Roivant, Priovant reports Phase 3 VALOR study results for brepocitinib

Roivant and Priovant Therapeutics announced results from the Phase 3 VALOR study evaluating brepocitinib in dermatomyositis. On the primary endpoint, brepocitinib 30 mg achieved a week 52 mean TIS of 46.5 compared to 31.2 for placebo. A statistically significant difference between brepocitinib 30 mg and placebo on mean TIS was seen at all time points, including as early as week 4. This result represents the first ever positive outcome for a 52-week placebo-controlled trial in DM, and the first ever positive registrational trial for a targeted therapy in DM. Brepocitinib also demonstrated clinically meaningful and statistically significant improvement over placebo on all nine key secondary endpoints. Dose-dependent response between brepocitinib 30 mg and brepocitinib 15 mg was seen consistently across the primary and secondary endpoints. Approximately 75% of patients entered the VALOR study on background steroids, with a mean baseline dose of 12.2 mg/day in the brepocitinib 30 mg arm and 11.3 mg/day in the placebo arm. Of these patients on background steroids, 62% of brepocitinib 30 mg patients achieved a steroid dose less than or equal to2.5 mg/day by the end of the studyand 42% of brepocitinib 30 mg patients were able to come off steroids altogether. Even against this backdrop, clinical improvement in the brepocitinib 30 mg arm was rapid, deep, lasting and broad, both in absolute terms and relative to placebo: Brepocitinib demonstrated clinically meaningful and statistically significant improvement relative to placebo on the CDASI, MMT-8 and HAQ-Disability Index. More than two-thirds of brepocitinib 30 mg patients experienced at least a moderate response, and nearly half experienced a major response; of patients who entered the trial with moderate-to-severe skin disease, 44% on brepocitinib 30 mg achieved cutaneous clinical remission by week 52, compared to 21% on placebo. Both TIS and CDASI achieved statistically significant separation from placebo as early as week 4 and sustained that separation at every assessment out to one year; median time to a TISgreater than or equal to40 response was approximately 8 weeks. The observed brepocitinib 30 mg safety profile was consistent with previous brepocitinib clinical trials. Adverse events of special interest, which included malignancy, cardiovascular events, and thromboembolic events, did not occur with greater frequency in the brepocitinib 30 mg arm than the placebo arm. Priovant plans to file an NDA for brepocitinib in dermatomyositis in the first half of 2026.