TTR Amyloidosis (FAC)

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]

We presented results from a retrospective natural history study evaluating disease progression in transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) patients with familial amyloidotic cardiomyopathy (FAC). These results were presented at a meeting with members of the Association of Black Cardiologists (ABC) at the American College of Cardiology (ACC) Annual Scientific Session, held March 14 – 16, 2015. Amongst other findings, study results showed a mean decline of 140 meters in 6-minute walk distance (6-MWD) over an 18-month period in FAC patients with mild-to-moderate heart failure. Also at the ACC meeting, we presented complete results from our Phase 2 clinical trial with revusiran, an investigational RNAi therapeutic for the treatment of FAC. Consistent with preliminary results presented last year, revusiran achieved an up to 98.2% knockdown of serum TTR – the disease-causing protein – and was found to be generally well tolerated. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=901611" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/FAC-NH-Presentation-for-ABC-meeting_15Mar20151.pdf" type="(2 MB PDF)"]View the FAC Natural History Study presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Revusiran-ACC-15Mar2015.pdf" type="(406 KB PDF)"]View the complete revusiran Phase 2 results[/spotlight-link]

On December 12, we hosted an R&D Day in New York City. Alnylam management and key opinion leaders discussed the latest progress as well as plans for the future development of our RNAi therapeutics pipeline. At this event, we announced our pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease. [spotlight-link icon="podcast" href="http://edge.media-server.com/m/p/njherf95" type=" "] Listen to the webcast replay[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/MASTER-RD-DAY-DECK_Capella.pdf" type="(12.3 MB PDF)"] View the complete presentation[/spotlight-link]

We announced positive initial data from our Phase 2 clinical trial with revusiran (ALN-TTRsc), an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR cardiac amyloidosis. Initial results from the pilot Phase 2 study demonstrated that revusiran was generally well tolerated in patients with significant disease burden.  In addition, data showed an up to 98.2% knockdown of both mutant and wild-type forms of TTR – the disease-causing protein.  As would be expected with the short treatment duration of five weeks, there were no significant changes observed in exploratory clinical measurements. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=883025" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Revusiran_Ph2-AHA_ABC_11152014.pdf" type="(1.5 MB PDF)"] View our presentation[/spotlight-link]

We presented new data from multiple clinical and pre-clinical studies at the 10th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held October 12 – 15, 2014 in San Diego.  Among multiple presentations, we presented additional data from our Phase 1 trial with ALN-TTRsc showing...

We have presented pre-clinical data with RNAi therapeutics targeting TTR for the treatment of TTR-mediated amyloidosis (ATTR).  These data were presented at the International Symposium on Amyloidosis (ISA) held April 27 – May 1, 2014.  These data showed that the degree of TTR knockdown in a mouse disease model was highly correlated with regression of TTR tissue deposits.  Further, comparative studies performed with the TTR stabilizer tafamidis and a TTR-specific antisense oligonucleotide (ASO) showed RNAi therapeutics targeting TTR to have superior pharmacologic profiles. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=843369" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Preclinical-Evaluation-of-RNAiTx-for-TTR-Poster-ISA2014.pdf" type="(0.7 MB PDF)"] View our poster [/spotlight-link]

We have reported positive clinical results from our Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). Data were presented at the Heart Failure Society of America 17th Annual Scientific Meeting being held September 22 – 25. These results demonstrated that ALN-TTRsc achieved robust, consistent, and statistically significant (p<0.01) knockdown of serum TTR protein levels of up to 94%. In addition, knockdown of TTR was found to be rapid, dose-dependent, and durable.  ALN-TTRsc was found to be generally safe and well tolerated in this study. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-TTRsc-PhaseI-Results-HFSA-Sept2013.pdf" type="(1.8 MB PDF)"] View our presentation [/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-TTRsc-PhI-HFSA-Poster-Sep2013.pdf" type="(0.7 MB PDF)"] View our poster [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=792320" type=" "] Read our press release[/spotlight-link]

We initiated dosing in our Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).  The Phase I trial of ALN-TTRsc is being conducted in the U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects.  The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=749037" type=" "] Read our press release[/spotlight-link][spotlight-link icon="release" href="http://www.alnylam.com/our-pipeline/clinical-trials/" type=" "] Learn more about our ALN-TTR program[/spotlight-link]

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