Cassava Sciences Licenses Simufilam Method of Treatment Patent
Cassava Sciences (NASDAQ: SAVA) has secured a license agreement with Yale University for intellectual property rights, including an exclusive license to a US method of treatment patent for simufilam as a potential treatment for seizures related to rare neurodevelopmental disorders, particularly tuberous sclerosis complex (TSC).
The licensed patent stems from research led by Dr. Angélique Bordey at Yale, published in Science Translational Medicine in 2020. The study demonstrated that simufilam, Cassava's proprietary small molecule drug candidate targeting filamin A, showed promising results in reducing TSC-related seizure frequency in animal models.
The company plans to conduct preclinical studies in collaboration with the TSC Alliance to evaluate simufilam's potential as a treatment for TSC-related seizures and determine future development steps.
Cassava Sciences (NASDAQ: SAVA) ha ottenuto un accordo di licenza con l'Università di Yale per i diritti di proprietà intellettuale, inclusa una licenza esclusiva per un brevetto statunitense relativo a un metodo di trattamento per simufilam, considerato un potenziale trattamento per le crisi epilettiche legate a disturbi neuroevolutivi rari, in particolare il complesso di sclerosi tuberosa (TSC).
Il brevetto licenziato deriva da ricerche condotte dalla Dr.ssa Angélique Bordey presso Yale, pubblicate in Science Translational Medicine nel 2020. Lo studio ha dimostrato che simufilam, il candidato farmaco di piccole molecole proprietario di Cassava, mirato a filamina A, ha mostrato risultati promettenti nella riduzione della frequenza delle crisi legate al TSC in modelli animali.
L'azienda prevede di condurre studi preclinici in collaborazione con l'alleanza TSC per valutare il potenziale di simufilam come trattamento per le crisi legate al TSC e determinare i futuri passi di sviluppo.
Cassava Sciences (NASDAQ: SAVA) ha asegurado un acuerdo de licencia con la Universidad de Yale para los derechos de propiedad intelectual, incluida una licencia exclusiva para una patente de método de tratamiento en EE. UU. para simufilam como un posible tratamiento para las convulsiones relacionadas con trastornos neurodesarrollo raros, particularmente el complejo de esclerosis tuberosa (TSC).
La patente licenciada proviene de una investigación liderada por la Dra. Angélique Bordey en Yale, publicada en Science Translational Medicine en 2020. El estudio demostró que simufilam, el candidato a fármaco de pequeñas moléculas propietario de Cassava, dirigido a la filamina A, mostró resultados prometedores en la reducción de la frecuencia de convulsiones relacionadas con el TSC en modelos animales.
La empresa planea llevar a cabo estudios preclínicos en colaboración con la Alianza TSC para evaluar el potencial de simufilam como tratamiento para las convulsiones relacionadas con el TSC y determinar los pasos de desarrollo futuros.
카사바 사이언스(Cassava Sciences) (NASDAQ: SAVA)는 예일 대학교와 지적 재산권에 대한 라이센스 계약을 체결했으며, 여기에는 시무필람(simufilam)에 대한 미국 치료 방법 특허에 대한 독점 라이센스가 포함됩니다. 이는 드문 신경 발달 장애와 관련된 발작, 특히 결절성 경화증 복합체(TSC)에 대한 잠재적 치료제로 고려되고 있습니다.
라이센스된 특허는 예일 대학교의 앙젤리크 보르데이 박사가 주도한 연구에서 비롯되었으며, 2020년 Science Translational Medicine에 발표되었습니다. 이 연구는 카사바의 독점 소분자 약물 후보인 시무필람이 필라민 A를 표적으로 하여 TSC와 관련된 발작 빈도를 동물 모델에서 줄이는 데 유망한 결과를 보였음을 보여주었습니다.
회사는 TSC 연합과 협력하여 시무필람의 TSC 관련 발작 치료 가능성을 평가하고 향후 개발 단계를 결정하기 위한 전임상 연구를 수행할 계획입니다.
Cassava Sciences (NASDAQ: SAVA) a conclu un accord de licence avec l'Université de Yale pour les droits de propriété intellectuelle, y compris une licence exclusive pour un brevet américain relatif à une méthode de traitement pour simufilam, considéré comme un traitement potentiel pour les crises liées à des troubles neurodéveloppementaux rares, en particulier le complexe de sclérose tubéreuse (TSC).
Le brevet sous licence provient de recherches menées par la Dr Angélique Bordey à Yale, publiées dans Science Translational Medicine en 2020. L'étude a démontré que simufilam, le candidat médicament à petites molécules propriétaire de Cassava, ciblant la filamine A, a montré des résultats prometteurs dans la réduction de la fréquence des crises liées au TSC dans des modèles animaux.
L'entreprise prévoit de réaliser des études précliniques en collaboration avec l'Alliance TSC pour évaluer le potentiel de simufilam en tant que traitement des crises liées au TSC et déterminer les étapes de développement futures.
Cassava Sciences (NASDAQ: SAVA) hat eine Lizenzvereinbarung mit der Yale-Universität über geistige Eigentumsrechte abgeschlossen, einschließlich einer exklusiven Lizenz für ein US-Patent für eine Behandlungsmethode für simufilam als potenzielle Behandlung von Anfällen im Zusammenhang mit seltenen neurodevelopmentalen Störungen, insbesondere dem tuberösen Sklerosekomplex (TSC).
Das lizenzierte Patent stammt aus einer Forschung, die von Dr. Angélique Bordey an Yale geleitet wurde und 2020 in Science Translational Medicine veröffentlicht wurde. Die Studie zeigte, dass simufilam, Cassavas proprietärer Wirkstoffkandidat, der auf Filamin A abzielt, vielversprechende Ergebnisse bei der Reduzierung der Anfallshäufigkeit im Zusammenhang mit TSC in Tiermodellen zeigte.
Das Unternehmen plant, in Zusammenarbeit mit der TSC-Allianz präklinische Studien durchzuführen, um das Potenzial von simufilam als Behandlung für TSC-bezogene Anfälle zu bewerten und zukünftige Entwicklungsschritte zu bestimmen.
- Secured exclusive patent license for new therapeutic application
- Expands potential market into rare neurodevelopmental disorders
- Positive preclinical data showing efficacy in animal models
- Still in early preclinical stage for TSC indication
- Additional studies required before potential clinical trials
Insights
Cassava Sciences' licensing agreement with Yale University marks a strategic expansion of its simufilam development program beyond Alzheimer's disease into rare neurodevelopmental disorders, specifically targeting seizures related to tuberous sclerosis complex (TSC).
This diversification is particularly significant given Cassava's recent challenges in their Alzheimer's program, which has faced skepticism and controversy. With a market cap of just
The TSC market, while smaller than Alzheimer's, offers several strategic advantages:
- Potential for orphan drug designation, providing 7 years of market exclusivity and development incentives
- Clearer efficacy endpoints (seizure reduction) compared to the complex cognitive measures in Alzheimer's trials
- Potentially faster and less expensive development pathway
- Less competitive landscape than Alzheimer's market
The Science Translational Medicine publication provides scientific credibility that has been questioned in Cassava's Alzheimer's program. Working with the TSC Alliance also adds legitimacy and access to patient networks for future clinical trials.
For investors, this creates a potential secondary value driver while reducing overall program risk through indication diversification. However, this remains very early-stage, requiring substantial preclinical work before potential clinical trials. The company's cash resources (
- Cassava enters into a license agreement with Yale University granting intellectual property rights, including rights to an issued US method of treatment patent for potential treatment with simufilam of seizures related to rare neurodevelopmental disorders
- The licensed patent is based on research led by Yale University and published in Science Translational Medicine showing that treatment with simufilam appeared to meaningfully reduce seizure frequency in an animal model
- Cassava will prioritize initial development efforts on tuberous sclerosis complex (TSC)-related seizures
AUSTIN, Texas, Feb. 27, 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 it has entered into an agreement with Yale University (Yale) for a license to intellectual property rights, including an exclusive license to an issued US method of treatment patent (US 12,186,3071) for simufilam as a potential treatment for seizures related to rare neurodevelopmental disorders including tuberous sclerosis complex (TSC). Simufilam is Cassava’s proprietary small molecule drug candidate that targets filamin A.
The intellectual property that Cassava has licensed is based on the promising research and development work of Angélique Bordey, PhD, Professor of Neurosurgery and Vice Chair of Research, Neurosurgery at Yale, and her collaborators. In a paper published in Science Translational Medicine2 in 2020, the researchers showed that treatment with simufilam (also known as PTI-125) appeared to meaningfully reduce TSC-related seizure frequency in an animal model. These data formed the basis of the US method of treatment patent issued to Yale on January 7, 20251, which is included in the license to Cassava.
“We are pleased to enter into a license agreement with Yale University, based on the research of Dr. Angélique Bordey and her team. Dr. Bordey’s research opens the door to a potential new therapeutic application for simufilam in the treatment of seizures related to rare neurodevelopmental disorders, including tuberous sclerosis complex,” said Rick Barry, President and Chief Executive Officer of Cassava Sciences. “We plan to conduct preclinical studies in collaboration with the TSC Alliance to further evaluate simufilam’s potential as a treatment for TSC-related seizures and define next steps.”
About Simufilam
Simufilam is a proprietary, investigational oral small molecule that targets 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.2
Initially, Cassava will focus 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 TSC3.
Resources:
1. US Patent 12,186,307 B2
2. Science Translational Medicine. 2020 Feb 19: https://pubmed.ncbi.nlm.nih.gov/32075941/
3. 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 for central nervous system disorders, including Alzheimer’s disease and tuberous sclerosis complex (TSC)-related seizures. Simufilam is a proprietary, investigational oral small molecule that targets 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 van der Weid
svonderweid@lifesciadvisors.com
Media
media@cassavasciences.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 include but are not limited to statements regarding: the plan to conduct preclinical studies of simufilam relating to TSC-related epilepsy, opportunities for simufilam with respect to other neurodevelopmental disorders, the potential for simufilam as a treatment for TSC-related seizures and Cassava’s development priorities. 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 conduct or complete preclinical and clinical studies on expected timelines and the results of such studies; 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, 2023 and Quarterly Report on Form 10-Q for the period ended September 30, 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 preclinical studies and 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.
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FAQ
What is the new patent license Cassava Sciences (SAVA) acquired from Yale University?
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