United Therapeutics says phase 3 ADVANCE OUTCOMES study met its primary endpoint

United Therapeutics (UTHR) announced that its long-term pivotal phase 3 ADVANCE OUTCOMES study met its primary endpoint, with ralinepag reducing the risk of a clinical worsening event by 55% compared with placebo in patients with PAH. Ralinepag demonstrated durable efficacy in delaying disease progression during the study, in which 80% of patients were on dual background therapy and 70% of patients were considered World Health Organization/New York Heart Association Functional Class II at baseline. Statistically significant improvements relative to placebo were also observed in important secondary endpoints, including six-minute walk distance and change in N-terminal pro-B-type natriuretic peptide, with ralinepag increasing the odds of achieving clinical improvement by 47% from baseline to Week 28. Benefits were consistent across all patient subgroups, including time since diagnosis, disease etiology, baseline 6MWD, and use of background therapies, reinforcing the robustness of the treatment effect and the potential broad therapeutic relevance of ralinepag. Treatment with ralinepag was well-tolerated and the safety profile was consistent with known prostacyclin-related adverse events. No new safety signals were observed.