Vertex to buy cystic fibrosis drug from Concert Pharmaceuticals

CTP-656 is an investigational cystic fibrosis 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 cystic fibrosis (CF). As part of the agreement, Vertex will pay Concert $160m in cash for all worldwide development and commercialization rights to CTP-656.

If CTP-656 is approved as part of a combination regimen to treat CF, Concert could receive up to an additional $90m in milestones based on regulatory approval in the U.S. and reimbursement in the UK, Germany or France.

The 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.

Vertex executive vice president and chief medical officer Jeffrey Chodakewitz said: "Our vision is to develop the most effective and convenient medicines for people with CF.

"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."

CTP-656 was developed by Concert through the application of deuterium chemistry to modify Vertex's CFTR potentiator, ivacaftor. Ivacaftor was discovered by Vertex scientists and is approved in the US, Europe, Canada and Australia for people with CF who have specific mutations in the CFTR gene.

CTP-656 has the potential to play a key role in future once-daily combination regimens to treat CF. Concert is currently conducting a Phase 2 study of CTP-656 in people with CF who have gating mutations. As part of the agreement, Vertex will acquire rights to all of Concert's other CF research and preclinical programs.