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 C3G and immune complex membranoproliferative glomerulonephritis, or IC-MPGN, both diseases affecting the kidney, and PNH, a blood disorder, as well as several more prevalent indications.
The Company’s first-generation factor D inhibitor, ACH-4471, has demonstrated preliminary clinical proof-of-concept in patients with C3 glomerulopathy, or C3G, a disease affecting the kidney, and paroxysmal nocturnal hemoglobinuria, or PNH, a blood disorder, and has advanced into phase II clinical trials in both diseases.In addition to ACH-4471, 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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May 17, 2019
Events & Presentations
Jun 20, 2019 at 11:00 am EDT Jun 20, 2019
11:00 am EDTThe JMP Securities Life Sciences Conference
Jun 17, 201924th Congress of the European Hematology Association (EHA) in Amsterdam, Netherlands
May 17, 2019The New Era of Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria meeting in Napoli, Italy. Sponsored by AIEPEN Onlus, the Italian PNH Association.