Cystic fibrosis drug enters clinical trials

Concert Pharmaceuticals has initiated a phase one multiple ascending dose clinical trial with CTP-656 for cystic fibrosis

CTP-656 (deuterium-modified ivacaftor) is a treatment for cystic fibrosis for use as monotherapy as well as in combination with other CFTR modulators. Ivacaftor is commercially available under the name Kalydeco.

James Cassella, chief development officer of Concert Pharmaceuticals, said: “CTP-656 has the potential to be an important new treatment, expanding therapeutic options for the cystic fibrosis community.”

The multiple ascending dose phase one 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.

Concert said it expects results from the study in the first half of 2016.

Cassella said: “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.”