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Ref Type abstract
Authors M. Javle L.A. Kankeu Fonkoua A. Mahipal C-Y. Liao C. Fountzilas D. Li M.S. Pelster S. Goel P. Peng C. Sun H. Wang K. Hennessy X. Fu L. Neo Y. Shan P. Huang D. Wu F. Wu J. Fan S.A. Piha-Paul
Title 95MO Tinengotinib in patients with advanced, fibroblast growth factor receptor (FGFR) inhibitor refractory/relapsed cholangiocarcinoma
Abstract Text FGFR inhibitors (FGFRi) have a proven role for the management of FGFR-altered cholangiocarcinoma (CCA) after chemotherapy. However, disease progression occurs in 6-8 months. Secondary polyclonal mutations in the FGFR2 kinase domain represent a prominent acquired resistance mechanism. Tinengotinib was identified as a novel FGFRi with high potency to a variety of FGFR2 kinase domain mutations and has shown promising clinical results in CCA pts who have progressed on prior FGFRi. Here, we present pooled data from three trials. Methods Pts received tinengotinib orally once daily for 28 days per cycle. Pooled analyses of treatment-related adverse events (TRAEs), overall response rate (ORR), disease control rate (DCR), and median progression-free survival (mPFS) in the stratified CCA population were performed. Results A total of 73 advanced CCA pts were enrolled between Dec 2019 - Mar 2023. Median age was 60 years (24-81), 60% were female, 100% pts had ≥ 1 prior therapy including 47% pts with prior FGFRi, 97% had stage IV disease at screening. TRAEs were reported in 63 (86.3%) pts. 28 (38.4%) were Grade (G) 1-2, 32 (43.8%) were G3, 3 (4.1%) were G4, no G5 was observed. The most common TRAEs (≥20%) were hypertension (43.8%, G3 19.2%), stomatitis (32.9%, G3 4.1%), diarrhea (30.1%, G3 4.1%) and palmar-plantar erythrodysesthesia (24.7%, G3 2.7%). Among 58 evaluable pts, the ORR and DCR were 20.7% and 75.9%, respectively. In 29 pts with FGFR2 alteration who received prior FGFRi, the ORR and DCR were 34% and 89.7%, respectively. 21 pts who acquired resistance to prior FGFRi had ORR of 38.1%, DCR of 95.2% and mPFS of 6.90 months (95%CI, 4.90-9.20). 10 pts who developed FGFR2 kinase domain mutations (N549D/H/K/T, V564F/I/L, E565A/G/K) detected by liquid biopsy at baseline had ORR of 50%, DCR of 90% and mPFS of 7.03 months (95%CI, 0.95-11.86). Conclusions These pooled results suggest that tinengotinib has a manageable toxicity profile in CCA pts. Notable clinical benefit was noted with tinengotinib in FGFR-altered CCA pts with acquired resistance to prior FGFRi. A global phase III clinical trial is planned to further evaluate the efficacy and safety of tinengotinib for CCA with FGFRi resistance.


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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
FGFR2 act mut cholangiocarcinoma predicted - sensitive Tinengotinib Clinical Study Actionable In a combined analysis of 3 clinical trials, Tinengotinib (TT-00420) resulted in an overall response rate (ORR) of 20.7% and disease control rate (DCR) of 75.9% in cholangiocarcinoma patients (n=58), and in patients with FGFR2 mutations (n=29), an ORR of 34% and DCR of 89.7% and ORR of 38.1%, DCR of 95.2%, and median progression-free survival of 6.9 mo in patients with prior FGFR inhibitor resistance (n=21) (Ann Oncol (2023) 34 (suppl_2): S215-S216; NCT03654547, NCT04742959, NCT04919642). detail...