Concert Pharmaceuticals announces CTP-656 solid dose Phase I results confirmed superior pharmacokinetic profile to Kalydeco

Concert is using this tablet formulation of CTP-656 in its ongoing multiple ascending dose Phase I clinical trial and intends to use the same formulation in a Phase II clinical trial.

“We are pleased that the tablet dosage formulation of CTP-656 performed well and confirmed the superior pharmacokinetic profile compared to standard of care. We believe this formulation is suitable for late stage clinical development,” said Roger Tung, President and CEO of Concert Pharmaceuticals. “These CTP-656 results are in line with our goal of providing superior benefits to patients including a more convenient, once-daily medicine that has the potential for improved efficacy.”

Concert has completed Part 1 of a Phase I multiple ascending dose trial to assess CTP-656 in healthy volunteers. Part 1 of the trial, initiated in November 2015, compared a single dose of 150 mg of CTP-656 in a tablet formulation against a single dose 150 mg commercial tablet of Kalydeco in eight subjects, following a high-fat meal. The results confirmed CTP-656’s superior pharmacokinetic profile compared to Kalydeco, including a longer half-life, reduced rate of clearance, substantially increased exposure and greater plasma levels at 24 hours. CTP-656’s half-life of approximately 15 hours supports the potential for once-daily dosing.

With both the solid dose and suspension formulation, the overall metabolite exposure profile of CTP-656 differed from that of Kalydeco. For CTP-656, the greatest exposure in plasma was to parent drug, which is the most active species, whereas with Kalydeco the greatest plasma exposure was to a less-active metabolite.

Part 2 of the multiple ascending dose Phase I trial will assess three doses of CTP-656, ranging between 75 mg and 225 mg, to be administered daily for seven days, compared to placebo. Top-line results are expected in the second quarter of 2016.