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Data on BioMarin's BMN-701

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BioMarin Pharmaceutical Inc. (BMRN) recently announced positive results on BMN-701 from the phase I/II study, POM-001. BMN-701 is a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) that is being developed for the treatment of late-onset Pompe disease.

Results from the POM-001 study surpassed the company's previously stated requirements for the advancement of the candidate to the next level. Results from the study revealed that around 19% of the patients showed improvement in the 6-minute walk distance.

BMN-701 also demonstrated a 14.1% relative improvement in Maximal Expiratory Pressure (:MEP) along with a 27.0% relative improvement in Maximal Inspiratory Pressure (:MIP) from pre-treatment baseline to week 24.

BioMarin plans to initiate a phase II/III study on the candidate by year end in patients suffering from late-onset Pompe disease who have previously being treated with Sanofi’s (SNY) Myozyme/Lumizyme (alglucosidase alfa). The study will evaluate the efficacy of the candidate by the respiratory parameter MIP along with other parameters such as MEP and six-minute walk test.

We note that BioMarin has several data read-outs and trial initiations lined up this year. The company also plans to initiate two late stage studies this year for PEG-PAL (:PKU) and BMN-673 (solid tumors) among others.

Moreover, BioMarin plans to seek US approval for Vimizim (:GALNS) soon for the treatment of patients suffering from mucopolysaccharidosis Type IVA (MPS IVA) or morquio A syndrome. The company expects potential approval of the candidate by the end of 2013. BioMarin also plans to submit the EU application for the candidate in the second quarter this year.

BioMarin currently carries a Zacks Rank #3 (Hold). However, other biopharma stocks such as Cytokinetics Inc. (CYTK) and Array BioPharma Inc. (ARRY) currently look more attractive carrying a Zacks Rank #1 (Strong Buy) and Zacks Rank #2 (Buy), respectively.

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