MIAMI, Florida – Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) (“Telomir” or the “Company”), a biotechnology company developing small-molecule therapeutics targeting epigenetic and metabolic drivers of cancer and age-related disease, today announced the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its lead candidate, Telomir-1 (Telomir-Zn), for the treatment of advanced and metastatic Triple-Negative Breast Cancer (TNBC).
The IND submission includes data from completed IND-enabling pharmacology, toxicology, and manufacturing studies. Subject to IND clearance, the Company plans to initiate a Phase 1/2 clinical trial evaluating Telomir-1 as an oral monotherapy in patients with advanced or metastatic TNBC.
Mechanism and Scientific Rationale
Telomir-Zn is a first-in-class metal-modulating epigenetic agent designed to restore transcriptional control in tumor cells by targeting intracellular iron-zinc homeostasis, an upstream regulatory node governing several histone demethylases (KDMs) activity, mitochondrial function, and oxidative stress response.
Preclinical studies indicate that Telomir-1 reduces intracellular redox-active iron while increasing zinc availability. This shift suppresses iron-dependent KDMs activities, driving accumulation of repressive histone methylation marks, downregulating oncogenic transcriptional programs, and disrupting cancer cell metabolism. The approach does not rely on nonspecific cytotoxic mechanisms, potentially offering a more targeted profile.
Preclinical Efficacy and Safety
Across multiple preclinical TNBC models, Telomir-Zn demonstrated:
- Reduction in tumor growth in aggressive TNBC models
- Reduction in metastatic dissemination in a chemotherapy-resistant setting
- Iron-dependent tumor cell mortality across human TNBC cell lines
- Enhanced activity in combination with chemotherapy in select models
Telomir-Zn has completed IND-enabling GLP safety studies demonstrating:
- No treatment-related adverse or dose-limiting toxicities
- Favorable cardiovascular, respiratory, and phototoxicity profiles
- Consistent systemic exposure and predictable pharmacokinetics
The program is supported by multi-level preclinical studies spanning cellular, in vivo, and safety studies, providing a supportive translational framework for clinical evaluation.
Phase 1/2 Clinical Development Plan
Following IND clearance, the Company plans to initiate a Phase 1/2 clinical study evaluating Telomir-Zn as an oral monotherapy in patients with advanced or metastatic Triple-Negative Breast Cancer.
The Phase 1 portion will utilize a standard 3+3 dose-escalation design to evaluate safety, tolerability, dose-limiting toxicities, and determination of the recommended Phase 2 dose.
The Phase 2 portion will evaluate preliminary antitumor activity using a Simon two-stage design, with objective response rate (ORR) as the primary endpoint, along with duration of response (DoR), progression-free survival (PFS), and continued safety.
The study is designed to identify a clinically meaningful signal of activity.
The Company is also evaluating biomarker strategies aligned with Telomir-Zn’s mechanism of action to support patient selection and clinical response assessment.
The Company is currently in discussions with multiple leading U.S.-based cancer centers regarding participation in the planned clinical program.
Clinical Context and Unmet Need
According to Breastcancer.org, breast cancer remains one of the leading causes of cancer-related death globally, with hundreds of thousands of deaths reported each year.
Triple-Negative Breast Cancer accounts for approximately 10-15% of all breast cancer cases and represents one of the most aggressive subtypes, with limited treatment options and poor clinical outcomes.
In advanced disease, median overall survival remains approximately 11-13 months, with five-year survival rates of approximately 12-15%. Despite available therapies-including chemotherapy, immune checkpoint inhibitors such as Keytruda, and antibody-drug conjugates such as Trodelvy-response rates typically range from 20% to 40%, and most patients either do not respond or experience rapid disease progression.
Therapies that can meaningfully improve response rates or durability of response in TNBC have the potential to significantly impact both patient outcomes and the treatment landscape.
The global market for TNBC therapeutics is estimated to be in the multi-billion-dollar range, based on published market research reports, reflecting the significant unmet need for more effective and durable treatment options.
Management Commentary
“This IND submission marks a critical transition from preclinical proof-of-concept to clinical development for Telomir-Zn,” said Erez Aminov, Chief Executive Officer of Telomir Pharmaceuticals.
“TNBC patients with advanced disease have few durable treatment options, and we believe that targeting the biological mechanisms driving treatment resistance, specifically iron-dependent epigenetic dysregulation, represents a differentiated and scientifically grounded approach. We look forward to advancing this program into the clinic.”
Dr. Itzchak Angel, Chief Scientific Advisor at Telomir Pharmaceuticals, added:
“Epigenetic dysregulation, including aberrant histone modification mediated by iron-dependent KDMs, is a central driver of oncogenic transcriptional programs in aggressive cancers such as TNBC. Our data support a mechanistic framework in which modulating intracellular metal homeostasis resets this dysregulation, enabling transcriptional repression of tumor-supporting gene networks without cytotoxic stress. We are excited to evaluate this biology in patients.”
Next Steps
Subject to IND clearance, Telomir plans to initiate its Phase 1/2 clinical trial in advanced TNBC and continue advancing biomarker-driven development strategies.
In parallel, the Company is continuing to expand its preclinical program, including evaluation of Telomir-Zn in additional TNBC animal models and further characterization of its mechanism of action.
The Company has submitted scientific manuscripts to peer-reviewed journals and plans to present data at scientific conferences, including the AACR Annual Meeting 2026.
About Telomir Pharmaceuticals
Telomir Pharmaceuticals, Inc. (NASDAQ:TELO) is a preclinical-stage biotechnology company developing small-molecule therapeutics designed to target fundamental epigenetic and metabolic mechanisms implicated in cancer, aging, and degenerative disease. The Company’s lead program, Telomir-1 (Telomir-Zn), has demonstrated activity in preclinical studies involving modulation of intracellular metal homeostasis, redox balance, epigenetically regulated gene expression, mitochondrial function, and genomic stability. For more information please visit https://telomirpharma.com/.
Contact Information
Krystina Quintana
Email: [email protected]
Phone: (786) 396-6723