BridgeBio posts early data for gene therapy targeting Canavan disease

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The shares of BridgeBio Pharma (NASDAQ:BBIO) traded higher in the morning hours Wednesday after the clinical-stage biotech announced encouraging data from the first two subjects who received the company’s Canavan disease candidate BP-812 in a Phase 1/2 clinical trial.

An ultra-rare and fatal disorder, Canavan disease is caused by a mutation of the ASPA gene that codes for aspartoacylase (ASPA), a protein that breaks down a compound called N-acetylaspartate (NAA). Genetically inherited Canavan disease has no approved therapies.

Highlights of the results include a 77% decline of NAA in the cerebrospinal fluid of the first patient six months after the treatment.

Meanwhile, the second patient was found to have an 89% reduction of NAA in CSF at month three post-treatment.

A decline in brain NAA signals that the AAV9-based gene therapy candidate BP-812 reached its target behind the blood-brain-barrier expressing the functional ASPA enzyme, BridgeBio (BBIO) said.

So far, none of the patients have experienced a drug-related serious adverse event, and BBP-812 was well tolerated, the company added.

BridgeBio (BBIO) anticipates more data from the trial later this year.

Read: Seeking Alpha contributor Avisol Capital Partners thinks that BridgeBio (BBIO) has yet to de-risk its business model.