Potential New Cystic Fibrosis Therapy, CTP-656, Begins Phase 1 Study

Potential New Cystic Fibrosis Therapy, CTP-656, Begins Phase 1 Study

Concert Pharmaceuticals, Inc., a clinical stage biopharmaceutical company focused on utilizing its DCE Platform® (deuterated chemical entity platform) to create novel small molecule drugs, recently announced that it has initiated a Phase 1 multiple ascending dose clinical trial for its drug candidate CTP-656 for the treatment of cystic fibrosis (CF).

CTP-656 is a novel potentiator that was developed by applying deuterium chemistry to modify ivacaftor, which is known commercially as Kalydeco® and marketed by Vertex Pharmaceuticals. Concert is initially developing CTP-656, which may enable once-daily dosing for CF patients, as a potential monotherapy in class III (gating) mutations (e.g. G551D) of the gene that encodes for cystic fibrosis transmembrane conductance regulator (CFTR), a protein that regulates components of sweat, mucus clearance, and digestion.

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 clinicaltrials.gov.

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.

Cystic fibrosis (CF), is a genetic disorder that affects mostly the lungs but also the pancreas, liver, kidneys, and intestine. Long-term complications of the disease include difficulty breathing and coughing up mucus as a result of frequent lung infections.

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