Concert Pharmaceuticals Reports Positive Topline Results for Second CTP‑543 Phase 3 Clinical Trial in Alopecia Areata
THRIVE-AA2 Study Met Primary Endpoint for Scalp Hair Regrowth
Key Secondary Endpoint Met for Patient Reported Outcome on Hair Satisfaction
Key Secondary Endpoint Met for Hair Regrowth as Early as 12 Weeks
CTP-543 Has Potential to be Best-in-Class for the Treatment of Alopecia Areata
Company Completes Pivotal Program with Two Positive Phase 3 Trials and Expects to File New Drug Application in First Half of 2023
“It is a new era of innovation for developing treatment options for patients with alopecia areata, many of whom often suffer physically and mentally with this challenging autoimmune disease,” said
“With the successful completion of our two THRIVE-AA Phase 3 trials, Concert is now moving rapidly to prepare our NDA for submission to the
Patients enrolled in THRIVE-AA2 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 87.9 (corresponding to approximately 12% 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 20 percent or less scalp hair loss) was 38.3 percent in the 12 mg twice-daily dose group and 33.0 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 key secondary endpoints were the percentage of responders on a Satisfaction of Hair Patient Reported Outcome (SPRO) 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. 47% of patients in the 8 mg twice-daily group and 52% of patients in the 12 mg twice-daily group reported being “satisfied” or “very satisfied,” as compared to 2% of patients in the placebo group. The treatment difference for both groups relative to placebo was statistically significant. SALT scores of 20 or less at Weeks 20, 16 and 12 were statistically significant in both dose groups.
The safety profile seen with CTP-543 in THRIVE-AA2 was consistent with previous studies. The most common (≥5%) side effects in any dose group were COVID-19 infection, nasopharyngitis, increased creatine kinase levels, acne and headache. No pulmonary embolisms or deep vein thromboses were observed in the trial. Two patients treated with the 8 mg twice-daily dose and two patients treated with the 12 mg twice-daily dose developed herpes zoster (shingles). Five serious adverse events were reported in five patients, with only one in the 8 mg twice-daily dose group that was assessed as possibly related to treatment.
Concert expects to submit the full results from this study for future scientific publication and presentation. These data, along with data from the first Phase 3 clinical trial, THRIVE-AA1, are intended to form the basis of a New Drug Application (NDA) planned to be submitted to the
THRIVE-AA2 (NCT04797650) is a randomized, double-blind, placebo-controlled clinical trial in 517 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 limited treatment options available for 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 and the planned timing for filing 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, including safety profiles, 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 the timing of the submission of an NDA, the availability of 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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