Cassava Sciences announces the best results over 12 months
- Cognition scores improved by 3.2 points on ADAS-Cog, from baseline at month 12 (p
- Two independent biostatisticians analyzed changes in ADAS-Cog scores, from baseline to month 12
- No behavioral disturbance on NPI in more than 50% of study subjects by month 12
- Pivotal Phase 3 clinical program launch remains on track for Q4 2021, with special protocol evaluations from the FDA
AUSTIN, Texas, September 22, 2021 (GLOBE NEWSWIRE) – Cassava Sciences, Inc. (Nasdaq: SAVA) today announced key clinical data from a pre-planned interim analysis of an ongoing open label study with its candidate – drug simufilam in patients with mild to moderate Alzheimer’s disease.
In a study funded by the National Institutes of Health (NIH), ADAS-Cog11 scores improved an average of 3.2 points from baseline (p
“I feel energized and encouraged by the clinical data,” said Rémi Barbier, President and CEO. “We look forward to the launch of a pivotal randomized, double-blind, placebo-controlled Phase 3 clinical program with simufilam in people with Alzheimer’s disease. “
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 (SD ± 6.3; p
- 68% of study subjects improved on ADAS-Cog at 12 months; these study subjects improved an average of 6.8 points (SD ± 3.8)
- An additional 20% of study subjects lost less than 5 points on ADAS-Cog at 12 months; these study subjects decreased by an average of 2.5 points (SD ± 1.3)
Independent published meta-analysis of patients with mild to moderate Alzheimer’s disease reports an average decrease of 5.5 points over 12 months1 among study subjects who received placebo in randomized controlled trials.
Study subjects entered the open-label study with a clinical diagnosis of mild to moderate Alzheimer’s disease, a mini-mental state examination (MMSE) ranging from 16 to 26.
The drug is well tolerated. There were no serious drug-related adverse events during the 12-month interim analysis.
Chain of custody for clinical data
Investigation sites collect clinical data from study subjects. The sites enter their clinical data directly into an electronic data entry (EDC) system managed by an external data management provider. The data management provider also manages the clinical database. At Cassava Sciences’ request, the data management vendor forwarded the baseline and 12 month ADAS-Cog scores directly to two independent consultant biostatisticians for analysis. Both consultants hold doctorates in statistics and provide consulting expertise in support of medical research. One consultant is based in Texas, the other in Arizona. The two statisticians independently came to the same statistical conclusions about changes in ADAS-Cog scores from baseline to month 12.
Neuropsychiatric inventory (NPI) at 12-month interim analysis
Alzheimer’s disease is often accompanied by behavioral disturbances, such as anxiety, agitation or delusions. These may become more common as the disease progresses. The Neuropsychiatric Inventory (NPI) is a widely used clinical tool to measure behavior changes related to dementia. At baseline, 34% of these study subjects had no neuropsychiatric symptoms on NPI. At 12 months, more than 50% had no neuropsychiatric symptoms at NPI.
Clinical strategy around an open study
Long-term safety data is a regulatory requirement. To collect this data, some drug development companies are conducting an open label study at the end of a Phase 3 clinical trial program. Cassava Sciences believes it is prudent to conduct an open label study. before undertake a large, complex and expensive phase 3 clinical program in Alzheimer’s disease: if an investigational drug for Alzheimer’s disease does not show long-term safety or therapeutic benefit in a large, open-label study Such a large, well-designed drug is unlikely to be successful under the more rigorous conditions of a randomized controlled trial. However, the treatment effects seen in an open label study are not evidence of the safety or effectiveness of the drug, nor can open data predict the clinical success of a Phase 3 program. Evidence of safety and efficacy will always be based on the results of a pivotal, randomized, double-blind, placebo-controlled phase 3 clinical program, which has not yet been conducted with simufilam.
About the open study
In March 2020, Cassava Sciences launched a long-term, open-label study to evaluate simufilam in patients with Alzheimer’s disease. This study is funded by a research grant from the National Institutes of Health (NIH). The open-label study aims to monitor the long-term safety and tolerability of simufilam 100 mg twice daily for 12 months or more. Another goal of the study is to measure cognitive changes using ADAS-Cog and to assess the presence and severity of dementia-related behaviors using the Neuropsychiatric Inventory (NPI). Approximately 200 study subjects are now enrolled in the open study from 16 investigation sites in the United States and Canada. The study dropout rate is currently less than 10%.
Next step: Phase 3 clinical program under the FDA Special protocol evaluations
Cassava Sciences is advancing simufilam in a Phase 3 clinical program in Alzheimer’s disease. The start of the study is on track for the fourth quarter of 2021. On August 24, 2021, Cassava Sciences announced that it had reached an agreement with the United States Food and Drug Administration (FDA) for a special assessment. of the protocol (SPA) for its two phase 3 studies.
Simufilam (sim-uh-FILL-am) is a patented small molecule (oral) drug that restores normal form and function of altered filamin A (FLNA), a scaffold protein, in the brain. The alteration of FLNA in the brain disrupts the normal functioning of neurons, leading to Alzheimer’s disease, neurodegeneration and neuroinflammation. The science behind simufilam is published in peer-reviewed journals, including Journal of Neuroscience, Neurobiology of aging, Journal of Biological Chemistry, Neuroimmunology and Neuroinflammation and Journal of Alzheimer’s Disease Prevention. Simufilam is substantially supported by peer-reviewed research grants from the National Institutes of Health (NIH).
Cassava Sciences owns the worldwide development and commercial rights for its research programs in Alzheimer’s disease and related technologies, without royalty obligation to any third party.
About Cassava Sciences, Inc.
The mission of Cassava Sciences is to discover and develop innovations for chronic and neurodegenerative diseases. Over the past 10 years, Cassava Sciences has combined cutting edge technology with new knowledge in neurobiology to develop new solutions for Alzheimer’s disease. For more information, please visit: https://www.CassavaSciences.com.
For more information, contact:
Eric Schoen, Chief Financial Officer
Caution regarding forward-looking statements: This press release includes forward-looking statements, including, but not limited to, those regarding the timing of the launch of our pivotal Phase 3 program with simufilam in Alzheimer’s disease and its likelihood of success, interpretation 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 cognitive decline in people with Alzheimer’s disease and the oral or written comments of our collaborators regarding simufilam and its clinical development.
Drug development involves a high degree of risk, and historically only a small number of research and development programs result in a product being commercialized. Clinical results from our early stage clinical trials may not be indicative of complete results or the results of later stage or larger scale clinical trials and do not guarantee regulatory approval. You should not place undue reliance on these statements or the scientific data that we present or publish. These statements are based on our current expectations and projections regarding future events.
These statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including, but not limited to, risks associated with the launch, the conduct or completion of our clinical studies on time, to demonstrate the specificity, safety, efficacy or potential health benefits of our product candidates, the severity and duration of health care precautions health taking into account the COVID-19 pandemic, any unanticipated impacts of the pandemic on our business operations, including those described in the section titled “Risk Factors” in our Annual Report on Form 10-K for the fiscal year ended December 31, 2020 and future reports to be filed with the SEC. The foregoing sets out many factors, but not all, that could cause actual results to differ from the expectations of any forward-looking statement. In light of these risks, uncertainties and assumptions, the forward-looking statements and the events discussed in this press release are inherently uncertain and may not occur, and actual results could differ materially and adversely from those anticipated or implied in the statements. prospective. Therefore, you should not rely on forward-looking statements as predictions of future events. Except as required by law, we disclaim any intention or responsibility to update or revise any forward-looking statements contained in this press release.
The contents of this press release are at our sole responsibility and do not represent the official views of the National Institutes of Health (NIH).
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1 Progression of Meta-analysis Model in Alzheimer’s disease (Ito, et al., Pfizer Global Research), Alzheimer’s and dementia 6 (2010) 39-53