Shots:

• The first three patients have been dosed in the P-I/II (CANaspire) trial evaluating the safety, tolerability, and PD activity of BBP-812 (IV) in 18 patients with Canavan disease. The results incl. safety, efficacy & updates on PD data are expected in H1’23
• The results showed that patients treated with BBP-812 @6 & 3mos. achieved 77% & 89% reduction of NAA in CSF, 15% & 53% reduction in NAA in brain white matter by MRS imaging; 45% & 81% in urine NAA, was well-tolerated by all 3 participants with no reported treatment-related serious AEs
• The consistent reductions in CSF and brain NAA were observed to support the ability of BBP-812 to reach the CNS and express the active aspartoacylase (ASPA) enzyme

Ref: BridgeBio  | Image: BridgeBio