Vertex purchases Concert Pharmaceuticals' investigational cystic fibrosis drug CTP-656 for $160 million

Vertex Pharmaceuticals on Monday announced an agreement to acquire Concert Pharmaceuticals' experimental cystic fibrosis ​drug​​ CTP-656 ​under a deal ​potentially ​worth as much as $250 million​, including a cash payment of $160 million​. The drugmaker noted that the CFTR potentiator ​​is a longer-acting version of its cystic fibrosis drug Kalydeco (ivacaftor), which has been approved in several markets for patients who carry specific mutations in the CFTR gene.

According to Vertex, CTP-656, which was developed using deuterium chemistry to modify Kalydeco, could have a critical role in future once-daily regimens for the treatment of cystic fibrosis.  Chief medical officer Jeffrey Chodakewitz remarked "we look forward to exploring once-daily regimens that combine CTP-656 with other potential medicines from our broad CF pipeline that treat the underlying cause of the disease."

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Under the agreed terms, Vertex will receive all global development and commercialisation rights to CTP-656, with Concert Pharmaceuticals eligible to receive up to $90 million in milestones based on regulatory clearance in the US as part of a combination regimen and reimbursement in France, Germany or the UK. Vertex will additionally gain rights to Concert Pharmaceuticals' other research and preclinical programmes in cystic fibrosis. Shares in Concert Pharmaceuticals surged as much as 34 percent on the news.

Concert Pharmaceuticals initiated a mid-stage study of CTP-656 for patients with cystic fibrosis and gating mutations in December 2016. The therapy was awarded orphan drug designation by the FDA the following month.

For related analysis, read ViewPoints: Vertex kills two birds with one deal.

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