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Targeted Agent NTX-301

NTX-301 is a nucleoside-based DNMT1 inhibiting targeted anticancer therapy for various blood cancers/solid cancers caused by abnormalities in DNA methyl-transferase (DNMT) that regulates the gene expression patterns in our body. The indications are MDS (myelodysplastic syndrome), AML (acute myeloid leukemia), T-ALL (T-cell acute lymphocytic leukemia) among blood cancers, and ovarian and bladder cancers among solid cancers.

Unlike the existing DNMT1 inhibitors, which are effective only in blood cancers, NTX-301 shows excellent efficacy in various solid cancers.

Blood Cancer (MDS/AML/T-ALL)

MDS/AML occur more often in the elderly, the sector of population, who have limited treatment options. Their 5-year survival rate is below 15%. 30~40% of T-ALL patients relapse, have limited treatment options, and have poor prognosis.

  • Erythrocyte

  • Neutrophil

  • Lymphocyte

  • Monocyte

  • Platelet

  • MDS/AML 5-Year Survival Rate (>65 Years Old)

  • Relapsed T-ALL Patient Overall Survival Rate


Ovarian/Bladder Cancer have serious resistance issues and low overall survival rate.

  • Worldwide Incidences and Deaths of Ovarian Cancer

  • 5-Year Survival Rate of Platinum-resistant Ovarian Cancer

  • Worldwide Incidences and Deaths of Bladder Cancer

  • Overall Survival Rate of Platinum-resistant Bladder Cancer

Distinction and Competitive Advantage

  • Better efficacy than current DNMT1 inhibitors

    Powerful efficacy through its dual mechanism of action (DNMT 1 inhibition + DNA damage repair inhibition)

    Has stable PK profile, as NTX-301 is less metabolized by Decitabine's metabolic enzyme (Cytidine Deaminase)

  • Better safety profile than current DNMT1 inhibitors

    Improved safety profile compared to current DNMT1 inhibitors through its selective action in cancer cells

    Increased dosage enables enhanced treatment effect compared to other treatments

  • Improve the patient convenience and drug compliances

    Oral administration that allows ease of use


Name of society Title Content Date Presentation Materials
ASH 2021 A Study on The Preclinical Efficacy, Underlying Mechanisms, and Sensitivity Markers of A Novel Hypomethylating Agent NTX-301 in Acute Myeloid Leukemia Identification of safety for humans, co-administration potential, efficacy indicators, and biomarkers Dec. 2021
ASCO 2021 Phase I Trial of 5-aza-4'-thio-2'-deoxycytidine (Aza-TdC) in Patients with Advanced Solid Tumors Identification of safety for humans, oral administration potential, target engagement, and efficacy indicators Jun. 2021

Clinical trial status

Pipeline Indication
Phase 1
Phase 2
Phase 3
NTX-301 Blood Cancer (MDS/AML)
US - Single Agent (UAB)
Blood Cancer (T-ALL)
US - Single Agent (UAB)
Solid Tumor (MEN 1 Syndrome, etc.)
US - Single Agent (NCI)
Solid Tumor (Ovarian/Bladder)
AUS - Combi (SOCRU, etc.)