Cassava Sciences is forging ahead with a second phase 3 trial of its troubled Alzheimer's drug just three days after revealing a federal investigation into the company.
The Texas biotech has been through the ringer since August with concerns bubbling over claims that the company manipulated data for the experimental Alzheimer's med called simufilam. Cassava is cooperating with unnamed government agencies that have asked for corporate information and documents, the company revealed in a Monday filing with the U.S. Securities and Exchange Commission.
The agency in question is the SEC itself, the Wall Street Journal reported Wednesday. The National Institutes of Health, which has awarded more than $5.5 million in grants to Cassava since March 2020, is also taking a look at the claims, the Journal reported.
Investors appeared to find a little bit of upshot on the start of another late-stage trial. Shares hovered around 2.5% higher, at $48.21 apiece, as of 12:20 p.m. ET Thursday. That's a drastic drop from around $117 a pop in mid-August.
Cassava's troubles began in August when a citizens petition questioned the company's scientific integrity related to clinical trials conducted with simufilam. The petition urged the FDA to halt the studies. The authors are two physicians—a former neuroscience research chief at Johnson & Johnson and Eli Lilly, and the other a cardiologist at Weill Cornell Medicine—who shorted Cassava's stock and believe images included in multiple scientific journals were manipulated using Photoshop, according to the Journal. Other scientists claimed some images in articles about simufilam's trial results were copied and pasted.
The company and its outspoken CEO Remi Barbier have tried rebutting the petition's claims. But those efforts were dealt a blow when Quanterix, a digital health company cited in Cassava's attempt at correcting the record, denied any involvement in preparing some of the data being scrutinized.
Billions in market value were lost. The wild Wall Street ride for Cassava has made it the sixth-best performing U.S. stock of 2021, according to the Journal.
Despite all those woes, Cassava initiated the first phase 3 trial of simufilam in October, and now, the company is starting a second late-stage study of the oral drug.
"Developing a new drug solution for Alzheimer’s is a daunting task during the best of times. During these times of outlandish allegations made against us by short-sellers, we stand committed to translate what we believe is a promising scientific breakthrough into a potentially meaningful treatment for people with Alzheimer’s disease. The rest is noise," Barbier said in a statement announcing the new trial.
The studies come after Cassava rolled out some data from a 12-month interim analysis of the drug in September. The company claimed in the analysis that simufilam improved cognition scores compared to baseline in the first 50 patients to complete 12 months of treatment.
In the first phase 3 study, which began patient dosing this month, Cassava will test the safety and efficacy of a 100 mg dose of the treatment to enhance cognition and slow cognitive and functional decline. The trial, named RETHINK-ALZ, will be conducted in 750 patients for the course of 52 weeks.
The second phase 3 trial will study both a 100 mg dose and a 50 mg regimen for 78 weeks. About 1,000 patients with mild-to-moderate Alzheimer's in the U.S. and Canada will take part. International locations are slated to be added later.
The Cassava drama is just one chapter in this year's drama surrounding Alzheimer's disease drug development. The FDA controversially approved Biogen's Aduhelm in June based on biomarker data that suggests it could impact a known hallmark of the disease. But the approval was not based on the actual efficacy of the therapy—which will have to be proven for Biogen to keep the marketing authorization.
Different than Biogen's Aduhelm, Cassava's simufilam attempts to treat the debilitating disease by stabilizing a scaffolding protein referred to as filamin A. The petition claimed there is no link between that protein and the neurodegenerative disease.