Telomir Pharmaceuticals Confirms Telomir-1 Restores Vision and Retinal Structure in Age-Related Macular Degeneration (AMD) Animal Model Using FDA-Recognized Surrogate Endpoints

Rhea-AI Summary

Telomir Pharmaceuticals (NASDAQ:TELO) announced breakthrough preclinical results for Telomir-1, their oral therapeutic for age-related macular degeneration (AMD). In a 14-day study using a genetically modified zebrafish model, Telomir-1 demonstrated remarkable vision restoration and retinal regeneration capabilities. The study showed significant improvements in multiple FDA-recognized surrogate endpoints. Key results include: restoration of vision and coordinated swimming behavior, complete recovery of inner nuclear layer thickness, improved ganglion cell layer integrity, 50% reduction in reactive oxygen species levels, and zero mortality in treatment groups. The zebrafish model exhibited 15% retinal degeneration pre-treatment, affecting critical retinal layers. This breakthrough represents a potential paradigm shift in AMD treatment, as no oral drug has previously demonstrated such comprehensive retinal restoration and vision recovery in AMD models.

Positive

• Successful restoration of vision and retinal structure in AMD animal model
• Demonstrated 50% reduction in reactive oxygen species (ROS) levels
• Complete recovery of inner nuclear layer thickness and improved ganglion cell layer integrity
• Zero mortality in treatment groups compared to 15% mortality in untreated animals
• Novel oral administration route, potentially offering easier treatment delivery compared to traditional eye injections

Negative

• Results are only from preclinical animal studies, not yet tested in humans
• Requires further clinical trials to prove efficacy and safety in human patients
• Potential regulatory hurdles for novel therapeutic approach

Insights

Ophthalmology and Pharmaceutical Research Expert

Telomir's preclinical AMD results show promising vision restoration in zebrafish, but human efficacy remains unproven with many development hurdles ahead.

Telomir's preclinical study represents an intriguing but very early-stage development in AMD therapeutics. The zebrafish model combining WRN, ND6, and Sen57 mutations creates a reasonable approximation of human AMD pathology, displaying both structural degeneration and functional vision impairment. The reported outcomes after Telomir-1 administration are noteworthy across multiple FDA-recognized surrogate endpoints, particularly the restoration of inner nuclear layer thickness, ganglion cell layer integrity, and plexiform layer structure.

What distinguishes this approach is the oral delivery method, which could offer significant advantages over current AMD treatments requiring invasive intravitreal injections. Additionally, the drug appears to work through a regenerative mechanism rather than merely slowing degeneration, which would represent a substantial therapeutic advancement if replicated in humans.

However, several critical limitations must be considered. First, zebrafish have remarkable innate regenerative capabilities that humans lack, potentially overestimating therapeutic effects. Second, the 14-day treatment window is extremely short, leaving questions about durability and long-term efficacy. Third, the press release lacks detailed information on mechanism of action, dose-response relationships, or pharmacokinetic data that would strengthen scientific credibility.

The reduction in reactive oxygen species (ROS) by up to 50% suggests an antioxidant effect, but this alone doesn't explain the structural regeneration observed. Most importantly, the vast biological differences between zebrafish and human retinas mean these results, while promising, have a high probability of failing to translate to clinical success. The therapeutic landscape for AMD is littered with preclinical successes that failed in human trials.

This represents an interesting technology with potential, but remains many years and numerous clinical hurdles away from commercialization, with significant risk of failure during this lengthy development process.

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 / ACCESS Newswire / May 29, 2025 / 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."

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 Pharmaceuticals' (TELO) AMD drug study?

Telomir-1 demonstrated vision restoration and retinal regeneration in zebrafish AMD models, with complete recovery of retinal layer thickness, 50% reduction in oxidative stress, and improved vision response after 14 days of oral treatment.

How does Telomir-1 differ from current AMD treatments?

Telomir-1 is unique as an oral drug candidate, whereas most current AMD treatments require eye injections. It has shown ability to restore vision and regenerate retinal structure, not just manage symptoms.

What were the specific improvements shown in TELO's AMD animal study?

The study showed restoration of coordinated swimming behavior, recovery of retinal layer thickness, improved ganglion cell layer integrity, 50% reduction in reactive oxygen species, and zero mortality in treatment groups.

What is the current development stage of Telomir-1 for AMD treatment?

Telomir-1 is currently in preclinical development, having shown positive results in zebrafish models. Human clinical trials have not yet begun.

What are the potential advantages of Telomir's (TELO) oral AMD treatment?

As an oral medication, Telomir-1 could offer easier administration compared to current eye injections, while potentially providing both vision restoration and retinal regeneration benefits.