C3G is a rare renal disease which is believed to be the result of over-activity of the alternative pathway. As ACH-4471 has been shown in vitro to inhibit alternative pathway activity, decreasing the formation of C3 protein fragments, the Company has initiated multiple phase 2 studies evaluating ACH-4471 for patients with this devastating disease. Achillion is also supporting a natural history study of C3G conducted by the Imperial College of London to add to the understanding of this disease. There is currently no cure available for C3G, no approved treatment to prevent disease progression and a poor prognosis for patients, of whom approximately 30-50% require dialysis or transplant 10 years after diagnosis.
PNH is a rare, acquired, life-threatening disease of the blood (hemolytic anemia), with an estimated prevalence of 8,000 – 10,000. The current approved treatment or supportive care is eculizumab RBC transfusion. Our goal is to provide consistent and potent inhibition of complement for all PNH patients. Our potent and specific factor D inhibitors have demonstrated activity independent of C5 inhibition.