Clinical DevelopmentLRMR continues to execute clinical development of nomlabofusp in Friedreich's ataxia and remains on track for a BLA submission by year-end 2025.
Financial StabilityLRMR ended 2024 with $183.5M in cash, cash equivalents, and marketable securities, providing runway into the second quarter of 2026.
Regulatory ProgressThe FDA has designated skin cell frataxin levels as a Reasonably Likely Surrogate Endpoint that may support an accelerated approval.