Aptose Biosciences Granted Orphan Drug Designation By U.S. FDA for APTO-253 In Acute Myeloid Leukemia

Industry News

The U.S. FDA has granted Orphan Drug Designation to APTO-253, an investigational drug of Aptose Biosciences, a Ontario, Canada based biotechnology company for the treatment of acute myeloid leukemia (AML).  AML is a rare form of cancer derived from myeloid stem cells in the bone marrow.  It is a challenging blood cancer to treat and currently there are few treatment options.  It has been shown in the literature that epigenetic suppression of the Krüppel-like factor 4 (KLF4) gene may be a key oncogenic event leading to AML.  APTO-253 is a targeted inducer of the KLF4 tumor suppressor gene.  Preclinical studies have demonstrated favorable outcomes with APTO-253 against AML cells.  According to Aptose Biosciences, APTO-253 is currently in a Phase Ib clinical trial in patients with relapsed or refractory hematologic malignancies.


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