Investment and Company Research
Opportunity Research
COMPANY UPDATE

RECENT TRADING HISTORY FOR RGBP

(Source: www.StockCharts.com)

Investment and Company Research
Opportunity Research
COMPANY REPORT

July 17, 2025

 

REGEN BIOPHARMA, INC.

(OTC – RGBP)

Industry: BioPharma

Price Target: $0.30

 

 

COMPANY OVERVIEW

The View from 30,000 Feet

Our initiation of coverage report on Regen BioPharma, Inc. (OTCID: RGBP, RGPP) was originally published in October 2023. While the Company’s positioning offered great potential in 2023, in our view, Regen’s current standing is overwhelmingly compelling. In fact, we do not believe we have come across a greater risk/reward profile than Regen in more than 15 years. Regen’s market cap does not even come close to reflecting the proper value for these shares, at current prices. Therefore, we believe substantial upside exists in Regen’s shares. As Regen evolves from an underfollowed preclinical stage firm to a clinical phase firm with multiple, additional IND submissions with the FDA, we expect to see a further revaluation beyond our current $0.30 price target.

The Pipeline

Leveraging an impressive array of IP, the Company plans to rapidly advance novel technologies through pre-clinical and Phase I/ II clinical trials.

Regen has an FDA-cleared IND (Investigational New Drug) application for HemaXellerate™, the Company’s innovative stem cell-derived therapy. The Company plans to launch a Phase I clinical trial in the coming months. The primary indication is to treat chemotherapy patients who have developed a potentially terminal side effect, severe aplastic anemia. There is no direct, approved therapy for this condition while undergoing chemotherapy and Regen could be awarded an Orphan Drug Designation (ODD) and possibly a Fast Track Designation for this product, which would be major coups for Regen. The Company is evaluating expanded applications for this groundbreaking therapy in a market poised for significant growth.

Regen boasts a dynamic and deep pipeline. In addition to HemaXellerate™, Regen has two IND submissions at the FDA. These include dCellVax, an indication for breast cancer and tCellVax, an indication for solid tumors. Moreover, the Company has 3 additional preclinical therapies targeting solid and liquid tumors.

At present, Regen has 11 patent awards with 17 pending. Importantly, much of the Company’s pipeline focuses on small molecules that activate and inhibit a novel gene (NR2F6) which controls how the immune system reacts to cancer cells and to inflammatory responses. Thus, Regen’s development pathways target innovative treatments using autologous cell therapies (HemaXellerate™), and gene silencing utilizing RNA and DNA-based immunotherapy and small molecules in the immune-oncology space.

Looking Ahead

Investors should focus their attention on the Phase I clinical trial candidate and related, upcoming milestones. These include the launch of the trial, patient enrollment, followed by the closing of the trial, data review, and the future release of top-line data. With the commencement or conclusion of each milestone, Regen’s shares should enjoy a bump in valuation, which is typical for early-stage biopharma companies. Plus, we believe that the Company could move preclinical products in its pipeline toward the same development pathway as HemaXellerate.™.

We believe that the Phase I trial could commence in early 1Q26, thus, we project our price target could be met at that time.

Valuation

As we alluded above, in our view, these shares are underfollowed and thus, undervalued. It is a rare find that a Company with an FDA-cleared IND and a robust pipeline and deep IP should trade at such a low level. It has been our experience that a biopharma firm with an active Phase I clinical trial candidate can trade at a valuation of $15-20M. If the candidate is awarded Orphan Drug Designation, which we believe could be in the cards here, the average market cap could emerge at a premium to this range. Therefore, when the Street discovers Regen, which may not occur until trial enrollment commences, we foresee a future revaluation that is normalized to industry standards. In the meantime, our near term $0.30 price target reflects a roughly $10M market cap, which is still a meaningful discount to the $15-20M typical range for active Phase I clinical trial candidate firms, and could prove to be conservative, in hindsight.

REGEN’S LEAD CANDIDATE

HemaXellerate™ is a personalized cellular therapeutic product designed to stimulate blood production in patients whose bone marrow is not properly functioning. Bone marrow damage can occur from radiation, chemotherapy, or chronic conditions. HemaXellerate is an innovative stem-cell derived product that offers substantial potential in treating chemotherapy-induced bone marrow suppression, a market management values at $1 billion annually. This is evidenced by the success of products like Neulasta, which address similar unmet needs.

The HemaXellerate process is straightforward. Belly fat is removed by liposuction and the trial will feature only one treatment, with outcomes measured six months following the treatment. As outlined below, neutrophil and hemoglobin levels are measured as response criteria, which mirrors certain other treatments. There will likely be 12-15 patients enrolled in two California-based sites and a CRO (Clinical Research Organization) has been selected to run the trial. The primary endpoint, as with most Phase I trials, is to determine safety and, on a secondary basis, the degree of efficacy. We believe the process could commence around year-end, with enrollment to begin in 1Q26. We anticipate that results could be released in under 18 months following enrollment. If all goes well, a Phase 2B trial with many more patients would be initiated.

However, we believe that based on potential future data and the current circumstances regarding the seriousness of the condition and current treatments, a partner firm could approach Regen and co-develop the drug, going forward. This would be a major bonus for Regen and certainly a boost for shareholders. A Phase IIb trial would likely be initiated and funded by the partner.

As noted above, one of the challenging side effects of chemotherapy is severe aplastic anemia, the primary indication in which the Company is targeting.

Severe Aplastic Anemia Primer

Severe Aplastic Anemia (SAA) is a life-threatening bone marrow failure disorder that can occur, on rare occasions, with patients undergoing chemotherapy treatments. If afflicted with SAA, patients’ bone marrow stops producing enough red cells, white cells, and platelets. This stage of anemia is considered severe based on certain results indicating dangerous platelet and neutrophils counts, along with the associated reduction in bone marrow cellularity.  Symptoms of SAA include fatigue, infections, and bleeding due to pancytopenia.

While causes include an autoimmune attack on bone marrow stem cells, the toxicity of chemotherapy can prompt the condition as well. Still, in more than half of all cases, the direct cause is idiopathic, or unknown. SAA is diagnosed via bone marrow biopsy or a complete blood count that breaks down white blood cells.

With respect to treatment options, bone marrow stem-cell transplants are very expensive and there are challenges finding a donor. With FDA clearance to begin clinical trials, HemaXellerate™ has the potential to redefine the treatment landscape—not just for aplastic anemia but for a wide range of hematological disorders.

Current FDA approved therapies have issues. For example, there are immunosuppressive therapies, but they tend to be secondary indications for SAA. An ATG, or anti-thymocyte globulin therapy, is an immunotherapy designed to control T-cell immune responses and suppress the immune attack on bone marrow.  These are used mainly in transplants and aren’t always well tolerated and can be combined with cyclosporine which may have its own poor side effect profile of their own, especially for chemotherapy patients. Other treatments may seek to alter a platelet count. 

The bottom line is that the available treatments are secondary in nature and there is no FDA approved therapy for SAA in chemotherapy patients. Since it is a rare condition, it is likely that Regen receives Orphan Drug Designation which is considered by the FDA when a condition affects fewer than 200,000 patients. A cursory review of the total sales of these secondary treatments and hospitalizations for all SAA patients (in the thousands) affirms management’s market size projection of nearly $1 billion in annual sales. While the chemo-specific indication represents fewer patients, an ODD could also lead to a Fast Track designation, thereby speeding time to market. In any event, an ODD designation ultimately translates into much higher price tags for approved drugs. Therefore, it is possible that even for the chemo segment, the Company’s lead candidate could be worth hundreds of millions in annual sales.

THE BUSINESS

Regen BioPharma focuses on creating immune checkpoint medicines.  An immune checkpoint is a mechanism by which certain cells of the immune system, typically T cells, are kept from being fully activated. (CAR-T cells are lymphoid cells that are genetically engineered in a laboratory. They have a new receptor so they can bind to cancer cells and kill them. Different types of cancers have different antigens. Each kind of CAR-T cell therapy is made to fight a specific kind of cancer antigen.)

This type of restraint on the immune system is important in the normal functioning of the immune system. However, it is now well-established that many cancers have an ability to trick immune cells into up regulating their checkpoints and thus shut down the ability of these immune cells to kill the tumor. Several drugs which target checkpoints, termed checkpoint inhibitors, are currently used as standard of care in certain cancers. Regen has been focusing its research on a novel immune checkpoint called NR2F6.

RGBP boasts platform technologies that have led to the development of cellular therapies (including CAR-T cells) as well as RNA and DNA technologies for the treatment of various cancers, and personalized stem cells for which the Company received IND clearance. It has submitted two other INDs for cancer therapies whose status are presently dormant but may be re-visited. Clearly, Regen has a valuable, diverse pre-clinical pipeline spanning cell therapies, RNA and DNA therapeutics and small molecule drugs

NR2F6

NR2F6 can be defined as an intracellular immune checkpoint that suppresses adaptive anti-cancer immune responses. It may have properties that when targeted for next-generation immunological regimens, may delay cancer progression and help improve survival. Hence, the patents RGBP owns regarding NR2F6 and cancer therapies. For example, RGBP was granted patents on shRNA that is designed to inhibit NR2F6 expression in CAR-T Cells and make these CAR-T cells have long-term, durable effectiveness. Separately, blocking NR2F6 in CAR-T cells should also make these cells more effective at killing solid tumors.

dCellVax

CellVax is a dendritic cell-based immunotherapy that stimulates the patient’s immune system through a process called “gene silencing”. The Company has an IND filed with the FDA for this therapy, with features of a proposed Phase I trial as follows:

• 10 advanced breast cancer patients
• Efficacy endpoints at 6 and 12 months
• Establishment of safety will allow for rapid expansion of patient numbers.
• Currently addressing FDA questions with Dr. Santosh Kesari, head of UCSD Neuro-Oncology program

tCellVax

tCellVax is a cell-based immunotherapy that stimulates the patient’s immune system through gene silencing of NR2F6 (Solid Tumors). This is the third IND on file with the FDA. Testing has included:

• Ex vivo siRNA silencing of NR2F6 in PBMC
• Silencing using tCellVax induces T cell activation (release of checkpoint inhibition)
• Initial indications: solid tumors
• Currently addressing FDA questions with Dr. Santosh Kesari

DiffronC

DiffronC is a novel form of therapy called differentiation therapy that is expected to have much milder toxicity than chemotherapy. The mechanism of action is to correct the specific genes that prevent the myelodysplastic syndrome stem cell from producing mature blood cells. By silencing genes, including the Company’s discovered cancer stem cell target gene, differentiation of cancer cells can be induced. The initial indication is treatment of myelodysplastic syndrome while other indications include solid tumors and acute leukemia.

FINANCIALS SNAPSHOT

There are key line items we scrutinize when reviewing pre-revenue companies and following a review of the Company’s financial statements, we believe that from a financial perspective, the Company has operated in textbook fashion—which is a major positive. The balance sheet has no long-term debt. Moreover, the largest expense is a modest $1.2M in accrued expenses—which is a small amount considering the nature of the business model. This figure is affirmed by the low quarterly burn rate of around $150,000, as outlined in the income statement as it prepares for major, future revenue closings.

Separately, we should note that Acadia is in the process of selling thousands of acres of land acquired for potential microgrid use that is no longer a viable option. Such financial transactions will be noted in future filings.

Looking ahead, we preliminarily forecast CY25 pre-development revenue of $3.8M, which reflect services performed in three NY counties.  Our current expectation is that the Company could begin to operate profitably on a quarterly operating basis sometime in 2026, depending upon the timing and year-to-date scale of the project(s) implementation.

For 2026, our estimates suggest that revenue could reach $12M, reflecting more counties in the core NY project come online and other project pre-development deployments increase in size and scope. A bonus contribution could occur from a potential waste-to-energy opportunity as well. Thus, our preliminary expectation is that the Company could begin to operate profitably on a 5-10% quarterly operating basis sometime in 2026, depending upon the timing and year-to-date scale of the project(s) implementation.  We would not be surprised to see revenue reach tens of millions in 2027, depending upon funding timing and scale.

While we have elected to currently provide high level revenue and operating profit to reflect variability in funding and implementation timing by its prospective customers and partners, we plan to compile a detailed, projected P&L as visibility normalizes later this year.

THE REGEN LEADERSHIP TEAM

David Koos, PhD, DBA, Chairman, Chief Executive Officer

David Koos has over 30 years of investment banking and venture capital experience. He has a deep knowledge of start-up businesses, public markers and SEC reporting companies. Dr. Koos has extensive relationships with large and small financial institutions, hedge funds and entities that Regen BioPharma expects to leverage for company growth.

Dr. Koos has a Ph.D. in Sociology and a Doctor of Business Administration degree with an emphasis on finance. Additionally, he has authored / co-authored numerous peer reviewed journal articles.

Harry Lander, Ph.D., Chief Scientific Consultant

Dr. Lander has over 30 years of professional scientific, business and financial management experience related to biomedical research. As a trained biochemist and immunologist, Dr. Lander bridges the gap between science and business.  He has had extensive experience in establishing the Sidra Medical and Research Center in Qatar as well as establishing Weill Cornell Medical College – Qatar and has deep experience with growing biotechnology/pharmaceutical companies. Dr. Lander is currently a Managing Partner of Dyo Biotechnologies, LTD where he oversees strategic consulting for biotechnology projects in Southeast Asia. Dr. Lander was President and Chief Scientific Officer of Regen BioPharma, Inc. Formerly he has served as Research Chief / Administration for Sidra Medical and Research Center (Doha, Qatar) and Assistant Provost for Weill Cornell Medical College (Cornell University). He has extensive managerial and financial experience running complex organizations and establishing new ones.  He founded, managed and sold The Gramercy Group, LLC, a NASDAQ market making firm in 2003 (currently Chardan Capital Management) and had multiple SEC licenses.

Scientific Advisory Board Members

Ravinder Reddy, Ph.D.: Dr. Reddy is currently a Professor of Radiology and the Director of the Center for Advanced Metabolic Imaging in Precision Medicine at the University of Pennsylvania. His research interests are in studying molecular and structural changes in various diseases including cancer. He has published important papers on the mechanisms of CAR T-cell toxicities as well as ways to monitor immunotherapy effectiveness. Dr. Reddy is the recipient of several prestigious biomedical research awards including grants from the Whitaker Foundation, DANA Foundation and National Institutes of Health. He is also a member of many prestigious professional organizations and societies and serves on the editorial boards of several journals in the field. With more than 200 peer-reviewed papers and 15 patents, Dr. Reddy has been inducted as a Fellow of the International Society of Magnetic Resonance in Medicine and has been named to the Council of Distinguished Investigators of the Academy of Radiology Research.

Mohammad Haris, Ph.D.: Dr. Haris is an Associate Professor in the Center for Advanced Metabolic Imaging in Precision Medicine, Department of Radiology, Perelman School of Medicine, at the University of Pennsylvania. His research focuses on development of novel quantitative and molecularly specific metabolic imaging technologies to improve outcomes for patients with cancer and neurological/ neurodegenerative disorders. With more than 100 peer reviewed publications and multiple patents, Dr. Haris has extensive expertise in examining and understanding the mechanisms of cancer growth and the tumor microvasculature with publications in high profile journals such as Nature Medicine, Cell, and Nature Biomed Eng. and Molecular Cancer. Dr. Haris received his Ph.D. in Biomedical Imaging and Master’s in Biochemistry from India and did his postdoctoral training at the University of Pennsylvania.

Stefano Bertuzzi, Ph. D, MPH: Dr. Bertuzzi, is currently the Executive Director of the American Society for Cell Biology and has been named Executive Director and CEO of the American Society for Microbiology, effective January 4, 2016. Before leading the American Society for Cell Biology, Dr. Bertuzzi was a senior scientific executive at the National Institutes of Health where he served as Director of the Office of Science Policy, Planning, and Communications, and as a science policy advisor to the NIH Director. Dr. Bertuzzi received his Ph.D. in Molecular Biotechnology from Milan, Italy and his master’s in public health from the Bloomberg School of Public Health at the Johns Hopkins University in Baltimore with a specialization in health policy.

Jonathan Baell, PhD: Dr. Baell is a professor or Medicinal Chemist at Monash University (Australia) whose research is focused on the discovery of new medicines for treating diseases with an unmet medical need. A proven record of undertaking original research with a total of 120 publications and patents. He is published in top-ranked journals in the field (e.g. 12 in Journal of Medicinal Chemistry, Nature Chemical Biology, Nature). He has 40 granted patents listed at www.lens.org.

He has won a national award for discovery of a new class of potential multiple sclerosis therapeutics. Additionally, he has achieved successful outcomes in a diverse array of drug discovery arenas, from rational design of peptidomimetics to heterocyclic drug discovery.

For the period 2012-16, Dr. Baell is Chief Investigator on competitive grants that totaled $4.73M. In addition, he has been awarded a prestigious NHMRC Senior Research Fellowship for the period 2012-2016. In 2011 and elected as the Australian representative to the Board of Directors for the Inaugural International Chemical Biology Society and appointed Chair of Membership.

Rohit Duggal, Ph.D.: An experienced industry executive with specialization in establishing and advancing biotherapeutic products targeting cancer. Dr. Duggal has more than 23 years of industry experience that spans cellular immunotherapy, immuno-oncology, oncolytic virotherapy, translational oncology and antiviral drug discovery. Currently VP of Research at TRL, he was the head of R&D and site head of ONK Therapeutics Inc. Before that, Rohit led the primary lymphocyte product development program at NantKwest.  At Sorrento Therapeutics he helped develop the immuno-oncology franchise that included antibodies in different formats.  Rohit obtained his Ph.D. from Texas A&M University.

Hinrich Gronemeyer, Ph.D.: Research Director at the IGBMC in Strasbourg-Illkirch and Research Director (Class ‘Exceptional’) of the French National Institute of Health and Medical Research (INSERM). His nearly 200 publications received an average citation of 83.34. Hinrich Gronemeyer contributed to pioneering work on nuclear receptors and their therapeutic intervention by design of small molecule ligands.

Additional SAB members can be found on the Company website.

FINANCIALS SNAPSHOT

As evidenced by its quarterly financials, Regen runs a lean ship, with very modest operating losses. Importantly, the Company has no long-term debt, which is somewhat unusual in this space. Perhaps that is why we are confident that management can secure a favorable funding arrangement for general corporate purposes and R&D.

Too many companies have a significant percentage of shares outstanding in the public float. Conversely, regen only has about 59% of its outstanding shares in the current public float. In our view, this is a major positive from a fundamental and trading perspective.

CONCLUSION

Regen is on the cusp of making a major leap from the preclinical biopharma stage to a clinical stage biopharma. The Company’s lead candidate, HemaXellerate, is an innovative stem cell-derived therapy and the Company plans to launch a Phase I clinical trial in the coming months. HemaXellerate’s primary indication is to treat chemotherapy patients who have developed a potentially terminal side effect, severe aplastic anemia. The only approved therapy is a costly stem cell transplant, which needs a matched donor and can lead to graft-versus-host disease. Still, the Regen therapy represents $1 billion in market size.  Regen could be awarded an Orphan Drug Designation (ODD) for this product, which would be a major coup for the Company. Regen is also evaluating expanded applications for this groundbreaking therapy. With two other filed INDs and a deep pipeline Regen is no one-trick pony. The Company has been awarded 11 patents with 17 patents pending. 

We believe that these shares are grossly undervalued based on the IP alone. When taking into account Regen’s migration to the clinical stage, this status becomes even more pronounced, given the typical market cap assigned to its peers. Our price target of $0.30 reflects a meaningful discount to the typical market caps enjoyed by active Phase I clinical trial peers. However, if the drug is awarded Orphan Drug Designation, our target could prove to be conservative.

Investment and Company Research
Opportunity Research
COMPANY REPORT

July 08, 2025

 

ACADIA ENERGY CORPORATION

(OTC – AECX)

Industry: Clean Energy

6-12 Mo. Price Target: $0.15

 

 

COMPANY OVERVIEW

The View from 30,000 Feet

In our view, New York-based Acadia Energy Corporation (OTC: AECX) is uniquely positioned to emerge as a leading and enviable player in large-scale, and innovative diverse energy integration projects in the U.S. Importantly, the Company’s approach should begin to bear fruit later this year, taking Acadia Energy from a pre-revenue firm to a revenue-generating company, thereby providing a major boost in its valuation and share price. We preliminarily forecast that Acadia Energy could record $3.8M in pre-development and operating revenue in 2025 and $12M in revenue in 2026, with a 5-10% operating margin.

Specifically, this energy systems integrator is primed to lead and operate large-scale microgrid networks for communities in upstate New York, a multi-billion-dollar opportunity in the coming years. Acadia Energy benefits from state mandates that call for a significant reduction in greenhouse gases and specific electricity mandates regarding  energy generation sources.

These mandates state that New York must achieve a 40% reduction in greenhouse gas emissions by 2030 and an 85% reduction by 2050, using 1990 as the baseline year. The remaining 15% of emissions may be offset through approved methods such as carbon capture, reforestation, or other negative emissions technologies—leading to net-zero emissions economy-wide by 2050. In the electricity sector, the law requires that 70% of electricity come from clean sources by 2030, and that the grid be entirely zero-emissions by 2040. The law also requires that at least 35% (ideally 40%) of the benefits of state climate investments be directed toward disadvantaged communities. These communities are often disproportionately affected by pollution and economic inequality. While these targets may be adjusted due to delays, funding questions, etc., the opportunity remains huge.

Acadia Energy’s Core Advantages

For nearly a decade, the Company and its leadership have established a true public-private partnership with key counties and towns in upstate New York. As the counties move forward and come online, Acadia Energy is well positioned to lead these local energy projects. Thus, the Company will establish and operate a microgrid network that will feature renewable and non-renewable energy and storage, thereby ensure reliability and reduce energy costs. To date, feasibility studies have been completed or are underway in key counties and in addition to co-development recognition by the New York Power Authority, many counties and towns are in support of Acadia Energy.

The leadership team has decades of experience managing large scale public and private projects and has partnered with leading engineering firms, core vendors, and others to build an enviable team in the clean energy systems integration industry.

Financials

As evidenced by its quarterly filings, Acadia Energy has been preparing for project commencement in upstate New York for some time. The Company has run a lean ship with a low burn rate and no long-term debt on the books. In addition to its opportunities in New York, management has explored leveraging its experience and expertise in the clean energy arena to include waste-to-energy and decarbonization projects, which could diversify future revenue streams. Moreover, as projects are launched, we believe that the Company’s replicable model could enable it to foster similar business opportunities in neighboring states. Meanwhile, given the nature of the business model, we currently forecast outsized top-line growth in 2026 and beyond, with quarterly operating profit to occur sometime in 2026. If pre-development revenues exceed our $3.8M and $12M projections for 2025 and 2026, quarterly operating margins could be north of 10% later in the year..

Valuation

We believe that Acadia Energy is grossly undervalued since there has been little awareness about the Company until now, when it is on the cusp of making the leap from pre-revenue to revenue generation. It is common that publicly traded companies that evolve from the pre-revenue stage to the revenue stage are afforded a major boost in valuation ahead of the evolution and can also enjoy one after the first quarterly report, if the financials are favorable. In our view, by the end of 2025, Acadia Energy will be valued based on a revenue multiple on 2026 projected sales. Given the potential variance in forecasts, and by applying a below market price/revenue multiple to be conservative, we believe that these shares could reach the $0.15 mark by year-end, reflecting a roughly  5.0x price/revenue multiple. This multiple is a reasonable target considering our projected top-line growth from 2025 to 2026. Moreover, just a few months ago, these shares traded at $0.18 when the revenue stage runway was further out. Therefore, we believe upside could exist in our target, as we get closer to year-end, with a potential trading range of $0.20 – $0.30 next year as well. Future trading ranges will be directly related to pre-development revenue from additional counties, along with specific development milestones in three counties we believe will serve as revenue generators in late 2025.

INDUSTRY OVERVIEW

Acadia Energy is well positioned to emerge as an energy integration leader by leveraging expertise in microgrid deployment and implementation. Against this backdrop, it is instructive to provide an overview of the segment and related events/projects in the U.S.

Market Size and Market Drivers

According to a report by the IMARC Group, the U.S. microgrid market is expected to grow from an estimated $7.9 billion in 2024 to $24.4 billion in 2033, a 13.3% CAGR. Various industry sources cite more than 500 operational microgrid projects while the EIA states 12 GW of battery storage was deployed by the end of 2024.

Deployment growth is being led by municipalities, remote communities, university campuses, industrial parks, hospitals, military bases, and by targeted state government mandates. By allowing towns and companies to produce and store their own electricity, microgrids lessen reliance on the main grid and cut customers’ long-term energy expenses by 20-30%. Moreover, by managing their own power supplies and demand, they can provide backup power in emergencies and not lose money for extended downtime. The operator merely disconnects from the main grid during power outages, if it integrates clean energy and storage.

Additionally, by optimizing energy use through localized management and storage, they can effectively manage peak demand and save energy expenses.

Another economic feature of microgrids is the reduction in operational costs. In an interesting quirk, a microgrid may also be able to sell excess energy back to the utility, further producing local economic benefits. Separately, commercial customers may no longer be subject to peak demand expenses, thus lowering their bills.

For those entities that utilize clean energy sources, reduced carbon emissions are generated and while it fosters goodwill locally, it can result in carbon credits as well.

As is the case with high growth, high opportunity industries, failings by other parties are driving remote communities, campuses, and others to seek out alternative or supplementary solutions to fix or avoid major critical issues. For example, extreme weather conditions and events in recent years have taxed state and local utilities. Reliable access to electricity is no longer a given as the utilities have proven to be routinely unreliable in the face of climate or weather disasters. Thus, the desire to have reliable sources of power and energy make the use of microgrids a no-brainer for local, rural communities, campuses, and managers of critical facilities. This is especially the case when clean energy sources are integrated into these large-scale projects.

On a secondary basis, innovation in management and control systems have fostered deployment as they have improved microgrid efficiency and reliability while enabling entities to meet stated sustainable energy objectives and initiatives.

Lifting Up the Hood…

A microgrid is a small-scale, local energy system designed to operate independently or in conjunction with a local utility’s main power grid. It can include a mix of low emissions energy sources such fuel cells, geothermal, nuclear, solar panels, wind turbines, or linear generators. Users of a microgrid or group of related microgrids are typically connected through a local network. Microgrids are usually connected to the main grid or used as a backup to reduce costs and main grid stress. They are connected to clean energy storage devices to ensure reliable and sustainable electricity.

At the system level, a microgrid is managed by a microgrid controller, which is a software/hardware system. The controller monitors local energy supply and demand, balances the load, and optimizes when to use the stored energy, versus buying from the main grid.

The main players in the microgrid production space include Schneider Electric, Siemens, GE, Honeywell, Eaton and others. In addition to private operators, in places like California and New Jersey, utilities are being required to develop public-serving microgrids for critical care facilities such as hospitals or remote areas/communities. They are developed and operated by the local utilities for reasons similar to the factors we cited above. Moreover, these microgrids are deployed to meet state regulatory mandates.

The industry isn’t crazy about utilities owning distributed energy systems. Still, there are few dozens of these projects around the country as the industry remains dominated by the non-utility private sector. The overwhelming majority of these are in California, with other notable ones on the East Coast. California-based projects tend to be deployed in fire-vulnerable communities, the ones on the East Coast are often domiciled in areas hit by hurricanes and heavy storms. The State of Hawaii has a mandate to eventually engage in a 100% clean energy systems policy across the islands.

The top 5 states in the country for current microgrid project deployments are: California, New York, Texas, Massachusetts, and Alaska. In California, 85% of the electricity used by the University of California San Diego (UCSD) campus is provided via microgrids. New York has an atypical project, the Brooklyn Microgrid, whereby residents can buy/sell energy via a blockchain. Alaska has several stand-alone microgrid projects providing electricity in rural villages.

A townwide residential microgrid network can provide electricity for hundreds to thousands of customers although 100-500 daily users in a 1-5MW microgrid size is more typical, per microgrid. Interestingly, the UCSD 45MW network has 45,000 daily users with a 45MW load.

New York and Acadia Energy

The Climate Leadership and Community Protection Act (CLCPA) is New York State’s landmark climate law. Widely regarded as one of the most ambitious climate policies in the United States at the time, the CLCPA aims to dramatically reduce greenhouse gas emissions while fostering clean energy development and prioritizing environmental justice.

At its core, the CLCPA mandates that New York achieve a 40% reduction in greenhouse gas emissions by 2030 and an 85% reduction by 2050, using 1990 as the baseline year. The remaining 15% of emissions may be offset through approved methods such as carbon capture, reforestation, or other negative emissions technologies—leading to net-zero emissions economy-wide by 2050.

In the electricity sector, the law requires that 70% of electricity come from clean sources by 2030, and that the grid be entirely zero-emissions by 2040.. Another major component of the CLCPA is its emphasis on climate justice. The law requires that at least 35% (ideally 40%) of the benefits of state climate investments be directed toward disadvantaged communities, which are often disproportionately affected by pollution and economic inequality. A Just Transition Working Group was also established to guide workforce development and ensure that communities and workers impacted by the transition away from fossil fuels are supported.

We should note that comments by Governor Hochul in 2024 indicate that these objectives may be aggressive and will be adjusted. Still, the opportunity is huge.

How does Acadia Energy fit into this playing field?

There are billions of dollars in play at the federal and state level. However, as is often the case with government funding, they are always subject to revision, delays, etc. At present, the Company is exclusively focused on specific communities in Upstate New York and is well-positioned to emerge as the winner for these opportunities over the next 2-3 years. Management has spent years cultivating community relationships and partnerships with the appropriate entities and figures in the communities. Plus, the Company already meets the disadvantaged community guidelines and has been directly targeting this market. Its unique model creates jobs, spurs economic growth, and meets the objectives of the CLCPA and the underlying communities.

Finally, the Company has developed its own microgrid financial and technology platform and microgrids and as an energy systems integrator, is prepared to be agnostic with vendors, as needed.

THE BUSINESS

Acadia Energy is an innovative energy integration company focusing on design, development, funding and operation of transformative projects, spanning intelligent microgrids, and clean energy resources. Microgrids are a key component to the sustainability and modernization of our nation’s energy infrastructure. They provide resiliency, redundancy, reliability, surety and security to today’s antiquated and failing utility grid system that powers our daily lives. Acadia Energy approaches its business with a focus on sustainability and its inter-relationship with public policy.

The Company’s objective is to provide municipal governments and local communities with a reliable, lower cost of power and a continuing revenue stream. This approach can help create a pathway to attracting new commerce, job creation, sustainability and a better quality of life. Since 2016, Acadia Energy has demonstrated a deep commitment to work with local, state and federal governments to develop the critical infrastructure microgrid marketplace. Acadia Energy’s core management team has decades of experience providing mission critical solutions to government, police, fire and federal authorities and the management team leverages that expertise to collaborate with municipal partners to create next generation energy solutions.

The executive and project management team at Acadia Energy, along with key partners, have extensive development experience in designing, developing, constructing, and operating utility-scale solar projects, as well as advanced microgrids, photovoltaic solar, waste to energy, and other distributed generation resources.

Acadia Energy has been recognized by the New York Power Authority (NYPA) as a microgrid co-developer. Effective February 1, 2025, NYPA has the capability of co-developing and owning generation assets.  Acadia has been selected as a co-development partner with NYPA on its pipeline of microgrid and Sustainability Hubs throughout Central New York (CNY), currently valued at over $2.0 billion.

Acadia Energy is currently in discussions with regulatory agencies such as the NY Public Service Commission (PCS), Department of Public Service (DPS), NYSERDA, and others regarding the implementation of microgrids. Although the State’s strategic CLCPA has received with an enthusiastic response within the state and around the world, many regulatory hurdles are still in place. Acadia is the first to address these hurdles and the PSC staff is addressing these issues with Acadia and its partners.

As part of its unique approach, the Acadia Energy team have worked to integrate innovative technologies into Sustainability Hubs. These sustainable environments have the potential to reduce energy costs, and support a “pure” food supply, mitigating the challenges associated with climate change, while stimulating local economies, and bringing jobs to rural communities, with a shared revenue stream.

Sustainability Hubs, when located near existing or planned industrial centers, offer a valuable asset: power that can be transmitted via private lines, avoiding costly transmission and delivery (T&D) fees that represent more than half the total energy bill. Acadia Energy can accomplish this by running its own lines from the power generation site to the end-user and amortizing the cost of delivering that power over the life of the installation, typically 25 years.

The Opportunity

Through its non-profit partner, TakeChargeNY, Acadia Energy has created public-private energy models with county leadership that include properties that are suitable for green energy generation and acceptable to the community. Moreover, in partnering with county leadership, Acadia Energy ensures that the pricing of the energy generated by its Sustainability Hubs would have low income and distressed community customers in mind and would be priced based on a county/Acadia decision.

As more commercial, public sector, institutions and residents transition away from fossil fuel to power their homes and businesses, the demands on our aging electric grid become more pressing. The inevitable move to electric vehicles places even greater strains on an already-overloaded grid.

It is estimated that upgrades to the grid will cost the State of New York more than $60 billion; the first $6 billion has already been approved. Upgrades mean more than just hanging wires. Improved load management on the grid is needed. New transmission and distribution systems must be established statewide. Natural integration of multiple new sources, like Acadia’s microgrids, is part of the solution.

Unfortunately, the current situation for those in upstate New York is inequitable. Improving the carrying capacity of the grid in upstate communities does not generate goodwill when the electrons are going to power homes and businesses downstate. This is a clear case of upstate subsidizing downstate energy needs which could be curtailed under Acadia leadership with the various counties.

The Acadia Energy Advantage

Acadia Energy, in our view, is well-positioned to lead the charge on behalf of and in conjunction with Upstate New York counties. The Company and its leadership have spent nearly a decade fostering relationships in a true, public-private partnership. The unique business model has involved leadership to  become embedded in the communities and demonstrate their sincere desire to improve not just power generation reliability and costs but the quality of life of the residents. No other entity has spent more time and capital in this venture. There have been “carpetbaggers” from outside of the State of New York that have stepped in to play a small role in random solar and other deployments. However, we do not believe that they are deficient when matched up against Acadia Energy.

Plus, we do not believe that other firms can demonstrate the decades of experience in large scale, complex, systems integration projects. These features are critical to success.

Our review of competing firms that also apply an energy integration approach affirm our thesis that Acadia Energy is in the catbird seat in its home state of New York. Peers such as BoxPower, Scale Microgrid, and GridAlternatives, while notable players in their own right, are unlikely to win business in Upstate New York away from Acadia Energy. These companies are not direct comps as they either are not full integrators, target tribal lands, or utilize a technology or engineering approach that may not satisfy the communities in this region. The only firm that may have an outside shot is Ameresco which has modest projects underway or operational.

The Process

Management has targeted several counties and towns that would commence later this year. Feasibility analyses have been launched or completed and each of these counties planning departments have provided Letters of Intent and Support.

Acadia Energy will work with TakeChargeNY in a collaborative manner to identify the desired service area, how the power should be allocated between residents and business, and results in a power purchase agreement amongst the stakeholders.

Once the microgrid locations have been identified Acadia Energy provides a development plan, which includes local labor agreements. One benefit to the community is the assurance that, to the extent possible, the local workforce will be utilized to complete the installation.

Acadia Energy then contracts with an experienced engineering company to perform the installation of generation sources, and interconnection with the utility in the region. Batteries, energy storage (CORESS Acadia’s proprietary storage) and fuel cells for baseload will be part of each project. This is especially important to the behind-the-meter users, such as manufacturing facilities, which rely upon steady supply of power whether the sun is out or not.

Acadia Energy will engage a leading engineering firm with experience installing clean energy infrastructure. Working with the local community, Acadia and their engineering consultant will seek to utilize the local workforce, prioritizing subcontractors from MWBE/Veterans communities.

Once the microgrid is operational, energy will be allocated according to the preferences expressed by the host county. This is defined by a percentage behind-the-meter, using power lines installed by Acadia Energy, to local institutional and industrial off-takers, as well as a percentage directed to residents (at a discounted rate to be negotiated with the prevailing utility).

Revenue generated by the facility is also apportioned according to the contract with each county. The municipal entity share can be used as they see fit. This could include a contribution to the general fund, reduction of county property taxes, or allocated towards new benefits, such as electric vehicle chargers for a school or municipal bus fleet.

Milestones

As evidenced by the graphic below, Acadia three-year plan is highly targeted, and in our view, achievable within this timeframe. As the project reaches its various stages, we believe that the Company will take this replicable model to neighboring states and communities. In addition, management has already explored leveraging its experience in systems integration and clean energy to engage in waste-to-energy conversion projects abroad. Once again, if one of these projects commences, Acadia Energy could offer similar terms in other nations and locales, thereby diversifying its business.

FINANCIALS SNAPSHOT

There are key line items we scrutinize when reviewing pre-revenue companies and following a review of the Company’s financial statements, we believe that from a financial perspective, the Company has operated in textbook fashion—which is a major positive. The balance sheet has no long-term debt. Moreover, the largest expense is a modest $1.2M in accrued expenses—which is a small amount considering the nature of the business model. This figure is affirmed by the low quarterly burn rate of around $150,000, as outlined in the income statement as it prepares for major, future revenue closings.

Separately, we should note that Acadia is in the process of selling thousands of acres of land acquired for potential microgrid use that is no longer a viable option. Such financial transactions will be noted in future filings.

Looking ahead, we preliminarily forecast CY25 pre-development revenue of $3.8M, which reflect services performed in three NY counties.  Our current expectation is that the Company could begin to operate profitably on a quarterly operating basis sometime in 2026, depending upon the timing and year-to-date scale of the project(s) implementation.

For 2026, our estimates suggest that revenue could reach $12M, reflecting more counties in the core NY project come online and other project pre-development deployments increase in size and scope. A bonus contribution could occur from a potential waste-to-energy opportunity as well. Thus, our preliminary expectation is that the Company could begin to operate profitably on a 5-10% quarterly operating basis sometime in 2026, depending upon the timing and year-to-date scale of the project(s) implementation.  We would not be surprised to see revenue reach tens of millions in 2027, depending upon funding timing and scale.

While we have elected to currently provide high level revenue and operating profit to reflect variability in funding and implementation timing by its prospective customers and partners, we plan to compile a detailed, projected P&L as visibility normalizes later this year.

THE ACADIA ENERGY LEADERSHIP TEAM

The Acadia Energy management team may be one of the most dedicated groups we have encountered in recent years. Moreover, the group has a wealth of expertise and experience in key areas, and in our view, rivals that of many NASDAQ and NYSE listed companies.

John Bay, Founder, President

John founded Acadia in 2016 to originally find energy-based solutions to the economic crises he saw in his adopted home of upstate and western New York. has spent over 35 years in developing technology solutions as a system integrator. He has specialized the energy, wireless, telecommunications, application software and internet technology development creating innovative, leading-edge solutions for these markets. Previously he founded Paradigm4, a corporate enterprise with more than 240 employees, leading to the successful completion some of the country’s largest mission-critical system integration projects. He has held upper management positions throughout his career, including P&L divisional responsibilities for wireless and telecommunications at MCI WorldCom.  He served on the Technical Advisory board of AT&T wireless and was instrumental in the Microsoft/Qualcomm endeavor know as Wireless Knowledge. Throughout his career, John has owned, operated, and managed corporate enterprises specializing in large, complex system integration projects and has successfully completed over $600 million in projects.

Steve Infanti, Chief Financial Officer

Mr. Infanti has had a long-distinguished career in business, development and consulting. He began his career with Coopers & Lybrand CPA’s, specializing in Construction and Manufacturing. He left public accounting to pursue interests in Construction and Development, first as a Chief Financial Officer and then as a Chief Executive Officer and co-owner of an Engineering News Record, National, Top 400 Construction Company, in the Building and Heavy Highway sectors. Mr. Infanti also has had interests in real estate development firms and partnerships, as well as other business ventures and investments. For the last 15 years, he has provided consulting services for business owners, hospitals and financial institutions as President and CEO of his business consulting company.

​Mr. Infanti has also served in a leadership capacity on numerous Boards encompassing community, not for profit, healthcare and business organizations. He received a Bachelor of Science degree in accounting from LeMoyne College. He has been a certified public accountant since 1974.

Charles Schwerin, Director of Community Relations

Chuck Schwerin is a serial entrepreneur, having co-founded several medical device and biotech startups, and mentored other entrepreneurs in his role as Managing Director of Business Services at Ithaca Area Economic Development.

He holds three patents on geographic information technology and was an adjunct professor, teaching entrepreneurship at Binghamton University. Chuck is also on the Board of the Edward L. Rose Land Conservancy for whom he publishes a quarterly newsletter.

Christopher Cheney, Director of Property Management

Christopher is a proven property management professional with deep experience in real estate, economic development, and insurance. Have served on boards of directors and committees in both private and public sectors. Currently serving on the Cayuga County Public Utilities Service Agency advisory committee.

James Hibbert, Director of Investor Relations

Jim has over 30 years of experience in the Investor and Public Relations marketplace. Jim has acted directly with law firms, auditors, and investment banks on developing and filing SEC regulatory documentation utilized in the formation and uplift of companies to the OTC, NASDAQ, and other public trading platforms. He has also assisted in securing corporate financing usually required with corporate uplifts.

As Acadia’s face to the public markets, Jim’s daily activities are primarily focused on interfacing with shareholders, raising corporate awareness, and disseminating corporate news.

Dennis Elsenbeck, Advisory Consultant

Dennis provides consulting services on a broad range of energy-related opportunities encompassing a forward view of supply, distribution, and demand options. In his leadership role with a major U.S. utility for nearly 30 years, he brings insight, analytics and business perspectives on long-term policies and the economic landscape. He assists Acadia regulatory compliance on energy transactions; regulatory counseling involving Public Service Commission proceedings; energy procurement and utility negotiations involving government entities and municipalities.

RISK FACTORS

As is the case with its peers, the Company’s near-term risk is related to federal and state government funding timing and terms for the commencement of clean energy projects in upstate New York. Present uncertainty could lead to delays which would impact revenue booking timing scale and project implementation. Still, we believe that the Company is insulated from much of the current uncertainty, due in part to their technology agnostic approach.  Plus, to offset challenging  potentialities, management has wisely run concurrent tracks of prospective clean waste-to-energy business to include Caribbean opportunities. As these referenceable projects launch, we believe it would offset any domestic funding delays that may occur. Moreover, we believe that the international projects could lead to complementary opportunities domestically, thereby negating any funding government funding issues.

As the Company evolves from a pre-revenue stage to a meaningful revenue generation stage, Acadia Energy may need to educate or re-educate the domestic markets about the company approach benefits on a local and state level. An unforeseen customer risk could emerge such as the desire of municipalities to select a different path to executing clean energy projects, relative to current methods. Competitive risks may also include lower pricing, more effective sales/marketing, and overall greater system integration business model efficiency.

The aforementioned risks could come from larger competitors, existing firms, or new entrants. Still, these future concerns are consistent with firms of Acadia Energy’s size and standing. Moreover, we believe that the Company’s seasoned management team is prepared to overcome these hurdles and generate significant interest, leading to broad deployment and significant, future top-line growth.

Volatility and liquidity are typical concerns for microcap stocks that trade on the over the counter (OTC) stock market. Management may seek to raise funds to fund corporate development, product revenue, and overall expansion. An overriding financial benefit as a public company is the favorable access to and the availability of capital to fund product and project launches, consistent marketing campaigns and other initiatives. Since the proceeds of any future funding would be used in large part to advance development, infrastructure, and revenue, we believe that any dilutive effect from such a funding could be offset by related increases in market value.

CONCLUSION

Clean energy integrator Acadia Energy’s share price is poised for a re-valuation as it evolves from a pre-revenue firm to a meaningful revenue generator. We believe that the Company could achieve $3.8M in revenue by year-end 2025.

The Company is uniquely positioned to emerge as a major player in energy systems integration in upstate New York. Current opportunities represent tens of billions of dollars in revenue. Acadia Energy benefits from key New York state mandates. For example, a significant majority of electricity must come from clean sources. Acadia Energy owns key competitive advantages over its peers. The leadership team has spent nearly a decade fostering a true public-private partnership with counties and towns in upstate New York. Plus, management has decades of experience in large scale integration projects.

Separately, the Acadia Energy approach is a replicable model for other projects in the US. In addition, the Company is exploring other clean energy opportunities abroad, potentially diversifying the revenue base.

Our 6–12-month price target of $0.15 represents a 5.0x price/revenue multiple on 2026 forecasted revenue of $12M, a more than 200% lap from our $3.8M forecast for 2025. These shares were above our target price a few months ago, thus we believe upside exists.

RECENT TRADING HISTORY FOR ACADIA ENERGY

(Source: www.Stockta.com)

Investment and Company Research
Opportunity Research
COMPANY REPORT

July 01, 2025

 

SIGYN THERAPEUTICS, INC.

(OTC – SIGY)

Industry: Medical Devices

6 Mo. Price Target: $9.00

 

 

COMPANY OVERVIEW

The View from 30,000 Feet

When we initiated coverage of Sigyn Therapeutics (OTCQB: SIGY) in March 2021, we were still in the throes of Covid-19, fears of infectious diseases, etc. However, even then, management had the vision to target a therapy to treat sepsis. Following strong in vitro data, and with improvements in the industry, Sigyn is continuing on this path, along with developing a treatment for other critically ill patients that face another condition that has a high level of mortality, end stage renal disease (ESRD) patients. In our view, milestones and events should serve as a series of valuation drivers for these shares over the next six months, and with even greater potential in the next 12-18 months.

Sigyn’s flagship platform, Sigyn Therapy™, is a proprietary blood purification technology designed to overcome the limitations of previous drug and device candidates to treat acute inflammatory conditions. The Company’s current focus is for treating for two of the most challenging indications. These include sepsis and the treatment of endotoxemia and concurrent inflammation in dialysis patients, specifically those with ESRD.

Sepsis plays a role in nearly 1 in 3 of the 1.7 million patients diagnosed with sepsis. It is the biggest culprit of deaths in hospital settings and there is no FDA approved treatment for the condition. A peer is set to announce results of its latest study that they hope will indicate that it can deplete endotoxins, which is a driver of the condition. This would be great news for the industry and Sigyn, given the fact that in vitro studies indicate an even broader capability.

On the ESRD side, 550,000 are on dialysis each year and the mortality rate is a daunting 70% after 5 years. Again, endotoxins and excessively produced inflammatory cytokines are hallmarks of the condition. Given its foundation in blood purification technology, ESRD treatment is a no-brainer for Sigyn, in our view.

Looking Ahead

The Company has a highly targeted development pathway for its led platform. These include two Breakthrough Device submissions, feasibility study and other events. Thus, the Company will have an active 12-18 months.

Our price target of $9.00 is roughly 3x the current share price and we believe it could be reached within six months, if not sooner. This thesis is based upon industry events we outline in this report, the submissions, and potential awards. The small 1.6 million share count and fewer than 250,000 shares n the public float also make these shares susceptible to a big move on favorable news. At the end of the day, a $4.4 million market valuation for a company with a deep product portfolio and a series of favorable events ahead is the definition of undervalued. However, we believe that a higher profile will be afforded the Company and therefore the shares and investors will be the beneficiary. Moreover, our price target could prove to be conservative when compared with a proper peer who trades at a significantly higher valuation. As future milestones are met, we believe the shares will emerge at a more normalized valuation. Furthermore, if data from a potential, future human ESRD trial is favorable in the next 2-3 years, we believe that Sigyn would emerge as a takeover candidate.

THE SIGYN PIPELINE

Sigyn Therapeutics, Inc. is developing next-generation medical devices to address life-threatening conditions with no FDA-approved treatments. In our view, Sigyn Therapy™, the Company’s core platform, is on track to achieve breakthrough results for two of the most challenging indications. These include sepsis and the treatment of endotoxemia and concurrent inflammation in dialysis patients, specifically those with end stage renal disease (ESRD).

Sigyn Therapy™: A High-Level View

Sigyn Therapy™ is a formulation of adsorbent components that provide 200,000+ square meters (~50 acres) of surface area on which to bind and eliminate circulating therapeutic targets. Sigyn Therapy™ is highly efficient, capable of processing the entire bloodstream of an average-sized person approximately 15 times during a four-hour treatment.

Key features of this therapy could be considered without a peer. These include its breadth and capacity to eliminate circulating therapeutic targets; the rate at which it processes the bloodstream; and its ability to be deployed on dialysis and CRRT machines already located in hospitals and clinics. However, throughput and ease of deployment are just highlights of the hardware’s general functionality. The use of Sigyn Therapy™ during in vitro studies have validated the clearance of bacterial toxins (including endotoxin), inflammatory cytokines, hepatic toxins, and infectious viral pathogens from human blood plasma.

Sepsis Treatment

Each year, 1.7 million patients in the U.S. are stricken with sepsis, a severe condition usually contracted while in the hospital. Sepsis is the leading cause of death in U.S. hospitals and a $62 billion annual healthcare burden. Sepsis is a life-threatening condition triggered by the body’s overwhelming response to bacterial components such as endotoxin (endotoxemia) and it continues to be a major global health issue, impacting millions. If not recognized and treated promptly, sepsis can result in multiple organ failure, shock, and death.

Yet, it has not been addressed with an FDA-approved therapy since Xigris was withdrawn from market in 2011. According to the CDC, 350,000 patients die from sepsis during hospitalization and the CDC estimates that sepsis plays a role in the deaths of 1 in 3 patients. Clearly, this is the textbook case for the definition of an unmet need but early treatment can likely improve outcomes.

The leading FDA candidate to treat a sepsis-related indication is an endotoxin-depletion device being advanced by Canadian firm Spectral Medical, Inc. (EDT.TO, OTC-EDTXF) will enable improved outcomes for patients with Endotoxic Septic Shock (ESS). ESS is a life-threatening condition that arises when the body’s response to infection spirals into a storm of systemic inflammation. It is characterized by a dangerous drop in blood pressure and a cascade of multiple organ failure caused by the presence of endotoxin. Spectral Medical believes that “ESS affects approximately 140,000 patients each year with a mortality rate > 50%.”

Not to be outdone, Sigyn Therapy™ in vitro studies have validated the ability of to deplete endotoxin, just like Spectral Medical. However, the Sigyn studies validated the ability to deplete nine other sepsis-related targets from human blood plasma as well. Sigyn Therapy’s ability to target a broad range of harmful agents highlights its potential to treat sepsis and other inflammatory disorders.

End Stage Renal Disease Treatment

Endotoxemia and concurrent inflammation contribute to shorten the lives of end-stage renal disease (ESRD) dialysis patients. Sadly, during dialysis treatment, this can occur when bacterial products contaminate dialysis fluid or pass through dialysis membranes. On the heels of the aforementioned in vitro studies regarding endotoxemia depletion, Sigyn Therapy™ aims to extend the lives of dialysis patients.

In the United States, 550,000 ESRD patients receive ~85 million dialysis treatments each year. Tragically, the first-year mortality rate of ESRD patients on dialysis is 20-25% and 60-70% die within 5-years of dialysis initiation. Surprisingly, the leading cause of ESRD patient deaths is cardiovascular disease, with a prevalence of 70–80%. Sigyn Therapy™ targets hallmark drivers of cardiovascular disease, including endotoxin and excessively produced inflammatory cytokines.

Dual Development Path

Going forward, Sigyn may elect to run two generally simultaneous development pathways. To accelerate clinical development, the Company plans to submit documentation with the FDA to receive a Breakthrough Device designation for the treatment of sepsis and one for the treatment of endotoxemia and concurrent inflammation in dialysis patients. Management brings relevant experience, including oversight of the first therapy to receive two Breakthrough Device designations from FDA.

Beyond an opportunity to obtain a Breakthrough Device designation, the Company will submit an Investigational Device Exemption (IDE) to FDA to support a feasibility study whose target enrollment is 12–15 ESRD patients. The study will integrate Sigyn Therapy™ in series with regularly scheduled dialysis treatments to evaluate safety and monitor changes in endotoxin and inflammatory cytokine levels during treatment. Favorable results could advance first in-human studies as well.

Higher Sigyn Valuations

It is instructive to review how industry events and milestones along Sigyn’s development pathways can provide  a series of materially positive boosts in the Company’s valuation. Spectral Medical may be a year or so ahead of Sigyn with respect to its development of sepsis treatment as it relates to the presence of endotoxins. Sometime in this quarter, Spectral Medical will announce results from a recent study and we believe they could be favorable.

If they are, we believe that Sigyn would be a beneficiary of such an event.

As we noted above, Sigyn’s in vitro studies have already indicated the presence of endotoxins in sepsis patients similar to Spectral Medical. However, Sigyn was found to have validated the ability to deplete nine other sepsis-related targets as well. As of this writing, Sigyn’s low market cap is a small fraction of the Spectral Medical’s market cap. Not only could a rising tide Sigyn’s valuation but its profile as well, given the Company’s current pathway.

In the ESRD arena, the valuation drivers may be even more pronounced, considering there is no other peer following the Company’s development path. If Sigyn study results are favorable, we believe that would provide a major boost to overall valuation. Advancement in ESRD treatment offers a real benefit for patients and a business case for the industry as well.

A therapy that extends ESRD patient lives would offer significant quantifiable value to a U.S. dialysis industry dominated by Fresenius Medical Care and DaVita, Inc. Based on the size of their in-network patient populations, each month of extended life represents a revenue opportunity of approximately $1 billion to each company. Likewise, each week of reduced hospitalization (patients are out of network when hospitalized) could enable Fresenius and DaVita to each recoup approximately $250 million in what would have been lost revenues.

A Clear Milestone Path

Looking ahead, investors should take notice of the Sigyn-driven and other industry-driven milestone and events. Sigyn has a clear development path. These include two Breakthrough Device submissions, which we believe could be granted with 60 days following the submission. Such events would be followed by the ESRD feasibility study, which if favorable, could lead to a unique IDE submission.

Interestingly, such a submission could be aided by a tactical approach by Sigyn management. The Company has also identified several candidate opportunities for Sigyn Therapy™ to be designated as a Humanitarian-Use Device (HUD). An HUD designation provides an alternative regulatory pathway for a medical device to treat a disease or condition that manifests in fewer than 8,000 individuals in the United States each year. Upon receipt of a HUD designation, a Company is permitted to submit a Humanitarian Device Exemption (HDE) marketing application that is exempt from FDA effectiveness requirements. In the absence of efficacy data, Sigyn Therapeutics will seek to leverage safety data from its ESRD study in combination with results from in vitro studies already conducted.

Other, Valuable Pipeline Therapies

While Sigyn Therapy™ remains management’s core focus, we believe value exists for the Company’s oncology portfolio.

In our view, the full lineup of the Company’s therapeutic candidates is enviable and represents a deep portfolio in the field of extracorporeal blood purification. To optimize the benefit of drugs to treat cancer, the Company invented the  ImmunePrepTM platform to enhance the performance of immunotherapeutic antibodies; ChemoPrepTM  to improve the delivery of chemotherapy; and ChemoPureTM to reduce chemotherapy toxicity. 

As the lead candidate Sigyn Therapy™ gains development traction, it is possible that management may seek a partner to further development of the oncology portfolio, thereby monetizing these underdeveloped assets.

SIGYN LEADERSHIP TEAM

James A. Joyce, Co-Founder, Chairman, Chief Executive Officer

James “Jim” Joyce has 30+ years of diverse public market experience, which includes two decades of public company CEO and Corporate Board leadership roles. He is also an inventor or co-inventor underlying 18 pending or issued patents.

Prior to establishing Sigyn Therapeutics, Mr. Joyce was the founder and former Chairman and CEO of Aethlon Medical (NASDAQ – AEMD), a therapeutic technology company that he navigated from single shareholder start-up to Nasdaq-traded Company with 8000+ shareholders. During his tenure at Aethlon, Mr. Joyce oversaw the development of the Hemopurifier®, a first-in-class blood purification technology to address life-threatening viruses and cancer-promoting exosomes. Under his leadership, the Hemopurifier® became the first therapeutic candidate to be awarded two FDA “Breakthrough Device” designations and was the first and only device to receive “Emergency Use Authorization” (EAU) approval from both the FDA and Health Canada to treat Ebola virus. Time Magazine named the Hemopurifier® one of the “11 Most Remarkable Advances in Healthcare” and designated the device to its “Top 25 Best Inventions” award list. The Hemopurifier® has since been cleared by the FDA to treat severe COVID-19 infections in a clinical setting.

Under Mr. Joyce’s leadership, the Hemopurifier® was the subject of two Department of Defense (DOD) contract awards and a National Cancer Institute (NCI) contract. Mr. Joyce led the completion of approximately $100 million of equity financings on behalf of Aethlon Medical and established preclinical and clinical collaborations with more than twenty government and non-government research institutes.

Based on the use of the Hemopurifier® to treat HIV and Hepatitis-C infected individuals in India, Mr. Joyce was the recipient of the “Spirit of India Award” sponsored by the Bill & Melinda Gates Foundation and awarded each year by the American India Foundation to the American business leader who has demonstrated a commitment to accelerate social and economic change in India.

Mr. Joyce testified before Congress and lobbied Capitol Hill to promote the Hemopurifier® as a broad-spectrum countermeasure against bioterror and pandemic threats, which contributed to expanding the government-wide definition of treatment countermeasure to be inclusive of medical devices under U.S. law.

Mr. Joyce is also the founder and former Executive Chairman of Exosome Sciences, Inc. (ESI), a company focused on the discovery of exosomal biomarkers to diagnose and monitor cancer and neurological disorders. Inspired by the death of a former teammate, Mr. Joyce established a collaboration with the Boston University CTE Center to test his hypothesis that circulating exosomes transported tau protein cargos (exosomal tau or TauSome) that could provide a basis for a non-invasive blood test to diagnose and monitor neurological tauopathies, including chronic traumatic encephalopathy (CTE) and Alzheimer’s disease. As a result, ESI was invited to participate in the first NIH funded clinical study of CTE, which revealed TauSome levels to be approximately 9x higher in 78 former NFL players as compared to the same age group control subjects. The study results (co-authored by Mr. Joyce) were published in the Journal of Alzheimer’s Disease. Follow-on clinical studies are being conducted. Mr. Joyce established a collaboration with the Boston University Alzheimer’s Disease Center that also demonstrated TauSome levels to be significantly elevated in diagnosed Alzheimer’s patients.

Prior to founding Aethlon Medical and Exosome Sciences, Mr. Joyce operated James Joyce & Associates. He was the founder and former CEO of Mission Labs, Inc. and a principal at London Zurich Securities. Upon graduating from the University of Maryland, Mr. Joyce was first employed as a member of the Denver Broncos Football Club of the National Football League.

Annette Marleau, Ph.D. Chief Scientific Officer

Dr. Marleau is a recognized thought leader in the development of therapeutic blood purification technologies to address cancer.  Prior to joining Sigyn Therapeutics, Dr. Marleau was Chief Technology Officer at Immunicom, Inc., where she led R&D endeavors to establish a pipeline of blood purification candidates to treat cancer. She also served as Director of Research at Aethlon Medical, Inc., where she oversaw preclinical programs that facilitated the first-in-human clinical investigation of the Aethlon Hemopurifier® as an adjunct cancer therapy. 

Dr. Marleau has been awarded more than $6 million in NIH grants and contracts to serve as Principal Investigator for pre-clinical and clinical programs to advance blood purification technologies.  Additionally, she co-authored two FDA-cleared Investigational Device Exemptions, co-authored a regulatory submission that resulted in an FDA “Breakthrough Device” award, and is an inventor on pending and issued patents underlying blood purification therapies targeting cancer, inflammatory disorders, and life-threatening infectious diseases. 

Dr. Marleau completed a fellowship in immunology at Scripps Research Institute in La Jolla, CA. She is a graduate of Western University (PhD), Ontario Veterinary College at University of Guelph (Master of Science), and University of Waterloo (Bachelor of Science) in Canada.

Eric Lynam, Head of Clinical Affairs

Mr. Lynam, who has played key roles in developing, contracting and conducting over 190 clinical trials, will oversee clinical studies of Sigyn Therapy™ in the United States and abroad.  Previously, Mr. Lynam was the Director of Scientific and Medical Affairs for Pharmatech Incorporated until its recent acquisition by Caris Life Sciences.  While at Pharmatech, Mr. Lynam pioneered a high efficiency, patient centered clinical trials system, providing on-demand trial access to a network of more than 1,500 investigators. 

Craig P. Roberts, Co-Founder, Board Member

Mr. Roberts is an inventor of life-saving therapeutic technologies, which includes a Percutaneous Adult Extracorporeal Membrane Oxygenation (ECMO) system that was licensed and subsequently sold to C.R. Bard. Mr. Roberts is also an inventor of the IMPACT System, which received CE Mark clearance in the European Union and was subsequently registered in 32 countries and deployed to treat cytokine storm associated conditions, including sepsis, acute respiratory distress syndrome (ARDS), acute liver failure, severe pneumonia, and H5N1 bird flu virus infection. The IMPACT system was comprised of multiple cartridges and designed to reduce the presence of inflammatory cytokines from human blood plasma. As a Clinical Perfusionist, Craig has conducted more than 4,000 extracorporeal procedures, including adult and pediatric cardiopulmonary bypass, cardiac assist devices, ECMO (artificial lung), vascular access catheter systems, and continuous renal replacement therapy.

Richa Nand, Board Member

Richa Nand is a senior legal executive with more than 20 years of experience as an intellectual property (“IP”) attorney and strategic business advisor for biotechnology and medical device companies. She is the founder of Insight Patents, a legal and consulting firm providing IP and transactional corporate services for the life sciences industry. Ms. Nand previously served as Vice President of Corporate Development and Legal at Bird Rock Bio – a Johnson & Johnson-backed biopharmaceutical company in San Diego – and Vice President of Intellectual Property and Licensing; Director of Business Development; and In-House Patent Counsel at Cytori Therapeutics. Prior to law school, she was a biomedical researcher at Cedars Sinai Medical Center in Beverly Hills, California. Ms. Nand received a Bachelor of Science degree in Microbiology and Molecular Genetics from the University of California, Los Angeles, and a Juris Doctor degree from Boston University School of Law.

Jim Dorst, Board Member

Jim Dorst has more than 30 years of senior management experience in finance, operations, planning and business transactions at both private and public companies. He was most recently Director of Corporate Development at SYNNEX/Concentrix, where he was primarily responsible for mergers and acquisitions. Mr. Dorst was previously Chief Operating Officer (“COO”) and Chief Financial Officer (“CFO”) at SpectraScience, Inc.; CFO of Aethlon Medical, Inc. and Vice President of Finance and Operations for Verdisoft Corporation. In addition, he previously served as Senior Vice President of Finance and Administration at SeeCommerce; CFO and COO of Omnis Technology Corp; and CFO and Senior Vice President of Information Technology at Savoir Technology Group, Inc. Mr. Dorst practiced as a Certified Public Accountant with Coopers & Lybrand (now PricewaterhouseCoopers LLP); and holds a Master of Science degree in Accounting and a Bachelor of Science degree in Finance from the University of Oregon.

Christopher Wetzel, Board Member

Christopher Wetzel has more than 25 years of leadership experience in various aspects of the healthcare delivery system and since 2004, has served as Chief Executive Officer for the Surgery Center at Hamilton in New Jersey. His career has focused on building organizations, increasing operational efficiency, increasing profitability, maximizing revenue, and managing change in the complex and high-growth healthcare environment. Mr. Wetzel applied his broad background in strategy, finance, and operations to guide various entities starting new ventures, entering new markets, and reengineering business processes. He is a long-term investor in the extracorporeal therapy space. Mr. Wetzel received a Master of Business Administration degree in Healthcare Management and a Bachelor of Science degree in Nursing from Thomas Jefferson University (formerly Philadelphia University).

Michael Ryan, Board Member

Mr. Ryan is a seasoned executive, entrepreneur and investor within the early-stage technology and life science industry. Mr. Ryan is one of the Founder Directors of Irrus Investments, Ltd., a role he has held since 2011. Irrus Investments is the largest angel investment syndicate in Ireland with an emphasis on life science companies. To date, Irrus has invested over €40million in 35 early-stage life science and technology companies in Ireland, UK, Sweden and USA. Mr. Ryan previously served as Chief Executive Officer and Board Member of Sedana Medical, from 2011 until shortly before the Company launched on the Nasdaq owned First North stock exchange in Stockholm in 2017. Prior to this, he was the main shareholder and Chief Executive Officer of Artema Medical AB, where he helped orchestrate the Company’s acquisition by Datascope Corporation. Mr. Ryan holds a B.Eng in Mechanical Engineering and a Masters in Industrial Engineering from University College Dublin.

Scientific Advisory Board

H. David Humes, MD

David Humes, MD is an internationally recognized nephrologist and physician scientist. He received his B.A. in mathematics/physics from University of California, Berkeley and his M.D. from University of California, San Francisco as a Regent Scholar. He completed further training at UCSF, University of Pennsylvania and Harvard. He has been appointed to the faculty at Harvard Medical School and University of Michigan. Currently he is Professor of Internal Medicine at University of Michigan after serving as Chairman of the Department. He is considered a world-renowned scholar in the areas of nephrology, acute renal failure, cell therapy, biomaterials, device formulation, tissue engineering, and extracorporeal therapy. He has published over 250 scientific articles and book chapters, edited 6 medical textbooks, and has over 70 issued or applied patents. He has served on numerous editorial boards of scientific and medical journals. He has founded 4 spinout biomedical companies raising over $100 million of private equity. His research has been funded with over $40 million of grants from federal agencies and foundations. His accomplishments have been recognized as an Established Investigator of the American Heart Association, President’s Award of National Kidney Foundation, A.N. Richards Distinguished Achievement Award in Nephrology, Special Recognition Award from Association of Professors of Medicine, and Kolff Award in Regenerative Medicine. He is an elected member of the American Society of Clinical Investigation and the American Association of Physicians and a Fellow of American Association for the Advancement of Science and American Institute for Biological and Medical Engineering.

Alexander S. Yevzlin, MD, FASN

Dr. Yevzlin graduated magna cum laude from Dartmouth College. He did his residency in Internal Medicine at the University of Michigan and fellowship in Nephrology at Northwestern. Dr. Yevzlin is currently Professor of Medicine and director of Interventional Nephrology at the University of Michigan. Dr. Yevzlin has presented and published over 150 abstracts, invited lectures, and manuscripts. He is an internationally recognized leader in the field of Interventional Nephrology, having edited the first three textbooks on the subject, and is a past President of the American Society of Diagnostic and Interventional Nephrology. In addition to his academic contributions, Dr. Yevzlin has been involved the invention, design, and reduction to practice of multiple medical devices in his role as chief medical officer, chief science officer, and founder of multiple start-up biotech companies.

Ajay Verma, MD, PhD

Ajay Verma is a neurologist, neuroscientist, drug developer, inventor and biotech science advisor. Ajay most recently headed R&D efforts at Yumanity Therapeutics, developing drugs against novel targets for treating neurodegenerative diseases. Prior to that he was the EVP of Research and Experimental Medicine at Codiak Biosciences. He has also served as CMO at United Neuroscience (now called Vaxxinity), VP of Neurology at Biogen and Novartis, and Director of Neuroscience Experimental Medicine at Merck. His drug development experience spans small molecule, peptide/protein, antibody, oligonucleotide, vaccine and exosome drug platforms. He has largely focused on translational and early clinical development in neurology indications using precision drug development approaches that leverage biomarkers and experimental medicine paradigms. Ajay is interested in developing drugs for established as well as novel targets in Neurology. Prior to his Biopharma career, Ajay was s Professor of Neurology at the Uniformed Services University of the Health Sciences. He also worked as a staff neurologist at the Walter Reed Army Medical Center for 11 years after completing his neurology residency there. Ajay received his M.D. and Ph.D. from Johns Hopkins University, where he trained in the laboratory of Dr. Solomon Snyder. Ajay received his B.S. in Zoology from the University of Maryland. 

Donald J. Hillebrand, MD

Dr. Hillebrand is the Associate Medical Director of Liver Transplantation at Saint Luke’s Hospital of Kansas City and Associate Professor of Medicine at the University of Missouri Kansas City (UMKC).  Dr. Hillebrand has authored more that 20 Papers (published or in press/preparation) as well as 7 books (and/or book chapters). He is a recognized leader in the field of Hepatology and Liver Transplantation. In addition to his academic and clinical contributions, Dr. Hillebrand has served as a Principal Investigator in various clinical studies to evaluate extracorporeal treatments for liver disorders. Previously, he served on the Editorial Board of ASAIO Journal, was a Section Editor for the ASAIO Journal-Liver Transplantation, and a Reviewer for the European Journal of Gastroenterology & Hepatology.  Dr. Hillebrand graduated with distinction in Microbiology from Iowa State University.  He did his residency in Internal Medicine at Iowa State University Hospitals and Clinics and fellowships in Gastroenterology, ­Hepatology and Liver Transplantation at Iowa State University Hospitals and Clinics.

Eric W. Stroup

Eric Stroup has nearly 30 years of experience in the medical device field. He spent 26 years with Fresenius Medical Care, in various research and development leadership roles, most recently as the Senior Vice President of the Dialyzers, Membranes and Adsorbers Product Engineering Center. During his career, Eric has made significant contributions as a member of Senior Management in R&D. Eric collaborated with regional and global stakeholders to develop innovative product pipelines, drive key product development, technical design, and engineering milestones with a fiscally disciplined, business-oriented approach in his execution.

Among many notable accomplishments, he has a demonstrated history of product delivery. During his leadership, the Dialyzer R&D team was responsible for eight 510(k) decisions from the FDA for submissions in the last ten years (16 cleared 510(k)’s since 2000). These submissions supported a product family that sold greater than 55 million units annually in North America, representing a >70% market share and providing >$375Million in revenue annually.

Eric built the Fresenius Medical Care Dialyzer Global R&D team in North America from scratch (including the completion of the Ogden Design Center) and established a wining culture across its locations in the EMEA, NA, and AP regions. Eric has an extraordinary commitment to deliver results and an innovative approach to driving outcomes.

FINANCIALS

Management runs a tight ship at Sigyn, given the series of studies and FDA submission preparation, along with prototyping and limited manufacturing. It is expected that Sigyn will reach a number of milestones over the next 18 months, including two Breakthrough Device Designations, the ESRD feasibility study, along with other potential submissions and studies, based on Sigyn Therapy™ preliminary data. These events may require a nominal fund-raise of around $1.5 million, which would fund the submissions and ESRD feasibility study. If the data is favorable, as expected, a first in-human study in late 2026 may be prepared for a launch, accompanied by a financing, and perhaps coincide with a NASDAQ uplisting.

RISK FACTORS

In our view, the Company’s near-term risks are related to the award of one or two Breakthrough Device designations. The greater near-term clinical risk would be unfavorable or inconclusive data from the Company’s upcoming ESRD study. It is also possible that results are favorable, but not statistically significant related to efficacy or QoL.

A secondary risk is related to timing of the launch of the upcoming trials and studies. These risks are consistent with those facing firms of similar size and status to Sigyn. Moreover, we believe that prior study results, along with Sigyn’s strategic approach reduces the risks outlined above.

Volatility and liquidity are typical concerns for microcap stocks that trade on the over the counter (OTC) stock market. It is also possible that the share count could increase to fund future clinical trials and studies. However, an overriding financial benefit as a public company is the favorable access to and the availability of capital to fund research and development, product studies and launches, and other initiatives. Since the proceeds of any future funding would be used, in large part, to advance major business development, we believe that any dilutive effect from such a funding could be offset by related increases in market value.

VALUATION AND CONCLUSION

Sigyn is poised to change the way acute, life-threatening inflammatory conditions induced by endotoxemia and other concurrent inflammation. Many of these conditions have no approved therapy and represent billions in market size. Primary targeted conditions include end stage renal disease and sepsis, the #1 cause of deaths in hospitals worldwide. The Company also has a deep oncology portfolio.

Sigyn has reported favorable in vitro results and is poised to submit multiple Breakthrough Device submissions this year. The Company could be awarded these designations in the coming quarter. A planned ESRD feasibility study should commence in the next 12 months. Data could be derived from this trial within 6 months following the end of the study.

Our six-month price target of $9.00 reflects both the current undervalued and underfollowed status of Sigyn versus peers, but also the value of future milestones. In our view, Sigyn’s primary platform offers valuable, hidden features which could serve as a valuation driver. Looking ahead, future milestones could eventually lead to an M&A event.

Investment and Company Research
Opportunity Research
COMPANY REPORT

OKYO: WIND AT ITS BACK

Since our January 2025 company report, OKYO Pharma Limited (NASDAQ – OKYO) has been a stellar performer, nearly doubling its share price while achieving a new 52-week high earlier this week. Moreover, these shares have enjoyed a commensurate doubling of its average daily volume, led by a series of recent milestones that we outlined in our previous report. With potentially ground-breaking developmental news on the horizon during 3Q25, we believe that these shares have just begun their ascent toward our new, $8.00 price target.

OKYO may have discovered a first-of-its-kind treatment for a debilitating ocular pain condition called neuropathic corneal pain (NCP) that has no FDA approved, effective treatment. NCP is a debilitating condition affecting many tens of thousands worldwide, and is characterized by chronic, severe eye discomfort. A Phase 2 clinical trial for NCP commenced in October 2024 with top-line results slated to be released during 3Q25, an accelerated timeline from the original end of year estimate.

Urcosimod, the recently assigned official name by USAN for OKYO’s lead candidate, OK-101, was developed using a membrane-anchored-peptide (MAP) technology to produce a novel long-acting drug candidate for treating ocular front-of-the-eye diseases.

Urcosimod is a lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor which is typically found on immune cells of the eye and neurons and glial cells found on the dorsal root ganglion. ChemR23 is generally recognized as being responsible for the inflammatory response. Urcosimod was shown to produce anti-inflammatory and pain-reducing activities in mouse models of both dry eye disease and a neuropathic corneal pain animal model; and is designed to combat washout through the inclusion of the lipid ‘anchor’ contained in the candidate drug molecule to enhance the residence time of urcosimod within the ocular environment.

We estimate that the potential size of the market could be $6.4 billion or greater.

Recent Events have Served as Fundamental Drivers

In March 2025, OKYO filed a Fast Track designation application with the FDA for urcosimod, and on May 1, 2025, OKYO announced the FDA has granted Fast Track designation for urcosimod to treat NCP. Receipt of this designation typically leads to accelerating further clinical development as turn-around time for meetings with the FDA is shortened, as well as the potential for a rolling FDA review of the New Drug Application (NDA) to treat NCP.

On March 31, 2025, OKYO announced successfully establishing that urcosimod has been shown to be stable for over two and a half years in single-use ampoules used for administration of the drug to patients. Achieving this objective is a key, core component in meeting CMC (Chemistry, Manufacturing and Controls) requirements for a successful New Drug Application with the FDA. The FDA requires long-term shelf life of drugs as part of the approval process.

Early Closure of Phase 2 Trial a Major Plus

In late April 2025, OKYO reached its most significant step to date with respect to the urcosimod Phase 2 trial designed to treat 48 patients with NCP. After the completion of 17 patients of the intended 48 patients designed for the study, OKYO leadership elected to close the trial early to enable initiating analysis of the clinical data. This decision to close the trial was driven by OKYO’s strong desire to access the currently masked data in the randomized, placebo-controlled trial, and to use it to start planning further development of urcoisimod, assuming the top-line data are positive. Notably, OKYO pointed out that patients participating in the trial have all been diagnosed with long term chronic neuropathic corneal pain and had previously been treated with multiple therapies with very limited or no response.

Significant interest was also reported to be seen in the trial from long-term sufferers of NCP, and OKYO believed it should plan to expand the development program and move forward with a multicenter trial.

The implications of the early trial completion plus desire to immediately analyze clinical results and release top-line data suggest two immediate outcomes, pending positive top-line results, in our view. One, it has been reported by OKYO that a considerable number of potential patients have expressed an interest in participating in a future registrational trial. Two, a number of patients also have requested compassionate use of urcosimod which OKYO announced it is seeking to arrange with Tufts Medical Center, subject to the necessary FDA consents. While this is not a demonstration of evidentiary efficacy of urcosimod, it is a compelling set of observations that suggests a potential drug effect and illustrates the challenging condition in which the patients suffer and the need for the Fast Track designation by the FDA.

LOOKING AHEAD

Management believes that concluding the trial early enables OKYO to save time in advancing the program to a meeting with FDA to discuss further development of urcosimod. Moreover, the analysis of the data from the 17 patients in the study could lead to the release of top-line results in the first half of 3Q25. Considering that urcosimod has already demonstrated favorable safety and placebo-like tolerability in OKYO’s previous 240 patient Phase 2 trial, we believe that OKYO leadership has reasons to be encouraged.

If top-line results are favorable when announced in the next quarter, the Company would likely seek to request an end-of-phase 2 meeting with FDA to explore accelerated plans for the drug’s further clinical development. Topics would likely include future trial design and protocols, primary and secondary endpoints, along with subject size and the number of sites. At this stage it is too early to speculate on the timing and roadmap of future development. However, the key takeaway for investors is that the acceleration of the release of potentially favorable top-line Phase 2 data, assuming the data are positive, should shorten the time that we believe these shares would take to reach our upgraded, $8.00 price target.

Against this backdrop, if an updated roadmap is announced due to favorable data, timelines, and prospective outcomes bode well for OKYO.

Our original $5.00 price target reflected a longer Phase 2 trial that would lead to later published results. However, we have elected to raise our price target to reflect a change in status, in our view. These changes include the acceleration of the closing of the trial, the desire of some patients to utilize the drug on a compassionate basis, and the Fast Track designation. These are all new events since our January 2025 report. As a first-mover that has the potential to publish favorable results for a debilitating condition that has a serious unmet need, and represents a multi-billion-dollar market, we believe these shares could reach the $8 level or higher, in short order. Furthermore, we believe that given the M&A activity in the ocular space, a deal with a partner could occur, and that the Company would benefit from the aforementioned re-valuation of these shares.

RECENT TRADING HISTORY FOR OKYO

(Source: www.StockCharts.com)

Investment and Company Research
Opportunity Research
COMPANY REPORT

January 30, 2025

 

OKYO PHARMA LIMITED

(NASDAQ – OKYO)

Industry: BioPharma

12 Mo. Price Target: $5.00

 

 

COMPANY OVERVIEW

We initiated coverage of London-based OKYO Pharma Limited (NASDAQ: OKYO) on May 9, 2023. OKYO is a biotechnology company focused on the discovery and development of novel molecules to treat inflammatory ocular diseases and ocular pain. OKYO’s lead drug candidate OK-101, which is presently in clinical development, is administered topically to the eye and was first developed to treat dry eye disease (DED), an estimated $5.5 billion global market. The Company has achieved significant development milestones since our original report was published. In fact, OKYO may have discovered a first-of-its-kind treatment for a debilitating ocular pain condition called neuropathic corneal pain (NCP) that has no FDA approved drug. A Phase 2 clinical trial for NCP commenced in October 2024 with results slated to be released by year-end 2025. If results are favorable, we believe it will serve as a key catalyst to drive prospective M&A for the Company, and power OKYO’s share price to our updated 12-month price target of $5.00.

Development of OKYO’s Lead Clinical Drug OK-101

OK-101 was developed using a membrane-anchored-peptide (MAP) technology to produce a novel long-acting drug candidate for treating ocular front-of-the-eye diseases. OK-101 is a lipid conjugated chemerin peptide agonist of the ChemR23 G-protein coupled receptor which is typically found on immune cells of the eye and neurons and glial cells found on the dorsal root ganglion. ChemR23 is generally recognized as being   responsible for the inflammatory response. OK-101 was shown to produce anti-inflammatory and pain-reducing activities in mouse models of both dry eye disease and a neuropathic corneal pain animal model; and is designed to combat washout through the inclusion of the lipid ‘anchor’ contained in the candidate drug molecule to enhance the residence time of OK-101within the ocular environment.

Brilliant Pivot by OKYO Management

Most observers and clinicians would characterize the current DED market’s seven FDA-approved products as subpar, or underwhelming. There are issues with efficacy, slow onset of action and a plethora of side effects. In the beginning of 2024, OKYO released top-line data from the placebo controlled, randomized, multi-center, multi-arm, Phase 2 clinical trial of OK-101 to treat DED. The data from the trial demonstrated that OK-101 showed statistically significant reduction in a number of secondary endpoints, including ocular burning/stinging, blurred vision, and of particular importance, ocular pain.  Recognizing that patients with DED are prone to suffer from NCP, as well as the earlier positive neuropathic corneal pain animal model results, the Company had begun the process of filing an IND for OK-101 to treat NCP, a debilitating condition for which there is no FDA approved drug. Armed with these data, OKYO management elected to pivot from a DED-centric treatment approach to the NCP space where the Company carries first-mover advantage.  In February 2024, FDA cleared an IND for OK-101 for NCP.  OKYO is the first company to be granted an IND to treat NCP, a major unmet medical need. And on October 23, 2024, OKYO announced the dosing of the first patient in a Phase 2 trial of OK-101 to treat NCP.  OKYO is the first company to enroll patients in a clinical trial designed to enroll patients specifically diagnosed with the NCP condition.

The size of the NCP market has not been fully established, due in part to the sophisticated equipment required to diagnose the condition. Since it is not widely available, we have applied a preliminary patient size range in the US only. According to the National Eye Institute, 16.4 million Americans suffer from DED. Our current patient size range of 160,000 represents 1% of the US DED market. Using an estimated (low) annual patient cost of $40,000 for an approved OK-101 for NCP, we arrive at a potential market size of $6.4 billion.. As indicated by these figures, the receipt of a potential Orphan Drug Designation by the FDA for its drug candidate could be significant. However, it is possible that in future years, as patients can be tested on a wider scale, the patient figure may reach a level that exceeds the sub-200,000 patient figure required to maintain Orphan Drug designation.

We expect a series of milestones including enrollment updates to favorably impact OKYO’s shares throughout the year. For example, the Company anticipates completing enrollment of the 48-patient trial by the end of 2Q 2025.

Valuation

Given that the Company is primed to reach a key valuation-changing milestone with the release of Phase 2 data for NCP at year-end, we have elected to publish a price target that we believe could be achieved in the next 12 months. If multi-dose data and efficacy match OKYO’s primary and secondary objectives, we believe that these data could serve as a catalyst for a mid-tier – to top tier pharmaceutical firm to seek to enter into a transaction with OKYO. This transaction could be in the form of M&A or an investment and related licensing or partnership arrangement with OKYO in exchange for future R&D. We believe that given the safety profile, a compassionate care usage profile could be granted by the FDA, thereby enabling OKYO and a potential partner to benefit. Moreover, due to the nature of OK-101’s chemistry, administration, and its first-mover advantage, we strongly believe that the Company’s OK-101 (and a partner or an acquirer) could be well positioned to be the first FDA approved topical solution for the treatment of NCP.

Against this backdrop, in 2026, we envision a potential investment of millions for future R&D, with additional funds to be invested based on R&D and other milestones, and a potential right to acquire a majority stake in or an outright purchase of OKYO.

Our 12-month, pre-data release price target is $5.00 and is designed to reflect the potential valuation assessed following a successful Phase 2 trial for a first mover product slated to treat an unmet need that represents a large market opportunity. We believe that in 2026/2027, when a deal with a partner could occur, the Company would benefit from a re-valuation of these shares. This price target is affirmed by a future valuation estimate. This estimate assumes $75M in annual CY2029 sales for an FDA-approved OK-101 and an assigned 6.5x sales multiple, which represents a slight discount to the median multiple assigned to a series of ocular M&A transactions. We thus arrive at a future potential $487M valuation in an acquisition. Our 12-month price target of $5.00 reflects this figure discounted back four years at a rate of 30%, on a Net Present Value basis.

THE DED MARKET

[Analyst’s Note: While the objective of this report is to inform readers regarding the untapped potential of the NCP indication, it is important to note both the success of the OKYO trial and the fact that NCP patients are a combination of a subset of DED patients along with patients who develop the disease due to other causes such as surgery and ocular injury.]

Dry eye disease, or keratoconjunctivitis sicca, is a common and often chronic problem, particularly in older adults. With each blink of the eyelids, tears spread across the front surface of the eye, known as the cornea. Tears provide lubrication, reduce the risk of eye infection, wash away foreign matter in the eye and keep the surface of the eyes smooth and clear. Excess tears in the eyes flow into small drainage ducts in the inner corners of the eyelids, which drain into the back of the nose.

People with dry eyes either do not produce enough tears or their tears are of a poor quality due to a deficient tear film lipid layer, which increases tear evaporation. Advanced dry eyes may damage the front surface of the eye and impair vision. Dry eyes can develop for many reasons, including age, gender, medications, medical conditions, and other factors. Tear film instability triggers chronic inflammation of the ocular surface which leads to symptoms of constant pain, itchiness, burning, visual impairment, etc.

DED affects more than 35% of people aged 50+, with women representing two-thirds of this segment. One reason why this segment may be more predisposed than men could be related to hormonal changes that occur after menopause. These hormonal changes affect the quantity and quality of the tear film, or layer of tear fluid that protects the eye.

The prevalence of dry eye is expected to increase substantially going forward due to an aging population and the increased use of contact lenses and digital screen time.  On a global basis, the DED market is expected to grow from $5.2B in 2019 to $6.54B in 2027, according to Fortune Business Insights. The US market is projected to be around $2.4B.

Below is a table of the leading approved DED drugs and their producers, who, in our view, could serve as potential OKYO partners.

THE OKYO DIFFERENCE

History

Tracing its roots to 2018, OKYO is a biopharmaceutical company developing next-generation therapeutics to improve the lives of patients suffering from inflammatory eye diseases and ocular pain. Its research program is focused on a novel G Protein-Coupled Receptor, or GPCR, which management believes plays a key role in the pathology of inflammatory eye diseases representing high unmet medical need. OKYO’s therapeutic approach is focused on targeting inflammatory and pain modulation pathways that drive these conditions. OKYO is presently developing OK-101, its lead clinical product candidate, for the treatment of neuropathic corneal pain (NCP). The Company is also evaluating its potential in benefiting patients with dry eye disease (“DED”).

In 2018, OKYO successfully obtained (via assignment from Panetta Partners Limited, a related party) an exclusive license from On Target Therapeutics (OTTx) to patents owned or controlled by OTTx and a sub-license from OTTx to certain patents licensed by OTTx from Tufts Medical Center (TMC) to support its ophthalmic disease drug programs. These licenses gave OKYO the right to exploit the IP estate which is directed to compositions-of-matter and methodologies for treating ocular inflammation such as DED with lipid-linked chemerin analogues. OKYO also has a license from TMC to a separate IP estate for treating symptoms of neuropathic corneal pain, uveitis and associated pain. The scope of the TMC IP granted use through the sublicense with OTT is commensurate with the scope of use of the IP granted to OTT from TMC. This intellectual property, which includes 3 key patents on OK-101, covering areas including technology, dry eye, and neuropathic corneal pain, forms the intellectual property basis of OKYO’s OK-101 program.

The Science

The development of new drugs to treat DED has been particularly challenging due to the heterogeneous nature of the patient population suffering from DED, and due to the difficulties in demonstrating an improvement in both signs and symptoms of the disease in well-controlled clinical trials. OK-101 is designed to target a chemokine-like receptor 1, or CMKLR1, or CHEMR23, which is a G protein-coupled receptor expressed on macrophages, neutrophils, monocytes, plasmacytoid/myeloid dendritic cells, natural killer cells and nonhemopoietic cell types, such as endothelial and epithelial cells as well as neurons and glial cells in the dorsal root ganglion, and retina. Activation of CMKLR1 by its endogenous peptide ligand chemerin is known to generate a pro-inflammatory response, and peptide fragments of chemerin have been discovered to generate the opposite, producing an anti-inflammatory response. However, natural ligands for CMKLR1 have short half-lives due to rapid inactivation. Development of OK-101, a stable, high potency CMKLR1 agonist provided an important step toward the development of a new class of anti-inflammatory and anti-ocular pain therapeutics that can be applied to the treatment of ophthalmic diseases including NCP and DED.

OK-101: DED Trial

In July,2024 OKYO announced:

“…promising new categorical data analyses from the recent OK-101 Phase 2 trial in DED patients. These analyses have identified conjunctival staining and ocular pain as the highest potential “sign” and “symptom” co-primary endpoints to be explored in the next DED trial of OK-101. The data from this first in-human trial of OK-101 in patients with DED has established a clear road map for future clinical development in this indication. Through our analytical work we have concluded that conjunctival staining and ocular pain represent important and de-risked endpoints to be studied further to help underserved patients whose dry eye symptoms include a pain component. Furthermore, this trial demonstrated a favorable tolerability profile for OK-101, with an excellent eyedrop comfort score for a topically administered drug.”

“Notably, the number of patients showing both a reduction in conjunctival sum staining and in the pain symptom in the OK-101-treated group was 34.2% compared to 20.3% in the placebo-treated group, a 68% improvement. Similarly, the number of patients with reduction in conjunctival sum staining and burning/stinging symptoms were also numerically higher in the OK-101-treated group (32.9%) compared to the placebo-treated group (20.3%), with a 62% improvement.”

This Phase 2 trial featured 240 patients diagnosed with DED.

OK-101: NCP Trial

OK-101’s IND, which was cleared by FDA in February 2024, is the first IND granted by FDA to treat patients with NCP. OKYO announced the start of the OK-101 NCP trial with the announcement of dosing of the first patient in October 2024. Notably, the Company announced that OK-101 was the first drug candidate to enroll patients specifically diagnosed with NCP in a clinical trial. This trial is designed as a randomized, placebo-controlled, double-masked study to treat 48 NCP patients. The trial has three arms, utilizing 0.05% OK-101 along with 0.1% OK-101 and placebo, (16 patients per arms). The protocol calls for 5 visits over the course of 16 weeks, with a study duration of an expected 9-12 months. Pedram Hamrah, MD, one of the leading authorities on NCP in the US is the trial’s Principal Investigator which is being conducted at the Tufts Medical Center in Boston. Given the favorable safety profile, previous data, first-mover advantage and the ease of a topical administration, it is easy to be favorably disposed toward a positive outcome later this year.

What is Neuropathic Corneal Pain?

The publication Nature published an in-depth clinical paper on NCP and both its description and findings scream for the need to treat sufferers, some of whom have become suicidal as a result of the condition. The text below features excerpts from both the abstract and introduction. I have highlighted critical points for convenience. The link to the full document is found below.

“Corneal neuropathic pain (CNP) is a poorly defined disease entity characterised by an aberrant pain response to normally non-painful stimuli and categorised into having peripheral and central mechanisms, with the former responding to instillation of topical anaesthetic. CNP is a challenging condition to diagnose due to numerous aetiologies, an absence of clinical signs and ancillary tests (in vivo confocal microscopy and esthesiometry), lacking the ability to confirm the diagnosis and having limited availability. Symptomatology maybe mirrored by severe and chronic forms of dry eye disease (DED), often leading to misdiagnosis and inadequate treatment. In practice, patients with suspected CNP can be assessed with questionnaires to elicit symptoms. A thorough ocular assessment is also performed to exclude any co-existent ocular conditions. A medical and mental health history should be sought due to associations with autoimmune disease, chronic pain syndromes, anxiety and depression. Management begins with communicating to the patient the nature of their condition. Ophthalmologists can prescribe topical therapies such as autologous serum eyedrops to optimise the ocular surface and promote neural regeneration. However, a multi-disciplinary treatment approach is often required, including mental health support, particularly when there are central mechanisms. General practitioners, pain specialists, neurologists and psychologists may be needed to assist with oral and behavioural therapies. Less data is available to support the safety and efficacy of adjuvant and surgical therapies and the long-term natural history remains to be determined. Hence clinical trials and registry studies are urgently needed to fill these data gaps with the aim to improve patient care.”

“Corneal neuropathic pain (CNP) is being increasingly recognised, particularly in patients with a diagnosis of dry eye disease (DED) [1], for its impact on a patient’s quality of life [2, 3]. The impacts can be mild with minimal effects on activities of daily living to severe with the patient experiencing debilitating symptoms that can lead to a deterioration in their physical and social well-being [4]. Reports have emerged of its occurrence and burden following cataract and refractive surgery [5,6,7] and in those with neurotrophic keratopathy [8], chronic pain syndromes [9, 10] and autoimmune diseases [11, 12]. The overarching feature of CNP is a heightened experience of pain without commensurate clinical signs [4, 13, 14].”

A range of symptoms can be produced by CNP with many overlapping those of DED, such that it is frequently misdiagnosed as dry eye [13, 18]. 

https://www.nature.com/articles/s41433-024-03060-x

Since DED patients can also suffer from neuropathic corneal pain, it can conceivably make those patients more resistant to anti-inflammatory drugs. Hence, those DED patients suffering from NCP would also benefit from a drug that comprises both anti-inflammatory and neuropathic pain-reducing characteristics. Enter OKYO. The ChemR23 receptor on leukocytes targeted by OK-101 was also shown to be expressed on neurons and glial cells in the dorsal root ganglion and spinal cord. Recent data utilizing a mouse model of NCP demonstrated that OK-101, given topically to the eyes of mice, was able to reduce neuropathic pain in these animals. These as well as other data in animal models and in in vitro studies indicate that topically administered OK-101 peptide is capable of not only producing an anti-inflammatory response but also capable of reducing neuropathic corneal pain. Thus, OK-101 may be well-positioned to improve the quality of life of NCP sufferers. Hence, OK-101 serves as a potentially promising candidate for the treatment of both inflammation and pain for those suffering from NCP.

The present size of the NCP market will ultimately depend on whether this disease is accorded orphan disease status by FDA. Strict diagnosis for NCP presently requires a microscopic technique called confocal microscopy for directly imaging the ocular nerves to determine nerve damage. Since this technique is not widely available by ophthalmologists, there is presently no accurate measure of the number of people with the condition. To make an estimate of the NCP market size, we have applied a preliminary patient size range in the US only. According to the National Eye Institute, 16.4 million Americans suffer from DED. Our current patient size range of 160,000 represents 1% of the US DED population.

It is possible that the winds may be in OKYO’s favor. First, OK-101 to treat NCP may be designated as an Orphan Drug if it is found by the FDA to have fewer than 200,000 patients with the condition. Given the gray area outlined above and affirmed in the Nature piece, OKYO stands a good chance to be granted such designation. It could potentially lead to outsized pricing, if and until a re-assessment of the figures occurs whereby pricing might normalize.

This assumes an FDA approval, of course. Another wind at OKYO’s back is the fact that a compassionate care usage may be in order at some point due to the quality-of-life issues and suicide rates due to the debilitating condition, etc.

Using an estimated (low) annual patient cost of $40,000 for an approved OK-101 for NCP with an Orphan Drug Designation, we arrive at a potential market size of $6.4 billion, at the lower patient level. As indicated by these figures, the receipt of a potential Orphan Drug Designation by the FDA for its drug candidate could be significant. However, we also take the approach that in future years, as patients can be tested on a wider scale, the 2% or higher figure may evolve as an accurate patient estimate, albeit with a potentially, slightly lower cost for the topical solution.

THE OKYO PHARMA LEADERSHIP TEAM

In our view, the OKYO team is an enviable one, given the strength of its members’ history of success in their respective industries.

Gabriele Cerrone, Non-Executive Chairman

Mr. Cerrone has a successful track record and extensive experience in the financing and restructuring of micro-cap biotechnology companies. He has founded ten biotechnology companies in oncology, infectious diseases and molecular diagnostics, and has taken nine of these companies to the NASDAQ Market and two to the Main Market and AIM Market in London. Mr. Cerrone is Executive Chairman and Founder of Tiziana Life Sciences plc (NASDAQ: TLSA) a neuroinflammatory focused therapeutics company. Mr. Cerrone also co-founded Cardiff Oncology, Inc. (NASDAQ: CRDF), an oncology company and served as its Co-Chairman; he was a co-founder and served as Chairman of both Synergy Pharmaceuticals, Inc. (NASDAQ: SGYP) and Callisto Pharmaceuticals, Inc. (AMEX: CLSP), and was a Director of and led the restructuring of Siga Technologies, Inc. (NASDAQ: SIGA). Mr. Cerrone also co-founded FermaVir Pharmaceuticals, Inc. and served as Chairman of the Board until its merger in September 2007 with Inhibitex, Inc. Mr. Cerrone served as a director of Inhibitex, Inc. until its US$2.5bn sale to Bristol Myers Squibb Co in 2012.

Mr. Cerrone is also the Co-Founder of Rasna Therapeutics Limited (OTCMKTS: RASP), a company focused on the development of therapeutics for leukaemias; Co-Founder of Hepion Pharmaceuticals, Inc. (Nasdaq: HEPA); Executive Chairman and Co-Founder of Gensignia Life Sciences, Inc., a molecular diagnostics company focused on oncology using microRNA technology; and Executive Chairman and founder of Accustem Sciences plc; and founder of BioVitas Capital Ltd.

Gary S. Jacob, PhD, Chief Executive Officer

Dr. Jacob has over 35 years of extensive experience in the pharmaceutical and biotechnology industries across multiple disciplines, including research and development, operations, business development, capital financing activities and senior management expertise. He has developed broad and influential contacts throughout the biopharmaceutical, financial, banking and investor communities. Dr. Jacob is the Co-Founder and former CEO and Chairman of Synergy Pharmaceuticals. During his time at Synergy, he served as Chairman, Chief Executive Officer and Executive Chairman, and is the co-inventor of Synergy’s FDA-approved drug Trulance® which is currently marketed in the U.S. by Bausch Health, Inc. to treat functional GI disorders. Dr. Jacob is also the former CEO and Managing Director of Immuron Inc., an Australian biotechnology company dual-listed on the Australian ASX exchange and on NASDAQ, as well as the former Chairman of the Board of Hepion Pharmaceuticals, Inc., a public NASDAQ listed company. He is currently a board member of Actavia, a public biotechnology company, and was also on the Board of Directors of Cardiff Oncology, Inc., a NASDAQ listed public oncology company. He served as Chief Executive Officer and Director of Callisto Pharmaceuticals, Inc. from May 2003 until January 2013.

Prior to his involvement with Callisto and Synergy, Dr. Jacob was at Monsanto/G.D. Searle, where he was Director of Glycobiology and a Monsanto Science Fellow, specializing in the field of Glycobiology and drug discovery. Dr. Jacob holds over 30 patents and is the co-inventor of two pharmaceutical drugs which are FDA approved. Dr. Jacob earned a B.S. cum laude in Chemistry from the University of Missouri, St. Louis and holds a Ph.D. in Biochemistry from the University of Wisconsin, Madison.

Raj Patil, PhD, Chief Science Officer

Dr. Patil brings 30 years of ophthalmic experience and a powerful combination of academic scholarship and pharmaceutical R&D excellence.

Dr. Patil previously worked with Ora Inc, as Vice President of Research & Development, where he was responsible for driving all anterior and posterior segment research of Ora’s R&D Institute. Earlier in his career, he worked at iVeena Delivery Systems as Vice President of Advanced Ocular Delivery Systems. His tenure at iVeena included a two-year sabbatical in Singapore, where he served as an Associate Professor of Ophthalmology at DUKE/NUS Medical School and Principal Investigator at Singapore Eye Research Institute.

Dr. Patil also held a number of leadership roles at Alcon/Novartis Institute of Biomedical Research, including Associate Director of Research and Head of Molecular Pharmacology glaucoma and retina research. Prior to joining the business world, Dr. Patil served as an Associate Professor of Ophthalmology, Cell Biology & Genetics at the University of Nebraska Medical Centre in Omaha and as an Assistant Professor of Ophthalmology, Molecular Biology & Pharmacology at Washington University in St. Louis.

Dr. Patil received his PhD in Biochemistry from the National Chemical Laboratory/University of Pune, India and completed his postdoctoral training in Biochemistry and Molecular Biology at the University of Michigan, Ann Arbor, MI. He is the recipient of the Olga Keith Wiess Special Scholar Award from the Research to Prevent Blindness Foundation, and a NIH Director’s New Innovator Award. Dr. Patil has authored over 50 peer-reviewed research articles and serves as a reviewer and editorial board member for numerous journals and is frequently invited to lecture at academic and industry events.

Keeren Shah, Chief Financial Officer

Keeren Shah serves as our Chief Financial Officer. Ms. Shah currently also serves as the Finance Director of Tiziana Life Sciences LTD, Accustem Sciences Limited and Rasna Therapeutics Inc., having previously served as the Group Financial Controller for all businesses from June 2016 to July 2020. Prior to joining the Company, Ms. Shah spent 10 years at Visa, Inc. as a Senior Leader in its finance team where she was responsible for key financial controller activities, financial planning and analysis, and core processes as well as leading and participating in key transformation programmes and Visa Inc.’s initial public offering. Before joining Visa, Ms. Shah also held a variety of finance positions at other leading companies including Arthur Andersen and BBC Worldwide. She holds a Bachelor of Arts with honours in Economics and is a member of the Chartered Institute of Management Accountants.

Willy Simon, Non-Executive Director

Willy Jules Simon is a banker and worked at Kredietbank N.V. and Citibank London before serving as an executive member of the Board of Generale Bank NL from 1997 to 1999 and as the chief executive of Fortis Investment Management from 1999 to 2002. He acted as chairman of Bank Oyens & van Eeghen from 2002 to 2004. Willy Simon has been the chairman of Bever Holdings, a company listed in Amsterdam, since 2006 and Chairman of Ducat Maritime since 2015. He is also a non-executive director of Tiziana Life Sciences plc.

John Brancaccio, Non-Executive Director

Mr. Brancaccio, a retired CPA, is a financial executive with extensive international and domestic experience in pharmaceutical and biotechnology for privately and publicly held companies. From 2000 to 2002, Mr. Brancaccio was the Chief Financial Officer/Chief Operating Officer of Eline Group, an entertainment and media company. From May 2002 until March 2004, Mr. Brancaccio was the Chief Financial Officer of Memory Pharmaceuticals Corp., a biotechnology company. From April 2004 until May 2017, Mr. Brancaccio was the Chief Financial Officer of Accelerated Technologies, Inc., an incubator for medical device companies. Mr. Brancaccio is currently a director of Cardiff Oncology, Inc., Hepion Pharmaceuticals, Inc., Rasna Therapeutics, Inc., and Tiziana Life Sciences plc.

Bernard Denoyer, Non-Executive Director

Bernard F. Denoyer has 49 years of financial management experience including his service as Senior Vice President, Finance and Secretary of development stage Synergy Pharmaceuticals, Inc, from July 2008 until FDA approval and his retirement in June 2017. Between 2004 and January 2013 Mr. Denoyer concurrently served as Principal Financial Officer of Synergy’s former parent company, Callisto Pharmaceuticals, Inc. From October 2000 to December 2003, Mr. Denoyer was an independent consultant. Prior to this, Mr. Denoyer served as Chief Financial Officer and Senior Vice President of META Group, Inc. Mr. Denoyer earned his CPA with Ernst & Young in 1975. He received a master’s Certificate of Accounting from the Kellogg Graduate School of Management in 1974, an MBA in Finance with honours from Columbia Business School in 1972 and a BA in Economics from Fairfield University in 1969. Mr. Denoyer is fluent in French and studied in Paris at l’Istitut d’Etude Politique et Economique in 1968. He is currently serving on the Board of Trustees for two not-for-profits, St. Edmunds Retreat, Inc. and Midwestern Connecticut Council on Alcoholism, Inc.

SCIENTIFIC ADVISORY BOARD

Napoleone Ferrara, MD, Board Member

Dr. Ferrara is a Professor at the University of California San Diego Medical Center and a member of The National Academy of Sciences and has received numerous prestigious awards, including the Lasker Award and the Breakthrough Prize in Life Sciences. His research on understanding the role of angiogenesis and vascular endothelial growth factor (VEGF) in cancer development, led to the discovery that VEGF is a key mediator of angiogenesis associated with intraocular neovascular syndromes. This pioneering research led to the clinical development of a humanized anti-VEGF Fab (Ranibizumab, Lucentis®), which has also been approved as a therapy for neovascular age-related macular degeneration (AMD), retinal vein occlusion and diabetic macular edema. Ranibizumab and other anti-VEGF agents have had a dramatic impact on the development of therapies for these blinding disorders. When Lucentis® (Ranibizumab) received FDA approval in late June 2006, the new macular degeneration drug was celebrated as a major medical breakthrough. Dr. Ferrara’s research also led to the development and approval of humanized anti-VEGF mAbs (Bevacizumab; Avastin®) for cancer treatment, with Avastin® being one of the bestselling cancer drugs over the last two decades. Lucentis® and Avastin® collectively achieved over $9 billion in sales last year.

Pedram Hamrah, MD, FRCS, FARVO, Board Member

Pedram Hamrah, MD is Co-Director of the Cornea Service and Director of the Center for Translational Ocular Immunology at New England Eye Center at Tufts Medical Center in Boston. Dr Hamrah’s research interests focus on corneal immunology and neuroscience, ocular imaging (immuno-imaging), ocular surface diseases and corneal neuropathic pain. He is currently on faculty at the departments of Ophthalmology and Bioengineering at Tufts University, where he is the director of clinical research and director of the Center for Translational Ocular Immunology. In addition, he is a faculty member at the immunology, neuroscience, and cell, molecular and developmental biology graduate programs at the Sackler School of Graduate Biomedical Sciences at Tufts. Throughout his career, he has focused on discovery, patient care and teaching. Dr. Hamrah currently serves on over a dozen editorial boards, is the associate editor for The Ocular Surface and TVST, section editor for Eye and assistant editor at Ocular Immunology and Inflammation.

Jay S. Pepose, MD, PhD, FARVO

Dr. Pepose, a specialist in refractive surgery and corneal and external diseases, is the founder and Medical Director of the Pepose Vision Institute and held the Bernard Becker Chair in Ophthalmology and Visual Sciences at Washington University School of Medicine in St. Louis. He is a consultant to numerous ophthalmic drug and device companies and serves as a Director and Chief Medical Advisor for Ocuphire Pharma. Dr. Pepose has been involved in over 40 clinical research trials, including registration trials for dry eye drugs, and has been the recipient of R-01 grant support from the National Eye Institute. He has served on the editorial boards of numerous prestigious journals, including the American Journal of Ophthalmology, Investigative Ophthalmology & Visual Science (IOVS), Cornea, and The Journal of Refractive Surgery and has over 200 peer reviewed publications. Dr. Pepose, an ARVO Gold Fellow, is a recipient of the Cogan Award from the Association for Research in Vision and Ophthalmology (ARVO) and the Life Achievement Honor Award from the American Academy of Ophthalmology. Dr. Pepose received an A.B. and M.A. in neurophysiology from Brandeis University and completed the M.D.-Ph.D. program at UCLA School of Medicine. He completed ophthalmology residency at the Wilmer Institute at the Johns Hopkins Medical Center and fellowship training at Georgetown University Medical Center.

Victor Perez, MD

Dr Victor Perez is a Director of Cornea Research at Bascom Palmer Eye Institute, at the University of Miami Miller School of Medicine. He is a clinician-scientist investigator in the field of ocular immunology and ocular surface diseases and served as Director of Duke Eye Center’s Ocular Immunology Center before joining Bascom Palmer Eye Institute.

Anat Galor, MD

Dr. Anat Galor is a cornea and uveitis trained specialist with dual appointments at the Bascom Palmer Eye Institute and the Miami VA medical center. Dr. Galor completed an ophthalmology residency at the Cole Eye Cleveland Clinic, a uveitis fellowship at the Wilmer Eye Institute, and a cornea and external diseases fellowship at Bascom Palmer Eye Institute. Dr. Galor currently runs the ocular surface pain program at the Bascom Palmer Eye Institute and the Miami VA and has focused her research on understanding mechanisms of pain in dry eye, with an emphasis on studying new diagnostic and treatment modalities.

Mark Milner, MD

Dr. Mark Milner is Director of Cornea at Goldman Eye in Palm Beach Gardens, Florida with a focused interest in dry eye disease and dysfunctional tear syndrome. Dr. Milner completed his ophthalmology residency at the New York Eye and Ear Infirmary and his fellowship in cornea, external disease and uveitis at Francis I. Proctor Foundation at the University of California, San Francisco. Dr. Milner served as an Associate Clinical Professor at Yale University Medical School, Department of Ophthalmology, and was previously the Director of the Cornea Clinic at The Veterans Administration Medical Center in West Haven, CT.

FINANCIALS SNAPSHOT

Given that OKYO is pre-revenue and has a lean infrastructure, line items in the P&L are few, as they generally just encompass research and development and general and administrative expenses. The Company has a March fiscal year and we have endeavored to project expenses to coincide with the continuation and conclusion of the Phase II trial. It should be noted that OKYO plans to raise funds this year to complete the cost of the clinical trial, which is lower than many in this industry segment. A $1.4M non-dilutive funding occurred on January 21, 2025, which is a great shot in the arm.

Going forward, we believe that the final trial results will be available at year-end 2025 and that a subsequent Phase III registration trial should likely commence within the next 6 months after this trial. At this juncture, we preliminarily forecast that a filing of a new drug application on OK-101 to treat NCP could occur by end of 2007, with marketing approval by late 2028.  Given the chronic nature of NCP, we deem it possible that the Company and a partner could generate up to several hundred million in sales in the first full three years. Still, for valuation purposes, considering the number of future years a forecast would entail, our first full-year top-line sales estimate for 2029 is $75 million.

RISK FACTORS

In our view, the Company’s biggest risk is related to the prospective clinical trial success of OKYO’s lead candidate, OK-101. If results of the ongoing Phase II NCP clinical trial are comparable to the Company’s other data, it is likely to be deemed a success, leading to negotiations with FDA for the development of the Phase 3 trial program. However, it is always possible that data related directly to primary and secondary endpoints provide upside and downside surprises. The next major risk is related to the ability of the Company to successfully attract a joint venture partner that could help fund a Phase 3 trial via research development investments and potentially pay royalties to market an approved product in the US. In our view, given that existing therapies for DED are widely considered to be subpar, favorable top-line NCP data could attract a number of prospective partners that have historically demonstrated a model that features external investment and M&A as a primary R&D path.

Other risks could come from competing firms with drugs under development that may be migrated to an NCP trial and subsequently demonstrate greater efficacy in their clinical trials. Clearly, these competitors are not yet in the clinic, which puts OKYO presently in the lead. Nevertheless, these other potential competitors could attract partners, making the execution of favorable joint ventures potentially challenging for the Company.

The aforementioned risks could come from larger competitors, existing firms, or new entrants. Still, these future concerns are consistent with firms of OKYO’s size and standing. Moreover, we believe that OKYO’s seasoned management team is prepared to overcome these hurdles and execute its R&D and business development objectives.

Volatility and liquidity are typical concerns for small cap and microcap stocks. An overriding financial benefit as a public company is the favorable access to and the availability of capital to fund research and development, product launches, marketing campaigns and other initiatives. Since the proceeds of any future funding for OKYO would be used in large part to advance its lead drug candidate, we believe that any dilutive effect from such a funding could be offset by related, future increases in market value.

VALUATION

Given that the Company is primed to reach a key valuation-changing milestone with the release of Phase 2 data for NCP at year-end, we have elected to publish a price target that we expect could be achieved in the next 12 months. If multi-dose data and efficacy match OKYO’s primary and secondary objectives, we believe that these data could serve as a catalyst for a mid-tier – to top tier pharmaceutical firm to seek to enter into a transaction with OKYO. This transaction could be in the form of M&A or an investment and related licensing or partnership arrangement with OKYO in exchange for future R&D. We believe that given the safety profile, a compassionate care usage profile could be granted by the FDA, thereby enabling OKYO and a potential partner to benefit. Moreover, due to the nature of OK-101’s chemistry, administration, and its first-mover advantage, we strongly believe that the Company’s OK-101 (and a partner or an acquirer) could be well positioned to be the first FDA approved topical solution for the treatment of NCP.

Against this backdrop, in 1H26, we envision a potential investment for future R&D, with additional funds to be invested based on R&D and other milestones, and a potential right to acquire a majority stake in or an outright purchase of OKYO.

Our 12-month, pre-data release price target is $5.00 and is designed to reflect the potential valuation assessed following a successful Phase 2 trial for a first mover product slated to treat an unmet medical need that represents a large market opportunity. We believe that in 2026/2027, when a deal with a partner could occur, the Company would benefit from a re-valuation of these shares. This price target is affirmed by a future valuation estimate. This estimate assumes $75M in annual CY2029 sales for an FDA-approved OK-101 and an assigned 6.5x sales multiple, which represents a slight discount to the median multiple assigned to a series of ocular M&A transactions. We thus arrive at a future potential $487M valuation in an acquisition. Our 12-month price target of $5.00 reflects this figure discounted back four years at a rate of 30%, on a Net Present Value basis/

As noted above, there has been a flurry of ocular company M&A by Big Pharma that have carried outsized valuations. As outlined in Table I, the deals we described were sold for a median of 7.6x trailing twelve-month revenue.

CONCLUSION

OKYO Pharma is focused on developing OK-101 to treat NCP which presently has no FDA approved drug to treat this debilitating ocular disease. OKYO Pharma commenced its Phase 2 trial of OK-101 in October 2024 and results are expected by year-end 2025. OKYO is the first company to be granted an investigational new drug (IND) application for NCP by FDA for clinical trials in NCP patients, and the first company to launch a clinical trial in NCP patients specifically diagnosed with NCP. This trial is on the heels of a favorable DED Phase 2 trial of OK-101 which notably demonstrated statistical significance in an ocular pain secondary endpoint.

The potential size of the NCP market assuming NCP receives Orphan Drug designation is $6.4 billion. The market opportunity for the first drug to receive FDA approval to treat this major unmet medical need is based on a 160,000 US potential patient size.

We believe that given its positioning, and assuming data are favorable, OKYO will likely also attract a potential joint venture development partner or an acquirer by 2026/2027. We believe the results from this trial represent a major binary event, serving as a major catalyst for a re-valuation of the stock. Our 12-month, pre-data release price target for OKYO is $5.00. This target is based on the prospective value of OK-101, projected sales multiples discounted back four years, and the median valuation of ocular biopharma M&A deals.

RECENT TRADING HISTORY FOR OKYO

(Source: www.StockCharts.com)