Spotlight

On December 16, 2016, we hosted our fourth consecutive R&D Day in New York City. Alnylam management and key opinion leaders discussed our most advanced clinical programs, reviewing all the latest data for patisiran, fitusiran and givosiran. [spotlight-link icon="podcast" href="https://event.webcasts.com/starthere.jsp?ei=1128976" type=""] Access the replay[/spotlight-link][spotlight-link icon="presentation" href=" http://216.92.19.121/rdday2016/#presentations" type=""]...

On December 10, 2015, we hosted our third consecutive R&D Day in New York City. Alnylam management and key opinion leaders discussed our most advanced clinical programs, reviewing all the latest data and providing guidance on development plans for the ATTR amyloidosis programs, fitusiran (ALN-AT3), ALN-CC5, ALN-AS1, and ALN-PCSsc. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=946453" type=" "]Read our press release[/spotlight-link][spotlight-link icon="podcast" href="http://edge.media-server.com/m/p/fy9m5tbr" type=" "] Access the replay[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/RD-Day-2015_Full-Day.pdf" type="(14.5 MB PDF)"] View the complete presentation[/spotlight-link]

We reported positive initial clinical results from our Phase 1/2 trial of ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. The new clinical data are being presented at the 20th Congress of the European Hematology Association (EHA) held June 11 – 14, 2015. Initial study results from 12 healthy volunteer subjects showed that subcutaneous administration of a single dose of ALN-CC5 resulted in potent, dose-dependent, durable, and statistically significant knockdown of serum C5 of up to 96%. In addition, single dose administration of ALN-CC5 achieved inhibition of serum complement activity of up to 92%, including an up to 61% inhibition of serum hemolytic activity. Further, ALN-CC5 has been found to be generally well tolerated to date. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=917666" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-CC5-EHA2015_FINAL.pdf" type="(677 KB PDF)"] View our presentation[/spotlight-link]

We presented pre-clinical data on ALN-AAT, an investigational RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the treatment of AAT deficiency-associated liver disease. New data, presented at the Digestive Disease Week (DDW) meeting, held May 16 – 19, 2015, showed a robust knockdown of serum AAT of up to 93% in non-human primates (NHPs) with monthly subcutaneous dosing. This level of knockdown was highly durable, lasting for greater than 30 days following the final dose. Further, ALN-AAT was found to have a wide therapeutic index based on results from GLP toxicology studies. In addition, study results were reported from a transgenic mouse model of alpha-1 liver disease, where mice overexpress the human Z-AAT protein. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=913507" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-AAT_DDW_051715.pdf" type="(7 MB PDF)"] View the presentation [/spotlight-link]

We presented initial 12-month clinical data from our ongoing Phase 2 open-label extension (OLE) study with patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin (TTR)-mediated amyloidosis in patients with familial amyloidotic polyneuropathy (FAP). Study results, presented at the 67th Annual Meeting of the American Academy of Neurology (AAN) being held April 18 – 25, showed a mean 2.5 point decrease in modified Neuropathy Impairment Score (mNIS+7) at 12 months in patients who had reached the 12-month endpoint (N=20) at the time of data cutoff. This decrease in neuropathy progression compares favorably to the 13 to 18 point increase in mNIS+7 at 12 months that can be estimated from the literature in untreated FAP patients with similar baseline characteristics. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=907541" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/PATISIRAN-12MONTHOLE-AAN-FINAL_Capella.pdf" type="(497 KB PDF)"] View our presentation[/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 presented positive initial Phase 1 data for ALN-AT3, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders. Preliminary results, presented at the 56th Annual Meeting of the American Society of Hematology (ASH), showed that subcutaneous administration of ALN-AT3 given once weekly for three weeks at low doses of 15 (N=3) or 45 (N=1) micrograms/kg (mcg/kg) resulted in an up to 57% knockdown of AT in hemophilia subjects.  The effects of ALN-AT3 lasted for about 60 days after a single dose. ALN-AT3 was found to be well tolerated in both healthy volunteers and hemophilia subjects enrolled in the study.  These initial results show preliminary evidence for potency and durability of RNAi therapeutics at mcg/kg subcutaneous doses in human studies, and are the first clinical data to be reported for Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=886691" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-AT3_Phase-1_ASH_120814.pdf" type="(1.1 MB PDF)"] View our ALN-AT3 Phase 1 presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ASH_RNA_Symposium_Dec2014.pdf" type="(2.0 MB PDF)"] View our pre-clinical presentation[/spotlight-link]

We presented pre-clinical with ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, at the American Society of Hematology (ASH) Meeting. Data showed an up to 99.2% knockdown of serum C5 and up to 96.2% inhibition of serum hemolytic activity in non-human primates (NHPs) with continued dosing for over seven months. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=886432" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-CC5_ASH_Dec2014_panel-by-panel.pdf" type="(0.9 MB PDF)"] View our poster[/spotlight-link]

We presented pre-clinical data from our investigational RNAi therapeutic programs toward genetically validated targets in development for the treatment of cardiovascular metabolic diseases, including: ALN-PCSsc targeting PCSK9 for the treatment of hypercholesterolemia; ALN-AC3 targeting apolipoprotein C3 (apoC3) for the treatment of hypertriglyceridemia; and ALN-ANG targeting angiopoietin-like 3 (ANGPTL3) for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia.  The data, presented at the American Heart Association (AHA) Scientific Sessions 2014, included new pre-clinical multi-dose data in non-human primates (NHPs) with over six months of dosing for ALN-PCSsc, showing robust and clamped knockdown of PCSK9 of up to 92% and reductions in LDL-C of up to 77% with a once-monthly subcutaneous dosing regimen.  These studies confirm the potential for a once-monthly, and possibly once-quarterly, low volume subcutaneous dose regimen, thus highlighting the emerging profile of our ESC-GalNAc conjugate delivery technology. We believe that ALN-PCSsc represents an innovative, differentiated, and well-validated approach for the treatment of hypercholesterolemia. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=883469" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/CardioMetabolic_AHA_Poster_111714.pdf" type="(1.1 MB PDF)"] View our poster[/spotlight-link]

We have expanded our infectious disease pipeline with two new RNAi therapeutic programs. First, we are advancing ALN-HDV, an RNAi therapeutic targeting the hepatitis delta viral (HDV) genome in development for the treatment of HDV infection. We are also advancing ALN-PDL, an RNAi therapeutic targeting hepatocyte-expressed programmed death ligand 1 (PD-L1) in development for the treatment of chronic liver infections. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=882104" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/20141111_AASLD_HepB-final_Breakout.pdf" type="(1.5 MB PDF)"] View our poster[/spotlight-link]

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