Clinical Data ConcernsConcerns remain regarding the durability of effect and the robustness of the data due to the small sample size and open-label nature.
Pipeline UncertaintyAnalyst remains on the sidelines until additional clarity on the potential of the pipeline programs is obtained and reiterates a Market Perform rating.
Regulatory ApprovalThe company is on track to initiate the Phase 1 LUMINA study of AMX00114 (anti-calpain-2 ASO) in ALS in Canada, but this dose was not yet given the go-ahead by the FDA, which requested more information leading to a clinical hold in the US.