Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is a clinical-stage biopharmaceutical company focused on advancing its orally administered factor D inhibitors into late-stage development and commercialization. Factor D is an essential protein within the amplification loop of the alternative pathway of the complement system. Achillion is focusing its drug development activities on alternative pathway-mediated, rare diseases where there are no approved therapies or where existing therapies are inadequate for patients.
Experts believe the alternative pathway plays a critical role in a number of disease conditions including rare orphan conditions such as PNH, a blood disorder, C3G and immune complex membranoproliferative glomerulonephritis, or IC-MPGN, both diseases affecting the kidney, as well as several more prevalent indications.
The Company’s first-generation factor D inhibitor, danicopan (ACH-4471), has demonstrated preliminary clinical proof-of-concept in patients with paroxysmal nocturnal hemoglobinuria, or PNH, a blood disorder, and C3 glomerulopathy, or C3G, a disease affecting the kidney, and has advanced into phase II clinical trials in both diseases. In addition to danicopan, we have two potent and specific orally-administered second generation factor D inhibitors in phase I clinical trials, ACH-5228 and ACH-5548.
To advance its investigational drugs into phase 3 and commercialization, the Company plans to work closely with key stakeholders including patients, payors, regulators and healthcare professionals.
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