Trial now testing long-term effects of approved ATTR-CM treatment
Developer aims to determine if drug can recover heart function in patients
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The first participant has been dosed in a clinical trial evaluating the long-term effects of Attruby (acoramidis), an approved treatment for transthyretin amyloid cardiomyopathy (ATTR-CM), on heart function and structure in adults with the progressive condition, which is marked by accumulating heart damage.
Developer Bridgebio, which markets the oral therapy in the U.S., is seeking to determine whether or not Attruby may be “capable of reversing progression and actively restoring heart health” in people with ATTR-CM, according to a company press release. With dosing started, the trial is now fully underway.
The post-marketing Phase 3b/4 study, called ASCEND-ATTR (NCT07695701), is recruiting an estimated 150 ATTR-CM patients, ages 18 to 80, at one site in the U.S. It’s slated to run through 2030.
The trial builds on results from a now-completed substudy of the Phase 3 ATTRibute-CM trial (NCT03860935), which showed that Attruby may improve heart structure and function in patients. The findings also suggested the drug may reduce the accumulation of toxic clumps of the transthyretin (TTR) protein that cause the condition.
This dosing milestone follows an announcement by the developer earlier this month that patient enrollment had started in ASCEND-ATTR.
Attruby approved for ATTR-CM in US and EU
“Serial [heart] imaging from the ATTRibute-CM … substudy gave us the first real signal that TTR [protein] stabilization can do more than slow disease progression, it may allow the heart to recover function and remodel favorably over time,” said Ahmad Masri, MD, a researcher at Oregon Health and Science University.
Masri added that “ASCEND-ATTR will allow us to study these structural and functional changes [over time] and in far greater depth, across a notably larger patient [group] and with two complementary imaging modalities, to better understand the extent to which favorable remodeling can be achieved with long-term [Attruby] treatment.”
In ATTR-CM, misfolded, unstable TTR protein forms toxic aggregates, known as amyloid deposits, that primarily build up in the heart, which can ultimately result in heart failure.
Attruby, sold by Bayer as Beyonttra in the European Union, is an oral medication that works by stabilizing the TTR protein, preventing its breakdown, and reducing the accumulation of amyloid deposits. It is approved to lower the risk of cardiovascular-related death and hospitalization in adults with ATTR-CM.
The treatment’s approvals were supported mainly by results from the ATTRibute-CM study, in which Attruby outperformed a placebo in improving survival and heart-related outcomes over about 2.5 years.
Continued evaluation in ATTRibute-CM’s open-label extension study (NCT04988386), where all participants are receiving Attruby, showed sustained survival and heart-related benefits for up to 4.5 years.
The treatment’s effects on heart structure and health in ATTRibute-CM’s substudy were analyzed using cardiac magnetic resonance (CMR) imaging. Those results suggested the treatment may restore heart health and reverse amyloid deposits, thereby reducing ATTR-CM progression.
In the ASCEND trial, patients will take Attruby at its approved dose for 36 months, or about three years. The trial’s main goal is to assess changes in heart function via CMR. Secondary goals are changes in other CMR measures of heart function, structure, and amyloid burden. Exploratory outcomes include echocardiographic measures and several blood biomarkers.
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