BioMarin Pharmaceutical Inc has halted its Phase 3a study of Neutralase for the reversal of anticoagulation by heparin in primary Coronary Artery Bypass Graft (CABG) surgery and that it has terminated its Neutralase program for all indications.
The decision to halt the Phase 3 study resulted from a recommendation from an independent Data Safety Monitoring Board (DSMB) and was based on a review of data from enrolled patients, which indicated with high probability that Neutralase would not demonstrate favorable safety and efficacy. Given the expected risk/benefit profile for Neutralase, BioMarin has decided to stop development of the drug for all indications.
BioMarin's portfolio includes product candidates for genetic diseases, including Aryplase for mucopolysaccharidosis VI that is in a Phase 3 clinical trial expected to be completed at the end of the first quarter of 2004, and a product candidate for Phenylketonuria (PKU) that the company expects to advance into clinical-stage development in 2004.
Vibrilase, an enzyme product candidate for treatment of serious burns, is nearing completion of a Phase 1b study, and NeuroTrans is in preclinical testing as a method to deliver both enzymes and other therapeutic molecules to the brain via traditional intravenous infusion. Aldurazyme for mucopolysaccharidosis I is approved in both the United States and European Union. Aldurazyme is manufactured by BioMarin and is marketed by BioMarin's joint venture partner, Genzyme Corporation.