The primary objective of the study is to evaluate the efficacy of MN-166 on patient’s functional activity measured by ALSFRS-R score and survival in ALS subjects. The secondary objectives are to evaluate the efficacy on muscle strength measured by hand-held dynamometry, quality of life measured by ALSAQ-5, safety and tolerability, and to characterize the pharmacokinetics (PK) of MN-166 using population PK modeling.

Amyotrophic lateral sclerosis (ALS) is a progressive, degenerative neurological disorder. It affects lower motor neurons and upper motor neurons and, frequently, prefrontal neurons.  

Riluzole is believed to delay disease progression and prolong survival by a few months. Edaravone has shown benefit in slowing disease progression, but its effect for survival is uncertain. There is a great need for safe, effective conventionally administered therapy for this fatal disease.

MN-166 is an orally available small molecule that penetrates the central nervous system well. It inhibits macrophage migration inhibitory factor and phosphodiesterases 3,4, and 10 with demonstrated neuroprotective action and glial cell attenuation in multiple in vitro and in vivo models. Based on findings from a completed Phase 1b/2a trial in ALS subjects, we hypothesize MN-166 in combination with riluzole can slow disease progression more effectively than riluzole alone.

This is a Phase 2b/3, multicenter, randomized, double-blind (12 months) placebo-controlled study followed by an open-label extension phase (6 months) in subjects with ALS on riluzole.  Subjects who meet entry criteria will be randomly assigned 1:1 to 1 of two treatment groups, MN-166 or matching placebo. We plan to enroll 230 subjects at 30 sites in US, Canada and Europe. (NCT04057898)

Study enrollment status and baseline characteristics of enrolled subjects will be available at the time of final presentation during Â鶹´«Ã½Ó³»­ 2020.