Pegtibatinase

Phase 3Protein / enzyme

TTravere Therapeutics, Inc.

Pegtibatinase is one of 2 small molecule programs targeting Homocystinuria. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

HomocystinuriaRecruiting

HomocystinuriaPhase 3

Recruiting

Competition

Competitive Landscape

1 competitor in Homocystinuria

#DrugCompanyPhaseTrials

Acetadote

AArbor Pharmaceuticals, Inc.

Approved

—2

Pegtibatinase

TTravere Therapeutics, Inc.

Phase 3

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

Timeline

Expected Milestones

Q3 2027

PreclinicalRecruiting

Homocystinuria

NCT03406611

Phase 3Recruiting

Homocystinuria

NCT06247085

Clinical

Trials by Indication[1]

Homocystinuria3 trials

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

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effects on Clinical Outcomes of Pegtibatinase (TVT-058) Administered Subcutaneously in Subjects With Cystathionine Beta Synthase-Deficient Homocystinuria (COMPOSE)

Recruiting·TTravere Therapeutics, Inc.·Preclinical·NCT03406611·39 enrolled

A Phase 3, Parallel-Group Treatment, Blinded, Randomized, Placebo-Controlled Study To Assess The Efficacy And Safety Of Pegtibatinase Administered Subcutaneously In Addition To Standard Of Care In Participants With Classical Homocystinuria Due To Cystathionine Beta Synthase Deficiency (HARMONY)

Recruiting·TTravere Therapeutics, Inc.·Phase 3·NCT06247085·70 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

Q3 2027

Trials by Indication[1]

Homocystinuria3 trials

References

Drug Info

Quick Facts

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