Sanfilippo primarily affects the central nervous system and causes deterioration of neurological function.
Abeona Therapeutics traded Monday at $1.38, up 6%. The stock has slumped 33% in the last six months.
The company released magnetic resonance imaging MRI data from the Phase 1/2 Transpher A clinical study.
The study “indicated that ABO-102 increased grey matter, corpus callosum and amygdala volumes in the brain in three young patients with Sanfilippo Syndrome Type A at 24 months as compared to afflicted patients without treatment,” Abeona Therapeutics said in a statement.
“Brain volume loss is characteristic in children with MPS IIIA and is associated with long-term cognitive and physical disability,” said Vishwas Seshadri, head of research & clinical Development at Abeona Therapeutics.
“Specifically, grey matter is important for cognitive development, corpus callosum for motor function, and amygdala for fear learning as well as social/emotional development,” Seshadri added.
“The new MRI data shows the potential of ABO-102 to increase brain grey matter, corpus callosum and amygdala volumes and is consistent with previously reported results of preservation of neurocognitive development in these three young patients in the Transpher A study,” Seshadri said.