Today, scientists presented final results from our Phase I clinical trial of ALN-TTRo1 at the XIII International Symposium on Amyloidosis held in Groningen. 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.

ALN-TTR01 uses the identical siRNA as ALN-TTR02 our lead RNAi therapeutic for the treatment of ATTR, but ALN-TTR02 employs a proprietary second-generation LNP formulation that has demonstrated improved potency and tolerability based on pre-clinical and clinical studies.  ALN-TTR02 is currently being tested in a Phase I clinical study in normal healthy volunteers with results expected in the third quarter of 2012.  Further, the we expects to start a Phase II study of ALN-TTR02 in ATTR patients in the second half of 2012 and start pivotal studies in 2013.