Concert Pharmaceuticals Announces Clinical Hold on CTP-543

Concert Pharmaceuticals received notice from the U.S. FDA that its CTP-543 Phase 2a clinical trial for alopecia areata has been placed on clinical hold.

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The FDA had previously informed the Company that it could initiate the Phase 2a clinical trial. FDA informed the Company that a review of certain recently completed non-clinical toxicology studies is required before proceeding. The FDA did not cite a safety concern; however, they intend to review these additional non-clinical data as support for the one-year dosing duration as planned in the Phase 2a trial.

At the request of the FDA, the Company intends to submit the requested non-clinical study reports promptly. Concert is working diligently with the FDA to resume enrollment in the Phase 2a trial as quickly as possible.

CTP-543 was discovered by applying Concert’s deuterium chemistry technology to modify ruxolitinib, a drug which selectively inhibits Janus kinases 1 and 2 (JAK1 and JAK2) and is commercially available under the name Jakafi in the United States for the treatment of certain blood disorders. Ruxolitinib has been used to treat alopecia areata in academic settings, including an investigator-sponsored clinical trial, and has been reported to promote hair growth in individuals with moderate-to-severe disease.

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