7 November 2017 - - Lexington, Massachusetts-based clinical stage biopharmaceutical company Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) has completed enrollment of the first cohort of its Phase 2a trial evaluating CTP-543, the company said.
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 inhibitor that is commercially available under the brand name Jakafi for the treatment of certain blood disorders.
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 or 8 mg) or placebo twice daily.
The primary outcome measure will utilize the severity of alopecia tool 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.
An independent Data Monitoring Committee (DMC) will conduct an interim safety data review from the first cohort after patients have completed three months of dosing.
Based on this review, the DMC will provide its recommendation regarding initiating the second cohort in the trial, which will be administered 8 mg of CTP-543 or placebo twice daily for 24 weeks.
CTP-543 has been well-tolerated in clinical evaluation to date and has demonstrated a non-clinical safety profile consistent with therapeutic JAK inhibition.
Concert Pharmaceuticals focuses on applying its DCE (deuterated chemical entity) Platform to create novel medicines designed to address unmet patient needs.
The company's approach starts with approved drugs in which deuterium substitution has the potential to enhance clinical safety, tolerability or efficacy. Concert has a broad pipeline of innovative medicines targeting autoimmune and inflammatory diseases and central nervous systems disorders.