Yesterday, BioMarin Pharmaceutical Inc. (BMRN) said it started a mid-stage study of PEG-PAL for the treatment of phenylketonuria (PKU). The company expects initial results from the study in mid-2010. It is co-developing the candidate with Merck Serono (MRK).

The primary objective of the open-label, multi-center study, to be conducted in up to 35 patients, is to evaluate the effect of PEG-PAL on blood phenylalanine (Phe) concentrations in PKU patients. The secondary objectives are to evaluate its safety and tolerability, immune response and steady state pharmacokinetics of subcutaneous injections of multiple dose levels.

BioMarin, which competes with companies such as Enzon Pharmaceuticals Inc. (ENZN), Shire Plc (SHPGY) and Altus Pharmaceuticals Inc. (ALTU), already has Kuvan in the market for treating PKU. The primary difference between PEG-PAL and Kuvan is the mode of administration, with Kuvan being an oral formulation and PEG-PAL an injectable.

As a reminder, Kuvan received approval from the US Food and Drug Administration in December 2007 for the treatment of mild to moderate forms of the genetic disease PKU. The disorder affects at least 50,000 diagnosed patients in the developed world. About half of these patients are estimated to have mild to moderate form of the disease, caused by a deficiency of the phenylalanine hydroxylase (PAH) enzyme. PAH is required for the metabolism of phenylalanine, an essential amino acid found in most protein-containing foods.

Last December, Kuvan received marketing approval from the European Commission for the treatment of hyperphenylalaninemia (HPA) in PKU or Tetrahydrobiopterin (BH4) deficiency. HPA results from abnormal rise of blood Phe levels emanating from disorders of Phe metabolism. PKU and BH4deficiency account for the majority of HPA cases.

Since Kuvan is approved for only mild to moderate forms of the disease, there is significant unmet demand for the treatment of PKU. BioMarin is also developing a prodrug, BMN168, for the treatment of PKU. This drug would have patent protection till 2026/2027, which is a major advantage. Successful development of BMN168 and PEG-PAL will help the company to reach the entire PKU market. It will also have products capable of driving future growth.

Currently, we are Neutral on BioMarin.

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