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Decitabine

Generic Name
Decitabine
Brand Names
Dacogen, Inqovi 5 Tablet Pack
Drug Type
Small Molecule
Chemical Formula
C8H12N4O4
CAS Number
2353-33-5
Unique Ingredient Identifier
776B62CQ27
Background

Myelodysplastic syndromes (MDS) are a heterogeneous group of hematopoietic neoplasms with variable underlying etiology and presentation, including neutropenia and thrombocytopenia. Further mutations leading to increased proliferation of cancerous cells can eventually lead to secondary acute myeloid leukemia, which has a poor prognosis. Among treatment options, nucleoside analogues such as decitabine and azacitidine integrate into cellular DNA and inhibit the action of DNA methyltransferases, leading to global hypomethylation and related downstream therapeutic benefits.

Decitabine was developed by MGI Pharma/SuperGen Inc. and was approved by the FDA for the treatment of MDS on February 5, 2006. It was first marketed under the name Dacogen®. It is also available as an oral combination product together with the cytidine deaminase inhibitor cedazuridine.

Indication

Decitabine is indicated for the treatment of patients with myelodysplastic syndromes (MDS) including all French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia), as well as for MDS scored as belonging to the intermediate-1, intermediate-2, or high-risk group in the International Prognostic Scoring System.

Associated Conditions
Chronic Myelomonocytic Leukemia, Myelodysplastic Syndrome

Global Clinical Trials for Myelodysplastic Syndrome Show Significant Industry Investment in 2025

• A comprehensive review of global Myelodysplastic Syndrome clinical trials reveals extensive research activity across G7 and E7 countries, with major pharmaceutical companies leading development efforts. • The analysis highlights significant participation from industry leaders including Bristol-Myers Squibb, Novartis, and Sanofi, demonstrating strong commercial interest in MDS therapeutics. • The report indicates evolving clinical trial landscapes across multiple regions, with detailed tracking of trial phases, enrollment trends, and success rates over the past five years.

Phase 2 Trial Shows Promise: Subcutaneous Dacogen Achieves 33% Response Rate in Myelofibrosis Patients

• A multicenter phase 2 trial demonstrated that subcutaneous Dacogen (decitabine) achieved a 33% overall response rate in myelofibrosis patients, with responses lasting a median of seven months. • The study enrolled 21 patients who received 0.3 mg/kg of subcutaneous Dacogen daily for 10 days, marking the first prospective evidence supporting low-dose extended schedule treatment in this population. • While the treatment showed promise in managing cytopenias, significant myelosuppression was observed, suggesting the need for further investigation of alternative dosing schedules.

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• Series A venture financing for epigenetic drug development has reached $342M in early 2024, marking a dramatic 375% increase from 2023 and setting a five-year record. • Avenzo Therapeutics secured the largest Series A round of $150M to advance CDK2 inhibitor ARTS-021, currently in Phase I/II trials for solid tumors and metastatic breast cancer. • The surge in investment follows FDA's approval of Vertex's Casgevy, highlighting growing confidence in epigenetic approaches as safer alternatives to traditional gene editing.

Nucleus RadioPharma Secures $56M Series A to Expand Radiopharmaceutical Manufacturing Network

• Nucleus RadioPharma, a Mayo Clinic and Eclipse venture, has raised $56 million in Series A funding to establish a network of radiopharmaceutical manufacturing facilities across the United States. • The company aims to modernize clinical development, manufacturing, and supply chain for radiotherapeutics and diagnostics, addressing current limited production capacity and fragmented supply issues. • The funding round was led by Eclipse and GE HealthCare, with participation from multiple healthcare institutions including Mayo Clinic, Fox Chase Cancer Center, and Mercy Health.
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