Inqovi (decitabine/cedazuridine) / Otsuka - LARVOL DELTA

Testing the Addition of an Anti-cancer Drug, ASTX727 (Cedazuridine, Decitabine), to Chemotherapy (Paclitaxel) and Immunotherapy (Pembrolizumab) for Metastatic Triple-Negative Breast Cancer (clinicaltrials.gov) - P1 | N=32 | Suspended | Sponsor: National Cancer Institute (NCI) | Recruiting ➔ Suspended

Trial suspension • Breast Cancer • HER2 Breast Cancer • Hormone Receptor Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • ER • HER-2 • PD-L1 • PGR

Taiho Oncology to Share Data in Five Presentations, Including Two Oral Presentations, at the 2025 American Society of Clinical Oncology Annual Meeting (PRNewswire) - "The oral presentations will feature data regarding ASTX727 (oral decitabine-cedazuridine), an agent approved for myelodysplastic syndromes (MDS), paired with venetoclax for the treatment of acute myeloid leukemia (AML) and the novel investigational drug candidate zipalertinib for the treatment of non-small cell lung cancer (NSCLC) harboring EGFR exon 20 insertion mutations. The company will also share data on three real-world studies of two FDA-approved therapies: trifluridine and tipiracil paired with bevacizumab for the later-line treatment of adult patients with metastatic colorectal cancer and futibatinib for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma."

EGFR exon 20 • P2 data • Real-world • Acute Myelogenous Leukemia • Cholangiocarcinoma • Colorectal Cancer • Myelodysplastic Syndrome • Non Small Cell Lung Cancer

A phase I/Ib study of olaparib and ASTX727 in BRCA 1/2- and HRD-mutated tumors. (ASCO 2025) - P1 | "Clinical Trial Registration Number: NCT06177171 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

P1 data • Oncology • BRCA • BRCA1 • BRCA2

An all-oral regimen of decitabine-cedazuridine (DEC-C) plus venetoclax (VEN) in patients (pts) with newly diagnosed acute myeloid leukemia (AML) ineligible for intensive induction chemotherapy: Results from a phase 2 cohort of 101 pts. (ASCO 2025) - P1/2 | "Clinical Trial Registration Number: NCT04657081 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Clinical • P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Phase II trial of 10-day ASTX727 (decitabine/cedazuridine) in combination with venetoclax for relapsed or refractory acute myeloid leukemia. (ASCO 2025) - P2 | "Clinical Trial Registration Number: NCT04975919 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Combination therapy • P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

The phase II NIBIT-ML1 study of nivolumab plus ipilimumab and ASTX727 or nivolumab plus ipilimumab in PD-1 resistant metastatic melanoma: Tumor methylation landscape and correlation with clinical outcomes. (ASCO 2025) - P2 | "Clinical Trial Registration Number: NCT04250246 The abstract will be released to the public on June 1, 2025 at 8:00 AM EDT"

Clinical • Clinical data • Late-breaking abstract • Metastases • P2 data • Melanoma • Oncology • Solid Tumor • PD-1

Comparing New Treatments for People With Newly Diagnosed Acute Myeloid Leukemia That Has an IDH2 Gene Change (A MyeloMATCH Treatment Trial) (clinicaltrials.gov) - P2 | N=93 | Recruiting | Sponsor: National Cancer Institute (NCI) | Not yet recruiting ➔ Recruiting | Initiation date: Apr 2025 ➔ Dec 2025

Enrollment open • Trial initiation date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology

Testing the Addition of an IDH2 Inhibitor, Enasidenib, to Usual Treatment (Cedazuridine-Decitabine) for Higher-Risk Myelodysplastic Syndrome (MDS) With IDH2 Mutation (A MyeloMATCH Treatment Trial) (clinicaltrials.gov) - P2 | N=54 | Recruiting | Sponsor: National Cancer Institute (NCI) | Not yet recruiting ➔ Recruiting | Initiation date: Apr 2025 ➔ Dec 2025

Enrollment open • Trial initiation date • Hematological Malignancies • Multiple Myeloma • Myelodysplastic Syndrome • Oncology

Venetoclax and ASTX727 for the Treatment of Relapsed, Refractory, or Newly Diagnosed Acute Myeloid Leukemia (clinicaltrials.gov) - P2 | N=85 | Active, not recruiting | Sponsor: M.D. Anderson Cancer Center | Recruiting ➔ Active, not recruiting

Enrollment closed • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Oral decitabine cedazuridine with and without venetoclax in higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia: a propensity score-matched study. (PubMed, Blood Cancer J) - "The median overall survival was 24 and 19 months for the DEC-C-Ven and DEC-C cohorts, respectively (P = 0.89), and the median event-free survival durations were 18 and 10 months (P = 0.026). In conclusion, the addition of Ven resulted in improved response rates and outcomes in specific subgroups; prospective clinical trials are needed to confirm these findings."

Clinical • Journal • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Transplantation

Oral decitabine in acute myeloid leukemia: assessing efficacy, safety, and future implications for older patients. (PubMed, Expert Rev Hematol) - "For 20 years, hypomethylating agents (HMAs), as single agents and recently as a backbone with venetoclax, have been used in this setting. The oral combination of decitabine and cedazuridine (C-DEC), which is therapeutically and pharmacologically equivalent to the intravenous (IV) formulation (IV-DEC), has expanded the therapeutic arsenal for AML, allowing for better convenience of administration...To summarize the most recent developments as up-to-date as possible, we considered, with a few exceptions, only papers published over the last three years until March 2025. Compared to IV-DEC, C-DEC provides similar therapeutic exposure and efficacy with an optional safety profile, enhanced treatment adherence, reduced treatment burden, and required healthcare facilities compared to those associated with IV-DEC, providing a precious tool to manage older and unfit AML patients."

Journal • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

DEC-C and Thioguanine for R/R AML (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: Columbia University | N=48 ➔ 0 | Trial completion date: Dec 2027 ➔ Apr 2024 | Recruiting ➔ Withdrawn | Trial primary completion date: Dec 2027 ➔ Apr 2024

Enrollment change • Trial completion date • Trial primary completion date • Trial withdrawal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

KCP-8602-801: Study of the Safety, Tolerability and Efficacy of KPT-8602 in Participants with Relapsed/Refractory Cancer Indications (clinicaltrials.gov) - P1/2 | N=277 | Completed | Sponsor: Karyopharm Therapeutics Inc | Active, not recruiting ➔ Completed

Trial completion • Acute Myelogenous Leukemia • Castration-Resistant Prostate Cancer • Colon Adenocarcinoma • Colon Cancer • Colorectal Adenocarcinoma • Colorectal Cancer • Genito-urinary Cancer • Hematological Disorders • Hematological Malignancies • Leukemia • Multiple Myeloma • Myelodysplastic Syndrome • Oncology • Prostate Adenocarcinoma • Prostate Cancer • Solid Tumor • KRAS

Economic Burden of Intravenous Decitabine Administration in Patients Affected by Acute Myeloid Leukemia Ineligible for Induction Chemotherapy and Impact of Oral Formulation Introduction: A Micro-Costing Study in Italy. (PubMed, Clinicoecon Outcomes Res) - "Recently, the oral formulation of decitabine-cedazuridine demonstrated comparable efficacy and safety to IV decitabine, and pharmacokinetic equivalence...The first scenario estimated a total saving for the Italian NHS of € 1.09 million over three years, the second scenario estimated a potential additional impact of - € 3418.6/patient due to transfusions. The administration of oral versus IV decitabine is expected to generate cost savings for the Italian NHS in terms of drug administration, CVC and infection management, in patients with AML ineligible for induction chemotherapy."

HEOR • Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Oncology

Early Intervention in High Risk CCUS (clinicaltrials.gov) - P1 | N=108 | Recruiting | Sponsor: Maximilian Stahl, MD | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Oncology

A Phase 1B/2A Trial of Combination of ASTX727 With ASTX029 in Acute Myeloid Leukemia (clinicaltrials.gov) - P1/2 | N=0 | Withdrawn | Sponsor: M.D. Anderson Cancer Center | N=42 ➔ 0 | Trial completion date: May 2029 ➔ Mar 2025 | Suspended ➔ Withdrawn | Trial primary completion date: May 2027 ➔ Mar 2025

Enrollment change • Trial completion date • Trial primary completion date • Trial withdrawal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • KRAS • NF1 • PTPN11

“Grouped application…extension of indication to include treatment of adult patients with myelodysplastic syndromes…Extension of indication to include treatment of adult patients with chronic myelomonocytic leukemia (CMML) for INAQOVI based on final results from studies ASTX727-01, ASTX727-02, ASTX727-04... As a consequence, sections 4.1, 4.2, 4.8, 5.1 and 5.2 of the SmPc are updated" (European Medicines Agency) - CHMP Final Minutes of the meeting on 14- 17 Oct 2024: “The committee adopted a 2nd request for supplementary information with a specific timetable”

CHMP • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Oncology

A plain language summary of the ASCERTAIN trial: oral decitabine and cedazuridine versus intravenous decitabine for MDS or CMML. (PubMed, Future Oncol) - No abstract available

Journal • Chronic Myelomonocytic Leukemia

ASTX727-06: A Study for Participants Who Participated in Prior Clinical Studies of ASTX727 (Standard Dose) (clinicaltrials.gov) - P2 | N=332 | Enrolling by invitation | Sponsor: Taiho Oncology, Inc. | Trial completion date: Dec 2025 ➔ Dec 2027 | Trial primary completion date: Dec 2025 ➔ Dec 2027

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Entrectinib in Combination With ASTX727 for the Treatment of Relapsed/Refractory TP53 Mutated Acute Myeloid Leukemia (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: OHSU Knight Cancer Institute | Trial completion date: Jun 2025 ➔ Jun 2026 | Trial primary completion date: Dec 2024 ➔ Dec 2025

Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • MAPK1 • MAPK3 • NTRK • RUNX1 • TP53

Dose-Escalation Study of E7727, an Oral Cytidine Deaminase Inhibitor With Oral Decitabine in Subjects With Solid Tumors (clinicaltrials.gov) - P1 | N=35 | Recruiting | Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Active, not recruiting ➔ Recruiting | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Jun 2025 ➔ Jan 2026

Enrollment open • Trial completion date • Trial primary completion date • Oncology • Solid Tumor

ASTX660-03: A Study of Tolinapant in Combination With Oral Decitabine/Cedazuridine and Oral Decitabine/Cedazuridine Alone in Participants With Relapsed/Refractory Peripheral T-cell Lymphoma (R/R PTCL) (clinicaltrials.gov) - P1/2 | N=33 | Active, not recruiting | Sponsor: Taiho Oncology, Inc. | N=132 ➔ 33 | Trial primary completion date: Dec 2025 ➔ Dec 2024

Enrollment change • Trial primary completion date • Follicular Lymphoma • Hematological Malignancies • Hepatosplenic T-cell Lymphoma • Lymphoma • Natural Killer/T-cell Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma

Maintenance therapy with oral decitabine plus cedazuridine after allogeneic stem cell transplantation for myelodysplastic syndrome. (PubMed, Haematologica) - "Overall, patients achieved a median 2-year relapse-free survival (RFS) of 66.7% (95% CI, 40.4-83.4%) and 2-year overall survival of 72.2% (95% CI, 45.6%-87.4%), with relapses occurring predominantly in TP53- mutated cases. Prospective clinical trials are essential to confirm the best tolerated dose with potential to improve transplant outcomes."

Journal • Bone Marrow Transplantation • Hematological Malignancies • Infectious Disease • Myelodysplastic Syndrome • Oncology • Pneumonia • Respiratory Diseases • Transplantation • TP53

Decitabine/Cedazuridine and Enzalutamide for the Treatment of Metastatic Castrate Resistant Prostate Cancer (clinicaltrials.gov) - P1 | N=8 | Terminated | Sponsor: Roswell Park Cancer Institute | Trial completion date: Mar 2026 ➔ Jan 2025 | Active, not recruiting ➔ Terminated; PI closed study

Trial completion date • Trial termination • Castration-Resistant Prostate Cancer • Genito-urinary Cancer • Oncology • Prostate Cancer • Solid Tumor • RB1 • TP53

Roll-over Study for Patients Who Have Completed a Prior Novartis-sponsored Sabatolimab (MBG453) Study and Are Judged by the Investigator to Benefit From Continued Treatment With Sabatolimab. (clinicaltrials.gov) - P2 | N=33 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Nov 2025 ➔ Feb 2028 | Trial primary completion date: Nov 2025 ➔ Feb 2028

Trial completion date • Trial primary completion date • Chronic Myelomonocytic Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology