Baricitinib Efficacy Through 4 Years in Responders and Safety up to 5 Years in Adults with Severe Alopecia Areata: Final Results from BRAVE-AA1 and BRAVE-AA2

Long-term efficacy and safety data are imperative for therapies used to treat severe alopecia areata (AA) because the disease is chronic, loss of response can be devastating to patients, and the safety profile needs to support long-term therapy. Baricitinib, a selective JAK inhibitor, has been approved for severe AA in adults and is being studied in pediatric patients. This study evaluates the final long-term efficacy and safety of baricitinib in adults with severe AA. Efficacy was assessed for patients randomized to baricitinib 4 mg or 2 mg who achieved response (Severity of Alopecia Tool [SALT] score ≤ 20) at Week 52 (4 mg, N = 133; 2 mg, N = 72) and stayed on the same dose through Week 200. Proportions of patients maintaining a SALT score ≤ 20 and achieving a Clinician-Reported Outcome Measure for Eyebrow/Eyelash (EB/EL) Hair Loss™ score of 0 or 1 with ≥ 2-point improvement from baseline were reported using the modified last-observation-carried-forward method. Safety is reported as incidence rates (IR) per 100 patient-years at risk for the All BARI AA group (N = 1303) and Extended BARI AA group (N = 948) through 248 weeks of exposure For baricitinib 4 mg and 2 mg, respectively, among Week 52 responders, 115/133 (86.5%) and 61/72 (84.7%) maintained a SALT score ≤ 20 at Week 200. Regrowth of EB/EL hair continued beyond Week 52 and was maintained through Week 200. In up to 5 years of exposure with baricitinib, the IR for serious infections, nonmelanoma skin cancer malignancies, deep vein thrombosis/pulmonary embolism, and major adverse cardiovascular events remained low relative to background rates. No deaths occurred, and adverse event rates remained stable over time. Within this population, ~ 85% of responders maintained SALT scores ≤ 20 through approximately 4 years with continued benefit for EB/EL. Baricitinib’s safety profile remained consistent over 5 years of treatment, in line with prior BRAVE program findings. BRAVE-AA1, ClinicalTrials.gov number, NCT03570749, start date, September 24, 2018; BRAVE-AA2, ClinicalTrials.gov number, NCT03899259, start date, July 8, 2019. Alopecia areata is an autoimmune disease caused when the immune system mistakenly attacks hair follicles, causing patchy or complete hair loss on the scalp, face, and body. The disease can be emotionally devastating to those affected, and severe cases do not usually improve without treatment. Since the disease often requires long-term treatment to maintain hair regrowth, studies examining the long-term benefits and risks of treatment are important. Baricitinib is a medication taken by mouth which is approved to treat severe alopecia areata in adults and is also being studied for use in children. This report describes the results from two studies in adults with alopecia areata, BRAVE-AA1 and BRAVE-AA2, that tested baricitinib's efficacy in regrowing hair and maintaining that regrowth over 4 years of treatment. Among patients who regrew their hair after 1 year of treatment with baricitinib, 85% maintained that regrowth for approximately 4 years of continued treatment. Hair regrowth in other areas such as the eyebrows and eyelashes continued to improve and be maintained over time. The observed profile remains consistent with the previously reported baricitinib safety profile in the BRAVE program. Overall, this long-term study provides evidence that ~ 9 out of 10 adults with severe alopecia areata who are treated with baricitinib maintain hair regrowth once they achieve it, and the safety profile remains consistent for up to 5 years of treatment.