Heterozygous Familial Hypercholesterolemia

6
Pipeline Programs
5
Companies
4
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
1
0
3
2
Early DiscoveryClinical DevelopmentMarket

On Market (1)

Approved therapies currently available

U
KYNAMROApproved
mipomersen sodium
Unknown Company
subcutaneous2013

Competitive Landscape

5 companies ranked by most advanced pipeline stage

Regeneron
RegeneronTARRYTOWN, NY
2 programs
1
1
PraluentPhase 4
LMTPhase 3
Sanofi
SanofiPARIS, France
1 program
1
LMTPhase 31 trial
Active Trials
NCT01709500Completed249Est. Jan 2015
Ionis Pharmaceuticals
1 program
1
mipomersen sodiumPhase 31 trial
Active Trials
NCT00706849Completed124Est. May 2010
Madrigal Pharmaceuticals
Madrigal PharmaceuticalsWEST CONSHOHOCKEN, PA
1 program
1
MGL-3196Phase 21 trial
Active Trials
NCT03038022Completed116Est. Jan 2018
Amgen
AmgenTHOUSAND OAKS, CA
1 program
Lp(a) and Aortic Valve CalcificationN/A1 trial
Active Trials
NCT02976818Completed173Est. Feb 2020

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
SanofiLMT
Ionis Pharmaceuticalsmipomersen sodium
Madrigal PharmaceuticalsMGL-3196
AmgenLp(a) and Aortic Valve Calcification

Clinical Trials (4)

Total enrollment: 662 patients across 4 trials

NCT01709500SanofiLMT

Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy)

Start: Dec 2012Est. completion: Jan 2015249 patients
Phase 3Completed
NCT00706849Ionis Pharmaceuticalsmipomersen sodium

Efficacy and Safety Study of ISIS 301012 (Mipomersen) as Add-on in Familial Hypercholesterolemic Patients With Coronary Artery Disease

Start: Jul 2008Est. completion: May 2010124 patients
Phase 3Completed
NCT03038022Madrigal PharmaceuticalsMGL-3196

Study of MGL-3196 in Patients With Heterozygous Familial Hypercholesterolemia (HeFH)

Start: Feb 2017Est. completion: Jan 2018116 patients
Phase 2Completed
NCT02976818AmgenLp(a) and Aortic Valve Calcification

Lp(a) and Aortic Valve Calcification

Start: Apr 2017Est. completion: Feb 2020173 patients
N/ACompleted

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
5 companies competing in this space