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Enzyme replacement therapy may help manage and treat rare, genetic disorders which otherwise may result in high morbidity and mortality.

Recombinant Protein Administered to First Infant with ENPP1 Deficiency

Phase 1b trial of INZ-701 aimed to improve quality of life in patients with rare, genetic diseases 

Inozyme Pharma Inc.
Published:Jul 04, 2023
|2 min read
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BOSTON, MA — Inozyme Pharma, Inc., a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, recently announced dosing of the first patient in its ENERGY-1 trial, a Phase 1b clinical trial of INZ-701 in infants with ENPP1 deficiency.

INZ-701—a recombinant Fc-fusion protein—is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton. In preclinical studies, the experimental therapy has shown the potential to prevent pathologic mineralization and overgrowth of smooth muscle cells inside blood vessels, which can drive morbidity and mortality in devastating genetic disorders. INZ-701 is currently in Phase 1/2 clinical trials for treating treat ENPP1 deficiency and ABCC6 deficiency.

“Infants diagnosed with ENPP1 deficiency face a high mortality risk in the first months of life. Those who survive this critical period often develop severe symptoms that adversely affect lifelong health and quality of life. I am excited to serve as a principal investigator in this trial as a first step towards delivering a potentially lifesaving therapy for this patient population,”, said David R. Weber, MD, MSCE, medical director of the Center of Bone Health, Division of Endocrinology and Diabetes at the Children’s Hospital of Philadelphia (CHOP).

The ENERGY-1 trial: What it entails

ENERGY-1 is a Phase 1b, single-arm, open-label clinical trial designed to primarily assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in infants with ENPP1 deficiency. The trial is expected to enroll up to eight infants between the ages of one and 12 months across multiple sites in the United States and Europe. Patients will receive subcutaneous doses of INZ-701 during the treatment period of 52 weeks and may continue to receive INZ-701 in an extension period beyond 52 weeks. 

The doses range from 0.2 mg/kg once weekly through 0.6 mg/kg twice weekly, with the ability to increase the dose further depending on the results of PK/PD and safety data. Additional outcome measures include the evaluation of plasma inorganic pyrophosphate (PPi) levels, survival, growth, development, functional performance, cardiac function, and exploratory biomarkers.

The company plans to provide a program update on global development plans for INZ-701 in patients with ENPP1 deficiency. The update will cover regulatory agreements on pivotal trial designs, including plans for an upcoming, pivotal trial of INZ-701 in pediatric patients, an overview of the ENPP1 deficiency opportunity, and ongoing patient identification efforts.

“Initiation of the ENERGY-1 trial in infants is an important milestone as we continue to advance INZ-701 with the goal of improving the lives of patients with ENPP1 deficiency across all age groups. We are committed to a global program to identify and treat all newborns with this condition,” said Kurt Gunter, MD, senior vice president and chief medical officer of Inozyme.

- This press release was originally published on the Inozyme Pharma Inc. website