"We are very encouraged by these preliminary findings, especially given the safety concerns as we entered the Phase 2 trial," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "Assuming the continuation of positive safety and efficacy trends in this ongoing study, we believe these data could support advancement to a Phase 3 trial at the end of 2011 with blood Phe level reductions as a primary endpoint for product registration. Getting to this stage has involved a tremendous amount of work by both the BioMarin team and our various collaborators. As with any program, we developed and tested many candidates before settling on PEG-PAL. Among the collaborators that helped us in this process we would like to particularly thank the Stevens laboratory at The Scripps Research Institute, Scriver laboratory at McGill University, and the local and national PKU communities."

The Phase 2 clinical trial is an open-label, multi-center study to be conducted in a series of dose-escalating cohorts from 0.001 mg/kg.  The primary treatment period of eight once weekly injections at a fixed dose will be followed by dose and frequency optimization and an extension period where doses can be increased up to 2.0 mg/kg/week.

The primary objective is to evaluate the effect of PEG-PAL on blood Phe concentrations in subjects with PKU.  The secondary objectives are to evaluate the safety and tolerability, immune response and steady state pharmacokinetics of subcutaneous injections of multiple dose levels of PEG-PAL.

SOURCE BioMarin Pharmaceutical Inc.