New Data by Academic Researchers Highlights Biological Activity of Simufilam on Filamin A

  • New in vitro data from Europe show that simufilam can reverse altered filamin A protein (FLNA) in pituitary tumor cells, leading to improved cell signaling.
  • Data suggest FLNA is a central factor influencing pituitary tumor cell behavior.
  • Data to be presented at the European Society of Endocrinology, May 13-16th.

AUSTIN, Texas, May 08, 2023 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (Nasdaq: SAVA), a clinical-stage biotechnology company, today announced new data from European researchers that highlight the bioactivity of simufilam on the filamin A (FLNA) protein. Prior research has shown that FLNA is altered in pituitary tumor cells, leading to impaired cell signaling. New in vitro data now show that simufilam can reverse this FLNA alteration in pituitary tumor cells, leading to improved cell signaling. The data also suggest that improved cell signaling may enhance the clinical efficacy of an FDA-approved drug used to manage a type of pituitary tumor.

“These findings take filamin A research to the next level,” said Remi Barbier, President & CEO. “They show an in vitro drug effect for simufilam in a pathway outside of neurodegeneration. This bioactivity is thematically consistent with the proposed mechanism of action of simufilam in Alzheimer’s disease, namely, simufilam reverses a filamin A alteration. To be clear, we’re inspired by this promising research, but that’s as far as it goes. We have no immediate plans to initiate by ourselves a clinical program in cancer.”

Prior research publications have demonstrated the essential role of the filamin A (FLNA) protein in signaling of numerous receptors, including dopamine receptors, calcium-sensing receptors, calcitonin receptors, insulin receptors, acetylcholine receptors and somatostatin receptors. Today’s new data by researchers in Europe shows a functional interaction between simufilam, FLNA and somatostatin receptors. Specifically, the researchers show simufilam treatment significantly reduced levels of phosphorylation (minus 28% ± 13% after 10 min, p<0.01 vs basal) at a site on FLNA in human pituitary tumor cells. The reduced FLNA phosphorylation was accompanied by improved cell signaling of somatostatin receptors. The authors also show that cells co-treated with simufilam plus an FDA-approved drug used to manage a type of pituitary tumor significantly increased cell death (+37% ± 9%, p<0.01 vs basal, p<0.05 vs. either drug alone) in a rat pituitary tumor cell line. These in vitro data suggest FLNA is a central factor influencing pituitary tumor cell behavior. The data also suggest co-treatment with simufilam may enhance somatostatin analogs, a class of FDA-approved drugs used to manage a type of pituitary tumor; however, there is not enough information from which scientific conclusions can be drawn regarding a relationship between simufilam and the treatment of any disease in humans.

Oral Presentation Details
When: May 15th, 2023

Where: 25th European Congress of Endocrinology, Istanbul, Turkey.

Abstract Title: A Novel Filamin A-binding Molecule May Significantly Enhance Somatostatin Receptor Type 2 Antitumoral Actions in Growth Hormone-secreting PitNET Cells.

Senior Authors: Prof. Erika Peverelli, PhD, and Prof. Giovanna Mantovani, PhD, Università di Milano, Italy and Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, U.O. di Endocrinologia, Milano, Italy.

External Web Link to Abstract:

Disclosures: Cassava Sciences provided simufilam drug to the Peverelli lab at no cost pursuant to a Material Transfer Agreement. Cassava Sciences did not fund Dr. Peverelli’s research. L. Burns, Ph.D., one of 15 co-authors of the abstract, is employed by Cassava Sciences as Senior VP of Neuroscience and holds equity in Cassava Sciences. Cassava Sciences owns intellectual property rights in the field of FLNA-binding compounds, including simufilam, and methods for inhibiting the growth of cancer cells.

About Simufilam
Simufilam is a novel drug candidate designed to treat and slow the progression of Alzheimer’s disease. Simufilam binds tightly to an altered conformation of the filamin A protein (FLNA) that is present in the brain of the Alzheimer’s patient and is critical to the toxicity of Aβ42. Simufilam is wholly owned by Cassava Sciences, without royalty or payment obligation to any third party.

About Cassava Sciences, Inc.
Cassava Sciences is a clinical-stage biotechnology company based in Austin, Texas. Our mission is to detect and treat neurodegenerative diseases, such as Alzheimer’s disease. Our novel science is based on stabilizing—but not removing—a critical protein in the brain. Our product candidates have not been approved by any regulatory authority, and their safety, efficacy or other desirable attributes have not been established. For more information, please visit our website:

For More Information Contact:

Eric Schoen, Chief Financial Officer
(512) 501-2450

Cautionary Note Regarding Forward-Looking Statements:
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, that may include but are not limited to: our strategy and plans; the treatment of Alzheimer’s disease dementia; the safety or efficacy of simufilam in patients; the release of evidential data by a third-party related to the biological activity of simufilam; 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.

Simufilam and SavaDx are investigational product candidates. They are not approved by any regulatory authority and their safety, efficacy or other desirable attributes have not been established in patients.

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. You should not place undue reliance on any scientific data we or others present or publish. This press release may also contain science data and drug information based on the work of third-party researchers or other publicly available information. We have not independently verified the accuracy or completeness of the data and information obtained from these sources. Accordingly, we make no representations as to the accuracy or completeness of such research, data or information. You are cautioned not to give undue weight to such research, data or information. The content of this press release is solely our responsibility and does not represent the official views of any third-party researcher, university, clinical group practice or any country’s government agency.

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 of simufilam to enhance somatostatin analogs used to treat pituitary tumors; to demonstrate the specificity, safety, efficacy or potential health benefits of our product candidates; any unanticipated impacts of inflation 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 December 31, 2022, 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 contained in this news release. 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

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