Filana Therapeutics : Corporate Presentation May 2026

Published: 2026-05-07 14:54:09 ET FLNA

Published on 05/07/2026 at 02:54 pm EDT

May 2026

Data from two mouse models provide preclinical evidence in TSC-related epilepsy.

Strong safety database of >2,000 patients who were treated in prior clinical trials with simufilam with some patients receiving treatment for as long as 24 months1.

Refining proof-of-concept trial protocol. Product supply in place.

New method of treatment patent issued in 2025 and in-licensed from Yale2.

Expanded team of experienced neurology experts3,4.

Sources: 1. Filana Therapeutics information on file, 2. https://www.filanatx.com/news-releases/news-release-details/cassava-sciences-licenses-simufilam-method-treatment-patent, 3. https://www.filanatx.com/news-releases/news-release-details/cassava-appoints-angelique-bordey-phd-enhance-preclinical , 4. https://www.filanatx.com/news-releases/news-release-details/cassava-sciences-appoints-dr-joseph-hulihan-chief-medical.

We are driven by our mission to develop transformative medicines to improve the lives of patients with CNS disorders such as TSC-related epilepsy, and other diseases associated with dysregulation or overexpression of filamin A.

Management Team

Richard Barry

President C Chief Executive Officer

Angelique Bordey, PhD Senior Vice President, Neuroscience

Eric Schoen

Chief Financial Officer

Christopher Cook Chief Operating C Legal Officer

Joseph Hulihan, MD

Chief Medical Officer

Board of Directors

Claude Nicaise, MD, Chairman

Robert Anderson

Richard Barry Dawn C. Bir

Pierre Gravier, MS Robert Gussin, PhD

Michael O'Donnell

Patrick Scannon, MD/PhD

Simufilam is a novel potential filamin A-modulating agent. The Company is planning a proof-of-concept clinical study to explore its use as a first-in-class treatment for TSC-related epilepsy. In partnership with our advisors, we are working diligently to address the FDA's requests for information detailed in the Clinical Hold Letter received in December 2025, including the submission of additional pre-clinical data and protocol design modifications. The Company intends to submit a response to FDA as soon as practicable. We look forward to sharing a progress update in the coming months.

Simufilam has been studied in more than 2,000 subjects with a good overall safety profile observed

across prior clinical trials, with some patients receiving treatment for as long as 24 months2.

Program based on a new method of treatment patent issued in 2025 and in-licensed from Yale. Builds on work conducted by Angélique Bordey, PhD, Vice Chair, Department of Neurosurgery, Yale School of Medicine. Dr. Bordey joined Filana Therapeutics as Senior Vice President, Neuroscience.

Preclinical data from two separate mouse models support simufilam's anti-seizure activity, in work

led by Dr. Bordey, both at Yale and in collaboration with the TSC Alliance3,4.

Expanded, experienced team of CNS experts.

Sources:1. Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations - PMC, 2. Cassava information 3. https://www.filanatx.com/news-releases/news-release-details/cassava-sciences-licenses-simufilam-method-treatment-patent 4. https://www.filanatx.com/news-releases/news-release-details/cassava-sciences-reports-positive-preclinical-study-evaluating. Notes: The Yale Mouse Model, as referred to herein, relates to the RhebCA model developed by Dr. Bordey at Yale, per reference 1

Simufilam is a Novel Filamin A Modulator

Simufilam's Value Proposition in TSC-Related Epilepsy

Filamin A regulates diverse aspects of neuronal cell development1.

Simufilam represents a potential first-in-class oral small molecule that potentially modulates filamin A function2.

Multiple commercial-scale GMP batches of simufilam have been reproducibly manufactured on behalf of Filana Therapeutics.

Potential to reduce the frequency of seizures.

Generally well-tolerated safety profile, based on data from >2,000 subjects treated across prior clinical trials.

The combination of mTOR activation and filamin A overexpression causes cell overgrowth, abnormal connectivity, and brain malformations, leading to seizures1.

Filamin A represents a novel target, independent of mTOR.

Simufilam is a potential first-in-class filamin A-modulating agent with the potential to treat TSC-related epilepsy2.

PDK

AKT

Mutant TSC1/TSC2

Increased RHEB activity

P13K

Receptors

mTOR inhibitors (everolimus, rapamycin)

Increased mTOR

activity

Increased Filamin A expression

simufilam (PTI-125)

Cell overgrowth Abnormal connectivity Brain malformation

Seizures

Sources: Image is derived from https://www.cassavasciences.com/static-files/a80aa7bc-ede1-4ef7-9a57-69d93774460a, 1. Man A, Di Scipio M, Grewal S, Suk Y, Trinari E, Ejaz R, Whitney R. The Genetics of Tuberous Sclerosis Complex and Related mTORopathies: Current Understanding and Future Directions. Genes (Basel). 2024 Mar 4;15(3):332. doi: 10.3390/genes15030332. PMID: 38540392; PMCID: PMC10970281, 2. Longbo Zhang et al., Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations.Sci. Transl. Med.12,eaay0289(2020).DOI:10.1126/scitranslmed.aay0289

TSC-Related Epilepsy

Tuberous Sclerosis Complex (TSC)

is a genetic disorder caused by mutations in the TSC1 or

TSC2 gene and can involve multiple organs.

The disorder may present at any age and is often diagnosed based on specific clinical criteria and/or genetic testing.

Epilepsy is one of the most common neurologic manifestations associated with TSC.

Source: 1. Rare Disease Org website : https://rarediseases.org/rare-diseases/tuberous-sclerosis, 2. Conte E, Boccanegra B, Dinoi G, Pusch M, De Luca A, Liantonio A, Imbrici P. Therapeutic Approaches to Tuberous Sclerosis Complex: From Available Therapies to Promising Drug Targets. Biomolecules. 2024 Sep 21;14(9):1190. doi: 10.3390/biom14091190. PMID: 39334956; PMCID: PMC11429992

How TSC presents

Brain

Eye

Lung

Heart

Renal

Skin

TSC-related epilepsy is the most common medical condition in people with TSC1, with seizure onset typically occurring in the first year of life2.

80% to G0% of TSC patients experience seizures at some point in their life3.

Brain malformations, otherwise known as cortical tubers, are a hallmark of disease, found in the brain of more than 80% of TSC patients4.

Sources 1. https://www.massgeneral.org/neurology/tsc/patient-education/how-tsc-affects-brain-seizures, 2.Conte E, Boccanegra B, Dinoi G, Pusch M, De Luca A, Liantonio A, Imbrici P. Therapeutic Approaches to Tuberous Sclerosis Complex: From Available Therapies to Promising Drug Targets. Biomolecules. 2024 Sep 21;14(9):1190. doi: 10.3390/biom14091190. PMID: 39334956; PMCID: PMC11429992, 3. https://www.tscalliance.org/understanding-tsc/what-is-tsc/ , 4. https://www.massgeneral.org/neurology/tsc/patient-education/how-tsc-affects-brain-anatomy, Image: Reference #4.

50K

U.S. Prevalence of TSC1

~40-45K

80-90% of patients with TSC have seizures2,3

~25-28k

>60% of treated patients are refractory

to antiepileptic therapy3

Current treatments include antiepileptic agents4.

Two therapies have been approved by FDA specifically for TSC-related epilepsy: everolimus5 and cannabidiol6.

Use of multiple agents and surgical intervention underscore the unmet need for improved therapy in people with TSC-related epilepsy7.

Sources: 1. https://www.tscalliance.org/understanding-tsc/what-is-tsc/, 2. Longbo Zhang et al., Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations.Sci. Transl. Med.12,eaay0289(2020).DOI:10.1126/scitranslmed.aay0289, 3. Chu-Shore CJ, Major P, Camposano S, Muzykewicz D, Thiele EA. The natural history of epilepsy in tuberous sclerosis complex. Epilepsia. 2010 Jul;51(7):1236-41. doi: 10.1111/j.1528-1167.2009.02474.x. Epub 2009 Dec 22. PMID: 20041940; PMCID: PMC3065368, 4. https://www.tscalliance.org/understanding-tsc/clinical-manifestations/brain-neurological-function/epilepsy-seizure-disorders/epilepsy-treatment-options-for-individuals-with-tsc/,6. Everolimus/AFINITOR is distributed by Novartis Pharmaceuticals,. 6. Cannabidiol/Epidiolox is distributed by Jazz Pharmaceutical, 8. Betts, K. A., Stockl, K. M., Yin, L., Hollenack, K. A., C Wang, M. (2020). 7. Economic Burden Associated with Tuberous Sclerosis Complex in Patients with Epilepsy. Value in Health, 23, S262-S262.

Open-label multi-center study to assess the effects of simufilam for the treatment of TSC-related epilepsy

Primary Endpoints:

Screening

Treatment

Initial Assessment

Safety, tolerability, pharmacokinetics

Secondary Endpoints:

Change in seizure frequency at study conclusion compared to baseline

Baseline evaluation of seizure frequency

All patients to receive simulfilam + standard of care

After planned treatment

Open Label Extension:

Available to patients who complete the initial treatment period and elect to continue

Preclinical Research

Studies conducted in the lab of Dr. Angélique Bordey (the Bordey Lab) at Yale identified filamin A overexpression in patients with TSC and FCDII, suggesting that filamin A could be a new therapeutic target in TSC-related epilepsy1,2,3.

The Bordey Lab showed that simufilam reduced seizure frequency in the Yale mouse model. Simufilam also reduced neuron overgrowth and brain malformations in these mice1,3.

Attenuation of the increase in seizure activity was later shown in another mouse model of TSC-related

seizures provided by the TSC Alliance (the "TSCA mouse model").

Simufilam appears to modulate filamin A function3, a new potential mechanism of action in TSC-related epilepsy. Filamin A plays a role in brain development, including cell growth and neuronal connectivity2.

Planning a clinical proof of concept study to evaluate simufilam in patients with TSC-related epilepsy, collaborating closely with the TSC Alliance and key opinion leaders.

Sources: 1. Zhang, Longbo; Bartley, Christopher M.; Gong, Xuan; Hsieh, Lawrence S.; Lin, Tiffany V.; Feliciano, David M.; Bordey, Angélique. "MEK-ERK1/2-Dependent FLNA Overexpression Promotes Abnormal Dendritic Patterning in Tuberous Sclerosis Independent of mTOR." Neuron, vol. 84, no. 1, 2014, pp. 78-91, 2. Hsieh, L., Wen, J., Claycomb, K. et al. Convulsive seizures from experimental focal cortical dysplasia occur independently of cell misplacement. Nat Commun 7, 11753 (2016). https://doi.org/10.1038/ncomms11753, 3. Longbo Zhang et al., Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations.Sci. Transl. Med.12,eaay0289(2020).DOI:10.1126/scitranslmed.aay0289. Note: The Lab of Angélique Bordey is referred to as the Bordey Lab in this document.

Yale Mouse Model2

TSCA Mouse Model2

Mouse Model

In utero expression of a constitutively active RHEB, a key regulator of mTOR signaling.

Transgenic mice with embryonic, conditional Tsc1 deletion (cKO, human GFAP promoter).

Characteristics Connecting Brain Abnormalities and Seizures

Cortical tuber-like malformations lead to spontaneous seizures.

Widespread forebrain abnormalities lead to spontaneous seizures.

Sources: 1. Summarized based on the TSC Alliance presentation on June 26, 2025: https://www.filanatx.com/news-releases/news-release-details/cassava-presents-promising-preclinical-simufilam-data-tsc, .2. In this presentation, the RhebCA mouse model is referred to as the Yale Mouse Model, the Tsc1 cKO mouse model is referred to as the TSCA Mouse Model. CKO = conditional knock-out .

In utero

RHEB

expression

Treatment

Assessment

Seizure activity worsens with time, consistent with increasing cell overgrowth and dendritic abnormalities.

Evaluate multiple age cohorts and treatment and control regimens.

EEG seizure measurements

for a range of 5-40 days.

Main Endpoints:

neuron cell body overgrowth, dendrite morphology and seizure activity

Sources 1. Summarized from Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations - PMC, 2.Illustrated timeline is not to scale.

Partial Reversal of Cell Overgrowth and Dendritic Hypertrophy1,2

Reduction in seizure activity3

YMM mice + Flna shRNA

Control mice + control shRNA

YMM mice+ control shRNA

Neuron

Body

Dendrites

Control shRNA Flna shRNA

Sources: 1. Summarized from Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations - PMC, (STM) Fig 3B, 2. STM Fig 3C, 3. STM, Fig 3G. Notes: RhebCA mice have been referred to as the Yale Mouse Model (YMM) herein, FLNA shRNA = Filamin A short hairpin (sh) RNA (also shFLNA), Luc = Luciferase, GFP = a flouescent reporter, green fluorescent protein

YMM + Vehicle

YMM + Simufilam

60% reduction in seizure frequency1

Control mice +

Vehicle

Neuron

Body

Dendrites

Vehicle Simufilam

Sources 1. Summarized from Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations - PMC, (STM) Fig 4B, 2. STM, Fig 4C. Notes: RhebCA mice have been referred to as the Yale Mouse Model (YMM) herein, FLNA shRNA = Filamin A short hairpin (sh) RNA (also shFLNA), Luc = Luciferase, GFP = a flouescent reporter, green fluorescent protein, PTI-125 = simufilam

Simufilam Reduced Seizure Activity In Yale Mouse Model

Treatment Groups

Baseline Phase Treatment Phase

Vehicle Vehicle

Mice

Vehicle-Treated Group

P61 Days

Vehicle Simufilam

Mice

Simufilam-Treated Group

P61 Days

Seizure activity in untreated adult mice worsened with time, consistent with an increase in cell overgrowth and network abnormalities

Sources: 1. Summarized fromFilamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations - PMC, (STM) Fig 5I . Notes: RhebCA mice have been referred to as the Yale Mouse Model (aka YMM) herein, D = treatment day. P61 refers to 61 days after birth.

Disclaimer

Filana Therapeutics Inc. published this content on May 07, 2026, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on May 07, 2026 at 18:53 UTC.