EXCLUSIVE: Telomir Pharmaceuticals Says Preclinical Drug Candidate Prevents Premature Aging In Patient-Derived Cells From Children With Progeria

Telomir Pharmaceuticals Inc. TELO on Wednesday announced new preclinical data showing that its lead candidate, Telomir-1, prevented cellular aging in human progeria cell lines obtained from the Progeria Research Foundation.

In this study, conducted by Smart Assays, Telomir-1 was tested in cells taken directly from a child with HGPS.

The study evaluated cell viability, reactive oxygen species (ROS), and intracellular calcium signaling—a marker of mitochondrial dysfunction—under normal and stress-induced conditions.

Also Read: EXCLUSIVE: Telomir Pharma To Raise $3 Million Via Equity Financing To Fund Its Rare Disease IND

Key preclinical findings

Telomir-1 increased survival in a dose-dependent manner, both under basal conditions and even under stress conditions induced by copper and iron—two metal ions known to accelerate aging by generating oxidative damage and destabilizing DNA and telomeres.

Progeria cells exhibited abnormally high levels of reactive oxygen species (ROS), a hallmark of cellular aging. Telomir-1 normalized these levels under basal conditions and even when ROS was further elevated by toxic metal exposure.

Iron-induced calcium overload—a signal of mitochondrial damage and a known feature of HGPS—was significantly reduced with Telomir-1, indicating restored mitochondrial regulation and improved cellular energy balance.

Why It Matters

Progeria, or Hutchinson-Gilford Progeria Syndrome (HGPS), is an ultra-rare pediatric disorder caused by a mutation in the LMNA gene. This mutation produces an abnormal progerin protein, which drives rapid biological aging in children.

The only FDA-approved therapy for progeria, Zokinvy (lonafarnib), is a farnesyltransferase inhibitor that has been shown to extend lifespan by an average of 4.3 years.

The company says that Zokinvy does not reverse the underlying disease pathology or halt cardiovascular deterioration, which remains the leading cause of death.

Telomir-1 is designed to regulate intracellular metal ions, reduce oxidative stress, restore mitochondrial function, extend telomere length, reverse muscle loss, and reset age-associated DNA methylation patterns—critical biological pathways implicated in progeria and broader age-related diseases.

The new data also build on previously reported studies in zebrafish and C. elegans nematodes harboring the wrn gene mutation (a model of adult progeria, or Werner syndrome), where Telomir-1 significantly extended lifespan, restored telomere length, reversed muscle degeneration, and normalized molecular age markers.

Telomir is currently finalizing IND-enabling studies for Telomir-1 and plans to engage with the U.S. Food and Drug Administration (FDA) to explore regulatory pathways, including the potential for orphan drug designation. The company is evaluating multiple rare disease indications for initial clinical development.

Price Action: TELO stock traded higher by 2.69% to $1.91 premarket at the last check on Wednesday.

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