The Food and Drug Administration (FDA) has granted Orphan Drug designation to TT-101 for the treatment of idiopathic pulmonary fibrosis (IPF).

TTI-101 is an orally-delivered, small molecule, STAT3 (signal transducer and activator of transcription 3) inhibitor. STAT3 protein has been shown to contribute to the development and progression of fibrosis in the lungs of patients with IPF.

Preclinical studies have demonstrated that direct inhibition of STAT3 signaling by TTI-101 reverses fibrosis and restores lung function in IPF models.

“This designation provides further support for the development of TTI-101, which has shown promise in treating diseases in which STAT3 plays a critical role,” said Imran Alibhai, PhD, CEO of Tvardi. “Tvardi is dedicated to pursuing treatment of STAT3-driven diseases with TTI-101, beginning with a phase 1 trial in oncology and now advancing to phase 2 clinical trials in both IPF and oncology.”

The Company recently announced the receipt of Orphan Drug designation for TTI-101 for the treatment of hepatocellular carcinoma.


Tvardi Therapeutics’ TTI-101 receives Orphan Drug designation for idiopathic pulmonary fibrosis. News release. May 26, 2022.