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 ProgramsThere is some near-term uncertainty about the next steps for the program, particularly regarding the design and timelines of a potentially registrational trial.
Regulatory ApprovalThe company is on track to initiate the Phase 1 LUMINA study of AMX00114 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.