ARTICLE | Clinical News

Abeona reports top-line Phase I/II data for ABO-102 in mucopolysaccharidosis IIIA

December 1, 2017 9:53 PM UTC

In October, Abeona Therapeutics Inc. (NASDAQ:ABEO) reported top-line data from the first cohort of 3 patients with mucopolysaccharidosis IIIA (MPS IIIA, Sanfilippo type A syndrome) in a Phase I/II trial showing that IV ABO-102 (AAV-SGSH) reduced cerebral spinal fluid (CSF) heparan sulfate levels by 69.3% at 1 year post-injection (p<0.001). Additionally, ABO-102 normalized liver volumes by 80% (p<0.005), stabilized deep brain architecture in the thalamus and putamen, as measured by MRI (p<0.05), and stabilized Leiter-R non-verbal IQ and Vineland Adaptive Behavior Scale scores at 1 year post-injection. Data were presented at the Alliance for Regenerative Medicine’s Cell & Gene meeting in La Jolla.

The open-label, international trial is evaluating safety as its primary endpoint. Secondary endpoints include increase in CSF and blood leukocyte SGSH enzyme activity levels, reduced liver and spleen volumes as measured by MRI, improved adaptive functioning or arrest of decline in adaptive functioning as measured by the Vineland Adaptive Behavior Scale, improved cognitive ability or arrest of cognitive deterioration as measured by the Leiter International Performance scale and the Mullen Scales of Early Learning, and reduction of urine glycosaminoglycans or heparan sulfate...