How Saniona Discovers New Medicines for Rare Diseases
Saniona’s Ion Channel Drug Discovery Engine
The process of discovering a new drug and conducting clinical trials to establish it is safe and effective may take several years and cost hundreds of millions of dollars.1 Additionally, some medicines may fail along the way. In fact, it is estimated that in the discovery research process (before a medicine is ready to be tested in humans), it takes 5,000–10,000 compounds to get just five that will be worthy of advancing into the clinical development process.2 This is why the Saniona scientific team’s experience and history are so important. Our team have been pioneers in discovery research related to ion channels for more than 20 years. We have developed a proprietary ion channel drug discovery engine that has generated a library now consisting of more than 20,000 potential lead molecules targeting various ion channels. We are confident that some of them will become new medicines – and the time and money we have invested in their discovery gives us a tremendous advantage over many start-ups.
Ion channels are critical to many functions including the activity of muscles and nerves. They are used to move charged ions like potassium or chloride into or out of a cell. Ion channel modulation is a validated approach to treating disease and has been used in medicines for diabetes, hypertension, epilepsy and more.
This is where our drug discovery process begins. We start by identifying the disease we want to treat, and we begin to research the ion channel targets known to be associated with that disease.
Initial Biological Screening of Compound Library
Once we understand the ion channel targets we need to modulate, we set up cellular test systems that can be used to screen the compounds in our library against those targets. When a compound matches a target, it’s called a “hit.”
Our proprietary ion channel drug discovery engine draws on our expertise in assay design, electrophysiological approaches, advanced imaging methodology, and various other proprietary techniques, databases and methods.
Discovery Chemistry and In Vitro Pharmacology Aided by Artificial Intelligence (AI)
Based on hits identified from the compound library, we conduct an iterative medicinal chemistry process to optimize the chemical and biological properties of the hit compounds. This research is conducted in test tubes, in our labs. We also need to optimize properties related to drug absorption, distribution, excretion and metabolism – essentially, we are analyzing not only what the drug could do to your body but what your body does to the drug (i.e., how fast do you metabolize and break it down). With the aid of artificial intelligence, we often synthesize several hundreds of new chemical entities before the optimal compound is found in the repeating cycle of iterative processes.
In Vivo Pharmacology and Efficacy
At this stage, we start investigating if the drug will have a beneficial effect on the disease we have decided to treat. We also study in more detail how the drug might affect the whole body, as well as relevant tissues and organs within the body. We also analyze how the effect correlates to the amount of drug in blood samples to estimate an appropriate human dose, and how many times a day it might need to be taken.
An optimized and selected compound undergoes a variety of tests related to toxicology (understanding potential side effects), formulation (deciding if it will be a tablet or a capsule or an injection, etc.) and scale-up (creating processes to make enough of the medicine to conduct clinical trials). We work with a network of partners to complete these steps.
Only after all of these steps have been completed successfully is a compound ready for clinical trials, where it is evaluated in humans. To learn more about the clinical development process, please see our companion Drug Development infographic at www.saniona.com.
- Tufts Center for the Study of Drug Development. Impact Report. Vol. 20, No. 3, May/June 2018.
- Drug Discovery and Development. Britannica. https://www.britannica.com/technology/pharmaceutical-industry/Obstacles-in-drug-development. Last accessed February 2021.