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Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

5
Pipeline Programs
2
Companies
7
Clinical Trials
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
6 programs
1
1
1
2
INZ-701Phase 31 trial
INZ-701Phase 3
INZ-701Phase 21 trial
INZ-701Phase 1/21 trial
INZ-701Phase 1
+1 more programs
Active Trials
NCT07745179Completed23Est. Feb 2025
NCT04686175Completed9Est. Dec 2024
NCT06739980Withdrawn0Est. Mar 2028
+1 more trials
BioMarin Pharmaceutical
1 program
INZ-701PHASE_33 trials
Active Trials
NCT07473973Active Not Recruiting12Est. Oct 2026
NCT06283589Completed11Est. Dec 2024
NCT05734196Active Not Recruiting16Est. Oct 2026

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
BioMarin PharmaceuticalINZ-701
Inozyme PharmaINZ-701
Inozyme PharmaINZ-701
Inozyme PharmaINZ-701
BioMarin PharmaceuticalINZ-701
BioMarin PharmaceuticalINZ-701
Inozyme PharmaNatural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 Deficiency

Clinical Trials (7)

Total enrollment: 98 patients across 7 trials

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

Start: Mar 2025Est. completion: Oct 202612 patients
Phase 3Active Not Recruiting

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

SEAPORT 1: Study of INZ-701 in Adults With Hemodialysis-Dependent End-Stage Kidney Disease

Start: Feb 2024Est. completion: Dec 202411 patients
Phase 1Completed

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

Start: Jun 2023Est. completion: Oct 202616 patients
Phase 1Active Not Recruiting
NCT07745179Inozyme PharmaNatural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 Deficiency

Natural History Study: ENPP1 Deficiency or the Early-Onset Form of ABCC6 Deficiency

Start: Dec 2018Est. completion: Feb 202523 patients
N/ACompleted

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 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.