Concert Pharmaceuticals has announced that it has initiated a clinical trial for its drug candidate CTP-656 for the treatment of cystic fibrosis.

What makes Concert’s experimental therapy potentially effective is its ease of use, which could improve compliance among CF patients who currently take ivacaftor. “CTP-656 has the potential to be an important new treatment, expanding therapeutic options for the cystic fibrosis community,” said James Cassella, PhD, Chief Development Officer of Concert Pharmaceuticals, in a recent press release. “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 1 clinical trial will be conducted in two distinct parts. Thirty-eight healthy volunteers will be enrolled to assess the composite safety, tolerability and pharmacokinetics. The first part of the trial will evaluate a single dose pharmacokinetic of CTP-656 (150 mg) in comparison to Kalydeco (150 mg). The second part of the trial will evaluate three distinct doses of CTP-656, commencing at 75 mg and advancing to 300 mg, given daily for seven days in comparison to placebo. More information about this Phase 1 clinical trial can be found at

The company is expecting to report top-line data from this clinical trial during the first half of 2016; in the second half of 2016, the company plans to commence a single Phase 2 clinical trial in a population of patients with CF associated with gating or minimal function CFTR mutations.

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