- Treatment reversed tremors, ataxia, anxiety-like behavior, liver and kidney pathology damage, reduced copper accumulation, normalized ALT, AST, and bilirubin levels, and improved survival.
- Building on previous data in Werner syndrome, showing Telomir-1 reset the epigenetic clock, extended telomere length, restored gene expression, reversed muscle loss, and rescued survival-alongside data in Age Related Macular Degeneration (AMD) demonstrating retinal regeneration and restored vision-the drug continues to show broad regenerative potential across rare genetic degenerative diseases
MIAMI, FL – Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), or the “Company,” a preclinical-stage biotechnology company focused on reversing biological aging and degenerative diseases, today announced compelling new preclinical data demonstrating that its lead drug candidate, Telomir-1, significantly reversed neurological, hepatic and kidney symptoms in a clinically relevant animal model of Wilson’s disease.
Wilson’s disease is a rare and potentially fatal genetic disorder caused by mutations in the ATP7B gene, which impair the body’s ability to eliminate excess copper. As copper builds up-primarily in the liver and brain-it leads to inflammation, tissue damage, and multi-organ dysfunction. Patients may experience liver failure, psychiatric disturbances, tremors, and progressive neurological decline. Current treatments involve lifelong copper chelation or liver transplantation, highlighting the urgent need for safer, disease-modifying therapies.
These new results build on earlier in vitro studies that confirmed Telomir-1’s high binding affinity for copper and its ability to exchange and regulate key ions. The current in vivo findings now confirm that Telomir-1’s copper-regulating properties translate into meaningful behavioral, physiological and histological improvements in a genetic animal model of Wilson’s disease.
In the ATP7B C271X -/- zebrafish model, which mimics human Wilson’s disease Telomir-1 demonstrated dose-dependent and statistically significant reversal of major disease features:
Key Findings from the Study:
- Up to a 4- to 5-fold reduction in episodic tremor events
- Normalization of swim distance, swim velocity, and exploratory behavior
- Reversal of ataxia-like motor behaviors (e.g., abnormal body bends and turn angles)
- Approximately 50% reduction in copper accumulation in dry liver tissue
- Marked improvement in liver and kidney histopathology, with liver and kidney scores reduced to near-normal levels
- Normalization of ALT, AST, and bilirubin-three critical liver biomarkers. ALT and AST are enzymes elevated during liver injury, while bilirubin builds up when detoxification is impaired. Telomir-1 restored these to wild-type levels, indicating protection of liver function and copper-induced damage
- Improved survival under high copper exposure conditions
“We’ve now seen Telomir-1 generate breakthrough results across some of the most challenging age-related and genetic diseases-including AMD, Wilson’s disease, cancer, progeroid and Werner syndromes, and early findings suggesting reversal of key factors in type 2 diabetes,” said Erez Aminov, Chairman and CEO of Telomir. “In our AMD model, Telomir-1 restored vision and regenerated retinal structure using FDA-recognized surrogate endpoints. In Werner syndrome, it reset the epigenetic clock and reversed hallmarks of accelerated aging. And in Wilson’s disease, we saw a reversal of neurological, liver and kidney damage. These results point to the broad therapeutic potential of Telomir-1 across diseases driven by cellular degeneration. We believe this positions Telomir-1 as a powerful new platform for longevity, neuroprotection, and regenerative medicine.”
“These recent findings establish Telomir-1 as a potent disease-modifying compound in a clinically relevant model of Wilson’s disease,” added Dr. Angel, Chief Scientific Advisor. “Its ability to reverse behavioral and neurological dysfunction, normalize histological and functional biomarkers, and extend survival underscores its therapeutic promise across both rare and age-related disorders.”
Telomir Pharmaceuticals is currently advancing Telomir-1 through IND-enabling studies and expects to file its first IND for a rare disease indication by year-end, with human clinical trials planned for the first half of 2026.
Contact Information
Helga Moya
[email protected]
(786) 396-6723