- INZ-701 was well-tolerated and significantly increased plasma pyrophosphate (PPi) levels in patients with end-stage kidney disease (ESKD) undergoing hemodialysis; low PPi levels are associated with calciphylaxis, a rare, life-threatening complication of ESKD -

- Company plans to initiate a registrational trial in calciphylaxis in 2025 subject to regulatory alignment and sufficient funding -

BOSTON, Oct. 24, 2024 (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 positive interim data from its ongoing Phase 1 SEAPORT 1 trial of INZ-701 in patients with end-stage kidney disease (ESKD) undergoing hemodialysis (NCT06283589). These data will be presented at the American Society of Nephrology's (ASN) Kidney Week 2024, which is being held October 24-27, 2024, in San Diego.

"The interim data from the SEAPORT 1 trial demonstrate that INZ-701 significantly raised PPi levels in patients with end-stage kidney disease and was well-tolerated in this high-risk population,” said Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme Pharma. "Low PPi levels are linked to the development of calciphylaxis, a rare and life-threatening complication of end-stage kidney disease, as well as the associated morbidity and mortality. Pending alignment with regulatory authorities and sufficient funding, these encouraging results provide a strong foundation for advancing INZ-701 into a registrational trial in calciphylaxis.”

"Calciphylaxis represents one of the most challenging complications of end-stage kidney disease, with profound impacts on patient quality of life and survival. The interim results from the SEAPORT 1 trial are encouraging, showing that INZ-701 elevated PPi levels in patients with end-stage kidney disease. PPi levels are critically deficient in patients with calciphylaxis and by addressing this underlying deficiency, INZ-701 has the potential to modify the course of this disease and offer hope where no approved therapies currently exist,” added Sagar Nigwekar, MD, MMSc, Co-Director, Kidney Research Center, Massachusetts General Hospital.

Key Findings from the Interim Analysis of SEAPORT 1

The open-label trial investigated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of INZ-701 in adults with ESKD and low PPi levels undergoing hemodialysis. On average, ESKD patients have lower PPi levels compared with healthy individuals, and those with calciphylaxis have even lower plasma PPi levels. The trial screened hemodialysis patients for PPi levels and identified the population with significantly reduced levels (