CNS Pharmaceuticals, Inc. 10-K
Table
of Contents
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C., 20549
FORM 10-K
For the fiscal year ended December 31, 2025
OR
For the transition period from _________________
to ___________________
Commission File Number: 001-39126
CNS Pharmaceuticals, Inc.
(Exact Name of Registrant as Specified in its Charter)
2100 West Loop South, Suite 900
Houston, Texas77027
(Address of Principal Executive Offices) (Zip Code)
Registrant’s Telephone Number, including
Area Code: 800-946-9185
Securities registered pursuant to Section 12(b) of the Exchange
Act:
Title of each class Trading Symbol(s) Name of each exchange on which registered
Common Stock CNSP The NASDAQ Stock Market LLC
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
periods as 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 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 the definitions
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 included 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 Act). YES ☐ NO ☒
The aggregate market value of the registrant’s voting equity
held by non-affiliates of the registrant, computed by reference to the price at which the common stock was last sold as of the last business
day of the registrant’s most recently completed second fiscal quarter, was $5.0 million. In determining the market value of the
voting equity held by non-affiliates, securities of the registrant beneficially owned by directors, officers and 10% or greater shareholders
of the registrant have been excluded. This determination of affiliate status is not necessarily a conclusive determination for other purposes.
The number of shares of the registrant’s common stock outstanding
as of March 31, 2026 was 811,449.
DOCUMENTS INCORPORATED BY REFERENCE
Portions of this registrant’s definitive proxy statement for
its 2026 Annual Meeting of Stockholders to be filed with the SEC no later than 120 days after the end of the registrant’s fiscal
year are incorporated herein by reference in Part III of this Annual Report on Form 10-K.
TABLE OF CONTENTS
Page
PART I
ITEM 1. Business 1
ITEM 1A. Risk Factors 15
ITEM 1B. Unresolved Staff Comments 30
ITEM 1C. Cybersecurity 31
ITEM 2. Properties 32
ITEM 3. Legal Proceedings 32
ITEM 4. Mine Safety Disclosures 32
PART II
ITEM 6. [RESERVED] 33
ITEM 7A. Quantitative and Qualitative Disclosures About Market Risks 36
ITEM 8. Financial Statements and Supplementary Data 37
ITEM 9A. Controls and Procedures 57
ITEM 9B. Other Information 58
ITEM 9C. Disclosure Regarding Foreign Jurisdictions that Prevent Inspections 58
PART III
ITEM 10 Directors, Executive Officers and Corporate Governance 59
ITEM 11 Executive Compensation 59
ITEM 14 Principal Accountant Fees and Services 60
PART IV
ITEM 15 Exhibits, Financial Statement Schedules 61
Exhibit Index 61
Signatures 65
i
References in this Form 10-K to “we”,
“us”, “its”, “our” or the “Company” are to CNS Pharmaceuticals, Inc., as appropriate to
the context.
Cautionary Statement About
Forward-Looking Statements
We make forward-looking statements under the “Risk
Factors,” “Business,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations”
and in other sections of this report. In some cases, you can identify these statements by forward-looking words such as “may,”
“might,” “should,” “would,” “could,” “expect,” “plan,” “anticipate,”
“intend,” “believe,” “estimate,” “predict,” “potential” or “continue,”
and the negative of these terms and other comparable terminology. These forward-looking statements, which are subject to known and unknown
risks, uncertainties and assumptions about us, may include projections of our future financial performance based on our growth strategies
and anticipated trends in our business. These statements are only predictions based on our current expectations and projections about
future events. There are important factors that could cause our actual results, level of activity, performance or achievements to differ
materially from the results, level of activity, performance or achievements expressed or implied by the forward-looking statements. In
particular, you should consider the numerous risks and uncertainties described under “Risk Factors”.
While we believe we have identified material risks,
these risks and uncertainties are not exhaustive. Other sections of this report may describe additional factors that could adversely impact
our business and financial performance. Moreover, we operate in a very competitive and rapidly changing environment. New risks and uncertainties
emerge from time to time, and it is not possible to predict all risks and uncertainties, nor can we assess the impact of all factors on
our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained
in any forward-looking statements.
Although we believe the expectations reflected
in the forward-looking statements are reasonable, we cannot guarantee future results, level of activity, performance or achievements.
Moreover, neither we nor any other person assumes responsibility for the accuracy or completeness of any of these forward-looking statements.
You should not rely upon forward-looking statements as predictions of future events. We are under no duty to update any of these forward-looking
statements after the date of this report to conform our prior statements to actual results or revised expectations, and we do not intend
to do so.
Forward-looking statements include, but are not
limited to, statements about:
· our ability to obtain additional funding to develop our product candidates;
· the need to obtain regulatory approval of our product candidates;
· the success of our clinical trials through all phases of clinical development;
· our ability to commercialize our product candidates;
ii
· market acceptance of our product candidates;
· competition from existing products or new products that may emerge;
· potential product liability claims;
· our ability to adequately support future growth; and
We caution you not to place undue reliance on the
forward-looking statements, which speak only as of the date of this report in the case of forward-looking statements contained in this
report.
You should not rely upon forward-looking statements
as predictions of future events. Our actual results and financial condition may differ materially from those indicated in the forward-looking
statements. We qualify all of our forward-looking statements by these cautionary statements. Although we believe that the expectations
reflected in the forward looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.
Therefore, you should not rely on any of the forward-looking statements. In addition, with respect to all of our forward-looking statements,
we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of
1995.
iii
PART I
Item 1. Business.
Overview
CNS Pharmaceuticals, Inc. (“CNS,” the “Company,”
“we,” “us,” or “our”) is a biotechnology company focused on developing innovative therapies for serious
diseases in neurology and oncology. Together, these therapeutic areas represent hundreds of billions of dollars in annual global pharmaceutical
spending, encompass some of the most prevalent and difficult to treat diseases affecting humanity, and are characterized by significant
unmet medical need. Significant progress has been made in the biological and molecular understanding of these diseases, enabling a shift
to high-value disease-modifying therapies and precision medicine approaches. Together with advancements in diagnostic capabilities to
aid in patient selection, there is the potential to improve the probability of success and reduce development timelines. We believe this
combination of scale, urgency, and scientific development creates compelling opportunities for differentiated therapeutic approaches and
novel mechanisms of action. Our executive team was purposefully built to focus on high-value therapeutic opportunities in these therapeutic
areas and the Company is working to build a differentiated portfolio of assets with best-in-class potential to address significant unmet
medical needs. CNS Pharmaceuticals is committed to advancing novel treatments that have the potential to improve patient outcomes.
Recent Developments
On January 1, 2026, the Company appointed Rami Levin,
MBA, as President & Chief Executive Officer. Following Mr. Levin’s appointment, we engaged an independent life science focused
advisory firm to conduct a comprehensive data-driven analysis of the Company including our pipeline, development priorities and long-term
positioning (the “Strategic Analysis”). While this Strategic Analysis was being completed, we rebuilt the Company’s
executive leadership team by appointing a new Chief Business Officer, Chief Financial Officer, Chief Medical Officer and Chief Technology
Officer effective March 2, 2026. This executive team was assembled to execute on the findings and recommendations of the Strategic Analysis
and develop and execute a new corporate strategy.
On March 11, 2026, we announced a new corporate
growth strategy focused on building a high-value pipeline in neurology and oncology. This strategy follows the comprehensive, data-driven
strategic review that incorporated clinical probability-of-success modeling, competitive landscape assessments, regulatory pathway analyses,
and risk-adjusted return evaluations. Based on this work, we are pursuing a disciplined approach to identify, acquire or in-license differentiated
preclinical and clinical-stage assets with strong biological rationale, validated or emerging clinical data, and clear development and
regulatory pathways. We are prioritizing opportunities with near- to mid-term value inflection points, meaningful commercial potential,
and relevance to our team’s expertise, while maintaining capital discipline. We have initiated a global asset search and are actively
evaluating opportunities aligned with these criteria, with a focus on areas where investor interest, partnering activity, and long-term
value creation potential are strongest.
Also on March 11, 2026, as part of our new corporate
strategy, we announced that we are pivoting from the Company’s historical singular focus on glioblastoma multiforme (“GBM”).
The Company’s prior development programs centered on two investigational compounds, TPI 287 and Berubicin, both of which were designed
to penetrate the blood-brain barrier and target cancers of the central nervous system such as GBM. We believe patients with GBM need better
treatment options and that TPI 287 and Berubicin have the potential to address those needs, but they are not in line with our new strategic
direction. As a result, we intend to explore out-licensing opportunities for TPI 287 and Berubicin.
Market Opportunity: Neurology & Oncology
Neurological disorders represent a staggering public
health crisis of global proportions. The World Health Organization (“WHO”) and the Global Burden of Disease study identify
neurological conditions as among the leading causes of disability and death worldwide. According to WHO, neurological disorders affect
more than one billion people globally and are the leading cause of disability-adjusted life years (“DALYs”) when all conditions
are aggregated across the spectrum of central nervous system (“CNS”) disease. Neurological conditions share several characteristics
that make them particularly challenging to treat and commercially significant: they are largely chronic and progressive, they carry high
caregiver burdens, they predominantly affect aging populations, and many remain without adequate disease-modifying treatments.
Cancer is among the foremost causes of morbidity and
mortality worldwide, imposing an enormous burden on individuals, healthcare systems, and economies globally. According to the WHO, cancer
accounts for approximately 10 million deaths per year, which accounted for nearly one in six deaths globally. In 2022, the International
Agency for Research on Cancer (“IARC”) recorded nearly 20 million new cancer cases and approximately 9.7 million cancer-related
deaths worldwide. In the United States alone, the National Cancer Institute (“NCI”) estimated that approximately 2,041,910
new cancer cases will be diagnosed in 2025, with approximately 618,120 deaths attributable to the disease.
Our new strategic focus on neurology and oncology positions
us in two of the largest, fastest-growing, and most scientifically dynamic sectors in biopharmaceuticals. Together, these therapeutic
areas represent hundreds of billions of dollars of investment. The global neurological therapeutics market exceeds $138 billion while
the global oncology therapeutics market is projected to reach $400 billion in the next decade. However, several large neurology and oncology
indications still lack disease-modifying therapies or have poor outcomes. These large commercial opportunities have the potential to be
addressed by leveraging the recent advancements in the understanding of these diseases and breakthroughs in drug development.
As a result, there are significant research and development
efforts being undertaken across academia, non-profit institutions, governmental agencies, emerging biotechnology companies and multinational
pharmaceutical companies to address these disease indications. In 2025, neurology and oncology focused companies received nearly 50 percent
of the venture capital investments in therapeutics. They were also the most active therapeutic areas based on the number of business development
transactions completed from 2022 to 2025. Oncology was the most active therapeutic area with 457 announced transactions completed during
this period and neurology was the second most active with 205 announced completed transactions. During this period, total mergers &
acquisitions and licensing transactions exceeded $100 billion in total value for both neurology and oncology. This activity spanned stage
of development from preclinical through approved products as well as therapeutic modality, which we believe demonstrates the potential
value that can be created in these therapeutic areas with novel and differentiated assets.
Based on this, we believe that 1) there are a significant
number of assets in neurology and oncology that are available for in-licensing, and 2) there is an established group of potential strategic
commercial partners. Several global biopharmaceutical companies including but not limited to AstraZeneca, Astellas Pharma, Bristol Myers
Squibb, Biogen, GlaxoSmithKline, Merck, Novartis, Pfizer and Takeda have established franchises in neurology and/or oncology and have demonstrated
track records of acquiring or partnering with companies developing product candidates in our target areas.
Neurology Market Drivers
The need for differentiated and novel neurology therapeutics
is being driven by multiple factors including aging global demographics. The global population aged 65 and over is projected to more than
double from approximately 700 million in 2020 to 1.5 billion by 2050, according to UN demographic projections. Because the incidence of
most major neurodegenerative diseases, such as Alzheimer's disease and Parkinson's disease, increases sharply with age, this demographic
shift is expected to dramatically expand patient populations and healthcare costs worldwide. As an example, the U.S. Alzheimer's Association
has estimated that if no disease-modifying treatment is found, the number of Americans with Alzheimer's could grow to 13 million by 2050.
However, significant unmet medical need remains across nearly all areas of neurodegeneration. Until 2023 and 2024, there were no approved
therapies that demonstrated an ability to slow underlying neurodegeneration in Alzheimer’s disease. Current treatments for Parkinson's
disease manage symptoms but do not arrest neurodegeneration. No approved neuroprotective or disease-modifying therapies exist for amyotrophic
lateral sclerosis (“ALS”) or Huntington's disease, among many others. According to the National Institute of Neurological
Disorders and Stroke, there are a total of 131 neurologic disorders. However, a 2024 analysis by Thomas et al. published in Neurology
(A Comprehensive Review of Novel FDA-approved Neurological Medications from 2018-2023, P7-4.009, Vol. 102) found that only 32 novel neurological
therapies were approved by the FDA from 2018 – 2023 for patients with migraines (9), multiple sclerosis (4), Alzheimer’s disease
(3), Parkinson’s disease (3), Epilepsy/seizures (3), ALS (2), Neuromyelitis Optica (2), and several others for rare neurological
conditions. This unmet need underpins both the scientific urgency, medical need and the commercial opportunity.
Advancement in the biological understanding of these
diseases, aided by biomarker discovery and enhanced diagnostic capabilities, are also leading to advancement in the field and uncovering
new opportunities for drug development. The development of blood-based biomarkers for neuroinflammation, including amyloid tau protein
for Alzheimer’s, has dramatically reduced the cost and complexity of patient stratification in clinical trials, increasing feasibility
and accelerating development timelines. Fluid and imaging biomarkers are increasingly accepted by the FDA as surrogate endpoints, potentially
lowering the bar for early-stage proof of concept. Small and mid-sized biotechnology companies now originate the majority of novel neurology
compounds entering clinical development. According to the industry research and contract research organization, IQVIA, neurology is among
the fastest-growing areas of Phase II/III clinical trial activity.
In addition, neurological disorders have the potential
for accelerated regulatory pathways including breakthrough therapy, fast track, and priority review designations, which have the potential
to shorten development and approval timelines and enable increased interaction with the FDA. Finally, several commercial neurology therapies
are facing loss of exclusivity (“LOE”) by 2030. Based on these demographic, biological, medical, pharmacoeconomic and commercial
considerations, we believe neurology is a therapeutic area on which to focus and deploy our resources.
Oncology Market Drivers
Cancer rates increase dramatically with age, and
all major developed-market nations are experiencing rapid population aging. As noted above, IARC projects 33 million new cases per year
by 2050. This represents a 65% increase over 2022 levels and is being driven in large part by demographic shifts rather than changes in
per-capita risk. In the United States alone, the National Cancer Institute (NCI) estimated that approximately 2,041,910 new cancer cases
will be diagnosed in 2025, with approximately 618,120 deaths attributable to the disease. Estimated national expenditures for cancer care
in the United States reached approximately $208.9 billion in 2020 and are expected to grow materially as the population ages. As a result,
the global oncology pharmaceutical market is the largest and fastest-growing category within biopharmaceuticals.
The past decade has witnessed transformative advances
in cancer biology and drug development. Immune checkpoint inhibition, which is led by anti-programmed cell death 1 (anti-PD-1/PD-L1) and
anti-cytotoxic T-lymphocyte associated protein 4 (“CTLA-4”) antibodies have become foundational to the treatment of dozens
of cancers. These therapies generate over $40 billion in annual worldwide sales and have catalyzed billions of dollars in follow-on investment.
Chimeric antigen receptor T-cell (“CAR-T”) therapy has achieved durable remissions in subsets of hematologic malignancies.
Antibody-drug conjugate (“ADC”) technologies have experienced a major resurgence, with multiple approvals in breast cancer,
bladder cancer, lung cancer, and other solid tumors. However, there continues to be a need for targeted therapies that can improve overall
survival, quality of life and overall outcomes of patients with cancer. Several new modalities are emerging as promising cancer treatments
including but not limited to protein degraders, bispecific antibodies, and next-generation immunotherapies. We believe the robust investment
in cancer drug discovery has created an opportunity to identify and select assets, including those with an underlying validated platform
technology, to establish a differentiated oncology pipeline.
Recent treatment approaches in oncology also demonstrate
the shift towards precision medicine and the development of therapies with improved efficacy and safety profiles compared to non-targeted
chemotherapies and radiation. Genomic profiling using next-generation sequencing (“NGS”), liquid biopsy, and companion diagnostics
has transformed the standard of care for many cancers, enabling the matching of patients to targeted therapies based on specific molecular
alterations. This trend drives demand for biomarker-guided therapy development and creates opportunities for companies developing therapies
against specific genomic targets (e.g., KRAS, EGFR, ALK, BRAF, HER2, FGFR, RET, and many others). As of 2025, over 50 cancer-specific
molecular targets have FDA-approved companion diagnostics. Precision medicine has uncovered a broad array of validated biological targets
that continues to expand. This shift has created clearly defined addressable patient populations for which there are established clinical
benchmarks and potential accelerated regulatory pathways.
We expect neurology and oncology will continue
to evolve rapidly, which will reshape the competitive landscape in these areas, thereby creating opportunities for us to build our pipeline
and focus our future development efforts. Leveraging our team’s collective experience in these therapeutic areas and relevant functions,
we believe we are well positioned to capitalize on evolving market dynamics and a broad set of assets to build a pipeline that is novel,
differentiated and has the potential to be best-in-class.
Our Team
To execute our neurology and oncology focused strategy,
the Company appointed a new executive team in the first quarter of 2026 that brings decades of hands-on experience in neurology and oncology,
including rare diseases and across several therapeutic modalities. Our executive team has diverse experience in clinical development,
regulatory affairs, chemistry, manufacturing and controls (“CMC”), business development, finance, capital markets, commercialization
and company transformation.
The Company’s newly formed executive team includes:
· Rami Levin, Chief Executive Officer, appointed January 1, 2026
· Lynne Kelley, M.D., FACS, Chief Medical Officer, appointed March 2, 2026
· Dylan Wenke, Chief Business Officer, appointed March 2, 2026
· Steve O’Loughlin, Chief Financial Officer, appointed March 2, 2026
· Eric Faulkner, Chief Technology Officer, appointed March 2, 2026
Rami Levin, MBA, brings nearly 30 years of global
leadership experience across oncology, CNS, rare diseases, endocrinology, and cell and gene therapy, with a proven track record of scaling
organizations, advancing late-stage clinical programs, and driving transformative value creation. Previously, Mr. Levin held senior
leadership roles as President & Chief Executive Officer of Saniona and CEO of ImStem Biotechnology. He led strategic turnarounds,
sharpened pipeline focus, secured significant financing, and advanced multiple programs toward clinical and regulatory milestones. He
has also held senior commercial and operational leadership roles at Sobi, Merck Serono, and Schering AG, where he successfully launched
and scaled global brands including Rebif®, Betaseron®, Kuvan®, Orfadin®, and Gamifant®. Earlier in his career, he
played a central role in global brand strategy and lifecycle management for Rebif®, one of the world’s leading multiple sclerosis
therapies. Mr. Levin holds an MBA from the Recanati Business School at Tel Aviv University and a BSc in Biology from Tel Aviv University.
Lynne Kelley, M.D., served as Chief Medical Officer
at multiple public and private biotechnology and medical device companies, including TISSIUM, Servier Pharmaceuticals, X4 Pharmaceuticals
and Senseonics. She has led clinical development programs resulting in INDs, NDAs, PMAs, and breakthrough and orphan drug designations,
and has presented safety and efficacy data to FDA advisory committees resulting in unanimous approval votes. Dr. Kelley has also played
key roles in corporate strategy, fundraising and M&A, including contributing to multi-billion-dollar oncology transactions. Earlier
in her career, Dr. Kelley held senior leadership roles at Becton Dickinson, where she oversaw global medical affairs and regulatory strategy
across devices, drugs and combination products. She began her career in academic medicine as an Assistant Professor of Surgery and Interventional
Radiology at Yale University. Dr. Kelley holds an MD from Dartmouth Medical School and a BA in Biology from Boston University. She is
board certified in General Surgery and Vascular Surgery and is a Fellow of the American College of Surgeons.
Dylan Wenke, MBA, is an experienced corporate development
and strategic transactions leader with a strong track record in biotechnology business development, licensing and financing. He has originated
and executed high-value transactions across multiple therapeutic areas and modalities. Previously, he held senior business development
roles at Corbus Pharmaceuticals, Bluejay Therapeutics and EuMentis Therapeutics, where he led portfolio-shaping transactions, financings
and global partnering initiatives. Dylan holds an MBA from the Kelley School of Business at Indiana University and a B.Sc. in Chemistry
from the University of North Florida.
Steve O’Loughlin brings nearly 20 years of
corporate finance, capital markets, and business development experience in the biopharmaceutical industry. Most recently, he served as
Chief Financial Officer of Actinium Pharmaceuticals, Inc. (NYSE American: ATNM), a clinical-stage radiopharmaceutical company, where he
played a central role in the company’s growth and strategic development over a decade-long tenure. At Actinium, Mr. O’Loughlin
oversaw SEC and NYSE compliance, investor relations, FP&A, corporate governance, and treasury management. He coordinated the execution
of multiple capital markets and business development transactions including the exclusive European license agreement for a Phase 3 radiopharmaceutical
asset with Immedica Pharma AB, generating $35 million in upfront proceeds and up to $417 million in potential milestone payments, as well
as a preclinical research collaboration with Astellas Pharma, Inc. Prior to Actinium, Mr. O’Loughlin served as Vice President of
Finance and Corporate Development at Protea Biosciences Group, Inc., where he executed capital raises, supported the growth of the Company’s
bioanalytical services business built around its proprietary LAESI® mass spectrometry imaging technology and drove strategic
collaborations with leading research institutions including Memorial Sloan Kettering Cancer Center and Yale University. Mr. O’Loughlin
began his corporate career at Caliber I.D. (formerly Lucid Technologies) supporting the commercial launch of the FDA approved VivaScope® diagnostic
imaging device and the Company’s IPO. Mr. O’Loughlin started his career in investment banking at Jesup & Lamont and Forge
Financial Group focused on the life sciences industry. Mr. O’Loughlin holds a B.S. in Business with a concentration in Finance from
Ramapo College of New Jersey.
Eric Faulkner, MS, MBA, brings over 30 years of
global leadership, technical operations, quality management, product launch, and commercial experience within the biotechnology / pharma
industry. Mr. Faulkner has extensive experience in product development, validation, manufacturing operations, supply chain, commercialization,
and quality management systems across the following therapeutic areas: CNS, endocrinology, immunology, neurology, oncology, rare diseases,
and gene therapy. Previously, Mr. Faulkner was CTO at IO Biotech where he built and led the Technical Operations organization on the BLA
enabling submission of Cylembio®, a therapeutic cancer vaccine. Mr. Faulkner also held senior roles at Homology Medicines as Head
of CMC Management & External Manufacturing for the IND submissions of multiple AAV vector programs. In addition, Mr. Faulkner was
also the Product Operations Lead for the Rare Disease Business Unit at Shire (Takeda) overseeing product launch, commercialization and
life cycle management activities for Elaprase® (intrathecal delivery), Gattex®, Natpara®, and Plenadren®. Mr. Faulkner
also spent over 15 years at Biogen involved in the product development, technology transfers, validation, registration, and commercialization
of Avonex®, Avonex Pen®, Tysabri®, and Tecfidera®. Mr. Faulkner obtained an MBA from Boston University, a MS in Biotechnology
and Biomedical Science from the University of Massachusetts, Boston, and a BS in Biology from the State University of New York, Fredonia.
Mr. Faulkner is a Lean Six Sigma Green Belt and is an ISO 13485 certified auditor.
Our Strategy
Our goal is to build a pipeline of neurology and
oncology focused assets that have the potential to be best-in-class and to improve outcomes for patients. We are executing a decisive
strategic pivot to reposition the company for long-term success. Our strategy to achieve this is to:
Legacy GBM Assets
TPI 287
TPI 287 is an investigational chemotherapy agent
belonging to the abeotaxane class of compounds and is structurally related to the taxane family of microtubule-stabilizing agents. Taxanes
such as paclitaxel and docetaxel are widely used in oncology but historically have demonstrated limited penetration across the blood-brain
barrier, which has restricted their use in treating cancers involving the central nervous system.
TPI 287 was designed as a synthetic, lipophilic
taxane derivative capable of penetrating the blood-brain barrier and achieving therapeutic concentrations within the central nervous system.
Like other taxanes, TPI 287 stabilizes microtubules, disrupting cell division and inducing apoptosis in rapidly proliferating tumor cells.
TPI 287 has been evaluated in multiple early-phase
clinical studies involving more than 300 patients across several oncology indications, including glioblastoma, metastatic breast cancer
with brain metastases, non-small cell lung cancer, castration-resistant prostate cancer and neuroblastoma.
A Phase 1/2 clinical study evaluated TPI 287 in
combination with bevacizumab in patients with recurrent glioblastoma. In that study, the combination demonstrated an objective response
rate of approximately 54%, including two complete responses, and a disease control rate of approximately 92%. The regimen was generally
well tolerated in the study population.
In July 2024, the Company entered into an Exclusive
License Agreement with Cortice Biosciences, Inc. pursuant to which the Company obtained exclusive rights to certain intellectual property
related to TPI 287 in the United States, Canada, Mexico and Japan.
The Company is currently exploring out-licensing
opportunities for TPI 287 to advance its development.
Berubicin
Berubicin is an investigational anthracycline chemotherapy
agent originally discovered at The University of Texas M.D. Anderson Cancer Center.
Anthracyclines represent one of the most widely
used classes of chemotherapy agents in oncology. Historically, however, anthracyclines have demonstrated limited penetration of the blood-brain
barrier, which has restricted their use in treating cancers of the central nervous system.
Preclinical and early clinical studies have suggested
that Berubicin may be capable of penetrating the blood-brain barrier and achieving therapeutic concentrations in brain tumors.
Berubicin was previously evaluated in Phase 1 clinical
trials conducted by Reata Pharmaceuticals in patients with recurrent malignant gliomas. In that study, 25 patients were evaluable for
response and the trial observed one complete response, one partial response and several cases of stable disease, representing a disease
control rate of approximately 44%. One patient who achieved a complete response remained disease-free more than 17 years following treatment
before passing away from causes unrelated to his GBM diagnosis.
The Company subsequently conducted a randomized
Phase 2 superiority clinical trial, known as CNS-201, evaluating Berubicin compared to lomustine in patients with recurrent glioblastoma
who had failed first-line therapy. In March 2025, the Company announced topline results from the primary analysis of the trial. While
Berubicin demonstrated clinical activity and outcomes that appeared comparable to lomustine across several endpoints, the trial did not
meet its primary end point in showing superiority compared to lomustine in overall survival.
The Company’s current plan for the Berubicin
program is to complete the trial, close the trial sites and prepare the Clinical Study Report for the CNS-201 trial. As with TPI 287,
we are currently exploring out-licensing opportunities that would enable further development of Berubicin.
Competition
The biotechnology and pharmaceutical industries
are characterized by intense and rapidly evolving competition. We face significant competition from a variety of companies, including
major pharmaceutical and biotechnology companies, early stage biotechnology companies, academic research institutions and other public
and private research organizations. Many of our competitors have substantially greater financial, technical, manufacturing, marketing,
and human resources than we do. Our competitors may succeed in developing products that are safer, more effective, or less costly than
any product candidates we may develop, which could render our product candidates non-competitive or obsolete.
Our strategic expansion into neurology brings us
into competition with one of the broadest and most active therapeutic areas in the pharmaceutical industry. Neurological disorders including,
but not limited to, neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, and ALS, as well as epilepsy, multiple
sclerosis, and neuropathic pain conditions represent large markets with substantial commercial interest. Competitors in neurology include
major pharmaceutical companies with dedicated CNS franchises, such as Biogen, UCB, Novartis, AbbVie, and Eisai, as well as a large and
growing number of clinical-stage biotechnology companies. The neurology space has experienced a significant resurgence of investment and
pipeline activity, driven in part by recent regulatory approvals for novel disease-modifying therapies in Alzheimer's disease and other
conditions, which has further increased competition for development resources, clinical investigators, and patient populations in clinical
trials.
The broader oncology market is among the most competitive
segments in the pharmaceutical industry. Large, well-capitalized companies such as AstraZeneca, Bristol-Myers Squibb, Roche, Merck &
Co., Pfizer, Eli Lilly, and Johnson & Johnson, among many others, maintain extensive oncology pipelines with significant clinical,
regulatory, and commercial infrastructure. In addition, numerous mid-size and emerging biotechnology companies are actively developing
oncology programs across modalities including targeted therapies, immunooncology agents, cell and gene therapies, radiopharmaceuticals,
and RNA-based medicines. We expect competition in oncology to intensify as scientific understanding of tumor biology continues to advance
and as additional modalities enter clinical development. Our ability to compete will depend on our ability to identify differentiated
targets and mechanisms of action, advance product candidates through clinical development efficiently, establish intellectual property
positions that provide meaningful market exclusivity, and enter into strategic partnerships or licensing arrangements that augment our
resources and expertise.
Our strategy depends on our ability to identify
and secure assets that are differentiated. In addition to pharmaceutical and biotechnology companies with established pipelines, we also
face competition from companies that also seek to acquire or in-license assets including companies such as Roivant, Ligand Pharmaceuticals,
Fortress Biosciences and several other private and public companies. These companies have more established track records of acquiring
or in-licensing assets, advancing assets through development and regulatory requirements and commercial capabilities than we do.
Many of our current or future competitors have
significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, conducting clinical
trials, obtaining regulatory approvals, obtaining reimbursement for and marketing of approved products than we do. Mergers and acquisitions
in the biotechnology, pharmaceutical and diagnostic industries may result in even more resources being concentrated among a smaller number
of our competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative
arrangements with large and established companies. These competitors also compete with us in recruiting and retaining qualified personnel
and establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary
to, or necessary for, our programs.
Intellectual Property
Our intellectual property is comprised of patents
and know-how related to our legacy assets TPI 287 and Berubicin. Existing TPI 287 patents will expire in 2028. When we licensed TPI 287
from Cortice on July 29, 2024, it had previously been granted Orphan Drug Designation (“ODD”) by the FDA. On June 10, 2020,
the FDA granted Orphan Drug Designation for Berubicin for the treatment of malignant gliomas. ODD from the FDA is available for drugs
targeting diseases with less than 200,000 cases per year. ODD may enable market exclusivity of 7 years from the date of approval of a
New Drug Application (“NDA”) in the United States. During that period the FDA generally could not approve another product
containing the same drug for the same designated indication. Orphan drug exclusivity will not bar approval of another product under certain
circumstances, including if a subsequent product with the same active ingredient for the same indication is shown to be clinically superior
to the approved product on the basis of greater efficacy or safety, or providing a major contribution to patient care, or if the company
with orphan drug exclusivity is not able to meet market demand. We do not hold or license any patents related to Berubicin and the ODD
now constitutes our primary intellectual property protections although the Company is exploring if there are other patents that could
be filed related to Berubicin to extend additional protections.
On July 24,
2021, the Company received Fast Track Designation from the FDA for Berubicin. Fast Track Designation is designed to facilitate the
development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
We are exploring the possibility to file additional
patent applications that potentially will allow for further increase of the exclusive market protection for use of TPI 287 and Berubicin.
However, we can provide no assurance that we will be able to file or receive additional patent protection. The failure to receive such
additional patent protection will reduce the barrier to entry for competition for TPI 287 and Berubicin, which may adversely affect our
ability to out-license either of these programs.
Governmental Regulation
Government authorities in the United States, at
the federal, state and local level, and in other countries extensively regulate, among other things, the research, development, testing,
manufacture, quality control, approval, labeling, packaging, storage, record-keeping, promotion, advertising, distribution, post-approval
monitoring and reporting, marketing and export and import of products such as those we are developing. The pharmaceutical drug product
candidates that we develop must be approved by the FDA before they may be marketed and distributed.
In the United States, the FDA regulates pharmaceutical
products under the Federal Food, Drug, and Cosmetic Act, and implementing regulations. Pharmaceutical products are also subject to other
federal, state and local statutes and regulations. The process of obtaining regulatory approvals and the subsequent compliance with appropriate
federal, state, local and foreign statutes and regulations require 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 administrative or judicial sanctions. FDA and related enforcement activity could include refusal to approve
pending applications, withdrawal of an approval, a clinical hold, 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. Any agency or judicial enforcement action could have a material adverse effect on us. The process required by the FDA before
a pharmaceutical product may be marketed in the United States generally involves the following:
· Submission to the FDA of an NDA for a new pharmaceutical product;
· FDA review and approval of the NDA.
The lengthy process of seeking required approvals
and the continuing need for compliance with applicable statutes and regulations require the expenditure of substantial resources and approvals,
and continued compliance is inherently uncertain.
Before testing any compounds with potential therapeutic
value in humans, the pharmaceutical product candidate enters the preclinical testing stage. Preclinical tests include laboratory evaluations
of product chemistry, toxicity and formulation, as well as animal studies to assess the potential safety and activity of the pharmaceutical
product candidate. These early proof-of-principle studies are done using sound scientific procedures and thorough documentation. The conduct
of the single and repeat dose toxicology and toxicokinetic studies in animals must comply with federal regulations and requirements including
good laboratory practices. The sponsor must submit the results of the preclinical tests, 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. The IND automatically
becomes effective 30 days after receipt by the FDA, unless the FDA has concerns and notifies the sponsor. In such a case, the IND sponsor
and the FDA must resolve any outstanding concerns before the clinical study can begin. If resolution cannot be reached within the 30-day
review period, either the FDA places the IND on clinical hold or the sponsor withdraws the application. The FDA may also impose clinical
holds on a pharmaceutical product candidate at any time before or during clinical studies for various reasons. Accordingly, we cannot
be sure that submission of an IND will result in the FDA allowing clinical studies to begin, or that, once begun, issues will not arise
that suspend or terminate such clinical study.
Clinical studies involve the administration of
the pharmaceutical product candidate to healthy volunteers or patients under the supervision of qualified investigators, generally physicians
not employed by or under the clinical study sponsor’s control. Clinical studies are conducted under protocols detailing, among other
things, the objectives of the clinical study, dosing procedures, subject selection and exclusion criteria, how the results will be analyzed
and presented and the parameters to be used to monitor subject safety. Each protocol must be submitted to the FDA as part of the IND.
Clinical studies must be conducted in accordance with GCP. Further, each clinical study must be reviewed and approved by an independent
institutional review board (“IRB”) at, or servicing, each institution at which the clinical study will be conducted. An IRB
is charged with protecting the welfare and rights of study participants and considers such items as whether the risks to individuals participating
in the clinical studies 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 study subject or his or her legal representative and must monitor the clinical study until
completed.
Human clinical studies are typically conducted
in three sequential phases that may overlap or be combined. While such designations are not officially defined by the regulatory agencies
(including the FDA), the generally accepted meanings are:
Post-approval studies, or Phase 4 clinical studies,
may be conducted after initial marketing approval. These studies are often used to gain additional experience from the treatment of patients
in the intended therapeutic indication. The FDA also may require Phase 4 studies, Risk Evaluation and Mitigation Strategies (“REMS”)
and post-marketing surveillance, among other things, to monitor the effects of an approved product or place conditions on an approval
that could restrict the distribution or use of the product.
Progress reports detailing the results of the clinical
studies must be submitted at least annually to the FDA and written IND safety reports must be submitted to the FDA and the investigators
for serious and unexpected adverse events or any finding from tests in laboratory animals that suggests a significant risk for human subjects.
Phase 1, Phase 2 and Phase 3 clinical studies may not be completed successfully within any specified period, if at all. The FDA or the
sponsor or its data safety monitoring board may suspend a clinical study at any time on various grounds, including a finding that the
research subjects or patients are being exposed to an unacceptable health risk. Similarly, an IRB can suspend or terminate approval of
a clinical study at its institution if the clinical study is not being conducted in accordance with the IRB’s requirements or if
the pharmaceutical product has been associated with unexpected serious harm to patients.
Concurrent with clinical studies, companies may
complete additional animal studies and must also develop additional information about the chemistry and physical characteristics of the
pharmaceutical 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 pharmaceutical product candidate and, among
other things, must develop methods for testing the identity, strength, quality and purity of the final pharmaceutical product. Additionally,
appropriate packaging must be selected and tested and stability studies must be conducted to demonstrate that the pharmaceutical product
candidate does not undergo unacceptable deterioration over its shelf life.
The results of product development, preclinical
studies and clinical studies, along with descriptions of the manufacturing process, analytical tests conducted on the chemistry of the
pharmaceutical product, proposed labeling and other relevant information are submitted to the FDA as part of an NDA requesting approval
to market the product. The submission of an NDA is subject to the payment of substantial user fees. A waiver of such fees may be obtained
under certain limited circumstances.
The FDA reviews all NDAs submitted before it accepts
them for filing and may request additional information rather than accepting an NDA for filing. Once the submission is accepted for filing,
the FDA begins an in-depth review of the NDA. Under the goals and policies agreed to by the FDA under the Prescription Drug User Fee Act
(“PDUFA”), the FDA has 10 months after the 60-day filing date in which to complete its initial review of a standard review
NDA and respond to the applicant, and six months after the 60-day filing date for a priority review NDA. The FDA does not always meet
its PDUFA goal dates for standard and priority NDAs.
After the NDA submission is accepted for filing,
the FDA reviews the NDA application to determine, among other things, whether the proposed product is safe and effective for its intended
use, and whether the product is being manufactured in accordance with cGMP to assure and preserve the product’s identity, strength,
quality and purity. The FDA may refer applications for novel pharmaceutical products or pharmaceutical products which present difficult
questions of safety or efficacy to an advisory committee, typically a panel that includes clinicians and other experts, for review, evaluation
and 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. During the pharmaceutical product approval
process, the FDA also will determine whether a REMS is necessary to assure the safe use of the pharmaceutical product. If the FDA concludes
that a REMS is needed, the sponsor of the NDA must submit a proposed REMS; the FDA will not approve the NDA without a REMS, if required.
Before approving an NDA, the FDA will inspect the
facilities at which the product is manufactured. The FDA will not approve the product 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 will typically inspect one or more clinical sites as well as the site where the pharmaceutical
product is manufactured to assure compliance with GCP and cGMP. If the FDA determines the application, manufacturing process or manufacturing
facilities are not acceptable, it will outline the deficiencies in the submission and often will request additional testing or information.
In addition, the FDA will require the review and approval of product labeling.
The NDA review and approval process is lengthy
and difficult, and the FDA may refuse to approve an NDA if the applicable regulatory criteria are not satisfied or may require additional
clinical data or other data and information. Even if such data and information is submitted, the FDA may ultimately decide that the NDA
does not satisfy the criteria for approval. Data obtained from clinical studies are not always conclusive and the FDA may interpret data
differently than we interpret the same data. The FDA will issue a complete response letter if the agency decides not to approve the NDA.
The complete response letter usually describes all of the specific deficiencies in the NDA identified by the FDA. The deficiencies identified
may be minor, for example, requiring labeling changes, or major, for example, requiring additional clinical studies. Additionally, the
complete response letter may include recommended actions that the applicant might take to place the application in a condition for approval.
If a complete response letter is issued, the applicant may either resubmit the NDA, addressing all of the deficiencies identified in the
letter, or withdraw the application.
If a product receives regulatory approval, the
approval may be significantly limited to specific diseases and dosages or the indications for use may otherwise be limited, which could
restrict the commercial value of the product. Further, the FDA may require that certain contraindications, warnings, or precautions be
included in the product labeling. In addition, the FDA may require Phase 4 testing which involves clinical studies designed to further
assess pharmaceutical product safety and effectiveness and may require testing and surveillance programs to monitor the safety of approved
products that have been commercialized.
Expedited Development and Review Programs
The FDA’s Fast Track program is intended to expedite
or facilitate the process for reviewing new pharmaceutical products that meet certain criteria. Specifically, new pharmaceutical products
are eligible for Fast Track designation if they are intended to treat a serious condition and demonstrate the potential to address unmet
medical needs for the condition. Fast Track designation applies to the combination of the product and the specific indication for which
it is being studied. Unique to a Fast Track product, the FDA may consider for review sections of the NDA on a rolling basis before the
complete application is submitted, if the sponsor provides a schedule for the submission of the sections of the NDA, if the FDA determines
that the schedule is acceptable and if the sponsor pays any required user fees upon submission of the first section of the NDA. On
July 24, 2021, the Company received Fast Track Designation from the FDA for Berubicin. The Company believes that TPI 287 has the potential
to qualify for Fast Track Designation as well. However, at this time, we do not intend to develop Berubicin or TPI 287 further and are
instead focused on evaluating opportunities to out-license these programs.
Any product submitted to the FDA for market, including
a Fast Track program, may also be eligible for other FDA programs intended to expedite development and review, such as priority review
and accelerated approval. Any product is eligible for priority review if it is intended to treat a serious condition and it offers a significant
improvement in the treatment, diagnosis or prevention of a disease compared to marketed products. The FDA will attempt to direct additional
resources to the evaluation of an application for a new pharmaceutical product designated for priority review in an effort to facilitate
the review. Additionally, accelerated approval may be available for a product intended to treat a serious condition that provides meaningful
therapeutic benefit over existing treatments, which means the product may be approved on the basis of adequate and well-controlled clinical
studies establishing that the product has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit, or
on the basis of an effect on an intermediate clinical endpoint. As a condition of accelerated approval, the FDA may require the sponsor
to perform adequate and well-controlled post-marketing clinical studies. In addition, the FDA currently requires pre-approval of promotional
materials for products receiving accelerated approval, which could impact the timing of the commercial launch of the product. Fast Track
designation, priority review and accelerated approval do not change the standards for approval but may expedite the development or approval
process.
Post-Approval Requirements
Any pharmaceutical products for which the Company
receives FDA approvals are subject to continuing regulation by the FDA, including, among other things, cGMP compliance, record-keeping
requirements, reporting of adverse experiences with the product, providing the FDA with updated safety and efficacy information, product
sampling and distribution requirements, complying with certain electronic records and signature requirements and complying with FDA promotion
and advertising requirements, which include, among others, standards for direct-to-consumer advertising, prohibitions on promoting pharmaceutical
products for uses or in patient populations that are not described in the pharmaceutical product’s approved labeling (known as “off-label
use”), industry-sponsored scientific and educational activities and promotional activities involving the internet. Failure to comply
with FDA requirements can have negative consequences, including adverse publicity, enforcement letters from the FDA, actions by the U.S.
Department of Justice and/or U.S. Department of Health and Human Services’ Office of Inspector General, mandated corrective advertising
or communications with doctors, and civil or criminal penalties. Although physicians may prescribe legally available pharmaceutical products
for off-label uses, manufacturers may not directly or indirectly market or promote such off-label uses.
We expect to rely on third parties for the production
of clinical and commercial quantities of our products. Manufacturers of our products are required to comply with applicable FDA manufacturing
requirements contained in the FDA’s cGMP regulations. cGMP regulations require, among other things, quality control and quality
assurance, as well as the corresponding maintenance of records and documentation. Pharmaceutical product manufacturers and other entities
involved in the manufacture and distribution of approved pharmaceutical products are required to register their establishments with the
FDA and certain state agencies and are subject to periodic unannounced inspections by the FDA and certain state agencies for compliance
with cGMP and other laws. Accordingly, manufacturers must continue to expend time, money and effort in the area of production and quality