FDA Clears IND for Telomir-Zn in Advanced or Metastatic Triple-Negative Breast Cancer
The FDA has cleared Telomir Pharmaceuticals' IND for Telomir-Zn in advanced or metastatic triple-negative breast cancer. The company plans to initiate a first-in-human Phase 1/2 trial in the first half of 2026.
The U.S. Food and Drug Administration (FDA) has cleared Telomir Pharmaceuticals' Investigational New Drug (IND) application for Telomir-Zn, its lead small-molecule candidate, for the treatment of patients with advanced or metastatic triple-negative breast cancer (TNBC). The company plans to initiate a first-in-human Phase 1/2 clinical trial in the first half of 2026, anchored by a leading U.S. academic medical center.
The FDA's clearance reflects review of a comprehensive IND submission that included pharmacology, toxicology, manufacturing data, and the company's first-in-human Phase 1/2 clinical study protocol (TELO-001). The IND package was supported by completed IND-enabling studies, including pharmacokinetic data demonstrating systemic exposure and preclinical data indicating activity in models of triple-negative breast cancer.
The planned Phase 1/2 trial (TELO-001) is a first-in-human, multicenter, open-label study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of Telomir-Zn as an oral monotherapy in adult patients with advanced or metastatic TNBC who have received prior systemic therapy. The study plans to enroll approximately 76 patients. Phase 1 will use a modified 3+3 dose-escalation design to evaluate safety, tolerability, and dose-limiting toxicities, determine the maximum tolerated dose and recommended Phase 2 dose, and assess pharmacokinetics, pharmacodynamics, and preliminary antitumor activity. Phase 2 will use Simon's two-stage design to assess preliminary efficacy, with objective response rate as the primary endpoint and duration of response, progression-free survival, overall survival, and safety as secondary endpoints.
The study includes an integrated biomarker program to evaluate pharmacodynamic activity, target engagement, and potential predictors of response. Biomarker analyses will assess changes in epigenetic regulation, including global DNA methylation, gene re-expression of epigenetically silenced tumor suppressor pathways, and histone modification patterns, as well as telomere dynamics and associated epigenetic signatures.
Telomir-Zn is an investigational small molecule designed to represent a novel therapeutic approach targeting modulation of intracellular metal homeostasis and downstream epigenetic regulation. Unlike conventional cytotoxic chemotherapy, immune checkpoint inhibition, antibody-drug conjugates, or classic epigenetic drugs, Telomir-Zn is being developed to target metal-dependent epigenetic vulnerabilities that may contribute to tumor progression, treatment resistance, genomic instability, and cancer-associated aging biology. Preclinical studies indicate that Telomir-Zn alters intracellular iron and zinc balance, modulates iron-dependent chromatin-regulating enzymes, and influences gene expression and cellular pathways associated with tumor biology, genomic stability, and cellular aging.
Telomir Pharmaceuticals is a clinical-stage biotechnology company developing small-molecule therapeutics targeting epigenetic and metabolic drivers of cancer and age-related disease. The company is listed on the NASDAQ exchange under the ticker symbol TELO. As of the reported period, it had a market capitalization of $39.19 million, had not yet generated revenue, and carried no long-term debt.