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MDS

8
Pipeline Programs
9
Companies
11
Clinical Trials
0
Approved Products

Pipeline by Development Stage

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

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Aprea Therapeutics
Aprea TherapeuticsPA - Doylestown
1 program
1
APR-246 + azacitidinePhase 31 trial
Active Trials
NCT03745716Completed154Est. Jan 2022
UNION therapeutics
UNION therapeuticsDenmark - Hellerup
2 programs
1
1
HetrombopagPhase 2/31 trial
Azacitidine or Ruxolitinib combined with SelinexorPhase 21 trial
Active Trials
NCT06664970Enrolling By Invitation39Est. Sep 2026
NCT05024877Unknown50Est. Dec 2023
Nordic Pharma
Nordic PharmaFrance - Paris
2 programs
1
lenalidomidePhase 21 trial
Individual Molecular MRD Monitoring for MDS Patients After Allo-SCTN/A1 trial
Active Trials
NCT02872662Completed308Est. Nov 2021
NCT00761449Completed28Est. Dec 2009
Astex Pharmaceuticals
Astex PharmaceuticalsUK - Cambridge
1 program
1
ASTX727Phase 21 trial
Active Trials
NCT04857645Completed57Est. Apr 2025
Otsuka
OtsukaJapan - Tokushima
1 program
1
BusulfanPhase 21 trial
Active Trials
NCT00796562Completed107Est. Nov 2016
Sandoz
SandozAustria - Kundl
3 programs
1
Azacitidine and LDE255Phase 11 trial
Non Interventional Study on Iron Toxicity After First Allo-transplant in MDS/CMMLN/A1 trial
transplantationN/A1 trial
Active Trials
NCT06267898Completed222Est. May 2015
NCT02757989Completed79Est. Jun 2024
NCT02323139Completed23Est. Aug 2018
Innovent Biologics
Innovent BiologicsChina - Jiangsu
1 program
1
IBI188Phase 11 trial
Active Trials
NCT04511975Suspended32Est. Apr 2022
Novartis
NovartisBASEL, Switzerland
3 programs
Non Interventional Study on Iron Toxicity After First Allo-transplant in MDS/CMMLN/A
transplantationN/A
Azacitidine and LDE255PHASE_1
Neovii Biotech
Neovii BiotechGermany - Grafelfing
1 program
transplantationN/A

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Aprea TherapeuticsAPR-246 + azacitidine
UNION therapeuticsHetrombopag
UNION therapeuticsAzacitidine or Ruxolitinib combined with Selinexor
Astex PharmaceuticalsASTX727
OtsukaBusulfan
Nordic Pharmalenalidomide
Innovent BiologicsIBI188
SandozAzacitidine and LDE255
Nordic PharmaIndividual Molecular MRD Monitoring for MDS Patients After Allo-SCT
Sandoztransplantation
SandozNon Interventional Study on Iron Toxicity After First Allo-transplant in MDS/CMML

Clinical Trials (11)

Total enrollment: 1,099 patients across 11 trials

NCT03745716Aprea TherapeuticsAPR-246 + azacitidine

APR-246 & Azacitidine for the Treatment of TP53 Mutant Myelodysplastic Syndromes (MDS)

Start: Jan 2019Est. completion: Jan 2022154 patients
Phase 3Completed
NCT05024877UNION therapeuticsHetrombopag

Hetrombopag for Low/Intermediate-1 Risk MDS With Thrombocytopenia

Start: Oct 2021Est. completion: Dec 202350 patients
Phase 2/3Unknown
NCT06664970UNION therapeuticsAzacitidine or Ruxolitinib combined with Selinexor

A Multicenter Study on the Treatment of MDS/MPN Overlap Syndrome With AZA or Rux Combined With Selinexor

Start: Nov 2024Est. completion: Sep 202639 patients
Phase 2Enrolling By Invitation
NCT04857645Astex PharmaceuticalsASTX727

ASTX727 and Donor Lymphocyte Infusions After Allogenic Stem Cell Transplantation in Very High Risk MDS or AML Patients

Start: Jun 2021Est. completion: Apr 202557 patients
Phase 2Completed
NCT00796562OtsukaBusulfan

Safety Study of Bone Marrow Transplant Using Mismatched Tissue Followed by Chemotherapy

Start: Dec 2008Est. completion: Nov 2016107 patients
Phase 2Completed
NCT00761449Nordic Pharmalenalidomide

Lenalidomide in High-risk MDS and AML With Del(5q) or Monosomy 5

Start: Oct 2007Est. completion: Dec 200928 patients
Phase 2Completed
NCT04511975Innovent BiologicsIBI188

A Study Evaluating the Safety and Efficacy of IBI188 in Combination With AZA in Subjects With Newly Diagnosed MDS

Start: Aug 2020Est. completion: Apr 202232 patients
Phase 1Suspended
NCT02323139SandozAzacitidine and LDE255

A Study of LDE255 in Combination With Azacitidine for High Risk Myelodysplastic Syndrome Patients

Start: Feb 2015Est. completion: Aug 201823 patients
Phase 1Completed
NCT02872662Nordic PharmaIndividual Molecular MRD Monitoring for MDS Patients After Allo-SCT

Individual Molecular MRD Monitoring for MDS Patients After Allo-SCT

Start: Aug 2016Est. completion: Nov 2021308 patients
N/ACompleted
NCT02757989Sandoztransplantation

Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome Low Risk

Start: May 2016Est. completion: Jun 202479 patients
N/ACompleted
NCT06267898SandozNon Interventional Study on Iron Toxicity After First Allo-transplant in MDS/CMML

Non Interventional Study on Iron Toxicity After First Allo-transplant in MDS/CMML

Start: Nov 2009Est. completion: May 2015222 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

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