Ferring Pharmaceuticals announced the signing of an agreement giving the company the option to secure global commercialization rights to nadofaragene firadenovec/Syn3 (rAd-IFN/Syn3), a novel gene therapy being developed by FKD Therapies Oy (FKD) as a treatment for patients with high-grade non-muscle invasive bladder cancer (NMIBC), who are unresponsive to Bacillus Calmette-Guérin (BCG) therapy. This option is exercisable on marketing approval from the US FDA. Ferring will create a new US oncology division with the specialist knowledge and presence to introduce novel advanced therapies to the market.
rAd-IFN/Syn3 is currently undergoing Phase 3 development in the US under the sponsorship of Finnish gene therapy specialists FKD. The results of the earlier Phase 2 trial, 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. The ongoing Phase 3 study is designed to establish the efficacy and safety of the product. rAd-IFN/Syn3 has been awarded Fast Track and Breakthrough Therapy designations by the FDA.
“We are excited about the potential to commercialize rAd-IFN/Syn3, a novel gene therapy for bladder cancer patients,” said Michel Pettigrew, President of the Executive Board and Chief Operating Officer, Ferring Pharmaceuticals. “The gene therapy sector is growing rapidly and building a presence in this specialized area is a very positive opportunity for Ferring.”
Bladder cancer is one of the most frequently occurring cancers with an estimated 430,000 new cases being reported worldwide each year. It is the fourth most common cancer in men in the US and is the most expensive cancer to treat on a life-time basis, with a high burden on patients, their relatives and healthcare systems. In high-grade NMIBC patients, BCG is the gold standard treatment and although effective, over 60% of cases eventually re-occur. The outcome for such patients is poor, with total cystectomy (complete removal of the bladder) to prevent the cancer spreading to other organs generally being the next treatment option. As such, the BCG unresponsive population is one of high unmet clinical need.
“Today, bladder cancer patients have very limited medical options and new treatments that delay or prevent total removal of the bladder and improve clinical outcomes are urgently needed for patients,” said Professor Klaus Dugi, Chief Medical Officer, Ferring Pharmaceuticals. “Phase 2 clinical results for rAd-IFN/Syn3 were very encouraging and we look forward to the Phase 3 data.”
Gene therapy is one of a new class of therapeutic treatments known as advanced therapy medicinal products. rAd-IFN/Syn3 is built on adenoviral vector technology, a non-integrating vector, and results in enhanced expression of the therapeutic protein interferon alfa 2b. To date, it has completed three clinical trials in the US.
rAd-IFN/Syn3 (nadofaragene firadenovec/Syn3) 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. This novel gene therapy approach turns the patient's own bladder wall cells into multiple interferon microfactories, enhancing the body's natural defenses against the cancer. The Phase 3 trial for rAd-IFN/Syn3 opened in 2016 with up to 150 patients to be enrolled across 35 centers in the US.