BridgeBio Pharma Inc. (BBIO), a biopharmaceutical company focused on genetic diseases, will be presenting additional data on clinical outcomes from ATTRibute-CM at the European Society of Cardiology (ESC) Congress tomorrow.
ATTRibute-CM is a phase III study of the company's investigational drug Acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Acoramidis demonstrated a significant impact on mortality, hospitalizations, and quality of life in in ATTR-CM participants in the trial.
Transthyretin amyloid cardiomyopathy is a rare but severe cause of restrictive cardiomyopathy caused by the accumulation of transthyretin fibrils in the myocardium. It can present with new or worsening heart failure or new conduction system disease. (Source: NIH).
The New Drug Application for Acoramidis was submitted to the FDA in February of this year based on positive results from the ATTRibute-CM trial. The U.S. regulatory agency's decision on Acoramidis is due on November 29, 2024.
The other core investigational drugs in the pipeline are:
-- Encaleret for autosomal dominant hypocalcemia type 1 (ADH1), which is under a phase III trial, dubbed CALIBRATE. Topline data from this study is anticipated next year.
-- BBP-418 for limb-girdle muscular dystrophy type 2I/R9, which is under a phase III trial, dubbed FORTIFY, with top-line results from the interim analysis expected in 2025.
-- Low-dose Infigratinib for achondroplasia, under a phase III trial, dubbed PROPEL 3, with enrollment expected to be completed this year. The same compound is also being explored in the indication of hypochondroplasia and is under an observational run-in for ACCEL 2, phase II study.
Cash Position
The company ended June 30, 2024, with $587 million in cash, cash equivalents, marketable securities and short-term restricted cash.
BBIO has traded in a range of $21.62 to $44.32 in the last 1 year. The stock closed yesterday's trading at $24.90, down 0.32%.
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December 19, 2025 15:10 ET U.S. inflation data and interest rate decisions by major central banks were the highlights of this busy week for economics news flow. Employment data and survey results on the housing markets also gained attention in the U.S. In Europe, the European Central Bank and Bank of England announced their policy decisions and macroeconomic projections.