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PAX-1-002 is an orally bioavailable small molecule formulation of sodium metaarsenite developed by Komipharm (Kominox). It functions as a telomerase inhibitor, targeting the enzyme responsible for maintaining telomere length in immortalized cancer cells. By inhibiting telomerase activity, PAX-1-002 induces telomere shortening, DNA damage, and subsequent apoptosis in malignant cells. The drug is currently being evaluated in Phase 2 clinical trials for the treatment of glioblastoma multiforme (GBM), including both newly diagnosed and recurrent or relapsed cases. It has also been investigated in other oncology indications such as prostate cancer and lymphoma under the development code KML-001.
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