Inozyme Pharma Announces Positive Interim Data from Ongoing Phase 1/2 Trials of INZ-701 in Adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE)

- Data from ongoing trial suggest clinical benefit for ENPP1 Deficiency, including improvement in key biomarkers, patient reported outcomes (PROs) and functional outcomes -

- Improvement in the Global Impression of Change (GIC) observed in all three dose cohorts in ABCC6 Deficiency (PXE) trial -

- INZ-701 was generally well tolerated and exhibited a favorable safety and immunogenicity profile in both trials -

- Company to host conference call and webcast today at 8:00 a.m. ET -

BOSTON, Sept. 26, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or the “Company”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced positive interim safety, pharmacokinetic (PK), pharmacodynamic (PD) and exploratory efficacy data from the Company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency (PXE, pseudoxanthoma elasticum).

"We are pleased that these trials have achieved their primary goals of demonstrating that INZ-701 was generally safe and well tolerated and meaningfully increased PPi levels in patients with ENPP1 Deficiency and ABCC6 Deficiency. We have also observed trends of clinical improvement which gives us confidence that we can design an informative pivotal trial in adults with ENPP1 Deficiency,” said Kurt Gunter, M.D., senior vice president and chief medical officer of Inozyme Pharma. “The trends towards improvement in clinically meaningful markers of bone metabolism in adults with ENPP1 Deficiency point to the potential for INZ-701 to treat rickets in our planned pivotal trial in pediatric patients with the condition.”

“Data from these first-in-human trials of INZ-701 are highly encouraging for both patients and physicians in the ENPP1 Deficiency and ABCC6 Deficiency communities. I am particularly excited to see early signs that INZ-701 may address symptoms in the adult population of ENPP1 Deficiency, which could translate to clinical benefits in the infant and adolescent patients who are in urgent need of a therapeutic option,” said Michael Levine, M.D., Professor Emeritus, Pediatrics and Medicine and Chief Emeritus, Division of Endocrinology and Diabetes at the Center for Bone Health at the Children’s Hospital of Philadelphia Research Institute.