Baricitinib is First JAK-Inhibitor to Demonstrate Hair Regrowth in Phase 3 Alopecia Areata (AA) Trial

Eli Lilly and Company (NYSE: LLY) and Incyte (NASDAQ:INCY) announced today top-line results from BRAVE-AA2, a Phase 3 study evaluating the efficacy and safety of once-daily baricitinib 2-mg and 4-mg in adults with severe alopecia areata (AA). Both doses of baricitinib met the primary efficacy endpoint at Week 36, demonstrating a statistically significant improvement in scalp hair regrowth compared to those randomized to placebo. AA is an autoimmune disease that causes patchy hair loss on the scalp, face and sometimes on other areas of the body that can progress. Baricitinib has received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of AA. This classification aims to expedite the development and review of drugs that are intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over already available therapies on a clinically significant endpoint(s). There are currently no FDA-approved treatments for AA.

"These positive results are very promising and suggest that baricitinib has the potential to address the urgent needs of people living with alopecia areata," said Brett King, M.D., Ph.D., associate professor of Dermatology at Yale School of Medicine. "This level of high-quality research is needed to advance our understanding and the treatment of this frequently devastating disease."

This multicenter, randomized, double-blind, placebo-controlled study included 546 adults with a Severity of Alopecia Tool (SALT) score ≥ 50 (i.e., who had ≥50% scalp hair loss) and a current episode of severe AA lasting at least six months but no more than eight years. The study included a diverse patient population from Argentina, Australia, Brazil, China, Israel, Japan, South Korea, Taiwan and the U.S.

Safety outcomes of baricitinib in BRAVE-AA2 were consistent with its established safety profile in patients with rheumatoid arthritis (RA) and atopic dermatitis (AD). No deaths, major adverse cardiovascular events (MACE) or venous thromboembolic events (VTEs) were reported in the study.

BRAVE-AA2 is the first Phase 3 study with positive results in patients with AA. Data from an additional Phase 3 study of baricitinib in AA will be available in the first half of this year. Detailed results from the BRAVE program will be presented at an upcoming medical conference and published in a peer-reviewed journal later this year. AA is the second potential treatment indication in dermatology for baricitinib after AD.

"For patients who suffer from alopecia areata, it is not a cosmetic condition, it is a devastating autoimmune disease that can have significant psychological effects. They lose much more than just hair," said Lotus Mallbris, M.D., Ph.D., vice president of immunology development at Lilly. "We are looking forward to sharing the totality of data from the overall clinical development program for baricitinib as a potential first-in-disease treatment for alopecia areata."

"Significant unmet need exists in the treatment of alopecia areata," said Abby Ellison, research director at the National Alopecia Areata Foundation (NAAF). "We appreciate Lilly's important work in this area and are excited that these data could bring us closer to a potential new treatment option for patients."

Baricitinib is an oral JAK inhibitor discovered by Incyte and licensed to Lilly. It is approved and commercially available as OLUMIANT in the U.S. and more than 70 countries as a treatment for adults with moderate to severe active RA and in the European Union and Japan for the treatment of adult patients with moderate to severe AD who are candidates for systemic therapy. Baricitinib is also being investigated in systematic lupus erythematosus (SLE), juvenile idiopathic arthritis (JIA) and COVID-19.