Myelodysplastic Syndromes

Oncology
45
Pipeline Programs
27
Companies
49
Clinical Trials
7 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
13
5
20
1
4
2
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
571%
Monoclonal Antibody
114%
Vaccine
114%
+ 39 programs with unclassified modality

On Market (1)

Approved therapies currently available

U
AZACITIDINEApproved
azacitidine
Unknown Company
Nucleoside Metabolic Inhibitor [EPC]intravenous, subcutaneous2022

Competitive Landscape

26 companies ranked by most advanced pipeline stage

E
EisaiChina - Liaoning
1 program
1
decitabinePhase 41 trial
Active Trials
NCT01011283Terminated26
Astex Pharmaceuticals
Astex PharmaceuticalsUK - Cambridge
4 programs
1
1
2
ASTX727Phase 31 trial
GuadecitabinePhase 31 trial
Decitabine/CedazuridinePhase 21 trial
ASTX727 + Day 2 FoodPhase 11 trial
Active Trials
NCT03813186CompletedEst. Dec 2019
NCT05835011TerminatedEst. Aug 2023
NCT03306264CompletedEst. May 2023
+1 more trials
M&
Merck & Co.RAHWAY, NJ
2 programs
1
1
LuspaterceptPhase 35 trials
VORINOSTATPhase 1/2Small Molecule1 trial
Active Trials
NCT00776503Completed52Est. Nov 2011
NCT04143724Recruiting99Est. Jun 2035
NCT03682536Active Not Recruiting363Est. Sep 2027
+3 more trials
Pierre Fabre
Pierre FabreFrance - Aignan
1 program
1
Reduced Intensity ConditioningPhase 31 trial
Active Trials
NCT01203228TerminatedEst. Feb 2015
Taiho Oncology
Taiho OncologyNJ - Princeton
4 programs
1
2
1
AzacitidinePhase 2/31 trial
ASTX727 LDPhase 1/21 trial
DEC-CPhase 1/21 trial
InqoviPhase 11 trial
Active Trials
NCT04980404Active Not RecruitingEst. Nov 2026
NCT03502668CompletedEst. Jan 2026
NCT04742634RecruitingEst. Nov 2033
+1 more trials
Takeda
TakedaTOKYO, Japan
4 programs
3
AzacitidinePhase 21 trial
ElriterceptPhase 21 trial
TAK-226Phase 21 trial
PevonedistatN/ASmall Molecule1 trial
Active Trials
NCT04484363No Longer Available
NCT02610777Completed120Est. Jul 2021
NCT04419649Recruiting160Est. Oct 2031
+1 more trials
Boehringer Ingelheim
Boehringer IngelheimINGELHEIM, Germany
4 programs
3
1
Best Supportive CarePhase 21 trial
AzacitidinePhase 11 trial
AzacitidinePhase 11 trial
VolasertibPhase 1Small Molecule1 trial
Active Trials
NCT02201329Completed5Est. Sep 2015
NCT01957644Terminated16Est. Dec 2016
NCT02721875Terminated1Est. Jul 2016
+1 more trials
Roche
RocheSTAVANGER NORWAY, Norway
3 programs
1
2
LenalidomidePhase 21 trial
Mycophenolate mofetilPhase 21 trial
AtezolizumabPhase 1Monoclonal Antibody1 trial
Active Trials
NCT02508870Completed46Est. Jun 2019
NCT01718379Completed132Est. Jun 2016
NCT00551291Completed10Est. Jun 2009
One Biosciences
One BiosciencesFrance - Paris
3 programs
3
Eltrombopag/RevoladePhase 21 trial
Eltrombopag/RevoladePhase 21 trial
Luspatercept Injection [Reblozyl]Phase 21 trial
Active Trials
NCT02928419TerminatedEst. Sep 2018
NCT02912208Active Not RecruitingEst. Oct 2026
NCT05924100RecruitingEst. Dec 2029
Novartis
NovartisBASEL, Switzerland
2 programs
1
1
MBG453Phase 21 trial
GimatecanPhase 11 trial
Active Trials
NCT00493571Completed16Est. May 2014
NCT04823624Terminated10Est. Nov 2025
Teva
TevaIsrael - Petach Tikva
2 programs
1
1
Obatoclax mesylatePhase 21 trial
OmacetaxinePhase 1/21 trial
Active Trials
NCT02835794Withdrawn0Est. Dec 2018
NCT00413114Completed24Est. Nov 2009
Amgen
AmgenTHOUSAND OAKS, CA
1 program
1
AMG 531Phase 21 trial
Active Trials
NCT00418665Completed39Est. Oct 2010
argenx
argenxBelgium - Zwijnaarde
1 program
1
AzacitidinePhase 2
Curis
CurisMA - Lexington
1 program
1
CA-4948Phase 21 trial
Active Trials
NCT05178342TerminatedEst. Jul 2024
OD
1 program
1
Epoetin alfaPhase 2
Rigel Pharmaceuticals
Rigel PharmaceuticalsSOUTH SAN FRANCISCO, CA
1 program
1
OlutasidenibPhase 2Small Molecule1 trial
Active Trials
NCT06566742RecruitingEst. Aug 2029
Kura Oncology
Kura OncologySAN DIEGO, CA
1 program
1
TipifarnibPhase 2Small Molecule1 trial
Active Trials
NCT02779777Terminated16Est. Aug 2018
Providence Therapeutics
1 program
1
amifostine trihydratePhase 21 trial
Active Trials
Karyopharm Therapeutics
1 program
1
selinexorPhase 21 trial
Active Trials
NCT02228525CompletedEst. Apr 2021
J&
Johnson & JohnsonNEW BRUNSWICK, NJ
1 program
1
BortezomibPhase 1/21 trial
Active Trials
NCT00411905Completed45Est. Aug 2011
Jazz Pharmaceuticals
Jazz PharmaceuticalsCA - Palo Alto
2 programs
2
CPX-351Phase 11 trial
CPX-351Phase 11 trial
Active Trials
NCT03572764Active Not Recruiting20Est. Mar 2027
NCT03393611Completed14Est. Nov 2021
Pfizer
PfizerNEW YORK, NY
1 program
1
ARRY-614, p38/Tie2 inhibitor; oralPhase 11 trial
Active Trials
NCT01496495Completed71Est. Mar 2015
BioCity Biopharma
BioCity BiopharmaChina - Wuxi
1 program
1
BC3402Phase 11 trial
Active Trials
NCT05970822UnknownEst. Dec 2025
Alliance Pharmaceuticals
1 program
1
K562/GM-CSF cell vaccinePhase 1Vaccine1 trial
Active Trials
NCT00361296Terminated9Est. Jan 2010
Remix Therapeutics
Remix TherapeuticsMA - Watertown
1 program
1
REM-422Phase 11 trial
Active Trials
NCT06297941Recruiting100Est. Jun 2027
Vera Therapeutics
Vera TherapeuticsBRISBANE, CA
1 program
Peri-Transfusion QOL AssessmentN/A1 trial
Active Trials
NCT03660228CompletedEst. Jun 2021

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Eisaidecitabine
Merck & Co.Luspatercept
Astex PharmaceuticalsASTX727
Astex PharmaceuticalsGuadecitabine
Pierre FabreReduced Intensity Conditioning
Taiho OncologyAzacitidine
TakedaTAK-226
Rigel PharmaceuticalsOlutasidenib
Astex PharmaceuticalsDecitabine/Cedazuridine
One BiosciencesLuspatercept Injection [Reblozyl]
NovartisMBG453
CurisCA-4948
TakedaElritercept
Merck & Co.Luspatercept
Kura OncologyTipifarnib

Showing 15 of 48 trials with date data

Clinical Trials (49)

Total enrollment: 1,448 patients across 49 trials

To Demonstrate Superiority of Decitabine Over Azacitidine in Subjects With Intermediate- or High-risk MDS.

Start: Nov 200926 patients
Phase 4Terminated

A Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to IPSS-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Participants Who Require Red Blood Cell Transfusions and Are ESA Naïve

Start: Jan 2019Est. completion: Sep 2027363 patients
Phase 3Active Not Recruiting

Study of ASTX727 vs IV Decitabine in Participants With MDS, CMML, and AML

Start: Feb 2018Est. completion: May 2023
Phase 3Completed

Guadecitabine (SGI-110) vs Treatment Choice in Adults With MDS or CMML Previously Treated With HMAs

Start: Jan 2017Est. completion: Nov 2020
Phase 3Completed
NCT01203228Pierre FabreReduced Intensity Conditioning

Dose-reduced Versus Standard Conditioning in MDS/sAML

Start: May 2004Est. completion: Feb 2015
Phase 3Terminated

A Multi-phase Study of ASTX030 (Azacitidine and Cedazuridine) in Myeloid Neoplasm Alone or in Combination With Venetoclax in AML (AZTOUND Study)

Start: May 2020Est. completion: May 2028
Phase 2/3Recruiting

A Study of TAK-226 for Transfusion-Dependent Anemia in Japanese Patients With Lower-Risk Myelodysplastic Syndromes

Start: Feb 2026Est. completion: Jan 203327 patients
Phase 2Not Yet Recruiting

A Phase 2 Study Evaluating Olutasidenib in Patients With IDH1-mutated Clonal Cytopenia of Undetermined Significance and Lower-risk Myelodysplastic/Syndromes/Chronic Myelomonocytic Leukemia.

Start: Dec 2024Est. completion: Aug 2029
Phase 2Recruiting
NCT05835011Astex PharmaceuticalsDecitabine/Cedazuridine

A Study of Oral Decitabine/Cedazuridine in Combination With Magrolimab in Participants With Intermediate- to Very High-Risk Myelodysplastic Syndromes (MDS)

Start: Jun 2023Est. completion: Aug 2023
Phase 2Terminated
NCT05924100One BiosciencesLuspatercept Injection [Reblozyl]

Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to MDS With del5q, Refractory/Resistant/Intolerant to Prior Treatments, RBC-TD

Start: Nov 2022Est. completion: Dec 2029
Phase 2Recruiting

MBG453 in Lower Risk MDS

Start: Jan 2022Est. completion: Nov 202510 patients
Phase 2Terminated

Treatment of Anemia in Patients With Very Low, Low or Intermediate Risk Myelodysplastic Syndromes With CA-4948

Start: Jan 2022Est. completion: Jul 2024
Phase 2Terminated

A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)

Start: Aug 2020Est. completion: Oct 2031160 patients
Phase 2Recruiting

Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia

Start: Nov 2019Est. completion: Jun 203599 patients
Phase 2Recruiting

Tipifarnib in Subjects With Myelodysplastic Syndromes

Start: Jun 2016Est. completion: Aug 201816 patients
Phase 2Terminated

An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML)

Start: Apr 2016Est. completion: Jul 2021120 patients
Phase 2Completed
NCT02928419One BiosciencesEltrombopag/Revolade

Efficacy of Eltrombopag Plus Lenalidomide Combination Therapy in Patients With IPSS Low and Intermediate-risk Myelodysplastic Syndrome With Isolated del5q

Start: May 2015Est. completion: Sep 2018
Phase 2Terminated

Phase I/II Trial to Investigate BI 836858 in Myelodysplastic Syndromes

Start: Jan 2015Est. completion: Nov 201927 patients
Phase 2Terminated

Extension Study to Evaluate the Safety and Efficacy of Luspatercept in Participants With β-Thalassemia Previously Enrolled in A536-04 (A536-06/MK-6143-004)

Start: Oct 2014Est. completion: Jun 2020
Phase 2Completed

Extension Study to Evaluate Long-Term Effects of Luspatercept in Patients With Myelodysplastic Syndromes (MDS) (A536-05/MK-6143-003)

Start: Oct 2014Est. completion: Mar 2020
Phase 2Completed

Selective Inhibitor of Nuclear Export (SINE) Selinexor (KPT-330) in Patients With Myelodysplastic Syndromes

Start: Aug 2014Est. completion: Apr 2021
Phase 2Completed

Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)

Start: Feb 2013Est. completion: Nov 2015
Phase 2Completed

Study of Luspatercept for the Treatment of Anemia in Patients With Myelodysplastic Syndrome (MDS) (MK-6143-001)

Start: Jan 2013Est. completion: Oct 2018
Phase 2Completed
NCT02912208One BiosciencesEltrombopag/Revolade

Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes

Start: Jun 2011Est. completion: Oct 2026
Phase 2Active Not Recruiting
NCT01718379RocheLenalidomide

Lenalidomide in Subject With Low and Intermediate-1 Risk MDS and Without Chromosome 5 Abnormality.

Start: Jul 2010Est. completion: Jun 2016132 patients
Phase 2Completed
NCT00551291RocheMycophenolate mofetil

A Study of NeoRecormon (Epoetin Beta), CellCept (Mycophenolate Mofetil) and Prednisone in Patients With Low or Intermediate Myelodysplastic Syndromes.

Start: Aug 2007Est. completion: Jun 200910 patients
Phase 2Completed

A Safety and Efficacy Study to Evaluate AMG 531 Treatment in Subject With Myelodysplastic Syndrome Receiving Revlimid

Start: Dec 2006Est. completion: Oct 201039 patients
Phase 2Completed
NCT00413114TevaObatoclax mesylate

Safety and Efficacy of Obatoclax Mesylate (GX15-070MS)for the Treatment of Myelodysplastic Syndromes (MDS)

Start: Dec 2006Est. completion: Nov 200924 patients
Phase 2Completed

Amifostine in Treating Patients With Advanced Myelodysplastic Syndrome

Start: Aug 1997
Phase 2Unknown

Pre-emptive Therapy With DEC-C to Improve Outcomes in MDS Patients With Measurable Residual Disease Post Allogeneic Hematopoietic Cell Transplant

Start: May 2022Est. completion: Nov 2033
Phase 1/2Recruiting

Phase 1-2 Study of Low Dose ASTX727 (ASTX727 LD) in Lower Risk MDS

Start: Jul 2018Est. completion: Jan 2026
Phase 1/2Completed
NCT02835794TevaOmacetaxine

A Clinical Trial of Omacetaxine, Azacitidine, and Growth-Colony Stimulating Factor (G-CSF) for Myelodysplastic Syndromes (MDS)

Start: Aug 2016Est. completion: Dec 20180
Phase 1/2Withdrawn

Vorinostat and Low Dose Cytarabine for High Risk Myelodysplasia

Start: May 2008Est. completion: Nov 201152 patients
Phase 1/2Completed

Bortezomib and Low Dose Cytarabine in the Treatment of High-risk Myelodysplastic Syndromes

Start: Jun 2006Est. completion: Aug 201145 patients
Phase 1/2Completed

Study of REM-422 in Patients With AML or Higher Risk MDS

Start: Apr 2024Est. completion: Jun 2027100 patients
Phase 1Recruiting

Study of BC3402 in Combination With Azacitidine in Patients With MDS and CMML

Start: Jun 2023Est. completion: Dec 2025
Phase 1Unknown

Inqovi Maintenance Therapy in Myeloid Neoplasms

Start: Sep 2021Est. completion: Nov 2026
Phase 1Active Not Recruiting

CPX-351 (Vyxeos™) for Transplant Eligible, Higher Risk Patients With Myelodysplastic Syndrome

Start: Dec 2018Est. completion: Mar 202720 patients
Phase 1Active Not Recruiting

Effect of Food on Blood Levels of ASTX727

Start: Nov 2018Est. completion: Dec 2019
Phase 1Completed

Trial of Volasertib With or Without Azacitidine in Patients With Myelodysplastic Syndromes

Start: Apr 2016Est. completion: Jul 20161 patients
Phase 1Terminated
NCT02508870RocheAtezolizumab

A Study of Atezolizumab Administered Alone or in Combination With Azacitidine in Participants With Myelodysplastic Syndromes

Start: Sep 2015Est. completion: Jun 201946 patients
Phase 1Completed

Volasertib in Combination With Azacitidine in Japanese Patients With Myelodysplastic Syndrome or Chronic Myelomonocytic Leukemia

Start: Aug 2014Est. completion: Sep 20155 patients
Phase 1Completed

Phase I Dose Escalation Trial of Volasertib in Combination With Azacitidine in Patients With MDS or CMML

Start: Nov 2013Est. completion: Dec 201616 patients
Phase 1Terminated

CPX-351 Salvage Therapy Followed by Haplo-Cord Transplant for Relapsed/Refractory Leukemia or Myelodysplastic Syndrome

Start: Nov 2012Est. completion: Nov 202114 patients
Phase 1Completed
NCT01496495PfizerARRY-614, p38/Tie2 inhibitor; oral

A Study of ARRY-614 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes

Start: Jan 2012Est. completion: Mar 201571 patients
Phase 1Completed
NCT00361296Alliance PharmaceuticalsK562/GM-CSF cell vaccine

Vaccine Therapy in Treating Patients With Myelodysplastic Syndromes

Start: Sep 2007Est. completion: Jan 20109 patients
Phase 1Terminated

Phase I Study of Gimatecan in Patients With Myelodysplastic Syndromes

Start: Aug 2007Est. completion: May 201416 patients
Phase 1Completed
NCT04484363TakedaPevonedistat

Expanded Access Program With Pevonedistat (Given With Azacitidine) for Adults With Higher-risk Myelodysplastic Syndromes

N/ANo Longer Available
NCT03660228Vera TherapeuticsPeri-Transfusion QOL Assessment

Peri-Transfusion QOL Assessments (PTQA): A New Paradigm of Transfusion Decision Support for Patients With MDS

Start: Oct 2018Est. completion: Jun 2021
N/ACompleted

Related Jobs in Oncology

Phase Legend

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

Key Insights

4 late-stage (Phase 3) programs — potential near-term approvals
7 actively recruiting trials targeting 1,448 patients
27 companies competing in this space