Pegtibatinase

Summary

Pegtibatinase (CHEMBL4297799) is a phase-3 clinical-stage enzyme; indicated across 1 condition including homocystinuria.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Enzyme
• Indications: 1 condition
• Clinical trials: 3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297799
Name	Pegtibatinase
Type	Enzyme
Max phase	3

Also known as: 20nhs peg-cbs, Ot-58, OT-58, Pegtibatinase, TVT-058, PEGTIBATINASE

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 disease in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
homocystinuria	3	MONDO:0004737	MONDO:0004737

Clinical trials

Total trials: 3.

Phase distribution

Phase	Trials
PHASE3	2
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06247085	PHASE3	RECRUITING	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
NCT06431893	PHASE3	ENROLLING_BY_INVITATION	A 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)
NCT03406611	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).