Concert Pharmaceuticals has announced that it has initiated enrollment of the second cohort of its phase 2a clinical trial evaluating CTP-543. 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 Janus Kinase (JAK) inhibitor.
An independent Data Monitoring Committee (DMC) conducted an interim safety data review of the first cohort of the phase 2a trial, following 12 weeks of dosing with 4 mg of CTP-543 or placebo twice daily. Based on this review, the DMC provided its recommendation to continue with the current cohort and to initiate dosing of the second cohort, whereby patients will be administered 8 mg of CTP-543 or placebo twice daily for 24 weeks. The Company expects to report topline data from Phase 2a trial in the fourth quarter of 2018.
“The alopecia areata community is eager for an effective and well-characterized treatment for this important autoimmune disease and we are extremely pleased that novel treatments, including CTP-543, are advancing in clinical trials,” said Dory Kranz, president and chief executive officer of the National Alopecia Areata Foundation.
“We are pleased that the CTP-543 trial is progressing as planned as we continue to advance the evaluation of our innovative product candidate for alopecia areata,” said James Cassella, Ph.D., chief development officer of Concert Pharmaceuticals. “There is a significant unmet medical need for alopecia areata and we intend to be at the forefront of advancing a new oral treatment for patients.”
The phase 2a trial is a double-blind, randomized, placebo-controlled trial to evaluate the safety and efficacy of CTP-543 in adults with moderate-to-severe alopecia areata. Approximately 90 patients are being enrolled in the study and sequentially randomized to receive one of two doses of CTP-543 (4 mg or 8 mg) or placebo twice daily. The primary outcome measure will utilize the severity of alopecia tool (SALT) after 24 weeks of dosing. Patient-reported outcome measures will be assessed as secondary endpoints. If appropriate, the protocol may be amended to explore higher doses of CTP-543. Additional information about the trial is available on www.clinicaltrials.gov.
CTP-543 has been well-tolerated in clinical evaluation to date and has demonstrated a non-clinical safety profile consistent with therapeutic JAK inhibition.