Filana Therapeutics, Inc.

Filana Therapeutics, Inc. is a clinical-stage biotechnology company based in Austin, Texas, focused on developing medicines for central nervous system (CNS) disorders. The company's mission is to improve the lives of patients with conditions such as Tuberous Sclerosis Complex (TSC)-related epilepsy and other diseases linked to the dysregulation or overexpression of filamin A. Their scientific approach involves modulating the activity of a critical brain protein for patients with certain CNS disorders, including TSC. The company's lead therapeutic drug candidate, simufilam, is a proprietary small molecule oral treatment drug. Simufilam was under clinical evaluation for Alzheimer's disease in two Phase 3 clinical studies through 2024, but all ongoing clinical trials for simufilam in Alzheimer's disease were discontinued, with the phase out of this development program completed in the second quarter of 2025. Simufilam is currently being studied for the treatment of TSC-related epilepsy. Discovered and designed in-house, simufilam has been characterized by academic collaborators since approximately 2008. It is intended to modulate the activity of filamin A, a protein that regulates neuronal cell development. Published studies by the company and its collaborators indicate that mTOR activation combined with filamin A overexpression leads to cell overgrowth, abnormal connectivity, and brain malformations, causing seizures in TSC patients. Filamin A is a novel target for TSC-related epilepsy, independent of mTOR, and simufilam is a potential first-in-class filamin A-modulating agent for this condition. The company also explores other potential indications for simufilam, both CNS-related and otherwise, where preclinical experiments suggest a benefit. Filana Therapeutics, Inc. owns or controls worldwide rights to its drug development program.