Why BridgeBio Pharma Shares Are Trading Higher Today - BridgeBio Pharma (NASDAQ:BBIO)

BridgeBio Pharma Inc 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.

BridgeBio Pharma Posts Promising Data From Gene Therapy Trial In Ultra-Rare Disease