Item 1A. Risk Factors.
In addition to the other information contained in this Form 10-K, the following risk factors should be considered carefully in evaluating our company’s business. Our business, financial condition or results of operations could be materially and adversely affected by any of these risks. Additional risks not presently known to us or that we currently deem immaterial may also adversely affect our business, financial condition or results of operations.
Risk Factor Summary
Our business is subject to certain risks. The risks described under the heading “Risk Factors” immediately following this summary may have an adverse effect on our business, cash flows, financial condition and results of operations or may cause us to be unable to successfully execute all or part of our strategy. Below are the principal factors that make our business speculative or risky:
Risks Related to Our Financial Position and Need for Additional Capital
We are a clinical stage biopharmaceutical company and have not generated any revenue to date from drug sales, and may never become profitable.
We have incurred significant operating losses in recent periods and anticipate that we will incur continued losses for the foreseeable future.
If we are unable to raise capital when needed or on attractive terms, we would be forced to delay, scale back or discontinue some of our product candidate development programs or commercialization efforts.
Our independent registered public accounting firm has expressed substantial doubt about our ability to continue as a going concern.
Our ability to utilize our net operating loss carryforwards and certain other tax attributes may be limited.
Risks Related to Drug Development and Regulatory Approval
We depend heavily on the success of our core product candidates, OST-HER2 and OST-tADC. We may not be able to obtain regulatory approval for, or successfully commercialize, any of our current or future product candidates.
Delays or difficulties in enrolling patients in clinical trials could delay regulatory approval and increase development costs.
If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals for both our current or future product candidates, we will not be able to commercialize, or will be delayed in commercializing, our current or future product candidates, and our ability to generate revenue will be materially impaired.
Our current or future product candidates may cause adverse or other undesirable side effects that could delay or prevent their regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following marketing approval, if any.
We may not be able to obtain or maintain orphan drug designation or exclusivity for any product candidates and, even if we do, that exclusivity may not prevent the FDA, EMA or other regulatory authorities from approving other competing products.
Even if we receive regulatory approval for any of our current or future product candidates, we will be subject to ongoing obligations and continued regulatory review, which may result in significant additional expense. Additionally, our current or future product candidates, if approved, could be subject to labeling and other restrictions and market withdrawal, and we may be subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our drugs.
Manufacturing our current or future product candidates is complex and we may encounter difficulties in production. If we encounter such difficulties, our ability to provide supply of our current or future product candidates for preclinical studies and clinical trials or for commercial purposes could be delayed or stopped.
Our future growth may depend, in part, on our ability to penetrate foreign markets, where we would be subject to additional regulatory burdens and other risks and uncertainties that could materially adversely affect our business.
Risks Related to Intellectual Property
If we or those from whom we in-license patents are unable to obtain and maintain patent and other intellectual property protection for our technology and product candidates or if the scope of the intellectual property protection obtained is not sufficiently broad, our competitors could develop and commercialize technology and drugs similar or identical to ours, and our ability to successfully commercialize our technology and drugs may be impaired.
If our trademarks and trade names for our products or company name are not adequately protected in one or more countries where we intend to market our products, we may delay the launch of product brand names, use different trademarks or tradenames in different countries, or face other potentially adverse consequences to building our product brand recognition.
If we are unable to adequately protect and enforce our trade secrets, our business and competitive position would be harmed.
We may initiate, become a defendant in, or otherwise become party to lawsuits to protect or enforce our intellectual property rights, which could be expensive, time-consuming and unsuccessful.
We may not obtain or grant licenses or sublicenses to intellectual property rights in all markets on equally or sufficiently favorable terms with third parties.
If we fail to comply with our obligations in any agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
Any owned, co-owned or in-licensed patent covering our current or future product candidates or other valuable technology could be narrowed or found invalid or unenforceable if challenged in court or before administrative bodies in the U.S. or abroad, including the USPTO and the EPO.
Risks Related to Management and Our Operations
In our industry in particular, our future success depends on our ability to retain key scientific employees and to attract, retain and motivate qualified personnel.
Our internal computer systems, or those of our third-party CROs or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our current or future product candidates’ development programs.
We will incur increased costs as a result of operating as a public company, and our management will be required to devote substantial time to new compliance initiatives.
Risks Related to Our Financial Position and Need for Additional Capital
We are a clinical stage biopharmaceutical company and have not generated any revenue to date from drug sales, and may never become profitable.
Our ability to achieve profitability depends on our ability to generate revenue from product sales. To date, while we have generated interest in potential research collaborations, we have not generated any commercial revenue from our product candidates, including our lead product candidate OST-HER2 and our OST-tADC platform. We do not expect to generate revenue from drug sales in the near future. We will not generate revenue unless and until we successfully complete the development of, obtain regulatory approval for, and commercialize one or more of our product candidates. Our lead product candidate, OST-HER2, has completed a Phase IIb clinical trial in patients with recurrent, fully resected pulmonary metastatic Osteosarcoma and we are currently pursuing regulatory interactions with the FDA and regulatory authorities in the United Kingdom and European Union regarding potential approval pathways. These pathways may include the FDA’s Accelerated Approval Program and conditional approval pathways in other jurisdictions. However, there can be no assurance that any such approvals will be granted, that confirmatory trials will be successful or that we will ultimately be able to commercialize OST-HER2. Our OST-tADC platform remains in the preclinical stage of development.
Even if we pursue these regulatory pathways, we face significant risks and uncertainties that may prevent us from generating revenue, including, but not limited to:
our ability to attract and retain qualified scientific, clinical and management personnel;
our ability to successfully develop our product candidates, including completing preclinical studies and clinical trials, generating positive safety and efficacy data, and conducting any confirmatory trials that may be required to support accelerated or conditional approvals;
our ability to obtain regulatory approval for our product candidates from the FDA and other regulatory authorities;
the costs, timing and uncertainties associated with advancing additional development programs we may identify internally or through collaborations or other strategic arrangements;
our ability to identify, develop and utilize biomarkers that support regulatory submissions or demonstrate clinical benefit;
our ability to establish and maintain relationships with third-party manufacturers and suppliers for clinical and potential commercial supply, and to accurately forecast and meet supply requirements;
our ability to enter into and maintain collaboration, licensing, manufacturing, distribution and other strategic arrangements on favorable terms;
our ability to establish commercialization capabilities, obtain adequate reimbursement and coverage from third-party payors, and achieve market acceptance among physicians, patients and payors if our products are approved;
our ability to compete effectively with existing or future therapies; and
our ability to obtain, maintain and enforce intellectual property protection and regulatory exclusivity for our product candidates.
In addition, while OST-HER2 has received Rare Pediatric Disease Designation and we may become eligible to receive a Priority Review Voucher if certain regulatory approvals are obtained within specified timeframes, there can be no assurance that we will meet the requirements to receive such a voucher or that it will provide any financial or strategic benefit to us.
Because of the numerous risks and uncertainties associated with drug development and commercialization, we may never generate significant revenue from product sales or achieve profitability.
We have incurred significant operating losses in recent periods and anticipate that we will incur continued losses for the foreseeable future.
Since our inception, we have devoted substantially all of our resources to the research and development of our product candidates, including OST-HER2 and our OST-tADC platform. As a result, we have incurred significant operating losses and negative cash flows from operations. For each of the years ended December 31, 2025 and 2024, we incurred operating losses and negative cash flows from operations. Since July 2018, we have financed our operations primarily through public and private offerings of our securities, from which we have raised aggregate gross proceeds of approximately $41.1 million. We have not generated any revenue from product sales and do not expect to do so unless and until one or more of our product candidates receives regulatory approval and is successfully commercialized. We expect to continue to incur significant expenses and operating losses for the foreseeable future. Our historical losses, together with expected future losses, have had and will continue to have an adverse effect on our stockholders’ equity and working capital. Our expenses are expected to increase substantially as we:
advance the development of our product candidates, including completing preclinical studies and conducting clinical trials, including any confirmatory trials that may be required to support potential regulatory approvals;
prepare regulatory submissions and continue interactions with regulatory authorities in the United States and internationally;
manufacture clinical trial materials and establish or expand relationships with third-party manufacturers to support clinical development and potential commercialization;
continue research and development activities to expand and advance our product pipeline, including our OST-tADC platform;
seek regulatory approval for our product candidates and prepare for potential commercialization;
expand our internal capabilities, including hiring additional scientific, clinical, regulatory and management personnel;
maintain, expand, and protect our intellectual property portfolio; and
incur additional legal, accounting, insurance, investor relations and other expenses associated with operating as a public company.
As a result, we will need to generate significant revenue to achieve profitability, and we may never achieve or sustain profitability.
If we are unable to raise capital when needed or on attractive terms, we would be forced to delay, scale back or discontinue some of our product candidate development programs or commercialization efforts.
The development of biopharmaceutical product candidates is capital intensive and subject to significant uncertainty. We are currently advancing our lead product candidate, OST-HER2, toward potential regulatory submission and approval, and our OST-tADC platform remains in preclinical development. We expect our expenses to increase substantially as we continue to support regulatory interactions, prepare for potential BLA submissions, conduct additional analyses, initiate any required confirmatory clinical trials, and expand our research and development activities. If OST-HER2 receives regulatory approval, we also expect to incur significant costs related to commercialization, including manufacturing scale-up, sales, marketing, distribution and medical affairs, whether independently or with collaborators.
Our capital requirements will depend on many factors, including regulatory outcomes, the scope and timing of any additional clinical trials that may be required by regulatory authorities, the costs of manufacturing and process development for our product candidates, the pace of expansion into additional indications or geographies, and our ability to establish collaborations or strategic partnerships. In particular, despite the positive results from our Phase IIb clinical trial, regulatory authorities may require additional clinical data, including from randomized controlled trials, which would significantly increase our funding needs and extend development timelines.
We will require substantial additional capital to support our ongoing operations and execute our business strategy. However, we may be unable to obtain financing on acceptable terms, or at all. Market conditions, including volatility in the biotechnology sector, and our clinical, regulatory and commercial progress may adversely impact our ability to raise capital. If we are unable to secure adequate funding when needed, we may be required to delay, reduce or terminate development programs, including for OST-HER2 or our OST-tADC platform, limit our ability to pursue additional indications or regulatory approvals in other jurisdictions, delay or forgo commercialization efforts, or curtail our operations, any of which could materially harm our business, financial condition and results of operations.
Raising additional capital may cause dilution to our existing stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.
We may seek additional capital through a combination of public and private securities offerings, including our at-the-market offering program, as well as other debt financings, strategic collaborations and alliances and licensing arrangements. The terms of any financing may adversely affect the holdings or the rights of our stockholders and the issuance of additional securities, whether equity or debt, by us, or the possibility of such issuance, may cause the market price of our shares to decline. The sale of additional equity or convertible securities would dilute all of our stockholders. The incurrence of indebtedness would result in increased fixed payment obligations and we may be required to agree to certain restrictive covenants, such as limitations on our ability to incur additional debt, limitations on our ability to acquire, sell or license intellectual property rights and other operating restrictions that could adversely impact our ability to conduct our business. We could also be required to seek funds through arrangements with collaborators or otherwise at an earlier stage than otherwise would be desirable and we may be required to relinquish rights to some of our technologies or current or future product candidates or otherwise agree to terms unfavorable to us, any of which may have a material adverse effect on our business, operating results and prospects.
Our independent registered public accounting firm has expressed substantial doubt about our ability to continue as a going concern.
We have incurred significant operating losses and negative cash flows from operations since our inception and expect to continue to incur substantial losses for the foreseeable future as we advance the development of our product candidates, including OST-HER2, and continue preclinical development of our OST-tADC platform. To date, we have financed our operations primarily through the issuance of equity and equity-linked securities, including common stock, preferred stock, convertible promissory notes and warrants in public and private offerings.
We do not currently generate product revenue and do not expect to generate any product revenue in the near term. In addition, we do not expect our existing collaboration or licensing arrangements, if any, to provide significant cash inflows sufficient to fund our operations. Our ability to generate revenue, if any, will depend on the successful development, regulatory approval and commercialization of our product candidates, which is subject to significant uncertainty and may not occur.
As a result of our recurring losses from operations, negative cash flows, and net capital deficiency, our independent registered public accounting firm has included an explanatory paragraph in its report on our consolidated financial statements expressing substantial doubt about our ability to continue as a going concern for at least 12 months from the date of issuance of the consolidated financial statements. Our consolidated financial statements do not include any adjustments that might result from the outcome of this uncertainty.
We will need to raise substantial additional capital to fund our operations and remain a going concern. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that such funding, if available, will be obtainable on acceptable terms. If we are unable to raise additional capital or otherwise address our liquidity needs, we may be required to significantly delay, scale back or discontinue the development of our product candidates, including OST-HER2, or otherwise curtail our operations. In such case, there can be no assurance that we will be able to continue as a going concern. In addition, the inclusion of a going concern explanatory paragraph in our auditor’s report may adversely affect our ability to obtain financing on acceptable terms, or at all, and could negatively impact the market price of our common stock.
Our ability to utilize our net operating loss carryforwards and certain other tax attributes in the future may be limited.
Under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended, or the Code, if a corporation undergoes an “ownership change” (generally defined as a greater than 50 percentage points (by value) in the ownership of its equity over a three-year period), the corporation’s ability to use its pre-change tax attributes to offset its post-change income may be limited. We have experienced such ownership changes in the past, and we may experience ownership changes in the future as a result of this offering or subsequent shifts in our stock ownership, some of which are outside our control. As of December 31, 2025 and 2024, we had federal and state NOLs of approximately $33,561,091 and $22,236,580, respectively, federal and state research and development tax credits of $268,568 and $268,568, respectively, and general business credit carryforwards of approximately $3,492,199 and $1,672,876, respectively. Our ability to utilize these NOLs and tax credit carryforwards may be limited by an “ownership change.” If we undergo future ownership changes, many of which may be outside of our control, our ability to utilize our NOLs and tax credit carryforwards could be further limited by Sections 382 and 383 of the Code. There is also a risk that due to regulatory changes, such as suspensions on the use of NOLs, or other unforeseen reasons, our existing NOLs could expire or otherwise become unavailable to offset future income tax liabilities. Additionally, our NOLs and tax credit carryforwards could be limited under state law. For these reasons, even if we attain profitability, we may be unable to use a material portion of our NOLs and other tax attributes.
Risks Related to Drug Development and Regulatory Approval
We depend heavily on the success of our lead product candidates, OST-HER2 and OST-tADC. We may not be able to obtain regulatory approval for, or successfully commercialize, any of our current or future product candidates.
We currently have no products approved for sale and may never successfully develop marketable product candidates. Our business depends heavily on the successful development, regulatory approval, and commercialization of our current and future immunotherapy product candidates for Osteosarcoma, of which our lead candidate, OST-HER2, is in Phase IIb clinical development. OST-tADC remains in preclinical development and will require substantial additional preclinical and clinical testing, as well as regulatory approvals, before we may commercialize it. The preclinical studies and clinical trials of our product candidates, as well as the manufacturing and marketing of any approved product, are subject to extensive and rigorous regulation by government authorities in the United States, the European Economic Area (EEA), and other jurisdictions in which we intend to test or market our product candidates. Before obtaining regulatory approvals, we must demonstrate through preclinical studies and clinical trials that each product candidate is safe and effective for its intended indication. Drug development is a lengthy, expensive, and uncertain process, and delay or failure can occur at any stage. Even with sufficient funding, there can be no assurance that any of our product candidates will be successfully developed, approved, or commercialized.
We cannot market our product candidates in the United States without approval of a BLA from the FDA, in the EEA without approval of a MAA from the EMA, or in other foreign jurisdictions without the requisite approvals. Obtaining regulatory approval is complex, time-consuming, costly, and uncertain, and the FDA, EMA, or other foreign authorities may delay, limit, or deny approval for many reasons, including but not limited to:
inability to demonstrate that our product candidates are safe and effective for their target indications;
preclinical or clinical trial results that fail to meet required statistical or clinical significance;
disagreement with the number, design, size, conduct, or implementation of our studies;
requests to conduct additional preclinical studies or clinical trials;
non-approval of formulation, labeling, or specifications;
actions by CROs that materially adversely affect study results;
regulatory authorities’ determination that our data are insufficient to demonstrate that clinical benefits outweigh safety risks;
disagreement with our interpretation of study data;
non-acceptance of data generated at certain study sites;
advisory committee recommendations that are negative or impose conditions;
requirements to implement a Risk Evaluation and Mitigation Strategy (REMS);
determinations that manufacturing processes or facilities do not comply with regulatory requirements, including cGMPs; or
changes in regulatory policies, requirements, or procedures.
Many of these factors are beyond our control and could prevent or delay regulatory approval, limit the indications for which a product may be approved, or impose significant post-approval obligations. Any such setback could materially and adversely affect our business, prospects, and ability to generate revenue.
Delays or difficulties in enrolling patients in clinical trials could prevent or delay regulatory approval and increase development costs.
We may not be able to initiate or continue clinical trials for our current or future product candidates if we are unable to locate and enroll a sufficient number of eligible patients to participate in these trials as required by the FDA or similar regulatory authorities outside the United States. This challenge is particularly significant because we are focused on patients with rare Osteosarcoma, which has a relatively low prevalence, with an incident rate of approximately 1,000 individuals affected per year in the United States. Identifying patients who meet our eligibility criteria, including those with specific molecular or driver gene profiles, may be difficult and could lead to slower-than-expected enrollment. Some of our competitors have ongoing clinical trials for current or future product candidates that treat the same patient populations as our current or future product candidates, and patients who would otherwise be eligible for our clinical trials may instead enroll in clinical trials of our competitors’ current or future product candidates.
While we successfully enrolled 41 patients in our Phase IIb clinical trial of OST-HER2, patient enrollment in future trials may be slower or more limited due to the rarity of the disease, competition for patients from other clinical trials, or other factors outside our control, including:
the willingness of participants to enroll in our clinical trials and available support in our countries of interest;
the obtaining of informed consent from parents or guardians of pediatric patients which meet evolving regulatory requirements in the United States and other countries;
the severity of the disease under investigation;
the eligibility criteria for the clinical trial in question;
the availability of an appropriate screening test;
the perceived risks and benefits of the product candidate under study;
the efforts to facilitate timely enrollment in clinical trials;
the patient referral practices of physicians;
the ability to monitor patients adequately during and after treatment; and
the proximity and availability of clinical trial sites for prospective patients.
Failure to enroll a sufficient number of patients in future trials could result in significant delays, increased development costs, or the need to suspend or abandon one or more clinical trials. Such delays may also impair our ability to seek participation in expedited regulatory programs, such as the FDA’s priority review or fast track designations, and could materially impact the timing, cost, and success of regulatory approvals and commercialization.
If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals for both our current or future product candidates, we will not be able to commercialize, or will be delayed in commercializing, our current or future product candidates, and our ability to generate revenue will be materially impaired.
Our current or future product candidates and the activities associated with their development and commercialization, including their design, testing, manufacture, safety, efficacy, recordkeeping, labeling, storage, approval, advertising, promotion, sale, distribution, import and export are subject to comprehensive regulation by the FDA and other regulatory agencies in the United States and by comparable authorities in other countries. Before we can commercialize any of our current or future product candidates, we must obtain marketing approval. We have not received approval to market any of our current product candidates and may not obtain regulatory approvals for our future product candidates, if any, from regulatory authorities in any jurisdiction and it is possible that none of our current or future product candidates or any current or future product candidates we may seek to develop in the future will ever obtain regulatory approval. We have only limited experience in filing and supporting the applications necessary to gain regulatory approvals and expect to rely on third-party CROs and/or regulatory consultants to assist us in this process. Securing regulatory approval requires the submission of extensive preclinical and clinical data and supporting information to the various regulatory authorities for each therapeutic indication and line of treatment to establish the product candidate’s safety and efficacy. Securing regulatory approval also requires the submission of information about the drug manufacturing process to, and inspection of manufacturing facilities by, the relevant regulatory authority. Our current or future product candidates may not be effective, may be only moderately effective or may prove to have undesirable or unintended side effects, toxicities or other characteristics that may preclude our obtaining marketing approval or prevent or limit commercial use.
The process of obtaining regulatory approvals, both in the United States and abroad, is expensive, may take many years if additional clinical trials are required, if approval is obtained at all, and can vary substantially based upon a variety of factors, including the type, complexity and novelty of the current or future product candidates involved. Changes in marketing approval requirements or policies during the development period, changes in or the enactment of additional statutes or regulations, or changes in regulatory review for each submitted NDA or BLA. The FDA and comparable authorities in other countries have substantial discretion in the approval process and may refuse to accept any application or may decide that our data is insufficient for approval and require additional preclinical, clinical or other studies. Our current or future product candidates could be delayed in receiving, or fail to receive, regulatory approval for many reasons, including the following:
the FDA or comparable foreign regulatory authorities may disagree with the design or implementation of our clinical trials;
we may be unable to demonstrate to the satisfaction of the FDA or comparable foreign regulatory authorities that a product candidate is safe and effective for its proposed indication or that it is suitable to identify appropriate patient populations;
the results of clinical trials may not meet the level of statistical significance required by the FDA or comparable foreign regulatory authorities for approval;
we may be unable to demonstrate that a product candidate’s clinical and other benefits outweigh its safety risks;
the FDA or comparable foreign regulatory authorities may disagree with our interpretation of data from preclinical studies or clinical trials;
the data collected from clinical trials of our current or future product candidates may not be sufficient to support the submission of an NDA, a BLA or other submission or to obtain regulatory approval in the United States or elsewhere;
the FDA or comparable foreign regulatory authorities may fail to approve the manufacturing processes or facilities of third-party manufacturers with which we contract for clinical and commercial supplies; and
the approval requirements or policies or regulations of the FDA or comparable foreign regulatory authorities may significantly change in a manner rendering our clinical data insufficient for approval.
In addition, even if we were to obtain approval, regulatory authorities may approve any of our current or future product candidates for fewer or more limited indications than we request, may not approve the price we intend to charge for our drugs, may grant approval contingent on the performance of costly post-marketing clinical trials, or may approve a product candidate with a label that does not include the labeling claims necessary or desirable for the successful commercialization of that product candidate. Any of the foregoing scenarios could materially harm the commercial prospects for our current or future product candidates, and our ability to generate revenues will be materially impaired.
Our current or future product candidates may cause adverse or other undesirable side effects that could delay or prevent their regulatory approval, limit the commercial profile of an approved label, or result in significant negative consequences following marketing approval, if any.
Undesirable side effects caused by our current or future product candidates could cause us to interrupt, delay or halt preclinical studies or could cause us or regulatory authorities to interrupt, delay or halt clinical trials and could result in a more restrictive label or the delay or denial of regulatory approval by the FDA or other regulatory authorities. While we have initiated clinical trials for OST-HER2, and although there have been limited side effects with this therapy to date, it is likely that there may be adverse side effects associated with its use. Results of our trials could reveal a high and unacceptable severity and prevalence of these or other side effects. In such an event, our trials could be suspended or terminated and the FDA or comparable foreign regulatory authorities could order us to cease further development of or deny approval of our current or future product candidates for any or all targeted indications. The drug-related side effects could affect patient recruitment or the ability of enrolled patients to complete the trial or result in potential product liability claims. Any of these occurrences may significantly harm our business, financial condition and prospects.
Further, our current or future product candidates could cause undesirable side effects in clinical trials related to on-target toxicity, or exaggerated and adverse pharmacologic effects at the target of interest in the test system. If on-target toxicity is observed, or if our current or future product candidates have characteristics that are unexpected, we may need to abandon their development or limit development to more narrow uses or subpopulations in which the undesirable side effects or other characteristics are less prevalent, less severe or more acceptable from a risk-benefit perspective. In our industry, many compounds that initially showed promise in early stage testing for treating cancer have later been found to cause side effects that prevented further development of the compound.
Clinical trials by their nature utilize a sample of the potential patient population. For example, the 41 patients in our Phase IIb trial of OST-HER2 may include a limited number of patients with side effects. With a limited number of patients and limited duration of exposure, rare and severe side effects of our current or future product candidates may only be uncovered with a significantly larger number of patients exposed to the product candidate. If our current or future product candidates are tested in large numbers of patients or if they receive marketing approval and we or others identify undesirable side effects caused by such current or future product candidates after such approval, a number of potentially significant negative consequences could result, including:
regulatory authorities may place a hold on an ongoing clinical trial or may refuse to allow a future clinical trial to be conducted;
regulatory authorities may withdraw or limit their approval of current or future product candidates;
we may or a regulatory authority might require that the product or products be recalled;
regulatory authorities may require the addition of labeling statements, such as a “boxed” warning or a contraindication;
we may be required to create a medication guide outlining the risks of such side effects for distribution to patients;
we may be required to change the way such current or future product candidates are distributed or administered, conduct additional clinical trials or change the labeling of the current or future product candidates;
regulatory authorities may require a REMS plan to mitigate risks, which could include medication guides, physician communication plans, or elements to assure safe use, such as restricted distribution methods, patient registries and other risk minimization tools;
we may be subject to regulatory investigations and government enforcement actions;
we may decide to remove such current or future product candidates from the marketplace; and
we could be sued and held liable for injury caused to individuals exposed to or taking our current or future product candidates.
We believe that any of these events could prevent us from achieving or maintaining market acceptance of the affected current or future product candidates and could substantially increase the costs of commercializing our current or future product candidates, if approved, and significantly impact our ability to successfully commercialize our current or future product candidates and generate revenues.
A breakthrough therapy designation by the FDA for our current or future product candidate does not convey any advantage in, or shorten the duration of, the regulatory review or approval process, and it does not increase the likelihood that our current or future product candidates will receive marketing approval.
In May 2024, we submitted a request to the FDA for breakthrough therapy designation for OST-HER2, and we may seek a breakthrough therapy designation for some of our other current or future product candidates. A breakthrough therapy is defined as a drug that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. For drugs that have been designated as breakthrough therapies, interaction and communication between the FDA and the sponsor of the trial can help to identify the most efficient path for clinical development while minimizing the number of patients placed in ineffective control regimens. Drugs designated as breakthrough therapies by the FDA are also eligible for accelerated approval.
Designation as a breakthrough therapy is within the discretion of the FDA. Accordingly, even if we believe that one of our current or future product candidates meets the criteria for designation as a breakthrough therapy, the FDA may disagree and instead determine not to make such designation. In any event, the receipt of a Breakthrough Therapy Designation for a product candidate does not convey any advantage in, or shorten the duration of, regulatory review or approval compared to drugs considered for approval under conventional FDA procedures and does not assure ultimate approval by the FDA. In addition, even if one or more of our current or future product candidates qualify as breakthrough therapies, the FDA may later decide that the drugs no longer meet the conditions for qualification.
A fast track designation by the FDA does not convey any advantage in, or shorten the duration of, the regulatory review regulatory review or approval process.
If a drug is intended for the treatment of a serious or life-threatening condition and the drug demonstrates the potential to address unmet medical needs for this condition, the drug sponsor may apply for fast track designation. The FDA has broad discretion whether or not to grant this designation, so even if we believe that a particular product candidate is eligible for this designation, we cannot assure you that the FDA would decide to grant it. Even though we have received fast track designation for OST-HER2 and may receive fast track designation again in the future for certain current or future product candidates, this designation does not convey any advantage in, or shorten the duration of, regulatory review or approval compared to conventional FDA procedures. The FDA may withdraw fast track designation if it believes that the designation is no longer supported by data from our clinical development program.
We may not be able to obtain or maintain orphan drug designation or exclusivity for any product candidates and, even if we do, that exclusivity may not prevent the FDA, EMA or other regulatory authorities from approving other competing products.
OST-HER2 received orphan drug designation for Osteosarcoma in the United States, and we may seek orphan drug designation (“ODD”) for other current or future product candidates. We have submitted the required information to the FDA in order to re-establish ODD for OST-HER2 in 2026. Regulatory authorities in some jurisdictions, including the United States and the European Union, may designate drugs for relatively small patient populations as orphan drugs. Under the Orphan Drug Act of 1983, 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 generally defined as a patient population of fewer than 200,000 individuals in the United States.
Generally, 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 may be entitled to a period of marketing exclusivity, during which the FDA or EMA generally cannot approve another marketing application for the same active ingredient for the same indication. The applicable period is seven years in the United States and ten years in the European Union, although EU exclusivity may be reduced to six years if a drug no longer meets the orphan criteria or is considered sufficiently profitable. Orphan drug exclusivity may be lost if the FDA or EMA determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient drug supply to meet patient needs.
Even if we obtain orphan drug exclusivity for a product, that exclusivity may not effectively protect the product from competition. Drugs with different active ingredients can be approved for the same condition, and the FDA or EMA may approve the same drug for the same condition if a later product demonstrates clinical superiority, such as being safer, more effective, or making a major contribution to patient care. In addition, we may not be the first to obtain marketing approval for a particular orphan indication. Approval of another product for the same active ingredient and indication could prevent or delay our ability to obtain orphan drug exclusivity, materially affecting our competitive position.
Regulatory authorities may also revise orphan drug regulations or policies, and it is uncertain how any such changes could impact our business or exclusivity rights.
Although we have obtained rare pediatric disease designation for OST-HER2 for Osteosarcoma patients, we may not be eligible to receive a priority review voucher in the event that FDA approval does not occur within the timeframe required by the applicable statutory provisions or if future law changes eliminate or further modify the PRV Program.
The Rare Pediatric Disease Priority Review Voucher Program (“PRV Program”) is intended to incentivize pharmaceutical sponsors to develop drugs for rare diseases. A sponsor who obtains approval of an NDA or BLA for a rare disease may be eligible for a Priority Review Voucher (“PRV”) under this program, which may be redeemed by the owner of such PRV to obtain priority review for a marketing application. A PRV is fully transferrable and can be sold to any sponsor, who in turn can redeem the PRV for priority review of a marketing application in six months, compared to the standard timeframe of approximately ten months. Under current law, as amended by the Consolidated Appropriations Act, 2026, the PRV Program is authorized through September 30, 2029. The FDA may not award PRVs under this program after that date. Eligibility for a PRV generally requires both rare pediatric disease designation and approval of a marketing application for the designated rare pediatric disease. Even if OST-HER2 receives rare pediatric disease designation and is ultimately approved, we may not receive a PRV if the BLA is not approved in time to meet statutory requirements or if the PRV Program is subsequently modified or allowed to expire. In addition, even if we are eligible for and obtain a PRV, there can be no assurance that we will be able to realize significant value from the voucher, as the market for PRVs and their perceived value may vary over time and may be affected by changes in regulatory policies, business conditions or the supply of available PRVs.
Even if we receive regulatory approval for any of our current or future product candidates, we will be subject to ongoing obligations and continued regulatory review, which may result in significant additional expense. Additionally, our current or future product candidates, if approved, could be subject to labeling and other restrictions and market withdrawal and we may be subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our drugs.
If the FDA or a comparable foreign regulatory authority approves any of our current or future product candidates, the manufacturing processes, labeling, packaging, distribution, adverse event reporting, storage, advertising, promotion and recordkeeping for the drug will be subject to extensive and ongoing regulatory requirements. These requirements include submissions of safety and other post-marketing information and reports, registration, as well as continued compliance with cGMPs and GCPs for any clinical trials that we conduct post-approval. Any regulatory approvals that we receive for our current or future product candidates may also be subject to limitations on the approved indicated uses for which the drug may be marketed or to the conditions of approval, or contain requirements for potentially costly post-marketing testing, including Phase IV clinical trials, and surveillance to monitor the safety and efficacy of the drug. Later discovery of previously unknown problems with a drug, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things:
restrictions on the marketing or manufacturing of the drug, withdrawal of the drug from the market, or drug recalls;
fines, warning or other letters or holds on clinical trials;
refusal by the FDA to approve pending applications or supplements to approved applications filed by us, or suspension or revocation of drug license approvals;
drug seizure or detention, or refusal to permit the import or export of drugs; and
injunctions or the imposition of civil or criminal penalties.
The FDA’s policies may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our current or future product candidates. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval that we may have obtained, which would adversely affect our business, prospects and ability to achieve or sustain profitability.
Positive results from early preclinical studies and clinical trials of our current or future product candidates are not necessarily predictive of the results of later preclinical studies and clinical trials of our current or future product candidates. If we cannot replicate the positive results from our earlier preclinical studies and clinical trials of our current or future product candidates in our later preclinical studies and clinical trials, we may be unable to successfully develop, obtain regulatory approval for and commercialize our current or future product candidates.
Positive results from our preclinical studies of our current or future product candidates, and any positive results we may obtain from our early clinical trials of our current or future product candidates, may not necessarily be predictive of the results from required later preclinical studies and clinical trials. Similarly, even if we are able to complete our planned preclinical studies or clinical trials of our current or future product candidates according to our current development timeline, the positive results from our preclinical studies and clinical trials of our current or future product candidates may not be replicated in subsequent preclinical studies or clinical trial results. For example, our later-stage clinical trials could differ in significant ways from our Phase IIb clinical trial of OST-HER2, which could cause the outcome of these later-stage trials to differ from our earlier-stage clinical trials. For example, these differences may include changes to inclusion and exclusion criteria, final dosage formulation, efficacy endpoints and statistical design.
Many companies in the pharmaceutical and biotechnology industries have suffered significant setbacks in late-stage clinical trials after achieving positive results in early-stage development, and we cannot be certain that we will not face similar setbacks. These setbacks have been caused by, among other things, preclinical findings made while clinical trials were underway or safety or efficacy observations made in preclinical studies and clinical trials, including previously unreported adverse events. Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that believed their product candidates performed satisfactorily in preclinical studies and clinical trials nonetheless failed to obtain FDA approval. If we fail to produce positive results in our planned preclinical studies or clinical trials of any of our current or future product candidates, the development timeline and regulatory approval and commercialization prospects for our current or future product candidates, and, correspondingly, our business and financial prospects, would be materially adversely affected.
Manufacturing our current or future product candidates is complex and we may encounter difficulties in production. If we encounter such difficulties, our ability to provide our current or future product candidates for preclinical studies and clinical trials or for commercial purposes could be delayed or stopped.
The process of manufacturing of our current or future product candidates is complex and highly regulated. We do not have our own manufacturing facilities or personnel and currently rely, and expect to continue to rely, on third parties based in the United States, Europe and Asia for the manufacture of our current or future product candidates. These third-party manufacturing providers may not be able to provide adequate resources or capacity to meet our needs and may incorporate their own proprietary processes into our product candidate manufacturing processes. We have limited control and oversight of a third-party’s proprietary process, and a third-party may elect to modify its process without our consent or knowledge. These modifications could negatively impact our manufacturing, including product loss or failure that requires additional manufacturing runs or a change in manufacturer, both of which could significantly increase the cost of and significantly delay the manufacture of our current or future product candidates. As our current or future product candidates progress through preclinical studies and clinical trials towards approval and commercialization, it is expected that various aspects of the manufacturing process will be altered in an effort to optimize processes and results. Such changes may require amendments to be made to regulatory applications which may further delay the timeframes under which modified manufacturing processes can be used for any of our current or future product candidates and additional bridging studies or trials may be required.
Our future growth may depend, in part, on our ability to penetrate foreign markets, where we would be subject to additional regulatory burdens and other risks and uncertainties that could materially adversely affect our business.
We are not permitted to market or promote any of our current or future product candidates in foreign markets before we receive regulatory approval from the applicable regulatory authority in that foreign market, and we may never receive such regulatory approval for any of our current or future product candidates. To obtain separate regulatory approval in many other countries we must comply with numerous and varying regulatory requirements of such countries regarding safety and efficacy and governing, among other things, clinical trials and commercial sales, pricing and distribution of our current or future product candidates, and we cannot predict success in these jurisdictions. If we obtain approval of our current or future product candidates and ultimately commercialize our current or future product candidates in foreign markets, we would be subject to additional risks and uncertainties, including:
differing regulatory requirements in foreign countries, which may cause obtaining regulatory approvals outside of the United States to take longer and be more costly than obtaining approval in the United States;
the burden of complying with complex and changing foreign regulatory, tax, accounting and legal requirements;
different medical practices and customs in foreign countries affecting acceptance in the marketplace;
import or export licensing requirements;
reduced protection of intellectual property rights and the existence of additional potentially relevant third-party intellectual property rights;
economic weakness, including inflation, or political instability in particular foreign economies and markets;
compliance with tax, employment, immigration and labor laws for employees living or traveling abroad;
foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country;
workforce uncertainty in countries where labor unrest is more common than in the United States;
potential liability under the Foreign Corrupt Practices Act of 1977 or comparable foreign regulations;
production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; and
business interruptions resulting from geopolitical actions, including war and terrorism.
Foreign sales of our current or future product candidates could also be adversely affected by the imposition of governmental controls, political and economic instability, trade restrictions and changes in tariffs.
We may in the future conduct clinical trials for current or future product candidates outside the United States, and the FDA and comparable foreign regulatory authorities may not accept data from such trials.
We may in the future choose to conduct one or more clinical trials outside the United States, including in Europe. The acceptance of study data from clinical trials conducted outside the United States or another jurisdiction by the FDA or comparable foreign regulatory authority may be subject to certain conditions or may not be accepted at all. In cases where data from foreign clinical trials are intended to serve as the basis for marketing approval in the United States, the FDA will generally not approve the application on the basis of foreign data alone unless (i) the data are applicable to the United States population and United States medical practice and (ii) the trials were performed by clinical investigators of recognized competence and pursuant to GCP regulations. Additionally, the FDA’s clinical trial requirements, including sufficient size of patient populations and statistical powering, must be met. Many foreign regulatory authorities have similar approval requirements. In addition, such foreign trials would be subject to the applicable doctrines or local laws of the foreign jurisdictions where the trials are conducted. There can be no assurance that the FDA or any comparable foreign regulatory authority will accept data from trials conducted outside of the United States or the applicable jurisdiction. If the FDA or any comparable foreign regulatory authority does not accept such data, it would result in the need for additional trials, which could be costly and time-consuming, and which may result in current or future product candidates that we may develop not receiving approval for commercialization in the applicable jurisdiction.
We may not be successful in our efforts to identify or discover additional product candidates or we may expend our limited resources to pursue a particular product candidate or indication and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success.
The success of our business depends primarily upon our ability to identify, develop and commercialize our product candidates. Although some of our current product candidates are in preclinical and clinical development, our scientific hypotheses may be incorrect or our research programs may fail to identify other potential product candidates for clinical development for a number of reasons. Our research methodologies may be unsuccessful in identifying potential product candidates, or our potential product candidates may be shown to have harmful side effects or may have other characteristics that may make the products unmarketable or unlikely to receive marketing approval.
Because we have limited financial and management resources, we focus on a limited number of research programs and product candidates and are currently focused on our lead core product candidate OST-HER2 and our other core product candidate OST-tADC, both targeting the treatment of Osteosarcoma. As a result, we may forego or delay pursuit of opportunities with other current or future product candidates or for other indications that later prove to have greater commercial potential. Our resource allocation decisions may cause us to fail to capitalize on viable commercial drugs or profitable market opportunities. Our spending on current and future research and development programs and current or future product candidates for specific indications may not yield any commercially viable drugs. If we do not accurately evaluate the commercial potential or target market for a particular product candidate, we may relinquish valuable rights to that product candidate through future collaboration, licensing or other royalty arrangements in cases in which it would have been more advantageous for us to retain sole development and commercialization rights to such product candidate.
If any of these events occur, we may be forced to abandon our development efforts for a program, which would have a material adverse effect on our business and could potentially cause us to cease operations. Research programs to identify new product candidates require substantial technical, financial and human resources. We may focus our efforts and resources on potential programs or current or future product candidates that ultimately prove to be unsuccessful.
Although a larger number of Osteosarcoma patients reside outside the United States, our ability to generate meaningful revenues in those jurisdictions may be limited due to pricing controls, reimbursement limitations, and other market access challenges.
The incidence of new cases of Osteosarcoma is approximately 1,000 individuals annually in the United States and approximately 20,000 globally. Although the global patient population is larger, our ability to generate revenues outside the United States may be limited by pricing regulations, reimbursement restrictions, and market access barriers. In many countries, particularly in the European Union and other developed markets, the pricing of prescription pharmaceuticals is subject to governmental control, and pricing and reimbursement approvals may be required prior to or following marketing authorization. Pricing negotiations with governmental authorities can be lengthy and may delay the commercial launch of a product candidate. In some jurisdictions, obtaining reimbursement or pricing approval may require the submission of health economic data or the conduct of additional clinical studies to demonstrate cost-effectiveness relative to existing therapies. If reimbursement for our product candidates is unavailable, limited in scope, or subject to significant restrictions, or if pricing is set at unsatisfactory levels, our ability to generate revenues in those markets may be materially adversely affected. In addition, in certain regions, including parts of Africa and the Middle East, limited healthcare infrastructure and diagnostic capabilities may constrain our ability to identify and treat patients, thereby limiting commercial opportunities. In the United States, there have been significant efforts to control drug pricing. For example, the Inflation Reduction Act of 2022 introduced measures that allow the federal government to negotiate prices for certain drugs under Medicare and impose rebates tied to inflation. These and other pricing reforms may reduce the prices we are able to charge for any approved products and adversely affect our revenues.
Risks Related to Commercialization
Even if we receive marketing approval for our current or future product candidates, our current or future product candidates may not achieve broad market acceptance, which would limit the revenue that we generate from their sales.
The commercial success of our current or future product candidates, if approved by the FDA or other applicable regulatory authorities, will depend upon the awareness and acceptance of our current or future product candidates among the medical community, including physicians and patients, as well as reimbursement and coverage by third party payors including Medicare and Medicaid. Market acceptance of our current or future product candidates, if approved, will depend on a number of factors, including, among others:
the efficacy of our current or future product candidates as demonstrated in clinical trials, and, if required by any applicable regulatory authority in connection with the approval for the applicable indications, to provide patients with incremental health benefits, as compared with other available medicines;
limitations or warnings contained in the labeling approved for our current or future product candidates by the FDA or other applicable regulatory authorities;
the clinical indications for which our current or future product candidates are approved;
availability of alternative treatments already approved or expected to be commercially launched in the near future;
the potential and perceived advantages of our current or future product candidates over current treatment options or alternative treatments, including future alternative treatments;
the willingness of the target patient population to try new therapies or treatment methods and of physicians to prescribe these therapies or methods;
the need to dose such product candidates in combination with other therapeutic agents, and related costs;
the strength of marketing and distribution support and timing of market introduction of competitive products;
pricing and cost effectiveness;
the effectiveness of our sales and marketing strategies;
our ability to increase awareness of our current or future product candidates;
our ability to obtain sufficient third-party coverage and reimbursement, including from federal healthcare programs such as Medicare and Medicaid; or
the ability or willingness of patients to pay out-of-pocket in the absence of third-party coverage.
If our current or future product candidates are approved but do not achieve an adequate level of acceptance by patients, physicians and payors, we may not generate sufficient revenue from our current or future product candidates to become or remain profitable. Before agreeing to cover and reimburse our products, third party payors may require us to demonstrate that our current or future product candidates, in addition to treating these target indications, are not only safe but cost effective compared to alternative therapies. Our efforts to educate the medical community, patient organizations and third-party payors about the benefits of our current or future product candidates may require significant resources and may never be successful.
We face substantial competition, which may result in others discovering, developing or commercializing drugs before or more successfully than we do.
The development and commercialization of new drugs is highly competitive and constantly evolving. We face competition with respect to our current product candidates and will face competition with respect to any product candidates that we may seek to develop or commercialize in the future from major pharmaceutical companies, specialty pharmaceutical companies, biotechnology companies, academic institutions, government agencies and other public and private research organizations worldwide. Some of these competitors currently market or are actively developing therapies for rare diseases and cancers, including Osteosarcoma, and some programs may be based on scientific approaches that are similar to ours, while others may employ entirely different approaches.
Specifically, if OST-HER2 receives marketing approval for the treatment of Osteosarcoma, it may face competition from other product candidates in development for these indications, including programs from AstraZeneca, Y-mAbs Therapeutics, MD Anderson Cancer Center and others. The competitive landscape is dynamic: competitors’ programs may advance or be discontinued, and new entrants may emerge, which could materially affect our ability to capture or maintain market share.
Many of the companies against which we are competing or may compete in the future have significantly greater financial, technical, regulatory and commercial resources than we do, including greater expertise in research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining regulatory approvals and reimbursement, and marketing approved drugs. Mergers and acquisitions in the pharmaceutical and biotechnology industries may result in further consolidation and concentration of resources among a smaller number of competitors. Smaller or early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies. These competitors may also compete with us in recruiting and retaining qualified scientific, sales, marketing, and management personnel, establishing clinical trial sites and enrolling patients in clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs.
Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize drugs that are safer, more effective, have fewer or less severe side effects, are more convenient or are less expensive than any drugs that we or our collaborators may develop. Our competitors also may obtain FDA or other regulatory approval for their drugs more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we or our collaborators are able to enter the market. The key competitive factors affecting the success of all of our current or future product candidates, if approved, are likely to be their efficacy, safety, convenience, price, the level of generic competition and the availability of reimbursement from government and other third-party payors.
Product liability lawsuits against us could cause us to incur substantial liabilities and could limit commercialization of any current or future product candidates that we may develop.
We will face an inherent risk of product liability exposure related to the testing of our current or future product candidates in human clinical trials and will face an even greater risk if we commercially sell any current or future product candidates that we may develop. If we cannot successfully defend ourselves against claims that our current or future product candidates caused injuries, we could incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may result in:
decreased demand for any current or future product candidates that we may develop;
injury to our reputation and significant negative media attention;
withdrawal of clinical trial participants;
significant costs and resources to defend the related litigation;
substantial monetary awards to trial participants or patients; and
the inability to commercialize any current or future product candidates that we may develop.
Although we maintain product liability insurance coverage, it may not be adequate to cover all liabilities that we may incur. We anticipate that we will need to increase our insurance coverage when we initiate a large global trial and if we successfully commercialize any product candidate. Insurance coverage is increasingly expensive. We may not be able to maintain product liability insurance coverage at a reasonable cost or in an amount adequate to satisfy any liability that may arise.
Even if we are able to commercialize any current or future product candidates, such drugs may become subject to unfavorable pricing regulations or third-party coverage and reimbursement policies, which would harm our business.
The regulations that govern regulatory approvals, pricing, and reimbursement for new drugs vary widely from country to country. Some countries require approval of the sale price of a drug before it can be marketed, and in many countries, the pricing review period begins after marketing approval is granted. In certain markets, including the European Union, prescription pharmaceutical pricing remains subject to continuing governmental control even after initial approval. As a result, we may obtain marketing approval for a product candidate in a particular country but be subject to price regulations that delay commercial launch or limit the revenues we are able to generate from the sale of the product candidate in that country. Adverse pricing limitations may hinder our ability to recoup our investment in one or more current or future product candidates, even if approved.
In the United States, there have been significant legislative and regulatory efforts to control drug pricing. For example, the Inflation Reduction Act of 2022 includes provisions that permit the U.S. Department of Health and Human Services, through the CMS, to negotiate prices for certain high-expenditure drugs covered under Medicare, impose inflation-based rebates, and redesign certain aspects of the Medicare Part D program. While the full implementation and long-term impact of these measures are still evolving, they may reduce the prices we are able to charge for any approved products and adversely affect our revenues and profitability. Additional federal or state healthcare reform measures may also be adopted in the future that could further impact pricing and reimbursement.
Our ability to successfully commercialize any current or future product candidates will depend in part on the extent to which coverage and reimbursement for these product candidates and related treatments are available from government authorities, private health insurers, and other organizations. Government authorities and other third-party payors decide which medications they will cover and establish reimbursement levels. Factors payors consider in determining reimbursement include whether the product is a covered benefit, safe and effective, medically necessary, appropriate for the patient, and cost-effective, and whether it is considered experimental or investigational.
A primary trend in the U.S. healthcare industry and elsewhere is cost containment. Government authorities and third-party payors have sought to control costs by limiting coverage, setting reimbursement levels, requiring rebates and discounts, and challenging the prices charged for drugs. We cannot be sure that coverage will be available for any product candidate that we commercialize or, if coverage is available, the level of reimbursement. Reimbursement levels may impact the demand for, or the price of, any product candidate for which we obtain marketing approval.
There may be significant delays in obtaining reimbursement for newly approved drugs, and coverage may be more limited than the purposes for which the drug is approved by the FDA or comparable foreign regulatory authorities. Moreover, eligibility for reimbursement does not imply that any drug will be paid for in all cases or at a rate that covers our costs, including research, development, manufacturing, and distribution. Reimbursement rates may vary based on the use of the drug, the clinical setting, and comparisons to lower-cost therapies, and may be incorporated into bundled payments for other services. Net prices for drugs may also be reduced by mandatory discounts or rebates required by government healthcare programs or private payors. In the United States, coverage and reimbursement decisions for Medicare are made by CMS, and private payors often follow Medicare coverage policies and payment limitations in setting their own reimbursement practices. Our inability to obtain timely and adequate coverage and reimbursement for any approved products could have a material adverse effect on our business, financial condition, and results of operations.
Healthcare reform measures may have a material adverse effect on our business and results of operations.
The United States and many foreign jurisdictions have enacted and continue to consider legislative and regulatory changes affecting the healthcare system that could prevent or delay marketing approval of our current or future product candidates, restrict or regulate post-approval activities, and affect our ability to profitably commercialize any products for which we obtain marketing approval. Changes in regulations, statutes, or the interpretation of existing requirements could require, among other things: (i) changes to our manufacturing arrangements; (ii) additions or modifications to product labeling; (iii) the recall or discontinuation of products; or (iv) additional recordkeeping, reporting, or compliance obligations. Any such changes could adversely affect our operations and increase our costs.
In the United States, there have been significant efforts to control healthcare costs and drug pricing. For example, the Inflation Reduction Act of 2022 includes provisions that, among other things, allow the U.S. Department of Health and Human Services to negotiate prices for certain drugs covered under Medicare, impose inflation-based rebates, and redesign certain aspects of the Medicare Part D program. The implementation and long-term effects of these measures are still evolving, but they may reduce the revenues we are able to generate from any approved products. In addition, other federal and state legislative and regulatory proposals aimed at controlling drug pricing, increasing transparency, or reforming reimbursement systems have been introduced and may be enacted in the future.
Our revenue prospects may also be affected by changes in healthcare spending and policy in the United States and abroad. We operate in a highly regulated industry, and new laws, regulations, or judicial decisions, or new interpretations of existing laws, regulations, or decisions, related to healthcare availability, pricing, coverage, or reimbursement may negatively impact our business, financial condition, and results of operations. We cannot predict the likelihood, nature, or extent of future healthcare reform measures or their potential impact on our business. If we or any third parties we engage are unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we or such third parties fail to maintain regulatory compliance, we may lose any regulatory approvals that we may obtain and may not achieve or sustain profitability.
If, in the future, we are unable to establish sales and marketing and patient support capabilities or enter into agreements with third parties to sell and market our current or future product candidates, we may not be successful in commercializing our current or future product candidates if and when they are approved, and we may not be able to generate any revenue.
We do not currently have a sales or marketing infrastructure and have limited experience in the sales, marketing, patient support or distribution of drugs. To achieve commercial success for any approved product candidate for which we retain sales and marketing responsibilities, we must build our sales, marketing, patient support, managerial and other non-technical capabilities or make arrangements with third parties to perform these services. In the future, we may choose to build a focused sales and marketing infrastructure to sell, or participate in sales activities with our collaborators for, some of our current or future product candidates if and when they are approved.
There are risks involved with both establishing our own sales and marketing and patient support capabilities and entering into arrangements with third parties to perform these services. For example, recruiting and training a sales force is expensive and time consuming and could delay any drug launch. If the commercial launch of a product candidate for which we recruit a sales force and establish marketing capabilities is delayed or does not occur for any reason, we would have prematurely or unnecessarily incurred these commercialization expenses. This may be costly, and our investment would be lost if we cannot retain or reposition our sales and marketing personnel.
Factors that may inhibit our efforts to commercialize our current or future product candidates on our own include:
our inability to recruit and retain adequate numbers of effective sales and marketing personnel;
the inability of sales personnel to obtain access to physicians or persuade adequate numbers of physicians to prescribe any future drugs;
the lack of complementary drugs to be offered by sales personnel, which may put us at a competitive disadvantage relative to companies with more extensive product lines; and
unforeseen costs and expenses associated with creating an independent sales and marketing organization.
If we enter into arrangements with third parties to perform sales, marketing, patient support and distribution services, our drug revenues or the profitability of these drug revenues to us are likely to be lower than if we were to market and sell any current or future product candidates that we develop ourselves. In addition, we may not be successful in entering into arrangements with third parties to sell and market our current or future product candidates or may be unable to do so on terms that are favorable to us. We likely will have little control over such third parties, and any of them may fail to devote the necessary resources and attention to sell and market our current or future product candidates effectively, or they may engage in practices that pose legal risks to us under applicable anti-kickback, fraud and abuse and other healthcare laws and regulations. If we do not establish sales and marketing capabilities successfully, either on our own or in collaboration with third parties, we will not be successful in commercializing our current or future product candidates.
Our relationships with prescribers, customers and third-party payors will be subject to applicable anti-kickback, fraud and abuse and other healthcare laws and regulations, which could expose us to significant penalties and adversely affect our business.
Although we do not currently have any products on the market, if we begin commercializing our current or future product candidates, we will become subject to additional healthcare statutory and regulatory requirements and enforcement by federal, state, and foreign governmental authorities. Healthcare providers, including physicians, play a primary role in the recommendation and prescription of any products for which we obtain marketing approval. Our arrangements with healthcare providers, third-party payors, and customers will expose us to broadly applicable fraud and abuse and other healthcare laws and regulations that constrain the business and financial relationships through which we market, sell, and distribute our products. Restrictions under applicable federal and state healthcare laws and regulations include, among others, the following:
the federal Anti-Kickback Statute prohibits, among other things, knowingly and willfully soliciting, offering, receiving, or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward referrals or the purchase, order, or recommendation of any good or service reimbursable under federal or state healthcare programs such as Medicare, Medicaid, and TRICARE. The term “remuneration” has been interpreted broadly to include anything of value, and a person or entity need not have actual knowledge of the statute or specific intent to violate it to be found in violation;
the federal False Claims Act imposes civil and criminal penalties, including through whistleblower (qui tam) actions, against individuals or entities for knowingly presenting, or causing to be presented, false or fraudulent claims for payment to the federal government or for making false statements to avoid an obligation to pay money to the government. Manufacturers may be held liable even when they do not submit claims directly if they are deemed to “cause” the submission of false claims. The statute provides for treble damages and significant per-claim penalties, and has been used to pursue a wide range of pharmaceutical company activities, including alleged improper promotional practices, off-label promotion, and pricing-related conduct;
HIPPA imposes criminal and civil liability for executing schemes to defraud healthcare benefit programs or making false statements in connection with the delivery of or payment for healthcare benefits, items, or services;
federal physician payment transparency requirements (commonly referred to as the Open Payments program) require manufacturers of drugs, biologics, and medical supplies reimbursable under federal healthcare programs to report annually to the U.S. Department of Health and Human Services information regarding payments and other transfers of value to physicians, certain non-physician healthcare providers (such as physician assistants and nurse practitioners), and teaching hospitals, as well as ownership and investment interests held by such individuals;
HIPAA, as amended by HITECH, and its implementing regulations, impose obligations on covered entities and their business associates with respect to safeguarding the privacy, security, and transmission of individually identifiable health information, including breach notification requirements and expanded enforcement authority; and
analogous state and foreign laws and regulations, including state anti-kickback and false claims laws, transparency laws, and data privacy and security laws, many of which differ from federal requirements and may not be preempted, thereby complicating compliance efforts.
Ensuring that our business arrangements with third parties comply with applicable healthcare laws and regulations could involve substantial costs. The regulatory and enforcement environment in the healthcare industry remains active, and governmental authorities have increased their focus on compliance with these laws. It is possible that governmental authorities will conclude that our business practices do not comply with current or future statutes, regulations, or case law. If our operations are found to be in violation of any of these laws or any other applicable regulations, we may be subject to significant civil, criminal, and administrative penalties, including damages, fines, exclusion from government-funded healthcare programs such as Medicare and Medicaid, and the curtailment or restructuring of our operations. In addition, if any of the physicians or other providers or entities with whom we do business are found to be non-compliant, they may be subject to sanctions, which could also adversely affect our business.
We may face potential liability and increased regulatory scrutiny if we obtain, use or fail to adequately protect identifiable patient health information in connection with our clinical trials and operations.
In the course of our clinical development activities, we may obtain or have access to sensitive personally identifiable information, including protected health information, from clinical trial participants, healthcare providers, research institutions, contract research organizations, and other third parties. While most healthcare providers and research institutions are subject to privacy and security regulations under HIPPA, as amended by HITECH, we may, depending on the nature of our relationships, be deemed a business associate or otherwise contractually subject to certain HIPAA obligations. Even where we are not directly subject to HIPAA, we may be subject to criminal liability under HIPAA in certain circumstances, including if we knowingly obtain or misuse individually identifiable health information in a manner that violates applicable law.
In addition, we are or may become subject to a variety of other federal, state, and foreign privacy and data protection laws and regulations that govern the collection, use, storage, disclosure, and protection of personal information. These include, for example, U.S. state privacy laws such as the CCPA, as amended by the CPRA, and similar laws in other jurisdictions, as well as international data protection regulations such as the European Union’s GDPR, to the extent we conduct clinical trials or otherwise process personal data outside the United States. These laws are complex, evolving, and may impose significant compliance obligations, including requirements related to consent, data minimization, cross-border data transfers, breach notification, and individual rights.
We and our collaborators, including CROs, CMOs, and other third-party service providers, may also be subject to data breach notification laws and consumer protection laws that require us to notify affected individuals, regulatory authorities, and others in the event of a data breach involving personal information. Failure to comply with these requirements could result in significant penalties, litigation, and reputational harm. We face an increasing risk of cybersecurity incidents and data breaches, including those resulting from unauthorized access, system failures, or cyberattacks. Any such incident could compromise the confidentiality, integrity, or availability of our data, including clinical trial data, disrupt our operations, delay our development programs, and expose us to regulatory enforcement actions and liability. Ensuring compliance with applicable privacy, data protection, and cybersecurity laws and regulations may require us to expend substantial resources. Claims that we or our third-party partners have violated individuals’ privacy rights or failed to adequately protect personal information, even if unfounded, could be costly to defend and could result in adverse publicity, which could materially harm our business, financial condition, and results of operations.
If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs that could have a material adverse effect on the success of our business.
We are subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and the handling, use, storage, treatment and disposal of hazardous materials and wastes. Our operations involve the use of hazardous and flammable materials, including chemicals and biological and radioactive materials. Our operations also produce hazardous waste products. We generally contract with third parties for the disposal of these materials and wastes. We cannot eliminate the risk of contamination or injury from these materials. In the event of contamination or injury resulting from our use of hazardous materials, we could be held liable for any resulting damages, and any liability could exceed our resources. We also could incur significant costs associated with civil or criminal fines and penalties. Although we maintain workers’ compensation insurance to cover us for costs and expenses we may incur due to injuries to our employees resulting from the use of hazardous materials, this insurance may not provide adequate coverage against potential liabilities. We do not maintain insurance for environmental liability or toxic tort claims that may be asserted against us in connection with our storage or disposal of biological, hazardous or radioactive materials.
If the market opportunities for OST-HER2 and our other current and future product candidates are smaller than we believe they are, our revenue may be adversely affected and our business may suffer. Moreover, because the target patient populations we are seeking to treat are small, we must be able to successfully identify patients and capture a significant market share to achieve profitability and growth.
We focus our research and product development on treatments for osteosarcoma, a rare disease. The incidence of new cases of Osteosarcoma is approximately 1,000 individuals in the United States annually and approximately 20,000 individuals globally. Given the limited number of patients with the diseases we are targeting, our ability to achieve meaningful revenue will depend on our ability to accurately identify eligible patients, successfully commercialize our product candidates, and capture a significant share of the addressable market. Our estimates of the number of patients who may be eligible for treatment with OST-HER2 or any of our other current or future product candidates are based on a variety of sources, including published literature, clinical experience, and internal analyses, and may prove to be inaccurate. Although we have completed enrollment in our Phase IIb clinical trial for OST-HER2, which enrolled a limited number of patients consistent with the rare nature of the disease, our understanding of the addressable patient population remains subject to significant uncertainty. New studies or real-world data may change the estimated incidence, prevalence, or treatable population for osteosarcoma, and the number of patients ultimately eligible for our therapies may be lower than expected. In addition, identifying, diagnosing, and referring patients with osteosarcoma, particularly in earlier stages or in certain geographic regions, can be challenging, and we may not be successful in reaching all patients who could potentially benefit from our therapies. Even if we obtain regulatory approval and achieve meaningful market penetration, the small size of the target patient population may limit our ability to generate sufficient revenue to achieve or sustain profitability.
Furthermore, because there are limited standard-of-care treatments specifically directed at Osteosarcoma, the pricing and reimbursement landscape for OST-HER2 and any other product candidates we may develop is uncertain. While therapies for rare diseases may in some cases support premium pricing, there can be significant variability in reimbursement decisions by governmental authorities and third-party payors. If we are unable to obtain adequate reimbursement at levels sufficient to support our anticipated commercial infrastructure, our ability to successfully market and sell OST-HER2 and any of our other current or future product candidates would be adversely affected.
Risks Related to Our Dependence on Third Parties
We rely, and expect to continue to rely, on third parties to conduct our ongoing and planned clinical trials for our current and future product candidates. If these third parties do not successfully carry out their contractual duties, comply with regulatory requirements or meet expected deadlines, we may not be able to obtain marketing approval for or commercialize our current and potential future product candidates and our business could be substantially harmed.
We do not have the ability to independently conduct clinical trials. We rely on medical institutions, clinical investigators, contract laboratories, and other third parties, including collaboration partners, to conduct or otherwise support our clinical trials for OST-HER2 and expect to rely on them when we begin clinical trials for OST-tADC and other current or future product candidates. We rely heavily on these parties for execution of clinical trials and control only certain aspects of their activities. Nevertheless, we are responsible for ensuring that each of our clinical trials is conducted in accordance with the applicable protocol, legal and regulatory requirements and scientific standards, and our reliance on CROs will not relieve us of our regulatory responsibilities. For any violations of laws and regulations during the conduct of our clinical trials, we could be subject to untitled and warning letters or enforcement action that may include civil penalties up to and including criminal prosecution.
We and any third parties that we contract with are required to comply with regulations and requirements, including GCP, for conducting, monitoring, recording and reporting the results of clinical trials to ensure that the data and results are scientifically credible and accurate, and that the trial patients are adequately informed of the potential risks of participating in clinical trials and their rights are protected. These regulations are enforced by the FDA, the Competent Authorities of the Member States of the European Economic Area and comparable foreign regulatory authorities for any drugs in clinical development. The FDA enforces GCP requirements through periodic inspections of clinical trial sponsors, principal investigators and trial sites. If we or the third parties we contract with fail to comply with applicable GCP, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials before approving our marketing applications. We cannot assure you that, upon inspection, the FDA will determine that any of our current or future clinical trials will comply with GCP. In addition, our clinical trials must be conducted with current or future product candidates produced under cGMP regulations. Our failure or the failure of third parties that we contract with to comply with these regulations may require us to repeat some aspects of a specific, or an entire, clinical trial, which would delay the marketing approval process and could also subject us to enforcement action. We also are required to register certain ongoing clinical trials and provide certain information, including information relating to the trial’s protocol, on a government-sponsored database, ClinicalTrials.gov, within specific timeframes. Failure to do so can result in fines, adverse publicity and civil and criminal sanctions.
Although we intend to design the clinical trials for our current or future product candidates, or be involved in the design when other parties sponsor the trials, we anticipate that third parties will conduct all of our clinical trials. As a result, many important aspects of our clinical development, including their conduct and timing, will be outside of our direct control. Our reliance on third parties to conduct future clinical trials will also result in less direct control over the management of data developed through clinical trials than would be the case if we were relying entirely upon our own staff. Communicating with outside parties can also be challenging, potentially leading to mistakes as well as difficulties in coordinating activities.
These factors may materially adversely affect the willingness or ability of third parties to conduct our clinical trials and may subject us to unexpected cost increases that are beyond our control. If our CROs do not perform clinical trials in a satisfactory manner, breach their obligations to us or fail to comply with regulatory requirements, the development, marketing approval and commercialization of our current or future product candidates may be delayed, we may not be able to obtain marketing approval and commercialize our current or future product candidates, or our development programs may be materially and irreversibly harmed. If we are unable to rely on clinical data collected by our CROs, we could be required to repeat, extend the duration of, or increase the size of any clinical trials we conduct and this could significantly delay commercialization and require significantly greater expenditures.
If any of our relationships with these third-party CROs terminate, we may not be able to enter into arrangements with alternative CROs on commercially reasonable terms, or at all. If our CROs do not successfully carry out their contractual duties or obligations or meet expected deadlines, if they need to be replaced or if the quality or accuracy of the clinical data they obtain are compromised due to the failure to adhere to our clinical protocols, regulatory requirements or for other reasons, any clinical trials such CROs are associated with may be extended, delayed or terminated, and we may not be able to obtain marketing approval for or successfully commercialize our current or future product candidates. As a result, we believe that our financial results and the commercial prospects for our current or future product candidates in the subject indication would be harmed, our costs could increase and our ability to generate revenue could be delayed.
The third parties upon whom we rely for the supply of the active pharmaceutical ingredient, or API, drug product and drug substance used in our core product candidates are limited in number, and the loss of any of these suppliers could significantly harm our business.
The API drug product and drug substance used in our core product candidates are supplied to us from a small number of suppliers, and in some cases sole source suppliers. Our ability to successfully develop our current or future product candidates, and to ultimately supply our commercial drugs in quantities sufficient to meet the market demand, depends in part on our ability to obtain the API, drug product and drug substance for these drugs in accordance with regulatory requirements and in sufficient quantities for commercialization and clinical testing. We do not currently have arrangements in place for a redundant or second-source supply of all API, drug product or drug substance in the event any of our current suppliers of such API, drug product and drug substance cease their operations for any reason.
For all of our current or future product candidates, we intend to identify and qualify additional manufacturers to provide such API, drug product and drug substance prior to submission of an NDA or a BLA to the FDA and/or an MAA to the EMA. We are not certain, however, that our single-source and dual source suppliers will be able to meet our demand for their products, either because of the nature of our agreements with those suppliers, our limited experience with those suppliers or our relative importance as a customer to those suppliers. It may be difficult for us to assess their ability to timely meet our demand in the future based on past performance. While our suppliers have generally met our demand for their products on a timely basis in the past, they may subordinate our needs in the future to their other customers.
Establishing additional or replacement suppliers for the API, drug product and drug substance used in our current or future product candidates, if required, may not be accomplished quickly. If we are able to find a replacement supplier, such replacement supplier would need to be qualified and may require additional regulatory approval, which could result in further delay. While we seek to maintain adequate inventory of the API, drug product and drug substance used in our current or future product candidates, any interruption or delay in the supply of components or materials, or our inability to obtain such API, drug product and drug substance from alternate sources at acceptable prices in a timely manner could impede, delay, limit or prevent our development efforts, which could harm our business, results of operations, financial condition and prospects.
Our success is dependent on our executive management team’s ability to successfully pursue business development, strategic partnerships and investment opportunities as our company matures. We may also form or seek strategic alliances or acquisitions or enter into additional collaboration and licensing arrangements in the future, and we may not realize the benefits of such collaborations, alliances, acquisitions or licensing arrangements.
We are party to licensing arrangements with the Trustees of the University of Pennsylvania and BlinkBio, Inc., and may in the future form or seek strategic alliances or acquisitions, create joint ventures, or enter into additional collaboration and licensing arrangements with third parties that we believe will complement or augment our development and commercialization efforts with respect to our current product candidates and any future product candidates that we may develop.
Going forward, we are seeking strategic partners for the further development and potential commercialization of our non-core and out-licensed programs, including OST-tADC. However, identifying appropriate partners is competitive, and the negotiation of these arrangements is complex, time-consuming, and resource-intensive. Potential partners may be unwilling to commit to such transactions on acceptable terms, or at all, particularly if they determine that our product candidates do not have sufficient clinical validation, commercial potential, or likelihood of regulatory approval. Even if we are successful in entering into such arrangements, we may not realize the anticipated benefits. Strategic transactions typically involve significant risks, including the potential for loss of control over certain development or commercialization activities, reliance on third parties to meet development, regulatory and commercialization milestones, disagreements or disputes with partners, which could delay or terminate development programs, reduced economic returns as a result of profit-sharing, milestone payments or royalties, and the diversion of management attention and internal resources.
In addition, these transactions may require us to incur substantial costs, record non-recurring charges, assume contingent liabilities, or issue equity securities that dilute our existing stockholders. If we are unable to successfully identify and execute strategic transactions, or if any such transactions fail to achieve their intended objectives, our ability to advance our product candidates, including OST-HER2 and our other programs, may be adversely affected, and our business, financial condition and results of operations could be materially harmed.
As a result, we may not realize the anticipated benefits of our existing collaboration and licensing arrangements or any future strategic transactions, including partnerships, acquisitions, or licensing arrangements, particularly if we are unable to effectively integrate such arrangements with our existing operations and company culture. Any such failure could delay development timelines, disrupt our business, or otherwise adversely affect our results of operations. In addition, we cannot assure that any strategic transaction or collaboration will generate the expected revenues, cost savings, or other anticipated benefits that justified our entry into such arrangement. Further, delays in identifying or entering into new collaboration or strategic partnership agreements with respect to our current or future product candidates may delay or limit the development and commercialization of such product candidates, including in specific geographies or for particular indications, which could materially harm our business prospects, financial condition and results of operations.
Our manufacturing process needs to comply with FDA regulations relating to the quality and reliability of such processes. Any failure to comply with relevant regulations could result in delays in or termination of our clinical programs and suspension or withdrawal of any regulatory approvals.
In order to produce our product candidates for clinical trials and our products, if any, for commercial purposes, either at our own facility or at a third-party’s facility, we and our third-party vendors will need to comply with the FDA’s cGMP regulations and guidelines. As part of our ongoing quality and process improvement efforts, we conducted a gap analysis of our cGMP quality system and it identified certain key areas for necessary remediation, including with regard to documentation requirements. We may encounter difficulties in achieving compliance with quality control and quality assurance requirements and may experience shortages in qualified personnel. We are subject to inspections by the FDA and comparable foreign regulatory authorities to confirm compliance with applicable regulatory requirements. Any failure to follow cGMP or other regulatory requirements, including any failure to remedy the issues identified in the cGMP gap analysis, or delay, interruption or other issues that arise in the manufacture, fill-finish, packaging, or storage of our product candidate as a result of a failure of our facilities or the facilities or operations of third parties to comply with regulatory requirements or pass any regulatory authority inspection could significantly impair our ability to develop and commercialize our current or future product candidates, including leading to significant delays in the availability of our product candidates for our clinical trials or the termination of or suspension of a clinical trial, or the delay or prevention of a filing or approval of marketing applications for our current or future product candidates. Significant non-compliance could also result in the imposition of sanctions, including warning or untitled letters, fines, injunctions, civil penalties, failure of regulatory authorities to grant marketing approvals for our current or future product candidates, delays, suspension or withdrawal of approvals, license revocation, seizures or recalls of products, operating restrictions and criminal prosecutions, any of which could damage our reputation and our business.
If our third-party manufacturers use hazardous and biological materials in a manner that causes injury or violates applicable law, we may be liable for damages.
Our research and development activities involve the controlled use of potentially hazardous substances, including chemical materials, by our third-party manufacturers. Our manufacturers are subject to federal, state and local laws and regulations in the United States governing the use, manufacture, storage, handling and disposal of medical and hazardous materials. Although we believe that our manufacturers’ procedures for using, handling, storing and disposing of these materials comply with legally prescribed standards, we cannot completely eliminate the risk of contamination or injury resulting from medical or hazardous materials. As a result of any such contamination or injury, we may incur liability or local, city, state or federal authorities may curtail the use of these materials and interrupt our business operations. In the event of an accident, we could be held liable for damages or penalized with fines, and the liability could exceed our resources. We do not have any insurance for liabilities arising from medical or hazardous materials. Compliance with applicable laws and regulations is expensive, and current or future regulations may impair our research, development and production efforts, which could harm our business, prospects, financial condition or results of operations.
Risks Related to Intellectual Property
If we and the third parties from whom we in-license intellectual property are unable to obtain, maintain, protect, or enforce patent and other intellectual property rights for our technology and product candidates, or if the scope of such protection is not sufficiently broad, our competitors could develop and commercialize similar or identical technologies and products, and our ability to successfully commercialize our product candidates may be impaired.
The patent position of biotechnology and pharmaceutical companies is highly uncertain and involves complex legal and factual questions, and has been the subject of extensive litigation in recent years. Our commercial success depends in part on our ability, and the ability of our licensors and other partners, to obtain, maintain, protect, and enforce intellectual property rights in the United States and other jurisdictions for our current and future product candidates, including OST-HER2, OST-tADC, our non-core programs, and our proprietary technologies and know-how.
We rely on a combination of owned, co-owned and in-licensed patents and patent applications to protect our intellectual property. For example, we in-license and co-own certain intellectual property relating to OST-HER2 from the University of Pennsylvania, and we in-license intellectual property relating to OST-tADC from BlinkBio, Inc. The intellectual property we own includes six granted U.S. utility patents and a number of foreign patents and pending patent applications. The patents and patent applications if granted are expected to expire between 2029 and 2038, not including any patent term extension. The intellectual property licensed from BlinkBio, Inc. includes six granted U.S. utility patents and a number of foreign patents and pending patent applications. The patents cover methods of use of silicon-based drug conjugates and silanol based therapeutic payloads. The patents and pending patent applications if granted are expected to expire between 2036 and 2037, not including any patent term extension. For additional information about our patents, see “ Business — Our Intellectual Property .”
The degree of patent protection required to successfully commercialize our product candidates may be unavailable or limited. We cannot assure that any of the patents we own or in-license, or any pending patent applications, will issue with claims of sufficient scope to protect our product candidates or provide a meaningful competitive advantage. If the scope or strength of our intellectual property protection is reduced or challenged, it could adversely affect our ability to attract collaborators or commercial partners.
Third parties may have developed or may develop technologies that compete with ours, and they may have filed or may file patent applications, or may obtain patents, that overlap with or conflict with our intellectual property. Because patent applications are typically not published until 18 months after filing and scientific publications often lag behind discoveries, we cannot be certain that we or our licensors were the first to make the inventions claimed in our patents or pending applications. As a result, the validity, enforceability, scope, and commercial value of our intellectual property cannot be predicted with certainty.
Even if our patents are issued, they may be challenged, invalidated, narrowed, or held unenforceable in administrative proceedings or litigation in the United States or abroad. Such challenges could result in the loss of exclusivity, freedom to operate, or other competitive advantages. Furthermore, given the time required for clinical development and regulatory review, patent protection for our product candidates may expire before or shortly after commercialization, thereby limiting our ability to realize the full commercial value of our intellectual property. Any failure to obtain, maintain, protect, or enforce our intellectual property rights, or any loss or narrowing of such rights, could allow third parties to use our technology or develop competing products and could materially adversely affect our business, financial condition, results of operations, and prospects.
If our trademarks and trade names for our products or company name are not adequately protected in one or more countries where we intend to market our products, we may delay the launch of product brand names, use different trademarks or tradenames in different countries, or face other potentially adverse consequences to building our product brand recognition.
Our trademarks or trade names may be challenged, infringed, diluted, circumvented or declared generic or determined to be infringing on other marks. We intend to rely on both registration and common law protection for our trademarks. We may not be able to protect our rights to these trademarks and trade names or may be forced to stop using these names, which we need for name recognition by potential partners or customers in our markets of interest. During the trademark registration process, we may receive Office Actions from the USPTO or from comparable agencies in foreign jurisdictions objecting to the registration of our trademark. Although we would be given an opportunity to respond to those objections, we may be unable to overcome such rejections. In addition, in the USPTO and in comparable agencies in many foreign jurisdictions, third parties are given an opportunity to oppose pending trademark applications and/or to seek the cancellation of registered trademarks. Opposition or cancellation proceedings may be filed against our trademark applications or registrations, and our trademark applications or registrations may not survive such proceedings. If we are unable to obtain a registered trademark or establish name recognition based on our trademarks and trade names, we may not be able to compete effectively and our business may be adversely affected.
If we are unable to adequately protect and enforce our trade secrets, our business and competitive position would be harmed.
In addition to the protection afforded by patents we may own, co-own or in-license, we seek to rely on trade secret protection, confidentiality agreements, and license agreements to protect proprietary know-how that may not be patentable, processes for which patents are difficult to enforce and any other elements of our product discovery and development processes that involve proprietary know-how, information, or technology that may not be covered by patents. Although it is our policy to require all of our employees, consultants, advisors and any third parties who have access to our proprietary know-how, information or technology to enter into confidentiality and assignment of inventions agreements, trade secrets can be difficult to protect and we have limited control over the protection of trade secrets used by our collaborators and suppliers. We cannot be certain that we have or will obtain these agreements in all circumstances and we cannot guarantee that we have entered into such agreements with each party that may have or have had access to our trade secrets or proprietary information.
Moreover, any of these parties might breach the agreements and intentionally or inadvertently disclose our trade secret information and we may not be able to obtain adequate remedies for such breaches. In addition, competitors may otherwise gain access to our trade secrets or independently develop substantially equivalent information and techniques. Further, the laws of some foreign countries do not protect proprietary rights and trade secrets to the same extent or in the same manner as the laws of the United States. As a result, we may encounter significant problems in protecting and defending our intellectual property both in the United States and abroad. If we are unable to prevent unauthorized material disclosure of our intellectual property to third parties, we will not be able to establish or maintain a competitive advantage in our market, which could materially adversely affect our business, financial condition, results of operations and future prospects.
Enforcing a claim that a party illegally disclosed or misappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable. If we choose to go to court to stop a third-party from using any of our trade secrets, we may incur substantial costs. These lawsuits may consume our time and other resources even if we are successful. Although we take steps to protect our proprietary information and trade secrets, including through contractual means with our employees and consultants, third parties may independently develop substantially equivalent proprietary information and techniques or otherwise gain access to our trade secrets or disclose our technology. If any of our trade secrets were to be lawfully obtained or independently developed by a competitor or other third-party, we would have no right to prevent them from using that technology or information to compete with us.
We may initiate, become a defendant in, or otherwise become party to lawsuits to protect or enforce our intellectual property rights, which could be expensive, time-consuming and unsuccessful.
Competitors may infringe any patents we may own, co-own or in-license. In addition, any patents we may own, co-own or in-license also may become involved in inventorship, priority, validity or unenforceability disputes. To counter infringement or unauthorized use, we may be required to file infringement claims, which can be expensive and time-consuming. We may not prevail in any lawsuits that we initiate, and the damages or other remedies awarded, if any, may not be commercially meaningful. In addition, in an infringement proceeding, a court may decide that one or more of any patents we may own, co-own or in-license is not valid or is unenforceable or that the other party’s use of our technology that may be patented falls under the safe harbor to patent infringement under 35 U.S.C. § 271(e)(1). There is also the risk that, even if the validity of these patents is upheld, the court may refuse to stop the other party from using the technology at issue on the grounds that any patents we may own, co-own or in-license do not cover the technology in question or that such third-party’s activities do not infringe the patent applications or any patents we in-license or may in the future own or co-own. An adverse result in any litigation or defense proceedings could put one or more of any patents we may own, co-own or in-license at risk of being invalidated, held unenforceable, or interpreted narrowly and could put those patent applications at risk of not issuing. Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing, patient support or distribution activities. We may not have sufficient financial or other resources to conduct such litigation or proceedings adequately. Some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources and more mature and developed intellectual property portfolios. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could have a material adverse effect on our ability to compete in the marketplace.
Post-grant proceedings provoked by third parties or brought by the USPTO may be necessary to determine the validity or priority of inventions with respect to the patent applications or any patents we in-license or may in the future own or co-own. These proceedings are expensive and an unfavorable outcome could result in a loss of our current patent rights and could require us to cease using the related technology or to attempt to license rights to it from the prevailing party. Our business could be harmed if the prevailing party does not offer us a license on commercially reasonable terms. In addition to potential USPTO post-grant proceedings, we may become a party to patent opposition proceedings in the EPO, or similar proceedings in other foreign patent offices or courts where these patents may be challenged. The costs of these proceedings could be substantial, and may result in a loss of scope of some claims or a loss of the entire patent. An unfavorable result in a post-grant challenge proceeding may result in the loss of our right to exclude others from practicing one or more of our inventions in the relevant country or jurisdiction, which could have a material adverse effect on our business. Litigation or post-grant proceedings within patent offices may result in a decision adverse to our interests and, even if we are successful, may result in substantial costs and distract our management and other employees. We may not be able to prevent, misappropriation of our trade secrets or confidential information, particularly in countries where the laws may not protect those rights as fully as in the United States.
We may not be able to detect infringement against any patents we may own, co-own or in-license. Even if we detect infringement by a third-party of any patents we may own, co-own or in-license, we may choose not to pursue litigation against or settlement with the third-party. If we later sue such third-party for patent infringement, the third-party may have certain legal defenses available to it, which otherwise would not be available except for the delay between when the infringement was first detected and when the suit was brought. Such legal defenses may make it impossible for us to enforce any patents we may own, co-own or in-license against such third-party.
Intellectual property litigation and administrative patent office patent validity challenges in one or more countries could cause us to spend substantial resources and distract our personnel from their normal responsibilities.
Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses, and could distract our technical and management personnel from their normal responsibilities. In addition, there could be public announcements of the results of hearings, motions or other interim proceedings or developments and if securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of our common stock. Such litigation or proceedings could substantially increase our operating losses and reduce the resources available for development activities or any future sales, marketing, patient support or distribution activities. We may not have sufficient financial or other resources to conduct such litigation or proceedings adequately. As noted above, some of our competitors may be able to sustain the costs of such litigation or proceedings more effectively than we can because of their greater financial resources. Uncertainties resulting from the initiation and continuation of patent litigation or other proceedings could compromise our ability to compete in the marketplace, including compromising our ability to raise the funds necessary to continue our clinical trials, continue our research programs, license necessary technology from third parties, or enter into development collaborations that would help us commercialize our current or future product candidates, if approved. Any of the foregoing events would harm our business, financial condition, results of operations and prospects.
We may be unable to obtain patent or other intellectual property protection for our current or future product candidates or our future products, if any, in all jurisdictions throughout the world, and we may not be able to adequately enforce our intellectual property rights even in the jurisdictions where we seek protection.
We may not be able to pursue patent coverage of our current or future product candidates in all countries. Filing, prosecuting and defending patents on current or future product candidates in all countries throughout the world would be prohibitively expensive, and intellectual property rights in some countries outside the United States can be less extensive than those in the United States. In addition, the laws of some foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the United States. Consequently, we may not be able to prevent third parties from practicing our inventions in all countries outside the United States, or from selling or importing products made using our inventions in and into the United States or other jurisdictions. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and further, may export otherwise infringing products to territories where we have patent protection, but where enforcement is not as strong as that in the United States. These products may compete with our current or future product candidates and our current intellectual property rights may not be effective or sufficient to prevent them from competing.
Many companies have encountered significant problems in protecting and defending intellectual property rights in foreign jurisdictions. The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents, trade secrets and other intellectual property protection, particularly those relating to pharmaceutical products, which could make it difficult for us to stop the infringement of any patents we may own, co-own or in-license or marketing of competing products in violation of our proprietary rights generally.
Many countries have compulsory licensing laws under which a patent owner may be compelled to grant licenses to third parties. In addition, many countries limit the enforceability of patents against government agencies or government contractors. In these countries, the patent owner may have limited remedies, which could materially diminish the value of such patent. If we are forced to grant a license to third parties with respect to any patents we may own or license that are relevant to our business, our competitive position may be impaired, and our business, financial condition, results of operations, and prospects may be adversely affected.
We may not obtain or grant licenses or sublicenses to intellectual property rights in all markets on equally or sufficiently favorable terms with third parties.
It may be necessary for us to use the patented or proprietary technology of third parties to commercialize our products, in which case we would be required to obtain a license from these third parties. The licensing of third-party intellectual property rights is a competitive area, and more established companies may pursue strategies to license or acquire third-party intellectual property rights that we may consider attractive or necessary. More established companies may have a competitive advantage over us due to their size, capital resources and greater clinical development and commercialization capabilities. In addition, companies that perceive us to be a competitor may be unwilling to assign or license rights to us. We also may be unable to license or acquire third-party intellectual property rights on terms that would allow us to make an appropriate return on our investment or at all. If we are unable to license such technology, or if we are forced to license such technology on unfavorable terms, our business could be materially harmed. If we are unable to obtain a necessary license, we may be unable to develop or commercialize the affected current or future product candidates, which could materially harm our business, and the third parties owning such intellectual property rights could seek either an injunction prohibiting our sales, or, with respect to our sales, an obligation on our part to pay royalties or other forms of compensation. Even if we are able to obtain a license, it may be non-exclusive, thereby giving our competitors access to the same technologies licensed to us. Any of the foregoing could harm our competitive position, business, financial condition, results of operations and prospects.
If we fail to comply with our obligations in any agreements under which we may license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose license rights that are important to our business.
We may from time to time be party to license and collaboration agreements with third parties to advance our research or allow commercialization of current or future product candidates. Such agreements may impose numerous obligations, such as development, diligence, payment, commercialization, funding, milestone, royalty, sublicensing, insurance, patent prosecution, enforcement and other obligations on us and may require us to meet development timelines, or to exercise commercially reasonable efforts to develop and commercialize licensed products, in order to maintain the licenses. In spite of our best efforts, our licensors might conclude that we have materially breached our license agreements and might therefore terminate the license agreements, thereby removing or limiting our ability to develop and commercialize products and technologies covered by these license agreements.
Any termination of these licenses, or if the underlying patents fail to provide the intended exclusivity, could result in the loss of significant rights and could harm our ability to commercialize our current or future product candidates, and competitors or other third parties would have the freedom to seek regulatory approval of, and to market, products identical to ours and we may be required to cease our development and commercialization of certain of our current or future product candidates. Any of the foregoing could have a material adverse effect on our competitive position, business, financial conditions, results of operations, and prospects.
In addition, the agreements under which we may license intellectual property or technology from third parties are likely to be complex, and certain provisions in such agreements may be susceptible to multiple interpretations. The resolution of any contract interpretation disagreement that may arise could narrow what we believe to be the scope of our rights to the relevant intellectual property or technology, or increase what we believe to be our financial or other obligations under the relevant agreement, either of which could have a material adverse effect on our business, financial condition, results of operations and prospects. Moreover, if disputes over intellectual property that we may license prevent or impair our ability to maintain future licensing arrangements on acceptable terms, we may be unable to successfully develop and commercialize the affected current or future product candidates, which could have a material adverse effect on our business, financial conditions, results of operations and prospects.
Changes in patent law could diminish the value of patents in general, thereby impairing our ability to protect our current or future product candidates.
As is the case with other biopharmaceutical companies, our success is heavily dependent on intellectual property, particularly patents. Obtaining and enforcing patents in the biopharmaceutical industry involves both technological and legal complexity and is therefore costly, time consuming and inherently uncertain. Recent patent reform legislation in the United States and other countries, including the Leahy-Smith America Invents Act, or Leahy-Smith Act, signed into law on September 16, 2011, could increase those uncertainties and costs. The Leahy-Smith Act includes a number of significant changes to U.S. patent law. These include provisions that affect the way patent applications are prosecuted, redefine prior art and provide more efficient and cost-effective avenues for competitors to challenge the validity of patents. In addition, the Leahy-Smith Act has transformed the U.S. patent system into a “first inventor to file” system. The first-inventor-to-file provisions, however, only became effective on March 16, 2013. Accordingly, it is not yet clear what, if any, impact the Leahy-Smith Act will have on the operation of our business. However, the Leahy-Smith Act and its implementation could make it more difficult to obtain patent protection for our inventions and increase the uncertainties and costs surrounding the prosecution of the patent applications that we have in-licensed and the enforcement or defense of such issued patents, all of which could harm our business, results of operations and financial condition.
The U.S. Supreme Court has and other courts have ruled on several patent cases in recent years, either narrowing the scope of patent protection available in certain circumstances or weakening the rights of patent owners in certain situations. Additionally, there have been recent proposals for additional changes to the patent laws of the United States and other countries that, if adopted, could impact our ability to obtain patent protection for our proprietary technology or our ability to enforce our proprietary technology. Depending on future actions by the U.S. Congress, the U.S. courts, the USPTO and the relevant law-making bodies in other countries, the laws and regulations governing patents could change in unpredictable ways that would weaken our ability to obtain new patents or to enforce our existing patents and patents that we might obtain in the future.
Intellectual property rights do not guarantee commercial success of current or future product candidates or other business activities. Numerous factors may limit any potential competitive advantage provided by our intellectual property rights.
The degree of future protection afforded by our intellectual property rights, whether owned, co-owned or in-licensed, is uncertain because intellectual property rights have limitations, and may not adequately protect our business, provide a barrier to entry against our competitors or potential competitors, or permit us to maintain our competitive advantage. Moreover, if a third party has intellectual property rights that cover the practice of our technology, we may not be able to fully exercise or extract value from our intellectual property rights. The following examples are illustrative:
patent applications that we own, co-own or may in-license may not lead to issued patents;
patents, should they issue, that we may own, co-own or in-license, may not provide us with any competitive advantages, may be narrowed in scope, or may be challenged and held invalid or unenforceable;
others may be able to develop and/or practice technology, including compounds that are similar to the chemical compositions of our current or future product candidates, that is similar to our technology or aspects of our technology but that is not covered by the claims of any patents we may own, co-own or in-license, should any patents issue;
third parties may compete with us in jurisdictions where we do not pursue and obtain patent protection;
we, or our future licensors or collaborators, might not have been the first to make the inventions covered by a patent application that we own, co-own or may in-license;
we, or our future licensors or collaborators, might not have been the first to file patent applications covering a particular invention;
others may independently develop similar or alternative technologies without infringing, misappropriating or otherwise violating our intellectual property rights;
our competitors might conduct research and development activities in the United States and other countries that provide a safe harbor from patent infringement claims for certain research and development activities, as well as in countries where we do not have patent rights, and may then use the information learned from such activities to develop competitive products for sale in our major commercial markets;
we may not be able to obtain and/or maintain necessary licenses on reasonable terms or at all;
third parties may assert an ownership interest in our intellectual property and, if successful, such disputes may preclude us from exercising exclusive rights, or any rights at all, over that intellectual property;
we may choose not to file a patent in order to maintain certain trade secrets or know-how, and a third-party may subsequently file a patent covering such trade secrets or know-how;
we may not be able to maintain the confidentiality of our trade secrets or other proprietary information; and
we may not develop or in-license additional proprietary technologies that are patentable.
Should any of these events occur, they could significantly harm our business, financial condition, results of operations and prospects.
Risks Related to Employee Matters, Managing Growth and Other Risks Related to Our Business
Our current operations are located in Maryland; and we or the third parties upon whom we depend may be adversely affected by natural disasters and our business continuity and disaster recovery plans may not adequately protect us from a serious disaster.
Our current operations are located in Maryland. Any unplanned event, such as flood, fire, explosion, earthquake, extreme weather condition, medical epidemics, power shortage, telecommunication failure or other natural or man-made accidents or incidents that result in us being unable to fully utilize our facilities, or the manufacturing facilities of our third-party contract manufacturers, may have a material and adverse effect on our ability to operate our business, particularly on a daily basis, and have significant negative consequences on our financial and operating conditions. Loss of access to these facilities may result in increased costs, delays in the development of our product candidates or interruption of our business operations. Natural disasters or pandemics could further disrupt our operations, and have a material and adverse effect on our business, financial condition, results of operations and prospects. If a natural disaster, power outage or other event occurred that prevented us from using all or a significant portion of our headquarters, that damaged critical infrastructure, such as the manufacturing facilities of our third-party contract manufacturers, or that otherwise disrupted operations, it may be difficult or, in certain cases, impossible, for us to continue our business for a substantial period of time. The disaster recovery and business continuity plans we have in place may prove inadequate in the event of a serious disaster or similar event. We may incur substantial expenses as a result of the limited nature of our disaster recovery and business continuity plans, which could have a material adverse effect on our business. As part of our risk management policy, we maintain insurance coverage at levels that we believe are appropriate for our business. However, in the event of an accident or incident at these facilities, we cannot assure our investors that the amounts of insurance will be sufficient to satisfy any damages and losses. If the manufacturing facilities of our third-party contract manufacturers are unable to operate because of an accident or incident or for any other reason, even for a short period of time, any or all of our research and development programs may be harmed. Any business interruption may have a material and adverse effect on our business, financial condition, results of operations and prospects.
Our future success depends on our ability to retain key executives and to attract, retain and motivate qualified personnel.
We are highly dependent on the research and development, clinical and business development expertise of Paul A. Romness, MPH, our Chairman, President and Chief Executive Officer, and Robert G. Petit, Ph.D., our Chief Medical Officer and Chief Scientific Officer, as well as the other principal members of our management, scientific and clinical teams. Although we have entered into employment agreements or arrangements with our executive officers, each of them may terminate their employment with us at any time. We do not maintain “key person” insurance for any of our executives or other employees. In addition, we rely on consultants and advisors, including scientific and clinical advisors, to assist us in formulating our research and development and commercialization strategy. Our consultants and advisors may be employed by employers other than us and may have commitments under consulting or advisory contracts with other entities that may limit their availability to us. If we are unable to continue to attract and retain high quality personnel, our ability to pursue our growth strategy will be limited.
Recruiting and retaining qualified scientific, clinical, manufacturing and sales and marketing personnel will also be critical to our success. The loss of the services of our executive officers or other key employees could impede the achievement of our research, development and commercialization objectives and seriously harm our ability to successfully implement our growth strategy. Further, replacing executive officers and key employees may be difficult and may take an extended period of time because of the limited number of individuals in our industry with the breadth of skills and experience required to successfully develop, gain regulatory approval of and commercialize drugs. Competition to hire from this limited pool is intense, and we may be unable to hire, train, retain or motivate these key personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnology companies for similar personnel. We also experience competition for the hiring of scientific and clinical personnel from universities and research institutions. Failure to succeed in clinical trials may make it more challenging to recruit and retain qualified scientific personnel.
We will need to develop and expand our company, and we may encounter difficulties in managing this development and expansion, which could disrupt our operations.
As of March 26, 2026, we had four full-time employees, one part-time employee and a limited number of regulatory and other consultants. We expect to increase our number of employees and the scope of our operations. To manage our anticipated growth and expansion, we must continue to implement and improve our managerial, operational and financial systems, expand our facilities and continue to recruit and train additional qualified personnel. Also, our management may need to divert a disproportionate amount of its attention away from its day-to-day activities and devote a substantial amount of time to managing these growth-oriented activities. Due to our limited resources, we may not be able to effectively manage the expansion of our operations or recruit and train additional qualified personnel. This may result in weaknesses in our infrastructure, give rise to operational mistakes, loss of business opportunities, loss of employees and reduced productivity among remaining employees. The physical expansion of our operations may lead to significant costs and may divert financial resources from other projects, such as the development of our current or future product candidates. If our management is unable to effectively manage our expected growth and expansion, our expenses may increase more than expected, our ability to generate or increase our revenue could be reduced and we may not be able to implement our growth strategy. Our future financial performance and our ability to commercialize our current or future product candidates, if approved, and compete effectively will depend, in part, on our ability to effectively manage the future growth and expansion of our company.
Failure to maintain effective internal controls in accordance with Section 404 of the Sarbanes-Oxley Act could have a material adverse effect on our business, results of operation or financial condition. In addition, current and potential stockholders could lose confidence in our financial reporting, which could have a material adverse effect on the price of the common stock.
Effective internal controls are necessary for us to provide reliable financial reports and effectively prevent fraud. We are required to document and test our internal control procedures in order to satisfy the requirements of Section 404 of the Sarbanes-Oxley Act, which requires annual management assessments of the effectiveness of our internal control over financial reporting. In addition, if we fail to maintain the adequacy of our internal controls, as such standards are modified, supplemented or amended from time to time, we may not be able to ensure that we can conclude on an ongoing basis that we have effective internal controls over financial reporting in accordance with Section 404. Our management has concluded that our internal control over financial reporting was not effective as of December 31, 2025 due inadequate segregation of duties as a result of limited personnel and insufficient written policies and procedures for accounting, information technology and financial reporting (no control procedures in place). Disclosing deficiencies or weaknesses in our internal controls, failing to remediate these deficiencies or weaknesses in a timely fashion or failing to achieve and maintain an effective internal control environment may cause investors to lose confidence in our reported financial information, which could have a material adverse effect on the price of the common stock. If we cannot provide reliable financial reports or prevent fraud, our operating results could be harmed.
Unstable market and economic conditions may have serious adverse consequences on our business, financial condition and stock price.
Global financial markets have experienced extreme volatility and disruptions in the past several years, including severely diminished liquidity and credit availability, declines in consumer confidence, declines in economic growth, increases in inflation and unemployment rates and uncertainty about economic stability. There can be no assurance that further deterioration in financial markets and confidence in economic conditions will not occur. Our general growth strategy may be adversely affected by any such economic downturn, volatile business environment or continued unpredictable and unstable market conditions. If the current equity and credit markets deteriorate, or do not improve, it may make any necessary debt or equity financing more difficult, more costly and more dilutive. Failure to secure any necessary financing in a timely manner and on favorable terms could have a material adverse effect on our growth strategy, financial performance and stock price and could require us to delay, scale back or discontinue the development and commercialization of one or more of our product candidates or delay our pursuit of potential in-licenses or acquisitions. In addition, there is a risk that one or more of our current service providers, manufacturers and other partners may not survive these difficult economic times, which could directly affect our ability to attain our operating goals on schedule and on budget.
Our internal computer systems, or those of our third-party CROs or other contractors or consultants, may fail or suffer security breaches, which could result in a material disruption of our current or future product candidates’ development programs.
Despite the implementation of security measures, our internal computer systems and those of our third-party CROs and other contractors and consultants are vulnerable to damage from computer viruses, unauthorized access, natural disasters, terrorism, war and telecommunication and electrical failures. While we have not experienced any such system failure, accident, or security breach to date, if such an event were to occur and cause interruptions in our operations, it could result in a material disruption of our programs. For example, the loss of clinical trial data for our current or future product candidates could result in delays in our regulatory approval efforts and significantly increase our costs to recover or reproduce the data. To the extent that any disruption or security breach results in a loss of or damage to our data or applications or other data or applications relating to our technology or current or future product candidates, or inappropriate disclosure of confidential or proprietary information, we could incur liabilities and the further development of our current or future product candidates could be delayed.
We may be unable to adequately protect our information systems from cyberattacks, which could result in the disclosure of confidential or proprietary information, including personal data, damage our reputation, and subject us to significant financial and legal exposure.
We rely on information technology systems that we or our third-party providers operate to process, transmit and store electronic information in our day-to-day operations. In connection with our product discovery efforts, we may collect and use a variety of personal data, such as name, mailing address, email addresses, phone number and clinical trial information. A successful cyberattack could result in the theft or destruction of intellectual property, data or other misappropriation of assets, or otherwise compromise our confidential or proprietary information and disrupt our operations. Cyberattacks are increasing in their frequency, sophistication and intensity, and have become increasingly difficult to detect. Cyberattacks could include wrongful conduct by hostile foreign governments, industrial espionage, wire fraud and other forms of cyber fraud, the deployment of harmful malware, denial-of-service, social engineering fraud or other means to threaten data security, confidentiality, integrity and availability. A successful cyberattack could cause serious negative consequences for us, including, without limitation, the disruption of operations, the misappropriation of confidential business information, including financial information, trade secrets, financial loss and the disclosure of corporate strategic plans. Although we devote resources to protect our information systems, we realize that cyberattacks are a threat, and there can be no assurance that our efforts will prevent information security breaches that would result in business, legal, financial or reputational harm to us, or would have a material adverse effect on our results of operations and financial condition. Any failure to prevent or mitigate security breaches or improper access to, use of, or disclosure of our clinical data or patients’ personal data could result in significant liability under state (e.g., state breach notification laws), federal (e.g., HIPAA, as amended by HITECH), and international law (e.g., the EU GDPR) and may cause a material adverse impact to our reputation, affect our ability to use collected data, conduct new studies and potentially disrupt our business.
We rely on our third-party providers to implement effective security measures and identify and correct for any such failures, deficiencies or breaches. We also rely on our employees and consultants to safeguard their security credentials and follow our policies and procedures regarding use and access of computers and other devices that may contain our sensitive information. If we or our third-party providers fail to maintain or protect our information technology systems and data integrity effectively or fail to anticipate, plan for or manage significant disruptions to our information technology systems, we or our third-party providers could have difficulty preventing, detecting and controlling such cyber-attacks and any such attacks could result in losses described above as well as disputes with physicians, patients and our partners, regulatory sanctions or penalties, increases in operating expenses, expenses or lost revenues or other adverse consequences, any of which could have a material adverse effect on our business, results of operations, financial condition, prospects and cash flows. Any failure by such third parties to prevent or mitigate security breaches or improper access to or disclosure of such information could have similarly adverse consequences for us. If we are unable to prevent or mitigate the impact of such security or data privacy breaches, we could be exposed to litigation and governmental investigations, which could lead to a potential disruption to our business.
Our employees, principal investigators, CROs and consultants may engage in misconduct or other improper activities, including non-compliance with regulatory standards and requirements and insider trading.
We are exposed to the risk that our employees, principal investigators, CROs and consultants may engage in fraudulent conduct or other illegal activity. Misconduct by these parties could include intentional, reckless and/or negligent conduct or disclosure of unauthorized activities to us that violate the regulations of the FDA and other regulatory authorities, including those laws requiring the reporting of true, complete and accurate information to such authorities; healthcare fraud and abuse laws and regulations in the United States and abroad; or laws that require the reporting of financial information or data accurately. In particular, sales, marketing, patient support and business arrangements in the healthcare industry are subject to extensive laws and regulations intended to prevent fraud, misconduct, kickbacks, self-dealing and other abusive practices. These laws and regulations may restrict or prohibit a wide range of pricing, discounting, marketing and promotion, sales commission, customer incentive programs and other business arrangements. Activities subject to these laws also involve the improper use of information obtained in the course of clinical trials or creating fraudulent data in our preclinical studies or clinical trials, which could result in regulatory sanctions and cause serious harm to our reputation. We have adopted a code of conduct applicable to all of our employees, but it is not always possible to identify and deter misconduct by employees and other third parties, and the precautions we take to detect and prevent this activity may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with these laws or regulations. Additionally, we are subject to the risk that a person could allege such fraud or other misconduct, even if none occurred. If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business, including the imposition of civil, criminal and administrative penalties, damages, monetary fines, possible exclusion from participation in Medicare, Medicaid and other federal and state healthcare programs, debarment from participation in any FDA-related activities, contractual damages, reputational harm, diminished profits and future earnings, and curtailment of our operations, any of which could adversely affect our ability to operate our business and our results of operations.
Efforts to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations will involve substantial costs. Because of the breadth of these laws and the narrowness of the statutory exceptions and safe harbors available, it is possible that some of our business activities could be subject to challenge under one or more of such laws. It is possible that governmental authorities will conclude that our business practices may not comply with current or future statutes, regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our operations are found to be in violation of any of these laws or any other governmental regulations that may apply to us, we may be subject to significant criminal, civil and administrative sanctions including monetary penalties, damages, fines, disgorgement, individual imprisonment, and exclusion from participation in government funded healthcare programs, such as Medicare and Medicaid, debarment from participation in any FDA-related activities, additional reporting requirements and oversight if we become subject to a corporate integrity agreement or similar agreement to resolve allegations of non-compliance with these laws, reputational harm, and we may be required to curtail or restructure our operations, any of which could adversely affect our ability to operate our business and our results of operations.
Use of artificial intelligence, machine learning and algorithmic tools involves risks that could materially and adversely affect our business, results of operations and development timelines.
We use, and expect to increasingly use, artificial intelligence (“AI”), machine learning (“ML”) and other data-driven models to support discovery, clinical trial design and operations, manufacturing planning, and other aspects of our business. The effectiveness of these systems depends on the quality and completeness of the underlying data, proper training and validation of models, human oversight, and adherence to applicable regulatory expectations. Errors, bias, software vulnerabilities, or misuse of AI, ML, or other algorithmic tools could compromise scientific conclusions, produce flawed clinical trial protocols, generate inaccurate operational or manufacturing forecasts, or result in breaches of data integrity or privacy. Such events could delay development, regulatory review, or commercialization, or could otherwise adversely affect patient safety.
Regulatory authorities, including FDA, the EMA and other global agencies, are increasingly scrutinizing the use of AI and ML in drug development, including claims regarding AI-assisted decision making. If our public statements regarding AI capabilities are inaccurate, incomplete, or become outdated, or if regulators adopt new expectations regarding AI governance, model validation, data transparency, or reporting in drug development, we could face investigations, enforcement actions, fines, or litigation. Such regulatory scrutiny could also require operational rework, additional documentation or validation, and delays in regulatory submissions or approvals.
In addition, malfunction, misuse, bias, or inaccuracies in AI, ML, or other algorithmic tools could materially and adversely affect the quality and reliability of our research and development programs, clinical data, manufacturing processes, and commercial operations. Any of these outcomes could materially delay development timelines, increase costs, negatively impact regulatory review, and reduce our ability to successfully develop, obtain marketing approval for, or commercialize our product candidates, which could have a material adverse effect on our business, financial condition, and prospects.
Geopolitical events, trade restrictions, sanctions and export controls could adversely affect our clinical operations, supply chain, data flows and financial transactions.
Our development, manufacturing, and supply chains, as well as certain aspects of our research and clinical programs, may be adversely affected by geopolitical events, including military conflicts, escalating international tensions, trade or investment restrictions, sanctions regimes, export controls, or retaliatory measures by foreign governments. Such actions could limit our ability, or the ability of our CROs, CMOs and other third-party partners, to source active pharmaceutical ingredients, starting materials, consumables, equipment, or software, to transfer biological materials, clinical samples, or data across international borders, or to make or receive payments in affected jurisdictions.
Geopolitical events may also increase cybersecurity threats targeting life sciences companies, potentially compromising sensitive research, patient data, or operational systems. Any of these factors could result in delays in our clinical trials, interruptions in the supply of materials or products, increased costs, or the need to rapidly identify and qualify alternative suppliers, manufacturing sites, or trial locations. Such disruptions could materially and adversely affect our business, results of operations, development timelines, and prospects for successfully developing and commercializing our product candidates.
Risks Related to Our Common Stock
A significant number of additional shares of our common stock may be issued pursuant to outstanding preferred stock, warrants, stock options and convertible securities, which issuances could substantially dilute existing stockholders and may depress the market price of our common stock.
We have issued, and may issue in the future, a significant number of shares of our common stock upon the conversion or exercise of outstanding preferred stock, warrants, stock options and other convertible securities.
In connection with the PIPE Financing completed in December 2024 and January 2025, we issued shares of our Series A Preferred Stock, each share of which is convertible into a number of shares of common stock at a conversion ratio equal to (x) the original issue price of the Series A Preferred Stock divided by (y) the conversion price of the Series A Preferred Stock. The Series A Preferred Stock was initially convertible at a 1:1 ratio, subject to adjustment as set forth in the Certificate of Designation, Preferences, Rights and Limitations of Series A Senior Convertible Preferred Stock. The number of shares of common stock into which the Series A Preferred Stock may be converted is also subject to potential increase pursuant to applicable resets and anti-dilution adjustments.
During 2025 and the first quarter of 2026, we completed three warrant exercise inducement and exchange transactions pursuant to which holders exercised certain existing warrants for cash and received new warrants to purchase a number of shares of our common stock equal to the number of shares received upon such exercise. In March 2026, we completed the Bridge Financing that included the issuance of the Bridge Notes and the Bridge Warrants. The Bridge Notes are convertible into shares of our common stock under certain circumstances. If we complete a “Qualified Offering,” defined as a registered public offering or registered direct offering resulting in at least $2.5 million in gross proceeds from new money investments, the outstanding principal, together with all accrued and unpaid interest, will automatically convert into the securities sold in such offering at the offering price. Additionally, prior to any such Qualified Offering or repayment of the Bridge Notes, holders may elect to convert the Bridge Notes, in whole or in part, into shares of our common stock at a conversion price equal to 90% of the average daily volume-weighted average price of our common stock during the 10 trading days immediately preceding the holder’s conversion notice, subject to adjustment.
As of the date of this Form 10-K, (i) 1,401,786 shares of our common stock are issuable upon the conversion of outstanding shares of our Series A Preferred Stock, with a conversion ratio of 3.571429 per share, (ii) 10,399,522 shares of our common stock are issuable upon the exercise of outstanding warrants, (iii) 6,771,250 shares of our common stock are issuable upon the exercise of outstanding and vested stock options under our 2023 Incentive Compensation Plan and (iv) 1,392,805 shares of our common stock are issuable upon the conversion of outstanding Bridge Notes (assuming full conversion of such notes into common stock at a conversion price of $1.39, which was the closing price of our common stock on March 26, 2026).
As a result of these features, declines in the market price of our common stock or future issuances of securities at lower prices could result in additional shares of our common stock becoming issuable upon conversion or exercise of these securities. The issuance of such shares would substantially dilute the ownership interests and voting power of existing stockholders.
In addition, we have agreed, or may agree, to file registration statements covering the resale of shares of common stock issuable upon conversion or exercise of these securities. The availability of a significant number of shares for sale in the public market, or the perception that such sales could occur, could adversely affect the market price of our common stock and make it more difficult for us to raise additional capital in the future.
Sales of a substantial number of shares of our common stock, including those issued pursuant to the Sales Agreement, could cause the market price of our common stock to decline.
The sale of a substantial number of shares of our common stock in the public market, or the perception that such sales may occur, could cause the market price of our common stock to decline. Although we cannot predict the exact number of shares that may be sold pursuant to the Sales Agreement or the price at which any sales may occur, the issuance and sale of up to $17,469,838 of our common stock pursuant to the Sales Agreement may result in the issuance of 12,568,228 additional shares (based on an assumed offering price of $1.39 per share, the closing price of our common stock on the NYSE American on March 26, 2026). Based on our shares outstanding as of March 26, 2026, and assuming full issuance of such shares, we would have 52,101,455 shares of common stock outstanding (excluding any shares issuable upon the conversion or exercise, as applicable, of outstanding convertible notes, preferred stock, warrants, or stock options). A substantial majority of the outstanding shares of our common stock are, and all of the shares sold pursuant to the Sales Agreement upon issuance will be, freely tradable without restriction or further registration under the Securities Act, unless such shares are owned or purchased by “affiliates” as that term is defined in Rule 144 under the Securities Act.
In addition, as of the date of this Form 10-K, there were outstanding (i) 392,500 shares of Series A Preferred Stock convertible into an aggregate of 1,401,786 shares of common stock, (ii) warrants to purchase an aggregate of 10,399,522 shares of common stock, (iii) options to purchase an aggregate of 6,771,250 shares of our common stock, of which options to purchase 2,866,750 shares of our common stock were then exercisable, and (iv) $2,200,000 in aggregate principal amount of convertible notes convertible into 1,392,805 shares of common stock (including the conversion of accrued interest and assuming a conversion price of $1.39, which was the closing price of our common stock on March 26, 2026). The shares of our common stock issuable upon conversion or exercise, as applicable, of such securities may be immediately eligible for resale in the open market. Any such sales, or the perception that such sales could occur, could cause the market price of our common stock to decline and may make it more difficult for us to raise capital in the future.
It is not possible to predict the aggregate proceeds resulting from sales made under the Sales Agreement.
Subject to certain limitations in the Sales Agreement and compliance with applicable law, we have the discretion to deliver a placement notice to the Sales Agents at any time throughout the term of the Sales Agreement. The number of shares that are sold through the Sales Agents, if any, after delivering a placement notice will fluctuate based on a number of factors, including the market price of our common stock during the sales period, the limits we set with the Sales Agents in any applicable placement notice, and the demand for our common stock during the sales period. Because the price per share of each share sold will fluctuate during the sales period, it is not currently possible to predict the aggregate proceeds to be raised in connection with those sales.
The common stock offered pursuant to the Sales Agreement will be sold in “at the market offerings,” and investors who buy shares at different times will likely pay different prices.
Investors who purchase shares pursuant to the Sales Agreement at different times will likely pay different prices, and so may experience different levels of dilution and different outcomes in their investment results. We will have discretion, subject to market demand, to vary the timing, prices, and number of shares sold pursuant to the Sales Agreement. In addition, subject to the final determination by our board of directors, there is no minimum or maximum sales price for shares to be sold pursuant to the Sales Agreement. Investors may experience a decline in the value of the shares they purchase pursuant to the Sales Agreement as a result of sales made at prices lower than the prices they paid.
Paul A. Romness, MPH, and our other executive officers, directors and their affiliates exercise significant influence over our company, which will limit your ability to influence corporate matters and could delay or prevent a change in corporate control.
Paul A. Romness, MPH, our Chairman, President and Chief Executive Officer, beneficially owns approximately 8.3% of the outstanding shares of common stock of our company, and other executive officers and directors beneficially own another approximately 3.4% of our outstanding shares. The existing holdings of Mr. Romness and other executive officers, directors and their affiliates represent beneficial ownership in the aggregate of approximately 11.7% of our outstanding common stock. As a result, these stockholders will be able to influence our management and affairs and the outcome of matters submitted to our stockholders for approval, including the election of directors and any sale, merger, consolidation or sale of all or substantially all of our assets. These stockholders may have interests, with respect to their common stock, that are different from those of other investors and the concentration of voting power among these stockholders may have an adverse effect on the price of our common stock. In addition, this concentration of ownership might adversely affect the market price of our common stock by:
delaying, deferring or preventing a change of control our company;
impeding a merger, consolidation, takeover or other business combination involving our company; or
discouraging a potential acquirer from making a tender offer or otherwise attempting to obtain control of our company.
We are incurring increased costs as a result of operating as a public company, and our management is required to devote substantial time to new compliance initiatives.
As a recently public company, we are incurring significant legal, accounting and other expenses that we did not incur as a private company. We are subject to the reporting requirements of the Exchange Act, which requires, among other things, that we file with the SEC annual, quarterly and current reports with respect to our business and financial condition. In addition, the Sarbanes-Oxley Act of 2002, as well as rules subsequently adopted by the SEC and the NYSE American to implement provisions of the Sarbanes-Oxley Act, impose significant requirements on public companies, including requiring establishment and maintenance of effective disclosure and financial controls and changes in corporate governance practices. Further, in July 2010, the Dodd-Frank Wall Street Reform and Consumer Protection Act was enacted. There are significant corporate governance and executive compensation related provisions in the Dodd-Frank Act under which the SEC adopted additional rules and regulations in these areas, such as “say on pay” and proxy access. Recent legislation permits emerging growth companies to implement many of these requirements over a longer period and up to five years from the pricing of our July 2024 initial public offering. We intend to take advantage of this new legislation but cannot guarantee that we will not be required to implement these requirements sooner than budgeted or planned and thereby incur unexpected expenses. Stockholder activism, the current political environment and the current high level of government intervention and regulatory reform may lead to substantial new regulations and disclosure obligations, which may lead to additional compliance costs and impact the manner in which we operate our business in ways we cannot currently anticipate.
We expect the rules and regulations applicable to public companies to substantially increase our legal and financial compliance costs and to make some activities more time-consuming and costly. If these requirements divert the attention of our management and personnel from other business concerns, they could have a material adverse effect on our business, financial condition and results of operations. The increased costs will increase our net loss and may require us to reduce costs in other areas of our business or increase the prices of our products or services. For example, we expect these rules and regulations to make it more difficult and more expensive for us to obtain director and officer liability insurance and we may be required to incur substantial costs to maintain the same or similar coverage. We cannot predict or estimate the amount or timing of additional costs we may incur to respond to these requirements. The impact of these requirements could also make it more difficult for us to attract and retain qualified persons to serve on our board of directors, our board committees or as executive officers.
We have not paid, and do not intend to pay, dividends on our shares of common stock and, therefore, unless our common stock appreciates in value, our investors may not benefit from holding our shares.
We have not paid any cash dividends on our shares of common stock, and we do not anticipate paying any cash dividends on our common stock in the foreseeable future. As a result, investors in our common stock will not be able to benefit from owning these shares unless their market price becomes greater than the price paid by such investors and they are able to sell such shares. We cannot assure you that you will ever be able to resell our common stock at a price in excess of the price paid.
