Core Viewpoint - Inozyme Pharma, Inc. is advancing its clinical-stage biopharmaceutical development with the presentation of topline data from ongoing Phase 1/2 clinical trials of INZ-701 for treating ENPP1 Deficiency and ABCC6 Deficiency at upcoming medical conferences [1] Group 1: Clinical Trials and Presentations - The European Calcified Tissue Society Congress (ECTS) 2024 will feature presentations on INZ-701's impact on bone and mineral metabolism biomarkers and clinical outcomes in adults with ENPP1 Deficiency [2][3] - The Endocrine Society's Annual Meeting (ENDO) 2024 will also include presentations on the safety and exploratory efficacy of INZ-701 in adults with ABCC6 Deficiency [3][4] Group 2: ENPP1 Deficiency Overview - ENPP1 Deficiency is a rare condition with a prevalence of approximately 1 in 64,000 pregnancies, leading to severe complications such as generalized arterial calcification of infancy and osteomalacia [4][5] - There are currently no approved therapies for ENPP1 Deficiency, highlighting the unmet medical need [4][5] Group 3: ABCC6 Deficiency Overview - ABCC6 Deficiency affects approximately 1 in 25,000 to 1 in 50,000 individuals and can lead to conditions like pseudoxanthoma elasticum, with no approved therapies available [7][8] - Pediatric patients may develop severe neurological and cardiovascular complications due to ongoing vascular calcification [7][8] Group 4: INZ-701 Development - INZ-701 is an enzyme replacement therapy designed to treat rare vascular and soft tissue disorders by metabolizing ATP to generate pyrophosphate (PPi), a natural inhibitor of mineralization [9][10] - The ongoing Phase 1/2 trials for both ENPP1 and ABCC6 Deficiencies primarily assess the safety, tolerability, and pharmacokinetic and pharmacodynamic profiles of INZ-701 [5][8]

BOSTON, May 08, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY) ("the Company" or "Inozyme"), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced Doug Treco, Ph.D., CEO and Chairman of Inozyme, will participate in a fireside chat at the Bank of America Securities 2024 Health Care Conference on Wednesday, May 15, 2024 from 6:40-7:10pm ET / 3:40-4:10pm PT. A live webcast of t ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number: 001-39397 INOZYME PHARMA, INC. (Exact name of registrant as specified in its charter) Delaware 38-4024528 (State or other jurisdiction of incorporation or organization) 321 Summer Street, Suite 400 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE S ...

Financial Performance - Cash, cash equivalents, and short-term investments were $166.2 million as of March 31, 2024, expected to fund operations into Q4 2025[6] - Research and Development (R&D) expenses increased to $19.1 million for Q1 2024, up from $11.9 million in the prior-year period, representing a 60% increase[6] - General and Administrative (G&A) expenses decreased to $5.2 million for Q1 2024, down from $6.5 million in the prior-year period, a reduction of approximately 20%[6] - Net loss for Q1 2024 was $23.3 million, or $0.38 loss per share, compared to a net loss of $17.4 million, or $0.40 loss per share in the prior-year period[6] - Total assets as of March 31, 2024, were $176.9 million, down from $200.8 million as of December 31, 2023[17] Clinical Trials and Research - Interim data from SEAPORT-1 Phase 1 trial of INZ-701 in patients with end-stage kidney disease is expected in Q4 2024[1] - Positive topline safety and immunogenicity data reported for INZ-701 in adults with ABCC6 Deficiency, indicating clinical improvements[3] - Anticipated initiation of pivotal trial in pediatric patients with ABCC6 Deficiency in Q1 2025, subject to regulatory review and funding[3] - Positive topline data from Phase 1/2 trial of INZ-701 in adults with ENPP1 Deficiency maintained through 48 weeks[4] - Estimated incidence of calciphylaxis is approximately 3.5 per 1,000 patients with end-stage kidney disease, with around 5,000 new patients presenting annually[9]

- Interim data from SEAPORT-1, a Phase 1 trial of INZ-701 in patients with end-stage kidney disease receiving hemodialysis, on track for fourth quarter of 2024 - - Interim data from ENERGY-1, a Phase 1b trial of INZ-701 in infants with ENPP1 Deficiency, on track for second half of 2024 - - Cash, cash equivalents, and short-term investments as of March 31, 2024, expected to fund operations into the fourth quarter of 2025 - BOSTON, May 07, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY) ("the Co ...

- Favorable safety and immunogenicity profile in ABCC6 Deficiency, with clinical improvements in vascular pathology, visual function and patient reported outcomes (PROs) - - Natural history study highlights stroke as common feature among patients with early-onset ABCC6 Deficiency - - Favorable safety, immunogenicity and clinical outcome data were maintained through 48 weeks in Cohorts 1-3 in ENPP1 Deficiency; Data from Cohort 4 support once weekly dosing in ongoing and future clinical trials - - Company to ...

BOSTON, April 05, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY) ("Company" or "Inozyme"), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced that it granted stock options to three new employees to purchase shares of the Company's common stock, pursuant to the Company's 2023 Inducement Stock Incentive Plan, as inducement material to the new employees entering into employm ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____________________ to _____________________ Commission File Number: 001-39397 INOZYME PHARMA, INC. (Exact name of registrant as specified in its charter) Delaware ...

Exhibit 99.1 Inozyme Pharma Reports Full Year 2023 Financial Results and Provides Business Highlights – Topline data from ongoing Phase 1/2 trials of INZ-701 in adults with ABCC6 Deficiency and ENPP1 Deficiency expected in early April – – Phase 1 trial of INZ-701 in end-stage kidney disease (ESKD) patients receiving hemodialysis (SEAPORT-1) now underway (NCT06283589), with topline data expected in Q4 2024 – – Launched global patient registry (PROPEL) in partnership with GACI Global to advance understanding ...

```markdown [PART I. FINANCIAL INFORMATION](index=6&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This section presents the unaudited condensed consolidated financial statements and management's discussion and analysis [Item 1. Financial Statements (Unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents Inozyme Pharma's unaudited condensed consolidated financial statements and related notes [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) This section details the company's financial position, including assets, liabilities, and stockholders' equity Condensed Consolidated Balance Sheets (amounts in thousands) | Metric | September 30, 2023 | December 31, 2022 | | :-------------------------------- | :------------------- | :------------------ | | **Assets** | | | | Cash and cash equivalents | $46,895 | $32,915 | | Short-term investments | $145,547 | $94,951 | | Total current assets | $200,856 | $131,393 | | Total assets | $205,702 | $139,195 | | **Liabilities** | | | | Total current liabilities | $12,275 | $14,715 | | Long-term debt, net | $32,065 | $4,139 | | Total liabilities | $45,489 | $20,801 | | **Stockholders' Equity** | | | | Total stockholders' equity | $160,213 | $118,394 | | Accumulated deficit | $(264,386) | $(214,761) | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) This section outlines the company's revenues, expenses, and net loss over specific periods Condensed Consolidated Statements of Operations and Comprehensive Loss (amounts in thousands, except per share data) | Metric | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :------------------------------------------------ | :------------------------------ | :------------------------------ | :----------------------------- | :----------------------------- | | Research and development expenses | $13,341 | $12,191 | $36,864 | $34,012 | | General and administrative expenses | $4,733 | $4,721 | $15,973 | $15,130 | | Total operating expenses | $18,074 | $16,912 | $52,837 | $49,142 | | Loss from operations | $(18,074) | $(16,912) | $(52,837) | $(49,142) | | Interest income, net | $1,416 | $737 | $3,254 | $1,118 | | Net loss | $(16,638) | $(16,372) | $(49,625) | $(48,517) | | Net loss per share (basic and diluted) | $(0.29) | $(0.38) | $(1.02) | $(1.36) | | Weighted-average common shares outstanding | 56,758,395 | 43,657,718 | 48,494,175 | 35,755,695 | [Condensed Consolidated Statements of Stockholders' Equity](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders'%20Equity) This section tracks changes in the company's equity, including common stock and accumulated deficit Changes in Stockholders' Equity (amounts in thousands, except share data) | Metric | Balance at Dec 31, 2022 | Balance at Sep 30, 2023 | | :------------------------------------ | :---------------------- | :---------------------- | | Common Stock Shares | 40,394,363 | 61,760,748 | | Common Stock Amount | $4 | $6 | | Additional Paid-in Capital | $333,356 | $424,671 | | Accumulated Deficit | $(214,761) | $(264,386) | | Total Stockholders' Equity | $118,394 | $160,213 | Key Equity Changes (Nine Months Ended Sep 30, 2023) - **Stock-based compensation**: **$5,364**[22](index=22&type=chunk) - **Shares issued** in at-the-market offering: **3,553,995 shares** for **$21.2 million net proceeds**[57](index=57&type=chunk) - **Issuance of common stock**, **net of issuance costs** (July 2023 offering): **14,375,000 shares** for **$64.4 million net proceeds**[53](index=53&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) This section summarizes cash inflows and outflows from operating, investing, and financing activities Condensed Consolidated Statements of Cash Flows (amounts in thousands) | Metric | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :------------------------------------------ | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(52,188) | $(43,361) | | Net cash used in investing activities | $(47,176) | $(8,621) | | Net cash provided by financing activities | $113,453 | $72,788 | | Net increase in cash, cash equivalents, and restricted cash | $14,089 | $20,806 | | Cash, cash equivalents, and restricted cash at end of period | $47,206 | $44,398 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) This section provides detailed explanations and disclosures supporting the unaudited financial statements [1. Organization and Basis of Presentation](index=11&type=section&id=1.%20Organization%20and%20Basis%20of%20Presentation) This note describes the company's business, financial position, and management's assessment of its going concern - Inozyme Pharma, Inc. is a clinical-stage rare disease biopharmaceutical company focused on developing novel therapeutics for diseases impacting vasculature, soft tissue, and skeleton, specifically targeting ENPP1 and ABCC6 Deficiencies with its lead product candidate, INZ-701[23](index=23&type=chunk)[24](index=24&type=chunk) - The company incurred **net losses** of **$49.6 million** for the nine months ended September 30, 2023, with an **accumulated deficit** of **$264.4 million**. **Cash, cash equivalents, and short-term investments** totaled **$192.4 million** as of September 30, 2023[26](index=26&type=chunk) - Management believes current **cash, cash equivalents, and short-term investments** are sufficient to **fund operations** for at least 12 months from the 10-Q filing date, but acknowledges the need for additional funding due to recurring losses and negative cash flows[27](index=27&type=chunk)[28](index=28&type=chunk) [2. Summary of Significant Accounting Policies](index=12&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) This note outlines the key accounting principles and methods used in preparing the financial statements - The condensed consolidated financial statements include Inozyme Pharma, Inc. and its wholly-owned subsidiaries, with all intercompany transactions eliminated[29](index=29&type=chunk) - No material changes occurred in the Company's significant accounting policies during the nine months ended September 30, 2023, compared to the audited consolidated financial statements for the year ended December 31, 2022[30](index=30&type=chunk) - The Company mitigates credit risk by diversifying its portfolio of U.S. Treasury and government agency debt securities and limiting exposure by institution, maturity, and investment type[32](index=32&type=chunk) [3. Recent Accounting Pronouncements](index=12&type=section&id=3.%20Recent%20Accounting%20Pronouncements) This note discusses the impact of recently adopted accounting standards on the company's financial reporting - The Company adopted ASU No. 2016-13, Financial Instruments-Credit Losses (Topic 326), effective January 1, 2023, with no impact on its financial statements[37](index=37&type=chunk) [4. Short-Term Investments](index=13&type=section&id=4.%20Short-Term%20Investments) This note details the composition and fair value of the company's short-term investment portfolio Short-Term Investments (amounts in thousands) | Description | September 30, 2023 Fair Value | December 31, 2022 Fair Value | | :------------------------------ | :---------------------------- | :--------------------------- | | U.S. Treasury securities | $57,788 | $16,615 | | U.S. government agency debt securities | $87,759 | N/A (replaced commercial paper) | | Commercial paper | N/A (replaced by U.S. government agency debt) | $78,336 | | Total | $145,547 | $94,951 | - The Company's **short-term investments increased** from **$94.951 million** at December 31, 2022, to **$145.547 million** at September 30, 2023, with a shift from commercial paper to U.S. government agency debt securities[38](index=38&type=chunk) [5. Fair Value Measurement](index=13&type=section&id=5.%20Fair%20Value%20Measurement) This note explains the methodologies and classifications used for measuring the fair value of financial assets Fair Value Measurements at Reporting Date (amounts in thousands) | Description | September 30, 2023 Total | September 30, 2023 Level 1 | September 30, 2023 Level 2 | | :---------------------------------------------------------------- | :----------------------- | :----------------------- | :----------------------- | | Money market funds | $41,289 | $41,289 | $— | | U.S. government agency debt securities | $87,759 | $— | $87,759 | | U.S. Treasury securities | $62,757 | $62,757 | $— | | **Total assets** | **$191,805** | **$104,046** | **$87,759** | | Description | December 31, 2022 Total | December 31, 2022 Level 1 | December 31, 2022 Level 2 | | :---------------------------------------------------------------- | :----------------------- | :----------------------- | :----------------------- | | Money market funds | $26,587 | $26,587 | $— | | Commercial paper | $78,336 | $— | $78,336 | | U.S. Treasury securities | $16,615 | $16,615 | $— | | **Total assets** | **$121,538** | **$43,202** | **$78,336** | - The Company's financial assets measured at **fair value increased** from **$121.5 million** at December 31, 2022, to **$191.8 million** at September 30, 2023, with a significant portion classified as Level 1 (quoted prices in active markets) and Level 2 (observable inputs)[40](index=40&type=chunk) [6. License Agreement](index=14&type=section&id=6.%20License%20Agreement) This note describes the company's intellectual property license agreement with Yale University and associated payments - The Company has a license agreement with Yale University for intellectual property related to ectonucleotide pyrophosphatase/phosphodiesterase enzymes, which forms the basis for the INZ-701 development program[41](index=41&type=chunk) - Payments to Yale include annual license maintenance fees (tens of thousands), up to **$3.0 million** for specified net product sales or development/commercialization milestones per therapeutic/prophylactic product, and low single-digit percentage royalties on worldwide net sales[41](index=41&type=chunk) - Milestone payments of approximately **$0.3 million** each were made in January and March 2022 following the first patient dosing and completion of the first cohort in the Phase 1/2 clinical trial of INZ-701 for ENPP1 Deficiency[41](index=41&type=chunk) [7. Commitments and Contingencies](index=15&type=section&id=7.%20Commitments%20and%20Contingencies) This note outlines the company's future lease obligations and potential legal contingencies Future Lease Payments (amounts in thousands) | Year Ending December 31, | Amount | | :----------------------- | :----- | | 2023 (remaining 3 months) | $249 | | 2024 | $1,016 | | 2025 | $944 | | **Total** | **$2,209** | - The Company has operating leases for office and laboratory space in Boston, Massachusetts, expiring in 2025, with **total future lease payments** of **$2.209 million** as of September 30, 2023[42](index=42&type=chunk)[43](index=43&type=chunk)[46](index=46&type=chunk) - The Company is not currently a party to any material legal proceedings and has not incurred material costs from indemnification agreements to date[44](index=44&type=chunk)[45](index=45&type=chunk) [8. Convertible Debt](index=15&type=section&id=8.%20Convertible%20Debt) This note details the terms and outstanding balance of the company's convertible debt agreement - The Company entered into a Loan Agreement with K2 HealthVentures LLC in July 2022, providing up to **$70.0 million** in term loans. As of September 30, 2023, **$32.5 million** in principal term loans were outstanding, with **$37.5 million** remaining available[46](index=46&type=chunk)[124](index=124&type=chunk) - The term loan matures on August 1, 2026, with interest-only payments for the first 36 months, followed by interest and principal payments. The variable **interest rate** was **9.60%** as of September 30, 2023[47](index=47&type=chunk) Long-Term Debt Summary (amounts in thousands) | Metric | September 30, 2023 | | :-------------------------- | :------------------- | | Gross proceeds | $32,500 | | Unamortized debt issuance costs | $(435) | | Carrying value | $32,065 | Future Principal Payments (amounts in thousands) | Fiscal Year | Amount | | :---------- | :----- | | 2023 | $— | | 2024 | $— | | 2025 | $10,479 | | 2026 | $24,052 | | **Total** | **$34,531** | [9. Stockholders' Equity](index=17&type=section&id=9.%20Stockholders'%20Equity) This note provides information on changes in common stock, additional paid-in capital, and stock-based compensation - In July 2023, the Company completed an underwritten public offering of **14,375,000 shares** of common stock at **$4.80 per share**, generating approximately **$64.4 million** in **net proceeds**[53](index=53&type=chunk) - As of September 30, 2023, the Company had sold **3,553,995 shares** of common stock through an 'at-the-market' offering program, yielding aggregate **net proceeds** of **$21.2 million**[57](index=57&type=chunk) Stock-Based Compensation Expense (amounts in thousands) | Period | Research and Development | General and Administrative | Total | | :-------------------------------- | :----------------------- | :------------------------- | :---- | | Three Months Ended Sep 30, 2023 | $839 | $833 | $1,672 | | Three Months Ended Sep 30, 2022 | $931 | $918 | $1,849 | | Nine Months Ended Sep 30, 2023 | $2,546 | $2,817 | $5,363 | | Nine Months Ended Sep 30, 2022 | $2,923 | $2,863 | $5,786 | [10. Net Loss per Share](index=18&type=section&id=10.%20Net%20Loss%20per%20Share) This note explains the calculation of basic and diluted net loss per share for the reporting periods Net Loss per Share Attributable to Common Stockholders (basic and diluted) | Period | Net Loss (thousands) | Net Loss per Share | Weighted-Average Shares Outstanding | | :-------------------------------- | :------------------- | :----------------- | :-------------------------------- | | Three Months Ended Sep 30, 2023 | $(16,638) | $(0.29) | 56,758,395 | | Three Months Ended Sep 30, 2022 | $(16,372) | $(0.38) | 43,657,718 | | Nine Months Ended Sep 30, 2023 | $(49,625) | $(1.02) | 48,494,175 | | Nine Months Ended Sep 30, 2022 | $(48,517) | $(1.36) | 35,755,695 | - The basic and diluted **net loss per share** are the same due to **net losses** in all periods, making potentially dilutive securities anti-dilutive[65](index=65&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=20&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's financial condition, operational results, clinical development of INZ-701, and liquidity outlook [Overview](index=20&type=section&id=Overview) This section introduces Inozyme Pharma's focus on rare diseases and its lead product candidate, INZ-701 - Inozyme Pharma is a clinical-stage rare disease biopharmaceutical company focused on developing novel therapeutics for diseases impacting vasculature, soft tissue, and skeleton, specifically targeting ENPP1 and ABCC6 Deficiencies with its lead product candidate, INZ-701[68](index=68&type=chunk) - The lead product candidate, INZ-701, is a soluble, recombinant fusion protein designed to correct defects in the ENPP1 and ABCC6 pathway, which regulates calcium deposition and inhibits blood vessel obstruction[69](index=69&type=chunk) - INZ-701 has received Orphan Drug Designation from the FDA and EMA for ENPP1 and ABCC6 Deficiencies, and Fast Track and Rare Pediatric Disease designations from the FDA for ENPP1 Deficiency[69](index=69&type=chunk) [Executive Summary](index=20&type=section&id=Executive%20Summary) This section highlights recent regulatory updates and positive interim clinical trial data for INZ-701 - In July 2023, the Company announced a regulatory update and alignment with the FDA and EMA on its global development strategy for INZ-701 for ENPP1 Deficiency[71](index=71&type=chunk) - In September 2023, positive interim safety, pharmacokinetic, pharmacodynamic, and exploratory efficacy data were reported from Phase 1/2 trials of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency, showing clinical benefit and sustained **PPi increase**[71](index=71&type=chunk) - The first site for the ENERGY-3 pivotal trial in pediatric patients with ENPP1 Deficiency was opened in September 2023, with patient recruitment underway[71](index=71&type=chunk) [Clinical Overview](index=21&type=section&id=Clinical%20Overview) This section details the progress and results of clinical trials for INZ-701 in various indications [ENPP1 Deficiency](index=21&type=section&id=ENPP1%20Deficiency) This section reports on clinical trial progress and efficacy data for INZ-701 in ENPP1 Deficiency - The Phase 1/2 clinical trial of INZ-701 in adults with ENPP1 Deficiency showed rapid, significant, and sustained **increases in plasma PPi** for up to 18 months, with potential for once-weekly dosing due to a long **half-life** (approx. **126 hours**)[77](index=77&type=chunk) - Exploratory efficacy data from the adult ENPP1 Deficiency trial indicated notable improvements in patient-reported and clinician-reported Global Impression of Change (**GIC scores**), with no patient deterioration[77](index=77&type=chunk)[78](index=78&type=chunk) - The ENERGY-1 trial for infants with ENPP1 Deficiency dosed its first patient in June 2023, with interim data expected in the second half of 2024. The ENERGY-3 pivotal trial for pediatric patients began recruitment in September 2023, with topline data anticipated in mid-2025[80](index=80&type=chunk)[82](index=82&type=chunk) [ABCC6 Deficiency](index=22&type=section&id=ABCC6%20Deficiency) This section presents clinical trial data and future development plans for INZ-701 in ABCC6 Deficiency - The Phase 1/2 clinical trial of INZ-701 in adults with ABCC6 Deficiency demonstrated a dose-dependent and sustained **increase in plasma PPi levels**, with drug accumulation suggesting potential for once-weekly dosing[90](index=90&type=chunk) - All patients in the ABCC6 Deficiency trial showed improvements in clinician-reported **GIC scores**, and seven of nine patients improved on patient-reported **GIC scores**[90](index=90&type=chunk) - Topline data from the adult ABCC6 Deficiency trial is expected in Q1 2024, with plans to initiate a Phase 3 clinical trial in Q4 2024, subject to regulatory review and funding[91](index=91&type=chunk) [Expanded Access Program](index=23&type=section&id=Expanded%20Access%20Program) This section describes the use of INZ-701 for pediatric patients under an expanded access program - In February 2023, the Company dosed its first pediatric patient with ENPP1 Deficiency under an expanded access program, allowing INZ-701 use outside clinical trials for serious conditions with no alternative treatments[92](index=92&type=chunk) [Future Development Plans; Other Potential Indications for INZ-701](index=23&type=section&id=Future%20Development%20Plans%3B%20Other%20Potential%20Indications%20for%20INZ-701) This section outlines plans to explore INZ-701 for additional diseases of pathologic mineralization - Beyond ENPP1 and ABCC6 Deficiencies, the Company plans to explore INZ-701 for other diseases of pathologic mineralization and intimal proliferation, including calciphylaxis and calcifications in end-stage kidney disease (ESKD)[93](index=93&type=chunk) - A Phase 1 clinical trial (SEAPORT-1) for INZ-701 in ESKD patients receiving hemodialysis is planned for initiation in H1 2024, with interim data expected in Q4 2024[72](index=72&type=chunk)[94](index=94&type=chunk) [Our Operations](index=24&type=section&id=Our%20Operations) This section discusses the company's operational focus, expected losses, and future funding requirements - The Company has not yet commercialized any products or generated revenue from product sales, with operations focused on capital raising, R&D, preclinical studies, early-stage clinical trials, and manufacturing arrangements[95](index=95&type=chunk) - Significant **operating losses** are expected to continue, and substantial additional funding will be required to support ongoing operations, including clinical trials, manufacturing scale-up, and potential commercialization[96](index=96&type=chunk)[97](index=97&type=chunk)[98](index=98&type=chunk) - The Company expects its existing **cash, cash equivalents, and short-term investments** to **fund cash flow requirements into the fourth quarter of 2025**[100](index=100&type=chunk) [Financial Operations Overview](index=25&type=section&id=Financial%20Operations%20Overview) This section provides an overview of the company's key financial expense categories and interest income [Research and Development Expenses](index=25&type=section&id=Research%20and%20Development%20Expenses) This section details the nature and expected trends of the company's research and development expenditures - **Research and development costs** are expensed as incurred and primarily relate to the discovery and development of INZ-701, including fees to CROs, manufacturing scale-up, personnel, and regulatory compliance[102](index=102&type=chunk)[103](index=103&type=chunk)[104](index=104&type=chunk) - **R&D expenses** are expected to **increase substantially** due to the global development strategy, ongoing and planned clinical trials of INZ-701, manufacturing scale-up, and potential development of additional product candidates[105](index=105&type=chunk) [General and Administrative Expenses](index=26&type=section&id=General%20and%20Administrative%20Expenses) This section describes the components and anticipated changes in general and administrative costs - **General and administrative expenses** primarily cover salaries, benefits, **stock-based compensation** for executive, finance, and administrative functions, as well as professional fees and facilities costs[106](index=106&type=chunk) - These expenses are anticipated to **increase** with headcount expansion, costs associated with being a public company, and preparation for potential commercial operations, including sales and marketing[106](index=106&type=chunk) [Interest Income, net](index=26&type=section&id=Interest%20Income,%20net) This section explains the sources of net interest income, including investments and debt interest - **Interest income, net**, includes income from bank deposits and investments, offset by interest expense from the Loan Agreement[107](index=107&type=chunk) [Other Income (Expense), net](index=26&type=section&id=Other%20Income%20(Expense),%20net) This section clarifies the components of other income and expense, primarily from marketable securities and foreign exchange - **Other income (expense), net**, primarily consists of **interest income** on marketable securities and foreign exchange gains or losses[108](index=108&type=chunk) [Results of Operations](index=26&type=section&id=Results%20of%20Operations) This section compares the company's financial performance across different reporting periods [Comparison of the Three Months Ended September 30, 2023 and 2022](index=26&type=section&id=Comparison%20of%20the%20Three%20Months%20Ended%20September%2030,%202023%20and%202022) This section analyzes the financial performance for the three-month periods ended September 30, 2023 and 2022 Results of Operations (Three Months Ended September 30, amounts in thousands) | Metric | 2023 | 2022 | Change | | :-------------------------- | :----- | :----- | :----- | | Research and development | $13,341 | $12,191 | $1,150 | | General and administrative | $4,733 | $4,721 | $12 | | Total operating expenses | $18,074 | $16,912 | $1,162 | | Loss from operations | $(18,074) | $(16,912) | $1,162 | | Interest income, net | $1,416 | $737 | $679 | | Other income (expense), net | $20 | $(197) | $(217) | | Net loss | $(16,638) | $(16,372) | $266 | [Research and Development Expenses_3M](index=27&type=section&id=Research%20and%20Development%20Expenses_3M) This section details the changes in research and development expenses for the three-month period - **Research and development expense increased** by **$1.2 million** (**9.4%**) for the three months ended September 30, 2023, compared to the same period in 2022, driven by a **$0.7 million increase** in INZ-701-related R&D and a **$0.4 million increase** in personnel-related expense[110](index=110&type=chunk)[111](index=111&type=chunk) [General and Administrative Expense_3M](index=27&type=section&id=General%20and%20Administrative%20Expense_3M) This section explains the trends in general and administrative expenses for the three-month period - **General and administrative expense** remained relatively flat for the three months ended September 30, 2023, compared to the same period in 2022[112](index=112&type=chunk) [Interest Income, net_3M](index=27&type=section&id=Interest%20Income,%20net_3M) This section analyzes the changes in net interest income for the three-month period - **Net interest income increased** by approximately **$0.7 million** for the three months ended September 30, 2023, primarily due to a **$1.6 million increase** in **interest income** from higher rates and larger cash balances, partially offset by a **$0.9 million increase** in Loan Agreement interest expense[113](index=113&type=chunk) [Other Income (Expense), net_3M](index=27&type=section&id=Other%20Income%20(Expense),%20net_3M) This section discusses the changes in other income (expense), net, for the three-month period - **Other income (expense), net, decreased** by approximately **$0.2 million** for the three months ended September 30, 2023, compared to 2022, mainly due to unrealized losses on Euro-denominated cash balances in the prior year[114](index=114&type=chunk) [Comparison of the Nine Months Ended September 30, 2023 and 2022](index=27&type=section&id=Comparison%20of%20the%20Nine%20Months%20Ended%20September%2030,%202023%20and%202022) This section analyzes the financial performance for the nine-month periods ended September 30, 2023 and 2022 Results of Operations (Nine Months Ended September 30, amounts in thousands) | Metric | 2023 | 2022 | Change | | :-------------------------- | :----- | :----- | :----- | | Research and development | $36,864 | $34,012 | $2,852 | | General and administrative | $15,973 | $15,130 | $843 | | Total operating expenses | $52,837 | $49,142 | $3,695 | | Loss from operations | $(52,837) | $(49,142) | $3,695 | | Interest income, net | $3,254 | $1,118 | $2,136 | | Other expense, net | $(42) | $(493) | $(451) | | Net loss | $(49,625) | $(48,517) | $1,108 | [Research and Development Expenses_9M](index=28&type=section&id=Research%20and%20Development%20Expenses_9M) This section details the changes in research and development expenses for the nine-month period - **Research and development expense increased** by approximately **$2.9 million** (**8.4%**) for the nine months ended September 30, 2023, compared to the same period in 2022, primarily due to a **$1.4 million increase** in personnel-related expense and a **$1.3 million increase** in INZ-701-related R&D[116](index=116&type=chunk)[117](index=117&type=chunk) [General and Administrative Expense_9M](index=28&type=section&id=General%20and%20Administrative%20Expense_9M) This section explains the trends in general and administrative expenses for the nine-month period - **General and administrative expense increased** by **$0.8 million** (**5.6%**) for the nine months ended September 30, 2023, compared to 2022, mainly due to a **$0.7 million increase** in personnel-related expenses from a former CEO's transition and separation agreement[118](index=118&type=chunk) [Interest Income, net_9M](index=28&type=section&id=Interest%20Income,%20net_9M) This section analyzes the changes in net interest income for the nine-month period - **Net interest income increased** by approximately **$2.1 million** for the nine months ended September 30, 2023, compared to 2022, driven by a **$4.2 million increase** in **interest income** from higher rates and larger cash balances, partially offset by a **$2.0 million increase** in Loan Agreement interest expense[119](index=119&type=chunk) [Other Expense, net_9M](index=28&type=section&id=Other%20Expense,%20net_9M) This section discusses the changes in other expense, net, for the nine-month period - **Other expense, net, decreased** by approximately **$0.5 million** for the nine months ended September 30, 2023, compared to 2022, due to lower unrealized losses on marketable securities[120](index=120&type=chunk) [Liquidity and Capital Resources](index=28&type=section&id=Liquidity%20and%20Capital%20Resources) This section examines the company's ability to meet its financial obligations and fund future operations [Sources of Liquidity](index=28&type=section&id=Sources%20of%20Liquidity) This section identifies the primary sources of funding for the company's operations - The Company funds operations primarily through convertible preferred stock sales, common stock offerings, pre-funded warrants, and borrowings under its Loan Agreement[121](index=121&type=chunk) - In August 2023, an underwritten offering of **14,375,000 common shares** generated approximately **$64.4 million** in **net proceeds**[125](index=125&type=chunk) Total Cash, Cash Equivalents, and Short-Term Investments (amounts in thousands) | Metric | September 30, 2023 | December 31, 2022 | | :------------------------------------------ | :------------------- | :------------------ | | Cash and cash equivalents | $46,895 | $32,915 | | Short-term investments | $145,547 | $94,951 | | **Total** | **$192,442** | **$127,866** | [Cash Flows](index=29&type=section&id=Cash%20Flows) This section summarizes the company's cash flow activities from operations, investing, and financing Cash Flow Summary (Nine Months Ended September 30, amounts in thousands) | Metric | 2023 | 2022 | | :------------------------------------------ | :----- | :----- | | Net cash used in operating activities | $(52,188) | $(43,361) | | Net cash used in investing activities | $(47,176) | $(8,621) | | Net cash provided by financing activities | $113,453 | $72,788 | | Net increase in cash, cash equivalents, and restricted cash | $14,089 | $20,806 | [Net Cash Used in Operating Activities](index=30&type=section&id=Net%20Cash%20Used%20in%20Operating%20Activities) This section analyzes the factors contributing to cash used in operating activities - **Net cash used in operating activities increased** by approximately **$8.8 million** for the nine months ended September 30, 2023, primarily due to a **$3.9 million increase** in payments against accounts payable and accrued expenses and a **$3.3 million increase** in **net loss** adjusted for non-cash items[128](index=128&type=chunk) [Net Cash Used in Investing Activities](index=30&type=section&id=Net%20Cash%20Used%20in%20Investing%20Activities) This section details the changes in cash used for investing activities, primarily marketable securities - **Net cash used in investing activities increased** by approximately **$38.6 million** for the nine months ended September 30, 2023, as an **$85.5 million increase** in **marketable securities purchases** outpaced a **$46.7 million increase** in maturities[129](index=129&type=chunk) [Net Cash Provided by Financing Activities](index=30&type=section&id=Net%20Cash%20Provided%20by%20Financing%20Activities) This section explains the sources of cash provided by financing activities, including debt and equity - **Net cash provided by financing activities increased** by **$40.7 million** for the nine months ended September 30, 2023, primarily due to a **$23.5 million increase** from **long-term debt issuance** and a **$17.4 million increase** from common stock and pre-funded warrant issuances[130](index=130&type=chunk) [Funding Requirements](index=30&type=section&id=Funding%20Requirements) This section outlines the company's future capital needs and potential financing strategies - The Company expects to incur substantial financial resources for ongoing and planned activities, including clinical trials, R&D, and potential commercialization, necessitating additional funding[131](index=131&type=chunk) - Existing **cash, cash equivalents, and short-term investments** are projected to **fund operations into the fourth quarter of 2025**, but this estimate is subject to change and potential faster capital consumption[133](index=133&type=chunk) - Future financing may involve debt or equity agreements with covenants that limit operations, or collaborations that require relinquishing rights to technologies or product candidates[132](index=132&type=chunk) [Critical Accounting Estimates](index=31&type=section&id=Critical%20Accounting%20Estimates) This section highlights key accounting estimates and assumptions that significantly impact financial reporting - The preparation of financial statements requires management to make estimates and assumptions, particularly for research and development expenses[31](index=31&type=chunk)[134](index=134&type=chunk) - No material changes to critical accounting estimates occurred during the three and nine months ended September 30, 2023, compared to the prior Annual Report on Form 10-K[134](index=134&type=chunk) [Contractual Obligations, Commitments and Contingencies](index=31&type=section&id=Contractual%20Obligations,%20Commitments%20and%20Contingencies) This section updates on the company's contractual obligations and potential liabilities - No material changes to contractual obligations and commitments occurred during the three and nine months ended September 30, 2023, except for additional borrowings under the Loan Agreement in February and June 2023[135](index=135&type=chunk) [Emerging Growth Company Status](index=31&type=section&id=Emerging%20Growth%20Company%20Status) This section explains the company's election to use extended transition periods for new accounting standards - The Company has elected to use the extended transition period for complying with new or revised accounting standards as an 'emerging growth company' under the Jumpstart Our Business Startups Act of 2012[136](index=136&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=31&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section details the company's exposure to market risks, primarily interest rate fluctuations on investments and variable-rate debt - The Company's primary market risk exposure is **interest rate sensitivity**, affecting its cash equivalents (money market funds, U.S. government agency debt securities) and short-term investments (U.S. Treasury securities, U.S. government agency debt securities)[137](index=137&type=chunk) - An immediate **100 basis point change** in **interest rates** would not materially affect the fair market value of the investment portfolio or financial results due to the short-term and low-risk nature of investments[137](index=137&type=chunk) - The **outstanding principal** of **$32.5 million** under the Loan Agreement bears a **variable interest rate**, which was **9.60%** as of September 30, 2023[138](index=138&type=chunk) [Item 4. Controls and Procedures](index=32&type=section&id=Item%204.%20Controls%20and%20Procedures) Management confirmed the effectiveness of disclosure controls and procedures, with no material changes to internal control over financial reporting - Management, with CEO and CFO participation, concluded that disclosure controls and procedures were effective at the reasonable assurance level as of September 30, 2023[141](index=141&type=chunk) - No material changes in internal control over financial reporting occurred during the period covered by this Quarterly Report on Form 10-Q[142](index=142&type=chunk) [PART II. OTHER INFORMATION](index=33&type=section&id=PART%20II.%20OTHER%20INFORMATION) This section provides additional information not covered in the financial statements, including risk factors and equity sales [Item 1A. Risk Factors](index=33&type=section&id=Item%201A.%20Risk%20Factors) This section directs readers to the Annual Report for risk factors, noting potential new or immaterial risks - Readers should carefully consider risk factors discussed in the Annual Report on Form 10-K for the year ended December 31, 2022, which are qualified by information in this Quarterly Report[145](index=145&type=chunk) - The Company acknowledges that additional risks not currently known or deemed immaterial could materially adversely affect its business, financial condition, or future results[145](index=145&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=33&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section confirms no unregistered equity sales and updates on the use of IPO proceeds for clinical development and R&D - No unregistered equity securities were issued during the three months ended September 30, 2023[146](index=146&type=chunk) - The Company received approximately **$115.9 million** in **net proceeds** from its July 2020 IPO[148](index=148&type=chunk) - As of September 30, 2023, approximately **$110.9 million** of IPO **net proceeds** have been used for clinical development of INZ-701, preclinical R&D, working capital, and general corporate purposes, with no material change to the planned use[149](index=149&type=chunk) [Item 6. Exhibits](index=34&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed with the Form 10-Q, including corporate governance documents and executive certifications - The exhibits include the Restated Certificate of Incorporation, Amended and Restated Bylaws, certifications from the Principal Executive Officer and Principal Financial Officer (pursuant to Sarbanes-Oxley Act Sections 302 and 906), and various Inline XBRL taxonomy documents[151](index=151&type=chunk)[152](index=152&type=chunk) [SIGNATURES](index=35&type=section&id=SIGNATURES) This section contains the official certifications and signatures of the company's authorized officers [Signatures](index=35&type=section&id=Signatures) This section contains the official signatures of the Chief Executive Officer and Chief Financial Officer, certifying the report filing - The report is signed by Douglas A. Treco, Chief Executive Officer, and Sanjay S. Subramanian, Chief Financial Officer, on November 7, 2023[156](index=156&type=chunk) ```