BridgeBio Pharma presented positive initial outcomes from the ATTRibute-CM open-label extension (OLE) study of acoramidis in ATTR-CM at the American Heart Association (AHA) Scientific Sessions. The OLE study, involving 389 participants who completed the 30-month ATTRibute-CM Phase 3 study, showed sustained benefits of acoramidis treatment. The preliminary results from this ongoing OLE study were also simultaneously published in Circulation.
Sustained Improvement in Cardiovascular Outcomes
Data from the ATTRibute-CM OLE study indicate that continuous treatment with acoramidis leads to a sustained improvement in cardiovascular outcomes. Specifically, the study demonstrated a statistically significant reduction in all-cause mortality (ACM) of 36% by Month 36 (p=0.009) and 34% by Month 42 (p=0.006), as assessed by the Stratified Cox proportional hazards model. Additionally, a significant reduction of composite ACM and cardiovascular hospitalization (CVH) by 46% at Month 36 (p<0.0001) and 48% at Month 42 (p<0.0001) was observed, building upon the previously presented 42% reduction at Month 30 in ATTRibute-CM.
Early and Continued Benefit
The OLE data also provided evidence of early benefit in patients who crossed over from placebo to acoramidis after Month 30, reinforcing the early separation seen previously in ATTRibute-CM. This suggests that earlier intervention with acoramidis can lead to better outcomes for patients with ATTR-CM.
Safety and Tolerability
Acoramidis continues to be well-tolerated, with no new clinically significant safety signals identified in this long-term evaluation. This is an important factor for a chronic condition like ATTR-CM, where long-term treatment is often necessary.
Clinical Implications
"Results from the ATTRibute-CM OLE continue to showcase the potential of acoramidis, with ongoing data across the study suggesting that early intervention with this stabilizer leads to early separation from placebo, with sustained benefit for patients with ATTR-CM," said Daniel Judge, M.D., professor of medicine and cardiology at the Medical University of South Carolina. "The prescribing community is eager to have another important treatment option given the remaining high unmet need for ATTR-CM patients."
Regulatory Status
Based on the positive results from ATTRibute-CM, BridgeBio submitted a New Drug Application to the U.S. Food and Drug Administration, which has been accepted with a PDUFA action date of November 29, 2024. A Marketing Authorization Application has also been submitted to the European Medicines Agency, with a decision expected in 2025. BridgeBio has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe.
