Alnylam Reports Phase 1/2 Results For Its Drug Candidate ALN-CC5

Shares of Alnylam (ALNY) closed down 1.85% today after the company reported phase 1/2 results for its clinical drug candidate known as ALN-CC5. This drug candidate targets complement component C5 diseases such as paroxysmal nocturnal hemoglobinuria -- PNH -- and atypical hemolytic-uremic syndrome -- aHUS. Complements are proteins in the body that are responsible for controlling the immune system and when they malfunction it can lead to the diseases listed above. 

One thing to note though is that Alnylam is co-developing this product with a division of Sanofi (SNY), known as Genzyme. Alnylam announced that it achieved a 96% knockdown of Complement C5 and 92% of serum complement activity. In addition the drug was able to 61% inhibition of hemolytic activity. This trial was only done as a single dose so the efficacy can be higher. This is because Alnylam expects multidose data for this phase 1/2 trial to come out before the end of 2015. 

The results were pretty good but to determine if they are efficacious compared to other competitors'drugs we will have to wait and see the new multi-dose data by year end. Acompetitor that already targets both diseases mentioned above is Alexion (ALXN). Alexion's drug that targets those diseases is known as Soliris. So unless Alnylam can advance better multi-dose data with ALN-CC5, even though it may make it to market, it will have a serious competitor in this space.