Cassava Sciences's (SAVA) Simufilam Improves Cognition and Behavior in Alzheimer’s Disease in Interim Analysis of Open-label Study

February 2, 2021 8:31 AM EST

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Cassava Sciences, Inc. (Nasdaq: SAVA) today announced results of an interim analysis from an open-label study of simufilam, its lead drug candidate for the treatment of Alzheimer’s disease. Patients’ cognition and behavior scores both improved following six months of simufilam treatment, with no safety issues.

In a clinical study funded by the National Institutes of Health and conducted by Cassava Sciences, six months of simufilam treatment improved cognition scores by 1.6 points on ADAS-Cog11, a 10% mean improvement from baseline to month 6. In these same patients, simufilam also improved dementia-related behavior, such as anxiety, delusions and agitation, by 1.3 points on the Neuropsychiatric Inventory, a 29% mean improvement from baseline to month 6.

Alzheimer’s is a progressive disease. Over time, a patient’s cognition will always worsen. “Experience based on longitudinal studies of ambulatory patients with mild to moderate Alzheimer’s disease suggest that scores on ADAS-cog decline by 6 - 12 points per year”, according to FDA’s Prescription Information sheet for ARICEPT® (donepezil), a drug approved for the treatment of dementia of the Alzheimer’s type1.“We could not be more pleased with these interim results,” said Remi Barbier, President & CEO. “We would have been satisfied to show simufilam stabilizes cognition in patients over 6 months. An improvement in cognition and behavior tells us this drug candidate has potential to provide lasting treatment effects for people living with Alzheimer’s disease. It’s an exciting development.”

The safety profile of simufilam in the interim analysis was consistent with prior human studies. There were no drug-related serious adverse events. Adverse events were mild and transient.

“Today’s data once again suggests simufilam could be a transformative, novel therapeutic,” added Nadav Friedmann, PhD, MD, Chief Medical Officer. “It appears the drug’s unique mechanism of action has potential to provide a treatment benefit following 6 months of dosing.”

About the Interim AnalysisCassava Sciences’ on-going, one-year, open-label, multi-center study is evaluating the long-term safety and tolerability of simufilam 100 mg twice daily in 100 patients with mild-to-moderate Alzheimer’s disease. This study was initiated March 2020 and is now approximately 80% enrolled. Today’s pre-planned interim analysis summarizes clinical data at the midway point of enrollment, i.e., the first 50 patients who have completed at least 6 months of drug treatment.

ADAS-Cog (Alzheimer's Disease Assessment Scale-Cognitive Subscale) is a standard test for assessing changes in cognition in Alzheimer’s disease trials. NPI (Neuropsychiatric Inventory) is a widely used tool for measuring changes in dementia-related behavior. The Mini-Mental State Exam (MMSE) is a widely used test of cognitive function among the elderly. The interim analysis shows mean baseline scores of 15.5 on ADAS-Cog11, 4.5 on NPI and 22.1 on MMSE.

Much of the value of the open-label study is to gain data to support simufilam’s long-term safety profile in patients. Interim efficacy data from an open-label study has limitations compared to efficacy data from a fully completed, large, randomized controlled clinical trial, or from a fully enrolled open-label study. However, prior clinical research in Alzheimer’s disease conducted by other sponsors can serve as a contextual reference for estimates of an expected rate of decline in cognition in placebo patients:

  • In 2019, a randomized controlled trial of aducanumab (Biogen) was conducted in >1,000 patients with early Alzheimer’s disease.2 In this Phase 3 study (EMERGE), patients on placebo showed a mean decline in cognition of approximately 1.4 points on ADAS-Cog13, a 6.3% decline, from baseline to month 6. Mean baseline ADAS-Cog13 score was 22.2. Mean baseline MMSE was 26.4.
  • A randomized controlled study of ARICPET® (donepezil, Eisai) was conducted in >400 patients with mild-to-moderate Alzheimer’s disease.3 In this Phase 3 study, patients on placebo showed a mean decline in cognition of approximately 1.9 points on ADAS-Cog, a 7.3% decline, from baseline to week 24. Mean baseline ADAS-Cog score was 26. MMSE range was 10-26.

Next StepsCassava Sciences believes today’s data and prior clinical results support advancing simufilam into a Phase 3 clinical program in Alzheimer’s disease. Initiation of a Phase 3 trial remains on schedule for 2nd half 2021.

Cassava Sciences and the U.S. Food and Drug Administration (FDA) recently concluded a successful end-of-phase 2 (EOP2) meeting for the simufilam drug development program. Details of the EOP2 meeting will be announced Q1 2021 after official FDA meeting minutes are finalized.

Based on today’s results and inbound demand from Alzheimer’s patients and their caregivers, the enrollment target for the open-label study will be increased by up to 50 additional patients, to a total target of approximately 150 patients. The Company is also in discussions with its scientific and clinical advisors about other potential enhancements to the open-label program.

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