Clinical AdvancementsNomlabofusp has the potential to be the first therapy to address frataxin deficiency, which is the root cause of disease in Friedreich's ataxia.
Financial StabilityLRMR ended 2024 with $183.5M in cash, cash equivalents, and marketable securities, which provides runway into 2Q26.
Regulatory ApprovalThe FDA is open to considering skin frataxin levels as a potential novel surrogate endpoint for Accelerated Approval.