United Therapeutics has announced promising results from its pivotal Phase 3 ADVANCE OUTCOMES study of ralinepag, a new oral treatment for pulmonary arterial hypertension (PAH). Ralinepag is a highly selective prostacyclin (IP) receptor agonist that works through multiple pathways — vasodilatory, anti-proliferative, and anti-inflammatory. It is taken once daily orally, yet achieves sustained receptor activity similar to parenteral (intravenous) therapies, which are far more burdensome for patients.
The study enrolled 687 PAH patients, 80% of whom were already on dual background therapy and showed positive results across all its endpoints.
The primary endpoint was time to first adjudicated clinical worsening event, defined as death, nonelective hospital admission for worsening PAH, initiation of parenteral or inhaled prostacyclin pathway agent for treatment of worsening PAH, disease progression, or unsatisfactory long-term clinical response. Secondary endpoints included changes from Baseline to Week 28 in NT-proBNP, 6MWD, and WHO/NYHA FC; shift and proportion of patients with improved risk status, clinical improvement, health-related quality of life (SF-36) as measured by patient-reported outcomes; time to first all-cause nonelective hospitalization; time to all-cause mortality; heart rate recovery following completion of the 6MWT; and safety and tolerability.
Read more at this link on the United Therapeutics press release
United Therapeutics plans to present the full study results at an upcoming international medical conference and intends to submit a New Drug Application (NDA) to the FDA by the second half of 2026.