Vertex Pharmaceuticals will pay up to $250 million for Concert Pharmaceuticals’ cystic fibrosis drug CTP-656. Currently in Phase II clinical trials, the drug is a deuterated version of Vertex’s own compound ivacaftor, which it sells under the name Kalydeco.
Like ivacaftor, CTP-656 is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator. Substituting deuterium atoms for specific hydrogens in the structure generates a novel compound with altered pharmacokinetic properties. Notable is its longer half-life, which means that CTP-656 may need to be given only once per day versus two times daily for Kalydeco.
For Concert, the deal is a validation of its deuterium chemistry approach to drug design and an opportunity for CTP-656 to advance into late-stage development and commercialization. Vertex already leads the cystic fibrosis drug market with Kalydeco and Orkambi, a CFTR potentiator combination it launched in 2015. Sales of the two products, which are used by people with specific mutations in the CFTR gene, reached $1.7 billion in 2016.
Vertex will acquire rights to all of Concert’s other cystic fibrosis research and preclinical programs. Concert intends to use the money to help support itself through 2021, expand its pipeline, and advance its deuterated JAK 1/2 inhibitor CTP-543, now in Phase II testing against the autoimmune disease alopecia areata.
The deal brings at least three benefits to Vertex, Leerink stock analyst Geoffrey C. Porges told clients in a report. It removes a potential competitor and increases the company’s cystic fibrosis franchise with a new compound that can improve its existing therapy combinations and extend its intellectual property protection by at least five years.
Concert’s shareholders and regulators must still approve the sale of the drug to Vertex.