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.
Myelodysplastic syndromes (MDS) are a group of hematopoietic stem cell malignancies characterized by ineffective blood cell production, leading to low blood cells counts and related anemia, bleeding and infection. MDS patients have also a risk of progression to acute myeloid leukemia. A prior Phase 2 clinical trial of tipifarnib was sponsored by Johnson & Johnson and conducted in patients with intermediate to high risk MDS. The results of this study suggest that tipifarnib may produce durable responses in the treatment of patients with intermediate- to high-risk MDS.
According to the ACS, the annual incidence of MDS is approximately 13,000 patients in the United States, the majority of which are 60 years of age or older. The estimated prevalence is over 60,000 patients. Approximately 75% of patients fall into the IPSS-R risk categories of Very Low, Low, and Intermediate, collectively known as lower risk MDS.
Tipifarnib is currently being evaluated in a Phase 2 clinical trial for the treatment of patients with lower risk MDS.