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Vertex buys Concert’s cystic fibrosis drug

Acquisition validates deuterated drug design and expands firm’s position in disease area

by Ann M. Thayer
March 8, 2017 | A version of this story appeared in Volume 95, Issue 11

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.

CTP-656 (deuterated ivacaftor)
Structure of CTP-656 (deuterated ivacaftor).
CTP-656 (deuterated ivacaftor)

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 CTP-656 may need to be given only once per day versus two times daily for Kalydeco.

For Concert, the deal validates its deuterium chemistry approach to drug design and presents an opportunity for CTP-656 to advance toward 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 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 support itself through 2021 and advance its deuterated JAK 1/2 inhibitor CTP-543, now in Phase II testing to treat 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, adds a new compound that can improve the firm’s existing cystic fibrosis therapy combinations, and extends its intellectual property protection by at least five years.


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