BridgeBio Pharma Reveals Early Positive Data For Canavan Disease

BridgeBio Pharma BBIO announced early positive data from the the first two participants dosed in CANaspire clinical study for the treatment of Canavan disease.

CANaspire is a Phase 1/2 open-label study designed to evaluate the safety, tolerability, and pharmacodynamic activity of AAV9 gene therapy candidate, BBP-812, for the treatment of Canavan disease.

The company said that the data readout from the first two CANaspire participants show rapid and robust post-treatment decreases in NAA in urine, 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) enzyme.

As the data reported are still early and the final safety and efficacy profile of the investigational gene therapy remains to be fully established, the company believes these data show the potential of BBP-812.

BridgeBio Pharma is planning to present these early positive data readouts on July 8, 2022, during Research Day at the National Tay Sachs & Allied Diseases Association Annual Family Conference in Denver, Colorado.

A full Phase 1/2 data readout for Canavan disease, including safety and efficacy data and updates on the pharmacodynamic data, is expected later in 2022. 

Canavan disease is an ultra-rare, disabling and fatal neurodevelopmental disorder disease with no approved therapy.

BridgeBio Pharma shares are trading higher 9 percent at $8.63