Clinical-stage biotech Concert Pharmaceuticals (NASDAQ:CNCE) traded ~7% higher in the pre-market Monday after the company announced that its second Phase 3 trial for CTP-543 reached primary endpoint with statistical significance in patients with alopecia areata.
An autoimmune disorder alopecia areata causes patchy or complete scalp hair loss. The multinational THRIVE-AA2 trial, was designed to evaluate the safety and efficacy of CTP-543 in 517 adult patients in the U.S., Canada and Europe.
Based on topline data, the trial approached primary efficacy endpoint of percentage of patients achieving an absolute Severity of Alopecia Tool (SALT) score of 20 or less at Week 24 of treatment with statistical significance. The efficacy was seen for both 8 mg twice-daily and 12 mg twice-daily dose of the oral therapy compared to the placebo.
However, two patients in the 8 mg twice-daily arm and two patients in the 12 mg twice-daily arm developed herpes zoster (shingles). Out of five serious adverse events, only one in the 8 mg twice-daily dose group was found to be treatment-related.
The data are yet to undergo the peer-review which typically precedes the publication in a medical journal.
Concert (CNCE) expects to file a New Drug Application (NDA) to the FDA in the 1H 2023 seeking marketing application for CTP-543 based on these data and data from THRIVE-AA2, the first late-stage trial for the therapy.