In January 2012, we presented positive preliminary data from our ALN-PCS Phase I study at Brigham and Women’s Hospital in Boston. Data show that ALN-PCS demonstrated robust, statistically significant, and dose-dependent lowering of both PCSK9 and LDL cholesterol levels.Read our Press Release ALN-PCS Phase I Data Presentation (3.6MB PPT) Listen to Conference Call
Specifically, the administration of ALN-PCS resulted in statistically significant RNAi silencing of PCSK9 of up to 66% and reductions of up to >50% in levels of LDL-C, or “bad” cholesterol, a clinically validated endpoint. These RNAi effects were rapid and durable after a single dose, supporting a once-monthly dose administration regimen expected in future studies. ALN-PCS was also found to be safe and well tolerated.
We believe these results are the first-ever data showing clinical efficacy of an RNAi therapeutics, specifically as measured by reductions in LDL-C, a clinically validated endpoint associated with coronary artery disease and myocardial infarction. Further, we believe that these data represent important progress in our overall “Alnylam 5×15” efforts and our execution on this exciting product strategy. Importantly, they are the first clinical data to be reported using our second-generation LNP technology, demonstrating a major improvement in potency and strongly supporting the advancement of this platform moving forward.