After years of controversy and allegations of doctored data, Cassava is moving on from Alzheimer’s.
Cassava Sciences has officially ended development of simufilam in Alzheimer’s disease, marking the end of a tumultuous era defined by controversy and failed clinical trials.
The company reported the end of the Alzheimer’s program Tuesday with the release of topline results from the Phase III REFOCUS-ALZ trial. Simufilam did not reduce cognitive or functional decline as compared to placebo in patients who had mild to moderate disease.
The program will be phased out completely by the end of the second quarter, Cassava said.
“We are disappointed that the results of REFOCUS-ALZ and RETHINK-ALZ showed no treatment benefit for patients with mild-to-moderate Alzheimer’s disease. These results were unambiguous,” CEO Rick Barry said in a statement.
The decision comes after years of challenges and upheaval for the company, which once touted simufilam as a breakthrough. In 2021, a whistle-blower claimed that Cassava doctored data for the drug, leading a prominent medical journal to pull some papers related to the drug. But others stood up for the company and its work. Cassava has also enjoyed the support of dedicated shareholders.
The SEC and DOJ both investigated Cassava, leading former CEO Remi Barbier to step down in July 2024. The SEC eventually sued the company for publishing misleading data on simufilam.
New CEO Barry pledged to release the next round of data on simufilam with more transparency. He followed through and in November 2024, the Phase III results from the ReThink-ALZ trial showed no effect on cognitive and functional decline in patients with mild-to-moderate Alzheimer’s disease. The drug also missed all secondary and biomarker endpoints.
Tuesday’s results add a period to the clinical failure, but Cassava had already signaled a decision to switch directions. In February the company licensed the rights to study simufilam in specific situations for seizures in neurodevelopmental disorders.
Now, the future of that program also appears shaky. Barry noted in Tuesday’s announcement the commencement of preclinical studies for tuberous sclerosis complex (TSC)-related epilepsy but said “we maintain ongoing strategic expense management efforts.”
CFO Eric Schoen said the company is “well-capitalized” with about $128.6 million in cash and cash equivalents as of December 31, 2024.
