Cassava Sciences Reports Positive Preclinical Study Evaluating Simufilam for TSC-Related Epilepsy
AUSTIN, Texas -- Cassava Sciences, Inc., a clinical-stage biotechnology company focused on developing novel treatments for central nervous system (CNS) disorders, today reported positive preclinical results of a study evaluating simufilam in a well-accepted mouse model of tuberous sclerosis complex (TSC)-related epilepsy.
Most individuals with TSC experience lifelong seizures associated with severe comorbidities, and nearly two-thirds of those patients are refractory to available treatments. Simufilam, a proprietary oral small molecule, demonstrated a beneficial effect on seizure activity with a positive dose response in this model. Importantly, the results are consistent with findings published in Science Translational Medicine of prior research conducted with simufilam at Yale School of Medicine in another mouse model. This new research further supports the potential use of simufilam as a novel, first-in-class treatment for TSC-related epilepsy.
The study was conducted in collaboration with the TSC Alliance and the TSC Preclinical Consortium using an animal model of TSC-related epilepsy, the Tsc conditional knockout (CKO) mouse line (Tsc1-CKO). These mice develop spontaneous seizures and are used by the TSC Alliance to evaluate the effectiveness and safety of novel and repurposed therapeutics in the potential treatment of TSC-related epilepsy. The study was conducted by PsychoGenics, Inc., the TSC Preclinical Consortium’s research partner.
The Tsc1-CKO mice were treated with several doses of simufilam. Seizure activity was monitored for approximately three weeks after onset, and simufilam was evaluated against treatment with vehicle alone. The data showed that simufilam attenuated the progression of seizure activity, with a statistically significant correlation between simufilam dose and the number of seizures by the end of the study. Not all parameters measured reached statistical significance. The Company intends to present data and analyses in an upcoming scientific conference and publication.
“We are excited that this independent study bolsters previous findings in a separate animal model of focal onset epilepsy. Based on these data, we believe that simufilam could represent a new approach to addressing the persistent unmet need in the TSC community,” said Angélique Bordey, PhD, Senior Vice President, Neuroscience of Cassava.
“Epilepsy is considered the most prevalent and challenging manifestation of TSC. Patients urgently need more treatment options. Positive results from two independent preclinical animal studies enhance our conviction as we prepare to advance simufilam to the clinic for this new indication,” said Rick Barry, President and Chief Executive Officer of Cassava. “We intend to initiate a proof-of-concept study of simufilam in TSC-related epilepsy in the first half of 2026.”
