Telomir Pharmaceuticals Inc.

06/11/2025 | Press release | Distributed by Public on 06/11/2025 07:31

Material Event (Form 8-K)

Item 8.01 Other Events

Telomir Pharmaceuticals Announces Telomir-1 Demonstrates Dose-Dependent Restoration of Neurological, Liver and Kidney Function in Wilson's Disease Animal Model

On June 11, 2025, Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) announced new preclinical data demonstrating that its lead small molecule candidate, Telomir-1, significantly improved neurological, behavioral, liver- and kidney-related outcomes in a clinically relevant animal model of Wilson's disease (ATP7B C271X -/- zebrafish).

Wilson's disease is a rare and potentially life-threatening genetic disorder caused by mutations in the ATP7B gene, which impair the body's ability to eliminate excess copper. This results in progressive copper accumulation-primarily in the liver and brain-causing inflammation, tissue damage, and organ dysfunction over time. Patients may experience liver failure, movement disorders, cognitive decline, and psychiatric symptoms. If left untreated, the condition can be fatal. Current therapies involve lifelong copper chelation or liver transplantation, underscoring the need for safer, disease-modifying treatments.

In this preclinical study, Telomir-1 demonstrated dose-dependent and statistically significant reversal of multiple disease-relevant endpoints in the Wilson's disease model:

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 behaviors, including abnormal body bends and turn angles
Approximately 50% reduction in copper accumulation in dry liver tissue
Significant and dose-dependent improvement in liver and kidney histopathology, with liver and kidney scores reduced to near-normal levels
Normalization of ALT, AST, and bilirubin-three critical markers of liver health. ALT and AST are enzymes that spike in response to liver injury, while bilirubin is a waste product that builds up when the liver cannot properly filter toxins. Elevated levels of all three are commonly seen in Wilson's disease due to copper-induced liver damage. Telomir-1 restored these values to levels indistinguishable from wild-type animals, indicating protection of liver function and improved detoxification capacity.
Improved survival under high copper exposure conditions

The study demonstrated that Telomir-1 produced a statistically significant and dose-dependent reversal of neurological dysfunction, liver and kidney damage, copper accumulation, and mortality in a clinically relevant genetic model of Wilson's disease. These findings support Telomir-1's potential as a disease-modifying treatment targeting both hepatic and neurological manifestations of copper toxicity and justify further development toward human studies in rare genetic disorders involving copper dysregulation.

Telomir Pharmaceuticals is operating at full speed and currently advancing Telomir-1 toward an IND submission in a rare disease indication by year-end, with human clinical trials planned for the first half of 2026.

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