Clinical BridgeBio's acoramidis shows positive results in phase 3 trial...

BridgeBio’s acoramidis shows positive results in phase 3 trial for heart disease

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Late-stage study results from BridgeBio’s phase 3 trial of its heart disease drug, acoramidis, reveal that the drug successfully met study goals and primary endpoints in the trial with patients suffering from transthyretin amyloid cardiomyopathy.

The trial proved that the experimental drug was approximately 1.8 times better than the placebo in preventing death and showed improvement in patients when walking for a duration of 6 minutes compared to the placebo.

In comparison to the placebo, the drug not only reduced the frequency of heart disease-related hospitalizations but also improved survival. The drug was able to cut heart disease-related hospitalizations by half and improve survival in patients by over 80%. In the case of the placebo, the percentage went down by 7% to 74% when it came to survival chances.

Endpoints in the study were measured in groups, and when it came to morbidity and mortality, a 1.7 figure win ratio denoted improvement in mortality and morbidity, which was less than the overall win ratio of 1.8. The change in mortality when broken down by individual endpoint was not statistically significant and had a p-value of 0.057.

Side effects for both acoramidis and the placebo were recorded as high. While over 37% of the treated patients demonstrated a severe treatment-emergent side effect, for the placebo, the number went as high as over 45%.

Over 60% of patients that were given the placebo ended up needing hospitalization due to side effects, whereas for patients who were given acoramidis, the number fell to approximately 50%. Across the board, there were more side effects recorded for the placebo.

Despite this, share prices for the company soared. Last Friday, the closing price for the shares was at $18.22, and since the study results were made public, prices rose by 60% up to $29.17.

The California-based biotech has announced that by the end of this year, it will be sharing all the data with the Food and Drug Administration as part of its marketing application, and by next year, it will start sending out applications in other markets like that in Europe. When it comes to commercially launching the drug, the biotech will be seeking the help of a partner, shareholders explained.

In order to test the drug as a primary prevention therapy, BridgeBio will be proceeding with phase 4 of the trial, and CEO Neil Kumar has further hinted at the possibility of a double-blind, head-to-head study against competitor drug Vyndaqel. Pfizer’s Vyndaqel is an already approved drug for cardiomyopathy in the U.S.

BridgeBio’s new drug will also give competition to Alnylam Pharmaceuticals’ patisiran, which is currently under regulatory review. According to RBC Capital Markets analyst Luca Issi, the positive phase 3 results also benefit other organizations developing similar drugs such as Alnylam and Ionis Pharmaceutical.

Transthyretin amyloid cardiomyopathy is a rare heart disease that can result in heart failure due to clumps of transthyretin protein buildup in the heart. The disease commonly manifests in abnormal patients when abnormal deposits of a protein called amyloid clog the areas of the heart muscle.

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