Inozyme announces ‘positive’ results from Phase 1/2 trials of INZ-701

Inozyme Pharma announced positive topline safety, pharmacokinetic, pharmacodynamic and exploratory efficacy data from the company’s ongoing Phase 1/2 clinical trials of INZ-701 in adults with ABCC6 Deficiency and ENPP1 Deficiency. “We are excited by the excellent safety and preliminary efficacy profile of INZ-701 in adults with ABCC6 Deficiency,” said Douglas Treco, Ph.D., CEO of Inozyme Pharma. “Our investigations into the natural history of this disease have identified a substantial and previously overlooked pediatric population with a high risk of stroke. We believe these patients represent a critical unmet need in this genetic disease and that changes observed in adults treated with INZ-701 will translate to clinical benefits in a future trial in children.” “The high risk of ischemic stroke in pediatric patients with ABCC6 Deficiency and its devastating consequences represents a serious unmet need in this population,” commented Professor Zulf Mughal, M.D., Consultant in Paediatric Bone Disorders at Al Jalila Children’s Specialty Hospital, Dubai, UAE. “I am very encouraged to see that INZ-701 may improve vascular pathology and believe that this effect may translate to clinical benefits in patients of all ages.”