Concert Pharmaceuticals announces initiation of CTP-354 Phase 1 trial in healthy volunteers

Jun 10 2013

Concert Pharmaceuticals, Inc. today announced that it has initiated a Phase 1 clinical trial with CTP-354. CTP-354 is a novel GABA_A receptor subtype-selective modulator that has demonstrated no sedation at therapeutic doses in preclinical models in contrast to existing GABA_A receptor non-selective agonists, such as benzodiazepines. Concert is developing CTP-354 for the potential treatment of spasticity and chronic pain. The Phase 1 single ascending dose study will evaluate the safety, tolerability and pharmacokinetics of CTP-354 in healthy volunteers. The company also intends to conduct a Positron Emission Tomography (PET) study to assess brain receptor occupancy of CTP-354 in healthy volunteers. Concert expects to report top-line results of the Phase 1 single ascending dose study by year-end.

“Based on CTP-354's preclinical profile, we believe it has the potential to offer patients effective treatment for these chronic conditions with an improved therapeutic profile. We look forward to assessing CTP-354 in our clinical program.”

"There is a significant unmet need for effective and better tolerated agents in the treatment of both spasticity and chronic pain, particularly treatments that would reduce or avoid the adverse events of sedation and ataxia that are commonly associated with existing treatments," said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "Based on CTP-354's preclinical profile, we believe it has the potential to offer patients effective treatment for these chronic conditions with an improved therapeutic profile. We look forward to assessing CTP-354 in our clinical program."

The Phase 1 study is a randomized, double-blind, placebo controlled study to assess single ascending doses of CTP-354 in healthy adult volunteers in the United States. The program advanced into Phase 1 clinical testing following successful preclinical studies with CTP-354 (previously referred to as C-21191). CTP-354 was developed based on an earlier generation of investigational GABA_A receptor modulators that demonstrated promising receptor subtype selectivity, but lacked a favorable pharmacokinetic profile. Concert's selective incorporation of deuterium in CTP-354 significantly improved pharmacokinetic properties in preclinical models while maintaining the desirable pharmacological activity seen with the earlier generation modulators.

SOURCE Concert Pharmaceuticals Inc.