Aeglea BioTherapeutics, Inc., a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics to benefit people with rare metabolic diseases, shared additional data from the PEACE Phase 3 study during a poster presentation at the Society for Inherited Metabolic Disorders (SIMD) Annual Meeting being held in Orlando, FL from April 10-13. The poster, titled Pegzilarginase in Arginase 1 Deficiency: Results of the PEACE Pivotal Phase 3 Clinical Trial (Abstract #30), will also be available on the publications section of Aeglea’s website and includes new data on patient-level outcomes, additional secondary endpoints and previously announced topline results.
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Pegzilarginase is a novel, recombinant human arginase 1 enzyme that has been shown in clinical trials to normalize the elevated levels of the amino acid arginine in patients with Arginase 1 Deficiency (ARG1-D), a rare, progressive disease characterized by high levels of arginine. People living with ARG1-D experience severe spasticity-related mobility limitations, seizures, developmental delay, intellectual disability and early mortality.
“Our clinical development program for pegzilarginase has provided the first quantitative insights into the disease burden of ARG1-D and sheds new light on both the severity of the disease and the devastating effect of uncontrolled arginine levels in these patients,” said Anthony G. Quinn, M.B., Ch.B., Ph.D., president and chief executive officer of Aeglea. “The data from PEACE show the ability of pegzilarginase to markedly improve arginine control and its impact on a broad range of disease-related abnormalities. We are pleased with these results and believe pegzilarginase has the potential to change the lives of the patients and families living with ARG1-D.”
Results from the PEACE Phase 3 Study
PEACE is a global, randomized, double-blind, placebo-controlled trial that enrolled 32 patients with ARG1-D aged two years and older. The study was designed to assess the effects of treatment with pegzilarginase (n=21) versus placebo (n=11) from baseline through a prespecified 24-week treatment period.