Immedica Pharma has announced the successful outcome from the Pegzilarginase Phase 3 Study, presented by its partner Aeglea BioTherapeutics.
“I am pleased to see that these study results give hope for improved treatment options for patients with the severe and debilitating disease arginase-1 deficiency. Immedica plans to submit an EMA-application in 2022 which, if approved, will mark an important milestone in Immedica’s ambition to bring new innovative medicines for the patient group within Urea Cycle Disorders,” says Anders Edvell, CEO, Immedica Pharma.
PEACE is the first placebo-controlled clinical trial ever conducted in Arginase 1 Deficiency (ARG1-D) and pegzilarginase is the first potential therapy to normalize the markedly elevated plasma arginine levels in these patients. Based on the results of this trial, Aeglea plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the first half of 2022. Additionally, Aeglea will work with Immedica Pharma AB to submit marketing authorization applications in those territories.
ARG1-D is a rare, progressive and debilitating disease characterized by high levels of the amino acid arginine. The disease manifestations include spasticity, developmental delay, intellectual disability and seizures. The functional disability and impact on daily life creates a significant burden for patients and caregivers. There are currently no FDA-approved treatments that address elevated arginine, the key driver of ARG1-D. Current standard of care includes severe dietary protein restriction and essential amino acid supplementation, which does not effectively or sustainably reduce high arginine levels.
“I would like to thank the patients and their caregivers, investigators and staff, and our employees for their contributions to the study. The dramatic reduction in plasma arginine levels and the positive trend in GMFM-E are very encouraging and represent an important step in our mission to bring a transformative therapy to this underserved patient community,” said Anthony G. Quinn, M.B., Ch.B., Ph.D., president and chief executive officer of Aeglea. “We believe that today’s announcement demonstrates validation of our scientific platform, overall pipeline and potential to address other rare metabolic diseases.”