On March 12, CTTQ Pharma, a core enterprise of Sino Biopharmaceutical Limited (1177.HK), announced that the Phase III clinical study of its independently developed Class 1 innovative drug, TQB3454 tablets, for the treatment of advanced bile duct cancer with IDH1 mutations, has completed its pre-specified interim analysis. The Independent Data Monitoring Committee (IDMC) determined that the primary endpoints of progression-free survival (PFS) and overall survival (OS) both met the pre-specified superiority criteria. The company has received written consent from the Center for Drug Evaluation (CDE), National Medical Products Administration (NMPA), and will submit a marketing application for this indication in the near future. 

The TQB3454-III-01 study (NCT05987358) is a randomized, double-blind, placebo-controlled, multicenter Phase III clinical study designed to evaluate the efficacy and safety of TQB3454 in patients with advanced bile duct cancer with IDH1 mutations who have failed previous treatment with gemcitabine and fluoropyrimidine-based combination regimens. The interim analysis results of this study showed that, compared to the control group, TQB3454 significantly reduced the risk of disease progression or death in patients with advanced bile duct cancer, and significantly prolonged their progression-free survival and overall survival. Its safety data were consistent with known risks, and no new safety signals were identified.

This is the second globally and the first domestically successful Phase III clinical study of an IDH1 inhibitor in the field of bile duct cancer. It is expected to fill the clinical gap in precision targeted therapy for IDH1-mutated bile duct cancer in China and provide a new treatment option for patients with this specific molecular subtype.

Due to its outstanding clinical value, TQB3454 was granted Breakthrough Therapy Designation by the CDE in April 2023. The company will announce detailed study data at a major international academic conference later this year. Biliary tract cancer (BTC) mainly includes bile duct cancer (including intrahepatic, perihilar, and distal bile duct cancer) and gallbladder cancer, accounting for about 3% of all digestive system tumors^[1]. Its incidence is increasing year by year and is relatively high in Asian countries. The main pathological histology type of BTC is adenocarcinoma. At initial diagnosis, it often exhibits advanced invasive characteristics, with a surgical resection rate of only 20%-30% and a post-resection recurrence rate as high as 50%-60%, leading to a very poor prognosis for patients^[2,3].

IDH1 inhibitors are an important component of precision therapy for bile duct cancer. Currently, no drug with the same target has been approved for the treatment of bile duct cancer in China, leaving a significant unmet clinical need. As the marketing application for TQB3454 progresses, this drug is expected to bring a new treatment option for patients with IDH1-mutated bile duct cancer in China, further advancing the development of precision medicine for bile duct cancer. 

References:
[1] ANDERSEN JB, SPEE B, BLECHACZ BR, et al.Genomicand genetic characterization of cholangiocarcinoma identi-fies therapeutic targets for tyrosine kinase inhibitors[J].Gas-troenterology, 2012, 142(4): 1021-1031.[2] Scott AJ, Sharman R, Shroff RT.Precision medicine in biliary tractcancer[J].J Clin Oncol, 2022, 40(24): 2716-2734.[3] ZHAO D Y, LIM K H. Current biologics for treatment ofbiliary tract cancers[J].J Gastrointest Oncol, 2017,8(3):430-440.

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