BridgeBio Pharma Posts Early Data From Neurological Movement Disorder Candidate

  • BridgeBio Pharma Inc BBIO 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.
  • Related: BridgeBio Inks Licensing Pact With Bristol Myers For SHP2 Inhibitor In Oncology.
  • 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.
  • Price Action: BBIO shares are up 0.38% at $6.66 during the market session on the last check Thursday.
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