BridgeBio Pharma Inc (NASDAQ:BBIO) announced updated data from its ongoing Phase 2 open-label extension (OLE) study of acoramidis (AG10) in symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM).
- Acoramidis was generally well-tolerated and resulted in sustained, near-complete TTR stabilization as measured by established ex vivo assays and increased serum TTR levels.
- Median N-terminal Pro-brain natriuretic peptide (NT-ProBNP) was stable or improving in trial participants throughout the OLE.
- Serum TTR levels were sustainably increased from baseline, with mean concentration rising from 21.55 mg/dL at baseline to 30.06 mg/dL at Month 30 (+41%).
- Related: BridgeBio Pharma Posts Updated Data From Muscular Dystrophy Trial, Plots Path To Market.
- Near-complete stabilization was verified with a mean stabilization of 102.5 ± 8.9% at Month 30.
- Median NT-proBNP was stable or improving in study participants. At Month 30, the median change from baseline in NT-proBNP was -437 pg/mL.
- 68% of participants with available samples at Month 30 (15/22) had NT-proBNP levels below their baseline, suggesting an improvement in their heart failure severity
- BridgeBio's Phase 3 study investigating acoramidis in ATTR-CM (ATTRibute-CM) is ongoing, with Month 30 topline data expected in mid-2023.
- In the Month 12 readout, no benefit of acoramidis relative to placebo was observed on the six-minute walk test. Still, improvements in the Kansas City Cardiomyopathy Questionnaire Overall Score, NT-proBNP, and serum TTR level were observed.
- Price Action: BBIO shares closed 6.90% higher at $10.85 on Friday
