Alnylam Pharmaceuticals Inc.
) is a biopharmaceutical company developing novel therapeutics
based on a biological pathway known as RNA (ribo nucleic acid)
interference (RNAi). Alnylam is developing several candidates
using this technology for the treatment of multiple diseases.
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ALN-PCS, a systemically administered RNAi therapeutic, is one of
the important candidates at Alnylam being developed for the
treatment of hypercholesterolemia. Positive results from a phase
I study on ALN-PCS were published in The Lancet. The study
evaluated a single dose of the candidate without concomitant
lipid-lowering agents such as statins.
Results from the study revealed that a rapid, dose-dependent
reduction in plasma PCSK9 of up to 84% from baseline was observed
in patients in the ALN-PCS arm compared to placebo. Moreover,
serum levels of low-density lipoprotein cholesterol (LDL-C) were
reduced by up to 57% from baseline in these patients compared to
placebo. Results also revealed that the candidate was generally
safe and well tolerated in this phase I study.
We remind investors that Alnylam had formed a global alliance
The Medicines Co.
) for the development and commercialization of the ALN-PCS RNAi
program earlier this year. As per the terms of the agreement
Alnylam is eligible to receive up to $180 million on the
achievement of development and commercial milestones apart from
an upfront cash payment of $25 million. The company could also
receive scaled double-digit royalties from The Medicines Company
on global sales of ALN-PCS products commercialized under this
Alnylam, along with The Medicines Company, is currently advancing
the development of another RNAi therapeutic candidate targeting
PCSK9, ALN-PCSsc. In preclinical studies, administration of
ALN-PCSsc resulted in more than 80% reduction in plasma PCSK9
with more than 50% reduction in LDL-C. Alnylam intends to
nominate a development candidate for ALN-PCSsc by year end.
We are encouraged by the pipeline progress at Alnylam. However,
most of the candidates at the company's pipeline are in the early
or middle stages of development. We expect investor focus to
remain on Alnylam's pipeline going forward.
Alnylam presently carries a Zacks Rank #2 (Buy). Stocks such as
Endo Health Solutions Inc.
) currently look better positioned with a Zacks Rank #1 (Strong