BioMarin Pharmaceutical Inc.
) announced that it has completed dosing the first patient in a
phase I/II study (n~22) on its pipeline candidate BMN 190. The
study is designed to evaluate patients suffering from neuronal
ceroid lipofuscinosis type II (NCL-II), a form of Batten disease.
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The primary objective of the 48 week open-label, dose-escalation
study is to assess the tolerability, safety and efficacy of the
candidate. The effectiveness in treating the disease will be
evaluated by using an NCL-II-specific rating scale score in
comparison with the historical data following 48 weeks of BMN190
therapy. The secondary key points of the study are to assess the
impact of BMN190 therapy on brain atrophy compared with the
historical data following 48 weeks treatment. The study also
intends to characterize pharmacokinetics as well as
The news on BMN 190 comes close on the heels of the research and
development (R&D) day at BioMarin where the company provided
an update on its pipeline. The most important pipeline candidate
at BioMarin is Vimizim. The candidate is being developed for the
treatment of patients suffering from mucopolysaccharidosis Type
IVA (MPS IVA) or morquio A syndrome.
The candidate is under review in the U.S. and the EU. BioMarin is
also seeking approval of the candidate in Brazil. The target date
in the U.S. is Feb 28, 2014. BioMarin stated that prior to the
final decision an advisory panel of the U.S. Food and Drug
Administration (FDA) will review Vimizim's marketing application
on Nov 19, 2013. The European approval of Vimizim is also on
track with the European Medicines Agency (EMA) validating Vimizim
marketing authorization application and granting it accelerated
review status. Consequently, BioMarin expects the EMA's Committee
for Medicinal Products for Human Use to render an opinion on the
marketing application before Dec 31, 2013.
Another interesting candidate in BioMarin's pipeline is PEG-PAL,
which is currently in phase III development for the treatment of
phenylketonuria (PKU). Data from the study is expected by the end
of next year.
BioMarin also threw light on the development of early-stage
candidates in its pipeline. The successful development and
commercialization of the robust pipeline will help drive
long-term growth at BioMarin, which currently has four marketed
BioMarin currently carries a Zacks Rank #3 (Hold). Companies like
Gilead Sciences Inc.
Biogen Idec Inc.
) are well placed with a Zacks Rank #1 (Strong Buy).