Achillion Pharmaceuticals

We are a clinical-stage biopharmaceutical company focused on advancing our oral small molecule complement system inhibitors into late-stage development and commercialization. Each of the product candidates in our portfolio was discovered in our laboratories and is wholly owned by us. We are focusing our product development activities on complement-mediated diseases where there are no approved therapies or where significant unmet medical need persists despite existing therapies. 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. We are currently advancing novel orally administered small molecules from our platform that target complement factor D, an essential protein of the alternative pathway. We believe that an overactive alternative pathway may play a critical role in a number of disease conditions including the therapeutic areas of nephrology, hematology, ophthalmology and neurology. Initially we are targeting C3 glomerulopathy, or C3G, and immune complex membranoproliferative glomerulonephritis, orIC-MPGN, two related rare diseases affecting the kidney, as well as paroxysmal nocturnal hemoglobinuria, or PNH, a blood disorder. We plan to expand our portfolio into additional indications where we believe an overactive alternative pathway plays an important role in disease pathogenesis.