Additional analyses from BridgeBio Pharma’s late-stage study show the oral drug candidate improved clinical outcomes in transthyretin amyloid cardiomyopathy patients.
BridgeBio Pharma on Wednesday unveiled additional findings from the Phase III ATTRibute-CM study of acoramidis, showing that the oral drug candidate’s effects on serum transthyretin levels correlate with improved clinical outcomes in patients with transthyretin amyloid cardiomyopathy.
The results came from five new analyses of ATTRibute-CM presented at the 2024 International Symposium on Amyloidosis (ISA) held in Minnesota this week.
One of these analyses showed that the acoramidis-related increase in transthyretin (TTR) was significantly associated with higher survival. For every 5-mg/dL hike in the biomarker at 28 days after treatment initiation, patients saw a 26% to 30% drop in the risk of death through 30 months of follow-up. Additional statistical modelling confirmed that the acoramidis-mediated jump in serum TTR levels is an independent predictor of survival.
TTR concentration was also associated with cardiovascular mortality and cardiovascular-related hospitalization, with every 1-mg/dL increase at 28 days correlating with a 5.5% and 4.7% drop in the likelihood of the respective outcomes.
Treatment with acoramidis also elicited an “early and profound” drop in the composite endpoint of cardiovascular mortality and cardiovascular hospitalization, according to BridgeBio. The drug candidate cut absolute risk of the composite outcome by 15.2% by month 30.
Acoramidis is an oral drug candidate that mimics a naturally occurring type of the TTR protein. In transthyretin amyloid cardiomyopathy (ATTR-CM), this protein is pathologically mutated causing it to form amyloid plaques that damage organs, including the heart. Acoramidis helps minimize this accumulation.
BridgeBio Cardiorenal President and CMO Jonathan Fox in a statement said the company is “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.”
The latest analyses presented at ISA come after BridgeBio announced in August 2023 that ATTRibute-CM had met its primary endpoint. Patients that were treated with acoramidis saw a “highly statistically significant” improvement in the composite outcome of all-cause mortality, cardiovascular-related hospitalizations and improvement from baseline in NT-proBNP levels and six-minute walk distance.
In July 2023, the biotech also posted topline data from the late-stage study, showing that 81% of acoramidis-treated patients were still alive at the 30-month follow-up, compared to only 74% in the placebo group.
BridgeBio submitted its New Drug Application for acoramidis in ATTR-CM in December 2023 and is expecting a decision by November 29, 2024. In preparation for the potential product launch, the biotech in January 2024 secured $1.25 billion in financing from Blue Owl Capital and the Canada Pension Plan Investment Board.
Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.
