Cassava Sciences, a biotech company based in Austin, Texas, has announced that its Alzheimer’s disease drug candidate, simufilam, has failed its first Phase 3 clinical trial, dealing a significant blow to the company’s future prospects. The company also revealed that its second Phase 3 study has been halted, causing shares to plummet by 84%.
Simufilam, a small-molecule drug designed to target filamin A, operates through a distinct mechanism compared to existing amyloid-targeting Alzheimer’s therapies. However, in the ReThink-ALZ study, simufilam failed to show a statistically significant improvement in either cognition or functional ability, marking a major setback for the drug.
Rick Barry, CEO of Cassava, expressed disappointment over the results, noting that cognitive decline in the placebo group was less pronounced than seen in other Alzheimer’s trials, which made it difficult for simufilam to demonstrate its potential benefits. “A result like this has implications for our second Phase 3 trial, ReFocus-ALZ,” Barry stated. “We have made the difficult decision to discontinue ReFocus-ALZ, given the nature of these results.”
In the study, which involved 804 participants, those treated with simufilam showed a 2.8-point change on the ADAS-Cog12 cognition scale after 52 weeks, compared to a 3.2-point change in the placebo group. This difference was not statistically significant. Similarly, on the ADCS-ADL scale for functional ability, the simufilam group experienced a 3.3% decline, which was nearly identical to the 3.8% decline in the placebo group.
Barry confirmed that the company is analyzing the data and plans to present more detailed findings at a future medical conference. However, with both Phase 3 trials now halted, the future of simufilam appears uncertain.
This setback follows a turbulent period for Cassava. Just weeks ago, the company settled with the U.S. Securities and Exchange Commission (SEC) for $40 million over allegations of misleading statements regarding the results of a previous Phase 2b trial of simufilam. While the company did not admit to wrongdoing, the settlement highlighted ongoing scrutiny of its practices. The settlement was followed by a surge in the company’s stock price, which had soared above $120 in mid-2021 before experiencing a steady decline. As of now, Cassava’s shares are trading at just $4.30.
Additionally, Hoau-Yan Wang, a scientist affiliated with Cassava and based at the City University of New York, was implicated in manipulating clinical trial results and agreed to a $50,000 settlement with the SEC. Former CEO Remi Barbier and ex-head of neuroscience Dr. Lindsay Burns also reached settlements, agreeing to pay $175,000 and $85,000, respectively.
In a statement, Cassava indicated that it does not anticipate any further legal action by the Department of Justice stemming from the SEC investigation.
Despite the challenges facing its lead drug, Cassava reported having approximately $150 million in cash at the end of the third quarter. However, the company’s pipeline remains limited, with no other major developments besides an early-stage Alzheimer’s diagnostic in the works.
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