Arginase I Deficiency

Pipeline Programs

Companies

Clinical Trials

Approved Products

Pipeline by Development Stage

Preclinical

Phase 1

Phase 1/2

Phase 2

Phase 2/3

Phase 3

On Market

Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Pre

P1/2

P2/3

Mkt

BioTherapeutics IncVA - Blacksburg

4 programs

PegzilarginasePhase 31 trial

AEB1102Phase 21 trial

AEB1102Phase 1/21 trial

Active Trials

NCT02488044Completed16Est. Feb 2019

NCT03378531Completed14Est. Dec 2022

NCT05676853Terminated3Est. Apr 2023

+1 more trials

Trial Timeline

Clinical trial activity over time

2021

2022

2023

2024

2025

2026

BioTherapeutics IncPegzilarginase

BioTherapeutics IncAEB1102

Clinical Trials (4)

Total enrollment: 65 patients across 4 trials

NCT05676853 BioTherapeutics IncPegzilarginase

A Study of Safety of Weekly Subcutaneous Pegzilarginase in Subjects With Arginase 1 Deficiency

Start: Apr 2023Est. completion: Apr 20233 patients

Phase 3Terminated

NCT03921541 BioTherapeutics IncPegzilarginase

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency

Start: Apr 2019Est. completion: Jan 202332 patients

Phase 3Completed

NCT03378531 BioTherapeutics IncAEB1102

A Study of AEB1102 (Pegzilarginase) in Patients With Arginase I Deficiency

Start: Dec 2017Est. completion: Dec 202214 patients

Phase 2Completed

NCT02488044 BioTherapeutics IncAEB1102

A Phase 1/2 Study of AEB1102 in Patients With Arginase I Deficiency

Start: Jun 2016Est. completion: Feb 201916 patients

Phase 1/2Completed

Phase Legend

Preclinical— Lab & animal studies

Phase 1— Safety & dosing

Phase 2— Efficacy testing

Phase 3— Large-scale trials

On Market— Approved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals

1 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.