Cassava Sciences Completes Interim Safety Review of Oral Simufilam Phase 3 Trials

Cassava Sciences, Inc., a biotechnology firm specializing in Alzheimer’s disease, has announced the completion of a third interim safety review of its drug candidate simufilam, currently in Phase 3 clinical trials. The Data and Safety Monitoring Board (DSMB), which routinely evaluates interim patient safety data, recommended that both ongoing Phase 3 studies proceed without modification.

Jim Kupiec, Chief Medical Officer at Cassava Sciences, expressed satisfaction with the completed safety review and anticipation for future announcements. "We look forward to announcing top-line efficacy, safety, and biomarker data for our 12-month Phase 3 study before the end of 2024," Kupiec said.

The DSMB, comprised of independent clinical research experts, is responsible for the interim safety assessment of the trials. It does not evaluate drug efficacy. Simufilam, a novel small molecule drug candidate, is being tested for the treatment of Alzheimer’s disease dementia. It is currently undergoing late-stage clinical evaluation in two pivotal Phase 3 trials, both of which are fully enrolled.

The first Phase 3 trial (NCT04994483) includes a 52-week treatment period with 804 Alzheimer’s patients randomized into the trial. Top-line results from this study are expected by the end of 2024. The second Phase 3 trial (NCT05026177) involves a 76-week treatment period with 1,125 patients randomized. Results from this study are anticipated around mid-2025.

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

This announcement follows prior interim safety MRI data released in October 2023, suggesting that simufilam is not associated with treatment-emergent amyloid-related imaging abnormalities (ARIA). In addition, the DSMB meetings in September 2023 and March 2024 recommended the continuation of both Phase 3 trials as planned. Final safety data are expected at the conclusion of the Phase 3 program.

Cassava Sciences, a clinical-stage biotechnology company based in Austin, Texas, focuses on detecting and treating neurodegenerative diseases, such as Alzheimer’s disease. Simufilam, their small molecule oral drug candidate, is designed to target a specific site on filamin A, a scaffolding protein essential for certain receptor interactions in the brain. The company believes that simufilam disrupts amyloid-β42 binding to brain receptors, potentially impacting the Alzheimer’s disease process. Cassava Sciences holds exclusive, worldwide rights to its investigational products and related technologies, with no royalty obligations to third parties.