pharma · Nasopharyngeal Carcinoma · Hepatocellular Carcinoma
Xiyuan Hospital of China Academy of Chinese Medical Sciences
Chinese Academy of is a pharma organization headquartered in TAIZHOU, CN. Primary therapeutic focus areas include Nasopharyngeal Carcinoma, Hepatocellular Carcinoma, COVID-19, Breast Cancer, Coronary Artery Disease. Nova
Phase 2 · small molecule · AML
Decitabine (DACOGEN) is a small-molecule antineoplastic agent developed by Xiyuan Hospital of China Academy of Chinese Medical Sciences for acute myeloid leukemia (AML). The program, identified as IIT2016007, is currently in Phase 2 development and has been terminated as of the latest milestone on 30 May 2025. Decitabi
Internal code IIT2016007（Decitabine）
Decitabine (DACOGEN) is a small-molecule antineoplastic agent developed by Xiyuan Hospital of China Academy of Chinese Medical Sciences for acute myeloid leukemia (AML). The program, identified as IIT2016007, is currently in Phase 2 development and has been terminated as of the latest milestone on 30 May 2025. Decitabine is administered intravenously and functions as an antineoplastic and immunomodulating agent (ATC class L01). The drug has already achieved regulatory approval in the European Union (EMA approval on 1 October 2024) and the United States, where multiple generic manufacturers hold approved applications. The clinical development program in China is registered under NCT03021395, though the specific rationale for termination and detailed clinical outcomes remain undisclosed. The competitive landscape for AML therapeutics includes approved agents such as Vyxeos Liposomal (Jazz Pharmaceuticals) and multiple targeted therapies. Decitabine's development by a Chinese research institution reflects ongoing efforts to establish clinical evidence for this established agent in specific patient populations, though the termination status suggests the program did not proceed to completion.
Acute myeloid leukemia remains a serious hematologic malignancy with significant unmet medical needs, particularly in elderly patients and those with specific molecular subtypes. Decitabine addresses an important therapeutic area where DNA methyltransferase inhibition offers a distinct mechanism compared to conventional chemotherapy. The drug's approval in major markets (US, EU) demonstrates established safety and efficacy, yet continued clinical investigation in China suggests regional interest in optimizing treatment protocols or exploring specific patient populations. The termination of this Phase 2 program, however, indicates that the clinical development objectives were not met or the sponsor determined alternative strategies were more appropriate. From a market perspective, decitabine competes within a crowded AML therapeutic space that includes targeted agents (FLT3 inhibitors, IDH inhibitors, BCL-2 inhibitors) and conventional hypomethylating agents. The presence of multiple generic manufacturers in the US market indicates mature market penetration and commoditized pricing. The clinical relevance of this terminated program lies in understanding why a well-established, approved agent required additional Phase 2 investigation in China and why that investigation was discontinued—factors that may reflect regulatory requirements, commercial considerations, or clinical efficacy concerns specific to the studied population.
Drug Class: Antineoplastic and immunomodulating agent (ATC L01)
Modality: Small molecule
Route of Administration: Intravenous
Brand Name: DACOGEN
International Nonproprietary Name (INN): Decitabine
Mechanism of Action: Not disclosed in available facts; however, decitabine is a cytidine analogue and DNA methyltransferase inhibitor used in hematologic malignancies.
Molecular Target: Not disclosed in available facts.
Related Therapies: Other hypomethylating agents (azacitidine); targeted AML therapies including FLT3 inhibitors, IDH inhibitors, and BCL-2 inhibitors.
First Approval: Decitabine was approved by the FDA and EMA prior to the Phase 2 program initiation; EMA approval documented on 1 October 2024 (marketing authorization holder: Janssen-Cilag International N.V.; EMEA product number EMEA/H/C/002221).
Patent Status: Not disclosed in available facts.
Also known as: AML, AML - acute myeloid leukaemia, AML - acute myeloid leukemia, ANLL, acute Nonlymphocytic leukaemia, acute Nonlymphocytic leukemia
Prevalence: Point prevalence: 1-5 / 10 000 (Europe) — source: Orphanet, validated.
Acute myeloid leukemia (AML) is a group of neoplasms arising from precursor cells committed to the myeloid cell-line differentiation. All of them are characterized by clonal expansion of myeloid blasts. AML manifests by fever, pallor, anemia, hemorrhages and recurrent infections.
ClinicalTrials.gov lists 1,453 registered studies for Acute Myeloid Leukemia (AACT aggregate).
Phase breakdown: PHASE2 (403), PHASE1 (378), NA (292), PHASE1/PHASE2 (203), PHASE3 (106), PHASE2/PHASE3 (31), EARLY_PHASE1 (23), PHASE4 (17)
Common investigational therapies:
Disease data sourced from MONDO Disease Ontology (MONDO:0018874), Orphanet — acute myeloid leukemia, NCT00037583, NCT00037596, NCT00038051, NCT00045942, NCT00048503, AACT (ClinicalTrials.gov aggregate), ClinicalTrials.gov, Open Targets Platform (CC BY 4.0).
Phase 2 trial initiated
Decitabine Phase 2 clinical trial in AML sponsored by Xiyuan Hospital, registered as NCT03021395.
Program terminated
Phase 2 development program terminated; specific reasons and clinical outcomes not disclosed.
The AML therapeutic landscape includes multiple approved agents competing for market share. Vyxeos Liposomal (Jazz Pharmaceuticals Ireland Limited) represents a liposomal formulation combining cytarabine and daunorubicin, approved for newly diagnosed AML in elderly patients. Imbruvica (Janssen-Cilag), while primarily a Bruton tyrosine kinase inhibitor, has applications in certain hematologic malignancies. Kyprolis (Amgen) targets proteasome inhibition in multiple myeloma and related conditions. Unituxin (United Therapeutics Europe Ltd) represents a monoclonal antibody approach to neuroblastoma and related malignancies. The competitive environment also includes generic decitabine formulations from multiple manufacturers (Accord Healthcare, Cipla, Dr Reddy's, Gland Pharma, Hetero Labs, Jiangsu Hansoh, Lupin, Otsuka, Sandoz, Sun Pharma, Wockhardt, Zydus), indicating market commoditization. The presence of established competitors and generic alternatives suggests that the terminated Phase 2 program faced challenges in demonstrating differentiation or clinical benefit sufficient to justify continued development in the Chinese market, particularly given decitabine's already-established regulatory status in major markets.
| Therapy | Company | Mechanism | Status |
|---|---|---|---|
| PFIZER AUSTRALIA PTY LTD | Pfizer Australia Pty Ltd | — | approved |
| IMBRUVICA | Janssen-Cilag Pty Ltd | — | approved |
| AFINITOR | Novartis Pharmaceuticals | — | approved |
| LYSODREN | S.A. | — | approved |
| INLYTA | Pfizer Australia Pty Ltd | — | approved |
| VYXEOS LIPOSOMAL (PREVIOUSLY VYXEOS) | Jazz Pharmaceuticals Ireland Limited | — | approved |
| UNITUXIN | United Therapeutics Europe Ltd | — | approved |
| KYPROLIS | Amgen | — | approved |
| OFEV | Boehringer Ingelheim Pty Ltd | — | approved |
| PACLITAXEL ACCORD | Accord Healthcare Pty. | — | approved |
| LYNOZYFIC | Regeneron UK Limited | — | approved |
| ARX-IMATINIB | Alphapharm Pty Ltd | — | approved |
| TRETINOIN | — | Retinoic acid receptor agonist | Approved |
| TAGRAXOFUSP | — | Interleukin-3 receptor subunit alpha binding agent | Approved |
| SARGRAMOSTIM | — | Granulocyte-macrophage colony-stimulating factor receptor agonist | Approved |
| OLUTASIDENIB | — | Isocitrate dehydrogenase [NADP] cytoplasmic inhibitor | Approved |
| MIDOSTAURIN | — | Protein kinase C (PKC) inhibitor | Approved |
| IVOSIDENIB | — | Isocitrate dehydrogenase [NADP] cytoplasmic inhibitor | Approved |
| IDARUBICIN HYDROCHLORIDE | — | DNA topoisomerase II alpha inhibitor | Approved |
| GLASDEGIB MALEATE | — | Smoothened homolog antagonist | Approved |
Additional associated therapies sourced from Open Targets Platform (CC0), linked to NovaPharmaNews drug profiles where matched.
United States (FDA): Decitabine is approved; multiple generic applications (ANDA) and original new drug applications (NDA021790, NDA205582) are documented. Approved manufacturers include Accord Healthcare, Cipla, Dr Reddy's, Gland Pharma, Hetero Labs, Jiangsu Hansoh Pharmaceuticals, Lupin, Otsuka, Sandoz, Sun Pharma, Wockhardt, and Zydus Pharmaceuticals.
European Union (EMA): Decitabine approved on 1 October 2024 under marketing authorization holder Janssen-Cilag International N.V. (EMEA product number EMEA/H/C/002221).
China (NMPA): Decitabine is in clinical trials status; NCT03356080 is registered in China. The Phase 2 program (IIT2016007, NCT03021395) sponsored by Xiyuan Hospital was terminated as of 30 May 2025.
Japan (PMDA): Regulatory status not disclosed in available facts.
Decitabine is an antineoplastic and immunomodulating agent used in the treatment of acute myeloid leukemia (AML). It is administered intravenously and functions as a DNA methyltransferase inhibitor.
Yes, decitabine is FDA-approved. Multiple generic manufacturers hold approved applications (ANDA), and original new drug applications (NDA021790, NDA205582) are documented in the FDA database.
Yes, decitabine (DACOGEN) was approved by the European Medicines Agency on 1 October 2024. The marketing authorization holder is Janssen-Cilag International N.V. (EMEA product number EMEA/H/C/002221).
The Phase 2 program (IIT2016007) is sponsored by Xiyuan Hospital of China Academy of Chinese Medical Sciences. The program was terminated as of 30 May 2025.
The primary trial identifier is NCT03021395. An additional trial (NCT03356080) is registered in China for decitabine in AML.
Decitabine is administered intravenously.
Decitabine is a cytidine analogue and DNA methyltransferase inhibitor; specific molecular details are not disclosed in available facts, though the drug is classified as an antineoplastic and immunomodulating agent (ATC class L01).
Multiple manufacturers produce generic decitabine in the US, including Accord Healthcare, Cipla, Dr Reddy's, Gland Pharma, Hetero Labs, Jiangsu Hansoh Pharmaceuticals, Lupin, Otsuka, Sandoz, Sun Pharma, Wockhardt, and Zydus Pharmaceuticals.
The Phase 2 program (IIT2016007) sponsored by Xiyuan Hospital was terminated as of 30 May 2025. Specific reasons for termination are not disclosed.
Competing approved therapies include Vyxeos Liposomal (Jazz Pharmaceuticals), Imbruvica (Janssen-Cilag), Kyprolis (Amgen), Unituxin (United Therapeutics), and multiple targeted agents including FLT3 inhibitors, IDH inhibitors, and BCL-2 inhibitors.
Decitabine is in clinical trials status in China. The Phase 2 program was terminated on 30 May 2025; full regulatory approval status is not disclosed.
The indication is acute myeloid leukemia (AML). Specific patient population details (treatment-naïve vs. relapsed/refractory) are not disclosed.
Janssen-Cilag International N.V. holds the marketing authorization for DACOGEN in the European Union (EMEA product number EMEA/H/C/002221).
Decitabine is a small-molecule antineoplastic agent classified in ATC class L01 (Antineoplastic and immunomodulating agents).
No partner is listed for the Xiyuan Hospital-sponsored Phase 2 program (IIT2016007). The program is independently sponsored.
No expected next milestones are disclosed. The program is terminated as of 30 May 2025.
Decitabine → Drug → Target → Indication → Company → Trials → Competitors
Strategic Implications: The termination of the Phase 2 program by Xiyuan Hospital suggests that the clinical development objectives in China were not achieved or that the sponsor reassessed the commercial viability of pursuing additional clinical evidence for an already-approved agent. Given decitabine's established regulatory status in the US and EU, the rationale for a Phase 2 investigation in China likely reflected either regional regulatory requirements, market access considerations, or exploration of specific patient populations (e.g., treatment-naïve vs. relapsed/refractory AML). The termination indicates that these objectives were not met.
Competitive Implications: The presence of multiple generic manufacturers in the US market and established competitors in the AML space (Vyxeos Liposomal, targeted agents) suggests limited commercial differentiation for decitabine in mature markets. The terminated Chinese program may reflect recognition that additional clinical data would not substantially improve market positioning against newer targeted therapies or combination approaches.
Future Catalysts: No future milestones are disclosed. The program status is terminated, and no expected next milestones are documented.
Expected Milestones: None anticipated; program is terminated as of 30 May 2025.
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Intelligence compiled from public regulatory and clinical sources (FDA, EMA, ClinicalTrials.gov and company disclosures). Figures may be editorial or analyst estimates; verify against primary sources before relying on them.