BridgeBio Pharma Presents P-I/II (CANaspire) Trial Results of BBP-812 for the Treatment of Canavan Disease at CNS 2022
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
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