Three companies with Phase II data for amyotrophic lateral sclerosis believe they have identified at least two ways to improve the odds of clinical success, even though the disease has poorly understood biology, heterogeneous presentation and progression, and no markers of progression or treatment response.

The first is to develop compounds with known mechanisms of action that target features of the disease found in all ALS patients. The second is to employ a range of strategies to identify and augment signals of effectiveness in Phase II trials.