James V. Cassella
Patients with moderate to severe alopecia areata who were assigned to CTP-543, an oral Janus kinase inhibitor from Concert Pharmaceuticals, achieved a 50% relative reduction in Severity of Alopecia Tool score at 24 weeks, according to data presented at the European Academy of Dermatology and Venereology Congress.
“I believe we are seeing the strongest effect of a JAK inhibitor on alopecia areata reported to date, based on the magnitude of effect that we see and the time courses of these doses,” James V. Cassella, PhD, study author and chief development officer at Concert Pharmaceuticals, told Healio Dermatology. “Especially with the 12-mg twice daily dosing.”
In the double blind, randomized, placebo-controlled trial, adults were randomly assigned to twice-daily CTP-543 at 4 mg (n = 29), 8 mg (n = 38) or 12 mg (n = 36), or placebo (n = 44) for 24 weeks. At baseline, all patients had at least 50% hair loss according to the Severity of Alopecia Tool (SALT).
The primary endpoint was the percentage of patients who achieved a 50% relative reduction in SALT from baseline to week 24. Secondary endpoints included proportion of patients achieving a 75% or 90% relative reduction in SALT, and Patient Global Impression of Improvement.
At week 24, 47% of patients in the 8-mg group and 58% in the 12-mg group achieved a 50% or greater improvement in SALT from baseline (P < .001), compared with 21% in the 4-mg group and 9% in the placebo group. Additionally, patients in the 8-mg group who responded to treatment averaged a 78% improvement in SALT, and those in the 12-mg group averaged an 86% improvement.
Similar results were reported for those achieving 75% or greater change in SALT (29% for 8 mg vs. 7% for placebo, P < .05; and 42% for 12 mg vs. 7% for placebo, P < .001) and 90% or greater change in SALT (16% for 8 mg vs. 0% for placebo, P < .05; and 36% for 12 mg vs. 0% for placebo, P < .001).
Per the Patient Global Impression of Improvement, 58% of patients in the 8-mg group and 78% of those in the 12-mg group considered their alopecia to be “much improved” or “very much improved” at study conclusion compared with 21% of patients in the placebo group (P < .001).
“From a clinical development standpoint, we have identified a non-effective dose and two effective doses, which gives us a profile that looks like the best [treatment] to date,” Cassella added. “With this, given the players in the space, I think it sets a new bar for clinical efficacy in treating alopecia areata.”
According to Cassella, treatment was mostly well-tolerated, with the majority of patients in the 12-mg group enrolling in a long-term open-label extension study. – by Stacey L. Adams and Abigail Sutton
Cassella JV, et al. CTP-543, an oral JAK inhibitor, achieves primary endpoint in phase 2 randomized, placebo-controlled dose-ranging trial in patients with moderate-to-severe alopecia areata. Presented at: 28th European Academy of Dermatology and Venereology Congress; Oct. 9-13, 2019; Madrid.
Disclosure: Cassella is an employee of Concert Pharmaceuticals.