Sumifilam

Data show that simufilam reduced seizure activity in a preclinical mouse model Presentation also highlights favorable human safety data from 1,929 patient Phase 3 program June 30, 2025 -- Cassava Sciences, Inc. (NASDAQ: SAVA, “Cassava”, the “Company”), a clinical-stage biotechnology company focused on developing novel, investigational treatments for central nervous system (CNS) disorders, today announced that the Company presented a poster at the TSC International Research Conference (TSC 2025) held June 26-28, 2025 in Bethesda, MD. The poster, available on the Company website via this link, detailed two important observations that support Cassava’s plan to initiate human clinical studies in H1 2026 to evaluate simufilam as a treatment for Tuberous Sclerosis Complex (TSC)-related epilepsy: Preclinical proof-of-concept: Treatment with simufilam alleviated neuronal abnormalities and reduced seizure frequency by 60% compared to vehicle in a mouse model of focal onset seizures. Clinical safety: Data from two human Phase 3 studies in Alzheimer’s disease demonstrated a favorable safety profile. “My research and collaboration with the TSC Alliance demonstrate our commitment to address the high unmet need in people with TSC-related epilepsy. We are excited to share this research with the TSC community, including the preclinical data showing that simufilam significantly reduced seizure frequency. Additional pre-clinical studies are underway to further confirm these promising findings,” said Angélique Bordey, PhD, Senior Vice President, Neuroscience of Cassava. “TSC leads to lifelong epilepsy and severe neurological issues for the approximately 50,000 patients in the US living with this rare disease. The data we presented at TSC 2025 mark the Company’s first presentation of simufilam’s positive results in a preclinical model. These compelling preclinical data build on favorable safety findings from prior human clinical studies and support our plan to begin our first clinical study in TSC-related epilepsy in the first half of 2026,” said Rick Barry, President and Chief Executive Officer of Cassava. Title: Simufilam, a small molecule, reduced seizure activity in a mouse model of focal cortical malformations and has demonstrated a favorable clinical safety profile. Brief Background: Tuberous sclerosis complex (TSC)-related epilepsy is a genetic disorder of the intracellular signaling pathway “mechanistic target of rapamycin” or mTOR, caused by mutations in the TSC1 or TSC2 gene. The presence of focal malformations in the brains of TSC patients is associated with lifelong epilepsy, leading to severe neurological issues. Recent evidence shows that expression of filamin A is increased in patients with TSC and in mouse models that recapitulate TSC and a related mTOR disorder, focal cortical dysplasia type II. Simufilam is a proprietary, investigational oral small molecule that is hypothesized to modulate the function of the filamin A protein. Studies conducted in the lab of Angélique Bordey (the “Bordey lab”), at Yale School of Medicine, form the basis of the preclinical data. The Bordey lab showed that treating mice with simufilam, before or after seizure onset, alleviated neuronal abnormalities and reduced seizure frequency by 60% compared to vehicle-treated mice: Simufilam significantly reduced seizure frequency (pSignificantly more mice achieved seizure freedom with simufilam (3/29 vehicle (saline) treated mice v. 11/32 simufilam treated mice, p=0.0343). While the effective dose in mice was 20 mg/kg/day, chronic oral toxicology studies showed no adverse effects at doses up to 50 mg/kg/day in rats and 1250 mg/kg/day in mice. Looking to the future, additional preclinical studies are underway with the TSC Alliance preclinical consortium and other collaborators to explore further simufilam's mechanism of action and its potential to treat TSC-related epilepsy. Data from two Phase 3 studies of simufilam in 1,929 patients with mild-to-moderate Alzheimer’s disease were also presented in the poster: Simufilam demonstrated a favorable safety profile. Non-serious adverse events were typically mild and not considered simufilam-related. None of the serious adverse events in these studies, or any other study, were assessed to be study-drug related. Simufilam is a proprietary, investigational oral small molecule that is hypothesized to modulate the function of the filamin A protein. Tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II are neurodevelopmental disorders caused by mutations in the mechanistic target of rapamycin (mTOR) pathway genes. These mutations lead to focal malformations of the developing cortex and seizures in 80% to 90% of patients. Nearly two-thirds of TSC patients do not respond to antiepileptic drugs and experience lifelong seizures, leading to a spectrum of neurocognitive and psychological disabilities and poor quality of life. Current treatments, including antiepileptic drugs, mTOR analogs, and surgery, are not fully effective, are associated with serious adverse events, and/or are invasive.1 Cassava is initially focused on developing simufilam as a potential treatment for TSC-related seizures. According to the TSC Alliance, the disorder affects an estimated 1 in 6,000 live births. Approximately 50,000 people in the United States and more than one million worldwide live with TSC2. Resources: Science Translational Medicine. 2020 Feb 19: https://pubmed-ncbi-nlm-nih-gov.libproxy1.nus.edu.sg/32075941/ Cassava Sciences, Inc. (NASDAQ: SAVA), is a clinical-stage biotechnology company focused on developing novel, investigational treatments, including simufilam, for central nervous system disorders, such as tuberous sclerosis complex (TSC)-related epilepsy, and potentially for additional pipeline indications. Simufilam is a proprietary, investigational oral small molecule that is hypothesized to modulate the function of the filamin A protein. The Company is based in Austin, Texas. The content above comes from the network. if any infringement, please contact us to modify.