Achillion maintains a robust discovery program with world-class chemistry and virology expertise

Currently, the Company's principal early-stage programs include the following:

HCV Inhibitor Program - Similar to the current treatment paradigm in HIV, Achillion believes combination therapy for the treatment of chronic HCV infection will benefit from drugs that inhibit HCV replication through complementary mechanisms of action. Achillion has leveraged its experience in HCV drug discovery to identify inhibitors that are distinct in its NS3 protease, NS5A, and NS5B polymerase inhibitor programs that meet target product profiles. Across preclinical and clinical studies, Achillion has demonstrated that these inhibitors have the potential to be efficacious against HCV.

Antibacterial Program - Achillion has extensive expertise in discovering and advancing multiple antibacterial candidates to nomination for preclinical development. Currently, Achillion is developing new compounds that demonstrate excellent inhibition of the essential bacterial enzyme DNA gyrase, and have improved metabolic stability. In particular, preliminary data show potent target inhibition of the Mycobacterium tuberculosis gyrase enzyme which has translated in vitro to robust antibacterial activity against drug-resistant tuberculosis. Furthermore, Achillion is working to identify compounds that possess potent gram negative antibacterial activity and have the necessary attributes for advancement toward preclinical studies.

Complement Factor D Platform - Achillion is leveraging its expertise in structure-based drug design to develop small molecule therapeutics with well-characterized biologic activity for immune-related diseases. In particular, Achillion is working to identify novel complement factor D inhibitor compounds that can be administered orally as potential treatments of complement-related diseases including paroxysmal nocturnal hemoglobinura (PNH), atypical hemolytic uremic syndrome (aHUS), and myasthenia gravis. Achillion has announced plans to nominate the first lead small molecule compound from this program and initiate clinical development during 2014.

To learn more about the proprietary platforms and discovery technologies that have enabled Achillion to make its discoveries, click here.

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