Concert Pharma's CF drug tops Vertex's top-seller in early study

Concert's James Cassella

Concert Pharmaceuticals ($CNCE), at work on a modified version of Vertex Pharmaceuticals' ($VRTX) star cystic fibrosis therapy, said its treatment lasted longer than the reference drug in a small, early-stage trial, stoking hopes for future clinical success.

The big idea at Concert is taking approved or previously studied drugs and reformulating them with deuterium to boost their safety, tolerability and efficacy, creating new--and patentable--products in the process. The company has applied that principle to Vertex's ivacaftor, approved on its own as Kalydeco and as part of a combination therapy called Orkambi, crafting a deuterium-modified drug called CTP-656.

And, looking at results from a 10-patient trial, Concert believes its CF experiment is progressing well. In the study, CTP-656 charted a better pharmacokinetic profile than ivacaftor, Concert said, exhibiting a longer half-life, increased exposure and higher plasma concentrations after 24 hours. That's clear evidence of the effects of deuterium, the company said, and Concert believes the results point to potentially easier dosing with CTP-656 over Vertex's drug.

The Phase I data sent Concert's share price up more than 20% as investors look forward to more clinical results for CTP-656. The company is plotting another Phase I trial testing ascending doses of its drug for the fourth quarter, expecting to take CTP-656 into Phase II next year.

"We intend to develop CTP-656 to improve patient outcomes and expand therapeutic options as a potential single agent treatment, as well as to enable new combination medications so that patients with cystic fibrosis can have a range of treatment options," Concert Chief Development Officer James Cassella said in a statement.

Concert pulled off an $84 million IPO last year on the strength of its deuterium-powered pipeline, which includes a Phase II treatment for diabetic nephropathy and a Phase I drug for spasticity. The company is also working through partnerships with Avanir Pharmaceuticals ($AVNR), Jazz Pharmaceuticals ($JAZZ) and Celgene ($CELG).

- read the results