Vertex Pharmaceuticals, a Boston, Massachusetts-headquartered biotechnology company, has agreed to acquire a cystic fibrosis (CF) drug candidate from Concert Pharmaceuticals, a Lexington, Massachusetts-headquartered pharmaceutical company, in a deal worth $250 million.

Under the agreement, Vertex will pay Concert $160 million in cash for all worldwide development and commercialization rights to the drug candidate, CTP-656. If CTP-656 is approved as part of a combination regimen to treat CF, Concert could receive up to an additional $90 million in milestones based on regulatory approval in the US and reimbursement in the UK, Germany, or France.

CTP-656 is an investigational CF transmembrane conductance regulator (CFTR) potentiator that has the potential to be used as part of future once-daily combination regimens of CFTR modulators that treat the underlying cause of CF, according to Vertex. The drug candidate was developed by Concert through the application of deuterium chemistry to modify Vertex’s CFTR potentiator, ivacaftor. Ivacaftor, marketed under the brand name Kalydeco, was discovered by Vertex scientists and is approved in the US, Europe, Canada, and Australia for treating people with CF who have specific mutations in the CFTR gene.

Concert is currently conducting a Phase II study of CTP-656 in people with CF who have CFTR gating mutations. As part of the agreement, Vertex will acquire rights to all of Concert’s other CF research and preclinical programs. This agreement is subject to approval by Concert’s shareholders and the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. Concert’s board of directors unanimously supports the transaction and recommends that Concert’s shareholders vote in favor of it.

Kalydeco (ivacaftor) is a prescription medicine for treating CF in patients age 2 years and older who have one of the following mutations in their CF gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H.

Source: Vertex Pharmaceuticals