GlobeNewswire Inc | Jan 4, 2021 06:00AM EST

January 04, 2021

U.S. Food and Drug Administration cleared Investigational New Drug Application

United Kingdom Medicines and Healthcare Products Regulatory Agency authorized Clinical Trial Application

Program addressing rare mineralization disorders expected to enroll first subject in H121 and provide preliminary safety and biomarker data in H221

BOSTON, Jan. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Companys 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.

About the INZ701-101 Phase 1/2 Clinical Trial of INZ-701 in Adults with ENPP1 Deficiency

The Phase 1/2 clinical trial is a multi-center, open-label, first-in-human, 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.

Additional details can be found:

https://clinicaltrials.gov/ct2/show/NCT04686175

About INZ-701

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.

AboutInozyme Pharma

Inozyme Pharma, Inc. (Nasdaq: INZY), is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat ENPP1 and ABCC6 deficiencies. ENPP1 and ABCC6 deficiencies are chronic, systemic, and progressive diseases occurring over the course of a patients lifetime, starting as early as fetal development and spanning into adulthood. ENPP1 and ABCC6 deficiencies are estimated to occur in approximately one in 200,000 and one in 50,000 births, respectively.

Inozyme Pharmawas founded in 2017 byJoseph Schlessinger, Ph.D.,Demetrios Braddock, M.D., Ph.D., andAxel Bolte, MSc, MBA, with technology developed byDr. Braddockand licensed fromYale University. For more information, please visitwww.inozyme.com.

Cautionary Note Regarding Forward-Looking Statements

Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials, our research and development programs, the availability of preclinical study and clinical trial data, and the timing of our regulatory applications. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, should, target, will, would and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on managements current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Companys ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the Risk Factors section, as well as discussions of potential risks, uncertainties and other important factors, in the Companys most recent filings with theSecurities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof and should not be relied upon as representing the Companys views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

InvestorsBrian Luque, Director, Investor Relations(951) 206-1200ir@inozyme.com

Media:Alex Van Rees, SmithSolve(973) 442-1555 ext. 111alex.vanrees@smithsolve.com