TTR-mediated Amyloidosis

8
Pipeline Programs
1
Companies
8
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
2
0
4
0
1
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
3100%
+ 5 programs with unclassified modality

On Market (1)

Approved therapies currently available

Alnylam Pharmaceuticals
ONPATTROApproved
patisiran
Alnylam Pharmaceuticals
intravenous2018

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Alnylam Pharmaceuticals
8 programs
2
4
1
1
patisiranPhase 3RNA Therapeutic1 trial
ALN-TTR02Phase 21 trial
ALN-TTRSCPhase 21 trial
PatisiranPhase 2RNA Therapeutic1 trial
RevusiranPhase 21 trial
+3 more programs
Active Trials
NCT02939820Approved For Marketing
NCT01559077Completed17Est. Nov 2012
NCT01814839Completed85Est. May 2015
+5 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Alnylam Pharmaceuticalspatisiran
Alnylam PharmaceuticalsRevusiran
Alnylam PharmaceuticalsALN-TTRSC
Alnylam PharmaceuticalsALN-TTR02
Alnylam PharmaceuticalsPatisiran
Alnylam PharmaceuticalsALN-TTRSC
Alnylam PharmaceuticalsALN-TTR02

Clinical Trials (8)

Total enrollment: 434 patients across 8 trials

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Start: Nov 2013Est. completion: Aug 2017225 patients
Phase 3Completed

A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

Start: Oct 2014Est. completion: Feb 201725 patients
Phase 2Completed

Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

Start: Dec 2013Est. completion: Jan 201526 patients
Phase 2Completed

The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)

Start: Oct 2013Est. completion: Aug 201627 patients
Phase 2Completed

Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

Start: May 2012Est. completion: Jan 201429 patients
Phase 2Completed

A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers

Start: Mar 2013Est. completion: May 201585 patients
Phase 1Completed

Trial to Evaluate Safety, Tolerability, and Parmacokinetics of ALN-TTR02 in Healthy Volunteer Subjects

Start: Mar 2012Est. completion: Nov 201217 patients
Phase 1Completed

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

N/AApproved For Marketing

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
1 companies competing in this space