BioMarin Pharmaceutical Inc.
) recently reported positive results from the much awaited phase
III study (MOR-004) of GALNS (2mg/kg) for the treatment of
patients suffering from mucopolysaccharidosis Type IVA (MPS IVA)
or morquio A syndrome. Data from the study revealed that there
was a significant change in six-minute walk distance in
comparison to placebo at the end of 24 weeks. The news positively
impacted the stock price.
Data from the study also revealed an improving trend in
three-minute stair climb and pulmonary function in patients
treated with GALNS compared to placebo, at the end of 24 weeks.
Moreover, patients treated with GALNS showed consistent and
robust reduction in urinary keratan sulfate (KS) levels compared
The company intends to seek regulatory approvals for the
indication in the first quarter of 2013 based on the positive
results from the MOR-004 study. At the third quarter conference
call, the company stated that it expects the US Food and Drug
Administration (FDA) to approve GALNS in the fourth quarter of
2013. BioMarin also announced positive data from the MOR-005
extension study on GALNS.
We are pleased with BioMarin's efforts to develop its
pipeline. We believe that the company can swing to profitability
on the back of the successful development of GALNS.
We note that in September this year, BioMarin reported
positive preliminary results from a phase II program of PEG-PAL
(PEGylated recombinant Phenylalanine Ammonia Lyase). We note that
this is another important pipeline program for the company.
BioMarin has an agreement with Merck Serono, a division of
), since 2005 for the development and commercialization of
PEG-PAL. The agreement was later revised in 2007. BioMarin plans
to initiate a phase III study of PEG-PAL in the second quarter of
2013 after the end of phase II discussion with the US regulatory
We have a long-term Neutral recommendation on BioMarin, which
carries a Zacks #3 Rank (Hold) in the short run.
BIOMARIN PHARMA (BMRN): Free Stock Analysis
(MKGAF): ETF Research Reports
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