UPI-928 Granted Orphan Drug Status for Acute Myeloid Leukemia
Update Pharma announced that the FDA has granted Orphan Drug Status for the use of UPI-928 for the treatment of Acute Myeloid Leukemia (AML).
UPI-928 is an agent with many mechanisms of action, including direct cytotoxic actions, immunologic mechanisms, and genomic effects. Immunologically, UPI-928 has been found to activate immune responses in a manner that can cure tumors in allogeneic transplant models. Further, it binds to DNA at a site that displaces telomerase binding proteins, which enables tumor cells to regain their mortality, become senescent, and die of old age. Due to these multiple mechanisms of action, UPI-928 may have a role in combination regimens that bridge the use of immune and cytotoxic agents.
“UPI-928 is an anthracene that lacks the lifetime dosage limit associated with other anthracyclines and has been useful in the treatment of refractory or recurrent patients who were heavily pretreated with anthracyclines, which makes it a potentially meaningful drug for the treatment of AML,” mentioned John Rothman, PhD.
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