Ferring, Blackstone spin out gene therapy with $570M in funding

In phase 2, 35% of patients who received Ferring Pharmaceuticals' nadofaragene firadenovec were free of high-grade disease at one year. (Blackstone)

Ferring Pharmaceuticals has spun out its late-phase gene therapy nadofaragene firadenovec with the support of Blackstone Life Sciences. The two organizations have put up $570 million to support the spinout as it works to bring the bladder cancer drug to the U.S. and other markets. 

Nadofaragene firadenovec consists of a virus designed to introduce a gene into bladder cells, thereby hijacking the molecular machinery for production of the cancer-fighting interferon alpha-2b protein. Ferring picked up an option on the therapy from FKD Therapies last year and is set to share data from a phase 3 trial early next month. 

The FDA has already accepted a filing for approval and granted it priority review, putting Ferring on course to secure clearance to sell nadofaragene firadenovec in the U.S. next year. With the launch date looming, Ferring has teamed with Blackstone to fund the commercial rollout.

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The result is FerGene, a spinout that is starting life with $400 million from Blackstone and up to $170 million from Ferring. FerGene will use some of the money to support the anticipated U.S. introduction of nadofaragene firadenovec for use in patients with high-grade, non-muscle invasive bladder cancer that is unresponsive to Bacillus Calmette-Guérin (BCG).

Another slice of the money will go toward global development of nadofaragene firadenovec. Ferring may handle commercialization of the gene therapy outside of the U.S. 

Blackstone also plans to offer more than just money to FerGene.

“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," Nick Galakatos, head of Blackstone Life Sciences, said in a statement.

Ferring is yet to share data from the 150-patient phase 3 trial of nadofaragene firadenovec, which wrapped up earlier this year, but the Blackstone-backed spinout and FDA filing suggest it generated results capable of making a success of the gene therapy. In phase 2, 35% of BCG-unresponsive patients who received nadofaragene firadenovec every three months were free of high-grade disease at one year.

The data were encouraging given the poor treatment options of people with bladder cancer that is unresponsive to BCG. Most patients treated with BCG suffer a recurrence of their bladder cancer. At that point, bladder removal is often seen as the best of a bad bunch of treatment options.  

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