Core Viewpoint - Cassava Sciences, Inc. reported positive preclinical results for simufilam in treating tuberous sclerosis complex (TSC)-related epilepsy, indicating its potential as a novel treatment option [1][2][6] Company Summary - Cassava Sciences is a clinical-stage biotechnology company focused on developing treatments for central nervous system disorders, including TSC-related epilepsy [13] - Simufilam is a proprietary oral small molecule believed to modulate the activity of the filamin A protein, which is crucial for neuronal development [13] - The company plans to initiate a proof-of-concept study for simufilam in TSC-related epilepsy in the first half of 2026 [6][9] Research Findings - The study utilized the Tsc1 conditional knockout (CKO) mouse model, which is recognized for evaluating the effectiveness of therapeutics for TSC-related epilepsy [3][4] - Simufilam demonstrated a beneficial effect on seizure activity, showing a statistically significant correlation between the dose of simufilam and the number of seizures [4] - The results align with previous findings from Yale School of Medicine, reinforcing the potential of simufilam as a first-in-class treatment for TSC-related epilepsy [2][5] Industry Context - Tuberous sclerosis complex affects approximately 50,000 individuals in the US, with epilepsy being the most common condition associated with TSC [12] - Between 80% and 90% of TSC patients experience seizures, and two-thirds have refractory epilepsy, highlighting the urgent need for new treatment options [12] - Current therapies are not fully effective and can lead to serious adverse events, emphasizing the potential market opportunity for simufilam [12]