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 aims to improve the lives of patients with conditions like 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, such as TSC. The company's lead therapeutic drug candidate, simufilam, is a proprietary small molecule oral treatment drug. Simufilam was discovered and designed in-house and has been characterized by academic collaborators since approximately 2008. It is being studied for the treatment of TSC-related epilepsy and 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, resulting in seizures in TSC patients. Filamin A represents a novel target for TSC-related epilepsy, independent of mTOR. Simufilam is a potential first-in-class filamin A-modulating agent. The company continues to explore 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.