Ferring, Blackstone back gene-therapy spin-out with $570 million in funding

Ferring Pharmaceuticals and Blackstone Life Sciences on Monday announced the formation of a joint venture, dubbed FerGene, with over $570 million in investment to fund the development of the gene therapy nadofaragene firadenovec, also known as rAd-IFN/Syn3, for certain patients with bladder cancer. The companies noted that Blackstone will invest $400 million in FerGene, while Ferring will contribute up to $170 million.

"Bringing a novel gene therapy to the market requires dedicated focus and capabilities," remarked Ferring chairman Frederik Paulsen, adding that FerGene "will have the resources and team needed to help us potentially bring nadofaragene firadenovec to patients." The therapy is currently in late-stage development for patients with high-grade, non-muscle invasive bladder cancer that is unresponsive to Bacillus Calmette-Guérin (BCG) therapy.

Ferring holds an option to secure global commercialisation rights to nadofaragene firadenovec under an agreement signed last year with Trizell Group's FKD Therapies, which developed the therapy. Ferring and Blackstone said that if nadofaragene firadenovec eventually gains FDA approval, FerGene would market the product.

Phase III data of nadofaragene firadenovec in BCG-unresponsive non-muscle invasive bladder cancer will be presented next month at the Society of Urologic Oncology (SUO) annual meeting. Stephen Boorjian, coordinating investigator for the trial, said the work "expands the search for effective alternatives to radical cystectomy for patients with high-grade, BCG-unresponsive, non-muscle invasive bladder cancer, and offers the potential to meaningfully improve future patient care." The companies noted that an earlier mid-stage trial, published in the Journal of Clinical Oncology, showed that 35% of patients who had been given one dose of nadofaragene firadenovec every three months, were free of high-grade disease at one year.

According to Ferring and Blackstone, the adenovirus vector-based gene therapy containing the gene interferon alfa-2b is administered by catheter into the bladder every three months. This leads to the cells secreting "high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer," they said. "We believe, and Ferring also believes, that this can change the standard of care in bladder cancer," remarked Nicholas Galakatos, senior managing director at Blackstone.

The FDA has already granted nadofaragene firadenovec a breakthrough therapy designation and accepted a biologics license application for the treatment, which it will assess under a priority review. The therapy, also known as TR002, is being studied in a Phase III trial for use against malignant pleural mesothelioma as well.

Meanwhile, since launching its life sciences unit last year, Blackstone has also formed the biotechnology start-up Anthos Therapeutics with Novartis focused on next-generation targeted therapies for cardiovascular disease, with Blackstone investing $250 million in that venture.

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