
Cassava Sciences Reports Topline Phase 3 REFOCUS-ALZ Data
Simufilam did not show a significant reduction in co-primary endpoints of cognitive or functional decline versus placebo in patients with mild-to-moderate Alzheimer's disease
Simufilam continued to demonstrate an overall favorable safety profile
Cassava's Alzheimer's disease development program with simufilam will be completely discontinued by the end of Q2 2025
AUSTIN, Texas, March 25, 2025 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (NASDAQ: SAVA, 'Cassava', the 'Company'), a clinical-stage biotechnology company focused on developing a novel, investigational treatment for central nervous system disorders, including Alzheimer's disease (AD) dementia and tuberous sclerosis complex (TSC)-related epilepsy, today shared topline results from the Phase 3 REFOCUS-ALZ study of simufilam in mild-to-moderate AD.
Topline data indicate that REFOCUS-ALZ did not meet each of the prespecified co-primary, secondary and exploratory biomarker endpoints. The co-primary endpoints were the change in cognition and function from baseline to the end of the double-blind treatment period at week 76, assessed by the ADAS-COG12 and ADCS-ADL scales, comparing simufilam to placebo. REFOCUS-ALZ enrolled 1,125 patients and was discontinued on November 25, 2024, following the report that a prior 52-week Phase 3 study, RETHINK-ALZ, did not meet its co-primary endpoints. A large portion of subjects enrolled in REFOCUS-ALZ completed their final study visit prior to the termination of the trial. Simufilam continued to demonstrate an overall favorable safety profile.
'We are disappointed that the results of REFOCUS-ALZ and RETHINK-ALZ showed no treatment benefit for patients with mild-to-moderate Alzheimer's disease. These results were unambiguous. Working with patients, their families and their caregivers has brought a special dignity to our Phase 3 Alzheimer's disease clinical trial program and to each of us at Cassava. We are deeply grateful for the dedication and committed efforts of study investigators and site teams, who enabled us to conduct these trials with integrity and scientific rigor and whose efforts provided a clear data read out,' said Rick Barry, President and Chief Executive Officer of Cassava.
'Cassava will discontinue all efforts to develop simufilam for Alzheimer's disease and we expect to phase out the program by the end of Q2 2025,' continued Mr. Barry. 'We remain dedicated to our mission of developing novel medicines for central nervous system disorders. While we have initiated preclinical studies to evaluate simufilam's potential as a treatment for TSC-related epilepsy, we maintain ongoing strategic expense management efforts.'
Eric Schoen, Chief Financial Officer of Cassava commented, 'We remain focused on the interests of Cassava shareholders and are committed to enhancing shareholder value. Cassava is well-capitalized with approximately $128.6 million in cash and cash equivalents as of December 31, 2024.'
Summary Study Results:
Primary Endpoint Data
Co-Primary Endpoints
LS means change from baseline to the end of the double-blind treatment period
N=372 N=376 N=372
ADAS-COG12 (±SE) 4.97 (± 0.46) 4.70 (± 0.46) 0.27 (± 0.63) P=0.67
5.26 (± 0.46) 4.70 (± 0.46) 0.56 (± 0.63) P=0.37
N=373 N=376 N=373
ADCS-ADL (±SE) - 6.27 (± 0.57) - 5.32 (± 0.57) - 0.95 (± 0.79) P=0.23
- 6.43 (± 0.57) - 5.32 (± 0.57) - 1.10 (± 0.79) P=0.16
*Based on the intent-to-treat population
BID = twice daily
ADAS-COG12 = The Alzheimer's Disease Assessment Scale – Cognitive Subscale (a lower number represents less cognitive impairment)
ADCS-ADL = Alzheimer's Disease Cooperative Study – Activities of Daily Living (a higher number represents less functional impairment)
Safety Data:
The table below provides a high-level summary of the patient demographic and safety data. Simufilam continued to demonstrate an overall favorable safety profile.
Metrics for Simufilam and Placebo Simufilam 100 mg BID Simufilam 50 mg BID Placebo BID
Baseline*
N=374 N=376 N=375
Age, mean (SD), in years 73.6 ± 8.2 74.5 ± 7.6 73.7 ± 7.9
Sex, n (%) female 208 (55.6%) 207 (55.1%) 214 (57.1%)
MMSE Score (No.%,)
21-27 240 (64.2%) 242 (64.4%) 235 (62.7%)
16-20 134 (35.8%) 134 (35.6%) 138 (36.8%)
Race/Ethnicity
White 326 (87.2%) 326 (86.7%) 313 (83.5%)
Black 17 (4.5%) 23 (6.1%) 21 (5.6%)
Asian 28 (7.5%) 21 (5.6%) 32 (8.5%)
Other 3 (0.8%) 6 (1.6%) 9 (2.4%)
Safety**
N=374 N=376 N=373
Any Adverse Event (AE) 286 (76.5%) 288 (76.6%) 282 (75.6%)
Serious AEs 43 (11.5%) 61 (16.2%) 45 (12.1%)
Death 2 (0.5%) 6 (1.6%) 3 (0.8%)
AEs leading to discontinuation from the study 32 (8.6%) 34 (9.0%) 17 (4.6%)
Most Frequent AEs ≥ 5.0%
1: COVID-19 45 (12.0%) 49 (13.0%) 40 (10.7%)
2: Urinary Tract Infection 32 (8.6%) 41 (10.9%) 34 (9.1%)
3: Fall 32 (8.6%) 43 (11.4%) 51 (13.7%)
4: Dizziness 26 (7.0%) 11 (2.9%) 23 (6.2%)
5: Diarrhea 14 (3.7%) 19 (5.1%) 15 (4.0%)
*Based on the intent-to-treat population
**Based on the safety population
BID = twice daily
AD = Alzheimer's disease
MMSE = Mini-Mental State Examination
About REFOCUS-ALZ
REFOCUS-ALZ (NCT05026177) is a Phase 3 trial designed as a multi-center, double-blinded, placebo-controlled, randomized parallel group study to evaluate the safety and efficacy of two doses of simufilam compared to a placebo in a study involving over 75 clinical trial sites in the U.S., Canada, Puerto Rico and South Korea. The clinical trial sites that conducted REFOCUS-ALZ were completely distinct from the clinical trial sites that conducted RETHINK-ALZ. REFOCUS-ALZ randomized approximately 1,125 people utilizing the same eligibility criteria as RETHINK-ALZ. Subjects were randomized 1:1:1 to receive simufilam, dosed in 50 mg or 100 mg tablets, or a matched placebo, dosed orally twice daily (BID) for 76 weeks. On November 25, 2024, the Company announced plans to discontinue the REFOCUS-ALZ study and its intention to report topline data from that trial, including the complete 52-week dataset and a large portion of 76-week data.
The prespecified co-primary endpoints for this study included the change in cognition and function from baseline to the end of the double-blind treatment period at week 76, assessed by the ADAS-COG12 and ADCS-ADL scales, comparing each dose of simufilam to placebo. Secondary endpoints included several well validated measures of neuropsychiatric symptoms and caregiver burden. Safety was evaluated by adverse event monitoring, as well as standard laboratory and ECG assessments. The study also included an evaluation of changes in plasma and cerebrospinal fluid biomarkers from baseline to week 76, including P-tau217 (phosphorylated tau at threonine 217), GFAP (glial fibrillary acidic protein) and NFL (neurofilament light chain), as well as an evaluation of various brain volumes using MRI (magnetic resonance imaging) and amyloid and tau deposition using PET (positron emission tomography) scans from baseline to week 76.
About Simufilam
Simufilam is a proprietary, investigational oral small molecule that targets the filamin A protein.
About Cassava Sciences, Inc.
Cassava Sciences, Inc. (NASDAQ: SAVA), a clinical-stage biotechnology company focused on developing novel, investigational treatments for central nervous system disorders, including Alzheimer's disease and tuberous sclerosis complex (TSC)-related epilepsy. Simufilam is a proprietary, investigational oral small molecule that targets the filamin A protein. The Company is based in Austin, Texas.
For More Information Contact:
Investors
Sandya von der Weid
Media
Company
Eric Schoen, Chief Financial Officer
(512) 501-2450
Cautionary Note Regarding Forward-Looking Statements:
This news release contains forward-looking statements that include but are not limited to statements regarding: REFOCUS-ALZ and RETHINK-ALZ, the timing for discontinuation of our Alzheimer's disease development program, our plans for the development of investigational treatments for central nervous system disorders, our plans to conduct preclinical studies of simufilam relating to seizures in TSC, the potential for simufilam as a treatment for TSC-related epilepsy, our strategic expense management efforts and the timing of anticipated milestones. These statements may be identified by words such as 'anticipate', 'before', 'believe', 'could', 'expect', 'forecast', 'intend', 'may', 'pending', 'plan', 'possible', 'potential', 'prepares for', 'will', and other words and terms of similar meaning.
Such statements are based 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 efficiently discontinue the Company's Alzheimer's disease development program, the ability to advance preclinical studies related to TSC-related epilepsy, and other risks inherent in drug discovery and development or specific to Cassava Sciences, Inc., as described in the section entitled 'Risk Factors' in our Annual Report on Form 10-K for the year ended December 31, 2024, and future reports to be filed with the SEC. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from expectations in any forward-looking statement. In light of these risks, uncertainties and assumptions, the forward-looking statements and events discussed in this news release are inherently uncertain and may not occur, and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. Accordingly, you should not rely upon forward-looking statements as predictions of future events. Except as required by law, we disclaim any intention or responsibility for updating or revising any forward-looking statements. For further information regarding these and other risks related to our business, investors should consult our filings with the SEC, which are available on the SEC's website at www.sec.gov.
All of our pharmaceutical assets under development are investigational product candidates. They have not been approved for use in any medical indication by any regulatory authority in any jurisdiction and their safety, efficacy or other desirable attributes, if any, have not been established in any patient population. Consequently, none of our product candidates is approved or available for sale anywhere in the world.
Our clinical results from earlier-stage clinical trials may not be indicative of future 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.
We are in the business of new drug discovery, development and commercialization. Our research and development activities are long, complex, costly and involve a high degree of risk. Holders of our common stock should carefully read our Annual Report on Form 10-K and Quarterly Reports on Form 10-Q in their entirety, including the risk factors therein. Because risk is fundamental to the process of drug discovery, development and commercialization, you are cautioned to not invest in our publicly traded securities unless you are prepared to sustain a total loss of the money you have invested.
Hashtags

Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
26 minutes ago
- Yahoo
Pandamonium Doughnuts in Champaign closing near end of June
CHAMPAIGN, Ill. (WCIA) — After 12 years in town, Pandamonium Doughnuts in Champaign is closing towards the end of the month. In an Instagram post Wednesday night, the donut shop put out a statement announcing that they will be closing near the end of June. In the statement, the business said the decision came from a desire to spend more time with family. Cat cafe opening soon in Champaign 'We've poured our hearts into this journey, but now, we're ready to shift our focus to a new chapter — one centered on family, connection, and being fully present for those we love most,' part of the statement read. 'This journey, which began over 12 years ago in our home kitchen with nothing more than a doughnut obsession has been one of the greatest adventures of our lives.' At this time, the shop does not have a concrete 'last day' set but it will fall sometime around the last week of June or first week of July. To read the full statement, visit the donut shop's Instagram page. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.


Business Insider
31 minutes ago
- Business Insider
Palantir Just Popped 40%, But Is Snowflake Still the Quiet Killer in AI?
Palantir (PLTR) ripped higher in May, up 40.3% in a month when the Nasdaq basically slept. The spark? A fresh partnership with Databricks (yes, the same Databricks Nvidia just backed), plus a string of government wins across immigration, defense, and even the IRS. That's vintage Palantir: big, bold, and all-in on public sector scale. Confident Investing Starts Here: Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter And the market loved it. This is Palantir at its most potent—throwing elbows in the AI analytics ring and still landing contracts with agencies that don't mess around. The company's vision of 'AI with consequences' is resonating. That's translating into real revenue and serious stock action. Snowflake Stays Cool, But Don't Mistake It For Passive While Palantir was printing headlines, Snowflake (SNOW) barely budged. But that doesn't mean nothing's happening. Snowflake is the definition of stealth strength. It's the clean, enterprise-friendly data infrastructure play—plugged deep into AWS, Azure, and Google Cloud. And here's the twist: analysts say Snowflake's commercial reach may actually outpace Palantir's total addressable market over the long haul. Snowflake lives in the subscription SaaS world. Palantir lives on custom, high-touch deployments. That's the clash: scalability vs. depth. Two AI Strategies. One Collision Course. Palantir is going hyper-targeted: government, defense, complex systems, all infused with AI. Snowflake is building universal tools for every corporate data team on Earth. Different lanes, same race. If the AI wave stays high and wide, Snowflake's architecture might have more runway. But if regulation and geopolitical tension put data and defense front and center? That's Palantir's jungle. The Verdict Comes Down to Lightning vs. Ice Palantir's got momentum, headlines, and that sweet Databricks nod. But Snowflake's commercial footprint is enormous—and quietly expanding. One's loud, the other's lethal. Investors need to ask: do you want the stock that can 2x on a single AI headline? Or the one that could 10x silently over five years because it's baked into every Fortune 500 data stack? Right now, the market's hyped on Palantir. But the AI war is just warming up. And Snowflake isn't showing its hand yet. What Do Analysts Think Is the Better Buy: Palantir or Snowflake? Palantir is going hyper-targeted: government, defense, complex systems, all infused with AI. Snowflake is building universal tools for every corporate data team on Earth. Different lanes, same race. Investors can compare Palantir and Snowflake using the TipRanks Stock Comparison Tool. Snowflake (SNOW) currently trades at $208.24. Analysts project a robust upside for the data cloud giant. Its average price target hits $226.74, signaling an 8.88% potential climb. The consensus stands firm at a 'Strong Buy'. Snowflake also boasts a Smart Score of 5. Its market capitalization reaches $69.48 billion, with a P/E ratio of -50.12. Palantir (PLTR) charts a different course. Trading at $132.81, analysts foresee a downturn for the AI analytics firm. Its average price target hovers at $101.06, indicating a 23.91% potential downside. The consensus leans towards a 'Hold' rating. Palantir holds a Smart Score of 4. The company commands a significantly larger market capitalization at $313.42 billion, with a P/E ratio of 576.68. This direct comparison highlights Wall Street's current preferences. Snowflake draws strong buy signals and projected upside. Palantir, despite recent surges, faces a 'Hold' consensus and forecasted decline. The market clearly evaluates these AI titans on distinct metrics.
Yahoo
41 minutes ago
- Yahoo
OSF Healthcare now offering scalp cooling for cancer patients
PEORIA, Ill. (WMBD) — A new service being offered at OSF Healthcare is helping patients going through chemotherapy. It's called scalp cooling, and it's a first-of-its-kind service being offered in our area. It's a non-invasive, free treatment designed to reduce hair loss during chemotherapy. It works by lowering the temperature of your scalp down to 65 degrees before, during and after chemo. Scalp cooling helps cancer patients keep as much of their hair as possible, which in turn, helps their mental health. Our 2025 Remarkable Women winner, Patricia Cutiletta, had a huge part in bringing this service to our area. 'Well, it has nothing to do with vanity,' Cutiletta said. 'I had an oncologist who said to me, oh, that's for vain people. It's not. It's for people who want to preserve some sense of identity. It's for people who want to continue as much of their normal lifestyle as they can. And it's for people who want something that they can try and control.' The scalp cooling service is part of OSF Healthcare's START program. Which stands for survive, thrive and recover together. There's more than just scalp cooling. Patients can also get personalized care to help navigate their 'new normal' with confidence. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.