Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Cardiovascular
4
Pipeline Programs
2
Companies
3
Clinical Trials
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
3
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
2100%
+ 1 programs with unclassified modality

On Market (1)

Approved therapies currently available

Alnylam Pharmaceuticals
AMVUTTRAApproved
vutrisiran
Alnylam Pharmaceuticals
subcutaneous2022

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Alnylam Pharmaceuticals
2 programs
2
1
VutrisiranPhase 3RNA Therapeutic1 trial
VutrisiranPhase 3RNA Therapeutic1 trial
Active Trials
NCT06679946Enrolling By Invitation700Est. Mar 2028
NCT04153149Active Not Recruiting655Est. Dec 2026
Intellia Therapeutics
1 program
1
NTLA-2001Phase 31 trial
Active Trials
NCT06128629Active Not Recruiting1,200Est. Apr 2028

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
Alnylam PharmaceuticalsVutrisiran
Intellia TherapeuticsNTLA-2001
Alnylam PharmaceuticalsVutrisiran

Clinical Trials (3)

Total enrollment: 2,555 patients across 3 trials

NCT06679946Alnylam PharmaceuticalsVutrisiran

A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Start: Dec 2024Est. completion: Mar 2028700 patients
Phase 3Enrolling By Invitation
NCT06128629Intellia TherapeuticsNTLA-2001

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Start: Dec 2023Est. completion: Apr 20281,200 patients
Phase 3Active Not Recruiting
NCT04153149Alnylam PharmaceuticalsVutrisiran

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Start: Nov 2019Est. completion: Dec 2026655 patients
Phase 3Active Not Recruiting

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

3 late-stage (Phase 3) programs — potential near-term approvals
RNA Therapeutic is the dominant modality (100% of programs)
2 companies competing in this space

Related Indications

Heart Failure(334)Hypertension(333)Coronary Artery Disease(295)Atrial Fibrillation(288)Pulmonary Arterial Hypertension(103)Acute Coronary Syndrome(62)Pulmonary Hypertension(55)Essential Hypertension(32)Coronary Heart Disease(28)Aortic Valve Stenosis(27)