Shanghai-based oncology biopharma firm Abbisko Therapeutics (HKEX: 02256) announced on Monday that it has received Breakthrough Therapy Designation from China's Center for Drug Evaluation (CDE) for irpagratinib (ABSK011), its highly selective FGFR4 inhibitor, for the treatment of hepatocellular carcinoma (HCC) patients with FGF19 overexpression.

The designation follows encouraging Phase I clinical data and targets a critical unmet need in advanced or unresectable HCC, particularly in patients who have failed immune checkpoint inhibitor (ICI) and multi-target kinase inhibitor (mTKI) therapies. FGF19 overexpression, present in roughly 30% of HCC cases, is linked to poor prognosis.

Irpagratinib is the first targeted agent leveraging molecularly defined biomarkers for precision treatment in HCC. No FGFR4 inhibitor has yet received global regulatory approval. Abbisko recently initiated a pivotal registrational study at leading Chinese institutions including Tongji Hospital and Nanjing Tianyinshan Hospital.

A combination study with atezolizumab, an anti-PD-L1 therapy from F. Hoffmann-La Roche and Roche (China), is underway. Data presented at the 2024 ESMO GI Congress showed a 50% objective response rate at the 220 mg BID dose in FGF19+ patients post-ICI treatment.

Founded in 2016, Abbisko has a strong pipeline in precision and immuno-oncology.