Overview
Telomir Pharmaceuticals, Inc, a preclinical-stage biotechnology company focused on reversing biological aging and degenerative diseases, 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.
About the condition: 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.
- Treatment effects- Treatment reversed tremors, ataxia, anxiety-like behavior, liver and kidney pathology damage, reduced copper accumulation, normalized ALT, AST, and bilirubin levels, and improved survival.
Telomir-1: A Breakthrough in Regenerative Medicine & Copper Regulation
- Broad regenerative potential – Reverses aging-related degeneration in Werner syndrome and AMD, restoring function.
- Epigenetic reset – Resets the clock, extends telomeres, restores gene expression, and rescues survival.
- Retinal regeneration – Restores vision in AMD, demonstrating systemic benefits.
- Copper-binding properties – High affinity for copper, regulating key ion exchanges.
- Wilson’s disease breakthrough – In vivo findings confirm copper regulation leads to significant physiological and behavioral improvements.
- Disease reversal in zebrafish model – Dose-dependent, statistically significant impact on major disease features in ATP7B C271X model.
Following are 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.
Words from the CEO: Telomir
- 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.""
Statement from the chief scientific advisor
- 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.""
The Telomir Pharmaceuticals
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.
