Preclinical data has demonstrated that the oral small molecule OLX-07010 inhibits tau aggregation in a model for inherited tauopathies such as progressive supranuclear palsy (PSP) and frontotemporal dementia (FTD).
Earlier this year, the company Oligomerix announced the first-in-human dosing of OLX-07010 in a Phase Ia clinical trial.
“While there has been significant progress in the efforts to develop effective treatments for Alzheimer’s disease as demonstrated with the recent FDA full approval of Eisai and Biogen’s lecanemab and the potential for full approval of Lilly’s donanemab, there still remains a high unmet need for a therapeutic drug that is oral and self-administered, and that can treat patients in both developed and developing nations globally,” said William Erhardt, President and Head of Development & Operations at Oligomerix.
The self-association of tau protein into progressively larger aggregates and tau tangles is a common pathological process in Alzheimer’s disease (AD) and multiple rare inherited neurodegenerative diseases such as PSP and FTD.
Since tau tangles and amyloid plaques are hallmarks of AD, treating both could be more beneficial to patients than targeting a single pathology. Additionally, because tau pathology has been highly correlated with disease progression in AD, the researchers targeted tau self-association, the first step in tau aggregation.
Compared to controls, oral treatment with OLX-07010 over four months significantly prevented the development of tau aggregates in a mouse model.
“This study validates Oligomerix’s approach for inhibiting tau aggregation in a mouse model relevant to rare human neurodegenerative diseases such as PSP and FTD, which is caused by mutations in tau,” commented James Moe, CEO and Head Discovery & Strategy at Oligomerix, an author and Principal Investigator of the studies. “Our studies demonstrate that targeting the self-interaction of tau is an effective approach for inhibiting the accumulation of all sizes and forms of tau aggregates in disease.”
Edited by Diana Spencer, Senior Digital Content Editor, Drug Discovery World