Achillion Announces Upcoming Oral Presentation at the 57th Annual Meeting of the American Society of Hematology and Nomination of ACH-4471, a Novel Small Molecule Factor D Inhibitor, for Clinical Development

Blue Bell, Pennsylvania, UNITED STATES

- Presentation will highlight ACH-4471, a highly potent, specific, and orally-administered factor D inhibitor, and preclinical data on its ability to block hemolysis of paroxysmal nocturnal hemoglobinuria (PNH) cells; mitigate the accumulation of C3 fragments; and its inhibitory effect against atypical hemolytic uremic syndrome (aHUS) -

- Achillion will host a Complement Factor D Symposium during ASH on Sunday, December 6, 2015 at 8:00 p.m. ET to discuss the complement system, the role of factor D, and the profile of ACH-4471 -

NEW HAVEN, Conn., Dec. 05, 2015 (GLOBE NEWSWIRE) --  Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced that an upcoming oral presentation discussing the Company’s novel small molecule factor D inhibitors will take place on Sunday, December 6, 2015 during the 57th Annual Meeting of the American Society of Hematology (ASH) in Orlando, FL, December 5 – 8. Achillion also announced today the nomination of its first orally bioavailable, highly potent and specific factor D inhibitor, ACH-4471, which is expected to enter clinical development during the first quarter of 2016.

"We believe our molecular level understanding of factor D has enabled us to develop exquisitely targeted inhibitors that can potentially provide highly effective and differentiated treatment for complement-mediated diseases," commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion. "We look forward to completing a safety and proof-of-mechanism study with ACH-4471 during the first half of 2016, which should enable us to assess the potential of ACH-4471 to positively impact clinical outcomes in patients in additional studies during the second half of 2016."

The abstract, entitled “Small Molecule Factor D Inhibitors Block Complement Activation in Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome,” details research conducted by Dr. Robert Brodsky and his team at Johns Hopkins University School of Medicine, with three highly potent and specific factor D inhibitors from Achillion’s complement inhibitor platform, including ACH-4471.

Following the oral presentation, Achillion will host a Complement Factor D Symposium featuring a review of complement immuno-biology by Dr. Scott Barnum, Department of Microbiology at the University of Alabama at Birmingham, a presentation by Dr. Peter Densen, Professor of Internal Medicine – Infectious Disease, University of Iowa, discussing relative risks associated with immune modulation, and a review of Achillion’s complement factor D program highlighting ACH-4471 by Dr. David Apelian, Executive Vice President and Chief Medical Officer, Achillion.

ASH 2015 Oral Presentation:

Abstract: #275
Title: Small Molecule Factor D Inhibitors Block Complement Activation in Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome
Presenter: Dr. Eleni Gavriilaki
Session Name: 101. Red Cells and Erythropoiesis, Structure and Function, Metabolism, and Survival, Excluding Iron: Heme and Anemia
Session Date: Sunday, December 6, 2015
Session Time: 4:30 PM - 6:00 PM
Presentation Time: 5:30 PM
Room: Orange County Convention Center, W414AB

Complement Symposium at ASH 2015

The Company will be hosting a Complement Symposium on Sunday, December 6, 2015 in Orlando, FL, during the 2015 ASH Annual Meeting. A live webcast and replay of the event will be available from Achillion’s investor website, beginning Sunday, December 6, 2015 at 8:00 p.m. Eastern time.

Reprints of the oral presentation and symposium slides will all be made available for download from the Company’s website, following the presentations at ASH.

Abstracts for the 2015 ASH Annual Meeting conference are available online and can be accessed at

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 dry age-related macular degeneration (dry 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. Achillion nominated ACH-4471 for clinical development in December 2015, and anticipates initiation of a Phase 1 healthy volunteer trial during the first half of 2016.

About Achillion Pharmaceuticals

Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) is a science-driven, patient-focused company leveraging its strengths across the continuum from discovery to commercialization to provide 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 becoming a fully-integrated pharmaceutical company preparing to bring life-saving medicines to patients with rare diseases. More information is available at

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 an oral presentations about its complement factor D inhibition program at the ASH annual meeting in 2015, to host a symposium discussing factor D and ACH-4471, the potential therapeutic benefits of its complement factor D compounds under development, and ability to advance ACH-4471 into clinical development during the first quarter of 2016. 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 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2015, 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.



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