Clinical Trial ResultsEarly trial results included partial reversal in C-peptide phenotype and improvements in visual acuity, which suggest a promising effect of the drug.
Market PotentialWolfram syndrome may have a higher prevalence than previously thought, indicating a larger potential market.
Regulatory MilestoneIncreased C-peptide in treated patients with reduction in HbA1C, allows the company to negotiate for a surrogate biomarker for Wolfram syndrome which might allow for a potential accelerated approval pathway.