New BridgeBio Data Back Acoramidis Benefit in ATTR-CM

BridgeBio Pharma recently announced promising results from the Phase III ATTRibute-CM study, revealing that acoramidis significantly increased serum transthyretin (TTR) levels, which is linked to better cardiovascular outcomes in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). These findings were shared at the International Symposium of Amyloidosis (ISA).

Acoramidis is a small-molecule, orally-administered TTR stabilizer currently under review by the U.S. Food and Drug Administration (FDA), with an anticipated decision deadline of November 29. Earlier in the year, Bayer secured exclusive rights to market acoramidis in Europe, contingent on regional approval.

The marketing applications for acoramidis in both the U.S. and Europe are founded on the ATTRibute-CM study results, which showed an 81% absolute survival rate among patients receiving acoramidis. Additionally, these patients experienced an average annual frequency of 0.29 for cardiovascular-related hospitalizations.

New data from the study reinforced the connection between elevated serum TTR levels and improved clinical outcomes, including a lower risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalizations (CRH). Specifically, by day 28 of acoramidis treatment, increased serum TTR levels were observed, which were sustained and linked to a reduction in the risk of all-cause mortality by up to 51%, cardiovascular mortality by 5.5%, and cardiovascular-related hospitalizations by 4.7% over a 30-month period.

Furthermore, acoramidis treatment markedly improved the composite endpoint encompassing CVM and CVH, achieving a 15.2% absolute risk reduction and a 38.2% hazard reduction by the 30-month mark.

Jonathan Fox, the chief medical officer, commented on the findings: "We remain encouraged by the potential benefits of targeting near-complete TTR stabilization, the resulting increases in serum TTR, and the corresponding statistically significant benefits on clinical event outcomes. We are committed to bringing acoramidis to the ATTR-CM community as quickly as possible, working toward our November 29 PDUFA date."

These developments highlight the potential impact of acoramidis on improving the health outcomes of patients with ATTR-CM, emphasizing the drug's role in enhancing TTR stability and reducing cardiovascular-related risks.