Concert Pharmaceuticals, Inc. announced the publication of safety and efficacy data from the randomized, double-blind, placebo-controlled dose-ranging Phase 2 clinical trial for CTP-543 in the Journal of the American Academy of Dermatology (JAAD). CTP-543 is an investigational oral Janus Kinase (JAK) inhibitor being evaluated in ongoing clinical trials for the treatment of adults with moderate to severe alopecia areata, an autoimmune disease in which the immune system attacks hair follicles and results in hair loss. The publication reports clinically meaningful and statistically significant scalp hair regrowth after 24 weeks of treatment with CTP-543 in both the 8 mg twice-daily and 12 mg twice-daily dose groups in patients with alopecia areata.

The publication highlights the establishment of two doses of CTP-543 with statistically significant hair regrowth and a generally well tolerated safety profile in patients with moderate to severe alopecia areata. Additionally, patient-reported outcomes of improvement following treatment with CTP-543 were statistically significant. The robust clinical results observed in the trial supported the Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) and the progression of CTP-543 into the THRIVE-AA pivotal Phase 3 clinical program.

The Company recently reported positive topline data from the THRIVE-AA1 trial, and topline data from the THRIVE-AA2 trial is expected in the third quarter of 2022. The Phase 2 trial was a randomized, double-blind, placebo-controlled, sequential dose trial to evaluate the safety and efficacy of CTP-543 in adult patients with moderate to severe alopecia areata. A total of 149 patients were randomized to receive one of three doses of CTP-543 (4 mg, 8 mg or 12 mg) or placebo, administered twice-daily.

The primary outcome measure utilized the Severity of Alopecia Tool (SALT) after 24 weeks of dosing. Patients treated with either 8 mg twice-daily or 12 mg twice-daily of CTP-543 met the primary efficacy endpoint with statistically significant differences (p <0.001) relative to placebo in the percentage of patients achieving a = 50% relative change from baseline at 24 weeks using the SALT score. The 8 mg twice-daily and 12 mg twice-daily groups were also significantly different from placebo in the number of patients achieving = 75% and = 90% relative change in SALT from baseline at 24 weeks.

At Week 24, patients treated with 8 mg twice-daily and 12 mg twice-daily compared to placebo also rated significantly greater improvement in their alopecia areata on the Patient Global Impression of Improvement Scale. For patients in the 4 mg cohort, the relative reduction in their overall SALT score from baseline was not significantly different from placebo. In the Phase 2 study, CTP-543 was generally well tolerated and safety results were consistent with the known safety profile of JAK inhibitors.

There was one serious adverse event reported in the 12 mg dose group where the patient was able to complete the trial after a brief dose interruption. CTP-543 is an oral selective inhibitor of Janus kinases JAK1 and JAK2. Alopecia areata is an autoimmune disease in which the immune system attacks hair follicles, resulting in partial or complete loss of hair on the scalp and body.

Alopecia areata may affect up to approximately 1.5 million Americans at any given time1. The scalp is the most commonly affected area, but any hair-bearing site can be affected alone or together with the scalp. Onset of the disease can occur throughout life and affects both women and men.

Alopecia areata can be associated with serious psychological consequences, including anxiety and depression. There are currently no drugs approved by the FDA for the treatment of alopecia areata. The FDA selected alopecia areata as one of eight new disease areas that it focused on under its Patient-Focused Drug Development Initiative (PFDDI) in 2016-2017.

The goal of the PFDDI is to bring patient perspectives into an earlier stage of product development.