Drugmaker Cassava believes its experimental Alzheimer’s therapy, which sent the company’s stock values surging with a recent phase 2 study announcement, could beat Biogen’s aducanumab to FDA approval. Alzheimer’s experts are hedging their bets.
Wall Street traders seem to have gained a new favorite potential Alzheimer’s treatment as shares of Cassava Science, a small and virtually unknown company, tripled recently on very early positive data on a drug called simufilam.
Interestingly, however, Alzheimer’s researchers and advocates are not the company’s largest fans, having heard little to nothing about simufilam, a drug that targets scaffolding proteins that help relay messages between cell membranes and the cell nucleus.
While Cassava’s stock price has dropped since its most recent highs, it is still significantly higher than what many observers would expect for a company with only nine staff members.
The stock rally started on Feb. 2 when Cassava issued a news release touting results of an interim analysis. The company said the study revealed that people who had received the drug demonstrated improved cognition and behavioral scores while showing no safety issues.
Cassava said it has met with the FDA to seek approval for a full phase 3 trial of simufilam. A new statement from the company outlined its plans for seeking FDA approval.
In the month before Cassava’s news release, the company was the focus of a very enthusiastic write-up in a market newsletter called Seeking Alpha. It was the start of the market buildup.
Calling the stock “drastically underpriced,” Seeking Alpha analyst Joe Springer said the company “could be the best-performing stock of 2021.”
Alzheimer’s experts are not so sure.
Meanwhile, George Vradenburg, chairman of UsAgainstAlzheimer’s, an advocacy group, called the market enthusiasm for Cassava a sign that stock traders are “begging for success right now.” Indeed, there are more than 100 drugs in the Alzheimer’s pipeline currently, but to date, disease modifying therapies have so far shown a 99-percent failure rate. “Too much reporting is based on press releases designed to pump up capital markets,” Vradenburg told Being Patient.
As to simufilam, Vradenburg said the company’s news releases were overly positive given that the drug has been tested on a very small population of 50 in a phase 2 study that has not even been fully recruited.
Dr. Jason Karlawish, a professor of medicine at the University of Pennsylvania, said the drug and the company was total news to him.
“A study design that is ‘open label’ is questionable — it means no control group and no blinding,” he explained. “Without those, a study is at high risk of bias. A controlled and blinded study is key to show efficacy.”
Rebecca M. Edelmayer, director of scientific engagement at the Alzheimer’s Association, called the company’s preliminary report “potentially encouraging” but noted many of the same concerns.
“The company’s data report is interesting,” Edelmayer said, “but it is important to first understand that it is an interim analysis of an open-label study with only 50 participants. It is not the final results and it is not a blinded, placebo-controlled trial.”
Cassava has received a total of $13 million in grants from the National Institute on Aging to study simufilam. In its interim report, Cassava said that simufilam improved cognition scores and decreased dementia-related behaviors such as anxiety, delusions and agitation.
Cassava President Remi Barbier said the company “could not be more pleased with these interim results.”
“We would have been satisfied to show simufilam stabilizes cognition in patients over six months. An improvement in cognition and behavior tells us this drug candidate has potential to provide lasting treatment effects for people living with Alzheimer’s disease. It’s an exciting development.”
Exciting but perhaps preliminary. Dr. James Ellison, the Swank Foundation Endowed Chair in Memory Care and Geriatrics at Christiana Care in Delaware, said that preliminary results like those being touted by Cassava are “often misleading.”
In addition, he noted the company claims that simufilam “might be the first disease modifying drug for Alzheimer’s disease to be FDA-approved.”
That, Dr. Ellison said, “seems a big claim given that the FDA is scheduled to announce an opinion on aducanumab in June.”