Rethink-ALZ.com, New Website on Simufilam Phase 3 Studies, Is Live

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by Margarida Maia PhD |

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Cassava Sciences has launched a new website, called Rethink-ALZ.com, to make it easier for anyone interested to access information about two Phase 3 clinical studies of simufilam, the company’s oral investigational therapy for mild-to-moderate Alzheimer’s disease.

The trials, dubbed RETHINK-ALZ and REFOCUS-ALZ, are ongoing in the U.S. and Canada, and both are currently recruiting patients with Alzheimer’s.

“I think clinical sites around the country are quite excited by the potential of oral simufilam to impact Alzheimer’s disease,” James Kupiec, MD, chief clinical development officer at Cassava, said in a press release.

“Rethink-ALZ.com is dedicated to enhance patient experience and enrollment for both of our on-going Phase 3 studies of oral simufilam in Alzheimer’s disease,” he said.

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1st Phase 3 Simufilam Trial Enrolling Patients; 2nd Expected by Year’s End

The website includes a pre-screener questionnaire to be filled by patients with Alzheimer’s or their caregivers. The questionnaire gathers initial information about those interested in participating in the clinical studies to determine if entering a trial is actually right for them.

To be considered for either of the studies, participants must be 50 to 87 years old, have a diagnosis or suspicion of Alzheimer’s, and have experienced a gradual change in memory for six months or more. They also must have a caregiver who can be available as a study partner during the entire duration of the trial, expected to last one to 1.5 years.

Participants also must be fully vaccinated against COVID-19 or have had a previous infection with the COVID-19-causing virus. Additional criteria also may apply, the website states.

Study locations span from Florida to Washington in the U.S., and from British Columbia to Nova Scotia in Canada, and participants have the option to select their nearest among the many available.

Simufilam, previously known as PTI-125, is a small molecule designed to restore the normal shape and function of an altered form of a protein called filamin A. Altered filamin A causes two other proteins — beta-amyloid and tau — to clump together to form the toxic deposits that drive the death of nerve cells (neurons) in the brain of patients with Alzheimer’s.

The RETHINK-ALZ Phase 3 study (NCT04994483) will evaluate how safe simufilam is when taken as a 100-milligram (mg) pill by mouth twice a day for 52 weeks (about one year). It also will assess how well the therapy candidate works to slow the decline of patients’ abilities to perform cognitive and day-to-day tasks when compared with a placebo.

Secondary endpoints include neuropsychiatric symptoms, and stress or burden experienced by caregivers.

The study plans to enroll up to 750 patients with mild-to-moderate Alzheimer’s. More information about contacts and locations can be found here.

The other Phase 3 study (NCT05026177) is called REFOCUS-ALZ. It will evaluate how safe simufilam is when taken as a 50-mg or 100-mg pill twice daily for 76 weeks (about 1.5 years), and how well it works versus a placebo. Primary and secondary endpoints are similar to those of RETHINK-ALZ.

REFOCUS-ALZ is expected to enroll more than 1,000 patients, starting in North America and eventually in other parts of the globe. More information about contacts and locations can be found here.

Simufilam also is being tested in a Phase 2 study (NCT04388254), launched in 2020 in Canada and the U.S., in which patients receive 100 mg pills twice daily during a one-year open-label phase. Then, participants are randomly assigned to either continue taking simufilam or to be switched to a placebo for six months, after which all participants enter a final six-month open-label phase of simufilam. In an open-label trial, both participants and researchers know the medications that patients are receiving.

Interim data has shown that six months of dosing with simufilam improved cognition and behavior in patients with mild-to-moderate Alzheimer’s.