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Tideglusib

Phase 2

Arrhythmogenic Cardiomyopathy | Small molecule | Cardiovascular |Rhythm Pharmaceuticals, Inc.|Last Updated: Mar 12, 2026

Target and mechanism

Molecular targetGSK3B
Target classInhibitor
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment120

FDA Designations

No designations recorded

Clinical trial landscape

Tideglusib · 1 trial · 2 indications

Phase 2 1
NCT06174220Targeted Therapy With Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic CardiomyopathyArrhythmogenic Cardiomyopathy
RECRUITING120 Analytics
PHASE2RECRUITING
Targeted Therapy With Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic Cardiomyopathy
Arrhythmogenic CardiomyopathyUnlock trial analytics

Study Endpoints

Primary Endpoints

PVC burden
Baseline and 6 months

Change in mean PVC count per 24 hours on 7-day Holter

Secondary Endpoints

Ventricular strain
Baseline and 6 months
Implantable cardioverter-defibrillator (ICD) therapies
Baseline and 6 months
Sustained ventricular tachycardia (VT) events
Baseline and 6 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
TideglusibACTIVE_COMPARATORRandomization to Tideglusib 1g po daily or matching placebo
PlaceboPLACEBO_COMPARATORRandomization to matching placebo 1g po daily

Interventions

NameTypeDescription
TideglusibDRUGTideglusib 1g po daily
PlaceboDRUGMatching placebo 1g po daily
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites17

Inclusion Criteria: * A pathogenic or likely pathogenic desmosomal (PKP2, DSG2, DSC2, DSP, or JUP\*) rare variant OR the TMEM43-p.S358L variant \*JUP carriers must be homozygous or compound heterozygous * Mean ≥ 500 PVCs per 24 hours on a baseline screening 7-day Holter monitor * Clinical ACM di...

Countries:Canada
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Competitive Landscape -Arrhythmogenic Right Ventricular Cardiomyopathy 3 trials (matched to "Arrhythmogenic Cardiomyopathy")

CompanyTickerTrialsLead PhaseDrugs
Lexeo Therapeutics, Inc.LXEO3PHASE1LX2020
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Frequently asked questions about Tideglusib

What is Tideglusib used for in Arrhythmogenic Cardiomyopathy?

Tideglusib is an investigational small molecule being studied for the treatment of Arrhythmogenic Cardiomyopathy, including Arrhythmogenic Right Ventricular Cardiomyopathy. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.

What does Tideglusib target?

Tideglusib is a glycogen synthase kinase-3 (GSK-3) inhibitor. It is being evaluated in a targeted therapy approach for Arrhythmogenic Cardiomyopathy, with biomarker-selected patient enrollment in the ongoing clinical trial.

Who makes Tideglusib?

Tideglusib is being developed by Rhythm Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol RYTM. The company is conducting a Phase 2 clinical trial for this drug in Arrhythmogenic Cardiomyopathy.

What phase is Tideglusib in?

Tideglusib is in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The ongoing Phase 2 trial is actively recruiting participants in Canada.

What clinical trials is Tideglusib in?

Tideglusib is being studied in a single Phase 2 clinical trial registered as NCT06174220, titled "Targeted Therapy With Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic Cardiomyopathy." This randomized, double-blind, placebo-controlled trial is recruiting 120 adult participants in Canada.

Is Tideglusib the same as a GSK-3 inhibitor?

Yes, Tideglusib is a glycogen synthase kinase-3 (GSK-3) inhibitor. The ongoing Phase 2 trial specifically evaluates this targeted therapy approach for Arrhythmogenic Cardiomyopathy, and the study is designed to enroll patients selected based on biomarkers.