AbbVie announced positive topline results from the first of two pivotal studies of the Phase 3 UP-AA clinical program evaluating the safety and efficacy of upadacitinib (RINVOQ®; 15 mg and 30 mg, once daily) in adult and adolescent patients with severe alopecia areata (AA) with a mean baseline SALT score of 83.8 (approximately 16% scalp hair coverage).
In Study 2, both doses of upadacitinib achieved the primary endpoint, with 44.6% and 54.3% of patients treated with upadacitinib 15 mg and 30 mg, respectively, reaching 80% or more scalp hair coverage (SALT score ≤ 20) at week 24, compared to 3.4% of patients receiving placebo (p<0.001).
"Often misunderstood as a cosmetic issue, AA is a systemic immune-mediated disease that can cause total hair loss, involving the scalp, eyebrows and eyelashes. People living with AA may face difficulties in managing their disease, which can significantly affect their quality of life," said Kori Wallace, M.D., Ph.D., vice president, global head of immunology clinical development, AbbVie. "UP-AA is the first pivotal program to have ranked and met the rigorous standard of SALT=0, indicating complete scalp hair regrowth. These data underscore AbbVie's commitment to advancing novel treatments that have the potential to improve the lives of individuals with immune-mediated diseases."
36.0% and 47.1% of patients treated with upadacitinib 15 mg and 30 mg, respectively, reached 90% or more scalp hair coverage (SALT ≤ 10), compared to 1.4% of patients receiving placebo at week 24 (p<0.001). Additional key secondary endpoints that were met included percentage of subjects with improvements in eyebrows and eyelashes, as well as the percentage of subjects with complete scalp hair coverage (SALT=0) with both doses of upadacitinib at week 24.
"The sudden and often unpredictable hair loss people living with AA experience can profoundly impact their self-esteem and mental well-being," said Arash Mostaghimi, M.D., M.P.A., M.P.H., associate professor of dermatology and vice chair of clinical trials and innovation, Brigham & Women's Hospital, Harvard Medical School. "There is a pressing need for more treatments that help enable regrowth of scalp and non-scalp hair. I am encouraged by these results that demonstrate the potential of upadacitinib to be an important new treatment option."
The safety profile of both doses of upadacitinib in the 24-week, placebo-controlled period (Period A) was generally consistent with that observed in approved indications. Treatment-emergent serious adverse events occurred in 1.4% and 2.8% of patients in the upadacitinib 15 mg and 30 mg groups, respectively, and none in the placebo group. Discontinuations due to treatment-emergent adverse events (TEAEs) occurred in 0.7% and 1.4% of subjects in the upadacitinib 15 mg and 30 mg groups, respectively, and none in the placebo group. The most common TEAEs observed were acne, nasopharyngitis and upper respiratory tract infection.Serious infections were reported infrequently with 0.7% in the upadacitinib 15 mg group and 1.0% in the upadacitinib 30 mg group, and none in the placebo group. There were no adjudicated MACE, malignancies or deaths reported. One adjudicated venous thromboembolism was reported in the upadacitinib 15 mg group in a patient with multiple risk factors.
About UP-AA Clinical Trial
UP-AA M23-716 was conducted as a single protocol that includes two replicate pivotal studies (Study 1 and Study 2) with randomization, investigative sites, data collection, analysis and reporting independent for each study. The Phase 3 randomized, placebo-controlled, double-blind studies evaluate efficacy and safety of upadacitinib in adult and adolescent subjects with severe alopecia areata. In Study 1 and Study 2 Period A, participants are randomized to one of three groups to receive upadacitinib 15 mg, upadacitinib 30 mg or placebo for 24 weeks. In Study 1 and Study 2 Period B, participants originally randomized to upadacitinib dose groups in Period A will continue their same treatment in Period B for 28 weeks. Participants originally randomized to placebo in Period A will either remain on placebo in Period B, or be randomized in one of two groups, based off of their SALT score at week 24. In total, Study 1 and Study 2 Periods A and B span 52 weeks. Participants who complete Study 1 or Study 2, can join Study 3 and may be re-randomized to receive 1 of 2 doses of upadacitinib for up to 108 weeks.
About RINVOQ
Discovered and developed by AbbVie scientists, RINVOQ is a JAK inhibitor that is being studied in several immune-mediated inflammatory diseases. In human leukocyte cellular assays, RINVOQ inhibited cytokine-induced STAT phosphorylation mediated by JAK1 and JAK1/JAK3 more potently than JAK2/JAK2 mediated STAT phosphorylation. The relevance of inhibition of specific JAK enzymes to therapeutic effectiveness and safety is not currently known.
