Recently, Alnylam Pharmaceuticals Inc. (ALNY) announced the commencement of an early-stage stage study (n~32) of its RNAi (RNA [Ribo Nucleic Acid] interference) therapeutic program ALN-PCS for treating patients suffering from hypercholesterolemia.
Hypercholesterolemia indicates a situation where the cholesterol levels in the blood are higher than normal.
The randomized, single-blind, placebo-controlled, single-ascending dose study intends to evaluate the safety and tolerability of the candidate in patients suffering from high low-density lipoprotein cholesterol (LDLc or “bad” cholesterol). Moreover, the clinical activity of ALN-PCS is also expected to be assessed through the study on the basis of measurements of plasma PCSK9 protein and serum LDLc. Results from the study, which is being conducted in the UK, are expected by year end.
ALN-PCS is a part of Alnylam’s core product strategy – 5×15 introduced in January 2011. Alnylam 5×15 aims to develop RNAi therapeutic products for the treatment of genetically defined diseases addressing major unmet medical needs. Alnylam aims to move five RNAi therapeutic products into advanced clinical development by 2015. Management stated that ALN-PCS is the fourth RNAi therapeutic to move into human studies at Alnylam. The other important pipeline candidates include second generation ALN-TTR (being developed for transthyretin-mediated amyloidosis [ATTR]) and ALN-HPN (refractory anemia).
Furthermore, the company is developing ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection; ALN-VSP for the treatment of liver cancers; and ALN-HTT for the treatment of Huntington’s disease (HD), through existing or future partnerships. We believe the second half of this year and 2012 will be an important period for the company due to significant clinical catalysts.
We note that Alnylam has alliances with major players such as Merck, Inc. (MRK), Roche (RHHBY), Biogen Idec (BIIB), Medtronic (MDT), Takeda, Kyowa Hakko Kirin and Cubist Pharmaceuticals (CBST). The alliances further validate the potential and viability of the RNAi approach.
Currently, we have a Neutral stance on Alnylam.
(We are reissuing this article to correct a mistake. The original article, issued October 6, 2011, should no longer be relied upon.)
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