Delving into the Latest Updates on APT-110 with Synapse

Overview

Basic Info

Drug Type

miRNA

Synonyms-

Target

miR-22

Mechanism

miR-22 inhibitors(microRNA 22 inhibitors)

Therapeutic Areas

Digestive System DisordersEndocrinology and Metabolic Disease

Active Indication

Diabetes Mellitus, Type 2Metabolic dysfunction-associated steatotic liver diseaseObesity

Inactive Indication-

Originator Organization

AptamiR Therapeutics, Inc.

Active Organization

AptamiR Therapeutics, Inc.

Inactive Organization-

Drug Highest PhasePreclinical

First Approval Date-

Regulation-

Structure

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IntroductionWe previously demonstrated in primary cultures of human subcutaneous adipocytes and in a mouse model of diet-induced obesity that specific microRNA-22-3p antagomirs produce a significant reduction of fat mass and an improvement of several metabolic parameters. These effects are related to the activation of target genes such asKDM3A,KDM6B,PPARA,PPARGC1BandSIRT1involved in lipid catabolism, thermogenesis, insulin sensitivity and glucose homeostasis.Research design and methodsWe now report a dedicated study exploring over the course of 3 months the metabolic and energetic effects of subcutaneous administration of our first miR-22-3p antagomir drug candidate (APT-110) in adult C57BL/6 male mice. Body composition, various blood parameters and energy expenditure were measured at several timepoints between week 12 and week 27 of age.ResultsWeekly subcutaneous injections of APT-110 for 12 weeks produced a sustained increase of energy expenditure as early as day 11 of treatment, a significant fat mass reduction, but no change of appetite nor physical activity. Insulin sensitivity as well as circulating glucose, cholesterol and leptin were improved. There was a dramatic reduction of liver steatosis after 3 months of active treatment. RNA sequencing revealed an activation of lipid metabolism pathways in a tissue-specific manner.ConclusionsThese original findings suggest that microRNA-22-3p inhibition could lead to a potent treatment of fat accumulation, insulin resistance, and related complex metabolic disorders such as obesity, type 2 diabetes mellitus and non-alcoholic fatty liver disease.

100 Deals associated with APT-110

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Diabetes Mellitus, Type 2	Preclinical	US	AptamiR Therapeutics, Inc.	-
Metabolic dysfunction-associated steatotic liver disease	Preclinical	US	AptamiR Therapeutics, Inc.	-
Obesity	Preclinical	US	AptamiR Therapeutics, Inc.	-