Price TargetThe price target is raised to $15, reflecting formal FDA alignment and risk-mitigated timelines.
Regulatory ApprovalThe FDA has indicated openness to a BLA submission for accelerated approval that includes clinical data from the ongoing phase 1/2 studies of LX2006 pooled with new clinical data from the planned pivotal study.
Safety ProfileLX2006 has been generally well tolerated with no grade 3+ serious adverse events to date, and no clinically significant complement activation or signs of frataxin over-expression observed in cardiac tissue.