Inozyme Pharma presents data on INZ-701 for ENPP1 Deficiency at CHOP Cardiology Annual Meeting, February 22, 2025.
Quiver AI Summary
Inozyme Pharma, Inc. announced that Dr. Kurt Gunter, the company's Senior Vice President and Chief Medical Officer, will present data on the Expanded Access Program for their therapeutic candidate INZ-701, aimed at treating ENPP1 Deficiency in infants and children, at the CHOP Cardiology Annual Meeting in Orlando from February 19-23, 2025. ENPP1 Deficiency is a rare and severe disease affecting bones and blood vessels, often leading to significant health challenges for those affected. The presentation will cover the impact of INZ-701, an enzyme replacement therapy designed to improve critical levels of inorganic pyrophosphate and adenosine, which play vital roles in bone and cardiovascular health. Inozyme Pharma is focused on developing treatments for rare diseases linked to deficiencies in these pathways.
Potential Positives
- Dr. Kurt Gunter, Senior Vice President and Chief Medical Officer, will present significant data on INZ-701, indicating progress in the treatment of a serious rare disease, ENPP1 Deficiency.
- The Expanded Access Program (EAP) demonstrates Inozyme's commitment to addressing unmet medical needs in a condition with no approved therapies, potentially positioning the company as a leader in this niche market.
- ENPP1 Deficiency affects a vulnerable patient population, and the successful presentation of data may enhance awareness and interest from the medical community, investors, and stakeholders in Inozyme's therapeutic approaches.
Potential Negatives
- The press release highlights the severity of ENPP1 Deficiency and the lack of approved therapies, suggesting a pressing medical need that the company is currently addressing but which also emphasizes the high stakes involved in their research and development efforts.
- As the company is in the clinical development stage for its lead candidate, there may be significant uncertainty regarding the efficacy and safety of INZ-701, which could impact investor confidence.
- The mention of high mortality rates in infants with ENPP1 Deficiency may evoke concern regarding the urgency and potential implications of their therapeutic development, affecting the company's public image.
FAQ
What is ENPP1 Deficiency?
ENPP1 Deficiency is a serious, progressive rare disease affecting blood vessels, soft tissues, and bones, with significant morbidity and mortality.
What is INZ-701?
INZ-701 is an enzyme replacement therapy designed to increase levels of inorganic pyrophosphate and adenosine to treat diseases like ENPP1 Deficiency.
When and where will the data presentation take place?
The presentation will be held on February 22, 2025, at the CHOP Cardiology Annual Meeting in Orlando, Florida.
Who will present the data on INZ-701?
Kurt Gunter, M.D., Senior Vice President and Chief Medical Officer of Inozyme Pharma, will present the data on INZ-701.
Are there approved therapies for ENPP1 Deficiency?
No, currently there are no approved therapies for ENPP1 Deficiency, making INZ-701 a significant potential treatment option.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
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Full Release
BOSTON, Feb. 21, 2025 (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 Kurt Gunter, M.D., Senior Vice President and Chief Medical Officer, will present
recently announced
data from the company’s Expanded Access Program (EAP) evaluating INZ-701 in infants and children with ENPP1 Deficiency at the CHOP Cardiology Annual Meeting, held February 19-23, 2025, in Orlando, Florida.
Presentation Details:
Title:
Impact of the Enzyme Replacement Therapy, INZ-701, in Children with ENPP1 Deficiency: Experience from an Expanded Access Program
Presentation Number:
10 – Poster Session
Date:
Saturday, February 22, 2025
Time:
10:00 – 11:00am ET
Presenter:
Kurt Gunter, M.D., Senior Vice President and Chief Medical Officer
About ENPP1 Deficiency
ENPP1 Deficiency is a serious and progressive rare disease that affects blood vessels, soft tissues, and bones. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1), with about 50% of these infants not surviving beyond six months. Children with this condition typically develop autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults may develop osteomalacia, or softened bones. ARHR2 and osteomalacia cause pain and difficulty with movement. Additionally, patients may experience hearing loss, calcification in arteries and joints, and heart problems. ENPP1 Deficiency is an autosomal recessive disease and biallelic mutations are estimated to occur in approximately 1 in 64,000 pregnancies worldwide. Many individuals with just one copy of the mutated gene (monoallelic ENPP1 Deficiency) exhibit severe symptoms, suggesting that the worldwide prevalence of ENPP1 Deficiency is much higher than current estimates. Currently, there are no approved therapies for ENPP1 Deficiency.
About Inozyme Pharma
Inozyme Pharma is a pioneering clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics for rare diseases that affect bone health and blood vessel function. We are experts in the PPi-Adenosine Pathway, where the ENPP1 enzyme generates inorganic pyrophosphate (PPi), which regulates mineralization, and adenosine, which controls intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels). Disruptions in this pathway impact the levels of these molecules, leading to severe musculoskeletal, cardiovascular, and neurological conditions, including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL).
Our 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 the treatment of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. By targeting the PPi-Adenosine Pathway, INZ-701 aims to correct pathological mineralization and intimal proliferation, addressing the significant morbidity and mortality in these devastating diseases.
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Contacts
Investors:
Inozyme Pharma
Stefan Riley, Senior Director of IR and Corporate Communications
(617) 461-2442
stefan.riley@inozyme.com
Media:
Biongage Communications
Todd Cooper
(617) 840-1637
todd@biongage.com
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