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ATR 1072

Phase 1

Cardiac Glycogenosis | Small molecule | Neurology |Atrium Therapeutics, Inc.|Trials Updated: Sep 18, 2026

ATR 1072 development status

Highest phase Phase 1
Registered trials 1 across 1 sponsor since Jul 2026

ATR 1072 target and mechanism

Molecular targetPRKAG2
Target classGene
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment37

FDA Designations

No designations recorded

ATR 1072 clinical trials

ATR 1072 · 1 trial · 4 indications

Phase 1 1
NCT07738107ATR 1072 in Participants With PRKAG2 SyndromeCardiac Glycogenosis
RECRUITING37 Analytics
PHASE1RECRUITING
ATR 1072 in Participants With PRKAG2 Syndrome
Cardiac GlycogenosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Incidence of treatment-emergent adverse events (TEAEs)
From first dose through end of study (approximately 48 weeks per participant)

Incidence and severity of treatment-emergent adverse events (TEAEs).

Secondary Endpoints

Change from baseline in cardiac muscle tissue concentration of ATR 1072 siRNA
Through Week 24
Plasma pharmacokinetic (PK) parameters of ATR 1072
Through Week 24
Plasma pharmacokinetic parameters of ATR 1072
Through week 48
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part A - MAD Dose Escalation Description Three sequential ascending doseEXPERIMENTALThree sequential ascending dose cohorts (Dose Level 1, Dose Level 2 and Dose Level 3) with approximately 4 evaluable participants per cohort. ATR 1072 administered intravenously (IV) every 6 weeks (Q6W) for up to 8 doses (48-week treatment period).
Part B - Expansion Cohort at the Recommended Phase 2 DoseEXPERIMENTALApproximately 25 participants enrolled at the recommended phase 2 dose determined in Part A. ATR 1072 administered IV Q6W to further evaluate safety, PK, and pharmacodynamics.

Interventions

NameTypeDescription
ATR 1072DRUGATR 1072 is an investigational therapy that delivers an siRNA to reduce PRKAG2 gene expression. Administered as an intravenous (IV) infusion every 6 weeks (Q6W). Formulated as a sterile liquid for injection.
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Adults 18 to 65 years of age. 2. Pathogenic, likely pathogenic, or variant of uncertain significance (VUS) in PRKAG2 confirmed by genetic testing. 3. Clinical manifestations consistent with PRKAG2 Syndrome. 4. Able and willing to comply with study procedures. 5. Able and will...

Countries:United States
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Recent Changes (Last 90 Days)

LOWSep 19, 2026NCT07738107Status: NOT_YET_RECRUITING → RECRUITING
LOWSep 19, 2026NCT07738107Status: NOT_YET_RECRUITING → RECRUITING
LOWSep 19, 2026NCT07738107Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 31, 2026NCT07738107NEW_TRIAL: changed
LOWJul 31, 2026NCT07738107NEW_TRIAL: changed

Frequently asked questions about ATR 1072

What is ATR 1072?

ATR 1072 is an investigational small molecule developed by Atrium Therapeutics, Inc. for Cardiac Glycogenosis, a neurological therapeutic area condition. It is currently in Phase 1 clinical development and is being studied in participants with PRKAG2 syndrome, a genetic disorder that can cause hypertrophic cardiomyopathy and Wolff-Parkinson-White syndrome.

What is ATR 1072 used for in Cardiac Glycogenosis?

ATR 1072 is being developed for Cardiac Glycogenosis, specifically in participants with PRKAG2 syndrome. The Phase 1 trial enrolls adults with this condition, which can include hypertrophic cardiomyopathy and Wolff-Parkinson-White syndrome. It is investigational and has not been approved for any use.

What does ATR 1072 target?

ATR 1072 targets PRKAG2, a gene that encodes the gamma-2 subunit of AMP-activated protein kinase. Mutations in PRKAG2 cause Cardiac Glycogenosis, the condition ATR 1072 is being studied in. The drug is a small molecule designed to address the effects of this genetic target.

Who makes ATR 1072?

ATR 1072 is developed by Atrium Therapeutics, Inc., which trades under the ticker RNA. The company is running the clinical program for this investigational small molecule in Cardiac Glycogenosis associated with PRKAG2 syndrome.

What phase is ATR 1072 in?

ATR 1072 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA or any regulatory agency. The Phase 1 program includes one active, recruiting trial with a total planned enrollment of 37 participants.

What clinical trials is ATR 1072 in?

ATR 1072 is being studied in one Phase 1 trial, NCT07738107, titled ATR 1072 in Participants With PRKAG2 Syndrome. The trial is recruiting in the United States, enrolls 37 adults aged 18 and older of all sexes, and uses a controlled design without randomization or double blinding.