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="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=929318" type=" "]Read our press release[/spotlight-link]
[spotlight-link icon="presentation" href="https://www.alnylam.com/web/assets/ALN-PCSsc-Phase-1_Poster_08302015.pdf" type="(0.4 MB PDF)"]View the poster[/spotlight-link]
[spotlight-link icon="presentation" href="https://www.alnylam.com/web/assets/ALN-PCSsc-Phase-1_Presentation_08302015.pdf" type="(1.7 MB PDF)"]View the presentation[/spotlight-link]