AUSTIN, Texas — September 22, 2026
Filana Therapeutics announced that the U.S. Food and Drug Administration has lifted the clinical hold on its investigational new drug application for simufilam, enabling the company to advance a planned Phase 2a proof-of-concept study in patients with Tuberous Sclerosis Complex (TSC)-related epilepsy. Filana expects patient screening to begin by the first quarter of 2027. During the clinical hold, the company completed trial-readiness activities, including engaging a clinical research organization, identifying clinical trial sites, recruiting potential investigators and securing investigational drug supply..
Phase 2a Study Evaluates Simufilam in TSC Epilepsy
Filana’s planned Phase 2a study is a 16-week multicenter, randomized, double-blind trial evaluating two doses of simufilam in patients aged 12 to 55 with refractory TSC-related seizures. The study is planned to involve approximately 40 participants across 13 U.S. sites, with eligible participants able to enter a 48-week extension study after completing the double-blind treatment period. The trial will assess safety, tolerability and pharmacokinetics, along with seizure-related measures including seizure frequency, seizure intensity and duration, nighttime seizures and sleep-related outcomes. Data from the study are intended to inform the future development of simufilam, including potential studies in younger patient populations.
Preclinical Findings Support Simufilam Development
Simufilam is an oral small molecule designed to modulate the filamin A protein, and its development in TSC-related epilepsy is supported by findings from two preclinical mouse models. Research conducted at Yale School of Medicine reported that simufilam reduced seizure frequency in a mouse model of focal-onset seizures involving TSC-related pathology. In a separate study conducted through the TSC Alliance Preclinical Consortium using a TSC-knockout mouse model, simufilam was reported to attenuate progression of seizure activity in a dose-dependent manner. These findings are preclinical and do not establish clinical efficacy in patients. Filana holds an exclusive worldwide license from Yale University covering intellectual property supporting the use of simufilam in TSC-related epilepsy.
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Filana Advances Clinical Program in TSC Epilepsy
The FDA’s decision to lift the clinical hold allows Filana to move toward clinical evaluation of simufilam in a population with significant unmet treatment needs. TSC is a rare genetic disorder caused by mutations in the TSC1 or TSC2 genes that can disrupt the mTOR pathway and lead to tumor growth in multiple organs. Epilepsy is reported to affect approximately 80% to 90% of people with TSC, while more than 60% are reported to remain refractory to antiepileptic therapy. Filana is developing simufilam as an investigational therapy for TSC-related epilepsy and is collaborating with the TSC Alliance and the broader TSC community to support patient engagement and clinical study execution.
Source :Filana Therapeutics, press release
