In this connection, Ferring Pharmaceuticals has launched a new company FerGene, which will focus on the global development and US commercialisation of nadofaragene firadenovec.
The gene therapy is being developed for patients having high-grade, Bacillus Calmette-Guérin (BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC). Currently, the gene therapy is in late phase 3 development.
Nadofaragene firadenovec has been given breakthrough therapy designation by the US Food and Drug Administration (FDA) and its biologics license application (BLA) had been accepted for filing by the regulator and given priority review. It is an adenovirus vector-based gene therapy having the gene interferon alfa-2b and is administered using a catheter into the bladder every three months.
Ferring Pharmaceuticals chairman Frederik Paulsen said: “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.
“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’s phase 3 clinical trial data will be released in early December by Colin Dinney from the University of Texas MD Anderson Cancer Center (MDACC), who has led its development. Nigel Parker of FKD Therapies is co-heading the development programme.
If approved by the FDA, the marketing authorisation of nadofaragene firadenovec will be held by FerGene. The new gene therapy company will receive an investment of $400m from Blackstone Life Sciences and up to $170m from Ferring Pharmaceuticals.
Blackstone Life Sciences head Nick Galakatos said: “This innovative partnership with Ferring illustrates the unique value of Blackstone Life Sciences in bringing transformative therapies to market.
“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 US and around the world.”