Edaravone is a free radical scavenger currently used in Asia for the treatment of stroke and Amyotrophic Lateral Sclerosis (ALS) and in the USA for the treatment of ALS. It is a unique antioxidant as it is able to cross the blood-brain-barrier, a common problem with other antioxidants. In addition, edaravone inhibits lipid peroxidation as efficiently as some well-known antioxidants such as vitamin E and vitamin C and it reacts with peroxynitrite much faster than uric acid, a good physiological peroxynitrite scavenger. Treeway has developed an oral formulation of edaravone (TW001) and currently a Phase 3 trial in ALS patients in Europe is ongoing. It is hypothesized that an antioxidant therapy might be a promising treatment strategy for Alzheimer’s Disease (AD) as oxidative stress plays an important role in the development and progression of this disease. Current treatment options for AD however, do not target oxidative stress. TW001 has the potential to slow down AD progression, thus improving the patient’s quality of life. In the long-term, TW001 has the potential to be used in a combination therapy with the aim to completely halt disease progression.

Treeway TW001AD BV received a funding from the ADDF, reference GC-2013807, to perform a Phase II study to determine the preliminary efficacy and safety of oral edaravone (TW001) in Alzheimer Patients. This project started in November 2020, and progress has been made since then. Ethical approval to start the clinical trial has been obtained, however, additional budget will be necessary for the current program for the following reasons:

-       Significant delay and therefore additional costs in the program due to the COVID-19 pandemic

-       Inclusion of additional: 

• clinical sites in two more countries; 
• biomarkers and cognitive functional composite (CFC).

This grant application is an extension of the project GC-2013807.