- SEC Filing

Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), a preclinical-stage biotechnology company developing therapies that target the root mechanisms of cancer, aging, and age-related diseases, today announced new in vitro results that expand understanding of its lead drug candidate, Telomir-1.

The studies, conducted by Eurofins Discovery, showed that Telomir-1 potently inhibits UTX (KDM6A), a histone demethylase that interacts closely with DNA methylation to control which genes are switched on or off. Abnormal UTX activity has been linked to silencing of tumor suppressors and inappropriate activation of disease-driving genes. This dysregulation is observed in cancer, autoimmune disease, aging, neurodegeneration, autism spectrum disorder, and metabolic dysfunction. By inhibiting UTX, Telomir-1 demonstrated the potential to reset faulty DNA methylation patterns and restore more normal gene regulation.

As previously reported, Telomir-1 also inhibited additional epigenetic enzymes — FBXL10, FBXL11, and JMJD3 — that are implicated in tumor progression, inflammation, metabolic dysfunction, and neurodevelopmental disorders. In prior prostate cancer studies in vivo, Telomir-1 reactivated silenced tumor suppressors STAT1 and TMS1 by reversing abnormal DNA methylation, providing functional evidence of its ability to reset gene programs that are often disabled in cancer.

Importantly, Telomir-1 showed no activity against GCN5L2 (KAT2A), a broad acetyltransferase enzyme whose inhibition has been associated with widespread toxicity. This selectivity may provide Telomir-1 with a cleaner safety margin compared to other epigenetic drugs.

The new in vitro data also showed that Telomir-1 had low-level inhibitory activity against Tankyrases (PARP5A and PARP5B). Tankyrases regulate the Wnt/β-catenin pathway, one of the body's master growth-control circuits that cancers frequently hijack as a "fuel line" for unchecked proliferation and treatment resistance. Unlike potent Tankyrase inhibitors, which can shorten telomeres and pose systemic safety risks, Telomir-1's modest activity may allow disruption of cancer's fuel line without compromising telomere biology. Supporting this distinction, previously reported data from a validated Werner Syndrome accelerated-aging in vivo model showed that Telomir-1 significantly elongated telomeres beyond healthy levels while also reversing abnormal DNA methylation, restoring youthful gene regulation, and resetting the epigenetic clock.

Taken together, the results support Telomir-1's emerging profile as a potential first-in-class epigenetic therapy with a dual mechanism of action: