We are a pre-clinical biotechnology company developing an oral small molecule to treat the side effects of cancer treatment and age-related conditions. (Incorporated in Florida)

We are a pre-clinical-stage pharmaceutical company focused on the development and commercialization of TELOMIR-1, a novel small molecule being developed to function as an oral in situ therapeutic treatment for human stem cells. Based on our pre-clinical studies and if approved by the FDA and comparable foreign regulators, we believe that TELOMIR-1 may potentially serve as a metal enzyme inhibitor of essential metals such as zinc and copper. These essential metals play an important role in the production and function of many enzymatic reactions and the modulation of key cellular pathways. In particular, zinc is essential to the function of pro-inflammatory cytokines such as Interleukin-17, or IL-17, that play a role in a host of age-related inflammatory conditions such as hemochromatosis and osteoarthritis as well as in post-chemotherapy health problems. IL-17 is a type of pro-inflammatory cytokine, and while the pro-inflammatory properties of IL-17 are key to its host-protective capacity, unrestrained IL-17 signaling is associated with immunopathology, inflammatory disease and cancer progression. Our initial focus will be on treatments to inhibit the production of pro-inflammatory cytokines, such as IL-17, by oral administration of TELOMIR-1 as a therapeutic treatment for stem cells in situ. To the best of our knowledge, there is no approved oral IL-17 inhibitor. Our goal is to advance the clinical development of TELOMIR-1 in the United States for the treatment of age-related inflammatory conditions such as hemochromatosis and osteoarthritis, as well as in post-chemotherapy recovery, with our initial targeted indications being hemochromatosis and post-chemotherapy recovery.

Pluripotent stem cells are a type of stem cells that have the ability to undergo self-renewal and to give rise to various cell types of the tissues of the body. Based on pre-clinical studies, we believe that TELOMIR-1 may have the potential to protect stem cells in situ by reducing the overload of metals such as zinc and copper that accompany age-related inflammatory conditions and certain cancers by modulating pro-inflammatory cytokines such as IL-17. If demonstrated by future clinical trials and approved by the FDA and comparable foreign regulators, we believe TELOMIR-1 may protect the stem cells by elongating and stimulating the telomeres to sustain self-renewal of stem cells. Telomeres are repetitive DNA sequences at the end of chromosomes that protect the chromosomes from becoming frayed or tangled. Each time a cell divides, the telomeres become slightly shorter, and eventually they become so short that the cell can no longer divide, with the result being that the cell dies. Effectively, telomeres protect the ends of our chromosomes by forming a cap, much like the plastic tip on shoelaces, thereby allowing the chromosome to be replaced properly during cell division.

TELOMIR-1 is currently under investigation to provide a potential therapeutic intervention against age-related inflammatory conditions such as hemochromatosis, as well as for post-chemotherapy recovery, by interrupting and preventing the IL-17 induced inflammatory pathways that create the systemic imbalance of cellular metals. Our pre-clinical studies suggest that TELOMIR-1 may inhibit the concentration and accumulation of metals such as zinc and copper in the serum that drive the hyperactivity of pro-inflammatory cytokines such as IL-17. Our studies suggest that TELOMIR-1 may achieve this outcome by selectively binding to metal ions in a dose dependent manner, slowing enzyme reactivity, and protecting and lengthening telomeres in the human chromosome. If demonstrated in clinical trials and approved by the FDA and comparable foreign regulators, we believe that TELOMIR-1 has potential as a non-toxic oral enzyme inhibitor that may regulate the overactivity of the enzymes caused by excessive metal reactivity.

We were organized as a Florida corporation in August 2021 for the purpose of pursuing the development and commercialization of TELOMIR-1 in the United States in human applications. We were originally incorporated under the name “Metallo Therapies Inc.” and changed our name to “Telomir Pharmaceuticals, Inc.” in October 2022. We are an early-stage company that had net losses of $0.14 million and $0.85 million for the years ended December 31, 2021 and 2022, respectively.

In Situ Therapeutic Treatment of Stem Cells

Stem cells have the potential to renew themselves. They can develop into many different cell types in the body during early and adult life. Pluripotent stem cells have the ability to differentiate into all of the cells of the adult body. Since pluripotent stem cells are undifferentiated, they do not have any tissue-specific characteristics that allow them to perform specialized functions. Given their unique regenerative abilities and limited quantities in the adult human body, in situ treatment and protection of stem cells may provide an important therapeutic mechanism for treatment of disease.

Metal Overload and Telomere Length

Research studies have demonstrated that metal overload in stem cells could severely impair the proliferation of pluripotent stem cells through excessive DNA damage. Furthermore, many pro-inflammatory cytokine-induced conditions create an imbalance of cellular metals such as zinc and copper. This metal imbalance may impact the length of telomeres in stem cells and impact their ability to sustain self-renewal.

Hemochromatosis

According to the CDC, hemochromatosis is a disorder in which the body builds up too much iron in the skin, heart, liver, pancreas, pituitary glands, and joints. This overload of iron is toxic to the body, and over time, the high levels of iron can damage tissues and organs and lead to conditions such as liver damage, liver cancer, heart problems, arthritis, and diabetes. Other conditions associated with high iron levels include inflammatory conditions, chronic kidney disease, and autoimmune disorders. Hemochromatosis is a life-long condition requiring regular treatment to avoid long-term serious effects with poor pharmacologic options. The most used treatment for hemochromatosis is phlebotomy, a procedure to remove some of the patient’s blood. Phlebotomy is relatively inexpensive, well accepted, and well tolerated, but it requires regular visits to health care professionals and blood draws and may not be appropriate for all patients. Additionally, iron chelators, which are molecules that bind to iron to help the body excrete it, are sometimes used, but they have limited use due to gastrointestinal and kidney toxicity issues. Iron chelators can be natural or synthetic and are used to treat iron overload, which can occur after regular blood transfusions. Despite the presence of these potential treatment options, we believe significant unmet need remains such that an innovative therapy achieving reduction in iron overload with a new profile could be subject to widespread use.

We are pursuing TELOMIR-1 as a metal enzyme inhibitor that, if approved by the FDA and comparable foreign regulators, may selectively bind metal ions to inhibit pro-inflammatory cytokines such as IL-17 and protect telomeres and that aims to increase the average telomere length, or ATL, by selectively binding iron and reducing blood iron levels, therefore supporting the ability to treat hemochromatosis. On average, about 750,000 US patients express one or more iron overload symptoms. There are 2 types of hemochromatosis, with the following patient mix: 150,000 primary hemochromatosis and 65,000 secondary hemochromatosis confirmed diagnoses in the United States. Over 150,000 patients have sought treatment since 2018, with most receiving phlebotomy, which is the withdrawal of blood to bring iron to normal levels. At present, 99% of hemochromatosis patients receive phlebotomy, with a portion of patients also receiving iron chelators as a co-treatment. Note that in the last 12 months, there have been 76,000 confirmed diagnoses of hereditary hemochromatosis and approximately 1% of hemochromatosis patients received deferasirox or deferoxamine off-label for 12 weeks (1 year for primary).

Certain sub-groups of hemochromatosis patients may benefit from TELOMIR-1, in addition to phlebotomy, which is the current standard of care for the treatment of hemochromatosis, if contraindicated for this treatment method. Lastly, iron chelation is used off-label, poorly tolerated and has toxic long-term effects. In pre-clinical stage testing, TELOMIR-1 may have more effective metal complexing than Doxycycline, an FDA-approved metalloproteinases (“MMP”) inhibitor. However, pre-clinical data may prove inaccurate and is not necessarily indicative of future results. MMP inhibitors act as “brakes” for enzymes in our body. MMP enzymes sometimes become too active and break down compounds and structures they should not, such as the body’s tissues. MMP inhibitors are compounds that typically slow down or stop these overactive enzymes, preventing them from causing damage to our tissues. They act as a control switch to keep the body in balance and protect it from harm.

Post-Chemotherapy Recovery

Post-chemotherapy recovery from adverse effects of antineoplastic treatments is often important for cancer therapy success. While chemotherapy treatment can be highly effective for cancer, it can also come with many side effects, as chemotherapy drugs destroy both cancerous and healthy cells. We are investigating the use of TELOMIR-1 as a potential complementary treatment for patients receiving chemotherapy in the form of a twice daily, oral regimen to inhibit pro-inflammatory cytokines and to reduce blood iron levels, enabling potentially more effective adherence and improved outcomes. In exploratory early discovery studies, proof of concept was demonstrated, and the animal studies are pending. Post-chemotherapy recovery space contains several treatments that are used symptomatically based on the nature or severity of side effects.

We believe that post-chemotherapy recovery presents several potential unmet commercial needs for TELOMIR-1. There is potential to enable a more rapid recovery of patients receiving chemotherapy, improving adherence with chemotherapy regimens. Additionally, as current recovery management is based on severity and treated as needed, we believe that TELOMIR-1 could potentially be used as a preventative measure. As a complementary treatment, side effect reduction in post-chemotherapy is important to minimize patient burden while improving treatment outcomes.

Note: Net loss and revenue figures are for the year that ended Dec. 31, 2022.

(Telomir Pharmaceuticals filed its S-1 on Nov. 14, 2023, without disclosing terms for its IPO.)