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Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced the publication of safety and efficacy data from the randomized, double-blind, placebo-controlled Phase 2 clinical trial of 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, an autoimmune disease in which the immune system attacks the hair follicles and causes hair loss. The publication reports clinically significant and statistically significant scalp hair regrowth after 24 weeks of treatment with CTP-543 in the 8 mg twice daily and 12 mg twice daily dose groups in patients with CTP-543. alopecia areata.
“We have made so much progress in alopecia areata in such a short time, and it is absolutely exciting to see JAK inhibitors progress as a potential treatment for alopecia areata,” said Brett King, MD, Department of Dermatology , Yale University School of Medicine. and lead author of the publication. “The therapeutic response in patients as early as 12 weeks in this trial is significant, and we hope that with continued development, CTP-543 will become a treatment option for the many people suffering from this serious autoimmune disease.”
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. Additionally, the patient-reported improvement results after CTP-543 treatment were statistically significant.
The robust clinical results seen in the trial supported U.S. Food and Drug Administration (FDA) Breakthrough Therapy Designation and the progression of CTP-543 into the pivotal Phase 3 THRIVE-AA clinical program. The company recently reported positive frontline data from the THRIVE-AA1 trial, and the first data from the THRIVE-AA2 trial is expected in the third quarter of 2022.
“We are very pleased that the results of this trial are being published in this very prestigious journal and that its wide readership in the field of dermatology will see the first published results of CTP-543 in patients with alopecia areata,” said James V. Cassella. , Ph.D., director of development for Concert Pharmaceuticals and co-author of the publication. “Today J.A.D. The publication further highlights the growing number of publications showing the importance of the JAK mechanism in alopecia areata and the potential therapeutic utility of CTP-543 as a new treatment option for this important medical condition.
CTP-543 Phase 2 Trial Results
The Phase 2 trial was a randomized, double-blind, placebo-controlled, dose-sequential trial to evaluate the safety and efficacy of CTP-543 in adult patients with moderate to severe alopecia. 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, given twice daily. The primary endpoint used the Alopecia Severity Tool (SALT) after 24 weeks of treatment.
Patients treated with 8 mg twice daily or 12 mg twice daily of CTP-543 reached the primary efficacy endpoint with statistically significant differences (p
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 interruption of treatment.
A copy of the publication entitled “Phase 2 randomized, dose-ranging trial of CTP-543, a selective Janus Kinase inhibit, in moderated-to-severe alopecia areata” is available on line on the J.A.D. website and additional materials are available at Mendeley.
About CTP-543 and alopecia areata
CTP-543 is an oral selective inhibitor of the Janus kinases JAK1 and JAK2.
Alopecia areata is an autoimmune disease in which the immune system attacks the hair follicles, resulting in partial or complete loss of hair on the scalp and body. Alopecia areata can affect up to approximately 1.5 million Americans at any one time1. The scalp is the most commonly affected area, but any hairy site can be affected alone or with the scalp. The 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 FDA-approved drugs for the treatment of alopecia areata.
The FDA selected alopecia areata as one of eight new disease areas it focused on as part of its Patient-Oriented Drug Development Initiative (PFDDI) in 2016-2017. The goal of PFDDI is to bring patient perspectives to an early stage of product development. Following the FDA’s September 2017 Patient-Oriented Drug Development meeting on alopecia areata, the FDA has summarized feedback shared by patients and patient representatives in a Patient’s voice report. Additional information on the PFDDI is available on line.