VivoSeq
Hematology

Amyloidosis

67

Pipeline Programs↗

30

Active Trials↗

29

Companies Involved↗

Amyloidosis is a moderately active landscape with 67 programs across 29 companies. The most active players are Prothena Biosciences Limited, Novo Nordisk, Karyopharm Therapeutics Inc. 7 therapies are approved with 10 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

66/100

Pipeline Velocity

34/100

Mech. Concentration

14/100

Completion Rate

80/100

Attrition Rate

13/100

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.

P

Prothena Biosciences Limited

1 program

Phase 1Novo Nordisk logo

Novo Nordisk

1 program

Phase 1K

Karyopharm Therapeutics Inc

1 program

Phase 1Johnson & Johnson logo

Johnson & Johnson

3 programs

Phase 2AbbVie logo

AbbVie

2 programs

Phase 2P

Prothena Biosciences Ltd.

1 program

Phase 2

Pipeline

Programs by Phase

Preclinical5

Ixazomib Citrate

Takeda

Diflunisal

Cidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

Bortezominb and Dexamethasone

Zhi-Hong Liu, M.D.

First-Line Dara-Borcyd Treatment

Peking University First Hospital

Stapokibart

Chengdu Kangnuoxing Biopharma,Inc.

Phase 114

Carfilzomib

Virionyx Corporation Limited

Tolcapone

Bausch Health Americas, Inc.

Selinexor

Karyopharm Therapeutics Inc

Melphalan-Flufenamide (melflufen)

Oncopeptides AB

Florbetaben F18

University of Chicago

Phase 231

Patisiran

Alnylam Pharmaceuticals

Daratumumab

Johnson & Johnson

Idelalisib

Gilead

Doxycycline

Pfizer's Upjohn has merged with Mylan to form Viatris Inc.

Dexamethasone

Aspen Global Incorporated

Phase 310

Lenalidomide

Karuna Therapeutics, Inc., a Bristol Myers Squibb company

Thalidomide

Bristol-Myers Squibb

Cyclophosphamide

Baxter Healthcare Corporation

Ixazomib

Takeda

I-124 Evuzamitide

Sharmila Dorbala

Approved7

Tafamidis

K-Group Alpha, Inc., a wholly owned subsidiary of Zentalis Pharmaceuticals, Inc.

Beta Blocker

Weill Medical College of Cornell University

Tofacitinib

Prism Pharmaceuticals

Trimetazidine

IRCCS San Raffaele

Acitretin

GSK

Analysis

Modality Breakdown

81%

Small molecule

58 of 72 classified programs — the dominant modality here.

19%ALL OTHERSMonoclonal antibody710%RNA therapy34%Protein / enzyme23%Peptide11%Cell therapy11%

Small molecule leads; the ring shows all 72 classified programs, each share of the total. Modality classified for 99% of 77 pipeline programs.

Analysis

Trial Status

106

clinical trials

Completed57 · 54%
Recruiting20 · 19%
Terminated9 · 8%
Active, not recruiting5 · 5%
Withdrawn5 · 5%
Unknown status4 · 4%
Other6 · 6%

Clinical Trials

Trial Activity

106 trials

Daratumumab for Patients With Light Chain Amyloidosis
Recruiting·NNanjing University School of Medicine·N/A·NCT06376214·100 enrolled
Test-retest Study With [18F]FBB in Cardiac Amyloidosis
Recruiting·LLife Molecular Imaging GmbH·Phase 1·NCT06790394·15 enrolled
A Prospective Cohort Study on Primary Cutaneous Amyloidosis
Recruiting·—·Phase 4·NCT06998875·56 enrolled
Study Evaluating the Efficacy and Safety of CM336 Injection in the Treatment of Light-Chain Amyloidosis
Recruiting·KKeymed Biosciences Co.Ltd·Phase 2·NCT07039578·90 enrolled
BCMA/CD3 Bispecific Antibody Treatment for Newly Diagnosed Amyloidosis
Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 2·NCT07151690·21 enrolled
Venetoclax, MLN9708 (Ixazomib Citrate) and Dexamethasone for the Treatment of Relapsed or Refractory Light Chain Amyloidosis
Recruiting·National Cancer Institute (NCI)·Phase 1·NCT04847453·24 enrolled
A Study of AT-02 in Subjects With Systemic Amyloidosis.
Recruiting·AAttralus, Inc.·Phase 2·NCT05951049·120 enrolled
Daratumumab Combined With Venetoclax and Dexamethasone for Newly Diagnosed Light-Chain Amyloidosis With Translocation (11;14)
Recruiting·PPeking Union Medical College Hospital·Phase 2·NCT06629818·36 enrolled
Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load
Recruiting·National Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT06974877·30 enrolled
A Phase II Trial of Teclistamab in Participants With Previously Treated Immunoglobulin Light-chain (AL) Amyloidosis
Recruiting·EEuropean Myeloma Network B.V.·Phase 2·NCT06649695·30 enrolled
Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis
Recruiting·AAttralus, Inc.·Phase 1·NCT05521022·100 enrolled
Daratumumab Maintenance Therapy for Improving Survival in Patients With Light Chain Amyloidosis, EMILIA Trial
Recruiting·MMayo Clinic·Phase 2·NCT05898646·96 enrolled
Safety, Efficacy, and Pharmacokinetics of Tafamidis in Patients With Transthyretin-mediated Amyloidosis Post Orthotopic Heart Transplantation
Recruiting·UUniversity of Texas Southwestern Medical Center·Phase 4·NCT05489523·25 enrolled
A Study to Evaluate the Safety and Efficacy of SCTC21C in Combination With Bortezomib, Cyclophosphamide, and Dexamethasone in Patients With Newly Diagnosed Systemic Light-Chain Amyloidosis (NDSLCA)
Recruiting·SSinocelltech Ltd.·Phase 3·NCT07388602·90 enrolled
FKC288 for Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis
Recruiting·NNanjing University School of Medicine·Phase 1·NCT05978661·12 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)