Inozyme Pharma Announces Updates on Global Development Strategy of INZ-701 for the Treatment of ENPP1 Deficiency

- Change in plasma pyrophosphate (PPi) as primary endpoint in the U.S. and co-primary endpoint in the EU for planned ENERGY-3 pivotal trial in pediatric patients -

- ENERGY-3 pivotal trial in pediatric patients expected to initiate in October 2023, topline data expected to be reported in mid-2025 -

- Updated cash runway expected to fund cash flow requirements into Q1 2025 -

- Company to host conference call today at 8am ET -

BOSTON, July 26, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or the “Company”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced a regulatory update for its global development strategy of INZ-701 for the treatment of ENPP1 Deficiency following recent meetings with the United States (U.S.) Food and Drug Administration (FDA) and the Paediatric Committee (PDCO) of the European Medicines Agency (EMA).

“We are pleased to have finalized our pediatric pivotal trial design with PPi, a well-established natural inhibitor of mineralization, as a primary endpoint in the U.S. and a co-primary endpoint in the EU. We have already observed that INZ-701 meaningfully increased PPi levels in our ongoing trial of INZ-701 in adults with ENPP1 Deficiency and, based on our discussions with regulators in the U.S. and EU, we believe we have a clear path forward in our clinical development program for the treatment of ENPP1 Deficiency,” said Douglas A. Treco, Ph.D., CEO of Inozyme Pharma.

ENERGY-3 Trial Design – Planned Pivotal Trial in Pediatric Patients with ENPP1 Deficiency

The Company plans to initiate the ENERGY-3 pivotal trial, a multicenter, randomized, open label trial in pediatric patients with ENPP1 Deficiency in October 2023. The ENERGY-3 trial is expected to enroll up to 33 patients between the ages of one and less than 13 years across multiple sites globally and is designed primarily to assess the efficacy and safety of INZ-701 in pediatric patients with ENPP1 Deficiency. Enrollment criteria for the trial include a confirmed genetic diagnosis of ENPP1 Deficiency, radiographic evidence of skeletal abnormalities and low plasma PPi. Patients will be randomized in a 2:1 ratio to an INZ-701 arm or a control arm (conventional therapy, i.e., oral phosphate and active vitamin D) for 52 weeks, followed by an open label extension period during which all patients may receive INZ-701. INZ-701 will be administered at a 2.4 mg/kg once weekly dose via subcutaneous (SC) injection.