Ipsen, Blueprint Medicines in licence deal for BLU-782 for the treatment of Fibrodysplasia Ossificans progressiva (FOP)

Ipsen and Blueprint medicines have entered a deal which expands Ipsen’s rare diseases portfolio to include BLU-782, a highly selective investigational ALK2 inhibitor for the treatment of FOP, in which Blueprint Medicines is eligible to receive up to $535 million, including a $25 million upfront payment and up to $510 million in potential development.

PARIS & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Regulatory News:

Ipsen (Euronext: IPN; ADR: IPSEY) and Blueprint Medicines Corporation (NASDAQ: BPMC) today announced Ipsen, through its subsidiary Clementia Pharmaceuticals, and Blueprint Medicines have entered into an exclusive, worldwide license agreement for the development and commercialization of BLU-782, an oral, highly selective investigational ALK2 inhibitor being developed for the treatment of fibrodysplasia ossificans progressiva (FOP).

The agreement enhances Ipsen’s Rare Diseases portfolio and advances Blueprint Medicines’ goal of rapidly and efficiently developing BLU-782 as a potential treatment for patients with FOP. Ipsen has demonstrated its commitment to leadership in this complex ultra-rare genetic disorder through the ongoing late-stage clinical development of palovarotene, an investigational retinoic acid receptor gamma (RARγ) agonist. With the addition of BLU-782, which recently completed dosing in a Phase 1 study in healthy volunteers, Ipsen has the potential to offer the broadest possible suite of treatment options for patients with FOP.

“Our strategy has been to build a leading Rare Diseases franchise, and through the recent acquisition of Clementia, we gained a first-in-class asset in palovarotene. Now, with the addition of Blueprint Medicines’ BLU-782, we have two strong complementary drug candidates. We will continue to develop and deliver valuable treatments for patients around the world living with FOP and other rare diseases,” said David Meek, CEO, Ipsen.

“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. “We are inspired by the FOP community, including the patients, families, clinicians and advocacy groups we have had the fortune to work with, as we have advanced this program from an idea to BLU-782, the first investigational therapy targeting ALK2, the genetic driver of FOP, to enter clinical development. We are also grateful for the dedication and drive of our team at Blueprint Medicines whose tireless commitment has brought BLU-782 this far.”

Subject to the terms of the license agreement, Blueprint Medicines will be eligible to receive up to $535 million in upfront, milestone and other payments, including an upfront cash payment of $25 million and 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. In addition, Ipsen will pay Blueprint Medicines tiered percentage royalties ranging from the low- to mid-teens on worldwide aggregate annual net sales of licensed products, subject to adjustment in specified circumstances under the license agreement.

About BLU-782

BLU-782 was designed by Blueprint Medicines to selectively target mutant ALK2, the underlying cause of FOP, using Blueprint Medicines’ proprietary scientific platform. Blueprint Medicines recently completed dosing in a Phase 1 clinical trial of BLU-782 in healthy volunteers and reported preliminary data at the American Society of Bone and Mineral Research Annual Meeting in September 2019, which showed that BLU-782 was well-tolerated at all doses tested. Previously reported preclinical data in a well-characterized, genetically accurate FOP model showed that BLU-782 prevented injury- and surgery-induced heterotopic ossification, reduced edema and restored healthy tissue response to muscle injury. The FDA has granted a rare pediatric disease designation, orphan drug designation and fast track designation to BLU-782.

About fibrodysplasia ossificans progressiva (FOP)

FOP is a rare, severely disabling genetic disorder characterized by progressive heterotopic ossification (HO), or the abnormal transformation of muscle, ligaments and tendons into bone. HO 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.