PHILADELPHIA, PA — FORE Biotherapeutics announced that its investigational therapy plixorafenib has cleared an interim efficacy analysis in the Phase 2 FORTE basket study for patients with recurrent or progressive BRAF V600-mutated primary central nervous system (CNS) tumors. Following review, the Independent Data Monitoring Committee recommended the trial continue as planned.
The analysis, conducted after the first 25 patients had sufficient response data, confirmed ongoing tumor regressions and met the study’s pre-specified efficacy threshold. FORE expects to complete enrollment and release topline results for this CNS basket in the second half of 2026.
“We are very pleased this study passed the interim analysis, reinforcing that tumor regressions continue to be observed,” said Stacie Peacock Shepherd, M.D., Ph.D., Chief Medical Officer at FORE. She noted that plixorafenib’s differentiated mechanism, known as a “paradox breaker,” could address the shortcomings of earlier BRAF inhibitors.
Macarena de la Fuente, M.D., Chief of Neuro-Oncology at the University of Miami Sylvester Comprehensive Cancer Center and lead CNS investigator, highlighted the drug’s tolerability and potential to reduce side effects common with current therapies, such as rash, fever, or cardiac complications. “We anticipate plixorafenib has the potential to transform the treatment paradigm for people with BRAF-altered recurrent or refractory primary CNS tumors,” she said.
The primary endpoint is overall response rate, supported by duration of response, in up to 50 patients. If successful, FORE believes the results could support a New Drug Application under the FDA’s Accelerated Approval pathway.
Plixorafenib is also being studied in solid tumors with BRAF fusions and rare BRAF V600 mutations as part of the broader FORTE trial, a global Phase 2 master protocol.
BRAF-altered CNS tumors remain a high unmet need, with limited treatment options and poor outcomes. FORE’s progress marks a potential step toward a new standard of care in this difficult-to-treat patient population.
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