On 29 July, the biopharmaceutical company Cassava Sciences announced positive results from an interim analysis of its open-label study of simufilam in Alzheimer’s disease (AD). Simufilam is a small molecule drug targeting the altered form of filamin A found in the AD brain to restore its normal shape and function. The open-label study is evaluating the safety and tolerability of simufilam (100 mg twice-daily) for 12 months or longer in people with mild to moderate AD. This study was initiated in March 2020 and has completed its target enrolment of 150 participants.
The pre-planned interim analysis was conducted with data of the first 50 participants who have completed 9 months of drug treatment. Results showed that participants who received simufilam had improved cognition and behaviour scores. Simufilam improved cognition scores 3.0 points on ADAS-Cog11, an 18% mean improvement, baseline to month 9 and reduced dementia-related behavior on the Neuropsychiatric Inventory (NPI) test. The company reported that over 50% of study participants had no neuropsychiatric symptoms at month 9, compared to 34% at baseline. In addition, simufilam was found to be safe and well-tolerated. Adverse events were mild and transient. Cassava Sciences plans to initiate a Phase III clinical program with simufilam in people with mild to moderate AD.