A Phase 1 Study of Oral Cedazuridine and Decitabine Combination (ASTX727, NSC# 820631) and Filgrastim as Maintenance Therapy Post-Hematopoietic Stem Cell Transplant in Children With High-Risk Acute Myeloid Leukemia

This phase I trial tests the safety, side effects, and best dose of ASTX727 and filgrastim for the treatment of children with high risk acute myeloid leukemia that has come back after a period of improvement (recurrent) or that does not respond to treatment (refractory) who have undergone allogenic hematopoietic stem cell transplantation. ASTX727 is a combination of cedazuridine and decitabine. 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. Filgrastim stimulates the production of neutrophils (a type of white blood cell) which can help to prevent infection. Giving ATSX727 and filgrastim may be safe and tolerable in treating children with high risk, recurrent or refractory acute myeloid leukemia who have undergone allogenic hematopoietic stem cell transplantation.

Start Date30 Oct 2026

Sponsor / Collaborator

National Cancer Institute

NCT07437950

/ Not yet recruitingPhase 2IIT

A Randomized Phase II Trial of ASTX727 With Standard Duration Versus Shorter Duration of Venetoclax in Genomically Heterogenous AML Among Adults Aged 60 or Older and Less Fit for Intensive Therapy: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment trial compares ASTX727 with standard duration versus shorter duration of venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML). ASTX727 is a combination of 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. 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. Shorter duration venetoclax may be as effective as standard duration venetoclax when given with ASTX727 for the treatment of newly diagnosed AML.

Start Date14 Oct 2026

Sponsor / Collaborator

National Cancer Institute

NCT07153497

/ Not yet recruitingPhase 2IIT

A Randomized Phase 2 Trial of Olutasidenib-Based Therapies in Patients With Newly Diagnosed IDH1-Mutant Myeloid Malignancies: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment substudy tests the addition of olutasidenib to usual treatment in patients with higher-risk myelodysplastic syndrome (MDS) or patients with acute myeloid leukemia (AML) with a mutation in the IDH1 gene. Olutasidenib blocks the protein made by the mutated IDH1 gene. Blocking this protein may help keep cancer cells from growing. For patients with MDS, olutasidenib will be added to decitabine-cedazuridine (also called ASTX727). Decitabine is in a class of medications called hypomethylating agents and is the standard treatment for MDS. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. The cedazuridine makes it possible to take the decitabine by mouth. Adding olutasidenib to the usual treatment for MDS (ASTX727) may increase the likelihood of going into remission. For patients with AML, olutasidenib and ASTX727 will be combined with venetoclax, a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. Venetoclax may stop the growth of cancer cells by blocking BCL-2, a protein needed for cancer cell survival. Adding olutasidenib to the usual treatment for AML (ASTX727 and venetoclax) may increase the likelihood of going into remission. For low risk MDS, the substudy tests whether giving olutasidenib alone helps improve blood counts.

Start Date27 May 2026

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

10 Feb 2026·Blood Advances

Decitabine-cedazuridine in patients with MDS and TP53 mutations

Article

Author: Urrutia, Samuel ; Griffiths, Elizabeth A. ; Garcia-Manero, Guillermo ; Sano, Yuri ; Savona, Michael ; McCloskey, James ; Bataller, Alex ; Sasaki, Koji ; Keer, Harold N. ; Oganesian, Aram ; Montalban-Bravo, Guillermo ; Kantarjian, Hagop ; Yee, Karen ; Zeidan, Amer

Patients with myelodysplastic syndromes (MDS) harboring TP53 mutations have poor outcomes. The objective of this study is to evaluate patients with TP53 mutations treated in the phase 2/3 studies of decitabine-cedazuridine (DEC-C) in MDS. We divided patients into three groups: TP53wt, TP53single-hit, and TP53multi-hit. We then performed propensity matching of patients who were treated with DEC-C vs a cohort of patients treated with parenteral hypomethylating agents (HMA). 180 patients were analyzed of which 73 (40.5%) had TP53 mutations, 23 (12%) with TP53multi-hit. Patients with TP53multi-hit more frequently had complex cytogenetics (69.5%), and had fewer median number of co-mutations (2.5, IQR 2-3) compared to TP53single-hit(3, IQR 1-5), or TP53wt (4, IQR 3-6) (p=0.002). Patients with TP53multi-hit had a higher chance of lack of response with 39.1% vs 28.1% for TP53single-hit (p=0.2). Patients with TP53mult-hit lost response earlier at 8.2 months, vs 13.2 months for TP53single-hit, and 15.1 months for TP53wt (p=0.1). Median overall survival (mOS) was 11.5 months (95% CI: 8.6 - 19.1) for TP53mult-hit, 22.1 months (95% CI: 14.6 - 35.9) for TP53single-hit, and 31.7 months (95% CI: 19.5 - 51.1) for TP53wt (log-rank, p < 0.005). Propensity scores matched 47 TP53mutpatients treated with DEC-C and 47 TP53mut patients treated with single agent parenteral HMA. Median survival was 13.1 months (95% CI: 8.4 - 21.3) for DEC-C vs 8.0 months (95% CI: 5.2 - 13.0) for single agent parenteral HMA (log-rank, p=0.047). In patients with MDS harboring TP53mut, DEC-C may improve overall survival compared to parenteral HMA.

02 Nov 2025·Expert Review of Anticancer Therapy

Oral decitabine and cedazuridine for the treatment of myelodysplastic syndromes: an integrated review of clinical, economic and patient-centered evidence

Review

Author: Salimi, Tehseen ; Zeidan, Amer M. ; Zhao, Ruizhi ; Epstein, Robert S.

INTRODUCTION:

Myelodysplastic syndromes/neoplasms (MDS) are a heterogenous group of myeloid cancers that impose a substantial negative impact on patient health-related quality of life. As MDS predominately affects older individuals, who are especially susceptible to the debilitating nature of the disease and its burdensome symptoms, treatment decisions should consider therapeutic value from multiple perspectives to balance clinical efficacy with patient-centered outcomes. This comprehensive approach is known as relative medical value.

AREAS COVERED:

Although improved outcomes have been observed with hypomethylating agents (HMAs) in patients with higher-risk MDS, parenteral administration of HMA requires frequent infusion clinic visits and is associated with substantial time burden, especially in older patients, impacting treatment persistence. Suboptimal use of HMAs in MDS may lead to poorer outcomes and higher healthcare costs, underscoring the need for patient-centered treatment options that improve persistence.

EXPERT OPINION:

Oral decitabine and cedazuridine (DEC-C) is an approved treatment for higher-risk MDS and may reduce patient burden associated with parenteral HMA therapy. Oral DEC-C has the potential to improve treatment persistence and clinical outcomes and reduce healthcare resource utilization and costs. This review integrates the available clinical, patient-centered, and economic evidence on the relative medical value of oral DEC-C treatment for MDS.

01 Sep 2025·Lancet Haematology

Oral decitabine and cedazuridine maintenance after haematopoietic stem-cell transplantation in very high-risk acute myeloid leukaemia or myelodysplastic syndrome (GFM-DACORAL-DLI): a multicentre, single-arm, phase 2 trial

Article

Author: de Latour, Régis Peffault ; Chevret, Sylvie ; Charbonnier, Amandine ; Chermat, Fatiha ; D'Aveni, Maud ; Carre, Martin ; Thépot, Sylvain ; Labussière, Hélène ; Stamatoullas, Aspasia ; Mokeddem, Chama ; Petit, Raphael ; Ravinet, Aurélie ; Rubio, Marie-Thérèse ; Paul, Franciane ; Adès, Lionel ; Alani, Mustafa ; Huynh, Anne ; Raffoux, Emmanuel ; Chevallier, Patrice ; Sébert, Marie ; Desseaux, Kristell ; Robin, Marie ; Garnier, Alice ; Mediavilla, Clémence ; Himberlin, Chantal ; Fenaux, Pierre

BACKGROUND:

The combination of a hypomethylating agent with donor lymphocyte infusion as maintenance therapy after haematopoietic stem-cell transplantation (HSCT) in acute myeloid leukaemia and myelodysplastic syndrome might reduce the risk of relapse. We aimed to evaluate the activity and safety of oral decitabine and cedazuridine (ASTX727) as maintenance after allogeneic HSCT in patients with acute myeloid leukaemia or myelodysplastic syndrome at very high risk of relapse post-transplantation.

METHODS:

We conducted a multicentre, single-arm, phase 2 study (GFM-DACORAL-DLI) at 12 centres in France. We enrolled patients aged 18-70 years with an Eastern Cooperative Oncology Group performance status of 0-2, without contraindication for HSCT, and with very high-risk disease (poor or very poor prognosis according to the revised International Prognostic Scoring System for myelodysplastic syndrome, adverse risk according to the 2017 European LeukemiaNet classification for acute myeloid leukaemia; unfavourable genetics; and acute myeloid leukaemia post-myelodysplastic syndrome or post-myeloproliferative neoplasm, or relapsing less than 2 years after complete response). Patients were included 5-45 days before transplantation. ASTX727 was orally administered at escalating doses (100 mg cedazuridine plus 35 mg decitabine starting at 1 day per cycle and increasing to 3 days) from day 40 post-HSCT, up to ten cycles. Donor lymphocyte infusion was recommended when patients had no contraindication after cycle 4. The primary endpoint was disease-free survival at 1 year after HSCT, assessed in the first 28 enrolled patients treated with ASTX7277. Safety was assessed in all participants who received at least one course of ASTX727. This study was registered with ClinicalTrials.gov (NCT04857645); enrolment is complete but follow-up is ongoing.

FINDINGS:

Between Sept 28, 2021, and March 1, 2023, 59 patients were screened and 51 patients underwent allogeneic HSCT (median age 62·0 years [IQR 56·5-65·0]; 22 [43%] female, 29 [57%] male). 34 patients received maintenance treatment with ASTX727; seven of them received at least one donor lymphocyte infusion. 14 (41%) patients completed the ten cycles. Median follow-up was 12·6 months (IQR 10·3-14·3). Among the first 28 enrolled patients treated with ASTX727, disease-free survival at 1 year after HSCT was 70·4% (95% CI 55·1-89·9). The most frequent grade 3 or worse adverse events were haematological, occurring in 25 (74%) of 34 patients (21 [62%] neutropenia, eight [24%] thrombocytopenia, four [12%] anaemia). Serious adverse events occurred in 14 (41%) of 34 patients, and were haematological in eight patients and gastrointestinal in three patients. One treatment-related death, due to thrombocytopenia, occurred.

INTERPRETATION:

ASTX727 could be a potential treatment option after HSCT in patients with acute myeloid leukaemia or myelodysplastic syndrome at very high risk of relapse. Further investigation is warranted to establish the efficacy and safety of this therapeutic approach.

FUNDING:

Taiho Oncology and Astex Pharmaceuticals.

News (Medical) associated with Decitabine/Cedazuridine

02 Feb 2026

·www.biospace.com

FDA Action Alert: Sanofi/Regeneron, Merck, REGENXBIO and More

Taylor Tieden for BioSpace In what is shaping up to be a back-loaded month, the FDA is set to release a slew of regulatory decisions in February, including two that would expand the labels of blockbuster drugs. REGENXBIO Readies for Hunter Decision After Delay Kicking off the month is REGENXBIO, which by Feb. 8 is expecting the FDA’s verdict on its Hunter syndrome gene therapy RGX-121. The Maryland-based biotech had originally expected a decision on RGX-121 by Nov. 9, but the FDA extended the deadline by three months to allow time to review longer-term clinical data submitted by REGENXBIO. Also known as mucopolysaccharidosis II, Hunter syndrome is a rare condition caused by mutations in the iduronate-2-sulfatase ( IDS ) gene, resulting in a dysfunctional enzyme that under healthy circumstances helps degrade waste inside the cell. The toxic buildup of cellular waste, in turn, leads to hallmark symptoms of Hunter syndrome, such as developmental delay and growth abnormalities. RGX-121 delivers a functioning copy of the IDS gene and restores the body’s ability to produce healthy levels of the corresponding enzyme. Data from the Phase I/II/III CAMPSIITE study, released in February 2024, showed an 86% median reduction in DS26, a disease biomarker indicative of brain disease activity. DS26 levels approached normal in the study, REGENXBIO said at the time. RGX-121 has the potential to become the first one-time therapy that targets the underlying cause of Hunter syndrome, according to an August 2025 news release from the company. Merck Looks to Expand Keytruda Into Ovarian Cancer Merck is seeking to add another indication to the more than 40 for which its blockbuster cancer drug Keytruda is already approved. By Feb. 20, the FDA is expected to release its decision on the proposed combination of the PD-1 inhibitor with chemotherapy, with or without Roche’s Avastin, for the treatment of platinum-resistant recurrent ovarian cancer. To support its application, Merck submitted data from the Phase III KEYNOTE-B96 trial, which in October last year demonstrated a 30% improvement in progression-free survival versus placebo, and a 24% drop in the risk of death from all causes. Nearly all patients experienced toxicities related to the study’s investigational regimen, leading to death in 0.9% of patients. In comparison, 1.6% of placebo counterparts died due to side effects. Keytruda has not yet been approved to treat any kind of ovarian cancer. Vanda Awaits Verdict for Bysanti in Bipolar I, Schizophrenia Vanda Pharmaceuticals is proposing its atypical antipsychotic Bysanti for the treatment of acute bipolar I disorder and schizophrenia. The FDA’s decision is expected on Feb. 21. Bysanti’s active ingredient is milsaperidone, an active metabolite of iloperidone, which is also owned by Vanda and is approved under the brand name Fanapt for the same indications that Bysanti is targeting. In a March 2025 news release , Vanda said that when taken orally, Bysanti converts to Fanapt and that the two drugs have been shown in clinical studies to be “bioequivalent at both low and high doses.” The company backed this claim with pharmacokinetic data showing comparable changes in levels over time between the two drugs. Given this chemical similarity, Vanda is supporting Bysanti’s application with studies described in Fanapt’s label . The safety package for Bysanti will also include data from Fanapt’s clinical development as well as post-marketing experience with the drug, covering more than 80,000 patient-years of exposure. Taiho Proposes Combo Regimen of Inqovi for Leukemia Taiho Oncology is combining its cancer drug Inqovi with AbbVie and Roche’s BCL-2 inhibitor Venclexta for the treatment of acute myeloid leukemia (AML). A verdict is expected on Feb. 25. Specifically, Taiho is proposing the combination in patients who are unable to undergo intensive induction chemotherapy. In the Phase IIb ASCERTAIN-V study, the investigational regimen elicited a complete response rate of 46.5%. Lowering the bar to include complete responders with incomplete hematologic recovery bumped this rate up to 63.4%, according to a May 2025 publication in the Journal of Clinical Oncology . Inqovi combines two drugs: the nucleoside metabolic inhibitor decitabine and the cytidine deaminase inhibitor cedazuridine. The first chemical triggers the death of cancer cells, while the second increases the overall systemic exposure of decitabine. Inqovi was first approved by the FDA in July 2020 for the treatment of myelodysplastic syndromes. If approved for this indication, Inqovi would become the first all-oral alternative to current AML therapies, Taiho CMO Harold Keer said in a July 2025 news release . Eton Targets Diabetes Insipidus With Oral Solution By Feb. 25, the FDA is expected to release its decision on Eton Pharmaceuticals’ ET-600, an oral desmopressin solution, for the treatment of diabetes insipidus. Affecting 1 in 25,000 people worldwide , diabetes insipidus is a rare disease characterized by the over-production of urine. The condition develops when the body fails to make enough vasopressin, an antidiuretic hormone that helps maintain fluid balance in the body. Desmopressin is a synthetic form of vasopressin. Pivotal data in March 2025 showed that ET-600 is bioequivalent to an FDA-approved reference product containing the same active ingredients. Compared to commercial products, however, ET-600, if approved, “can accommodate the precise and titratable doses necessary for pediatric patients,” CEO Sean Brynjelsen said at the time. Following Delay, Ascendis Awaits Decision on Dwarfism Drug Late last year, the FDA pushed back by three months its decision date for Ascendis Pharma’s investigational achrondroplasia drug TransCon CNP. That extension is nearing its end, with a verdict expected by Feb. 28. While it qualifies as a rare disease— achondroplasia affects around 15,000 to 40,000 newborns—it is nevertheless the most common cause of dwarfism. The disease is caused by a gain-of-function mutation in the FGFR3 gene, which ultimately suppresses the activity of cells involved in bone development. A crucial player in this pathway is C-type natriuretic peptide, a hormone that counteracts FGFR3. TransCon CNP works by mimicking the function of this hormone and helps spur bone growth in patients. In the Phase II ACcomplisH trial, treatment improved annualized growth velocity to 5.42 cm per year, as compared with 4.35 cm per year in placebo comparators. A 2023 publication in The Lancet noted that this treatment benefit was statistically significant. If approved, TransCon CNP is expected to pose a substantial challenge to BioMarin’s achondroplasia medicine Voxzogo. In its third-quarter earnings call in October last year, BioMarin walked back its previously set revenue goal of $4 billion by 2027, with CFO Brian Mueller pointing specifically to the “impact of potential Voxzogo” competitors. BioMarin Targets Younger PKU Population for Palynziq Looking to broaden its patient population, BioMarin is seeking a label expansion for its enzyme substitution therapy Palynziq, with a target action date of Feb. 28. Specifically, the California company is looking to lower the age of eligibility for the drug to include adolescents from 12 through 17 years with phenylketonuria (PKU). Palynziq is currently only indicated for adults who have uncontrolled blood phenylalanine levels. Data from the Phase III PEGASUS study, released in September last year, showed that Palynziq treatment lowered blood phenylalanine levels by 49.7% on average at 72 weeks, as compared with dietary adjustments alone. Mean baseline phenylalanine was 1,026.4 µmol/L and nearly half of patients had concentrations above 1,000 µmol/L. Nearly half of treated patients saw at least a 50% reduction in phenylalanine levels at 72 weeks, BioMarin added at the time. PKU is a genetic disease that renders the phenylalanine hydroxylase enzyme dysfunctional, in turn disrupting the normal metabolism of phenylalanine, commonly found in many protein-containing foods. Patients with this condition suffer from the complications of phenylalanine buildup, such as seizures, tremors and intellectual disabilities. Sanofi, Regeneron Eye Another Indication for Dupixent Capping off this list is Sanofi and Regeneron’s blockbuster drug Dupixent, which the partners are proposing for the treatment of allergic fungal rhinosinusitis (AFRS). The FDA’s verdict is expected on Feb. 28. AFRS is a distinct subtype of chronic rhinosinusitis characterized by an excessive inflammatory reaction at the sinuses, driven by a hypersensitivity to fungi. Patients often suffer from nasal congestion and thick mucus discharge, and the condition can lead to nasal polyps, poor quality of life and even bone deformation around the sinus cavities. Current treatments include surgery and a lengthy course of systemic steroids. Sanofi and Regeneron are supporting their expansion bid with data form the Phase III LIBERTY-AFRS-AIMS study, which in November last year demonstrated a 50% improvement in nasal congestion at 52 weeks, as compared with 9.8% in placebo comparators. Dupixent likewise hit key secondary endpoints, including patient-reported nasal obstruction, nasal polyp size and the need for corticosteroids or surgery.

Clinical ResultPhase 3Drug ApprovalPhase 2

13 Nov 2025

·www.prnewswire.com

Taiho Oncology Announces 14 Presentations, Including Six Oral Presentations, at the 67th American Society of Hematology Annual Meeting and Exposition

Six oral presentations will detail new data on the outcomes of all-oral regimens containing azacitidine and cedazuridine or decitabine and cedazuridine in patients with myelodysplastic syndromes, chronic myelomonocytic leukemia or acute myeloid leukemia PRINCETON, N.J., Nov. 13, 2025 /PRNewswire/ -- Taiho Oncology, Inc., a company developing and commercializing novel treatments for hematologic malignancies and solid tumors, announced the presentation of new data from 14 studies across company-sponsored and company-funded externally led studies at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition, to be held Dec. 6-9, 2025, in Orlando, Florida. Taiho Oncology will share findings from clinical trials in two oral presentations and evidence from real-world studies in two online abstracts. The oral presentations will highlight results from the Phase 2 part of ASTX030-01, a randomized, open-label, crossover trial comparing oral combination of azacitidine and cedazuridine to subcutaneous azacitidine and the Phase 2 portion of the Phase 1/2 trial ASTX727-03 of low-dose versus standard-dose oral decitabine and cedazuridine in patients with lower-risk myelodysplastic syndromes (MDS). The two online posters will present real-world data on U.S. adults with acute myeloid leukemia (AML), describing treatment patterns and outcomes with hypomethylating agent (HMA) plus venetoclax, and evaluating the time burden of initiating parenteral HMA-based therapy in routine practice. Additionally, 10 presentations – including four oral presentations – led by independent investigators will feature data from Taiho-funded investigator-initiated or collaborative studies. These presentations will highlight: Results from a UK multicenter Phase 2 randomized trial comparing ASTX727 with hydroxycarbamide or best supportive care in advanced MDS/ myeloproliferative neoplasm (MPN). Data from a Phase 2 study evaluating the all-oral combination of ASTX727, venetoclax and revumenib in newly diagnosed AML patients with NPM1m, KMT2Ar or NUP98r AML mutations. Results of a Phase 1/2 clinical trial of the combination of oral decitabine/cedazuridine with the venetoclax in patients with untreated high-risk MDS and chronic myeloid leukemia (CMML). Safety and efficacy results from a Phase 1/2 trial evaluating an all-oral combination of ASTX727, venetoclax and gilteritinib in both relapsed refractory and newly diagnosed FLT3-mutated AML, high-risk MDS or MPN. "As the leader of oral HMA development, we're excited that these data will be featured in six oral presentations at the ASH Annual Meeting," said Harold Keer, MD, PhD, Taiho Oncology Chief Medical Officer. "These data highlight our increasing understanding of novel oral regimens in hematology. We are invested in developing treatments that have the potential to improve flexibility and lower the treatment burden for patients, and collaborating with leading institutions, evidenced by our continued support of independent clinical research to advance cancer care. We look forward to participating in the essential scientific exchange that contributes to progress in hematology at the ASH Annual Meeting." Data being presented by Taiho Oncology and its partners at the 2025 ASH Annual Meeting and Exposition include: Company-Sponsored Studies Oral Presentations Title: A phase 2 dose confirmation trial of oral ASTX030, a combination of oral azacitidine with cedazuridine among patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, and acute myeloid leukemia Abstract Number: 491 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML Session Date: Dec . 7, 2025 Session Time: 9:30 a.m. to 11a.m. Presentation Time: 10:15 to 10:30 a.m. Location: OCCC - Valencia Room W415D Presenter: Guillermo Garcia-Manero, MD Title: Low-dose oral decitabine and cedazuridine among patients with low-risk myelodysplastic syndromes Abstract Number: 790 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Treatment Advances in Lower risk Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms Session Date: Dec . 8, 2025 Session Time: 10:30 a.m. to 12 p.m. Presentation Time: 11:15 to 11:30 a.m. Location: OCCC - Valencia Room W415A Presenter: Guillermo Garcia-Manero, MD Online Abstracts Title: Time burden of treatment with parenteral HMAs in combination with venetoclax among patients with newly diagnosed AML Abstract Number: 6990 Lead Author: Amer Zeidan, MD Title: Real-World Characteristics, Treatment Modifications, and Outcomes for AML Patients Receiving HMAs + Venetoclax (1L) Abstract Number: 8176 Lead Author: Amer Zeidan, MD Company-Funded Externally Led Studies Oral Presentations Title: Phase II Study of the All-Oral Combination of Revumenib (SNDX-5613) with Decitabine/Cedazuridine (ASTX727) and Venetoclax (SAVE) in Newly Diagnosed AML Abstract Number: 47 Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens Session Date: Dec . 6, 2025 Session Time: 9:30 to 11 a.m. Presentation Time: 10:30 to 10:45 a.m. Location: OCCC - Valencia Room W415A Presenter: Wei-Ying Jen, BM BCh, FRCPath Title: Oral decitabine/cedazuridine in combination with venetoclax in treatment-naïve high-risk myelodysplastic syndrome or chronic myelomonocytic leukemia: Updates of a Phase 1/2 clinical trial Abstract Number: 237 Session Name: 637 . Myelodysplastic Syndromes : Clinical and Epidemiological: Treatment Advances in Higher Risk Myelodysplastic Syndromes Session Date: Dec . 6, 2025 Session Time: 2 to 3:30 p.m. Presentation Time: 2:30 to 2:45 p.m. Location: OCCC - Chapin Theater W415BC Presenter: Alex Bataller, MD, PhD Title: ASTX727 delivers superior response rates and associated survival benefit versus hydroxycarbamide/best supportive care in CMML and other MDS/MPN overlap syndromes: First results from the Phase 2 UK multicenter randomized ammo trial Abstract Number: 488 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Moving the Needle Through Novel Approaches in MDS and CMML Session Date: Dec. 7, 2025 Session Time: 9:30 to 11 a.m. Presentation Time: 10:45 to 11 a.m. Location: OCCC - Valencia Room W415D Presenter: Daniel H. Wiseman, MB, ChB, PhD Title: Phase I/II Study of Decitabine/Cedazuridine (ASXT727), Venetoclax, and Gilteritinib for Patients with FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome Abstract Number: 997 Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Frontline treatment with intensive or lower-intensity regimens Session Date: Dec . 8, 2025 Session Time: 4:30 to 6 p.m. Presentation Time: 4:30 to 4:45 p.m. Location: OCCC - Chapin Theater W320 Presenter: Roberta S. Azevedo, MD Poster Presentations Title: Translational Investigation of Tolinapant (ASTX660) in Acute Myeloid Leukemia Using Integrated Clinical, Bioinformatic, and Pharmacological Approaches Abstract Number: 2552 Session Name: 802. Chemical Biology and Experimental Therapeutics: Poster I Session Date: Dec. 6, 2025 Session Time: 5:30 to 7:30 p.m. Location: OCCC - West Halls B3-B4 Title: A Randomized Phase II Trial of ASTX727 and Venetoclax With or Without Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy (MM1OA-S03) Abstract Number: 3466 Session Name: 617 . Acute Myeloid Leukemias: Commercially Available Therapies: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: Trial in progress: A Phase I Study Evaluating the Safety of cirtuvivint (CIRT) as Monotherapy and in Combination with ASTX727 in Patients with MDS and AML Abstract Number: 3431 Session Name: 616 . Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: Trial in Progress - A Randomized Study of ASTX727 With or Without Ladademstat in Accelerated/Blast-Phase Myeloproliferative Neoplasms Abstract Number: 3827 Session Name: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: A Comparison of Overall Survival and Quality of Life in MDS Patients Treated with Azacitidine vs Decitabine: A Propensity-Matched Registry Study Abstract Number: 3862 Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster II Session Date: Dec. 7, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 Title: A Multiarm Phase 1b Study of Personalized Oral Maintenance Therapy with Decitabine/Cedazuridine (ASTX727) Plus Physician's Choice of Venetoclax, Gilteritinib, Enasidenib, or Ivosidenib in Acute Myeloid Leukemia Abstract Number: 5228 Session Name: 617. Acute Myeloid Leukemias: Commercially Available Therapies: Poster III Session Date: Dec. 8, 2025 Session Time: 6 to 8 p.m. Location: OCCC - West Halls B3-B4 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 Taiho Pharmaceutical Co., Ltd. Taiho Oncology Contact: Leigh Labrie (609) 664-9878 [email protected] SOURCE Taiho Oncology 21% more press release views with Request a Demo

Phase 2ASHClinical ResultPhase 1Cell Therapy

07 Nov 2025

·www.globenewswire.com

ORYZON Reports Financial Results and Corporate Update for Quarter Ended September 30, 2025

The net result as of September 30, 2025 improved by $1.1M compared to September 2024, following termination of a Convertible Bonds financing facility with Nice&Green CNS(Vafidemstat) Received feedback from the FDA on Phase III PORTICO-2 trial in BPD; Company to revise protocol and resubmitReinforcing company’s clinical, strategy and regulatory teamsExpansion of ongoing Phase IIb in schizophrenia into other EU countries continuesPreparations continue for new Phase II trial in aggression in Autism Spectrum Disorder Oncology – Hematology(Iadademstat) Positive data in 1L AML to be presented at ASH with 100% ORR (88% CR)Positive data in R/R FLT3+ AML to be presented at ASH. RP2D selectedInitiated enrollment in new Phase II trial in myeloproliferative neoplasms Enrollment in Phase I in MDS continues activelyInitiated enrollment in Phase Ib in sickle cell diseaseNew Phase II trial in essential thrombocythemia in preparation MADRID and CAMBRIDGE, Mass., Nov. 07, 2025 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and a European leader in epigenetics, today reported financial results for the nine months ended September 30, 2025 and provided a corporate update on recent developments. “We have secured over $60 million in the first half of 2025, marking a significant turnaround for Oryzon,” said Dr. Carlos Buesa, Oryzon’s Chief Executive Officer. “These proceeds enable us to advance our clinical programs with a renewed strategic focus on CNS, particularly in our studies in BPD, schizophrenia, and ASD.” “Following the FDA’s feedback, the Company has strengthened its clinical, strategic, and regulatory teams by incorporating highly experienced professionals with deep expertise in late-stage clinical development and FDA-EMA interactions,” continued Dr. Buesa. “As part of our strategic plan to become a CNS-focused company and to further strengthen our balance sheet, we are exploring potential partnerships for our promising oncology-hematology asset iadademstat. We continue to enhance its value through ongoing collaborations and the generation of new clinical evidence under our CRADA agreement with the NCI, which requires only a minimal financial commitment from the Company.” “The impressive data in first-line AML from the triple combination of venetoclax, azacitidine, and iadademstat — showing no dose-limiting toxicities and a 100% overall response rate — clearly illustrates the success of this strategy,” Dr. Buesa added. “We expect that additional promising results in MDS and other studies will continue to demonstrate the clinical relevance of iadademstat across different hematologic malignancies. We believe that the data to be presented at ASH, together with forthcoming results in SCD, will further support our efforts to identify the right partner to ensure that this drug ultimately reaches patients.” Third Quarter and Recent Highlights Vafidemstat: Following submission of the clinical trial protocol for the PORTICO-2 Phase III trial with vafidemstat in Borderline Personality Disorder (BPD) to the U.S. Food & Drug Administration (FDA) for approval in June, the Company received written feedback from the FDA in October. The FDA’s guidance covers different elements such as the selection of study endpoints and certain non-clinical considerations. Oryzon will incorporate these insights and resubmit the revised Phase III protocol.As part of our strategic preparations to advance our late-stage pipeline and to enhance dialogue with the FDA and EMA, the Company has strengthened its clinical, strategic, and regulatory teams by incorporating highly experienced professionals as Senior Advisors: Dr. Iman Barilero, who brings extensive CNS experience from her nine-year tenure as Global Head of Regulatory Affairs at Lundbeck and will be acting as a Chief Regulatory Officer; Dr. Christopher Breder, MD PhD, a veteran drug developer who also spent several years of his career as a Medical Officer at the FDA; and Dr. Raymond Sanchez, former CMO of Cerevel Therapeutics. The Company plans to incorporate additional experts to further reinforce its development capabilities.These appointments follow the recent incorporation of distinguished clinical and academic experts into our Clinical Advisory Board (CAB), including Dr. Alan Schatzberg (Chair of the Department of Psychiatry and Behavioral Sciences at Stanford University from 1991 to 2010 and current Director of the Stanford Mood Disorders Center), Dr. Eric Hollander (Professor of Psychiatry and Behavioral Sciences at the Albert Einstein College of Medicine and Director of the Autism and Obsessive-Compulsive Spectrum Program), Dr. Emil Coccaro (Professor of Psychiatry at The Ohio State University College of Medicine and former Chair of Psychiatry & Behavioral Neuroscience at The University of Chicago (2004-2016)), and Dr. Sarah Fineberg (Assistant Professor of Psychiatry at Yale University). Oryzon is preparing a new Phase II trial to evaluate vafidemstat for the treatment of aggression in patients with autism spectrum disorder (ASD). This trial, named HOPE-2, plans to include, inter alia, genetically-defined ASD subpopulations, such as individuals with Phelan-McDermid syndrome (PMS), and will initially be conducted in Spain as part of the activities supported by the recently granted Med4Cure IPCEI EU initiative. In parallel with its clinical development efforts in ASD and PMS, Oryzon is collaborating as a sponsor of the first-ever PMS Burden of Illness study, led by CureShank, a research advocacy organization founded by families of individuals affected by PMS. This study, recently launched, aims to characterize the direct and indirect burden of PMS to patients, caregivers, and the US healthcare system.The EVOLUTION Phase IIb clinical trial evaluating vafidemstat in patients with schizophrenia continues to enroll participants. This study aims to assess the efficacy of vafidemstat, with a primary focus on improving negative symptoms. As secondary endpoints, the trial will evaluate vafidemstat’s efficacy in improving cognitive impairment and positive symptoms in schizophrenia. Initially conducted only in Spain, the trial is now being expanded to additional EU countries.Oryzon has continued to strengthen its patent portfolio for vafidemstat during this quarter, with an additional “Decision to grant” communication from the European patent office. The allowed claims cover the use of vafidemstat for the treatment of aggressiveness and social withdrawal associated with CNS diseases, including claims specifically aimed at the treatment of aggressiveness associated with BPD, ASD, Alzheimer’s disease and other conditions, as well as claims directed to treating social withdrawal associated with diseases such as schizophrenia or ASD. Once formally granted, this patent will remain in force until at least 2038, not including any potential patent term extension. Additional patents in this family have already been granted or allowed in Europe, Australia, Canada, Hong Kong, Israel, South Korea, Malaysia, the Philippines, and Russia, with applications pending in other countries. Iadademstat: The iadademstat triplet combination with venetoclax and azacitidine achieved an overall response rate (ORR) of 100% (n=8) in a Phase I dose finding clinical trial in patients with newly diagnosed acute myeloid leukemia (AML). The study, which continues active patient enrollment, has been accepted for presentation at the upcoming American Society of Hematology (ASH) Annual Meeting (December 6–9, 2025, Orlando, Florida, USA). As reported in the ASH abstract, 88% of patients achieved complete remission (CR) and 12.5% achieved morphologic leukemia-free state (MLFS). After a median follow-up of nine months, the estimated six-month overall survival (OS) rate was 88%, and no dose-limiting toxicities were observed. This investigator-initiated study (IIS) is led by the Oregon Health & Science University (OHSU) Knight Cancer Institute.A second study in the same clinical setting, sponsored by the National Cancer Institute (NCI) and conducted under our Cooperative Research and Development Agreement (CRADA) with the NCI, continues to actively recruit patients and is expected to provide additional data to further substantiate the promising early results in first line AML. The iadademstat combination with gilteritinib achieved a 67% overall response rate (8/12 patients) and a 58% complete response rate (CR + CRh + CRi; 7/12 patients) among the 12 evaluable patients treated at the dose currently under expansion in the open-label, multicenter Phase Ib FRIDA clinical trial in patients with relapsed or refractory (R/R) AML harboring a FLT3 mutation (FLT3mut+). The activity observed at this dose was superior to both historical and real-world data reported for gilteritinib monotherapy. Three patients have proceeded to hematopoietic stem cell transplantation (HSCT). The study, which continues to enroll patients, has been accepted for presentation at ASH-2025. The study is being conducted in the United States and will enroll up to approximately 45 patients. As reported in the ASH abstract, 34 patients had been enrolled at the data cut-off for abstract submission, with four dose level cohorts evaluated in the escalation phase. The combination was tolerable at the tested doses, and the study has progressed to the expansion phase at one selected pharmacologically active dose. Updated data will be presented at the congress.A new randomized Phase II study of iadademstat in combination with ASTX727 (oral decitabine + cedazuridine) in patients with accelerated/blast phase (AP/BP) myeloproliferative neoplasms (MPNs), sponsored by the NCI under the CRADA with Oryzon, has recently started to enroll patients. The study has a dose escalation phase to identify the Recommended Phase 2 Dose (RP2D) of iadademstat + ASTX727, followed by a randomized phase which will investigate the efficacy of iadademstat + ASTX727 compared to ASTX727 monotherapy. A Trial-in-progress (TIP) abstract has been accepted for presentation at ASH-2025.Enrollment has continued in the IIS Phase I dose-finding trial of iadademstat in combination with azacitidine in myelodysplastic syndrome (MDS), led by the Medical College of Wisconsin (MCW), and in the Phase I/II trial of iadademstat plus immune checkpoint inhibitors in patients with extensive-stage small cell lung cancer (SCLC) who have initially received standard of care chemotherapy and immunotherapy, conducted and sponsored by the NCI under the CRADA with Oryzon.Beyond oncology, Oryzon has expanded clinical evaluation of iadademstat into non-malignant hematological disorders, with a first trial in sickle cell disease (SCD). This multicenter, open-label Phase Ib trial, named RESTORE (REgulation of Sickling ThrOugh Reprogramming Epigenetics), will evaluate the safety and tolerability of iadademstat in adult patients with SCD, and determine its Recommended Phase 2 dose (RP2D), as well as to evaluate iadademstat’s effect on inducing fetal hemoglobin (HbF) expression. Increases in HbF have already been recognized by the FDA as a clinically meaningful endpoint for the treatment of SCD. The trial, recently approved by the European Medicines Agency (EMA), has started to enroll patients. The study is conducted across several sites in Spain and aims to enroll approximately 40 adult patients. A new trial, which will evaluate iadademstat in essential thrombocythemia (ET), is currently in preparation, with Clinical Trial Application (CTA, the EU equivalent to an IND) submission to EMA planned for Q425.Oryzon has strengthened its patent portfolio for iadademstat during this quarter, with “Decision to grant” communications from the European and Australian Patent Offices for patent applications entitled “Combinations of iadademstat for cancer therapy”. The allowed claims protect the use of iadademstat in combination with PD1 or PD-L1 inhibitors for the treatment of cancer, including small cell lung cancer (SCLC). Once formally granted, the patents will remain in force until at least 2040, excluding potential patent term extensions. A corresponding patent has already been granted in Russia, with applications pending in the United States, Japan, China, and other territories. Earlier stage programs: ORY-4001, Oryzon’s highly selective histone deacetylase 6 (HDAC6) inhibitor nominated as a clinical candidate for the treatment of certain neurological diseases such as Charcot-Marie-Tooth disease (CMT), Amyotrophic Lateral Sclerosis (ALS) and others, continues to progress through IND enabling studies to prepare it for clinical studies. Financial Update: Third quarter 2025 Financial Results Research and development (R&D) expenses were $3.9 million and $9.6 million for the quarter and nine months ended September 30, 2025, compared to $1.9 and $7.1 million for the quarter and nine months ended September 30, 2024. General and administrative expenses were $1.2 and $3.9 million for the quarter and nine months ended September 30, 2025, compared to $0.9 and $3.1 million for the quarter and nine months ended September 30, 2024. Net losses were $1.2 and $4.6 million for the quarter and nine months ended September 30, 2025, compared to net losses of $1.1 and $3.8 million for the quarter and nine months ended September 30, 2024. The result is as expected, given the biotechnology business model where companies in the development phase typically have a long-term maturation period for products and do not have recurrent income. Negative net result was $1.5 million (–$0.02 per share) for the nine months ended September 30, 2025, compared to a negative net result of $2.5 million (–$0.04 per share) for the nine months ended September 30, 2024. Cash, cash equivalents, and marketable securities totaled $40.4 million as of September 30, 2025. ORYZON GENOMICS, S.A.BALANCE SHEET DATA (UNAUDITED)1(Amounts in thousands US $) September 30th, 2025 September30th, 2024 Cash and cash equivalents40,430 8,442Marketable securities0 0Total Assets171,036 122,661 Deferred revenue0 0Total Stockholders' equity136,974 96,854 ORYZON GENOMICS, S.A. STATEMENTS OF OPERATIONS (UNAUDITED)1 (US $, amounts in thousands except per share data) Three Months EndedSeptember 30th Nine Months Ended September 30th 20252024 20252024 Collaboration Revenue00 00 Operating expenses: Research and Development3,8571,915 9,6277,076 General and administrative1,232879 3,8903,051 Total operating expenses5,0892,794 13,51710,127 Loss from Operations-5,089-2,794 -13,517-10,127 Other income, net3,8941,671 8,8796,312 Net Loss -1,195-1,123 -4,638-3,815 Net Financial & Tax1,590-256 3,1621,272 Net Result 395-1,379 -1,476-2,543 Loss per share allocable to common stockholders: Basic 0.01-0.02 -0.02-0.04 Weighted average Shares outstanding Basic 75,197,02663,383,939 72,523,99462,336,626 1 Spanish GAAP * Exchange Euro/Dollar (1.1741 for 2025 and 1.1196 in 2024) About OryzonFounded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company and the European leader in epigenetics, with a strong focus on personalized medicine in CNS disorders and oncology. Oryzon’s team is composed of highly qualified professionals from the pharma industry located in Barcelona, Boston, and San Diego. Oryzon has an advanced clinical portfolio with two LSD1 inhibitors, vafidemstat in CNS (Phase III-ready) and iadademstat in oncology (Phase II). The company has other pipeline assets directed against other epigenetic targets like HDAC-6 where a clinical candidate ORY-4001, has been nominated for its possible development in CMT and ALS. In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases. For more information, visit www.oryzon.com About IadademstatIadademstat (ORY-1001) is a small oral molecule, which acts as a highly selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers (see Maes et al., Cancer Cell 2018 Mar 12; 33 (3): 495-511.e12.doi: 10.1016 / j.ccell.2018.02.002.). A FiM Phase I/IIa clinical trial with iadademstat in R/R AML patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi (see Salamero et al, J Clin Oncol, 2020, 38(36): 4260-4273. doi: 10.1200/JCO.19.03250). Iadademstat has shown encouraging safety and strong clinical activity in combination with azacitidine in a Phase IIa trial in elder 1L AML patients (ALICE trial) (see Salamero et al., ASH 2022 oral presentation & The Lancet Haematology, 2024, 11(7):e487-e498). Iadademstat is currently being evaluated in combination with gilteritinib in the ongoing Phase Ib FRIDA trial in patients with relapsed/refractory AML with FLT3 mutations, and in combination with azacitidine and venetoclax in 1L AML in an investigator-initiated study led by OHSU and in a trial sponsored by the U.S. National Cancer Institute (NCI) under the Cooperative Research and Development Agreement (CRADA) signed between Oryzon and the NCI to collaborate on further clinical development of iadademstat in different types of hematologic and solid cancers. Beyond hematological cancers, the inhibition of LSD1 has been proposed as a valid therapeutic approach in some solid tumors such as small cell lung cancer (SCLC), neuroendocrine tumors (NET), medulloblastoma and others. In a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients (CLEPSIDRA trial), preliminary activity and safety results have been reported (see Navarro et al., ESMO 2018 poster). Iadademstat is in a Phase I/II randomized trial in 1L ED-SCLC in combination with ICI sponsored by NCI and led by the Memorial Sloan Kettering Cancer Center. Oryzon is further expanding the clinical development of iadademstat in oncology through additional CRADA and investigator-initiated studies. In addition, Oryzon is expanding iadademstat’s clinical development into non-oncological hematology indications, with trials in sickle cell disease (enrolling) and essential thrombocythemia (trial in preparation). Iadademstat has orphan drug designation for SCLC in the US and for AML in the US and EU. About Vafidemstat Vafidemstat (ORY-2001) is an oral, CNS-optimized LSD1 inhibitor. The molecule acts on several levels: it reduces cognitive impairment, including memory loss and neuroinflammation, and at the same time has neuroprotective effects. In animal studies vafidemstat not only restores memory but reduces the exacerbated aggressiveness of SAMP8 mice, a model for accelerated aging and Alzheimer’s disease (AD), to normal levels and also reduces social avoidance and enhances sociability in murine models. In addition, vafidemstat exhibits fast, strong, and durable efficacy in several preclinical models of multiple sclerosis (MS). Oryzon has performed two Phase IIa clinical trials in aggressiveness in patients with different psychiatric disorders (REIMAGINE, see Ferrer et al, Psychiatry & Clin Neurosci, 2025, doi.org/10.1111/pcn.13800) and in aggressive/agitated patients with moderate or severe AD (REIMAGINE-AD), with positive clinical results reported in both. Additional finalized Phase IIa clinical trials with vafidemstat include the ETHERAL trial in patients with Mild to Moderate AD, where a significant reduction of the inflammatory biomarker YKL40 was observed after 6 and 12 months of treatment, and the pilot, small-scale SATEEN trial in Relapse-Remitting and Secondary Progressive MS, where anti-inflammatory activity was also observed. Vafidemstat has also been tested in a Phase II in severe Covid-19 patients (ESCAPE) assessing the capability of the drug to prevent ARDS, one of the most severe complications of the viral infection, where it showed significant anti-inflammatory effects in severe Covid-19 patients. Following completion of the global, randomized, double blind Phase IIb PORTICO trial in Borderline Personality Disorder (BPD), with final data presented at ECNP-2024, vafidemstat is advancing as a Phase III-ready asset for agitation/aggression in BPD (PhIII protocol submitted). Vafidemstat is also being investigated in a double-blind, randomized, placebo-controlled Phase IIb trial in negative symptoms of schizophrenia (EVOLUTION trial, recruitment ongoing). The company is also deploying a CNS precision medicine approach with vafidemstat in genetically defined patient subpopulations of certain CNS disorders, as well as in neurodevelopmental syndromes, and is preparing a clinical trial in aggression in autistic conditions like Phelan-McDermid syndrome. FORWARD-LOOKING STATEMENTS This communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives, and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates” and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees, or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forward‐looking statements, whether as a result of new information, future events, or otherwise. This document does not constitute an offer or invitation to purchase or subscribe shares in accordance with the provisions of Regulation (EU) 2017/1129 of the European Parliament and of the Council of 14 June 2017, and/or the restated text of the Securities Market Law, approved by Law 6/2023 of 17 March, and its implementing regulations. Nothing in this document constitutes investment advice. In addition, this document does not constitute an offer of purchase, sale or exchange, nor a request for an offer of purchase, sale or exchange of securities, nor a request for any vote or approval in any jurisdiction. The shares of Oryzon Genomics, S.A. may not be offered or sold in the United States of America except pursuant to an effective registration statement under the Securities Act of 1933 or pursuant to a valid exemption from registration. Spain Oryzon IR & Media, Europe & US Patricia Cobo/Mario Cordera Emili Torrell Sandya von der Weid Atrevia Chief BD Officer LifeSci Advisors, LLC +34 91 564 07 25 +34 673 33 97 65 +34 93 515 1313 +41 78 680 05 38 pcobo@atrevia.com mcordera@atrevia.com etorrell@oryzon.com svonderweid@lifesciadvisors.com

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-	Decitabine/Cedazuridine	L01XY	DB15694

R&D Status

Approved

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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

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Indication	Highest Phase	Country/Location	Organization	Date
Anemia, Refractory	Phase 3	United States	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Austria	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Canada	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Czechia	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	France	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Germany	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Hungary	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Italy	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	Spain	Astex Pharmaceuticals, Inc.	15 Feb 2018
Anemia, Refractory	Phase 3	United Kingdom	Astex Pharmaceuticals, Inc.	15 Feb 2018

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04655755 (ASH2025)	Phase 1/2	High Risk Myelodysplastic Syndrome \| Chronic Myelomonocytic Leukemia First line	69	Oral decitabine/cedazuridine + venetoclax	qfoaldtxdf(kzxhsnzciq) = Patients with ASXL1mut had a median OS not reached (vs 18 months in patients with ASXL1wt, P=0.008) fwgsbzmipf (mqxwwwyyjx ) View more	Positive	06 Dec 2025
NCT05010772 (ASH2025)	Phase 1	Acute Myeloid Leukemia Maintenance	35	ASTX727 35/100 mg PO	nkmqsfxpxg(lofqtrbklk) = restvkrpql jptgnkpknn (idfucdjizh ) View more	Positive	06 Dec 2025
				ASTX727 35/100 mg PO + venetoclax 400 mg (azole adjusted) PO	nkmqsfxpxg(lofqtrbklk) = chhnuoewov jptgnkpknn (idfucdjizh ) View more
ISRCTN30808508 (AMMO, ASH2025)	Phase 2	Myeloproliferative Disorders	77	ASTX727	emzagrntav(skdzwiyxvv) = sonygmqsvg vorwnbacxt (qfewjyvksc, 39.3 - 66.3) View more	Positive	06 Dec 2025
				Hydroxycarbamide/Best Supportive Care	emzagrntav(skdzwiyxvv) = ytxoiqebxq vorwnbacxt (qfewjyvksc, 14.9 - 49.4) View more
NCT06661915 (ASH2025)	Phase 2	Myeloproliferative Disorders	50	ASTX727 + iadademstat	rqfpgcjxlc(sprovoknat) = urpalzslcu parflexfmf (pltyqdoris )	Positive	06 Dec 2025
				ASTX727	rqfpgcjxlc(sprovoknat) = uffdtuetvg parflexfmf (pltyqdoris )
NCT06802146 (ASH2025)	Phase 1	CCUS Clonal Cytopenia of Undetermined Significance	138	DEC/CED	uitgeocafd(canlmzycta) = lwiphmjrfy lvdlvlcbru (amffpruyxo ) View more	Positive	06 Dec 2025
NCT05360160 (SAVE, ASH2025)	Phase 2	Acute Myeloid Leukemia NPM1 mutation \| KMT2A rearrangement	17	Decitabine/cedazuridine + venetoclax + revumenib	dftatuzafs(ibqgystlde) = QTc prolongation occurred in 8 (47%) pts; 3 were grade 2 (18%), the rest were grade 1 (29%) yyllqctmwo (avoxgwhsfk ) View more	Positive	06 Dec 2025
NCT04340843 (ETCTN/NCI 10330, CTgov)	Phase 2	Chondrosarcoma \| Central Nervous System Neoplasms \| Brain metastases	19	ASTX727+Belinostat (Treatment (Belinostat, ASTX727))	pfduucuizd = ftwgpzkeso brmccztriv (avwljqiiyy, jbcpvtrpxf - gzukzqrbva) View more	-	16 Sep 2025
				SGI-110+Belinostat (Treatment (Belinostat, SGI-110))	cyhfovvqkf = vlxsgpscga lubnsmvjzq (yhvfkhrvjg, cunephmhov - tgjxtugzlz) View more
NCT04657081 (ASCO2025) Manual	Phase 2	Acute Myeloid Leukemia First line	101	decitabine-cedazuridine (DEC-C)+venetoclax	iourdeaqra(fzmduuvlxg) = yhkfxtmtsp wivlxqnaef (mgumcdhesp, 36.5 - 56.7) View more	Positive	23 May 2025
NCT04657081 (S135, EHA2025)	Phase 1/2	Acute Myeloid Leukemia	189	DECITABINE-CEDAZURIDINE (DEC-C) (DEC-C + VEN)	hhglpbwryy(kctxvnxnga) = lecnwnmodl pmkvihypwx (slwhrvronz, 36.5 - 56.7) View more	Positive	14 May 2025
NCT05010772 (4277, ASH2024)	Phase 1	Acute Myeloid Leukemia complex cytogenetics \| antecedent MDS/MPN \| therapy-related AML ... View more	31	ASTX727 alone	isuqordbnl(teesulajyb) = cadnccsxiw iluyweludx (gltvidhjzh ) View more	Positive	09 Dec 2024
				ASTX727 plus venetoclax	isuqordbnl(teesulajyb) = fzkpynpmbu iluyweludx (gltvidhjzh ) View more

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