EXOME

all the information, none of the junk | biotech • healthcare • life sciences

Ferring and Blackstone Team Up to Fund Gene Therapy Firm With $570M

Xconomy Boston — 

Ferring Pharmaceuticals and Blackstone Life Sciences have formed a new company to commercialize a bladder cancer gene therapy that is currently in late-stage clinical testing.

The new company, FerGene, is a subsidiary of Swiss pharmaceutical company Ferring, which is investing $170 million in the new unit. Blackstone has committed $400 million. In addition to US commercialization, FerGene will also handle clinical tests of the therapy to support its approval in other markets around the world.

FerGene’s gene therapy, nadofaragene firadenovec, was developed for patients whose fast-growing non-muscle invasive bladder cancer does not respond to Bacillus Calmette-Guérin (BCG), an immunotherapy used to treat the cancer. While BCG treatment can be effective, tumors can still reoccur. In such cases, patients often undergo removal of the bladder and certain reproductive organs to prevent the cancer from spreading.

The FerGene therapy is intended to boost expression of interferon alpha b, a therapeutic protein. The therapy uses a virus to ferry the gene for making that protein into the cells of the bladder wall. Cells pick up the gene and translate its DNA sequence, which Ferring says results in the secretion of large quantities of interferon alpha b, a naturally occurring protein that the body uses to fight cancer.

Ferring last year licensed global rights to nadofaragene firadenovec from Finland-based gene therapy developer FKD Therapies Oy (FKD), which initially developed the treatment. At that time, Ferring said it planned to create a US oncology division to bring the treatment to the market.

According to Phase 2 data published in the Journal of Clinical Oncology in 2017, 35 percent of the 40 bladder cancer patients who received one dose of the gene therapy were free of high-grade disease after one year. Of those 14 patients, two experienced a recurrence of their cancer: one at 21 months, and the other at 28 months. One patient died 17 months into the trial, a death attributed to a tumor in the upper urinary tract.

The Phase 3 test of the gene therapy enrolled about 150 patients. Data from that study are scheduled to be presented on Dec. 5 at the annual meeting of the Society of Urologic Oncology in Washington, DC.

Blackstone Life Sciences is the division of private equity giant Blackstone (NYSE: BX) that invests in medicines and healthcare technologies. The PE firm launched the dedicated life sciences investing business last year after acquiring Clarus Ventures, a life sciences investment firm that has poured money into more than 50 private and public companies spanning biotechnology, medical devices, and diagnostics. Clarus, which has offices in Cambridge, MA, and South San Francisco, had raised $2.6 billion since its 2005 founding.

Photo by Flickr user Caroline Davis2010 via a Creative Commons license