The FDA has granted a rare pediatric disease designation, orphan drug designation and fast track designation to BLU-782. 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.
Blueprint pharma trial#
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. BLU-782 was designed by Blueprint Medicines to selectively target mutant ALK2, the underlying cause of FOP, using Blueprint Medicines' proprietary scientific platform.
Blueprint pharma license#
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. 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.
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. 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 (RAR?) agonist. 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 and Blueprint Medicines Corporation 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).
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