Achillion Pharmaceuticals

Factor D Inhibitors

ACH-4471

ACH-4471 is a potent and highly specific factor D inhibitor nominated as the first compound to be advanced into clinical development. Primary and secondary pharmacology studies, as well as non-clinical toxicity studies up to 28 days, have been completed. ACH-4471 was dosed orally in non-human studies and demonstrated the ability to block the activation of the alternative pathway. ACH-4471 was been shown to substantially decrease hemolysis and C3b deposition on red blood cells from patients with PNH, and achieved dose-dependent inhibition of cell killing in Atypical hemolytic uremic syndrome (aHUS) in the modified HAM assay. Achillion is currently conducting a number of clinical trials evaluating the potential of ACH-4471 for the treatment of C3 Glomerulopathy (C3G) and paroxysmal nocturnal hemaglobinuria (PNH).

For additional information on Achillion’s C3G clinical trials, please visit:

  • Proof of Concept Study for a 12 Month Treatment in Patients With C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN):NCT03459443
  • Proof of Concept Study for 6 Month Treatment in Patients With C3 Glomerulopathy (C3G): NCT03369236
  • Proof-of-Mechanism Study to Determine the Effect of ACH-0144471 on C3 Levels in Patients With C3G or IC-MPGN: NCT03124368

 

For additional information on Achillion’s PNH clinical trials, please visit:

  • A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab (PNH):NCT03472885
  • A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)): NCT03053102
  • A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH): NCT03181633