The FDA has lifted the clinical hold on Filana Therapeutics’ investigational new drug application for simufilam, allowing the company to begin a planned Phase 2a study in tuberous sclerosis complex-related epilepsy.
Filana expects to begin patient screening by the first quarter of 2027. The company said it has engaged a contract research organization, identified clinical trial sites, recruited potential investigators and secured investigational drug supply.
The planned 16-week study will be a multicenter, randomized, double-blind trial evaluating two doses of oral simufilam in patients with refractory seizures associated with tuberous sclerosis complex, or TSC. Filana plans to enroll 40 patients aged 12 to 55 years at 13 U.S. sites.
Participants who complete the double-blind treatment phase will be eligible to enter a 48-week extension study. The trial will assess safety, tolerability, pharmacokinetics and seizure-related measures, including seizure frequency, seizure intensity and duration, nighttime seizures and sleep-related outcomes.
TSC is a rare genetic disorder linked to mutations in the TSC1 or TSC2 genes. The mutations affect the mechanistic target of rapamycin pathway and can cause tumors in multiple organs. Epilepsy is the most common health issue associated with TSC, affecting an estimated 80% to 90% of patients, according to the company.
Filana said TSC-related epilepsy affects approximately 45,000 people in the United States and that more than 60% of patients remain refractory to antiepileptic therapies despite available treatments.
Simufilam is an oral small molecule designed to modulate filamin A. Filana’s planned clinical program is supported by preclinical findings in two mouse models, including research showing reduced seizure frequency or slowed progression of seizure activity.
Filana holds an exclusive worldwide license from Yale University covering intellectual property supporting simufilam’s use in TSC-related epilepsy.
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