About Achillion Pharmaceuticals

Achillion is a science-driven, patient-focused biopharmaceutical company seeking to leverage its capabilities across the continuum from discovery through commercialization by discovering and developing small molecule therapeutics aimed at meeting the needs of patients with complement-mediated diseases.

 

Achillion has discovered and developed 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 comprised of three pathways: the alternative pathway, the lectin pathway and the classical pathway.

 

Achillion is currently advancing three novel small molecules from this platform: ACH-4471, ACH-5228, and ACH-5548, all of 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, or C3G, immune complex membranoproliferative glomerulonephritis, or IC-MPGN, both diseases affecting the kidney, and paroxysmal nocturnal hemoglobinuria, or PNH, a blood disorder, as well as several more prevalent indications. To date, Achillion’s lead drug candidate, ACH-4471, has demonstrated preliminary proof-of-concept in phase II trials for patients with C3G and those with untreated PNH.

 

Achillion is headquartered in New Haven, Connecticut. Achillion's common stock trades on the NASDAQ Global Select Market under the symbol: ACHN.

 

To learn more visit us at www.achillion.com.