Alnylam Pharmaceuticals, Inc.
) 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
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.
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We note that Alnylam has a development and commercialization
) 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
Alnylam, a biopharmaceutical company, presently carries a Zacks
Rank #2 (Buy). However, biopharmaceutical stocks such as
Jazz Pharmaceuticals Public Limited Company
) look better positioned carrying a Zacks Rank #1 (Strong