“CTP-656 has the potential to be an important new treatment, expanding therapeutic options for the cystic fibrosis community,” said James Cassella, Chief Development Officer of Concert Pharmaceuticals. “We believe the development of CTP-656 as a once-daily dose could help address adherence issues documented with the current standard of care by offering a simplified treatment option.”
The multiple ascending dose Phase I study will be conducted in two parts and enroll 38 healthy volunteers to assess safety, tolerability and pharmacokinetics of CTP-656 in a tablet formulation. The first part will assess a single dose pharmacokinetic comparison of 150 mg of CTP-656 versus 150 mg of Kalydeco. The second part will assess three doses of CTP-656, starting at 75 mg and up to 300 mg daily for 7 days compared to placebo.
Concert expects to report top-line results from the Phase I multiple ascending dose study in the first half of 2016. The company intends to initiate a single Phase II trial in patients with cystic fibrosis associated with gating or minimal function CFTR mutations in the second half of 2016.
