Concert Pharmaceuticals announced final topline results from its recently completed dose-ranging Phase 2 trial evaluating CTP-543 in patients with moderate-to-severe alopecia areata, an autoimmune disorder that results in patchy or complete hair loss. The primary endpoint was reached in two of the three CTP-543 dose levels tested. A phase 3 trial is expected to begin in 2020.
The Phase 2 trial was a double-blind, randomized, placebo-controlled, sequential dose trial in adult patients with moderate-to-severe alopecia areata. A total of 149 patients were randomized to receive one of three doses of CTP-543 (4 mg, 8 mg, or 12 mg) or placebo, administered twice-daily.
Patients treated with either 8 mg twice-daily or 12 mg twice-daily of CTP-543 met the primary efficacy endpoint with statistically significant differences relative to placebo in the percentage of patients achieving a ≥ 50% relative change from baseline at 24 weeks using the Severity of Alopecia Tool (SALT). The 8 mg twice-daily and 12 mg twice-daily groups were also significantly different from placebo in the number of patients achieving ≥ 75% and ≥ 90% relative change in SALT from baseline at 24 weeks. At Week 24, patients treated with 8 mg twice-daily and 12 mg twice-daily compared to placebo also rated significantly greater improvement in their alopecia areata on the Patient Global Impression of Improvement Scale. Treatment with CTP-543 was generally well tolerated. The most common (≥10%) side effects in the 12 mg CTP-543 dose group were headache, nasopharyngitis, upper respiratory tract infection, and acne.
About 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 650,000 Americans at any given time. 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. There are currently no drugs approved by the FDA for the treatment of alopecia areata.
CTP-543 is an oral selective inhibitor of Janus kinases JAK 1 and JAK 2. Janus kinases (JAKs) are a group of enzymes that play an important role in the immune system. Research has shown that inhibiting JAKs can be beneficial in treating certain immune-mediated diseases.
The U.S. FDA has granted Fast Track designation for CTP-543.Source: Concert Pharmaceuticals