[Asia Economy Reporter Hyunseok Yoo] Pinobio announced on the 15th that it has disclosed preclinical results of the targeted anticancer agent NTX-301 at the American Society of Hematology (ASH) in collaboration with the Korea Research Institute of Chemical Technology.


NTX-301 is a nucleoside-based DNMT1 inhibitor targeting hematologic and solid cancers. It is a best-in-class targeted anticancer drug candidate that addresses insufficient efficacy, resistance development, and high side effects seen with existing drugs decitabine and azacitidine.


According to the announcement, NTX-301 demonstrated superior anticancer efficacy and excellent safety compared to existing DNMT1 inhibitors in preclinical studies. In preclinical in vitro experiments, it strongly inhibited cancer cell growth and selectively activated specific DNA affecting cancer growth, proliferation, and metastasis within cancer cells, showing an improved safety profile over existing drugs.


Additionally, the combination with AbbVie’s Venclexta (active ingredient venetoclax) showed excellent synergy. Venclexta is used as a standard treatment for elderly MDS/AML patients. In animal experiments with transplanted cancer cells, the group treated with the combination of NTX-301 and venetoclax achieved complete remission at lower doses compared to the group treated with azacitidine and venetoclax, and survival periods were also extended.


Jung Doo-young, CEO of Pinobio, stated, “Having secured excellent preclinical data, we expect good results from the ongoing Phase 1 clinical trial in the U.S. targeting MDS and AML patients. We will do our best to make NTX-301 an innovative treatment for patients with rare and intractable diseases such as MDS and AML.”



Meanwhile, NTX-301 has completed a Phase 1 clinical trial in patients with advanced solid tumors, and high safety and excellent efficacy indicators were presented at this year’s American Society of Clinical Oncology (ASCO). Pinobio is currently conducting a U.S. Phase 1 clinical trial for MDS/AML hematologic cancers, a U.S. Phase 1/2 clinical trial for solid tumors, and an Australian Phase 1/2 combination therapy clinical trial for ovarian and bladder cancers.


This content was produced with the assistance of AI translation services.

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