Ferring and Blackstone Invest $570 Million in Bladder Cancer Gene Therapy


Ferring Pharmaceuticals and Blackstone Life Sciences jointly invested $570 million in nadofaragene firadenovec (rAd-IFN/Syn3), a gene therapy for a type of bladder cancer. FerGene, a Ferring subsidiary, was created to commercialize the drug in the U.S. and advance global development.

Specifically, the therapy is for patients with high-grade, Bacillus Calmette-Guerin (BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC). Bladder cancer is diagnosed worldwide in about 430,000 people annually, making it the ninth most common cancer. In the U.S., bladder cancer is the sixth most common, affecting about 699,450 people and 80,000 new cases diagnosed in the U.S. alone each year.

BCG is the standard treatment in high-grade NMIBC patients, and although viewed as effective, the cancers re-occur in about 60% of patients. In that setting, radical cystectomy, which is complete removal of the bladder and some reproductive organs, is the recommended treatment. Because of this, the BCG unresponsive populations is viewed by the industry and U.S. Food and Drug Administration (FDA) as an area of high unmet clinical need.

Nadofaragene firadenovec is an adenovirus vector-based gene therapy. It contains the gene interferon alfa-2b, which is administered by catheter into the bladder every three months. The virus enters the bladder wall cells, where it releases the active gene. The bladder cells integrate the gene, churning out high quantities of interferon alfa-2b protein. This protein occurs naturally in the body to fight cancer.

“Bringing a novel gene therapy to the market requires dedicated focus and capabilities, and FerGene, a Ferring company, will have the resources and team needed to help us potentially bring nadofaragene firadenovec to patients,” said Frederick Paulsen, chairman of Ferring. “Through this new joint financing model between Ferring and Blackstone Life Sciences, we aim to ensure more people with high-grade, BGC unresponsive, non-muscle invasive bladder cancer may benefit from this novel gene therapy if approved.”

The gene therapy is currently in late Phase III clinical trials. The FDA granted it Breakthrough Therapy designation and its Biologics License Application (BLA) has been accepted for filing and granted Priority Review.

The results from the Phase III trial will be presented on December 5 at the Society of Urologic Oncology (SUO) 20th Annual Meeting held in Washington, DC. Colin Dinney, professor and chair of the Department of Urology at the University of Texas MD Anderson Cancer Center will make the presentation.

Dinney co-heads the gene therapy development program with Nigel Parker of FKD Therapeutics Oy. FKD is a specialist gene therapy company headquartered in Finland.

Once the FDA approves the therapy, FerGene will own the marketing authorization.

Blackstone is investing $400 million in FerGene and Blackstone is investing up to $170 million. Ferring will likely launch and commercialize the gene therapy outside the U.S.

“This innovative partnership with Ferring illustrates the unique value of Blackstone Life Sciences in bringing transformative therapies to market,” said Nick Galakatos, head of Blackstone Life Sciences. “Our expertise and experience in hands-on clinical development and early commercialization will help further advance this promising therapy for bladder cancer patients in the U.S. and around the world.”

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