Telomir Pharmaceuticals has announced compelling preclinical findings demonstrating that its lead compound Telomir-1 fully reverses epigenetic gene silencing in aggressive human prostate cancer cells, achieving superior efficacy compared to standard chemotherapy agents Paclitaxel and Rapamycin in restoring critical tumor suppressor function.
Superior Epigenetic Restoration
In a 21-day study using a mouse model implanted with aggressive human prostate cancer cells (PC3 xenograft), Telomir-1 administered orally completely reversed hypermethylation of STAT1, a master regulator of immune surveillance and programmed cell death. The reversal occurred in a dose-dependent fashion, while Paclitaxel showed no effect and Rapamycin achieved only partial reduction of STAT1 hypermethylation.
STAT1 is frequently silenced in advanced cancers through promoter hypermethylation, effectively disabling one of the body's key defense mechanisms by shutting down immune detection and blocking signals that would normally trigger cancer cell death. By restoring STAT1 activity, Telomir-1 may be reawakening the body's built-in tumor suppressor system, allowing immune cells to once again recognize, target, and eliminate cancer.
"This study provides novel and direct molecular evidence of Telomir-1's ability to reprogram cancer epigenetics," said Erez Aminov, CEO of Telomir. "By potentially restoring the function of key tumor suppressor genes like STAT1, we're not just slowing tumor growth-we're turning the immune system back on. These results highlight Telomir-1's potential as a powerful new approach to treating aggressive cancers as combination therapy with cancer immunomodulators."
Dual Tumor Suppressor Activation
Beyond STAT1, Telomir-1 also reduced hypermethylation of TMS1 (also known as ASC or PYCARD), a pro-apoptotic tumor suppressor commonly silenced in prostate cancer. While Paclitaxel and Rapamycin showed comparable or greater effects on TMS1 methylation, Telomir-1 is unique in its ability to modulate both STAT1 and TMS1 simultaneously—two genes that together regulate immune response and apoptosis.
TMS1 plays a central role in inflammasome activation, contributing to tumor cell death and supporting 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, which can impair immune surveillance and allow cancer cells to persist undetected.
"PC3 cells are notoriously aggressive and resistant to treatment," said Dr. Itzchak Angel, Chief Scientific Advisor at Telomir. "The potential simultaneous reactivation of STAT1 and TMS1 by inhibition of the regulatory hypermethylation suggests that Telomir-1 may help overcome two of the biggest hurdles in cancer biology-immune evasion and apoptosis resistance. This dual action could help explain the tumor suppression observed in our earlier studies."
Safety Profile Reinforced
Critically, Telomir-1 did not elongate telomeres in the cancerous prostate cells (PC3), a finding that reinforces its cancer safety profile. While telomere elongation is therapeutic in degenerative and aging-related diseases, longer telomeres in cancer cells can fuel unchecked growth. PC3 cells already maintain short but stable telomeres via telomerase—a hallmark of malignancy. The fact that Telomir-1 exerted strong biological effects without increasing telomere length supports its selectivity in targeting telomere maintenance where appropriate.
Building on Previous Success
These findings build upon results previously announced in March 2025, where Telomir-1 reduced tumor volume by approximately 50% and eliminated chemotherapy-induced mortality when combined with Paclitaxel in the same prostate cancer model. That study also suggested Telomir-1's metal ion-regulating properties may mitigate oxidative stress, further protecting healthy cells from chemotherapy toxicity.
Clinical Development Path
Telomir is conducting ongoing research to evaluate Telomir-1 across multiple therapeutic areas, including oncology, Wilson's disease, age-related macular degeneration (AMD), 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," added Aminov. "Our team is committed to advancing this program thoughtfully and strategically as we move toward clinical development."
