Bio-Path Holdings, a biotechnology company based in Houston, TX, received Orphan Drug Designation from the FDA for its lead compound Liposomal Grb-2 for the treatment of acute myeloid leukemia (AML).  AML is a rare and fast-growing cancer of the blood plasma cells in the bone marrow.  Grb-2, Growth Receptor Bound protein 2, is an adaptor protein important in cancer cell progression and inhibition of Grb-2 function has been shown to be effective at suppressing tumor growth by interrupting cancer cell signaling.  Liposomal Grb-2 is a neutral-charged, liposome-incorporated antisense directed to reduce Grb-2 expression.  It is currently in a Phase 2 trial in combination with Ara-C for AML and is also being evaluated for chronic myelogenous leukemia.

 

References:

http://www.biopathholdings.com/pdf/ODD_for_AML20150428_000.pdf