PEGZILARGINASE
Pegzilarginase met the primary endpoint of arginine reduction in the PEACE Phase 3 study in patients with Arginase 1 Deficiency.
In our PEACE Phase 3 clinical trial, treatment with pegzilarginase was shown to markedly reduce blood arginine levels in patients with ARG1-D. Pronounced and sustained plasma arginine reduction was accompanied by a positive trend in the Gross Motor Function Measure Part E (GMFM-E), a key clinical assessment of a patient’s mobility. In the PEACE clinical trial, pegzilarginase was well-tolerated and safety data were consistent with results from previous clinical trials.
In data from a Phase 1/2 clinical trial and it’s open-label extension study, treatment with pegzilarginase was shown to rapidly and sustainably reduce blood arginine levels in patients with ARG1-D. The data also showed that pegzilarginase improved clinical outcomes, including mobility and motor function. In the Phase 1/2 clinical trial, pegzilarginase was well tolerated, with most treatment-related adverse events being mild and manageable with standard measures.
Pegzilarginase is investigational. Safety and efficacy have not been established by any agency.
In a Phase 3 pivotal clinical trial, treatment with pegzilarginase was shown to markedly reduce blood arginine levels in people with ARG1-D.

Pegzilarginase has received several regulatory designations, including Fast Track, Breakthrough, Orphan Drug and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration as well as Orphan Drug Designation from the European Medicines Agency.
Advancing Multiple Development Programs
