Safety and Efficacy of Tipifarnib in Head and Neck Cancer With HRAS Mutations and Impact of HRAS on Response to Therapy

Summary

Objectives

KO-TIP-007 is an international, multicenter, open-label, 2 cohort, non-comparative,
pivotal study evaluating the efficacy of tipifarnib in HRAS mutant HNSCC (AIM-HN) and the
impact of HRAS mutations on response to first line systemic therapies for HNSCC (SEQ-HN).
KO-TIP-007 has 2 study cohorts. The first study cohort, named AIM-HN, includes HNSCC
subjects with HRAS mutations. AIM-HN subjects will receive treatment with tipifarnib and
the outcome of this cohort will be evaluated for ORR by an independent review facility.

The second study cohort, SEQ-HN, is an observational sub-study including HNSCC patients
in whom HRAS mutations were not identified (wild type HRAS HNSCC) and who consent to
provide first line outcome data and additional follow up.

HNSCC patients in whom HRAS mutations are identified and who meet eligibility criteria
will be offered participation in AIM-HN. HNSCC patients in whom HRAS mutations are not
identified may participate in SEQ-HN only. These patients will be followed and the
comparison of outcomes of HRAS mutant and HRAS wild type HNSCC will address the
exploratory objective to determine the effect of HRAS mutation on the ORR of first line
systemic therapy in patients with recurrent/metastatic HNSCC. Outcome data from
subsequent lines of therapy will be collected.