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PAX-1-003 is an orally bioavailable small molecule telomerase inhibitor being developed by Komipharm (also known as Kominox). The drug is an arsenic-based compound, specifically sodium metaarsenite, which acts by targeting the telomerase enzyme. Telomerase is responsible for maintaining telomere length and is overexpressed in approximately 90% of human cancer cells, allowing them to bypass replicative senescence. By inhibiting telomerase activity, PAX-1-003 induces telomere shortening, leading to chromosomal instability and apoptosis in malignant cells. It is currently being evaluated in Phase 1 clinical trials for a broad range of metastatic solid tumors, including lung-to-brain metastases and a basket study encompassing 14 different metastatic cancer types such as breast, gastric, and prostate cancers.
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