Achillion Pharmaceuticals

Pipeline

Factor D Inhibitors

 

Achillion has discovered and is developing a complement inhibitor platform, directed at advancing small molecule compounds that have the potential to be used in the treatment of immune-related diseases associated with the alternative pathway of the complement system. The complement system is a part of the human innate immune system and is believed to be comprised of three pathways: the alternative pathway, the lectin pathway and the classical pathway. Achillion is advancing novel small molecules from this platform which target complement factor D, an essential protein within the amplification loop of the alternative pathway. Experts believe the alternative pathway plays a critical role in a number of disease conditions including rare orphan conditions such as C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN), both diseases affecting the kidney, and paroxysmal nocturnal hemoglobinuria (PNH), a blood disorder, as well as several more prevalent indications.

 

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