EXCLUSIVE: Telomir Pharmaceuticals Lead Drug Shows Promising Signs In Animal Study For Rare Premature Aging Disorder

Telomir Pharmaceuticals Inc. TELO on Thursday released new preclinical data for its lead candidate, Telomir-1.

The data demonstrated that Telomir-1, administered orally, significantly increases telomere length, reverses body weight and muscle loss, and resets cellular aging markers in a validated animal model of Werner Syndrome (WS)—a rare genetic disorder also known as adult-onset progeria.

Adult progeria is a rare genetic disorder characterized by premature aging starting in late adolescence or early adulthood.

Also Read: EXCLUSIVE: Telomir Pharma To Raise $3 Million Via Equity Financing To Fund Its Rare Disease IND

Werner Syndrome is caused by mutations in the wrn gene, which plays a critical role in DNA repair and telomere maintenance.

These findings confirm and build upon the company's previously reported results from a preclinical C. elegans study, which demonstrated that Telomir-1 restored lifespan and normalized physiological decline in animals with a wrn gene mutation.

Key Preclinical Findings

In this study, Telomir-1 reversed age-related hypomethylation at two chromosomal regions, restoring methylation patterns to above-normal wild-type levels.

This suggests that Telomir-1 may help restore healthy gene regulation and reset the body's epigenetic aging clock, reducing the risk of dysfunction in key biological systems.

Telomeres are the protective caps at the ends of chromosomes that shorten with each cell division and under stress. Telomere shortening is considered one of the hallmarks of aging.

In the study, Telomir-1 increased telomere length by about three-fold compared to the shortened length in the mutated animals. At the higher dose, telomere length significantly exceeded wild-type (healthy) levels, suggesting restoration and potential enhancement of chromosomal integrity.

In the wrn-mutant zebrafish model, animals exhibited a 50–60% reduction in body weight and muscle volume. After 14 days of Telomir-1 treatment, these physical markers were restored to levels statistically indistinguishable from healthy controls — indicating functional recovery and metabolic improvement.

Reactive oxygen species (ROS), which damage cells and accelerate aging, were elevated in untreated animals. Telomir-1 reduced ROS levels by up to 50%, suggesting improved cellular resilience.

Roughly 15% of untreated animals died during the 14-day study period, whereas no deaths occurred in any Telomir-1-treated groups.

In March, Telomir Pharmaceuticals revealed preclinical findings demonstrating that Telomir-1 reduces tumor size by approximately 50% in a prostate cancer animal model with aggressive cancer cells.

The company says that Telomir-1 actively suppresses cancer growth and protects against chemotherapy-induced toxicity and mortality when combined with Paclitaxel—a widely used chemotherapy but often associated with severe toxicity and side effects.

Price Action: TELO stock closed at $2.11 on Wednesday.

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