On June 15, 2025 Abbisko Therapeutics (HKEX Code: 02256) reported that it has completed first patient dosing in a registrational study of irpagratinib, a self-developed and highly-selective small molecule FGFR4 inhibitor, for the treatment of Hepatocellular Carcinoma (HCC) (Press release, Abbisko Therapeutics, JUN 15, 2025, View Source [SID1234653901]).
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In May, irpagratinib received Breakthrough Therapy Designation from the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA). It is the first therapeutic agent to utilize molecularly defined biomarkers for precision-targeted treatment of HCC.
The vast majority of patients with advanced HCC treated with current standard-of-care therapies—including immune checkpoint inhibitors (ICIs) and multi-targeted kinase inhibitors (mTKIs)— experience disease progression within one year. Additionally, approximately 30% of HCC patients exhibit FGF19 overexpression, a biomarker associated with more aggressive tumor biology and poorer prognosis. The registrational study of irpagratinib (ABSK-011-205) is a multi-center, randomized, double-blind, placebo-controlled clinical trial designed to evaluate the efficacy and safety of irpagratinib in combination with Best Supportive Care (BSC) versus placebo in combination with BSC, in patients with advanced or unresectable HCC who exhibit FGF19 overexpression and have previously been treated with ICIs and mTKIs. Eligible patients will be randomized in a 2:1 ratio to receive irpagratinib or placebo.
About Irpagratinib (ABSK-011)
Irpagratinib is a highly-selective FGFR4 small molecule inhibitor designed to target overexpression of the FGF19 signaling pathway. Several epidemiological studies indicate that approximately 30% of HCC patients worldwide exhibit FGF19 overexpression. Development of targeted therapies against FGFR4 represent an innovative and novel approach to the treatment of HCC.
To date, no FGFR4 inhibitor has been granted regulatory approval globally. According to Frost & Sullivan, irpagratinib is expected to become the first breakthrough treatment for the treatment of HCC patients with FGF19 overexpression.
In addition to monotherapy, Abbisko Therapeutics is exploring irpagratinib in combination with atezolizumab, an anti-PD-L1 antibody manufactured by F. Hoffmann-La Roche and Roche (China), in a Phase II study. At the previous 2024 ESMO (Free ESMO Whitepaper) GI Congress, Abbisko presented clinical data demonstrating 220mg irpagratinib BID in combination with atezolizumab achieved a 50% objective response rate (ORR) in FGF19+ HCC patients who had previously received immune checkpoint inhibition therapy.