BMN 401 / BioMarin - LARVOL DELTA

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency (clinicaltrials.gov) - P3 | N=2 | Terminated | Sponsor: Inozyme Pharma | N=12 ➔ 2 | Active, not recruiting ➔ Terminated; Following review of the totality of program data, the sponsor decided to discontinue development of INZ701. The decision was not related to safety.

Enrollment change • Trial termination • ENPP1

Efficacy and Safety of BMN 401 in Children With ENPP1 Deficiency: 52-Week Results From the Pivotal ENERGY 3 Trial (ASBMR 2026) - No abstract available

Clinical • Late-breaking abstract • ENPP1

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency (clinicaltrials.gov) - P2 | N=22 | Active, not recruiting | Sponsor: Inozyme Pharma | Recruiting ➔ Active, not recruiting | N=200 ➔ 22 | Trial completion date: Dec 2030 ➔ Oct 2026 | Trial primary completion date: Nov 2030 ➔ Oct 2026

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • ENPP1

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency (clinicaltrials.gov) - P3 | N=12 | Active, not recruiting | Sponsor: Inozyme Pharma | Recruiting ➔ Active, not recruiting | Trial completion date: Nov 2028 ➔ Oct 2026 | Trial primary completion date: Sep 2028 ➔ Oct 2026

Enrollment closed • Trial completion date • Trial primary completion date • ENPP1

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency (clinicaltrials.gov) - P1 | N=16 | Active, not recruiting | Sponsor: Inozyme Pharma | Recruiting ➔ Active, not recruiting | Trial completion date: Nov 2027 ➔ Oct 2026 | Trial primary completion date: Nov 2027 ➔ Oct 2026

Enrollment closed • Trial completion date • Trial primary completion date • ENPP1

Generalized Arterial Calcification of Infancy (GACI): State of the Art and Clinical Perspectives. (PubMed, J Cardiovasc Dev Dis) - "This review provides a comprehensive overview of the pathophysiology, genetic basis, and clinical features of GACI. In addition, we summarize current and emerging therapeutic strategies, including enzyme replacement therapy with recombinant ENPP1 (INZ-701), critically discussing available preclinical and early clinical evidence, as well as current limitations."

Journal • Review • Cardiovascular • Congestive Heart Failure • Heart Failure • Hypertension • ABCC6 • ENPP1

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency (clinicaltrials.gov) - P1 | N=16 | Recruiting | Sponsor: Inozyme Pharma | Trial completion date: Apr 2026 ➔ Nov 2027 | Trial primary completion date: Mar 2026 ➔ Nov 2027

Trial completion date • Trial primary completion date • ENPP1

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency (clinicaltrials.gov) - P3 | N=12 | Recruiting | Sponsor: Inozyme Pharma

New P3 trial • ENPP1

Calciphylaxis diagnosis, management and future directions: a comprehensive update on behalf of the European Renal Association CKD-MBD Working Group. (PubMed, Clin Kidney J) - "Recent investigational therapies, including SNF472 and INZ-701, target key calcification pathways and offer promise. Looking ahead, future research should prioritize the development and validation of diagnostic criteria, and prognostic tools integrating clinical risk factors with biomarkers. In addition, we propose the routine inclusion of patient-reported experience measures in calciphylaxis studies to better capture treatment impact in this vulnerable population."

Journal • Review • Calciphylaxis • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Inflammation • Pain • Rare Diseases • Renal Disease • Thrombosis • MGP

Recombinant ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) decreases vascular calcification and prevents osteomalacia in a rat model of chronic kidney disease. (PubMed, JBMR Plus) - "Moreover, INZ-701 treatment significantly prevented the osteoid volume increase observed in vehicle-treated rats, addressing another critical clinical manifestation of CKD-MBD. These results underscore the potential of INZ-701 to reduce vascular calcification and bone mineralization abnormalities in CKD."

Journal • Preclinical • Cardiovascular • Chronic Kidney Disease • Nephrology • Orthopedics • Renal Disease • ENPP1

The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: Inozyme Pharma | N=40 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • FGF23

The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency (clinicaltrials.gov) - P3 | N=27 | Active, not recruiting | Sponsor: Inozyme Pharma | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2025 ➔ Feb 2026 | Trial primary completion date: May 2025 ➔ Jan 2026

Enrollment closed • Trial completion date • Trial primary completion date • ENPP1

Inozyme Pharma Reports Full Year 2024 Financial Results and Announces Strategic Prioritization of ENPP1 Deficiency Pivotal Program and Recent Business Highlights (GlobeNewswire) - "The Company anticipates completing the one-year dosing period for all patients by January 2026, with topline data expected in first quarter of 2026."

P3 data: top line • Trial status • Rare Diseases

Inozyme Pharma to Present Recently Announced Interim Data for INZ-701 in Infants and Young Children with ENPP1 Deficiency at CHOP Cardiology 2025 (GlobeNewswire) - "Inozyme Pharma, Inc...today announced that...data from the company’s Expanded Access Program (EAP) evaluating INZ-701 in infants and children with ENPP1 Deficiency at the CHOP Cardiology Annual Meeting, held February 19-23, 2025, in Orlando, Florida."

Clinical data • Genetic Disorders • Rare Diseases

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency (clinicaltrials.gov) - P1/2 | N=9 | Completed | Sponsor: Inozyme Pharma | Active, not recruiting ➔ Completed

Trial completion • ENPP1

The SEAPORT 1 Study: Evaluation of the Safety and Tolerability of INZ-701 in Adults With End-Stage Kidney Disease Undergoing Hemodialysis (clinicaltrials.gov) - P1 | N=11 | Completed | Sponsor: Inozyme Pharma | Active, not recruiting ➔ Completed

Trial completion • Calciphylaxis • Chronic Kidney Disease • Nephrology • Rare Diseases • Renal Disease

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE (clinicaltrials.gov) - P1/2 | N=10 | Completed | Sponsor: Inozyme Pharma | Trial primary completion date: Nov 2024 ➔ Jul 2024 | Active, not recruiting ➔ Completed | Trial completion date: Dec 2024 ➔ Jul 2024

Trial completion • Trial completion date • Trial primary completion date • ABCC6

The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency (clinicaltrials.gov) - P2 | N=40 | Not yet recruiting | Sponsor: Inozyme Pharma

New P2 trial

The SEAPORT 1 Study: Evaluation of the Safety and Tolerability of INZ-701 in Adults With End-Stage Kidney Disease Undergoing Hemodialysis (clinicaltrials.gov) - P1 | N=11 | Active, not recruiting | Sponsor: Inozyme Pharma | Trial primary completion date: Dec 2024 ➔ Jul 2024

Trial primary completion date • Calciphylaxis • Chronic Kidney Disease • Nephrology • Rare Diseases • Renal Disease

SEAPORT 1: An Open-label Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of INZ-701 in Participants with ESKD Undergoing Hemodialysis: Interim Analysis (KIDNEY WEEK 2024) - "Median PPi at screening in 13 patients with ESKD on hemodialysis was substantially lower than what is reported in healthy volunteers (1002-2169 nM; n=10), which supports previous findings implicating PPi in the pathophysiology of ESKD. INZ-701 safety, tolerability, pharmacokinetics, and pharmacodynamics results will be presented."

Clinical • PK/PD data • Calciphylaxis • Chronic Kidney Disease • Diabetes • Diabetic Nephropathy • Infectious Disease • Metabolic Disorders • Nephrology • Pain • Rare Diseases • Type 2 Diabetes Mellitus • ENPP1

Novel treatment for PXE: Recombinant ENPP1 enzyme therapy. (PubMed, Mol Ther) - "Recent studies demonstrated that INZ-701, a recombinant human ENPP1, the principal PPi-generating enzyme, restored plasma PPi levels and prevented ectopic calcification in the muzzle skin of an Abcc6-/- mouse model of PXE...When Abcc6-/- mice were injected weekly from 5 to 15 weeks of age, BL-1118 dose-dependently increased steady-state plasma ENPP1 activity and PPi levels and significantly reduced ectopic calcification in the muzzle skin and kidneys. These results suggest that BL-1118 is a promising enzyme therapy for PXE, a disease currently lacking preventive or therapeutic options."

Journal • ABCC6 • ENPP1

The SEAPORT 1 Study: Evaluation of the Safety and Tolerability of INZ-701 in Adults With End-Stage Kidney Disease Undergoing Hemodialysis (clinicaltrials.gov) - P1 | N=15 | Active, not recruiting | Sponsor: Inozyme Pharma | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2024 ➔ Jan 2025 | Trial primary completion date: Aug 2024 ➔ Dec 2024

Enrollment closed • Trial completion date • Trial primary completion date • Calciphylaxis • Chronic Kidney Disease • Nephrology • Rare Diseases • Renal Disease

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency (clinicaltrials.gov) - P2 | N=200 | Recruiting | Sponsor: Inozyme Pharma | Not yet recruiting ➔ Recruiting

Enrollment open

The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency (clinicaltrials.gov) - P1 | N=16 | Recruiting | Sponsor: Inozyme Pharma | Trial completion date: Apr 2025 ➔ Apr 2026 | Trial primary completion date: Mar 2025 ➔ Mar 2026

Trial completion date • Trial primary completion date • ENPP1

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency (clinicaltrials.gov) - P2 | N=200 | Not yet recruiting | Sponsor: Inozyme Pharma

New P2 trial