BOSTON, June 13, 2018 /PRNewswire/ — Samus Therapeutics, Inc. (“Samus” or the “Company”), a privately held Boston-based biopharmaceutical company developing novel therapeutics and biomarkers targeting the epichaperome, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PU-H71 for the treatment of myelofibrosis. The Company also announced today that it has dosed the first patient in a Phase 1b dose-escalation study of PU-H71 in patients with myelofibrosis.
PU-H71, along with PU-AD, are novel therapeutics that, with high specificity, target the epichaperome, a foundational protein complex present in multiple disease states, including cancer and neurological disorders. Under conditions of abnormal stress, cells become biochemically ‘rewired’ to form a network of high-molecular-weight complexes known as epichaperomes. In cancer, these can function as a network to enhance cellular survival, irrespective of tissue of origin or genetic background. PU-H71 specifically targets the epichaperome, present in over half of all cancers tested by Samus and the Chiosis group at Memorial Sloan Kettering Cancer Center, to precisely interfere with the epichaperome function in diseased cells and to not affect normal cells. (Nature Reviews Cancer, 2018, doi:10.1038/s41568-018-0020-9; Nature, 538, 397-401, 2016).