Program snapshot

Simufilam - tuberous sclerosis complex (TSC)-related epilepsy.

TSC Program Update: The Company is actively addressing FDA’s request for information detailed in the Clinical Hold Letter received in December 2025, including the submission of additional pre-clinical data and protocol design modifications. The Company intends to submit a response to FDA as soon as practicable.

Modality: Oral small molecule.  

Mechanism: Modulation of filamin A (FLNA) function (first-in-class potential).  

Recent data: Simufilam attenuated the progression of seizure activity in a dose- dependent manner in Tsc1 conditional KO mice (independent study with the TSC Alliance Preclinical Consortium); earlier preclinical work showed that simufilam treatment of treating mice with focal onset seizures due to TSC-related pathology , before or after seizure onset, alleviated neuronal abnormalities and reduced seizure frequency by 60-70% compared to vehicle-treated mice.  

Clinical Trial Information: Filana Therapeutics is planning to launch an open-label proof of concept study to assess the safety and tolerability of simufilam in people living with TSC-related epilepsy.  The study will evaluate the effect of simufilam on the frequency and severity of epileptic seizures and other symptoms.