Marie Daghlian

French biopharmaceutical Ipsen has placed another bet on rare diseases by obtaining an exclusive global license to Blueprint Medicine’s BLU-782, an oral, highly selective investigational ALK2 inhibitor being developed for the treatment of rare bone disease fibrodysplasia ossificans progressiva.

Fibrodysplasia ossificans progressiva (FOP) is a disabling genetic disorder where muscle, ligaments, and tendons abnormally transform into bone. The transformation may be spontaneous or associated with painful episodic disease flare-ups that are usually precipitated by soft tissue injury. As the disease progresses, extra-skeletal bone increasingly restricts joints, resulting in severe disability and loss of mobility, compromised respiratory function, and increased risk of early death. FOP is caused by a mutation in the gene for ALK2, which is known as ACVR1, leading to inappropriate activation of the bone morphogenetic pathway.

Under the terms of their agreement, Ipsen subsidiary Clementia Pharmaceuticals and Blueprint Medicines have entered into an exclusive, worldwide license agreement for the development and commercialization of BLU-782 as a potential treatment for patients with FOP. Blueprint Medicines will receive $25 million upfront and is eligible for up to $510 million in potential milestone payments related to specified development, regulatory and sales-based milestones for licensed products in up to two indications, including FOP. Blueprint Medicines is also eligible for royalties on worldwide aggregate annual net sales of licensed products.

The deal comes eight months after Ipsen paid $1 billion upfront for Clementia Pharmaceuticals for its late-stage experimental treatment palovarotene, an investigational retinoic acid receptor gamma (RARγ) agonist in development for FOP and rare bone diseases. Ipsen says the deal enhances and complements its rare diseases portfolio, while Blueprint Medicines says the deal will advance its goal of rapidly and efficiently developing BLU-782 as a potential treatment for patients with FOP.

BLU-782 selectively targets mutant ALK2, the underlying cause of FOP. Blueprint Medicines recently completed dosing in a phase 1 clinical trial of BLU-782 in healthy volunteers and reported preliminary data in September 2019, which showed that it was well tolerated at all doses tested. The U.S. Food and Drug Administration has granted BLU-782 Rare Pediatric Disease, Orphan Drug, and Fast Track designations.

“We admire Ipsen’s track record of successful global clinical development in this complex, ultra-rare genetic disorder and believe this expertise, combined with Ipsen’s global infrastructure and commitment to transforming the treatment of FOP, will accelerate the development of BLU-782 globally,” said Jeff Albers, CEO, Blueprint Medicines.

Photo: Jeff Albers, CEO, Blueprint Medicines

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