On Monday, BridgeBio Pharma, Inc. (NASDAQ:BBIO) presented initial outcomes from the ATTRibute-CM open-label extension (OLE) study of acoramidis in ATTR-CM at the American Heart Association Scientific Sessions.
ATTRibute-CM was designed to evaluate the efficacy and safety of acoramidis, an investigational small-molecule stabilizer of transthyretin (TTR) gene.
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The OLE study involves 330 participants who completed the 30-month ATTRibute-CM Phase 3 study.
Key initial results from the OLE study show that continuous treatment with acoramidis led to:
- A confirmed sustained improvement relative to placebo in time to the first event ( cardiovascular-related hospitalizations (CVH) or All-Cause Mortality (ACM)) starting at Month 3 in ATTRibute-CM
- A statistically significant reduction in ACM alone of 36% by Month 36 (p=0.009) and 34% by Month 42 (p=0.006).
- A significant reduction of composite ACM and CVH by 46% at Month 36 and 48% at Month 42, building upon the previously presented 42% reduction at Month 30 in ATTRibute-CM.
- Acoramidis continues to be well tolerated, with no new clinically significant safety signals identified in this long-term evaluation.
The OLE data build on previously reported results from ATTRibute-CM in which acoramidis demonstrated clinically important treatment effects on mortality, CVH, and quality of life, further supporting that greater transthyretin (TTR) stabilization can improve clinical outcomes for patients.
This included a 50% reduction in the cumulative frequency of CVH relative to placebo at Month 30.
BridgeBio's marketing application is under FDA review, with a PDUFA action date of November 29, 2024. It also has a marketing application to the European Medicines Agency, with a decision expected in 2025.
BridgeBio has granted Bayer AG (OTC:BAYRY) exclusive rights to commercialize ceramides for ATTR-CM in Europe.
William Blair writes that reducing overall mortality is crucial for competing in the ATTR-CM market against Pfizer Inc's (NYSE:PFE) tafamidis, which already includes proven mortality benefits on its label from the ATTR-ACT trial.
The analyst writes, "While Bridge Bio continues to publish analyses of acoramidis demonstrating the TTR stabilizer is safe and efficacious, we deem an on-label claim to all-cause mortality benefit as an important factor for market share in the lucrative, but competitive, ATTR-CM space."
Price Action: BBIO stock is up 0.85% at $22.54 at the last check on Tuesday.
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