Tvardi Therapeutics (TVARDI) recently announced that TTI-101, an oral small-molecule STAT3 inhibitor, has received fast-track FDA status for the treatment of relapsed/refractory locally advanced, unresectable, or metastatic hepatocellular carcinoma (HCC).






Liver cancer is the sixth most common cancer worldwide, with about 900,000 patients diagnosed each year. Hepatocellular carcinoma, the most common form of liver cancer, has limited treatment options and poor overall survival, with a 5-year survival rate of about 18%.
TTI-101 is an oral small-molecule STAT3 inhibitor. STAT3 is a key regulatory protein that has long been recognized as a major target in oncology. STAT3 plays a key role in the pathogenesis of HCC by promoting tumor formation and immunosuppressing tumor microenvironment. Inhibition of STAT3 signaling is expected to have a synergistic therapeutic effect on cancer cells. In preclinical trials, TTI-101 exhibited excellent pharmacokinetic characteristics, attenuated pY705-STAT3 phosphorylation, and showed anticancer activity in animal models of head and neck, lung, breast, and liver cancers.



TTI-101 is in its first human phase 1 clinical trial in patients with advanced solid tumors who have failed all treatments. To date, TTI-101 monotherapy has been shown to be well tolerated and clinically active in a wide range of tumors, including multiple durable radiographic objective responses.
This fast-track status will facilitate the rolling review of clinical data and ultimately shorten the application review time for TTI-101 approval for HCC patients. TTI-101 has previously received orphan drug status for the treatment of hepatocellular carcinoma and idiopathic pulmonary fibrosis.

"We are pleased that the FDA granted TTI-101 fast-track status for HCC," said Imran Alibhai, PhD, CEO of Tvardi. "This is an endorsement of the compelling safety and efficacy profile we have seen in patients with posterior line HCC in Phase 1 trials. "This fast-track status comes at a perfect time as we are about to begin enrolling patients in the Phase 2 HCC basket trial, which will test TTI-101 as monotherapy or in combination with existing approved therapies in patients with first-line, second-line, and posterior HCC."

Reference Materials: [1] FDA Grants for the letter of Tvardi Therapeutics 'TTI-101 for Hepatocellular Carcinoma 19, 2022, from https://tvarditherapeutics.com/2022/10/19/fda-grants-fast-track-designation-to-tvardi-therapeutics-tti-101-for-hepatocel lular-carcinoma/

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