BioMarin Pharmaceutical Inc.
) recently submitted a Clinical Trial Application (CTA) with the
Medicines and Healthcare Products Regulatory Agency (MHRA) in the
UK for BMN-190. BMN-190 is being developed for the treatment of
patients suffering from neuronal ceroid lipofuscinosis type 2
(NCL-2), a form of batten disease.
BioMarin intends to begin enrolling for a phase I/II study on
BMN-190 in mid-2013. BioMarin is expected to enrol around 22
subjects globally for the study. The subjects will be evaluated
for 48 weeks.
The study will evaluate the safety and tolerability of BMN-190
in NCL-2 patients. The study will also evaluate the effectiveness
of BMN-190 using an NCL-2-specific rating scale score in
comparison to natural history data.
Meanwhile, BioMarin recently submitted a Biologics License
Application (BLA) to the US Food and Drug Administration (FDA)
for Vimizim (BMN-110, elosulfase alfa) for the treatment of
patients suffering from mucopolysaccharidosis Type IVA (MPS IVA)
or morquio A syndrome. BioMarin also intends to seek approval for
Vimizim in the European Union (EU) for the same indication
towards the end of this month.
Vimizim is the most important pipeline candidate at BioMarin.
The company expects Vimizim candidate to be approved by the end
of 2013. We believe that the company can swing to profitability
in the event of Vimizim gaining approval.
We note that BioMarin has several data read-outs and trial
initiations lined up this year. We expect investor focus to stay
on these results.
BioMarin, a biopharma stock, currently carries a Zacks Rank #3
(Hold). However other biopharma stocks such as
Array BioPharma Inc.
) currently look better positioned. While Athersys and
Cytokinetics carry a Zacks Rank #1 (Strong Buy), Array carries a
Zacks Rank #2 (Buy).
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