Thursday, December 22, 2016
Concert Pharmaceuticals, Inc. announced the initiation of a U.S.-based Phase 2 clinical trial evaluating CTP-656 (deuterated ivacaftor), a next generation CFTR potentiator being developed for the treatment of cystic fibrosis. CTP-656 was created based on Concert’s application of deuterium chemistry to modify ivacaftor, which is commercially available under the brand name Kalydeco® and is the current standard of care for cystic fibrosis patients with gating mutations. Topline results of the Phase 2 trial of CTP-656 are expected by year-end 2017.
“We are excited to initiate our Phase 2 trial with CTP-656 in individuals with cystic fibrosis,” said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. “In contrast to Kalydeco, Phase 1 clinical results from CTP-656 support dosing it once-daily with food, without regard to the fat content of the food, potentially enabling CTP-656 to support improved adherence and provide real world benefits for patients with cystic fibrosis.”
The Phase 2 clinical trial is a randomized, parallel-group, double-blind, placebo-controlled, clinical trial to evaluate the safety and efficacy of CTP-656 in cystic fibrosis patients with gating mutations. Patients enrolled in the 28-day study will receive one of three doses (20 mg, 100 mg and 150 mg) of CTP-656 once-daily or placebo. There will also be an open-label Kalydeco comparator arm in the trial. Approximately 30-40 patients will be enrolled in the Phase 2 trial which will be conducted in multiple centers in the U.S. The primary endpoint of the Phase 2 trial is a change from baseline in sweat chloride at Day 28. Secondary endpoints include change in percent predicted forced expiratory volume (FEV1) and change from baseline in CFQ-R Respiratory Domain. The study is being conducted within the Cystic Fibrosis Foundation’s Therapeutic Development Network (TDN). Additional information about the Phase 2 clinical trial is available at www.clinicaltrials.gov.
In Phase 1 clinical evaluations, CTP-656 provided superior key exposure parameters compared to Kalydeco. CTP-656 was well-tolerated and its safety profile was comparable to that of Kalydeco. No serious adverse events were reported in Phase 1 studies.
