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Technology to Increase Selectivity for
cancer cells and Enhance Efficacy
NTX-301
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Targeted Agent NTX-301

NTX-301 is a nucleoside-based DNMT1 inhibitory 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) among blood cancers, and ovarian and bladder cancers among solid cancers.

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

Indications

Hematological Malignancy (MDS/AML)

MDS/AML have high incidence rate in the older adults and have limited treatment options. The 5-year survival rate from diagnosis is less than 15%.

Ovarian
Cancer
Bladder
Cancer

Ovarian/bladder cancers have serious issue of developing resistance to platinum-based chemotherapy. Once resistance occurs, the survival rate is extremely low.

Overcoming the limitations of competing drugs

NTX-301 overcomes resistance mechanisms of competing DNMT1 inhibitors through the Thio- modification in the nucleoside structure

Distinction and Competitive Advantage

  • Strong Efficacy

    Dual MoA inhibiting DNMT1 and DNA repair induces strong efficacy

    Stable PK profile can be achieved by evading the metabolic enzymes of Decitabine

  • Good Safety Profile

    Cancer-selective activation feature leads to improved safety profile

    Increased safety margin allows higher dosage and effective treatment compared to existing DNMT1 inhibitors

  • Convenient Use

    Oral administration is easier for patients to take the drug

Clinical trial status

Project Indication Development Stage

Discovery
Preclinical
Phase 1
Phase 2
Phase 3
 Clinical Site
NTX-301 Solid tumor
- US Phase 1b/2a(ongoing)
- AUS Phase 1a(finished)
Hematological Malignancy
(MDS/AML/CMML)
- US Phase 1a(ongoing)