Inozyme to Present Phase 1/2 INZ-701 Data for ENPP1 and PXE at Medical Conferences

Inozyme Pharma Inc., a clinical-stage company listed on the Nasdaq under the ticker INZY, has announced that it will be presenting new data from its Phase 1/2 clinical trials of INZ-701 at two significant medical conferences. INZ-701 is being developed for treating ENPP1 Deficiency and ABCC6 Deficiency, conditions resulting in pathologic mineralization and intimal proliferation.

The first presentation will take place at the European Calcified Tissue Society Congress (ECTS) 2024, scheduled from May 25-28 in Marseille, France. Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer at Inozyme, will present two oral abstracts on May 26. The first session, entitled "Impact of INZ-701 on Bone and Mineral Metabolism Biomarkers and Clinical Outcomes in Adults with ENPP1 Deficiency," will discuss the results from a 48-week Phase 1/2 open-label study. The second session, "Safety and Exploratory Efficacy of INZ-701 in Adults with ABCC6 Deficiency Manifesting as Pseudoxanthoma Elasticum," will also disclose findings from a 48-week Phase 1/2 open-label study.

The second presentation will be held during the Endocrine Society’s Annual Meeting (ENDO) 2024, which takes place from June 1-4 in Boston, Massachusetts. Kurt Gunter, M.D., Senior Vice President and Chief Medical Officer, will share the presentation titled "Impact of INZ-701 on Bone and Mineral Metabolism Biomarkers and Clinical Outcomes in Adults with ENPP1 Deficiency," summarizing the 48-week Phase 1/2 open-label study results.

ENPP1 Deficiency is an inherited condition that severely impacts the vasculature, soft tissue, and skeleton, with a prevalence of roughly 1 in 64,000 pregnancies globally. It can cause severe complications ranging from generalized arterial calcification of infancy (GACI Type 1) and autosomal-recessive hypophosphatemic rickets type 2 (ARHR2) in children to osteomalacia in adults, which leads to pain and mobility issues. There are no approved therapies currently available for this debilitating condition.

The Phase 1/2 clinical trial for ENPP1 Deficiency enrolled nine adult patients across North America and Europe. The primary objectives were to assess the safety and tolerability of INZ-701, as well as to determine its pharmacokinetic (PK) and pharmacodynamic (PD) profiles. Initial dosing ranged from 0.2 mg/kg to 1.8 mg/kg over 32 days, administered subcutaneously twice a week. Following dose optimization, a fourth cohort received 1.2 mg/kg once a week. The open-label Phase 2 extension is designed to evaluate long-term safety and efficacy over a 48-week period, with a focus on skeletal, vascular, and physical function outcomes.

ABCC6 Deficiency, affecting about 1 in 25,000 to 1 in 50,000 individuals worldwide, also has severe vascular and soft tissue implications. Infants with this deficiency often present with GACI Type 2, similar to ENPP1 Deficiency, and may develop serious conditions such as stroke or cardiovascular diseases as they age. Pseudoxanthoma elasticum (PXE) is the characteristic presentation in older individuals.

For ABCC6 Deficiency, the Phase 1/2 trial enrolled ten adult patients in the US and Europe. The study mirrors the ENPP1 trial with regard to safety, tolerability, and PK/PD profiles, using the same dosing strategy. Long-term evaluations in the Phase 2 extension include assessments of vascular and ophthalmologic health, as well as patient-reported outcomes.

INZ-701 is a recombinant Fc fusion protein designed as an enzyme replacement therapy. It aims to manage conditions related to vascular, soft tissue, and skeletal pathologies by metabolizing adenosine triphosphate (ATP) to generate inorganic pyrophosphate (PPi) and adenosine monophosphate (AMP), thus preventing abnormal mineralization and intimal proliferation. Preclinical and early clinical data suggest that INZ-701 is well tolerated and efficacious in increasing PPi levels.

Inozyme Pharma continues its efforts to address severe rare diseases with no current treatment options, focusing on INZ-701's potential to mitigate the life-altering impacts of ENPP1 and ABCC6 deficiencies.