Achillion Presents Novel Research on Factor D Inhibition at the XXVIth International Complement Workshop

NEW HAVEN, Conn., Sept. 06, 2016 -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today 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 XXVI^th 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 XXVI^th International Complement Workshop will be available from the Resources section of the Achillion website at http://www.achillion.com.

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).

About Achillion Pharmaceuticals

Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is a science-driven, patient-focused company seeking to leverage its strengths across the continuum from discovery to commercialization in its goal of providing better treatments for people with serious diseases. The company employs a highly-disciplined discovery and development approach that has allowed it to pursue best-in-class oral antiviral therapy for chronic hepatitis C (HCV) and build a platform of potent and specific complement inhibitors. Achillion is rapidly advancing its efforts to become a fully-integrated pharmaceutical company with a goal of bringing life-saving medicines to patients with rare diseases. More information is available at http://www.achillion.com.

Cautionary Note Regarding Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other important factors that could cause actual results to differ materially from those indicated by such forward-looking statements. Achillion may use words such as “expect,” “anticipate,” “project,” “intend,” “plan,” “aim,” “believe,” “seek,” “ estimate,” “can,” “focus,” “will,” “look forward,” “goal,” “potential,” and “may” and similar expressions to identify such forward-looking statements. These forward-looking statements also include statements about: the potential for the Company’s complement factor D inhibitor compounds, including ACH-4471, to serve as a novel approach to treating alternative pathway complement-mediated diseases such as C3G and PNH; the potential for the ACH-4471 data to inform future trials and study protocols; the Company’s development plans and timelines for ACH-4471; the Company’s belief that its platform could play a role in addressing the needs of specified types of patients; and statements regarding the Company’s plans, prospects and strategies. Among the important factors that could cause actual results to differ materially from those indicated by such forward-looking statements are risks relating to, among other things Achillion’s ability to: replicate in later clinical studies any favorable findings in preclinical or early-stage healthy volunteers studies of ACH-4471; advance the preclinical and clinical development of its complement factor D inhibitors under the timelines it projects in current and future preclinical studies and clinical trials; obtain and maintain patent protection for its drug candidates and the freedom to operate under third party intellectual property; demonstrate in any current and future clinical trials the requisite safety, efficacy and combinability of its drug candidates; obtain and maintain necessary regulatory approvals; establish commercial manufacturing arrangements; identify, enter into and maintain collaboration agreements with third-parties; compete successfully in the markets in which it seeks to develop and commercialize its product candidates and future products; manage expenses; manage litigation; raise the substantial additional capital needed to achieve its business objectives; and successfully execute on its business strategies. These and other risks are described in the reports filed by Achillion with the U.S. Securities and Exchange Commission, including its Quarterly Report on Form 10-Q for the fiscal quarter ended June 30, 2016, and its subsequent SEC filings.

In addition, any forward-looking statement in this press release represents Achillion’s views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Achillion disclaims any duty to update any forward-looking statement, except as required by applicable law.

Investors:
Glenn Schulman
Executive Director, Investor Relations
Achillion Pharmaceuticals, Inc.
Tel. (203) 752-5510
[email protected]

Media:
Liz Power
Senior Director, Public Relations
Achillion Pharmaceuticals, Inc.
Tel: (203) 752-5509
[email protected]