United Therapeutics announces TETON-1 study met primary efficacy endpoint

United Therapeutics (UTHR) announced that its TETON-1 study evaluating the use of nebulized Tyvaso for the treatment of idiopathic pulmonary fibrosis met its primary efficacy endpoint, demonstrating superiority over placebo for the change in absolute forced vital capacity by 130.1 mL from baseline to week 52. Nebulized Tyvaso achieved statistical significance for reducing the risk of clinical worsening and showed numerical improvement in other important secondary endpoints relative to placebo, including time to first acute exacerbation of IPF and changes in percent predicted FVC, King’s Brief Interstitial Lung Disease quality of life questionnaire score, and diffusion capacity of lungs for carbon monoxide. Benefits of nebulized Tyvaso were observed across all subgroups, such as use of background therapy, smoking status, and supplemental oxygen use. Treatment with nebulized Tyvaso was well-tolerated, and the safety profile was consistent with previous Tyvaso studies and known prostacyclin-related adverse events. No new safety signals were observed. 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 and most secondary endpoints, including time to first clinical worsening and first acute exacerbation of IPF and changes in percent predicted FVC, K-BILD score, and DLCO. Overall survival at week 52 trended in favor of Tyvaso but did not meet statistical significance.