BridgeBio Pharma Shares Preliminary Data From Gene Therapy Candidate In Ultra Rare Disorder

BridgeBio Pharma Shares Preliminary Data From Gene Therapy Candidate In Ultra Rare Disorder
  • BridgeBio Pharma Inc BBIO presented pharmacodynamic, tolerability, and preliminary functional efficacy data from the first three participants dosed in the CANaspire Phase 1/2 trial of BBP-812.
  • BBP-812 is an investigational intravenous gene therapy for Canavan disease, an ultra-rare neurodevelopmental disorder.
  • The disease is caused by an inherited mutation of the ASPA gene that codes for aspartoacylase, a protein that breaks down a compound called N-acetylaspartate (NAA). 
  • At Month 3 post-treatment, Participant 3 showed an 89% reduction of NAA in cerebrospinal fluid (CSF) and a 45% reduction in urine NAA.
  • In addition, Participant 2 showed a continued decline in urine NAA at Month 6, with an 85% decrease compared to pre-treatment.
  • In June, BridgeBio Pharma reported, at Month 6 post-treatment, Participant 1 showed a 77% reduction of NAA in CSF, a 15% reduction in NAA in brain white matter by MRS imaging, and a 45% reduction in urine NAA.
  • At Month 3 post-treatment, Participant 2 showed an 89% reduction of NAA in CSF, a 53% reduction in NAA in brain white matter by MRS imaging, and an 81% reduction in urine NAA.
  • BBP-812 has been well-tolerated by all 3 participants, with no reported treatment-related serious adverse events.
  • Price Action: BBIO shares are down 1.84% at $10.43 on the last check Friday.
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