NANJING, China and GAITHERSBURG, Md., Dec. 4, 2025 — TransThera Sciences Inc. (“TransThera”) announced today the publication of clinical findings from a U.S.-based Phase 2 trial investigating tinengotinib in patients with Cholangiocarcinoma (CCA) in The Lancet Gastroenterology and Hepatology, which has an Impact Factor of 38.6.
Cholangiocarcinoma (CCA) is a highly aggressive cancer of the bile ducts, frequently driven by FGFR2 fusions—genomic alterations that can be targeted by FGFR inhibitors such as pemigatinib and futibatinib. However, secondary FGFR2 mutations often lead to resistance to these therapeutic agents.
The multicenter, open-label Phase 2 trial (NCT04919642) enrolled patients with FGFR2 fusion-positive CCA who had either initial resistance or had acquired resistance to prior FGFR inhibitor (FGFRi) therapy. The study also included patients with other FGFR alterations or FGFR wild-type tumors. Tinengotinib demonstrated clinical effectiveness in patients with FGFR2 fusion-positive CCA who had developed FGFRi resistance, as well as in those with other FGFR-altered subtypes.
Dr. Milind Javle of The University of Texas MD Anderson Cancer Center, the corresponding author of the publication, commented: “Currently, two FDA-approved therapies target FGFR2 fusions in CCA. Nevertheless, resistance remains a significant clinical challenge. Therefore, there is an urgent need for next-generation FGFR inhibitors capable of overcoming this resistance. Tinengotinib, a multi-kinase FGFR inhibitor, is engineered to block both FGFR and the compensatory pathways contributing to resistance. In this phase 2 study, tinengotinib yielded durable responses and significant clinical benefit. These encouraging results provide a strong rationale for proceeding with a Phase 3 registration study.”
Dr. Jean Fan, TransThera’s Chief Medical Officer, also stated: “We are extremely pleased that these clinical trial results have received peer recognition and were published in such a prestigious journal. This study offers critical insights into treatment strategies for patients with FGFR-altered, chemotherapy- and FGFR inhibitor–refractory or relapsed CCA, including a comparison of tinengotinib versus a physician’s chosen treatment. We are committed to advancing global enrollment and providing new treatment options for patients suffering from metastatic cholangiocarcinoma.”
Disclaimer: This article serves as a press release by TransThera to disclose the company’s latest developments. It is not intended as a product promotion advertisement and does not constitute the company’s or investment advice.
About Tinengotinib
Tinengotinib is an internally discovered, registrational clinical-stage multi-kinase inhibitor that exerts antitumor effects by targeting FGFRs and VEGFRs, mitotic kinases Aurora A/B, and Janus kinases (JAK). Ongoing clinical trials in the US and China have indicated tinengotinib’s potential efficacy across various solid tumor types. The FDA has granted it Orphan Drug Designation (ODD) and Fast Track Designation (FTD) for the treatment of CCA. In China, it received Breakthrough Therapy Designation (BTD) from the National Medical Products Administration (NMPA) and was approved for inclusion in the Priority Review and Approval Procedure by the NMPA for CCA. The European Medicines Agency (EMA) also granted it Orphan Drug Designation (ODD) for the treatment of biliary tract cancer.
About TransThera
TransThera is a biopharmaceutical company in the registrational clinical stage, driven by clinical demand, focused on discovering and developing innovative small molecule therapies for oncology, inflammatory, and cardiometabolic diseases. Further supported by in-depth research in translational medicine and drug design, TransThera aims to develop first-in-class or best-in-class drug candidates strategically positioned to address urgent global clinical requirements. For more information, please visit
SOURCE TransThera Sciences (Nanjing) Inc.