BridgeBio Reports Encouraging Results from ATTRibute-CM Study
A recent study has shown that participants who transitioned from tafamidis and placebo to acoramidis in the ATTRibute-CM open-label extension experienced significant improvements in serum transthyretin (TTR) levels. Within the first month of treatment, there was a notable average increase of 3.0 mg/dL in serum TTR (n=21), followed by a further increase of 3.4 mg/dL by Month 6 (n=18).
Link Between Serum TTR and Patient Outcomes
A key finding from the study indicated that elevated serum TTR levels at Day 28 were associated with a reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalization (CVH) in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). This connection highlights the importance of stabilizing serum TTR as an indicator of clinical improvement.
Effects of Acoramidis on Clinical Outcomes
The study revealed that acoramidis, a potent TTR stabilizer, significantly improves clinical outcomes for patients. Some specific benefits noted in the study included:
- An early and sustained improvement in the time to the first event (either CVH or ACM), beginning as early as Month 3.
- A substantial 42% reduction in the composite events of CVH and ACM at Month 30 compared to those on placebo.
- A striking 50% decrease in the cumulative frequency of CVH events by Month 30.
Scientific Findings from the ATTRibute-CM Study
During a recent presentation, Mathew Maurer, M.D., from Columbia University Irving Medical Center, discussed the analysis comparing participants receiving acoramidis with those in the tafamidis and placebo groups. The initial findings, previously shared, indicated that by Day 28, higher TTR levels not only correlated with improved patient survival but also had a significant positive impact on participants' quality of life.
Ongoing Developments and Future Prospects
BridgeBio's collaboration with the Cardiovascular Data Science (CarDS) Lab is focused on leveraging advanced artificial intelligence tools in the TRACE AI Network Study. This initiative aims to implement comprehensive screening strategies across various health systems to identify undiagnosed cases of ATTR-CM, particularly within specific socioeconomic and demographic populations.
Given the encouraging results from the ATTRibute-CM study, BridgeBio has proactively submitted a New Drug Application to the U.S. Food and Drug Administration (FDA), which is currently under review. This submission is in line with a Marketing Authorization Application to the European Medicines Agency, with decisions expected over the coming year.
About BridgeBio Pharma, Inc.
BridgeBio Pharma, Inc. is a biopharmaceutical company focused on discovering, developing, and delivering transformative therapies for genetic diseases. Founded in 2015, BridgeBio's experienced team is dedicated to advancing genetic medicine. The company is actively involved in both early and advanced clinical development programs.
Frequently Asked Questions
What is the ATTRibute-CM study?
The ATTRibute-CM study is a Phase 3 clinical trial assessing the efficacy and safety of acoramidis for patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
What were the main findings regarding serum TTR levels?
The study revealed that patients transitioning to acoramidis showed increases in serum TTR levels, which correlated with improved survival rates and lower hospitalization risks.
How does acoramidis compare to tafamidis?
Acoramidis has shown superior stabilization of TTR levels compared to tafamidis, leading to significantly better clinical outcomes for patients with ATTR-CM.
What does the future hold for the treatment of ATTR-CM?
BridgeBio is actively seeking regulatory approvals for acoramidis, suggesting a promising future for more effective treatments for patients with ATTR-CM.
Who can be contacted for more information about BridgeBio?
For further inquiries, Vikram Bali at BridgeBio can be contacted via email at contact@bridgebio.com or by phone at (650)-789-8220.
