Program snapshot

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

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.  

Planned Phase 2a Trial Design: Filana’s Phase 2a study is a 16-week multicenter, randomized, double-blind clinical trial to evaluate two doses of simufilam in patients with refractory TSC-related seizures. The study is planned to be conducted at 13 sites in the U.S. with a target enrollment of 40 subjects aged 12 to 55. All subjects who complete the double-blind treatment phase are eligible to participate in a 48-week extension study.
The study is designed to assess safety, tolerability, pharmacokinetics, and seizure-related measures, including seizure frequency, seizure intensity and duration, nighttime seizures, and sleep-related outcomes. Data from the study are expected to help inform the development path for simufilam in TSC-related epilepsy, including the design of future studies in younger patient populations.