Royalty Pharma to Pay Potential $500M for Royalties on Ferring’s Gene Therapy

Pictured: Businessmen shaking hands after closing a deal/iStock, AmnajKhetsamtip

Pictured: Businessmen shaking hands after closing a deal/iStock, AmnajKhetsamtip

Swiss company Ferring Pharmaceuticals got a major boost from the world’s largest buyer of biopharma royalties with Thursday's announcement that Royalty Pharma is paying $300 million upfront—and offering $200 million in manufacturing-milestone payments—for access to Ferring’s Adstiladrin, an FDA-approved gene therapy treatment for bladder cancer.

The announcement noted that the milestone payment is “contingent on certain manufacturing goals that are expected to be achieved in 2025 for the FDA-approved intravesical gene therapy that Ferring will make available next month through an early experience program” for patients suffering from high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC).

Royalty Pharma will receive an initial 5.1% royalty on net sales of Adstiladrin in the U.S., which will increase to 8% after Ferring receives its milestone payment and is expected to end by mid-2030.

Jean-Frédéric Paulsen, Ferring’s executive chairman, said the investment “is yet another demonstration of the value and confidence in our gene therapy Adstiladrin to address significant unmet medical needs for patients.”

The gene therapy was approved by the FDA in December 2022 and in June 2023 the regulator also approved a manufacturing scale-up process. The company’s goal is to release Adstiladrin in the U.S. by the second half of 2023.

The gene therapy is a non-replicating adenovirus vector-based treatment that works by bringing the gene-encoding interferon alfa-2b protein into the bladder. Internal cellular machinery then results in cells producing and releasing interferon alfa-2b protein, which is a recombinant analog of a natural cancer-fighting protein. The treatment targets adult patients with BCG-unresponsive NMIBC.

Bladder cancer is the sixth-most common cancer in the U.S., with NMIBC representing 75% of new cases, according to the European Association of Urology. BCG is the first-line standard of care, but many patients end up developing BCG-unresponsive forms of the cancer, prompting the need for an alternative treatment. Adstiladrin, which is administered once every three months into the bladder via a urinary catheter, is the first gene therapy-based treatment for this form of cancer.

Royalty Pharma CEO Pablo Legorreta said in a statement Thursday that the company believes the gene therapy has “blockbuster potential and we are pleased to provide funding to support the launch of Adstiladrin and help Ferring reach as many patients as possible with this important therapy in the United States.” Legorreta also noted that this represents Royalty Pharma’s first investment in a gene therapy.

Connor Lynch is a freelance writer based in Ottawa, Canada. Reach him at lynchjourno@gmail.com.

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