We presented data on DMPK and safety of GalNAc-siRNA conjugates at the DIA/FDA Oligonucleotide-Based Therapeutic Conference, held September 9 – 11, 2015, in Washington, D.C.   [spotlight-link icon="presentation" href="" type="(2.1 MB PDF)"]View the DMPK presentation[/spotlight-link] [spotlight-link icon="presentation" href="" type="(2.1 MB PDF)"]View the Toxicology/Safety presentation[/spotlight-link]  

We presented new pre-clinical data supporting the selection of a Development Candidate (DC) for ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO), also referred to as hydroxyacid oxidase 1 (HAO1), for the treatment of primary hyperoxaluria type 1 (PH1).  These data were presented at the 48th European Society of Paediatric Nephrology (ESPN) Annual meeting – held September 3 – 5, 2015, in Brussels – showing up to 99% silencing of the HAO1 mRNA and up to 98% mean reduction of urinary oxalate in animal models of PH1. [spotlight-link icon="release" href="" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="" type="(1.4 MB PDF)"]View the presentation[/spotlight-link]

Alnylam and The Medicines Company reported positive initial results from the ongoing Phase 1 clinical trial with ALN-PCSsc, an investigational RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia.  Subcutaneous administration of ALN-PCSsc resulted in an up to 83% lowering of LDL-C, with an up to 64 ± 5% mean maximum reduction, comparable to published results for anti-PCSK9 monoclonal antibodies (Zhang XL., et al., BMC Med, 2015).  Similar reductions in LDL-C were seen in patients on and off concomitant statin therapy.  The effects of ALN-PCSsc were highly durable, with clinically significant and clamped reductions in LDL-C maintained for over 140 days, supportive of a once-quarterly and possibly bi-annual subcutaneous dose regimen. Maximal lowering effects on LDL-C were consistently achieved at a dose of 300 mg associated with a low injection volume of 1.5 mL. Importantly, ALN-PCSsc was generally well tolerated with no clinically significant adverse events to date. [spotlight-link icon="release" href="" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="" type="(0.4 MB PDF)"]View the poster[/spotlight-link] [spotlight-link icon="presentation" href="" type="(1.7 MB PDF)"]View the presentation[/spotlight-link]

We presented positive interim clinical data from our ongoing Phase 1 study for ALN-AT3, a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders. Data were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress held June 20 – 25, 2015. New clinical results from 12 subjects with severe hemophilia show that subcutaneous administration of ALN-AT3 achieved potent and dose-dependent knockdown of AT of up to 86%. AT knockdown was highly durable, with effects lasting over two months after the last dose, supporting further evaluation of a once-monthly subcutaneous dose regimen. In addition, AT knockdown was associated with statistically significant increases in thrombin generation with a mean increase of up to 350% and marked improvements in whole blood clotting; these results demonstrate a re-balancing of hemostasis in severe hemophilia subjects. Furthermore, in an exploratory post-hoc analysis, a reduced frequency of bleeding was observed at higher AT knockdown levels with a maximum bleed-free interval of 114 days. Very importantly, ALN-AT3 continues to be generally well tolerated. [spotlight-link icon="release" href="" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="" type="(614 KB PDF)"]View our Phase 1 data 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="" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="" type="(677 KB PDF)"] View our presentation[/spotlight-link]


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