Clinical Efficacy And Biomarker SupportExtended clinical data showed normalization of a cerebrospinal fluid biomarker alongside stabilization of cognitive measures and organ sizes with treatment, supporting a favorable benefit profile.
Non-dilutive Financing PotentialExisting capital, a committed venture debt facility, and restored eligibility to receive and sell a Priority Review Voucher create potential non-dilutive funding that can ease financing pressure.
Regulatory Pathway ClarityFDA feedback indicated integrated clinical and natural history data together with a cerebrospinal fluid biomarker could support an accelerated approval pathway, reducing regulatory uncertainty.