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Inozyme Pharma, Inc. - INZY STOCK NEWS

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Overview of Inozyme Pharma, Inc.

Inozyme Pharma, Inc. (INZY) is a clinical-stage biopharmaceutical company dedicated to developing novel enzyme replacement therapies for rare diseases. Specializing in disorders of paradoxical mineralization and intimal proliferation, the company targets severe metabolic conditions that affect bone health, vascular function, and soft tissue integrity. Its innovative approach centers on the modulation of the PPi-Adenosine pathway, a critical biochemical mechanism involved in preventing pathologic calcification and abnormal vascular smooth muscle proliferation.

Core Therapeutic Focus

At the heart of Inozyme Pharma is its commitment to addressing disorders such as ENPP1 Deficiency and ABCC6 Deficiency, both of which lead to devastating clinical manifestations including arterial calcification and musculoskeletal complications. The company is also actively investigating applications in calciphylaxis, a rare and life-threatening complication associated with end-stage kidney disease. By harnessing its expertise in enzyme replacement therapy, Inozyme aims to offer a transformative treatment option where no approved therapies currently exist.

Innovative Technology and Product Candidate

The company’s lead candidate, INZ-701, is a soluble, recombinant, genetically engineered fusion protein designed to correct deficiencies in the PPi-Adenosine pathway. INZ-701 works by metabolizing extracellular ATP to generate inorganic pyrophosphate (PPi), a natural inhibitor of pathological mineralization, and by producing adenosine, which plays a role in regulating intimal proliferation. This dual mechanism offers significant potential to mitigate the underlying causes of critical rare diseases that affect bone, vascular, and soft tissue function.

Research, Development, and Clinical Strategy

Inozyme Pharma is committed to a robust clinical development program, conducting early-phase trials to evaluate the safety, tolerability, as well as the pharmacokinetic and pharmacodynamic profiles of INZ-701. The company’s research strategy is underpinned by in-depth natural history studies and retrospective analyses, which have enhanced understanding of these rare conditions and refined clinical trial design. By leveraging advanced biotechnological techniques alongside strategic clinical collaborations, Inozyme Pharma is positioned to deliver meaningful insights into disease mechanisms and potential therapeutic benefits.

Scientific Expertise and Market Position

With a strong foundation in molecular biology and genetic engineering, Inozyme Pharma demonstrates expertise in the design and development of enzyme replacement therapies. The company is recognized for its scientific rigor and methodical approach to drug development, which is reflected in its comprehensive evaluation of the PPi-Adenosine pathway. This focus not only supports its lead candidate but also positions Inozyme as a key player in the increasingly competitive rare disease therapeutic landscape. The firm’s activities, including multiple clinical studies, strategic research partnerships, and participation in scientific forums, underscore its commitment to advancing treatments for debilitating metabolic disorders.

Therapeutic Impact and Unmet Need

Rare diseases such as ENPP1 Deficiency and ABCC6 Deficiency present high unmet medical needs due to their severe impact on patient quality of life, including significant skeletal, cardiovascular, and neurological challenges. In addressing these conditions, Inozyme Pharma’s work is not only scientifically innovative but also critically important in the broader context of rare disease management. Its therapeutic candidate, INZ-701, offers a novel mechanism of action that could potentially alter the course of these diseases by restoring a key regulatory pathway, thereby reducing morbidity and improving patient outcomes.

Conclusion

Inozyme Pharma, Inc. combines advanced biotechnological innovation with deep expertise in rare disease pathology to address some of the most challenging conditions in modern medicine. Through its targeted approach to enzyme replacement therapy and its focus on the PPi-Adenosine pathway, the company continues to push the boundaries of therapeutic discovery in the rare disease field. This comprehensive strategy leverages both scientific insight and clinical precision to potentially transform treatment paradigms for patients suffering from debilitating disorders affecting bone health and vascular integrity.

Rhea-AI Summary

BOSTON, June 07, 2022 – Inozyme Pharma (Nasdaq: INZY) announced the appointment of Dr. Kurt Gunter as Chief Medical Officer. Dr. Gunter brings over 30 years of experience in regulatory affairs and clinical development, previously serving at Athenex and Kuur Therapeutics. He will lead Inozyme's clinical strategy for INZ-701, currently in Phase 1/2 trials for ENPP1 and ABCC6 Deficiencies. These are serious rare diseases with no approved therapies, making INZ-701 a vital potential treatment. His expertise is expected to enhance Inozyme's efforts to address unmet medical needs.

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Inozyme Pharma (Nasdaq: INZY), a biopharmaceutical company focused on rare diseases, announced that its CEO, Axel Bolte, will present at the Jefferies Healthcare Conference on June 8, 2022, at 2:30 PM ET. The presentation will discuss the company’s advancements in developing therapies for abnormal mineralization diseases, particularly those related to ENPP1 and ABCC6 deficiencies. A replay of the presentation will be available on the Investor Relations section of Inozyme's website.

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Inozyme Pharma reported positive preliminary data for its Phase 1/2 trial of INZ-701 in ENPP1 Deficiency, showing significant increases in plasma pyrophosphate levels. The company expects topline data later in 2022. Additionally, dosing in the ABCC6 Deficiency trial is progressing on schedule with preliminary data anticipated soon. As of March 31, 2022, cash reserves stood at $97.8 million, supplemented by a $68.3 million offering, extending operational runway into Q4 2023. However, R&D expenses rose to $11.8 million, contributing to a net loss of $16.9 million for the quarter.

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Inozyme Pharma (Nasdaq: INZY), a clinical-stage biopharmaceutical firm, announced that CEO Axel Bolte will present at two investor conferences. The first is the Bank of America Securities 2022 Healthcare Conference on May 10, 2022, at 7:40 p.m. ET. The second is the H.C. Wainwright Global Investment Conference, with a prerecorded presentation available on-demand starting May 24, 2022, at 7 a.m. ET. Replays can be accessed from Inozyme's Investor Relations section on their website, emphasizing its commitment to developing therapeutics for rare diseases linked to mineralization.

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Inozyme Pharma, Inc. (Nasdaq: INZY) has announced the pricing of an underwritten offering of approximately 16.3 million shares of its common stock at $3.69 per share, along with pre-funded warrants for an additional 3.5 million shares at $3.6899 each. The offering aims to raise gross proceeds of about $73 million before expenses. The transaction is expected to close on April 19, 2022. Jefferies LLC and Cowen and Company, LLC are the joint book-running managers for this offering. A shelf registration statement related to these securities was previously filed with the SEC.

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Inozyme Pharma (Nasdaq: INZY) has begun dosing its first patient in a Phase 1/2 clinical trial for INZ-701, targeting ABCC6 Deficiency. Meanwhile, the second cohort in the trial for ENPP1 Deficiency is fully enrolled, with topline results anticipated in the second half of 2022. Preliminary safety and biomarker data from the ABCC6 trial are expected by Q2 2022. The trials aim to assess the safety and efficacy of INZ-701, which has shown promise in earlier models by increasing plasma pyrophosphate levels and preventing soft tissue calcification, significant factors in both conditions.

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Inozyme Pharma (Nasdaq: INZY) announced that its CEO Axel Bolte will participate in a fireside chat at the 21st Annual Needham Virtual Healthcare Conference on April 13, 2022, at 8:45 AM ET. The event will provide insights into the company’s development of INZ-701, a novel therapy targeting rare genetic diseases caused by ENPP1 and ABCC6 deficiencies. A replay of the chat will be available for 30 days in the Investor & News section of Inozyme’s website.

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Inozyme Pharma reported positive preliminary data from its Phase 1/2 clinical trial of INZ-701 for treating ENPP1 Deficiency, with three patients in the lowest dose cohort (0.2 mg/kg) showing significant increases in plasma pyrophosphate (PPi) levels. The PPi levels rose to a mean of 1356 nM, a 5-fold increase from baseline. INZ-701 was well-tolerated without serious adverse events. The trial is progressing to the next dose level (0.6 mg/kg), and topline data is expected in the second half of 2022, addressing a critical medical need as no approved therapies currently exist for this condition.

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Inozyme Pharma (Nasdaq: INZY) reported a net loss of $56.6 million, or $2.40 per share, for the year ended December 31, 2021. R&D expenses decreased to $37.7 million from $46.5 million in 2020, while G&A expenses rose to $18.9 million. The company is advancing its clinical programs for INZ-701, with patient dosing underway for ENPP1 Deficiency and initial patient enrollment expected for ABCC6 Deficiency. Preliminary safety and biomarker data are anticipated in the first half of 2022. Cash reserves stood at $111.8 million, sufficient to fund operations into Q1 2023.

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Inozyme Pharma (Nasdaq: INZY) announced the appointment of Sanjay S. Subramanian as CFO and Soojin Kim, Ph.D., as CTOO. Subramanian brings over 20 years of financial expertise, previously raising over $200 million at Ocugen. Kim, with more than 25 years in pharmaceutical R&D, will enhance the company's manufacturing operations for INZ-701, currently in Phase 1/2 trials for ENPP1 Deficiency. Both executives aim to advance Inozyme's mission in treating rare diseases caused by abnormal mineralization.

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FAQ

What is the current stock price of Inozyme Pharma (INZY)?

The current stock price of Inozyme Pharma (INZY) is $0.8755 as of April 3, 2025.

What is the market cap of Inozyme Pharma (INZY)?

The market cap of Inozyme Pharma (INZY) is approximately 54.6M.

What is the core focus of Inozyme Pharma, Inc.?

Inozyme Pharma is focused on developing novel enzyme replacement therapies for rare diseases, particularly those affecting bone health, vascular function, and soft tissues through targeting the PPi-Adenosine pathway.

Which conditions are primarily targeted by the company?

The company primarily targets conditions such as ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis, which are characterized by pathologic mineralization and abnormal vascular smooth muscle proliferation.

What is INZ-701?

INZ-701 is a recombinant, genetically engineered fusion protein that functions as an enzyme replacement therapy aimed at restoring the balance in the PPi-Adenosine pathway to counteract disorders of mineralization and intimal proliferation.

How does INZ-701 work in the body?

INZ-701 metabolizes extracellular ATP to generate inorganic pyrophosphate (PPi), which inhibits abnormal mineralization, and produces adenosine, which helps regulate vascular smooth muscle proliferation.

What stage of development is Inozyme Pharma currently in?

Inozyme Pharma is a clinical-stage biopharmaceutical company actively conducting early-phase trials and natural history studies to evaluate the safety and efficacy of its therapeutic candidates.

Why is the PPi-Adenosine pathway significant?

The PPi-Adenosine pathway plays a crucial role in regulating mineralization and vascular smooth muscle proliferation. Disruptions in this pathway can lead to severe conditions such as ENPP1 and ABCC6 Deficiencies, making it a key target for therapeutic intervention.

How does Inozyme Pharma differentiate itself from its competitors?

The company differentiates itself through its specialized focus on the PPi-Adenosine pathway and the development of targeted enzyme replacement therapies, supported by deep scientific expertise and comprehensive clinical research.

What is the potential impact of Inozyme Pharma's research?

By addressing the underlying molecular mechanisms of rare metabolic disorders, Inozyme Pharma's research has the potential to significantly improve clinical outcomes and quality of life for patients suffering from debilitating conditions with limited treatment options.

Are there any approved therapies for the targeted diseases?

Currently, there are no approved therapies that effectively address the underlying causes of ENPP1 Deficiency, ABCC6 Deficiency, or calciphylaxis, which underscores the importance of Inozyme Pharma's innovative approach.
Inozyme Pharma, Inc.

Nasdaq:INZY

INZY Rankings

INZY Stock Data

54.61M
63.25M
1.2%
92.62%
6.83%
Biotechnology
Pharmaceutical Preparations
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United States
BOSTON