Galimedix Therapeutics announced the results of a new study at the Society for Neuroscience conference demonstrating that a single application of the investigational compound MRZ-99030, also known as GAL-101, causes sustained prevention of misfolded amyloid beta molecules from aggregating into toxic forms in vitro, neutralizing their ability to be toxic to neural tissues.
“These results are groundbreaking in the prevention of misfolded amyloid beta from creating toxic oligomers and their effects within the body,” Dr. Christopher Parsons, co-author of the study, senior editor of the journal, Neuropharmacology, and co-founder of Galimedix, said in a company news release. “While these results were presented at a neuroscience meeting, designed to bring additional light to new advances in neurodegenerative diseases, toxic oligomers of amyloid beta have been shown to play a significant role in the advancement of ophthalmic diseases, including glaucoma and dry age-related macular degeneration (dry AMD).”
“This poster shows how powerful a compound MRZ-99030 may be. Even after a thousand-fold dilution below predicted therapeutic levels, it prevented the formation of these toxic oligomers and even reversed their toxic effects by rapidly capturing the misfolded proteins into harmless “blobs” that continued their work even after the drug dropped below minimal levels. In clinical use, this may prevent or reverse the development of these diseases,” Dr. Parsons said.
Investigators had been aware that MRZ-99030 in low concentrations has proven to block the neural toxicity of amyloid beta in animal and in vitro models and was demonstrated safe in a phase 1 study in 70 human subjects. In this study, a therapeutic concentration of MRZ-99030 was shown to rapidly clear the toxic molecules into “blobs,” preventing further toxic effect on cells, and reversing the deficit in neural function caused by the toxic levels of misformed amyloid beta. The “blobs” were collected and resuspended in solution with the original concentration of misfolded amyloid beta with ten times lower concentration of MRZ-99030, which still blocked the toxicity to cells and reversed deficit. The dilution step was then repeated four more times, ultimately resulting in a concentration of MRZ-99030 at least 1,000 times lower than the predicted therapeutic level, while misfolded amyloid beta was kept at original toxic levels. Even then, toxicity was again blocked and deficit to neural function was reversed. GAL-101 is currently advancing toward Phase 2 studies in both glaucoma and dry AMD.
“GAL-101 eye drops may potentially provide sustained prevention of formation of toxic amyloid beta oligomers, clearing the system of these pathological factors, which has been shown in animal studies to lead to gradual removal of toxic beta amyloid deposits, and which the current “Hot Topic” poster has shown, could potentially reduce neural deficit and improve function,” added Dr. Andrew Pearlman, CEO and founder of Galimedix.