Achillion Pharma (ACHN) Announces Presentation of ACH-4471 Data in Factor D Inhibition

September 6, 2016 6:09 AM EDT

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Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) announced the presentation of two posters which detailed novel preclinical research into complement biology and the inhibition of factor D by ACH-4471 during the XXVIth International Complement Workshop, September 4 - 8, 2016, in Kanazawa, Ishikawa, Japan.

Researchers from Achillion presented a poster entitled “Assessment of complement-mediated bacterial killing and the effect of a small molecule factor D inhibitor in vitro” that described the effect of inhibiting different complement pathways on bacterial killing via two critical mechanisms: lysis and opsonophagocytosis. A second poster from Achillion, entitled “Comparison of complement functional assays: Differential sensitivities of hemolysis and Wieslab assays to levels of complement proteins C5, factor B, and factor D,” detailed the similarities and differences between inhibition of the alternative versus classical pathway in in vitro assays of complement activity.

“As we build upon our broad knowledge of the complement system, we continue to learn more about the unique role of the alternative pathway, its distinctions from the classical and lectin pathways, and its potential advantages in the treatment of rare diseases,” commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion. "With this important and exciting research, we continue to highlight the potential of factor D inhibition and look forward to advancing our first orally-administered small molecule, ACH-4471, into phase 2 clinical trials for both C3G and PNH during the fourth quarter of this year."

Reprints of the posters presented at the XXVIth International Complement Workshop will be available from the Resources section of the Achillion website at

About the Achillion Complement Factor D Platform

Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop small molecule drug candidates that are oral inhibitors of complement factor D. Factor D is an essential serine protease involved in the complement pathway, a part of the innate immune system. Achillion's complement platform is focused on seeking to advance small molecule compounds that inhibit factor D and can potentially be used in the treatment of immune-related diseases in which complement alternative pathway plays a critical role. Potential indications being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and dry age-related macular degeneration (dry AMD).

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