Shots:

• The company reported the PD data from the two patients dosed in the P-I/II (CANaspire) trial to evaluate BBP-812 (IV) in patients with Canavan disease
• The results showed rapid & robust post-treatment reduction in NAA in urine (~50% & 81%) @6 & 3mos. post-treatment; CSF (77% & 89%) and brain (15% & >50%) as shown by MRS imaging suggesting that the therapy is producing functional ASPA enzyme & reached its intended target behind the blood-brain-barrier, well-tolerated with no treatment-related SAEs. The results will be presented at NTSAD 2022
• The safety, efficacy & PD updates from the P-I/II trial are expected in 2022. In the preclinical PoC studies, the therapy recovers survival & regular motor function in the same indication

Ref: GlobalNewswire | Image: BridgeBio