BridgeBio Reports Serum TTR Increase After Switching to Acoramidis in ATTRibute-CM Study

BridgeBio Pharma, Inc., a biopharmaceutical firm specializing in genetic diseases, unveiled new data from its Phase 3 ATTRibute-CM and open-label extension (OLE) study of acoramidis at the European Society of Cardiology (ESC) 2024 conference. This study primarily investigates the efficacy and safety of acoramidis, an advanced oral stabilizer of transthyretin (TTR) used for treating transthyretin amyloid cardiomyopathy (ATTR-CM).

Mathew Maurer, M.D. from Columbia University Irving Medical Center, presented the findings on serum TTR levels. Participants who transitioned from tafamidis and placebo to acoramidis in the OLE study showed a mean serum TTR increase of 3.0 mg/dL at Month 1 and 3.4 mg/dL at Month 6. This is significant because TTR is vital for transporting thyroxine and vitamin A, and elevated TTR levels correlate with reduced heart failure and better survival outcomes.

Earlier, BridgeBio revealed results showing that acoramidis treatment led to increased serum TTR levels by Day 28, which were maintained and linked with lowered risks of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalizations (CVH) through Month 30. Specifically, a 5 mg/dL increase in serum TTR at Day 28 was associated with a 30.9% and 26.1% reduction in death risk by logistic and Cox proportional hazards models, respectively. Also, each 1 mg/dL increase in serum TTR resulted in a 5.5% reduction in cardiovascular death risk and a 4.7% decrease in first CVH risk over 30 months.

Dr. Maurer emphasized that switching from tafamidis to acoramidis boosted serum TTR levels, enhancing patient outcomes in those with ATTR-CM. This could serve as a basis for monitoring TTR levels and selecting specific treatments in a future landscape with multiple therapy options for ATTR-CM.

In collaboration with the Cardiovascular Data Science (CarDS) Lab, Dr. Rohan Khera from Yale School of Medicine discussed the application of artificial intelligence tools in the TRACE AI Network Study. This initiative, supported by BridgeBio, aims to provide a scalable screening toolkit for ATTR-CM across various health systems to determine the potential prevalence of undiagnosed ATTR-CM in patients undergoing routine cardiovascular evaluations, particularly among diverse socioeconomic and demographic groups.

Following the successful results from ATTRibute-CM, BridgeBio submitted a New Drug Application to the U.S. Food and Drug Administration (FDA), which has been accepted with a decision expected on November 29, 2024. Additionally, a Marketing Authorization Application was submitted to the European Medicines Agency, with a decision anticipated in 2025. BridgeBio has granted Bayer exclusive rights to commercialize acoramidis for ATTR-CM in Europe.

BridgeBio Pharma Inc. is dedicated to discovering, developing, and delivering transformative medicines for patients suffering from genetic diseases. The company’s pipeline includes a range of development programs from early science to advanced clinical trials. Founded in 2015, BridgeBio's team is committed to applying advances in genetic medicine to benefit patients as swiftly as possible.

In March 2024, BridgeBio partnered with Bayer, granting them exclusive rights to commercialize acoramidis in Europe for treating ATTR-CM. This collaboration combines Bayer’s extensive expertise in cardiovascular diseases with BridgeBio’s leadership in the emerging field of ATTR-CM, leveraging Bayer’s established cardiovascular infrastructure in Europe.

BridgeBio continues to be a significant player in the genetic medicine landscape, focusing on delivering innovative treatments and improving the quality of life for patients with genetic diseases.