Alnylam Pharmaceuticals, Inc.
) recently announced a restructuring plan which included the layoff
of almost one third of its workforce. Alylam arrived at this
difficult decision because it wanted to focus on its pipeline
candidates with the maximum potential.
The proposed restructuring is expected to slash cash operating
expenses by $20 million in 2012 as against a one-time restructuring
cost of $4 million which will be recorded in the first quarter of
Alnylam, which currently has no product on the market, develops
novel therapeutics based on a biological breakthrough technology
known as RNA (Ribo Nucleic Acid) interference (RNAi). The company
wants to focus its resources on its core product strategy, Alnylam
5x15, which is aimed at developing five RNAi therapeutic products
for the treatment of genetically defined diseases addressing major
unmet medical needs.
The five programs under the strategy include ALN-PCS (for
hypercholesterolemia), ALN-TTR (transthyretin (TTR) mediated
amyloidosis or ATTR), ALN-HPN (refractory anemia), ALN-APC
(hemophilia) and ALN-TMP (hemoglobinopathies). Among these, Alnylam
aims to accelerate development of ALN-TTR and ALN-APC which it
believes holds the most promise.
Under the ALN-TTR program, Alnylam is currently conducting a
phase I study of ALN-TTR01, a systemically delivered RNAi
therapeutic that employs a first-generation lipid nanoparticles
(LNP) formulation to treat ATTR. ATTR is an orphan disease which is
difficult to be addressed by currently available therapeutic
approaches. In late November 2011, Alnylam presented encouraging
data on ALN-TTR01 which showed that it led to statistically
significant reductions in serum TTR protein levels for the
treatment of ATTR. Overall, the candidate was well tolerated with
no serious adverse events observed in the study. This is the first
time an RNAi has silenced a disease-causing gene in clinical
trials, thus highlighting the potential of RNAi as a new class of
Alnylam however intends to accelerate development of ALN-TTR02
which is a second generation delivery formulation for the treatment
of ATTR. Alnylam has chosen ALN-TTR02 as its go-to market product
over ALN-TTR01. Alnylam filed an Investigational New Drug (
) application for ALN-TTR02 in early January 2012 and expects to
initiate a phase I study in the UK in the first half of 2012. Data
from the trial is expected in the third quarter of 2012 with phase
II studies scheduled to begin in the second half.
Further, ALN-APC is in preclinical development and is expected
to move into clinical stages in the first half of 2013.
We currently have a Neutral recommendation on Alnylam. The stock
carries a Zacks #3 Rank ("Hold") in the short term.
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