Ipsen Partners with Blueprint Medicines in $510M Deal to Advance Precision Therapy BLU-782 for Fibrodysplasia Ossificans Progressiva

Today, Blueprint Medicines, a company dedicated to developing precision therapies, announced that it has entered into a research and development agreement with Ipsen through its subsidiary Clementia Pharmaceuticals. The two parties will jointly develop the ALK2 inhibitor BLU-782 for the treatment of patients with fibrodysplasia ossificans progressiva (FOP). This collaboration not only advances Blueprint Medicines’ goal of rapidly developing BLU-782 but also strengthens Ipsen’s R&D pipeline in the field of rare diseases.

Fibrodysplasia Ossificans Progressiva (FOP) is an extremely rare genetic disorder caused by mutations in the ACVR1 gene, also known as ALK2. The ACVR1 protein belongs to the bone morphogenetic protein (BMP) type I receptor family, which regulates skeletal and muscular growth and development. FOP is also commonly referred to as "Stone Man Syndrome," a term reflecting the progressive immobilization of patients as their bodies become stone-like. The hallmark symptom of this rare disease is the heterotopic ossification of soft, elastic muscles and connective tissues into bone following minor trauma-induced inflammation.

▲Skeletal specimen from a patient with fibrodysplasia ossificans progressiva (FOP) (Image source: Mütter Museum official website)

BLU-782, developed by Blueprint Medicines, is a highly selective oral ALK2 inhibitor that directly targets ACVR1 mutants to inhibit abnormal protein activity. It has currently demonstrated favorable tolerability in Phase 1 clinical trials. Preclinical data indicate that BLU-782 has the potential to prevent heterotopic ossification caused by injury and surgery, reduce edema, and restore the resilience of healthy tissue following muscle injury. Previously, the FDA granted BLU-782 Rare Pediatric Disease Designation, Orphan Drug Designation, and Fast Track Designation.

Under the terms of the agreement, Ipsen will leverage its expertise and clinical infrastructure in rare disease treatment to accelerate the development of BLU-782. Blueprint Medicines will receive a $25 million upfront payment and is eligible for up to $510 million in research, regulatory, and sales milestone payments, as well as tiered royalties on net sales.

“We believe that Ipsen’s expertise and clinical experience in treating rare genetic diseases can facilitate the global development of BLU-782,” said Mr. Jeff Albers, Chief Executive Officer of Blueprint Medicines. “We are pleased to see BLU-782, the first investigational therapy targeting ALK2, the driver gene of FOP, enter the clinical development stage. We also extend our gratitude to the Blueprint Medicines team for their relentless efforts in the development of BLU-782.”

“Previously, through the acquisition of Clementia Pharmaceuticals, we obtained the development rights to palovarotene for the prevention of flare-ups of heterotopic ossification in patients with fibrodysplasia ossificans progressiva (FOP),” said David Meek, Chief Executive Officer of Ipsen. “With this agreement, we have now also secured the development rights to BLU-782. We remain committed to developing and delivering valuable therapies for patients with FOP and other rare diseases.”

References:

[1] Ipsen and Blueprint Medicines Announce Exclusive Global License Agreement to Develop and Commercialize BLU-782 for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP), Retrieved October 16, 2019, from https://www.businesswire.com/news/home/20191015006093/en

▽Follow [WuXi AppTecDe】WeChat Official Account