Cassava Sciences Announces Top-line Results of 12-month Interim Analysis from Open-label Study Evaluating Simufilam in Alzheimer’s Disease
- Cognition Scores Improved 3.2 Points on ADAS-Cog, Baseline to Month 12 (p<0.001)
- Two Independent Biostatisticians Analyzed Changes in ADAS-Cog Scores, Baseline to Month 12
- No Behavior Disorders on NPI in Over 50% Of Study Subjects at Month 12
- Initiation of Pivotal Phase 3 Clinical Program Remains On-track for Q4 2021, with Special Protocol Assessments from FDA
In a study funded by the
“I feel energized and encouraged by the clinical data,” said
In the first 50 study subjects who completed 12 months of open-label treatment with simufilam:
- ADAS-Cog11 scores improved an average of 3.2 points from baseline (S.D. ± 6.3; p<0.001)
- 68% of study subjects improved on ADAS-Cog at 12 months; these study subjects improved an average of 6.8 points (S.D. ± 3.8)
- An additional 20% of study subjects declined less than 5 points on ADAS-Cog at 12 months; these study subjects declined an average of 2.5 points (S.D. ± 1.3)
An independent, published meta-analysis of patients with mild-to-moderate Alzheimer’s disease reports an average decline of 5.5 points over 12 months1 amongst study subjects who were administered placebo in randomized, controlled trials.
Study subjects entered the open-label study with a clinical diagnosis of mild-to-moderate Alzheimer’s, Mini-Mental State Examination (MMSE) range 16-26.
Drug is well-tolerated. There are no drug-related serious adverse events through the 12-month interim analysis.
Chain of Custody for Clinical Data
Investigator sites collect clinical data from study subjects. Sites enter their clinical data directly into an electronic data capture (EDC) system managed by an outside data management vendor. The data management vendor also maintains the clinical database. At Cassava Sciences’ request, the data management vendor transmitted baseline and month-12 ADAS-Cog scores directly to two independent consulting biostatisticians for analysis. Both consultants hold PhD’s in statistics and provide consulting expertise in support of medical research. One consultant is based in
Neuropsychiatric Inventory (NPI) at the 12 Month Interim Analysis
Alzheimer’s is often accompanied by behaviors disorders, such as anxiety, agitation or delusions. These may become more frequent as disease progresses. The Neuropsychiatric Inventory (NPI) is a clinical tool widely used to measure changes in dementia-related behavior. At baseline, 34% of these study subjects had no neuropsychiatric symptoms on the NPI. At 12 months, over 50% had no neuropsychiatric symptoms on the NPI.
Clinical Strategy Around Open-label Study
Long-term safety data is a regulatory requirement. To collect these data, some drug development companies conduct an open-label study at the conclusion of a Phase 3 clinical testing program.
About the Open-label Study
Next Step: Phase 3 Clinical Program under FDA Special Protocol Assessments
Simufilam (sim-uh-FILL-am) is a proprietary, small molecule (oral) drug that restores the normal shape and function of altered filamin A (FLNA), a scaffolding protein, in the brain. Altered FLNA in the brain disrupts the normal function of neurons, leading to Alzheimer’s pathology, neurodegeneration and neuroinflammation. The underlying science for simufilam is published in peer-reviewed journals, including
Cassava Sciences’ mission is to discover and develop innovations for chronic, neurodegenerative conditions. Over the past 10 years,
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Cautionary Note Regarding Forward-Looking Statements: This press release includes forward looking statements including but not limited to those regarding the timing of the initiation of our pivotal Phase 3 program with simufilam in Alzheimer’s disease and its likelihood of success, the interpretation of clinical data generated in a 12-month interim analysis of an open-label study, the clinical safety profile of simufilam, the occurrence of neuropsychiatric symptoms in people with Alzheimer’s disease, the publication of an analysis regarding the expected rate of cognitive decline in people with Alzheimer’s disease and oral or written comments made by our employees regarding simufilam and its clinical development.
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1 Disease Progression Meta-analysis Model in Alzheimer’s disease (Ito, et al.,
Source: Cassava Sciences, Inc.