Alnylam Pharmaceuticals Inc. (ALNY) announced positive results from its Phase I study of ALN-PCS, an RNAi therapeutic targeting PCSK9 for the treatment of severe hypercholesterolemia. ALN-PCS is a PCSK9 synthesis inhibitor that reduces intracellular and extracellular levels of PCSK9 resulting in lowered plasma levels of low-density lipoprotein cholesterol or LDL-C , or "bad" cholesterol.
The trial results demonstrated that administration of a single dose of ALN-PCS, in the absence of concomitant lipid-lowering agents such as statins, lead to statistically significant and durable reductions of PCSK9 plasma levels of up to 84% and lowering of LDL-C of up to 50%. Also, the new data highlight continued improved efficacy and tolerability for Alnylam's second-generation lipid nanoparticle or LNP delivery technology.
Phase I study's primary objective was to evaluate the safety and tolerability of a single dose of ALN-PCS, while the secondary objectives included assessment of pharmacodynamic effects of the drug on plasma PCSK9 protein levels and evaluation of clinical efficacy as measured by LDL-C levels.
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