Positive Results From Clinical Trial Of PEG-PAL For Biomarin

BioMarin has declared results from the Phase-I clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), for the treatment of phenylketonuria (PKU). The company expects to initiate the phase-II clinical study in late June or early July. FDA has already reviewed the phase-II clinical trial protocol.

The phase-I clinical trial was an open-label, multi-center study conducted in 25 PKU patients in a series of five dose-escalating cohorts ranging from 0.001 to 0.1 mg/kg. Each cohort received a single dose, and a 6-week follow-up period.

Substantial blood Phe level reductions in the range of 36% to 97% (mean of 62%) were observed in all patients in the fifth dosing cohort (0.1 mg/kg) with a mean baseline blood Phe level of 1113 umol/L. No notable blood Phe level reductions were observed in the first four dosing cohorts (0.001 to 0.03 mg/kg).

Hank Fuchs, Chief Medical Officer, BioMarin, said: We are encouraged to see significant reductions in blood Phe levels in all patients in the fifth dosing cohort of the Phase 1 trial. Importantly, there were no serious reactions, and both the number and severity of the mild to moderate reactions were in-line with expectations of an enzyme of this nature.

The Phase 2 study will answer the critical questions of optimal dose, dosing regimen and tolerability, he added.