Filana Therapeutics Announces FDA Lift on Clinical Hold for Simufilam Phase 2a Study

Filana Therapeutics, Inc. (NASDAQ: FLNA) announced on September 22, 2026, that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on its investigational new drug (IND) application. This action clears the way for the company to initiate a planned Phase 2a proof-of-concept study evaluating simufilam in patients with tuberous sclerosis complex (TSC)-related epilepsy.

During the period the clinical hold was in place, the company completed key trial-readiness activities. These included engaging a leading clinical research organization, identifying clinical trial sites, and recruiting potential investigators. The company states that with investigational drug supply now in place, the implementation of the Phase 2a study is able to progress expeditiously toward site initiation and patient enrollment.

The Phase 2a study is designed as a 16-week, multicenter, randomized, double-blind clinical trial. It will evaluate two doses of simufilam in patients with refractory TSC-related seizures. The trial 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 subsequent 48-week extension study.

The study will assess safety, tolerability, pharmacokinetics, and seizure-related measures. These measures include seizure frequency, seizure intensity and duration, nighttime seizures, and sleep-related outcomes. Data from the trial is expected to inform the development path for simufilam in TSC-related epilepsy, including the design of future studies in younger patient populations.

Simufilam is an oral small molecule intended to modulate the filamin A protein. The clinical evaluation is supported by preclinical findings from the laboratory of Angélique Bordey, PhD, at Yale School of Medicine, and studies conducted with the TSC Alliance Preclinical Consortium. Filana holds an exclusive worldwide license from Yale University to intellectual property supporting the use of simufilam in TSC-related epilepsy.