FKD Therapies Oy

Ferring Pharma's CSO says the company's commitment to reproductive and maternal health products is unchanged even as R&D priorities shift. After more than 25 years, Ferring Pharmaceuticals suddenly shuttered a San Diego research institute in March just months after finishing a major renovation. It's all part of the plan, says Chief Scientific Officer Armin Metzger, Ph.D. The research executive, now in his seventh year with Ferring, said that the company is making a concerted pivot toward external innovation to make the best use of capital following two FDA approvals at the end of 2022. It's a shift that Metzger describes as a "fundamental" change. "We will still have internal discovery, but [be] much, much more external focused, giving more flexibility of allocating funds," he said in an interview with Fierce Biotech. The clear-eyed assessment of the company’s priorities set the record straight as to why the Ferring Research Institute, one of the company’s three main R&D hubs, closed after 27 years. The move resulted in nearly 90 staffers being laid off and came after Ferring finished renovations on the institute just last year.  “[T]he turning point was that we got two FDA approvals of two first-in-class assets in one year,” said Metzger. “And this leads to the adaptation, again of the strategy—how we deliver innovation to the patients—and it is not an easy decision-making process.”  In November 2022, Ferring nabbed FDA approval for the first fecal microbiota product, Rebyota, for adults with C. difficile following antibiotic treatment or recurrent infection. Just a few weeks later, gene therapy Adstiladrin was approved for bladder cancer.  The new strategy is not a complete departure from Ferring’s existing work, as those now-approved assets originated from deals. Ferring acquired Rebiotix in April 2018, tacking on the C. difficile treatments, and then a month later, the Swiss pharma signed a commercialization agreement with FKD Therapies for the bladder cancer treatment. “I think a company of our size, with the investment needed now on the later stage, we can't entertain technology platforms in-house,” said Metzger, who added that Ferring will instead rely on "partnerships across the globe.” Ferring’s main therapeutic areas are remaining unchanged, however, including maternal and reproductive health, which Metzger called “the core” of the company. One of the priorities is bringing the infertility treatment Rekovelle to the U.S. after it nabbed approval in Europe and Switzerland. He expects feedback from the FDA in the second half of the year.  In addition to Rekovelle, infertility treatments LutrePulse and Menopur are under development, along with Pabal, a med for postpartum hemorrhage.  But the clinical-stage work is in limbo. A phase 2 trial for a treatment to address inadequate milk production, merotocin, was shuttered earlier this year due to low enrollment and a phase 2 infertility treatment is now absent from the company’s pipeline. A spokesperson for Ferring did not immediately respond to a request for comment on the missing program.  Regardless, Metzger says that in the next year, the company hopes to bolster its portfolio, including potentially boosting the indications for merotocin. This could also mean combination opportunities and female and male infertility assets from Ferring's reproductive medicine pipeline.

Bladder cancer can be treated with several types of drugs. Gene therapy is now part of the mix. The FDA has approved a Ferring Pharmaceuticals gene therapy as a treatment for patients whose cancer is at high risk of progressing by invading muscle tissue and spreading throughout the body. The regulatory decision announced Friday covers the treatment of adults whose bladder cancer does not respond to Bacillus Calmette-Guérin (BCG), an immunotherapy that’s commonly used as a first-line treatment. The Ferring therapy, known in development as nadofaragene firadenovec, is designed to get the body to produce a particular therapeutic protein that fights the cancer. Engineered versions of the protein Interferon-alfa 2b are already used for indications that include hepatitis B and C infections, hairy cell leukemia, and melanoma. Ferring’s gene therapy, which will be marketed under the brand name Adstiladrin, turns the cells lining a patient’s bladder into tiny factories that churn out high quantities of human interferon-alfa 2b. Delivered via an adenovirus, the therapy brings to bladder wall cells a gene that encodes interferon-alfa 2b. The protein-making machinery of the cells translate the DNA sequence and then express the therapeutic protein, which is intended to provide anti-tumor effects. The FDA decision for the Ferring gene therapy was based on the results of an open-label, single-arm Phase 3 study that enrolled 157 patients. The gene therapy, which is dosed via a catheter, was administered once every three months for up to 12 months. Results showed that 51% of patients who received Adstiladrin achieved a complete response, meaning the disappearance of all signs of the cancer, at three months. The median duration of that response was 9.7 months; 46% of those responders maintained that response for at least one year. Bladder discharge, fatigue, bladder spasm, urinary urgency, and blood in the urine were among the most common adverse reactions reported from the clinical trial. The results were published by The Lancet Oncology in 2020. Adstiladrin was initially developed by FKD Therapies. Ferring, a privately held company based in Saint-Prex, Switzerland, licensed global rights to the gene therapy in 2019; the following year, Ferring and Blackstone Life Sciences formed a company called FerGene to continue development and commercialization of the bladder cancer treatment. The partners backed FerGene with a combined $570 million. An application seeking FDA approval of Adstiladrin was turned down in 2020 due to manufacturing issues. Two months ago, Ferring and Blackstone restructured their alliance to give Ferring full control of FerGene and its bladder cancer gene therapy. In a news release announcing regarding the FDA approval, Ferring said it expects Adstiladrin will become commercially available in the second half of 2023 after the company expands its manufacturing capacity. Approval of Adstiladrin is the second affirmative FDA decision for Ferring in the past month. In late November, the agency approved Ferring’s Rebyota, the first microbiota-based live biotherapeutic for preventing recurrence of Clostridioides difficile infection in adults. Image: Getty Images, magicmine

Dive Brief:After years of testing and an initial rejection from the Food and Drug Administration, Ferring Pharmaceuticals on Friday won U.S. approval of its gene therapy for a form of high-risk bladder cancer.Bladder cancer is one of the most common forms of cancer. Most newly diagnosed cases are classified as non-muscle invasive bladder cancer, or NIMBC. Ferrings treatment, called Adstiladrin, is for high-risk NIMBC that is not responsive to Bacillus Calmette-Gurin therapy.Adstiladrins approval was based on the results of a Phase 3 trial of 157 patients. While its now cleared for use, Ferring doesnt expect to make it available on the U.S. market until the second half of next year as it works to expand its manufacturing capacity.Dive Insight:Also known as nadofaragene firadenovec, Adstiladrin has been in development since 2011, originating at a research institute in Finland. Its original owner, FKD Therapies Oy, licensed the treatment to Ferring in 2018.One year later, Ferring partnered with Blackstone Life Sciences to invest $570 million in development of the therapy for high-risk BCG-unresponsive NMIBC. At the time, the two companies set up a subsidiary called FerGene to commercialize the drug in the U.S.In December 2019, Ferring revealed positive results from a Phase 3 trial, showing that just over half achieved a complete response, or disappearance of all signs of cancer, following treatment. According to the FDA, the median duration of response was 9.7 months and 46% of those who responded remained in remission for at least one year.The companys first attempt to secure approval ended with a complete response letter from the FDA over manufacturing issues, however. FerGene later restructured.Adstiladrin is an adenovirus vector-based gene therapy that contains the gene interferon alfa-2b. Administered via a catheter into the bladder once every three months, the therapy delivers the gene to bladder cells, turning the patients own bladder wall cells into interferon microfactories, according to Ferring.The approval follows several other recent gene therapy OKs from the FDA. Bluebird bio recently received clearances for its drugs Zynteglo and Skysona, while CSL recently won approval of its hemophilia B treatment Hemgenix.Ferring, meanwhile, had a microbiota-based treatment cleared by the FDA for a recurrent type of gut infection earlier this month. '