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BridgeBio Reports Results of BBP-812 in the P-I/II (CANaspire) Trial for the Treatment of Canavan Disease

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BridgeBio Reports Results of BBP-812 in the P-I/II (CANaspire) Trial for the Treatment of Canavan Disease

BridgeBio Reports Results of BBP-812 in the P-I/II (CANaspire) Trial for the Treatment of Canavan Disease

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

Click here to­ read the full press release 

Neha

Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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