Telomir-1 boosts organ recovery in Wilson’s disease preclinical study

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.

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