(By Balaseshan) Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY) said it has began dosing in its phase 1 clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).
The phase 1 trial of ALN-TTRsc is being conducted in the U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects.
The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc. Secondary objectives include assessment of clinical activity of the drug as measured by serum TTR levels. Alnylam expects to present data from this trial in mid-2013.
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Upon completion of the phase 1 trial, the company plans to start a phase 2 clinical study of ALN-TTRsc in FAC patients by the end of 2013 and, assuming positive results, expects to start a pivotal trial for ALN-TTRsc in FAC patients in 2014.
ATTR is caused by mutations in the TTR gene which cause abnormal amyloid protein deposits to accumulate in various tissues including peripheral nerves and heart, resulting in neuropathy and/or cardiomyopathy.
ATTR represents a major unmet medical need with significant morbidity and mortality; familial amyloidotic polyneuropathy (FAP) affects about 10,000 people worldwide and familial amyloidotic cardiomyopathy (FAC) affects at least 40,000 people worldwide.
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ALN-TTRsc, which is being developed for the treatment of FAC, is a subcutaneously administered RNAi therapeutic that comprises an siRNA conjugated to a GalNAc ligand that enables receptor-mediated delivery to the liver.
Alnylam entered into an exclusive alliance with Genzyme, a Sanofi company, to develop and commercialize RNAi therapeutics, including ALN-TTR02 and ALN-TTRsc, for the treatment of ATTR in Japan and the broader Asian-Pacific region. Alnylam intends to develop and commercialize the ALN-TTR program in North and South America, Europe, and rest of the world.
ALNY closed Friday's regular session at $25.00. The stock has been trading between $9.51 and $25.80 for the past 52 weeks.