Our lead drug candidate, tipifarnib, is an inhibitor of protein farnesylation, a key cell signaling process implicated in cancer initiation and development. Tipifarnib has been studied in more than 5,000 patients and has demonstrated anti-cancer activity in certain patient subsets and a well-established safety profile. Clinical and preclinical data suggest that in the right therapeutic context, tipifarnib has the potential to provide significant benefit to cancer patients with limited treatment options.
Chronic myelomonocytic leukemia (CMML) is a disorder of bone marrow stem cells in which an increase in white blood cells, or monocytosis, is a defining feature. The clinical presentation of CMML varies from predominantly myelodysplastic, an ineffective production of blood cells, to predominantly myeloproliferative, an overproduction of blood cells. A prior Phase 2 clinical trial of tipifarnib sponsored by Johnson & Johnson in adult patients with intermediate to high risk MDS, included 19 patients with CMML (according to the French American British (FAB) classification). The results of this study suggest that tipifarnib may produce durable responses as a single agent in patients with CMML.
CMML is primarily a disease of the elderly with a median age at diagnosis of 65 to 75 years, and an estimated annual incidence of approximately 1,280 patients in the United States. The prognosis of CMML is poor, with a median survival of 2 to 3 years and a 15% to 20% risk of transformation to acute myeloid leukemia, or AML. Management of CMML typically focuses on supportive care as therapeutic options are limited.
Tipifarnib is currently in a Phase 2 clinical trial for the treatment of patients with CMML.