Business
BridgeBio Pharma Announces Early Positive Data for BBP-812, its Investigational AAV9 Gene Therapy for Canavan Disease
- These results are the first reported demonstration of rapid and robust treatment changes in key disease markers associated with the severity of disease -
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- These results are the first reported demonstration of rapid and robust treatment changes in key disease markers associated with the severity of disease - Initial pharmacodynamic results for two participants show unprecedented decreases in N-acetylaspartate (NAA) in the brain and urine, suggesting the therapy is producing functional ASPA enzyme - If successful, BridgeBio’s gene therapy could be the first therapeutic option for children born with Canavan disease, a devastating and fatal neurodevelopmental disorder PALO ALTO, Calif., June 22, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced promising pharmacodynamic data from the first two participants dosed in CANaspire, its Phase 1/2 clinical trial of BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease. Canavan disease is an ultra-rare and fatal disease with no approved therapies. “Taken together, these robust decreases in urine, cerebrospinal fluid (CSF) and brain N-acetylaspartate (NAA), along with MRI signs of new myelination reported by the principal investigator are exciting and suggest we are on the right track when it comes to potentially making a difference for patients with this disease, and we look forward to gathering more data as the trial progresses,” said Genevieve Laforet, M.D., Ph.D., vice president of clinical development at Aspa Therapeutics, the BridgeBio Gene Therapy affiliate company developing the gene therapy for Canavan disease. “We are continuing to recruit and dose new participants for CANaspire and we are grateful to be able to collaborate with the advocacy organizations in the Canavan community in the pursuit of potential meaningful therapeutic advances for children with this cruel and fatal disorder.” Data from the first two CANaspire participants show rapid and robust post-treatment decreases in NAA in urine, and importantly, in CSF and brain tissue as shown by magnetic resonance spectroscopy (MRS), to a degree not seen in available natural history data. Reduction in brain NAA is an early signal suggesting that BBP-812 administered IV has reached its intended target behind the blood-brain-barrier and is expressing functional aspartoacylase (ASPA) enzy...