For HCPs

About Attruby

Clinical Results

Attruby (acoramidis) made a proven impact on the combination of survival and hospitalizations at Month 30, and has data through 54 months across wild-type and variant patients.1

ATTRibute-CM Study Design

The ATTRibute-CM study evaluated the efficacy and safety of Attruby (acoramidis) in adults with hereditary or wild-type transthyretin-mediated cardiac amyloidosis for efficacy (N=611) and safety (N=632) over 30 months in a phase 3, randomized (2:1), double-blind, placebo-controlled study.* Concomitant use of tafamidis was allowed after 12 months. The primary endpoint was a 4-part hierarchical composite (ACM, CVH, CFB in NT-proBNP, CFB in 6MWD). Secondary endpoints included CFB in 6MWD, CFB in KCCQ-OS, CFB in serum TTR, ACM, CVM,† cumulative frequency of CVH,† CFB in NT-proBNP,† and safety assessments‡.2,3,5

See full Study Design

*Efficacy assessment included 611 participants in the prespecified mITT population (eGFR .30 mL/min/1.73 m2). A total of 632 patients underwent randomization. Of these patients, 21 who had stage 4 CKD were excluded from the primary analysis in the mITT population.3
†These secondary endpoints are prespecified and non-alpha protected.5,13
‡Based on ITT population of 632 patients, including 21 who had stage 4 CKD. The ITT population was defined as all randomized participants who received at least 1 dose of study drug and had at least 1 postbaseline efficacy evaluation and included participants who had a baseline eGFR <30 mL/min/1.73 m2.3

Primary Endpoint

Strength

Attruby significantly reduced the combination of ACM and CVH2,3

The primary composite endpoint at Month 30 included:

  • All-cause mortality (ACM)
  • Cumulative frequency of CV-related hospitalizations (CVH)

(P=0.018, assessed using the stratified Finkelstein-Schoenfeld test)

Speed

Attruby showed a rapid observable difference at 3 months

Time to First Event (ACM or CVH)2

Kaplan-Meier plot of time to first event of ACM or CVH with Attruby versus placebo

1-month post hoc exploratory analysis

Explore post hoc exploratory analysis at 1 month of mean cumulative CV outcomes with Attruby vs placebo.

Learn More

Additional Data

Strength

Post Hoc Analysis

Forty-two percent relative risk reduction

In the composite of ACM and recurrent CVH through 30 months4*

Secondary Endpoints

Strength

Hospitalizations

Fifty percent relative risk reduction

In CVH frequency at Month 30

Mean number of CVH events per year was 0.3 for Attruby (acoramidis) vs 0.6 for placebo2,3

Prespecified, non-alpha controlled

Strength

Survival

Eighty-one percent

of patients on Attruby (acoramidis) remained alive at 30 months vs 74% with placebo2†‡

See outcomes in ATTRv-CM patients
  • *RRR was calculated using the negative binomial regression model. The total number of events for Attruby (acoramidis) compared with placebo (2:1 randomized) was 79 vs 52 for ACM, respectively, and 182 vs 170 for CVH, respectively.4
  • †Cox HR=0.77 (95% CI: 0.54-1.10); CMH P=0.057.5
  • ‡Based on ITT population of 632 patients, including 21 who had stage 4 CKD. The ITT population was defined as all randomized participants who received at least 1 dose of study drug and had at least 1 postbaseline efficacy evaluation and included participants who had a baseline eGFR <30 mL/min/1.73 m2.3

Open-Label Extension Trial

Open-Label Extension (OLE)
Study Design7

No new safety concerns were observed in the OLE trial at Month 54

  • Participants who completed treatment at Month 30 were invited to enroll in the OLE
  • Participants who previously received placebo were switched to Attruby (acoramidis), while treated patients continued on Attruby (acoramidis)
  • Primary objective was to collect long-term safety data
  • Secondary objectives include continued monitoring of clinical outcomes (ACM, CVH, 6MWD, and KCCQ-OS)

The OLE study is unblinded, non-controlled, and included a self-selected population, which may have led to baseline imbalance and survivor bias. This non-prespecified, interim analysis was not powered to make definitive conclusions.

See full Study Design

Strength

Long-term data through 54 months

No new safety concerns were observed in the OLE trial at Month 54.1

At Month 54, for patients who started treatment on Attruby (acoramidis), a 45% RRR in ACM was observed1*

Time to ACM From Baseline Through Month 541,2,6,7†

Graph of survival probability with Attruby® compared with placebo through Month fifty-four

The OLE study is unblinded, non-controlled, and included a self-selected population, which may have led to baseline imbalance; this non-prespecified interim analysis was not powered to make definitive conclusions.6

  • *Compared with those who started on placebo.1
  • †Data are for the full analysis set, which included the mITT population in ATTRibute-CM defined as all participants who were randomized to Attruby (acoramidis) or placebo, received ≥ 1 dose of Attruby (acoramidis) or placebo, had ≥1 efficacy evaluation after baseline, and had baseline eGFR of ≥30 mL/min/1.73 m2. Data were analyzed using a stratified Cox proportional hazards model.1,6
  • ‡Assessed by the Cox proportional hazards model.1,6

  • §Assessed by the Cochran-Mantel-Haenszel test.5

Serum TTR & Outcomes

In two post-hoc exploratory analyses of the ATTRibute-CM study, early increases in serum TTR were associated with improved outcomes at Month 30

5 mg/dL at Day 28 associated with 32% risk reduction in all-cause mortality and 19% risk reduction in CV hospitalizations
See early rise in serum TTR at 4 weeks

Exploratory analyses based on participants from ATTRibute-CM for whom serum TTR data were available at both baseline and Day 28. May not reflect broader patient population. Effect not generalizable to other TTR stabilizers.

  • *Independent study in 557 patients with ATTR-CM from the ATTRibute-CM study. Univariate analyses in both the overall population and the Attruby (acoramidis)-treated population; change in serum TTR remained an independent predictor of
ACM even after adjusting for baseline demographic variables, diuretic use, NYHA class, baseline serum TTR, TTR variant or wild-type status, and National Amyloidosis Centre stage.8

  • †Analysis of the mlTT population of ATTRibute-CM. Combined outcomes of serum TTR CFB to Day 28 and Month 30, and first CVH or cardiovascular mortality, excluding those events that occurred prior to Day 28. Patient stratification was based on treatment group, baseline TTR (<20 or ≥20 mg/dL), and original randomization factors of TTR genotype (wild-type vs variant), NT-proBNP (≤3000 or >3000 pg/mL), and eGFR (<45 or ≥45 ml/min/1.73 m2).9

KCCQ-OS/6MWD

KCCQ-OS

Strength

Attruby (acoramidis) made a measurable difference on health-related quality of life1

Secondary Endpoint

  • At 30 months, the Least Squares mean difference in change from baseline in KCCQ-OS was 10 points in CFB in KCCQ-OS compared with placebo (95% CI: 5.97-13.91; P<0.0001)2,3*

At Month 30:

  • -3.1 points mean CFB in KCCQ-OS in the Attruby (acoramidis) arm of ATTRibute-CM compared to -10.8 points with placebo.5

In ATTRibute-CM, at month 3, a separation in Least Squares mean change from baseline in KCCQ-OS Score was observed between Attruby (acoramidis) and placebo.

Both patients who received Attruby and patients on placebo experienced a decline in their KCCQ-OS. At Month 30, LS mean difference (95% CI) in change from baseline in KCCQ-OS was 10 points (P<0.0001).

Through Month 54 (OLE study):

  • -4.8 points mean CFB in KCCQ-OS with continuous Attruby (acoramidis)1

The OLE study is unblinded, non-controlled, and included a self-selected population, which may have led to baseline imbalance; this non-prespecified, interim analysis was not powered to make definitive conclusions.6

6MWD

Strength

Secondary Endpoint
At Month 30:

About 131 feet

Change in Least Squares mean in 6MWD (95% CI: 69-190; P<0.0001) compared with placebo2†

†Equivalent to a difference of 40 meters.5

NT-proBNP

Strength

Secondary Endpoint

Change in NT-proBNP favored Attruby (acoramidis) ↓52.9% of placebo3*

Elevated levels of NT-proBNP are an indicator of heart stress.12

  • *Prespecified, non-alpha protected secondary endpoint.5,13

ATTRv-CM Patients

Strength

Consistent treatment benefit across variant and wild-type ATTR-CM

Attruby (acoramidis) was proven to significantly reduce the combination of ACM and cumulative frequency of CVH at Month 30 of the ATTRibute-CM trial to (P=0.018), assessed using the stratified F-S test)14

Prespecified subgroup analysis in patients with ATTRv-CM,

Fifty-nine percent relative risk reduction

In composite of ACM or first CVH observed with Attruby (acoramidis)
vs placebo at Month 30.14 This analysis was descriptive and exploratory in nature. Results are limited by small sample size

PRESPECIFIED SUBGROUP ANALYSIS

Composite of ACM or First CVH14

Forest plot showing risk of ACM or first CVH with Attruby® vs placebo across ATTR-CM subgroups

Stability

Change From Baseline in Serum TTR Level Through Month 30, mITT Population15†‡

Graph showing change from baseline in serum TTR levels with Attruby® compared to placebo for ATTRv-CM patients

These analyses were descriptive and exploratory in nature. Results are limited by small sample size.

Select secondary outcomes in ATTRv-CM patients treated with Attruby (acoramidis) (n=39) vs placebo (n=20)16:

Sixty-two percent relative risk reduction

In frequency of CVH with Attruby (acoramidis) vs placebo (95% CI: 0.139-1.027)17

  • 20-Point difference in LS mean CFB in KCCQ-OS score16

These analyses were descriptive and exploratory in nature. Results are limited by small sample size.

The Importance of TTR (transthyretin)

Watch on YouTube
  • *Indicate nominal statistical significance.
  • †From mlTT population; in total, 59/611 participants were categorized as having ATTRv-CM at randomization. Subsequently, mutations were identified in the clinical database in 56/611 participants. Serum TTR concentrations were determined using a standardized clinical assay for serum prealbumin performed in a central laboratory. Data are shown for participants who had non-missing CFB values. Serum TTR levels were measured at baseline, Day 28, Month 3, and every 3 months thereafter. Observed percentage changes from baseline at various timepoints were summarized by descriptive statistics in the mlTT population.15
  • ‡Concomitant tafamidis permitted after Month 12.15

6MWD=6-minute walk distance; ACM=all-cause mortality; ATTR-CM=transthyretin amyloid cardiomyopathy; ATTRv=variant transthyretin-mediated amyloidosis; ATTRwt=wild-type transthyretin-mediated amyloidosis; CFB=change from baseline; CKD=chronic kidney disease; CMH=Cochran-Mantel-Haenszel; CV=cardiovascular; CVH=cardiovascular-related hospitalization; CVM=cardiovascular-related mortality; eGFR=estimated glomerular filtration rate; F-S test=Finkelstein-Schoenfeld test; HR=hazard ratio; ITT=intent-to-treat; KCCQ-OS=Kansas City Cardiomyopathy Questionnaire Overall Summary; LS mean=least-squares mean; mITT=modified intent-to-treat; NT-proBNP=N-terminal pro–B-type natriuretic peptide; NYHA=New York Heart Association; OLE=open-label extension; QoL=quality of life; RR=relative risk; RRR=relative risk reduction; SEM=standard error of the mean; TTR=transthyretin.

References:

  1. Soman P, Cuddy SAM, Gillmore JD, et al. Long-term durability of acoramidis efficacy in transthyretin amyloid cardiomyopathy: open-label extension of the ATTRibute-CM randomized clinical trial. JAMA Cardiol. 2026;11(5):446-454. doi:10.1001/jamacardio.2026.0819
  2. Attruby. Prescribing information. BridgeBio Pharma, Inc.; 2024.
  3. Gillmore JD, Judge DP, Cappelli F, et al. Efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy. N Engl J Med. 2024;390(2):132-142. doi:10.1056/NEJMoa2305434
  4. Judge DP, Heitner SB, Falk RH, et al. Transthyretin stabilization by AG10 in symptomatic transthyretin amyloid cardiomyopathy. J Am Coll Cardiol. 2019;74(3):285-295. doi:10.1016/j.jacc.2019.03.012
  5. Data on file. BridgeBio Pharma, Inc.; 2024-2026.
  6. Judge DP, Gillmore JD, Alexander KM, et al. Long-term efficacy and safety of acoramidis in ATTR-CM: initial report from the open-label extension of the ATTRibute-CM trial. Circulation. 2025;151(9):601-611. doi:10.1161/CIRCULATIONAHA.124.072771
  7. Judge DP, Gillmore JD, Alexander KM, et al. Long-term efficacy and safety of acoramidis in ATTR-CM: initial report from the open-label extension of the ATTRibute-CM trial. Circulation. 2025;151(suppl 1):1-4. doi:10.1161/CIRCULATIONAHA.124.072771
  8. Maurer MS, Judge DP, Gillmore JD, et al. Early increase in serum transthyretin by acoramidis independently predicts improved survival in TTR amyloid cardiomyopathy. J Am Coll Cardiol. 2025;85(20):1911-1923. doi:10.1016/j.jacc.2025.03.542
  9. Ambardekar AV, Sarswat N, Wright R, et al. Acoramidis, serum transthyretin, and cardiovascular outcomes in transthyretin amyloid cardiomyopathy: insights from the ATTRibute-CM trial. J Card Fail. 2026;32(5):864-876. doi:10.1016/j.cardfail.2026.02.045.
  10. Sarswat N, Ambardekar A, Taubel J, et al. Acoramidis-mediated early increase in serum transthyretin is associated with lower cardiovascular-related hospitalizations and mortality: insights from the ATTRibute-CM study. Poster presented at: American College of Cardiology Annual Scientific Meeting; March 29-31, 2025; Chicago, IL.
  11. Spertus JA, Jones PG, Sandhu AT, Arnold SV. Interpreting the Kansas City Cardiomyopathy Questionnaire in clinical trials and clinical care: JACC state-of-the-art review. J Am Coll Cardiol. 2020;76(20): 2379-2390. doi:10.1016/j.jacc.2020.09.542
  12. Suzuki S, Sugiyama S. The molar ratio of N-terminal pro-B-type natriuretic peptide/B-type natriuretic peptide for heart failure-related events in stable outpatients with cardiovascular risk factors. Internal Medicine. 2018;57(18):2621-2630. doi:10.2169/internalmedicine.0471-17
  13. Gillmore JD, Judge DP, Cappelli F, et al. Efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy [protocol]. N Engl J Med. 2024;390:132-142. doi:10.1056/NEJMoa2305434
  14. Alexander KM, Davis MK, Akinboboye O, et al. Efficacy of acoramidis in wild-type and variant transthyretin amyloid cardiomyopathy: results from ATTRibute-CM and its open-label extension. JAMA Cardiol. 2026;11(1):57-67. doi:10.1001/jamacardio.2025.4477 
  15. Maurer M, Sarswat N, Grogan M, et al. Acoramidis improves serum TTR levels in patients with wild-type or variant transthyretin amyloid cardiomyopathy—Results from ATTRibute-CM. Presented at: Annual Congress of the Heart Failure Association of the European Society of Cardiology; May 17-20, 2025; Belgrade, Serbia.
  16. Fontana M, Kumar V, Sheridan P, et al. Descriptive analysis of unmet need in a contemporary cohort of tafamidis-treated patients with ATTR-CM. Poster presented at: Heart Failure Society of America Annual Scientific Meeting; September 27-30, 2024; Atlanta, GA.
  17. Gillmore JD, Judge DP, Cappelli F, et al. Efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy. N Engl J Med. 2024;390(2)(suppl 1):1-34. doi: 10.1056/NEJMoa2305434

Important Safety Information and Indication

Important Safety Information

Adverse Reactions
Diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation.
Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively).

Laboratory Tests
Mean increase in serum creatinine of 0.2 and 0.0 mg/dL and a mean decrease in eGFR of 8.2 and 0.7 mL/min/1.73 m2 was observed in the adults with ATTR-CM treated with Attruby versus placebo, respectively, at Day 28 and then stabilized. These changes were reversible after treatment discontinuation.

Use in Specific Populations

Pregnancy & Lactation: There are no data on the use of Attruby in pregnant women. Animal data have not shown developmental risk associated with the use of Attruby in pregnancy. There are no available data on the presence of Attruby in either human or animal milk or the effects of the drug on the breastfed infant or maternal milk production.

Indication

Attruby® (acoramidis) is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization.

Please see Full Prescribing Information including Patient Information.

You’re about to leave AttrubyHCP.com and visit an external site. BridgeBio is not responsible for the content or privacy practices of the site you’re about to redirect to.

BridgeBio is committed to protecting and respecting your privacy. All information collected will be handled in accordance with our Privacy Policy. By clicking "Submit," you agree to share your contact information and certain health information with BridgeBio, BridgeBio affiliates, and companies working with BridgeBio or BridgeBio affiliates (collectively, "BridgeBio"). You agree that BridgeBio may contact you by mobile or online digital media, mail, email, fax, telephone call, and text messages (including autodialed and prerecorded calls and messages) for marketing purposes, including targeted online marketing, or to otherwise provide you with information about BridgeBio's products, treatment, or other offers. You can unsubscribe from these communications at any time by clicking "Unsubscribe" in the communications you receive.

More information on DDIs1

Avoid concomitant use of Attruby (acoramidis) with UGT inducers and strong CYP3A inducers, as these can potentially decrease Attruby exposure.

Consider more frequent monitoring for evidence of increased exposure to CYP2C9 substrates when Attruby (acoramidis) is coadministered with sensitive CYP2C9 substrates.

CYP=cytochrome P450; DDI=drug-drug interaction; UGT=uridine diphosphate glucuronosyltransferase.

Established Safety Profile4

Patients With 1 or More Events Attruby
n=421, % (n)		 Placebo
n=211, % (n)
Any TEAEs 98.1% (413) 97.6% (206)
Any treatment-emergent SAEs 54.6% (230) 64.9% (137)
Any treatment-related TEAEs 11.9% (50) 5.2% (11)
Severe TEAEs 37.3% (157) 45.5% (96)

SAE=serious adverse event; TEAE=treatment-emergent adverse event.