Ipsen acquires Blueprint drug and moves further into ultra-rare disease

pharmafile | October 17, 2019 | News story | Research and Development  

Ipsen has announced that through its subsidiary Clementia Pharmaceuticals, it has entered into an exclusive, worldwide license agreement for the development and commercialisation of BLU-782.

BLU-782 is an oral, highly selective investigation ALK2 inhibitor being developed for the treatment of fibrodysplasia ossificans progressive (FOP).

The agreement ultimately enhances Ipsen’s rare disease portfolio and advances Blueprint Medicines’ goal of rapidly and efficiently developing BLU-782 as a potential treatment for FOP.

Ipsen has already demonstrated its commitment to leadership in ultra-rare disease through the ongoing late-stage clinical development of palovarotene, an investigation retinoic acid receptor gamma (RARy) agonist.

BLU-782 has recently completed dosing in a phase 1 study in healthy volunteers and Ipsen now has the potential to offer the broadest possible collection of treatment option for patients with FOP.

David Merck, CEO at Ipsen, said: “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.”

Under the terms of the 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. Additionally Ipsen will pay Blueprint Medicines tiered percentage royalties ranging from the low-to mid-teens on worldwide aggregate annual net sales of licensed products.

The FDA has already granted a rare paediatric disease designation, orphan drug designation and fast track designation to BLU-782.

Jeff Albers, CEO of Blueprint Medicines, added: “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.”

“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.”

Nik Kiran

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