Elevar Therapeutics, the U.S. subsidiary of HLB (028300.KQ), has begun dosing the first patient in a global Phase 2 trial to expand the tumor-agnostic indication of lirafugratinib, the company said Friday. Lirafugratinib is a fibroblast growth factor receptor 2 (FGFR2) fusion and rearrangement targeted cancer therapy currently under review by the U.S. Food and Drug Administration (FDA) for approval as a bile duct cancer drug.
Tumor-agnostic therapies are precision-medicine-based cancer drugs that treat patients with specific gene mutations regardless of the organ in which the cancer originated. Through this Phase 2 trial, Elevar is expected to verify the potential to expand the treatment area beyond bile duct cancer to various solid tumors by targeting FGFR2 mutations.
The Phase 2 trial evaluates the efficacy and safety of lirafugratinib in patients with confirmed FGFR2 fusion or rearrangement among those with unresectable, locally advanced, or metastatic solid tumors who have prior treatment experience. The primary endpoint is the objective response rate (ORR), and the trial is being conducted as a multicenter study in five countries including the United States, Korea, the United Kingdom, Spain, and France. The first patient was recently dosed at Samsung Medical Center, and patient enrollment and dosing have also proceeded at the Moffitt Cancer Center in the United States.
In earlier global Phase 1/2 trials, lirafugratinib confirmed meaningful anticancer activity in 13 types of FGFR2 fusion and rearrangement solid tumor patient groups, excluding bile duct cancer. Based on an integrated dataset combining those trial data with the results of this global Phase 2 trial, Elevar plans to conduct an interim analysis after securing data on five or more patients per tumor type across at least seven tumor types.
"Lirafugratinib is a next-generation targeted cancer therapy that precisely targets FGFR2 mutations, and it holds the potential to expand the treatment area beyond bile duct cancer to various solid tumors," Elevar CEO Kim Dong-gun said. "Now that patient recruitment has begun in Korea and the United States, we will accelerate the progress of the global trial and pursue our strategy to expand the tumor-agnostic indication without disruption."
Meanwhile, lirafugratinib has received Orphan Drug Designation (ODD) and Breakthrough Therapy Designation (BTD) from the FDA for its bile duct cancer indication. A priority review is currently underway for the same indication. The final decision on approval will be made by Sept. 27 this year.
