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Just caught that BridgeBio (BBIO) had their big presentation at MDA Conference a couple weeks back with those Phase 3 Fortify trial results for LGMD2I/R9. They showed the interim analysis actually demonstrated efficacy in this rare genetic disorder - that's pretty significant for such an underserved patient population. Stock was already up around 3% when they closed that day at $66.54, hit $67.53 in after-hours trading.
What's interesting is they didn't just drop one presentation. Besides Katherine Mathews delivering the main late-breaking data on the Fortify study from Iowa, Yale collaborators also presented on their high-throughput assay work for measuring ribitol response across FKRP variants. Plus four posters covering disease burden, treatment patterns, and the whole translational journey of BBP-418. Feels like they're building a pretty comprehensive case for this therapy.
BBP-418 is supposedly one of the most advanced investigational options for this condition. Hard to underestimate how big that is when you're looking at millions of patients worldwide with rare genetic diseases that basically have no effective treatments. The stock's been trading between $28 and $85 over the past year, so there's been some volatility. You following this one?