SAN2668
A potential first-in-class therapy for severe pediatric epilepsies
SAN2668 is Saniona’s lead clinical candidate for the treatment of severe pediatric epilepsies, including Developmental Epileptic Encephalopathies (DEEs). These syndromes are often drug-resistant, lack approved therapies, and have lifelong consequences for patients and families.
SAN2668
Designed with selective pharmacology targeting the relevant GABAA receptor subtypes involved in seizure control, SAN2668 offers a precision approach to seizure prevention while minimizing liability for tolerance development, sedation, cognitive impairment, and motor side effects. Its profile supports the potential for best-in-class efficacy for children with difficult-to-treat epilepsy syndromes.
SAN2668 is progressing toward Phase 1 clinical trials in 2026. In addition to safety and tolerability the planned clinical assessments include pharmacodynamic and target engagement studies to provide early validation of its mechanism of action and its ability to achieve therapeutic receptor occupancy levels associated with robust anti-seizure efficacy. These data will inform rational dose selection for Phase 2, supporting an optimal balance between efficacy and tolerability in pediatric populations.
The program reflects Saniona’s commitment to advancing transformative ion channel modulators for rare and severe CNS disorders.
SAN2668 STATUS
SAN2668 is progressing toward Phase 1 clinical trials in late 2026
R&D WEBINAR – JUNE 2026
SAN2668 (GABAA PAM) for the Treatment of Developmental and Epileptic Encephalopathies
In this R&D webinar, Saniona and a leading clinical expert examine the significant unmet need in Developmental and Epileptic Encephalopathies (DEE) and introduce SAN2668, a subtype-selective GABAA positive allosteric modulator in development for these severe, treatment-resistant epilepsies. The session covers the DEE treatment landscape, SAN2668’s mechanism of action and preclinical data, and the planned clinical development pathway
LEARN ABOUT DEE FROM A LEADING SPECIALIST
“What's exciting for us as clinicians is targeting the spike-and-wave pathology. Many DEEs are characterized by persistent epileptiform activity that contributes to cognitive and behavioral impairment - so the potential impact here is not only on seizures, but on behavior and communication.”
Bambino Gesù Children’s Hospital, Rome