A Randomized Phase II Trial of Enasidenib-Based Therapies Versus Cedazuridine-Decitabine in Higher-Risk IDH2-Mutated Myelodysplastic Syndrome: A MyeloMATCH Treatment Trial

This phase II MyeloMATCH treatment trial compares the usual treatment of cedazuridine-decitabine (ASTX727) to the combination treatment of ASTX727 and enasidenib in treating patients with higher-risk, IDH2-mutated myelodysplastic syndrome (MDS). ASTX727 is a combination of two drugs, decitabine and cedazuridine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Enasidenib is an enzyme inhibitor that may stop the growth of cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with enasidenib may be effective in treating patients with higher-risk IDH2-mutated MDS.

Start Date27 Dec 2025

Sponsor / Collaborator

National Cancer Institute

NCT06672146

/ RecruitingPhase 2IIT

A Randomized Phase II Trial of ASTX727 and Venetoclax Compared With ASTX727, Venetoclax, and Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment trial studies how well ASTX727 and venetoclax plus enasidenib works compared to ASTX727 and venetoclax alone for the treatment of older patients with newly diagnosed acute myeloid leukemia (AML) or younger patients who are considered unfit for standard treatment, and who have an abnormal change (mutation) in the IDH2 gene. This gene mutation can cause AML to grow and spread. This trial is being done to see if adding enasidenib to the usual treatment can help more patients with the IDH2 gene get rid of AML. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Enasidenib works by stopping the growth and spread of tumor cells that have the IDH2 mutation. Giving ASTX727 and venetoclax plus enasidenib may work better in treating AML patients with the IDH2 mutation.

Start Date27 Dec 2025

Sponsor / Collaborator

National Cancer Institute

NCT06484062

/ SuspendedPhase 1IIT

A Phase I Study Evaluating the Safety of Cirtuvivint as Monotherapy and in Combination With ASTX727 and ASTX727 + Venetoclax in Patients With Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML)

This phase I trial tests the safety, side effects, and best dose of SM08502 (cirtuvivint) alone and in combination with ASTX727 in treating patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). Cirtuvivint may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. ASTX727 is a combination of two drugs, decitabine and cedazuridine. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Giving cirtuvivint alone or in combination with ASTX727 may be safe, tolerable, and/or effective in treating patients with AML and MDS.

Start Date16 Sep 2025

Sponsor / Collaborator

National Cancer Institute

100 Clinical Results associated with Decitabine/Cedazuridine

100 Translational Medicine associated with Decitabine/Cedazuridine

100 Patents (Medical) associated with Decitabine/Cedazuridine

Literatures (Medical) associated with Decitabine/Cedazuridine

01 Nov 2024·BRITISH JOURNAL OF HAEMATOLOGY

Oral decitabine/cedazuridine versus intravenous decitabine for acute myeloid leukaemia: A randomised, crossover, registration, pharmacokinetics study

Article

Author: Sano, Yuri ; Novák, Jan ; Illes, Arpad ; Santini, Valeria ; Keating, Mary‐Margaret ; Fracchiolla, Nicola Stefano ; Metzelder, Stephan K. ; Lübbert, Michael ; Lunghi, Monia ; Platzbecker, Uwe ; Rodríguez‐Macías, Gabriela ; Oganesian, Aram ; Arnan, Montserrat ; Mayer, Jiří ; Krauter, Jürgen ; del Castillo, Teresa Bernal ; Koristek, Zdenek ; Geissler, Klaus ; Keer, Harold

Summary:

This study compared decitabine exposure when administered IV (DEC‐IV) at a dose of 20 mg/m2 for 5‐days with orally administered decitabine with cedazuridine (DEC‐C), as well as the clinical efficacy and safety of DEC‐C in patients with acute myeloid leukaemia (AML) who were ineligible for intensive induction chemotherapy. In all, 89 patients were randomised 1:1 to DEC‐IV or oral DEC‐C (days 1–5 in a 28‐day treatment cycle), followed by 5 days of the other formulation in the next treatment cycle. All patients received oral DEC‐C for subsequent treatment cycles until treatment discontinuation. Equivalent systemic decitabine exposures were demonstrated (5‐day area under the curve ratio between the two decitabine formulations of 99.64 [90% confidence interval 91.23%, 108.80%]). Demethylation rates also were similar (≤1.1% difference). Median overall survival (OS), clinical response and safety profile with oral DEC‐C were consistent with those previously observed with DEC‐IV. Next‐generation sequencing was performed to identify molecular abnormalities that impact OS and TP53 mutations were associated with a poor outcome. These findings support the use of oral DEC‐C in patients with AML.

01 Sep 2024·Clinical Lymphoma Myeloma & Leukemia

Oral Decitabine/Cedazuridine Is an Effective Ambulatory Therapy for Patients With Myelofibrosis Refractory to JAK2 Inhibitor Therapy

Article

Author: Dueck, Amylou ; Ginzburg, Yelena ; Handa, Shivani ; Mascarenhas, John O ; Sivakumar, Ganesh ; Tremblay, Douglas ; Hoffman, Ronald ; Kremyanskaya, Marina ; Srisuwananukorn, Andrew

BACKGROUND:

Outcomes are dismal for patients with myelofibrosis (MF) who are no longer responsive to JAK2 inhibitors (JAKi) and/or have increasing blast cell numbers. Although prior reports have suggested the benefits of intravenous decitabine (DAC) combined with ruxolitinib for patients with Myeloproliferative Neoplasm (MPN) accelerated/blast phase (AP/BP), decitabine-cedazuridine (DEC-C), an oral fixed-dose combination providing equivalent pharmacokinetic exposure, has not been evaluated in MF.

METHODS:

We conducted a retrospective analysis of 14 patients with high-risk MF refractory to ruxolitinib or MPN-AP (10-19% blasts) treated with DEC-C +/- JAKi at Mount Sinai Hospital from 2021 to 2024.

RESULTS:

The cohort was elderly (median age,76 years) and almost uniformly possessed high risk mutations with 13 of the 14 patients progressing on JAKi therapy. With a median follow-up of 9.4 months, the median overall survival (OS) was 29 months for the entire cohort. Median OS was 10.8 months for MPN-AP and was not reached for ruxolitinib refractory MF patients. All patients (n = 9) receiving > 4 cycles of DEC-C had clinical benefit exemplified by a reduction in blast cell numbers, spleen size, and lack of progression to MPN-BP (78%). Furthermore, 3/14 patients proceeded to allogeneic stem cell transplant. Myelosuppression was a common adverse event which was managed by reducing the number of days of administration of DEC-C from 5 to 3 per cycle.

CONCLUSIONS:

This report demonstrates the feasibility, tolerability, and clinical benefit of an exclusively ambulatory regimen for high-risk, elderly patients with advanced MF which warrants further evaluation in a prospective clinical trial.

01 Apr 2024·The Lancet. Haematology

Oral decitabine and cedazuridine plus venetoclax for older or unfit patients with acute myeloid leukaemia: a phase 2 study

Article

Author: Garcia-Manero, Guillermo ; Kadia, Tapan ; Short, Nicholas ; Pierce, Sherry ; Alvarado, Yesid ; Bataller, Alex ; Masarova, Lucia ; Jain, Nitin ; Islam, Rabiul ; Konopleva, Marina ; Kornblau, Steven ; Ravandi, Farhad ; Montalban-Bravo, Guillermo ; Bazinet, Alexandre ; Maiti, Abhishek ; Issa, Ghayas ; Abuasab, Tareq ; DiNardo, Courtney D ; Daver, Naval ; Kantarjian, Hagop ; Yilmaz, Musa ; Huang, Xuelin ; Jabbour, Elias ; Montalbano, Kathryn ; Rausch, Caitlin R

BACKGROUND:

Hypomethylating agents combined with venetoclax are effective regimens in patients with acute myeloid leukaemia who are ineligible for intensive chemotherapy. Decitabine and cedazuridine (ASTX727) is an oral formulation of decitabine that achieves equivalent area-under-curve exposure to intravenous decitabine. We performed a single centre phase 2 study to evaluate the efficacy and safety of ASTX727 plus venetoclax.

METHODS:

This study enrolled patients with newly diagnosed (frontline treatment group) acute myeloid leukaemia who were ineligible for intensive chemotherapy (aged ≥75 years, an Eastern Cooperative Oncology Group [ECOG] performance status of 2-3, or major comorbidities) or relapsed or refractory acute myeloid leukaemia. Being aged 18 years or older and having an ECOG performance status of 2 or less were requirements for the relapsed or refractory disease treatment cohort, without any limits in the number of previous lines of therapy. Treatment consisted of ASTX727 (cedazuridine 100 mg and decitabine 35 mg) orally for 5 days and venetoclax 400 mg orally for 21-28 days in 28-day cycles. The primary outcome was overall response rate of ASTX727 plus venetoclax. Living patients who have not completed cycle one were not evaluable for response. Safety was analysed in all patients who started treatment. This study was registered on ClinicalTrials.gov (NCT04746235) and is ongoing. The data cutoff date for this analysis was Sept 22, 2023.

FINDINGS:

Between March 16, 2021, and Sept 18, 2023, 62 patients were enrolled (49 frontline and 13 relapsed or refractory) with a median age of 78 years (IQR 73-82). 36 (58%) were male; 53 (85%) were White, 4 (6%) Black, 2 (3%) Asian and 3 (5%) other or did not answer. 48 (77%) of 62 patients were European LeukemiaNet 2022 adverse risk, 24 (39%) had antecedent myelodysplastic syndromes, 12 (19%) had previously failed a hypomethylating agent, ten (16%) had therapy-related acute myeloid leukaemia, and 11 (18%) had TP53 mutations. The median follow-up time was 18·3 months (IQR 8·8-23·3). The overall response rate was 30 (64%) of 47 patients (95% CI 49-77) in frontline cohort and six (46%) of 13 patients (19-75) in relapsed or refractory cohort. The most common grade 3 or worse treatment-emergent adverse events were febrile neutropenia in 11 (18%) of 62 patients, pneumonia in eight (13%), respiratory failure in five (8%), bacteraemia in four (6%), and sepsis in four (6%). Three deaths occurred in patients in remission (one sepsis, one gastrointestinal haemorrhage, and one respiratory failure) and were potentially treatment related.

INTERPRETATION:

ASTX727 plus venetoclax is an active fully oral regimen and safe in most older or unfit patients with acute myeloid leukaemia. Our findings should be confirmed in larger multicentric studies.

FUNDING:

MD Anderson Cancer Center Support Grant, Myelodysplastic Syndrome/Acute Myeloid Leukaemia Moon Shot, Leukemia SPORE, Taiho Oncology, and Astex Pharmaceuticals.

News (Medical) associated with Decitabine/Cedazuridine

06 May 2025

·www.prnewswire.com

Actinium Announces Enrollment of First Patient in the Iomab-ACT Commercial CAR-T Trial at the University of Texas Southwestern Medical Center

- Initial clinical data expected in the second half of 2025 from this first-of-its-kind trial to administer a targeted radiotherapy conditioning agent with a commercial CAR-T therapy - Iomab-ACT supported by results of NIH funded trial with MSK showing effective lymphodepletion of targeted immune cells resulting in negligible rates of CAR-T toxicities ICANS and CRS and CAR T-cell persistence with a novel CD19 CAR-T therapy - Iomab-ACT has the potential to increase the addressable market for CAR-T therapies, which generated $4 billion in sales in 2024, by enabling improved access and better patient outcomes compared to current chemotherapy conditioning agents NEW YORK, May 6, 2025 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today announced that the first patient was enrolled on the trial studying Iomab-ACT targeted conditioning with a commercial CAR-T therapy at the University of Texas Southwestern Medical Center (UTSW) (NCT06768905). Initial clinical data from this trial is expected in the second half of 2025. Actinium is developing Iomab-ACT as a targeted radiotherapy conditioning agent intended to replace non-targeted chemotherapeutic conditioning agents such as Fludarabine and Cyclophosphamide (Flu/Cy) to address serious CAR-T related toxicities including immune effector cell-associated neurotoxicity (ICANS) and cytokine release syndrome (CRS), to potentially improve patient access and outcomes. Currently, there are seven CAR-T therapies approved for certain leukemias and lymphomas and multiple myeloma, that over 150,000 patients are diagnosed with annually. In 2024, the seven approved CAR-T therapies generated over $4 billion in sales and CAR-T therapies are forecasted to reach $12 billion in annual sales in 2030. Dr. Farrukh Awan, Professor of Medicine, Division of Hematology Oncology at UTSW said, "We are thrilled to initiate patient enrollment to study Iomab-ACT targeted radiotherapy conditioning with a commercial CAR-T therapy. Iomab-ACT is supported by compelling preclinical and clinical data, and we believe it has immense potential to eliminate the need for chemotherapy-based conditioning, which is a major barrier for many patients seeking CAR-T treatment. Despite the positive impact CAR-T therapy has had on patient outcomes, there is still significant room for improvement. We are optimistic that Iomab-ACT can transform CAR-T therapy conditioning if this trial demonstrates it has the ability to increase patients access and reduce the rates and severity of ICANS and CRS and also potentially improve patient outcomes. We are excited to begin treating patients with Iomab-ACT and eager to present our preliminary findings later this year." Iomab-ACT targets CD45, a cell surface marker expressed on immune cells relevant to CAR-T therapy including lymphocytes and is the only clinical stage conditioning agent targeting CD45. Preclinical data demonstrated that Iomab-ACT can selectively target immune cells implicated in CAR-T toxicities, while sparing bone marrow stem cells, red blood cells and platelets. Preclinical and clinical data also showed that Iomab-ACT produces transient lymphodepletion that aligns with the CAR-T treatment process. This data supported the first clinical trial of Iomab-ACT with a novel CD19 CAR-T therapy in collaboration with Memorial Sloan Kettering Cancer Center (MSK) in patients heavily pretreated with relapsed and refractory B-cell Acute Lymphoblastic Leukemia (B-ALL) or Diffuse Large B-cell Lymphoma (DLBCL). In this study, no patients (0/4) developed ICANS of any grade, and minimal CRS. Iomab-ACT also demonstrated transient depletion of peripheral blood lymphocytes and monocytes, persistence of CAR T-cells up to 8 weeks and minimal non-hematologic toxicities. These positive findings supported the continued advancement of Iomab-ACT and the initiation of the commercial CAR-T trial at UTSW. Sandesh Seth, Actinium's Chairman and CEO, stated, "This is a pivotal moment for our Iomab-ACT CD45 targeted radiotherapy conditioning program. Iomab-ACT is a highly differentiated conditioning agent that has produced promising initial clinical results where multiple targeted conditioning approaches including monoclonal antibodies and antibody drug conjugates directed against a variety of targets have not achieved clinical success to date. Based on the promising initial outcomes from the pilot study of Iomab-ACT with a novel CD19 CAR-T, we are incredibly excited by the potential of this commercial CAR-T trial and future development path. With initial clinical data expected beginning in the second half of this year, we are making strong progress to achieving our goal of establishing Iomab-ACT as a universal targeted conditioning regimen for CAR-T and other cellular therapies." Targeted Radiotherapy CAR-T Conditioning Opportunity A multi-billion-dollar market opportunity exists for better conditioning in other areas of cellular therapy, such as CAR-T. Currently, there are seven CAR T-cell therapies targeting CD19 for lymphoma and leukemia and BCMA for multiple myeloma that are approved by the FDA with total sales of over $4.0 billion in 2024. The pipeline of CAR-T therapies in development has rapidly expanded, with the addressable patient population expected to nearly double and reach approximately 93,000 patients in the U.S. by 2030 based on the current pipeline of cellular therapies. The addressable market for Iomab-ACT is in line with the patient population for cellular therapies that is approximately150,000 patients annually across the indications in which CAR-T therapies are approved, as all patients receive conditioning of some type. We believe a potential blockbuster revenue opportunity exists for Iomab-ACT assuming it can provide clinical benefits related to adverse events related to CAR-T, longer duration of response or improved survival outcomes. About Actinium Pharmaceuticals, Inc. Actinium is a pioneer in the development of targeted radiotherapies intended to meaningfully improve patient outcomes. Actinium is advancing its lead product candidate Actimab-A, a CD33 targeting therapeutic, as potential backbone therapy in acute myeloid leukemia (AML) and other myeloid malignancies leveraging the mutation agnostic alpha-emitter radioisotope payload Actinium-225 (Ac-225). Actimab-A has demonstrated potential activity in relapsed and refractory acute myeloid leukemia (r/r AML) patients in combination with the chemotherapy CLAG-M including high rates of Complete Remissions (CR) and measurable residual disease (MRD) negativity leading to improved survival outcomes and is being advanced to a pivotal Phase 2/3 trial. In addition, Actinium is engaged with the National Cancer Institute (NCI) under the Cooperative Research and Development Agreement (CRADA) for development of Actimab-A in AML and other myeloid malignancies. The first clinical trial under the CRADA will evaluate the triplet combination comprised of Actimab-A, Venetoclax (Abbvie/Roche) an oral Bcl-2 inhibitor and ASTX-727 (Taiho Oncology, an Otsuka holdings company) a novel oral hypomethylating agent (HMA) in frontline acute myeloid leukemia (AML) patients. Additionally, Actinium is developing Actimab-A as a potential pan tumor therapy in combination with PD-1 checkpoint inhibitors including KEYTRUDA® and OPDIVO® by depleting myeloid derived suppressor cells (MDSCs), which represents a potential multi-billion-dollar addressable market. ATNM-400 is Actinium's novel non-PSMA targeting Ac-225 radiotherapy for prostate cancer, which is supported by preclinical data demonstrating higher efficacy than Pluvicto (PSMA-617-Lutetium-177) and potent efficacy in Pluvicto resistant prostate cancer models. Iomab-ACT, Actinium's next generation conditioning candidate, is being developed with the goal of improving patient access and outcomes for potentially curative cell and gene therapies. Iomab-B is an induction and conditioning agent prior to bone marrow transplant in patients with r/r AML, which Actinium is seeking a potential strategic partner for the U.S. In addition, the company's R&D efforts are primarily focused on advancing several preclinical programs for solid tumor indications. Actinium holds 230 patents and patent applications including several patents related to the manufacture of the isotope Ac-225 in a cyclotron. For more information, please visit: Forward-Looking Statements This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time. Investors: [email protected] SOURCE Actinium Pharmaceuticals, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ImmunotherapyCell TherapyClinical Study

23 Apr 2025

·www.prnewswire.com

Taiho Oncology to Share Data in Five Presentations, Including Two Oral Presentations, at the 2025 American Society of Clinical Oncology Annual Meeting

Two oral presentations of phase 2 study findings from novel treatment options: an all-oral decitabine-cedazuridine combination for AML and zipalertinib for NSCLC that harbors exon 20 insertion mutations Additional presentations to include real-world data on the safety and effectiveness of two approved regimens: trifluridine-tipiracil paired with bevacizumab for metastatic colorectal cancer and futibatinib for cholangiocarcinoma PRINCETON, N.J., April 23, 2025 /PRNewswire/ -- Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, announced today that it will present new data from five studies, including two oral presentations, at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, to be held May 30 through June 3 at McCormick Place in Chicago. 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. "ASCO provides an invaluable opportunity to bring together the leading minds in oncology and discuss emerging new therapies and our understanding of real-world outcomes of existing therapies," said Harold Keer, MD, PhD, Chief Medical Officer, Taiho Oncology. "Our data at ASCO will continue to demonstrate our unwavering commitment to meaningful innovation that can potentially improve the lives of patients with cancer, their families and their caregivers." Details about these presentations can be found below: Oral Presentations Title: Efficacy of zipalertinib in NSCLC patients with EGFR exon 20 insertion mutations who received prior platinum-based chemotherapy with or without amivantamab Abstract Number: 8503 Session Name: Lung Cancer – Non-Small Cell Metastatic Session Type: Oral Abstract Session Session Date: June 1, 2025 Presentation Time: 8 a.m. – 11 a.m. CDT Location: Arie Crown Theater | Live Stream Presenter: Helena A. Yu, MD, Memorial Sloan Kettering Cancer Center Title: 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 Abstract Number: 6504 Session Name: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant Session Type: Oral Abstract Session Session Date: June 2, 2025 Presentation Time: 3 p.m. – 6 p.m. CDT Location: S100a | Live Stream Presenter: Amer Zeidan, MBBS, Yale Cancer Center Poster Presentation Title: Real-world treatment patterns and outcomes with trifluridine/tipiracil monotherapy or in combination with bevacizumab in metastatic colorectal cancer Abstract Number: 3580 Session Name: Gastrointestinal Cancer—Colorectal and Anal Session Type: Poster Presentation Session Date: May 31, 2025 Presentation Time: 9 a.m. – 12 p.m. CDT Location: Hall A - Posters and Exhibits | On Demand Presenter: Donald Richards, MD, PhD, of Texas Oncology Online Abstracts Title: Real-world Clinical Outcomes of Patients with Metastatic Colorectal Cancer (mCRC) Treated with Trifluridine-Tipiracil + Bevacizumab by Performance Status Abstract Number: E15606 Session Type: Publication Only Publication Date: May 22, 2025 Publication Time: 5 p.m. EDT Lead Author: Maliha Nusrat, MD, MS, Memorial Sloan Kettering Cancer Center Title: Real-World patient characteristics and treatment patterns among cholangiocarcinoma patients treated with FGFR inhibitors Abstract Number: E16262 Session Type: Publication Only Publication Date: May 22, 2025 Publication Time: 5 p.m. EDT Lead Author: Amit Mahipal, MD, University Hospitals Seidman Cancer Center and Case Comprehensive Cancer Center About Taiho Oncology, Inc. The mission of Taiho Oncology, Inc. is to improve the lives of patients with cancer, their families and their caregivers. The company specializes in the development and commercialization of orally administered anti-cancer agents for various tumor types. Taiho Oncology has a robust pipeline of small-molecule clinical candidates targeting solid-tumor and hematological malignancies, with additional candidates in pre-clinical development. Taiho Oncology is a subsidiary of Taiho Pharmaceutical Co., Ltd. which is part of Otsuka Holdings Co., Ltd. Taiho Oncology is headquartered in Princeton, New Jersey and oversees its parent company's European and Canadian operations, which are located in Baar, Switzerland and Oakville, Ontario, Canada. For more information, visit , and follow us on LinkedIn and X. Taiho Oncology and the Taiho Oncology logo are registered trademarks of Otsuka Holdings Co., Ltd. or its subsidiaries. Taiho Oncology Contact: Leigh Labrie (609) 664-9878 [email protected] SOURCE Taiho Oncology WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2ASCODrug ApprovalClinical Result

27 Mar 2025

·pipelinereview.com

Actinium Pharmaceuticals Announces ATNM-400 a Novel Non-PSMA Targeting First in Class Prostate Cancer Radiotherapy Leveraging Actinium-225

– 99.8% tumor growth inhibition achieved with a single dose of ATNM-400 in preclinical prostate cancer models – ATNM-400 accumulated in tumors for up to 144 hours and showed minimal uptake in healthy tissues in prostate cancer xenograft model – Initial ATNM-400 preclinical data to be presented at AACR including results in Pluvicto resistant prostate cancer models NEW YORK, NY, USA I March 27, 2025 I Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a pioneer in the development of targeted radiotherapies, today announced ATNM-400, a novel, non-PSMA targeting, first in class radiotherapy for prostate cancer utilizing the Actinium-225 (Ac-225) radioisotope. Initial preclinical data from ATNM-400 will be presented at the American Association for Cancer Research (AACR) Annual Meeting being held on April 25 – 30, 2025, in Chicago, IL. The ATNM-400 AACR abstract will include the results from in vitro and in vivo studies including biodistribution imaging and efficacy analyses with various dose levels of ATNM-400. Actinium continues to advance ATNM-400 with additional data expected from Pluvicto-resistant prostate cancer models at AACR. Pluvicto (Lu-177-PSMA-617) is a prostate-specific membrane antigen (PSMA) directed targeted radiotherapy that uses the beta-particle emitting radioisotope Lutetitium-177 (Lu-177) that is approved for patients with metastatic prostate cancer. Pluvicto is marketed and sold by Novartis and generated sales of $1.39 billion in 2024. ATNM-400 is differentiated from Pluvicto as it targets a different marker than PSMA that has been shown to be overexpressed in patients with prostate cancer and uses the alpha-particle emitter Ac-225, which is more potent than Lu-177 but has a shorter path length, which could result in fewer off-target effects. Sandesh Seth, Actinium’s Chairman and CEO, said, “The current era of radiotherapy is built on the clinical and commercial success of Pluvicto in prostate cancer. The field is now looking to address patients that do not respond or progress after Pluvicto therapy. We believe ATNM-400 can address this high unmet need and we are incredibly excited by our data to date. As anticipated, we have seen robust tumor control and ATNM-400 has shown to be well tolerated in preclinical studies, which we believe is due to the precise and potent cell-killing of Ac-225. We are also highly excited by the results of our biodistribution studies that showed selective tumor uptake with minimal uptake in normal tissues. By focusing on a non-PSMA target, we also believe ATNM-400 has the potential to address some of the toxicities reported with Pluvicto and other PSMA targeting radiotherapies such as xerostomia. We are eager to present our ATNM-400 data at AACR and to continue to advance this highly novel prostate cancer candidate.” Highlights from the abstract include: ATNM-400 AACR Presentation Details Title: ATNM-400 is a novel Actinium-225 antibody radioconjugate with strong efficacy in preclinical models of prostate cancer Abstract Number: 578 Session: PO.ET08.01 – Theranostics and Radiotheranostics Date & Time: April 27, 2025 – 2:00 pm – 5:00 pm About Actinium Pharmaceuticals, Inc. Actinium is a pioneer in the development of targeted radiotherapies intended to meaningfully improve patient outcomes. Actinium is advancing its lead product candidate Actimab-A, a CD33 targeting therapeutic, as potential backbone therapy in acute myeloid leukemia (AML) and other myeloid malignancies leveraging the mutation agnostic alpha-emitter radioisotope payload Actinium-225 (Ac-225). Actimab-A has demonstrated potential activity in relapsed and refractory acute myeloid leukemia (r/r AML) patients in combination with the chemotherapy CLAG-M including high rates of Complete Remissions (CR) and measurable residual disease (MRD) negativity leading to improved survival outcomes and is being advanced to a pivotal Phase 2/3 trial. In addition, Actinium is engaged with the National Cancer Institute (NCI) under the Cooperative Research and Development Agreement (CRADA) for development of Actimab-A in AML and other myeloid malignancies. The first clinical trial under the CRADA will evaluate the triplet combination comprised of Actimab-A, Venetoclax (Abbvie/Roche) an oral Bcl-2 inhibitor and ASTX-727 (Taiho Oncology, an Otsuka holdings company) a novel oral hypomethylating agent (HMA) in frontline acute myeloid leukemia (AML) patients. Additionally, Actinium is developing Actimab-A as a potential pan tumor therapy in combination with PD-1 checkpoint inhibitors including KEYTRUDA ® and OPDIVO ® by depleting myeloid derived suppressor cells (MDSCs), which represents a potential multi-billion-dollar addressable market. Iomab-ACT, Actinium’s next generation conditioning candidate, is being developed with the goal of improving patient access and outcomes for potentially curative cell and gene therapies. Iomab-B is an induction and conditioning agent prior to bone marrow transplant in patients with r/r AML, which Actinium is seeking a potential strategic partner for the U.S. ATNM-400 is Actinium’s novel non-PSMA targeting Ac-225 radiotherapy for prostate cancer, which is supported by preclinical data and is being advanced to clinical trials. In addition, the company’s R&D efforts are primarily focused on advancing several preclinical programs for solid tumor indications. Actinium holds 230 patents and patent applications including several patents related to the manufacture of the isotope Ac-225 in a cyclotron. For more information, please visit: https://www.actiniumpharma.com/ SOURCE: Actinium Pharmaceuticals

AACRClinical StudyRadiation Therapy

100 Deals associated with Decitabine/Cedazuridine

External Link

KEGG	Wiki	ATC	Drug Bank
-	Decitabine/Cedazuridine	L01XY	DB15694

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Acute Myeloid Leukemia	European Union	Otsuka Pharmaceutical Netherlands B.V.	15 Sep 2023
Acute Myeloid Leukemia	Iceland	Otsuka Pharmaceutical Netherlands B.V.	15 Sep 2023
Acute Myeloid Leukemia	Liechtenstein	Otsuka Pharmaceutical Netherlands B.V.	15 Sep 2023
Acute Myeloid Leukemia	Norway	Otsuka Pharmaceutical Netherlands B.V.	15 Sep 2023
Chronic Myelomonocytic Leukemia	Australia	Otsuka Australia Pharmaceutical Pty Ltd.	29 Oct 2020
Myelodysplastic Syndromes	United States	Taiho Oncology, Inc.	07 Jul 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Myelodysplastic-Myeloproliferative Diseases	Phase 2	United States	The University of Texas MD Anderson Cancer Center	31 Jan 2025
Myeloproliferative Disorders	Phase 2	United States	The University of Texas MD Anderson Cancer Center	31 Jan 2025
Acute Lymphoblastic Leukemia	Phase 2	United States	The University of Texas MD Anderson Cancer Center	01 Jul 2024
Acute Lymphoblastic Leukemia	Phase 2	United States	Astex Pharmaceuticals, Inc.	01 Jul 2024
Hematologic Neoplasms	Phase 2	United States	The University of Texas MD Anderson Cancer Center	01 Jul 2024
Hematologic Neoplasms	Phase 2	United States	Astex Pharmaceuticals, Inc.	01 Jul 2024
Hematopoietic stem cell transplantation	Phase 2	United States	The University of Texas MD Anderson Cancer Center	01 Jul 2024
Hematopoietic stem cell transplantation	Phase 2	United States	Astex Pharmaceuticals, Inc.	01 Jul 2024
Leukemia	Phase 2	United States	The University of Texas MD Anderson Cancer Center	01 Jul 2024
Leukemia	Phase 2	United States	Astex Pharmaceuticals, Inc.	01 Jul 2024

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05010772 (4277, ASH2024)	Phase 1	Acute Myeloid Leukemia complex cytogenetics \| antecedent MDS/MPN \| therapy-related AML ... View more	31	ASTX727 alone	fkubhtxexz(tmluqagvkv) = ifdmohfasd raijzquhmt (fsooawduwg ) View more	Positive	09 Dec 2024
				ASTX727 plus venetoclax	fkubhtxexz(tmluqagvkv) = jhgmnagvtm raijzquhmt (fsooawduwg ) View more
NCT04746235 (2896, ASH2024)	Phase 2	Acute Myeloid Leukemia TP53mutation \| FLT3-ITD \| N/KRASmutation	60	ASTX727 35/100 mg + Venetoclax 400 mg	fyetagrnvw(ienstuqojw) = aufnsulsqu npralwoxie (txgdhvrzua ) View more	Positive	08 Dec 2024
NCT05360160 (ASH2024) Manual	Phase 1/2	Acute Myeloid Leukemia NPM1 Mutation \| KMT2A Rearrangement \| NUP98 Rearrangement	26	Revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax (SAVE)	cupyczbyye(iybmmizkuo) = bicccnpjyr pzqjlfbkqb (efwoyasulf ) View more	Positive	07 Dec 2024
NCT05835011 (CTgov)	Phase 2	Myelodysplastic Syndromes	2	Decitabine/Cedazuridine+Magrolimab	pibkryacow = utthpiwulo ekamnfkhvl (tulszgsrms, edofmtgtde - vzbfhyowwi) View more	-	02 Oct 2024
NCT02103478 \| NCT03306264 (ASCERTAIN, EHA2024) Manual	Phase 2/3	Myelodysplastic Syndromes	180	DEC-CDEC-C (ASTX727)	dfwysasvjl(hbmufilrtc) = elprzdzkir gvopxzjcsy (kwfvnenpyw ) View more	Positive	14 May 2024
				DEC-Ccitabine	dfwysasvjl(hbmufilrtc) = lcrfjrxgtk gvopxzjcsy (kwfvnenpyw )
MYDAS-T MDS05 \| ES-3000 (EHA2024)	Phase 1/2	Myelodysplastic Syndromes	8	ES-3000 60mg TID	mmruitavgw(pmfqucdwqq) = One patient progressed and subsequently died of infective complications (colitis) attributable to progressive disease in cycle 2 lhyhzsqssn (qhqcxdjyim ) View more	-	14 May 2024
EHA2024	Not Applicable	Chronic Myelomonocytic Leukemia	50	Azacitidine	hfvaupfuro(utjtshzqcy) = cxdibndqjx tpzpsgbmeo (shtgvgfxbx ) View more	-	14 May 2024
				Decitabine-cedazuridine	hfvaupfuro(utjtshzqcy) = jdvelktgnx tpzpsgbmeo (shtgvgfxbx ) View more
NCT04746235 (Pubmed) Manual	Phase 2	Acute Myeloid Leukemia	62	ASTX727 (cevenetoclax 100 mg and decitabine 35 mg) plus venetoclax 400 mg	ekyahrejfh(owoytsawjp) = gvcjixhqaf odtrkvkaki (hjzcsibose ) View more	Positive	01 Apr 2024
				ASTX727 + venetoclax 400 mgvenetoclax 400 mg (newly diagnosed acute myeloid leukaemia)	ekyahrejfh(owoytsawjp) = kulxzsqiqn odtrkvkaki (hjzcsibose, 49 - 77)
NCT03306264 (ASCERTAIN, Pubmed 1 \| Pubmed 2 \| Lancet Haematol) Manual	Phase 3	Chronic Myelomonocytic Leukemia \| Myelodysplastic Syndromes	133	ASTX727	tbnmikszth(fbbomknuze) = fpqyoxngxv dguoemfjni (ehicefjtil, 92·66 - 105·60) View more	Positive	01 Jan 2024
				decitabine	cvnqffnlmc(mzgzoektbp) = ukatdjooqy zvwwirmvpv (hhdjkzrlha )
NCT04746235 (ASH2023)	Phase 2	Acute Myeloid Leukemia	52	ASTX727 (Decitabine/Cedazuridine) plus Venetoclax	rxtbsiotrz(zgvmaftuyw) = dpzxcitoke ubrgyingqb (uujeoxtomv ) View more	-	11 Dec 2023

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Chat with Hiro

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis