Cassava Sciences Inc (SAVA)

[TonyJoe1957]

Biotechnology brings new approach and fresh hope to Alzheimer’s research - nj.com
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As our scientific understanding of Alzheimer’s disease gets better, so do the options for treating it, and one of the more promising is a new biotech drug being tested here in New Jersey.

The Princeton Medical Institute is looking for older adults with mild to moderate Alzheimer’s disease to enroll in a clinical trial for a medicine designed to work in a novel way, targeting an obscure protein that seems to trigger the progression of the disease.

The drug, simufilam, showed encouraging results in a preliminary study, significantly improving cognition in Alzheimer’s patients, while reducing the psychiatric symptoms associated with the disease.

Now, researchers like Dr. Jeffrey T. Apter are looking to validate that result in a much larger, Phase III study that will involve some 1,500 patients at Alzheimer’s clinics across the country.

“I got interested in simufilam when I saw some of the science behind it,” says Apter, founder and president of Global Medical Institutes and lead principal investigator for PMI. “It’s exciting. This is the only Alzheimer’s drug I know of in development that improves cognition. It’s not only preventative. It’s improving cognition and helping with memory at the same time.”

The drug is being developed by Cassava Sciences, a biotech company based in Austin, Texas. It reflects a recent emphasis on broadening Alzheimer’s research beyond its traditional focus on trying to clear away the beta-amyloid “plaque” that is often associated with the disease.

Simufilam does not directly target beta-amyloid. Rather, it’s a small molecule that helps restore the function of a protein called filamin A, which is known to “misfold” in the brain of people with Alzheimer’s. This misfolding is believed to contribute to inflammation and build-up of the beta-amyloid plaque. Simufilam works to return the protein to its proper shape and function.

In a Phase II study involving 150 people, early results showed significant benefits, including an 84% reduction in beta-amyloid plaque and a 38% reduction in tau, the so-called “tangles” in the brain that also are the hallmarks of Alzheimer’s.

The results, which were presented at the Alzheimer’s Association International Conference in July, also showed that:

Roughly two-thirds of people in the trial saw their cognition improve, while others experienced cognitive decline that was slower than expected.
The percentage of Alzheimer’s patients experiencing psychiatric symptoms such as anxiety or delusions dropped from 66% to 50%.
The preliminary research involved an “open-label” approach, meaning everyone who participated got the drug. Experts say these results should be viewed with caution, because the outcome wasn’t measured against a group of patients who took a placebo.

The Phase III clinical trial now beginning at the Princeton Medical Institute and other sites will not be subject to that concern. It will test the validity of the drug across a much larger population of patients, and will include a placebo group.

The study is open to adults between the ages of 50 and 87 who have mild or moderate Alzheimer’s disease. The trial has not begun yet, but Apter is pre-screening possible candidates. He’s looking to recruit about a dozen volunteers for the study at his site in Princeton.

Participants will be randomly assigned to receive either a placebo or 100 milligrams tablets of simufilam twice a day for a year.

For more information or to be considered for a clinical trial, call the Princeton Medical Institute at 609-921-6050 or visit the PMI website.

Apter has worked in Alzheimer’s research for more than two decades and was involved in clinical trials that led to FDA approval of such early Alzheimer’s drugs as Aricept and Namenda. He is an attending physician at Princeton Health and Penn Medicine and was recently appointed to the board of Alzheimer’s New Jersey.

The field of Alzheimer’s research is emerging from a long period of frustration and disappointment as one failed clinical trial followed another. Most of that research focused on medicines that targeted beta-amyloid plaque, leading some researchers — including Apter — to question what’s called the “amyloid theory.”

His efforts have shifted recently to medications that target Alzheimer’s in other ways. The new Cassava drug is one such example. Another is a medication being developed by the pharmaceutical firm Janssen, a subsidiary of New Brunswick-based Johnson & Johnson, that targets tau in the brain. The Janssen study, called Autonomy, is a Phase II clinical trial that involves an infusion once a month to see if the medication can slow abnormal tau from spreading in the brain and delay memory loss in the early stage of Alzheimer’s disease.

Research on beta-amyloid drugs continues, and one of those drugs, Aduhelm, recently overcame questions about its effectiveness to become the first new drug approved by the FDA for the treatment of Alzheimer’s in nearly two decades.

Apter says Alzheimer’s is a complex disease that involves many factors, including not just the amyloid plaque and the tau tangles, but also inflammation. He’s encouraged by the early results for simufilam because it showed an effect on all of those, including brain inflammation.

“We’re excited about this drug, because it stops beta-induced neuroinflammation,” Apter says. “This type of study gives us a lot of optimism, because it’s not just addressing amyloid. It’s multi-factorial in addressing Alzheimer’s.”

Like many researchers, he expects the eventual treatment of Alzheimer’s to involve some combination of medications, which varies from patient to patient. “It probably won’t just be one drug,” Apter says.

That’s one reason why patients who express an interest in a particular trial at the Princeton Medical Institute are evaluated for other studies as well.

“We’re running many studies, and not every study is for every person,” Apter says. “We try to find the best study for them.”

He says having the Cassava drug and the Janssen drug in the pipeline bodes well for the future of Alzheimer’s treatment. “These are the most promising drugs in development right now,” Apter says.

Tony Dearing may be reached at [e-mail]. Follow him on Twitter @TonyDearing. Find NJ.com on Facebook.

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