BridgeBio Pharma announced in a recent press release that the first ACT-EARLY clinical trial participant has now received a dose of the study drug, acoramidis. ACT-EARLY is the first study designed to test whether early treatment with acoramidis can prevent the development of transthyretin amyloidosis (ATTR) in people who carry a high-risk genetic variant.
The drug is already approved in the U.S., Europe, Japan and the U.K. to treat adults who have been diagnosed with ATTR cardiomyopathy (ATTR-CM). This approval was based on earlier research which showed that acoramidis helped reduce the chances of death and heart-related hospital stays in patients with ATTR-CM.
“I have met many families of those diagnosed with hereditary ATTR and one question often asked is what can be done for asymptomatic carriers of the genetic variant causing ATTR,” said Muriel Finkel, president of Amyloidosis Support Groups, a non-profit organization dedicated to the support of amyloidosis patients and caregivers. “Since there is currently no approved therapy to delay or prevent disease onset, this underserved, at-risk population must wait for the development of symptoms before therapy can be prescribed.”
ACT-EARLY aims to find out whether starting treatment earlier — before symptoms begin — might lead to better outcomes.
Read more about treatment and care for ATTR-CM
The researchers plan to enroll around 600 participants aged between ages 18 and 75 who have genetic changes known to cause ATTR but haven’t developed any symptoms yet. Participants will be randomly assigned to receive either acoramidis or a placebo (a look-alike pill with no active drug). Acoramidis works by stabilizing the transthyretin (TTR) protein to help prevent it from forming harmful clumps that can build up in the body and cause disease.
The goal of the study is to see if acoramidis can prevent or delay the accumulation of TTR in the heart or nerves in people with the genetic risk. Researchers will track how long it takes for signs of heart or nerve damage to appear, as well as how participants respond to the medication over time. They will also monitor changes in heart scans, nerve tests and TTR protein levels in the blood.
The study is currently enrolling participants in the U.S., Bulgaria, Italy, the Netherlands and Spain. More information is available at clinicaltrials.gov.
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