Canavan Disease
Statistically significant change in gross motor function has been seen and motor milestones achieved in patients with high dose BBP-812 following 12 months compared to the natural course of the disease observed in those in the natural history study CANinform. Patients given low dose had significant and sustained reduction in N-acetylaspartate (NAA) levels in urine, cerebrospinal fluid (CSF), and brain with encouraging trends of more NAA reduction in those given high dose. As NAA builds-up in those with Canavan Disease due to an inability to break it down, this reduction is important and indicates that BBP-812 may have a meaningful impact on the disease.