AUSTIN, Texas — Filana Therapeutics, Inc. (NASDAQ: FLNA, “Filana Therapeutics”, the “Company”), a biotechnology company currently focused on developing simufilam for the treatment of Tuberous Sclerosis Complex (TSC)-related epilepsy, today 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.”
“Drug-resistant epilepsy remains a challenge for a majority of individuals affected by TSC,” said Kari Luther Rosbeck, President and Chief Executive Officer of the TSC Alliance. “Research investigating different molecular pathways involved in TSC offers hope for potential new options for those living with the disease. The TSC Alliance applauds Filana for their innovative work in exploring a new target mechanism and their plans to launch a clinical trial to test the effectiveness of that potential new treatment to help address a major unmet need for the TSC community.”
Planned Phase 2a Trial Design
Filana’s Phase 2a study is a 16-week multicenter, randomized, double-blind clinical trial to evaluate two doses of simufilam in patients with refractory TSC-related seizures. The study 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 48-week extension study.
The study is designed to assess safety, tolerability, pharmacokinetics, and seizure-related measures, including seizure frequency, seizure intensity and duration, nighttime seizures, and sleep-related outcomes. Data from the study are expected to help inform the development path for simufilam in TSC-related epilepsy, including the design of future studies in younger patient populations.
Scientific Rationale
Simufilam is an oral small molecule intended to modulate the filamin A protein. The clinical evaluation of simufilam in TSC-related epilepsy is supported by findings from two preclinical mouse models. Initial studies were conducted in the laboratory of Angélique Bordey, PhD, Rothberg Professor of Neurosurgery at Yale School of Medicine and Senior Vice President, Neuroscience at Filana. Those studies showed that simufilam reduced seizure frequency in a mouse model of focal onset seizures involving TSC-related pathology1. In a separate study conducted with the TSC Alliance Preclinical Consortium using a well-accepted TSC-knockout mouse model, simufilam attenuated the progression of seizure activity in a dose-dependent manner2.
Filana holds an exclusive worldwide license from Yale University to intellectual property supporting the use of simufilam in TSC-related epilepsy.
About TSC and TSC-Related Epilepsy
TSC is a rare genetic disorder resulting from a mutation in the TSC1 or TSC2 gene. These mutations affect the mechanistic target of rapamycin (mTOR) pathway and can cause tumors to grow in multiple organs3,4. Epilepsy is the most common health issue affecting the TSC community, with 80% to 90% of TSC patients experiencing seizures5. TSC-related epilepsy affects approximately 45,000 people in the U.S.1,3. Most patients start having seizures within their first year of life1. Even with multiple approved treatments, more than 60% of TSC patients remain refractory to antiepileptic therapy6.
About Filana Therapeutics, Inc.
Filana Therapeutics, Inc. (NASDAQ: FLNA), is a biotechnology company focused on developing novel, investigational therapies to modulate the filamin A protein for the treatment of central nervous system disorders, such as tuberous sclerosis complex (TSC)-related epilepsy, and other diseases associated with dysregulation or overexpression of filamin A.
For more information, please visit: https://www.FilanaTx.com.
References
- Zhang L, Huang T, Teaw S, Nguyen LH, Hsieh LS, Wong X, Burns LH, Bordey A. Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations. Science Translational Medicine. 2020;12(531):eaay0289. DOI: 10.1126/scitranslmed.aay0289
- Stansley B, Islam MM, Aguiar DJ, Fuchs Z, Catron M, Morairty S, et al. The small molecule simufilam dose-dependently attenuates the worsening of seizures in a mouse model of tuberous sclerosis complex. Epilepsia. 2026;00:1–13. DOI: 10.1002/epi.70227
- https://www.tscalliance.org/understanding-tsc/what-is-tsc/
- https://www.tscalliance.org/understanding-tsc/genetics/
- Crino P, Nathanson K, Henske EP. The tuberous sclerosis complex. N Engl J Med. 2006;355(13):1345-1356. DOI: 10.1056/NEJMra055323
- Chu-Shore CJ, Major P, Camposano S, Muzykewicz D, Thiele EA. The natural history of epilepsy in tuberous sclerosis complex. Epilepsia. 2010;51(7):1236–1241. DOI: 10.1111/j.1528-1167.2009.02474.x
For More Information Contact
Investors
Mike Moyer
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Filana Therapeutics
Eric Schoen, Chief Financial Officer
(512) 501-2450
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