Acoramidis may improve cardiac mass, function in transthyretin amyloid cardiomyopathy
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Key takeaways:
- Cardiac MRI data suggest acoramidis could improve outcomes for people with symptomatic transthyretin amyloid cardiomyopathy.
- Tissue imaging showed a “clinically important degree of regression.”
ATLANTA — Data from a small substudy with cardiac MRI suggest acoramidis may improve both cardiac mass and function for adults with symptomatic transthyretin amyloid cardiomyopathy, building on previous positive data, researchers reported.
The findings were presented at the American College of Cardiology Scientific Session.
As Healio previously reported, data from the phase 3 ATTRibute-CM trial showed that acoramidis (BridgeBio), an investigational, next-generation, small molecule stabilizer of transthyretin (TTR), demonstrated consistent benefits on survival, hospitalization and other measures of illness severity. New data published in The New England Journal of Medicine in January showed that acoramidis reduced risk for all-cause mortality and CV-related hospitalizations in symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM), in addition to improving functional and quality of life measures.
“Our phase 3 trial represented a unique opportunity to, in a longitudinal way, explore the structural and functional consequences of this potentially disease-modifying therapy,” Jonathan Fox, MD, PhD, president and chief medical officer for cardiovascular and renal diseases at BridgeBio Pharma, told Healio. “When we designed the trial, we thought this was an opportunity to do something that had not been done before.”
For the cardiac magnetic resonance substudy of ATTRibute-CM, Fox and colleagues analyzed data from 31 adults enrolled from a U.K. site who underwent cardiac MRI at baseline and month 30. The median age of participants was 76 years; median left ventricular ejection fraction at baseline was 52% for participants in the acoramidis group and 50% for those in the placebo group.
At month 30, researchers found that LV mass index was reduced from baseline for participants who received acoramidis but increased for those who received placebo (mean, –2 g/m2 vs. 5.6 g/m2). Similarly, LV stroke volume index improved from baseline to 30 months for participants in the acoramidis group but remained unchanged for the placebo group (mean, 4.9 mL/m2 vs. 0 mL/m2), and LVEF improved for the acoramidis group but worsened in the placebo group (mean, 4.6% vs. – 8.2%). LV global longitudinal strain increased by 0.1% in the acoramidis group and 2.2% in the placebo group.
Additionally, researchers noted that an extracellular volume reduction of 5% or greater, a threshold that is considered to indicate clinically meaningful regression, was observed in 12.5% of participants who received acoramidis but none who received placebo.
“Despite the small sample size and exploratory nature of the study, the results were remarkable,” Fox said during an interview. “While the tissue imaging suggested a clinically important degree of regression, the reduction in cardiac mass and the improvements in cardiac hemodynamics were even more impressive. We expected to observe evidence that active treatment might slow the progression of the disease, which was the broad expectation. We were surprised and excited the data suggested clinically important directional improvement in cardiac mass, function and amyloid burden.”
Fox said the imaging data support the potential for acoramidis to become an important disease-modifying therapyoption for patients if FDA-approved.
“Patients and their physicians need choices,” Fox said. “For any disease, there is no single therapy that is right for everyone. We believe the totality of the program evidence, going back to the early clinical studies, positions acoramidis as an attractive treatment option for patients.”
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Razvi Y, et al. Abstract 1074-05. Presented at: American College of Cardiology Scientific Session; April 6-8, 2024; Atlanta.
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