Pegtibatinase

Phase 3Protein / enzyme

TTravere Therapeutics, Inc.

Pegtibatinase is one of 5 small molecule programs targeting Homocystinuria. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

HomocystinuriaActive, not recruiting

HomocystinuriaPhase 3

Active, not recruiting

Competition

Competitive Landscape

4 competitors in Homocystinuria

#DrugCompanyPhaseTrials

Acetadote

AArbor Pharmaceuticals, Inc.

Approved

—2

Cysteine Hydrochloride

Novartis

Phase 2

2 3

Cystadane

RRECORDATI GROUP

Phase 2

1 4

Tylenol

KKenvue Brands

Phase 2

1 5

Pegtibatinase

TTravere Therapeutics, Inc.

Phase 3

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

Timeline

Expected Milestones

Q4 2026

Phase 1/2Active, not recruiting

Homocystinuria

NCT03406611

Clinical

Trials by Indication[1]

Homocystinuria3 trials

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

Active, not recruiting·TTravere Therapeutics, Inc.·Phase 3·NCT06247085·70 enrolled

Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)

Active, not recruiting·TTravere Therapeutics, Inc.·Phase 1/2·NCT03406611·32 enrolled

A Phase 3 Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

Enrolling by invitation·TTravere Therapeutics, Inc.·Phase 3·NCT06431893·100 enrolled

Sources

References

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

Details

Drug Info

Quick Facts

Modality: Protein / enzyme[2]
Highest Phase: Phase 3[1]
Company: TTravere Therapeutics, Inc.

Pegtibatinase

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Trials by Indication[1]

Homocystinuria3 trials

References

Drug Info

Quick Facts

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