Alnylam Pharmaceuticals, Inc.
) 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
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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
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
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
) 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
Anthera Pharmaceuticals, Inc.
), are comparatively well placed. While Actelion carries a Zacks
Rank #1 (Strong Buy), Anthera carries a Zacks Rank #2 (Buy).