Concert Pharmaceuticals Reports Positive Topline Results for First CTP-543 Phase 3 Clinical Trial in Alopecia Areata
THRIVE-AA1 Study Meets Primary Endpoint for Scalp Hair Regrowth and Key Secondary Endpoints at Both Doses
Statistically Significant Hair Regrowth Observed as Early as Eight Weeks
CTP-543 Has Potential to be Best-in-Class for the Treatment of Alopecia Areata
Topline Data from Second Phase 3 Trial, THRIVE-AA2, Expected Third Quarter of 2022
The key secondary endpoints were the percentage of responders on a Hair Satisfaction Patient Reported Outcome (PRO) scale at Week 24 and the percentage of patients achieving absolute SALT scores of 20 or less at each of Weeks 20, 16, 12 and 8. All key secondary endpoints were met with statistical significance in both dose groups.
“Today marks an important milestone in advancing new treatments for alopecia areata, and I’m so happy to see such positive results from the first Phase 3 trial with CTP-543,” said
“With these compelling Phase 3 data, we believe that CTP-543 has the potential to be a best-in-class treatment for patients with alopecia areata, a disease that has long been ignored. We are extremely grateful to the patients and teams of clinical research professionals who participate in our trials,” said
Patients enrolled in THRIVE-AA1 were required to have at least 50 percent scalp hair loss due to alopecia areata, as measured by SALT. A SALT score of 100 represents total scalp hair loss whereas a score of 0 represents no scalp hair loss. The average baseline SALT score across all patients was approximately 85.9 (corresponding to less than 15% average scalp hair coverage).
A statistically significant proportion of patients treated with either 8 mg twice-daily or 12 mg twice-daily of CTP-543 experienced greater scalp regrowth compared to placebo. The proportion of patients achieving a SALT score of 20 or less (meaning that 80 percent or more scalp hair coverage was achieved) was 41.5 percent in the 12 mg twice-daily dose group and 29.6 percent in the 8 mg twice-daily dose group, compared to 0.8 percent of patients in the placebo group, at the 24-week endpoint. The treatment difference for both dose groups of CTP-543 relative to placebo was statistically significant (p<0.0001).
The safety profile seen with CTP-543 in THRIVE-AA1 was consistent with previous studies. The most common (≥5%) side effects in any dose group were headache, acne, upper respiratory infection, increased creatine kinase levels, COVID-19 infection and nasopharyngitis. Upper respiratory infections were greater in the placebo group than in either of the CTP-543 dose groups. No pulmonary embolisms or deep vein thromboses were observed in the trial. One patient treated with the 8 mg twice-daily dose and one patient treated with the 12 mg twice-daily dose developed herpes zoster (shingles). Serious adverse events were reported in nine patients, with only one patient (in the 8 mg twice-daily dose group) having events (2) that were assessed as possibly related to treatment. Four patients who reported serious adverse events were in the placebo group.
Concert expects to submit the full results from this study for future scientific publication and presentation. These data, along with data from THRIVE-AA2, a second Phase 3 clinical trial, are intended to form the basis of a New Drug Application (NDA) planned to be submitted to the
THRIVE-AA1 (NCT04518995) is a randomized, double-blind, placebo-controlled clinical trial in 706 adult patients age 18-65 with moderate to severe alopecia areata at sites in the
About CTP-543 and Alopecia Areata
CTP-543 is an investigational oral selective inhibitor of Janus kinases JAK1 and JAK2. The FDA has granted CTP-543 Breakthrough Therapy designation for the treatment of adult patients with moderate to severe alopecia areata and Fast Track designation for the treatment of alopecia areata.
Alopecia areata is an autoimmune disease in which the immune system attacks hair follicles, resulting in partial or complete loss of hair on the scalp and body. Alopecia areata may affect up to approximately 1.5 million Americans at any given time.1 The scalp is the most commonly affected area, but any hair-bearing site can be affected alone or together with the scalp. 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 drugs approved by the FDA for the treatment of alopecia areata.
The FDA selected alopecia areata as one of eight new disease areas that it focused on under its Patient-Focused Drug Development Initiative (PFDDI) in 2016-2017. The goal of the PFDDI is to bring patient perspectives into an earlier stage of product development. Following the FDA’s Patient-Focused Drug Development meeting held in
Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations, plans and prospects, including, among others, statements about our expectations regarding the development of CTP-543, the potential for CTP-543 to be a best-in-class treatment for the treatment of alopecia areata, the timing of availability of clinical trial data and the timing of regulatory filings, including an NDA for CTP-543, and any other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation, timing and design of future clinical trials, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals and other factors discussed in the “Risk Factors” section of our most recent Quarterly Report on Form 10-Q filed with the
1 Benigno M. Clinical, Cosmetic and Investigational Dermatology 2020
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