- Aeglea BioTherapeutics (AGLE -4.4%) announces positive preliminary data from a Phase 1/2 open-label clinical trial evaluating AEB1102 (pegzilarginase) in patients with Arginase 1 deficiency, a rare inherited disorder characterized by the complete or partial absence of an enzyme called arginase which leads to the accumulation of ammonia in the blood. The results were presented at the Society for Inherited Metabolic Disorders Annual Meeting in San Diego, CA.
- Repeat dose data from two adult patients and ascending dose data from one pediatric patient showed sustained lowering of plasma arginine with repeated weekly IV doses of pegzilarginase. Also, elevated levels of arginine-derived metabolites at baseline were rapidly decreased and reductions maintained.
- Pegzilarginase was well-tolerated in the adults but the pediatric patient experienced an infusion-related reaction and withdrew due to time constraints between school and the study.
- The company expects to report adult and pediatric repeat dose data in Q3.
- Pegzilarginase is an arginine degrader derived from human Arginase 1. The company says it has a potential immunogenicity advantage over microbial enzymes because its human origin makes it less likely to provoke an immune response when administered.
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