- BridgeBio Pharma Inc (NASDAQ:BBIO) announced promising pharmacodynamic data from the first two participants dosed in CANaspire Phase 1/2 trial of BBP-812, gene therapy for Canavan disease.
- Canavan disease is an ultra-rare inherited disorder that damages the ability of nerve cells (neurons) in the brain to send and receive messages.
- Data from the first two participants show rapid and robust post-treatment decreases in N-acetylaspartate (NAA) in urine and, significantly, in cerebrospinal fluid (CSF) and brain tissue, to a degree not seen in available natural history data.
- Also Read: BridgeBio Pharma Posts Early Data From Neurological Movement Disorder Candidate.
- Reduction in brain NAA is an early signal suggesting that BBP-812 administered IV has reached its target behind the blood-brain barrier and is expressing functional aspartoacylase enzyme.
- There is evidence in the scientific literature that lower NAA levels are associated with milder disease.
- BBP-812 has been well-tolerated, and no participants have experienced a treatment-related serious adverse event.
- The company said that while the data reported are still early and the final safety and efficacy profile of the investigational gene therapy remains to be fully established.
- BridgeBio believes these data show the potential of BBP-812.
- Price Action: BBIO shares are up 6.65% at $8.50 during the market session on the last check Wednesday.
