BridgeBio Pharma Stock Down, Month 12 Topline Results from Phase 3 ATTRibute-CM Study Released
Dec 27, 2021 By MarketDepth
BridgeBio Pharma, Inc. (NASDAQ: BBIO) stock crushed about 70% after the biotech company announced results from Month 12 (Part A) of ATTRibute-CM, an ongoing global Phase 3 study investigating acoramidis for the treatment of symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). The mean observed decline in 6MWD at Month 12 in participants receiving acoramidis or placebo with baseline eGFR ≥ 30 mL/min/1.73m2 were 9 meters and 7 meters, respectively. Decline observed in both arms of ATTRibute-CM was similar to expected functional decline in healthy elderly adults.1 The declines were also substantially less than the >40 meter annual declines observed in previous untreated arms reviewed by the company.2 The decline in the ATTRibute-CM placebo group was more than 70% lower than the decline observed in the ATTR-ACT treatment group.2
“This result is disappointing and baffling. I am, along with many others, searching for answers regarding the 6MWD. The results do not appear to be due to a baseline imbalance. The hypotheses we are currently evaluating include context bias, training bias, and an evolution in diagnosis and standard of care. The drug does appear to be pharmacologically active and well-tolerated, and we observed improvement on quality of life with promising trends on adverse events leading to death. The drug seems to be doing what we are asking of it. If we observe enough clinical outcome events at Month 30, I am still hopeful that we will demonstrate the benefit of acoramidis treatment.”Neil Kumar, Ph.D., founder and CEO of BridgeBio
The ATTRibute-CM independent data monitoring committee recommends continuing the study based on unblinded data reviews. Despite the unexpected performance of the six-minute walk test, the trial’s steering committee co-chairs and the Company agree that there is potential for acoramidis to demonstrate benefit on the Month 30 endpoint which includes all-cause mortality and cardiovascular hospitalizations.
“Although these results were not what we hoped, the most important moment in this trial will be the Part B readout at 30 months, where we will see the effects of acoramidis on all-cause mortality and cardiovascular hospitalizations. From what I’ve seen so far, I remain enthusiastic about getting to that endpoint.”Daniel Judge, M.D., professor in the Division of Cardiology at the Medical University of South Carolina, and co-chair of the ATTRibute-CM Steering Committee
1 Enright, P.L. et al. Chest 2003.
2 Maurer, M.S. et al. NEJM 2018.; Lane, T. et al., Circulation 2019.