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Alnylam Pharmaceuticals, Inc. (ALNY - Analyst Report) recently announced positive interim results from a phase II study on ALN-TTR02. ALN-TTR02 is a ribo nucleic acid interference (RNAi) therapeutic targeting the transthyretin (TTR) gene for treating TTR-mediated amyloidosis (ATTR).

Alnylam is evaluating the safety and tolerability of ALN-TTR02 (0.01 to 0.30 mg/kg doses) in the phase II study. Alnylam will also assess clinical activity based on serial measurement of circulating serum levels of wild-type and mutant TTR. The company is conducting the study in 10 different countries including Portugal, France, Sweden, Germany, Spain, Brazil and the US.

A rapid, dose-dependent, and durable knockdown of serum TTR levels was observed in the first 19 patients enrolled and analyzed in this study. The study also revealed that several doses of ALN-TTR02 were generally safe and well tolerated.

Meanwhile, Alnylam plans to initiate an open-label extension (OLE) study on ALN-TTR02 in patients from the phase II study shortly. The company will evaluate the neuropathy impairment score in these patients in the extension study along with other clinical objectives.

Alnylam has plans to present data from this study in 2014. Moreover, the company has plans to initiate a phase III pivotal study on ALN-TTR02 in patients suffering from familial amyloidotic polyneuropathy (FAP) by the end of this year.

We note that Alnylam has a development and commercialization agreement with Sanofi (SNY - Analyst Report) 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.

We are encouraged by the company’s progress with its lead pipeline candidate, ALN-TTR02. Moreover, the orphan drug status granted to ALN-TTR02 by the US Food and Drug Administration (FDA) ensures seven years of marketing exclusivity once the candidate is approved.

Alnylam, a biopharmaceutical company, presently carries a Zacks Rank #2 (Buy). However, biopharmaceutical stocks such as Jazz Pharmaceuticals Public Limited Company (JAZZ - Analyst Report) and Santarus, Inc. look better positioned carrying a Zacks Rank #1 (Strong Buy).

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