Alexion Reports Positive SBC-103 Phase 1/2 Data - Quick Facts

(RTTNews) - Alexion Pharmaceuticals, Inc. (ALXN) said its researchers presented new data from an ongoing open-label, Phase 1/2 trial of intravenous SBC-103, an investigational enzyme replacement therapy, in children with mucopolysaccharidosis IIIB, a genetic rare lysosomal storage disease.The preliminary evidence, based on brain scans and neurocognitive assessments at 24 weeks, showed the potential for disease stabilization in patients with mucopolysaccharidosis IIIB treated with SBC-103.

The company said, during 24 weeks of treatment with SBC-103 at the highest dose of 3 mg/kg, most adverse events were mild in severity and no patient discontinued the study.

Martin Mackay, Executive Vice President and Global Head of R&D at Alexion, said: "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."