A Novel Small Molecule MYC Inhibitor
APTO-253 is a novel small molecule that inhibits expression of the MYC oncogene, leading to cell cycle arrest and apoptosis in human-derived solid tumor and hematologic cancer cells. APTO-253 is under development as a novel therapy for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).
About MDS and AML
MDS represents a group of closely related diseases arising from impaired hematopoietic stem cells in the bone marrow. Visually, the cells may appear abnormal, or dysplastic and patients with MDS may initially present with anemia, neutropenia, thrombocytopenia, or other hematological symptoms.
AML is a type of cancer that initiates in the bone marrow when stem cells or progenitor cells lose cell cycle control, anti-apoptotic factor, or other means to limit rampant proliferation. Leukemic cells can rapidly spread from the marrow to the bloodstream and quickly crowd out normal cells as they infiltrate other organs and tissue systems.
Addressing an Unmet Medical Need
Approximate MDS incidence per year or 4.4 to 4.6 cases per 100,000 people in the U.S.
Of MDS cases progress to AML, according to the American Cancer Society.
The estimated prevalence for AML as of January 1, 2010, according to the Leukemia & Lymphoma Society.
Patients with new AML diagnoses with about 10,460deaths in 2014, as projected by the American Cancer Society.