In November 2011, we presented positive preliminary data from our ALN-TTR01 Phase I study at the VIIIth International Symposium on Familial Amyloidotic Polyneuropathy. These data show that administration of ALN-TTR01 resulted in statistically significant reductions in serum TTR protein levels in ATTR patients and demonstrate human proof of concept for RNAi therapeutics.Read our press release ALN-TTR01 PhI Prelim Results (821 KB PPT) Listen to replay of conference call (7.1 MB mp3
Lowering of serum TTR protein was found to be dose dependent, rapid, and durable after just a single dose. To date, ALN-TTR01 has been generally safe and well tolerated in this study.
We are committed to bringing this important medicine to ATTR patients in need, and look forward to the continued advancement of our ALN-TTR program, including our upcoming ALN-TTR02 regulatory filing. These data are extremely encouraging and represent a key milestone in the advancement of RNAi therapeutics. Importantly, we believe this result marks the first time RNAi has been shown to silence a disease-causing gene in humans.
Over the past year, we have made very meaningful progress in our transformation from a platform company into a product company focused on advancing RNAi therapeutic programs to patients.
- We launched our ‘Alnylam 5×15’ strategy, where we introduced our focus on genetically defined diseases and targets.
- We also demonstrated clinical activity and human proof-of-RNAi mechanism with ALN-VSP for the treatment of liver cancers.
- And, in the coming weeks, we’ll be reporting data from our Phase I ALN-PCS program using our second generation LNP technology, where we have the potential to demonstrate silencing of PCSK9 protein and effects on LDL cholesterol levels, as well as safety and tolerability.
Taking these events in aggregate with the data presented from our ALN-TTR01 clinical study, we believe we have the opportunity to continue to show that RNAi can be harnessed to make innovative medicines for important diseases.