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Research in ALS has always been extraordinarily difficult, due in part to the significant heterogeneity of the disease. Prior to 2017, 86 clinical trials exploring more than 50 compounds were conducted, yet no Phase 3 studies in ALS demonstrated a meaningful positive effect on slowing or eliminating functional disease progression.

Clinical research of our ALS treatment diverged from typical clinical development design strategies previously used for ALS studies. It required demonstration of clinically meaningful efficacy within a limited time period of only six months after first dose, instead of endpoints measured in years. It prioritized the importance of day-to-day capabilities — the ability to climb stairs, swallow food and others, using the ALSFRS-R functional rating scale.

Innovative efforts implemented by the development program helped to advance the conversation about current and future paradigms for ALS clinical trial studies.

We know people with ALS and their families never give up and neither did we. Our determined researchers went to work on a disease that was not well understood. Each clinical trial became an opportunity to learn more about ALS and how edaravone could benefit patients.

Our focus remains on bringing treatment to people living with ALS as we strive to make a meaningful difference in their lives. We continue to expand our work to deepen our understanding of ALS, explore potential biomarkers to aid in further research and seek new answers.

MTPC group companies have invested in a private biotech company focused on discovering and developing new precision therapies to target distinct subtypes of ALS, including a device to remove toxic proteins and a drug that restores a dysfunctional waste clearance system in cells. MTPC group companies also have in place several research partnerships to investigate potential effects on ALS.