Alnylam Pharmaceuticals Inc.
) recently announced that its candidate ALN-TTR02 has fared well in
a phase I study (n=17). The candidate is being developed for the
treatment of transthyretin (TTR)-mediated amyloidosis (ATTR). The
data was presented at a seminar in Boston University School of
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The randomized, single-blind, placebo-controlled and
single-ascending dose study assessed the safety and tolerability
profile of ALN-TTR02. The candidate was found to be safe and well
tolerated with an impressive pharmacologic profile. Results from
the study revealed that patients treated with ALN-TTR02 experienced
reduction of up to 94% of serum TTR following a solitary dose.
We remind investors that last month the US Food and Drug
Administration (FDA) granted orphan drug status to ALN-TTR02. The
candidate, ALN-TTR02, received orphan drug status to treat patients
suffering from familial amyloidotic polyneuropathy (FAP). The
disorder refers to one of the predominant clinical manifestations
of TTR- ATTR.
We note that the orphan drug designation is granted in the US only
to drugs that treat a disease affecting less than 200,000 people in
the country. Moreover, the orphan drug status for ALN-TTR02
provides Alnylam with seven years of marketing exclusivity (from
the day ALN-TTR02 is cleared by the FDA) in the US for the
The company also initiated a phase II trial to evaluate clinical
activity, safety and tolerability of multiple once-monthly doses of
ALN-TTR02 in patients suffering from ATTR. Subject to positive
results, the company plans to initiate pivotal trial in 2013.
Apart from Alnylam,
) is also developing its candidate, Vyndaqel (tafamidis), to treat
TTR-FAP. We note that TTR-FAP received a complete response letter
(CRL) from the FDA in June 2012. The drug is approved in the EU.
Apart from ALN-TTR02, Alnylam is also developing ALN-TTRsc, for the
utilization of GalNAc-conjugate delivery technology and
subcutaneous dose administration, into clinical development. The
company intends to file a New Drug Application (IND) for ALN-TTRsc
in the second half of 2012 with data expected in the first half of
2013. Furthermore, the company is developing ALN-RSV01 for the
treatment of respiratory syncytial virus infection; ALN-VSP for the
treatment of liver cancer.