Filana Outlines Simufilam TSC Epilepsy Rationale, Plans FDA Data Submission
Filana Therapeutics detailed the biological rationale for its investigational filamin A modulator simufilam in TSC-related epilepsy, highlighting mTOR pathway involvement and 60% of patients refractory to current antiepileptics. The company will submit additional preclinical data and protocol modifications to FDA to lift its clinical hold and expects an update soon.
1. Conference Presentation
At the Eilat XVIII meeting in Madrid, Filana presented the scientific rationale for simufilam in TSC-related epilepsy, emphasizing its modulation of filamin A and downstream effects on the mTOR pathway. The presentation underscored the high unmet need in TSC patients, with 80–90% experiencing seizures and over 60% remaining refractory to existing antiepileptic therapies.
2. FDA Hold Response and Next Steps
Filana is responding to the December 2025 FDA clinical hold by preparing additional preclinical studies and modifying its trial protocol. The company plans to submit this information in the coming months and anticipates sharing a program update once the FDA reviews its response.