Telomir Pharmaceuticals Announces Telomir-1 Demonstrates Dose-Dependent Restoration of Neurological, Liver and Kidney Functions in Preclinical Wilson's Disease Model
- Significant dose-dependent improvements in Wilson's disease model with 4-5x reduction in tremors and 50% reduction in copper accumulation
- Successful normalization of critical liver biomarkers (ALT, AST, bilirubin) to wild-type levels
- Demonstrated broad therapeutic potential across multiple conditions including AMD, Wilson's disease, and Werner syndrome
- Clear development timeline with IND filing planned for year-end and human trials in early 2026
- Still in preclinical stage with no human trial data available
- Results limited to zebrafish model studies
- Multiple regulatory hurdles ahead before potential commercialization
Insights
Telomir-1 shows promising preclinical results for Wilson's disease, adding to its potential as a multi-indication regenerative therapy.
Telomir has released compelling preclinical data for their lead candidate Telomir-1 in Wilson's disease, a rare genetic disorder causing copper accumulation and multi-organ damage. Using the ATP7B C271X -/- zebrafish model, the company demonstrated dose-dependent improvements across multiple clinically relevant endpoints.
The most significant findings include 4-5x reduction in tremors, normalized motor function, ~50% reduction in copper accumulation in liver tissue, improved liver/kidney histopathology, normalization of key liver biomarkers (ALT, AST, bilirubin), and improved survival under high copper conditions.
What makes these results particularly notable is that Telomir-1 appears to address both the underlying copper regulation dysfunction and reverse established tissue damage. Current Wilson's disease treatments like chelation therapy or zinc supplementation mainly prevent further copper accumulation but don't repair existing damage.
This data expands Telomir-1's potential beyond its previously reported effects in Werner syndrome (where it extended telomeres and reset the epigenetic clock) and Age-Related Macular Degeneration (where it demonstrated retinal regeneration). The consistent regenerative effects across multiple disease models suggest a fundamental mechanism targeting cellular aging and degeneration.
The company plans to file its first IND for a rare disease indication by year-end, with human trials expected in H1 2026. While promising, investors should note these remain preclinical results in animal models, with human efficacy and safety yet to be established.
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 / ACCESS Newswire / June 11, 2025 / 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 tissueMarked 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.
Cautionary Note Regarding Forward-Looking Statements
This press release, statements of Telomir's management or advisors related thereto, and the statements contained in the news story linked in this release contain "forward-looking statements," which are statements other than historical facts made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These risks and uncertainties include, but are not limited to, the potential use of the data from our studies, our ability to develop and commercialize Telomir-1 for specific indications, and the safety of Telomir-1.
Any forward-looking statements in this press release are based on Telomir's current expectations, estimates and projections only as of the date of this release. These and other risks concerning Telomir's programs and operations are described in additional detail in its Annual Report on Form 10-K for the fiscal year ended December 31, 2024, which is on file with the SEC. Telomir explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.
Contact Information
Helga Moya
info@telomirpharma.com
(786) 396-6723
SOURCE: Telomir Pharmaceuticals, Inc
View the original press release on ACCESS Newswire
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