• Ferring launches new company FerGene, with a focus on the global development and US commercialization of nadofaragene firadenovec, for high-grade, Bacillus Calmette-Guérin unresponsive, non-muscle invasive bladder cancer patients
  • Blackstone Life Sciences will invest $400 million USD and their expertise to accelerate the future development and commercialization of nadofaragene firadenovec, an investigational gene therapy
  • The FDA has accepted the Biologics License Application (BLA) for filing and granted Priority Review for nadofaragene firadenovec
  • Phase 3 results to be presented at Society of Urologic Oncology 20th Annual Meeting in Washington DC on December 5, 2019

San Francisco, CA (UroToday.com) — Ferring Pharmaceuticals and Blackstone Life Sciences today announced the joint investment of over $570 million USD in nadofaragene firadenovec (rAd-IFN/Syn3), an investigational novel gene therapy in late stage development for patients with high-grade, Bacillus Calmette-Guérin (BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC).

FerGene, a new gene therapy company and Ferring subsidiary, has been created to potentially commercialize nadofaragene firadenovec in the US and to advance the global clinical development. FerGene’s goal is to bring this promising therapy to a patient population which has seen little improvement in their standard of care over the past twenty years. Blackstone will invest $400 million USD and Ferring will invest up to $170 million USD in FerGene. Ferring will also potentially launch and commercialize nadofaragene firadenovec outside of the US.

“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 Frederik Paulsen, Chairman, Ferring Pharmaceuticals. 

“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.”

Nadofaragene firadenovec, currently in late Phase 3 development, has been granted Breakthrough Therapy designation and had its Biologics License Application (BLA) accepted for filing and granted Priority Review by the FDA.

“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,” said Nick Galakatos, Ph.D., Head of Blackstone Life Sciences.

“Through FerGene, Blackstone and Ferring’s goal is to successfully commercialize and further develop this adenovirally mediated interferon alfa-2b gene therapy, a potential breakthrough treatment for high-grade, BCG unresponsive, non-muscle invasive bladder cancer patients,” said Paris Panayiotopoulos, Blackstone Life Sciences Managing Director.

Phase 3 clinical trial results will be presented at the Society of Urologic Oncology (SUO) 20th Annual Meeting in Washington, DC on December 5, 2019 by Dr. Colin Dinney, Professor and Chairman of the Department of Urology at the University of Texas MD Anderson Cancer Center (MDACC) and a founder and past president of the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC). Dr Dinney pioneered the development of nadofaragene firadenovec and co-heads the development program alongside Dr. Nigel Parker6 of FKD Therapies Oy (FKD). Upon the potential FDA approval, FerGene will hold the marketing authorization of nadofaragene firadenovec.

FKD is a specialist gene therapy company based in Finland focused on the development and regulatory filing of nadofaragene firadenovec, which has been studied in the Phase 3 trial in 33 centers across the US, in conjunction with the SUO-CTC.

“We are excited to present the Phase 3 data at the upcoming SUO meeting,” said Dr. Stephen A. Boorjian, the Coordinating Investigator for the trial and the Carl Rosen Professor of Urology at Mayo Clinic in Rochester, Minnesota. “This trial expands the search for effective alternatives to radical cystectomy for those patients with high-grade, BCG unresponsive, non-muscle invasive bladder cancer, and offers the potential to meaningfully improve future patient care.”

Reference: 

1. AIV Institute for Molecular Sciences, Kuopio, Finland.

Source: “Ferringusa » Ferring And Blackstone Life Sciences Invest Over $570 Million USD In Novel Gene Therapy For Bladder Cancer Patients”. 2019. Ferringusa.Com

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