UNITED STATES
SECURITIES AND
EXCHANGE COMMISSION
Washington, D.C.
20549
Form 10-K
For the fiscal year ended
December 31, 2025
For the transition period from ________to_________
Commission file number
001-41952
Telomir
Pharmaceuticals, Inc.
(Exact name of registrant
as specified in its charter)
(State or other jurisdiction of (I.R.S. Employer
incorporation or organization) Identification No.)
(Address of principal executive offices) (Zip Code)
Registrant’s
telephone number, including area code: 786-396-6723
Securities registered pursuant
to Section 12(b) of the Act:
Title of each class Trading Symbol(s) Name of exchange on which registered
Common stock, no par value TELO The Nasdaq Capital Market
Securities
registered pursuant to Section 12(g) of the Act: None
Indicate by check mark if the registrant is a well-known seasoned
issuer, as defined in Rule 405 of the Securities Act.
Yes ☐No☒
Indicate by check mark if the registrant is not required to file reports
pursuant to Section 13 or Section 15(d) of the Act.
Yes ☐No☒
Indicate by check mark
whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during
the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject
to such filing requirements for the past 90 days.
Yes☒ No ☐
Indicate by check mark
whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation
S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit
such files).
Yes☒
No ☐
Indicate by check mark whether
the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging
growth company. See definition of “large accelerated filer,” “accelerated filer,” “smaller reporting company,”
and “emerging growth company” in Rule 12b-2 of the Exchange Act.
Large accelerated filer ☐ Accelerated filer ☐
Non-accelerated filer ☒ Smaller reporting company ☒
Emerging growth company ☒
If an emerging growth company,
indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised
financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark
whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness of its internal
control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting
firm that prepared or issued its audit report. ☒
If securities are registered
pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant in the filing reflect
the correction of an error to previously issued financial statements. ☐
Indicate by check mark
whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by
any of the registrant’s executive officers during the relevant recovery period pursuant to §240.10D-1(b). ☐
Indicate by check mark
whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐No☒
The aggregate market value
of the voting and non-voting common equity held by non-affiliates as of June 30, 2025, was $18.1 million based on the closing sale price
of the company’s common stock on such date of $1.25 per share, as reported by the NASDAQ Capital Market.
As of March 12, 2026, there were 34,380,971 shares of common stock
issued and outstanding.
Telomir Pharmaceuticals, Inc.
Annual Report on Form 10-K
For the fiscal year ended December 31, 2025
TABLE OF CONTENTS
Page
Cautionary Note on Forward-Looking Statements 3
PART I 6
Item 1. Description of Business 6
Item 1A. Risk Factors 22
Item 1B. Unresolved Staff Comments 49
Item 1C. Cybersecurity 49
Item 2. Description of Property 49
Item 3. Legal Proceedings 49
Item 4. Mine Safety Disclosure 49
Item 5. Market for Common Equity and Related Stockholder Matters 50
Item 6. [Reserved] 50
Item 7A. Quantitative and Qualitative Disclosures About Market Risk 57
Item 8. Financial Statements 57
Item 9A. Controls and Procedures 57
Item 9B. Other Information 58
Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections 58
PART III 59
Item 10. Directors, Executive Officers and Corporate Governance 59
Item 11. Executive Compensation 59
Item 14. Principal Accountant Fees and Services 60
Item 15. Exhibits, Financial Statement Schedules 61
Signatures 62
Unless we have indicated otherwise, or the context
otherwise requires, references in this Annual Report to “TELO,” the “Company,” “we,” “us”
and “our” or similar terms refer to Telomir Pharmaceuticals, Inc., a Florida corporation.
From time to time, we may use our website, our
Facebook page at https://www.facebook.com/people/Telomir-Pharmaceuticals-Inc/100087267737318/#, our Twitter at https://x.com/TelomirPharma,
and on our LinkedIn account at https://www.linkedin.com/company/telomir-pharmaceuticals-inc/posts/?feedView=all to distribute
material information. Our financial and other material information is routinely posted to and accessible on the Investors section of
our website, available at www.telomirpharma.com. Investors are encouraged to review the Investors section of our website because
we may post material information on that site that is not otherwise disseminated by us. Information that is contained in and can be accessed
through our website, our Facebook page, our Twitter posts and our LinkedIn posts are not incorporated into, and does not form a part
of, this Annual Report.
CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS
This Annual Report on Form 10-K contains forward-looking
statements (as defined in Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934,
as amended (the “Exchange Act”) that reflect our current expectations and views of future events. In some cases, you can
identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,”
“plan,” “anticipate,” “could,” “intend,” “target,” “project,”
“contemplate,” “believe,” “estimate,” “predict,” “potential”, or “continue”
or the negative of these terms or other similar expressions. In particular, statements about the markets in which we operate, including
expectations regarding our studies, growth of our various markets, and our expectations, beliefs, plans, strategies, objectives, prospects,
assumptions, or future events or performance contained in this Annual Report under the headings “Risk Factors,” “Management’s
Discussion and Analysis of Financial Condition and Results of Operations” and “Business” are forward-looking statements.
We have based these forward-looking statements
on our current expectations, assumptions, estimates and projections. While we believe these expectations, assumptions, estimates, and
projections are reasonable, such forward-looking statements are only predictions and involve known and unknown risks and uncertainties,
many of which are beyond our control. These and other important factors, including those discussed in this Annual Report under the headings
“Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations”
and “Business,” may cause our actual results, performance, or achievements to differ materially from any future results,
performance or achievements expressed or implied by these forward-looking statements, or could affect our share price. Important factors
that could cause actual results or events to differ materially and adversely from those expressed in forward-looking statements include,
but are not limited to, the following:
● the timing of anticipated regulatory filings;
● our future expenses, capital requirements and need for additional financing;
● our ability to recruit and enroll suitable patients in our clinical trials;
● developments relating to our competitors and our industry;
Given the risks and uncertainties set forth in
this Annual Report, you are cautioned not to place undue reliance on such forward-looking statements. The forward-looking statements
contained in this Annual Report are not guarantees of future performance and our actual results of operations, financial condition, and
liquidity, and the development of the industry in which we operate, may differ materially from the forward-looking statements contained
in this Annual Report. In addition, even if our results of operations, financial condition and liquidity, and events in the industry
in which we operate are consistent with the forward-looking statements contained in this Annual Report, they may not be predictive of
results or developments in future periods.
Any forward-looking statement that we make in
this Annual Report speaks only as of the date of such statement. Except as required by federal securities laws, we do not undertake any
obligation to update or revise, or to publicly announce any update or revision to, any of the forward-looking statements, whether as
a result of new information, future events or otherwise, after the date of this Annual Report.
Summary
Risk Factors
Our business is subject to numerous risks and
uncertainties that you should consider before investing in our company. You should carefully consider all of the risks described more
fully in the section titled “Risk Factors” in this Annual Report on page 22, before deciding to invest in our common stock.
If any of these risks actually occurs, our business, financial condition and results of operations would likely be materially adversely
affected.
Important factors that could cause actual results
or events to differ materially, but are not limited to, the following:
Risks Related to Our Intellectual Property
We depend on rights to Telomir-1 that are or
will be licensed to us.
We may not be able to adequately protect our
product candidates or our proprietary technology in the marketplace.
If third parties claim that our intellectual
property, products, processes, or anything else used by us infringes upon their intellectual property, our operating profits could be
adversely affected.
We have been granted a license to the right to
develop Telomir-1 in the United States in human and pet application, but we have not been granted a license to the rights to patents
covering Telomir-1 in foreign jurisdictions.
Risks Related to Our Operations and Financial
Condition
We are an early development-stage company with
no revenues and our financial condition raises substantial doubt as to our ability to continue as a going concern.
Because we have a limited operating history,
you may not be able to accurately evaluate our operations.
We will need to raise additional financing for
the continuation of our operations.
Our operating results may fluctuate, which could
have a negative impact on our ability to grow our client base, establish sustainable revenues and succeed overall.
We have yet to achieve a profit and will not
achieve a profit in the near future, if at all.
Certain of our executive officers are not be
employed by us on a full-time basis.
Conflicts of interest may arise between us and
MIRALOGX.
Risks Relating to Our Business and Our Industry
Our future success will largely depend on the
success of Telomir-1 and any future product candidates, which development will require significant capital resources and years of clinical
development effort.
We are dependent on our current and future product
candidates, some of which may not receive regulatory approval or be successfully commercialized.
Results of pre-clinical studies and earlier clinical
trials are not necessarily predictive indicators of future results.
We have limited marketing experience, and we
do not anticipate at this time establishing a sales force or distribution and reimbursement capabilities, and we may not be able to successfully
commercialize any of our product candidates if they are approved in the future.
We will need to further increase the size and
complexity of our organization in the future, and we may experience difficulties in managing our growth and executing our growth strategy.
We expect to face intense competition, often
from companies with greater resources and experience than we have.
We have significant and increasing liquidity
needs and may require additional funding.
Risks Related to Development and Regulatory
Approval of Our Product Candidates
Clinical trials for our product candidates are
expensive, time-consuming, uncertain, and susceptible to change, delay or termination. The results of clinical trials are open to differing
interpretations.
Any failure by us to comply with existing regulations
could harm our reputation and operating results.
The regulatory approval processes with the FDA
are lengthy and inherently unpredictable.
There is a high rate of failure for drug candidates
proceeding through clinical trials.
Risks Related to Our Reliance Upon Third Parties
We rely on, and expect to continue to rely on,
third parties to conduct clinical trials for our product candidates.
Our existing collaboration arrangements and any
that we may enter into in the future may not be successful, which could adversely affect our ability to develop and commercialize our
product candidates.
Risks Relating to the Ownership of Our Common
Stock
Because of the speculative nature of an investment
in our company, you may lose your entire investment.
Certain of our founding
stockholders, plus our existing officers and directors, control a substantial interest in us and thus may influence certain actions requiring
stockholder vote.
Risks Relating to Our Proposed Merger with
TELI Pharmaceuticals, Inc. (“TELI”)
Because TELI has a limited operating history,
you may not be able to accurately evaluate TELI’s operations.
TELI has significant and increasing liquidity
needs and will require additional funding.
TELI has yet to generate revenues or achieve
a profit and may not generate revenue or achieve a profit for many years, if at all.
TELI does not own rights to Telomir-1
TELI’s product candidates, if approved,
may not achieve the expected market acceptance and, consequently, limit TELI’s ability to generate revenue.
There are several conflicts of interests inherent
in the Merger.
PART I
ITEM 1. Description of Business
Overview
Telomir Pharmaceuticals, Inc. is a preclinical-stage
biotechnology company focused on the development of small-molecule therapeutics designed to address upstream biological drivers of aging
and age-related diseases rather than treating symptoms alone. Our research strategy targets fundamental cellular processes associated
with disease progression and functional decline, including metal-ion dysregulation, oxidative stress, epigenetic regulation, mitochondrial
dysfunction, and telomere integrity.
Telomir’s lead investigational compound,
Telomir-1 (Zn-Telomir), is a novel orally administered small molecule engineered to modulate intracellular metal balance—particularly
iron, copper, calcium, and zinc—through a regulated exchange mechanism rather than broad chelation. Our preclinical research has
evaluated Telomir-1 across multiple in vitro and in vivo model systems to characterize biological activity, safety profile, and potential
relevance across a range of disease pathways.
We have not yet conducted clinical trials in
humans. All findings described in this Business section are derived from preclinical studies and may not be predictive of clinical outcomes.
We plan to submit an IND application in 2026 and, if the IND becomes effective and other conditions are satisfied, to commence a Phase
1/2 clinical trial thereafter. The timing of these activities is subject to change and may be delayed due to regulatory, technical, manufacturing,
financing, or other factors
TELI Pharmaceuticals, Inc., a private related party company incorporated
under the laws of Delaware (“TELI”) and we entered into an Agreement and Plan of Merger and Reorganization, dated November
20, 2025, and as amended on February 4, 2026 (collectively, the “Merger Agreement”), pursuant to which a wholly owned subsidiary
of Telomir will merge with and into TELI, with TELI surviving as a wholly owned subsidiary of Telomir (the “Merger”), subject
to shareholder approval. At the effective time of the Merger (the “Effective Time”), each outstanding share of common stock
of TELI, $0.0001 par value per share (“TELI Common Stock”), will be converted into the right to receive such number of Telomir
Common Stock as is calculated based on the exchange ratio of the shares for the Merger (the “Exchange Ratio”). The Exchange
Ratio is calculated using the relative company valuations of each of TELO and TELI, as determined by Moore (as further described herein).
It is expected that shareholders of TELI will receive one share of Telomir Common Stock for each share of TELI Common Stock held (the
“Merger Share Consideration”). The Telomir Common Stock issued as the consideration will not be registered for trading under
the Securities Act. The Merger will result in an alignment of U.S. and non-U.S. rights to Telomir-1 within a single public company structure,
thereby simplifying global development and partnership efforts. As a result of the Merger, Telomir will own the entire worldwide intellectual
property portfolio and development programs related to Telomir-1
Scientific Rationale
Scientific research supports the role of reactive
metal accumulation and oxidative stress as contributors to progressive cellular dysfunction associated with aging and age-related diseases.
Iron and copper are essential for normal physiology; however, with aging and in certain disease states, regulation can weaken, contributing
to intracellular accumulation of reactive metal species.
Excess ferrous iron and reactive copper catalyze
the formation of reactive oxygen species, which can damage DNA, proteins, lipids, and mitochondria. Calcium dysregulation further contributes
to mitochondrial instability and activation of stress-related signaling pathways. Together, these processes contribute to genomic instability,
cellular senescence, and disease progression.
Our research premise is that restoring intracellular
balance across these pathways may support improved cellular function in preclinical systems relevant to aging and disease.
Telomir-1: Investigational Candidate and Mechanistic
Profile
Telomir-1 has been evaluated in preclinical model
systems for its potential effects on cellular homeostasis. In nonclinical studies conducted to date, Telomir-1 has demonstrated activity
consistent with modulation of oxidative stress pathways, redox balance under metal-induced stress conditions, mitochondrial structure
and function, intracellular calcium dynamics, and epigenetic regulatory processes. Observed findings also suggest potential interaction
with iron-dependent enzymatic pathways, including histone demethylases. These effects have been observed in controlled preclinical settings
and may reflect interconnected biological mechanisms; however, their relevance to human disease has not been established.
Epigenetic Regulation and DNA Methylation
Epigenetic regulation, including changes in DNA
methylation and histone methylation, plays a central role in gene expression, cellular identity, and disease biology. Abnormal epigenetic
patterns can contribute to inappropriate gene silencing, loss of genomic stability, and impaired stress responses. In disclosed preclinical
cancer and aging models, Telomir-1 has demonstrated the ability to influence DNA methylation patterns and inhibit iron-dependent histone
demethylases, including members of the KDM2, KDM5, and KDM6 families. These enzymes require ferrous iron as a cofactor and play key roles
in regulating chromatin structure and transcriptional programs.
Telomere Integrity and Genomic Stability
Telomeres are protective DNA-protein structures
located at the ends of chromosomes that shorten with age and under conditions of oxidative stress and cellular damage. Accelerated telomere
shortening is associated with cellular senescence, impaired regenerative capacity, and age-related disease.
In disclosed preclinical experimental systems
evaluating genomic stability and cellular aging, Telomir-1 has been associated with preservation of telomere integrity and observed telomere
elongation in certain model systems, together with normalization of accelerated aging phenotypes. These observations were made in controlled
preclinical models and do not represent evidence of clinical benefit in humans.
Preclinical Aging and Healthspan Observations
Across multiple preclinical studies, Telomir-1
has demonstrated biological activity relevant to aging and age-related decline, including reduction of oxidative stress and reactive
oxygen species, preservation of mitochondrial function, protection against stress-induced cell death, normalization of functional decline
in aging and progeria models, and improvements in mobility, survival, and healthspan parameters in model organisms.
Oncology Research
Cancer cells frequently rely on dysregulated
metal metabolism, oxidative stress, and epigenetic silencing to support uncontrolled proliferation and treatment resistance. Telomir-1
has been evaluated in multiple preclinical oncology models, including prostate cancer, triple-negative breast cancer, pancreatic cancer,
and leukemia. In these models, Telomir-1 has been associated with reduced tumor growth, altered metabolic activity, reversal of abnormal
DNA methylation, and modulation of pathways related to cancer cell survival. Telomir-1 has demonstrated anti-tumor activity rather than
tumor promotion in preclinical systems evaluating telomere biology.
IND-Enabling GLP Safety and Toxicology
We disclosed favorable results from IND-enabling
Good Laboratory Practice toxicology and safety pharmacology studies for Telomir-1. These studies evaluated cardiovascular safety, respiratory
safety, phototoxicity, and repeat-dose toxicology in animal models. Telomir-1 was reported to be well tolerated across completed GLP
safety studies, without treatment-related adverse or dose-limiting toxicities observed. Cardiovascular assessments demonstrated no test-article-related
changes in blood pressure, heart rate, electrocardiogram parameters, or body temperature, and no concerning cardiac safety signals. Repeated-dose
studies in rats and dogs were reported to be well tolerated, with findings described as limited, reversible, and non-adverse. No clinically
meaningful respiratory effects or phototoxicity were reported. Across oral administration studies, Telomir-1 demonstrated consistent
systemic exposure.
Development Strategy and Outlook
Telomir-1 remains in preclinical development.
We continue to advance regulatory preparation and development activities in support of a potential Investigational New Drug (“IND”)
application. We are targeting IND submission in 2026 and, subject to regulatory review, completion of required preclinical and manufacturing
activities, and availability of resources, plans to initiate a Phase 1/2 clinical trial thereafter. The timing of these activities is
subject to change and may be delayed due to regulatory, technical, operational, or financial factors. There can be no assurance that
preclinical findings will translate into safety, efficacy, disease modification, or therapeutic benefit in humans.
To date, we have completed multiple preclinical
studies on Telomir-1, including those demonstrating that Telomir-1 is non-mutagenic and possesses strong biological and metal-binding
properties (Graphic 1). Using advanced “in silico modeling,” driven by artificial intelligence to predict a compound’s
therapeutic potential, chemical and biological activity, and toxicity, we continue to uncover evidence supporting Telomir-1’s potential
to address metal-overload conditions. Additionally, recent independent in vitro studies have confirmed that Telomir-1 exhibits strong
binding affinities for copper and iron, with reduced binding affinity for zinc.
Graphic 1: Telomir-1 is capable of binding
to several metal ions
We collaborate with third-party organizations
to conduct research and advance development efforts. One such partner, InSilico Trials, utilizes in silico digital simulations to support
drug development through computational modeling. These techniques allow us to efficiently predict the safety and efficacy of potential
compounds, reducing the need for extensive clinical trials. Additionally, we work with Recipharm and Smart Assays to assess Telomir-1’s
binding properties and investigate ion competition and exchange under varying conditions, further refining its therapeutic potential.
An example of these findings is illustrated in
the Graphic 2 below, created in collaboration with Smart Assays Biotechnologies Ltd. The figure depicts the concentration of free iron
(Fe2+) in the presence of Telomir-1 (T1) and various ions. When Telomir-1 is introduced, the concentration of free iron decreases
(indicated by the shift from the blue point to the orange point). This is direct evidence for the binding of iron to T1, reducing its
free ion concertation in the solution. However, the addition of copper (Cu2+) at varying concentrations leads to an exchange
between bound iron and copper, resulting in the release of previously bound iron and restoration of free iron levels in solution. This
is evidenced by an increase in free iron concentration, corresponding to the amount of copper added. In contrast, zinc does not induce
an exchange effect on the binding of iron to Telomir-1, indicating a lower affinity of Zinc to Telomir-1.
Graphic 2: Ionic exchange between iron and
copper or zinc
Wilson’s disease
Wilson’s disease is a rare genetic disorder
affecting approximately 1 in 30,000 individuals worldwide. It is caused by mutations in the ATP7B gene, which disrupt the body’s
ability to regulate copper metabolism. This results in toxic copper accumulation, particularly in the liver and brain, leading to severe
complications such as liver failure, neurological damage, and psychiatric disorders. Without treatment, Wilson’s disease is fatal.
Current treatments, including chelating agents
(e.g., penicillamine and trientine) and zinc therapy, focus on reducing copper levels but have notable limitations:
These challenges highlight the urgent need for
novel therapies that address the root cause of Wilson’s disease while minimizing adverse effects. Telomir-1, with its targeted
copper-binding properties, has the potential to provide a safer, more effective treatment by directly addressing the underlying mechanisms
of the disease. Furthermore, Wilson’s disease qualifies as a candidate for orphan drug designation due to its rarity and life-threatening
nature, offering opportunities for accelerated development and additional regulatory and financial incentives.
We are actively investigating Telomir-1’s
potential in treating Wilson’s disease by studying its effects on copper toxicity through in vitro experiments and a mouse model
of the disease. These studies are currently ongoing.
Type 2 Diabetic (NIDDM)
In collaboration with the India-based research
organization Pentagrit, in 2024 Telomir conducted preclinical studies evaluating two forms of Telomir-1, administered orally at three
different doses, in zebrafish models of Type 2 diabetes mellitus induced by a high-calorie diet. The study assessed key metabolic indicators,
including fasting glucose levels, Oral Glucose Tolerance Test (OGTT) results, insulin concentrations, and HOMA-IR. The findings revealed:
A Novel Mechanism of Action
Telomir-1 introduces a novel mechanism of action
by addressing the cellular role of iron metabolism in chronic diseases such as Type 2 diabetes. Excess iron contributes to oxidative
stress, beta-cell damage, and insulin resistance. Telomir-1 is designed to normalize iron homeostasis, reducing oxidative stress and
enhancing insulin sensitivity. This differentiates it from current diabetes drugs that primarily treat symptoms without targeting the
underlying causes.
The study demonstrated significant reductions
in fasting plasma glucose to basal levels, improvements in glucose tolerance, and a reversal of insulin resistance to near pre-diabetic
levels. These results were accompanied by improved HOMA-IR values, a standard measure of insulin sensitivity and resistance, and increased
survival rates in treated models.
Global Impact of Type 2 Diabetes
According to the International Diabetes Federation,
over 800 million adults worldwide are affected by Type 2 diabetes (both diagnosed and undiagnosed), with annual healthcare costs exceeding
$966 billion as of 2021. Existing treatments primarily manage blood glucose and symptoms but fail to address the root causes of the disease.
These therapies are often associated with significant limitations, including minimal impact on insulin resistance, gastrointestinal issues,
risk of hypoglycemia, cardiovascular risks, and weight gain.
The findings from this zebrafish diabetes model
suggest that Telomir-1 could offer a transformative approach to managing Type 2 diabetes. By targeting the underlying mechanisms of insulin
resistance and oxidative stress through iron metabolism normalization, Telomir-1 represents a potential breakthrough in diabetes treatment.
A key indicator of its success was the significant reduction in HOMA-IR values, highlighting its ability to improve insulin sensitivity
and glucose regulation. The reversal of insulin resistance to near pre-diabetic levels underscores Telomir-1’s potential as a groundbreaking
therapy (Graphic).
Graphic 3: Effects of two forms of Telomir-1
on HOMA-IR, a measure of insulin resistance in Zebrafish
We are currently evaluating the potential in
NIDDM by studying the effect of Telomir-1 on several metabolic parameters in a rat model of NIDDM. These studies are ongoing.
Age reversal
We have carried out a preclinical study, conducted
in collaboration with Nagi Bioscience SA, utilized a sophisticated in vivo microfluidic-based assay to assess the effects of Telomir-1
on the nematode Caenorhabditis elegans, a well-established model for aging studies. The microfluidic platform allowed precise, automated
tracking of lifespan, health span, and age-related mobility decline in real-time, enabling the research team to accurately measure the
effects of Telomir-1 on these critical metrics.
Two forms of Telomir-1 were administered in two
concentrations, allowing the study to examine dose-dependent responses in treated subjects. The study found that Telomir-1 significantly
enhanced lifespan and health span parameters in aged microorganism populations. Key findings included:
Graphic 4: Effect of Telomir-1 on c.elegans
life span
Graphic 5: Effect of two forms of Telomir-1
on several motility parameters
Progeria Study: Telomir-1 Shows Promising
Age-Reversal Effects
We recently completed a study evaluating the
effects of Telomir-1 in a nematode model of Progeria (Caenorhabditis elegans). The study focused on nematodes with a mutation in the
wrn-1 gene, the equivalent of the human WRN gene, which is implicated in Werner Syndrome, a form of Progeria. In C. elegans, wrn-1 depletion
is associated with a significantly reduced mean and median lifespan compared to normal (wild-type) organisms.
The study demonstrated significant age-reversal
effects in wrn-1-mutated nematodes treated with Telomir-1. Treatment effectively restored longevity levels to those comparable
to wild-type organisms. Additional benefits included an extended healthy lifespan and normalization of physiological parameters such
as movement velocity and tail amplitude.
Further studies in a Progeria human cell line
are planned in collaboration with Smart Assays to build on these promising findings.
Oxidative Stress Study: Telomir-1 Shows potential to Reverse Oxidative
Stress and Provide Robust Cellular Protection
Oxidative stress is a key driver of aging and
disease progression, contributing to conditions such as Alzheimer’s, Age-related Macular Degeneration (AMD), cardiovascular diseases,
cancer, and diabetes. It also plays a critical role in the severity of viral infections, including avian influenza (bird flu), by increasing
inflammation, cellular damage, and impairing immune responses. Addressing oxidative stress is essential for mitigating these effects
and improving patient outcomes.
Telomir Pharmaceuticals recently conducted preclinical
studies in collaboration with Smart Assays Biotechnologies Ltd. to evaluate Telomir-1’s ability to combat copper-induced oxidative
stress. The study, performed in human cell lines, demonstrated:
These findings suggest Telomir-1’s potential
to preserve cellular integrity, combat oxidative damage, and regulate metal ion balance—critical factors in aging-related diseases
and viral infections. This breakthrough supports further clinical development to assess its full potential in disease intervention.
Graphic 6: Effect of Telomir-1 on Oxidative
Stress
Graphic 7: Effect of Telomir-1 on Copper Toxicity
Expanding Telomir-1’s Therapeutic Potential
Building on these findings, the Company continues to evaluate the
broader therapeutic potential of Telomir-1 across multiple disease areas characterized by metal dysregulation and oxidative stress. These
areas of scientific interest include Wilson’s disease, Type 2 diabetes, age-related macular degeneration, oncology, and epigenetic
regulation.
Recent studies have shown that Telomir-1 has potent anti-cancer effects.
In vivo evaluation using zebrafish tumor xenografts revealed heterogeneous antitumor responses. Telomir-Zn significantly reduced primary
tumor size in BT-549 and HCC1806 xenografts and decreased metastatic dissemination in HCC1806. Combination treatment with paclitaxel
produced greater tumor suppression compared with either monotherapy (Graphic 8-9).
Graphic 8. Antitumor efficacy of paclitaxel (PTX) and Telomir-1
in BT-549 zebrafish xenografts. (A)Primary tumor size and metastatic dissemination were evaluated in BT-549 xenografts after 3 days
of treatment. (B) Representative microscopy images of primary tumors treated with vehicle or Telomir-Zn 1-10 μM throughout the
evaluation time Data are normalized relative to the negative control group and presented as mean ± SEM. Statistical
Graphic 9. Antitumor efficacy of paclitaxel (PTX) and Telomir-Zn
in HCC1806 zebrafish xenografts.(A) Primary tumor size and metastatic dissemination were evaluated in HCC1806 xenografts after 3
days of treatment. (B) Representative microscopy images of disseminated tumor cells after 3 days of treatment with vehicle or Telomir-Zn
3 μM.
Data are normalized relative to the negative control group and presented
as mean ± SEM.
For metastasis data, statistical analysis was performed using one-way
ANOVA (p = 0.0617), followed by a two-tailed Student’s t-test. p < 0.05 indicates significance compared with the
vehicle group.
The Company is in the process of finalizing the initial clinical development
program for Telomir-1 and expects to provide additional details regarding the planned clinical indication in connection with the anticipated
submission of its Investigational New Drug (IND) application. Future development efforts may also explore additional indications based
on regulatory guidance, emerging scientific data, capital availability, and strategic considerations. There can be no assurance that
the Company will pursue development in any or all of these areas.
● Cancer Research: Exploring anti-cancer applications using xenograft studies.
Future Plans and Milestones
Telomir-1 has demonstrated significant potential
in reversing age-related and metabolic conditions while targeting underlying disease mechanisms. As the company continues to refine the
molecular understanding of Telomir-1, efforts are underway to identify the most impactful indication for its initial IND application.
We are optimizing manufacturing processes to
produce GMP-grade Telomir-1 for IND-enabling activities and potential future clinical trials. We have completed the preclinical safety
studies it currently believes are required to support an IND submission. We are targeting IND submission in 2026 and, if the IND becomes
effective and subject to completion of remaining regulatory, manufacturing, and operational activities, plans to initiate a Phase 1/2
clinical trial thereafter. The FDA may require additional studies or information prior to or following IND submission. The timing of
these activities is subject to change and may be delayed due to regulatory, technical, operational, financial, or other factors.
Through these initiatives, we are committed to
revolutionizing treatments for age-related and chronic diseases by addressing their root causes and paving the way for transformative
healthcare solutions.
Market Potential
The
market potential for Telomir-1 is substantial, given its focus on addressing the root causes of age-related diseases and promoting longevity.
Chronic diseases such as diabetes, cancer, Alzheimer’s, and cardiovascular disorders, which are closely tied to aging, account
for over 75% of healthcare spending in the United States, exceeding $4 trillion annually, according to the CDC. As the global population
ages, the demand for innovative therapies targeting the underlying mechanisms of these conditions is expected to grow significantly.
Furthermore, Verified Market Research reports that the anti-aging drug market was valued at approximately $91.05 billion in 2024 and
is projected to reach $160.24 billion by 2031, growing at a compound annual growth rate (CAGR) of 7.32%. With its novel mechanism of
action that regulates cellular metal imbalances and reduces oxidative stress, Telomir-1 is uniquely positioned to capitalize on these
expanding markets, offering transformative potential to enhance health span, slow disease progression, and redefine longevity treatments.
Pre-Clinical IND-Enabling Studies
We have successfully completed several IND-enabling
studies for Telomir-1, aiming to demonstrate its non-toxic profile. In collaboration with Frontage Laboratories, our research is aimed
at establishing the metabolism and maximum tolerated dose (MTD) of Telomir-1, providing critical data to support its development. Our
preclinical studies were conducted in collaboration with third-party organizations, including Frontage Laboratories, InSilico Trials,
Pharmaseed Ltd, Smart Assay Biotechnologies Ltd., Recipharm, Naji Biosciences, and Pantagrit. These partners played a key role in supporting
the execution and analysis of the research.
Our Clinical Development Plan
Upon completion of required preclinical studies
and supporting activities, we intend to submit an Investigational New Drug (“IND”) application to the U.S. Food and Drug
Administration (“FDA”) for Telomir-1. We have not yet selected a specific initial clinical indication and expects that the
development strategy may evolve based on regulatory guidance and ongoing scientific evaluation. We are targeting IND submission in 2026.
If the IND becomes effective and subject to regulatory feedback and operational readiness, we plan to initiate first-in-human clinical
development thereafter. The scope, design, and timing of any clinical trial will be determined in consultation with regulatory authorities
and may be modified based on FDA input. There can be no assurance that the FDA will permit us to proceed to clinical trials on the anticipated
timeline or at all.
Market Opportunity
Telomir-1 is under
investigation for its therapeutic potential across multiple areas, including Progeria, Type 2 Diabetes, Wilson’s disease, Age-related
Macular Degeneration (AMD), cancer, and other age-related conditions. Additionally, Telomir-1 is being explored for its potential in
mitigating the severity of viral infections such as avian influenza (bird flu) by addressing oxidative stress and immune dysfunction.
These indications are being carefully evaluated to identify the most impactful opportunities for clinical development and market entry.
Certain
of the therapeutic areas under evaluation represent established pharmaceutical markets in North America. According to Fortune Business
Insights, the U.S. Type 2 Diabetes market was valued at approximately $30.47 billion in 2022. Allied Market Research has projected that
the global age-related macular degeneration (AMD) market could reach approximately $18 billion by 2030, reflecting anticipated growth
over the forecast period. These estimates are based on third-party reports and are subject to assumptions and uncertainty. There can
be no assurance that Telomir-1, if approved, would address any of these markets or achieve commercial adoption.
The U.S oncology market, covering a broad range
of cancers, was valued at $74.1 billion in 2023, and is projected to reach $180.12 billion by 2033, growing at a CAGR of 9.2% from 2024
to 2033, as per Precedence Research. Meanwhile, the anti-aging and age-reversal market was valued at $91.05 billion in 2024 globally
and is projected to grow to $160.24 billion globally by 2031, with a CAGR of 7.32%, according to Verified Market Research.
Rare diseases such as Progeria and Wilson’s
disease, despite having smaller patient populations, represent highly lucrative opportunities due to significant unmet medical needs
and strong pricing potential. For instance, the annual treatment cost for Progeria exceeds $1 million, based on the price of Zokinvy
(lonafarnib), the FDA-approved drug for the condition. Similarly, treatments for Wilson’s disease, like Syprine (trientine hydrochloride),
can cost approximately $300,000 per year.
The global antiviral drugs market is experiencing
significant growth, driven by the increasing prevalence of viral infections and advancements in treatment options. In 2022, the market
was valued at approximately $49.8 billion and is projected to reach around $71.1 billion by 2032, reflecting a compound annual growth
rate (CAGR) of 3.73% during this period.
Specifically focusing on avian influenza (bird
flu), the treatment market was estimated at $22.06 billion in 2023 and is expected to grow at a CAGR of 8.18%, reaching approximately
$38.27 billion by 2030.
These figures highlight the substantial market
opportunity for innovative treatments addressing chronic, age-related, and viral diseases. With increasing demand for therapies targeting
oxidative stress, cellular degeneration, and immune resilience, Telomir-1 is well-positioned to address these critical unmet medical
needs. Its broad therapeutic potential, spanning rare diseases, metabolic disorders, neurodegeneration, and viral infections, represents
a significant market opportunity across North America and beyond, reinforcing its potential as a transformative solution in modern medicine.
Intellectual Property
We license the U.S. patent rights for the use
of Telomir-1 in human applications from MIRALOGX, LLC (“MIRALOGX”), a related party intellectual property development and
holding company. MIRALOGX has filed a Patent Cooperation Treaty (PCT) application, PCT/US2023/073106 on August 29, 2023. The application
designated the U.S. and will enter U.S. national phase. The application, if granted and subject to payment of patent maintenance fees,
would offer protection extending through at least August 29, 2043 in the U.S. The patent rights for Telomir-1 outside of the United States
are not included in our current patent rights.
Our license from MIRALOGX is set forth in an
Amended and Restated Exclusive License Agreement, dated August 11, 2023, between us and MIRALOGX, pursuant to which we obtained the exclusive
perpetual right and license under the above-described patent rights to make, have made, use, and sell “Licensed Products”
in the U.S. for human uses and pre-clinical studies and activities of any kind conducted in furtherance of obtaining regulatory approval
or commercialization for human uses (the “Initial MIRALOGX License Agreement”). On November 10, 2023, we and MIRALOGX entered
into the Amendment No. 1 to the Amended and Restated License Agreement, pursuant to which the field of use relating to the license was
amended to include therapeutic treatments and other medical or health uses in animals, in addition to humans, and related preclinical
studies and activities conducted in furtherance of obtaining regulatory approval for and commercialization of veterinary, in addition
to human, therapeutic treatments and uses (together with the “Initial MIRALOGX License Agreement, the “MIRALOGX License Agreement”).
“Licensed Product” is defined in the agreement as a drug product containing as an active agent 2,4,6-tris(3,4-dihydro-2H-pyrrol-2-yl)
pyridine or a pharmaceutically acceptable salt, ester, or solvate thereof. We also have the right to grant corresponding sublicenses
under the licensed patent rights. The MIRALOGX License Agreement provides for the payment to MIRALOGX of an 8% royalty (payable quarterly)
on our net sales of Licensed Products by us or our sublicensees and on non-royalty bearing milestone revenue. There are no up-front,
execution, or milestone payments in the license agreement. Further, no payments have been made to date under the agreement.
Government Regulation
The FDA and comparable regulatory authorities
in state and local jurisdictions impose substantial and burdensome requirements upon companies involved in the clinical development,
manufacture, marketing, and distribution of drugs. These agencies and other federal, state, and local entities regulate, among other
things, the research and development, testing, manufacture, quality control, safety, effectiveness, labeling, storage, record keeping,
approval, advertising and promotion, distribution, post-approval monitoring and reporting, sampling and export and import of our drug
candidates.
In the United States, the FDA regulates drugs
under the Federal Food, Drug, and Cosmetic Act, or FDCA, and its implementing regulations. The process of obtaining regulatory approvals
and the subsequent compliance with appropriate federal, state, local and foreign statutes and regulations requires the expenditure of
substantial time and financial resources. Failure to comply with the applicable U.S. requirements at any time during the product development
process, approval process or after approval, may subject an applicant to a variety of administrative or judicial sanctions, such as the
FDA’s refusal to approve pending New Drug Applications (NDAs), withdrawal of an approval, imposition of a clinical hold, issuance
of warning letters, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines,
refusals of government contracts, restitution, disgorgement or civil or criminal penalties. The process required by the FDA before a
drug may be marketed in the United States generally involves the following:
● submission to the FDA of an NDA;
● satisfactory completion of an FDA advisory committee review, if applicable;
Pre-clinical studies
Before testing any drug or biological product
candidate in humans, the product candidate must undergo rigorous pre-clinical testing. The pre-clinical developmental stage generally
involves laboratory evaluations of drug chemistry, formulation, and stability, as well as studies to evaluate toxicity in animals, to
assess the potential for adverse events (“AEs”) and, in some cases, to establish a rationale for therapeutic use. The conduct
of pre-clinical studies is subject to federal regulations and requirements, including GLP regulations for safety/toxicology studies.
An IND sponsor must submit the results of the pre-clinical studies, together with manufacturing information, analytical data, any available
clinical data or literature and a proposed clinical protocol, to the FDA as part of the IND.
An IND is a request for authorization from the
FDA to ship an investigation product and then administer it to humans and must be allowed to proceed by the FDA before human clinical
trials may begin. Some long-term pre-clinical testing, such as animal tests of reproductive AEs and carcinogenicity, may continue after
the IND is submitted. An IND automatically becomes effective 30 days after receipt by the FDA, unless the FDA raises concerns or questions
before that time related to one or more proposed clinical trials and places the trial on clinical hold. In such a case, the IND sponsor
and the FDA must resolve any outstanding concerns before the clinical trial can begin. As a result, submission of an IND may not result
in the FDA allowing clinical trials to commence.
Clinical trials
The clinical stage of development involves the
administration of the investigational product to healthy volunteers or patients under the supervision of qualified investigators, generally
physicians not employed by, or under control of, the trial sponsor, in accordance with GCPs, which include the requirement that all research
patients provide their informed consent for their participation in any clinical trial. Clinical trials are conducted under protocols
detailing, among other things, the objectives of the clinical trial, dosing procedures, subject selection and exclusion criteria and
the parameters to be used to monitor subject safety and assess efficacy. Each protocol, and any subsequent amendments to the protocol,
must be submitted to the FDA as part of the IND. Furthermore, each clinical trial must be reviewed and approved by an IRB for each institution
at which the clinical trial will be conducted to ensure that the risks to individuals participating in the clinical trials are minimized
and are reasonable in relation to anticipated benefits. The IRB also approves the informed consent form that must be provided to each