Cassava Sciences Licenses Simufilam Method of Treatment Patent
- 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 80% to 90% of patients.
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
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Media
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Company
Eric Schoen, Chief Financial Officer
(512) 501-2450
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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.
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