Cassava Sciences, Inc. (NASDAQ: SAVA) announced that the independent Data and Safety Monitoring Board (DSMB) has completed its third interim safety review of simufilam, the company's drug candidate for Alzheimer's disease. The DSMB recommended that the two ongoing Phase 3 trials continue without any modifications.
The recommendation comes after a routine, scheduled meeting where the DSMB, composed of independent clinical research experts, reviewed interim patient safety data. The board's assessment is focused solely on patient safety and does not include an evaluation of drug efficacy.
Phase 3 Trial Details
Simufilam is currently being evaluated in two pivotal Phase 3 trials. These trials are fully enrolled, with over 1,900 patients with mild-to-moderate Alzheimer’s disease participating across sites in the U.S., Puerto Rico, Canada, Australia, and South Korea.
The first Phase 3 trial (NCT04994483) involves 804 patients who were randomized to receive either simufilam or placebo over a 52-week treatment period. Top-line results from this trial are expected by the end of 2024.
The second Phase 3 trial (NCT05026177) includes 1,125 patients and has a 76-week treatment period, with top-line results anticipated around mid-year 2025.
Simufilam's Mechanism and Prior Findings
Simufilam is a novel, small molecule drug candidate designed to treat Alzheimer’s disease dementia. It targets a specific site on filamin A, a scaffolding protein believed to play a critical role in receptor interactions in the brain. Cassava Sciences believes simufilam can interrupt amyloid-β42 binding to receptors and potentially modify the course of Alzheimer's disease.
Interim safety MRI data, announced in October 2023, suggested that simufilam is not associated with treatment-emergent amyloid-related imaging abnormalities (ARIA), a known risk with some Alzheimer's therapies. Final safety data for simufilam are expected at the conclusion of the Phase 3 program.
Management Commentary
"We are pleased to have satisfactorily completed this safety review, the last before the expected top-line read out for our first Phase 3 study," said Jim Kupiec, Chief Medical Officer of Cassava Sciences. "We look forward to announcing top-line efficacy, safety and biomarker data for our 12-month Phase 3 study before the end of 2024."
Cassava Sciences is collaborating with Premier Research International, a global contract research organization (CRO), to conduct the Phase 3 program. The company retains exclusive worldwide rights to its investigational product candidates and related technologies.
