GTX-102

Phase 3Small molecule

Ultragenyx

GTX-102 is one of 6 small molecule programs targeting Angelman syndrome. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Angelman SyndromeActive, not recruiting

Angelman SyndromePhase 3

Active, not recruiting

Competition

Competitive Landscape

6 competitors in Angelman Syndrome

#DrugCompanyPhaseTrials

Lodosyn

LLaTonya J. Hickson

Approved

—2

Obudanersen

Ionis Pharmaceuticals

Phase 3

2 3

ION582

Ionis Pharmaceuticals

Phase 3

2 4

GTX-102

Ultragenyx

Phase 3

4 5

Rugonersen

OOak Hill Bio Ltd

Phase 3

2 6

Cystadane

RRECORDATI GROUP

Preclinical

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

Timeline

Expected Milestones

Q4 2027

Phase 3Active, not recruiting

Angelman Syndrome

NCT06617429

Q1 2029

Phase 3Active, not recruiting

Angelman Syndrome

NCT06415344

Q1 2030

Phase 2Active, not recruiting

Angelman Syndrome

NCT07157254

Clinical

Trials by Indication[1]

Angelman syndrome4 trials

A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome

Completed· Ultragenyx logo

Ultragenyx·Phase 1/2·NCT04259281·74 enrolled

A Long-term Extension Trial Investigating the Safety and Efficacy of GTX-102 in Patients With Angelman Syndrome

Active, not recruiting· Ultragenyx logo

Ultragenyx·Phase 3·NCT06415344·255 enrolled

A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects With Deletion- or Nondeletion-type Angelman Syndrome

Active, not recruiting· Ultragenyx logo

Ultragenyx·Phase 2·NCT07157254·60 enrolled

Show 1 more trial

A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects With Angelman Syndrome

Active, not recruiting· Ultragenyx logo

Ultragenyx·Phase 3·NCT06617429·129 enrolled

Sources

References

[1]
ClinicalTrials.gov — 4 trial records for GTX-102. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — GTX-102 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for GTX-102. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Ultragenyx company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: Ultragenyx

GTX-102

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2027

Q1 2029

Q1 2030

Trials by Indication[1]

Angelman syndrome4 trials

References

Drug Info

Quick Facts

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