Aptose Biosciences is a science-driven biotechnology company advancing first-in-class therapeutics to treat life-threatening cancers, such as acute myeloid leukemia (AML), high-risk myelodysplastic syndromes (MDS) and other hematologic malignancies. Aptose is developing targeted medicines for precision treatment of these diseases, based on a patient’s specific gene expression signature. The company is also creating proprietary companion diagnostic tools to identify predisposed sensitivities to targeted therapies for use in single-agent or combination regimens. In the treatment of cancer, this strategy is intended to optimize efficacy and quality of life by minimizing the cytotoxic side effects associated with conventional therapies.
Based on insights into the genetic and epigenetic profiles of certain cancers and patient populations, Aptose is building a pipeline of novel oncology therapies directed at dysregulated epigenetic processes and signaling pathways. Aptose appears to be the only company to seize on the Kruppel-Like Factor 4 (KLF4) gene’s distinctive expression profile in patients with AML and MDS. Notable scientific publications report that KLF4 is a master transcription factor forms super enhacer structures and guides cell identity. Moreover, KLF4 gene expression is epigenetically suppressed in patients with AML and in other hematologic cancers and that this gene suppression may serve as a key leukemogenic trigger. The Company’s lead program, APTO-253, can induce expression of the silenced KLF4 gene, providing a new therapeutic approach for the treatment of this disease. Furthermore, the c-Myc oncogene is known to epigenetically acquire super enhancers that dramatically upregulate c-Myc gene expression and contribute to oncogenesis. APTO-253 simultaneously can induce expression of the KLF4 master transcription factor and downregulate expression of the cMyc oncogene in AML cells, leading to apoptotic cell death in AML cells.
Aptose’s leadership team comprises accomplished industry, financial and clinical research professionals who are dedicated to building a comprehensive anticancer drug pipeline and clinical development programs focused on targeted therapeutics directed against dysregulated epigenetic processes.