Filana Therapeutics (FLNA) Investor presentation summary

Strategic focus and mission

• Pursuing development of simufilam, a novel filamin A modulator, for TSC-related epilepsy, building on preclinical and clinical data.

• Mission centers on transformative medicines for CNS disorders, especially those involving filamin A dysregulation.

• Expanded leadership and scientific team with expertise in neurology and CNS drug development.

Simufilam program and clinical development

• Simufilam targets filamin A, a novel mechanism independent of mTOR, aiming to reduce seizure frequency in TSC-related epilepsy.

• Proof-of-concept clinical study planned, pending FDA clearance after additional preclinical data and protocol modifications.

• Open-label, multi-center trial will assess safety, tolerability, pharmacokinetics, and seizure frequency changes.

• Over 1,200 subjects have been treated with simufilam in prior trials, showing a favorable safety profile.

Preclinical and scientific evidence

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

• Filamin A overexpression identified as a therapeutic target in TSC and FCDII; simufilam modulates this pathway.

• Simufilam demonstrated dose-dependent seizure reduction in TSCA mouse model.