Anthony Quinn, PhD, President and CEO of Aeglea BioTherapeutics, discusses the PEACE study, a phase 3 clinical trial of pegzilarginase for the treatment of arginase 1 (ARG1) deficiency.
ARG1 deficiency is a rare genetic disorder characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells. Arginase is one of six enzymes that play a role in the urea cycle. The lack of the arginase enzyme results in hyperargininemia in the blood and cerebrospinal fluid, and can lead to hyperammonemia in the blood as well. Children most often experience spasticity (especially in the lower limbs) but can also experience seizures, short stature, and intellectual disability.
As Dr. Quinn explains, the PEACE study is a pivotal, double-blind, placebo-controlled trial of pegzilarginase, an investigational enzyme replacement therapy for patients with ARG1 deficiency. The study duration is 6 months and patients are randomized during this time 2:1 to receive either pegzilarginase or placebo. After 6 months, patients who received placebo are offered to receive pegzilarginase. The primary endpoint of the study is plasma-arginine reduction. Mobility assessments will also be performed to further demonstrate the connection between plasma-arginine reduction and clinical benefit.
Baseline patient characteristic data from this study was recently presented at International Congress of Inborn Errors of Metabolism (ICIEM) 2021. It was reported that the demographic and disease features of the ARG1 deficiency patients enrolled in the PEACE study at baseline are generally consistent with insights from the previous Phase 1/2 cohort and published case series. Specifically, patients exhibited markedly elevated plasma arginine (despite treatment with standard of care), spasticity, seizures, and mobility deficits. Topline data from the PEACE study is expected next month, in December 2021.
To learn more about ARG1 deficiency and other rare metabolic disorders, visit https://checkrare.com/diseases/metabolic-disorders/
