VivoSeq
Metabolic & Endocrine

Homozygous Familial Hypercholesterolemia

29

Pipeline Programs↗

5

Active Trials↗

20

Companies Involved↗

Homozygous Familial Hypercholesterolemia is an emerging landscape with 29 programs across 20 companies. The most active players are Beijing Suncadia Pharmaceuticals Co., Ltd, Cerenis Therapeutics, SA, NeuroBo Pharmaceuticals Inc.. 19 programs are in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

52/100

Pipeline Velocity

11/100

Mech. Concentration

55/100

Completion Rate

82/100

Attrition Rate

15/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

B

Beijing Suncadia Pharmaceuticals Co., Ltd

1 program

Phase 2C

Cerenis Therapeutics, SA

1 program

Phase 2N

NeuroBo Pharmaceuticals Inc.

1 program

Phase 2A

Akcea Therapeutics

1 program

Phase 2Sanofi logo

Sanofi

2 programs

Phase 3Takeda logo

Takeda

2 programs

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

81%

Small molecule

26 of 32 classified programs — the dominant modality here.

19%ALL OTHERSMonoclonal antibody39%RNA therapy39%

Small molecule leads; the ring shows all 32 classified programs, each share of the total. Modality classified for 100% of 32 pipeline programs.

Analysis

Trial Status

34

clinical trials

Completed23 · 68%
Terminated3 · 9%
Recruiting2 · 6%
Withdrawn2 · 6%
Not yet recruiting2 · 6%
Active, not recruiting1 · 3%
Other1 · 3%

Clinical Trials

Trial Activity

34 trials

Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Recruiting·Novartis logoNovartis·Phase 3·NCT06597006·9 enrolled
A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
Recruiting·Arrowhead Pharmaceuticals logoArrowhead Pharmaceuticals·Phase 3·NCT07037771·60 enrolled
Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)
Active, not recruiting·Arrowhead Pharmaceuticals logoArrowhead Pharmaceuticals·Phase 2·NCT05217667·18 enrolled
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Not yet recruiting·Arrowhead Pharmaceuticals logoArrowhead Pharmaceuticals·Phase 3·NCT07473843·12 enrolled
Evaluate the Long-term Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
Not yet recruiting·BBeijing Suncadia Pharmaceuticals Co., Ltd·Phase 2·NCT07133815·55 enrolled
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia
Completed·Novartis logoNovartis·Phase 3·NCT04659863·13 enrolled
Effect of CER-001 on Plaque Volume in Homozygous Familial Hypercholesterolemia (HoFH) Subjects
Completed·CCerenis Therapeutics, SA·Phase 2·NCT01412034·23 enrolled
An Efficacy and Safety Study of Alirocumab in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Completed·Sanofi logoSanofi·Phase 3·NCT03510715·18 enrolled
Safety and Tolerability of Repatha® (Evolocumab) in Indian Participants With Homozygous Familial Hypercholesterolemia
Completed·Amgen logoAmgen·Phase 4·NCT03403374·30 enrolled
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
Completed·KKastle Therapeutics, LLC·Phase 3·NCT00607373·51 enrolled
Dose-escalating Safety Study of ISIS 301012 in Homozygous Familial Hypercholesterolemia Subjects on Lipid Lowering Therapy
Completed·KKastle Therapeutics, LLC·Phase 2·NCT00280995·12 enrolled
Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
Completed·AAegerion Pharmaceuticals, Inc.·Phase 3·NCT00943306·19 enrolled
A Study to Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Completed·AstraZeneca logoAstraZeneca·Phase 3·NCT02434497·9 enrolled
Study in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Completed·Regeneron logoRegeneron·Phase 3·NCT03156621·69 enrolled
Efficacy and Safety of Gemcabene in Patients With Homozygous Familial Hypercholesterolemia on Stable, Lipid-Lowering Therapy (COBALT-1)
Completed·NNeuroBo Pharmaceuticals Inc.·Phase 2·NCT02722408·8 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)