ASTX727

Phase 2Small molecule

ASTX727 is one of 159 small molecule programs targeting Malignant Peripheral Nerve Sheath Tumor. It is currently in Phase 2.

Indications

Total Trials

Active Trials

158

Competitors

Pipeline

Status by Indication[3][1]

Indication

Malignant Peripheral Nerve Sheath TumorRecruiting

Acute Myeloid LeukemiaWithdrawn

Myelodysplastic SyndromesActive, not recruiting

Hepatic ImpairmentRecruiting

Renal ImpairmentRecruiting

PembrolizumabRecruiting

TumourRecruiting

Neurological CancerRecruiting

Malignant Peripheral Nerve Sheath TumorPhase 2

Recruiting

Acute Myeloid LeukemiaPhase 2

Withdrawn

Myelodysplastic SyndromesPhase 1

Active, not recruiting

Hepatic ImpairmentPhase 1

Recruiting

Renal ImpairmentPhase 1

Recruiting

PembrolizumabPhase 1

Recruiting

TumourPhase 1

Recruiting

Neurological CancerPhase 1

Recruiting

Competition

Competitive Landscape

235 competitors in Acute Myeloid Leukemia

#DrugCompanyPhaseTrials

Novantrone

Pfizer

Phase 3

34 2

Xospata

Astellas

Phase 3

24 3

Fludara

Sanofi

Phase 3

18 4

Vepesid

AAvyxa

Phase 3

52 5

Cerubidine

HHikma Pharmaceuticals LLC

Phase 3

42 6

Mylotarg

WWyeth-Lederle Vaccines

Phase 3

38 7

Vanflyta

Daiichi Sankyo

Phase 3

16 8

Cerubidine

HHikma Pharmaceuticals LLC

Phase 3

32 9

Neupogen

EEastern Cooperative Oncology Group

Cellcept

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 1Recruiting

Renal Impairment

NCT04953897

Phase 1Recruiting

Hepatic Impairment

NCT04953910

PreclinicalRecruiting

Acute Myeloid Leukemia

NCT05360160

Q2 2027

Phase 2Recruiting

Malignant Peripheral Nerve Sheath Tumor

NCT04872543

Q4 2027

PreclinicalRecruiting

MDS/MPN

NCT04061421

Q1 2028

Phase 1Active, not recruiting

BRCA1 Mutation

NCT06177171

Clinical

Trials by Indication[1]

Acute Myeloid Leukemia5 trials

A Phase 1B/2A Trial of Combination of ASTX727 With ASTX029 in Acute Myeloid Leukemia

Withdrawn·MM.D. Anderson Cancer Center·Phase 1/2·NCT06113289

A Study of ASTX660 as a Single Agent and in Combination With ASTX727 in Subjects With Relapsed/Refractory Acute Myeloid Leukemia (AML)

Terminated·AAstex Pharmaceuticals, Inc.·Phase 1·NCT04155580·68 enrolled

A Phase I-II Study Investigating the All Oral Combination of the Menin Inhibitor SNDX-5613 With Decitabine/Cedazuridine (ASTX727) and Venetoclax in Acute Myeloid Leukemia (SAVE)

Recruiting·MM.D. Anderson Cancer Center·Preclinical·NCT05360160·43 enrolled

Show 2 more trials

A Phase Ia/Ib Open-label, Multiple Dose, Study to Determine the Recommended Dose, Evaluate PKs, PDs, Safety, and Activity of Venetoclax in Combination With Oral Decitabine/Cedazuridine (ASTX727) in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML)

Recruiting·MM.D. Anderson Cancer Center·Phase 1·NCT06191978·40 enrolled

An Open-Label, Multicenter, Extension Study for Subjects Who Participated in Prior Clinical Studies of ASTX727 (Standard Dose)

Enrolling by invitation· Taiho Oncology, Inc. logo

Taiho Oncology, Inc.·Phase 2·NCT04093570·332 enrolled

Renal Impairment1 trial

Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Renal Impairment

Recruiting· Taiho Oncology, Inc. logo

Taiho Oncology, Inc.·Phase 1·NCT04953897·18 enrolled

Tumour1 trial

Lutathera and ASTX727 in Neuroendocrine Tumours

Recruiting·IImperial College London·Phase 1·NCT05178693·27 enrolled

Myelodysplastic/Myeloproliferative Neoplasm1 trial

Phase I/II Study of a Combination of Decitabine and Cedazuridine (ASTX727) and ASTX029, an ERK Inhibitor, for Patients With RAS Pathway Mutant Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms

Withdrawn·MM.D. Anderson Cancer Center·Phase 1/2·NCT06284460

Hepatic Impairment1 trial

Study to Evaluate the Pharmacokinetics and Safety of Oral Decitabine and Cedazuridine in Cancer Patients With Hepatic Impairment

Recruiting· Taiho Oncology, Inc. logo

Taiho Oncology, Inc.·Phase 1·NCT04953910·27 enrolled

11 more indications

Breast Cancer1 trial

Study of ASTX727 Plus Talazoparib in Patients With Triple Negative or Hormone Resistant/HER2-negative Metastatic Breast Cancer

Completed·KKathy Miller·Phase 1·NCT04134884·34 enrolled

Relapsed/Refractory1 trial

Study of the Safety, Tolerability and Efficacy of KPT-8602 in Participants With Relapsed/Refractory Cancer Indications

Completed·KKaryopharm Therapeutics Inc·Phase 1/2·NCT02649790·277 enrolled

Pembrolizumab1 trial

Combination Study of Guadecitabine/ASTX727 and Pembrolizumab

Recruiting·RRoyal Marsden NHS Foundation Trust·Phase 1·NCT02998567·60 enrolled

CMML1 trial

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

Completed·AAstex Pharmaceuticals, Inc.·Phase 3·NCT03306264·227 enrolled

Malignant Peripheral Nerve Sheath Tumor1 trial

A Study of ASTX727 in People With Malignant Peripheral Nerve Sheath Tumors (MPNST)

Recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT04872543·25 enrolled

High Risk MDS or AML Patients1 trial

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

Completed·GGroupe Francophone des Myelodysplasies·Phase 2·NCT04857645·57 enrolled

Toxicity1 trial

A Phase I/II Trial to Assess the Efficacy and Toxicity of ASTX727 (Oral Decitabine/Cedazuridine) for the Treatment of Hematological Neoplasms After Allogeneic Stem Cell Transplantation

Withdrawn·MM.D. Anderson Cancer Center·Phase 2·NCT06297629

Myelodysplastic Syndromes1 trial

Phase 1 Trial of ASTX727 in Subjects With Lower-risk Myelodysplastic Syndromes

Active, not recruiting· Otsuka logo

Otsuka·Phase 1·NCT03906695·30 enrolled

MDS/MPN1 trial

The ABNL-MARRO 001 Study: A Phase 1/2 Study of Active Myeloid Target Compound Combinations in MDS/MPN Overlap Syndromes.

Recruiting·MMichael Savona·Preclinical·NCT04061421·94 enrolled

BRCA1 Mutation1 trial

A Phase I/Ib Study of Olaparib and ASTX727 in BRCA1/2- and HRD-mutated Tumors

Active, not recruiting·PPamela Munster·Phase 1·NCT06177171·13 enrolled

Neurological Cancer1 trial

Phase I Trial of ASTX727 in Recurrent/Progressive Non-enhancing IDH Mutant Gliomas

Recruiting·MMassachusetts General Hospital·Phase 1·NCT03922555·18 enrolled

Sources

References

[1]
ClinicalTrials.gov — 20 trial records for ASTX727. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — ASTX727 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for ASTX727. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Taiho Oncology, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: Taiho Oncology, Inc.

ASTX727

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q2 2027

Q4 2027

Q1 2028

Trials by Indication[1]

Acute Myeloid Leukemia5 trials

Renal Impairment1 trial

Tumour1 trial

Myelodysplastic/Myeloproliferative Neoplasm1 trial

Hepatic Impairment1 trial

Breast Cancer1 trial

Relapsed/Refractory1 trial

Pembrolizumab1 trial

CMML1 trial

Malignant Peripheral Nerve Sheath Tumor1 trial

High Risk MDS or AML Patients1 trial

Toxicity1 trial

Myelodysplastic Syndromes1 trial

MDS/MPN1 trial

BRCA1 Mutation1 trial

Neurological Cancer1 trial

References

Drug Info

Quick Facts

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