Grb-2 (Growth factor Receptor Bound protein-2) is an adaptor protein that links tyrosine kinases such as bcr-abl with their downstream signaling molecules, including MAPK and AKT, which are critical regulators of cancer proliferation and survival. Liposomal Grb-2 seeks to suppress the function or expression of Grb-2, interrupt the vital signaling function and have a therapeutic effect.
The Phase I clinical trial is being conducted at the MD Anderson Cancer Center and is a dose-escalating study to determine the safety and tolerance of escalating doses of Liposomal Grb-2, as well as the optimal biologically active dose. The study seeks a total of 18 to 30 evaluable patients and will evaluate six doses of Liposomal Grb-2 in six cohorts. An evaluable patient is a patient who has been able to complete the four-week treatment cycle.
As of November 2013, the Company has successfully completed five cohorts of the study and has treated two patients in the sixth cohort. The drug has been well tolerated and possible anti-leukemia effects have been observed. Enrollment is currently on-going for the sixth cohort of the trial and Bio-Path anticipates that enrollment for the Phase I study will be completed in the first quarter of 2014.