Drug discovery
The exploratory research and drug discovery process includes the search for a new approach to treat a certain disease. Compounds are either searched for in existing compound collections or are de novo designed and synthesized.

Evaluation is to a great extent performed with assays that determine binding to target proteins, effects on relevant markers in cultured cells and preclinical disease models. Once potent and selective compounds have been identified these may need to be further optimized to ensure adequate pharmaceutical properties.

Preclinical development
In recent years Karo Bio has built up resources for preclinical development to enable selection of high quality drug candidates. In-house resources include in vitro models for ADME (absorption, distribution, metabolism and excretion) as well as resources for pharmacokinetics, bioanalysis and in vivo pharmacology. In-house competences for safety pharmacology and toxicology are also available.

In addition to these resources, Karo Bio utilizes contract organizations to characterize compounds. When a compound fulfills preset criteria for efficacy and safety in these models a candidate drug is selected. A documentation package for regulatory approval, before starting clinical trials, is prepared after additional external toxicology studies.

Clinical development
The department for clinical development is responsible for planning and execution of clinical studies. During the clinical trials the compound is evaluated for its safety and efficacy in treating or preventing a specific disease or condition. The results of these studies comprise the most important factors in the approval process.

The different steps in clinical development are described below.

• Clinical phase I
  First stage of clinical testing designed to assess the safety and tolerability of a drug in healthy human volunteers.
  
  
• Clinical phase II
  Phase II trials are performed on larger patient groups (20-300) and are designed to assess how well the drug works and to assess dosing requirements.
  
  
• Clinical phase III
Phase III studies are performed on even larger patient groups (300–3,000) or more, and are aimed at being the definitive assessment of how effective the drug is, in comparison with current 'gold standard' treatment.

Once all clinical documentation has been compiled and evaluated, a new drug application (NDA) is submitted to the regulatory authorities.