Tonix Pharmaceuticals stock halted ahead of FDA approval news
Telomir Pharmaceuticals , Inc. (NASDAQ:TELO) announced positive preclinical data for its lead candidate, Telomir-1, in human progeria cell lines. The tests demonstrated significant cellular protective activity in cells from a child with Hutchinson-Gilford Progeria Syndrome (HGPS). The announcement comes as the company’s stock trades near its 52-week low of $1.86, down significantly from its high of $8.40. According to InvestingPro analysis, the company maintains a market capitalization of approximately $55 million.
The study, conducted by SmartAssays using cells obtained from The Progeria Research Foundation, focused on three critical hallmarks of cellular aging and disease progression in progeria: loss of viability, oxidative stress, and mitochondrial dysfunction.
Key findings showed that Telomir-1 significantly increased cell viability under both normal conditions and in the presence of toxic levels of iron and copper. The compound normalized reactive oxygen species (ROS) that are typically elevated in progeria cells, and reversed calcium overload, a marker of mitochondrial damage.
Progeria is an ultra-rare pediatric disorder affecting fewer than 30 known patients in the United States. It is caused by a mutation in the LMNA gene, leading to rapid biological aging in children with an average life expectancy of 13 to 15 years. The only currently approved treatment, Zokinvy (lonafarnib), extends lifespan by approximately 4.3 years but does not reverse the underlying disease pathology.
These results build on prior studies in zebrafish and C. elegans models of Werner syndrome (adult progeria), where Telomir-1 extended lifespan and reversed various aging markers.
The company is finalizing IND-enabling studies and plans to engage with the FDA to explore regulatory pathways, including potential orphan drug designation, as it evaluates multiple rare disease indications for initial clinical development. InvestingPro data reveals the company faces financial challenges with a current ratio of 0.75, indicating potential liquidity constraints. Despite these challenges, analysts have set an ambitious price target of $15.50, suggesting significant upside potential if clinical development proves successful.
According to the SEC filing, this information was released by the company on Wednesday. For deeper insights into TELO’s financial health and additional investment metrics, InvestingPro subscribers have access to over 10 more exclusive ProTips and comprehensive financial analysis tools.
In other recent news, Telomir Pharmaceuticals has reported significant advancements in the development of its lead drug candidate, Telomir-1. The company announced promising results from a preclinical study of Telomir-1 in treating Wilson’s disease, a genetic disorder characterized by excessive copper accumulation. The study, conducted on an animal model, showed improvements in neurological, liver, and kidney functions. Additionally, Telomir Pharmaceuticals disclosed positive outcomes from a separate preclinical trial targeting Hutchinson-Gilford Progeria Syndrome (HGPS), where Telomir-1 demonstrated improvements in cell survival and reduced oxidative stress. In another study, Telomir-1 showed potential in reversing symptoms of Werner Syndrome by resetting biological aging markers and restoring healthy DNA methylation. The company also reported promising findings in a model of age-related macular degeneration (AMD (NASDAQ:AMD)), where Telomir-1 improved vision and retinal structure. These developments are part of Telomir Pharmaceuticals’ efforts to advance Telomir-1 towards clinical trials, with plans to file an Investigational New Drug (IND) application by the end of the year.
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