Complement-Mediated Disease

We reported new pre-clinical results in animal models of myasthenia gravis (MG) from ALN-CC5, an investigational RNAi therapeutic in development for the treatment of complement-mediated diseases, at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting, held September 14-17 in New Orleans, Louisiana. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/AANEM_ALN-CC5_MG_Presentation_09142016.pdf" type="(1 MB PDF)"]View the presentation[/spotlight-link]

We reported data from 6 patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) enrolled in Part C of our ongoing Phase 1/2 clinical study with ALN-CC5, an investigational RNAi therapeutic targeting complement component 5 (C5) for the treatment of complement-mediated diseases. These data were presented at the 21st Congress of the European Hematology Association (EHA) Meeting in Copenhagen, Denmark, held June 9-12, 2016.  In this part of the study, ALN-CC5 was evaluated as a monotherapy or as an adjunct to eculizumab, an approved anti-C5 monoclonal antibody indicated for the treatment of PNH. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=975341" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/EHA_CC5_Ph1-2_061116.pdf" type="(330 KB PDF)"]View the presentation[/spotlight-link]

We reported updated data from our ongoing Phase 1/2 clinical study with ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. Data were presented at the 53rd Congress of the European Renal Association - European Dialysis and Transplant Association (ERA-EDTA) in Vienna, Austria, held May 21-24, 2016.  Results showed that administration of ALN-CC5 in healthy volunteers (N=44) achieved up to 99% knockdown of serum C5 and a mean maximum of 86% serum hemolysis inhibition in the highest dose group, with mean levels consistently greater than 80% inhibition through the 13 weeks of treatment. Results also showed a mean maximum CH50 inhibition of 99.6% and maximum inhibition up to 100% relative to baseline.   The effects of ALN-CC5 were found to be highly durable, with C5 knockdown clamped at over 90% for more than six months following a single dose. C5 knockdown and complement inhibition results support the potential for a once-quarterly dosing regimen when used in combination with the monoclonal antibody, eculizumab. Importantly, ALN-CC5 was shown to be generally well tolerated, with no serious adverse events and no drug-related discontinuations to date. All adverse events were mild or moderate in severity. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ERA-EDTA_CC5_Ph-1_052216.pdf" type="(880 KB PDF)"]View the presentation[/spotlight-link]

We reported new data from our ongoing Phase 1/2 clinical study with ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases. Data were presented at the American Society of Hematology (ASH) 2015 Annual Meeting, held December 5 – 8, 2015.  Results showed that administration of ALN-CC5 achieved up to 99% knockdown of serum C5 and up to 98% inhibition of serum hemolytic activity, an assay for complement activity.  Further, ALN-CC5 administration resulted in low levels of residual C5, which – based on comparisons from separate studies – were at or below the estimated levels of free C5 observed at therapeutic doses of eculizumab, an approved anti-C5 monoclonal antibody. The effects were also found to be highly durable, with C5 knockdown and complement inhibition results supporting a once monthly and possibly a once quarterly subcutaneous dose regimen.  Importantly, ALN-CC5 was shown to be generally well tolerated, with no clinically significant, drug-related adverse events to date. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=945752" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ASH-2015_CC5_Hill_Poster_010615.pdf" type="(0.6 MB PDF)"]View the poster[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ASH-2015_CC5_Hill_Presentation_010615.pdf" type="(1.3 MB PDF)"]View the 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]

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 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 with ALN-CC5, a subcutaneously administered RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases, at the 25th International Complement Workshop held September 14 – 18, 2014, in Rio de Janeiro, Brazil. Data demonstrated potent and clamped C5 knockdown as well as robust inhibition of complement activity in non-human primates for up to 100 days with a subcutaneous, monthly dosing regimen. Further, in a rat model of membranous nephropathy, ALN-CC5 administration resulted in a significant reduction in proteinuria due to complement-mediated disease activity in the kidney. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=871024" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-CC5_ICW_BRK_091614.pdf" type="(1.2 MB PDF)"] View our poster[/spotlight-link]

We presented pre-clinical data with our Development Candidate for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases, at the 7th International Conference on Complement Therapeutics, held June 6 – 11, 2014, in Olympia, Greece.  These data demonstrate that ALN-CC5 led to an up to 98.7% knockdown of serum C5 and an up to 96.8% inhibition of complement activity in non-human primates (NHP) with weekly subcutaneous dose administration. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=853070" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Alnylam-ALN-CC5-ICCT-June2014.pdf" type="(0.5 MB PDF)"] View our presentation [/spotlight-link]

We presented pre-clinical data from three programs within our “Alnylam 5x15” RNAi therapeutic pipeline: ALN-AT3 for the treatment of hemophilia and rare bleeding disorders (RBD), ALN-CC5 for the treatment of complement-mediated diseases, and ALN-TMP for the treatment of β-thalassemia and iron overload disorders. These data were presented at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 – 10 in New Orleans. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=812683" type=" "]Read our ALN-AT3 press release[/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=812394" type=" "]Read our ALN-CC5/ALN-TMP press release[/spotlight-link]

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