Alnylam Pharmaceuticals, Inc. (NasdaqGS:ALNY - News) 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 this year.
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 therapeutics.
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 (NYSE:IND - News) 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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