On January 14^th, 2020, Betta announced that it recently received a notice from the Center for Drug Evaluation (CDE) of the NMPA that the investigational new drug (IND) application of BPI-43487 program has been accepted.

BPI-43487 is a novel chemical compound developed by Betta. BPI-43487 is an oral small molecule inhibitor with high selectivity and efficacy targeting fibroblast growth factor receptor 4 (FGFR4), and it is intended for to treat solid tumors such as hepatocellular carcinoma (HCC) with fibroblast growth factor 19 (FGF19) amplification and cholangiocellular carcinoma.

FGFR4 is a member of the FGFR tyrosine kinase receptor family, which is highly expressed in liver tissues. It can utilize endogenous FGF19 as its intracellular ligand to activate FGFR4 to promote liver cell proliferation and thus causes HCC. Clinical studies have confirmed that FGFR4 inhibitors can reduce the tumors of HCC or advanced solid tumor patients with activated FGF19 / FGFR4 pathway. BPI-43487 inhibits cancer proliferation by specifically targeting the catalytic activity of FGFR4, reducing the phosphorylation level of downstream proteins, and blocking the activation of downstream signaling pathways that regulate tumor cell proliferation and survival. As of now, all FGFR4 inhibitors for FGF19-amplified HCC patients are in their early clinical development stage, and none of them have been launched to the market. According to the Announcement on the Work Plan for the Reform of the Registration and Classification of Chemical Drugs (No. 51, 2016) issued by the former China Food and Drug Administration, BPI-43487 is thus categorized as a Category I innovative drug that has not been listed globally.

Dr. Jiabing Wang, senior vice president and chief scientific officer of Betta, emphasized that liver cancer is a malignant tumor with a high mortality rate second only to lung cancer in China, of which HCC is the most common type. The treatment option is limited for HCC and the 5-year survival rate of patients is less than 15%. Current treatment option for HCC is surgery for the early-stage disease. However, patients are often diagnosed with HCC at the advanced stage where surgical resection and chemotherapy are no longer suitable options, therefore researchers are constantly trying to find new mechanisms to combat HCC. Betta strives to expand the field of targeted oncology therapy. As an IND-approved project independently developed by Betta, BPI-43487 shows high potency on inhibiting FGFR4, has a good selectivity and in vivo metabolic profile, and a good safety profile according to the pre-clinical studies. We hope to start the clinical study of BPI-43487 as soon as possible and provide a new treatment option for HCC patients.

About Betta

Betta Pharmaceuticals Co., Ltd. (Stock code: 300558.SZ) is a national level high-tech enterprise, established by the experienced talent team of overseas returnees, with research and development (R&D) of innovative drugs as its core focus, integrating R&D, manufacturing and marketing. The company independently developed China's first small-molecule targeted anticancer drug, icotinib hydrochloride (trade name: Conmana). It was recognized as a major breakthrough in the Chinese public healthcare industry and has won China Patent Gold Award twice as well as the first National Scientific and Technological Progress Award in China's chemical and pharmaceutical industry. Until the third quarter in 2019, icotinib has benefited 230,000 patients of advanced lung cancer, with the cumulative sales reaching nearly 7 billion RMB. The company has established a comprehensive new drug R&D platform, with its R&D centers in Hangzhou and Beijing, and more than 30 innovative drugs under development, covering multiple tumor indications such as lung cancer, renal cancer, and breast cancer. The company has also reached strategic collaboration with leading domestic and foreign pharmaceutical companies such as Amgen, Xcovery, Tyrogenex, Merus, Mabworks, and InventisBio, to work together to promote the health and well-beings of the general public.