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BM001 is a small molecule inhibitor targeting the growth hormone/insulin-like growth factor-1 (GH/IGF1) axis. It acts by rendering the growth hormone receptor (GHR) dysfunctional, thereby inhibiting cancer cell proliferation. This mechanism mimics the natural resistance to cancer observed in individuals with Laron syndrome, who have GHR deficiency. BM001 has demonstrated potent antiproliferative effects in vitro and in vivo across several cancer types, including colorectal cancer (CRC), triple-negative breast cancer (TNBC), and non-small cell lung cancer (NSCLC). The compound is currently in preclinical development and was identified as a lead candidate by BIMINI Biotech[1][2][5]. Additionally, literature refers to BM001 as a 2,4-diaminopyrimidine–based compound with antifolate properties that can inhibit dihydrofolate reductase (DHFR) and impact folate metabolism pathways relevant for tumor cell survival[3].
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