United Therapeutics 宣布Tyvaso®治疗特发性肺纤维化的TETON-1试验达到关键终点

SILVER SPRING, Md.&RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)-- United Therapeutics Corporation (Nasdaq: UTHR), a public benefit corporation, today announced that its TETON-1 study evaluating the use of nebulized Tyvaso for the treatment of idiopathic pulmonary fibrosis (IPF) met its primary efficacy endpoint, demonstrating superiority over placebo for the change in absolute forced vital capacity (FVC) by 130.1 mL (Hodges-Lehmann [H-L] estimate, 95% confidence interval [CI], 82.2 to 178.1 mL; p <0.0001) 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 (K-BILD) score, and diffusion capacity of lungs for carbon monoxide (DLCO).

Benefits of nebulized Tyvaso were observed across all subgroups, such as use of background therapy (nintedanib, pirfenidone, or no 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 (H-L estimate, 95% CI, 79.7 to 144.0; p <0.0001) 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.

“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. We remain sincerely grateful to the patients, caregivers, and investigators who made this trial possible,” said Martine Rothblatt, Ph.D., Chairperson and Chief Executive Officer of United Therapeutics. “In the past seven months, our three pivotal studies, TETON-1, TETON-2, and the ADVANCE OUTCOMES study of ralinepag, met their primary endpoints with p-values <0.0001, an inflection point heralding a new era of even greater growth for United Therapeutics. These consecutive accomplishments were produced by our culture of innovation and our commitment to multiple shots on goal to advance treatments for rare cardiopulmonary and respiratory diseases.”

“IPF is a progressive, life-limiting disease for which existing treatments provide only modest benefit and are often accompanied by significant side effects. TETON-1 reinforces what was shown in TETON-2, with both studies demonstrating clinically meaningful treatment outcomes in IPF patients with or without background anti-fibrotic therapy. 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, M.D., Schar Chair, Advanced Lung Disease and Lung Transplant Program at Inova Fairfax Hospital and Chair of the TETON Steering Committee.

“The TETON program has achieved statistical significance in endpoints that have never been attained in other IPF clinical trials. These results provide a compelling body of evidence for nebulized Tyvaso’s differentiated direct lung delivery combined with multimodal activity across fibrotic, vascular, and inflammatory pathways that are not currently addressed by existing therapies,” said Peter Smith, Pharm.D., Senior Vice President, Product Development at United Therapeutics and the lead for the global TETON program.

United Therapeutics plans to seek priority review of a supplemental New Drug Application, to be submitted to theU.S.Food and Drug Administration (FDA)by the end of this summer, 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 TETON-1 study results and data from the integrated analyses of TETON-1 and TETON-2 will be presented at the American Thoracic Society Annual Meeting inOrlandoin May 2026. Full results of the TETON-2 study were recently published in the New England Journal of Medicine and are available online at NEJM.org.