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Tickers in this Article: BMRN, AGEN, CBM
BioMarin Pharmaceutical Inc. (BMRN) 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 morquio syndrome.

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).  

Our Recommendation

We have a long-term Neutral recommendation on BioMarin. The stock carries a Zacks Rank #3 (Hold). However, other pharma stocks such as Agenus Inc. (AGEN) and Cambrex Corporation (CBM) carry a Zacks Rank #1 (Strong Buy).

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