Treatment Emergent Serious Adverse Events

Full Analysis Set

ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints

Measurement is actual plasma concentration of ORGN001 at measured timepoints, starting at their current rcPMP dose. 6 patients started at a dose of 240 mcg/kg, 1 patient at 248 mcg/kg, and 1 patient at 280 mcg/kg. (EOI = End of Infusion). 1 patient is missing a pre-infusion dose measurement.

Analyses were performed on urine SSC, a biomarker of the MoCD pathway. Levels of SSC measured in urine were normalized to urine creatinine levels. The observed value, change, and percent change in urine and blood SSC levels from baseline were summarized by visit over time.

Not all patients had samples taken at each expected timepoint.

Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam.

All patients entering the study had complete examinations throughout the study to identify Normal vs Abnormal Neurologic Function on the parameters presented. Date shown here are from Baseline and final examination where all 8 patients have data.

Change from baseline in Seizure frequency

Number of patients with Seizures in observation period