Fosigotifator did not demonstrate a significant effect on disease progression, combining function and mortality measures, in either the primary or the exploratory high-dose groups.
Secondary endpoints, including the revised ALS functioning rating scale, respiratory function and health-related quality of life also showed no statistically significant improvement compared to the placebo. However, a slight signal was observed in muscle strength, with slower deterioration in both upper and lower extremities for patients in the high-dose group.
Despite the negative trial outcome for ALS, fosigotifator is still being studied for other conditions, including vanishing white matter disease and major depressive disorder.
