Earlier this year the alliance began a watch on trials announced which relate to treatment of aHUS.
Click here for the previous news item
An additional trial has been announced for a new complement inhibitor to be used for aHUS . Click here for the press release about it .
This is a phase 2 study by Alnylam Pharmaceuticals involving 12 aHUS patients not previously treated with a complement inhibitor.
The drug is known as Cemdisiran ( previously working name was ALN-CC5) and uses RNA Interference technology.
It works by stopping the production of C5 in the liver rather than blocking C5 from triggering the membrane attack complex excessively.
The purpose of the trial will be to test dose levels and safety and to produce the desired endpoint of platelet recovery to normal levels, and to address TMA , both significant to aHUS patients’ recovery.
The trial results will be known in late 2018.
Click below to view the Alnylam video on RNA Interference (RNAi) technology