“It is very satisfying that after only one year in lead optimization, we can initiate the final testing with the aim of selecting a candidate for preclinical development. The Kv7 channel field is one of the most promising target areas for the treatment of epilepsy, and we are experiencing great commercial interest in our program", says Thomas Feldthus, CEO of Saniona.

“Our Kv7 compound has a truly new and innovative mode-of-actions and selectivity profile, and the rapid identification of the compound was only possible due to our very robust research platform, IonBaseTM, characterized by fast turn-around chemistry, biological testing, database integration and IP-protection. I am convinced that our innovative Kv7 compound profile represents a new generation of effective and well tolerated epilepsy medicines with potential for use in other brain disorders including depression and bipolar disorder,” said Palle Christophersen, EVP Research.

Epilepsy, a brain disorder characterized by recurrent seizures, affects millions of people worldwide. There is considerable unmet need since about 30 percent of the patients are unresponsive to current medicines.

Kv7 channels mediate potassium ion transport across the cell membrane of neurons, which decreases the likelihood of generating uncontrolled nerve impulses. Activators of Kv7 channels are therefore very effective in dampening overactive neurons and thus prevent the generation of epileptic seizures. Mutations in Kv7 channels containing the Kv7.2 and Kv7.3 subunits are the second most common cause of inherited severe childhood epilepsies, which demonstrate their importance in controlling nerve cell activity.

The non-selective Kv7 activator retigabine has provided both clinical and commercial proof-of-concept for treatment of patients with resistant focal onset seizures. Retigabine has also shown anti-epileptic effect and developmental improvement in smaller investigator-driven studies with children with loss-of-function mutations in Kv7.2. However, retigabine has been withdrawn from the market due to compound specific and non-target related side-effects.

The Kv7 channel family comprise five subtypes, of which channels consisting of Kv7.2 and Kv7.3 subunits are selectively expressed in the brain. The Saniona program focuses on development of subtype selective Kv7.2/Kv7.3 activators, thus avoiding retigabine´s troublesome side effects on the CNS and urinary system, which caused a high drop-out rate in the clinical studies and eventually resulted in quite low adherence to the drug despite good efficacy.