Our continuous growth depends on the achievements of our Research and Development
To create the best conditions for the discovery and development of novel therapies, we follow an optimal development process to ensure progression of a target candidate from idea to registration on the market.
LEO Pharma has a fully integrated drug discovery platform with a strong interplay between various scientific disciplines including medicinal chemistry, bioinformatics, molecular and cell biology, pharmacology and pharmacokinetics.
From idea to drug candidate
Identification of new product opportunities requires an in-depth understanding of the molecular pathology of the disease we try to treat. World leading pioneering scientific knowledge in combination with curiosity and a profound interest in helping patients is a prerequisite for the success. We follow the frontline in pioneering science, treatments, competitor pipelines and moves, as well as the scientific community around our key focus indication.
In an iterative process including identification of new targets and experimental biological profiling, we work to understand how our compounds work on the molecular level; how they interact with the biological target and how this intervention will translate into the disease in patients. This profiling includes a variety of biochemical and cell-based assays.
In vivo pharmacologists test the molecules in target-relevant and mechanistic in vivo models. Molecules are differentiated and profiled in order to obtain the pharmacodynamic effect and the pharmacokinetic profile that is needed.
When the most interesting drug molecules have been selected, we focus on obtaining a thorough understanding of the biological effect of compounds. This includes profiling in more advanced models to identify the most promising compounds without any predictable adverse effects, to gain a thorough understanding of mechanism of action and to establish the optimal therapeutic regimen.
In the preclinical development phase, we assess the risk of the drug candidate to human health prior to, during and after clinical development. We plan, execute and compile non-clinical regulatory safety studies and submissions on development candidates or products to support clinical evaluation and marketing of our drugs.
All preclinical safety studies (e.g. toxicology and drug metabolism) are outsourced to specialised contract research organisations (CROs), and we have an established procurement function handling all contractual steps.
'Freedom to operate' analyses are performed to ascertain that no blocking patent rights seem to exist in relation to the drug candidates. We subsequently prepare patent applications covering the new compounds, therapeutic uses thereof, formulations and processes.
Potential new molecules continue to the pharmaceutical formulation phase, in which the choice of ingredients to complement the pharmaceutical substance, the dosage form (e.g. injectable, cream, ointment, tablet, gel, foam, etc.) and the manufacturing methods are designed and tested. The optimum pharmaceutical formulation secures the right absorption of the drug molecule into the human body with a minimum of side-effects.
The drug candidate has now advanced to the clinical testing in humans, which traditionally takes five to seven years.
In phase I, we test the drug in healthy volunteers starting with very small doses followed by higher until the doses are expected to be effective in patients. The safety and tolerability of the drug is assessed, and pharmacokinetic evaluations are performed.
In phase II, the drug is tested in few patients and the effect on the target disease is evaluated. In addition, the dosing of the drug and the safety in patients are investigated.
In phase III, the findings from phase II are confirmed in a large patient population. The purpose is to demonstrate efficacy and safety in statistically robust, unbiased studies, which usually include several hundred patients from many hospitals in several countries, and the trials are traditionally controlled and double-blind. We co-operate with more than 1,000 investigators worldwide to conduct the trials.
After successful phase III studies, the drug may obtain marketing authorisation, and is usually further investigated in several phase IV trials to define the exact role of the drug in treatment of diseases.
Throughout the drug development process, from early discovery through launch of a product, regulatory affairs provide input and strategies to ensure that the data and documentation developed are tailored to suit the final registration by the various health authorities worldwide. Our regulatory affairs department analyse and compile documentation from the clinical and non-clinical studies, quality data and the manufacturing process.
This documentation is electronically submitted to the regulatory authorities for assessment after which - if the data are found sufficient to prove the drug's quality, efficacy and safety - we are granted marketing authorisations.
The major part of regulatory activities is after launch of the new product; Changes in the manufacturing process require approval from the regulatory authorities, new indications, new pharmaceutical formulations or additional safety information requires changes in the labelling, and new markets and requirements from regulatory authorities require updates.
Throughout the life-cycle of LEO Pharma products, we continuously provide up-to-date information regarding benefits and risks of our products, based on information from many sources such as preclinical and clinical trials, case reports from healthcare providers, consumers, literature and quality complaints.
International and national legislation regulate the field strictly. LEO Pharma follows the requirements and we maintain a constant and focused safety surveillance of our products in all markets. Regulatory authorities are continuously kept informed about our evaluations and activities to sustain their confidence in LEO Pharma products.
In all phases, we investigate and document the toxicity of the drug and how administration of the drug interacts with physiological processes, including building knowledge on how the drug is absorbed, distributed, metabolised and excreted. This is paramount knowledge for documenting the clinical efficacy and safety of the new drug.