Filana Therapeutics Announces Publication in Epilepsia of Preclinical Simufilam Data
- Study in a well-accepted mouse model of severe
Tuberous Sclerosis Complex (TSC) showed that simufilam attenuated the progression of seizures in a dose-dependent manner - Results confirm and extend previously published preclinical findings
- TSC-related epilepsy affects approximately 45,000 people in the
U.S.
The study was led by
This dose escalation study evaluated simufilam’s ability to slow the worsening of seizures in the Tsc1 conditional knockout mouse (Tsc1-cKO)3, 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 formal publication of these data in Epilepsia supports our previously reported findings and reinforces the biological rationale for studying simufilam in TSC-related epilepsy,” said
“While we continue to address FDA’s questions related to the clinical hold, the publication of our data in Epilepsia adds to the biological rationale behind simufilam in TSC-related epilepsy,” said
Key Takeaways
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
Figure 3C from the article is a heatmap comparing the number of seizures per day recorded for each mouse in the study. Mice were administered vehicle or simufilam at one of three ascending doses.
Epilepsia paper, Figure 3C
Heatmap of the daily number of seizures per mouse during EEG recordings in each condition. EEG monitoring spanned 20 days, but seizure counts were quantified for the first 5 and last 10 days, and comparative analysis was performed between the first and last 5 days. Gray squares: Mice had died. The 5 day-gap indicates that analysis was not performed for this period.
TSC Program Status
One of the models offered by the
About
About TSC and TSC-related Epilepsy
TSC is a rare genetic disorder resulting from a mutation in the TSC1 or TSC2 gene. This affects the mechanistic target of rapamycin (mTOR) pathway and can cause tumors to grow in multiple organs4. 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
About Filana Therapeutics, Inc.
For more information, please visit: https://www.FilanaTx.com
References:
- 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. https://doi.org/10.1002/epi.70227
- 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. Sci. Transl. Med. (2020) 12(531). https://www.science.org/doi/10.1126/scitranslmed.aay0289
- https://www.tscalliance.org/understanding-tsc/what-is-tsc/
- https://www.tscalliance.org/researchers/preclinical-research/
- Crino P, Nathanson K,
Petri Henske ,E. The Tuberous Sclerosis Complex . N Engl J Med. (2006) 355 (13):1345-56. DOI: 10.1056/NEJMra055323 - Chu-Shore, C. J., Major, P., Camposano, S., Muzykewicz, D., & Thiele,
E. A . (2010). The natural history of epilepsy in tuberous sclerosis complex. Epilepsia. 2010; 51(7): 1236–1241. https://doi.org/10.1111/j.1528-1167.2009.02474.x
For More Information Contact:
Investors
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Cautionary Note Regarding Forward-Looking Statements:
This news release contains forward-looking statements that may include but are not limited to statements regarding: our ability to successfully engage with, and satisfactorily respond to, requests for additional information from the
Such statements are based on our current expectations and projections about future events. Such statements speak only as of the date of this news release and are subject to a number of risks, uncertainties and assumptions, including, but not limited to, those risks relating to our ability to provide FDA with additional information, including additional pre-clinical data, and modifying the protocol design proposed clinical trial, to satisfy completion of FDA’s review and release of full clinical hold, the ability to advance preclinical studies related to TSC-related epilepsy, and other potential indications, the ability to successfully carry out the Company’s obligations under the Yale License Agreement, the ability to initiate an initial proof-of-concept study of simufilam in TSC-related epilepsy, and other risks inherent in drug discovery and development or specific to Filana Therapeutics, Inc., as described in the section entitled “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2025 and subsequent reports to be filed with the SEC. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from expectations in any forward-looking statement. In light of these risks, uncertainties and assumptions, the forward-looking statements and events discussed in this news release are inherently uncertain and may not occur, and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. Accordingly, you should not rely upon forward-looking statements as predictions of future events. Except as required by law, we disclaim any intention or responsibility for updating or revising any forward-looking statements. For further information regarding these and other risks related to our business, investors should consult our filings with the SEC, which are available on the SEC's website at www.sec.gov.
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Epilepsia paper, Figure 3C
Heatmap of the daily number of seizures per mouse during EEG recordings in each condition. EEG monitoring spanned 20 days, but seizure counts were quantified for the first 5 and last 10 days, and comparative analysis was performed between the first and last 5 days. Gray squares: Mice had died. The 5 day-gap indicates that analysis was not performed for this period.
Source: Filana Therapeutics, Inc.