Filana Therapeutics presents Phase 2a simufilam study plans for TSC-related epilepsy
FLNA•Filana Therapeutics expects screening for its Phase 2a simufilam study in TSC-related epilepsy to begin by Q1 2027 after the FDA lifted a clinical hold. The randomized study plans to enroll about 40 patients at 13 U.S. sites.
1. Study design
The double-blind study will enroll approximately 40 patients ages 12-55 across 13 U.S. sites. Two simufilam doses will be added to standard care, with safety, tolerability and pharmacokinetics as primary endpoints.
2. Preclinical and safety data
Simufilam reduced seizure frequency by 60% in the Yale mouse model; a second model showed dose-dependent attenuation of increased seizure activity. More than 2,000 individuals received simufilam in prior clinical studies, with no drug-related serious adverse events reported.
