Inozyme Pharma, Inc. 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). INZ-701 will be administered at a 2.4 mg/kg once weekly dose via subcutaneous (SC) injection. INZ-701 is a single, multicenter, clinical trial with differences in the statistical treatment of endpoints, based on guidance from the FDA and PDCO, as follows: U.S. EU Primary Endpoint -- Change in plasma Co-Primary Endpoints -- Change in plasma PPi from baseline and Radadiographic Global Impression of Change (RGI-C) score (p<0.2); Secondary Endpoints - RGI-C score, Secondary Endpoints -- RSS, Growth R tickets Severity Score (RSS), Growth Z-score and PK Z-score and pharmacokinetics (PK).

Bed on r recommendations from the FDA, the primary endpoint of plma PPi should be supported by consistent trendnds in appropriate secondary endpoints. Based on the agreed Paediatric Investigational Plan (PIP) with PDCO, plasma PPi and RGI-C are co-primary endpoints, with a relaxed p-value of <0.2 for RGI-C. Planned Pivotal Trials in Infants and Adolescent/adult Patients with ENPP1 Deficiency The Company plans to conduct the ENERGY-2 pivotal trial, an open label, single arm trial in infants with ENPP1 Deficiency, based on the Paediatric Investigational Plan ("PIP") agreed upon by PDCO. Discussions are ongoing with the FDA regarding the design of a potential pivotal trial of INZ-701 in infants with ENPP1Deficiency in the U.S. Pending regulatory discussions and appropriate financial resources, the Company also plans to conduct the ENERgy-4 pivotal trial, a multicenter, randomized, controlled trial in adolescents and adults with ENPP1 Deficiency.

Basis for Planned Marketing Applications Based on regulatory feedback from the FDA and EMA, positive data from the ongoing and planned clinical trials of INZ-701 in patients with ENPP1 Defiencies, including comprehensive data demonstrating clinical impact of plasma PPi, could provide the basis for the Company's submission of marketing applications in both the U.S. and EU. INZ-701 is currently in clinical trials for the treatment of ENPP 1 Deficiency and ABCC6 Deficiency. These statements include, but are not limited to, statements relating to the timing and design of clinical trials, the potential benefits of INZ-701, the timing and contents of planned global development strategy, the availability and timing of clinical trial data, planned regulatory filings and the basis for such filings, the timing of the planned commercial launch of INZ-701, if approved, and the period over which believe that existing cash, cash equivalents and short term investments will be sufficient to fund cash flow requirements.

These risks and uncertainties include, but are not limited, risks associated with the Company's ability to conduct its ongoing pivotal trial.