Drug intelligence / Profile preview

ATR-1072

Development stage
Phase 2
Lead developer
Atrium Therapeutics
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Cytotoxic ADCs → Antibody-Drug Conjugates (ADCs) → Antibody Conjugates → Antibody-Based Therapeutics
01

Overview

ATR-1072 is an antibody oligonucleotide conjugate (AOC) therapeutic developed by Atrium Therapeutics for the treatment of PRKAG2 syndrome, a rare genetic cardiomyopathy. The drug consists of a monoclonal antibody conjugated to a small interfering RNA (siRNA) payload, utilizing the AOC platform to achieve tissue-selective delivery directly to the heart. ATR-1072 works through a sequence-specific gene silencing mechanism to reduce the expression of PRKAG2 mRNA. By lowering PRKAG2 levels, the therapeutic aims to normalize AMP-activated protein kinase (AMPK) activity and reduce the pathogenic accumulation of glycogen in cardiac tissue, with the ultimate goal of improving heart function. ATR-1072 is currently in IND-enabling studies, with an investigational new drug (IND) filing anticipated in the second half of 2026.

02

Targets

PRKAG2 (Protein kinase AMP-activated non-catalytic subunit gamma 2)

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