A recent study “Tipifarnib in Head and Neck Squamous Cell Carcinoma With HRAS Mutations” published in the Journal of Clinical Oncology evaluated the efficacy of the farnesyltransferase inhibitor tipifarnib in patients with recurrent and/or metastatic head and neck squamous cell carcinoma (R/M HNSCC) with HRAS mutations, a unique subset of the disease with high unmet needs.
The study enrolled 30 patients with R/M HNSCC in a single-arm, open-label phase II trial of tipifarnib for mutant HRAS malignancies. Mutant HRAS status for enrollment was documented by local, approved gene-sequencing platforms; all patients submitted tissue from the most recent tumor biopsy for central laboratory confirmation and variant allele frequency (VAF) determination with OncoKDM, our NGS data interpretation platform.
For patients with R/M HNSCC with high HRAS variant allele frequency (≥ 20%), tipifarnib treatment produced an objective response rate of 55% and a median overall survival of 15.4 months. The safety profile of tipifarnib was tolerable and manageable in this phase II trial. The results demonstrated encouraging clinical activity with tipifarnib for patients with R/M HNSCC with HRAS mutations for whom limited therapeutic options exist.
