The results are in, United Therapeutics stock surges on positive drug trial results

United Therapeutics Corporation (NASDAQ: UTHR) shares rose 12% Monday after the company announced its TETON-1 pivotal study of Tyvaso met its primary endpoint for treatment of idiopathic pulmonary fibrosis.

Nebulized Tyvaso demonstrated superiority over placebo for the change in absolute forced vital capacity by 130.1 mL and reduced the risk of clinical worsening in patients with idiopathic pulmonary fibrosis. The treatment effect exceeded results seen in the earlier TETON-2 study.

The therapy achieved statistical significance for reducing the risk of clinical worsening and showed numerical improvement in other secondary endpoints relative to placebo, including time to first acute exacerbation of IPF and changes in percent predicted FVC, K-BILD score, and DLCO.

Integrated analyses of TETON-1 and TETON-2 showed statistically significant treatment effects compared to placebo from baseline to week 52 for the primary endpoint of change in absolute FVC by 111.8 mL. Benefits were observed across all patient subgroups, including those using background therapy with nintedanib or pirfenidone.

Treatment with nebulized Tyvaso was well-tolerated, and the safety profile was consistent with previous Tyvaso studies. No new safety signals were observed.

United Therapeutics plans to seek priority review of a supplemental New Drug Application to be submitted to the FDA by the end of this summer to add IPF to the labeled indications for nebulized Tyvaso. Both the FDA and the European Medicines Agency have granted orphan designation for Treprostinil to treat IPF.