Powerful Discovery and Development Engine

Achillion employs a highly-disciplined discovery and development approach that has enabled it to generate a portfolio of oral antivirals for chronic hepatitis C (HCV), including three clinical-stage candidates (an NS5A inhibitor, a protease inhibitor, and an NS5B nucleotide polymerase inhibitor), and a proprietary library of over 1,000 potent and specific complement factor D inhibitors, as well as novel antibacterial compounds.

From early lead identification through clinical candidate selection, Achillion has coupled its knowledge base in genomic replication targets with an integrated drug discovery operation, powered by structure-guided design, to produce a prolific and sustainable discovery engine and facilitate the rapid advancement of its discovery programs.

Compound Synthesis, Hit Identification and Lead Optimization

Achillion’s anti-infective library of compounds contains a diverse set of molecules that have been synthesized for the principal purpose of inhibiting genomic replication in viruses and bacteria. Furthermore, Achillion’s complement inhibitor platform has generated a library of highly potent and specific inhibitors of the serine protease, factor D.

The Company has developed the following discovery tools that enable management of its compounds efficiently and advance its discovery programs. Explore:

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 and display competitive 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 a user-friendly interface developed in-house 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 a user-friendly interface developed in-house 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.

Achillion Pharmaceuticals