The FDA has granted a breakthrough therapy designation to tipifarnib for use in patients with recurrent or metastatic HRAS-mutated head and neck squamous cell carcinoma.
The FDA has granted a breakthrough therapy designation to tipifarnib for use in patients with recurrent or metastatic HRAS-mutated head and neck squamous cell carcinoma (HNSCC) with variant allele frequency (VAF) of 20% or higher following progressive disease on platinum-based chemotherapy.1 The decision is based on findings from the phase 2 RUN-HN trial (KO-TIP-001; NCT023383927) presented during the 2020 ASCO Virtual Scientific Program, in which tipifarnib elicited an objective response rate (ORR) of 50.0% (95% CI, 26.0%-74.0%) in response-evaluable patients with HRAS-mutant HNSCC and a high VAF.2
“We are very pleased that the FDA has awarded breakthrough therapy designation to tipifarnib, and we appreciate the agency’s affirmation of its potential to treat this devastating disease,” Troy Wilson, PhD, JD, president and chief executive officer of Kura Oncology, stated in a press release. “We remain focused on conducting our AIM-HN registration-directed trial and look forward to working closely with the FDA to bring this therapy to patients as soon as possible.”
The trial enrolled patients with solid tumors that harbored HRAS mutations. The first stage of the trial comprised 2 cohorts: patients with thyroid cancer (n = 13) and patients with a solid tumor other than thyroid cancer (n = 13). For the second stage of the trial, those in cohort 2 who had HNSCC moved into a HNSCC extension cohort, which could enroll up to 30 patients. Cohort 3 was also added, and this would enroll up to 20 patients with tumors other than HNSCC. Here, patients had to have a 35% or higher HRAS-mutant VAF or a 20% or higher HRAS VAF with serum albumin of 3.5 g/dL or higher.