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. 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 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.
The Company’s lead program, APTO-253, can induce expression of the Kruppel-Like Factor 4 (KLF4) gene, providing a new therapeutic approach for the treatment of AML and MDS. Aptose appears to be the only company to seize on the abnormal silencing of KLF4 gene expression in patients with AML and MDS. Notable scientific publications report that KLF4 is a master transcription factor forms super enhancer 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. 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 c-Myc oncogene in AML cells, leading to apoptotic cell death in AML cells.
In June 2016, Aptose effected a definitive agreement with South Korean company, CrystalGenomics, Inc. (CG), granting Aptose an exclusive option to research, develop and commercialize CG026806 (CG’806) in all countries of the world except Korea and China, for all fields of use. CG’806 is a highly potent, non-covalent small molecule therapeutic agent, exhibiting picomolar IC50 toward the FMS-like tyrosine kinase 3 with the Internal Tandem Duplication (FLT3-ITD) and single-digit nanomolar IC50’s against Bruton’s tyrosine kinase (BTK) and its C481S mutant (BTK-C481S). .
CG’806 may become an effective therapy in the FLT3-ITD subset of AML patients. FLT3-ITD occurs in approximately 30% of patients with AML. Importantly, CG’806 targets other oncogenic kinases which may also be operative in FLT3-ITD AML, thereby potentially allowing the agent to become an important therapeutic option for a difficult-to-treat patient population, among other hematologic malignancies.
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.