Transthyretin (TTR)-Mediated Amyloidosis

2
Pipeline Programs
1
Companies
2
Clinical Trials
0
Approved Products

Pipeline by Development Stage

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

Drug Modality Breakdown

RNA Therapeutic
1100%
+ 1 programs with unclassified modality

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Alnylam Pharmaceuticals
2 programs
1
1
RevusiranPhase 21 trial
patisiranPhase 1RNA Therapeutic1 trial
Active Trials
NCT02053454Completed12Est. Jun 2014
NCT02595983Completed12Est. Feb 2017

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Alnylam PharmaceuticalsRevusiran
Alnylam Pharmaceuticalspatisiran

Clinical Trials (2)

Total enrollment: 24 patients across 2 trials

NCT02595983Alnylam PharmaceuticalsRevusiran

The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant

Start: Oct 2015Est. completion: Feb 201712 patients
Phase 2Completed
NCT02053454Alnylam Pharmaceuticalspatisiran

A Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy Volunteers

Start: Jan 2014Est. completion: Jun 201412 patients
Phase 1Completed

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

RNA Therapeutic is the dominant modality (100% of programs)
1 companies competing in this space