Inozyme Pharma to Present Recently Announced Interim Data for INZ-701 in Infants and Young Children with ENPP1 Deficiency at CHOP Cardiology 2025

Rhea-AI Summary

Inozyme Pharma (Nasdaq: INZY) has announced that their Chief Medical Officer, Dr. Kurt Gunter, will present new data regarding INZ-701 at the CHOP Cardiology 2025 conference in Orlando, Florida. The presentation will focus on results from their Expanded Access Program (EAP) evaluating INZ-701's impact on infants and children with ENPP1 Deficiency.

The presentation, titled 'Impact of the Enzyme Replacement Therapy, INZ-701, in Children with ENPP1 Deficiency: Experience from an Expanded Access Program,' is scheduled for Saturday, February 22, 2025, from 10:00 to 11:00am ET during the poster session. The conference runs from February 19-23, 2025.

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.

For more information, please visit https://www.inozyme.com/ or follow Inozyme on LinkedIn, X, and Facebook.

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

FAQ

What will Inozyme Pharma present at CHOP Cardiology 2025?

Inozyme Pharma will present interim data from their Expanded Access Program (EAP) evaluating INZ-701 in infants and children with ENPP1 Deficiency.

When is Inozyme's INZ-701 presentation at CHOP Cardiology 2025?

The presentation is scheduled for Saturday, February 22, 2025, from 10:00 to 11:00am ET during the poster session.

What is the focus of INZY's Expanded Access Program?

The EAP focuses on evaluating INZ-701, an enzyme replacement therapy, in infants and young children with ENPP1 Deficiency.

Who will present the INZ-701 data at CHOP Cardiology 2025?

Dr. Kurt Gunter, Senior Vice President and Chief Medical Officer of Inozyme Pharma, will present the data.

What is the therapeutic focus of Inozyme's INZ-701?

INZ-701 is being developed for rare diseases that affect bone health and blood vessel function, specifically targeting ENPP1 Deficiency.