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Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

5
Pipeline Programs
2
Companies
5
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
1
1
0
2
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Inozyme Pharma
Inozyme PharmaMA - Boston
5 programs
1
1
1
2
INZ-701Phase 31 trial
INZ-701Phase 31 trial
INZ-701Phase 21 trial
INZ-701Phase 1/21 trial
INZ-701Phase 11 trial
Active Trials
NCT05734196Recruiting16Est. Nov 2027
NCT04686175Completed9Est. Dec 2024
NCT06739980Withdrawn0Est. Mar 2028
+2 more trials
BioMarin Pharmaceutical
1 program
INZ-701PHASE_3

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
Inozyme PharmaINZ-701
Inozyme PharmaINZ-701
Inozyme PharmaINZ-701
Inozyme PharmaINZ-701
Inozyme PharmaINZ-701

Clinical Trials (5)

Total enrollment: 64 patients across 5 trials

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

Start: Mar 2025Est. completion: Nov 202812 patients
Phase 3Recruiting

The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency

Start: Nov 2023Est. completion: Feb 202627 patients
Phase 3Active Not Recruiting

The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency

Start: Jan 2025Est. completion: Mar 20280
Phase 2Withdrawn

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

Start: Nov 2021Est. completion: Dec 20249 patients
Phase 1/2Completed

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency

Start: Jun 2023Est. completion: Nov 202716 patients
Phase 1Recruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

2 late-stage (Phase 3) programs, potential near-term approvals
2 actively recruiting trials targeting 64 patients
2 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.