Alnylam Pharmaceuticals Inc. ( ALNY ) recently presented data from an early stage study of its pipeline candidate ALN-TTR01 at a scientific conference in Japan. ALN-TTR01 is a systemically delivered RNA interference technology (RNAi) therapeutic that employs a first-generation lipid nanoparticles (LNP) formulation to treat transthyretin (TTR) mediated amyloidosis (ATTR).
Data from the trials showed that ALN-TTR01 led to statistically significant reductions in serum TTR protein levels for the treatment of ATTR. Overall, the candidate has proved to be well tolerated with no serious adverse events observed in the study. We believe the encouraging data further highlights the potential of RNAi as a new class of therapeutics. This is the first time an RNAi has silenced a disease-causing gene in clinical trials.
ALN-TTR01 is part of Alnylam's core product strategy, Alnylam 5x15, which aims at developing RNAi therapeutic products for the treatment of genetically defined diseases addressing major unmet medical needs. Under this strategy, the company aims to have five RNAi therapeutic programs into advanced clinical development by 2015. Apart from ALN-TTR01, this program includes ALN-PCS (for hypercholesterolemia), ALN-HPN (refractory anemia) and ALN-APC (hemophilia). The fifth program is expected to move into pre-clinical development later in 2011.
Alnylam is also developing ALN-TTR02 which is a second generation delivery formulation. Alnylam plans to file an Investigational New Drug ( IND ) application for ALN-TTR02 by year end. Results from the study are expected in 2012.
We currently have an Outperform recommendation on Alnylam. The stock carries a Zacks #2 Rank ("Buy") in the short term. We like the company's "5X15" initiative which aims at developing products for treating genetically defined diseases with unmet medical needs using the potentially revolutionary RNAi technology. We like the pipeline progress at Alnylam and believe that 2012 will be an important year for the company due to significant clinical catalysts. Moreover, the company has collaborations with some of the big pharmaceutical players like Merck ( MRK ), Medtronic ( MDT ), Novartis ( NVS ), Biogen Idec ( BIIB ), Roche ( RHHBY ), Takeda, Kyowa Hakko Kirin, and Cubist Pharmaceuticals ( CBST ) to further develop and utilize its core technology. These partnerships further validate the potential and viability of the RNAi approach.
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