BridgeBio doses first patient in 150-participant acoramidis ATTR-CM study

BridgeBio Pharma dosed the first participant in ASCEND-ATTR, a Phase 3b/4 study of acoramidis in roughly 150 adults with transthyretin amyloid cardiomyopathy.

"Serial cardiac imaging from the ATTRibute-CM CMR substudy gave us the first real signal that TTR stabilization can do more than slow disease progression, it may allow the heart to recover function and remodel favorably over time," Ahmad Masri, M.D., M.S. at Oregon Health and Science University, said. "ASCEND-ATTR will allow us to study these structural and functional changes prospectively and in far greater depth."

The single-arm, open-label study will perform cardiovascular magnetic resonance and echocardiography annually over 36 months. The primary endpoint is Month 36 CMR responder status based on improvement from baseline in left ventricular systolic function. Secondary endpoints include CMR measures of cardiac function, structure, and amyloid burden at Month 36, plus echocardiographic measures, circulating biomarkers, and imaging assessments at Months 12 and 24.

The trial builds on the Phase 3 ATTRibute-CM CMR substudy, which showed mean improvements from baseline in Left Ventricular Mass Index, Left Ventricular Stroke Volume Index, and Left Ventricular Ejection Fraction through month 30, with evidence of amyloid regression in a subset of patients. Acoramidis is the only selective small molecule, orally administered, near-complete (≥90%) transthyretin stabilizer, according to BridgeBio.

Attruby (acoramidis) is already approved to treat ATTR-CM in adults to reduce cardiovascular death and cardiovascular-related hospitalization. Reported adverse reactions include diarrhea (11.6 percent vs 7.6 percent for placebo) and upper abdominal pain (5.5 percent vs 1.4 percent). BridgeBio reported second-quarter revenue of $243.7 million, including $222.4 million from Attruby. The company's market capitalization stands at $15.72 billion.

The study's potential to demonstrate disease regression could strengthen acoramidis' competitive position against Pfizer's tafamidis (Vyndaqel) in the ATTR-CM market. Additional data from the CMR substudy of ATTRibute-CM and its open-label extension compared to a natural history cohort will be presented at the European Society of Cardiology Congress 2026.

BridgeBio shares traded at $80.40 as of Aug. 26, with a price-to-sales ratio of 21.91, below its historical median of about 39.6. GuruFocus's GF Value metric estimates intrinsic value at $148.10 per share, roughly 46 percent above the current price. Insiders sold $130.2 million worth of shares over the past 12 months with no insider purchases reported, while 10 premium gurus hold BBIO positions, with seven increasing and three reducing.

The first-participant dosing marks a clinical milestone for acoramidis, the company's key revenue driver. Investors will watch the ESC Congress 2026 data presentation and subsequent enrollment updates from ASCEND-ATTR as the next key events.

This article is for informational purposes only and does not constitute investment advice.