Small molecule drugs are low molecular weight organic compounds (typically below 900 daltons) that can interact with biochemical or biological targets like enzymes, cell surface receptors, nucleic acids and others in order to produce a therapeutic effect. By virtue of their small size, these molecules have favorable pharmacokinetic properties like better absorption, distribution, metabolism and excretion compared to biologics like antibodies or gene therapies. Some examples of commonly used small molecule drugs include paracetamol, aspirin, statins, antibiotics and antidepressants.

Target Identification and Validation

The first step in any drug discovery project is to identify and validate a biologically relevant target. This involves determining the biological role and importance of the target through various "omics" techniques like genomics, proteomics and metabolomics. Computational tools are also used to predict potential drug targets. Once a target is identified, assays are developed to test its involvement in the disease state. Knockout models can establish the validity of the target. With the validated target in hand, the drug discovery project can proceed to the next stage.

Hit Identification and Optimization

High-throughput screening (HTS) of vast chemical libraries is used to test hundreds of thousands to millions of Small Molecule Drug compounds in relevant biochemical or cellular assays to identify initial "hits" that interact with the target. These hit compounds typically have low potency and selectivity. Structure-activity relationship (SAR) studies are then used to iteratively optimize the hits into stronger "lead" compounds through systematic variation of their chemical structure. Physicochemical properties like solubility and permeability are also optimized at this stage to improve drug-like qualities. By the end of the hit-to-lead phase, a small set of optimized lead compounds emerge that are suitable for further testing.

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