Telomir Pharmaceuticals Demonstrates Telomir-1 Reverses Key Drivers of Cellular Decline in Human Cell Lines Supporting Therapeutic Potential in Autism and Spasmodic Dysphonia

Rhea-AI Summary

Telomir Pharmaceuticals (NASDAQ:TELO) has announced promising preclinical data for its oral drug candidate, Telomir-1, demonstrating its ability to reverse cellular decline in human cell lines. The drug showed significant improvements in cell viability, mitochondrial function, oxidative stress reduction, calcium signaling, and protection from metal-induced toxicity. Based on these results, Telomir is expanding research into autism spectrum disorder (ASD) and spasmodic dysphonia (SD). The company plans to participate in the FDA's Rare Disease Endpoint Advancement (RDEA) Pilot Program to support development in rare diseases like Progeria and Wilson's disease. The research particularly shows promise for ASD, which affects 1 in 36 children in the US, and SD, which impacts approximately 50,000 Americans.

Positive

• Preclinical data shows Telomir-1 successfully reverses multiple hallmarks of cellular decline
• Potential expansion into large market opportunities: autism (1 in 36 children in US) and spasmodic dysphonia (50,000 Americans)
• Participation in FDA's RDEA Pilot Program could accelerate rare disease drug development
• Multiple potential therapeutic applications including Progeria and Wilson's disease

Negative

• Still in early preclinical stage with only in vitro data
• No FDA-approved treatments currently exist for the targeted conditions, indicating potential regulatory challenges
• Complex development pathway targeting multiple diseases may require significant resources

Insights

Biotechnology Research Scientist

Telomir-1 shows promising cellular effects in laboratory studies but remains years from clinical use despite expanding disease targets.

Telomir Pharmaceuticals has released in vitro data demonstrating that Telomir-1 affects multiple cellular mechanisms linked to aging and disease. The studies show the compound improves cell viability, boosts mitochondrial function, reduces oxidative stress, restores calcium signaling, and protects against metal toxicity in human cell lines.

These findings represent very early-stage research. Laboratory cell studies are merely the first step in drug development, followed by disease-specific models, animal studies, and multiple phases of human clinical trials—a process typically requiring years and having high failure rates.

The company's expansion into autism spectrum disorder and spasmodic dysphonia appears scientifically plausible based on shared cellular mechanisms, but represents a significant leap without direct evidence in disease-specific models. While ASD has documented telomere shortening and mitochondrial dysfunction that align with Telomir-1's effects, the connection to SD is more tenuous, as the release acknowledges telomere involvement hasn't been directly studied in this condition.

The planned participation in the FDA's Rare Disease Endpoint Advancement Pilot Program suggests a strategic pivot toward orphan indications like progeria and Wilson's disease, which could benefit from accelerated pathways and market exclusivity. However, pursuing multiple indications simultaneously at this preliminary stage raises questions about development focus and resource allocation.

Neurological Disease Specialist

Early cellular research shows theoretical mechanisms for Telomir-1 in neurological conditions but lacks disease-specific evidence or clinical validation.

The cellular processes targeted by Telomir-1—mitochondrial dysfunction, oxidative stress, and calcium dysregulation—are indeed implicated in both autism and spasmodic dysphonia pathology. However, we must distinguish between targeting general cellular mechanisms and demonstrating actual efficacy in these specific conditions.

For autism spectrum disorder, affecting 1 in 36 children in the US, the current therapeutic landscape focuses almost exclusively on behavioral interventions. The biological abnormalities mentioned (shortened telomeres, mitochondrial dysfunction) provide reasonable mechanistic rationale, but many compounds that correct cellular abnormalities in laboratories fail to translate to clinical benefits in complex neurological conditions.

The connection to spasmodic dysphonia appears more speculative. This rare voice disorder, affecting approximately 50,000 Americans, currently relies on botulinum toxin injections for temporary symptom management. The press release acknowledges that telomere involvement hasn't been directly studied in SD, making this application more hypothetical.

The rare disease direction makes strategic sense given the regulatory advantages, particularly for progeria where accelerated aging and telomere biology are directly linked. For Wilson's disease, Telomir-1's protection against metal toxicity provides an interesting angle, though specific evidence in copper overload models would be needed to substantiate this approach.

These findings establish a foundation for further research but remain many steps removed from clinical application or proof of therapeutic benefit.

MIAMI, FLORIDA / ACCESS Newswire / May 7, 2025 / Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) ("Telomir"), a leader in age-reversal science, today announced new preclinical data showing that its lead oral drug candidate, Telomir-1, reverses multiple hallmarks of cellular decline across several human cell lines. The findings include improved mitochondrial activity, reduced oxidative stress, restored calcium balance, and protection from toxic metal effects-offering a mechanistic foundation for the Company's new research initiatives in autism spectrum disorder (ASD) and spasmodic dysphonia (SD).

The in vitro studies, conducted in collaboration with SmartAssays, demonstrated Telomir-1's ability to:

• Improve cell viability under stress, particularly in dividing cell populations.
  Maintaining cell survival is critical for tissue repair, immune defense, and slowing the progression of degenerative conditions.

• Boost mitochondrial function, indicating stronger energy production.
  Healthy mitochondria are essential for powering cells and preventing energy loss that drives aging and diseases like Alzheimer's, Parkinson's, and autism.

• Reduce reactive oxygen species (ROS), limiting oxidative damage.
  Excess ROS contributes to DNA damage, inflammation, neurodegeneration and cell death, all of which accelerate aging and chronic diseases.

• Restore calcium signaling, helping maintain healthy cellular functions and communication.
  Disrupted calcium balance impairs brain, muscle, and heart function and is a known trigger of cellular death and neurodegeneration.

• Protect cells from metal-induced toxicity, including iron and copper-both linked to ROS formation, calcium dysregulation and accelerated cellular aging and death.
  Iron and copper accumulation drive oxidative stress and mitochondrial failure.

"This is a strong demonstration of Telomir-1's ability to restore core cell functions under conditions that mimic aging and stress," said Erez Aminov, CEO of Telomir. "These biological improvements give us confidence to pursue exploratory models in autism and spasmodic dysphonia, where these same cellular disruptions are widely documented and involved in disease initiation or progression."

Scientific Rationale for Expanding into Autism and Spasmodic Dysphonia

The cellular mechanisms targeted by Telomir-1-oxidative stress, mitochondrial dysfunction, calcium imbalance, and metal-induced toxicity-have been demonstrated in proliferating cells and may be directly relevant to both autism and spasmodic dysphonia (SD).

• Autism spectrum disorder (ASD) affects 1 in 36 children in the United States. While current therapies focus on behavioral symptoms, there are no FDA-approved treatments that address the underlying cellular biology. Research has shown that individuals with ASD exhibit shortened telomeres, mitochondrial dysfunction, oligodendrocytes, myelination dysfunctions and elevated oxidative stress-biological disruptions Telomir-1 was specifically designed to target.

• Spasmodic dysphonia (SD) is a rare neurological voice disorder affecting about 50,000 Americans. It causes involuntary spasms in the vocal cords and gained public awareness when Robert F. Kennedy Jr. disclosed his diagnosis. SD is currently treated with repeated botulinum toxin injections, offering only temporary relief. While telomere shortening has not yet been directly studied in SD, the condition is marked by oxidative stress, neuroinflammation, metal ion accumulation and neurodegeneration-factors known to accelerate telomere attrition and neurodegeneration in other related neurological diseases.

"What's compelling is that Telomir-1 isn't just addressing one pathway-it's restoring balance, particularly in dividing cells, across multiple core stress systems that overlap with what has been reported in autism and SD," said Dr. Angel, Chief Scientific Advisor. "That's the foundation for our next stage of research."

Regulatory Strategy and Rare Disease Development

To support its rare disease pipeline, Telomir plans to participate in the FDA's Rare Disease Endpoint Advancement (RDEA) Pilot Program, which enables early regulatory dialogue on trial endpoints for underserved conditions. This initiative will support further development of Telomir-1 in rare diseases such as:

• Progeria, a genetic disorder characterized by accelerated aging and early mortality, where telomere shortening is a key pathogenic driver

• Wilson's disease, a rare metabolic disorder involving copper buildup in tissues, where Telomir-1's protective effects against metal-induced toxicity may offer preclinical therapeutic relevance

About Telomir Pharmaceuticals, Inc.

Telomir Pharmaceuticals, Inc. (Nasdaq:TELO) is a pre-clinical stage pharmaceutical company seeking to lead development in several areas, including age-reversal science. Telomir is focused on the development of Telomir-1, a novel small molecule metal ion regulator designed to lengthen the DNA's protective telomere caps, which are crucial in the aging process. Telomir's goal is to explore the potential of Telomir-1, starting with ongoing research in animals and then in humans.

Telomeres are the protective end caps of a chromosome made up of DNA sequences and proteins. As humans age, telomeres shorten, with metal reactivity accelerating the process, which presents humans and pet animals with an increased chance of contracting a number of degenerative and age-related diseases. Telomir's goal is to develop and gain regulatory approval for Telomir-1, proposed to be dosed orally, with the broader aim of promoting longevity and enhancing overall quality of life.

Telomir-1 is in preclinical development and has not yet been tested in humans. There is no assurance that Telomir-1 will proceed through development or will ultimately receive FDA approval for marketing.

Cautionary Note Regarding Forward-Looking Statements

This press release, statements of Telomir Pharmaceuticals' 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 TELO's Telomir-1 preclinical studies?

Telomir-1 demonstrated improvements in cell viability, mitochondrial function, reduced oxidative stress, restored calcium signaling, and protection from metal-induced toxicity in human cell lines.

What medical conditions is Telomir Pharmaceuticals (TELO) targeting with Telomir-1?

Telomir is targeting autism spectrum disorder (ASD), spasmodic dysphonia (SD), and rare diseases including Progeria and Wilson's disease.

How many people could benefit from TELO's treatment for autism and spasmodic dysphonia?

The treatment could potentially benefit 1 in 36 children in the US with autism spectrum disorder and approximately 50,000 Americans with spasmodic dysphonia.

What is Telomir's (TELO) regulatory strategy for drug development?

Telomir plans to participate in the FDA's Rare Disease Endpoint Advancement (RDEA) Pilot Program to support early regulatory dialogue on trial endpoints for underserved conditions.

What makes Telomir-1's approach unique for treating autism and spasmodic dysphonia?

Telomir-1 targets multiple cellular mechanisms simultaneously, including oxidative stress, mitochondrial dysfunction, calcium imbalance, and metal-induced toxicity, which are documented in both conditions.