Tuesday, BioMarin Pharmaceutical Inc. (BMRN) said that the first patient has initiated treatment in the mid-stage clinical study of PEG-PAL for the treatment of phenylketonuria or PKU. Initial results from the study are expected in mid-2010.
Hank Fuchs, M.D., Chief Medical Officer of BioMarin said, "We remain optimistic on this program and believe that, if approved, PEG-PAL may offer a significant benefit for many PKU patients, especially those who do not respond adequately to Kuvan." Kuvan is the first and only prescription medication to lower blood Phe levels.
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.
The primary objective of the study 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.
BMRN closed Tuesday's regular trading at $17.83 down $0.33 or 1.82% on a volume of 1.90 million shares on the Nasdaq.
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