BridgeBio shares dData on serum TTR increase when switching participants from placebo and tafamidis to acoramidis in ATTRibute-CM and its open-label extension.- BridgeBio Pharma.

ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of TTR.

The data on change from baseline in serum TTR levels in participants receiving acoramidis versus those receiving tafamidis in the placebo group in ATTRibute-CM at Month 30, as well as serum TTR levels in patients who transitioned from placebo and tafamidis to acoramidis in the OLE study, were presented by Mathew Maurer, M.D. of Columbia University Irving Medical Center. In participants who switched from tafamidis and placebo in the ATTRibute-CM study to acoramidis in its OLE, there was a mean of 3.0mg/dL increase in serum TTR at Month 1 of the OLE (n=21, p=0.01) and mean of 3.4mg/dL increase in serum TTR at Month 6 of the OLE (n=18, p=0.01).

TTR plays an important role in the body transporting thyroxine and vitamin A, and higher TTR levels are associated with less heart failure and better survival. BridgeBio previously shared results demonstrating that acoramidis treatment resulted in increased serum TTR levels by Day 28 that were sustained and were correlated with a reduced risk of ACM, CVM, and CVH in ATTR-CM participants through Month 30.

This includes the following results: i. For every 5mg/dL increase in serum TTR level at Day 28 after treatment initiation, the risk of death through Month 30 was reduced by 30.9% (by the logistic model) and 26.1% (by the Cox proportional hazards model), showing a statistically significant correlation between increasing serum TTR and decreasing risk of death. ii. For each 1 mg/dL, increase in serum TTR on Day 28 after treatment initiation, there was a 5.5% risk reduction in cardiovascular death observed through Month 30. iii. For each 1 mg/dL, increase in serum TTR at Day 28 after treatment initiation was associated with a 4.7% lower risk of a first cardiovascular hospitalization over 30 months.

“The data shared show that switching from tafamidis to acoramidis resulted in an increase in serum TTR, which has been associated with improved outcomes in patients with ATTR-CM,” said Dr. Maurer. “In a future arena of multiple disease modifying therapies for ATTR-CM, such data may provide a rationale for monitoring of patients with serum transthyretin levels and using these data in choosing a specific treatment.”

Additionally, as part of the ongoing partnership with the Cardiovascular Data Science (CarDS) Lab, Rohan Khera, M.D., M.S., cardiologist-data scientist at Yale School of Medicine will be presenting about the artificial intelligence tools being deployed in the TRACE AI Network Study. The BridgeBio-partnered initiative will provide a scalable screening toolkit for ATTR-CM across large, diverse health systems to quantify the potential prevalence of undiagnosed ATTR-CM among all patients undergoing routine cardiovascular evaluation, specifically among key socioeconomic and demographic subpopulations.

Based on the positive results from ATTRibute-CM, BridgeBio submitted a New Drug Application to the FDA which has been accepted with a PDUFA action date of November 29, 2024, and a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025. BridgeBio has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe.