Cassava Sciences Announces Positive End-of-Phase 2 Meeting with FDA and Outlines Pivotal Phase 3 Program for Simufilam in Alzheimer’s Disease
- Two Upcoming Phase 3 Studies and a Previously Completed Phase 2 Program Support a New Drug Application Filing for Simufilam in Alzheimer’s disease -
- Agreement Reached to Use ADAS-Cog as Co-Primary Efficacy Endpoint -
- Pivotal Phase 3 Program Remains On-track to be Initiated 2nd Half 2021 -
“For over 10 years we’ve been doing basic research and early drug development with simufilam,” said
“We appreciate the valuable guidance and flexibility FDA has provided,” added
Simufilam is a novel drug, discovered at
The EOP2 meeting took place mid-January. FDA attendees included
Official meeting minutes confirm that
Cassava Sciences’ pivotal Phase 3 clinical program consists of two double-blind, randomized, placebo-controlled studies, each described below.
Cassava Sciences’ first Phase 3 study is designed to evaluate disease-modifying effects of simufilam in Alzheimer’s disease. The goal is to demonstrate a slower rate of decline in cognition and health function in subjects treated with simufilam compared to placebo.
Details of the first Phase 3 study include:
- Approximately 1,000 subjects with mild-to-moderate Alzheimer’s disease to be enrolled.
- Subjects to be randomized (1:1:1) to simufilam 100 mg, 50 mg, or placebo BID.
- Subjects to be treated for 18 months.
- The co-primary efficacy endpoints are ADAS-Cog, a cognitive scale, and ADCS-ADL, a functional scale; both are widely used clinical tools in trials of Alzheimer’s disease.
- A secondary efficacy endpoint is iADRS, a widely used clinical tool in trials of Alzheimer’s disease that combines cognitive and functional scores from ADAS-Cog & ADCS-ADL.
- Other secondary endpoints include biomarkers of disease and NPI4, a clinical tool that assesses the presence and severity of dementia-related behavior.
- The Company plans to initiate the first pivotal Phase 3 study Q3 2021.
Cassava Sciences’ second Phase 3 study is designed to evaluate symptomatic improvement in Alzheimer’s disease. The goal is to demonstrate improved cognition and health function in subjects treated with simufilam compared to placebo.
Details of the second Phase 3 study include:
- Approximately 600 subjects with mild-to-moderate Alzheimer’s disease to be enrolled.
- Subjects to be randomized (1:1) to simufilam 100 mg or placebo BID.
- Subjects to be treated for 9 to 12 months.
- The co-primary efficacy endpoints are ADAS-Cog, a cognitive scale, and ADCS-ADL, a functional scale; both are widely used clinical tools in trials of Alzheimer’s disease.
- A secondary efficacy endpoint is iADRS, a widely used clinical tool in trials of Alzheimer’s disease that combines cognitive and functional scores from ADAS-Cog & ADCS-ADL.
- Other secondary endpoints include biomarkers of disease and NPI, a clinical tool that assesses the presence and severity of dementia-related behavior.
- The Company plans to initiate the second pivotal Phase 3 study Q4 2021.
FDA has provided further flexibility to
In addition to the planned pivotal Phase 3 clinical program, other clinical studies in support of simufilam’s safety and efficacy in Alzheimer’s disease are briefly described below.
Open-label Study
The Company plans to conduct a second interim analysis of the open-label study mid-year 2021, when approximately 50 subjects complete 12 months of drug treatment. Much like the first pre-planned interim analysis (6 months), the second pre-planned interim analysis (12 months) is expected to generate clinical data around long-term safety, cognition and dementia-related behavior.
Cognition Maintenance Study (CMS)
In Q2 2021,
Slide Deck
Cassava Sciences’ latest corporate presentation is available on its website under the Investors/Presentations page: https://www.CassavaSciences.com
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. As of 2020, there were approximately 50 million people worldwide living with dementia, a figure expected to increase to 150 million by 2050.5 The annual global cost of dementia is now above
About Simufilam
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
About
Cassava Sciences’ mission is to discover and develop innovations for chronic, neurodegenerative conditions. Over the past 10 years,
For More Information Contact:
eschoen@CassavaSciences.com
(512) 501-2450
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 an open-label study and Phase 2 program results; plans to conduct a second interim analysis of an open-label study and the timing thereof; planned enrollment and other changes to said open-label program; results of our EOP2 meeting with FDA including agreement on elements to support a New Drug Application filing for simufilam in Alzheimer’s disease; our intention to initiate a Phase 3 clinical program with simufilam and the timing, enrollment, duration and other details thereof; 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
This press release also contains information based on independent industry publications. We have not independently verified the accuracy or completeness of the information contained in these publicly available sources. Accordingly, we make no representations as to the accuracy or completeness of such information. You are cautioned not to give undue weight to such information.
1 ADAS-Cog = The Alzheimer’s Disease Assessment Scale – Cognitive Subscale, a measure of cognition
2 ADCS-ADL = Alzheimer’s Disease Cooperative Study – Activities of Daily Living, a measure of health function
3 iADRS = integrated Alzheimer’s Disease Rating Scale, a composite measure of cognition and health function
4 Neuropsychiatric Inventory (NPI)
5
https://www.alzint.org/about/dementia-facts-figures/dementia-statistics/
Source: Cassava Sciences, Inc.