Hereditary ATTR Amyloidosis with Cardiomyopathy (hATTR-CM)

We reported new clinical data from our ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of hereditary TTR-mediated amyloidosis (hATTR amyloidosis). These data were presented at the XV International Symposium on Amyloidosis held July 3 – 7, 2016 in Uppsala, Sweden. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=977940" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ISA-2016_Patisiran-Ph-2-OLE_070116.pdf" type="(480 KB PDF)"]View the initial 24-month patisiran Phase 2 OLE data presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ISA-2016_Correlation_070116.pdf" type="(360 KB PDF)"]View the TTR knockdown, mNIS +7 correlation data presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ISA-2016_APOLLO_070116.pdf" type="(370 KB PDF)"]View the APOLLO baseline demographics presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ISA-2016_Revusiran-Ph2-OLE_070116.pdf" type="(640 KB PDF)"]View the initial 12-month revusiran Phase 2 OLE data presentation[/spotlight-link]

We reported data from our ongoing Phase 2 open-label extension (OLE) studies with patisiran and revusiran, investigational RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis). [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=939975" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Patisiran-Ph-2-OLE-18-Month_EC-ATTR_110315.pdf" type="(720 KB PDF)"]View the initial 18-month patisiran Phase 2 OLE data presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Revusiran-Ph-2-OLE-6-Month_EC-ATTR_110315.pdf" type="(550 KB PDF)"]View the initial 6-month revusiran Phase 2 OLE data presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/DISCOVERY_EC-ATTR_110215.pdf" type="(350 KB PDF)"]View the DISCOVERY screening study presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/SSA-NH_EC-ATTR_110215.pdf" type="(670 KB PDF)"]View the SSA Natural History study presentation[/spotlight-link]

We presented pre-clinical data with ALN-TTRsc02, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis).  In pre-clinical studies, including those in non-human primates (NHPs), ALN-TTRsc02 achieved potent and highly durable knockdown of serum TTR of up to 99% with multi-month durability achieved after just a single dose, supportive of a potentially once quarterly dose regimen. Results from studies comparing TTR knockdown activity of ALN-TTRsc02 to that of revusiran showed that ALN-TTRsc02 has a markedly superior TTR knockdown profile.  Further, in initial rat toxicology studies, ALN-TTRsc02 was found to be generally well tolerated with no significant adverse events at doses as high as 100 mg/kg. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=936518" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-TTRsc02_OTS_101415.pdf" type="(1.1 MB PDF)"]View the presentation[/spotlight-link]

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