BridgeBio Pharma Inc BBIO has announced data from the Phase 2 study of BBP-418 in patients with limb-girdle muscular dystrophy type 2i (LGMD2i), a group of conditions that cause weakness and wasting of the muscles in the arms and legs.
- The Company plans to engage with regulatory health bodies in 2022 to discuss potential paths to approval and intends to initiate a Phase 3 trial in 2H of 2022.
- At the Muscular Dystrophy Association (MDA) 2022, BridgeBio is also presenting Phase 1 trial data of BBP-418 in healthy volunteers to support its LGMD2i program.
- The Phase 1 study demonstrated overall tolerability across a wide range of dosing. No dose-limiting toxicity was observed.
- Related: Sentynl Buys BridgeBio Pharma's Fosdenopterin Rights In Genetic Disorder.
- Participants showed an average 0.21 or 43% increase in the glycosylated alpha-dystroglycan (αDG) ratio to total αDG from baseline.
- An average of 70% reduction in creatine kinase (CK), a key marker of muscle breakdown, was observed after 90 days of treatment and an average of 77% reduction after 180 days.
- All cohorts demonstrated a 3% increase in 10MWT velocity at day 90 and a 4% increase at day 180 for cohorts 1 and 2.
- Price Action: BBIO shares are up 0.83% at $8.65 during the market session on the last check Monday.
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