BridgeBio Posts Early Data From Movement Disorder Candidate

 

• BridgeBio Pharma Inc 
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   has announced interim Phase 1 data from healthy volunteers, supporting the development of BBP-671 for pantothenate kinase-associated neurodegeneration (PKAN) and organic acidemias. 
• BBP-671 is being developed as a potential therapy for diseases in which coenzyme-A (CoA) metabolism is deficient, including PKAN, propionic acidemia (PA), and methylmalonic acidemia (MMA).
• BBP-671 was detected in healthy volunteer plasma and cerebrospinal fluid (CSF), which suggests BBP-671 is entering the brain.
• BBP-671 increased whole blood acetyl-CoA levels.
• The drug was generally well-tolerated following oral dosing in healthy volunteers, with no serious adverse events observed.
• Mild treatment-related emergent adverse events included headache, abdominal pain, and nausea.
• Asymptomatic neutropenia was observed in three individuals with repeat dosing of BBP-671.
• BridgeBio plans to move forward with the second part of its Phase 1 clinical study in patients with PA and MMA in 2H of 2022 and initiate a pivotal Phase 2/3 clinical study in PKAN in 2023.

https://www.benzinga.com/general/biotech/22/05/27425575/bridgebio-pharma-posts-early-data-from-neurological-movement-disorder-candidate