Acoramidis for Amyloid Cardiomyopathy

(ATTRibute-CM Trial)

No longer recruiting at 119 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Eidos Therapeutics, a BridgeBio company

Must not be taking: Calcium channel blockers

Disqualifiers: Acute myocardial infarction, Stroke, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: 30 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests acoramidis, a new medication, to determine its safety and effectiveness for individuals with Transthyretin Amyloid Cardiomyopathy (ATTR-CM), a heart condition that can lead to heart failure. Participants will receive either the medication or a placebo (a harmless pill with no medical effect) to compare outcomes such as heart health and hospital visits over time. The study seeks individuals diagnosed with ATTR-CM who have experienced heart failure symptoms, such as requiring ongoing diuretic treatment to manage symptoms. As a Phase 3 trial, this is the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications for ATTR-CM and calcium channel blockers like verapamil and diltiazem. However, you can continue using dihydropyridine calcium channel blockers and digitalis if needed for specific heart conditions.

Is there any evidence suggesting that acoramidis is likely to be safe for humans?

Studies have shown that acoramidis, the treatment being tested, is generally safe for people. Most individuals experience only mild side effects, such as stomach pain and diarrhea. Serious side effects, like an allergic reaction, are rare. Research in animals suggests that acoramidis does not cause harm during pregnancy. Overall, the treatment appears safe, with only a few mild side effects reported. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for ATTR-CM?

Acoramidis is unique because it specifically stabilizes the transthyretin protein, potentially preventing the buildup of amyloid deposits in the heart—a hallmark of transthyretin amyloid cardiomyopathy. Unlike standard treatments like tafamidis, which also aim to stabilize transthyretin but may have different binding affinities and efficacy levels, acoramidis offers a novel approach with its high potency in stabilizing the protein. Researchers are excited about acoramidis because it could significantly improve heart function and reduce hospitalizations related to heart complications, offering new hope for patients with this challenging condition.

What evidence suggests that acoramidis might be an effective treatment for ATTR-CM?

Research has shown that acoramidis may help treat transthyretin amyloid cardiomyopathy (ATTR-CM), a heart condition. In this trial, participants will receive either acoramidis or a placebo. Earlier studies found that people taking acoramidis experienced fewer heart problems than those taking a placebo, which contains no active medicine. Specifically, acoramidis reduced heart-related issues by 49% over 30 months. Patients also showed better overall health, with fewer hospital visits for heart problems and lower death rates. These results suggest that acoramidis could be a good option for managing ATTR-CM symptoms. ^{6 7 8 9 10}

Are You a Good Fit for This Trial?

Inclusion Criteria

You have had heart failure in the past and were hospitalized for it, or you currently have symptoms and are being treated with a diuretic medication.

You have symptoms of heart disease categorized as NYHA Class I-III due to ATTR cardiomyopathy.

On stable doses of cardiovascular medical therapy

Exclusion Criteria

You are taking certain types of calcium channel blockers that affect your heart's conduction system, such as verapamil or diltiazem. However, if you are taking dihydropyridine calcium channel blockers or digitalis to manage atrial fibrillation with rapid ventricular response, you may be eligible.

Biomarkers of myocardial wall stress, NT-proBNP level ≥8500 pg/mL at screening

Measure of kidney function, eGFR by MDRD formula <15 mL/min/1.73 m2

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive acoramidis HCl 800 mg or placebo twice daily for 30 months

30 months

Regular visits every 3 months for PK-PD substudy

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants may opt into continuation of acoramidis treatment long-term without placebo

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• acoramidis
• Placebo Oral Tablet

How Is the Trial Designed?

Group I: acoramidis HCl 800 mg Experimental Treatment 1 Intervention

Subjects will receive acoramidis HCl 800 mg twice daily. 6MWT primary outcome will be assessed at the end of 12 months. The hierarchical combination of All-Cause mortality, cumulative frequency of cardiovascular-related hospitalizations, change from baseline in NT-proBNP levels, and change from baseline in distance walked on the 6MWT will be assessed after 30 months of treatment.

Group II: Placebo Placebo Group 1 Intervention

Subjects will receive placebo to match twice daily. 6MWT primary outcome will be assessed at the end of 12 months. The hierarchical combination of All-Cause mortality, cumulative frequency of cardiovascular-related hospitalizations, change from baseline in NT-proBNP levels, and change from baseline in distance walked on the 6MWT will be assessed after 30 months of treatment.

Who Is Running the Clinical Trial?

Eidos Therapeutics, a BridgeBio company

Lead Sponsor

Trials

12

Recruited

2,400+

Published Research Related to This Trial

The Drug Adverse Reaction Database (DART) provides detailed information on 147 confirmed and 89 potential protein targets associated with adverse drug reactions (ADRs), which is crucial for understanding the mechanisms behind these reactions and aiding in drug discovery.

DART includes extensive data on 257 drugs and 1080 ligands, offering insights into their binding properties, physiological functions, and the specific adverse effects they may cause, making it a valuable resource for researchers and developers in pharmacology.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug Adverse Reaction Target Database (DART) : proteins related to adverse drug reactions. Ji, ZL., Han, LY., Yap, CW., et al.[2018]

In a clinical development program involving 1684 subjects and 2038 injections, OptiMARK demonstrated a safety profile comparable to Magnevist, with 31% of its injections associated with adverse events.

OptiMARK was found to be safe and well-tolerated, showing fewer adverse events compared to Magnevist (35%) and placebo (48%), indicating its potential as a reliable imaging agent.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The OptiMARK clinical development program: summary of safety data. Brown, JJ., Kristy, RM., Stevens, GR., et al.[2019]

The DIMDI provides comprehensive databases that contain valuable information on adverse drug reactions, which can help healthcare professionals and researchers understand the safety profiles of various medications.

Access to these databases is crucial for monitoring drug safety and improving patient care by identifying potential risks associated with drug treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Information on adverse drug reactions in databases hosted at the German Institute of Medical Documentation and Information (DIMDI)]. Bystrich, E.[2016]

The study found a high overlap of safety signals (SDRs) among the EudraVigilance Data Analysis System (EVDAS), FAERS, and VigiBase® databases, with median overlaps of 85% at the Preferred Term level and up to 97.7% when combining High Level Terms with Standardized MedDRA® Queries.

Despite the high overlap, the recall of signals in EVDAS was only about 59.4% to 87.9% of all signals generated across the databases, indicating that while the databases share many similar safety profiles, there are still differences in reporting that can affect signal detection.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Investigating Overlap in Signals from EVDAS, FAERS, and VigiBase&#174;. Vogel, U., van Stekelenborg, J., Dreyfus, B., et al.[2021]

The safety of marketed drugs is a significant concern, as some commonly prescribed medications can lead to serious or life-threatening side effects in patients.

The ChEMBL resource will provide a curated drug safety data set, including toxicity classifications and black box warnings, which will be freely available and regularly updated to aid in drug safety research and discovery.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug Safety Data Curation and Modeling in ChEMBL: Boxed Warnings and Withdrawn Drugs. Hunter, FMI., Bento, AP., Bosc, N., et al.[2023]

Citations

1.

jacc.org

jacc.org/doi/full/10.1016/j.jacc.2025.09.013

Effect of Acoramidis on Recurrent and Cumulative ...

Acoramidis significantly reduced the burden of cumulative CV outcomes in ATTR-CM over 30 months. Numerically fewer events were observed with acoramidis vs ...

2.

nejm.org

nejm.org/doi/full/10.1056/NEJMoa2305434

Efficacy and Safety of Acoramidis in Transthyretin Amyloid ...

In patients with transthyretin amyloid cardiomyopathy, the receipt of acoramidis resulted in a significantly better four-step primary hierarchical outcome.

3.

investor.bridgebio.com

investor.bridgebio.com/news/news-details/2025/Acoramidis-Begins-to-Reduce-Cumulative-Cardiovascular-Outcomes-Within-the-First-Month-of-Treatment-in-Patients-with-ATTR-CM/default.aspx

Acoramidis Begins to Reduce Cumulative Cardiovascular ...

Acoramidis significantly reduced the cumulative risk of CVM or recurrent CVH through Month 30 versus placebo with a 49% hazard reduction (p< ...

4.

acc.org

acc.org/latest-in-cardiology/journal-scans/2025/09/30/13/39/new-insights-into-outcomes-with-acoramidis

New Insights Into Outcomes With Acoramidis in ATTR-CM, ...

Results showed at 30 months that acoramidis significantly reduced the cumulative risk of cardiovascular mortality or recurrent cardiovascular ...

5.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40074465/

Efficacy of Acoramidis on All-Cause Mortality and ...

In participants with ATTR-CM, acoramidis reduced the composite of ACM or first CVH vs placebo, with an early effect driven by a reduction in CVH.

6.

webmd.com

webmd.com/drugs/2/drug-500597/attruby-acoramidis/details

Attruby (acoramidis) - Uses, Side Effects, and More

Common side effects include diarrhea and stomach pain. Serious side effects are rare, but can include an allergic reaction. Uses ...

7.

drugs.com

drugs.com/sfx/acoramidis-side-effects.html

Acoramidis Side Effects: Common, Severe, Long Term

The most frequent adverse events were gastrointestinal adverse reactions such as diarrhea and upper abdominal pain.

8.

rxlist.com

rxlist.com/acoramidis/generic-drug.htm

Acoramidis: Side Effects, Uses, Dosage, Interactions, ...

What Are Side Effects Associated with Using Acoramidis? Common side effects of Acoramidis include: abdominal pain; diarrhea. Serious side ...

9.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/label/2024/216540s000lbl.pdf

Attruby - accessdata.fda.gov

In pregnant rats, oral administration of acoramidis (0, 50, 350, and 1,000 mg/kg/day) throughout organogenesis did not result in any adverse effects on ...

10.

ema.europa.eu

ema.europa.eu/en/documents/product-information/beyonttra-epar-product-information_en.pdf

Beyonttra, INN-acoramidis - EMA

Acoramidis has not been studied in patients with hepatic impairment. 5.3 Preclinical safety data. Non-clinical data reveal no special hazard ...