A new drug shows promising results in trials, but questions remain about whether benefits outweigh side effects.

Findings suggest that the amyloid-targeting drug candidate slows cognitive decline in some people, but questions remain over its potential side effects

Results suggest that the amyloid-targeting drug candidate slows cognitive decline in some people, but questions remain over its potential side effects.

In July 2022, a bombshell dropped on the Alzheimer's disease research field. For years, researchers had searched for something that caused the disease’s telltale amyloid plaques — complex tangles of a protein called amyloid-beta (Aβ) frequently found in the brains of patients with neurodegenerative disorders. A series of studies published starting in the mid-2000s reported the discovery of a toxic form of Aβ in the brains of mouse models of Alzheimer's disease called Aβ*56. Researchers hoped that Aβ*56 was the protein that snowballed into those amyloid plaques. But a team of sleuths found that many of the papers describing Aβ*56 were fraudulent and contained an array of faked images and blots. The fraud seemed to call the entire idea of amyloids causing Alzheimer’s disease into question.

Predicting when new drugs come to market in the United States has never been an exact science, and it has become even harder since the start of the COVID-19 pandemic, experts told UPI. But some drugs on the horizon have the potential to make a major impact.

AB Science is initiating a Phase 3 trial to evaluate the safety and efficacy of its candidate therapy masitinib in patients with mild to moderate Alzheimer’s disease.

Newly announced findings from the phase 3 ACCORD trial (NCT04797715) showed that AXS-05 (Axsome Therapeutics), a novel, investigational n-methyl-D-aspartate (NMDA) receptor antagonist, met its primary end point of statistically significant delay in time to relapse and preventing relapse of agitation in patients with Alzheimer disease (AD) agitation.1

Biopharmaceutical company Axsome Therapeutics Inc. (AXSM:NASDAQ), which is focused on developing new medicines for use in the treatment of central nervous system (CNS) disorders, today announced that "AXS-05, a novel, oral, investigational NMDA receptor antagonist with multimodal activity, met the primary and key secondary endpoints in the ACCORD (Assessing Clinical Outcomes in Alzheimer's Disease Agitation) Phase 3 trial, by substantially and statistically significantly delaying the time to relapse and preventing relapse of agitation in patients with Alzheimer's disease, as compared to placebo."