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Alnylam Pharmaceuticals, Inc. (ALNY - Analyst Report) recently announced that results from phase I studies on ALN-TTR01 and ALN-TTR02 have been published in the New England Journal of Medicine. Both candidates target the transthyretin (TTR) gene and are being developed for the treatment of TTR-mediated amyloidosis (ATTR).

The phase I study on ALN-TTR01 (n=32) was a multi-center, randomized, single-blind, placebo-controlled, dose-ranging trial in patients suffering from ATTR. The study evaluated the safety and tolerability of a single dose of ALN-TTR01 in these patients. Results from the study revealed that a dose-dependent reduction in serum TTR levels (mean reduction of 38%) was observed in patients under ALN-TTR01 treatment. The candidate was also proved to be generally safe and well tolerated.

Alnylam’s phase I study on ALN-TTR02 (n=17) was also a multi-center, randomized, single-blind, placebo-controlled, dose-ranging trial in ATTR patients. Results from the study revealed a substantial knockdown of serum TTR in the patients who received 0.15 to 0.50 mg/kg doses of ALN-TTR02 compared to those under placebo. ALN-TTR02 was also found to be generally safe and well tolerated.

Alnylam is currently evaluating ALN-TTR02 in an open-label, multi-center, multi-dose, dose-escalation phase II study to evaluate the safety and tolerability of the candidate in ATTR polyneuropathy patients. Interim results from the study revealed that several doses of ALN-TTR02 resulted in robust and statistically significant reduction in serum TTR protein levels. The candidate was generally safe and well tolerated in this study as well. Moreover, Alnylam intends to begin an open-label extension study of ALN-TTR02 soon, in patients treated in the phase II study. Positive results from the phase II study will lead to the initiation of a phase III study on the candidate in patients suffering from familial amyloidotic polyneuropathy by year end.

Moreover, Alnylam has ALN-TTRsc under its RNAi therapeutics program for the treatment of ATTR. The company recently announced positive top-line results from a phase I study on the candidate in ATTR patients, where it was found to significantly reduce serum TTR protein levels. Alnylam plans to initiate a phase II study on ALN-TTRsc in the familial amyloidotic cardiomyopathy indication later this year. Positive results from the phase II study will lead to the initiation of a phase III study on the candidate in 2014.

We note that Alnylam has an exclusive collaboration agreement with Sanofi (SNY - Analyst Report) for the development and commercialization of RNAi therapeutics in Japan and the broader Asian-Pacific region. The company also has plans to develop and commercialize the ALN-TTR program in other countries. We expect investor focus to remain on Alnylam’s pipeline going forward.

Alnylam, a biopharmaceutical company, presently carries a Zacks Rank #3 (Hold). Other biopharma stocks, such as Actelion Ltd. (ALIOF) and Anthera Pharmaceuticals, Inc. (ANTH - Snapshot Report), are comparatively well placed. While Actelion carries a Zacks Rank #1 (Strong Buy), Anthera carries a Zacks Rank #2 (Buy).

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