BridgeBio Pharma (NASDQ:BBIO), a commercial-stage biopharmaceutical company developing medicines for patients suffering from genetic diseases and cancers with clear genetic drivers, recently reported updated results from its ongoing Phase 2 study for acoramidis. The study is an open-label extension study of its acoramidis molecule, in transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM).
An interim analysis of the continuing Phase 2 OLE study was completed utilizing available information up to August 31, 2021.
Ahmad Masri, M.D., MS, director of the Cardiac Amyloidosis Program at Oregon Health & Science University commented:
“Over approximately three years in this study, acoramidis continued to be well tolerated and potently stabilize TTR. In patients with advanced symptomatic disease that would be expected to decline rapidly, participants remained remarkably stable or improved with respect to key cardiac biomarkers. These results provide additional optimism for the results of the ongoing Phase 3 study of acoramidis expected next year.”
Acoramidis was well-tolerated and resulted in near-complete TTR stabilization as measured by established ex vivo tests and increased serum TTR levels, with median N-terminal Pro-brain natriuretic peptide (NT-ProBNP) remaining stable or improving throughout the OLE.
In INTR-CM patients, NT-ProBNP levels are highly predictive of mortality and typically rise with time in untreated individuals. The Phase 2 OLE trial findings suggest that acoramidis may be taken long term by ATTR-CM patients and show a reduction in disease progression among those who received it.
The Acoramidis Trials
Acoramidis (AG10) is currently investigational and is being designed to stabilize tetrameric transthyretin. This is aimed at stopping (at the beginning of the process), the series of molecular events that eventually leave to TTR amyloidosis, also known as ATTR. It is a small molecule that can be orally administered.
Currently in Phase 2 and Phase 3 studies, acoramidis aims to treat patients with ATTR and reduce symptoms. Acoramidis was designed to resemble a “rescue mutation” of the TTR gene (T119M), which is a naturally occurring variant that has been shown to prevent or reduce ATTR in people with harmful, or disease-causing, TTR gene mutations. By stabilizing their TTR, acorimidis can maintain the protective benefits of TTR and then go on to fight the root cause of the disease.
BridgeBio currently has a pipeline of over 30 development programs ranging from very early science to clinical trials like the one for acoramidis. The commercial focus for the company is now to deliver the first two of the approved therapies to market.
If acoramidis is approved, it would be the first drug on the market to target the underlying cause of ATTR, and would potentially offer patients a disease-modifying therapy that could improve or stabilize their condition.
