Kura Oncology Inc. said its lead product candidate tipifarnib in chronic myelomonocytic leukemia, or CMML, met its main objective in a phase 2 study.
Preliminary results from the trial showed that nine patients with RAS wild-type CMML who used tipifarnib achieved stable disease — meaning the disease did not shrink or grow — or better, including three objective responses. The main objective was met with an overall response rate of 33% in patients with RAS wild-type CMML.
CMML is a type of blood cancer in which cancer cells form in the bone marrow and invade the blood.
The study enrolled 24 patients, of whom 16 were evaluable for response as of Nov. 7. Nine patients had tumors with RAS wild-type status and seven were RAS mutant. The drug was generally well-tolerated in the study, with side effects consistent with its known safety profile.
"Although the signal observed in RAS wild-type patients is encouraging, these data are still immature and time to event endpoints cannot be yet fully evaluated," Kura Chief Medical Officer Antonio Gualberto said.
Kura is also evaluating tipifarnib in phase 2 trials in HRAS mutant head and neck squamous cell cancer, or HNSCC; peripheral T-cell lymphoma; and myelodysplastic syndrome. The company intends to initiate a registration-enabling study of tipifarnib in HRAS mutant HNSCC in 2018.
