BioMarin Pharmaceutical Inc.
) announced that it has started dosing for a phase II study
(n=24) on BMN 111, an analog of C-type Natriuretic Peptide (CNP),
for the treatment of children suffering from achondroplasia. The
news positively impacted BioMarin's shares.
The phase II open-label, sequential cohort, dose-escalation study
will primarily evaluate the safety and tolerability of daily
subcutaneous doses of BMN 111 in children (5-14 years) suffering
We remind investors that during its second quarter 2013
conference call on Jul 25, 2013, BioMarin stated that the U.S.
Food and Drug Administration (FDA) placed a partial clinical hold
on the development of BMN 111. The FDA had asked for additional
clinical pharmacokinetic and safety data in children suffering
However, in Sep 2013, BioMarin announced that the clinical hold
on the candidate has been lifted by the U.S. regulatory body
following the submission of modified phase II protocol and
withdrawal of the previous one. The regulatory authorities in the
U.S. as well as in Europe agreed that a phase II study for the
indication can be initiated without additional data.
We are encouraged by the successful study initiation after the
clinical hold. However, further objections from regulatory
authorities related to BMN 111 may put the candidate's future in
In a separate press release the company announced that it has
initiated Investigational New Drug (IND)-enabling studies on BMN
270 for the treatment of hemophilia A. BioMarin intends to start
clinical studies on the hemophilia A candidate early next year.
We are encouraged by the company's progress with its pipeline so
BioMarin currently carries a Zacks Rank #3 (Hold). Some
better-ranked stocks include
). All these stocks hold a Zacks Rank #1 (Strong Buy).
BIOMARIN PHARMA (BMRN): Free Stock Analysis
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