Paroxysmal nocturnal hemoglobinuria (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.
C3 Glomerulopathy (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, potentially decreasing the formation of C3 protein fragments, the company plans to initiate a phase 2 study of ACH-4471 in C3G patients by the end of 2016. Achillion also plans to support a natural history study of C3G with a leading international research institution to add to the understanding of this devastating 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.