BioMarin Pharmaceutical Inc.
) recently acquired privately held biotechnology company,
Zacharon Pharmaceuticals, which develops small molecules
targeting pathways of glycan and glycolipid metabolism.
BioMarin bought 100% of Zacharon's share capital for $10
million upfront and might make additional clinical, regulatory
and commercial milestone payments. Zacharon's pipeline consists
of two optimization programs -- inhibition of heparan sulfate
synthesis for MPS III (mucopolysaccharidosis) and other MPS
disorders, and inhibition of ganglioside synthesis for diseases
like Tay Sachs and Sandhoff.
We expect the Zacharon acquisition to expand BioMarin's drug
development programs. BioMarin has a robust pipeline with several
data readouts expected in the first half of 2013. In Nov 2012,
the company reported positive results on its lead pipeline
candidate, GALNS, indicated for the treatment of MPS IVA or
The company intends to seek regulatory approval for the
candidate soon. BioMarin also expects GALNS to be approved in the
US in the fourth quarter of 2013.
Another important candidate, PEG-PAL (PEGylated recombinant
phenylalanine ammonia lyase), is being developed for the
treatment of phenylketonuria (PKU). In Sept 2012, the company
presented positive preliminary results from a phase II program of
PEG-PAL and expects to commence a phase III study on the
candidate in the second quarter of 2013.
In addition to these late-stage candidates, the company has
BMN-673 for (genetically defined cancers and advanced
hematological malignancies) and BMN-701 (Pompe's disease).
We have a long-term Neutral recommendation on BioMarin. The
stock carries a Zacks Rank #3 (Hold). However, other pharma
stocks such as
) carry a Zacks Rank #1 (Strong Buy).
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