Simufilam fails to meet endpoints in phase 3 Alzheimer’s study, trial discontinued

Topline results from the phase 3 ReThink-ALZ study of simufilam in mild to moderate Alzheimer’s disease showed the investigational therapeutic did not meet any of its prespecified endpoints.

As a result, manufacturer Cassava Sciences Inc. stated in a press release that the clinical trial will be discontinued.

ReThink-ALZ sought to evaluate safety and efficacy of simufilam (PTI-125) in a multicenter, double-blind, placebo-controlled, randomized parallel group study conducted at more than 75 sites in the United States, Canada and Australia.

A total of 804 individuals with confirmed mild to moderate AD were randomly assigned on a 1:1 basis to receive either oral simufilam 100 mg or a matching placebo, twice daily for 52 weeks.

The co-primary endpoints were change in cognition and function from baseline to week 52, as measured by both the Alzheimer’s Disease Assessment Scale-Cognitive Subscale-12 (ADAS-COG12) and Alzheimer’s Disease Cooperative Study ADL scale (ADCS-ADL). Although simufilam did not achieve significant change, the company noted the drug demonstrated a favorable safety profile.

“The results are disappointing for patients and their families who are living with this disease and physicians who have been looking for novel treatment options,” Rick Barry, president and CEO of Cassava, said in the release. “A result like this has implications on our second phase 3 trial, ReFocus-ALZ. We have made the difficult decision to discontinue ReFocus-ALZ, given the nature of today’s reported results.”