Filana Therapeutics (FLNA) H.C. Wainwright 27th Annual Global Investment Conference summary

Leadership and strategic direction

• New executive leadership was established in July 2024 following regulatory settlements and management changes.

• The board and management team now include experts in neuroscience and drug development, notably Dr. Angélique Bordey and Dr. Joe Houlihan.

• The company is focusing on developing simufilam for TSC-related epilepsy, shifting from its previous Alzheimer's program.

• Collaboration with the TSC Alliance and leading clinicians is central to the new strategy.

Clinical and preclinical program updates

• Two fully enrolled phase III Alzheimer's trials failed, prompting a pivot to TSC-related epilepsy.

• Preclinical studies in mouse models showed significant seizure reduction, with up to 30% of mice seizure-free and a dose-dependent response.

• The safety profile of simufilam remains strong, with no drug-related serious adverse events in over 1,200 patients.

• Proof-of-concept phase II-A trial in TSC-related epilepsy is planned for the first half of 2026.

Market opportunity and unmet need

• TSC affects about 50,000 patients in the US, with 80-90% experiencing epilepsy; over 60% do not achieve adequate seizure control with current drugs.

• Existing treatments (Afinitor, EPIDIOLEX) are effective for some but have tolerability issues and leave many patients with unmet needs.

• There is a clear commercial and humanitarian rationale for developing new therapies targeting the root cause of TSC-related epilepsy.