Clinical Data 80% plasma arginine reduction (primary endpoint; p < 0.0001) accompanied by a positive trend in GMFM-E, a key clinical assessment of patient mobility
90.5% of patients achieved normal plasma arginine levels
Pegzilarginase was well-tolerated; no discontinuations due to adverse events
First potential therapy to address key driver of ARG1-D, a devastating ultra-rare disease
Plan to submit BLA in first half of 2022