December 16, 2024 — Abbisko Therapeutics (HKEX Code: 02256) announced that its self-developed, highly selective small molecule FGFR4 inhibitor, irpagratinib (ABSK011), has received approval from the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) to conduct a registrational clinical trial for the treatment of Hepatocellular Carcinoma (HCC). 

This study will serve as the key basis for approval , marking a significant milestone for irpagratinib in the treatment of advanced or unresectable HCC. The registrational study, ABSK-011-205, is designed as a multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating 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 have FGF19 overexpression and have previously received immunotherapy (ICI) and multi-targeted kinase inhibitor (mTKI) therapies.

Patients with advanced or unresectable HCC currently lack effective treatment options following treatment with ICI and mTKI therapies. Those with FGF19 overexpression often face significantly worse prognosis, and thus new treatment options are urgently needed. Abbisko Therapeutics is preparing to launch a pivotal study that holds the promise of bringing renewed hope and transformative possibilities to this group of patients with substantial unmet medical need.

About Irpagratinib (ABSK011)

Irpagratinib is a highly selective FGFR4 small molecule inhibitor designed to target overexpression of the FGF19 signaling pathway. Multiple epidemiological studies indicate that approximately 30% of HCC patients worldwide have overexpression of FGF19. The development of targeted therapies against this signaling pathway represents an innovative and novel approach to the treatment of HCC.

Currently, no FGFR4 inhibitor has been approved worldwide. According to Frost & Sullivan, irpagratinib is expected to become the first breakthrough treatment for the treatment of aHCC patients with FGF19 overexpression.

In addition to monotherapy, Abbisko Therapeutics is exploring a Phase II trial of irpagratinib in combination with atezolizumab, an anti-PD-L1 antibody manufactured by F. Hoffmann-La Roche and Roche (China). At the 2024 ESMO-GI Congress, Abbisko presented updated clinical data demonstrating 220mg BID irpagratinib in combination with atezolizumab achieved a 50% objective response rate (ORR) in FGF19+ HCC patients who had received prior ICI therapy.