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Telomir Pharmaceuticals Demonstrates Telomir-1 Reverses Key Drivers of Cellular Decline in Human Cell Lines Supporting Therapeutic Potential in Autism and Spasmodic Dysphonia
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
[email protected]
(786) 396-6723
SOURCE: Telomir Pharmaceuticals, Inc
View the original press release on ACCESS Newswire
P.Smith--AT