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First Trial of Alzheimer’s Drug Underway

Brain illustration


17 October 2017. The first clinical trial is underway of a therapy for stopping the progression of Alzheimer’s disease that purports to rebuild neural connections in the brain. The trial, sponsored by M3 Biotechnology Inc. in Seattle, is testing in humans for the first time its small-molecule drug code-named NDX-1017.

Alzheimer’s disease is a progressive neurodegenerative disease affecting growing numbers of older people worldwide. People with Alzheimer’s disease often have deposits of abnormal substances in spaces between brain cells, known as amyloid-beta peptides, as well as misfolded tangles of proteins inside brain cells known as tau. The Alzheimer’s Association says some 5.4 million individuals in the U.S. have the disorder, of which 5.2 million are age 65 or older. By 2050 that number is expected to increase to 16 million.

M3 Bio says NDX-1017 addresses receptors of proteins known as hepatocyte growth factors that stimulate growth of cells and tissue in several organs in the body. Among the functions provided by hepatocyte growth factors are self-repair of injured organs and protection against inflammation. In the case of NDX-1017, these properties are directed to the repair and protection of neurons, or nerve cells, in the brain.

The early-stage clinical trial is enrolling 92 individuals to test NDX-1017’s safety, tolerability, and chemical activity in the body. NDX-1017, given as injections under the skin, will be tested against a placebo among younger healthy adults, age 18 to 45, and older healthy individuals, age 60 to 75. The research team is testing single and multiple doses of the treatments at ascending levels, looking primarily for signs of adverse effects for up to 20 days. However, the team is also assessing concentrations of NDX-1017 in blood plasma over 48 hours and the length of time the drug remains in the blood.

M3 Bio began in 2013, while company co-founder and CEO Leen Kawas was a postdoctoral research associate in pharmacology and toxicology at Washington State University in Pullman. The company’s R&D is supported by grants from Alzheimer’s Drug Discovery Foundation and Dolby Family Ventures, as well as $12 million in equity raised in the company’s first venture funding round, according to the Seattle Times. Alzheimer’s Drug Discovery Foundation and Dolby Family Ventures, along with the W-Fund that supports tech start-ups in Washington State are also funding the clinical trial.

“Current drugs on the market for Alzheimer’s patients offer only symptomatic relief,” says Kawas in a company statement, “whereas we anticipate NDX-1017 will slow, halt and potentially restore lost function. The preclinical studies suggest we are on the right path, and we are excited to advance a much-needed brain regenerative therapy to alleviate the suffering of millions afflicted by the disease, and their families, around the world.”

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