25 Sep 2023, Shanghai—Abbisko Therapeutics Co., Ltd. (“Abbisko” hereafter) today announced that Irpagratinib (ABSK-011), a highly selective small molecule inhibitor of fibroblast growth factor receptor 4 (“FGFR4”) in patients with advanced hepatocellular carcinoma（HCC）has obtained investigational new drug (IND) approval from the U.S. FDA.

The approved study is “A Phase 1, Open-Label Study of ABSK-011 to Assess Safety, Tolerability, and Pharmacokinetics in Patients with Advanced Solid Tumors”. The study population was mainly FGF19-overexpressing HCC patients. This is the first clinical trial of Irpagratinib conducted by Abbisko outside of China.

In December 2022, Abbisko announced preliminary Phase I results of Irpagratinib in second-line HCC patients with FGF19 overexpression in China，it showed excellent safety and efficacy.

Abbisko will also present an update on Irpagratinib's Phase I clinical trial at the European Society of Oncology (ESMO) 2023 Annual Meeting in October.

Meanwhile, Abbisko is also conducting a Phase II trial of Irpagratinib in combination with the anti-PD-L1 antibody atezolizumab produced by Roche in advanced HCC patients with FGF19 overexpression in China's mainland, and first patient dose was completed in February 2022.

According to statistics from the International Agency for Research on Cancer (IARC) of the World Health Organization (WHO), primary liver cancer is the sixth most common cancer globally, with nearly 906,000 new cases and 830,000 deaths in 20201. In the US, there are more than 42,000 new cases and 31,000 deaths2. Notably, China accounts for approximately 50% of the incidence and mortality of liver cancer worldwide (more than 410,000 new cases and 391,000 deaths)3.

 IARC predicts that by 2040, new cases and deaths of liver cancer will further increase. Generally speaking, the treatment of liver cancer is difficult, the prognosis is poor, and the ratio of morbidity vs mortality is as high as 1:0.9, which seriously threatens human life and health. HCC is the main type of liver cancer, accounting for 85% to 90% of primary liver cancers4. HCC is highly malignant, and the existing treatment methods still cannot meet the long-term survival benefits. For the treatment of HCC, there is still a huge unmet clinical need.

About Irpagratinib

Irpagratinib, a highly selective FGFR4 small molecule inhibitor, intended for the treatment of advanced solid tumors, especially in the presence of abnormalities in the FGFR4 signaling pathway (such as: ligand FGF19 amplification/overexpression, FGFR4 mutation/amplification/fusion, etc.) of advanced HCC, cholangiocarcinoma, breast cancer, etc. The FGFR4 signaling pathway is a promising direction for the development of molecularly targeted therapies in HCC. Irpagratinib demonstrated improved potency and anti-tumor efficacy compared to competitors as well as favorable physical-chemical properties in preclinical studies. We believe that Irpagratinib is potentially a novel and leading FGFR4 inhibitor for the treatment of HCC patients with hyper-activation of FGF19/FGFR4 pathway, based on Frost & Sullivan’s global competitive landscape of FGFR4 inhibitors.

Reference：

1.     World Health Organisation: Globocan 2020 – Liver Factsheet. Available at: https://gco.iarc.fr/today/data/factsheets/cancers/11-Liver-fact-sheet.pdf

2.     World Health Organisation: Globocan 2020 – United States of America Factsheet. Available at: https://gco.iarc.fr/today/data/factsheets/populations/840-united-states-of-america-fact-sheets.pdf

3.     World Health Organisation: Globocan 2020 – China Factsheet. Available at: http://gco.iarc.fr/today/data/factsheets/populations/160-china-fact-sheets.pdf

4.     El-Serag HB, Rudolph KL. Hepatocellular carcinoma: epidemiology and molecular carcinogenesis. Gastroenterology. 2007;132(7):2557-2576. doi:10.1053/j.gastro.2007.04.061