Inozyme Pharma’s Chief Medical Officer to Present Data on INZ-701 for ENPP1 Deficiency at CHOP Cardiology Annual Meeting
Boston, MA, February 21, 2025 – Inozyme Pharma, Inc., a pioneering biopharmaceutical company dedicated to the development of innovative therapeutics for rare diseases that impact bone health and blood vessel function, announced that Kurt Gunter, M.D., the Senior Vice President and Chief Medical Officer, will share findings from the company’s Expanded Access Program (EAP) of INZ-701 in infants and children with ENPP1 Deficiency at the Children’s Hospital of Philadelphia (CHOP) Cardiology Annual Meeting. This prestigious event took place from February 19-23, 2025, in Orlando, Florida.
Background on INZ-701 and ENPP1 Deficiency
INZ-701 is an investigational enzyme replacement therapy for ENPP1 Deficiency, a rare metabolic disorder that results in impaired bone mineralization and vascular calcification. ENPP1 Deficiency is caused by mutations in the ENPP1 gene, which results in a deficiency of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1), an enzyme essential for maintaining healthy bone and blood vessels. INZ-701 is designed to replace the missing ENPP1 enzyme and help restore normal bone health and blood vessel function.
Data from Inozyme Pharma’s Expanded Access Program
During the CHOP Cardiology Annual Meeting, Dr. Gunter will present data from Inozyme’s Expanded Access Program (EAP), which was initiated in 2023 to provide access to INZ-701 for patients with ENPP1 Deficiency who were not eligible for clinical trials. The EAP is designed to evaluate the safety, efficacy, and real-world effectiveness of INZ-701 in a larger and more diverse patient population. The data to be presented will include clinical, biochemical, and radiological assessments of infants and children treated with INZ-701.
Impact on Individuals with ENPP1 Deficiency
For individuals with ENPP1 Deficiency, the presentation of data from Inozyme’s EAP could provide valuable insights into the safety and efficacy of INZ-701 in treating their condition. This information could help inform decisions about treatment options and improve the overall understanding of ENPP1 Deficiency and its management. By sharing this data at a major medical conference, Inozyme Pharma is contributing to the scientific community’s knowledge of this rare disease and the potential benefits of INZ-701 as a therapeutic option.
Global Implications
Beyond the individual level, the presentation of data from Inozyme Pharma’s EAP at the CHOP Cardiology Annual Meeting could have broader implications for the medical community and the world at large. Rare diseases, such as ENPP1 Deficiency, often receive less attention and funding than more common conditions. However, they can significantly impact the lives of those affected and their families. By advancing the development of new treatments, such as INZ-701, the biopharmaceutical industry can help improve the lives of individuals with rare diseases and contribute to a more equitable healthcare system.
Conclusion
Inozyme Pharma’s announcement of data from its Expanded Access Program for INZ-701 in ENPP1 Deficiency marks an important step forward in the development of this investigational therapeutic. The presentation of this data at the CHOP Cardiology Annual Meeting will provide valuable insights for the medical community and contribute to the ongoing efforts to improve the lives of individuals with ENPP1 Deficiency. This research underscores the importance of continued investment in the development of treatments for rare diseases and the role that biopharmaceutical companies, like Inozyme Pharma, play in advancing medical knowledge and improving healthcare for all.
- Inozyme Pharma’s Chief Medical Officer, Kurt Gunter, M.D., presented data from the company’s Expanded Access Program (EAP) for INZ-701 in ENPP1 Deficiency at the CHOP Cardiology Annual Meeting.
- ENPP1 Deficiency is a rare metabolic disorder that results in impaired bone mineralization and vascular calcification.
- INZ-701 is an investigational enzyme replacement therapy designed to replace the missing ENPP1 enzyme and restore normal bone health and blood vessel function.
- The data presented at the CHOP Cardiology Annual Meeting will include clinical, biochemical, and radiological assessments of infants and children treated with INZ-701.
- The presentation could provide valuable insights for individuals with ENPP1 Deficiency and their families, contributing to improved understanding and treatment options.
- Beyond the individual level, the presentation could have broader implications for the medical community and the healthcare system, highlighting the importance of continued investment in the development of treatments for rare diseases.
