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Life-changing heart disease drug proven safe and effective in phase 3 trial

The heart, a tireless worker, pumps over 100,000 times a day, tirelessly delivering oxygen and vital nutrients to our organs. (CREDIT: Creative Commons)

The heart, a tireless worker, pumps over 100,000 times a day, tirelessly delivering oxygen and vital nutrients to our organs, sustaining our bodily functions. Yet, for millions of people worldwide, this essential organ becomes a battleground as heart failure takes hold.

Heart failure affects an estimated 6.2 million individuals in the United States alone, with a staggering 64 million affected globally.


Among those grappling with heart failure, a hidden menace lurks – transthyretin amyloid cardiomyopathy, or ATTR-CM. This underdiagnosed, highly progressive, and often fatal condition affects up to one in five older patients with abnormally thickened hearts.

ATTR-CM, which can be hereditary or develop spontaneously, is characterized by the buildup of misfolded transthyretin (TTR) proteins in the heart. These rogue proteins weaken the heart muscle and, over time, lead to severe heart dysfunction, ultimately culminating in heart failure.


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One of the greatest challenges in the battle against ATTR-CM is the frequent misdiagnosis of patients, owing to limited awareness and symptoms that mimic other, more common heart-related conditions.

Moreover, the scarcity of approved treatments exacerbates the problem, with the sole available therapy for the most prevalent form of ATTR-CM carrying a staggering annual price tag of $225,000 and limited accessibility.


In a glimmer of hope, the ATTRibute-CM phase 3 trial has yielded promising results with a novel drug called acoramidis. Researchers report that acoramidis demonstrates remarkable efficacy and safety in treating ATTR-CM. It achieves near-complete stabilization of TTR proteins, potentially slowing or halting the progression of the disease. Building on the success of this 30-month randomized, multicenter, controlled study, BridgeBio Pharma, based in Palo Alto, California, is now seeking approval from the Food and Drug Administration (FDA) for its use in ATTR-CM patients.

Dr. Daniel Judge speaking with a colleague. (CREDIT: Sarah Pack)

Dr. Daniel Judge, a cardiologist at the Medical University of South Carolina (MUSC) and a co-leader of the trial's steering committee, expressed optimism about the potential impact of acoramidis. He said, “We hope that, when this drug receives approval from the FDA, it will create competition in the market and drive down the cost of these incredibly expensive medications.”


Dr. Judge, along with his colleague Dr. Isabella Graef of Stanford University, has dedicated years to researching and developing this groundbreaking treatment. Dr. Graef played a pivotal role in creating the new drug that specifically targets the misfolded proteins central to the disease. Dr. Judge, on the other hand, has been deeply involved in phase 2 and 3 clinical trials, conducting tests on the new drug at MUSC. Notably, MUSC was one of the first sites globally to enroll patients in the ATTRibute-CM phase 3 trial.

In the pictures above, the heart on the right has TTR amyloid. This makes the heart brighter and thicker. With amyloid in the heart, squeezing and relaxing is not as good as for a normal heart. (CREDIT: Dr. Daniel Judge)

The findings of the ATTRibute-CM investigators are remarkable – acoramidis effectively binds to circulating TTR proteins, preventing their accumulation as amyloid in the heart. Compared to a placebo, acoramidis reduced hospitalizations related to heart events, decreased cardiac congestion (as determined by blood tests), and improved patients' ability to walk longer distances in just six minutes.


In a presentation at the American Heart Association, Dr. Judge revealed that the number of patients needed to be treated with acoramidis to prevent one cardiovascular hospitalization in a year during the study was just five. Moreover, the incidence of adverse events in both the acoramidis and placebo groups was low and comparable, reaffirming the drug's safety. These findings hold immense promise for ATTR-CM patients, suggesting that acoramidis may not only halt disease progression but also increase survival rates.

Improvements in Efficacy Measures Observed With Tafamidis in ATTR-ACT. ATTR-ACT = Tafamidis in Transthyretin Cardiomyopathy Clinical Trial; ATTR-CM = transthyretin amyloid cardiomyopathy; KCCQ-OS = Kansas City Cardiomyopathy Questionnaire Overall Summary; NT-proBNP = N-terminal pro-B-type natriuretic peptide. (CREDIT: JACC Journals)

Dr. Judge, with over two decades of experience in cardiology and a focus on translational research targeting heart disease, has recruited and followed several patients participating in acoramidis clinical trials. He and his fellow investigators have witnessed the journey of this novel drug from translational testing to direct benefits for real patients grappling with ATTR-CM.


Reflecting on his journey, Dr. Judge remarked, “Early in my career, I was disappointed by the lack of any available treatments. I'm glad to see another successful trial for ATTR-CM. A patient whom I first met in 2017 had a prognosis of three years. Fast forward to 6.5 years later, and this same patient, who was one of the first participants enrolled at MUSC in the phase 2 trial, seems to be getting better due to acoramidis.”

The dream is that, if the disease can be stabilized, the heart can gradually improve, offering a brighter future for those battling ATTR-CM. Safely and effectively halting disease progression with acoramidis could enhance both the quantity and quality of life for ATTR-CM patients worldwide.

For more science and technology stories check out our New Discoveries section at The Brighter Side of News.


Note: Materials provided above by The Brighter Side of News. Content may be edited for style and length.


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