iAdore - Oral edaravone

An innovative platform trial

Within an innovative platform design study, we will evaluate the efficacy of multiple promising compounds, all of which have been shown to be safe in patients with ALS and have a high potential for therapeutic efficacy. Patients will be randomized into the three treatment arms based on their genetic profile. The primary aim is to assess the efficacy of the drugs versus placebo on overall survival. A large advantage of this design is that we can decrease the chance of being randomized to placebo, namely in a ratio of 2:1 instead of 1:1. In the future, other compounds may be added to this platform study.

iAdore – oral edaravone treatment arm

Edaravone is a formulation that reduces oxidative stress and is currently on the market in Japan and the United States in an intravenous formulation to treat ALS. As the chronic intravenous administration has important logistical drawbacks and safety concerns, an oral formulation of edaravone (TW001) was developed, which can be provided daily. TW001 has been shown to be safe and well-tolerated in three phase I studies. This treatment arm will investigate TW001 as treatment modality to prolong the life-expectancy in patients with ALS.