GlobeNewswire Inc | Mar 25, 2021 06:00AM EDT

March 25, 2021

Received Orphan Drug Designation by the U.S. Food and Drug Administration for INZ-701 for treatment of ABCC6 deficiency

Expect to initiate Phase 1/2 trials of INZ-701 for ENPP1 deficiency in the first half of the year and ABCC6 deficiency by mid-2021

Cash, cash equivalents, and investments expected to enable continued operations into second half of 2022

BOSTON, March 25, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization, today reported financial results for the full year ended December 31, 2020 and provided recent business highlights.

2020 was a foundational year for Inozyme as we laid the groundwork to become a clinical stage company focused on the biology of mineralization. We have significantly expanded our team and operational footprint, ensuring we have the resources required to bring INZ-701 - our lead product candidate - into two planned first-in-human clinical studies in patients with ENPP1 deficiency and ABCC6 deficiency, said Axel Bolte, MSc, MBA, co-founder, president, and chief executive officer of Inozyme Pharma. ENPP1 deficiency is a systemic, progressive, and chronic disease that occurs over a patients lifetime, starting as early as fetal development, and spanning into adulthood, with devastating and often fatal effect. ABCC6 deficiency is a calcification disorder with an onset in adolescence and progressive worsening and high morbidity.

Mr. Bolte continued, Based on discussions with U.S. and European regulatory authorities, we plan to initiate our Phase 1/2 clinical trial in ENPP1 deficiency in the first half of 2021 and our Phase 1/2 clinical trial in ABCC6 deficiency by mid-2021.

Recent Business Highlights

-- Granted Orphan Drug Designation for INZ-701 for ABCC6 Deficiency Inozyme recently received orphan drug designation by the U.S. Food and Drug Administration for INZ-701 for the treatment of patients with ABCC6 deficiency. -- Received Clearance to Proceed in U.S. and U.K. with Phase 1/2 Clinical Trial of INZ-701 for the Treatment of ENPP1 Deficiency the Company expects to initiate the Phase 1/2 trial in the first half of 2021 and to provide preliminary safety and biomarker data in the second half of 2021. -- Completed the Burden of Disease Patient Study this study provides an overview of the burden of disease in patients with ENPP1 deficiency and ABCC6 deficiency. The study has been accepted as poster presentations at three scientific conferences in the second quarter of 2021. -- Completed the Healthy Volunteer Pyrophosphate (PPi) Study this study validates the highly sensitive assay that will be used to evaluate INZ-701 in clinical trials. The results of this study will be presented at medical conferences later in the year. -- Advanced Gene TherapyResearch and Development Inozyme progressed research and preclinical proof-of-concept work in demonstrating the ability to raise PPi levels using a gene therapy approach in animal models.

Upcoming Anticipated Milestones

The Company also announced the following anticipated milestones for the INZ-701 clinical development program, subject to COVID-19-related restrictions:

-- ENPP1 DeficiencyH1 2021: Initiate prospective natural history studyH1 2021: Initiate Phase 1/2 clinical trialH2 2021: Report preliminary safety and biomarker data from Phase 1/2 clinical trial -- ABCC6 DeficiencyH1 2021: File Clinical Trial ApplicationsMid-2021: Initiate Phase 1/2 clinical trialBy the End of 2021: Report preliminary safety and biomarker data from Phase 1/2 clinical trial

Financial Results for the Year Ended December 31, 2020

-- Cash Position and Financial Guidance Cash, cash equivalents, and investments were$159.9 millionas ofDecember 31, 2020. Based on its current plans, the Company expects that its existing cash, cash equivalents, and investments will be sufficient to enable funding of its operating expenses and capital expenditure requirements into the second half of 2022. -- Research and Development (R&D) Expenses R&D expenses were$46.5 millionfor the year endedDecember 31, 2020, compared to$16.2 millionfor the year ended December 31, 2019. The increase was primarily due to an increase of$17.8 millionresulting from the non-recurring, non-cash purchase of in-process research and development intellectual property assets from Alexion Pharmaceuticals in exchange for stock of the Company inJuly 2020; costs associated with preclinical studies and clinical preparation activities with the Companys contract research organization; and growth in the number of R&D employees. -- General and Administrative (G&A) Expenses G&A expenses were$10.5 millionfor the year endedDecember 31, 2020, compared to$4.6 millionfor the year ended December 31, 2019. The increase was primarily due to the growth in the number of G&A employees; an increase in legal fees related to patents, new contracts, and operations as a public company; and generally higher fees in areas such as audit, tax, and information technology to support the Companys growth. -- Net Loss Net loss was$56.4 million, or$5.11loss per share, for the year endedDecember 31, 2020, compared to$19.7 million, or$16.67loss per share, for the year ended December 31, 2019.

AboutInozyme Pharma

Inozyme Pharma, Inc. (Nasdaq: INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. 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 the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

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, the timing of our regulatory applications and the period over which we believe that our existing cash, cash equivalents and investments will be sufficient to fund our operating expenses. 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 initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 deficiency and ABCC6 deficiency; 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.

Condensed Consolidated Balance Sheet Data(Unaudited)

(in thousands)

December 31, 2020 December 31, 2019Cash, cash equivalents, and investments $ 159,896 $ 47,132 Total assets 169,363 47,944 Total liabilities 11,260 3,236 Convertible preferred stock ? 77,927 Additional paid-in-capital 249,175 1,428 Accumulated deficit (91,076 ) (34,652 )Total stockholders' equity (deficit) 158,103 (33,219 )

Condensed Consolidated Statements of Operations and Comprehensive Loss(Unaudited)

(in thousands, except share and per share data)

YearEndedDecember 31, 2020 2019Operating expenses: Research and development $ 46,493 $ 16,220 General and administrative 10,548 4,586 Total operating expenses 57,041 20,806 Loss from operations (57,041 ) (20,806 )Other income (expense): Interest income 370 1,106 Other income (expense), net 247 (24 )Other income (expense), net 617 1,082 Net loss $ (56,424 ) $ (19,724 )Other comprehensive (loss) income: Unrealized (losses) gains on available-for-sale (3 ) 7 securitiesTotal other comprehensive (loss) income (3 ) 7 Comprehensive loss $ (56,427 ) $ (19,717 )Net loss attributable to common $ (56,424 ) $ (19,724 )stockholders?basic and dilutedNet loss per share attributable to common $ (5.11 ) $ (16.67 )stockholders?basic and dilutedWeighted-average common shares outstanding?basic 11,036,500 1,183,147 and diluted

InvestorsInozyme PharmaAxel Bolte, co-founder, president, and chief executive officerir@inozyme.com

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