. Ferring will launch and commercialize the therapy outside of the U.S.

NMIBC is an early type of bladder cancer. It is present in the outside layer of the bladder, meaning it hasn’t penetrated deeper into the bladder or spread to other parts of the body. There are about 80,000 new cases of bladder cancer in the U.S. annually, with about 70% being NMIBC. The recommended treatment for NMIBC is intravesical BCG—meaning the therapy is placed directly into the bladder--but between 30% and 50% of those high-grade cases will recur. The outcome for BCG unresponsive patients is poor, with removal of the bladder often the next treatment.

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The Phase III trial of 157 patients in the U.S. hit the primary endpoint, with 53% of patients whose bladder cancer was confined to the superficial layer achieving a clinical response (CR) at three months and 24% continuing to show a CR at 12 months. It also showed broad efficacy in the population with a 73% high-grade recurrence-free (HGRF) survival in patients with papillary disease at three months and 44% HGRF survival as 12 months.

“Currently, patients living with high-grade NMIBC who are unresponsive to BCG have few treatment options and often face bleak outcomes, including complete bladder removal, known as cystectomy,” said Colin PN Dinney, professor and chairman of the Department of Urology at The University of Texas MD Anderson Cancer Center. “Cystectomy is a complex and life-altering surgical procedure for patients, so these positive results from the Phase III trial of nadofaragene firadenovec are highly promising for patients. It would be gratifying to provide an alternative that addresses the critical unmet need for effective second-line therapy for patients facing radical cystectomy.”

The most common adverse events were fatigue, bladder spasm and discharge around the catheter, urinary urgency, blood in the urine, chills, fever, headache, painful urination, urinary tract infection, and diarrhea.

“As a practicing urologist and trial investigator, it’s encouraging to see these types of efficacy and safety results in patients with high-grade NMIBC, an area that’s been in need of new innovative treatment options for more than 20 years,” said Neal Shore, medical director, Carolina Urologic Research Center. “These robust clinical results further demonstrate the potential of nadofaragene firadenovec as a valuable treatment option for NMIBC patients.”

The FDA has already validated FKD’s BLA and granted it Priority Review.

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