BridgeBio Pharma, Inc. (BBIO) Thursday reported positive preliminary data from the CANaspire Phase 1/2 study of its gene therapy candidate, BBP-812 for the treatment of Canavan disease, a rare genetic neurological disorder.
Results from the study showed progressive and continued post-dose improvement in gross motor function and achievement of motor milestones in participants treated with BBP-812.
Significant and sustained reductions in N-acetylaspartate (NAA) levels, which will be elevated in Canavan disease patients, in urine, cerebrospinal fluid (CSF), and brain were shown in all participants who received low dose of BBP-812. Further reduction in urine NAA was observed in participants who received high dose BBP-812. These results were presented at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) in Rome, Italy.
"Compared to the natural course of Canavan disease, in which most children do not achieve developmental milestones beyond that of a 6-month old, the fact that some children are sitting independently, taking steps, or even walking post-dosing is truly remarkable," said Florian Eichler, director of the leukodystrophy service at Massachusetts General Hospital, Center for Rare Neurological Disease and principal investigator of CANaspire.
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