SAN RAFAEL, Calif., March 02, 2016 (GLOBE NEWSWIRE) — BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced positive 48-week results from its Phase 1/2 pivotal study for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children with CLN2 disease, a form of Batten disease. CLN2 disease is a rapidly progressing, fatal neurodegenerative disease with no approved treatments, where the majority of affected children lose their ability to walk and talk by approximately six years of age. The average rate of clinical decline for motor and language function in patients receiving cerliponase alfa treatment — the primary efficacy endpoint — was approximately 80% less than the expected rate of decline in the untreated population, preserving essential function in the majority of treated patients (p <0.0001). Treatment with 300 mg cerliponase alfa administered via intracerebroventricular (ICV) infusion every other week was generally safe and well-tolerated in 24 patients and resulted in disease stabilization in 65% (15 of 23) of patients treated over a 48-week period, based on the Hamburg Motor + Language CLN2 rating. BioMarin estimates the incidence of CLN2 disease is approximately one in 200,000 with approximately 1,200 to 1,600 children in BioMarin’s commercial territories. Full article