Results from a radiographic study describing skeletal features of pediatric patients with ENPP1 Deficiency will be presented
BOSTON, June 21, 2024 (GLOBE NEWSWIRE) — Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced a data presentation and sponsored symposium at the 11th International Conference on Children’s Bone Health (ICCBH), which is being held June 22-25, 2024, in Salzburg, Austria.
Presentation Details
Title: Expanding the spectrum of radiographic features in children with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency: Novel skeletal findings from an international collaboration
Format: Oral Presentation – Session 3
Date: Monday, June 24, 2024
Session Time: 11:00 – 12:00 CEST / 5:00 am – 6:00 am ET
Presenting Author: Leanne Ward, M.D., Professor of Pediatrics in the Faculty of Medicine, University of Ottawa
Symposium Details
Title: Recognizing ENPP1 Deficiency – An overlooked cause of hypophosphatemic rickets
Date: Saturday, June 22, 2024
Session Time: 13:45 – 14:45 CEST / 7:45 am – 8:45 am ET
Chair:
- Agnes Linglart, M.D., Professor of Paediatrics at the Bicetre Paris Saclay University and Hospital, and National Coordinator of the Centre of Reference for Rare Disorders of Calcium and Phosphate Metabolism and the Network for Rare Bone Diseases at OSCAR
Presenters:
- Zulf Mughal, M.D., Consultant in Paediatric Bone Disorders at Al Jalila Children’s Specialty Hospital, Dubai, UAE
- David Weber, M.D., MSCE, Attending Physician and Medical Director of the Center for Bone Health at Children’s Hospital of Philadelphia
About ENPP1 Deficiency
ENPP1 Deficiency is a progressively debilitating condition of the vasculature, soft tissue, and skeleton with a prevalence of approximately 1 in 64,000 pregnancies worldwide. Although ENPP1 Deficiency was initially described in patients with biallelic ENPP1 Deficiency (homozygous or compound heterozygous mutations), many patients with monoallelic ENPP1 Deficiency (heterozygous mutations) have clinical symptoms, potentially increasing the worldwide prevalence. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1) and approximately 50% of infants die within six months of birth. Children with ENPP1 Deficiency typically develop rickets, a condition diagnosed as autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults can develop osteomalacia (softened bones). ARHR2 and osteomalacia lead to pain and mobility issues. Patients can also exhibit signs and symptoms of hearing loss, arterial and joint calcification, and cardiovascular complications. There are no approved therapies for ENPP1 Deficiency.
About INZ-701
INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton. In preclinical studies, the experimental therapy has shown potential to prevent pathologic mineralization and intimal proliferation, which can drive morbidity and mortality in devastating genetic disorders such as ENPP1 Deficiency, ABCC6 Deficiency and calciphylaxis. INZ-701 is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency and calciphylaxis.
About Inozyme Pharma
Inozyme Pharma, Inc. is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases impacting the vasculature, soft tissue, and skeleton. Inozyme is developing INZ-701, an enzyme replacement therapy, to address pathologic mineralization and intimal proliferation, which can drive morbidity and mortality in these severe diseases. INZ-701 is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency and calciphylaxis.
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
(857) 330-8871
stefan.riley@inozyme.com
Media:
SmithSolve
Matt Pera
(973) 886-9150
matt.pera@smithsolve.com
