Benzinga | Jul 1, 2021 07:03AM EDT

Concert Pharmaceuticals Presents Update on CTP-543 Long-Term Extension Study in Alopecia Areata During 2nd JAK Inhibitors Drug Development Summit

Concert Pharmaceuticals, Inc. (NASDAQ:CNCE) today will present an update from its ongoing open label, long-term extension study of the investigational medicine CTP-543 in patients with moderate to severe alopecia areata, an autoimmune disorder that results in patchy or complete hair loss. The data show that, relative to previous Phase 2 study results of CTP-543, hair regrowth assessed by the Severity of Alopecia Tool (SALT) was maintained or improved in the great majority of patients through one year of continuous dosing with 12 mg twice-daily of CTP-543. Approximately 57% of participants receiving 12 mg of CTP-543 twice-daily following 52 weeks of dosing achieved a clinically-meaningful SALT score of 20 or less. Dosing in the long-term extension study is ongoing, and patients completing the ongoing Phase 3 THRIVE-AA trials are eligible to enroll in the study. These data will be presented virtually during the virtual JAK Inhibitors Drug Development Summit.

"With its Breakthrough Therapy designation, we are excited about the prospect of CTP-543 for the treatment of alopecia areata as we advance it through its Phase 3 clinical program. Through the Phase 3 program and long-term extension study we are building a robust body of data to support our New Drug Application with the FDA, which we plan to file in early 2023," said James V. Cassella, PhD, Concert's Chief Development Officer, who made the oral presentation at the Summit. "We are committed to understanding how alopecia areata affects the whole person and are working diligently to advance CTP-543 as one of the first FDA-approved therapies for this serious autoimmune condition."

"We believe Janus kinase inhibitors, or JAKs, are an important mechanism in the treatment of autoimmune disorders, including alopecia areata," said Maryanne Senna, MD, principal investigator of the Hair Academic Innovative Research Unit at Massachusetts General Hospital. "Through clinical research, we are at a point where we better understand the utility of this class of medicines and believe for the first-time that new treatments could be on the horizon."

CTP-543 has been generally well tolerated in the long-term extension study with only 4 of the 158 subjects entering the study discontinuing due to adverse events to date. Adverse events are consistent with those previously reported in the Phase 2 trials. Clinical labs for key hematology parameters appear stable across the long-term extension study relative to end of treatment in the Phase 2 trials.

Details from the oral presentation, entitled "Update on Long-Term Safety & Efficacy of CTP-543, an Oral JAK Inhibitor, for the Potential Treatment of Alopecia Areata" will be available at 11:55 a.m. ET in the Scientific Presentations section of Concert's website in connection with the meeting presentation.