In November 2024, Axonis announced the successful completion of an oversubscribed $115 million Series A financing, with proceeds being used to advance Axonis’ lead development candidate, AXN-027, through clinical proof-of-concept in patients. 

AXN-027 is a first-in-class oral small molecule designed to potentiate the function of KCC2, a major CNS chloride transporter essential for inhibitory neurotransmission, for the treatment of epilepsy and pain. Proceeds will also enable the development of next generation compounds targeting KCC2 in these and other indications, including psychiatric and neurodevelopmental disorders.