Concert Pharmaceuticals, Inc. announced that it has initiated its Phase 1 clinical program for CTP-543, which is being developed for the treatment of alopecia areata, an autoimmune disease for which there are currently no approved treatments. CTP-543 was discovered by applying Concert's deuterium chemistry technology to modify ruxolitinib, which is commercially available under the name Jakafi® in the United States for the treatment of myelofibrosis and polycythemia vera. Ruxolitinib has been used to treat alopecia areata in academic settings, including an investigator-sponsored clinical trial, and has been shown to promote hair growth in individuals with moderate to severe disease. Concert's Phase 1 program will assess the safety, tolerability, pharmacokinetic and pharmacodynamic profile of single- and multiple-ascending doses of CTP-543 in healthy volunteers.
"Alopecia areata is a disease that can have devastating effects on patients but which currently lacks approved and effective therapies. We are pleased to broaden Concert's pipeline of clinical drug candidates with the advancement of CTP-543 into clinical testing. CTP-543 is another example of how we have applied our novel deuterium platform to create medicines that represent new treatment options," said Roger Tung, Ph.D., President and Chief Executive Officer of Concert Pharmaceuticals. "Recent advances in our understanding of alopecia areata biology, combined with patient data from several investigator-initiated studies, give us confidence that CTP-543 has potential to be an effective treatment. We hope to move this program quickly through Phase 1 evaluation in order to initiate efficacy studies in patients with alopecia areata next year."
The Phase 1 program is expected to enroll approximately 80 healthy volunteers. Dosing has been initiated to assess the safety, tolerability, and pharmacokinetics of single-ascending oral doses of CTP-543 in a double-blind trial compared to placebo. Following successful completion of the first part of the Phase 1 trial, Concert will assess CTP-543 dosed orally once-daily or twice-daily for seven consecutive days in a double-blind, placebo-controlled, multiple-ascending dose trial. Pharmacodynamics of CTP-543 will also be assessed after a single dose and after 7 days of dosing in the multiple-ascending dose trial.
