Core Insights - Inozyme Pharma, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for rare diseases affecting bone health and blood vessel function [3][4] - The company will participate in a fireside chat at the Piper Sandler 36th Annual Healthcare Conference on December 4, 2024 [1][2] Company Overview - Inozyme Pharma specializes in the PPi-Adenosine Pathway, where the ENPP1 enzyme plays a crucial role in generating inorganic pyrophosphate (PPi) and adenosine, which are vital for regulating mineralization and controlling smooth muscle cell proliferation in blood vessels [3] - Disruptions in this pathway can lead to severe conditions such as ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL) [3] Lead Candidate - The lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) aimed at increasing levels of PPi and adenosine [4] - INZ-701 is currently in clinical development for treating ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis, targeting the underlying issues of pathological mineralization and intimal proliferation [4]

Group 1 - Inozyme Pharma, Inc. (INZY) has experienced a significant downtrend, with a 30% decline in stock price over the past four weeks, indicating excessive selling pressure [1] - The stock is currently in oversold territory, as indicated by its Relative Strength Index (RSI) reading of 28.72, suggesting a potential for a price reversal [3] - Analysts have raised earnings estimates for INZY by 4.2% over the last 30 days, indicating a strong consensus among sell-side analysts that could lead to price appreciation [3] Group 2 - The Zacks Rank for INZY is 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate revisions and EPS surprises, further supporting the potential for a turnaround [3]

Core Insights - Inozyme Pharma, Inc. is a clinical-stage biopharmaceutical company focused on developing therapeutics for rare diseases affecting bone health and blood vessel function [3][4] - The company will have presentations at two upcoming investor conferences, the Stifel 2024 Healthcare Conference and the Jefferies London Healthcare Conference [1][2] Company Overview - Inozyme Pharma specializes in the PPi-Adenosine Pathway, which involves the ENPP1 enzyme that generates inorganic pyrophosphate (PPi) and adenosine, both crucial for regulating mineralization and controlling smooth muscle cell proliferation in blood vessels [3] - Disruptions in this pathway can lead to severe conditions such as ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL) [3] Lead Candidate - The lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) aimed at increasing PPi and adenosine levels [4] - INZ-701 is currently in clinical development for treating ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis, targeting the underlying issues of pathological mineralization and intimal proliferation [4]

Product Development and Clinical Trials - INZ-701 is a lead product candidate designed to treat ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis by increasing levels of inorganic pyrophosphate (PPi) and adenosine[68]. - The company reported positive interim data from the Phase 1 SEAPORT 1 Trial of INZ-701 in patients with end-stage kidney disease (ESKD) receiving hemodialysis in October 2024, with plans to initiate a pivotal trial in 2025[70]. - The FDA has granted Orphan Drug Designation and Fast Track designation for INZ-701 for both ENPP1 and ABCC6 Deficiencies, indicating regulatory support for its development[69]. - The ongoing Phase 1/2 clinical trial of INZ-701 in adults with ENPP1 Deficiency has shown a significant increase in plasma PPi levels, reaching comparable levels to healthy subjects[75]. - The ENERGY 3 trial, a pivotal trial for INZ-701 in pediatric patients with ENPP1 Deficiency, is expected to complete enrollment by the end of 2024, with topline data anticipated in early 2026[72]. - The company plans to initiate the ENERGY 2 trial for infants with ENPP1 Deficiency in Q4 2024, focusing on changes in plasma PPi and survival as co-primary endpoints[81]. - Interim data from the ENERGY 1 trial in infants is expected to be reported in Q4 2024, assessing safety, tolerability, and pharmacodynamics of INZ-701[78]. - Significant reductions in fibroblast growth factor-23 and increases in bone-specific alkaline phosphatase levels were observed in the 1.8 mg/kg dose cohort of the Phase 1/2 trial, indicating restoration of proper bone mineralization[76]. - The first site for the ENERGY 3 trial was opened in September 2023, with patient recruitment underway and a milestone payment of $0.5 million incurred after the first patient was dosed[84]. - Enrollment for the ENERGY 3 trial is expected to be completed by the end of 2024, with topline data anticipated in early 2026[84]. - Positive data from ongoing and planned clinical trials of INZ-701 could support marketing applications in the US and EU, with a potential commercial launch for pediatric patients as early as the first half of 2027[85][86]. - Interim data from the Phase 1/2 trial of INZ-701 in adult patients with ABCC6 Deficiency showed a sustained increase in plasma PPi levels, with mean baseline levels at 947±193 nM and peak levels reaching 2169 nM[89]. - INZ-701 demonstrated a favorable safety profile in the Phase 1/2 trial, with no serious or severe adverse events reported, and a total of approximately 12+ patient-years of treatment across all cohorts[91]. - SEAPORT 1 trial for patients with ESKD receiving hemodialysis showed significant increases in PPi levels, with baseline levels at 619±74 nM and reaching 1551±270 nM by Day 24[96][95]. - INZ-701 also led to reductions in biomarkers of mineral metabolism in ESKD patients, suggesting potential benefits in mitigating vascular calcification risks[97]. - A pivotal trial of INZ-701 in patients with calciphylaxis is planned for 2025, subject to regulatory review and funding[100]. - The ADAPT long-term safety study for INZ-701 has begun enrolling patients who completed previous trials, with dosing set at 1.8 mg/kg for adults and 2.4 mg/kg for pediatric patients[100]. Financial Performance and Funding - The company has not yet commercialized any products and continues to incur significant operating losses, necessitating additional funding to support ongoing operations[104][107]. - As of September 30, 2024, the company expects existing cash and short-term investments to fund cash flow requirements for at least the next 12 months[109]. - Research and development expenses for the three months ended September 30, 2024, increased by $6.5 million to $19.9 million compared to $13.3 million in the same period of 2023[118]. - INZ-701-related research and development expenses rose by $5.7 million primarily due to a $3.0 million increase in chemistry, manufacturing, and controls expenses[120]. - Total operating expenses for the nine months ended September 30, 2024, were $76.9 million, an increase of $24.0 million from $52.8 million in the same period of 2023[124]. - The net loss for the three months ended September 30, 2024, was $24.6 million, compared to a net loss of $16.6 million for the same period in 2023[118]. - Interest income for the three months ended September 30, 2024, decreased by approximately $0.6 million to $1.8 million compared to $2.4 million in the same period of 2023[121]. - Interest expense increased by $0.5 million for the three months ended September 30, 2024, primarily due to borrowings under the Loan Agreement[122]. - Research and development expenses for the nine months ended September 30, 2024, increased by $23.9 million to $60.8 million compared to $36.9 million in the same period of 2023[125]. - INZ-701-related research and development expenses for the nine months ended September 30, 2024, increased by $20.2 million, primarily due to a $9.3 million increase in chemistry, manufacturing, and controls expenses[126]. - The company anticipates continued increases in research and development expenses as it executes its global development strategy and prepares for ongoing clinical trials[114]. - As of September 30, 2024, total cash, cash equivalents, and short-term investments amounted to $131.6 million, down from $188.6 million as of December 31, 2023[133]. - Net cash used in operating activities for the nine months ended September 30, 2024 was $(72.7) million, an increase of approximately $20.5 million compared to $(52.2) million for the same period in 2023[134][135]. - Net cash provided by investing activities increased by approximately $98.7 million for the nine months ended September 30, 2024, primarily due to an $85.0 million decrease in purchases of marketable securities[136]. - Net cash provided by financing activities decreased by $102.3 million for the nine months ended September 30, 2024, compared to the same period in 2023, mainly due to a $64.4 million decrease related to the July 2023 equity offering[137]. - The company has an aggregate of $45.0 million principal in term loans outstanding under the Loan Agreement, with an interest rate of 9.60% as of September 30, 2024[146][147]. - The company expects to incur significant commercialization expenses related to product manufacturing, sales, marketing, and distribution if marketing approval for INZ-701 or other product candidates is obtained[138]. - As of December 31, 2023, the company had sold 3,553,995 shares of common stock for aggregate net proceeds of $21.2 million under the Open Market Sale Agreement[130]. - The company anticipates needing substantial additional funding to support ongoing and planned activities, particularly for clinical trials and research and development[138]. - The company has filed a universal shelf registration statement allowing it to offer and sell up to $300.0 million of various securities, including common stock and preferred stock[130]. - Cash flows from operating activities are expected to be impacted by the company's ongoing clinical trials and research efforts, which may require more capital than currently anticipated[141].

Exhibit 99.1 Inozyme Pharma Reports Third Quarter 2024 Financial Results and Provides Business Highlights - Interim data from ENERGY 1, a Phase 1b trial of INZ-701 in infants with ENPP1 Deficiency, on track for fourth quarter of 2024 – - Topline data from ENERGY 3, a pivotal trial of INZ-701 in pediatric patients with ENPP1 Deficiency, expected in early 2026 - - Company plans to initiate registrational trials in calciphylaxis and ABCC6 Deficiency in 2025 subject to regulatory alignment and sufficient fundin ...

- Interim data from ENERGY 1, a Phase 1b trial of INZ-701 in infants with ENPP1 Deficiency, on track for fourth quarter of 2024 – - Topline data from ENERGY 3, a pivotal trial of INZ-701 in pediatric patients with ENPP1 Deficiency, expected in early 2026 - - Company plans to initiate registrational trials in calciphylaxis and ABCC6 Deficiency in 2025 subject to regulatory alignment and sufficient funding - - Cash, cash equivalents, and short-term investments as of September 30, 2024, expected to fund operat ...

- INZ-701 was well-tolerated and significantly increased plasma pyrophosphate (PPi) levels in patients with end-stage kidney disease (ESKD) undergoing hemodialysis; low PPi levels are associated with calciphylaxis, a rare, life-threatening complication of ESKD - - Company plans to initiate a registrational trial in calciphylaxis in 2025 subject to regulatory alignment and sufficient funding - BOSTON, Oct. 24, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinic ...

BOSTON, Oct. 17, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) ("the Company" or "Inozyme"), a clinical-stage biopharmaceutical company developing innovative therapeutics for rare diseases that affect bone health and blood vessel function, today announced that interim data from the Company's ongoing Phase 1 SEAPORT 1 of INZ-701 in patients with end-stage kidney disease (ESKD) receiving hemodialysis will be presented during a poster session at the American Society of Nephrology (ASN) Kidney We ...

BOSTON, Oct. 07, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) ("the Company" or "Inozyme"), a clinical-stage biopharmaceutical company developing innovative therapeutics for rare diseases that affect bone health and blood vessel function, today announced the appointment of Erik Harris to its Board of Directors, effective October 3, 2024. Mr. Harris, who currently serves as Chief Commercial Officer and Executive Vice President at Ultragenyx, brings to Inozyme over 20 years of commercial exper ...

Stifel initiated coverage on Inozyme Pharma Inc. INZY, saying, "This under-the-radar stock could be worth a look ahead of it." The company's lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to increase PPi and adenosine, enabling the potential treatment of multiple diseases caused by deficiencies in these molecules. It is currently in clinical development for ENPP1 Deficiency, ABCC6 Deficiency and calciphylaxis. In July, the FDA granted fast track designation ...