HUTCHMED’s Fanregratinib Achieves Primary Endpoint in Pivotal Liver Cancer Trial]

HUTCHMED’s Fanregratinib Achieves Primary Endpoint in Pivotal Liver Cancer Trial

HUTCHMED (China) Limited (HCM) announced that its investigational fibroblast growth factor receptor (FGFR) inhibitor fanregratinib met the primary endpoint in a pivotal Phase 2 registrational study for intrahepatic cholangiocarcinoma (IHCC).

Fanregratinib (HMPL-453) is a selective small-molecule inhibitor targeting FGFR 1, 2, and 3. Abnormal FGFR signaling drives tumor proliferation, angiogenesis, and therapy resistance in cancer cells.

IHCC is a bile duct cancer occurring within the liver, representing the second most common primary liver cancer. Often diagnosed at advanced stages, it affects the bile ducts connecting the liver, gallbladder, and small intestine (Source: Mayo Clinic).

The Chinese National Medical Products Administration (NMPA) accepted and granted priority review for fanregratinib’s new drug application in December 2025, based on Phase 2 results. The therapy targets advanced, metastatic or unresectable FGFR2 fusion/rearrangement-positive IHCC following prior chemotherapy and immunotherapy.

The single-arm, multi-center Phase 2 study demonstrated an objective response rate (ORR) of 42.5% by independent review, meeting its primary endpoint. Secondary analyses showed sustained clinical activity with rapid onset (median response: 1.4 months). Median duration of response was 6.9 months, disease control rate reached 83.9%, median progression-free survival was 6.9 months, and median overall survival reached 16.6 months.

The selective FGFR inhibitor showed a manageable safety profile. Grade 3 or higher drug-related adverse events occurred in 48.3% of patients, primarily elevated liver enzymes and palmar-plantar erythrodysesthesia syndrome (PPES).

Phase 2 results will be presented at the European Society for Medical Oncology (ESMO) Gastrointestinal Cancers Congress on July 4, 2024 in Munich, Germany.

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