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On Wednesday, Telomir Pharmaceuticals , Inc. (NASDAQ:TELO), a $61 million market cap biotechnology company, disclosed significant findings from a preclinical trial of its leading drug candidate, Telomir-1, in treating Wilson’s disease, a genetic disorder characterized by excessive copper accumulation in the body. According to InvestingPro data, the company’s stock has declined over 60% in the past year, reflecting the challenging environment for early-stage pharmaceutical companies. The study, conducted on an animal model, indicated that Telomir-1 could notably improve neurological, liver, and kidney functions.
Wilson’s disease leads to copper build-up, primarily affecting the liver and brain, which can result in severe tissue damage and organ dysfunction. Current treatment options are limited to copper chelation or liver transplantation. Telomir-1’s preclinical efficacy suggests it might offer a disease-modifying approach, addressing both neurological and hepatic symptoms.
The study revealed that Telomir-1, administered in varying doses, significantly reduced tremors, normalized movement and behavior, and decreased copper levels in the liver. Additionally, the treatment improved liver and kidney histopathology and key liver health markers—ALT, AST, and bilirubin—back to near-normal levels. While InvestingPro analysis indicates the company operates with moderate debt levels and faces short-term liquidity challenges, these results could be crucial for its future prospects. Notably, Telomir-1 also enhanced survival rates under high copper exposure conditions.
These findings underscore the potential of Telomir-1 as a therapeutic agent for Wilson’s disease, offering hope for a treatment that could modify the disease course. Following these promising results, Telomir Pharmaceuticals plans to submit an Investigational New Drug (IND) application by the end of the year and commence human clinical trials in the first half of 2026. Despite current losses, analysts maintain an optimistic outlook with a price target significantly above current levels. Get deeper insights into TELO’s financial health metrics and 8 additional key ProTips with InvestingPro.
This information is based on a press release statement filed with the Securities and Exchange Commission on June 11, 2025.
In other recent news, Telomir Pharmaceuticals announced promising preclinical results for its lead drug candidate, Telomir-1, in various studies targeting different conditions. The company reported significant improvements in reversing Wilson’s disease symptoms in a preclinical animal model, with notable reductions in copper accumulation and improvements in liver and kidney function. Additionally, Telomir-1 showed potential in reversing key aging markers in a study related to Werner Syndrome, indicating its broad therapeutic potential. The drug also demonstrated promising results in treating age-related macular degeneration (AMD (NASDAQ:AMD)), improving vision and retinal structure in an animal model.
Furthermore, Telomir Pharmaceuticals received a $3 million equity investment from The Bayshore Trust, aimed at supporting the submission of an Investigational New Drug (IND) application for Telomir-1 by the end of the year. In a separate development, the company appointed Alan Weichselbaum as its new Chief Financial Officer, highlighting his extensive experience in corporate finance. These recent developments underscore Telomir’s commitment to advancing its drug pipeline and addressing unmet medical needs in various conditions.
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