BioMarin Pharmaceutical Inc.
) recently announced preliminary results from a phase II program of
PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase).
PEG-PAL is an investigational enzyme substitution therapy which is
being developed to treat phenylketonuria (PKU).
The phase II program consisted of four primary studies involving
a total of 56 patients, above 16 years of age. Preliminary data
from the program showed that PEG-PAL was well tolerated and could
prove to be efficacious. Although two patients dropped out of the
study due to adverse reactions, BioMarin said that the cases were
not severe. Twelve more dropped out due to logistical reasons.
BioMarin expects to meet the US Food and Drug Administration
(FDA) to review data from the phase II program in the first quarter
of 2013. The company plans to initiate a phase III study of PEG-PAL
in the second quarter of 2013 after the end of phase II discussion
with the US regulatory body.
Primarily, the phase III program of PEG-PAL will include a study
for the evaluation of safety and blood phenylalanine (Phe) levels
in patients suffering from PKU.
BioMarin already markets Kuvan around the world which is
indicated to treat PKU. BioMarin recorded net product revenue of
$34.7 million from Kuvan in the second quarter of 2012. The company
expects to generate around $130-$140 million as net sales of the
product at the end of 2012.
We note that BioMarin has a partnership with Merck Serono, a
), since 2005 for the development and commercialization of both
Kuvan and PEG-PAL. The agreement was later revised in 2007.
We have a Neutral recommendation on BioMarin, which carries a
Zacks #3 Rank (Hold) in the short run. The company has multiple
data readouts in the near future. We expect investor focus to stay
on these results.
BIOMARIN PHARMA (BMRN): Free Stock Analysis
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