Pipeline Progress at Alnylam


Investors eagerly await updates on the pipelines of pharma/biotech companies. Such updates help investors interested in the above mentioned sector decide whether to invest or not in a particular company. Pipeline updates provide information on experimental drugs and sometimes give an insight into the commercial potential of the candidate once it is successfully developed and commercialized.  

Last week, Alnylam Pharmaceuticals Inc. (ALNY) initiated a phase II study on ALN-TTRsc in patients suffering from familial amyloidotic cardiomyopathy (FAC) or senile systemic amyloidosis (:SSA). ALN-TTRsc is a sub cutaneous ribo nucleic acid interference (RNAi) therapeutic targeting the transthyretin (:TTR) gene that is being developed for the treatment of TTR-mediated amyloidosis (:ATTR).

The open-label, multi-dose, phase II study (n=15) will primarily evaluate the general tolerability of ALN-TTRsc. Alnylam will also assess the measurement of knockdown of serum TTR levels. Moreover, the company will conduct additional tests like cardiac imaging, circulating cardiac biomarkers, 6-minute walk test, New York Heart Association (:NYHA) classification, and measures of heart failure symptoms and quality of life.

Patients successfully completing the phase II study will be eligible for an open-label extension (:OLE) study of ALN-TTRsc, which is expected to be initiated in mid 2014. The OLE study will be evaluating the general tolerability and clinical activity of the candidate with long-term dosing. Alnylam is expecting phase II study results in late 2014. In the event of positive phase II data, Alnylam will move ALN-TTRsc into phase III development by the end of next year in patients suffering from TTR cardiac amyloidosis.

We note that Alnylam has a development and commercialization agreement with Sanofi (SNY) for Alnylam’s RNAi therapeutics, which include ALN-TTR02 and ALN-TTRsc. The agreement is for the development and commercialization of these candidates in Japan and the broader Asian-Pacific region. Alnylam intends to develop and commercialize the ALN-TTR program in other countries as well.

ALN-TTRsc is an important candidate at Alnylam’s RNAi therapeutics program. The candidate has delivered encouraging results in its initial clinical studies. We are also encouraged by Alnylam’s other RNAi therapeutics candidates. 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, a biopharmaceutical company, carries a Zacks Rank #4 (Sell). Some better-ranked stocks in the same sector include Vanda Pharmaceuticals Inc. (VNDA) and Jazz Pharmaceuticals (JAZZ). Both stocks hold a Zacks Rank #1 (Strong Buy).

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