New Preclinical Data Being Presented at The Liver Meeting 2013

NEW HAVEN, CT, USA I November 2, 2013 I Achillion Pharmaceuticals, Inc. (ACHN) today announced a poster presentation detailing the preclinical profile of ACH-3422, a uridine-analog nucleotide prodrug being advanced for the potential treatment of chronic hepatitis C viral infection (HCV). The poster is being presented at the 64th Annual Meeting of the American Association for the Study of Liver Diseases (The Liver Meeting 2013) in Washington D.C.

The poster presentation, entitled, “Preclinical Characteristics of ACH-3422: A Potent Uridine Nucleotide Prodrug for Inhibition of Hepatitis C Virus NS5B RNA Polymerase,” (Poster 475; HCV Therapy: The Developmental Pipeline. Saturday, November 2, 2013: 2:00 PM — 7:30 PM ET. Poster Hall), details the potent and specific inhibition of HCV NS5B polymerase by ACH-3422, and the demonstrated low risk for mitochondrial toxicity based upon in vitro studies with human cells in static and proliferating conditions, and high efficiency in the conversion of ACH-3422 into the triphosphate within human hepatocyte cell lines. These attributes, combined with the previously reported 14-day animal toxicity study of ACH-3422, continue to support the advancement of this compound toward clinical studies for use in combination with other direct acting antiviral agents for the potential pan-genotypic treatment of chronic HCV.

“These data provide additional insight into the compelling profile of ACH-3422 as a potential NS5B nucleotide inhibitor for the broad treatment of HCV. Based upon our current development timelines, we anticipate initiating our first-in-human and proof-of-concept trials with ACH-3422 during the first half of 2014,” commented Milind Deshpande, Ph.D., President and Chief Executive Officer of Achillion. “We believe that the ability to potentially combine a potent nucleotide, such as ACH-3422, with our other proprietary assets, including our differentiated Phase 2 NS5A inhibitor, ACH-3102, and our Phase 2 protease inhibitors, including ACH-2684, provides extensive optionality for developing a simple and effective all-oral treatment regimen aimed at curing HCV across broad treatment populations.”

A reprint of the poster presentation can be accessed from the Resources section of our website at  http://www.achillion.com.

About ACH-3422

ACH-3422 is a small-molecule, nucleotide prodrug inhibitor of HCV NS5B polymerase. In vitro, ACH-3422 has demonstrated excellent potency, with activity demonstrated across all genotypes of HCV and an EC50 of approximately 50 to 65 nanomolar against genotype 1 HCV. To date, Achillion has completed 14-day safety studies in animals, where no significant findings were noted at the highest dose tested. ACH-3422 appears to have high oral bioavailability, rapid uptake and conversion of the prodrug into the monophosphate within the liver, and a pharmacokinetic profile supportive of once-daily dosing. Manufacturing and preclinical studies to support clinical development have been initiated, with the expectation of beginning first-in-human studies during the first half of 2014.

About Achillion Pharmaceuticals

Achillion is an innovative pharmaceutical company dedicated to bringing important new treatments to patients with infectious disease. Achillion’s discovery, clinical development, and commercial teams have advanced multiple novel product candidates with proven mechanisms of action into studies and toward the market. Achillion is focused on solutions for the most challenging problems in infectious disease including HCV and resistant bacterial infections. For more information on Achillion Pharmaceuticals, please visit www.achillion.com or call 1-203-624-7000.

SOURCE: Achillion Pharmaceuticals