PHILADELPHIA, PA — The U.S. Food and Drug Administration recently granted Breakthrough Therapy Designation to plixorafenib, an investigational drug from FORE Biotherapeutics, for the treatment of adults with BRAF V600E-mutated high-grade glioma, a form of aggressive brain cancer.
The designation was based on data from approximately 25 patients in a completed Phase 1/2a trial and an ongoing Phase 2 study evaluating the drug in central nervous system tumors.
Breakthrough Therapy Designation is intended to expedite the development and review of treatments for serious conditions that show early evidence of substantial improvement over existing therapies.
“High-grade gliomas are aggressive primary brain tumors associated with poor outcomes,” said Macarena da la Fuente, chief of neuro-oncology at the University of Miami Miller School of Medicine, citing the need for more effective and better-tolerated treatments.
Plixorafenib is a BRAF inhibitor designed to target genetic mutations linked to tumor growth, with a mechanism intended to avoid limitations seen in earlier therapies.
Clinical data presented previously showed a 67% overall response rate in a subgroup of patients with BRAF V600-mutated primary CNS tumors, along with extended duration of response and clinical benefit across tumor types.
The company is conducting the global Phase 2 FORTE trial, which includes multiple study arms evaluating plixorafenib in different patient populations, including recurrent or progressive brain tumors.
Interim results from the CNS tumor cohort met predefined criteria, allowing the study to continue under ongoing safety monitoring.
FORE Biotherapeutics said it expects topline results from the CNS portion of the FORTE trial by the end of 2026 and indicated that positive data could support a New Drug Application under the FDA’s accelerated approval pathway.
The drug has also received Fast Track and Orphan Drug designations for cancers involving BRAF mutations and central nervous system tumors.
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