United Therapeutics Corp. announced its experimental drug ralinepag reduced the risk of clinical worsening in patients with pulmonary arterial hypertension (PAH) by 55% in a large, late-stage study, meeting the trial's primary goal.
"In ADVANCE OUTCOMES, ralinepag reduced the risk of clinical worsening, decreased NT-proBNP levels, and improved exercise capacity in lower risk, heavily pretreated PAH patients, underscoring the potential benefits of a once-daily prostacyclin receptor agonist," said Vallerie V. McLaughlin, M.D., chair of the study's steering committee, in a statement.
The positive result (hazard ratio 0.45, p<0.0001) was consistent across all patient subgroups. The drug also hit key secondary goals, including a 24.3% reduction in NT-proBNP, a critical biomarker for heart failure, and a 20.4-meter improvement in the six-minute walk distance (6MWD) test compared to placebo.
The data positions ralinepag to potentially become the first once-daily oral prostacyclin receptor agonist approved for PAH, a life-threatening disease characterized by high blood pressure in the lungs. United Therapeutics said it intends to file for U.S. Food and Drug Administration approval in the second half of 2026.
Study Details and Competitive Landscape
The ADVANCE OUTCOMES study was a phase 3, event-driven trial that enrolled 687 PAH patients, who were randomized to receive either ralinepag or a placebo in addition to their existing therapy. A majority of patients were already on dual background therapy, indicating a relatively advanced and pre-treated population.
Ralinepag is a selective prostacyclin (IP) receptor agonist designed to mimic the effects of natural prostacyclin, a substance that promotes vasodilation and inhibits vascular remodeling. The company's data suggests ralinepag has a higher binding affinity and potency compared to selexipag, the active metabolite of Johnson & Johnson's approved PAH drug, Uptravi.
The safety profile of ralinepag was consistent with known side effects of the prostacyclin class, which can include headache, jaw pain, and nausea. No new safety signals were identified in the study.
The positive data could allow United Therapeutics to compete more effectively in the oral PAH treatment market. The results suggest ralinepag's efficacy and convenient once-daily dosing could make it a strong competitor to existing therapies.
The successful phase 3 results are a significant step for United Therapeutics in strengthening its PAH franchise. For investors, the key upcoming event will be the company's New Drug Application submission to the FDA, expected by the end of 2026.
This article is for informational purposes only and does not constitute investment advice.
