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27/10/2008 - BioMarin

BioMarin Initiates Phase 1 Clinical Study of PEG-PAL in PKU

The Phase 1 clinical trial is an open-label, multi-center study to be conducted in approximately 35 PKU patients in a series of seven dose- escalating cohorts ranging from 0.001 to 1.0 mg-kg, with each cohort receiving a single dose, and a 6-week follow-up period


NOVATO, Calif., May 20 -PRNewswire-FirstCall- -- BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced that the first patient has initiated treatment in the Phase 1 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). The study is expected to conclude enrollment in the fourth quarter of 2008.

┬┤┬┤We are dedicated to serving the PKU community and hope to address the entire spectrum of PKU patients between PEG-PAL and Kuvan. We believe PEG-PAL holds tremendous potential to bring blood Phe down to normal levels and may help patients who either do not respond to Kuvan or who wish to reduce blood Phe levels beyond what is possible with Kuvan,┬┤┬┤ said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. ┬┤┬┤Positive preclinical data shows sustained decreases in blood Phe levels in PKU mice without any notable safety concerns and we hope to see the same results in humans.┬┤┬┤

The Phase 1 clinical trial is an open-label, multi-center study to be conducted in approximately 35 PKU patients in a series of seven dose- escalating cohorts ranging from 0.001 to 1.0 mg-kg, with each cohort receiving a single dose, and a 6-week follow-up period.

The primary objective of the study is to assess the safety and tolerability of single, subcutaneous injections of PEG-PAL in subjects with PKU. The secondary objectives of the study are to evaluate the pharmacokinetics of single, subcutaneous injections of PEG-PAL administered at escalating doses and to evaluate the effect of PEG-PAL on blood Phe concentrations in subjects with PKU.

About PEG-PAL

PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) is an investigational enzyme substitution therapy for the treatment of PKU. Pharmacology studies conducted in the PKU mouse model demonstrated that weekly subcutaneous administrations of PEG-PAL resulted in a significant and stable decrease of plasma phenylalanine. BioMarin estimates that PEG-PAL could be a potential treatment option for a significant portion of the PKU population.


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