“My hope is that within five years, we will be able to reverse more than 80% of early Alzheimer’s Disease, and that later, we will prevent it completely."

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GEN spoke to Maria Maccecchini, PhD, to discuss Annovis Bio, Inc.'s clinical trial results along with the company’s future plans. The company’s Alzheimer’s clinical trial positive results are the first double-blind, placebo-controlled study that shows cognitive improvements in Alzheimer’s patients as measured by ADAS-Cog and functional improvements in Parkinsons patients as measured by the Unified Parkinson’s Disease Rating Scale (UPDRS).

GEN: How would you rate the clinical trial results that were just reported?

Maccecchini : They are dramatic because of how we attack Alzheimer’s or Parkinson’s disease. The same approach is used whether we are dealing with frontotemporal dementia or PD. After 10 years of nobody really believing in what we were doing, we showed that a toxic cascade, including high levels of neurotoxic proteins, kills nerve cells. Amyloid-beta, tau, and alpha-synuclein are all toxic. If you just remove one, you still have the others. It so happens that the drug I licensed from the NIH, ANVS 401, has an extremely different mechanism of action compared to other approaches. It specifically inhibits the translation of all three neurotoxic aggregating proteins by targeting the conserved regions of each of their 5’ UTRs.

These neurotoxic proteins do their damage by inhibiting axonal transport from the brain to the periphery and other nerve cells. We have beautiful data that shows how ANVS 401 fully restores the speed of the transport of neuroinformation. So if you have sluggish cells, the information will move slower. Whatever you do will be slower. If the cells become normal, the information will move faster. That is the short-term effect. We knew from animals that the drug has a short- and a long-term effect. Short-term it improves axonal transport and long-term protects nerve cells from dying. In humans we observed that the drug improves cognition and motor skills after one month.

They are applying for an orphan drug designation for Alzheimer’s and for Down Syndrome. I don’t know anything about the Down Syndrome indication but people with Down Syndrome are very likely to suffer with Alzheimer’s when they get older.

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I believe that can’t be done, but hope it can be done.

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Alzheimer’s takes a lot of resources to treat because it is such a long, slow deterioration of the brain, and the patient is helpless to do much for themselves for a long part of it. Money spent on research for a cure for Alzheimer’s is money well spent. Both my parents died of brain diseases with symptoms like Alzheimer’s, but with a different underlying mechanism.

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Very good news, thank you.

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I hope so. My grandfather died in dementia. I’m scared I’ll get it too.

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