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BioMarin Pharmaceutical Inc. (BMRN - Analyst Report) recently announced the completion of a phase I study, which evaluated its candidate BMN-111 in men (above 18 years) suffering from achondroplasia. Achondroplasia refers to the commonest form of dwarfism in humans. The double-blind, placebo-controlled study was initiated in February this year.

The study consisted of two parts. While the first part of the study was evaluating a chain of single subcutaneous doses, the second part included either ten days of fixed dosing or dose escalation.

Results from the study showed that BMN-111, an analog of C-type Natriuretic Peptide (CNP), was well tolerated. The adverse events observed in the study were mild in nature.

BioMarin now plans to initiate a proof-of-concept and dose finding phase II study on the candidate in mid-2013. The primary objective of the study will be to assess growth velocity along with medical complications of achondroplasia.

We note that BioMarin recently announced preliminary results from a phase II program of PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase), which is being developed to treat phenylketonuria (PKU). The company plans to initiate a phase III study of PEG-PAL in the second quarter of 2013.

The company has multiple data readouts lined up in the near future. BioMarin is expecting results from the much awaited phase III trial of GALNS in the fourth quarter of 2012. The candidate is being developed as an enzyme replacement therapy (ERT) for the treatment of lysosomal storage disorder mucopolysaccharidosis type IVA (MPS IVA). The company is also expecting results from a phase I/II trial of BMN-673 (solid tumors) in the same quarter.

Our Recommendation

We have a Neutral recommendation on BioMarin, which carries a Zacks #3 Rank (Hold) in the short run. We expect investor focus to stay on the results of the upcoming data readouts.

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