FLNA

We caution you not to place undue reliance on forward-looking statements because our future results may differ materially from those expressed or implied by them.
We do not undertake to update any forward-looking statement, whether written or oral, relating to the matters discussed in this Annual Report on Form 10-K, except as required by law.
Accordingly, we make no representations as to the accuracy or completeness of such data and information.
The contents of this Annual Report are solely our responsibility and do not necessarily represent any official views of NIH, the Department of Health and Human Services, or any other agency of the United States government, or any of our vendors, collaborators or unrelated third-parties .
Rigorous evidence for drug safety and efficacy is required for regulatory approval and is derived from one or more large, randomized, placebo-controlled studies.
Business Overview Filana Therapeutics, Inc.
(formerly Cassava Sciences, Inc.) is a clinical-stage biotechnology company based in Austin, Texas.
Our mission is to develop transformative medicines to improve the lives of patients with central nervous system (CNS) disorders, such as Tuberous Sclerosis Complex (TSC)-related epilepsy and other diseases associated with dysregulation or overexpression of filamin A.
Our science is based on modulating the activity of a critical protein in the brain for patients with certain CNS disorders, such as TSC.
Our lead therapeutic drug candidate, simufilam, was under clinical evaluation for the proposed treatment of Alzheimer’s disease in two Phase 3 clinical studies through 2024, when all ongoing clinical trials for simufilam in Alzheimer’s disease were discontinued.
The phase out of the Company’s Alzheimer's disease development program was completed in the second quarter of 2025.
Our lead therapeutic product candidate, simufilam, is a proprietary small molecule oral treatment drug being studied for the treatment of TSC-related epilepsy.
Simufilam is intended to modulate the activity of a protein called filamin A, which regulates diverse aspects of neuronal cell development.
Our and our collaborators’ published studies have demonstrated that the combination of mTOR activation and filamin A overexpression causes cell overgrowth, abnormal connectivity, and brain malformations, leading to seizures in patients with TSC.
While such seizures have historically been treated with mTOR inhibitors and other drugs, filamin A represents a novel target for the potential treatment of TSC-related epilepsy, independent of mTOR.
Simufilam is a potential first-in-class filamin A-modulating agent with the potential to treat TSC-related epilepsy.
We continue to explore other potential indications for simufilam, both CNS related and otherwise, where our pre-clinical experiments suggest that simufilam may provide a benefit.
We Own or Control Worldwide Rights to Our Drug Development Program Our success depends in part on our ability to obtain, maintain, and enforce intellectual property protection for our product candidates, including simufilam, and related technologies.
We seek to protect our proprietary position through a combination of patents, patent applications, trade secrets, know‑how, and other intellectual property rights.
We own or exclusively control a portfolio of issued patents and pending patent applications directed to simufilam and related diagnostic assets.
These intellectual property rights include patents covering composition of matter for simufilam in the United States, Europe, Australia, Israel, and Canada, as well as patents covering certain methods of use of simufilam in the United States, Europe, and Japan.
We also own pending patent applications covering diagnostic assets related to simufilam in the United States, Europe, Japan, China, Canada, and Australia, as well as additional pending patent applications covering other diagnostic assets in the United States, Europe, Japan, China, and Canada.
In the United States, our patent protection relating to simufilam, including its solid forms and methods of use for Alzheimer’s disease and other neurodegenerative diseases, consists of nine issued U.S. patents, with expiration dates ranging from 2029 to 2040, subject to potential patent term extensions and adjustments.
We have filed corresponding foreign patent applications for each of these U.S. patent families where we believe such protection is commercially appropriate.
In addition to our owned intellectual property, on February 26, 2025, we entered into a License Agreement (the “License Agreement”) with Yale University (“Yale”) pursuant to which we were granted exclusive worldwide rights, with rights to sublicense, to Yale’s interest in certain patent and other intellectual property rights that could be useful or necessary to the development and commercialization of simufilam for the treatment of TSC‑related epilepsy and other potential indications.
Under this exclusive, worldwide license, we have the right to sublicense the licensed intellectual property.
The license is subject to customary diligence obligations and includes milestone payments, royalties on net sales of licensed products, and other contingent payments, including payments tied to the transfer of any regulatory priority review voucher, if issued.
As a result, commercial sales of simufilam or related products, if any, may be subject to royalty obligations to Yale.
Except for intellectual property licensed from third parties, including pursuant to the License Agreement with Yale, we do not owe royalties or other payments to third parties with respect to our owned patents and patent applications covering simufilam.
Preclinical Studies with Simufilam in Tuberous Sclerosis Complex Preclinical research conducted at Yale indicates that simufilam (then PTI-125) may be effective in reducing TSC-related seizure activity.
A study conducted by Angelique Bordey, PhD, at Yale and published in the peer-reviewed journal Neuron in 2014 found overexpression of filamin A and neuronal abnormalities in brain tissue from TSC patients, as well as in brain tissue from mice that had been genetically altered to model TSC.
The study further found that genetically normalizing filamin A expression in the mice prevented neuronal abnormalities.
Bordey and published in the peer-reviewed journal Science Translational Medicine in 2020 likewise found elevated filamin A and neuronal abnormalities in brain tissue from patients with focal cortical dysplasia type II (FCDII), a genetic disorder with similar characteristics to TSC, as well as in the brains of mice that had been genetically altered to model TSC and FCDII.
That study showed that normalizing filamin A expression in the mice through genetic knockdown both limited the neuronal abnormalities and reduced the frequency of epileptic seizures in the mice.
Bordey and her colleagues also studied the effect of treatment with simufilam in the mice that had been altered to model TSC and FCDII.
The research showed that treatment with simufilam limited neuronal abnormalities and reduced seizure activity in the mouse model at a level similar to genetic knockdown of filamin A expression.
Treatment with simufilam did not, however, affect the level of filamin A in the brains of the mice.
Bordey joined the Company as Senior Vice President, Neuroscience, on May 1, 2025, while continuing her tenured academic position at Yale School of Medicine on a part time basis.
In connection with plans to initiate a clinical program in TSC-related epilepsy, the Company appointed Dr.
Joseph Hulihan as Chief Medical Officer in August 2025.

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Gone since FY2024

Accordingly, you should not place undue reliance upon any forward-looking statements.
We cannot assure you that we will realize the results or developments we expect or anticipate or, even if substantially realized, that they will affect us or our operations in the way we expect.
The forward-looking statements included in this Annual Report on Form 10-K are made only as of the date hereof.
We undertake no obligation to publicly update or revise any forward-looking statement as a result of new information, future events or otherwise, except as required by law.
Although we believe that such sources are reliable, we make no representations as to the accuracy or completeness of such data and information.
In addition, statements that “we believe” or similar statements reflecting our beliefs, views, and opinions on the relevant subject are based upon information available to us as of the date of this Annual Report.
While we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information.
These statements are inherently uncertain and involve a number of assumptions and limitations, and you are cautioned not to unduly rely upon these statements.
The contents of this Annual Report are solely our responsibility and do not represent any views of NIH, the Department of Health and Human Services, or any other agency of the United States government, or any of our vendors, collaborators or unrelated third-parties .
Other than our Phase 3 trials, all of our earlier-stage clinical trials have involved a relatively small number of patients and limited data.
Information and results generated from our early-stage studies do not constitute, and should not be interpreted as, evidence of safety or efficacy for simufilam in Alzheimer’s disease.
Rigorous evidence for drug safety and efficacy is required for regulatory approval and is derived from one or more large, randomized, placebo-controlled Phase 3 studies.
Unless otherwise noted, all clinical data in this Annual Report is statistically non-significant at a standard probability level of p<0.05.
In addition, from time to time, our scientific research may include the use of exploratory biomarkers, typically labelled ‘research use only.’ They are understood to mean non-safety-related, investigational diagnostic products that are in the research phase of development and have not been approved by any regulatory agency to be effective, sensitive, specific, accurate, predictive or linked to a specific diagnosis or indication.
At present there is no sufficiently reliable evidence that any observed treatment effect on such biomarkers is reasonably likely to predict clinical benefit.
National Clinical Trial (NCT) is an eight-digit identification number that http://www.ClinicalTrials.gov assigns a clinical study when it is registered with the National Library of Medicine, which is operated by the United States government.
Business Overview Cassava Sciences, Inc. is a clinical-stage biotechnology company based in Austin, Texas.
Our mission is to detect and treat central nervous system disorders, such as Alzheimer’s disease and Tuberous Sclerosis Complex (TSC)-related epilepsy.
Our novel science is based on stabilizing – but not removing – a critical protein in the brain for patients with certain central nervous system disorders, such as Alzheimer’s or TSC.
Our lead therapeutic drug candidate, simufilam, was under clinical evaluation for the proposed treatment of Alzheimer’s disease in Phase 3 clinical studies through 2024, when all ongoing clinical trials for simufilam in Alzheimer’s disease were discontinued.
We combine innovative technology with new insights in neurobiology to develop novel solutions targeting Alzheimer’s disease, TSC-related epilepsy, and other central nervous system disorders.
Our strategy is to leverage our unique scientific/clinical platform to develop first-in-class programs for treating central nervous system disorders.
In addition, we are in the early stages of exploring potential artificial intelligence (“AI”) capabilities and related data analytics to target improvements in productivity and efficiency in our business, enhancements to research and development activities, and advancements in statistical analysis capabilities.
We currently have two biopharmaceutical assets under development: ● our lead therapeutic product candidate, called simufilam, is a proprietary small molecule oral treatment drug being studied for the treatment of Alzheimer’s disease and TSC-related epilepsy; and ● our lead investigational diagnostic product candidate, called SavaDx, is a novel biomarker assay being studied for detection of the presence of Alzheimer’s disease from a small sample of blood.
Simufilam targets a protein called filamin A (FLNA) in the brain of patients with central nervous system disorders, such as Alzheimer’s or TSC.
Our and our collaborators' published studies have demonstrated that an altered form of FLNA is linked to neuronal dysfunction, neuronal degeneration and neuroinflammation.
In Alzheimer’s disease, we believe simufilam disrupts amyloid binding to the α7 nicotinic acetylcholine receptor (α7nAChR), which underlies our drug’s primary mechanism of action in Alzheimer’s disease.
Published pre-clinical studies further show that overexpression of FLNA in patients with TSC may be associated with epileptic seizure activity.
On February 26, 2025, we entered into a License Agreement (the “License Agreement”) with Yale to support our development and commercialization efforts for simufilam for the treatment of TSC-related epilepsy.
We solely own patents covering the composition of matter of our drug simufilam in the United States, Europe, Australia, Israel and Canada.
We solely own patents covering certain methods of use of our drug simufilam in the United States, Europe and Japan.
We solely own pending patent applications that cover diagnostic assets for simufilam in the United States, Europe, Japan, China, Canada, and Australia.
We solely own pending patent applications that cover other diagnostic assets in the United States, Europe, Japan, China and Canada.
We have no obligation to pay any royalty to any third party in connection with any of the foregoing described patents and patent applications.
In the United States, our patent protection with respect to simufilam, its solid forms, and uses of simufilam for Alzheimer’s disease and other neurodegenerative diseases includes nine issued United States patents, with terms expiring on dates ranging from 2029 to 2040, subject to any patent extensions that may be available for such patents.
Corresponding foreign filings have been made for each of the Unites States filings.
About Alzheimer ’ s Disease Alzheimer’s disease is a degenerative disease of the brain that affects cognition, function and behavior.
Over time, a patient’s cognition and health functions decline as the disease takes its toll.
With disease progression, patients move from mild to moderate to, eventually, severe Alzheimer’s disease.
Cognitive decline becomes more pronounced, and presumably more difficult to treat, in advanced stages of the disease.

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