United Therapeutics completes enrollment for phase 3 PAH drug study

Published 23/06/2025, 12:14
United Therapeutics completes enrollment for phase 3 PAH drug study

SILVER SPRING, Md. - United Therapeutics Corporation (NASDAQ:UTHR), a $13.1 billion pharmaceutical company currently identified as undervalued by InvestingPro analysis, announced Monday it has completed enrollment for its phase 3 ADVANCE OUTCOMES study evaluating ralinepag as a treatment for pulmonary arterial hypertension (PAH). The company maintains excellent financial health with a perfect Piotroski Score of 9.

The study enrolled 728 participants and will continue to accrue clinical worsening events through the end of 2025, with top-line data expected in the first half of 2026. The company’s strong financial position, with an impressive 89% gross profit margin and 19.85% revenue growth over the last twelve months, provides robust support for this extensive clinical program.

Ralinepag is being developed as a potential once-daily oral prostacyclin agonist for PAH patients. The drug previously demonstrated positive results in a phase 2 study, showing a 29.8% reduction in median pulmonary vascular resistance compared to placebo after 22 weeks of treatment.

In a subsequent open-label extension study, participants showed a mean increase of 36.3 meters in six-minute walk distance after two years, with over 85% maintaining stable functional class status. The company noted these results should be interpreted cautiously as the extension study was uncontrolled.

The ADVANCE OUTCOMES study’s primary endpoint is time to first clinical worsening event. Secondary endpoints include changes in various disease markers and quality of life measurements.

PAH is a life-threatening condition characterized by increased pressure in pulmonary arteries, affecting approximately 500,000 people worldwide, including around 50,000 in the United States.

Ralinepag remains an investigational compound not approved for any use in any country. For investors interested in deeper analysis, InvestingPro offers comprehensive research reports with detailed financial metrics and 12 additional exclusive ProTips about United Therapeutics’ investment potential.

In other recent news, Insmed Incorporated announced positive results from its Phase 2b study of Treprostinil Palmitil Inhalation Powder (TPIP) for pulmonary arterial hypertension. The study achieved its primary and all secondary efficacy endpoints, including a significant reduction in pulmonary vascular resistance and improvements in six-minute walk distance and NT-proBNP concentrations. Insmed plans to engage with the FDA for Phase 3 trials, expected to begin in 2025 for PH-ILD and 2026 for PAH. Meanwhile, Liquidia Technologies experienced a legal victory as a federal judge dismissed United Therapeutics’ cross-claims, clearing the way for the launch of its drug Yutrepia. This ruling removes legal barriers, allowing Liquidia to potentially launch Yutrepia soon, which could compete with United Therapeutics’ Tyvaso. Analysts at Raymond James maintained a Strong Buy rating for Liquidia following this court decision. Additionally, United Therapeutics received an Outperform rating from Leerink Partners, focusing on the potential of its Tyvaso treatment for idiopathic pulmonary fibrosis. TD Cowen also reaffirmed a Buy rating for United Therapeutics, citing confidence in the company’s pipeline and upcoming clinical trials.

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