Benzinga | May 3, 2021 08:37AM EDT

Aeglea BioTherapeutics Completes Patient Randomization For PEACE, Its Pivotal Phase 3 Clinical Trial Of Pegzilarginase For Treatment Of Arginase 1 Deficiency

Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics as innovative solutions for rare metabolic diseases, today announced that it has completed patient randomization for PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints), the pivotal Phase 3 clinical trial investigating pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D). ARG1-D is a rare, progressive disease characterized by high levels of arginine which results in severe mobility limitations due to spasticity, seizures, developmental delay, intellectual disability and early mortality. Topline data from PEACE are expected in the fourth quarter of 2021.

"Completing randomization in our pivotal Phase 3 PEACE study is an important milestone given the challenges our investigators and patient families have faced with COVID-19. Exceeding our enrollment target of 30 patients with 32 randomized during a global pandemic demonstrates the high levels of enthusiasm in the ARG1-D patient and physician community and underscores the significant need for effective treatment options for this patient population," said Anthony G. Quinn, M.B. Ch.B., Ph.D., president and chief executive officer of Aeglea. "Pegzilarginase has the potential to be the first FDA-approved therapy to address the driver of the disease, persistently high levels of arginine, and to improve the quality of life for ARG1-D patients and their families."

Pegzilarginase is a novel, recombinant human arginase 1 enzyme that has been shown to lower toxic levels of the amino acid arginine in patients with ARG1-D. PEACE is a global, randomized, double-blind trial designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks. The trial enrolled 32 ARG1-D patients aged two years and older, who had plasma arginine levels greater than 250 ?M and a baseline deficit in at least one clinical response assessment. The primary endpoint is assessment of plasma arginine reduction from baseline and the key secondary endpoints include the 2 Minute Walk Test and Gross Motor Function Measure Part E as assessments of clinically meaningful effects, in addition to safety and pharmacokinetics. Previous studies have demonstrated that plasma arginine control has the potential to improve the clinical status and to slow disease progression in patients with ARG1-D.

For more information on the PEACE study, please visit http://clinicaltrials.gov.