HQ Team
July 17, 2023: BridgeBio Pharma Inc., a Palo Alto, California-based biopharmaceutical company, reported its late-stage trial oral drug to treat a rare heart disease condition has yielded positive results.
The experimental study of acoramidis in transthyretin amyloid cardiomyopathy, or ATTR-CM, “bring much hope to amyloidosis patients,” said Muriel Finkel, President of Amyloidosis Support Groups, a non-profit organization dedicated to the support of amyloidosis patients and caregivers.
BridgeBio’s drug was 1.8 times better than placebo when measured for preventing death, hospitalization, and improvement in six minutes of walking distance as part of the main goal, according to a statement from BridgeBio.
Acoramidis also helped improve survival by 81% in patients on the treatment compared to 74% on the placebo and also reduced the frequency of heart disease-related hospitalization by 50%.
‘No safety concerns’
“No safety signals of potential clinical concern” were identified. The company intends to file a New Drug Application with the US Food and Drug Administration by the end of 2023.
Transthyretin amyloid cardiomyopathy is an underdiagnosed and potentially fatal disease of the heart muscle.
A late-breaker presentation, or, an informative, fast-paced 5-minute presentation, of new information or important findings not fully available before the general abstract deadline, has been accepted for the annual meeting of the European Society of Cardiology.
The phase III trial was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, small-molecule stabilizer of transthyretin (TTR).
“The çompany consistently observed a statistically significant treatment effect at 30 months across additional measured markers of morbidity, quality of life, and function,” according to the statement.
Maximize stabilization
A key objective in the rational drug design of acoramidis was to maximize TTR stabilization at clinically achieved blood concentrations. Acoramidis showed a 42% greater increase in serum TTR levels versus tafamidis, the other marketed stabilizer.
“No comparison can be made as to the potential effectiveness or safety of acoramidis and tafamidis, given that these are exploratory analyses, and the study was not prospectively designed for a head-to-head comparison.”
The company is “extremely encouraged by the robustly positive and consistent findings of the ATTRibute-CM study, which confirm our position that highly potent TTR stabilization has the potential to profoundly impact patients’ lives,” said Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal.
In ATTR-CM, a protein called transthyretin, which normally circulates in the bloodstream, builds up in the heart, nerves, and other organs.
When these amyloid deposits build up in the heart, the walls can become stiff, making the left ventricle unable to properly relax and fill with blood – called cardiomyopathy.
Hereditary ATTR-CM
As the condition progresses, the heart can become unable to adequately squeeze to pump blood out of the heart, ultimately leading to heart failure.
There are two types of ATTR-CM. In hereditary ATTR-CM (hATTR-CM), which can run in families, and there’s a variant in the transthyretin gene, which results in amyloid deposits in the heart, nerves, and sometimes the kidneys and other organs.
Symptoms may start as early as age 20 and as late as 80.
Hereditary ATTR-CM is more common in localized parts of Portugal, Sweden, and Japan.
There are a number of variants in different parts of the world. Some variants are more common in people of Irish ancestry while others are common among people of African descent.
Different variants may progress in different ways and involve different organs, according to the American Heart Association.
The most common variant in the US occurs in one in 25 of all African Americans and in older patients who may be misdiagnosed with high blood pressure-related heart disease.
Genetic testing may provide important information to roll out a treatment plan.
