EPI-743 /kg

Phase 2Small molecule

PPTC Therapeutics

EPI-743 /kg is one of 3 small molecule programs targeting Leigh syndrome. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Leigh SyndromeActive, not recruiting

Leigh SyndromePhase 2

Active, not recruiting

Competition

Competitive Landscape

4 competitors in Leigh Syndrome

#DrugCompanyPhaseTrials

Rapamune

PPrism Pharmaceuticals

Phase 2

1 2

EPI-743

EEdison Pharmaceuticals Inc

Phase 2

1 3

EPI-743 /kg

PPTC Therapeutics

Phase 2

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

Clinical

Trials by Indication[1]

Leigh syndrome1 trial

Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

Completed·PPTC Therapeutics·Phase 2·NCT01721733·35 enrolled

Sources

References

[1]
ClinicalTrials.gov — 1 trial record for EPI-743 /kg. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — EPI-743 /kg compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for EPI-743 /kg. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — PTC Therapeutics company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: PPTC Therapeutics

EPI-743 /kg

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Leigh syndrome1 trial

References

Drug Info

Quick Facts

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