Tideglusib

Phase 3Small molecule

AAMO Pharma Limited

Tideglusib is one of 47 small molecule programs targeting Myotonic Dystrophy. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Myotonic DystrophyCompleted

Progressive Supranuclear PalsyActive, not recruiting

Amyotrophic Lateral SclerosisNot yet recruiting

Myotonic DystrophyPhase 3

Completed

Progressive Supranuclear PalsyPreclinical

Active, not recruiting

Amyotrophic Lateral SclerosisPhase 2

Not yet recruiting

Competition

Competitive Landscape

55 competitors in Amyotrophic Lateral Sclerosis

#DrugCompanyPhaseTrials

Vimpat

Enablex

Nourianz

Kyowa Kirin Co., Ltd.

Approved

—4

Bosulif

PPrism Pharmaceuticals

Approved

1 5

Qalsody

Biogen

Approved

3 6

Atropen

FFresenius Kabi Taiwan Ltd.

Approved

—7

Quinidine Gluconate

WWyeth-Lederle Vaccines

Avodart

Transderm Scop

BBaxter Healthcare Corporation

Mirapex

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 2/3Recruiting

Myotonic Dystrophy

NCT05004129

Q3 2027

Phase 2Recruiting

Cardiomyopathy

NCT06174220

Clinical

Trials by Indication[1]

Myotonic Dystrophy4 trials

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

Recruiting·AAMO Pharma Limited·Phase 2/3·NCT05004129·76 enrolled

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

Completed·AAMO Pharma Limited·Phase 2·NCT02858908·16 enrolled

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Completed·AAMO Pharma Limited·Phase 2/3·NCT03692312·56 enrolled

Show 1 more trial

Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy

Available·AAMO Pharma Limited··NCT07119775

Progressive Supranuclear Palsy1 trial

Safety, Tolerability, and Efficacy of Two Different Oral Doses of NP031112 Versus Placebo in the Treatment of Patients With Mild-to-Moderate Progressive Supranuclear Palsy

Completed·NNoscira SA·N/A·NCT01049399·146 enrolled

Amyotrophic Lateral Sclerosis1 trial

Tideglusib for the Treatment of Amyotrophic Lateral Sclerosis

Not yet recruiting·UUniversity of Zurich·Phase 2·NCT05105958·98 enrolled

Autism Spectrum Disorder1 trial

Tideglusib vs. Placebo in the Treatment of Adolescents With Autism Spectrum Disorders

Completed·HHolland Bloorview Kids Rehabilitation Hospital·Phase 2·NCT02586935·83 enrolled

Cardiomyopathy1 trial

Targeted Therapy With Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic Cardiomyopathy

Recruiting·HHamilton Health Sciences Corporation·Phase 2·NCT06174220·120 enrolled

1 more indication

Mild-to-moderate Alzheimer's Disease Patients1 trial

Efficacy, Safety and Tolerability of Tideglusib to Treat Mild-to-Moderate Alzheimer's Disease Patients

Completed·NNoscira SA·Phase 2·NCT01350362·306 enrolled

Sources

References

[1]
ClinicalTrials.gov — 9 trial records for Tideglusib. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Tideglusib compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Tideglusib. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — AMO Pharma Limited company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: AAMO Pharma Limited

Tideglusib

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q3 2027

Trials by Indication[1]

Myotonic Dystrophy4 trials

Progressive Supranuclear Palsy1 trial

Amyotrophic Lateral Sclerosis1 trial

Autism Spectrum Disorder1 trial

Cardiomyopathy1 trial

Mild-to-moderate Alzheimer's Disease Patients1 trial

References

Drug Info

Quick Facts

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