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Updated Healthy Volunteer Data from Phase 1/2 Clinical Study with ALN-CC5 for the Treatment of Complement-Mediated Diseases

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.


We are encouraged by these data as we work to advance potential therapeutic options for patients with complement-mediated diseases such as Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS), and Myasthenia Gravis (MG), amongst others. We look forward to the continued development of ALN-CC5 as we strive to address unmet needs and improve the lives of patients living with complement-mediated diseases.