The licensing deal follows years of controversy for Cassava, as well as the high-profile late-stage failure of its Alzheimer’s disease drug simufilam.
Embattled Cassava Sciences on Thursday entered into a licensing deal with Yale University, granting the biotech intellectual property rights to study its Alzheimer’s disease drug candidate simufilam in a specific way for seizures in neurodevelopmental disorders.
In particular, the license will give Cassava the ability to test simufilam as a treatment for seizures in patients suffering from rare conditions such as tuberculosis sclerosis complex (TSC). The deal covers U.S. patent 12,186,307, which protects the method of providing patients with a specific and “effective” amount of a filamin A modulator. Aside from TSC, the patent also names focal cortical dysplasia as a potential avenue of application.
The patent, built on the work of Yale’s Vice Chair of neurosurgery research Angélique Bordey, “opens the door to a potentially new therapeutic application for simufilam,” Cassava CEO Rick Barry said in a statement. Cassava will run preclinical studies for simufilam in TSC, with an eye toward positioning the asset as a therapy for seizures.
Simufilam is a small-molecule drug that is said to work by binding to an altered version of filamin A, which is typically involved in neurodegeneration, as seen in Alzheimer’s disease.
Over the past few years, simufilam and Cassava have been at the center of controversy. In 2021, a whistleblower accused the biotech of doctoring data for simufilam, a claim that Cassava strongly denied. The Alzheimer’s space became split in half in the succeeding months: The journal publisher Springer pulled select simufilam papers, while the Journal of Prevention of Alzheimer’s Disease stood up for Cassava.
The SEC and DOJ eventually launched probes—which did not end well for the biotech. In July 2024, while both investigations were still ongoing, Barry’s predecessor Remi Barbier stepped down as Cassava’s chief executive. A few months later, in September 2024, the SEC sued the company for allegedly publishing misleading data for simufilam.
All the while, Cassava stood strong by simufilam’s side, maintaining that the molecule was indeed effective against Alzheimer’s. In November 2024, however, that all fell apart when simufilam’s Phase III trial ended in failure. Compared with placebo, Cassava’s drug was unable to significantly slow cognitive and functional decline in patients with mild-to-moderate Alzheimer’s disease. Simufilam also missed all pre-specified secondary and exploratory biomarker endpoints.
The late-stage flop forced Cassava to discontinue a second Phase III Alzheimer’s trial of simufilam, as well as an expanded access program.
All of this has happened against the backdrop of two successful Alzheimer’s drugs finally entering the market in Eisai and Biogen’s Leqembi and Eli Lilly’s Kisunla.
