On June 2, 2015 Aptose Biosciences reported that the U.S. Food and Drug Administration (FDA) has granted the company orphan drug designation for APTO-253 for the treatment of acute myeloid leukemia (AML) (Press release, Aptose Biosciences, JUN 2, 2015, View Source [SID:1234505214]). APTO-253, a first-in-class inducer of the KLF4 gene, is the company’s lead product candidate in a Phase Ib clinical trial in patients with AML, high-risk myelodysplastic syndrome (MDS) and other hematologic malignancies in which KLF4 silencing is reported as operative.
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"AML is a particularly challenging cancer of the blood and bone marrow for which there are currently few treatment options," said William G. Rice, Ph.D., Chairman, President and CEO. "APTO-253, with its unique mechanism of action, has the potential to emerge as an entirely new therapeutic approach for this patient population, and receiving orphan drug designation is a key regulatory milestone along the path."
Epigenetic suppression of the Krüppel-like factor 4 (KLF4) gene has been reported in the scientific literature as a key transforming event in AML. APTO-253 is a first-in-class, targeted inducer of the KLF4 tumor suppressor gene, and has demonstrated a favorable safety profile with no evidence of suppression of the normal bone marrow. Preclinical studies have shown potent single-agent activity to kill AML cells and strong synergy as part of a combination strategy with various marketed and investigational agents. APTO-253 is currently in a Phase 1b clinical study in patients with relapsed or refractory hematologic malignancies.
Orphan drug designation is granted by the FDA to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 individuals in the United States. Orphan drug status provides research and development tax credits, an opportunity to obtain grant funding, exemption from FDA application fees and other benefits. If APTO-253 is approved to treat AML, the orphan drug designation provides Aptose with seven years of marketing exclusivity.
About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer derived from myeloid progenitor or stem cells that typically mature into red blood cells, white blood cells or platelets. AML initiates in the bone marrow when stem or progenitor cells lose cell cycle control, anti-apoptotic factor or other means to limit rampant proliferations. Leukemic cells have the ability to rapidly spread from the marrow to the bloodstream. Further, these rapidly proliferating cells quickly crowd out normal cells as they infiltrate other organs and tissue systems.
AML is the most common type of acute leukemia among adults, with an annual incidence of more than 18,000 patients, and causing more than 10,000 deaths each year in the U.S. It is a particularly devastating blood cancer, with less than 25 percent of newly diagnosed patients surviving beyond five years.