BioMarin Pharmaceutical Inc.
) 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
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
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
) 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
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
Array BioPharma Inc.
) currently look more attractive carrying a Zacks Rank #1 (Strong
Buy) and Zacks Rank #2 (Buy), respectively.
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