Recently, BioMarin Pharmaceutical Inc. (BMRN) commenced a mid-stage study of its pipeline candidate GALNS (N-acetylgalactosamine 6-sulfatase) in children suffering from mucopolysaccharidosis IVA (MPS IVA), a rare genetic enzyme deficiency disorder.
The company intends to evaluate the safety and tolerability of the drug dosed at 2.0 mg/kg/week over a period of one year. The study will evaluate 10 to 15 children below 5 years of age. The open-label, multinational study will also evaluate the levels of urinary keratan sulfate (KS) and growth velocity.
Apart from the mid-stage study in children, GALNS is also being evaluated in a randomized, double-blind, placebo-controlled late-stage study (n~160) in the MPS IVA indication. BioMarin is enrolling patients in a late stage study with primary endpoint of six-minute walk distance. Enrollment is underway in the 24-week study. Top-line data from the study, to be conducted in 40 centers around the world, is expected in the second half of 2012.
Apart from GALNS, another promising candidate at BioMarin is PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). PEG-PAL is currently undergoing mid-stage studies. The company intends to provide an update on the candidate at its Research & Development day on December 8, 2011.
BioMarin intends to initiate a late-stage study with PEG-PAL for PKU in the second half of 2012. BioMarin has many other candidates in early stage development. The successful development and commercialization of this robust pipeline will help drive long-term growth at BioMarin.
Apart from the robust pipeline, BioMarin has four marketed products. The marketed products are Aldurazyme, Naglazyme, Kuvan and Firdapse.
BioMarin co-markets Aldurazyme with Sanofi-Aventis (SNY) for the treatment of MPS-I (mucopolysaccharidosis). Naglazyme is marketed for treating MPS-VI. Kuvan is marketed for treating patients suffering from PKU. Firdapse was launched in the EU in April 2010 for treating patients suffering from Lambert Eaton Myasthenic Syndrome (LEMS), a rare autoimmune disorder.
We currently have a Neutral recommendation on BioMarin. The stock carries a Zacks #3 Rank (short-term Hold rating).
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