BioMarin posts strong early-stage results for CLN2 disease drug candidate

Rare disease drug developer BioMarin Pharmaceutical (Nasdaq: BMRN) late yesterday announced positive 48-week results from its Phase I/II pivotal study for cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1) to treat children with CLN2 disease, a form of Batten disease.

The news pushed BioMarin’s shares up 2.5% to $89.66 in morning trading today.

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).