- 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.
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