Achillion Announces Acceptance of Late Breaking Abstract for ACH-4471 at the 21st Congress of the European Hematology Association

Blue Bell, Pennsylvania, UNITED STATES

- Interim results, including safety, pharmacokinetics and pharmacodynamics, to be presented from ongoing phase 1 single-ascending dose trial with ACH-4471 in healthy volunteers -

NEW HAVEN, Conn., May 19, 2016 (GLOBE NEWSWIRE) -- Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN) today announced the upcoming presentation of an e-poster summarizing a late breaking abstract accepted for the 21st Congress of the European Hematology Association (EHA). The e-poster presentation will report interim results from the ongoing phase 1 single-ascending dose healthy volunteer study being led by Roderick B. Ellis-Pegler and Christian Schwabe of Auckland Clinical Studies Ltd, Aukland, New Zealand and a group of researchers from Achillion. The study, initiated in February 2016, aims to assess the safety and tolerability of single-ascending oral doses of ACH-4471 and evaluation of its pharmacokinetic (PK) and pharmacodynamic (PD) profile and PK/PD relationship as measured by alternative pathway (AP) activity ex vivo in hemolysis and Wieslab assays. As described in the abstract, oral dosing of ACH-4471 resulted in rapid, nearly complete and sustained inhibition of complement alternative pathway activity.  ACH-4471 was well tolerated at the single dose levels examined to date with no drug-related serious adverse events, treatment emergent adverse events (TEAE) leading to study discontinuation, or study drug-related grade 3/4 TEAEs reported.

The congress is being held June 9 – 12, 2016 in Copenhagen, Denmark. Abstracts can be accessed on the EHA website at   and a reprint of the poster will be available following its presentation under the resources section of the Achillion website at

EHA E-Poster Information:

Abstract: EHA-4145
Title: An Orally Administered Small Molecule Factor D Inhibitor (ACH-4471) For Treatment of PNH and Complement Diseases: Preliminary Phase I Results in Healthy Volunteers
Session Name: 12. Bone marrow failure syndromes incl. PNH - Clinical
Display Date/Time: Friday, June 10, 09:30 CET to Saturday, June 11, 19:00 CET

About the Achillion Complement Factor D Platform

Achillion has leveraged its internal discovery capabilities and a novel complement-related platform to develop 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 plays a critical role. Potential indications being evaluated for these compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 Glomerulopathy (C3G), dry age-related macular degeneration (dry AMD), and chronic obstructive pulmonary disease (COPD). 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 treatments, as well as for patients suffering from other alternative pathway complement-mediated diseases. Achillion nominated ACH-4471 for clinical development in December 2015, and initiated clinical development in February 2016.

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

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,” and “may” and similar expressions to identify such forward-looking statements. These forward-looking statements also include statements about: the potential disease indications for the Company’s complement platform, and its belief that its platform could play a role in addressing the needs of specified types of patients; the Company’s plans and strategies; and its clinical development of ACH-4471. 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 March 31, 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.



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