Filana Therapeutics (FLNA) Corporate presentation summary

Mission and leadership

• Focused on developing transformative medicines for CNS disorders, including TSC-related epilepsy, by targeting filamin A dysregulation.

• Leadership team includes experienced executives and scientific advisors, such as Dr. Angélique Bordey and Dr. Joseph Hulihan.

Simufilam development and scientific rationale

• Simufilam is a novel, oral, first-in-class filamin A modulator with a strong safety profile from over 2,000 patients in prior trials.

• Preclinical studies in two mouse models (Yale and TSCA) show simufilam reduces seizure frequency and improves neuron morphology.

• Simufilam targets filamin A, a protein implicated in abnormal brain development and seizures in TSC, independent of mTOR pathways.

• Patent for simufilam's use in TSC-related epilepsy was issued in 2025 and licensed from Yale.

TSC-related epilepsy background and market

• TSC is a genetic disorder affecting multiple organs, with epilepsy as the most common neurological manifestation.

• 80–90% of TSC patients experience seizures, and over 60% are refractory to current antiepileptic therapies.

• U.S. prevalence of TSC is about 50,000, with 40–45k affected by epilepsy.

• Current FDA-approved therapies for TSC-related epilepsy are limited, highlighting unmet medical need.