Dr. Maccecchini’s presentation highlighted the Company’s data that demonstrates by lowering amyloid, tau, and alpha-synuclein, the Company’s lead candidate, ANVS401, restores axonal transport and nerve cell health.
Hundreds of recent clinical trials individually targeting amyloid, tau, or alpha-synuclein in AD, PD or Down syndrome (DS) have failed. While these proteins at elevated levels turn toxic, just targeting one is not enough to protect the brain from neurodegeneration. Overexpression of neurotoxic proteins drives downstream events that dysregulate axonal transport, lead to inflammation, nerve cell death, and loss of function.
To test this hypothesis, trisomic DS mice were treated with ANVS401, an orally available small molecule shown to reduce amyloid precursor protein (APP), tau, and alpha-synuclein. ANVS401 lowered APP and phospho-tau, reversed Rab5 hyperactivation and restored retrograde transport. In transgenic APP-AD mice, ANVS401 lowered APP and its fragments in the brain and fully restored long-term potentiation, memory, and learning. In transgenic alpha-synuclein PD mice, it lowered alpha-synuclein in the gut and in the brain and fully restored affected functions.
“Let me explain the science of axonal transport for non-scientists: If we think about it, it is obvious that the brain is our information processing, storage and distribution center. In order for us to function, our brain needs to direct everything we do, say, feel and think. To do so, the bodies of the nerve cells are located in the brain, they have long arms called axons that crisscross the body and at the end of the arms there are fingers, called synapses, that touch everything. The fingers need to communicate with the body and the body needs to communicate with the fingers, in order for the nerve cells, the brain and for us to function. That communication is done by sending packages of information up and down the arm. If that communication does not work, the information flow is blocked, the nerve cells get sick and the function associated with the sick nerve cells is lost. That is why in neurodegenerative diseases functions are lost. These functions can be in the brain: memory, learning, executive function; or in the body: speech, shaking, gate; or in the eye: sight,” stated Dr. Maccecchini. “Our approach to neurodegeneration is unique when compared to the long list of failed trials exclusively targeting amyloid. The NYAS conference, the first major event of its type dedicated to non-amyloid approaches to AD, provided a great opportunity to share our unique approach with the scientific community.”
ANVS401 inhibits more than one neurotoxic protein and, thereby, improves axonal transport and reverses the toxic cascade leading to nerve cell death. The Company is presently in two ongoing double-blind, placebo-controlled Phase 2a studies that were designed to measure the toxic cascade that leads to nerve cell death and expects preliminary data in both AD and PD patients in the first quarter of 2021.
Statements in this press release contain “forward-looking statements” that are subject to substantial risks and uncertainties. Forward-looking statements contained in this press release may be identified by the use of words such as “anticipate,” “expect,” “believe,” “will,” “may,” “should,” “estimate,” “project,” “outlook,” “forecast” or other similar words, and include, without limitation, statements regarding the timing, effectiveness and anticipated results of ANVS401 clinical trials. Forward-looking statements are based on
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