Alexion Pharmaceuticals reports that researchers have presented new data from an ongoing open-label, Phase 1/2 trial of intravenous (SBC-103 (rhNAGLU enzyme), an investigational enzyme replacement therapy, in children with mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo syndrome type B), a genetic, progressive, and devastating rare lysosomal storage disease.

Preliminary evidence, based on brain scans and neurocognitive assessments at 24 weeks, showed the potential for disease stabilization in patients with MPS IIIB treated with SBC-103.

“MPS IIIB is a devastating and life-threatening disorder, with no available treatments, and has a severe and progressive impact on the cognitive function of children suffering with the disease,” said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion.

“These new data presented today suggest the potential of SBC-103 to cross the blood-brain barrier when administered intravenously and provide preliminary evidence of potential dose-dependent disease stabilization at 24 weeks in children with MPS IIIB.”