- A unique oral drug candidate that is shown to restore vision and regenerate the retina in an animal model, addressing a major unmet need in ocular therapeutics
MIAMI, Florida – Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) (“Telomir” or the “Company”), an emerging leader in age-reversal science, today announced compelling preclinical results from a study evaluating its novel oral therapeutic, Telomir-1, in a genetically modified zebrafish model of age-related macular degeneration (AMD). Following a 14-day oral dosing regimen, Telomir-1 reversed central vision response and vision acuity, restored retinal degeneration and architecture, and significantly reduced oxidative stress-achieving improvements across several FDA-recognized surrogate endpoints relevant to AMD.
The study utilized the Sen57wrn-/-ND6-/+ zebrafish model, which combines genetic mutations associated with premature aging (WRN), mitochondrial dysfunction (ND6), and chronic senescence (Sen57). These animals exhibit progressive retinal degeneration, visual impairment, and oxidative stress-closely modeling dry AMD and geographic atrophy in humans.
Model Transformation: From Degeneration to Recovery
Before treatment, the aged (18-month-old) zebrafish demonstrated clear signs of neurodegeneration and visual impairment. Mutant animals showed sluggish, uncoordinated swimming behavior and delayed responses to visual stimuli such as light and movement-evidence of significant vision loss.
Microscopic analysis of their retinas revealed approximately 15% total retinal degeneration, affecting several critical layers:
- The inner nuclear layer (INL) was thinned. This layer acts as the retina’s central processing zone, where signals from light-sensitive photoreceptors are refined before being passed deeper into the eye.
- The ganglion cell layer (GCL) was degraded. This layer contains neurons that form the optic nerve, which sends visual information to the brain. Damage here disrupts vision at its source.
- The inner plexiform layer (IPL) volume was reduced. IPL is a retinal layer located between the inner nuclear layer (INL) and the ganglion cell layer (GCL). It may contribute to disease processes through inner retinal changes, inflammation, or vitreoretinal interactions.
- The outer plexiform layer (OPL) showed early deterioration. This layer connects photoreceptors to other retinal cells and is essential for detecting changes in light and contrast.
In addition to retinal damage, the diseased animals exhibited reactive oxygen species (ROS) levels nearly four times higher than healthy controls-indicating intense oxidative stress-and suffered a 15% mortality rate during the two-week study window.
After receiving Telomir-1, treated animals demonstrated marked recovery:
- The zebrafish resumed active, coordinated swimming and responded significantly better to light and movement, reflecting restored vision.
- Retinal architecture was structurally restored, including:
- Full recovery of INL thickness, restoring core signal processing in the retina.
- Improved GCL integrity, reactivating the transmission of visual information to the brain.
- Significant augmentation of IPL size, indicating improved processing of visual signals
- Improvement of OPL structure, maintaining input from photoreceptors.
- ROS levels were reduced by up to 50%, and no mortality occurred in any Telomir-1 treatment group.
Histological cross-sections confirmed Telomir-1’s ability to regenerate not only the inner retinal layers, but also additional retinal structures-supporting improved-laminar retinal restoration and function.
Collectively, these results demonstrate Telomir-1’s ability to restore visual function, reverse retinal degeneration, reduce oxidative stress, and improve survival-all from a short oral treatment regimen.
“This breakthrough reinforces our vision at Telomir: to redefine how we treat age-related diseases by going beyond symptom management and targeting the root mechanisms of degeneration,” said Erez Aminov, Chief Executive Officer of Telomir. “To our knowledge, no oral drug has ever demonstrated this level of retinal restoration and vision recovery in any AMD model-this is a meaningful leap forward for patients and the field.”
“The preclinical success achieved in this AMD model is truly remarkable,” added Dr. Angel, Chief Scientific Advisor of Telomir. “Telomir-1 when studied orally, restored both structure and function in the retina, demonstrating not just neuroprotection, but true regenerative capacity-a property rarely seen in ophthalmic drug development.”
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.
Contact Information
Helga Moya
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(786) 396-6723