Achillion Announces Upcoming Oral Presentation at the 57th Annual Meeting of the American Society of Hematology Detailing Novel Results With Factor D Inhibitors

- Highly potent, specific, and oral factor D inhibitors effectively block hemolysis of paroxysmal nocturnal hemoglobinuria (PNH) cells and mitigate the accumulation of C3 fragments –

- Inhibitory effect against atypical hemolytic uremic syndrome (aHUS) demonstrated by inhibitors in a recently published, novel assay for the rare disease –

- Achillion to host Complement Symposium during ASH on Sunday, December 6, 2015 discussing the complement system, alternative pathway biology, and the role of factor D -

NEW HAVEN, Conn., Nov. 05, 2015 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced that a submitted abstract has been accepted for oral presentation at the 57th Annual Meeting of the American Society of Hematology (ASH) in Orlando, FL, December 5 – 8, and for inclusion in the 2016 Highlights of ASH. 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 highly potent and specific factor D inhibitors from Achillion’s complement inhibitor platform.

Abstracts for the conference are available online at

Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion, commented, “The alternative pathway within the complement system plays a significant role in multiple serious and underserved diseases. This research conducted by Dr. Brodsky and his associates demonstrates that Achillion’s compounds effectively block the breakdown of PNH cells and mitigate the accumulation of C3 fragments on cells which leads to extravascular hemolysis. Furthermore, the effect observed with these inhibitors for aHUS was observed in the first reliable human, in vitro model of the disease, which was developed by Dr. Brodsky and his colleagues.”

Dr. Deshpande further stated, “Achillion’s factor D inhibitor compounds represent a novel approach to potentially treat patients with complement-related rare diseases, many of which are directly caused by dysregulation of the alternative pathway, including orphan diseases such as PNH and aHUS, as well as for higher prevalence indications such as dry age-related macular degeneration.”

Abstract #275

Title: Small Molecule Factor D Inhibitors Block Complement Activation in Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic Syndrome
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 webcast replay of the event will be available from Achillion’s investor website, beginning Sunday, December 6, 2015 at 11:00 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.

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.

About Achillion Pharmaceuticals

Achillion is seeking to apply its expertise in biology and structure-guided design with 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 chronic hepatitis C viral infection (HCV) and complement-related diseases through the inhibition of complement factor D. 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 poster presentations about its complement factor D inhibition program at the ASH annual meeting in 2015 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 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 June 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.

Company Contact: Glenn Schulman Achillion Pharmaceuticals, Inc. Tel. (203) 624-7000 Investors: Mary Kay Fenton Achillion Pharmaceuticals, Inc. Tel. (203) 624-7000 Investors: Tricia Truehart The Trout Group, LLC Tel. (646) 378-2953

Source:Achillion Pharmaceuticals, Inc.