Amyloidosis

10
Pipeline Programs
8
Companies
49
Clinical Trials
1 recruiting
1
Approved Products

Pipeline by Development Stage

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

Drug Modality Breakdown

Small Molecule
150%
RNA Therapeutic
150%
+ 9 programs with unclassified modality

On Market (1)

Approved therapies currently available

Alnylam Pharmaceuticals
ONPATTROApproved
patisiran
Alnylam Pharmaceuticals
intravenous2018

Competitive Landscape

8 companies ranked by most advanced pipeline stage

Alnylam Pharmaceuticals
1 program
1
1
PatisiranPhase 3RNA Therapeutic1 trial
Active Trials
NCT02510261Completed211Est. Nov 2022
BridgeBio Pharma
BridgeBio PharmaCA - Palo Alto
3 programs
1
2
AcoramidisPhase 31 trial
acoramidisPhase 31 trial
acoramidisPhase 11 trial
Active Trials
NCT04769479Completed14Est. Sep 2021
NCT06563895Recruiting582Est. Dec 2032
NCT03860935Completed632Est. May 2023
Ionis Pharmaceuticals
2 programs
1
Isis 420915/GSK 299872Phase 21 trial
99m-technetium pyrophosphate scintigraphyN/A1 trial
Active Trials
NCT05259072Terminated4Est. Dec 2023
NCT02627820Withdrawn0Est. Dec 2018
Sanofi
SanofiPARIS, France
1 program
1
Bendamustine HydrochloridePhase 21 trial
Active Trials
NCT04943302Withdrawn0Est. Jan 2026
GS
Gilead SciencesFOSTER CITY, CA
1 program
1
IdelalisibPhase 2Small Molecule1 trial
Active Trials
NCT02590588Terminated1Est. Mar 2017
J&
Johnson & JohnsonNEW BRUNSWICK, NJ
1 program
1
VELCADEPhase 1/25 trials
Active Trials
NCT00367718Withdrawn0
NCT00799539No Longer Available
NCT00298779Completed20
+2 more trials
Zentalis Pharmaceuticals
1 program
1
ZN-d5Phase 1/21 trial
Active Trials
NCT05199337CompletedEst. Feb 2024
Pfizer
PfizerNEW YORK, NY
1 program
Shear wave elastographyN/A1 trial
Active Trials
NCT04456582Unknown60Est. May 2021

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
BridgeBio PharmaAcoramidis
BridgeBio Pharmaacoramidis
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE
Alnylam PharmaceuticalsPatisiran
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE
Johnson & JohnsonVELCADE

Showing 15 of 46 trials with date data

Clinical Trials (49)

Total enrollment: 12,605 patients across 49 trials

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

Start: May 2025Est. completion: Dec 2032582 patients
Phase 3Recruiting

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

Start: Mar 2019Est. completion: May 2023632 patients
Phase 3Completed

Daratumumab, VELCADE (Bortezomib), Lenalidomide and Dexamethasone Compared to VELCADE, Lenalidomide and Dexamethasone in Subjects With Previously Untreated Multiple Myeloma

Start: Dec 2018Est. completion: Nov 2029709 patients
Phase 3Active Not Recruiting

A Study Comparing Daratumumab, VELCADE (Bortezomib), Lenalidomide, and Dexamethasone (D-VRd) With VELCADE, Lenalidomide, and Dexamethasone (VRd) in Participants With Untreated Multiple Myeloma and for Whom Hematopoietic Stem Cell Transplant is Not Planned as Initial Therapy

Start: Nov 2018Est. completion: Mar 2026395 patients
Phase 3Active Not Recruiting

A Study to Compare Daratumumab, Bortezomib, and Dexamethasone (DVd) vs Bortezomib and Dexamethasone (Vd) in Chinese Participants With Relapsed or Refractory Multiple Myeloma

Start: Nov 2017Est. completion: Feb 2024213 patients
Phase 3Completed

A Study of VELCADE (Bortezomib) Melphalan-Prednisone (VMP) Compared to Daratumumab in Combination With VMP (D-VMP), in Participants With Previously Untreated Multiple Myeloma Who Are Ineligible for High-Dose Therapy (Asia Pacific Region)

Start: Nov 2017Est. completion: Mar 2024220 patients
Phase 3Completed

Study of Subcutaneous and Intravenous Velcade in Combination With Dexamethasone in Chinese Subjects With Relapsed and Refractory Multiple Myeloma

Start: Sep 2016Est. completion: Nov 201881 patients
Phase 3Completed

A Study to Evaluate Daratumumab in Transplant Eligible Participants With Previously Untreated Multiple Myeloma

Start: Sep 2015Est. completion: Sep 20231,085 patients
Phase 3Completed

The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

Start: Jul 2015Est. completion: Nov 2022211 patients
Phase 3Completed

A Study of Combination of Daratumumab and Velcade (Bortezomib) Melphalan-Prednisone (DVMP) Compared to Velcade Melphalan-Prednisone (VMP) in Participants With Previously Untreated Multiple Myeloma

Start: Dec 2014Est. completion: Aug 2024706 patients
Phase 3Completed

Addition of Daratumumab to Combination of Bortezomib and Dexamethasone in Participants With Relapsed or Refractory Multiple Myeloma

Start: Aug 2014Est. completion: Jan 2024498 patients
Phase 3Completed

Bortezomib (Velcade®), Lenalidomide (Revlimid®) and IV Busulfan (Busilvex®) in Patients Under 65 Years Old

Start: Sep 2013Est. completion: Nov 2016460 patients
Phase 3Completed

Velcade (Bortezomib) Consolidation After Transplant

Start: Jan 2012Est. completion: Jan 2018256 patients
Phase 3Completed

A Study of Subcutaneous and Intravenous VELCADE in Patients With Previously Treated Multiple Myeloma

Start: Jul 2008Est. completion: Sep 2010222 patients
Phase 3Completed

Study of the Combination of Rituximab, Cyclophosphamide, Doxorubicin, VELCADE, and Prednisone or Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone in Patients With Newly Diagnosed Mantle Cell Lymphoma

Start: May 2008Est. completion: Jun 2017487 patients
Phase 3Completed

VELCADE-Thalidomide-Dexamethasone (VTD) vs Thalidomide-Dexamethasone (TD) Incorporated Into Double Autotransplantation for Untreated Multiple Myeloma (MM)

Start: May 2006Est. completion: Dec 2015480 patients
Phase 3Unknown

Study of VELCADE and Rituximab in Patients With Relapsed or Refractory B-cell Non-Hodgkin's Lymphoma

Start: Mar 2006Est. completion: Jul 2010676 patients
Phase 3Completed

VELCADE/Melphalan/Prednisone Versus Melphalan/Prednisone in Patients With Previously Untreated Multiple Myeloma

Start: Dec 2004
Phase 3Completed

Study of DOXIL/CAELYX (Pegylated Liposomal Doxorubicin) and VELCADE (Bortezomib) or VELCADE Monotherapy for the Treatment of Relapsed Multiple Myeloma

Start: Dec 2004Est. completion: Jun 2014646 patients
Phase 3Completed

Trial of VELCADE in Patients With Metastatic or Locally Recurrent Nasopharyngeal Carcinoma

0
Phase 2Withdrawn
NCT04943302SanofiBendamustine Hydrochloride

Isatuximab and Bendamustine in Systemic Light Chain Amyloidosis

Start: Sep 2022Est. completion: Jan 20260
Phase 2Withdrawn
NCT02627820Ionis PharmaceuticalsIsis 420915/GSK 299872

The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart

Start: Jan 2016Est. completion: Dec 20180
Phase 2Withdrawn

Idelalisib for Immunoglobulin M (IgM)-Associated Primary (AL) Amyloidosis

Start: Jan 2016Est. completion: Mar 20171 patients
Phase 2Terminated

Bendamustine, Bortezomib (Velcade ®) and Prednisone (BVP) in Patients Newly Diagnosed Multiple Myeloma

Start: Jul 2011Est. completion: Dec 201560 patients
Phase 2Completed

Study of Treatment for Newly Diagnosed Multiple Myeloma Patients Older Than 65 Years With Sequential Melphalan/Prednisone/Velcade (MPV) Followed by Revlimid/Low Dose Dexamethasone (Rd) Versus Alternating Velcade/Melphalan/Prednisone (MPV) With Revlimid/Low Dose Dexamethasone

Start: Feb 2011Est. completion: May 2016250 patients
Phase 2Completed

Study of the Combination of VELCADE, Rituximab, Cyclophosphamide, Doxorubicin, and Prednisone or Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone in Patients With Newly Diagnosed Non-Germinal Center B-Cell Subtype of Diffuse Large B-Cell Lymphoma

Start: Jan 2010Est. completion: Aug 2012164 patients
Phase 2Completed

Velcade Consolidation Bone Study

Start: Sep 2009Est. completion: Apr 2014106 patients
Phase 2Completed

A Study to Compare CNTO 328 (Anti-IL-6 Monoclonal Antibody) and VELCADE-Melphalan-Prednisone (VMP) With VMP Alone in Previously Untreated Multiple Myeloma Patients

Start: Jun 2009Est. completion: Apr 2013118 patients
Phase 2Completed

Treatment With Velcade (Bortezomib) Plus Dexamethasone (VD) or VD Plus Cyclophosphamide or VD Plus Lenalidomide in Patients With Multiple Myeloma Stabilized After 4 Cycles of VD

Start: May 2008Est. completion: Aug 2011163 patients
Phase 2Completed

A Study to Evaluate Two Different Regimens of VELCADE in Combination With Dexamethasone, Thalidomide and Cyclophosphamide (VDT vs VDTC) in Newly Diagnosed Multiple Myeloma

Start: Oct 2007Est. completion: May 200998 patients
Phase 2Completed

Velcade (Bortezomib), Adriamycin Dexamethasone (PAD) or Vincristine Adriamycin Dexamethasone in Second Line Treatment of Multiple Myeloma

Start: Dec 2006Est. completion: Jan 200830 patients
Phase 2Terminated

VTD Followed By MPT Maintenance As a First Line Treatment For The Patients With MM Who Are Non-Transplant Candidates

Start: Apr 2006Est. completion: Feb 200835 patients
Phase 2Completed

Sequential VAD and VTD Followed by HDT With ASCT and Velcade Maintenance for NDMM

Start: Mar 2006Est. completion: Jul 200862 patients
Phase 2Unknown

Study of Bortezomib Combined With ACVBP in Peripheral T-cell Lymphoma

Start: Jan 2006Est. completion: Apr 201160 patients
Phase 2Completed

A Phase 2 Study of Velcade™ in Subjects With Relapsed or Refractory Follicular B-Cell Lymphoma

Start: Sep 2005Est. completion: Jan 201087 patients
Phase 2Terminated

Study of Bortezomib (Velcade) in Combination With Dexamethasone to Treat Patients With Relapsed Hodgkin's Lymphoma

Start: Mar 200537 patients
Phase 2Terminated

Phase 1/2 Study of ZN-d5 for the Treatment of Relapsed or Refractory Light Chain (AL) Amyloidosis

Start: Nov 2021Est. completion: Feb 2024
Phase 1/2Completed

Bortezomib and CHOP in Patients With Advanced Stage Aggressive T Cell or Natural Killer (NK)/T Cell Lymphomas

Start: Apr 2006Est. completion: Oct 200955 patients
Phase 1/2Completed

Open-Label Phase 1/2 Study of VELCADE for Injection in Patients With Light-chain (AL)-Amyloidosis

Start: Jun 2005Est. completion: Sep 200970 patients
Phase 1/2Completed

Open-Label Study to Assess the Effect of Omeprazole on the Pharmacokinetics of VELCADE in Patients With Either Advanced Solid Tumors or Non-Hodgkin's Lymphoma.

20 patients
Phase 1Completed

A Single Dose Study to Evaluate the Pharmacokinetics of Acoramidis Modified Release Formulations in Healthy Subjects

Start: Mar 2021Est. completion: Sep 202114 patients
Phase 1Completed

A Study of JNJ-54767414 (HuMax CD38) (Anti-CD38 Monoclonal Antibody) in Combination With Backbone Treatments for the Treatment of Patients With Multiple Myeloma

Start: Feb 2014Est. completion: Jan 2024240 patients
Phase 1Completed

A Dose Escalation Study of the Histone Deacetylase Inhibitor (HDACi) JNJ 26481585 in Combination With VELCADE (Bortezomib) and Dexamethasone for Patients With Relapsed Multiple Myeloma

Start: Sep 2011Est. completion: Nov 201318 patients
Phase 1Completed

A Study to Further Assess Safety and Effectiveness Data of the Bortezomib(Velcade)/Melphalan/Prednisone (BMP) Regimen in Previously Untreated and Transplant Ineligible Multiple Myeloma Patients

N/ANo Longer Available
NCT05259072Ionis Pharmaceuticals99m-technetium pyrophosphate scintigraphy

carDIo-ttranSSfOrm nucLEar Imaging Study

Start: Jan 2022Est. completion: Dec 20234 patients
N/ATerminated
NCT04456582PfizerShear wave elastography

Noninvasive Assessment of Myocardial Stiffness by 2D-SWE Ultrasound Technique (Bidimensional Shear Wave Elastography) in Patients With Transthyretin Amyloidosis

Start: Jun 2020Est. completion: May 202160 patients
N/AUnknown

Bortezomib (Velcade) - Regulatory Post Marketing Surveillance (PMS)

Start: Jun 2006Est. completion: Mar 20121,121 patients
N/ACompleted

An Observational Study of Chinese Multiple Myeloma Patients Treated With Velcade

Start: Mar 2006Est. completion: May 2010532 patients
N/ACompleted

Expanded Access Protocol (EAP) to Provide Bortezomib to Patients With Multiple Myeloma Who Have Received at Least 2 Previous Lines of Therapy and Are Refractory to or Have Relapsed After Their Last Therapy for Multiple Myeloma

Start: Jan 2004Est. completion: Oct 2006641 patients
N/ACompleted

Phase Legend

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

Key Insights

3 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 12,605 patients
8 companies competing in this space