BridgeBio (BBIO) Goldman Sachs 45th Annual Global Healthcare Conference summary

Strategic approach and philosophy

• Emphasizes a Bayesian, evidence-weighted approach to decision-making, integrating molecular, cellular, and clinical data to guide program evaluation and development.

• Highlights the complexity of biological systems and the need to synthesize diverse data sources rather than rely solely on single-point clinical trial outcomes.

• Uses comparative analysis across programs, such as Infigratinib vs. Vismodegib and Acoramidis vs. Tafamidis, to inform strategic direction.

Clinical program updates

• Infigratinib phase II data in achondroplasia shows a 60% improvement in annualized height velocity (AHV) over prior trials, with statistically significant changes in proportionality.

• Data clustering indicates efficacy is not driven by outliers, and enrollment for phase III is progressing well, with readout expected next year.

• Regulatory pathway for Infigratinib remains on track, with 52-week data expected to be sufficient for review.

ATTR and Acoramidis developments

• Recent data at ISA conference linked higher serum TTR levels with reduced mortality and cardiovascular hospitalizations, supporting Acoramidis' clinical benefit.

• Ongoing and planned publications aim to further connect biomarker improvements to functional outcomes, with 1-year OLE follow-up data expected before the PDUFA in November.

• Regulatory interactions for Acoramidis have been positive, with labeling discussions set for late summer and commercial launch preparations underway.