NEW HAVEN, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) announced today that researchers presented new data from an ongoing open-label, Phase 1/2 trial of intravenous (IV) 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 (MRI) and neurocognitive assessments at 24 weeks, showed the potential for disease stabilization in patients with MPS IIIB treated with SBC-103. These data were presented at the 14th International Symposium on MPS and Related Diseases in Bonn, Germany.
MPS IIIB is caused by genetic mutations that result in a marked decrease in N-acetyl-α-D-glucosaminidase (NAGLU) enzyme activity, leading to abnormal accumulation of heparan sulfate (HS) in the brain and other organs, as well as progressive brain atrophy with cortical gray matter (CGM) volume loss. This results in severe neurocognitive decline, behavioral disturbances, speech loss, increasing loss of mobility, and premature death. At present there is no treatment for this disorder. MPS IIIB typically presents in children during the first few years of life, and these children have a greater than 50 percent mortality rate by 17 years of age.
“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.”
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