Exebryl-1®: POTENTIAL PROMISING THERAPY FOR
Exebryl-1® is a small molecule compound that has been shown to prevent the formation and accumulation of the soluble and insoluble forms of beta-amyloid protein, as well as amyloid plaques, found in Alzheimer's brains of disease victims.
During the last eight years, ProteoTech, through its world-renowned investigators of amyloid disease research, has worked on the development of disease-modifying small molecule therapeutics specifically targeting beta-amyloid protein accumulation and fibrillogenesis for the treatment of Alzheimer's disease.
Exebryl-1® was identified from a unique synthetic compound and analog library established at ProteoTech, focused on targeting specific amyloid proteins. In early studies, molecular modeling and structure-activity-relationship (SAR) studies identified a pharmacophore that led to the design, synthesis and testing of a significant number of small molecule analogs for beta-amyloid protein inhibitory activity using in vitro and transgenic animal models.
Exebryl-1® had impressive effects on beta-amyloid protein fibrillogenesis in a number of different in vitro assays and transgenic animal models, and was found to specifically reduce beta-amyloid protein fibril formation, deposition and accumulation and to induce removal from brain.
Initial studies suggest that Exebryl-1® may also have an important dual capacity by inhibiting and reducing tau protein from forming paired helical filaments, important in neurofibrillary tangle formation. The presence of neurofibrillary tangles in brain containing tau protein is an important pathological hallmark of Alzheimer's disease. Further studies are ongoing to confirm these exciting findings.
Exebryl-1® may be the first orally bioavailable small molecule drug that affects both amyloid plaque and neurofibrillary tangle accumulation, the two major characteristic and pathological lesions of Alzheimer's disease.
ProteoTech has already completed a Phase 1a Human Clinical Trial to determine the safety of this new compound prior to efficacy studies or the treatment of mild to moderate stages of Alzheimer's disease. The drug was found to be safe with little-to-no adverse effects reported. ProteoTech is currently optimizing Exebryl-1® using enhanced formulation technology before completing Phase 1b and Phase 2a proof-of-concept trials.
ProteoTech International Alzheimer's Conference Presentation - Exebryl-1®