ONO-2020 is a new type of drug targeting epigenetic pathways in Alzheimer’s Disease (AD) hopefully leading to better memory and cognition. The idea is that with the right amount of epigenetic modulation, there might be beneficial effects on AD related symptoms.
Epigenetics explores how the environment and our behavior modifies the expression of genes without changing DNA. Epigenetics could describe the behavioral differences in our offspring. Epigenetics is impacted by environmental factors; diet, stress, toxins, and lifestyle choices and is targeted by drug developers to change how cells function in progressive diseases, i.e. diabetes, cancer, and AD treatments.
The clinical trial is a phase II, double-blind, parallel-group, placebo-controlled study to assess safety, tolerability, pharmacokinetics, and efficacy of ONO-2020 in patients with mild to moderate AD. There is still a lot of research to be done to determine if ONO-2020 will be an important emerging drug to combat AD progression. This clinical trial is one step in a long process to prove the safety and efficacy of one of many evolving epigenetic modifiers in AD therapy.
Sources:
Shivraj Sohur U, O’Neill S, et al. (2025). A Study of ONO-2020 in Participants with Mild to Moderate Alzheimer’s Disease. Gov, Ono Pharmaceutical Co. Ltd. clinicaltrials.gov/study/NCT06881836?cond=Alzheimer%27s+Disease.
Jianbing Men, Xinyue Wang, Yunnuo Zhou, Yumeng Huang, Yue Zheng, Yingze Wang, Shuang Yang, Nan Chen, Nan Yan, Xiaoxu Duan. (2025). Neurodegenerative diseases: Epigenetic regulatory mechanisms and therapeutic potential. Cellular Signalling. 131(1).