Inozyme Pharma, a clinical-stage biopharmaceutical company, announced that the US Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application and that the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) has authorized its Clinical Trial Application (CTA) for a phase 1/2 clinical trial evaluating INZ-701 in adults with ENPP1 deficiency. The company expects to enroll the first subject in the first half of 2021 and provide preliminary safety and biomarker data in the second half of 2021.
“With these important regulatory clearances for our first-in-human clinical trial for INZ-701 in subjects with ENPP1 deficiency, we have transitioned from a research-stage to a clinical-stage company. This is a significant milestone in our mission to develop therapeutic breakthroughs in diseases of abnormal mineralization,” said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. “We are pleased to begin 2021 by ramping up study start up activities and look forward to dosing subjects in the first half of the year.”
The phase 1/2 clinical trial is a multi-center, open-label, first-in-human, and multiple ascending dose study in adults with ENPP1 deficiency. The trial is expected to enroll nine adult subjects across three dose cohorts with three subjects per cohort. Subjects will participate in a pre-dosing screening period followed by a four-week treatment period in which subjects will receive INZ-701 subcutaneously twice weekly. The phase 1/2 clinical trial will primarily investigate the safety and tolerability of INZ-701 and characterize its pharmacokinetic and pharmacodynamic profile, including plasma pyrophosphate (PPi) and other biomarker levels, to establish a recommended dosing regimen for further clinical development. Exploratory objectives include obtaining baseline measurements of calcification, patient reported outcomes and quality of life.
INZ-701 is a soluble, recombinant protein containing the extracellular domain of native human ENPP1 fused to the Fc domain of the immunoglobulin IgG1 that is designed to correct a defect in the mineralization pathway caused by ENPP1 and ABCC6 deficiencies. In preclinical studies conducted in ENPP1-deficient and ABCC6-deficient mouse models, dosing with INZ-701 resulted in normalized levels of PPi and reduced tissue calcification. The FDA has granted orphan drug, rare pediatric disease, and fast track designations to INZ-701 for the treatment of ENPP1 deficiency. The European Medicines Agency has also granted orphan drug designation to INZ-701 for the treatment of ENPP1 deficiency