What changed in this filing

/stocks — the workspaceLOADING

Item 1A. Risk Factors

2025-03-27 compared with 2024-03-26 · 341 added, 327 removed, 788 unchanged (46% of the section changed)

Investing in our securities involves a high degree of risk.

You should carefully consider the following risks and other information included or incorporated by reference in this Annual Report in evaluating us and our common stock.

You should carefully consider the following risks and other information included or incorporated by reference in this Annual Report in evaluating our company and our common stock.

Any of the following risks could materially and adversely affect our results of operations, our financial condition and the market price of our common stock.

9 unchanged sentences

These risks and uncertainties include, but are not limited to, the following:

◾ there is substantial doubt about our ability to continue as a going concern.

o there is substantial doubt about our ability to continue as a going concern.

We have a history of operating losses and expect to incur losses for the foreseeable future.

We may never generate revenues or, if we are able to generate revenues, achieve profitability;

◾ we have a limited operating history, and we expect a number of factors to cause our operating results to fluctuate on a quarterly and annual basis, which may make it difficult to predict our future performance;

◾ if preclinical studies or clinical trials for our product candidates cannot be initiated or completed or if they are delayed or unsuccessful, we will be unable to meet our future development and commercialization goals;

◾ the disorders we seek to treat have low prevalence and it may be difficult to identify patients with these disorders, which may lead to delays in enrollment for our trials or slower commercial revenue if approved, and we may also face enrollment challenges as a result of other factors;

◾ our product candidates are novel and still in development.

o we will need to raise additional capital and such financing may not be available to us in the necessary time frame, in amounts that we require, on terms that are acceptable to us, or at all;

o our ability to maintain compliance with the continued listing requirement of the Nasdaq Stock Market LLC (“Nasdaq”);

o we have a limited operating history, and we expect a number of factors to cause our operating results to fluctuate on a quarterly and annual basis which may make it difficult to predict future performance;

o if preclinical studies or clinical trials for our product candidates cannot be initiated or completed or if they are delayed or unsuccessful, we will be unable to meet our future development and commercialization goals;

o some of the disorders we seek to treat have low prevalence and it may be difficult to identify patients with these disorders, which may lead to delays in enrollment for our trials or slower commercial revenue if approved, and we may also face enrollment challenges as a result of other factors;

o our product candidates are novel and still in development.

If we are unable to successfully develop, receive regulatory approval for and commercialize our current or future product candidates, our business will be harmed;

◾ we have started testing one of our product candidates in clinical trials .

Success in early preclinical studies or clinical trials may not be indicative of results obtained in later preclinical studies and clinical trials;

◾ clinical trials required for our product candidates are expensive and time-consuming, and their outcomes are uncertain;

◾ we will need to raise additional capital, which may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates, and additional capital may not be available on favorable terms or at all, which may force us to delay, reduce the scope of or eliminate our research and development programs, reduce our commercialization efforts or curtail our operations;

◾ we are subject to extensive and costly government regulation;

◾ even if we obtain regulatory approval to market our product candidates, our product candidates may not be accepted by the market;

◾ we rely on a license to use the technology that is material to our business and if the agreement underlying the license were to be terminated or if other rights that may be necessary for commercializing our intended

products cannot be obtained, it would halt our ability to market our products and technology, as well as have an immediate material adverse effect on our business, operating results and financial condition;

◾ we are subject to stringent and evolving U.S.

o we have started testing one of our product candidates in clinical trials.

Success in preclinical studies or clinical trials may not be indicative of results obtained in later clinical trials;

o clinical trials required for our product candidates are expensive and time-consuming, and their outcomes are uncertain;

o we will need to raise additional capital, which may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies or product candidates.

Additional capital may not be available on favorable terms or at all which may force us to delay, reduce the scope of or eliminate our research and development programs, reduce our commercialization efforts or curtail our operations;

o we are subject to extensive and costly government regulations which are subject to change;

o even if we obtain regulatory approval to market our product candidates, our product candidates may not be accepted by the market;

o we rely on a license to use the technology that is material to our business and if the agreement underlying the license were to be terminated or if other rights that may be necessary for commercializing our intended products cannot be obtained, it would halt our ability to market our products and technology, as well as have an immediate material adverse effect on our business, operating results and financial condition;

o we are subject to stringent and evolving U.S.

and foreign laws, regulations, rules, contractual obligations, policies and other obligations related to data privacy and security.

Our actual or perceived failure to comply with such obligations could lead to regulatory investigations or actions, litigation, (including class actions) and mass arbitration demands, fines and penalties, disruptions of our business operations, reputational harm, loss of revenue or profits and other adverse business consequences;

◾ global and macroeconomic conditions, including worldwide economic, political and social instability could adversely affect our revenue, financial condition, or results of operations.

o global and macroeconomic conditions, including worldwide economic, political and social instability could adversely affect our revenue, financial condition, or results of operations.

Risks Related to Our Business

4 unchanged sentences

We have incurred losses in each year of our operations and we expect to continue to incur operating losses for the foreseeable future.

Since our inception we have incurred in operating losses which have adversely affected and are likely to continue to adversely affect our working capital, total assets and shareholders’ equity.

Since our inception, we have incurred operating losses which have adversely affected, and are likely to continue to adversely affect, our working capital, total assets and shareholders’ equity.

In addition, the impact of these events and conditions on our liquidity raise substantial doubt about our ability to continue as a going concern.

We plan to raise additional capital primarily through public and/or private equity financings and/or convertible debt financings.

We will need to raise additional capital and plan to raise additional capital primarily through public and/or private equity financings and/or convertible debt financings.

However, financing may not be available to us in the necessary time frame, in amounts that we require, on terms that are acceptable to us or at all.

11 unchanged sentences

We have no products that have generated any commercial revenue, do not expect to generate revenues from the commercial sale of products in the foreseeable future and might never generate revenues from the sale of products.

Our ability to generate revenue and achieve profitability will depend on, among other things, successful completion of preclinical development and testing and clinical trials of our product candidates;

Our ability to generate revenue and achieve profitability will depend on, among other things, successful completion of preclinical

development and testing and clinical trials of our product candidates;

obtaining necessary regulatory approvals from the FDA and comparable foreign regulatory authorities;

3 unchanged sentences

and raising sufficient funds to finance our activities.

Many of these factors will depend on circumstances beyond our

Many of these factors will depend on circumstances beyond our control.

We might not succeed at any of these undertakings.

6 unchanged sentences

Our financial condition has varied significantly in the past and will continue to fluctuate from quarter-to-quarter or year-to-year due to a variety of factors, many of which are beyond our control.

Factors relating to our business that may contribute to these fluctuations include, among other factors described elsewhere in this Annual Report:

Factors relating to our business that may contribute to these fluctuations, among other factors described elsewhere in this Annual Report, include:

● our ability to obtain additional funding to develop our product candidates, the extent to which we are able to obtain such funding on favorable terms, and changes to our operations or strategy that may be necessitated due to the need for additional funding;

3 unchanged sentences

● any delays in regulatory review and approval of product candidates in clinical development;

● our ability to obtain and maintain regulatory approval for our product candidates in the United States and foreign jurisdictions;

● our ability to obtain and maintain regulatory approval for our product candidates in the U.S.

and foreign jurisdictions;

● our ability to successfully commercialize product candidates for which we obtain regulatory approval within expected timelines or at all;

11 unchanged sentences

Risks Related to Product Development, Regulatory Approval, Manufacturing and Commercialization

We may conduct certain of our clinical trials for our product candidates outside of the U.S.

which, among other risks, exposes us to the possibility that the FDA and other comparable foreign regulatory authorities may not accept data from such trials, in which case our development plans will be delayed, which could materially harm our business.

We are currently conducting a first-in-human Phase 1 clinical trial in our Parkinson’s disease program in Australia.

Although the FDA may accept data from clinical trials conducted outside the United States, acceptance of this data is subject to certain conditions imposed by the FDA.

Where data from foreign clinical trials are intended to serve as the basis for marketing approval in the United States, the FDA will not approve the application on the basis of foreign data alone unless those data are applicable to the U.S.

population and U.S.

medical practice;

the studies were performed by clinical investigators of recognized competence;

and the data are considered valid without the need for an on-site inspection by the FDA or, if the FDA considers such an inspection to be necessary, the FDA is able to validate the data through an on-site inspection or other appropriate means.

For studies that are conducted only at sites outside of the United States and not subject to an IND, the FDA requires the clinical trial to have been conducted in accordance with GCPs, and the FDA must be able to validate the data from the clinical trial through an on-site inspection if it deems such inspection necessary.

For such studies not subject to an IND, the FDA generally does not provide advance comment on the clinical protocols for the studies, and therefore there is an additional potential risk that the FDA could determine that the study design or protocol for a non-U.S.

clinical trial was inadequate, which could require us to conduct additional clinical trials.

There can be no assurance the FDA will accept data from clinical trials conducted outside of the United States.

If the FDA does not accept data from our clinical trials of our product candidates conducted outside of the United States, it would likely result in the need for additional clinical trials, which would be costly and time consuming and delay or permanently halt our development of our product candidates.

Conducting clinical trials outside the United States also exposes us to additional risks, including risks associated with:

● additional foreign regulatory requirements;

● foreign exchange fluctuations;

● compliance with foreign manufacturing, customs, shipment and storage requirements;

● cultural differences in medical practice and clinical research;

● diminished protection of intellectual property in some countries.

By extension, clinical trials that are predominantly conducted in the United States or primarily based on feedback from the FDA may not result in sufficiently diverse patient populations to warrant approval in other countries (for example, Japan) or those other comparable foreign regulatory authorities may have differences of opinion on appropriateness of trial design or differences in interpretation of some data.

In those situations, approvals in other countries outside the United States may be delayed or never approved, which would materially detract from the commercial success of any impacted product candidates.

We are subject to extensive and costly government regulation, which are subject to change.

Product candidates employing our technology are subject to extensive and rigorous domestic government regulation including regulation by the FDA, the Centers for Medicare and Medicaid Services (“CMS”), other divisions of the United States Department of Health and Human Services (“HHS”), the United States Department of Justice (“DOJ”), state and local governments and their respective foreign equivalents.

The FDA and comparable foreign regulatory authorities regulate the research, development, preclinical studies and clinical trials, manufacture, safety, effectiveness, recordkeeping, reporting, labeling, storage, approval, advertising, promotion, sale, distribution, import and export of biopharmaceutical products.

If products employing our technologies are marketed abroad, they will also be subject to extensive regulation by foreign governments, whether they have obtained the FDA’s or comparable foreign regulatory authorities’ approval for a given product and its uses.

Such foreign regulation may be equally or more demanding than corresponding U.S.

Government regulation substantially increases the cost and risk of researching, developing, manufacturing and selling our products.

The regulatory review and approval process, which includes preclinical studies and clinical trials of each product candidate, is lengthy, expensive, and uncertain.

We or our collaborators must obtain and maintain regulatory authorization to conduct clinical trials as well as approval for each product we intend to market and the manufacturing facilities used for the products must be inspected and meet legal requirements.

Securing regulatory approval requires the submission of extensive preclinical and clinical data and other supporting information for each proposed therapeutic indication to establish the product’s safety and efficacy, and in the case of biologics also potency and purity, for each intended use.

The development and approval process takes many years, requires substantial resources and may never lead to the approval of a product.

Even if we can obtain regulatory approval for a particular product, the approval may limit the indicated medical uses for the product, may otherwise limit our ability to promote, sell, and distribute the product, may require that we conduct costly post-marketing surveillance, and/or may require that we conduct ongoing post marketing studies.

Material changes to an approved product, such as, for example, manufacturing changes or revised labeling, may require further regulatory review and approval.

Once obtained, any approvals may be withdrawn, suspended or varied.

For example, if there is a later discovery of previously unknown problems with the product, such as a previously unknown safety issue.

If we, our collaborators or our manufacturers fail to comply with applicable regulatory requirements at any stage during the regulatory process, such noncompliance could result in, among other things, delays in the approval of applications or supplements to approved applications;

refusal of a regulatory authority, including the FDA, to review pending market approval applications or supplements to approved applications;

warning letters;

import and/or export restrictions;

product recalls or seizures;

total or partial suspension of production, distribution, manufacturing or clinical trials;

civil penalties;

withdrawals, suspension or variation of previously approved marketing applications or licenses;

recommendations by the FDA or comparable foreign other regulatory authorities against governmental contracts;

and/or criminal prosecutions.

Moreover, the development of our product candidates may be delayed by other events beyond our control.

For example, action by the Trump administration to limit federal agency budgets or personnel, may result in reductions to the FDA’s budget, employees, and operations, which may lead to slower response times and longer review periods, potentially affecting our ability to progress development of our product candidates or obtain regulatory approval for our product candidates.

Our long-term success depends heavily on our ability to fund and complete research and development activities and obtain regulatory approval for our product candidates.

Only a small minority of all research and development programs ultimately result in commercially successful drugs.

Clinical failure can occur at any stage of clinical development.

Clinical and preclinical trials may produce negative or inconclusive results and we may decide, or regulators may require us, to conduct additional clinical or preclinical trials.

In addition, data obtained from trials are susceptible to varying interpretations and regulators may not interpret our data as favorably as we do.

This may delay, limit or prevent regulatory approval.

Success in preclinical testing and early clinical trials does not ensure that later clinical trials will generate the same results or otherwise provide adequate data to demonstrate the efficacy and safety of a drug candidate.

As part of development, we also must show that we can formulate and manufacture our product candidates in compliance with regulatory requirements .

We will need substantial additional financing to complete the development of our drug candidates and comply with the regulatory requirements governing this process.

Further, even if we complete the development of our drug candidates and gain marketing approvals from the FDA and comparable foreign regulatory authorities in a timely manner, we cannot be sure that such drug candidates will be commercially successful in the pharmaceutical market.

If the results of clinical trials, the anticipated or actual timing of marketing approvals or the market acceptance of any of our drug candidates, if approved, do not meet the expectations of investors or public market analysts, our business will be in jeopardy and the market price of our common stock would likely decline.

If preclinical studies or clinical trials for our product candidates cannot be initiated or completed or if they are delayed or unsuccessful, we will be unable to meet our future development and commercialization goals.

We rely and expect to continue to rely on third parties, including contract research organizations (“CROs”) and outside consultants, to conduct, supervise or monitor some or all aspects of preclinical studies and clinical trials involving our product candidates.

We have less control over the timing and other aspects of these preclinical studies and clinical trials than if we performed the monitoring and supervision entirely on our own.

Third parties may not perform their responsibilities for our preclinical studies and clinical trials on our anticipated schedule or, for clinical trials, consistent with a clinical trial protocol.

Delays in preclinical studies and clinical trials could significantly increase our product development costs and delay product commercialization.

2 unchanged sentences

● demonstrating sufficient safety and efficacy to obtain regulatory approval to commence a clinical trial;

● reaching agreement on acceptable terms with prospective CROs and study sites;

● reaching agreement on acceptable terms with prospective contract research organizations (“CROs”) and study sites;

● developing a stable formulation of a product candidate;

10 unchanged sentences

● nonclinical or clinical safety observations, including adverse events and SAEs.

We rely and expect to continue to rely on third parties including CROs and outside consultants to conduct, supervise or monitor some or all aspects of preclinical studies and clinical trials involving our product candidates.

We have less control over the timing and other aspects of these preclinical studies and clinical trials than if we performed the monitoring and supervision entirely on our own.

Third parties may not perform their responsibilities for our preclinical studies and clinical trials on our anticipated schedule or, for clinical trials, consistent with a clinical trial protocol.

If clinical trials are unsuccessful and we are not able to obtain regulatory approvals for our product candidates under development, we will not be able to commercialize these products and therefore may not be able to generate sufficient revenues to support our business.

The disorders we seek to treat have low prevalence and it may be difficult to identify patients with these disorders, which may lead to delays in enrollment for our trials or slower commercial revenue if approved, and we may also face enrollment challenges as a result of other factors.

Genetically defined disorders generally, and especially those for which our current product candidates are targeted, have low incidence and prevalence.

We expect to rely in part on relationships with clinical centers of

excellence, key opinion leaders and patient advocacy groups to assist in identifying eligible patients, and any deterioration of those relationships could impede our ability to successfully enroll patients.

Patient enrollment may be affected by other factors including:

Some of the disorders we seek to treat have low prevalence and it may be difficult to identify patients with these disorders, which may lead to delays in enrollment for our trials or slower commercial revenue if approved, and we may also face enrollment challenges as a result of other factors.

As our portfolio of drug candidates moves from preclinical testing to clinical testing and then through progressively larger and more complex clinical trials, we will need to enroll an increasing number of patients with the appropriate characteristics.

At times we have experienced difficulty enrolling patients and we may experience more difficulty as the scale of our clinical testing program increases.

The factors that affect our ability to enroll patients are largely uncontrollable and principally include the following:

● the size of the patient population;

● the severity of the disease under investigation;

● design of the study protocol;

● the nature of the clinical test and design of the study protocol;

● the eligibility criteria for the trial;

4 unchanged sentences

● the proximity and availability of clinical trial sites to prospective patients.

Our inability to enroll a sufficient number of patients with these diseases for our future clinical trials would result in significant delays and could require us to not initiate or to abandon clinical trials for one or more indications altogether.

Our inability to enroll a sufficient number of patients with these diseases in our future clinical trials would result in significant delays and could prevent us from initiating or cause us to abandon clinical trials for one or more indications altogether.

Enrollment delays in our clinical trials may result in increased development costs for our product candidates which would cause the value of our company to decline and limit our ability to obtain additional financing.

Additionally, the reported number of people in the indications we aim to treat, as well as the people with these diseases who have the potential to benefit from treatment with our product candidates, are based on estimates.

Additionally, the reported number of people in some of the indications we aim to treat, as well as the people with these diseases who have the potential to benefit from treatment with our product candidates, are based on estimates.

The total addressable market opportunity for our product candidates will ultimately depend upon, among other things, the final approved product labeling for each of our product candidates, if our product candidates are approved for sale in our target indications, acceptance by the medical community and patient access, drug pricing and reimbursement.

7 unchanged sentences

Also, third parties we rely on for preclinical development, such as the providers of supercomputer time needed for our Magellan™ platform and collaborators that provide us with materials and resources may fail to fulfill their obligations to us in a timely manner or at all and the development of our product candidates could be significantly delayed as a result.

In addition, we are still developing proof of concept for our product candidates in animals and positive data from animal models may not be predictive of positive human results and patients may have side effects that were not observed in animals.

In addition, we are still developing proof of concept for our product candidates in animals and positive data from animal models may not be predictive of positive human results.

Patients may have side effects that were not observed in animals.

Further, we and our product candidates are subject to extensive regulation by the FDA and comparable regulatory authorities in other countries governing, among other things, research, testing, clinical trials, manufacturing, labeling, promotion, selling, adverse event reporting and recordkeeping.

14 unchanged sentences

If our study data do not consistently or sufficiently demonstrate the safety or efficacy of any of our product candidates, then the regulatory approvals for such product candidates could be significantly delayed as we work to meet approval requirements, or, if we are not able to meet these requirements, such approvals could be withheld, varied or withdrawn.

Regulatory delays or rejections may also be encountered as a result of many other factors, including changes in regulatory policy during the period of product development.

Regulatory delays

or rejections may also be encountered as a result of many other factors including changes in regulatory policy during the period of product development.

The approach we are taking to discover and develop our product candidates is novel and may never lead to marketable products.

We have concentrated our efforts and research and development activities on our novel small molecules for potential treatment of rare and genetic diseases caused by protein misfolding and Magellan™, our target identification platform.

We have concentrated our efforts and research and development activities on our novel small molecules for potential treatment of rare and genetic diseases and on more prevalent neurodegenerative diseases caused by protein misfolding and Magellan™, our target identification platform.

Our future success depends on the successful development of such product candidates, including our ability to successfully complete IND-enabling and GLP-compliant preclinical studies, and the effectiveness of our platform.

3 unchanged sentences

In addition, decisions by other companies with respect to their therapeutic development efforts may increase skepticism in the marketplace regarding the potential for potential therapeutics.

There are currently no companies with approved drugs for these indications that have the ability to cross the blood-brain barrier.

There are currently no companies with approved disease modifying small molecule drugs for these indications that have the ability to cross the blood-brain barrier.

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.

Because we have limited financial and human resources, we are currently focusing primarily on development of our Parkinson’s and Gaucher disease programs.

Because we have limited financial and human resources, we are currently focusing primarily on development of our Parkinson’s program.

As a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that later prove to have greater commercial potential.

11 unchanged sentences

slower than expected rates of patient recruitment;

failure to recruit a sufficient number of patients, which is a common issue in studies for rare disorders such as the indications we are currently pursuing;

failure to recruit a sufficient number of patients, which is a common issue in studies for rare disorders such as some of the indications we are currently pursuing;

modification of clinical trial protocols;

9 unchanged sentences

This failure could cause us to abandon a product candidate and could delay development of other product candidates.

Any delay in, or termination of, our clinical trials would delay our ability to obtain regulatory approvals for and commercialize our product candidates and generate product revenues.

Any delay in or termination of our clinical trials would delay our ability to obtain regulatory approvals for and commercialization of our product candidates and generate product revenues.

Any change in or termination of our clinical trials could materially harm our business, financial condition and results of operations.

13 unchanged sentences

Because of this lack of experience, any clinical trials we may conduct may not be completed on time, if at all.

Large-scale trials require significant additional financial and management resources, monitoring and oversight, and reliance on third-party clinical investigators, consultants or contract research organizations (“CROs”).

Large-scale trials require significant additional financial and management resources, monitoring and oversight and reliance on third-party clinical investigators, consultants or CROs.

Relying on third-party clinical investigators, CROs and manufacturers, which are all also subject to governmental oversight and regulations, may also cause us to encounter delays that are outside of our control.

2 unchanged sentences

Failure to commence or complete, or delays in, future planned clinical trials, could prevent us from or delay us in commercializing our product candidates.

We are subject to extensive and costly government regulation.

Product candidates employing our technology are subject to extensive and rigorous domestic government regulation including regulation by the FDA, the Centers for Medicare and Medicaid Services, other divisions of the United States Department of Health and Human Services, the United States Department of Justice, state and local governments and their respective foreign equivalents.

The FDA and comparable foreign regulatory authorities regulate the research, development, preclinical studies and clinical trials, manufacture, safety, effectiveness, record-keeping, reporting, labeling, storage, approval, advertising, promotion, sale, distribution, import and export of biopharmaceutical products.

If products employing our technologies are marketed abroad, they will also be subject to extensive regulation by foreign governments, whether or not they have obtained the FDA’s or comparable foreign regulatory authorities’ approval for a given product and its uses.

Such foreign regulation may be equally or more demanding than corresponding United States regulation.

Government regulation substantially increases the cost and risk of researching, developing, manufacturing and selling our products.

The regulatory review and approval process, which includes preclinical studies and clinical trials of each product candidate, is lengthy, expensive, and uncertain.

We or our collaborators must obtain and maintain regulatory authorization to conduct clinical trials.

We or our collaborators must obtain regulatory approval for each product we intend to market, and the manufacturing facilities used for the products must be inspected and meet legal requirements.

Securing regulatory approval requires the submission of extensive preclinical and clinical data and other supporting information for each proposed therapeutic indication in order to establish the product’s safety and efficacy, and in the case of biologics also potency and purity, for each intended use.

The development and approval process takes many years, requires substantial resources, and may never lead to the approval of a product.

Even if we are able to obtain regulatory approval for a particular product, the approval may limit the indicated medical uses for the product, may otherwise limit our ability to promote, sell, and distribute the product, may require that we conduct costly post-marketing surveillance, and/or may require that we conduct ongoing post-marketing studies.

Material changes to an approved product, such as, for example, manufacturing changes or revised labeling, may require further regulatory review and approval.

Once obtained, any approvals may be withdrawn, suspended or varied, including, for example, if there is a later discovery of previously unknown problems with the product, such as a previously unknown safety issue.

If we, our collaborators, or our manufacturers fail to comply with applicable regulatory requirements at any stage during the regulatory process, such noncompliance could result in, among other things delays in the approval of applications or supplements to approved applications;

refusal of a regulatory authority, including the FDA, to review pending market approval applications or supplements to approved applications;

warning letters;

import and/or export restrictions;

product recalls or seizures;

total or partial suspension of production, distribution, manufacturing or clinical trials;

civil penalties;

withdrawals, suspension or variation of previously approved marketing applications or licenses;

recommendations by the FDA or comparable foreign other regulatory authorities against governmental contracts;

and/or criminal prosecutions.

We may conduct certain of our clinical trials for our product candidates outside of the U.S.

We have recently completed a first-in-human Phase 1 clinical trial in healthy volunteers for our Parkinson’s disease program in Australia.

During a pre-IND meeting with the FDA that took place in December 2024, we reviewed all of the clinical and preclinical data we have recorded for GT-02287 thus far, including the first-in-human Phase 1 study, and discussed our plans to submit an IND application for US expansion of clinical development for GT-02287 in support of eventual marketing approval in the U.S.

Where data from foreign clinical trials are intended to serve as the basis for marketing approval in the U.S., the FDA will not approve the application on the basis of foreign data alone unless those data are applicable to the U.S.

population and U.S.

medical practice.

Therefore, later stage clinical

trials designed to determine that GT-02287 is safe and effective for the purposes of FDA approval will be conducted in part in the U.S.

The Phase 1 study was performed by clinical investigators of recognized competence;

For studies that are conducted only at sites outside of the U.S.

and not subject to an IND, the FDA requires the clinical trial to have been conducted in accordance with GCPs and the FDA must be able to validate the data from the clinical trial through an on-site inspection if it deems such inspection necessary.

clinical trial was inadequate, which could require us to conduct additional clinical trials.

There can be no assurance the FDA will accept data from clinical trials conducted outside of the United States.

Conducting clinical trials outside the United States also exposes us to additional risks including risks associated with:

● additional foreign regulatory requirements;

● foreign exchange fluctuations;

● compliance with foreign manufacturing, customs, shipment and storage requirements;

● cultural differences in medical practice and clinical research;

● diminished protection of intellectual property in some countries.

By extension, clinical trials that are predominantly conducted in the U.S.

or primarily based on feedback from the FDA may not result in sufficiently diverse patient populations to warrant approval in other countries (for example, Japan) or those other comparable foreign regulatory authorities may have differences of opinion on appropriateness of trial design or differences in interpretation of some data.

In those situations, approvals in other countries outside the U.S.

may be delayed or never approved, which would materially detract from the commercial success of any impacted product candidates.

If we decide to pursue a Fast Track Designation or comparable foreign regulatory procedures for some of our product candidates, it may not lead to a faster development or regulatory review or approval process.

We may seek Fast Track Designation, or comparable foreign regulatory procedures, for one or more of our product candidates.

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 product sponsor may apply for FDA Fast Track Designation.

The FDA has broad discretion whether or not to grant this designation, so even if we believe a particular product candidate is eligible for this designation, the FDA may decide not to grant it.

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 product sponsor may apply for FDA Fast Track Designation.

The FDA has broad discretion whether to grant this designation, so even if we believe a particular product candidate is eligible for this designation, the FDA may decide not to grant it.

Even if we do receive Fast Track Designation, we may not experience a faster development process, review or approval compared to conventional FDA procedures.

2 unchanged sentences

If we decide to seek Orphan Drug Designation for some of our product candidates, we may be unsuccessful or may be unable to maintain the benefits associated with Orphan Drug Designation, including the potential for supplemental market exclusivity.

As part of our business strategy, we may seek Orphan Drug Designation for one or more of our product candidates, and we may be unsuccessful.

As part of our business strategy, we may seek Orphan Drug Designation for one or more of our product candidates which may be unsuccessful.

Regulatory authorities in some jurisdictions, including the United States and European countries, may designate drugs for relatively small patient populations as orphan drugs.

Under the Orphan Drug Act, the FDA may designate a drug 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, or a patient population greater than 200,000 in the United States where there is no reasonable expectation that the cost of developing the drug will be recovered from sales in the United States.

In the United States, Orphan Drug Designation entitles a party to financial incentives such as a tax credit.

Under the Orphan Drug Act, the FDA may designate a drug as an orphan drug if it is intended to treat a rare disease or condition, which is generally defined as a patient population of fewer than 200,000 individuals in the U.S.

or a patient population greater than 200,000 in the U.S.

where there is no reasonable expectation that the cost of developing the drug will be recovered from sales in the U.S.

In the U.S., Orphan Drug Designation entitles a party to financial incentives such as a tax credit.

Opportunities for grant funding toward clinical trial costs may also be available for clinical trials of drugs for rare diseases, regardless of whether the drugs are designated for the orphan use.

3 unchanged sentences

Even after obtaining approval, we may be limited in our ability to market our product.

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

In addition, exclusive marketing rights in the U.S.

may be limited if we seek approval for an indication broader than the orphan-designated indication or may be lost if the FDA later determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantities of the product to meet the needs of patients with the rare disease or condition.

Further, even if we obtain orphan drug exclusivity for a product, that exclusivity may not effectively protect the product from competition because different drugs with different principal molecular structural features can be approved for the same condition.

In the EU, Regulation (EC) No.

In the European Union (the “EU”), Regulation (EC) No.

141/2000, as implemented by Regulation (EC) No.

3 unchanged sentences

and (iii) there exists no satisfactory authorized method of diagnosis, prevention, or treatment of the condition that has been authorized in the EU, or even if such method exists, the product will be of significant benefit to those affected by that condition.

Orphan medicinal product designation, entitles an applicant to incentives such fee reductions or fee waivers, protocol assistance, and access to the centralized marketing authorization procedure.

Orphan medicinal product designation entitles an applicant to incentives such as fee reductions or fee waivers, protocol assistance, and access to the centralized marketing authorization procedure.

Upon grant of a marketing authorization, orphan medicinal products are entitled to a ten-year period of market exclusivity for the approved therapeutic indication, which means that the EMA cannot accept another marketing authorization application or accept an application to extend for a similar product and the European Commission cannot grant a marketing authorization for the same indication for a period of ten years.

7 unchanged sentences

A company may voluntarily remove a product from the register of orphan products.

Orphan Drug Designation in the United States, or orphan medicinal product designation the EU, neither shortens the development time or regulatory review time of a drug nor gives the drug any advantage in the regulatory review or approval process.

While we may seek Orphan Drug Designation in the United States, or orphan medicinal product designation the EU, for our product candidates, we may never receive such designation.

Orphan Drug Designation in the U.S.

or orphan medicinal product designation the EU neither shortens the development time or regulatory review time of a drug nor gives the drug any advantage in the regulatory review or approval process.

While we may seek Orphan Drug Designation in the U.S.

or orphan medicinal product designation the EU for our product candidates, we may never receive such designation.

We do not have, and may never obtain, the regulatory approvals we need to market our product candidates.

Following completion of clinical trials, the results are evaluated and, depending on the outcome, an NDA is submitted to the FDA to obtain the FDA’s approval of the product and authorization to commence commercial marketing.

Following completion of clinical trials, the results are evaluated and, depending on the outcome, a New Drug Application (“NDA”) is submitted to the FDA to obtain the FDA’s approval of the product and authorization to commence commercial marketing.

In responding to an NDA, the FDA may require additional testing or information, may require that the product labeling be modified, may impose post-approval study and other commitments or reporting requirements or other restrictions on product distribution, or may deny the application.

3 unchanged sentences

The timing of final review by the FDA and action varies greatly but can take years in some cases and may involve the input of an FDA advisory committee of outside experts.

Product sales in the United States may commence only when an NDA is approved.

Product sales in the U.S.

may commence only when an NDA is approved.

Comparable procedures and limitations are applicable in the EU and in other jurisdictions.

30 unchanged sentences

Even if physicians and patients would like to use our products, our products may not gain market acceptance among healthcare payors such as managed care formularies, insurance companies or government programs such as Medicare or Medicaid or comparable foreign programs.

Acceptance and use of our products will depend upon a number of factors including:

perceptions by members of the health care community, including physicians, about the safety and effectiveness of our drug or device product;

Acceptance and use of our products will depend upon a number of factors including perceptions by members of the health care community, including physicians, about the safety and effectiveness of our drug or device product;

cost-effectiveness of our product relative to competing products;

3 unchanged sentences

● cost-effectiveness;

● the safety and effectiveness of our products, including any significant potential side effects (including drowsiness and dry mouth), as compared to alternative products or treatment methods;

● the safety and effectiveness of our products, including any significant potential side effects as compared to alternative products or treatment methods;

● the timing of market entry as compared to competitive products;

5 unchanged sentences

Because we expect sales of our current product candidates, if approved, to generate substantially all of our product revenues for the foreseeable future, the failure of these products to find market acceptance would harm our business and require us to seek additional financing which may not be available.

Climate change, climate change-related regulation and sustainability concerns could adversely affect our businesses and the operations of our subsidiaries, and any actions we take or fail to take in response to such matters could damage our reputation.

Investor advocacy groups, certain institutional investors, investment funds, other market participants and other stakeholders have focused increasingly on the Environmental, Social and Governance ("ESG") practices of companies, including those associated with climate change.

These parties have placed increased importance on the importance on the implications of the social cost of their investments.

If our ESG practices do not meet investor or other industry stakeholder expectations and standards, which continue to evolve, our reputation and associate retention may be negatively impacted based on an assessment of our ESG practices.

Any sustainability disclosures we make may include our policies and practices on a variety of social and ethical matters, including corporate governance, environmental compliance, employee health and safety practices, human capital management, product quality, supply chain management, and workforce inclusion and diversity.

It is possible that stakeholders may not be satisfied with our ESG practices or the speed of their adoption, or that we may not sufficiently communicate our ESG practices to stakeholders.

We could also incur additional costs and require additional resources to monitor, report, and comply with various ESG practices.

In addition, investors may decide to refrain from investing in us as a result of their assessment of our approach to and consideration of the ESG factors.

In addition, we may face physical risks associated with climate change.

These physical risks include risks to our manufacturing and supply chain from flooding, severe storms, wildfires, droughts or extreme temperatures, all of which could increase costs and impair our ability to meet our operational demands in a timely manner.

To date, we have not experienced material losses or disruptions to our operations related to climate change, and we do not anticipate that these risks will have a material impact to our Company in the near term.

We are also subject to changing rules and regulations promulgated by a number of governmental and self-regulatory organizations, including the SEC, Nasdaq and the Financial Accounting Standards Board.

These rules and regulations continue to evolve in scope and complexity and many new requirements have been created in response to laws enacted by Congress, making compliance more difficult and uncertain.

Concern over severe weather may also result in new or additional legal or regulatory requirements designed to mitigate the effects of severe weather on the environment and businesses.

If such laws or regulations are more stringent than current legal or regulatory obligations, we may experience disruption in, or an increase in the costs associated with sourcing, manufacturing and distribution of our products, as well as an increase in costs associated with monitoring, tracking and reporting ESG related information to regulatory bodies, which may adversely affect our business, results of operations or financial condition.

These changing rules, regulations and stakeholder expectations may result in increased general and administrative expenses and increased management time and attention spent complying with or meeting such

regulations and expectations.

For example, the State of California recently passed the Climate Corporate Data Accountability Act and the Climate-Related Financial Risk Act that, if implemented, will impose broad climate-related disclosure obligations on certain companies doing business in California.

states’ legislatures are considering enactment of similar rules and regulations.

In addition, the European Union (“EU”) enacted the Corporate Sustainability Reporting Directive (“CSRD”) legislation in January 2023 which requires certain reporting and disclosure relating to ESG matters for companies whose business and assets exceed certain thresholds within EU countries.

Developing and acting on initiatives within the scope of ESG, and collecting, measuring and reporting ESG related information and metrics can be costly, difficult and time consuming and is subject to evolving reporting standards, including the SEC’s climate-related reporting requirements, and similar proposals by other international regulatory bodies.

We may also communicate certain initiatives and goals, regarding environmental matters, diversity, responsible sourcing and social investments and other ESG related matters, in our SEC filings or in other public disclosures.

These initiatives and goals within the scope of ESG could be difficult and expensive to implement, the technologies needed to implement them may not be cost effective and may not advance at a sufficient pace, and we could be criticized for the accuracy, adequacy or completeness of the disclosure.

Further, statements about our ESG-related initiatives and goals, and progress against those goals, may be based on standards for measuring progress that are still developing, internal controls and processes that continue to evolve, and assumptions that are subject to change in the future.

Risks Related to Our Financial Condition and Capital Requirements;

2 unchanged sentences

Until such time, if ever, as we can generate substantial product revenue, we expect to seek additional funding to meet our operational needs and capital requirements.

While we believe that our existing cash, cash equivalents and marketable securities will enable us to fund our operating expenses and capital expenditure requirements into the first quarter of 2025, we have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect, including if our business or operations change in a manner that consumes available resources more rapidly than we anticipate.

While we believe that our existing cash and cash equivalents will enable us to fund our operating expenses and capital expenditure requirements into the third quarter of 2025, we have based this estimate on assumptions that may prove to be wrong and we could exhaust our available capital resources sooner than we expect, including if our business or operations change in a manner that consumes available resources more rapidly than we anticipate.

Our requirements for additional capital will depend on many factors including:

11 unchanged sentences

Cash requirements may vary materially from those now planned because of changes in direction of our research and development programs, competitive and technical advances, patent developments, regulatory changes or other developments.

If adequate additional funds are not available when required, or if we are unsuccessful in entering into partnership agreements for further development of our pipeline, management may need to curtail our development efforts and planned operations to conserve cash.

If additional sources of financing are not available on favorable terms, or at all, including as a result of actions taken by central banks to counter inflation, volatility in the capital markets, liquidity concerns at and failures of banks and other financial institutions and related market uncertainty, or if we are unsuccessful in entering into partnership agreements for further development of our pipeline, management may need to curtail our development efforts and planned operations to conserve cash.

We expect to finance our operations through a combination of equity offerings, debt financings, government or private party grants, collaborations, strategic alliances and licensing arrangements.

We currently have on file with the SEC a shelf registration statement on Form S-3 which allows us to offer and sell our registered common stock, preferred stock, debt securities and or warrants from time to time pursuant to one or more offerings at prices and terms to be determined at the time of sale.

On September 6, 2024, we entered into an Equity Distribution Agreement (the “Distribution Agreement”) with Oppenheimer & Co.

(“Oppenheimer”), pursuant to which, from time to time, we may offer and sell through Oppenheimer up to $50.0 million of the common stock registered under the shelf registration statement pursuant to one or more “at the market” offerings.

From time to time, we have issued and sold shares of common stock pursuant to this agreement and as of December 31, 2024, we have $47 million of common stock remaining available for sale under the Distribution Agreement.

Sales of our common stock under the Distribution Agreement with Oppenheimer could be subject to business, economic or competitive uncertainties and contingencies, many of which may be beyond our control, and which could cause actual results from the sale of our common stock to differ materially from expectations.

To the extent additional capital is raised through the sale and issuance of shares or other securities convertible into shares, the ownership interest of our stockholders will be diluted.

Future issuances of our common stock or other equity securities, or the perception that such sales may occur, could adversely affect the trading price of our common stock and impair our ability to raise capital through future offerings of shares or equity securities.

No prediction can be made as to the effect, if any, that future sales of common stock or the availability of common stock for future sales will have on the trading price of our common stock.

We do not currently have any other committed external sources of funds.

1 unchanged sentence

In addition, if we obtain debt financing, a substantial portion of our operating cash flow may be dedicated to the payment of principal and interest on such indebtedness, thus limiting funds available for our business activities.

In addition, debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends.

Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions such as incurring additional debt, making capital expenditures or declaring dividends.

If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may be required to relinquish valuable rights to our technologies, future revenue streams or product candidates, grant licenses on terms that may not be favorable to us or commit to future payment streams.

We will require substantial additional funds to support our research and development activities, and the anticipated costs of preclinical studies and clinical trials, regulatory approvals and eventual commercialization.

Such additional sources of financing may not be available on favorable terms, if at all, including as a result of actions taken by central banks to counter inflation, volatility in the capital markets, liquidity concerns at and failures of banks and other financial institutions and related market uncertainty.

If we do not succeed in raising additional funds on acceptable terms or if we are unsuccessful in entering into partnership agreements for further development of our pipeline, we may need to curtail our development efforts and planned operations to conserve cash.

Our continued operations may be in jeopardy and we may be forced to cease operations and sell or otherwise transfer all or substantially all of our remaining assets.

We face intense competition in the markets targeted by our product candidates.

Many of our competitors have substantially greater resources than we do, and we expect that all of our product candidates under development will face intense competition from existing or future drugs.

We expect that all of our product candidates under development, if approved, will face intense competition from existing and future drugs marketed by large companies.

Many of our competitors have substantially greater resources than we do and we expect that all our product candidates under development will face intense competition from existing or future drugs.

We expect that all our product candidates under development, if approved, will face intense competition from existing and future drugs marketed by large companies.

These competitors may successfully market products that compete with our products, successfully identify product candidates or develop products earlier than we do, or develop products that are more effective, have fewer side effects or cost less than our products.

5 unchanged sentences

The market exclusivity period prevents a successful generic or biosimilar applicant from commercializing its product in the EU until ten (10) years have elapsed from the initial marketing authorization of the reference product in the EU.

The overall ten-year period may, occasionally, be extended for a further year to a maximum of eleven years if, during the first eight years following authorization of the reference product, the marketing authorization holder obtains an authorization for one or more new therapeutic indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit in comparison with existing

There is, however, no guarantee that a product will be considered by the EU’s regulatory authorities to be a new chemical/biological entity, and products may not qualify for data exclusivity.

The overall ten-year period may occasionally be extended for a further year to a maximum of eleven (11) years if, during the first (8) eight years following authorization of the reference product, the marketing authorization holder obtains an authorization for one or more new therapeutic indications which, during the scientific evaluation prior to their authorization, are held to bring a significant clinical benefit in comparison with existing therapies.

There is, however, no guarantee that a product will be considered by the EU’s regulatory authorities to be a new chemical/biological entity and therefore products may not qualify for data

In the EU there is also a special regime for biosimilars, or biological medicinal products that are similar to a reference medicinal product but that do not meet the definition of a generic medicinal product.

5 unchanged sentences

We compete with companies that are pursuing other forms of treatment for the same or similar indications we are pursuing including established pharmaceutical and biotechnology companies and that have greater financial and other resources.

While we are not currently aware of any other companies that are taking the same therapeutic approach to protein folding disorders similar to the ones we are pursuing, we are aware of companies developing products for the same target indications.

While we are not currently aware of any other companies that are taking the same therapeutic approach to protein folding disorders to the ones we are pursuing, we are aware of companies developing products for the same target indications.

Mergers and acquisitions in the biotechnology and pharmaceutical industries may result in even more resources being concentrated among a smaller number of competitors.

10 unchanged sentences

Our business and operations may be adversely affected by health epidemics or pandemics.

Our business and operations may be adversely affected by pandemics or epidemics, including due to business interruptions caused by travel restrictions, quarantines, “stay-at-home” and "shelter-in-place" orders, shutdowns requested or mandated by governmental authorities, or staffing shortages while employees quarantine as a result of exposure to or transmission of the virus.

In addition, health epidemics or pandemics could cause significant disruption in the operations of third-party manufacturers, CROs and other third parties upon whom we rely.

Our business and operations may be adversely affected by pandemics or epidemics, including business interruptions caused by travel restrictions, quarantines, “stay-at-home” and "shelter-in-place" orders, shutdowns requested or mandated by governmental authorities or staffing shortages while employees quarantine as a result of exposure to or transmission of the virus.

In addition, health epidemics or pandemics could cause significant disruption in the operations of third-party manufacturers, CROs and other parties upon whom we rely.

For example, the COVID-19 pandemic presented a substantial public health and economic challenge around the world and affected employees, patients, communities and business operations as well as the global economy and financial markets.

The COVID-19 pandemic and the resulting post-pandemic environment has impacted clinical site activation and patient enrollment.

Clinical trial sites have experienced limited capacity and staffing shortages in a post-COVID-19 environment, partially due to personnel having been reassigned during the pandemic, resulting in a backlog of patient enrollment and delayed site initiations across the industry.

Our inability to successfully recruit and retain patients and principal investigators and site staff could adversely impact our expected future clinical trial operations.

The COVID-19 pandemic and the resulting post-pandemic environment impacted clinical site activation and patient enrollment.

Clinical trial sites experienced limited capacity and staffing shortages in a post-COVID-19 environment, partially due to personnel having been reassigned during the pandemic, resulting in a backlog of patient enrollment and delayed site initiations across the industry.

Our inability to successfully recruit and retain patients and principal investigators and site staff in these circumstances could adversely impact our expected future clinical trial operations.

Risks Related to Our Intellectual Property

5 unchanged sentences

If we breach the terms of the Minoryx License, for example, by failing to comply with any material terms thereof, Minoryx may have the right to terminate the license.

If we were to lose our license under this agreement, we would not be able to market certain of our products and technology, which would likely require us to cease our current operations and have an immediate material adverse effect on our business, operating results and financial condition.

If we were to lose our license under this agreement, we would not be able to market certain of our product candidates and technology which would likely require us to cease our current operations and have an immediate material adverse effect on our business, operating results and financial condition.

Our success depends substantially upon our ability to obtain and maintain intellectual property protection relating to our products and technologies.

4 unchanged sentences

Even if patents do issue, we cannot be sure that the claims of these patents will be held valid or enforceable by a court of law, will provide us with any significant protection against competing products or will afford us a commercial advantage over competitive products.

If, at some point in the future, one or more products resulting from our product candidates is approved for sale by the FDA and we do not have adequate intellectual property protection for those products, competitors could duplicate them for approval and sale in the United States without repeating the extensive testing required of us to obtain FDA approval.

without repeating the extensive testing required of us to obtain FDA approval.

If we fail to protect our intellectual property rights, our ability to pursue the development of our technologies and products would be negatively affected.

1 unchanged sentence

If we do not adequately maintain or protect our intellectual property, competitors may be able to use our technologies to produce and market drugs in direct competition with us and erode our competitive advantage.

Furthermore, some foreign countries lack rules and methods for defending intellectual property rights and do not protect proprietary rights to the same extent as the United States.

Furthermore, some foreign countries lack rules and methods for defending intellectual property rights and do not protect proprietary rights to the same extent as the U.S.

Many companies have had difficulty protecting their proprietary rights in these foreign countries.

4 unchanged sentences

patents that may be issued or licensed may be challenged, invalidated, or circumvented, or otherwise may not provide us any competitive advantage;

our competitors, many of which have substantially greater resources than we and many of which have made significant investments in competing technologies, may seek, or may already have obtained, patents that will limit, interfere with, or eliminate our ability to make, use, and sell our potential products either in the United States or in international markets;

there may be significant pressure on the United States government and other international governmental bodies to limit the scope of patent protection both inside and outside the United States for treatments that prove successful as a matter of public policy regarding worldwide health concerns;

and countries other than the United States may have less robust patent laws than those upheld by United States courts, allowing foreign competitors the ability to exploit these laws to create, develop, and market competing products using our technologies and patents.

or in international markets;

there may be significant pressure on the U.S.

government and other international governmental bodies to limit the scope of patent protection both inside and outside the U.S.

for treatments that prove successful as a matter of public policy regarding worldwide health concerns;

and countries other than the U.S.

may have less robust patent laws than those upheld by U.S.

courts, allowing foreign competitors the ability to exploit these laws to create, develop, and market competing products using our technologies and patents.

Moreover, any patents issued to us may not provide us with meaningful protection or others may challenge, circumvent or narrow our patents.

2 unchanged sentences

While extensions of patent terms due to regulatory delays may be available, it is possible that, before any of our product candidates can be commercialized, any related patent, even with an extension, may expire or remain in force for only a short period following commercialization thereby reducing any advantages to us of the patent.

In addition, the PTO and patent offices in other jurisdictions have often required that patent applications concerning pharmaceutical and/or biotechnology-related inventions be limited or narrowed substantially to cover only the innovations specifically exemplified in the patent application, thereby limiting the scope of protection against competitive challenges.

In addition, the U.S.

Patent and Trademark Office (“PTO”) and patent offices in other jurisdictions have often required that patent applications concerning pharmaceutical and/or biotechnology-related inventions be limited or narrowed substantially to cover only the innovations specifically exemplified in the patent application, thereby limiting the scope of protection against competitive challenges.

Thus, even if we or our licensors are able to obtain patents, the patents may be substantially narrower than anticipated which could deprive us of rights necessary for the successful commercialization of our product candidates.

3 unchanged sentences

We have not conducted any formal search of patents issued to third parties, and third-party patents containing claims covering our product candidates that predate our patents may exist.

Because of the number of patents issued and patent applications filed in our technical areas or fields, our competitors or other third parties may assert that our product candidates are covered by United States or foreign patents held by them.

or foreign patents held by them.

In addition to patents, we rely on a combination of trade secrets, confidentiality, nondisclosure and other contractual provisions and security measures to protect our confidential and proprietary information.

2 unchanged sentences

In addition, others may independently develop similar proprietary information or techniques or otherwise gain access to our trade secrets which could impair any competitive advantage we may have.

Patent protection and other intellectual property protection is crucial to the success of our business and prospects, and there is a substantial risk that such protections will prove inadequate.

Patent protection and other intellectual property protection is crucial to the success of our business and prospects and there is a substantial risk that such protections may prove inadequate.

We may be involved in lawsuits to protect or enforce our patents, which could be expensive and time consuming.

4 unchanged sentences

An adverse determination in litigation to which we may become a party could subject us to significant liabilities, require us to obtain licenses from third parties or restrict or prevent us from selling our products in certain markets.

Although patent and intellectual property disputes might be settled through licensing or similar arrangements, the costs associated with such arrangements may be substantial and could include our paying large, fixed payments and ongoing royalties.

Although patent and intellectual property disputes might be settled through licensing or similar arrangements, the costs associated with such arrangements may be substantial and could include paying large, fixed payments and ongoing royalties.

Furthermore, the necessary licenses may not be available on satisfactory terms or at all.

5 unchanged sentences

Third parties may also assert that our patents are invalid in patent office administrative proceedings.

These proceedings include oppositions in the European Patent Office and inter partes review and post-grant review proceedings in the PTO.

The success rate of these administrative challenges to patent validity in the United States is higher than it is for validity challenges in litigation.

These proceedings include oppositions in the European Patent Office as well as inter partes review and post-grant review proceedings in the PTO.

The success rate of these administrative challenges to patent validity in the U.S.

is higher than it is for validity challenges in litigation.

Interference or derivation proceedings brought before the PTO may be necessary to determine priority of inventions disclosed in our patents or patent applications.

Determining whether a product infringes a patent, as well as priority of inventions and other patent-related disputes, involves complex legal and factual issues and the outcome is often uncertain.

During these proceedings, it may be determined that we do not have priority of invention for one or more aspects in our patents or patent applications and could result in the invalidation in part or whole of a patent or could put a patent application at risk of not issuing.

During these proceedings, it may be determined that we do not have priority of invention for one or more aspects in our patents or patent applications which could result in the invalidation in part or whole of a patent or could put a patent application at risk of not issuing.

Even if successful, an interference or derivation proceeding may result in substantial costs and distraction to our management.

7 unchanged sentences

If we infringe the rights of third parties, we could be prevented from selling products or forced to pay damages and defend against litigation.

If our products, methods, processes and other technologies infringe the proprietary rights of other parties, we could incur substantial costs and we may have to:

obtain licenses, which may not be available on commercially reasonable terms or at all;

If our products, methods, processes and other technologies infringe the proprietary rights of other parties, we could incur substantial costs and we may have to obtain licenses, which may not be available on commercially reasonable terms or at all;

abandon an infringing product candidate;

1 unchanged sentence

stop using the subject matter claimed in the patents held by others;

and/or defend litigation or administrative proceedings which may be costly whether we win or lose, and which could result in a substantial diversion of our financial and management resources.

In addition, because patent applications can take many years to issue and because publication schedules for pending applications vary by jurisdiction, there may be applications now pending of which we are unaware, and which may result in issued patents that our future products would infringe.

and/or defend litigation or administrative proceedings which may be costly whether we win or lose and could result in a substantial diversion of our financial and management resources.

In addition, because patent applications can take many years to issue and publication schedules for pending applications vary by jurisdiction, there may be applications now pending of which we are unaware and may result in issued patents that our future products would infringe.

Also, because the claims of published patent applications can change between publication and patent grant, there may be published patent applications that may ultimately issue with claims that we infringe.

We have licensed certain rights, assets and technology related to the Magellan™ platform from Minoryx and we believe that they owned all of such rights prior to our license.

We have licensed certain rights, assets and technology related to the Magellan™ platform from Minoryx and we believe that they owned all such rights prior to our license.

Although, to our knowledge, no third party has asserted a claim of infringement or other claim against us, others may hold or claim to hold proprietary or other rights that could prevent our Magellan™ platform from being developed or marketed.

25 unchanged sentences

In either scenario, our clinical trials supply could be delayed significantly as we establish alternative supply sources.

In some cases, the technical skills required to manufacture our products or product candidates may be unique or proprietary to the original CMO and we may have difficulty, or there may be contractual restrictions prohibiting us from, transferring such skills to a back-up or alternate supplier, or we may be unable to transfer such skills at all.

In addition, if we are required to change CMOs for any reason, we will be required to verify that the new CMO maintains facilities and procedures that comply with quality standards and with all applicable regulations.

We will also need to verify, such as through a manufacturing comparability study, that any new manufacturing process will produce our product candidates according to any specifications previously submitted to the FDA or another comparable foreign regulatory authority.

The delays associated with the verification of a new CMO could negatively affect our ability to develop product candidates or commercialize our products in a timely manner or within budget.

Furthermore, a CMO may possess technology related to the manufacture of our product candidate that such CMO owns independently.

Furthermore, a CMO may possess technology related to the manufacture of our product

candidate that such CMO owns independently.

This would increase our reliance on such CMO or require us to obtain a license from such CMO in order to have another CMO manufacture our product candidates.

16 unchanged sentences

Third-party manufacturers must be inspected by the FDA and comparable foreign regulatory authorities for cGMP compliance before they can produce commercial products.

We may be in competition with other companies for access to these manufacturers’ facilities and may be subject to delays in manufacture if the manufacturers give other clients higher priority than they give to us.

If we are unable to secure and maintain third-party manufacturing capacity, the development and sales of our products and our financial performance may be materially affected.

Manufacturers are obligated to operate in accordance with requirements mandated by the FDA or comparable foreign regulatory authorities.

1 unchanged sentence

This could result in higher costs to us or deprive us of potential product revenues.

ensure strict compliance with cGMP requirements and other requirements under federal drug laws, other government regulations and corresponding foreign laws, regulations and standards.

Drug manufacturers are subject to ongoing periodic unannounced inspections by the FDA, the Drug Enforcement Administration (“DEA”) and corresponding state and foreign regulatory authorities to monitor and ensure strict compliance with cGMP requirements and other requirements under federal drug laws, other government regulations and corresponding foreign laws, regulations and standards.

If we or our third-party manufacturers fail to comply with applicable regulations, sanctions could be imposed on us, including fines, injunctions, civil penalties, failure by the government or competent regulatory authorities to grant marketing approval of drugs, delays, suspension, variation or withdrawal of approvals, seizures or recalls of product, shutdown of the manufacturer, invalidation of drug lots or processes, operating restrictions, product recalls and criminal prosecutions.

7 unchanged sentences

In addition, a lack of development and marketing collaborations may lead to significant delays in introducing proposed products into certain markets and/or reduced sales of proposed products in such markets.

Changes in U.S.

and international trade policies, particularly with respect to China, may adversely impact our business and operating results.

One of our primary manufacturers and suppliers, WuXi AppTec (“WuXi”), is located in China and the subject of increased U.S.

government scrutiny.

Trade tensions and conflicts between the United States and China have been escalating in recent years and, as such, we are exposed to the possibility of product and material supply disruption and increased costs and expenses in the event of changes to the laws, rules, regulations and policies of the governments of the United States or China, or due to geopolitical unrest and unstable economic conditions.

government has recently made statements and taken certain actions that may lead to potential changes to U.S.

and international trade policies, including imposing several rounds of tariffs and export control restrictions affecting certain products manufactured in China.

Increased focus on relations with China has included U.S.

legislative proposals, such as the proposed BIOSECURE Act, which has been passed by the U.S.

House of Representatives and is pending before the U.S.

If enacted, the BIOSECURE Act would, among other things, prohibit U.S.

federal agencies from entering into or renewing any contract with any entity that uses biotechnology equipment or services produced or provided by a “biotechnology company of concern” to perform that contract with the government.

Although the proposed BIOSECURE Act has not been enacted and thus is subject to change through the legislative process, a version of the BIOSECURE Act passed by the U.S.

House of Representatives defines a “biotechnology company of concern” to include WuXi.

If adopted, the BIOSECURE Act could cause us to seek to exit some or all of our arrangements with WuXi (or any other China-based service provider determined to be “biotechnology companies of concern”) and accelerate the transition of these services to alternative companies or continue to engage redundant suppliers for the U.S.

Additionally, the legislation could adversely impact WuXi’s operations or financial position which, in turn, could impact its ability to perform under our agreements with it.

Our reliance on Chinese-based contract research organizations, such as WuXi, may also cause us to face additional risks due to geopolitical tensions between the U.S.

and China and related legal and regulatory restrictions and requirements, including measures directly affecting WuXi.

Any unfavorable government policies on international trade, such as export controls, capital controls or tariffs, may increase the cost of manufacturing our product candidates and platform materials, affect the demand for our drug products (if and once approved), the competitive position of our product candidates, and import or export of raw materials and finished product candidate used in our and our collaborators’ preclinical studies and clinical trials, particularly with respect to any product candidates and materials that we import from China, including pursuant to our arrangements with WuXi.

If any new tariffs, export controls, legislation and/or regulations are implemented, or if existing trade agreements are renegotiated or, in particular, if either the U.S.

or Chinese government takes retaliatory trade actions due to the recent trade tension, such changes could have an adverse effect on our business, financial condition and results of operations.

Any negative impact of the ability of our third party collaborators to deliver the materials we require to conduct our clinical operations due to political actions, supply chain disruptions or otherwise, may have a material adverse impact on our results of operations or financial condition.

Data provided by collaborators and others upon which we rely that has not been independently verified could turn out to be false, misleading, or incomplete.

2 unchanged sentences

If we fail to establish marketing, sales and distribution capabilities, or fail to enter into arrangements with third parties, we will not be able to create a market for our product candidates.

Our strategy for our product candidates is to control, directly or through contracted third parties, all or most aspects of the product development process, including marketing, sales and distribution.

Currently, we do not have any sales, marketing or distribution capabilities.

In order to generate sales of any product candidates that receive regulatory approval, we must either acquire or develop an internal marketing and sales force with technical expertise and with supporting distribution capabilities or make arrangements with third parties to perform these services for us.

The acquisition or development of a sales and distribution infrastructure would require substantial resources, which may divert the attention of our management and key personnel and defer our product development efforts.

To the extent that we enter into marketing and sales arrangements with other companies, our revenues will depend on the efforts of others.

These efforts may not be successful.

If we fail to develop sales, marketing and distribution channels, or enter into arrangements with third parties, we will experience delays in product sales and incur increased costs.

Sales of pharmaceutical products largely depend on the reimbursement of patients’ medical expenses by government health care programs and private health insurers.

Without the financial support of the government or third-party payors, the market for our products will be limited.

These third-party payors are increasingly challenging the price and examining the cost effectiveness of medical products and services.

Recent proposals to change the health care system in the United States have included measures that would limit or eliminate payments for medical products and services or subject the pricing of medical treatment products to government control.

Significant uncertainty exists as to the reimbursement status of newly approved health care products.

Third-party payors may not reimburse sales of our products or enable our collaborators to sell them at profitable prices.

Our business strategy might involve out-licensing product candidates to or collaborating with larger firms with experience in marketing and selling pharmaceutical products.

We may not be able to successfully establish marketing, sales, or distribution relationships and such relationships, if established, may not be successful.

Further, we may not be successful in gaining market acceptance for our products.

To the extent that we enter into any marketing,

sales, or distribution arrangements with third parties, our product revenues will be lower than if we marketed and sold our products directly, and any revenues we receive will depend upon the efforts of such third-parties.

If we are unable to establish such third-party sales and marketing relationships, or choose not to do so, we will have to establish and rely on our own in-house capabilities.

We, as a company, have no experience in marketing or selling pharmaceutical products and currently have no sales, marketing, or distribution infrastructure.

To market any of our products directly, we would need to develop a marketing, sales, and distribution force that both has technical expertise and the ability to support a distribution capability.

The establishment of a marketing, sales, and distribution capability would significantly increase our costs, possibly requiring substantial additional capital.

In addition, there is intense competition for proficient sales and marketing personnel, and we may not be able to attract individuals who have the qualifications necessary to market, sell, and distribute our products.

We may not be able to establish internal marketing, sales, or distribution capabilities.

If we are unable to, or choose not to establish these capabilities, or if the capabilities we establish are not sufficient to meet our needs, we will be required to establish collaborative marketing, sales, or distribution relationships with third parties.

If any of our existing or future collaborative partners do not satisfy their obligations, or if we are unable to enter into collaboration agreements with partners on favorable terms, we will be unable to develop our partnered product candidates.

We may not have day-to-day control over the activities of our existing and future collaborative partners with respect to any of our partnered product candidates.

Any collaborative partner may not fulfill its obligations under our collaboration agreements.

If a collaborative partner fails to fulfill its obligations under an agreement with us, we may be unable to assume the development of the products covered by that agreement or enter into alternative arrangements with a third party.

In addition, we may encounter delays in the commercialization of the product candidate that is the subject of the agreement.

Accordingly, our ability to receive any revenue from the product candidates covered by these agreements will be dependent on the efforts of our collaborative partner.

We could also become involved in disputes with a collaborative partner, which could lead to delays in or termination of our development and commercialization programs and time-consuming and expensive litigation or arbitration.

In addition, any such dispute could diminish our collaborators’ commitment to us and reduce the resources they devote to developing and commercializing our products.

Conflicts or disputes with our collaborators, and competition from them, could harm our relationships with our other collaborators, restrict our ability to enter future collaboration agreements and delay the research, development or commercialization of our product candidates.

If any collaborative partner terminates or breaches its agreement, or otherwise fails to complete its obligations in a timely manner, our chances of successfully developing or commercializing these product candidates would be materially and adversely affected.

We may not be able to enter into collaboration agreements with partners on terms favorable to us, or at all.

Our inability to enter into collaborative arrangements with collaborative partners, or our failure to maintain such arrangements, would limit the number of product candidates that we could develop and ultimately decrease our sources of any future revenues.

We face risks in connection with existing and future collaborations with respect to the development, manufacture and commercialization of our product candidates.

We face a number of risks in connection with our current and future collaborations.

Our collaboration agreements are subject to termination under various circumstances.

Our collaborators may change the focus of their development and commercialization efforts or may have insufficient resources to effectively assist in the development of our products.

Any future collaboration agreements may have the effect of limiting the areas of research and development that we may pursue, either alone or in collaboration with third parties.

Further, disagreements with collaborators, including disagreements over proprietary rights, contract interpretation, or the preferred course of development, might cause delays, might result in litigation or arbitration, or might result in termination of the research, development or commercialization of our products.

Any such disagreements would divert management attention and resources and be time-consuming and costly.

General Risk Factors

1 unchanged sentence

If we fail to maintain proper and effective system of internal controls over financial reporting in the future, our ability to produce accurate and timely financial statements could be impaired which could harm our operating results, investors’ views of us and, as a result, the value of our securities.

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

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

In addition, Section 404 of the Sarbanes-Oxley Act of 2002 (“Section 404”) and related SEC rules require management to furnish a report on the effectiveness of our internal control over financial reporting.

4 unchanged sentences

As a result, shareholders could lose confidence in our financial and other public reporting, which would harm our business and the market value of our common stock.

Global and macroeconomic conditions, including economic, political and social instability could adversely affect our revenue, financial condition, or results of operations.

The global credit and financial markets have recently experienced extreme volatility and disruptions, including severely diminished liquidity and credit availability, disruptions in access to bank deposits and lending commitments due to bank failures, supply chain disruptions, heightened interest rates and inflation, stock volatility, as well as uncertainty about economic stability.

Global and macroeconomic conditions, including economic, political and social instability, could adversely affect our financial condition or results of operations.

The global credit and financial markets have recently experienced extreme volatility and disruptions including severely diminished liquidity and credit availability, disruptions in access to bank deposits and lending commitments due to bank failures, declines in economic growth, increases in unemployment rates, supply chain disruptions, heightened interest rates and inflation, stock volatility and uncertainty about economic stability.

Such conditions may continue or worsen in the future.

The financial markets and the global economy may also be adversely affected by the current or anticipated impact of military conflict including Russia’s invasion of Ukraine and the conflict between Hamas and Israel, terrorism or other geopolitical events.

Sanctions imposed by the United States and other countries in response to such conflicts, including the war in Ukraine and the conflict between Hamas and Israel, may also adversely impact the financial markets and the global economy, and any economic countermeasures by affected countries and others could exacerbate market and economic instability.

In 2018 and 2019, the United States imposed tariffs on goods imported from China and certain other countries.

Although the United States and China signed a new trade agreement in January 2020, most of the previously-implemented tariffs on goods imported from China remain in place.

Sanctions imposed by the U.S.

and other countries in response to such conflicts, including sanctions imposed in connection with the war in Ukraine and the conflict between Hamas and Israel, the effect of tariffs and/or any resulting trade wars, increasing interest rates, or other factors may also adversely impact the financial markets and the global economy and any economic countermeasures by affected countries and others could exacerbate market and economic instability.

For example, in late 2024 and early 2025, the United States, Canada, China, and the European Union each announced either new tariffs, non-tariff barriers, or export controls.

Any of these risks, ensuing retaliation, or the further deterioration of trade relations between countries could have an adverse impact on our financial condition and results of operations.

Additional tariffs or further retaliatory trade measures taken by China or other countries in response could affect the demand for any of our products, impact the competitive position of our products, prevent us from being able to sell products in certain countries or otherwise adversely impact our results of operations.

Growing tensions, protectionist trade policies, and tariffs may also lead to a fragmentation of the global economy, a general reduction of international trade in goods and services, and a reduction in the integration of financial markets, any of which could materially and adversely affect our financial condition, or prospects.

There can be no assurance that further deterioration in credit and financial markets and confidence in economic conditions will not occur.

Our general business strategy as well as our suppliers’ ability to provide us with raw materials and components, may be adversely affected by any such economic downturn, volatile business environment or continued unpredictable and unstable market conditions, which could directly affect our ability to attain our operating goals on schedule and on budget, including requiring us to delay or abandon certain development plans and could have a material adverse effect on our growth strategy, financial performance and stock price.

In addition, there is a risk that one or more of our current suppliers, may not survive an economic downturn, which could directly affect our ability to attain our operating goals on schedule and on budget.

In addition, there is a risk that one or more of our current suppliers may not survive an economic downturn, which could directly affect our ability to attain our operating goals on schedule and within budget.

Changes in trade policies, including the imposition of tariffs or other trade restrictions, could materially impact our ability to obtain the raw materials, active pharmaceutical ingredients, and other components necessary for the manufacturing of our product candidates used in our clinical development activities.

Some of these materials may be sourced from foreign suppliers, and any increase in tariffs or duties on imported goods could significantly raise the cost of doing business.

Additionally, retaliatory tariffs, trade disputes, trade wars, or changes in international trade agreements may lead to supply chain disruptions, including delays in obtaining critical components or the need to seek alternative suppliers.

If we are unable to mitigate the impact of increased costs or supply chain disruptions, our financial condition, and ability to develop our product candidates in a timely manner, could be adversely affected.

We will need to expand our operations and increase the size of our company, and we may experience difficulties in managing growth.

As we advance our product candidates through preclinical studies and clinical trials, and develop new product candidates, we will need to increase our product development, scientific, regulatory and compliance and administrative headcount to manage these programs.

In addition, to continue to meet our obligations as a public company, and particularly after we will no longer qualify as an emerging growth company, we will need to increase our general and

administrative capabilities.

As we advance our product candidates through preclinical studies and clinical trials and develop new product candidates, we will need to increase our product development as well as our scientific, regulatory and compliance and administrative headcount to manage these programs.

In addition, to continue to meet our obligations as a public company, and particularly when we no longer qualify as an emerging growth company, we will need to increase our general and administrative capabilities.

Our management, personnel and systems currently in place may not be adequate to support this future growth.

11 unchanged sentences

While our senior executive officers are parties to employment agreements with us, these agreements do not guarantee that they will remain employed with us in the future.

In addition, these our arrangements with our senior executive officers include only limited, if any, restrictions on our senior executive officers’ ability to compete with us after their employment is terminated.

The competition for qualified personnel in the pharmaceutical field is intense, and there is a limited pool of qualified potential employees to recruit.

In addition, our arrangements with our senior executive officers include only limited, if any, restrictions on our senior executive officers’ ability to compete with us after their employment is terminated.

The competition for qualified personnel in the pharmaceutical field is intense and there is a limited pool of qualified potential employees.

Due to the intense competition for talent, we may be unable to continue to attract and retain qualified personnel necessary for the development of our business or to recruit suitable replacement personnel.

2 unchanged sentences

The value to employees of such awards may be significantly affected by movements in our stock price and current market conditions and extreme stock price volatility may diminish our ability to incentivize employees through the use of such awards.

If we are unsuccessful in our recruitment and retention efforts, our business may be harmed.

If we are unsuccessful in our recruitment and retention efforts, our business may be adversely affected.

Under applicable employment laws, we may not be able to enforce covenants not to compete and therefore may be unable to prevent our competitors from benefiting from the expertise of some of our former employees.

3 unchanged sentences

If we are unable to enforce these covenants not to compete, we may be unable to prevent our competitors from benefiting from the expertise of our former employees or consultants and our competitiveness may be diminished.

If we are unable to hire additional qualified personnel, our ability to grow our business may be harmed.

Over time we will need to hire additional qualified personnel with expertise in drug development, product registration, clinical, preclinical and nonclinical research, quality compliance, government regulation, formulation and manufacturing, financial matters and sales and marketing.

We compete for qualified individuals with numerous biopharmaceutical companies, universities and other research institutions.

Competition for such individuals is intense, and our search for such personnel may not be successful.

Attracting and retaining qualified personnel will be critical to our success.

Our relationships with customers, physicians, and third-party payors will be subject, directly or indirectly, to federal and state healthcare fraud and abuse laws, false claims laws, health information privacy and security laws and other healthcare laws and regulations including comparable foreign laws and regulations.

If we are unable to comply or have not fully complied with such laws, we could face substantial penalties.

Healthcare providers and third-party payors in the United States and elsewhere will play a primary role in the recommendation and prescription of any product candidates for which we obtain marketing approval.

Healthcare providers and third-party payors in the U.S.

and elsewhere will play a primary role in the recommendation and prescription of any product candidates for which we obtain marketing approval.

Our current and future arrangements with healthcare professionals, principal investigators, consultants, customers and third-party payors may subject us to various federal and state fraud and abuse laws and other healthcare laws including, without limitation, the federal Anti-Kickback Statute, the federal civil and criminal false claims laws and the law commonly referred to as the Physician Payments Sunshine Act and regulations, and foreign equivalent laws and regulations.

4 unchanged sentences

● federal civil and criminal false claims laws, including, without limitation, the False Claims Act and civil monetary penalty laws which prohibit, among other things, individuals or entities from knowingly presenting, or causing to be presented, claims for payment or approval from Medicare, Medicaid or other government payors that are false or fraudulent or making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government;

● HIPAA, which created new federal criminal statutes that prohibit a person from, among other things, knowingly and willfully executing a scheme or making false or fraudulent statements to defraud any healthcare benefit program, regardless of the payor (e.g., public or private);

● HIPAA, as amended by HITECH and its implementing regulations, and as amended again by the final HIPAA omnibus rule, Modifications to the HIPAA Privacy, Security, Enforcement, and Breach Notification Rules Under HITECH and the Genetic Information Nondiscrimination Act;

● the Health Insurance Portability and Accountability Act (“HIPAA”), which created new federal criminal statutes that prohibit a person from, among other things, knowingly and willfully executing a scheme or making false or fraudulent statements to defraud any healthcare benefit program, regardless of the payor (e.g., public or private);

● HIPAA, as amended by HITECH and its implementing regulations, and as amended again by the final HIPAA omnibus rule, Modifications to the HIPAA Privacy, Security, Enforcement, and Breach

Notification Rules Under HITECH and the Genetic Information Nondiscrimination Act;

Other Modifications to HIPAA, published in January 2013, which imposes certain requirements relating to the privacy, security and transmission of individually identifiable health information without appropriate authorization by entities subject to the rule, such as health plans, health care clearinghouses and certain health care providers, and their respective business associates and covered subcontractors;

14 unchanged sentences

Third-party payors decide which therapies they will pay for and establish reimbursement levels.

Third-party payors often rely upon Medicare coverage policy and payment limitations in setting their own coverage and reimbursement policies.

Third-party payors often rely upon Medicare coverage policy and payment limitations in setting their own coverage and

reimbursement policies.

However, decisions regarding the extent of coverage and amount of reimbursement to be provided for any product candidates that we develop will be made on a payor-by-payor basis.

One payor’s determination to provide coverage for a drug does not determine whether or not another payor will also provide coverage, and adequate reimbursement, for the drug.

One payor’s determination to provide coverage for a drug does not determine whether another payor will also provide coverage and adequate reimbursement for the drug.

Additionally, a third-party payor’s decision to provide coverage for a therapy does not imply that an adequate reimbursement rate will be approved.

Each payor determines whether or not it will provide coverage for a therapy, what amount it will pay the manufacturer for the therapy, and on what tier of its formulary it will be placed.

Each payor determines whether it will provide coverage for a therapy, what amount it will pay the manufacturer for the therapy and on what tier of its formulary it will be placed.

The position on a payor’s list of covered drugs, or formulary, generally determines the co-payment that a patient will need to make to obtain the therapy and can strongly influence the adoption of such therapy by patients and physicians.

2 unchanged sentences

Patients are unlikely to use our drugs unless coverage is provided, and reimbursement is adequate to cover a significant portion of the cost of our drugs.

Outside the United States, reimbursement and healthcare payment systems vary significantly by country, and many countries have instituted price ceilings on specific products and therapies.

Outside the U.S., reimbursement and healthcare payment systems vary significantly by country and many countries have instituted price ceilings on specific products and therapies.

For example, the EU provides options for EU Member States to restrict the range of medicinal products for which their national health insurance systems provide reimbursement and to control the prices of medicinal products for human use.

An EU Member State may approve a specific price for the medicinal product, it may refuse to reimburse a product at the price set by the manufacturer or it may instead adopt a system of direct or indirect controls on the profitability of the company placing the medicinal product on the market.

An EU Member State may approve a specific price for the medicinal product, refuse to reimburse a product at the price set by the manufacturer or it may instead adopt a system of direct or indirect controls on the profitability of the company placing the medicinal product on the market.

Many EU Member States also periodically review their reimbursement procedures for medicinal products which could have an adverse impact on reimbursement status.

8 unchanged sentences

In the United States and some foreign jurisdictions, there have been, and continue to be, several legislative and regulatory changes and proposed changes regarding the healthcare system that could prevent or delay marketing approval of product candidates, restrict or regulate post-approval activities, and affect our ability to profitably sell any product candidates for which we obtain marketing approval.

Among policy makers and payors in the United States and elsewhere, there is significant interest in promoting changes in healthcare systems with the stated goals of containing healthcare costs, improving quality and/or expanding access.

In the United States, the pharmaceutical industry has been a particular focus of these efforts and has been significantly affected by major legislative initiatives.

In March 2010, the ACA was passed, which substantially changed the way healthcare is financed by both the government and private insurers, and significantly impacts the U.S.

Among policy makers and payors in the U.S.

and elsewhere, there is significant interest in promoting changes in healthcare systems with the stated goals of containing healthcare costs, improving quality and/or expanding access.

In the U.S., the pharmaceutical industry has been a particular focus of these efforts and has been significantly affected by major legislative initiatives.

For example, the Affordable Care Act (“ACA”) of 2010 substantially changed the way healthcare is financed by both the government and private insurers and significantly impacts the U.S.

pharmaceutical industry.

The ACA has been subject to judicial and Congressional challenges.

For example, on June 17, 2021 the U.S.

Supreme Court dismissed a challenge on procedural grounds that argued the ACA is unconstitutional in its entirety because the “individual mandate” was repealed by Congress.

Prior to the U.S.

Supreme Court ruling, on January 28, 2021, President Biden issued an executive order that initiated a special enrollment period for purposes of obtaining health insurance coverage through the ACA marketplace.

The executive order also instructed certain governmental agencies to review and reconsider their existing policies and rules that limit access to healthcare, including among others, reexamining Medicaid demonstration projects and waiver programs that include work requirements, and policies that create unnecessary barriers to obtaining access to health insurance coverage through Medicaid or the ACA.

On August 16, 2022, President Biden signed the Inflation Reduction Act of 2022 (“IRA”) into law, which among other things, extends enhanced subsidies for individuals purchasing health insurance coverage in ACA marketplaces through plan year 2025.

The ACA has been subject to judicial and Congressional challenges but remains in place for all intents and purposes.

The Inflation Reduction Act of 2022 (“IRA”), which among other things, extends enhanced subsidies for individuals purchasing health insurance coverage in ACA marketplaces through plan year 2025.

The IRA also eliminates the “donut hole” under the Medicare Part D program beginning in 2025 by significantly lowering the beneficiary maximum out-of-pocket cost and through a newly established manufacturer discount program, it also allows the U.S.

1 unchanged sentence

The implementation of the IRA is currently subject to ongoing litigation challenging the constitutionality of the IRA's Medicare drug price negotiation program.

The effect of IRA on our business and the healthcare industry in general is not yet known.

Other legislative changes have been proposed and adopted since the ACA was enacted, including aggregate reductions to Medicare payments to providers of up to 2% per fiscal year, which went into effect in April 2013 and, due to subsequent legislative amendments to the statute, will remain in effect through 2032, unless additional Congressional action is taken.

Additionally, there has been heightened governmental scrutiny in the United States of pharmaceutical pricing practices in light of the rising cost of prescription drugs and biologics.

Such scrutiny has resulted in several recent congressional inquiries, Presidential executive orders and proposed and enacted federal and state legislation designed to bring more transparency to product pricing, review the relationship between pricing and manufacturer patient programs and reform government program reimbursement methodologies for products.

For example, in July 2021, the Biden administration released an executive order, “Promoting Competition in the American Economy,” with multiple provisions aimed at prescription drugs.

In response to Biden’s executive order, on September 9, 2021, the U.S.

Department of Health and Human Services (“HHS”) released a Comprehensive Plan for Addressing High Drug Prices that outlines principles for drug pricing reform and sets out a variety of potential legislative policies that Congress could pursue as well as potential administrative actions HHS can take to advance these principles.

In addition, the IRA, among other things, (1) directs HHS to negotiate the price of certain high expenditure single-source drugs and biologics covered under Medicare and (2) imposes rebates under Medicare Part B and Medicare Part D to penalize price increases that outpace inflation.

These provisions will take effect progressively starting in fiscal year 2023, although the Medicare drug price negotiation program is currently subject to legal challenges.

HHS has and will continue to issue and update guidance as these programs are implemented.

It is currently unclear how the IRA will be implemented but is likely to have a significant impact on the pharmaceutical industry.

In addition, in response to the Biden administration’s October 2022 executive order, on February 14, 2023, HHS released a report outlining three new models for testing by the Center for Medicare and Medicaid Innovation which will be evaluated on their ability to lower the cost of drugs, promote accessibility, and improve quality of care.

It is unclear whether the models will be utilized in any health reform measures in the future.

Although a number of these and other proposed measures may require authorization through additional legislation to become effective, and the Biden administration may reverse or otherwise change these measures, both the Biden administration and Congress have indicated that they will continue to seek new legislation measures to control drug costs.

The effect of IRA on our business and the healthcare industry

in general is not yet known.

With the recent change in administration, the future of the IRA and its effects remain uncertain.

Other legislative changes have been proposed and adopted since the ACA was enacted including aggregate reductions to Medicare payments to providers of up to 2% per fiscal year, which went into effect in April 2013 and will remain in effect through 2032 unless additional Congressional action is taken.

Moreover, changes to the political landscape in the United States may impact the market sentiment surrounding the pharmaceutical industry.

In addition, in order to obtain reimbursement for our products in some European countries, including some EU Member States, we may be required to compile additional data comparing the cost-effectiveness of our products to other available therapies.

Since retaking office, President Trump has signed several Executive Orders that may impact the health and pharmaceutical industry.

On January 20, 2025, President Trump began the action of withdrawing the United States from the World Health Organization.

This order also rescinded a prior executive order signed by formed President Biden that coordinated the federal government’s COVID-19 response efforts and implemented processes to respond to emerging pandemics.

In addition, President Trump has proposed reductions in federal research spending that may impact organizations such as the National Institutes of Health, the National Science Foundation and the Centers for Disease Control and Prevention.

Funding from government agencies and reimbursement programs such as the NIH, Medicare and Medicaid, including the overall availability and reimbursement rates under these programs, often fluctuates and is subject to the political process, which is often unpredictable.

For example, on February 7, 2025, the NIH issued Notice Number NOT-OD-25-068, a guidance document pronouncing that reimbursement for certain indirect costs would be capped at 15% for existing and future grant recipients, a rate that is lower than the in-place rate for many existing grant recipients.

Certain of our third party collaborators may depend on NIH grants and reimbursements to partially fund research.

Any reduction in the availability or rate of funding or reimbursement, or delays surrounding the approval of such funding or reimbursement, may adversely impact our ability to develop our product candidates.

The outcome of these orders is, and will likely remain, uncertain for the foreseeable future.

In addition, to obtain reimbursement for our products in some European countries including some EU Member States, we may be required to compile additional data comparing the cost-effectiveness of our products to other available therapies.

The Health Technology Assessment, or HTA, of medicinal products is becoming an increasingly common part of the pricing and reimbursement procedures in some EU Member States including those representing the larger markets.

8 unchanged sentences

If we are unable to maintain favorable pricing and reimbursement status in EU Member States for product candidates that we may successfully develop and for which we may obtain regulatory approval, any anticipated revenue from and growth prospects for those products in the EU could be negatively affected.

We expect that these and other healthcare reform measures that may be adopted in the future may result in more rigorous coverage criteria and in additional downward pressure on the price that we receive for any approved drug.

We expect that these and other healthcare reform measures that may be adopted in the future may result in more rigorous coverage criteria and additional downward pressure on the price that we receive for any approved drug.

Any reduction in reimbursement from Medicare or other comparable foreign programs may result in a similar reduction in payments from private payors.

The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize our drugs.

We expect that additional state and federal healthcare reform measures will be adopted in the future, any of which could limit the amounts that federal and state governments will pay for healthcare products and services, which could result in reduced demand for our product candidates or additional pricing pressures.

If we obtain approval to commercialize any approved products outside of the United States, a variety of risks associated with international operations could materially adversely affect our business.

If any of our product candidates are approved for commercialization outside of the United States, we intend to enter into agreements with third parties to market them on a worldwide basis or in more limited geographical regions.

We expect that we will be subject to additional risks related to entering into international business relationships, including:

6 unchanged sentences

● foreign currency fluctuations, which could result in increased operating expenses and reduced revenues, 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;

● workforce uncertainty in countries where labor unrest is more common than in the U.S.;

● production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad;

1 unchanged sentence

Foreign Corrupt Practices Act, the U.K.

Bribery Act 2010 and similar anti-bribery and anticorruption laws in other jurisdictions;

● business interruptions resulting from geopolitical actions, including war (such as Russia’s invasion of Ukraine and the conflict between Hamas and Israel) and terrorism, or natural disasters including earthquakes, hurricanes, floods and fires, economic or political instability, sanctions, or public health emergencies, and related shelter-in-place orders, travel, social distancing and quarantine policies, boycotts, curtailment of trade and other business restrictions;

Bribery Act 2010 or similar anti-bribery and anticorruption laws in other jurisdictions;

● business interruptions resulting from geopolitical actions including war (such as Russia’s invasion of Ukraine and the conflict between Hamas and Israel) and terrorism, natural disasters such as earthquakes, hurricanes, floods and fires, economic or political instability, sanctions, or public health emergencies, and related shelter-in-place orders, travel, social distancing and quarantine policies, boycotts, curtailment of trade and other business restrictions;

● difficulty in importing and exporting clinical trial materials and study samples.

16 unchanged sentences

Treasury Department’s Office of Foreign Assets Controls.

Export controls and trade sanctions laws and regulations may restrict or prohibit altogether the provision, sale, or supply of our product candidates to certain governments, persons, entities, countries, and territories, including those that are the target of comprehensive sanctions or an embargo.

We cannot ensure that all of our employees, agents, contractors or those of our affiliates, will comply with all applicable laws and regulations.

Export controls and trade sanctions laws and regulations may restrict or prohibit altogether the provision, sale, or supply of our product candidates to certain

governments, persons, entities, countries and territories including those that are the target of comprehensive sanctions or an embargo.

We cannot ensure that all our employees, agents, contractors or those of our affiliates will comply with all applicable laws and regulations.

Violations of anti-corruption, anti-money laundering, import and export control or sanctions laws and regulations could result in substantial civil and criminal fines and penalties, imprisonment, the loss of export or import privileges, debarment, breach of contract and fraud litigation, reputational harm and other consequences.

16 unchanged sentences

If we are unable to obtain insurance at an acceptable cost or otherwise protect against potential product liability claims, we will be exposed to significant liabilities which may materially and adversely affect our business and financial position.

If we are sued for any injury allegedly caused by our or our collaborators’ products, our liability could exceed our total assets and our ability to pay the liability.

If we are sued for any injury allegedly caused by our company’s or our collaborators’ products, our liability could exceed our total assets and our ability to pay the liability.

A product liability claim or series of claims brought against us would decrease our cash and could cause our stock price to fall.

10 unchanged sentences

Our data processing activities may subject us to numerous data privacy and security obligations, such as various laws, regulations, guidance, industry standards, external and internal privacy and security policies, contracts, and other obligations that govern the processing of personal information by us and on our behalf.

In the United States, federal, state, and local governments have enacted numerous data privacy and security laws, including data breach notification laws, personal information privacy laws, consumer protection laws (e.g., Section 5 of the Federal Trade Commission Act) and other similar laws (e.g., wiretapping laws).

For example, HIPAA, as amended by HITECH, imposes specific requirements relating to the privacy, security, and transmission of protected health information.

In the U.S., federal, state, and local governments have enacted numerous data privacy and security laws including data breach notification laws, personal information privacy laws, consumer protection laws (e.g., Section 5 of the Federal Trade Commission Act) and other similar laws (e.g., wiretapping laws).

For example, HIPAA, as amended by HITECH, imposes specific requirements relating to the privacy, security and transmission of protected

health information.

Several states have also enacted comprehensive data privacy laws, which either became effective in 2023 or will become effective within the next couple of years.

These state comprehensive data privacy laws provide individuals with certain rights concerning their personal information including the right to access, correct or delete certain personal information and opt-out of certain data processing activities such as targeted advertising, profiling, and automated decision-making.

One example of these comprehensive state data privacy laws is, the California Consumer Privacy Act of 2018, as amended by the California Privacy Rights Act of 2020 (“CPRA”) (collectively, “CCPA”), which applies to the personal information of consumers, business representatives, and employees who are California residents, and requires businesses to provide specific disclosures in privacy notices and honor requests of such California residents to exercise certain rights related to their personal information, such as those noted above.

One example of these comprehensive state data privacy laws is the California Consumer Privacy Act of 2018, as amended by the California Privacy Rights Act of 2020 (“CPRA”) (collectively, “CCPA”) which applies to the personal information of consumers, business representatives and employees who are California residents.

It requires businesses to provide specific disclosures in privacy notices and honor requests of such California residents to exercise certain rights related to their personal information such as those noted above.

The CCPA provides for administrative fines for noncompliance (up to $7,500 per violation) and allows private litigants affected by certain data breaches to recover significant statutory damages.

4 unchanged sentences

While some of these laws exempt data processed in the context of clinical trials, these developments may nonetheless further complicate compliance efforts, and increase legal risk and compliance costs for us and the third parties upon whom we rely.

Outside the United States, an increasing number of laws, regulations, and industry standards apply to data privacy and security.

For example, the European Union’s General Data Protection Regulation (EU GDPR) and the United Kingdom GDPR (UK GDPR) (collectively, GDPR) impose strict requirements for processing personal information, and violators of these laws face significant penalties.

For example, under GDPR, government regulators may impose temporary or definitive bans on data processing, as well as fines of up to 20 million euros under the EU GDPR (17.5 million British Pounds under the UK GDPR) or 4% of annual global revenue, in either case, whichever is greater, or we may be subject to private litigation related to processing of personal information brought by classes of data subjects or consumer protection organizations authorized at law to represent.

In addition, the Swiss Federal Act on Data Protection, or FADP, also applies to the collection and processing of personal information, including health-related information, by companies located in Switzerland, or in certain circumstances, by companies located outside of Switzerland.

Outside the U.S., an increasing number of laws, regulations and industry standards apply to data privacy and security.

For example, the European Union’s General Data Protection Regulation (the “EU GDPR”) and the United Kingdom GDPR (the “UK GDPR”) (collectively, the “GDPR”) impose strict requirements for processing personal information and violators of these laws face significant penalties.

For example, under the GDPR, government regulators may impose temporary or definitive bans on data processing as well as fines of up to 20 million euros under the EU GDPR (17.5 million British Pounds under the UK GDPR) or 4% of annual global revenue, in either case, whichever is greater, or we may be subject to private litigation related to processing of personal information brought by classes of data subjects or consumer protection organizations authorized at law to represent.

In addition, the Swiss Federal Act on Data Protection (the “FADP”) also applies to the collection and processing of personal information including health-related information, by companies located in Switzerland, or in certain circumstances, by companies located outside of Switzerland.

The FADP has been revised and adopted by the Swiss Parliament.

Companies must comply with the revised version of the FADP and its revised ordinances which may result in an increase of costs of compliance, risks of noncompliance and penalties for noncompliance.

In the ordinary course of business, we may transfer personal information from Europe and other jurisdictions to the United States or other countries.

In the ordinary course of business, we may transfer personal information from Europe and other jurisdictions to the U.S.

or other countries.

Europe and other jurisdictions have enacted laws requiring data to be localized or limiting the transfer of personal information to other countries.

In particular, the European Economic Area (“EEA”), the UK and Switzerland have significantly restricted the transfer of personal information to the United States and other countries whose privacy laws it generally believes are inadequate.

In particular, the European Economic Area (the “EEA”), the UK and Switzerland have significantly restricted the transfer of personal information to the U.S.

and other countries whose privacy laws they generally believe are inadequate.

Other jurisdictions may adopt similarly stringent interpretations of their data localization and cross-border data transfer laws.

Although there are currently various mechanisms that may be used to transfer personal information from the EEA and UK to the United States in compliance with law, such as the EEA and UK’s standard contractual clauses, the UK’s International Data Transfer Agreement / Addendum, and the EU-U.S.

Data Privacy Framework and the UK extension thereto (which allows for transfers for relevant U.S.-based organizations who self-certify compliance and participate in the Framework), these mechanisms are subject to legal challenges, and there is no assurance that we can satisfy or rely on these measures to lawfully transfer personal information to the United States.

If there is no lawful manner for us to transfer personal information from the EEA, the UK, or other jurisdictions to the United States, or if the requirements for a legally-compliant transfer are too onerous, we could face significant adverse consequences, including the interruption or degradation of our operations, the need to relocate part of or all of our business or data processing activities to other jurisdictions (such as Europe) at significant expense, increased exposure to regulatory actions, substantial fines and penalties, the inability to transfer data and work with partners, vendors and other third parties, and injunctions against our processing or transferring of personal information necessary to operate our business.

Some European regulators have prevented companies from transferring personal information out of Europe for allegedly violating the EU

GDPR’s cross-border data transfer limitations.

For example, in May 2023, the Irish Data Protection Commission determined that a major social media company’s use of the standard contractual clauses to transfer personal data from Europe to the United States was insufficient and levied a 1.2 billion Euro fine against the company and prohibited the company from transferring personal data to the United States.

Although there are currently various mechanisms that may be used to transfer personal information from the EEA and UK to the U.S.

in compliance with law such as the EEA and UK’s standard contractual clauses, the UK’s International Data Transfer Agreement / Addendum and the EU-U.S.

Data Privacy Framework and the UK extension thereto (which allows for transfers for relevant U.S.-based organizations who self-certify compliance and participate in the Framework), these mechanisms are subject to legal challenges and there is no assurance that we can satisfy or rely on these measures to lawfully transfer personal information to the U.S.

If there is no lawful manner for us to transfer personal information from the EEA, the UK or other jurisdictions to the U.S., or if the requirements for a legally-compliant transfer are too onerous, we could face significant adverse consequences including the interruption or degradation of our operations, the need to relocate part of or all of our business or data processing activities to other jurisdictions (such as Europe) at significant expense, increased exposure to regulatory actions, substantial fines and penalties, the inability to transfer data and work with partners, vendors and other third parties and injunctions against our processing or transferring of personal information necessary to operate our business.

Some European regulators have prevented companies from transferring personal information out of Europe for allegedly violating the EU GDPR’s cross-border data transfer limitations.

was insufficient and levied a 1.2 billion Euro fine against the company and prohibited the company from transferring personal data to the U.S.

Our employees and personnel may use generative artificial intelligence (“AI”) technologies to perform their work and the disclosure and use of personal information in generative AI technologies is subject to various privacy laws and other privacy obligations.

19 unchanged sentences

and imprisonment of company officials.

In particular, plaintiffs have become increasingly more active in bringing privacy-related claims against companies, including class actions and mass arbitration demands.

Plaintiffs have become increasingly more active in bringing privacy-related claims against companies including class actions and mass arbitration demands.

Some of these claims allow for the recovery of statutory damages on a per violation basis and, if viable, carry the potential for monumental statutory damages depending on the volume of data and the number of violations.

Any of these events could have a material adverse effect on our reputation, business, or financial condition, including but not limited to:

loss of customers;

Any of these events could have a material adverse effect on our reputation, business or financial condition including but not limited to the loss of customers;

interruptions or stoppages in our business operations (including, as relevant, clinical trials);

4 unchanged sentences

or revision or restructuring of our operations.

Issues relating to the use of artificial intelligence and machine learning could adversely affect our business and operating results.

Issues relating to the use of AI and machine learning could adversely affect our business and operating results.

Magellan™ is our platform technology that leverages AI-supported structural biology, proprietary algorithms and physics-based models powered by the cutting-edge CSCS Swiss National Supercomputing Centre to explore novel allosteric binding pockets on disease-implicating proteins.

Issues relating to the use of new and evolving technologies such as AI and machine learning may cause us to experience brand or reputational harm, competitive harm, legal liability, and new or enhanced governmental or regulatory scrutiny, and we may incur additional costs to resolve such issues.

Issues relating to the use of new and evolving technologies such as AI and machine learning may cause us to experience brand or reputational harm, competitive harm, legal liability and new or enhanced governmental or regulatory scrutiny.

We may incur additional costs to resolve such issues.

As with many innovations, AI presents risks and challenges that could undermine or slow its adoption and therefore harm our business.

For example, perceived or actual technical, legal, compliance, privacy, security, ethical

or other issues relating to the use of AI may cause public confidence in AI to be undermined, which could harm our business reputation.

For example, perceived or actual technical, legal, compliance, privacy, security, ethical or other issues relating to the use of AI may cause public confidence in AI to be undermined, which could harm our business reputation.

In addition, litigation or government regulation related to the use of AI may also adversely impact our and others’ abilities to develop and offer products that use AI, as well as increase the cost and complexity of doing so.

22 unchanged sentences

We also rely on third-party service providers to provide other products, services, or otherwise to operate our business.

Our ability to monitor these third parties’ information security practices is limited, and these third parties may not have adequate information

security measures in place.

Our ability to monitor these third parties’ information security practices is limited, and these third parties may not have adequate information security measures in place.

If our third-party service providers experience a security incident or other interruption, we could experience adverse consequences.

6 unchanged sentences

Certain data privacy and security obligations may require us to implement and maintain specific security measures, industry-standard or reasonable security measures to protect our information technology systems and sensitive information.

While we and our third party services providers have implemented security measures designed to protect against security incidents, there can be no assurance that these measures will be effective.

While we and our third-party service providers have implemented security measures designed to protect against security incidents, there can be no assurance that these measures will be effective.

We take steps to detect and remediate vulnerabilities in our information technology systems, but we may not be able to detect and remediate all vulnerabilities because the threats and techniques used to exploit the vulnerability change frequently and are often sophisticated in nature.

26 unchanged sentences

Future adverse developments with respect to specific financial institutions or the broader financial services industry may lead to market-wide liquidity shortages, impair our ability to access near-term working capital needs, and create additional market and economic uncertainty.

There can be no assurance that future credit and financial market instability and a deterioration in confidence in

economic conditions will not occur.

There can be no assurance that future credit and financial market instability and a deterioration in confidence in economic conditions will not occur.

Our general business strategy may be adversely affected by any such economic downturn, liquidity shortages, volatile business environment or continued unpredictable and unstable market conditions.

1 unchanged sentence

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 or abandon clinical development plans.

In addition, there is a risk that one or more of our current service providers, financial institutions, manufacturers, and other partners may be adversely affected by the foregoing risks, which could directly affect our ability to attain our operating goals on schedule and on budget.

In addition, there is a risk that one or more of our current service providers, financial institutions, manufacturers, and

other partners may be adversely affected by the foregoing risks, which could directly affect our ability to attain our operating goals on schedule and on budget.

In addition, any further deterioration in the macroeconomic economy or financial services industry, could lead to losses or defaults by our suppliers, which in turn, could have a material adverse effect on our current and/or projected business operations and results of operations and financial condition.

1 unchanged sentence

The market price for our common stock has been and likely will continue to be volatile, and your investment in our securities could decline in value.

Our stock price has been highly volatile since our IPO and is likely to continue to be volatile.

The stock market in general, and the markets for pharmaceutical, biopharmaceutical and biotechnology stocks in particular, have experienced extreme price and volume fluctuations that have been often unrelated or disproportionate to the operating performance of the issuer.

Our stock price has been highly volatile and is likely to continue to be so.

The stock market in general, and the markets for pharmaceutical, biopharmaceutical and biotechnology stocks in particular, have experienced extreme price and volume fluctuations that have often been unrelated or disproportionate to the operating performance of the issuer.

The market price for our common stock may be influenced by many factors, including:

● results from, and any delays in our preclinical studies and any other future clinical development programs, including any delays related to the health epidemics or pandemics or other factors outside of our control;

● results from, and any delays in our preclinical studies and any other clinical development programs, including any delays related to the health epidemics or pandemics or other factors outside of our control;

● actual or anticipated changes in estimates as to financial results, development timelines and other company milestones or recommendations by securities analysts;

14 unchanged sentences

● changes in accounting principles;

● general economic, industry and market conditions, heightened inflation and measures taken by central banks to combat inflation, exchange rate fluctuations, supply chain disruptions and increasing commodity, energy and fuel prices;

● general economic, industry and market conditions, exchange rate fluctuations, supply chain disruptions and fluctuating commodity, energy and fuel prices;

● the impact of political instability, natural disasters, events of terrorism and/or war, such as the war in Ukraine and the conflict between Hamas and Israel, and the corresponding tensions created from such conflict between Russia, the United States and countries in Europe as well as other countries such as China;

1 unchanged sentence

In the past, securities class action litigation has often been brought against companies that experience volatility in the market price of their securities and in particular, biotechnology and pharmaceutical companies.

Whether or not meritorious, litigation brought against us could result in substantial costs and a diversion of management’s attention and resources, which could adversely affect our business, operating results and financial condition.

Stock market volatility and declines in the price of our common stock also increase the likelihood that we may fail to meet the minimum price requirements for continued listing on the Nasdaq Global Market.

Whether meritorious, litigation brought against us could result in substantial costs and a diversion of management’s attention and resources which could adversely affect our business, operating results and financial condition.

Stock market volatility and declines in the price of our common stock also increase the likelihood that we may fail to meet the minimum bid price requirement of $1.00 for continued listing on the Nasdaq Global Market.

If the Nasdaq Global Market delists our securities from trading on its exchange for failure to meet the listing standards, we and our stockholders could face significant negative consequences, including:

4 unchanged sentences

We incur and will continue to 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 and corporate governance practices.

As a public company, and particularly after we will no longer qualify as an emerging growth company, we incur and will continue to incur significant legal, accounting and other expenses that we did not incur previously.

The Sarbanes-Oxley Act, the Dodd-Frank Wall Street Reform and Consumer Protection Act, the listing requirements of Nasdaq, and other applicable securities rules and regulations impose various requirements on U.S.

As a public company, and particularly after we will no longer qualify as an emerging growth company, we incur and will continue to incur significant legal, accounting and other expenses.

The Sarbanes-Oxley Act, the Dodd-Frank Wall Street Reform and Consumer Protection Act, the listing requirements of the Nasdaq, and other applicable securities rules and regulations impose various requirements on U.S.

reporting public companies, including the establishment and maintenance of effective disclosure and financial controls and corporate governance practices.

4 unchanged sentences

While we remain an emerging growth company, we will not be required to include an attestation report on internal control over financial reporting issued by our independent registered public accounting firm.

However, we are required, pursuant to Section 404 of the Sarbanes-Oxley Act, to furnish a report by management on, among other things, the effectiveness of our internal control over financial reporting.

However, pursuant to Section 404 of the Sarbanes-Oxley Act, we are required to furnish a report by management on, among other things, the effectiveness of our internal control over financial reporting.

The process to document and evaluate our internal control over financial reporting is both costly and challenging.

1 unchanged sentence

Despite our efforts, there is a risk that we will not be able to conclude, within the prescribed timeframe or at all, that our internal control over financial reporting is effective as required by Section 404.

If we identify one or more material weaknesses, it could result in an adverse reaction in the financial markets due to a loss of confidence in the reliability of our financial statements.

If we identify one or more material weaknesses, it could

result in an adverse reaction in the financial markets due to a loss of confidence in the reliability of our financial statements.

We are an “emerging growth company,” and the reduced reporting requirements applicable to emerging growth companies may make our common stock less attractive to investors.

1 unchanged sentence

For so long as we remain an emerging growth company, we are permitted and plan to rely on exemptions from certain disclosure requirements that are applicable to public companies that are not emerging growth companies.

These provisions include, but are not limited to:

being permitted to report only two years of audited financial statements and only two years of related selected financial data and management’s discussion and analysis of financial condition and results of operations disclosure;

These provisions include, but are not limited to, being permitted to report only two years of audited financial statements and only two years of related selected financial data and management’s discussion and analysis of financial condition and results of operations disclosure;

an exemption from compliance with the auditor attestation requirement in the assessment of our internal control over financial reporting pursuant to Section 404 of the Sarbanes-Oxley Act;

2 unchanged sentences

In addition, the JOBS Act permits emerging growth companies to take advantage of an extended transition period to comply with new or revised accounting standards applicable to public companies.

As a result, the information we provide might be different from the information that is available for other public companies.

As a result, the information we provide might differ from the information that is available for other public companies.

We cannot predict whether investors will find our common stock less attractive if we rely on these exemptions.

1 unchanged sentence

We will remain an emerging growth company until the earliest of (i) December 31, 2026, (ii) the first fiscal year after our annual gross revenue exceeds $1.235 billion, (iii) the date on which we have, during the immediately preceding three-year period, issued more than $1.00 billion in non-convertible debt securities, or (iv) the end of any fiscal year in which the market value of our common stock held by non-affiliates exceeds $700 million as of the end of the second quarter of that fiscal year.

We are currently listed on the Nasdaq Global Market.

If we are unable to maintain listing of our securities on the Nasdaq Global Market or any stock exchange, our stock price could be adversely affected and the liquidity of our stock and our ability to obtain financing could be impaired and it may be more difficult for our stockholders to sell their securities.

Although our common stock is currently listed on the Nasdaq Global Market, we may not be able to continue to meet the exchange’s minimum listing requirements or those of any other national exchange.

If we are unable to maintain listing on the Nasdaq or if a liquid market for our common stock does not develop or is sustained, our common stock may remain thinly traded.

The Listing Rules of the Nasdaq require listing issuers to comply with certain standards in order to remain listed on its exchange.

If, for any reason, we should fail to maintain compliance with these listing standards and the Nasdaq should delist our securities from trading on its exchange and we are unable to obtain listing on another national securities exchange, a reduction in some or all of the following may occur, each of which could have a material adverse effect on our stockholders:

● the liquidity of our common stock;

● the market price of our common stock;

● our ability to obtain financing for the continuation of our operations;

● the number of institutional and general investors that will consider investing in our common stock;

● the number of investors in general that will consider investing in our common stock;

● the number of market makers in our common stock;

● the availability of information concerning the trading prices and volume of our common stock;

● the number of broker-dealers willing to execute trades in shares of our common stock.

In the past, we have received notices from the Nasdaq’s Listing Qualifications Department indicating that we had not complied with certain of the Nasdaq Global Market’s continued listing standards.

While we have regained compliance for each instance, there can be no assurance that we will continue to maintain compliance with the Nasdaq listing requirements.

A delisting could substantially decrease trading in our common stock, adversely affect the market liquidity of our common stock as a result of the loss of market efficiencies associated with the Nasdaq and the loss of federal preemption of state securities laws, adversely affect our ability to obtain financing on acceptable terms, if at all, and may result in the potential loss of confidence by investors, suppliers, and employees and lead to fewer business development opportunities.

Additionally, the market price of our common stock may decline further, and stockholders may lose some or all of their investment.

In the event of a delisting, we anticipate that we would take actions to restore our compliance with the Nasdaq Global Market or another national exchange’s listing requirements, but we can provide no assurance that any such action taken by us would allow our common stock to remain listed on the Nasdaq Global Market, stabilize our market price, improve the liquidity of our common stock, prevent our common stock from dropping below the Nasdaq Global Market’s minimum bid price requirement, or prevent future non-compliance with the Nasdaq Global Market or another national exchange’s listing requirements.

Our ability to use our net operating loss carryforwards and certain other tax attributes may be limited.

As of December 31, 2024, we had net operating loss, or NOL, carryforwards of approximately $47 million.

We have incurred substantial losses during our history, do not expect to become profitable in the foreseeable future and may never achieve profitability.

Net operating losses, or NOLs, of our Swiss subsidiary can be carried forward for seven years and will begin to expire commencing from 2025 for the NOLs generated in 2017 under applicable Swiss tax law.

Net operating losses of our Swiss subsidiary can be carried forward for up to seven years and will begin to expire commencing from 2025 for the NOLs generated in 2018 under applicable Swiss tax law.

Under applicable U.S.

1 unchanged sentence

Our federal NOL carryforwards generated in tax years beginning after December 31, 2017, may be carried forward indefinitely, but the deductibility of such federal NOL carryforwards may be limited.

It is uncertain if and to what extent various states will conform to U.S.

federal income tax law with respect to the treatment of NOL carryforwards.

Similar provisions of state tax law may also apply to limit the use of any state NOLs.

In addition, under Sections 382 and 383 of the Internal Revenue Code of 1986, as amended, and corresponding provisions of state law, if a corporation undergoes an “ownership change,” generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation’s ability to use its pre-change NOL carryforwards and other pre-change tax attributes (such as research tax credits) to offset its post-change income or taxes may be limited.

We have experienced ownership changes in the past.

Utilization of the net operating loss carryforwards may be subject to an annual limitation due to ownership changes that may have occurred previously.

Such annual limitation could result in the expiration of net operating losses and credits before their utilization.

We have not yet completed a Section 382 analysis, and therefore, there can be no assurances over any previous ownership changes and their impacts on our ability to utilize loss carryforwards.

In addition, we may experience ownership changes in the future as a result of subsequent shifts in our stock ownership, some of which are outside of our control.

3 unchanged sentences

New tax laws, statutes, rules, regulations, or ordinances could be enacted at any time.

For instance, the recently enacted IRA imposes, among other rules, a 15% minimum tax on the book income of certain large corporations and a 1% excise tax on certain corporate stock repurchases.

For instance, the IRA imposes, among other rules, a 15% minimum tax on the book income of certain large corporations and a 1% excise tax on certain corporate stock repurchases.

Further, existing tax laws, statutes, rules, regulations, or ordinances could be interpreted differently, changed, repealed, or modified at any time.

Any such enactment, interpretation, change, repeal, or modification could adversely affect us, possibly with retroactive effect.

In particular, changes in corporate tax rates, the realization of our net deferred tax assets, the taxation of foreign earnings, and the deductibility of expenses under the Tax Cuts and Jobs Act, as amended by the Coronavirus Aid, Relief, and Economic Security Act or any future tax reform legislation, could have a material impact on the value of our deferred tax assets, result in significant one-time charges, and increase our future tax expenses.

future tax reform legislation, could have a material impact on the value of our deferred tax assets, result in significant one-time charges, and increase our future tax expenses.

We do not anticipate paying dividends on our common stock and, accordingly, stockholders must rely on stock appreciation for any return on their investment.

1 unchanged sentence

The declaration of dividends is subject to the discretion of our board of directors and limitations under applicable law and will depend on various factors including our operating results, financial condition, future prospects and any other factors deemed relevant by our board of directors.

You should not rely on an investment in our company if you require dividend income from your investment in our company.

You should not rely on an investment in our company if you require dividend income.

The success of your investment will likely depend entirely upon any future appreciation of the market price of our common stock which is uncertain and unpredictable.

9 unchanged sentences

The rights of the holders of our securities may be impaired by the potential issuance of preferred stock.

Our articles of incorporation give our board of directors the ability to designate and issue preferred stock in one or more series.

Our amended and restated certificate of incorporation (the “Amended Charter”) gives our board of directors the ability to designate and issue preferred stock in one or more series.

As a result, the board of directors may, without stockholder approval, issue preferred stock with voting, dividend, conversion, liquidation or other rights which could adversely affect the relative voting power and equity interest of the holders of common stock.

5 unchanged sentences

Our research coverage by industry and financial analysts is currently limited.

Even if our analyst coverage increases, if one or more of the analysts who cover us downgrade our

stock, our stock price would likely decline.

Even if our analyst coverage increases, if one or more of the analysts who cover us downgrade our stock, our stock price would likely decline.

If one or more of these analysts cease coverage of our company or fail to regularly publish reports on us, we could lose visibility in the financial markets which in turn could cause our stock price or trading volume to decline.

Anti-takeover provisions in our organizational documents and Delaware law might discourage or delay attempts to acquire us that you might consider favorable.

Our amended and restated certificate of incorporation (the “Amended Charter”) and amended and restated bylaws (the “Amended Bylaws”) contain provisions that may make the merger or acquisition of us more difficult without the approval of our board of directors.

Our Amended Charter and amended and restated bylaws (the “Amended Bylaws”) contain provisions that may make the merger or acquisition of us more difficult without the approval of our board of directors.

Among other things, these provisions:

3 unchanged sentences

Further, as a Delaware corporation, we are also subject to provisions of Delaware law which may impair a takeover attempt that our stockholders may find beneficial.

These anti-takeover provisions and other provisions under Delaware law could discourage, delay, or prevent a transaction involving a change in control of us, including actions that our stockholders may deem advantageous, or could negatively affect the market price of our common stock.

These anti-takeover provisions and other provisions under Delaware law could discourage, delay or prevent a transaction involving a change in control, including actions that our stockholders may deem advantageous or could negatively affect the market price of our common stock.

These provisions could also discourage proxy contests and make it more difficult for you and other stockholders to elect directors of your choosing and to cause us to take other corporate actions our stockholders desire.

12 unchanged sentences

These provisions may discourage us, or our stockholders through derivative litigation, from bringing a lawsuit against any of our current or former directors or officers for any breaches of their fiduciary duties even if such legal actions, if successful, might benefit us or our stockholders.

In addition, our Amended Charter and Amended Bylaws provide that we will, to the fullest extent permitted by Delaware law, indemnify our directors and officers for costs or damages incurred by them in connection with any threatened, pending, or completed action, suit, or proceeding brought against them by reason of their positions as directors and officers.

In addition, our Amended Charter and

Amended Bylaws provide that we will, to the fullest extent permitted by Delaware law, indemnify our directors and officers for costs or damages incurred by them in connection with any threatened, pending or completed action, suit or proceeding brought against them by reason of their positions as directors and officers.

We also intend to enter into indemnification agreements with each of our directors and executive officers.

1 unchanged sentence

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

What changed 10-K

Item 1A. Risk Factors