According to United Therapeutics, the Phase 3 TETON-1 trial of nebulized Tyvaso treprostinil inhalation solution in patients with idiopathic pulmonary fibrosis met its primary endpoint, demonstrating a 130.1 mL change in absolute FVC from baseline to week 52. The change in absolute FVC from baseline to week 52 demonstrated in the TETON-1 study is significantly higher than the 95.6 mL change demonstrated in the TETON-2 trial. The TETON-1 trial, which was initiated in 2021, enrolled 598 IPF patients who received either nebulized Tyvaso or nebulized placebo 4 times per day over 52 weeks.
United Therapeutics said that the TETON-1 trial demonstrated statistical significance “for reducing the risk of clinical worsening” and “showed numerical improvement in other important secondary endpoints relative to placebo.” The company said that combined analysis of the two studies showed statistically significant improvements for “most secondary endpoints, including time to first clinical worsening and first acute exacerbation of IPF and changes in percent predicted FVC” while “Overall survival at week 52 trended in favor of Tyvaso but did not meet statistical significance.”
Tyvaso inhalation solution was approved by the FDA for the treatment of pulmonary arterial hypertension in July 2009. In March 2021, the FDA approved use of the inhalation solution for the treatment of pulmonary hypertension with interstitial lung disease (PH-ILD). In the new announcement, United Therapeutics plans to submit an sNDA to add the IPF indication sometime this summer.
United Therapeutics Chairperson and CEO Martine Rothblatt commented, “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.
Senior Vice President, Product Development, Peter Smith said, “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.”
Read the United Therapeutics press release
