Final Operational Milestone #3
We have completed study of ten increasing doses of the small molecule drug candidate CFI-400945 fumarate in a first-in-human study in patients with advanced solid tumors. In this dose-escalation phase, a total of 47 patients were screened, 46 patients were enrolled, and 43 patients were treated. The 64 mg/day dose level was determined to be the maximum tolerated dose for CFI-400945 fumarate and the dose escalation phase of the trial was closed. One patient with adenoid cystic carcinoma, being treated at the 48 mg dose level, has had stable disease for 38 months, had 12% reduction in tumor size and is doing clinically well.
Beginning in December 2016, the dose expansion phase of the trial was open for enrollment at the 64 mg/day dose level. There have been 15 patients enrolled in this expansion phase at this point, mostly with gastrointestinal cancers, but unfortunately in this cohort, no significant efficacy was seen and as a consequence this cohort was closed to further enrollment.
In a continuation of the study independent of CIRM funding, a new cohort of patients is enrolling in an alternative dosing schedule that is hypothesized to improve overall exposure to the drug. In addition, efforts have been initiated to study this compound in a separate trial in patients with Acute Myeloid Leukemia.