Epilepsy is one of the most common neurological conditions in the world, with around 50 million people with the condition. There are a variety of approved medications for epilepsy and pain. but many patients cannot benefit from the drugs or have severe side effects such as depressive mood, psychosis and anxiety, according to Axonis’ website. This means more approaches are needed to help these patients.
Axonis was launched in 2020 based on research from the U.S. centers Boston Children’s Hospital and Harvard in addition to Canada’s Université Laval. The startup’s lead candidate is designed to enhance the effectiveness of Potassium-chloride co-transporter 2 (KCC2), a protein involved in nerve cell inhibitory transmission. By doing so, it can make nerve cells function normally and help patients who previously didn’t respond to treatments or suffer side effects.
The company’s lead program is due to reach clinical testing later this year, with phase 1 results due in early 2025.
The round, which was co-led by Cormorant Asset Management and venBio Partners, will be used to take Axonis’ lead programme through clinical proof-of-concept in patients with epilepsy and pain. It will also let the company work on separate pipeline programmes treating psychiatric and neurodevelopmental disorders.
“KCC2 is the major central nervous system (CNS)-specific chloride transporter. Unlike ion channels and other targets which are activated by ligands, KCC2 transporter biology is more complex which makes drug discovery difficult,” Axonis CEO Joanna Stanicka told Outsourcing Pharma, adding that the company’s drug discovery technology is backed by high expertise in this protein.
The epilepsy treatment market was worth around $10.15 billion in 2022 and is projected to grow to around $15.35 billion by 2030, with an annual growth rate of 5.1%. This growth is spurred by increasing approvals of new treatments in addition to improved diagnosis approaches. Some of the current drugs in this market include UCB’s Briviact and Pfizer’s Lyrica.
There are other efforts in development to target KCC2 such as directly activating the protein with small molecules, enhancing its expression in nerve cells with RNA or the gene editing tool CRISPR.
Many startups fighting epilepsy are in the medtech space, such as Epi-Minder, an Australian firm that bagged $16 million in a bridge financing round in 2022 to fund the development of its patient monitoring device. Earlier this year, the U.S. company Neurava closed a seed round of more than $2 million to kick off the development of wearable devices to save the lives of epilepsy patients.
“The progress in the precision neuroscience field has enabled major clinical successes in recent years, which has led to multi-billion dollar M&A deals for CNS companies, as well as hugely successful IPOs and private financings recently,” Stanicka said.
