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="https://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="https://www.alnylam.com/web/assets/ASH-2015_CC5_Hill_Presentation_010615.pdf" type="(1.3 MB PDF)"]View the presentation[/spotlight-link]