Monday, March 06, 2017
Concert Pharmaceuticals, Inc. announced that the results from the Company’s Phase 1 single and multiple ascending dose trial evaluating CTP-543 in healthy volunteers were presented in a poster session at the American Academy of Dermatology’s Annual Meeting in Orlando, FL. Concert is developing CTP-543 for the treatment of moderate-to-severe alopecia areata, an autoimmune disorder in which the immune system attacks hair follicles, resulting in patchy or complete hair loss. CTP-543 is a deuterium-modified analog of ruxolitinib, a drug which is commercially available in the U.S. under the brand name Jakafi® for the treatment of certain blood disorders. The results of the Phase 1 trial support the further evaluation of CTP-543 in patients with alopecia areata. Concert intends to initiate a Phase 2a trial in the first quarter of 2017, with topline results expected by year-end 2017.
“We are highly encouraged by the favorable safety and pharmacokinetic profile of CTP-543 observed in Phase 1, and we believe CTP-543 has the potential to become an important treatment option for individuals with moderate-to-severe alopecia areata,” said James Cassella, Ph.D., Chief Development Officer of Concert Pharmaceuticals. “There is a significant need to develop an effective treatment for alopecia areata and Concert is at the forefront with CTP-543.”
The Phase 1 program was designed to determine the safety, tolerability and pharmacokinetics of CTP-543 in a total of 77 healthy volunteers. In the single ascending dose portion of the program, oral doses of 8, 16, 32 and 48 mg CTP-543 were evaluated. For the multiple ascending dose portion, escalating doses of CTP-543 up to 32 mg daily (studied both as once-daily 32 mg doses and as twice-daily 16 mg doses) were administered for seven consecutive days.
In Phase 1, CTP-543 was well tolerated across all dose groups and there were no serious adverse events reported in the study. The most common reported adverse event was headache. There were no withdrawals or dose modifications related to CTP-543 in the trial.
In the trial, CTP-543 showed increased exposure with increasing doses. The safety and exposure observed with 16 mg of CTP-543 twice daily appeared to be comparable to published findings for the reported exposure of 20 mg ruxolitinib twice daily. CTP-543 had a half-life of approximately 3.3 hours, similar to that reported for non-deuterated ruxolitinib1. In an open-label clinical trial of 12 patients with moderate-to-severe alopecia areata, investigators at Columbia University reported that 20 mg of ruxolitinib administered orally twice daily resulted in significant hair regrowth in 75% of the patients2. Given the overall exposure and safety parameters observed with CTP 543 in the Phase 1 program, the Company has selected four doses (4, 8, 12 and 16 mg twice daily) to evaluate in its double-blind, placebo-controlled Phase 2a trial.