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Filana Therapeutics Announces Publication in Cells of Promising Preclinical Findings for Simufilam in TSC2-Deficient Cells

Globe Newswire•28/10/2026•08:00 ET
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Key Highlights

  • ➤Simufilam reduced elevated ATF4 expression by approximately 37% in TSC2-deficient cells
  • ➤Simufilam restored impaired mitochondrial membrane potential in TSC2-deficient cells
  • ➤Simufilam did not significantly suppress mTORC1 signaling in the study
  • ➤Patient screening for planned Phase 2a study expected to begin by first quarter of 2027
  • ➤Filana’s planned TSC-related epilepsy study remains preclinical; clinical benefits are unproven

Expert Statements

Rick Barry, President and Chief Executive Officer of Filana Therapeutics

“The Cells publication provides additional scientific support for our continued investigation of simufilam in TSC-related epilepsy. Together with previously reported seizure-related findings in preclinical models(2)(,3), this new research strengthens the rationale for advancing the study of simufilam in TSC-related epilepsy. We are preparing for our planned Phase 2a study, with patient screening expected to begin by the first quarter of 2027.”

Angélique Bordey, PhD, Senior Vice President, Neuroscience at Filana Therapeutics and Yale School of Medicine researcher

“While mTORC1 hyperactivity is a central feature of TSC, other molecular and cellular dysfunctions may also contribute to the disease. These findings provide new insight into simufilam’s effects on cellular stress responses and mitochondrial function. Importantly, these effects were observed without significant suppression of mTORC1 signaling. Further studies in disease-relevant human neuronal models may help clarify the relevance of these findings to TSC-related epilepsy.”

Findings provide new insight into simufilam’s effects on cellular stress responses and mitochondrial function

New research demonstrates a 37% reduction in elevated ATF4, consistent with attenuation of the ATF4-associated cellular stress response

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