BioMarin 'encouraged' by interim data from BMN 250 Phase 1/2 trial
BioMarin Pharmaceutical announced that it presented interim data from a Phase 1/2 trial for BMN 250, an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetylglucosaminidase with a peptide derived from insulin-like growth factor 2, for the treatment of Sanfilippo B syndrome or mucopolysaccharidosis IIIB at WORLDSymposium 2018. Discovered by BioMarin, BMN 250 is being studied in a multicenter, international clinical trial evaluating safety and tolerability, as well as cognitive function of patients with Sanfilippo B receiving BMN 250. Designed to restore functional NAGLU activity in the brain, BMN 250 is administered via intracerebroventricular infusion. "These studies are generating a robust data set to determine the safety and efficacy of BMN 250 in Sanfilippo B," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "While it is still very early, we are encouraged that the interim data is showing normalization of an important biomarker and liver size, as well as suggesting a stabilization in cognitive decline. We appreciate the support of the Sanfilippo community to study this experimental treatment, and we remain committed to contributing to the body of scientific knowledge of this devastating disease."