Myelodysplastic Syndrome

Oncology
21
Pipeline Programs
12
Companies
21
Clinical Trials
3 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
4
2
11
0
3
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
3100%
+ 18 programs with unclassified modality

On Market (1)

Approved therapies currently available

U
DECITABINEApproved
decitabine
Unknown Company
Nucleoside Metabolic Inhibitor [EPC]intravenous2020

Competitive Landscape

12 companies ranked by most advanced pipeline stage

Astex Pharmaceuticals
Astex PharmaceuticalsUK - Cambridge
2 programs
1
1
decitabinePhase 31 trial
ASTX727 Dose EscalationPhase 1/21 trial
Active Trials
NCT02103478CompletedEst. Dec 2019
NCT00043381CompletedEst. Apr 2003
Amgen
AmgenTHOUSAND OAKS, CA
1 program
1
Darbepoetin alphaPhase 31 trial
Active Trials
NCT01196715Completed360Est. Oct 2015
Takeda
TakedaTOKYO, Japan
1 program
1
ElriterceptPhase 31 trial
Active Trials
NCT07422480Recruiting300Est. Oct 2033
E
EisaiChina - Liaoning
8 programs
2
6
DecitabinePhase 21 trial
DecitabinePhase 21 trial
DecitabinePhase 21 trial
DecitabinePhase 21 trial
Decitabine, Arsenic Trioxide and Ascorbic AcidPhase 21 trial
+3 more programs
Active Trials
NCT00282399Terminated41Est. Feb 2009
NCT00941109Completed12Est. Sep 2012
NCT00067808Completed128Est. May 2009
+5 more trials
Genentech
GenentechCA - Oceanside
2 programs
2
ErlotinibPhase 2Small Molecule1 trial
ErlotinibPhase 2Small Molecule1 trial
Active Trials
NCT00570375Withdrawn0Est. Apr 2009
NCT00977548Completed39Est. Jun 2012
M&
Merck & Co.RAHWAY, NJ
1 program
1
Azacitidine and oral vorinostatPhase 2Small Molecule1 trial
Active Trials
NCT01748240Terminated21Est. Jul 2015
Sanofi
SanofiPARIS, France
1 program
1
ClofarabinePhase 21 trial
Active Trials
NCT00299156Completed65Est. Dec 2012
Karyopharm Therapeutics
1 program
1
SelinexorPhase 21 trial
Active Trials
NCT02431351WithdrawnEst. May 2019
Antengene
AntengeneChina - Shanghai
1 program
1
ATG-016Phase 1/21 trial
Active Trials
NCT04691141TerminatedEst. Sep 2023
PMV Pharma
PMV PharmaPRINCETON, NJ
1 program
1
AzacitidinePhase 11 trial
Active Trials
NCT06616636RecruitingEst. Aug 2029
Pfizer
PfizerNEW YORK, NY
1 program
1
SEA-CD70Phase 11 trial
Active Trials
NCT04227847Recruiting178Est. Jul 2028
Novartis
NovartisBASEL, Switzerland
1 program
SamplesN/A1 trial
Active Trials
NCT02809222Completed138Est. Mar 2021

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
TakedaElritercept
AmgenDarbepoetin alpha
Astex Pharmaceuticalsdecitabine
Karyopharm TherapeuticsSelinexor
Merck & Co.Azacitidine and oral vorinostat
GenentechErlotinib
Eisaidecitabine
GenentechErlotinib
EisaiDecitabine, Arsenic Trioxide and Ascorbic Acid
EisaiDecitabine
SanofiClofarabine
EisaiDecitabine
EisaiDecitabine
EisaiDecitabine
AntengeneATG-016

Showing 15 of 21 trials with date data

Clinical Trials (21)

Total enrollment: 1,624 patients across 21 trials

NCT07422480TakedaElritercept

A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS) Who Need Regular Blood Transfusions

Start: Apr 2026Est. completion: Oct 2033300 patients
Phase 3Recruiting
NCT01196715AmgenDarbepoetin alpha

Comparison Study of Standard Care Against Combination of Growth Factors Agents for Low-risk Myelodysplastic Syndromes

Start: Nov 2010Est. completion: Oct 2015360 patients
Phase 3Completed
NCT00043381Astex Pharmaceuticalsdecitabine

Decitabine Versus Supportive Care in Adults With Advanced-stage MDS

Start: Apr 2001Est. completion: Apr 2003
Phase 3Completed
NCT02431351Karyopharm TherapeuticsSelinexor

Efficacy & Safety Study of KPT-330 in Erythropoietin-Refractory Lower-Risk Myelodysplastic Syndrome Patients

Start: Nov 2015Est. completion: May 2019
Phase 2Withdrawn
NCT01748240Merck & Co.Azacitidine and oral vorinostat

Addition of Vorinostat to Azacitidine in Higher Risk MDS a Phase II add-on Study in Patients With Azacitidine Failure

Start: Mar 2013Est. completion: Jul 201521 patients
Phase 2Terminated
NCT00977548GenentechErlotinib

Erlotinib Study for Myelodysplastic Syndrome (MDS)

Start: Sep 2009Est. completion: Jun 201239 patients
Phase 2Completed
NCT00619099Eisaidecitabine

A Study of Decitabine Given Subcutaneously to Adults With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)

Start: May 200867 patients
Phase 2Completed
NCT00570375GenentechErlotinib

The Role of Erlotinib an Epidermal Growth Factor Receptor (EGFR) Inhibitor in the Treatment of Myelodysplastic Syndrome

Start: Nov 2007Est. completion: Apr 20090
Phase 2Withdrawn
NCT00621023EisaiDecitabine, Arsenic Trioxide and Ascorbic Acid

Cephalon Decitabine, Arsenic Trioxide and Ascorbic Acid for Myelodysplastic Syndrome

Start: Nov 2007Est. completion: Apr 20107 patients
Phase 2Completed
NCT00414310EisaiDecitabine

Decitabine (DAC) w/ or w/o Valproic Acid (VPA) in Myelodysplastic Syndrome (MDS) and Acute Myelogenous Leukemia (AML)

Start: Dec 2006Est. completion: May 2015153 patients
Phase 2Completed
NCT00299156SanofiClofarabine

Oral Clofarabine Study in Patients With Myelodysplastic Syndrome

Start: Mar 2006Est. completion: Dec 201265 patients
Phase 2Completed
NCT00260065EisaiDecitabine

A Study of Decitabine Given to Adults With Advanced-Stage Myelodysplastic Syndromes

Start: May 2005Est. completion: Dec 200899 patients
Phase 2Completed
NCT00113321EisaiDecitabine

Low-Dose Decitabine in Myelodysplastic Syndrome Post Azacytidine Failure

Start: Mar 2005Est. completion: Nov 200816 patients
Phase 2Terminated
NCT00067808EisaiDecitabine

Study of Three Different Schedules of Low-Dose Decitabine in Myelodysplastic Syndrome (MDS)

Start: Oct 2003Est. completion: May 2009128 patients
Phase 2Completed
NCT04691141AntengeneATG-016

A Study to Evaluating the Pharmacokinetics, Safety, and Efficacy of ATG 016 Monotherapy in IPSS-R Intermediate Risk and Above Myelodysplastic Syndrome (MDS)

Start: Feb 2021Est. completion: Sep 2023
Phase 1/2Terminated
NCT02103478Astex PharmaceuticalsASTX727 Dose Escalation

Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral Cytidine Deaminase Inhibitor (CDAi) in Patients With Myelodysplastic Syndromes (MDS)

Start: Oct 2014Est. completion: Dec 2019
Phase 1/2Completed
NCT06616636PMV PharmaAzacitidine

A Phase Ib Study of Rezatapopt in Combination With Azacitidine in Patients With TP53Y220C Mutant Myeloid Malignancies (Acute Myeloid Leukemia or Myelodysplastic Syndrome)

Start: Jan 2025Est. completion: Aug 2029
Phase 1Recruiting
NCT04227847PfizerSEA-CD70

A Safety Study of SEA-CD70 in Patients With Myeloid Malignancies

Start: Aug 2020Est. completion: Jul 2028178 patients
Phase 1Recruiting
NCT00941109Eisaidecitabine

Escalation Study to Determine Bioavailability of a Single Oral Dose of Decitabine in Patients With Myelodysplastic Syndrome (MDS)

Start: Dec 2009Est. completion: Sep 201212 patients
Phase 1Completed
NCT00282399EisaiSubcutaneous Decitabine

A Clinical Trial of Decitabine in Patients With Myelodysplastic Syndrome

Start: Oct 2006Est. completion: Feb 200941 patients
Phase 1Terminated
NCT02809222NovartisSamples

Plasmatic L-AScorbic Acid in MYelodyplastic Syndroms and Controls

Start: Oct 2016Est. completion: Mar 2021138 patients
N/ACompleted

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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

3 late-stage (Phase 3) programs — potential near-term approvals
3 actively recruiting trials targeting 1,624 patients
12 companies competing in this space

Related Indications

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