Saniona (SANION) R&D Day 2026 summary

Pipeline overview and program updates

• SAN2668 is the lead internal development program, targeting severe pediatric epilepsies, especially developmental and epileptic encephalopathies (DEEs), as a selective GABA PAM with a focus on both seizure control and comorbidities.

• The program aims to shift from seizure suppression to disease modification, addressing high unmet medical needs in neurology.

• Phase 1 is expected to start in late 2026, with a Phase 1B/2 pediatric DEE basket study planned for early 2028.

• SAN2668 is designed to maintain benzodiazepine-like efficacy while minimizing chronic use limitations and improving tolerability.

• Internal pipeline also includes SAN2219 for epilepsy and SAN2465 for major depressive disorder.

Clinical trial data and development milestones

• Preclinical data show robust seizure control across multiple models, including spike-wave discharges, focal seizures, and behavioral improvements in zebrafish DEE models.

• SAN2668 provides superior seizure control at higher receptor occupancy compared to clobazam.

• Phase 1 will use qEEG and PET biomarkers to confirm target engagement and inform dose selection, with translational biomarkers and a photosensitivity study planned.

• An open-label pediatric study will precede a randomized, placebo-controlled phase II trial measuring both seizure and non-seizure endpoints.

• Planned Phase 1 clinical development strategy and key preclinical data will be discussed at the upcoming R&D Day.

R&D strategy and innovation priorities

• The approach leverages validated biomarkers, including quantitative EEG and PET imaging, for rapid de-risking and objective evidence of activity.

• SAN2668 is engineered for selective potentiation of GABA α2/α3 subtypes, aiming to separate efficacy from sedation and cognitive burden.

• The development plan emphasizes early human efficacy signals and is informed by extensive clinical and regulatory experience in DEEs.

• Ion channel discovery platform underpins the pipeline and is validated through multiple research collaborations.

• Series of webinars planned to highlight differentiated CNS pipeline assets.