Concert Biopharmaceuticals Presents CTP-656 Phase 1 Data for Cystic Fibrosis Therapy at NACF Conference

Ana Pamplona, PhD avatar

by Ana Pamplona, PhD |

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Concert Pharmaceuticals will present data from a Phase 1 single ascending dose trial of experimental CF therapy CTP-656, a next generation potentiator developed for the treatment of cystic fibrosis during the 29th Annual North American Cystic Fibrosis Conference this week in Phoenix, Arizona. The study being presented is entitled ”Pharmacokinetic Studies of Deuterated Isotopologs of Ivacaftor in Preclinical Models and Healthy Volunteers.

Cystic Fibrosis (CF) is a rare but serious and well-characterized genetic chronic respiratory disease that results from mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and associated with chronic polymicrobial infections of the lung, such as Pseudomonas aeruginosa (Pa) and Staphylococcus aureus (S. Aureus). The Cystic Fibrosis Foundation estimates that globally 70,000 individuals suffer from cystic fibrosis.

Concert Pharmaceuticals is a clinical stage biopharmaceutical company focused on applying its DCE Platform® (deuterated chemical entity platform) to generate new small molecule drugs derived from already approved drugs, advanced clinical candidates or previously studied compounds with potential for improvement with deuterium replacement to increase clinical safety, tolerability and efficacy. Concert created CTP-656 using deuterium chemistry to alter ivacaftor to generate a new potentiator to be administrated to CF patients once a day.

In the Phase 1 healthy volunteer trial, researchers compared CTP-656 to current standard of care Kalydeco®. CTP-656 appeared to have a superior pharmacokinetic profile, such as reduced rate of clearance, longer half-life, significantly increased exposure and higher plasma levels at 12 and 24 hours, for once a day dosage and similar safety and efficacy as Kalydeco.

“With CTP-656 we aim to develop another treatment option to meaningfully enhance patient care,” said James Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals.

The single ascending dose Phase 1 clinical study was performed in 10 healthy volunteers using three doses (75, 150 and 300 mg) of CTP-656 as an aqueous suspension and were evaluated and compared to commercially available 150 mg of Kalydeco. The candidate CTP-656 showed an enhanced pharmacokinetic and metabolite profile when compared to Kalydeco. However, the in vitro potentiation of the CTP-656 was similar to ivacaftor.

During the last quarter of 2015, Concert will initiate a subsequent Phase 1 trial to assess several ascending doses of CTP-656 in healthy volunteers. This clinical study will include a single dose crossover study where a tablet formulation of CTP-656 will be compared to Kalydeco. These results will be announced in the first half of 2016.