HLB said on the 11th that lirafugratinib, a targeted anticancer drug under development as a treatment for cholangiocarcinoma, showed superior efficacy and safety to existing therapies in a phase 2 trial. Based on this, the company plans to file for U.S. Food and Drug Administration (FDA) approval this month to use lirafugratinib as a second-line treatment for cholangiocarcinoma.
HLB subsidiary Elevar Therapeutics on the 9th (local time) presented phase 2 results for lirafugratinib in cholangiocarcinoma patients with FGFR2 fusions or rearrangements, a cancer-causing genetic alteration, at the American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO GI 2026).
Antoine Hollebecque, professor at Gustave Roussy Cancer Center in France and the European principal investigator (PI) who delivered the presentation, said, "Lirafugratinib is a drug that precisely inhibits only FGFR2 and could be a meaningful treatment option for cholangiocarcinoma patients who have failed prior therapies."
In the trial, the objective response rate (ORR), the proportion of patients whose tumors visibly shrank, was 46.5%, and the disease control rate (DCR), the proportion whose disease did not worsen, was 96.5%. The median duration of response (mDOR) was 11.8 months. Even in the cohort that had failed other FGFR inhibitors, the drug showed anticancer activity, with an ORR of 23% and a DCR of 77%.
In particular, among 11 patients who had never received chemotherapy or an FGFR inhibitor, the ORR was 63%, the mDOR was 9.2 months, and the median progression-free survival (mPFS) was 11 months. Based on this, the academic community also suggested the potential for development as a first-line treatment for cholangiocarcinoma.
In terms of safety, lirafugratinib was generally assessed as predictable and manageable. Common adverse events included stomatitis and hand-foot syndrome, most of which were mechanism-of-action related.
The incidences of hyperphosphatemia and diarrhea, hallmark adverse events of FGFR inhibitors, were 20.7% and 21.6%, respectively. These rates are lower than those reported with the already approved cholangiocarcinoma treatments pemigatinib (hyperphosphatemia 60%, diarrhea 47%) and futibatinib (hyperphosphatemia 85%, diarrhea 39%).
Experts said the waterfall plot showing the degree of tumor shrinkage was particularly impressive. One expert said, "There are clear cases of deep tumor reduction, and the fact that the duration of response approaches one year is noteworthy." However, they also cautioned against making direct comparisons across studies.
Academia views lirafugratinib as potentially overcoming resistance to pan-FGFR inhibitors, suggesting it could become a new option for patients with limited treatments.
Buoyed by these positive assessments, Elevar plans to apply to the FDA this month for approval of lirafugratinib as a second-line treatment for cholangiocarcinoma. The drug was designated a breakthrough therapy in 2023, so it may be eligible for priority review.
Nam Kyung-suk, executive director of HLB Group's Bio Strategy and Planning Team, said, "These clinical results show that lirafugratinib is an effective targeted therapy that can be chosen after existing treatments," adding, "Along with the FDA filing, we will continue development as a tumor-agnostic therapy targeting FGFR fusions and rearrangements beyond cholangiocarcinoma."