Private equity giant Blackstone, the world’s third largest private equity investor, continues to move deeper into the life sciences vertical pairing with Ferring Pharmaceuticals to ink a joint $570 million investment to develop a novel gene therapy in the quest to address an unmet need for bladder cancer patients. FerGene, a new gene therapy company and Ferring subsidiary has been created to commercialize the target of this intriguing investmentnadofaragene 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).

The Deal

Blackstone offers $400 million while Ferring ponies up $170 million in FerGene—the latter will potentially launch and commercialize nadofaragene firadenovec outside of the United States. It represents yet another example of how private equity firms expand and extend their reach directly into drug development—a world of high risk and potentially supreme returns.

The Condition

Patients with high-grade, Bacillus Calmette- Guérin (BCG) unresponsive, non-muscle invasive bladder cancer (NMIBC) haven’t seen any progress in clinical development in two decades. Referred to as bladder cancer, it is one of the most frequently occurring cancers with 430,000 worldwide cases per yearThe fourth most common cancer among men in America, it represents the most expensive cancer to treat on a life-time basis, with a high burden on patients, their relatives and healthcare systems.

BCG represents the gold standard treatment and effective in many cases—but unfortunately, at least 60% of cases eventually re-occur. For these patients outcomes are poor with total cystectomy (complete removal of bladder) to prevent the cancer spreading to other organs generally being the next treatment option. The BCG unresponsive population represents one with a high unmet critical need. Consequently the Blackstone and Ferring duo, based on the underlying intellectual property secured a year earlier by Ferring, seek to capitalize and deliver a next generation treatment to markets.

What is Nadofaragene Firadenovec?

Developed by FDK Therapies Oy (FKD) in Kuopio Finland as a treatment for patients with high-grade non-muscle invasive bladder cancer (NMIBC), who are unresponsive to Bacillus Calmette-Guérin (BCG) therapy, the drug is currently in late Phase III development and has been granted Breakthrough Therapy designation by the FDA. A Biologics License Application (BLA) accepted for filing and granted Priority Review by the FDA as well. In 2018 Ferring structured a deal with FDK to commercialize the investigational target asset.

rAD-IFN/Syn3 (nadofaragene firadenovec) has been studied in 33 research sites in America during a Phase III trial.  Prior, Phase II trial results published in the Journal of Clinical Oncology, reported 35% of BCG unresponsive NMIBC bladder cancer patients given one dose of rAD-IFN/Syn3 every three months, were free of high-grade disease at one year.

It is an investigational gene therapy consisting of an adenovirus containing the gene interferon alfa-2b. It is administered by catheter into the bladder, where the virus enters the cells of the bladder wall. Inside the cells, the virus breaks down leaving the active gene  to do its work. The internal gene/DNA machinery of the cells picks up the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer.  Essentially, this novel gene therapy approach transforms the patient’s own bladder wall cells into multiple interferon microfactories, enhancing the body’s natural defenses against this deadly form of cancer.

Key Clinical Investigator Presents

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 Parker 6 of FKD Therapies Oy (FKD). Upon the potential FDA approval, FerGene will hold the marketing authorization of nadofaragene firadenovec.

Blackstone Comment

Nick Galakatos, PhD, Head of Blackstone Life Sciences commented “Nadofaragene firadenovec, currently in late Phase 3 development, has been granted Breakthrough Therapy designation “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,” 

Lead Research/Investigators

Dr. Colin Dinney, Professor and Chairman of Department of Urology, University of Texas MD Anderson Cancer Center (MDACC); Founder and past president of the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC).

Dr. Nigel Parker, FKD Therapies

Source: BusinessWire

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