BridgeBio: positive results from Phase 3 in transthyretin amyloid cardiomyopathy

 The primary endpoint was met (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001); this primary endpoint result consistently favored acoramidis treatment across key subgroups, including across both variant and wild-type ATTR patients as well as across New York Heart Association (NYHA) Class I, II, and III patients. In particular, in contrast to results observed in prior studies of TTR stabilizers, consistency against cardiovascular-related hospitalizations (CVH) was observed across all prespecified subgroups at 30 months

- Absolute values observed across all-cause mortality (ACM), cardiovascular mortality (CVM) and CVH showed that over 30 months, patients survived more and were hospitalized less than has been seen in prior controlled studies of ATTR-CM to the company’s knowledge

- The 81% survival rate on acoramidis approaches the survival rate in the age-matched US database (~85%)

- The 0.29 mean annual CVH rate on acoramidis approaches the annual hospitalization rate observed in the broader US Medicare population (~0.26)

- Assessment of measures of disease progression in the trial suggest that on acoramidis, 45% of subjects experienced an improvement from baseline in N-terminal prohormone of brain natriuretic peptide (NT-proBNP) versus 9% on placebo, and 40% of subjects experienced an improvement from baseline on 6-minute walk distance (6MWD) versus 24% on placebo; to the company’s knowledge, the proportions of treated patients improving on these measures over 30 months are higher than have been observed in prior controlled studies in ATTR-CM

- Acoramidis achieved near-complete stabilization of transthyretin (TTR) in both wild-type and variant ATTR patients; serum TTR was promptly and consistently elevated throughout the study

- In an exploratory post-hoc analysis of the relationship between on-treatment serum TTR levels and on-treatment measures of CVH, NT-proBNP, and Kansas City Cardiomyopathy Questionnaire (KCCQ), there was an association between the mean on-treatment TTR level and each of these three variables, consistent with the premise that ever-higher degrees of stabilization lead to ever-better outcomes for patients

- As was previously reported, in a comparative exploratory post hoc analysis enabled by tafamidis drop-in, albeit at low patient numbers, acoramidis showed a 42% greater increase in serum TTR levels relative to placebo + tafamidis

- Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified

- Company intends to file a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) by end of 2023

https://www.marketscreener.com/quote/stock/BRIDGEBIO-PHARMA-INC-60342525/news/BridgeBio-presents-detailed-positive-results-from-Phase-3-ATTRibute-CM-study-of-acoramidis-for-patie-44707947/