Aeglea BioTherapeutics Announces Design of Pivotal Phase 3 PEACE Trial Evaluating Pegzilarginase in Arginase 1 Deficiency

Aeglea BioTherapeutics (NASDAQ:AGLE), a clinical-stage biotechnology company that designs and develops innovative human enzyme therapeutics for patients with rare genetic diseases and cancer, today announced the design of its global pivotal Phase 3 PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints) trial to evaluate the safety and efficacy of pegzilarginase, the Company’s lead investigational therapy, in patients with Arginase 1 Deficiency (ARG1-D).

As quoted in the press release:

The Company has aligned the trial design and endpoints with input from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and plans to conduct a single, global pivotal trial to support registration. Aeglea expects to dose the first patient in the PEACE trial in the second quarter of 2019 and expects topline data will be available in the first quarter of 2021.

PEACE is a global, randomized, double-blind trial designed to assess the effects of treatment with pegzilarginase versus placebo over 24 weeks with a primary endpoint of statistically significant plasma arginine reduction from baseline. The primary endpoint is intended to assess the effectiveness of pegzilarginase in lowering plasma arginine levels given the evidence that improved plasma arginine control has the potential to improve the clinical status and slow disease progression in patients with ARG1-D. Secondary endpoints assessing changes in clinically meaningful outcomes including mobility, adaptive behavior, safety and pharmacokinetics will be used to describe the broader impact of pegzilarginase relative to placebo on multiple aspects of ARG1-D.

“Arginine is the key driver of this devastating disease and pegzilarginase is the first ever approach that has demonstrated substantial lowering of plasma arginine levels,” said Anthony G. Quinn, M.B Ch.B, Ph.D., president and chief executive officer of Aeglea. “Finalization of our pivotal trial protocol represents an important advance for ARG1-D patients who lack effective treatment options. Our expectation is that the data from the PEACE trial could be sufficient to support marketing applications for pegzilarginase in ARG1-D.”

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