AL Amyloidosis

Pipeline Programs

Companies

Clinical Trials

4 recruiting

Approved Products

Pipeline by Development Stage

Preclinical

Phase 1

Phase 1/2

Phase 2

Phase 2/3

Phase 3

On Market

Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Monoclonal Antibody

444%

ADC

444%

Bispecific Antibody

111%

+ 11 programs with unclassified modality

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Pre

P1/2

P2/3

Mkt

AstraZenecaCAMBRIDGE, United Kingdom

4 programs

CAEL-101Phase 31 trial

CAEL-101Phase 21 trial

Chimeric Fibril-Reactive Monoclonal Anti-body 11-1F4Phase 11 trial

Active Trials

NCT02245867Completed30Est. Jul 2017

NCT04304144Completed25Est. Nov 2023

NCT04504825Active Not Recruiting125Est. Oct 2027

+1 more trials

UNION therapeuticsDenmark - Hellerup

3 programs

TeclistamabPhase 2Monoclonal Antibody1 trial

A Registry of Chinese AL Amyloidosis Patients Treated With Subcutaneous or Intravenous DaratumumabN/AMonoclonal Antibody1 trial

Active Trials

NCT05277493Unknown500Est. Sep 2025

NCT07079423Recruiting35Est. Dec 2027

NCT06935162Recruiting30Est. Dec 2028

ViiV HealthcareNC - Durham

2 programs

Belantamab mafodotinPhase 2ADC

Belantamab mafodotin 2.5 mg/kgPhase 1/2ADC

ProthenaDUBLIN 2, Ireland

4 programs

non-interventionalN/A1 trial

NEOD001PHASE_21 trial

Active Trials

NCT02574676Completed343Est. Jul 2018

NCT02632786Completed129Est. Mar 2018

NCT02613182Terminated34Est. May 2018

+1 more trials

GSKLONDON, United Kingdom

2 programs

Belantamab mafodotin 2.5 mg/kgPHASE_1_2ADC1 trial

Belantamab mafodotinPHASE_2ADC1 trial

Active Trials

NCT05145816Recruiting37Est. Sep 2026

NCT04617925Completed35Est. Jul 2025

Bristol Myers SquibbPRINCETON, NJ

2 programs

PomalidomidePHASE_1_21 trial

IsatuximabPHASE_2Monoclonal Antibody1 trial

Active Trials

NCT01570387Completed27Est. Apr 2019

NCT05066607Unknown46Est. Mar 2026

Keymed BiosciencesChina - Chengdu

1 program

CM-336 BCMA/CD3 bispecific antibodyN/ABispecific Antibody1 trial

Active Trials

NCT06769555Withdrawn0Est. Aug 2028

OncopeptidesSweden - Stockholm

1 program

Melphalan-FlufenamidePHASE_11 trial

Active Trials

NCT04115956Terminated6Est. Jan 2022

GenentechCA - Oceanside

1 program

Venetoclax Oral Tablet, 200 mgPHASE_1_21 trial

Active Trials

NCT05451771Recruiting53Est. Sep 2026

Trial Timeline

Clinical trial activity over time

2021

2022

2023

2024

2025

2026

2027

2028

AstraZenecaCAEL-101

UNION therapeuticsTeclistamab

Bristol Myers SquibbIsatuximab

GSKBelantamab mafodotin

AstraZenecaCAEL-101

ProthenaNEOD001

GSKBelantamab mafodotin 2.5 mg/kg

GenentechVenetoclax Oral Tablet, 200 mg

Bristol Myers SquibbPomalidomide

OncopeptidesMelphalan-Flufenamide

AstraZenecaChimeric Fibril-Reactive Monoclonal Anti-body 11-1F4

Showing 15 of 18 trials with date data

Clinical Trials (18)

Total enrollment: 1,816 patients across 18 trials

NCT04512235 AstraZenecaCAEL-101

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)

Start: Nov 2020Est. completion: Apr 2027281 patients

Phase 3Active Not Recruiting

NCT04504825 AstraZenecaCAEL-101

A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)

Start: Aug 2020Est. completion: Oct 2027125 patients

Phase 3Active Not Recruiting

NCT07079423 UNION therapeuticsTeclistamab

Teclistamab in Newly Diagnosed Mayo Stage IIIB AL Amyloidosis

Start: Jun 2025Est. completion: Dec 202735 patients

Phase 2Recruiting

NCT06935162 UNION therapeuticsTeclistamab

Teclistamab in Previously Treated AL Amyloidosis

Start: May 2025Est. completion: Dec 202830 patients

Phase 2Recruiting

NCT05066607 Bristol Myers SquibbIsatuximab

Isatuximab Plus Pomalidomide and Dexamethasone Association for Patients With AL Amyloidosis Not in VGPR or Better After Any Previous Therapy

Start: Feb 2022Est. completion: Mar 202646 patients

Phase 2Unknown

NCT04617925 GSKBelantamab mafodotin

A Study of Belantamab Mafodotin in Patients With Relapsed or Refractory AL Amyloidosis

Start: Feb 2021Est. completion: Jul 202535 patients

Phase 2Completed

NCT04304144 AstraZenecaCAEL-101

A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis

Start: Mar 2020Est. completion: Nov 202325 patients

Phase 2Completed

NCT03154047 ProthenaNEOD001

Study in Subjects With Light Chain (AL) Amyloidosis

Start: Jun 2017Est. completion: May 201880 patients

Phase 2Terminated

NCT02632786 ProthenaNEOD001

The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis

Start: Mar 2016Est. completion: Mar 2018129 patients

Phase 2Completed

NCT02613182 ProthenaNEOD001

Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis

Start: Feb 2016Est. completion: May 201834 patients

Phase 2Terminated

NCT05145816 GSKBelantamab mafodotin 2.5 mg/kg

Phase 1/2a Study of Belantamab Mafodotin in Relapsed or Refractory AL Amyloidosis

Start: Feb 2024Est. completion: Sep 202637 patients

Phase 1/2Recruiting

NCT05451771 GenentechVenetoclax Oral Tablet, 200 mg

Venetoclax-Dexamethasone in Relapsed and/or Refractory t(11;14) Amyloidosis

Start: Oct 2022Est. completion: Sep 202653 patients

Phase 1/2Recruiting

NCT01570387 Bristol Myers SquibbPomalidomide

A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis

Start: Jun 2012Est. completion: Apr 201927 patients

Phase 1/2Completed

NCT04115956 OncopeptidesMelphalan-Flufenamide

A Clinical Study of Melphalan Flufenamide (Melflufen) and Dexamethasone for Patients With Immunoglobulin Light Chain (AL) Amyloidosis

Start: Aug 2020Est. completion: Jan 20226 patients

Phase 1Terminated

NCT02245867 AstraZenecaChimeric Fibril-Reactive Monoclonal Anti-body 11-1F4

Study of Chimeric Fibril-Reactive Monoclonal Antibody 11-1F4 in Patients With AL Amyloidosis

Start: Oct 2014Est. completion: Jul 201730 patients

Phase 1Completed

NCT06769555 Keymed BiosciencesCM-336 BCMA/CD3 bispecific antibody

BCMA/CD3 BiTE for RRAL or NDAL Amyloidosis With Insufficient Depth of Hematologic Response After Induction Therapy

Start: Jan 2025Est. completion: Aug 20280

N/AWithdrawn

NCT05277493 UNION therapeuticsA Registry of Chinese AL Amyloidosis Patients Treated With Subcutaneous or Intravenous Daratumumab

A Registry of Chinese AL Amyloidosis Patients Treated With Subcutaneous or Intravenous Daratumumab

Start: Mar 2022Est. completion: Sep 2025500 patients

N/AUnknown

NCT02574676 Prothenanon-interventional

Quality of Life (QOL) Registry for Patients With AL Amyloidosis

Start: Oct 2015Est. completion: Jul 2018343 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

2 late-stage (Phase 3) programs, potential near-term approvals

4 actively recruiting trials targeting 1,816 patients

9 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.