Filana Therapeutics Inc (FLNA) News & EventsFLNAFilana Therapeutics Inc

Filana Therapeutics Gets FDA Lift, Starts Phase 2a Trial for TSC-Related Epilepsy

Filana Therapeutics announced that the U.S. Food and Drug Administration, FDA, has lifted the clinical hold on the Company's investigational new drug, IND, application, enabling Filana to initiate a planned Phase 2a proof-of-concept study in patients aged 12 to 55 with TSC-related epilepsy. Patient screening is expected to begin by the first quarter of 2027. "With the clinical hold lifted, we can now execute our development plan for simufilam in TSC-related epilepsy," said Rick Barry, President and Chief Executive Officer of Filana. "While on hold, we completed key trial-readiness activities, including engaging a leading clinical research organization, identifying clinical trial sites, and recruiting potential investigators. With investigational drug supply in place, implementation of our Phase 2a study of simufilam is now able to progress expeditiously toward site initiation and patient enrollment."

Filana Therapeutics Publishes Preclinical Data on Epilepsy

Filana Therapeutics announced publication in Epilepsia of preclinical data showing that simufilam attenuated seizure progression in a well-accepted mouse model of severe TSC-related epilepsy. The results support the continued evaluation of simufilam for the treatment of TSC-related epilepsy. Simufilam is an oral small molecule intended to modulate filamin A protein. Top line results of the study were previously reported by the company in August 2025. The work was conducted in collaboration with the TSC Alliance and PsychoGenics, Inc., the clinical research partner of the TSC Preclinical Consortium. This dose escalation study evaluated simufilam's ability to slow the worsening of seizures in the Tsc1 conditional knockout mouse, an exceptionally severe and progressive model of TSC-related epilepsy. Seizures were monitored for approximately three weeks following onset. The data showed that simufilam attenuated the progression of seizure activity compared to vehicle, with a statistically significant correlation between simufilam dose and the number of seizures by the end of the study. The study evaluated simufilam in the Tsc1-cKO mouse, an exceptionally severe and rapidly progressive model of TSC-related epilepsy. In this model, seizures normally worsen steadily over time. Simufilam attenuated the expected worsening of seizures over time in both number and total duration. Attenuation of seizure worsening increased with drug exposure. Mice were administered vehicle or simufilam at one of three ascending doses. Filana Therapeutics is working to address the previously disclosed request for information from FDA that was contained in a December 2025 Clinical Hold Letter, including the submission of additional pre-clinical data and protocol design modifications. The company intends to submit a response to FDA following completion of these activities and expects to provide a program update in the coming months.

Filana Therapeutics CEO Emphasizes Commitment to Science and Patients

"At Filana Therapeutics, our name reflects our deep commitment to science and to patients affected by diseases tied to filamin A dysregulation, including TSC-related epilepsy. We are driven by the urgent need for new treatment options that can meaningfully improve patients' lives," said Rick Barry, President and Chief Executive Officer of Filana Therapeutics, Inc. "In partnership with our advisors, we are working diligently to address the FDA's requests and look forward to sharing a progress update in the coming months."