GlobeNewswire Inc | Nov 12, 2020 06:00AM EST

November 12, 2020

Submitted CTA for INZ-701 for the treatment of ENPP1 deficiency to United Kingdom regulatory agency

Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency

Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021

BOSTON, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today reported financial results for the third quarter ended September 30, 2020 and provided recent business highlights.

ENPP1 deficiency is a systemic, progressive and continuous disease occurring over the course of a patients lifetime, starting as early as fetal development and spanning into adulthood. The fact that INZ-701 had previously received orphan drug designation and now rare pediatric disease and fast track designations underscores the significant unmet medical need for a treatment for this disease, said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. Im pleased with the progress we have made with U.S. and European regulatory authorities, and we remain on track to initiate our planned Phase 1/2 clinical trials in the first half of 2021, subject to clearance of our regulatory applications.

Recent Business Highlights

-- Submitted Clinical Trial Application (CTA) for INZ-701 for the treatment of ENPP1 deficiency Inozyme recently submitted its first CTA to initiate a Phase 1/2 clinical trial of INZ-701 for the treatment of ENPP1 deficiency to the United Kingdoms Medicines and Healthcare products Regulatory Agency (MHRA). -- Received Rare Pediatric Disease Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) for INZ-701 for the treatment of ENPP1 deficiency The FDA grants rare pediatric disease designation to drugs for serious and life-threatening diseases in which the serious or life-threatening manifestations primarily affect children aged from birth through 18 years and affect fewer than 200,000 people in the U.S. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval of a biologics license application (BLA) for a rare pediatric disease product application may be eligible for a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product. Separately, Fast Track Designation facilitates the potential expedited development and review of a drug for the treatment of a serious or life-threatening disease and that has demonstrated the potential to address unmet medical needs. Benefits of this designation include frequent engagements with the FDA to discuss the drugs clinical development plan, eligibility for priority review, and a rolling review of a BLA. Previously, the FDA and the European Medicines Agency (EMA) had granted orphan drug designation to INZ-701 for the treatment of ENPP1 deficiency. -- Completed disease burden study in ENPP1 deficiency and ABCC6 deficiency Inozyme and GACI Global, a patient advocacy organization dedicated to bettering the lives of families affected by Generalized Arterial Calcification of Infancy and/or Autosomal Recessive Hypophosphatemic Rickets Type 2 (GACI/ARHR2), completed a study to characterize the burden of disease and understand the systemic progression of disease for the rare genetic diseases of both ENPP1 deficiency and ABCC6 deficiency from the perspective of a patient and/or parent. Inozyme expects to share data from this study in 2021.

Upcoming Anticipated Milestones, Subject to COVID-19 Dynamics

-- INZ-701 for ENPP1 deficiencyEarly 2021: Clearance of IND and CTAsH1 2021: Initiation of Phase 1/2 clinical trialH1 2021: Initiation of prospective natural history studyH2 2021: Preliminary safety and biomarker data from Phase 1/2 clinical trial -- INZ-701 for ABCC6 deficiencyEarly 2021: Clearance of CTAsH1 2021: Initiation of Phase 1/2 clinical trialH2 2021: Preliminary safety and biomarker data from Phase 1/2 clinical trial

Upcoming Investor Conference

-- Piper Sandler 32nd Annual Healthcare Conference, November 30 December 3, 2020

Third Quarter 2020 Financial Results

-- Cash Position and Financial Guidance Cash, cash equivalents and investments were $171.7 million as of September 30, 2020. Based on its current plans, the Company expects that its existing cash, cash equivalents and investments will be sufficient to enable it to fund its operating expenses and capital expenditure requirements at least into the second half of 2022. -- Research and Development (R&D) Expenses R&D expenses were $25.2 million for the third quarter ended September 30, 2020, compared to $3.3 million for the same period in 2019. The increase was primarily due to an increase of $17.8 million resulting from the non-recurring, non-cash purchase of in-process research and development intellectual property assets from Alexion in exchange for stock of the Company in July 2020, costs associated with preclinical studies and clinical preparation activities with the Companys CRO, and growth in the number of R&D employees. -- General and Administrative (G&A) Expenses G&A expenses were $3.1 million for the third quarter ended September 30, 2020, compared to $1.0 million for the same period in 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 $28.1 million, or $1.55 loss per share, for the third quarter ended September 30, 2020, compared to $4.0 million, or $3.38 loss per share, for the same period in 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 successfully resolve the clinical hold with regard to its planned Phase 1/2 clinical trial of INZ-701 for ENPP1 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)

September December 31, 30, 2019 2020Cash, cash equivalents and investments $ 171,709 $ 47,132 Total assets 178,993 47,944 Total liabilities 11,077 3,236 Convertible preferred stock ? 77,927 Additional paid-in-capital 247,872 1,428 Accumulated deficit (79,958 ) (34,652 )Total stockholders? equity (deficit) 167,916 (33,219 )

Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited)

(in thousands, except share and per share data)

ThreeMonthsEndedSeptember 30, NineMonthsEndedSeptember 30, 2020 2019 2020 2019Operating expenses: Research and development $ $ $ $ 25,174 3,317 39,457 10,941General and 3,142 1,003 6,313 3,097 administrativeTotal operating expenses 28,316 4,320 45,770 14,038 Loss from operations (28,316 ) (4,320 ) (45,770 ) (14,038 )Other income (expense): Interest income 64 288 306 892 Other income (expense), 157 (3 ) 158 (34 )netOther income (expense), 221 285 464 858 netNet loss $ ) $ ) $ ) $ ) (28,095 (4,035 (45,306 (13,180Other comprehensive (loss) income:Unrealized (losses) gainson available-for-sale (13 ) (2 ) (5 ) 8 securitiesTotal other comprehensive (13 ) (2 ) (5 ) 8 (loss) incomeComprehensive loss $ ) $ ) $ ) $ ) (28,108 (4,037 (45,311 (13,172Net loss attributable to common stockholders?basic $ (28,095 ) $ (4,035 ) $ (45,306 ) $ (13,180 )and dilutedNet loss per shareattributable to $ ) $ ) $ ) $ )commonstockholders?basic (1.55 (3.38 (6.57 (11.20and dilutedWeighted-average commonshares outstanding?basic 18,101,496 1,195,309 6,893,745 1,176,769 and diluted

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

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