Cassava Sciences’ Simufilam Improves Cognition and Behavior in Alzheimer’s Disease in Interim Analysis of Open-label Study
- Patients’ Cognition Improved 1.6 Points on ADAS-Cog11 -
- Patients’ Behavior Improved 1.3 Points on NPI -
- Improvements Maintained at 6 Months -
- Results Support Advancing Simufilam into Phase 3 Clinical Program -
In a clinical study funded by the
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
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
About the Interim Analysis
Cassava 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
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.
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.
About Alzheimer's Disease
Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking skills. Currently, there are no drug therapies to halt Alzheimer’s disease, much less reverse its course. In the
Simufilam 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
Simufilam and SavaDx were both developed in-house. Both product candidates are substantially funded by peer-review research grant awards from the
Cassava Sciences’ mission is to discover and develop innovations for chronic, neurodegenerative conditions. Over the past 10 years,
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Cassava Sciences’ open-label study of simufilam in Alzheimer’s disease is funded by clinical research grant #AG065152 from the
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Cassava Sciences Safe Harbor
This news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, relating to: our strategy and plans; the treatment of Alzheimer’s disease; the status of current and future clinical studies with simufilam, including the interpretation of an interim analysis of open-label study results; inherent limitations of the ADAS-Cog and NPI testing batteries; planned enrollment and other changes to the open-label program; our intention to initiate a Phase 3 clinical program with simufilam in 2nd half 2021; results of our EOP2 meeting with FDA and the timing of further announcements; verbal commentaries made by our employees; and potential benefits, if any, of the our product candidates. These statements may be identified by words such as “may,” “anticipate,” “believe,” “could,” “expect,” “forecast,” “intend,” “plan,” “possible,” “potential,” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Our clinical results from earlier-stage clinical trials may not be indicative of full results or results from later-stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements or any scientific data we present or publish.
Such statements are based largely on our current expectations and projections about future events. Such statements speak only as of the date of this news release and are subject to a number of risks, uncertainties and assumptions, including, but not limited to, those risks relating to the ability to conduct or complete clinical studies on expected timelines, to demonstrate the specificity, safety, efficacy or potential health benefits of our product candidates, the severity and duration of health care precautions given the COVID-19 pandemic, any unanticipated impacts of the pandemic on our business operations, and including those described in the section entitled “Risk Factors” in our Annual Report on Form 10-K for the year ended
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1 Source: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/020690s042,021720s014,022568s011lbl.pdf (2018)
2 Source: Biogen, EMERGE Phase III study, slide 24, https://investors.biogen.com/static-files/8e58afa4-ba37-4250-9a78-2ecfb63b1dcb (2020)
3 Source: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/020690s042,021720s014,022568s011lbl.pdf (2018)
4, 5 Source: Alzheimer’s Association. Disease Facts and Figures. https://www.alz.org/media/documents/alzheimers-facts-and-figures-2019-r.pdf
Source: Cassava Sciences, Inc.