Telomir says data show Telomir-1 reversed epigenetic silencing

In a regulatory filing, Telomir Pharmaceuticals (TELO) reported new preclinical results evaluating the effects of its lead candidate, Telomir-1, in a murine xenograft model using PC3 human prostate cancer cells. The data demonstrated that Telomir-1 reversed epigenetic silencing by DNA methylation of the STAT1 gene, a tumor suppressor and immune response regulator, in a dose-dependent manner. STAT1 is frequently silenced in advanced cancers via promoter hypermethylation, disrupting immune detection and apoptotic pathways. In this study, Telomir-1 administered orally daily for 21 days resulted in full reversal of STAT1 hypermethylation in tumor tissue. No such reversal was observed in the Paclitaxel group, and only partial demethylation was observed with Rapamycin In addition to STAT1, Telomir-1 also reduced hypermethylation of TMS1, a pro-apoptotic tumor suppressor commonly silenced in prostate cancer. While PTX and Rapamycin showed comparable or greater effects on TMS1 methylation, Telomir-1 is unique in its ability to modulate both STAT1 and TMS1-two genes that together regulate immune response and apoptosis. Importantly, TMS1 also plays a central role in inflammasome activation, which not only contributes to tumor cell death but also supports the immune system’s ability to detect and clear abnormal cells. When TMS1 is hypermethylated, this immune signaling pathway is disrupted-reducing caspase-1 activation and inflammatory cytokine release. This can impair immune surveillance and allow cancer cells to persist undetected. By reducing TMS1 hypermethylation, Telomir-1 may help restore immune recognition and enhance the tumor’s sensitivity to both chemotherapy and immunotherapy. Telomere length analysis in PC3 tumor tissue showed no measurable elongation following Telomir-1 administration. This finding is relevant given concerns that telomere-targeting compounds may promote tumor progression through telomere extension in malignant cells. In contrast, Telomir-1 demonstrated selective activity without altering telomere length in this model. Telomir is conducting ongoing research to evaluate Telomir-1 across multiple therapeutic areas, including oncology, Wilson’s disease, age-related macular degeneration, autism spectrum disorder, and dysphonia. The company is continuing preclinical development across these programs and plans to announce its initial IND indication at a future date. “We remain encouraged by the preclinical data and continue to explore Telomir-1’s potential across several disease areas. Our team is committed to advancing this program thoughtfully and strategically as we move toward clinical development,” said Erez Aminov, CEO of Telomir.