BioMarin Pharmaceutical Inc.
) recently announced positive results from the PKU-016 ASCEND
study. This is the largest randomized controlled study evaluating
the safety and therapeutic effects of Kuvan (sapropterin
dihydrochloride) on neuropsychiatric symptoms in subjects with
We note that Kuvan is already approved for the treatment of
patients suffering from PKU. BioMarin has a partnership with
Merck Serono, a division of
), for the development and commercialization of Kuvan since
PKU-016 is a randomized, placebo-controlled, 13-week outcomes
study in patients who were treated with Kuvan. The study
evaluated medically important symptoms similar to attention
deficit hyperactivity disorder (ADHD) in patients suffering from
PKU and whose blood levels of phenylalanine (Phe) were reduced by
The study results showed that Kuvan improved the attention
deficit hyperactivity rating scale (ADHD-RS) driven by a
statistically significant change in the inattention component of
the score. BioMarin now plans to submit data from the study to
the US Food and Drug Administration (FDA) to include this in the
BioMarin reported Kuvan net product revenues of $36.4 million
in the third quarter of 2012, up 19.3% year over year. The
company expects Kuvan net product revenue in the range of
$130-$140 million for the full year 2012.
BioMarin also mentioned in its press release that the results
from the study will help the company to design the phase III
PEG-PAL (poly ethylene glycol-phenylalanine ammonia lyase)
program. The candidate is being developed for the treatment of
severe PKU. In Sep 2012, BioMarin reported positive preliminary
results from an open-label, multi-center, phase II program of
We remind investors that BioMarin will report its fourth
quarter and full year results on Feb 21. We expect investor focus
to stay on the company's 2012 results.
BioMarin currently carries a Zacks Rank #3 (Hold). However
other bio pharma stocks such as
Alexion Pharmaceuticals Inc.
) carry a Zacks Rank #2 (Buy).
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