Achillion's research platform has transformed innovation into action, advancing novel therapeutics from the bench to patients

Discovered and developed in-house, Achillion's platform has generated complement factor D inhibitors, a portfolio of antivirals for HCV (including a NS5A inhibitor, a protease inhibitor, and NS5B nucleotide polymerase inhibitor), as well as antibacterial compounds. Achillion intends to continue applying its deep understanding of immunology, microbiology and synthetic chemistry to expand its product candidate portfolio.

From early lead identification through clinical candidate selection, Achillion has coupled its knowledge base in genomic replication targets with an integrated drug discovery infrastructure to aid in the rapid advancement of its discovery programs.

Compound Synthesis, Hit Identification and Lead Optimization - Achillion's focused compound library contains a diverse set of molecules that have been synthesized for the principal purpose of inhibiting genomic replication in viruses and bacteria. The Company has developed the following discovery tools that enable management of its compounds efficiently and advance its discovery programs:

  • AACP (Achillion Automated Chemistry Platform) is a proprietary software program that facilitates the synthesis of thousands of small molecules in parallel by automating several cumbersome steps involved thereby supporting the Company's drug discovery programs.
  • CART (Compound Acquisition and Repository Tracking) is a software tool that streamlines Achillion scientists' ability to select and acquire compounds for lead identification.
  • CHEM-ACH is a data mining software that allows proprietary compounds synthesized at Achillion to be cross-referenced against biological activities associated with them. Structure-activity relationships are studied with CHEM-ACH, greatly facilitating the design and synthesis of compounds for lead optimization.
  • CIDM (Competitive Intelligence & Data Mining) is a web application that Achillion uses to analyze publicly available information to display competitive information including clinical and preclinical development activities, intellectual property and scientific literature.
  • Complement Wiki is an in-house database of ongoing and completed complement-related drug development and clinical trial designs and results. It also has an in-house developed, user friendly interface for accessing and analyzing this data.
  • HCV Wiki is an in-house database of ongoing and completed HCV therapy clinical trial designs and results. It also has an in-house developed, user friendly interface for accessing and analyzing this data.
  • PSTS (Preclinical Study Tracking System) is a web interface which is used for accessing the details of Achillion's preclinical studies. It allows Achillion's scientists to enter, modify, and query preclinical study documents and facilitate the preclinical development process.

Preclinical Candidate Selection - A cornerstone of Achillion's approach to drug discovery and development is the early assessment of the drug-like properties associated with optimized lead compounds. Potency and activity against a given target are necessary but not sufficient predictors of eventual successful clinical development of a new drug. In order to perform an early assessment of the potential for successful development, prior to progression of a compound into late-stage preclinical studies in support of clinical trials, the Company aggressively evaluates compounds in numerous tests relating to safety, metabolism, pharmacokinetic properties and physical properties associated with the feasibility for an oral formulation.

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