Investors in the pharma/biotech sector eagerly wait for pipeline updates as they play an important role in deciding whether or not to invest in a particular company. Pipelines are of prime importance as far as pharma/biotech companies are concerned. These companies spend a significant amount in advancing their pipelines.
Earlier this week, Alnylam Pharmaceuticals Inc. (ALNY) initiated the DISCOVERY study (n=1000) evaluating the prevalence of transthyretin (TTR) mutations in patients suspected to be suffering from cardiac amyloidosis. The main objective of the study is to characterize the frequency of TTR mutations. Alnylam intends to use the results to identify and facilitate the diagnosis of familial TTR medicated amyloidotic cardiomyopathy (FAC).
Currently, Alnylam is evaluating the safety and tolerability of ALN-TTRsc in a phase II study in patients suffering from FAC and senile systemic amyloidosis (SSA). Patients who will complete the phase II study may be eligible for an open-label extension study for additional assessment of general tolerability and clinical activity with long-term dosing of the candidate.
The open label study is expected to start soon. Alnylam also plans to start a phase III study on the candidate in TTR cardiac amyloidosis patients by Dec 31, 2014. ALN-TTRsc is one of the most advanced candidates at Alnylam. Another interesting candidate in the company’s pipeline is Patisiran, which is currently being evaluated in a phase III study for the treatment of transthyretin-mediated amyloidosis (ATTR) patients suffering from familial amyloidotic polyneuropathy.
Alnylam is yet to have an approved product. With most of its candidates either in the early or mid-stages of development, we expect investor focus to remain on the company’s pipeline. Any hiccups in pipeline development will weigh heavily on the stock.
Alnylam carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the healthcare sector are Gilead Sciences (GILD), Medivation (MDVN) and Endo International (ENDP). All these stocks hold a Zacks Rank #1 (Strong Buy).
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