Concert Pharmaceuticals Reports Positive CTP-543 Results from Interim Analysis of Phase 2a Trial in Patients with Alopecia Areata
CTP-543 Achieved its Primary Endpoint in the 8 mg Twice-Daily Cohort
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“We are very pleased by these initial efficacy and tolerability data for
CTP-543 in patients with moderate-to-severe alopecia areata. We believe
that the extent of efficacy and tolerability of CTP-543 to date supports
its further development,” said
The primary efficacy endpoint in the 8 mg twice-daily cohort was met with 47% of patients achieving a ≥ 50% relative reduction in their overall severity of alopecia tool (SALT) score from baseline compared to placebo (p <0.001). For the 4 mg cohort, 21% of patients achieved a ≥ 50% relative reduction in their overall SALT score from baseline, however these differences were not significantly different from placebo. In the primary analysis, the response observed in the 8 mg twice-daily dose was significantly different than the 4 mg twice daily dose (p < 0.05). The average baseline SALT score across all patients enrolled in the trial was approximately 88.
Compared to placebo, a significant relative reduction in mean SALT score was first observed in the 8 mg cohort at Week 12 (p <0.05).
The most common side effects in the trial were headache, upper respiratory tract infection, cough, acne and nausea. No serious adverse events were reported.
The Phase 2a trial is a double-blind, randomized, placebo-controlled, sequential dose trial to evaluate the safety and efficacy of CTP-543 in adult patients with moderate-to-severe alopecia areata. Patients will be randomized to receive one of three doses of CTP-543 (4 mg, 8 mg, or 12 mg) or placebo twice-daily. The final cohort evaluating a 12 mg twice-daily dose of CTP-543 compared to placebo is ongoing. The primary outcome measure will utilize the severity of alopecia tool (SALT) after 24 weeks of dosing. Results from the complete Phase 2a trial, including the 12 mg cohort, are expected in the third quarter of 2019.
About CTP-543 and Alopecia Areata
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
Alopecia areata is an autoimmune disease that results in partial or
complete loss of hair on the scalp and body that may affect up to
650,000 Americans at any given time1. 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 U.S. Food and
Drug Administration (
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Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations,
plans and prospects, including statements about our expectations on the
clinical development of CTP-543, and other statements containing the
words "anticipate," "believe," "continue," "could," "estimate,"
"expect," "intend," "may," "plan," "potential," "predict," "project,"
"should," "target," "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 of future clinical 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 Securities and Exchange Commission and in other filings that we
make with the
1 Fricke M. Epidemiology and Burden of alopecia areata: a systemic review. Clinical, Cosmetic and Investigational Dermatology. 2015; Vol 8. 397-403.
Jakafi® is a registered trademark of