Axonis Therapeutics announced the successful completion of an oversubscribed $115 million Series A financing — proceeds that will be put towards advancing the company's lead development candidate, a first-in-class oral therapeutic for KCC2, through clinical proof-of-concept.
The Massachusetts-based biotech's lead asset, AXN-027, is an 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. KCC2 is a currently undrugged but highly validated CNS-specific target that can restore balance to disinhibited brain circuits to treat neurological disorders without disabling side effects.
According to Axonis, despite the approval of several drugs for epilepsy and pain, many patients can't tolerate or don't respond to these first-line therapies due to impaired synaptic inhibition. Axonis’ oral KCC2 potentiators are designed to restore functional inhibition and overcome drug resistance in patients.
Proceeds from the Series A will also enable the development of next generation compounds targeting KCC2 in other indications, including psychiatric and neurodevelopmental disorders.