BridgeBio Pharma Presents Updated Encouraging Clinical and Biomarker Data from its BBP-812 Canavan Disease Gene Therapy Program at the 2023 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

- Following treatment, the N-acetylaspartate (NAA) levels of CANaspire participants are consistent with levels seen in individuals with milder Canavan disease based on findings from the Company’s natural history study and reports in the scientific literature

- Sustained reductions in NAA were measured in the urine, cerebrospinal fluid (CSF), and brain of all participants and have been observed for over a year in the earliest dosed participants

- After dosing, brain magnetic resonance imaging (MRI) of participants demonstrated robust improvements in the formation of myelin, or white matter, which is essential for brain development

- Positive changes in sitting ability and head control have also been observed using multiple assessments including the Gross Motor Function Measure-88 (GMFM-88), a standardized clinical outcome measure used by physical therapists to assess movement function in children

- If successful, BridgeBio’s gene therapy for Canavan disease could be the first therapeutic option for children born with this devastating and fatal neurodevelopmental disorder

PALO ALTO, Calif., May 22, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, presented promising positive data from six 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 a rapidly progressive, rare neurological disease that affects children from birth and has no treatment options beyond supportive care. The changes in key biomarkers, brain myelination and – critically – clinical function observed in children receiving BBP-812 in our study are compelling, and as we continue to advance our program we are hopeful that we will see outcomes that make a meaningful difference in the lives of children with Canavan disease and their families,” said Genevieve Laforet, M.D., Ph.D., vice president of clinical development at Aspa Therapeutics, the BridgeBio affiliate that is developing BBP-812 for Canavan disease. “We are beyond grateful to the children and their families who are participating in CANaspire as well as the study investigators, who together are making it possible to explore the potential of BBP-812 as a therapy for Canavan disease. We intend to work with the FDA to move our program forward as rapidly as possible, including exploring available expedited programs such as accelerated approval.”