NOVATO, Calif., Sept. 22 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. announced today that the first patient has initiated treatment in the Phase 2 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). Initial top-line results are expected in mid-2010.
The Phase 2 clinical trial is an open-label, multi-center study to be conducted in up to 35 patients 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 eight weeks of dose and frequency optimization and an extension period where doses can be increased up to 2.0 mg/kg/week.
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.
About BioMarin
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BioMarin(R), Naglazyme(R) and Kuvan(R) are registered trademarks of BioMarin Pharmaceutical Inc.
SOURCE BioMarin Pharmaceutical Inc.
CONTACT: Investors, Eugenia Shen, +1-415-506-6570, or Media, Susan Berg,
+1-415-506-6594, both of BioMarin Pharmaceutical Inc.
Web site: http://www.bmrn.com/
