Telomir Pharmaceuticals Announces Telomir-1 Resets the Body's Epigenetic Clock, Reverses DNA Methylation, and Restores Youthful Gene Regulation in an Ultra-Rare Accelerated Aging Animal Model of Werner Syndrome

Rhea-AI Summary

Telomir Pharmaceuticals (NASDAQ:TELO) has announced breakthrough preclinical results for Telomir-1 in treating Werner Syndrome, a rare genetic aging disorder. The study, conducted using a zebrafish model, demonstrated remarkable anti-aging effects including three-fold telomere lengthening, DNA methylation reversal, and complete survival rates. The oral treatment restored muscle mass and body weight in affected animals, which typically show 50-60% reductions, back to normal levels. Additionally, Telomir-1 reduced oxidative stress by up to 50% and achieved 100% survival rate compared to 15% mortality in untreated animals. The drug showed potential in reversing key aging markers by resetting the body's epigenetic clock and restoring youthful gene regulation. These findings build upon previous successful results in C. elegans studies and position Telomir-1 as a promising therapeutic candidate for aging-related diseases.

Positive

• Treatment demonstrated three-fold increase in telomere length, exceeding healthy wild-type levels
• Achieved 100% survival rate in treated groups compared to 15% mortality in untreated animals
• Successfully restored muscle mass and body weight to normal levels after 14 days of treatment
• Reduced oxidative stress levels by up to 50%
• Reversed age-related DNA hypomethylation, restoring methylation patterns above normal levels

Negative

• Still in preclinical phase with no human trials yet
• Results limited to rare disease model (Werner Syndrome)
• No FDA-approved treatments exist for comparison
• Potential risks and long-term effects remain unknown

Insights

Biotechnology Research Scientist

Telomir-1 shows promising anti-aging effects in preclinical zebrafish models, potentially addressing multiple aging hallmarks through epigenetic regulation.

The preclinical data for Telomir-1 reveals compelling effects on reversing aging hallmarks in an ultra-rare disease model. What makes these findings scientifically significant is the multi-faceted impact on biological markers of aging. The ability to restore DNA methylation patterns is particularly noteworthy, as epigenetic dysregulation is a fundamental driver of aging across species. By reversing age-related hypomethylation, Telomir-1 appears to reset gene expression patterns to a more youthful state, potentially affecting hundreds of downstream cellular processes.

The three-fold increase in telomere length exceeding even wild-type levels is remarkable, as most interventions merely slow telomere attrition rather than substantially lengthen them. This suggests Telomir-1 may activate telomerase or alternative lengthening mechanisms in a novel way. The concurrent improvements in physical parameters - body weight, muscle mass, oxidative stress reduction, and 100% survival rate - indicate this isn't merely a molecular marker effect but translates to functional outcomes.

However, several critical scientific questions remain before extrapolating to human aging: First, the Sen57wrn-/-ND6-/+ zebrafish model, while useful, represents an accelerated aging phenotype that may respond differently than natural aging. Second, the reversal of hypomethylation to above normal wild-type levels warrants careful investigation for potential off-target effects, as excessive methylation could silence important genes. Third, the duration of these effects and potential rebound phenomena after treatment cessation remain unknown. Finally, the translation from zebrafish to human biology, particularly regarding telomere regulation and methylation patterns, contains significant uncertainties despite conserved mechanisms.

Pharmaceutical Investment Analyst

Telomir's zebrafish study data strengthens their early-stage anti-aging pipeline, though human trials remain distant with significant hurdles ahead.

These preclinical results significantly strengthen Telomir's scientific foundation by demonstrating multiple mechanisms of action for their lead compound. Most biotech startups struggle to show more than one viable pathway, but Telomir-1 appears to address at least three major hallmarks of aging: telomere attrition, epigenetic alterations, and mitochondrial dysfunction.

The zebrafish Werner Syndrome model provides a more translatable vertebrate system than their previous C. elegans studies, representing a critical step in the development pipeline. The comprehensive improvements across molecular, cellular, and organism-wide parameters suggest potential applications beyond rare diseases into broader age-related conditions - a massive market opportunity if eventually proven in humans.

However, investors should recognize several crucial limitations. Despite the encouraging data, Telomir-1 remains years away from human trials, with significant regulatory hurdles ahead. The press release specifically states they're still in preclinical phases. The zebrafish model, while useful, doesn't predict human efficacy or toxicity with high confidence. Additionally, the rare disease path they're likely pursuing (Werner Syndrome) represents a tiny market (<5,000 patients worldwide), though it provides a regulatory pathway to broader indications later.

The company must still complete IND-enabling studies, including rigorous toxicology, before FDA clearance for first-in-human trials. No details on formulation, dosing, or pharmacokinetics were provided, leaving open questions about how Telomir-1 would be delivered to humans. Financially, early-stage biotechs typically require substantial capital to reach clinical inflection points, so dilution risks remain despite positive preclinical data.

Treatment restored gene control, extended telomere length beyond healthy levels, reversed muscle and weight loss, reduced oxidative stress, and resulted in 100% survival - highlighting Telomir-1's potential to reverse key hallmarks of aging

MIAMI, FL / ACCESS Newswire / June 5, 2025 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO), or the "Company," an emerging leader in age-reversal science, today announced compelling new preclinical data demonstrating that its lead candidate, 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. 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.

Werner Syndrome: A Rare and Devastating Accelerated Aging Disorder

Werner Syndrome is a rare autosomal recessive disorder caused by mutations in the wrn gene, which plays a critical role in DNA repair and telomere maintenance. Patients typically begin showing signs of premature aging-such as graying hair, cataracts, diabetes, osteoporosis, and atherosclerosis-in their 20s or 30s. Median life expectancy is 40-50 years. There are currently no FDA-approved treatments.

To model this disease, the Company's study utilized the Sen57wrn-/-ND6-/+ zebrafish model, which replicates key features of Werner Syndrome, including telomere loss, mitochondrial dysfunction, retinal degeneration, muscle deterioration, and abnormal DNA methylation.

Key Preclinical Findings

1. DNA Methylation Reversal - Resetting the Genetic Clock
DNA methylation is one of the body's key mechanisms for controlling which genes are turned on or off. It works by attaching small chemical tags (called methyl groups) to DNA at locations known as CpG islands-which act like gene "on/off" switches. When methylation patterns are intact, cells know which genes to express and when.

However, with aging and disease, this regulatory system starts to break down - a phenomenon known as epigenetic drift. In these cases, genes that should be off may turn on inappropriately, and protective genes may be silenced. This malfunction in gene regulation is strongly linked to a wide range of chronic diseases including:

• Cancer (via silencing of tumor suppressor genes)

• Neurodegenerative diseases like Alzheimer's and Parkinson's

• Autoimmune diseases such as lupus and multiple sclerosis

• Metabolic conditions like Type 2 diabetes

• Premature aging disorders like progeria and Werner Syndrome

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.

2. Telomere Elongation - Rebuilding the Chromosomal Clock
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, compared with the shortened length in the mutated animals, Telomir-1 increased telomere length by about three-fold. At the higher dose, telomere length significantly exceeded wild-type (healthy) levels, suggesting not only restoration but also potential enhancement of chromosomal integrity

3. Muscle Mass and Body Weight Recovery
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.

4. Oxidative Stress Reduction
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.

5. Survival Benefit
Roughly 15% of untreated animals died during the 14-day study period, whereas no deaths occurred in any Telomir-1 treated groups - highlighting a systemic survival advantage.

"These results reinforce my conviction that Telomir-1 may represent one of the most important scientific developments in the field of aging," said Erez Aminov, Chairman and CEO of Telomir. "While we remain in the preclinical phase, the consistency and strength of the data demand serious attention. My full commitment is behind advancing Telomir-1 toward human studies in the most responsible and rigorous way. The potential here is too significant to ignore."

"This is one of the most comprehensive rejuvenation profiles we've seen in a vertebrate aging model," added Dr. Itzchak Angel, Chief Scientific Advisor. "The ability to reverse both genomic and epigenetic instability while improving survival supports the growing potential of Telomir-1 as a foundational therapy for aging-related diseases."

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 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 safety of Telomir-1. 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

FAQ

What are the key findings of Telomir-1 (TELO) in treating Werner Syndrome?

Telomir-1 showed three-fold telomere lengthening, DNA methylation reversal, restored muscle mass and body weight, 50% reduction in oxidative stress, and 100% survival rate in treated animals.

How does Telomir-1 affect telomere length in the Werner Syndrome study?

Telomir-1 increased telomere length by approximately three-fold, significantly exceeding normal wild-type levels in healthy subjects.

What is Werner Syndrome and how does it affect patients?

Werner Syndrome is a rare genetic disorder causing accelerated aging, with symptoms like premature graying, cataracts, diabetes, and osteoporosis appearing in patients' 20s or 30s, with median life expectancy of 40-50 years.

What was the survival rate improvement in Telomir's Werner Syndrome study?

The study showed 100% survival in Telomir-1 treated groups compared to an 85% survival rate in untreated animals over the 14-day study period.

How does Telomir-1 affect DNA methylation and gene regulation?

Telomir-1 reversed age-related hypomethylation at chromosomal regions, restoring methylation patterns above normal wild-type levels, potentially restoring healthy gene regulation.