Alnylam Pharmaceuticals Inc. (ALNY) recently initiated a phase I study on ALN-TTRsc. ALN-TTRsc is a ribo nucleic acid interference (RNAi) therapeutic that targets transthyretin (:TTR) for the treatment of TTR-mediated amyloidosis (:ATTR).
The company is developing the candidate for the treatment of familial amyloidotic cardiomyopathy (FAC). The company is expected to enrol around 40 healthy volunteers for the study. The study will evaluate the safety and tolerability of ALN-TTRsc in these volunteers.
The company expects data from the study in mid-2013. Depending on the results from the phase I study, Alnylam plans to initiate a phase II study on ALN-TTRsc in patients suffering from FAC by the end of this year and a pivotal study for ALN-TTRsc in FAC patients in 2014.
Alnylam is collaborating with Genzyme, a Sanofi (SNY) company, for the development and commercialization of RNAi therapeutics in Japan and the broader Asian-Pacific region. Alnylam is responsible for the same in North and South America, Europe, and the rest of the world.
Earlier this year, Alnylam had provided a brief update on its key “Alnylam 5x15” pipeline, which aims to develop RNAi therapeutic candidates and partner programs for the next couple of years. The company is expecting several study results and initiations based on its RNAi therapeutic candidates in the next two years.
We believe that the company is progressing well with its “Alnylam 5x15” pipeline. We expect investor focus to stay on the successful development of the pipeline, though most of the candidates are in early stages of development.
Alnylam carries a Zacks Rank #3 (Hold). However, other biopharmaceutical stocks such as Athersys Inc. (ATHX) and Array BioPharma Inc. (ARRY) currently carry a Zacks Rank #1 (Strong Buy) and Zacks Rank #2 (Buy) respectively.
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