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New Support for Experimental Alzheimer’s Drug

Merck’s verubecestat, which is currently being tested in two Phase 3 studies, is safe, according to newly published Phase 1 data.

Written byJef Akst
| 2 min read

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progress for Alzheimer's drugWIKIPEDIA, INTERNET ARCHIVE BOOK IMAGESVerubecestat, an experimental Alzheimer’s drug developed by Merck Labs, is currently being tested in two ongoing Phase 3 trials. New results published today (November 2) in Science Translational Medicine provide “a summary of the discovery and early-stage profiling of what we hope is going to be a new therapeutic for Alzheimer’s,” Merck pharmacologist Matthew Kennedy told Scientific American. “It represents well over a decade of investment in this project by many, many scientists.”

The drug aims to block the production of plaques that are thought to promote Alzheimer’s disease–associated neurodegeneration. It does so by attaching to BACE1 (beta-site amyloid precursor protein cleaving enzyme 1), one of two enzymes that cleaves amyloid precursor protein (APP) into amyloid-β. Verubecestat reduced levels of amyloid-β in the blood and brains of rats and monkeys with no signs of toxicity, and proved safe and tolerable in a small, Phase 1 human trial, according to the report. The early-stage study also hinted at the drug’s efficacy: verubecestat reduced the amount of amyloid-β found in patients’ cerebrospinal fluid.

“This is the first detailed report of what a BACE inhibitor does in humans,” Harvard Medical School’s Dennis Selkoe, who was not involved in the work, told ...

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Meet the Author

  • Jef (an unusual nickname for Jennifer) got her master’s degree from Indiana University in April 2009 studying the mating behavior of seahorses. After four years of diving off the Gulf Coast of Tampa and performing behavioral experiments at the Tennessee Aquarium in Chattanooga, she left research to pursue a career in science writing. As The Scientist's managing editor, Jef edited features and oversaw the production of the TS Digest and quarterly print magazine. In 2022, her feature on uterus transplantation earned first place in the trade category of the Awards for Excellence in Health Care Journalism. She is a member of the National Association of Science Writers.

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