Item 1A. Risk Factors 17
Item 1B. Unresolved Staff Comments 42
Item 1C. Cybersecurity 42
Item 2. Description of Property 42
Item 3. Legal Proceedings 42
Item 4. Mine Safety Disclosure 42
Item 5. Market for Common Equity and Related Stockholder Matters 43
Item 6. Selected Financial Data 43
Item 7A. Quantitative and Qualitative Disclosures About Market Risk 50
Item 8. Financial Statements 50
Item 9A. Controls and Procedures 50
Item 9B. Other Information 51
Item 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections 51
PART III 52
Item 10. Directors, Executive Officers and Corporate Governance 52
Item 11. Executive Compensation 58
Item 14. Principal Accountant Fees and Services 68
Item 15. Exhibits, Financial Statement Schedules 69
Signatures 70
Unless
we have indicated otherwise, or the context otherwise requires, references in this Report to “TELO,” the “Company,”
“we,” “us” and “our” or similar terms refer to Telomir Pharmaceuticals, Inc., a Florida corporation.
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.
PART
I
ITEM
1. Description of Business
Overview
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. In
situ stem cell therapy uses the body’s natural resources to regenerate damaged tissue and replace cells with new, functional
cells.
Specifically,
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. If demonstrated by future clinical trials and approved by the U.S. Food and Drug Administration, or FDA,
we believe TELOMIR-1 may also 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.
Based
on our pre-clinical studies to date, and if ultimately approved by the FDA, we believe that TELOMIR-1 may potentially serve as a metal
enzyme inhibitor of essential metals such as iron, zinc and copper. These essential metals play a role in a host of age-related inflammatory
conditions such as osteoarthritis and hemochromatosis (a condition that causes the body to absorb too much iron, which can damage organs
and tissues), as well as in post-chemotherapy health problems. 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 iron, zinc and copper that accompany age-related inflammatory
conditions and certain cancers by modulating pro-inflammatory cytokines such as Interleukin-17 (or IL-17).
Our
goal is to advance the clinical development of TELOMIR-1 in the United States as a potential therapeutic intervention for age-related
inflammatory conditions. Our initial development plan was to research TELOMIR-1 as a potential treatment for hemochromatosis followed
by the post-chemotherapy recovery indication. Due to the inability to define the preclinical mouse model for hemochromatosis and register
the investigator site, in March 2024 we shifted our research focus to make osteoarthritis our lead indication for TELOMIR-1. Our research
plan now includes hemochromatosis as the second indication followed by post-chemotherapy recovery. As our research progresses, we may
explore different indications or shift our indication focus as we deem necessary or as circumstances require.
TELOMIR-1
is currently under pre-clinical investigation, with the goal of submitting an Investigational New Drug Application (or IND) for Telomor-1
to the FDA which, if accepted, would allow us to move in human clinical trials. In particular, we are studying TELOMIR-1’s potential
to interrupt and prevent the IL-17 induced inflammatory pathways that create the systemic imbalance of cellular metals. 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.
To
date, we have completed several pre-clinical studies with respect to TELOMIR-1. Some of these studies were designed to demonstrate that
TELOMIR-1 is not mutagenic and has good biological and metal binding capabilities (Graphic 1). Using
“in silico modeling”, which deploys artificial intelligence-driven computational models to predict a compound’s therapeutic
potential, biological activities and toxicity, we are continuing to find evidence that the mechanism of action of TELOMIR-1 has the potential
to reverse age-related conditions such as osteoarthritis by lengthening DNA’s protective telomere caps.
Graphic
1. TELOMIR-1 Molecule
We
have collaborations with third parties who conduct our research for us. One of these is InSilicoTrials, a company founded by life science,
cybersecurity, and digital innovation experts, which utilizes in silico digital simulations. Through such collaborations, we specialize
in leveraging artificial intelligence and simulations to enhance drug development. Using in silico techniques, we analyze data to predict
safety and efficacy of potential compounds, which we believe is an efficient and cost-effective research and development advancement
with the potential to minimize the extensiveness of later clinical trials.
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 the regenerative abilities
and limited quantities of stem cells in the adult human body, in situ treatment and protection of stem cells may provide an important
therapeutic mechanism for treatment of disease.
The
graph below describes telomere elongation secondary to TELOMIR-1 stimulation and subsequent stem cell self-renewal. As noted, telomeres
shorten during the natural aging process. Based on our initial studies, we believe that TELOMIR-1may be able to delay or reverse age-related
conditions through telomere elongation.
Invitro
Human Stem Cell Therapy with Frontage Laboratories: Telomir Length Determination
Through
research studies with Frontage Laboratories, we are investigating in vitro human stem cell in human cell lines. Specifically, we are
engaged in the following in conjunction with Frontage Laboratories:
Cell
line selection. Based on the literature highlighting their appropriateness for telomere and aging research, Frontage acquires three
vials for each stem cell line: primary human umbilical vein endothelial cell, non-immortalized human fibroblast, and the human BM-derived
mesenchymal stem cell line. The cells then undergo an evaluation to determine telomere length and their capacity for cell renewal and
proliferation and assess cell death, as described below.
Telomere
length determination. A commercially available qPCR kit is employed to treat and assess the absolute telomere length of the cells.
Cells undergo treatment with different concentrations of TELOMIR-1, followed by harvesting at distinct time intervals, and subsequently
subjected to telomere length analysis.
Results
of our research with Frontage Laboratories is provided in the graph below. The graph shows that 50mM of TELOMIR-1 elongated telomeres
compared to untreated and vehicle cells.
Our
Strategy
Our
goal is to develop, gain US regulatory approval for and commercialize TELOMIR-1 as a new treatment option targeting the elongation of
telomeres for treatment of age-related inflammatory conditions, with osteoarthritis as our initial clinical focus followed by hemochromatosis.
Thereafter, we plan to expand the development of TELOMIR-1 to post-chemotherapy recovery therapeutic. The key elements of our strategy
to achieve this goal include:
Osteoarthritis
Background
According
to the CDC, osteoarthritis is the most common form of arthritis. Some people call it degenerative joint disease or “wear and tear”
arthritis. It occurs most frequently in the hands, hips, and knees. With osteoarthritis, the cartilage within a joint begins to break
down and the underlying bone begins to change. These changes usually develop slowly and get worse over time. Osteoarthritis can cause
pain, stiffness, and swelling. In some cases, it also causes reduced function and disability. Key impacted joints include hands, knees,
hips, and neck. Most patients with osteoarthritis often present with pain and stiffness in their joints as soft tissue damage progresses,
restricting their ability to participate in normal life activities. IL-17 promotes osteoarthritis disease progression by regulating
chondrocyte autophagy, senescence, and cartilage matrix degradation.
There
is no cure for osteoarthritis, so doctors usually treat osteoarthritis symptoms with various therapies, which may include: increasing
physical activity, physical therapy with muscle strengthening exercises, weight loss, medications, including over-the-counter pain relievers,
supportive devices such as crutches or canes and surgery (typically joint replacement, if other treatment options have not been effective).
Hemochromatosis
Background
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.
Post-Chemotherapy
Recovery Background
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 plan to investigate 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.
Pre-Clinical
IND-Enabling Studies
To
date, as shown in the table below, we have completed several IND-enabling studies with respect to TELOMIR-1 that were designed to demonstrate
that TELOMIR-1 is non-toxic. Our research studies with Frontage Laboratories have also helped us establish the metabolism and maximum
tolerated dose (or MTD) of TELOMIR-1.
Type of Study Species (in vitro studies) Purpose Results
We
are planning pre-clinical proof-of-concept studies for TELOMIR-1, including canine and rat osteoarthritis studies.
All
pre-clinical studies described in this Annual Report were conducted with the assistance of third parties, including Frontage Laboratories
of Exton Pennsylvania and InSilico Trials Technologies of Trieste, Italy, each of whom utilize
artificial intelligence-driven computer in-silico testing models.
Our
Clinical Development Plan
Following
completion of the toxicology studies and preclinical proof-of-concept studies pre-clinical development program, we plan to submit to
the FDA an IND, focused on investigating TELOMIR-1 for the treatment of osteoarthritis. We will consider a second IND for hemochromatosis
with FDA guidance.
Our
first IND application submission investigating TELOMIR-1 for the treatment of osteoarthritis is currently planned for the first quarter
of 2025. If allowed to proceed by the FDA, a Phase I double-blind, randomized, placebo-controlled trial to evaluate the safety, tolerability,
and pharmacokinetics of TELOMIR-1 in 40-60 healthy male and female adult subjects will be initiated approximately 30 days post-IND submission.
Our
second IND application will likely focus on investigating TELOMIR-1 for the treatment of hemochromatosis and is planned for submission
with guidance from the FDA. Additionally, post-chemotherapy recovery may be considered as a future contender with additional guidance
from the FDA.
Our
clinical development plans will depend on FDA acceptance of our IND applications. As appropriate and pursuant to discussions with the
FDA, we may periodically adjust the timeline for certain filings and associated clinical trials. It is important to note that the process
for conducting clinical trials is uncertain and there is no assurance that our clinical development activities will meet the planned
timelines set forth above.
Manufacture
of Product for Clinical Development Activities
Anthem
Biosciences, a leading contract development and manufacturing organization located in India, has been developing a large-scale synthesis
protocol for us and will be supplying quantities of TELOMIR-1 needed for our pre-clinical and clinical development activities. We are
currently in discussions with Frontage Laboratories to have TELOMIR-1 formulated into solid oral dosage forms for clinical trials. DAVOS
Pharm is a US intermediary between Anthem in India and Telomir in the US.
Market
Opportunity
TELOMIR-1,
if approved, is expected to initially compete in the osteoarthritis market followed by hemochromatosis. TELOMIR-1, if approved, may have
the potential to secondarily compete in the post-chemotherapy recovery market.
Potential
Market for Osteoarthritis
As
of 2022, according to the CDC, approximately 32.5 million adults in the United States have osteoarthritis or on average 10% of the population.
The patient count will rise to 78.4 million by 2040. As the U.S. population ages, it is anticipated that the burden of osteoarthritis
will grow, accounting for 5%-6% of all hospitalization related costs. Most patients manage their symptoms using a variety of pharmacologic
and non-pharmacologic tools depending on the severity of pain felt. For the approximately 25% of patients considered severe cases (i.e.
a pain score of 7 and above out of 10), a mix of pharmaceutical options exist, usually starting with corticosteroids, hyaluronic acid,
and prescription strength non-steroidal anti-inflammatory drugs (or NSAIDs). The market
for medications to treat osteoarthritis is worth $600 - $800/patient/annum or approximately $19.5 to $26 billion in annual costs spread
across multiple medications from NSAIDs to steroids. We are seeking to have TELOMIR-1 become a novel, first line treatment for osteoarthritis,
and compete for a material share of the estimated medical costs market.
Potential
Market in Hemochromatosis
Patients
with two mutated copies of the HFE gene (C282Y and H63D) are at the greatest risk for developing hemochromatosis, alongside patients
with a family history of hemochromatosis. As a result, approximately 13 million U.S. patients carry mutations for hemochromatosis but
only a fraction of them has symptoms, and not every symptomatic patient develops hemochromatosis. On average, about 750,000 U.S. 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. Today, we estimate that 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.
Potential
Market in Post-Chemotherapy
Patients
undergoing chemotherapy irrespective of the type of cancer are at risk. That said, older patients aged 65+ and above, have a higher risk
of developing post-chemotherapy side effects such as digestive issues, sleep and memory issues, fatigue, and body aches. Other risk factors
include the presence of metabolic conditions, dose levels of chemotherapy medication, blood count, and BMI.US CDC health data estimates
that 650,000 patients receive chemotherapy annually in the US, with 45% experiencing severe side effects and 40% having more moderate
side effects. Thus, approximately 158,000 patients were diagnosed with the adverse effects of chemotherapy (antineoplastic and immunosuppressive
drugs. Today, Neulasta is given to patients undergoing chemotherapy as a “catch-all” therapeutic to help manage side effects.
Neulasta is typically given to patients after chemotherapy for recovery.
Competition
We
are subject to competition from pharmaceutical and biotechnology companies and academic and research institutions. Nearly all of our
competitors have significantly more resources and experience than we do.
TELOMIR-1
will face competition in its initial target indication, osteoarthritis, as well as in its potential second target indication hemochromatosis
followed by post-chemotherapy recovery.
In
osteoarthritis, TELOMIR-1 would compete as a treatment with a variety of pharmacologic compounds, including NSAIDs, corticosteroids,
hyaluronic acid, opioids, stem cell injections, platelet injects, and nutritional supplements. Since there is presently no known cure,
the front line treatment for osteoarthritis is pain management, although some treatment options seek to reverse osteoarthritis, which
is what we are targeting for TELOMIR-1.
In
hemochromatosis, as noted above, the current standard of care is phlebotomy, which typically costs $80 - $300 per visit in community
health facilities or offered for free at blood banks. Based on historical treatment data, phlebotomy serves more than 95% of hemochromatosis
patients, as there are no FDA-approved therapeutics for this indication. Patients who use phlebotomy continue to embrace it given the
lack of clinical options, its broad accessibility and low treatment cost. However, this option is not available to patients with hemophilia
and other blood disorders. About 5% of patients are treated with iron chelators such as Exjade and Jadenu (and associated generics).
These options are more expensive with pricing for Exjade at $532 per day and Jadenu at $517 per day. The last treatment option, deferasirox
/ deferoxamine was used with only 7,000 patients (2018-2022) based on IQVIA claims and scripts data. Future clinical options for treating
hemochromatosis exist and remain limited. For example, 2 molecules are pre-clinical, 2 in Phase 1, 1 in Phase 2, and 1 is in Phase 3
clinicial trials.
In
post-chemo recovery, the current standard of care involves prescribing a small set of compounds designed to reduce patient
discomfort from side effects. Filgrastim, pegfilgrastim, sargramostim are some examples of prescribed medication. These medications
stimulate the growth of healthy white blood cells and granulocytes by subcutaneous injection. However, prices are high and can cost
around $4,000 to $6,400 per dose of injection. Jakafi, a tyrosine kinase inhibitor, is used in polycythemia vera and
graft-versus-host disease. Pricing for Jakafi is $16,200 per bottle, with a $270 wholesale acquisition cost (WAC) unit price. Thus,
given the cost, only a portion of the high-risk patients are given the script; these are usually patients who are older than 65, and
with a weakened immune system and low white blood cell count. Patients who use the class of medication do so to reduce
post-treatment infection risk, even if they suffer from fevers and aches because of usage. The development pipeline of competing
assets is robust but targeted at specific side effects such as iron deficiency or thrombotic constraints.
Intellectual
Property
We
license the U.S. patent rights for the use of TELOMIR-1 in human applications from MIRALOGX, LLC (“MIRALOGX”), an 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 our company
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.
U.S.
Government Regulation
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 clinical trial subject or his or her legal representative and must monitor the clinical
trial until completed. There also are requirements governing the reporting of ongoing clinical trials and completed clinical trial results
to public registries. Information about most clinical trials must be submitted within specific timeframes for publication on the www.clinicaltrials.gov
website. Information related to the product, patient population, phase of investigation, study sites and investigators and other aspects
of the clinical trial is made public as part of the registration of the clinical trial. Sponsors are also obligated to disclose the results
of their clinical trials after completion. Disclosure of the results of these trials can be delayed in some cases for up to two years
after the date of completion of the trial. Competitors may use this publicly available information to gain knowledge regarding the progress
of development programs.
Human
clinical trials are typically conducted in three sequential phases, which may overlap or be combined:
Post-approval
trials, sometimes referred to as Phase IV clinical trials, may be conducted after initial marketing approval. These trials are used to
gain additional experience from the treatment of patients in the intended therapeutic indication, particularly for long-term safety follow
up. In certain instances, the FDA may mandate the performance of Phase IV clinical trials as a condition of approval of an NDA or a Biologics
License Application (“BLA”).
Progress
reports detailing the results of the clinical trials must be submitted at least annually to the FDA and more frequently if significant
adverse events (“SAEs”) occur. The FDA or the sponsor may suspend or terminate a clinical trial at any time, or the FDA may
impose other sanctions on various grounds, including a finding that the research patients are being exposed to an unacceptable health
risk. Similarly, an IRB can refuse, suspend, or terminate approval of a clinical trial at its institution if the clinical trial is not
being conducted in accordance with the IRB’s requirements or if the drug has been associated with unexpected serious harm to patients.
Concurrently
with clinical trials, companies usually complete additional pre-clinical studies and must also develop additional information about the
physical characteristics of the drug or biological product as well as finalize a process for manufacturing the product in commercial
quantities in accordance with cGMP requirements. The manufacturing process must be capable of consistently producing quality batches
of the product candidate and, among other things, the sponsor must develop methods for testing the identity, strength, quality, potency,
and purity of the final biological product. Additionally, appropriate packaging must be selected and tested, and stability studies must
be conducted to demonstrate that the biological product candidate does not undergo unacceptable deterioration over its shelf life.
Marketing
Approval
Assuming
successful completion of the required clinical testing, the results of the pre-clinical studies and clinical trials, together with detailed
information relating to the product’s chemistry, manufacture, controls, and proposed labeling, among other things, are submitted
to the FDA as part of an NDA requesting approval to market the product for one or more indications. In most cases, the submission of
an NDA is subject to a substantial application user fee.
The
review process typically takes twelve months from the date the NDA is submitted to the FDA. The FDA conducts a preliminary review of
all NDAs within the first 60 days after submission to determine whether they are sufficiently complete to permit substantive review before
accepting them for “filing.” The FDA may request additional information rather than accept an NDA for filing. In this event,
the application must be resubmitted with the additional information and may be subject to an additional application user fee. The resubmitted
application is also subject to review before the FDA accepts it for filing. Once the submission is accepted for filing, the FDA begins
an in-depth substantive review. The FDA reviews an NDA to determine, among other things, whether the drug is safe and effective and whether
the facility in which it is manufactured, processed, packaged, or held meets standards designed to assure the product’s continued
safety, quality and purity. Under the current guidelines in effect in the Prescription Drug User Fee Act (PDUFA), the FDA has a goal
to review and act on the submission within ten months from the completion of the preliminary review of a standard NDA for a new molecular
entity.
The
FDA also may require submission of a REMS plan to ensure that the benefits of the drug outweigh its risks. The REMS plan could include
medication guides, physician communication plans, assessment plans, and/or elements to assure safe use, such as restricted distribution
methods, patient registries, or other risk minimization tools.
The
FDA may refer an application for a novel drug to an advisory committee. An advisory committee is a panel of independent experts, including
clinicians and other scientific experts, that reviews, evaluates and provides a recommendation as to whether the application should be
approved and under what conditions. The FDA is not bound by the recommendations of an advisory committee, but it considers such recommendations
carefully when making decisions.
Before
approving an NDA, the FDA typically will inspect the facility or facilities where the product is manufactured. The FDA will not approve
an application unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate
to assure consistent production of the product within required specifications. Additionally, before approving an NDA, the FDA may inspect
one or more clinical trial sites to assure compliance with GCP requirements.
After
evaluating the NDA and all related information, including the advisory committee recommendation, if any, and inspection reports regarding
the manufacturing facilities and clinical trial sites, the FDA may issue an approval letter, or, in some cases, a complete response letter.
A complete response letter generally contains a statement of specific conditions that must be met in order to secure final approval of
the NDA and may require additional clinical trials or pre-clinical studies in order for FDA to reconsider the application. Even with
submission of this additional information, the FDA ultimately may decide that the application does not satisfy the regulatory criteria
for approval. If and when those conditions have been met to the FDA’s satisfaction, the FDA will typically issue an approval letter.
An approval letter authorizes commercial marketing of the drug with specific prescribing information for specific indications.
Post-approval
requirements
Drugs
manufactured or distributed pursuant to FDA approvals are subject to pervasive and continuing regulation by the FDA, including, among
other things, requirements relating to recordkeeping, periodic reporting, product sampling and distribution, advertising and promotion
and reporting of adverse experiences with the product. After approval, most changes to the approved product, such as adding new indications
or other labeling claims are subject to prior FDA review and approval. There also are continuing annual user fee requirements for any
marketed products and the establishments at which such products are manufactured, as well as new application fees for supplemental applications
with clinical data.
Employees
and Human Capital Resources
As
of March 15, 2024, we had 1 full-time employee, our chief financial officer, and 8 part-time employees, of which one of our part-time
employees is engaged in research and development. None of our employees is represented by a labor union or are covered by a collective
bargaining agreement. We consider our relationship with our employees to be satisfactory. In addition, we utilize the services of contractors
and part-time outside consultants to support our organization’s needs. We expect to continue to build our team to ensure we can
effectively execute our development plans.
Legal
Proceedings
There
are no material proceedings to which any director or officer, or any associate of any such director or officer, is a party that is adverse
to our Company or any of our subsidiaries or has a material interest adverse to our Company or any of our subsidiaries. No director or
executive officer has been a director or executive officer of any business which has filed a bankruptcy petition or had a bankruptcy
petition filed against it during the past ten years. No current director or executive officer has been convicted of a criminal offense
or is the subject of a pending criminal proceeding during the past ten years. No current director or executive officer has been the subject
of any order, judgment or decree of any court permanently or temporarily enjoining, barring, suspending or otherwise limiting his involvement
in any type of business, securities or banking activities during the past ten years. No current director or officer has been found by
a court to have violated a federal or state securities or commodities law during the past ten years. From time to time, we may be named
in claims arising in the ordinary course of business.
We
anticipate that we will expend significant financial and managerial resources in the defense of our intellectual property rights in the
future if we believe that our rights have been violated. We also anticipate that we will expend significant financial and managerial
resources to defend against claims that our products and services infringe upon the intellectual property rights of third parties.
Corporation
Information
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.
Our
corporate headquarters is located at 855 N Wolfe Street, Suite 601, Baltimore, Maryland 21205. Our telephone number is (737) 289-0835.
Our
website address is www.telomirpharma.com. The information contained on, or that can be accessed through, our website is deemed not to
be incorporated in this Annual Report or to be part of this Annual Report. You should not consider the information contained on our website
to be part of this Annual Report.
ITEM
1A. Risk Factors
RISK
FACTORS
Investing
in shares of our common stock is very speculative and involves a high degree of risk. You should carefully consider the risks
and uncertainties described below, the section of this Annual Report entitled “Management’s Discussion and Analysis of Financial
Condition and Results of Operations” and our financial statements and related notes included elsewhere in this Annual Report. The
risks and uncertainties described below are not the only ones we face. Additional risks and uncertainties that we are unaware of, or
that we currently believe are not material, may also become important factors that affect us. If any of the following risks occur, our
business, operating results and prospects could be materially harmed. In that event, the price of our common stock could decline, and
you could lose part or all of your investment.
Important
factors that could cause actual results or events to differ materially, but are not limited to, the following:
● our use of the net proceeds from this offering;
● the timing of anticipated regulatory filings;
● the timing of availability of data from our clinical trials;
● our ability to recruit and enroll suitable patients in our clinical trials;
● developments relating to our competitors and our industry;
Risks
Related to Our Intellectual Property
We
depend on rights to TELOMIR-1 that are or will be licensed to us. We do not own the intellectual property rights to TELOMIR-1 and any
loss of our rights to it could prevent us from selling our product.
Within
our present and future pipeline of treatments, TELOMIR-1 is in-licensed from another company. We do not currently own any intellectual
property rights, including the patent application that underlies this license. Our rights to use TELOMIR-1 is subject to the negotiation
of, continuation of and compliance with the terms of this license. Thus, the non-provisional patent application is not written by us
or our attorneys, and we did not have control over the drafting and prosecution. The patent owner and our licensor might not have given
the same attention to the drafting and prosecution of these patents and applications as we would have if we had been the owner of the
patent application and had control over the drafting. We cannot be certain that drafting of the licensed patent application, or patent
prosecution, by the licensor have been or will be conducted in compliance with applicable laws and regulations or will result in valid
and enforceable patents and other intellectual property rights. This absence of control over the drafting, prosecution of patent and
applications, along with non-compliance with royalty payments and confidentiality breaches are just some of the ways that may result
in the Company’s’ loss of the license and inability to continue operations.
Significant
additional research and development activity, pre-clinical testing, and/or clinical testing TELOMIR-1 is required before we will have
a chance to achieve a viable product for licensing or commercialization. Our business currently depends entirely on the successful development,
regulatory approval, and licensing or commercialization of our product candidate, which may never occur.
Enforcement
of our licensed patent application or defense of any claims asserting invalidity of these patents is often subject to the control or
cooperation of our licensor. Legal action could be initiated against the owners of the intellectual property that we license and an adverse
outcome in such legal action could harm our business because it might prevent such companies or institutions from continuing to license
intellectual property that we may need to operate our business. In addition, such licensor may resolve such litigation in a way that
benefits it but adversely affects our ability to have freedom to operate to develop and commercialize TELOMIR-1.
We
may not be able to adequately protect our product candidates or our proprietary technology in the marketplace.
Our
success will depend, in part, on our ability to obtain patents, protect our trade secrets and operate without infringing on the proprietary
rights of others. We may rely upon a combination of patents, trade secret protection (i.e., know-how), trademarks, licenses, and confidentiality
agreements to protect the intellectual property of our product candidates. The strengths of patents in the pharmaceutical field involve
complex legal and scientific questions and can be uncertain. Where appropriate, we seek patent protection for certain aspects of our
products and technology. However, patent protection for naturally occurring compounds is exceedingly difficult to obtain, defend and
enforce. Filing, prosecuting and defending patents throughout the world would be prohibitively expensive, so our policy is to look to
patent technologies with commercial potential in jurisdictions with significant commercial opportunities. However, patent protection
may not be available for some of the products or technology we are developing. If we must spend significant time and money protecting,
defending, or enforcing our patents, designing around patents held by others or licensing, potentially for large fees, patents or other
proprietary rights held by others, our business, results of operations and financial condition may be harmed. We may not develop additional
proprietary products that are patentable.
The
patent positions of pharmaceutical products are complex and uncertain. Although we have sought and expect to continue to seek patent
protection for our product candidates, their methods of use, and methods of manufacture, any, or all of them may not be subject to effective
patent protection. If any of our products are approved and marketed for an indication for which we do not have an issued patent, our
ability to use our patents to prevent a competitor from commercializing a non-branded version of our commercial products for that non-patented
indication could be significantly impaired or even eliminated.
Publication
of information related to our product candidates by us, or others may prevent us from obtaining or enforcing patents relating to these
products and product candidates. Furthermore, others may independently develop similar products, may duplicate our products, or may design
around our patent rights. In addition, any of our issued patents may be opposed and/or declared invalid or unenforceable. If we fail
to adequately protect our intellectual property, we may face competition from companies who attempt to create a generic product to compete
with our product candidates. We may also face competition from companies who develop a substantially similar product to one of our product
candidates that is not covered by any of our patents.
Many
companies have encountered significant problems in protecting, defending and enforcing intellectual property rights in foreign jurisdictions.
The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents and other
intellectual property rights, particularly those relating to pharmaceuticals, which could make it difficult for us to stop the infringement
of our patents or marketing of competing products in violation of our proprietary rights generally. Proceedings to enforce our patent
rights in foreign jurisdictions could result in substantial cost and divert our efforts and attention from other aspects of our business.
Currently,
we do not own the rights to the intellectual property and technology that will be used to commercially develop our initial product
candidate, TELOMIR-1. MIRALOGX, which is a separate intellectual property development company owned by a trust established by the
Company’s founder, holds the patent rights to TELOMIR-1, which are currently comprised of a pending non-provisional patent
application. Pending the issuance of the non-provisional patent application, we will have an exclusive, license from MIRALOGX to
develop and commercialize TELOMIR-1 in the U.S. for human and non-human applications. The term of the license will continue through
the date of the expiration of the last-to-expire licensed patent or, if later, the date of the expiration of the last strategic
partnership/sublicensing agreement covering the licensed products. The licensed patent rights are expected to extend through 2043.
We expect additional patent terms may be awarded, including additional patent terms based on the time for regulatory review of drug
products. There are no up-front, execution, or milestone payments required under the license agreement. Further, no payments have
been made to date under the agreement. We are also required to pay an 8% royalty on net sales or revenue in exchange for an
exclusive, worldwide license to patent rights, and we may bring suit in our own name to enforce our patent rights under the license
agreement. In the event we are unable to enforce our rights under the agreement or are unable to detect unauthorized use of our
intellectual property, we may lose the benefit of the licensed rights used to commercially develop TELOMIR-1. MIRALOGX will control
the prosecution of the patent applications for TELOMIR-1.
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.
There
is a substantial amount of litigation, both within and outside the U.S., involving patent and other intellectual property rights in the
pharmaceutical industry. We may, from time to time, be notified of claims that we are infringing upon patents, trademarks, copyrights,
or other intellectual property rights owned by third parties, and we cannot provide assurances that other companies will not, in the
future, pursue such infringement claims against us, our commercial partners or any third-party proprietary technologies we have licensed.
If we were found to infringe upon a patent or other intellectual property right, or if we failed to obtain or renew a license under a
patent or other intellectual property right from a third party, or if a third party that we were licensing technologies from was found
to infringe upon a patent or other intellectual property rights of another third party, we may be required to pay damages, including
damages of up to three times the damages found or assessed, if the infringement is found to be willful, suspend the manufacture of certain
products or reengineer or rebrand our products, if feasible, or we may be unable to enter certain new product markets. Any such claims
could also be expensive and time consuming to defend and divert management’s attention and resources. Our competitive position
could suffer as a result. In addition, if we have declined or failed to enter into a valid non-disclosure or assignment agreement for
any reason, we may not own the invention or our intellectual property, and our products may not be adequately protected. Thus, we cannot
guarantee that our product candidates, or our commercialization thereof, does not and will not infringe any third party’s intellectual
property.
We
have been granted a license to the right to develop TELOMIR-1 in the United States in human application, but we have not been granted
a license to the rights to patents covering TELOMIR-1 in foreign jurisdictions.
We
have been granted a license to the right to develop TELOMIR-1 in the United States but not in countries outside the United States, as
MIRALOGX has retained all rights outside the United States and may license such rights to other parties. Accordingly, MIRALOGX potentially
could develop a competing product for such jurisdictions outside of the United States.
Risks
Related to Our Operations and Financial Condition
We
are an early development-stage company with no revenues.
As
very early development-stage enterprise that is focused on the development of a pre-clinical pharmaceutical product, we have generated
no revenue and have an accumulated deficit of $14.1 million and $1.0 million as of December 31, 2023 and December 31, 2022, respectively.
There can be no assurance that sufficient funds required to pursue our development program will be generated from operations or that
funds will be available from external sources, such as debt or equity financings or other potential sources. The lack of additional capital