Filana Therapeutics (FLNA) Corporate presentation summary

Strategic focus and mission

• Focused on developing transformative medicines for CNS disorders, particularly TSC-related epilepsy, leveraging expertise in filamin A biology.

• Mission-driven approach to address diseases associated with dysregulation or overexpression of filamin A.

Simufilam development and scientific rationale

• Simufilam is a novel, oral, first-in-class filamin A modulator targeting TSC-related epilepsy, a condition affecting 40-45k people in the US.

• Preclinical studies in two mouse models demonstrated simufilam's ability to reduce seizure frequency and improve neuron morphology.

• Filamin A identified as a new therapeutic target in TSC-related epilepsy, independent of mTOR pathway.

• Simufilam's mechanism involves modulating filamin A, which regulates neuronal development and connectivity.

Clinical and preclinical progress

• Proof-of-concept clinical study planned, with protocol refined and product supply secured.

• Preclinical data from Yale and TSC Alliance mouse models support anti-seizure activity.

• Simufilam showed a 60% reduction in seizure frequency in the Yale mouse model and dose-dependent effects in the TSCA model.

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