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Achillion Announces Upcoming Poster Presentations Discussing Novel Oral Complement Factor D Inhibitor Platform at the 56th Annual Meeting of the American Society of Hematology

AAQL, BIOGY

- Highly Potent and Selective Oral Factor D Inhibitors Represent a Novel Approach for Treating Complement-Related Diseases Including Paroxysmal Nocturnal Hemoglobinuria (PNH) -

NEW HAVEN, Conn., Nov. 6, 2014 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced that two abstracts are scheduled for presentation at the 56th Annual Meeting of the American Society of Hematology (ASH) in San Francisco, CA, December 6 – 9. The abstracts are now available at http://www.hematology.org/Annual-Meeting/.

Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion, commented, "Over the past year, we have successfully harnessed innovation from our talented R&D teams to expand into complement-related therapeutic areas. Our breakthrough came after we obtained high resolution 3D structures of factor D-inhibitor complexes which allowed us to design potent and specific inhibitors of complement factor D. These upcoming presentations represent the first look into our preclinical complement inhibitor program and we look forward to their inclusion at ASH."

Dr. Deshpande further commented, "Our complement factor D inhibitors possess desirable attributes including pico-molar potency in vitro, low potential for off-target effects, and complete inhibition of complement in non-human primates after oral dosing. We anticipate that our platform could play a role in addressing the needs of all PNH patients, including those patients who fail to respond or respond sub-optimally to the currently available treatment, as well as patients suffering from other complement-mediated diseases."

Publication No. 4817
Title: Novel small-molecule inhibitors targeting complement factor D for therapy of paroxysmal nocturnal hemoglobinuria
Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster III
Date: Monday, December 8, 2014 
Presentation Time: 6:00 PM - 8:00 PM
 
Publication No. 4819 
Title: Preclinical evaluation of orally bioavailable small-molecule inhibitors of complement factor D as a potential treatment for paroxysmal nocturnal hemoglobinuria
Session: 802. Chemical Biology and Experimental Therapeutics: Poster III 
Date: Monday, December 8, 2014 
Presentation Time: 6:00 PM - 8:00 PM 

Reprints of the poster presentations will be made available on the Company's website after presentation at ASH and can be accessed at www.achillion.com/resources.

About Complement Factor D Platform

Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop 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 advancing compounds that inhibit factor D, can be orally-administered, and can potentially be used in the treatment of immune-related diseases in which complement plays a critical role. Potential indications being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), myasthenia gravis, and age-related macular degeneration (AMD). Achillion anticipates that its platform could play a role in addressing the needs of all PNH patients, including patients who have suboptimal response to, or fail to respond to, the currently available treatment, as well as for patients suffering from other complement-mediated diseases.

About Achillion Pharmaceuticals

Achillion is seeking to apply expertise in biology and structure-guided design and a deep understanding of patient and clinician needs to develop innovative treatment solutions aimed at improving patients' lives. The company's scientific excellence, integrated capabilities and experienced team position it to successfully achieve its goal of advancing new products along the entire continuum from the bench to the patient. Achillion's pipeline is currently focused on small molecule therapeutics for infectious disease and complement-related diseases. 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, including statements with respect to the Company's plans to present poster presentations about its complement factor D inhibition program at the ASH annual meeting in 2014 and the potential therapeutic benefits of its complement factor D compounds under development. Achillion may use words such as "expect," "anticipate," "project," "intend," "plan," "aim," "believe," "seek," " estimate," "can," "focus," "will," and "may" and similar expressions to identify such forward-looking statements. 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: advance the preclinical and clinical development of its drug candidates, including ACH-3422, ACH-3102, sovaprevir and 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 appropriate third-parties; compete successfully with other companies that are seeking to develop improved therapies for the treatment of HCV; 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 Annual Report on Form 10-K for the year ended December 31, 2013, 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.

CONTACT: Company Contact:
         Glenn Schulman
         Achillion Pharmaceuticals, Inc.
         Tel. (203) 624-7000
         gschulman@achillion.com
         
         Media:
         Laurie Masonson
         Ogilvy PR
         Tel. (917)459-6164
         laurie.masonson@ogilvy.com
         
         Investors:
         Mary Kay Fenton
         Achillion Pharmaceuticals, Inc.
         Tel. (203) 624-7000
         mfenton@achillion.com
         
         Investors:
         Tricia Truehart
         The Trout Group, LLC
         Tel. (646) 378-2953
         ttruehart@troutgroup.com

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