Ipsen adds Blueprint's FOP rare disease drug to pipeline for $535m


Ipsen has added to its rare diseases pipeline after striking an agreement with US biotech Blueprint for a  licence to develop and market a drug for the rare disease fibrodysplasia ossificans progressiva (FOP).

The deal with Blueprint Medicines for BLU-782, an ALK2 inhibitor class drug, builds on Ipsen’s acquisition of the Canadian rare diseases biotech Clementia Pharmaceuticals for up to $1.31 billion in February.

That deal brought in palovarotene, a phase 3 drug also in development for FOP, as well as other rare diseases.

Massachusetts-based Blueprint will receive up to $535 million, including $25 million up front, and $510 if the drug meets certain development, regulatory, and sales-based targets and other payments, plus tiered percentage royalties.

In return France's Ipsen will gain a worldwide licence to develop and market BLU-782, and the companies say the arrangements will accelerate the development of the drug, which has just completed a phase 1 study in healthy volunteers.

Preclinical data showed BLU-782 prevented injury- and surgery-induced heterotopic ossification, reduced oedema and restored healthy tissue response to muscle injury.

The FDA has granted a rare paediatric disease designation, orphan drug designation and fast track designation to BLU-782, with the latter allowing for an expedited development and review.

FOP is a rare, severely disabling genetic disorder characterised 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-building pathway.

David Meek, CEO of 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.

Blueprint also develops cancer drugs – it is developing avapritinib for certain gastrointestinal tumours, and its BLU-667 is in development for cancers with RET mutations, with a filing in non-small cell lung cancer planned for early next year.