BridgeBio Pharma Inc (NASDAQ:BBIO) released results from ATTRibute-CM Phase 3 study of acoramidis in transthyretin amyloid cardiomyopathy, or ATTR-CM.
A highly statistically significant improvement in the primary endpoint (a hierarchical analysis prioritizing in order: all-cause mortality, then the frequency of cardiovascular-related hospitalization, then change from baseline in NT-proBNP, then change from baseline in 6-minute walk distance) demonstrated by a Win Ratio of 1.8 (p<0.0001).
On-treatment survival rate of 81% versus placebo survival rate of 74% (absolute risk reduction of 6.43%; relative risk reduction of 25%).
A statistically significant relative risk reduction of 50% (p<0.0001) was observed on the frequency of cardiovascular-related hospitalization.
The company consistently observed a statistically significant treatment effect at 30 months across additional measured markers of morbidity, quality of life, and function:
No safety signals of potential clinical concern were identified.
In comparative exploratory post hoc analyses enabled by tafamidis drop-in, albeit at low patient numbers, acoramidis showed a 42% greater increase in serum TTR levels and a 92% improvement in median NT-proBNP relative to placebo + Pfizer Inc’s (NYSE:PFE) Vyndaqel/Vyndamax (tafamidis).
The company intends to file a New Drug Application with the FDA by the end of 2023.
Price Action: BBIO shares are up 59.30% at $29.30 during the premarket session on the last check Monday.
