M3 Biotechnology Begins Testing of Alzheimer’s Therapy in Adults with the Disease

  • NDX-1017 is intented to halt or reverse Alzheimer's
  • It a completely different mechanism of action than other drug candidates currently in development
  • NDX-1017 addresses receptors of proteins known as hepatocyte growth factors


M3 Biotechnology, announced on 9/13/18 that is has begun testing its lead therapy in individuals with Alzheimer’s disease or mild cognitive impairment in the United States and France.
The investigational drug, NDX-1017, is novel because it is designed to halt or reverse the effects of Alzheimer’s disease, rather than just slow its progression.

“NDX-1017 is a novel therapy that employs a completely different mechanism of action than other drug candidates currently in development,” said Leen Kawas, PhD, CEO of M3 Biotechnology. “By amplifying key brain receptors, our novel therapy is designed to impact neurodegeneration and also promote the regeneration of brain tissue.”

Phase 1 testing for safety and tolerability in healthy young individuals is complete and testing in healthy elderly individuals is nearly complete. Preliminary results suggest the compound is well tolerated and exposure levels compare favorably to preclinical studies.

Preclinical trials of NDX-1017 have demonstrated the ability of the compound to promote neuro-regeneration and improve cognitive function. While the compound shows promise for a range of neurodegenerative diseases, including Parkinson’s, M3 Bio’s initial focus is in Alzheimer’s disease. The Phase 1 clinical trial is being conducted by Biotrial, Inc.

NDX-1017 is a small molecule that the company says 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.

In November of 2017, M3 Announced that it has raised $15.2 Million to support the development of  NDX-1017.