Targeting STAT3 to treat hepatocellular carcinoma, innovative therapy receives FDA fast track designation

Recently, Tvardi Therapeutics announced that its oral small molecule STAT3 inhibitor TTI-101 has obtained fast track qualification from the US FDA for the treatment of recurrent/refractory locally advanced, unresectable or metastatic hepatocellular carcinoma (HCC).

TTI-101 is an oral small molecule STAT3 inhibitor. STAT3 is a key regulatory protein that has long been considered a major target in oncology. STAT3 plays a key role in the pathogenesis of HCC by promoting tumor formation and immunosuppressive tumor microenvironment. Inhibition of STAT3 signaling is expected to have a synergistic therapeutic effect on cancer cells. In preclinical experiments, TTI-101 showed excellent pharmacokinetic characteristics, reduced the effect of pY705-STAT3 phosphorylation, and showed anticancer activity in animal models of head and neck cancer, lung cancer, breast cancer and liver cancer.

▲Mechanism of action of TTI-101 (Image source: Tvardi Therapeutics company website)

TTI-101 is undergoing the first human Phase 1 clinical trial in patients with advanced solid tumors who have failed all treatments. So far, TTI-101 monotherapy has shown good tolerability and clinical activity in a wide range of tumors, including multiple durable radiological objective responses.

The fast track status of TTI-101 will facilitate the rolling review of clinical data and ultimately shorten the review time for the application of TTI-101 for HCC patients. Previously, TTI-101 has been granted orphan drug status for the treatment of hepatocellular carcinoma and idiopathic pulmonary fibrosis.

“We are pleased that the FDA has granted Fast Track designation to TTI-101 for the treatment of HCC,” said Imran Alibhai, MD, PhD, Chief Executive Officer of Tvardi. “This is recognition of the compelling safety and efficacy we saw in our Phase 1 trial in patients with later-line HCC. This Fast Track designation comes at an ideal time as we are about to begin enrolling patients in our Phase 2 HCC basket trial, which will test TTI-101 as a monotherapy or in combination with existing approved therapies in patients with first-, second-, and later-line HCC.”

References: [1] FDA Grants Fast Track Designation to Tvardi Therapeutics’ TTI-101 for Hepatocellular Carcinoma. Retrieved October 19, 2022, from https://tvarditherapeutics.com/2022/10/19/fda-grants-fast-track-designation-to-tvardi-therapeutics-tti-101-for-hepatocellular-carcinoma/

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