Results from the TETON-1 study show nebulized Treprostinil (Tyvaso) met its primary endpoint, demonstrating a significant increase in lung function compared to placebo.
RT’s Three Key Takeaways:
- Primary Endpoint Met: Nebulized treprostinil demonstrated superiority over placebo by improving absolute forced vital capacity by 130.1 mL over 52 weeks in patients with idiopathic pulmonary fibrosis.
- Clinical Worsening Reduced: The study achieved statistical significance in reducing the risk of clinical worsening and showed numerical improvements in quality of life and time to first acute exacerbation.
- FDA Submission Planned: United Therapeutics intends to submit a supplemental New Drug Application to the Food and Drug Administration by the end of summer, seeking priority review for the new indication.
The TETON-1 study evaluating nebulized Tyvaso (treprostinil) for the treatment of idiopathic pulmonary fibrosis (IPF) met its primary efficacy endpoint, according to United Therapeutics Corp.
The study demonstrated superiority over placebo for the change in absolute forced vital capacity (FVC) by 130.1 mL from baseline to week 52 (p < 0.0001). Nebulized treprostinil also achieved statistical significance for reducing the risk of clinical worsening and showed numerical improvement in secondary endpoints, including time to first acute exacerbation of IPF, changes in percent predicted FVC, King’s Brief Interstitial Lung Disease quality of life questionnaire (K-BILD) score, and diffusion capacity of lungs for carbon monoxide (DLCO).
“The unprecedented results of TETON-1, which surpassed even the overwhelmingly positive results of TETON-2, represent a profound step forward for people living with IPF, a devastating disease with few treatment options,” said Martine Rothblatt, PhD, chairperson and CEO of United Therapeutics.
Benefits were observed across all patient subgroups, including those using background therapies such as nintedanib or pirfenidone, smoking status, and supplemental oxygen use. The safety profile was consistent with previous studies, and no new safety signals were observed.
Integrated analyses of the TETON-1 and TETON-2 studies showed a statistically significant change in absolute FVC by 111.8 mL compared to placebo (p < 0.0001). These findings also indicated significant effects on time to first clinical worsening and acute exacerbations.
“Together, both studies demonstrated not only better preservation of lung function, but also preservation of quality of life, as well as reduced disease worsening and reduced acute IPF exacerbations. These findings have the potential to fundamentally shift our approach to IPF management and could be a game changer,” said Steven D Nathan, MD, chair of the TETON steering committee, in a news release.
United Therapeutics plans to submit a supplemental New Drug Application to the US Food and Drug Administration (FDA) by the end of this summer. The company will seek priority review to add IPF to the labeled indications for nebulized Tyvaso based on data from the TETON-1 and TETON-2 studies. Both the FDA and the European Medicines Agency have granted orphan designation for treprostinil to treat IPF.
Additional results from the TETON-1 study and integrated data will be presented at ATS 2026. Full results of the TETON-2 study were recently published in the New England Journal of Medicine.
