Vertex to develop CTP-656 for potential use in future combination regimens aimed at treating the underlying cause of CF.

Concert Pharmaceuticals has signed a definitive asset purchase agreement with Vertex Pharmaceuticals under which Vertex will acquire CTP-656. 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 CF. Board of Directors unanimously support the transaction and recommend that Concert’s shareholders vote in favor of it.

As part of the agreement, Vertex will pay Concert $160 million 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 $90 million in milestones based on regulatory approval in the US 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 support the transaction and recommend that Concert’s shareholders vote in favour of it.

"With Vertex’s clinical and commercial expertise in CF, this agreement provides the optimal pathway to rapidly advance the development of CTP-656 for the benefit of CF patients," said Roger Tung, President and CEO of Concert Pharmaceuticals. "The financial strength provided to Concert by this agreement will allow us to advance CTP-543 into pivotal testing and broaden our proprietary development pipeline."

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 II 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.