tinengotinib (TT-00420) / TransThera Biosci - LARVOL DELTA

Tinengotinib for adults with advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 trial. (PubMed, Lancet Gastroenterol Hepatol) - P2 | "These findings suggest that tinengotinib might have activity in patients with cholangiocarcinoma with FGFR2 fusions that progressed following FGFR inhibitor therapy. Anti-tumour activity was also observed in patients with other FGFR alterations. The data from this phase 2 study supported the initiation of a phase 3 registration trial."

Journal • P2 data • Biliary Cancer • Cardiovascular • Cholangiocarcinoma • CNS Disorders • Dental Disorders • Dermatology • Hypertension • Oncology • Solid Tumor • Stomatitis • FGFR2

Survival and safety of tinengotinib in pooled patients with advanced, fibroblast growth factor receptor (FGFR) inhibitor refractory/relapsed cholangiocarcinoma (CCA) (ESMO 2025) - P1, P1/2, P2 | "Conclusions Tinengotinib shows promising efficacy with an acceptable safety profile in CCA pts with notable clinical benefit in FGFR2-altered CCA with prior FGFRi(s). A global phase III study is currently open to further assess the efficacy and safety of tinengotinib in FGFR2-altered CCA pts who progressed on chemotherapy and FGFRi."

Clinical • Metastases • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • FGFR • FGFR2

TransThera Raises HK$190 Million via H-Share Placing to Fund Tinengotinib Trials and Pipeline R&D (TipRanks) - "TransThera Sciences...is advancing a portfolio of innovative drug candidates centered on its core oncology asset Tinengotinib, alongside other molecules including TT-00973 and TT-01488, targeting specialized treatment needs and reinforcing its presence in the innovative drug development space."

Financing • Oncology

Tinengotinib in patients with advanced, metastatic cholangiocarcinoma: Overall survival results and biomarker correlative analysis from a phase 2 clinical trial. (ASCO-GI 2025) - P2, P3 | "Tinengotinib has shown promising anti-tumor efficacy in CCA pts with FGFR fusion after prior FGFRi and in those with primary FGFR mutations. An ongoing randomized phase III study will investigate tinengotinib vs. chemotherapy in FGFR inhibitor refractory CCA (NCT05948475)."

Biomarker • Clinical • Metastases • P2 data • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • ARID1A • BCOR • FGFR2 • MED12

The efficacy and safety of tinengotinib in patients with advanced or metastatic HR+/HER2- breast cancer or TNBC (SABCS 2023) - P1, P1/2 | "Tinengotinib for the treatment of HR+HER2- BC or TNBC, whether as monotherapy or in combination with nab-paclitaxel, had manageable side effects. Tinengotinib has shown promising clinical benefit in heavily pre-treated pts with refractory HR+HER2- BC or TNBC. Clinical benefit was similar across the subgroups of pts with HR+ HER2-zero and HR+ HER2 low disease."

Clinical • Metastases • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • Hormone Receptor Breast Cancer • Hormone Receptor Positive Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • CSF1R • FGFR1 • HER-2 • JAK1 • JAK2

Efficacy and safety results of FGFR1-3 inhibitor, tinengotinib, as monotherapy in patients with advanced, metastatic cholangiocarcinoma: Results from phase II clinical trial. (ASCO-GI 2024) - P2 | "Tinengotinib has promising clinical benefit for FGFR2 fusion CCA after prior FGFRi and for non-fusion FGFR alterations. Tinengotinib-related toxicities were manageable. An ongoing randomized, controlled phase III study will evaluate the clinical efficacy, safety, and pharmacodynamic effect of Tinengotinib vs Physicians’ choice in subjects with FGFR2-altered refractory/relapsed CCA after prior chemotherapy and FGFRi therapy."

Clinical • Metastases • Monotherapy • P2 data • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • FGFR2

First-In-Human Phase I Study of Tinengotinib (TT-00420), a Multiple Kinase Inhibitor, as a Single Agent in Patients With Advanced Solid Tumors. (PubMed, Oncologist) - "Tinengotinib was well tolerated with favorable pharmacokinetic characteristics. Preliminary findings indicated potential clinical benefit in FGFR inhibitor-refractory cholangiocarcinoma, HER2-negative breast cancer (including TNBC), and CRPC. Continued evaluation of tinengotinib is warranted in phase II trials."

Journal • Metastases • P1 data • Biliary Cancer • Breast Cancer • Cardiovascular • Castration-Resistant Prostate Cancer • Cholangiocarcinoma • Dermatology • Gastrointestinal Cancer • Genito-urinary Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • Hormone Receptor Breast Cancer • Hormone Receptor Positive Breast Cancer • Hypertension • Oncology • Prostate Cancer • Solid Tumor • Triple Negative Breast Cancer • FGFR2 • HER-2 • JAK1

Phase II study of tinengotinib in advanced cholangiocarcinoma: Analysis of molecular response and resistance mechanisms. (ASCO-GI 2026) - P2 | "These findings provide genomic evidence that tinengotinib may overcome resistance to prior FGFRi treatment in FGFR2 fus-positive CCA, as reflected by significantly reductions in ctDNA VAFs and frequent clearance of FGFR2 KD mutations. The emergence of resistance alterations including de novo FGFR2 mutations and bypass pathways activations indicates acquired resistance that may limit long-term benefit. These results support further investigation with expanded biomarker sampling to better characterize resistance evolution and guide therapy optimization in an ongoing Phase 3 trial."

Metastases • P2 data • Tumor mutational burden • Biliary Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Oncology • Solid Tumor • EGFR • FGFR2 • FUS • KRAS • TMB • TP53

TransThera Showcases Tinengotinib Phase II Data in Advanced Cholangiocarcinoma at ASCO GI 2026 (TipRanks) - "The poster detailed circulating tumor DNA biomarker analyses that evaluated tinengotinib in various FGFR2 fusion and FGFR-altered patient subgroups, including those with primary or acquired resistance to prior FGFR inhibitors, and showed findings that support continued development and expanded biomarker sampling in an ongoing Phase III trial, underscoring the company’s push to strengthen its position in precision oncology for cholangiocarcinoma."

Biomarker • P2 data • Cholangiocarcinoma

Efficacy and Safety of TT-00420 (Tinengotinib) Tablets Versus Chemotherapy in Patients With Advanced Intrahepatic Cholangiocarcinoma Harboring FGFR2 Fusions/Rearrangements or Mutations (clinicaltrials.gov) - P3 | N=138 | Not yet recruiting | Sponsor: TransThera Sciences (Nanjing), Inc.

New P3 trial • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • FGFR2

NEW DRUG APPLICATION FOR TINENGOTINIB TABLETS ACCEPTED BY THE NATIONAL MEDICAL PRODUCTS ADMINISTRATION (PRNewswire) - "It is intended for the treatment of adults with unresectable advanced or metastatic cholangiocarcinoma (CCA) who have received at least one prior systemic treatment and FGFR inhibitor treatment. Previously, Tinengotinib tablets have been included in...the List of Breakthrough Therapy Designation for this indication."

Breakthrough therapy • China filing • Cholangiocarcinoma

Tinengotinib in Previously Treated Cholangiocarcinoma: New Signals from a Multicentre Phase 2 Trial (OncoDaily) - "Between Nov 19, 2021, and March 5, 2024, 55 patients were enrolled across the four cohorts. Median age was 61 years, and all patients had received prior systemic therapy. Median follow-up was 11.3 months. Objective responses differed markedly across FGFR-defined cohorts: ORR 6.3% in cohort A1 (primary FGFR inhibitor resistance; one confirmed partial response); ORR 30.0% in cohort A2 (acquired FGFR inhibitor resistance; three confirmed partial responses); ORR 23.1% in cohort B (other FGFR alterations; three confirmed partial responses); ORR 0% in cohort C (FGFR wild-type)....Tinengotinib demonstrated a manageable safety profile. Grade 3 treatment-related adverse events most commonly included hypertension (31%), palmar-plantar erythrodysesthesia syndrome (13%), and stomatitis (11%)."

P2 data • Cholangiocarcinoma

Targeting high-risk MYC-overexpressed osteosarcoma with an Aurora kinase inhibitor:--results from a pilot umbrella trial. (PubMed, NPJ Precis Oncol) - P=N/A | "Patients were assigned to three arms: (A) PD-1 antibody plus gemcitabine and docetaxel; (B) PARP inhibitor combined with temozolomide; or (C) tinengotinib (TT-00420), a small-molecule aurora kinase inhibitor currently in clinical trials. This study demonstrated the feasibility of using genomic molecular subtyping to guide the precise treatment of osteosarcoma. We also revealed that the abnormal genomic and transcriptomic profiles caused by MYC amplification could be suppressed by tinengotinib."

Journal • Oncology • Osteosarcoma • Sarcoma • Solid Tumor • HRD • MYC

Tinengotinib in advanced or metastatic HR+/HER2- and TNBC: efficacy and biomarker correlative analysis from a phase Ib/II study (SABCS 2025) - P1/2 | "Tinengotinib exhibited promising clinical benefit and manageable safety profile for the treatment of HR+/HER2- BC or TNBC. The biomarker correlative analysis revealed that ROS1 mutations could be a potential predictive biomarker. Early reduction in ctDNA VAF may serve as a pharmacodynamic biomarker."

Biomarker • Clinical • Metastases • P1/2 data • Breast Cancer • HER2 Breast Cancer • HER2 Negative Breast Cancer • HER2 Positive Breast Cancer • Hormone Receptor Breast Cancer • Hormone Receptor Positive Breast Cancer • Triple Negative Breast Cancer • CDKN2A • CSF1R • CTNNA1 • DNMT3A • ERBB3 • FGFR2 • HER-2 • JAK1 • JAK2 • KMT2D • MLL2 • NF1 • PDGFRB • PIK3CA • PTEN • RAD51D • ROS1 • TP53

Inclusion of tinengotinib tablets in the list of products for priority review by the national medical products administration (The Manila Times) - "TransThera Sciences Nanjing, Inc. (the 'TransThera') announced that Tinengotinib tablets have been included in the List of Products for Priority Review by the Center for Drug Evaluation ('CDE') of the National Medical Products Administration ('NMPA') of the PRC, with the proposed indication for the treatment of adults with unresectable advanced or metastatic cholangiocarcinoma (CCA) who have received at least one prior systemic treatment and FGFR inhibitor treatment."

China filing • Priority review • Cholangiocarcinoma

TransThera Publishes Clinical Studies of Tinengotinib (TT-00420) against Cholangiocarcinoma on Lancet (PRNewswire) - "In a multicenter, open-label Phase 2 trial (NCT04919642), patients with FGFR2 fusion-positive CCA who had either primary resistance or developed acquired resistance to prior FGFR inhibitor (FGFRi) therapy were enrolled, along with patients harboring other FGFR alterations or FGFR wiled-type tumors. Tinengotinib demonstrated clinical activity in patients with FGFR2 fusion-positive CCA with acquired FGFRi resistance, as well as in those with other FGFR-altered subtypes."

P2 data • Cholangiocarcinoma

Unravelling Tinengotinib's Mechanistic Landscape in Triple-Negative Breast Cancer Via Network Pharmacology and in Silico Simulation Techniques. (PubMed, Cell Biochem Biophys) - No abstract available

Journal • Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer

VOLUNTARY ANNOUNCEMENT (HKEXnews) - "The board of directors of the Company...is pleased to announce that the phase II clinical trial of the Company’s core product Tinengotinib (TT-00420) in combination with Fulvestrant for the treatment of previously treated hormone receptor positive (HR+) and human epidermal growth factor receptor 2 negative or low expression (HER2-) relapsed or metastatic breast cancer has obtained Investigational New Drug (IND) approval from the National Medical Products Administration (NMPA) of the PRC on September 10, 2025."

New P2 trial • HER2 Negative Breast Cancer • Hormone Receptor Positive Breast Cancer

FIRST PATIENT DOSED IN PHASE II CLINICAL TRIAL OF TINENGOTINIB IN COMBINATION WITH AKESO’S (CADONILIMAB, PD-1/CTLA-4) / (IVONESCIMAB, PD-1/VEGF) (HKEXnews) - "This trial is an open-label, multicenter Phase II clinical study conducted in China to evaluate the efficacy and safety of (cadonilimab, PD-1/CTLA-4)/(ivonescimab, PD-1/ VEGF) in combination with Tinengotinib tablets for the treatment of advanced HCC."

Trial status • Hepatocellular Cancer

Study of TT-00420 (Tinengotinib) in Subjects With Cholangiocarcinoma Who Failed or Relapsed to Chemotherapy and FGFR Inhibitor (clinicaltrials.gov) - P2 | N=50 | Active, not recruiting | Sponsor: TransThera Sciences (Nanjing), Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Biliary Cancer • Cholangiocarcinoma • Oncology • Solid Tumor • FGFR2

Safety of TT-00420 (Tinengotinib) Monotherapy in Patients With Advanced Solid Tumors and Triple Negative Breast Cancer (clinicaltrials.gov) - P1 | N=48 | Completed | Sponsor: TransThera Sciences (Nanjing), Inc. | Active, not recruiting ➔ Completed

Monotherapy • Trial completion • Breast Cancer • Hormone Receptor Positive Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer

A Phase II Clinical Study of AK104/AK112 in Combination With TT-00420 Tablet for Advanced HCC. (clinicaltrials.gov) - P2 | N=100 | Not yet recruiting | Sponsor: Akeso

New P2 trial • Hepatocellular Cancer • Oncology • Solid Tumor

Tinengotinib (TT-00420) inhibits tumor growth and overcomes multidrug resistance in gastrointestinal cancers (IDDF 2025) - "Additionally, trastuzumab-resistant and immunotherapy-resistant models were established to evaluate TT-00420's potential in overcoming drug resistance. Western blot analysis confirmed that Aurora A played a crucial role in mediating its tumor-suppressive effects in resistant models.Conclusions TT-00420 is a highly promising anti-tumor agent, exhibiting potent efficacy against CRC and GC. Our findings provide a new therapeutic approach for gastrointestinal cancers and offer valuable insights for future clinical research."

Biliary Cancer • Breast Cancer • Cholangiocarcinoma • Colorectal Cancer • Gastric Cancer • Gastrointestinal Cancer • HER2 Breast Cancer • HER2 Positive Breast Cancer • Lung Cancer • Oncology • Small Cell Lung Cancer • Solid Tumor • FGFR • HER-2 • PD-1

TransThera Publishes Translational Studies of Tinengotinib (TT-00420) against Cholangiocarcinoma on Annals of Oncology (The Manila Times) - "The article discloses for the first time the co-crystal structure of tinengotinib with the FGFR2 kinase domain of its unique binding mode, in addition to kinetic studies to illustrate its higher affinity compared to first-generation FGFR inhibitors, in vitro and in vivo activities against clinically acquired FGFR2 resistance mutations, as well as a case report to demonstrate its clinical efficacy."

Preclinical • Cholangiocarcinoma

GRANT OF TINENGOTINIB (TT-00420) FAST TRACK DESIGNATION FOR TREATMENT OF MCRPC (HKEXnews) - "The board of directors of the Company (the 'Board') is pleased to announce that the U.S. Food and Drug Administration (the 'FDA') has granted the fast track designation of tinengotinib for treatment of patients with metastatic castration-resistant prostate cancer (mCRPC)....In the phase I/II studies of tinengotinib monotherapy, 13 mCRPC patients with measurable disease were enrolled. The overall response rate (ORR) was 46%, and the disease control rate (DCR) was 85%."

Fast track • Castration-Resistant Prostate Cancer