Innovative oral drug Phase III study failed BridgeBio's share price fell 72%

Article source: Medical Rubik's Cube Info

Author: Shi Bei

On December 27, BridgeBio announced that the ongoing Phase III ATTribute-CM study of acoramidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM) failed to meet the primary endpoint at 12 months

Acoramidis (AG10) is an oral small molecule under investigation, designed to effectively stabilize the tetramer transthyretin (TTR), thereby preventing a series of molecular events that cause TTR amyloidosis (ATTR) from the beginning

In the ATTribute-CM study, key clinical data for 632 patients showed that the average decrease in 6-minute walk distance (6MWD) observed in the acoramidis group and placebo group at the 12th month was 9 meters and 7 meters, respectively

The company stated that the decline observed in the ATTribute-CM study is similar to the expected decline in function of healthy elderly people, and is significantly lower than the previously untreated annual decline of >40 meters

In terms of secondary endpoints, compared with placebo, acoramidis significantly improved the KCCQ-OS (Kansas City Cardiomyopathy Questionnaire) score (p<0.

In terms of safety, about 85% of patients in the placebo group and 92% of patients in the acoramide group had adverse events

The independent data monitoring committee of the ATTRibute-CM study recommended that the study be continued on the basis of unblinded data review