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Clinical Trial
. 2022 Aug;87(2):306-313.
doi: 10.1016/j.jaad.2022.03.045. Epub 2022 Mar 29.

Phase 2 randomized, dose-ranging trial of CTP-543, a selective Janus Kinase inhibitor, in moderate-to-severe alopecia areata

Brett King  1 , Natasha Mesinkovska  2 , Paradi Mirmirani  3 , Suzanne Bruce  4 , Steve Kempers  5 , Emma Guttman-Yassky  6 , Janet L Roberts  7 , Amy McMichael  8 , Maria Colavincenzo  9 , Colleen Hamilton  10 , Virginia Braman  10 , James V Cassella  10
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Free article
Clinical Trial

Phase 2 randomized, dose-ranging trial of CTP-543, a selective Janus Kinase inhibitor, in moderate-to-severe alopecia areata

Brett King et al. J Am Acad Dermatol. 2022 Aug.
Free article

Abstract

Background: Janus kinase (JAK) activation is suggested to have a pathological role in alopecia areata (AA). CTP-543, a deuterated compound that selectively inhibits JAK1 and JAK2, is being developed as an oral treatment for AA.

Objective: To assess the safety and efficacy of a 24-week regimen of CTP-543 in patients with chronic, moderate-to-severe AA.

Methods: In this phase 2, randomized, double-blind, placebo-controlled, sequential-design trial, patients were randomized to receive CTP-543 (4 mg, 8 mg, or 12 mg) or placebo every 12 hours for 24 weeks.

Results: A dose-related increase was observed in the percentage of patients with ≥50% relative reduction in Severity of Alopecia Tool scores from baseline at week 24 (9% placebo, 21% 4 mg twice daily, 47% 8 mg twice daily, and 58% 12 mg twice daily), with statistical significance versus placebo (P < .001) observed for the 8-mg twice daily and 12-mg twice daily groups, with differences from placebo noted as early as 12 weeks after the initiation of treatment. Safety results were consistent with the known safety profiles of JAK inhibitors.

Limitations: These initial findings are from a relatively small controlled trial, and additional studies are needed to fully characterize the safety and efficacy of CTP-543 in adult patients with AA.

Conclusions: Patients treated with CTP-543 (8 or 12 mg, twice daily) had a significant reduction in the severity of AA.

Keywords: AA; JAK; JAK inhibitor; JAK1; JAK2; Janus kinase; SALT; alopecia; alopecia areata; alopecia totalis; alopecia universalis; hair loss; ophiasis; patchy hair loss.

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Conflict of interest statement

Conflicts of interest Dr King has served on advisory boards, is a consultant, and/or is a clinical trial investigator for Aclaris Therapeutics Inc, Almirall, Arena Pharmaceuticals, Bristol-Meyers Squibb, Concert Pharmaceuticals Inc, Dermavant Sciences Inc, Eli Lilly and Company, Incyte Corp, Pfizer Inc, TWi Biotechnology Inc, and Viela Bio. He is on speaker bureaus for Eli Lilly, Pfizer Inc, Regeneron, and Sanofi Genzyme. Dr Mesinkovska is an investigator for Concert Pharmaceuticals, Inc, AbbVie, Arcutis, Arena, Eli Lilly and Company, Pfizer Inc, Kadmon, Bristol Myers Squibb, Novartis, Promius, Leo, and Mertz; she is a consultant to and/or has served on advisory boards for Concert Pharmaceuticals, Inc, Eli Lilly and Company, Nutrafol, and the National Alopecia Areata Foundation; she has received an honorarium from Celgene. Dr Mirmirani reports research grants for conducting clinical trials from Concert Pharmaceuticals, Inc, Eli Lilly and Company, and Pfizer Inc. Dr Bruce is an investigator on alopecia trials for Concert Pharmaceuticals, Inc and Eli Lilly and Company. Drs Roberts and Kempers are investigators for Concert Pharmaceuticals, Inc. Dr Guttman-Yassky is an employee of Mount Sinai and has received research funds (grants paid to the institution) from AbbVie, Almirall, Amgen, AnaptysBio, Asana Biosciences, Boerhinger-Ingelhiem, Celgene, Dermavant, DS Biopharma, Eli Lilly and Company, Galderma, Ichnos Sciences, Innovaderm, Janssen, Kiniska, Kyowa Kirin, Leo Pharma, Novan, Pfizer Inc, Ralexar, Regeneron Pharmaceuticals, Inc, Sienna Biopharma, UCB, and Union Therapeutics; and is a consultant for AbbVie, Aditum Bio, Almirall, Amgen, Asana Biosciences, AstraZeneca, Boerhinger-Ingelhiem, Cara Therapeutics, Celgene, Concert Pharmaceuticals, Inc, DBV, Dermira, DS Biopharma, Eli Lilly and Company, EMD Serono, Escalier, Galderma, Ichnos Sciences, Incyte Kyowa Kirin, Leo Pharma, Mitsubishi Tanabe, Pandion Therapeutics, Pfizer Inc, RAPT Therapeutics, Regeneron Pharmaceuticals, Inc, Sanofi, Sienna Biopharma, Target PharmaSolutions, and Union Therapeutics. Dr McMichael is an investigator for Concert Pharmaceuticals, Inc, Aclaris Therapeutics, Allergan, Cassiopea, and Procter and Gamble and is a consultant to and/or has served on advisory boards for Pfizer Inc, Eli Lilly and Company, Galderma, Procter and Gamble, Revian, Almirall, and Allergan. Dr Colavincenzo is or has been an investigator for Eli Lilly and Company and Pfizer Inc. Authors Hamilton and Braman, and Dr Cassella are employees of Concert Pharmaceuticals, Inc and receive salary and stock options.

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