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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 3
INZ-701Phase 3
INZ-701Phase 21 trial
INZ-701Phase 1/21 trial
INZ-701Phase 1
+1 more programs
Active Trials
NCT07745179Completed23Est. Feb 2025
NCT04686175Completed13Est. Dec 2024
NCT06739980Withdrawn0Est. Mar 2028
BioMarin Pharmaceutical
1 program
INZ-701PHASE_34 trials
Active Trials
NCT07473973Terminated2Est. Aug 2026
NCT06283589Completed11Est. Dec 2024
NCT06046820Terminated27Est. Aug 2026
+1 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
BioMarin PharmaceuticalINZ-701
BioMarin PharmaceuticalINZ-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: 85 patients across 7 trials

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

Start: Mar 2025Est. completion: Aug 20262 patients
Phase 3Terminated

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

Start: Nov 2023Est. completion: Aug 202627 patients
Phase 3Terminated

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 202413 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: Sep 20269 patients
Phase 1Terminated
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

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
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.