SAN2219 is a candidate from Saniona’s GABA-A α2/α3 positive allosteric modulator (PAM) program and has demonstrated strong efficacy in multiple seizure models. Its differentiated pharmacological profile supports potential development for both common and rare forms of epilepsy.

Preclinical activities include synthesis optimization, scale-up, GMP manufacturing, and GLP toxicology studies. Saniona completed analytical development and synthesis optimization in 2024 and has now signed a contract to produce GLP toxicology batches in summer 2025.

“Epilepsy affects 50 million people globally, with 30% resistant to current treatments,” said Karin Sandager Nielsen. “There is a significant need for better therapies across both idiopathic epilepsy and rare pediatric syndromes. SAN2219 could help address these unmet needs by improving seizure control without the side effects that often limit current treatments. Our preclinical data indicates that SAN2219 combines strong seizure control with high tolerability and low risk of tolerance development - a rare profile in epilepsy therapies. This program builds on Saniona’s deep ion channel expertise that has repeatedly generated high-value programs, as exemplified by the recent collaboration on SAN711 with Acadia.”

SAN2219 is a selective activator of GABA-A α2/α3/α5 receptors, designed to inhibit excessive neural excitability in epilepsy. By avoiding activation of the α1 subunit targeted by benzodiazepines, SAN2219 offers robust seizure control with an improved safety profile, supporting both acute and chronic use.

About Epilepsy
Epilepsy is a chronic neurological disorder characterized by recurrent seizures caused by abnormal electrical activity in the brain. It affects approximately 50 million people worldwide, making it one of the most common neurological conditions. While many patients achieve seizure control with existing treatments, about 30% remain resistant to available therapies. Epilepsy can range from mild to severe, impacting quality of life and increasing the risk of injury, cognitive impairment, and comorbid conditions. There is a significant need for new treatment options, particularly for drug-resistant epilepsy and rare pediatric syndromes.

Rare pediatric epilepsies are severe and often debilitating forms of epilepsy that typically emerge in early childhood. Many are linked to specific genetic mutations affecting ion channels, neurotransmitter systems, or brain development. These conditions, including Dravet syndrome, Lennox-Gastaut syndrome, and CDKL5 deficiency disorder, are often resistant to standard antiepileptic drugs and associated with developmental delays, cognitive impairment, and other neurological complications. Because of their severity and limited treatment options, there is an urgent need for novel, targeted therapies that can provide better seizure control and improve long-term outcomes for affected children and their families.