7:02AM Alnylam Pharma presents final results from a Phase I clinical trial of ALN-TTR01; data shows that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels, including both wild-type and mutant TTR protein, in ATTR patients (ALNY) 10.60 : Co announced final results from its completed Phase I clinical trial with ALN-TTR01, an RNAi therapeutic targeting transthyretin for the treatment of TTR-mediated amyloidosis. The data were presented at the XIII International Symposium on Amyloidosis held May 6-10, 2012 in Groningen, The Netherlands. Data from this study show that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels, including both wild-type and mutant TTR protein, in ATTR patients. Knockdown of TTR, the disease-causing protein, was found to be dose dependent, rapid, and durable after just a single dose. The full time course for TTR knockdown reveals the potential for once monthly or possibly once every other monthly dose regimens in further studies. ALN-TTR was found to be generally safe and well tolerated in this study. "We believe these data with ALN-TTR01 provide key human proof of concept as we advance ALN-TTR02 as our 'go-to-market' RNAi therapeutic for the treatment of ATTR, a debilitating orphan genetic disease. ALN-TTR02 uses our proprietary second-generation LNP formulation which has demonstrated markedly improved potency in human clinical studies, and we look forward to presenting results from an ongoing Phase I clinical study in the third quarter of 2012. Alnylam is committed to bringing this high impact medicine to patients afflicted with ATTR." ALN-TTR01 was found to be generally safe and well tolerated in ATTR patients. Mild-to-moderate acute infusion reactions were observed in 5 of 24 (20.8%) patients receiving ALN-TTR01 and were readily managed by slowing of the infusion rate where necessary. There were no significant increases in liver function test parameters. All patients on study drug completed the study; there were no discontinuations except for one patient in the placebo group who underwent elective hospitalization for a liver transplant, and was scored as a serious adverse event unrelated to study drug. Co expects to start a Phase II study of ALN-TTR02 in ATTR patients in the second half of 2012 and expects to start pivotal studies in 2013.
