United Therapeutics Corporation Announces Full Results of TETON-2 Phase 3 Clinical Trial Published in The New England Journal of Medicine

Core Insights - United Therapeutics Corporation announced the full results of the TETON-2 Phase 3 clinical trial for nebulized Tyvaso (treprostinil), demonstrating significant preservation of lung function and reduction in clinical worsening events in patients with idiopathic pulmonary fibrosis (IPF) [1][2] Study Results - The TETON-2 study met its primary efficacy endpoint, showing a statistically significant improvement in absolute forced vital capacity (FVC) from baseline to week 52, with a median change of 49.9 mL in the Tyvaso group compared to 136.4 mL in the placebo group, resulting in a between-group difference of 95.6 mL (P<0.001) [1][2] - Nebulized Tyvaso reduced the risk of clinical worsening events by 29% (hazard ratio 0.71; P=0.02) compared to placebo, achieving statistical significance in this key secondary endpoint [1][2] - Improvements were also noted in secondary endpoints, including change in percent predicted FVC, quality of life as measured by the King's Brief Interstitial Lung Disease questionnaire (K-BILD), and diffusion capacity of lungs for carbon monoxide (DLCO) [1][2] Patient Population and Safety - The trial included 597 patients with a mean age of 71.7 years, 80.1% male, and 75.4% on background antifibrotic therapy, reflecting a general population of IPF patients [1][2] - Treatment with nebulized Tyvaso was well-tolerated, with adverse events primarily being cough, headache, and diarrhea, consistent with previous studies [1][2] Regulatory Plans - United Therapeutics plans to submit a supplemental New Drug Application (sNDA) to the FDA by the second half of 2026 to add IPF to the labeled indications for nebulized Tyvaso [1][2] Background on IPF and Tyvaso - Idiopathic pulmonary fibrosis (IPF) is a progressive lung disease characterized by scarring of lung tissue, leading to respiratory failure and death, affecting an estimated 100,000 patients in the United States [2] - Tyvaso is currently indicated for pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD) [2][3]