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Blocking a Key Step in the Neurodegenerative Process with Highly Specific Small Molecules

By blocking a key step in the neurodegenerative process, Galimedix’s technology prevents the formation of toxic amyloid beta (Aβ) oligomers and protofibrils instead of removing already formed toxic species like most products in development and on the market do. Pre-clinical data support that our product candidates can block formation and remove the whole spectrum of possible toxic Aβ oligomers and do this in both the brain and the retina, without affecting physiological neuronal function.

Current Understanding of Aβ Pathology as Cause of Neurodegeneration

Many studies have indicated that toxic Aβ oligomers and protofibrils are an underlying cause of neurodegenerative diseases of the retina in the eye. These toxic Aβ species have recently been validated as a key target in Alzheimer’s disease through the approval and and promising Phase 3 results of anti-Aβ drugs, lecanemab and donanemab.

As highlighted below, normal Aβ monomers are important to neuronal function but misfolding of these monomers can lead to the development of toxic forms of Aβ that can be highly damaging to the retina and brain cells. This process is outlined here.

Toxic Aβ damages neurons, which leads to activation of the complement cascade, loss of cell function, apoptosis and eventual cell death.

Aβ – A Key Target for Both Eye and Brain
Neurodegenerative Diseases

Evidence suggests the same mechanisms are involved in retinal diseases and the devastating brain condition, Alzheimer’s disease (AD). These striking similarities show that the accumulation of toxic Aβ is a key overlapping feature between three neurodegenerative disorders that affect millions of older adults worldwide: AD, glaucoma, and dry age-related macular degeneration (dry AMD). All three disorders are chronic, age-related disorders with no known cure and lead to irreversible neuronal damage and loss of function.

Product Candidates Designed to Prevent Toxic Aβ Oligomer Formation at the Source

Galimedix is developing cutting-edge orally and topically administered Aβ aggregation modulators that target the beginning of the Aβ peptide aggregation cascade. Our lead candidate GAL-101 acts upstream to most other Aβ-targeting approaches on the market and in advanced development, potentially enabling it to effectively impact disease progression without disturbing normal neuronal function.

As shown above, Galimedix’s molecules prevent the formation of all toxic Aβ oligomers that drive neurodegeneration at the source by binding to a specific motif only exposed in the misfolded monomers, triggering the clustering of these misfolded Aβ monomers and preventing the assembly of harmful oligomers. These clusters collect and detoxify Aβ. 

Trigger Effect Provides Lasting Efficacy Beyond Drug Administration

Of interest, the clusters bind further misfolded monomers so that this process continues even when there is no drug left in the tissue. This self-propagating detoxification or “trigger effect” results in a sustained effect lasting far longer than the time the drug remains after a single administration at therapeutic levels in the system. This trigger effect has been patented by Galimedix.