Prexigebersen (Liposomal Grb2 Antisense) for Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is characterized by the rapid accumulation of immature myeloid cells in the blood, resulting in a drop of the other cell types, such as red blood cells and platelets. AML incidence increases with age, with amajority of patients aged 60 or older. AML is the most common acute leukemia in adults. The National Cancer Institute estimates that approximately 20,000 new cases occur each year. There is a critically unmet need for non-toxic therapies for older, fragile AML patients who are unfit or ineligible for high-dose chemotherapy or a stem cell transplant. The cure rate is between 5 to 15% in older adults, and those who cannot receive the standard course of chemotherapy have an average survival rate of five to ten months.

Prexigebersen (Liposomal Grb2 antisense) is Bio-Path’s lead product candidate, a neutral-charge, liposome-incorporated antisense drug designed to inhibit protein synthesis of Grb2 (growth factor receptor bound protein 2). Grb2 plays an essential role in cancer progression via the RAS activation pathway.

Grb2 is an adapter protein that bridges signals between activated and mutated tyrosine kinases, such as Flt3, c-Kit, and Bcr-Abl, and the Ras pathway, leading to activation of downstream ERK and AKT proteins. Inhibition of Grb2 by prexigebersen represents a significant advance in treating cancers with activated tyrosine kinases using a target not druggable with small molecule inhibitors. Inhibition of Grb2 has been demonstrated to halt cell propagation and enhance cell killing by chemotherapeutic agents without added toxicity. Prexigebersen has received orphan drug designation from the FDA and from the European Medicines Agency for AML.

Completed and Planned Studies