Filana Therapeutics Announces Publication in Cells of Promising Preclinical Findings for Simufilam in TSC2-Deficient Cells
Yale researchers published preclinical findings on simufilam in the peer-reviewed journal Cells, showing a 37% reduction in elevated ATF4 and restored mitochondrial membrane potential in TSC2-deficient cells. The data suggest a mechanism distinct from direct mTOR inhibition, an established approach for TSC-associated seizures, and were funded by Alexion and the NIH without Filana funding.
Key figures
- Drug Name
- simufilam
- Phase Of Trial
- Phase 2a (planned)
- Atf4 reduction
- 37%
- Atf4 reduction pvalue
- 0.03
- Patient screening start
- Q1 2027
- Tsc epilepsy us patients
- approximately 45,000
- Mitochondrial membrane potential pvalue
- 0.007
AI analysis
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