Vertex Acquiring Cystic Fibrosis Treatment CTP-656 from Concert Pharmaceuticals

Vertex Pharmaceuticals is acquiring CTP-656, a potentiator treatment for the protein that is defective in cystic fibrosis, from Concert Pharmaceuticals.

CTP-656, a deuterium-modified version of the therapy ivacaftor, is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, meaning that it enhances the activity of the CFTR protein.

CTP-656 has received orphan drug designation in the United States, Europe, Canada and Australia. It has the potential to play a key role in once-a-day combination regimens of CFTR modulators as a treatment for cystic fibrosis.

Under the agreement, Vertex will give Concert $160 million for the worldwide rights to CTP-656. Concert will receive up to $90 million more if the United States approves CTP-656 as part of a combination regimen to treat cystic fibrosis, and if the United Kingdom, France or Germany agree to reimbursements for the therapy.

The deal also gives Vertex rights to all of Concert’s other cystic fibrosis research and preclinical programs.

“Our vision is to develop the most effective and convenient medicines for people with CF,” Jeffrey Chodakewitz, MD, executive vice president and chief medical officer at Vertex, said in a press release. “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.”

Concert developed CTP-656 by using deuterium chemistry to modify Kalydeco (ivacaftor).

Patients with a CFTR mutation develop thick, sticky mucus in their respiratory, digestive, and reproductive systems. Kalydeco acts on CFTR proteins at the cells’ surface to allow salt and water to move through the cells, improving hydration and mucus clearance.

CTP-656 is also a potential stand-alone therapy for CF associated with gating mutations of the CFTR gene.

Concert started a randomized, double-blind, placebo-controlled Phase 2 clinical trial (NCT02971839) in December 2016 to evaluate the effectiveness and safety of CTP-656, compared with Kalydeco, in 40 CF patients with gating mutations of the defective CFTR gene. The trial is being conducted in nine U.S. states, with main results expected by the end of the year.

Last year, Concert reported positive results from a Phase 1 clinical trial (NCT02599792) evaluating the safety and tolerability of increasing concentrations of CTP-656, compared with a single dose of Kalydeco. CTP-656 outperformed Kalydeco and was found to be well-tolerated.