China Biopharmaceutical (01177): TQB3454 tablet “IDH1 inhibitor” new drug marketing application accepted

Zhitong Finance App News, China Biopharmaceutical (01177) announced that the national Class 1 innovative drug TQB3454 “IDH1 Inhibitor” independently developed by the group subsidiary Zhengda Tianqing Pharmaceutical Group Co., Ltd. (Zhengda Tianqing) has submitted a new drug marketing application to the China National Drug Administration Drug Evaluation Center (CDE) and has been accepted for the treatment of advanced biliary tract cancer (BTC) with IDH1 mutations. With significant clinical benefits, TQB3454 was listed as a breakthrough treatment by CDE in April 2023 and included in the priority review and approval process in May 2026.

TQB3454 is an IDH1 inhibitor independently developed by the Group. It can specifically inhibit IDH1 mutase activity, reduce the level of 2-HG, a downstream carcinogenic metabolite, reverse the high methylation state of DNA and histone proteins caused by abnormal 2-HG elevation, and restore normal chromatin structure, thereby inducing mutant cells to restore normal differentiation and development processes and exert anti-tumor effects.

The marketing application is based on a randomized, double-blind, placebo-controlled, multicenter phase III clinical study (TQB3454-III-01) to evaluate the efficacy and safety of TQB3454 in patients with advanced biliary tract cancer with IDH1 mutations who have previously failed treatment with gemcitabine and fluorouracil. Research results showed that compared with placebo, TQB3454 can significantly reduce patients' risk of disease progression or death. Progression-free survival (PFS) and overall survival (OS) were significantly extended. The safety spectrum was consistent with known risks, and no new safety signals were found. This is the second phase III study of an IDH1 inhibitor with positive results in the field of biliary tract cancer in the world and the first in China.

Biliary tract cancer mainly includes cholangiocarcinoma (intrahepatic cholangiocarcinoma, hepatic cholangiocarcinoma, distal cholangiocarcinoma) and gallbladder cancer. It accounts for about 3% of all digestive system tumors, mainly adenocarcinoma. It is highly aggressive, has an extremely poor prognosis, and a 5-year survival rate of less than 5%. Globally, the incidence of biliary tract cancer is increasing year by year, with Asian countries having the highest incidence. Among biliary tract malignancies, IDH1 mutations mainly occur in intrahepatic cholangiocarcinoma, and the mutation rate is about 4.9% to 20.0%.

Currently, no same-targeted drugs have been approved for treatment of biliary tract cancer in China, and clinical needs are far from being met. TQB3454 is expected to fill the market gap in this field and provide a new and efficient treatment option for domestic IDH1 mutant biliary tract cancer patients.