Inozyme Pharma(INZY)2025-03-10 12:31Financial Position - The estimated cash, cash equivalents, and short-term investments for Inozyme Pharma, Inc. as of December 31, 2024, are approximately $113.1 million[4]. Clinical Trials and Results - Interim data from the ENERGY 1 trial showed that 80% of infants treated with INZ-701 survived beyond their first year, compared to a historical survival rate of approximately 50%[10]. - Substantial reductions or stabilization of arterial calcifications were observed in all surviving patients treated with INZ-701, with some instances of complete resolution[10]. - The ENERGY 3 pivotal trial has completed enrollment with 25 patients, providing over 90% power to detect meaningful differences in clinical endpoints[13]. - The planned ASPIRE pivotal trial in children with ABCC6 Deficiency is expected to enroll approximately 70 patients, with preliminary support from U.S. and EU regulators[15]. - The adult study of INZ-701 demonstrated positive improvements in vascular and retinal pathology after 48 weeks of treatment, supporting further development in pediatric populations[14]. - The Company anticipates completing the one-year dosing period for all patients in the ENERGY 3 trial by January 2026, with topline data expected in early 2026[13]. - INZ-701 was well-tolerated in infants and young children, with no serious treatment-related adverse events reported[10]. - The Company plans to continue regulatory engagement to finalize the ASPIRE trial protocol, aiming to initiate the trial in early 2026[16]. - The Company reported low, often transient, anti-drug antibody levels in some children and adults, with no impact on pharmacokinetics or pharmacodynamics[10].