Cassava Presents Promising Preclinical Simufilam Data at TSC Alliance Meeting
Cassava Sciences (NASDAQ: SAVA) presented promising preclinical data for simufilam in treating Tuberous Sclerosis Complex (TSC)-related epilepsy at the TSC International Research Conference. The research demonstrated that simufilam reduced seizure frequency by 60% compared to vehicle in a mouse model of focal onset seizures.
Key findings from the preclinical studies conducted at Yale School of Medicine showed statistically significant seizure reduction (p<0.0001) and improved seizure freedom rates (11/32 simufilam-treated mice vs 3/29 vehicle-treated mice, p=0.0343). The effective dose was 20 mg/kg/day, with no adverse effects observed at doses up to 50 mg/kg/day in rats and 1250 mg/kg/day in mice.
Additionally, the company presented favorable human safety data from two Phase 3 studies involving 1,929 patients with mild-to-moderate Alzheimer's disease. The safety profile showed only mild, non-serious adverse events typically unrelated to simufilam, with no serious adverse events assessed as study-drug related. Cassava plans to initiate human clinical studies for TSC-related epilepsy in H1 2026.
Cassava Sciences (NASDAQ: SAVA) ha presentato dati preclinici promettenti sull'uso di simufilam nel trattamento dell'epilessia correlata alla Sclerosi Tuberosa (TSC) durante la Conferenza Internazionale di Ricerca sulla TSC. La ricerca ha dimostrato che simufilam ha ridotto la frequenza delle crisi epilettiche del 60% rispetto al controllo in un modello murino di crisi focali.
I risultati principali degli studi preclinici condotti presso la Yale School of Medicine hanno evidenziato una riduzione statisticamente significativa delle crisi (p<0,0001) e un miglioramento nei tassi di libertà dalle crisi (11/32 topi trattati con simufilam contro 3/29 trattati con controllo, p=0,0343). La dose efficace è stata di 20 mg/kg/giorno, senza effetti avversi osservati fino a 50 mg/kg/giorno nei ratti e 1250 mg/kg/giorno nei topi.
Inoltre, l'azienda ha presentato dati favorevoli sulla sicurezza umana provenienti da due studi di Fase 3 che hanno coinvolto 1.929 pazienti con Alzheimer da lieve a moderato. Il profilo di sicurezza ha mostrato solo eventi avversi lievi e non gravi, generalmente non correlati a simufilam, senza eventi avversi gravi attribuibili al farmaco. Cassava prevede di avviare studi clinici sull'uomo per l'epilessia correlata alla TSC nella prima metà del 2026.
Cassava Sciences (NASDAQ: SAVA) presentó datos preclínicos prometedores sobre simufilam para el tratamiento de la epilepsia relacionada con el Complejo de Esclerosis Tuberosa (TSC) en la Conferencia Internacional de Investigación sobre TSC. La investigación mostró que simufilam redujo la frecuencia de las convulsiones en un 60% en comparación con el vehículo en un modelo murino de convulsiones de inicio focal.
Los hallazgos clave de los estudios preclínicos realizados en la Escuela de Medicina de Yale mostraron una reducción estadísticamente significativa de las convulsiones (p<0,0001) y una mejora en las tasas de libertad de convulsiones (11/32 ratones tratados con simufilam frente a 3/29 tratados con vehículo, p=0,0343). La dosis efectiva fue de 20 mg/kg/día, sin efectos adversos observados en dosis de hasta 50 mg/kg/día en ratas y 1250 mg/kg/día en ratones.
Además, la compañía presentó datos favorables de seguridad en humanos de dos estudios de Fase 3 que involucraron a 1.929 pacientes con enfermedad de Alzheimer leve a moderada. El perfil de seguridad mostró solo eventos adversos leves y no graves, generalmente no relacionados con simufilam, sin eventos adversos graves relacionados con el medicamento del estudio. Cassava planea iniciar estudios clínicos en humanos para la epilepsia relacionada con TSC en la primera mitad de 2026.
Cassava Sciences (NASDAQ: SAVA)는 TSC(결절성 경화증) 관련 간질 치료를 위한 시무필람의 유망한 전임상 데이터를 TSC 국제 연구 회의에서 발표했습니다. 연구 결과, 시무필람은 국소 발작 마우스 모델에서 대조군 대비 발작 빈도를 60% 감소시켰습니다.
예일 의과대학에서 수행된 전임상 연구의 주요 결과는 통계적으로 유의한 발작 감소(p<0.0001)와 발작 완전 관해율 개선(시무필람 투여 마우스 11/32 vs 대조군 3/29, p=0.0343)을 보여주었습니다. 효과적인 용량은 20 mg/kg/일이었으며, 쥐에서는 50 mg/kg/일, 마우스에서는 1250 mg/kg/일까지 부작용이 관찰되지 않았습니다.
또한, 회사는 경도에서 중등도 알츠하이머병 환자 1,929명을 대상으로 한 두 건의 3상 임상시험에서 긍정적인 인체 안전성 데이터를 발표했습니다. 안전성 프로필은 시무필람과 관련 없는 경미하고 심각하지 않은 이상 반응만을 보였으며, 심각한 이상 반응은 연구 약물과 관련이 없는 것으로 평가되었습니다. Cassava는 2026년 상반기에 TSC 관련 간질에 대한 인체 임상시험을 시작할 계획입니다.
Cassava Sciences (NASDAQ: SAVA) a présenté des données précliniques prometteuses sur le simufilam pour le traitement de l’épilepsie liée au Complexe de Sclérose Tuberuse (TSC) lors de la Conférence Internationale de Recherche sur la TSC. La recherche a montré que le simufilam réduisait la fréquence des crises de 60% par rapport au véhicule dans un modèle murin de crises focales.
Les résultats clés des études précliniques menées à la Yale School of Medicine ont démontré une réduction statistiquement significative des crises (p<0,0001) et une amélioration des taux de liberté de crises (11/32 souris traitées au simufilam contre 3/29 traitées au véhicule, p=0,0343). La dose efficace était de 20 mg/kg/jour, sans effets indésirables observés jusqu’à 50 mg/kg/jour chez les rats et 1250 mg/kg/jour chez les souris.
De plus, la société a présenté des données favorables sur la sécurité humaine issues de deux études de phase 3 impliquant 1 929 patients atteints de la maladie d’Alzheimer légère à modérée. Le profil de sécurité a révélé uniquement des événements indésirables légers et non graves, généralement non liés au simufilam, sans événements indésirables graves attribués au médicament de l’étude. Cassava prévoit de lancer des études cliniques humaines pour l’épilepsie liée à la TSC au premier semestre 2026.
Cassava Sciences (NASDAQ: SAVA) präsentierte vielversprechende präklinische Daten zu Simufilam bei der Behandlung von epileptischen Anfällen im Zusammenhang mit dem Tuberösen Sklerose-Komplex (TSC) auf der Internationalen TSC-Forschungskonferenz. Die Forschung zeigte, dass Simufilam die Anfallshäufigkeit in einem Mausmodell für fokale Anfälle um 60% im Vergleich zum Vehikel reduzierte.
Wesentliche Ergebnisse der präklinischen Studien an der Yale School of Medicine zeigten eine statistisch signifikante Reduktion der Anfälle (p<0,0001) sowie verbesserte Anfallsfreiheit (11/32 mit Simufilam behandelte Mäuse vs. 3/29 Vehikel-behandelte Mäuse, p=0,0343). Die wirksame Dosis betrug 20 mg/kg/Tag, ohne beobachtete Nebenwirkungen bei Dosen bis zu 50 mg/kg/Tag bei Ratten und 1250 mg/kg/Tag bei Mäusen.
Zusätzlich präsentierte das Unternehmen günstige Sicherheitsdaten am Menschen aus zwei Phase-3-Studien mit 1.929 Patienten mit leichter bis mittelschwerer Alzheimer-Krankheit. Das Sicherheitsprofil zeigte nur milde, nicht schwerwiegende Nebenwirkungen, die typischerweise nicht mit Simufilam in Verbindung gebracht wurden, und keine schwerwiegenden Nebenwirkungen, die als durch das Studienmedikament verursacht bewertet wurden. Cassava plant, im ersten Halbjahr 2026 klinische Studien am Menschen für TSC-bedingte Epilepsie zu starten.
- Significant 60% reduction in seizure frequency in preclinical mouse model
- Statistically significant improvement in seizure freedom rates (p=0.0343)
- Favorable safety profile demonstrated in large Phase 3 studies with 1,929 patients
- No serious adverse events related to simufilam in clinical studies
- High dosing tolerance shown in toxicology studies
- Human clinical trials for TSC-related epilepsy won't begin until H1 2026
- Additional preclinical studies still needed to confirm findings
Insights
Cassava's simufilam shows 60% seizure reduction in preclinical epilepsy model with established human safety data, expanding beyond Alzheimer's.
Cassava Sciences has presented compelling preclinical data showing simufilam reduced seizure frequency by
What makes this development particularly noteworthy is the dual-pronged approach Cassava is taking: leveraging both new preclinical efficacy data and existing human safety data from their Alzheimer's program involving 1,929 patients. This strategy could potentially streamline the development timeline for this new indication.
TSC is a rare genetic disorder affecting approximately 50,000 patients in the US that causes lifelong epilepsy and severe neurological issues. The disease mechanism involves mutations in TSC1 or TSC2 genes affecting the mTOR pathway, with evidence showing increased expression of filamin A - precisely the protein that simufilam is designed to modulate.
The scientific rationale appears robust, with the research conducted in the lab of Cassava's SVP of Neuroscience, Dr. Angélique Bordey, formerly at Yale School of Medicine. Additional preclinical studies are underway with the TSC Alliance preclinical consortium to further explore simufilam's mechanism and potential.
This represents a strategic expansion of simufilam's potential applications beyond Alzheimer's into a rare disease with high unmet need. With human clinical studies planned to begin in H1 2026, Cassava is building a more diversified pipeline that could provide additional value drivers beyond their core Alzheimer's program.
Data show that simufilam reduced seizure activity in a preclinical mouse model
Presentation also highlights favorable human safety data from 1,929 patient Phase 3 program
AUSTIN, Texas, June 30, 2025 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (NASDAQ: SAVA, “Cassava”, the “Company”), a clinical-stage biotechnology company focused on developing novel, investigational treatments for central nervous system (CNS) disorders, today announced that the Company presented a poster at the TSC International Research Conference (TSC 2025) held June 26-28, 2025 in Bethesda, MD.
The poster, available on the Company website via this link, detailed two important observations that support Cassava’s plan to initiate human clinical studies in H1 2026 to evaluate simufilam as a treatment for Tuberous Sclerosis Complex (TSC)-related epilepsy:
- Preclinical proof-of-concept: Treatment with simufilam alleviated neuronal abnormalities and reduced seizure frequency by
60% compared to vehicle in a mouse model of focal onset seizures. - Clinical safety: Data from two human Phase 3 studies in Alzheimer’s disease demonstrated a favorable safety profile.
“My research and collaboration with the TSC Alliance demonstrate our commitment to address the high unmet need in people with TSC-related epilepsy. We are excited to share this research with the TSC community, including the preclinical data showing that simufilam significantly reduced seizure frequency. Additional pre-clinical studies are underway to further confirm these promising findings,” said Angélique Bordey, PhD, Senior Vice President, Neuroscience of Cassava.
“TSC leads to lifelong epilepsy and severe neurological issues for the approximately 50,000 patients in the US living with this rare disease. The data we presented at TSC 2025 mark the Company’s first presentation of simufilam’s positive results in a preclinical model. These compelling preclinical data build on favorable safety findings from prior human clinical studies and support our plan to begin our first clinical study in TSC-related epilepsy in the first half of 2026,” said Rick Barry, President and Chief Executive Officer of Cassava.
Poster Title and Highlights:
Title: Simufilam, a small molecule, reduced seizure activity in a mouse model of focal cortical malformations and has demonstrated a favorable clinical safety profile.
Brief Background: Tuberous sclerosis complex (TSC)-related epilepsy is a genetic disorder of the intracellular signaling pathway “mechanistic target of rapamycin” or mTOR, caused by mutations in the TSC1 or TSC2 gene. The presence of focal malformations in the brains of TSC patients is associated with lifelong epilepsy, leading to severe neurological issues. Recent evidence shows that expression of filamin A is increased in patients with TSC and in mouse models that recapitulate TSC and a related mTOR disorder, focal cortical dysplasia type II.
Simufilam is a proprietary, investigational oral small molecule that is hypothesized to modulate the function of the filamin A protein.
Preclinical Data:
Studies conducted in the lab of Angélique Bordey (the “Bordey lab”), at Yale School of Medicine, form the basis of the preclinical data. The Bordey lab showed that treating mice with simufilam, before or after seizure onset, alleviated neuronal abnormalities and reduced seizure frequency by
- Simufilam significantly reduced seizure frequency (p<0.0001).
- Significantly more mice achieved seizure freedom with simufilam (3/29 vehicle (saline) treated mice v. 11/32 simufilam treated mice, p=0.0343).
While the effective dose in mice was 20 mg/kg/day, chronic oral toxicology studies showed no adverse effects at doses up to 50 mg/kg/day in rats and 1250 mg/kg/day in mice.
Looking to the future, additional preclinical studies are underway with the TSC Alliance preclinical consortium and other collaborators to explore further simufilam's mechanism of action and its potential to treat TSC-related epilepsy.
Human Safety Data:
Data from two Phase 3 studies of simufilam in 1,929 patients with mild-to-moderate Alzheimer’s disease were also presented in the poster:
- Simufilam demonstrated a favorable safety profile.
- Non-serious adverse events were typically mild and not considered simufilam-related.
- None of the serious adverse events in these studies, or any other study, were assessed to be study-drug related.
About Simufilam
Simufilam is a proprietary, investigational oral small molecule that is hypothesized to modulate the function of the filamin A protein.
About TSC
Tuberous sclerosis complex (TSC) and focal cortical dysplasia (FCD) type II are neurodevelopmental disorders caused by mutations in the mechanistic target of rapamycin (mTOR) pathway genes. These mutations lead to focal malformations of the developing cortex and seizures in
Nearly two-thirds of TSC patients do not respond to antiepileptic drugs and experience lifelong seizures, leading to a spectrum of neurocognitive and psychological disabilities and poor quality of life. Current treatments, including antiepileptic drugs, mTOR analogs, and surgery, are not fully effective, are associated with serious adverse events, and/or are invasive.1
Cassava is initially focused on developing simufilam as a potential treatment for TSC-related seizures. According to the TSC Alliance, the disorder affects an estimated 1 in 6,000 live births. Approximately 50,000 people in the United States and more than one million worldwide live with TSC2.
Resources:
- Science Translational Medicine. 2020 Feb 19: https://pubmed.ncbi.nlm.nih.gov/32075941/
- https://www.tscalliance.org/understanding-tsc/what-is-tsc/
About Cassava Sciences, Inc.
Cassava Sciences, Inc. (NASDAQ: SAVA), is a clinical-stage biotechnology company focused on developing novel, investigational treatments, including simufilam, for central nervous system disorders, such as tuberous sclerosis complex (TSC)-related epilepsy, and potentially for additional pipeline indications. Simufilam is a proprietary, investigational oral small molecule that is hypothesized to modulate the function of the filamin A protein. The Company is based in Austin, Texas.
For more information, please visit: https://www.CassavaSciences.com
For More Information Contact:
Investors
Sandya von der Weid
svonderweid@lifesciadvisors.com
Company
Eric Schoen, Chief Financial Officer
(512) 501-2450
ESchoen@CassavaSciences.com
IR@cassavasciences.com
Cautionary Note Regarding Forward-Looking Statements:
This news release contains forward-looking statements that may include but are not limited to statements regarding: our plans to conduct preclinical studies of simufilam relating to seizures/epilepsy in TSC, the potential for simufilam to continue to deliver preclinical efficacy and activity related to seizures and epilepsy in TSC, the expected timing of our plans to conduct clinical studies with simufilam in H1 2026, the potential for simufilam as a treatment for TSC-related epilepsy and other potential indications, the potential for simufilam to continue to deliver favorable safety results, 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 advance preclinical studies related to TSC-related epilepsy and other potential indications, the ability to successfully carry out the Company’s obligations under the Yale License Agreement, 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. These 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 or preclinical studies 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 and development. Our research and development activities are long, complex, and costly and involve a high degree of risk. Holders of our common stock should carefully read our Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q in their entirety, including the risk factors therein. Because risk is fundamental to the process of drug discovery and development, you are cautioned not to invest in our publicly traded securities unless you are prepared to sustain a total loss of the money you have invested.
FAQ
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