Simufilam

Sept. 22, 2026: Filana Therapeutics (NASDAQ: FLNA) said the FDA lifted the clinical hold on its IND for simufilam, allowing the company to start a planned Phase 2a proof-of-concept study in tuberous sclerosis complex (TSC)-related epilepsy. Patient screening is expected to begin by the first quarter of 2027.

The planned trial is a 16-week, multicenter, randomized, double-blind study in 40 patients ages 12 to 55 with refractory TSC-related seizures at 13 U.S. sites, evaluating two doses of simufilam. Endpoints include safety, tolerability, pharmacokinetics, and seizure-related measures such as seizure frequency, intensity and duration, nighttime seizures, and sleep-related outcomes. Patients completing the double-blind phase may enter a 48-week extension study.

Filana said it has engaged a CRO, identified trial sites, recruited potential investigators, and has drug supply in place. The company is also working with the TSC Alliance to support patient engagement and study execution.

The release cites preclinical support from two mouse models, including dose-dependent attenuation of seizure progression in a TSC-knockout model. Filana also said data from the Phase 2a study are expected to inform future development, including potential studies in younger patients.