BridgeBio shares preliminary data from CANaspire Phase 1/2 trial of BBP-812

BridgeBio (BBIO) Pharma presented positive preliminary data from eleven participants dosed in the CANaspire Phase 1/2 clinical trial of BBP-812, an investigational intravenous adeno-associated virus serotype 9 gene therapy in development for the treatment of Canavan disease. Highlights of the results presented at ESGCT 2024 include: Rapid and sustained reduction of NAA in urine and brain of participants post BBP-812 dosing; Urine NAA was reduced to levels associated with mild Canavan disease; NAA in CSF was reduced an average of 70% +/- 10% twelve months after receiving the low dose; Improved myelination on T-2-weighted magnetic resonance imaging observed in the majority of participants dosed with BBP-812; Progressive and continued post-dose improvement in gross motor function and achievement of motor milestones; In the low-dose cohort, these divergent trajectories result in statistically significant improvements in achieved motor function and milestones at 12-months after treatment with BBP-812; All participants demonstrated progress on administered developmental/motor assessments as measured by the GMFM-88, HINE-2, CDC milestones, or the Canavan Disease Rating Scale BBP-812 continues to be generally well-tolerated.