Binds to and inhibits farnesyltransferase enzyme.
By inhibiting the farnesylation of protein, it prevents activation of certain oncogenes and inhibits cell growth and angiogenesis.
The FDA has granted breakthrough therapy designation to the investigational drug
Tipifarnib approved for the treatment of patients with recurrent or metastatic HRAS-mutant head and neck squamous cell carcinoma (HNSCC) with variant allele frequency of at least 20% following disease progression on platinum-based chemotherapy.
Phase 2 RUN-HN clinical trial evaluated tipifarnib in patients with recurrent or metastatic HRAS-mutant HNSCC.
Participants received a starting dose of either 600 mg or 900 mg of tipifarnib administered orally twice daily on days 1 through 7 and 15 through 21 of 28-day treatment cycles until progression of disease (PD) or unacceptable toxicity.
Demonstrated an overall response rate of 50% among evaluable patients.
The median duration of response was 14.7 months.
Median overall survival was 15.4 months.
Has activity in AML and in a phase II study 252 patients receiving 600 mg of this agent bid for 21 days in 28 day cycles appears to benefit such patients, with 11 complete remissions (Johnson and Johnson).
In a phase II study of poor risk elderly patients with AML a 14% complete response rate was noted (Lancet JE).
Appears to be synergistic wit bortezomib, and purine nucleoside analogues cladribine and fludarabine.