BridgeBio Starts Dosing In Early-Stage Genetic Metabolism Disorder Trial

  • BridgeBio Pharma Inc BBIO has dosed the first patient in its Phase 1 trial of BBP-671, an investigational oral therapy for conditions caused by coenzyme A (CoA) deficiencies, including propionic acidemia (PA), methylmalonic acidemia (MMA), and pantothenate kinase-associated neurodegeneration (PKAN).
  • PA and MMA are rare metabolic disorders in which the body cannot properly break down certain proteins and lipids (fats).
  • BBP-671 is an investigational oral therapy intended to increase CoA levels by allosterically modulating pantothenate kinases, key enzymes in the CoA biosynthesis pathway. 
  • Related: BridgeBio Pharma Touts Encouraging Data From Dwarfism Candidate.
  • The first part of the study evaluated BBP-671 in healthy individuals, and the second part of the study is evaluating BBP-671 in PA and MMA patients. 
  • Interim data from healthy individuals were reported earlier this year. 
  • The first patient dosed in the second part of the trial has PA. In this part of the study, up to eight PA and eight MMA patients will receive BBP-671. 
  • Initial data from the second part are expected in 1H of 2023. The company also plans to initiate a pivotal Phase 2/3 clinical study in PKAN in 2023.  
  • Price Action: BBIO shares are down 0.09% at $10.59 during the premarket session on the last check Thursday.
Market News and Data brought to you by Benzinga APIs
Posted In: BiotechNewsHealth CareSmall CapGeneralBriefs
Benzinga simplifies the market for smarter investing

Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.

Join Now: Free!