BioMarin Pharmaceutical Inc.
) recently announced the completion of a phase I study, which
evaluated its candidate BMN-111 in men (above 18 years) suffering
from achondroplasia. Achondroplasia refers to the commonest form of
dwarfism in humans. The double-blind, placebo-controlled study was
initiated in February this year.
The study consisted of two parts. While the first part of the
study was evaluating a chain of single subcutaneous doses, the
second part included either ten days of fixed dosing or dose
Results from the study showed that BMN-111, an analog of C-type
Natriuretic Peptide (CNP), was well tolerated. The adverse events
observed in the study were mild in nature.
BioMarin now plans to initiate a proof-of-concept and dose
finding phase II study on the candidate in mid-2013. The primary
objective of the study will be to assess growth velocity along with
medical complications of achondroplasia.
We note that BioMarin recently announced preliminary results
from a phase II program of PEG-PAL (PEGylated recombinant
Phenylalanine Ammonia Lyase), which is being developed to treat
phenylketonuria (PKU). The company plans to initiate a phase III
study of PEG-PAL in the second quarter of 2013.
The company has multiple data readouts lined up in the near
future. BioMarin is expecting results from the much awaited phase
III trial of GALNS in the fourth quarter of 2012. The candidate is
being developed as an enzyme replacement therapy (ERT) for the
treatment of lysosomal storage disorder mucopolysaccharidosis type
IVA (MPS IVA). The company is also expecting results from a phase
I/II trial of BMN-673 (solid tumors) in the same quarter.
We have a Neutral recommendation on BioMarin, which carries a
Zacks #3 Rank (Hold) in the short run. We expect investor focus to
stay on the results of the upcoming data readouts.
BIOMARIN PHARMA (BMRN): Free Stock Analysis
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