Concert Pharmaceuticals initiates CTP-543 Phase II trial in alopecia areata

Trial designed to evaluate the safety and efficacy of CTP-543 after 12 months of dosing with the primary efficacy analysis at week 24.

Concert Pharmaceuticals has initiated a Phase IIa clinical trial evaluating 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 an orally-administered selective inhibitor of Janus kinases 1 and 2, known as JAK1 and JAK2, which are enzymes believed to be involved in this autoimmune disorder. The Phase IIa trial is designed to evaluate the safety and efficacy of CTP-543 after 12 months of dosing with the primary efficacy analysis at week 24. The company expects to report topline data in the first quarter 2018.

"Alopecia areata is a devastating autoimmune disease that impacts half a million or more patients in the US at any given time. There is a significant unmet medical need with no FDA-approved therapy," said Roger Tung, President and CEO of Concert Pharmaceuticals. "We intend to be at the forefront of advancing a new oral treatment for alopecia areata patients and look forward to assessing CTP-543's therapeutic efficacy in the Phase II trial."

"The treatment community, and patients suffering from this disease, have been eager for new treatments for alopecia areata. Though more work needs to be done to explore safety and efficacy, there is a growing and encouraging body of evidence supporting the Janus kinase mechanism in this indication," stated Julian Mackay-Wiggan, Associate Professor of Dermatology and Director of Clinical Research in Dermatology at Columbia University. "I believe this is a new era in which we are advancing treatments into clinical evaluation that target the underlying biology of the disease."

The Phase IIa trial is a double-blind, randomized, placebo-controlled, parallel dose trial to evaluate the safety and efficacy of CTP-543 in adult patients with moderate-to-severe alopecia areata. Approximately 100 patients will be randomized to receive one of four doses of CTP-543 (4, 8, 12 and 16 mg twice daily) or placebo. The primary outcome measure will utilize the severity of alopecia tool (SALT) after 24 weeks of dosing. The trial will include an additional 28 weeks of dosing where all patients enrolled in the study will receive CTP-543.