AptamiR Therapeutics

AptamiR Therapeutics is a US-based biotechnology company focusing on the development of targeting OligoNucleotide Therapeutic (ONT) drug candidates. The company's research is centered on two main projects: miRNASH and OvamiR. The miRNASH project aims to treat cardiometabolic disorders such as obesity, dyslipidemia, diabetes, and metabolic dysfunction-associated fatty liver disease (MAFLD) by addressing fat accumulation, inflammation, and necrosis. The OvamiR project develops targeting microRNA ONTs to treat various aspects of Epithelial Ovarian Cancer, including proliferation, migration, and chemoresistance.

AptamiR's strategy employs the "One Drug-Multiple Targets" concept, developing microRNA ONTs that simultaneously modulate several target genes involved in complex diseases. The company has achieved proof of efficacy for its first generation of microRNA-based ONTs using primary cultures of human cells and in specific animal models. AptamiR is developing a second generation of miRNA antagomirs with a Peptide Nucleic Acid backbone, conjugated to a fatty acid or short peptide for targeted delivery to adipocytes. This approach aims to lower the effective dose and improve safety and pharmacokinetic profiles.

In a study published in BMJ Open Diabetes Research & Care in 2020, AptamiR's first miR-22-3p antagomir drug candidate (APT-110) demonstrated significant fat mass reduction and improvement in metabolic parameters in mice over a 12-week period. The treatment increased energy expenditure, improved insulin sensitivity, and reduced liver steatosis. These findings suggest that microRNA-22-3p inhibition could potentially treat fat accumulation, insulin resistance, and related metabolic disorders.