What changed in this filing

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Item 1A. Risk Factors

2025-03-06 compared with 2024-03-25 · 95 added, 66 removed, 707 unchanged (19% of the section changed)

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Our consolidated financial statements as of December 31, 2024 did not include any adjustments that might result from the outcome of this uncertainty.

We expect that our current cash will be able to fund operations through the fourth quarter of 2024 and into the first quarter of 2025 but will not be sufficient to fund operations for twelve months from the date of the filing of this Annual Report.

We expect that our current cash will be able to fund operations into the third quarter of 2025 but will not be sufficient to fund operations for twelve months from the date of the filing of this Annual Report.

We will need to raise additional capital to operate our business and our failure to obtain funding when needed may force us to delay, reduce or eliminate certain of our development programs or commercialization efforts .

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As of December 31, 2024, our accumulated deficit totaled approximately $335.0 million on a consolidated basis.

Pursuant to the VCN Purchase Agreement, we have agreed to use reasonable efforts to commercialize VCN-01 and we agreed as a post- closing covenant to commit to fund VCN's research and development programs, including but not limited to VCN-01 PDAC Phase 2 clinical trial, VCN-01 RB pivotal trial and necessary G&A within a budgetary plan of approximately $27.8 million over the next three years.

We expect to incur additional operating losses in the future and therefore expect our cumulative losses to increase.

With the exception of the quarter ended June 30, 2010, and limited laboratory revenues from Adeona Clinical Laboratory, which we sold in March 2012, we have generated very minimal revenues.

We do not expect to derive revenue from any source in the near future until we or our potential partners successfully commercialize our products.

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For the foreseeable future we will have to fund all of our operations and capital expenditures from equity and debt offerings, cash on hand, licensing and collaboration fees and grants, if any.

We will need to raise additional capital to fund our operations and meet our current timelines and we cannot be certain that funding will be available on acceptable terms on a timely basis, or at all.

Based on our current plans, our cash and cash equivalents will be sufficient to complete our planned clinical trials of VCN-01 (in PDAC and retinoblastoma), Phase 1a/2a clinical trial of SYN-004, but will not be sufficient for additional trials of VCN-01, SYN-020 or SYN-004, which are expected to require significant cash expenditures.

The amount of government funding available for grants is dependent upon governmental budgets over which we have no control and which change with new administrations.

Based on our current plans, we expect that our current cash will be able to fund operations into the third quarter of 2025 but will not be sufficient to fund our operations for the next twelve months and will only be sufficient to complete our planned clinical trials of VCN-01 (in PDAC and retinoblastoma), but may not be sufficient for additional trials of VCN-01, SYN-020 or SYN-004, or to complete the last cohort of the Phase 1a/2a clinical trial of SYN-004, which are expected to require significant cash expenditures.

In addition, based on the significant anticipated cost of a Phase 3 clinical program in a broad indication for SYN-004, we expect it will not be feasible for us to initiate and complete this trial at this time without a partner given the capital constraints tied to our current market cap and share price.

As part of our strategic transformation into an oncology focused company, we are exploring value creation options for our SYN-004 and SYN-020 assets, including out-licensing or partnering.

We intend to focus our capital on our VCN-01 clinical trials and do not intend to provide further funding for our development of SYN-004 internally but intend to out-license or partner further development of SYN-004.

Further development of VCN’s product candidates will require additional funding.

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In addition, we could be forced to delay, discontinue or curtail product development, forego sales and marketing efforts, and forego licensing in attractive business opportunities.

Our ability to raise capital through the sale of securities may be limited by the rules of the SEC and NYSE American that place limits on the number and dollar amount of securities that may be sold.

Our ability to raise capital through the sale of securities may be limited by the rules of the SEC and NYSE American LLC (“NYSE American”) that place limits on the number and dollar amount of securities that may be sold.

There can be no assurances that we will be able to raise the funds needed, especially in light of the fact that our ability to sell securities registered on our registration statement on Form S-3 will be limited until such time the market value of our voting securities held by non-affiliates is $75 million or more.

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We anticipate a need for additional employees as we undertake later stage clinical trials.

We have also incurred certain obligations pursuant to the terms of the VCN Purchase Agreement including the assumption of $2.4 million of liabilities and have agreed to a post-closing covenant to commit to fund research and development of VCN-01 and OV pipeline programs, including but not limited to the VCN-01 PDAC Phase 2 trial, a VCN-01 RB pivotal trial and necessary G&A within a budgetary plan of approximately $27.8 million.

Further development of VCN-01 and pipeline OV product candidates will require additional expenditures.

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If we raise funds through collaborations and licensing arrangements, we might be required to relinquish significant rights to our technologies or tests or grant licenses on terms that are not favorable to us.

The amount of government funding available for grants is dependent upon governmental budgets over which we have no control and which change with new administrations.

The actual amount of funds we will need to operate is subject to many risk factors, some of which are beyond our control.

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If we cannot successfully execute on any of the factors listed above, our business may not succeed, we may never generate revenue and your investment will be adversely affected.

We have identified material weaknesses in our internal controls, and we cannot provide assurances that these weaknesses will be effectively remediated or that additional material weaknesses will not occur in the future

We have identified material weaknesses in our internal controls in the past, and we cannot provide assurances that additional material weaknesses will not occur in the future

If our internal control over financial reporting or our disclosure controls and procedures are not effective, we may not be able to accurately report our financial results, prevent fraud, or file our periodic reports in a timely manner, which may cause investors to lose confidence in our reported financial information and may lead to a decline in our stock price.

Our management is responsible for establishing and maintaining adequate internal control over our financial reporting, as defined in Rule 13a- 15(f) under the Exchange Act.

Based on our assessment, we have concluded that we did not maintain effective review controls at a sufficient level of precision with certain financial statement areas and over unusual transactions involving complex accounting and related.disclosure requirements.

We also did not maintain effective information technology general controls over user access, program change management, and segregation of duties, within certain key information systems supporting the Company’s accounting and financial reporting processes.

Based on our assessment, we have concluded that as of December 31, 2024 we have remediated our prior weakness over internal controls.

During 2023 and the first three quarters of 2024, we did not maintain effective review controls at a sufficient level of precision with certain financial statement areas and over unusual transactions involving complex accounting and related disclosure requirements.

Additionally, many of our business process controls dependent upon the information derived from these information systems were also ineffective, as we did not design and implement controls to validate the completeness and accuracy of underlying data utilized in the operation of those controls.

While we plan to take remedial action to address the material weaknesses, we cannot provide any assurance that such remedial measures, or any other remedial measures we take, will be effective.

While we have taken remedial action to address the material weaknesses and we now believe that our internal control over financial reporting is effective, we cannot provide any assurance that such remedial measures, or any other remedial measures we take, will continue to be effective.

If we fail to maintain effective internal control over financial reporting, we may not be able to accurately report our financial results, detect or prevent fraud, or file our periodic reports in a timely manner, which may, among other adverse consequences, cause investors to lose confidence in our reported financial information and lead to a decline in our stock price.

In addition, a material weakness will not be considered remediated until the applicable controls operate for a sufficient period of time and management has concluded, through testing, that these controls are designed and operating effectively.

Although management believes that the material weaknesses will be remediated by the end of the fiscal year there can be no assurance that the deficiencies will be remediated at such time or that the internal control over financial reporting, as modified, will enable us to identify or avoid material weaknesses in the future.

Although management believes that the material weaknesses have been remediated there can be no assurance that our internal control over financial reporting, as modified, will enable us to identify or avoid material weaknesses in the future.

We expect to seek to raise additional capital in the future, which may be dilutive to stockholders or impose operational restrictions.

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We may also enter into strategic transactions, issue equity as consideration for acquisitions or part of license issue fees to our licensors, compensate consultants or settle outstanding payables using equity that may be dilutive.

We are authorized to issue 350,000,000 shares of common stock, of which 17,148,049 shares of common stock were outstanding as of December 31, 2023.

At December 31, 2023, we had reserved 6,834,797 shares of common stock for issuance upon exercise of our outstanding options, and preferred shares.

In addition, at such date, we had 2,822,845 shares of our common stock reserved for future issuance under our equity incentive plans.

If all of these securities were to be exercised, the total number of shares of our common stock that we would be required to issue is 9,657,642, which in addition to the 17,148,049 shares outstanding, would leave 323,194,309 authorized but unissued shares of common stock available to be issued.

We are authorized to issue 350,000,000 shares of Common Stock, of which 2,782,449 shares of Common Stock were outstanding as of March 4, 2025.

If all of the unissued authorized shares were issued stockholders ownership percentage will be diluted.

In order to raise additional capital, we may in the future offer additional shares of our Common Stock or other securities convertible into or exchangeable for our Common Stock at prices that may not be the same as the price per share paid by existing stockholders, thereby subjecting such stockholders to dilution.

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Such a stock price decline could occur even when we have met any previously publicly stated revenue and/or earnings guidance we may provide.

If our acquired intangible assets become impaired, we may be required to record a significant charge to earnings.

If our acquired intangible assets become impaired in the future as was the case in 2024, we may be required to record a significant charge to earnings.

We regularly review acquired intangible assets for impairment when events or changes in circumstances indicate that the carrying value may not be recoverable.

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RISKS RELATED TO OUR BUSINESS

Prior to 2022 we had not conducted any cancer research and development activities and there can be no assurance that we will successfully be able to do so.

Prior to 2022 we had not conducted any research and development activities directed to cancer diagnosis, treatment or prevention and there can be no assurance that we will successfully be able to do so.

Prior to the VCN Acquisition, our focus was on the microbiome and our research and development was focused primarily on therapeutics for various microbiome related diseases.

Upon the VCN Acquisition, our focus has shifted to the use of oncolytic viruses to treat cancer.

Although, our members of management and scientific/development teams have experience in this field, we may not be successful as a company with such focus.

Although, members of our management and scientific/development teams have experience in the research and development of cancer treatments, we may not be successful as a company with such focus.

In the past Oncolytic Viruses have experienced certain safety and efficacy challenges.

Although current clinical trials of oncolytic virotherapies have supported their role as a potential treatment for cancer, there is the risk of virus-related toxicities in vivo and possible transmission to patients' contacts, such as other patients and health care workers.

Although current clinical trials of OVs have supported their role as a potential treatment for cancer, there is the risk of virus-related toxicities in vivo and possible transmission to patients' contacts, such as other patients and health care workers.

In recent years, clinical trials to address these concerns have been conducted.

Any such transmission by VCN-01 or a competitor would have an adverse impact on our future OV research and development efforts.

Likewise, a number of oncolytic virotherapies have previously failed to meet their primary endpoints in advanced clinical trials, potentially reducing investor and partner interest or confidence in the development of new such therapies, however well differentiated they are from previous products.

Likewise, a number of OVs have previously failed to meet their primary endpoints in advanced clinical trials, potentially reducing investor and partner interest or confidence in the development of new such therapies, however well differentiated they are from previous products.

Our research and development efforts may not result in commercially successful products and technologies, which may limit our ability to achieve profitability.

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In addition, we face significant competition in seeking appropriate strategic partners and the negotiation process is time-consuming and complex.

Moreover, we may not be successful in our efforts to establish a strategic partnership or other alternative arrangements for our product candidates because they may be deemed to be at too early of a stage of development for collaborative effort and third parties may not view our product candidates as having the requisite potential to demonstrate safety and efficacy.

Moreover, we may not be successful in our efforts to establish a strategic partnership or other alternative arrangements for our product candidates because they may be deemed to be at too early a stage of development for collaborative effort and third parties may not view our product candidates as having the requisite potential to demonstrate safety and efficacy.

If we license products or businesses, we may not be able to realize the benefit of such transactions if we are unable to successfully integrate them with our existing operations and company culture.

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The “IDIBELL/ICO License Agreement provides that the licensors have the right to revoke the IDIBELL/ICO License Agreement if VCN during a continuous period of two years abandons its research or development activities of the licensed patent or activities aimed at exploitation of the resulting products.

We entered into an option agreement with MGH to enter into an exclusive license to intellectual property and technology related to the use of IAP to maintain GI and microbiome health, diminish systemic inflammation, and treat age-related diseases.

There can be no assurance that we will be able to reach agreement on license terms or that the terms will be favorable to us.

This license agreement is expected to require us to meet certain diligence requirements and timelines in order to keep the license agreement in effect.

In addition, certain license agreements, including the one that may potentially be entered into with MGH, typically contain provisions requiring royalty free non-exclusive licenses to the U.S government if any federal funding was used to invent any of the patents being licensed.

In the event we or our sublicensee are not able to meet our diligence requirements contained in the license agreement with MGH or any other license agreement, we may not be able to retain the rights granted under our agreement or renegotiate with our arrangement institution on reasonable terms, or at all.

If any license were to terminate and we were to lose the right to commercialize our products, our business opportunity would be adversely affected.

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For us to research, develop, and test our product candidates, we would need to contract with outside researchers, in most cases those parties that did the original research and from whom we have licensed the technologies.

Our agreement with UT Austin allows UT Austin to terminate its agreement if we fail to comply with the terms of the agreement.

We can give no assurances that any of our issued patents licensed to us or any of our other patent applications will provide us with significant proprietary protection or be of commercial benefit to us.

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engage in certain research and development activities that require additional expenditures.

Our agreements with Washington University and MGH may require that we initiate certain studies and file or have accepted an NDA within a certain amount of time, each of which are costly and will require additional expenditures.

Our agreement with Washington University may require that we initiate certain studies and file or have accepted an NDA within a certain amount of time, each of which are costly and will require additional expenditures.

Although all manufacturing, preclinical studies and human clinical trials are expensive and difficult to design and implement, costs associated with the manufacturing, research and development of biologic product candidates are generally greater in comparison to small molecule product candidates.

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Should clinicians or regulatory authorities view alternative therapeutic regiments as more effective than our products, this might delay or prevent us from obtaining regulatory approval for our products, or it might prevent us from obtaining favorable reimbursement rates from payers, such as Medicare, Medicaid, hospitals and private insurers.

Not only do our product candidates compete with other product candidates being developed for similar or the same indications, we also compete for employees and for clinical trial sites.

Not only do our product candidates compete with other product candidates being developed for similar or the same indications, we also compete for employees, clinical trial sites, and participants in clinical trials.

We may seek to selectively establish collaborations, and, if we are unable to establish them on commercially reasonable terms, we may have to alter our development and commercialization plans.

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Although we believe additional manufacturers are available, if any of our manufacturers were to limit or terminate production or otherwise fail to meet the quality or delivery requirements needed to satisfy the supply commitments, the process of locating and qualifying alternate sources could require up to several months, during which time our production could be delayed.

Any curtailment in the availability of VCN-01, SYN-004 (ribaxamase) or SYN-020 could have a material adverse effect on our business, financial position and results of operations.

Any curtailment in the availability of our product candidates could have a material adverse effect on our business, financial position and results of operations.

In addition, because regulatory authorities must generally approve raw material sources for pharmaceutical products, changes in raw material suppliers may result in production delays or higher raw material costs.

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Furthermore, during the COVID-19 pandemic, many manufacturers prioritized the manufacture of COVID-19 related products, increasing the manufacturing lead times for non-COVID-19 related products.

If a pandemic should occur again and manufacturers prioritized the manufacture of pandemic related products, we may suffer a delay or interruption in the supply of clinical trial supplies.In addition, any could delay the completion of our clinical trials, increase the costs associated with conducting our clinical trials and, depending upon the period of delay, require us to commence new clinical trials at significant additional expense or to terminate a clinical trial.

If a pandemic should occur again and manufacturers prioritized the manufacture of pandemic related products, we may suffer a delay or interruption in the supply of clinical trial supplies.

In addition, any future pandemic or other disruption to clinical trial participation or the production of clinical trial materials could delay the completion of our clinical trials, increase the costs associated with conducting our clinical trials and, depending upon the period of delay, require us to commence new clinical trials at significant additional expense or to terminate a clinical trial.

We are responsible for ensuring that each of our contract manufacturers comply with the cGMP requirements of the FDA and other regulatory authorities from which we seek to obtain product approval.

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Any problems obtaining the standard of care drugs could result in a delay or interruption in our clinical trials.

For our planned Phase 2 clinical trial of VCN-01 in patients with PDAC, we are administering VCN-01 in combination with the already approved standard of care drugs, gemcitabine/nab-paclitaxel, for which there has recently been a supply shortage.

For our ongoing Phase 2 clinical trial of VCN-01 in patients with PDAC, we are administering VCN-01 in combination with the already approved standard of care drugs, gemcitabine/nab-paclitaxel, for which there has recently been a supply shortage.

Therefore, our success will be dependent upon the continued use of and ability to obtain the standard of care drugs.

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We have limited resources dedicated to designing, conducting and managing our preclinical studies and clinical trials.

We rely on, third parties, including CROs, consultants and principal investigators, to assist us in designing, managing, conducting, monitoring and analyzing the data from our preclinical studies and clinical trials.

We rely on, third parties, including clinical research organizations (CROs), consultants and principal investigators, to assist us in designing, managing, conducting, monitoring and analyzing the data from our preclinical studies and clinical trials.

We rely on these vendors and individuals to perform many facets of the clinical development process on our behalf, including conducting preclinical studies, the recruitment of sites and subjects for participation in our clinical trials, maintenance of good relations with the clinical sites, and ensuring that these sites are conducting our trials in compliance with the trial protocol and applicable regulations.

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We may fail to retain or recruit necessary personnel, and we may be unable to secure the services of consultants.

As of March 30, 2024, we employed 21 full-time employees, including employees located at Theriva Biologics’ offices in Barcelona, Spain.

As of March 6, 2025, we employed 20 full-time employees and 2 part-time employees, including employees located at Theriva Biologics’ offices in Barcelona, Spain.

We have also engaged clinical consultants to advise us on our clinical programs and regulatory consultants to advise us on our dealings with the FDA and other foreign regulatory authorities.

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Global health crises may adversely affect our planned operations.

Our business and the business of the supplier of our clinical product candidates and the suppliers of the standard of care drugs that are administered in combination with our product candidates could be materially and adversely affected by the risks, or the public perception of the risks, related to a pandemic or other health crisis, such as the recent outbreak of COVID-19.

We have experienced delays in patient enrollment due to the COVID-19 pandemic.

To date, we are on track to meet all of our previously announced future clinical milestones;

however, if the COVID-19 pandemic increases in severity or we should experience another pandemic, we could once again experience delays in patient enrollment and experience significant disruptions to our clinical development timelines.

We experienced delays in patient enrollment due to the COVID-19 pandemic.

If we should experience another pandemic, we could once again experience delays in patient enrollment and experience significant disruptions to our clinical development timelines.

If we experience delays in patient enrollment or patients drop outs and we deem it necessary or advisable to improve patient recruitment by, among other things, opening additional clinical sites, we could incur increased clinical program expenses.

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● interruption in global shipping that may affect the manufacture and transport of clinical trial materials, such as investigational drug product used in our clinical trials;

● changes in local regulations as part of a response to the COVID-19 coronavirus outbreak which may require us to change the ways in which our clinical trials are conducted, which may result in unexpected costs, or to discontinue the clinical trials altogether;

● changes in local regulations as part of a response to the a pandemic outbreak which may require us to change the ways in which our clinical trials are conducted, which may result in unexpected costs, or to discontinue the clinical trials altogether;

● delays in necessary interactions with local regulators, ethics committees and other important agencies and contractors due to limitations in employee resources or forced furlough of government employees;

● delay in the timing of interactions with the FDA and other regulatory agencies due to absenteeism by employees or by the diversion of their efforts and attention to approval of other therapeutics or other activities related to COVID-19.

In addition, a significant outbreak of contagious diseases in the human population could result in the complete or partial closure of one or more manufacturing facilities which could impact our supply of our product candidates or the standard of care drugs that are administered in combination with our product candidates.

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Pandemics could also impact members of our Board of Directors resulting in absenteeism from meetings of the directors or committees of directors, and making it more difficult to convene the quorums of the full Board of Directors or its committees needed to conduct meetings for the management of our affairs.

The extent to which the virus may continue to impact our business and clinical trials will depend on future developments, which are highly uncertain and cannot be predicted with confidence, such as the ultimate geographic spread of the disease, the duration of the outbreak, travel restrictions and social distancing in the United States and Spain, business closures or business disruptions and the effectiveness of actions taken in the United States, Spain, and other countries to contain and treat the disease.

The extent to which a pandemic may impact our business and clinical trials will depend on future developments, which are highly uncertain and cannot be predicted with confidence, such as the ultimate geographic spread of the disease, the duration of the outbreak, travel restrictions and social distancing in the United States and Spain, business closures or business disruptions and the effectiveness of actions taken in the United States, Spain, and other countries to contain and treat the disease.

We do not yet know the full extent of potential delays or impacts on our business, operations, or the global economy as a whole.

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Business disruptions could seriously harm our future revenue and financial condition and increase costs and expenses.

Our operations and those of our third-party suppliers and collaborators could be subject to earthquakes, power shortages, telecommunications failures, water shortages, floods, hurricanes or other extreme weather conditions, medical epidemics, labor disputes, war or other business interruptions.

Any interruption could seriously harm our ability to timely proceed with any clinical programs or to supply product candidates for use in our clinical programs or during commercialization.

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Additionally, supply chain disruptions impacted and may continue to impact our research activities.

Moreover, at the end of 2021 and into 2022, tensions between the United States and Russia escalated when Russia amassed large numbers of military ground forces and support personnel on the Ukraine-Russia border and, in February 2022, Russia invaded Ukraine.

In response, North Atlantic Treaty Organization, or NATO has deployed additional military forces to Eastern Europe and the Biden administration announced certain sanctions against Russia.

The invasion of Ukraine and the retaliatory measures that have been taken, or could be taken in the future, by the United States, NATO, and other countries have created global security concerns that could result in a regional conflict and otherwise have a lasting impact on regional and global economies, any or all of which could disrupt our supply chain, and despite the fact that we currently do not plan any clinical trials in Eastern Europe, may adversely impact the cost and conduct of our international clinical trials of our product candidates.

Continuing regional conflicts in Eastern Europe and the Middle East have created global security concerns that could have a lasting impact on regional and global economies, any or all of which could disrupt our supply chain, and despite the fact that we currently do not plan any clinical trials in Eastern Europe or the Middle East, may adversely impact the cost and conduct of our international clinical trials of our product candidates.

Unfavorable U.S.

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A severe or prolonged economic downturn could result in a variety of risks to our business, including weakened demand for our technologies and our ability to raise additional capital when needed on favorable terms, if at all.

Recently, the rate of inflation has increased throughout the U.S.

Inflation may adversely affect us by increasing the costs associated with performing research and development on internal research initiatives and partnered programs.

Ongoing inflation in world economies may adversely affect us by increasing the costs associated with performing research and development on internal research initiatives and partnered programs.

We may experience significant increases in the prices of labor, consumables, and other costs of doing business.

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Any of the foregoing could harm our business and we cannot anticipate all of the ways in which the current economic climate and financial market conditions could adversely impact our business.

In addition, the global macroeconomic environment could be negatively affected by, among other things, COVID-19 or other pandemics or epidemics, instability in global economic markets, increased U.S.

trade tariffs and trade disputes with other countries, instability in the global credit markets, supply chain weaknesses, instability in the geopolitical environment as a result of the withdrawal of the United Kingdom from the European Union, the Russian invasion of Ukraine, the war in the Middle East and other political tensions, and foreign governmental debt concerns.

In addition, the global macroeconomic environment could be negatively affected by, among other things, pandemics or epidemics, instability in global economic markets, instability in the global credit markets, supply chain weaknesses, instability in the geopolitical environment as a result of the withdrawal of the United Kingdom from the European Union, the Russian war with Ukraine, the war in the Middle East and other political tensions, and foreign governmental debt concerns.

Such challenges have caused, and may continue to cause, uncertainty and instability in local economies and in global financial markets.

Changes in U.S.

or international social, political, regulatory and economic conditions or in laws and policies governing trade, manufacturing, development and investment in the countries where we currently sell our products or conduct our business, could adversely affect our business, reputation, financial condition and results of operations.

Changes or proposed changes in U.S.

or other countries’ trade policies may result in restrictions and economic disincentives on international trade.

Tariffs, economic sanctions and other changes in U.S.

trade policy have in the past and could in the future trigger retaliatory actions by affected countries, and certain foreign governments have instituted or are considering imposing retaliatory measures on certain U.S.

Further, any emerging protectionist or nationalist trends (whether regulatory- or consumer-driven) either in the United States or in other countries could affect the trade environment.

We, like many other multinational corporations, conduct a significant amount of business that would be impacted by changes to the trade policies of the United States and foreign countries (including governmental action related to tariffs, international trade agreements, or economic sanctions).

Such changes have the potential to adversely impact the U.S.

economy or certain sectors thereof or the economy of another country in which we conduct operations, our industry and the global demand for our products, and as a result, could have a material adverse effect on our business, financial condition and results of operations.

We rely extensively on our information technology systems, and our systems and infrastructure face certain risks, including cybersecurity and data leakage risks.

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Commencement and completion of clinical trials may be delayed by several factors, including:

● obtaining an IND application with the FDA or foreign equivalent to commence clinical trials;

● obtaining permission to proceed under an IND application with the FDA or foreign equivalent to commence clinical trials;

● identification of, and acceptable arrangements with, one or more clinical sites;

● obtaining IRB or EC approval to commence clinical trials;

● obtaining IRB or IEC approval to commence clinical trials;

● obtaining IBC approval for use of a genetically modified organism;

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● inability or unwillingness of medical investigators to follow our clinical protocols;

● unwillingness of the FDA or foreign equivalent, IRBs/ECs, or IBCs to permit the clinical trials to be initiated.

In addition, we, IRBs/ECs or the FDA or foreign equivalent may suspend our clinical trials at any time if it appears that we are exposing participants to unacceptable health risks or if IRBs/ECs or the FDA or foreign equivalent finds deficiencies in our submissions or conduct of our trials.

● unwillingness of the FDA or foreign equivalent, IRBs/IECs, or IBCs to permit the clinical trials to be initiated.

In addition, we, IRBs/IECs or the FDA or foreign equivalent may suspend our clinical trials at any time if it appears that we are exposing participants to unacceptable health risks or if IRBs/IECs or the FDA or foreign equivalent finds deficiencies in our submissions or conduct of our trials.

The results of our clinical trials may not support our product candidate claims and the results of preclinical studies and completed clinical trials are not necessarily predictive of future results.

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Under the Orphan Drug Act of 1983, the (“Orphan Drug Act”), the FDA may designate a product as an orphan drug if it is a drug intended to treat a rare disease or condition, which is defined as a patient population of fewer than 200,000 individuals in the United States.

We received orphan drug designation from the FDA for VCN-01 for the treatment of retinoblastoma and from the EMA for the treatment of pancreatic cancer.

If a product with an orphan drug designation subsequently receives the first marketing approval for the indication for which it has such designation, the product is entitled to a seven-year period of marketing exclusivity, which precludes the FDA from approving another marketing application for the same drug for the same indication during that time period with some exceptions.

We have received orphan drug designation from both the FDA and EMA for VCN-01 for the treatment of retinoblastoma and for the treatment of pancreatic cancer.

If a product with an orphan drug designation subsequently receives the first marketing approval for the indication for which it has such designation, the product is entitled to a seven-year period of marketing exclusivity in the United States, which precludes the FDA from approving another marketing application for the same drug for the same indication during that time period with some exceptions.

A similar provision in the European Union allows 10 years of exclusivity in Europe.

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Orphan drug exclusivity may be lost in Europe under certain situations, such as the inability of the holder of the orphan drug designation to produce sufficient quantities of the drug to meet the needs of patients with the rare disease or condition or for certain other reasons.

Although we have been granted orphan drug designation for VCN-01 for the treatment of retinoblastoma and pancreatic cancer, this does not mean FDA will approve the NDA.

Even if we obtain FDA approval, we may not be able to obtain or maintain orphan drug exclusivity for VCN-01.

We may not be the first to obtain marketing approval of VCN-01 designation for the orphan-designated indication due to the uncertainties associated with developing pharmaceutical products.

In addition, exclusive marketing rights in the United States may be limited if we seek approval for an indication broader than the orphan-designated indication or may be lost if the FDA later determines that the request for designation was materially defective or if we are unable to assure sufficient quantities of the product to meet the needs of patients with the rare disease or condition.

Further, even if we obtain orphan drug exclusivity for a product, that exclusivity may not effectively protect the product from competition because different drugs with different active moieties may be approved for the same condition, or the competitive product is otherwise outside the scope of exclusivity.

Even after an orphan drug is approved, the FDA can subsequently approve the same drug with the same active moiety for the same condition if the FDA concludes that the later drug is clinically superior in that it is shown to be safer, more effective or makes a major contribution to patient care or the manufacturer of the product with orphan exclusivity is unable to maintain sufficient product quantity.

Orphan drug designation neither shortens the development time or regulatory review time of a drug nor gives the drug any advantage in the regulatory review or approval process, nor does it prevent competitors from obtaining approval of the same product candidate for indications other than those in which orphan drug designation have been granted.

Fast Track designation by the FDA may not actually lead to a faster development or regulatory review or approval process, and does not assure FDA approval of our product candidate and, even if we obtain FDA approval, we may not receive marketing approval, marketing exclusivity or other expected benefits.

In May 2024, the FDA granted Fast Track designation to VCN-01 for the treatment of pancreatic cancer.

However, the receipt of such a designation for a product candidate may not result in a faster development process, review or approval compared to drugs considered for approval under conventional regulatory procedures and does not assure that the product will ultimately be approved by the regulatory authority or that approval will be granted within any particular timeframe.

As a result, while we have received Fast Track designation for VCN-01 for the treatment of pancreatic cancer, we may not experience a faster development process, review or approval compared to conventional FDA procedures.

In addition, the FDA may withdraw Fast Track designation if it believes that the designation is no longer supported by data from our clinical development program.

Fast Track designation alone does not guarantee qualification for the FDA’s priority review procedures.

Although we have obtained rare pediatric disease designation for VCN-01 for the treatment of retinoblastoma, we may not be eligible to receive a priority review voucher in the event the FDA determines we no longer meet the criteria for designation, revokes the designation or FDA approval of a BLA for VCN-01 for retinoblastoma does not occur prior to September 30, 2026.

The FDA grants rare pediatric disease designation for rare diseases (fewer than 200,000 affected persons in the United States) that are serious and life-threatening and primarily affect children ages 18 years or younger.

The sponsor of an application for a rare pediatric disease drug product may be eligible for a voucher that can be used or sold to obtain a priority review for a subsequent application submitted under section 505(b)(1) of the FDCA or section 351 of the PHS Act.

The rare pediatric disease priority review voucher program was most recently reauthorized by Congress through December 20, 2024, with the potential for priority review vouchers to be granted through September 30, 2026.

We received rare pediatric disease designation from the FDA for VCN-01 on July 30, 2024.

Vouchers for rare pediatric disease drugs are awarded for qualifying applications when the drug receives approval.

Although VCN-01 has received rare pediatric disease designation for the treatment of retinoblastoma, VCN-01 may not receive a priority review voucher for a number of reasons:

VCN-01 may not receive approval for retinoblastoma prior to September 30, 2026;

VCN-01 may receive approval in adults, but not pediatric patients;

VCN-01 may not meet the eligibility requirements for a priority voucher at the time we seek approval for VCN-01.

Finally, a rare pediatric disease designation does not necessarily lead to faster development or regulatory review of the product or increase the likelihood that it will receive marketing approval.

The failure to maintain rare pediatric disease designation for VCN-01 or if FDA approval does not occur prior to September 30, 2026 could result in the inability to receive a priority review voucher which could adversely affect our business, financial condition and results of operations.

Our product candidates, if approved for sale, may not gain acceptance among physicians, patients and the medical community, thereby limiting our potential to generate revenues.

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● the availability of adequate third-party insurance coverage or reimbursement.

If any product candidate that we develop does not provide a treatment regimen that is as beneficial as, or is perceived as being as beneficial as, the current standard of care or otherwise does not provide patient benefit, that product candidate, if approved for commercial sale by the FDA or other regulatory authorities, likely will not achieve market acceptance.

If any product candidate that we develop is not as beneficial as, or is perceived as not being as beneficial as, the current standard of care or otherwise does not provide patient benefit, that product candidate, if approved for commercial sale by the FDA or other regulatory authorities, likely will not achieve market acceptance.

Our ability to effectively promote and sell any approved products will also depend on pricing and cost-effectiveness, including our ability to produce a product at a competitive price and our ability to obtain sufficient third-party coverage or reimbursement.

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We will have to compete with other pharmaceutical and biotechnology companies to recruit, hire, train and retain marketing and sales personnel.

If we are unable or decide not to establish internal sales, marketing and distribution capabilities, we will pursue collaborative arrangements regarding the sales and marketing of our products;

If we are unable to or decide not to, establish internal sales, marketing and distribution capabilities, we will pursue collaborative arrangements regarding the sales and marketing of our products;

however, there can be no assurance that we will be able to establish or maintain such collaborative arrangements.

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We cannot assure you that we will be able to maintain the continued listing standards of the NYSE American.

The NYSE American requires companies to meet certain continued listing criteria including a minimum stockholders’ equity of $6.0 million if an issuer has sustained losses from continuing operations and/or net losses in its five most recent years, as outlined in the NYSE American Company Guide.

The NYSE American Company Guide also states that the NYSE normally will not consider removing from listing securities of an issuer if it is in compliance with all of the following:a total value of market capitalization of at least $50.0 million;

The NYSE American Company Guide also states that the NYSE normally will not consider removing from listing securities of an issuer if it is in compliance with all of the following:

a total value of market capitalization of at least $50.0 million;

1,100,000 publicly-held shares;

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and 400 round lot shareholders.

If our common stock falls below $0.20 per share on a 30-trading-day average it will become subject to the continued listing evaluation and follow-up procedures set forth in Section 1009 of the NYSE American Company Guide which could, among other things, result in initiation of immediate delisting procedures.

In the event that we were to fail to meet the requirements of NYSE American per share price requirement or stockholders’ equity requirement and we could not timely cure such deficiency, our listing could become subject to NYSE American continued listing evaluation and follow-up procedures, which could result in delisting procedures.

In addition, the NYSE American has informed us that it can commence delisting proceedings and immediately suspend trading in the event that our Common Stock trades at levels viewed to be abnormally low and no longer suitable for listing pursuant to Section 1003(f)(v) of the NYSE American Company Guide.

Generally the NYSE American views trading at or below a price of $0.10 to be abnormally low.

New reverse stock split rules implemented by the NYSE American in January 2025 limit the circumstances under which reverse stock splits can be used in order to cure low trading price deficiencies, including the immediate suspension and delisting of any company that has effected one or more reverse stock splits over the prior two year period with a cumulative ratio of 200 shares or more to one.

Based on these rules, due to the reverse stock split effected in August 2024, we would be limited in effecting a reverse stock split to cure a low price deficiency.

As stated above, in the event that we were to fail to meet the requirements of NYSE American per share price requirement the NYSE American could commence delisting proceedings and immediately suspend trading of our Common Stock o the NYSE American or if we fail to meet other requirements such as the stockholders’ equity requirement and we could not timely cure such deficiency, our listing could become subject to NYSE American continued listing evaluation and follow-up procedures, which could result in delisting procedures.

We previously received notification from the NYSE American citing failure to comply with the minimum stockholders’ equity continued listing standard as set forth in Part 10, Section 1003 of the Company Guide.

Although in the past we have been able to cure previously cited deficiencies, there can be no assurance that we will continue to meet the NYSE American continued listing requirements.

In addition, in the future we may not be able to maintain minimum stockholders’ equity and/or issue additional equity securities in exchange for cash or other assets, if available, to maintain certain minimum stockholders’ equity required by the NYSE American.

In addition, in the future we may not be able to ensure that our Common Stock trades at levels not viewed to be abnormally low and no longer suitable for listing or maintain minimum stockholders’ equity and/or issue additional equity securities in exchange for cash or other assets, if available, to maintain certain minimum stockholders’ equity required by the NYSE American.

If we are delisted from the NYSE American then our Common Stock will trade, if at all, only on the over-the-counter market, such as the OTC Bulletin Board securities market, and then only if one or more registered broker-dealer market makers comply with quotation requirements.

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Our stock price has fluctuated in the past, has recently been volatile and may be volatile in the future.

By way of example, on February 14, 2023, the price of our common stock closed at $1.13 per share while on November 14, 2023, our stock price closed at $0.39 per share with no discernable announcements or developments by the company or third parties.

By way of example, on March 19, 2024, the price of our common stock closed at $11.00 per share while on March 20, 2024, our stock price closed at $15.50 per share with no discernable announcements or developments by the company or third parties.

We may incur rapid and substantial decreases in our stock price in the foreseeable future that are unrelated to our operating performance or prospects.

In addition, the outbreak of the novel strain of coronavirus (COVID-19) has caused broad stock market and industry fluctuations.

The stock market in general and the market for biotechnology and pharmaceutical companies in particular have experienced extreme volatility that has often been unrelated to the operating performance of particular companies.

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● general economic, industry and market conditions;

● other events or factors, including those resulting from such events, or the prospect of such events, including war, terrorism and other international conflicts, such as the recent Russian invasion of Ukraine as well as continued and new sanctions against Russia, which restrict a wide range of trade and financial dealings with Russia and Russian persons, public health issues including health epidemics or pandemics, such as the outbreak of the novel coronavirus (COVID-19), and natural disasters such as fire, hurricanes, earthquakes, tornados or other adverse weather and climate conditions, whether occurring in the United States or elsewhere, could disrupt our operations, disrupt the operations of our suppliers or result in political or economic instability.

● other events or factors, including those resulting from such events, or the prospect of such events, including war, terrorism and other international conflicts, which restrict a wide range of trade and financial dealings, public health issues including health epidemics or pandemics, such as the outbreak of the novel coronavirus (COVID-19), and natural disasters such as fire, hurricanes, earthquakes, tornados or other adverse weather and climate conditions, whether occurring in the United States or elsewhere, could disrupt our operations, disrupt the operations of our suppliers or result in political or economic instability.

These broad market and industry factors may seriously harm the market price of our common stock, regardless of our operating performance.

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Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 1A. Risk Factors