Cassava Presents Promising Preclinical Simufilam Data at TSC Alliance Meeting

Core Insights - Cassava Sciences, Inc. presented promising preclinical data on simufilam, showing a significant reduction in seizure activity in a mouse model, which supports its potential as a treatment for Tuberous Sclerosis Complex (TSC)-related epilepsy [1][3][4] - The company plans to initiate human clinical studies in the first half of 2026 to further evaluate simufilam's efficacy in treating TSC-related epilepsy [2][3][10] Preclinical Data - Simufilam reduced seizure frequency by 60% in a mouse model compared to vehicle-treated mice, with significant results (p<0.0001) [7][9] - The effective dose in mice was 20 mg/kg/day, and no adverse effects were observed at doses up to 50 mg/kg/day in rats and 1250 mg/kg/day in mice [7] - The treatment also led to a higher rate of seizure freedom in mice, with 11 out of 32 simufilam-treated mice achieving seizure freedom compared to 3 out of 29 vehicle-treated mice (p=0.0343) [9] Clinical Safety Data - Data from two Phase 3 studies involving 1,929 patients with mild-to-moderate Alzheimer's disease demonstrated a favorable safety profile for simufilam [11][15] - Non-serious adverse events were typically mild and not considered related to simufilam, with no serious adverse events assessed as study-drug related [15] Background on TSC - Tuberous Sclerosis Complex (TSC) affects approximately 50,000 patients in the US, leading to lifelong epilepsy and severe neurological issues [3][12] - Current treatments for TSC-related epilepsy are often ineffective, with nearly two-thirds of patients not responding to antiepileptic drugs [13][14] Future Directions - Additional preclinical studies are planned in collaboration with the TSC Alliance to explore simufilam's mechanism of action and its potential for treating TSC-related epilepsy [10]