• 1,929 patients randomized in a pair of Phase 3 trials to evaluate oral simufilam in Alzheimer's disease dementia.
• Top-line results for on-going, 52-week Phase 3 trial expected approximately year-end 2024.
• Top-line results for on-going, 76-week Phase 3 trial expected approximately mid-year 2025.

Simufilam is Cassava Sciences’ proprietary oral drug candidate. This investigational drug binds to altered filamin A protein in the brain and restores its normal shape and function. By targeting altered filamin A, simufilam may help patients with Alzheimer’s achieve better health outcomes.

“We completed target enrollment for our Phase 3 program in approximately two years,” said Remi Barbier, President and CEO. “This achievement exemplifies our unwavering commitment to develop a new treatment option for Alzheimer’s. We are so thankful to the patients and their families, clinical investigators and operational partners who are helping us reach this goal.”

A total of 1,929 patients were randomized in two on-going Phase 3 trials of simufilam. These are registrational studies (aka, pivotal), meaning if positive data and results are generated, they can support the filing of a new drug application (NDA) for simufilam in Alzheimer’s disease. Both Phase 3 studies received a Special Protocol Assessment (SPA) from the FDA.

The first Phase 3 trial (NCT04994483) has a 52-week treatment period; 804 Alzheimer’s patients were randomized into this study, as announced in October 2023. Top-line results for the 52-week Phase 3 study are currently expected approximately year-end 2024.

The second Phase 3 trial (NCT05026177) has a 76-week treatment period; 1,125 Alzheimer’s patients were randomized into this study. Top-line results for the 76-week Phase 3 study are currently expected approximately mid-year 2025.

Patients with mild-to-moderate Alzheimer’s disease dementia who met study eligibility criteria were recruited into the Phase 3 program from clinical sites in the U.S., Puerto Rico, Canada, Australia and South Korea. Cassava Sciences is conducting its on-going Phase 3 program in collaboration with Premier Research International, a global contract research organization (CRO).

Today’s news follows recently announced interim safety MRI data that suggests simufilam is not associated with treatment-emergent amyloid-related imaging abnormalities (ARIA). In addition, a September 2023 meeting of a Data and Safety Monitoring Board (DSMB) recommended that both Phase 3 studies of simufilam continue as planned, without modification. Final safety data are expected at the conclusion of the Phase 3 program.