HONG KONG, SHANGHAI & FLORHAM PARK, N.J., June 25, 2026
HUTCHMED (Nasdaq/AIM:HCM; HKEX:13) announced that pivotal Phase II registration study results for fanregratinib (HMPL-453) in patients with advanced intrahepatic cholangiocarcinoma (ICC) harboring FGFR2 fusion or rearrangement alterations will be presented at the ESMO Gastrointestinal Cancers Congress 2026 in Munich, Germany. The data support a New Drug Application (NDA) that was accepted for review and granted Priority Review by China’s National Medical Products Administration (NMPA) in December 2025. Fanregratinib is being developed as a targeted therapy for previously treated patients with advanced, metastatic, or unresectable ICC, a rare and aggressive form of bile duct cancer with limited treatment options after progression on standard therapies.
Strong Response Rates Demonstrate Clinical Benefit
The registration-enabling Phase II, multicenter, open-label study enrolled patients across 53 clinical sites in China who had previously received at least one systemic treatment regimen. All participants had undergone chemotherapy, while 72% had also received immunotherapy. The trial successfully achieved its primary endpoint, demonstrating an Independent Review Committee-assessed Objective Response Rate (ORR) of 42.5%. Additional efficacy results highlighted rapid and durable anti-tumor activity, with a median time to response of just 1.4 months, a Disease Control Rate (DCR) of 83.9%, and a median Duration of Response (DoR) of 6.9 months. The study also reported a median Progression-Free Survival (PFS) of 6.9 months and a median Overall Survival (OS) of 16.6 months, supporting the potential of fanregratinib to provide meaningful clinical benefit for patients with FGFR2-altered ICC.
Manageable Safety Profile Supports Continued Development
Fanregratinib demonstrated a manageable and predictable safety profile consistent with selective FGFR inhibition. Drug-related Grade 3 or higher adverse events occurred in 48.3% of patients, with the most common events including elevated liver enzymes and palmar-plantar erythrodysesthesia syndrome (PPES). Importantly, only 2.2% of patients discontinued treatment due to drug-related adverse events, and no treatment-related deaths were reported during the study. These findings suggest that fanregratinib may offer a favorable balance between efficacy and tolerability in a patient population with significant unmet medical needs and limited targeted treatment alternatives.
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Potential New Targeted Therapy for FGFR2-Altered ICC
Fanregratinib is a novel, highly selective inhibitor of FGFR1, FGFR2, and FGFR3, designed to block abnormal fibroblast growth factor receptor signaling that drives tumor growth, angiogenesis, and treatment resistance in multiple cancers. FGFR2 gene fusions and rearrangements are recognized oncogenic drivers in a subset of intrahepatic cholangiocarcinoma patients, making them an attractive target for precision oncology approaches. According to lead investigator Professor Jianming Xu of the Chinese PLA General Hospital, the study results represent an important advancement in the targeted treatment landscape for FGFR2-altered ICC. If approved, fanregratinib could become a valuable new treatment option for patients who have exhausted standard therapies, further strengthening HUTCHMED’s expanding oncology portfolio and commitment to developing innovative targeted cancer medicines.
Source: HUTCHMED press release
