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MIAMI - Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), a $61 million market cap biotechnology company currently rated as having WEAK financial health according to InvestingPro, announced Wednesday that its lead drug candidate, Telomir-1, demonstrated significant reversal of neurological, liver and kidney symptoms in a preclinical animal model of Wilson’s disease.
In the ATP7B C271X -/- zebrafish model, which mimics human Wilson’s disease, the drug showed dose-dependent improvements including reduced tremors, normalized swimming behavior, and approximately 50% reduction in copper accumulation in liver tissue.
The treatment also improved liver and kidney histopathology and normalized key liver biomarkers ALT, AST, and bilirubin, according to the company’s press release statement. While these results are promising, InvestingPro data shows the company faces financial challenges with short-term obligations exceeding liquid assets and a current ratio of 0.75.
Wilson’s disease is a rare genetic disorder caused by mutations in the ATP7B gene that impair the body’s ability to eliminate excess copper, leading to copper buildup primarily in the liver and brain. Current treatments involve lifelong copper chelation or liver transplantation.
"These recent findings establish Telomir-1 as a potent disease-modifying compound in a clinically relevant model of Wilson’s disease," said Dr. Angel, Chief Scientific Advisor at Telomir.
The company reports that these results build on previous studies showing Telomir-1’s effects in other conditions including Werner syndrome and Age-Related Macular Degeneration.
Telomir Pharmaceuticals is advancing the drug 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.
The preclinical-stage biotechnology company focuses on developing treatments for biological aging and degenerative diseases.
In other recent news, Telomir Pharmaceuticals has announced promising preclinical results for its lead compound, Telomir-1, in an animal model of Werner Syndrome, highlighting its potential in reversing key aging markers. The study demonstrated significant improvements, including increased telomere length and reduced oxidative stress, with all treated animals surviving. Additionally, Telomir Pharmaceuticals reported breakthrough findings in a preclinical study on age-related macular degeneration (AMD), showing significant improvements in vision and retinal structure in an animal model, supporting further development towards an Investigational New Drug submission.
In corporate developments, the company appointed Alan Weichselbaum as the new Chief Financial Officer, following the departure of Michelle Yanez. Weichselbaum brings extensive experience in corporate finance and capital markets to his new role. Financially, Telomir Pharmaceuticals secured a $3 million equity investment from The Bayshore Trust, its largest shareholder, at a premium over the closing share price. This investment is aimed at supporting the company’s operational goals, including the submission of an IND application for Telomir-1 by year-end. The company also plans to advance Telomir-Ag2, a compound with antimicrobial properties, in its development pipeline.
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