AVXL

We encourage you to carefully review the full risk factors contained in this report in their entirety for additional information regarding these risks and uncertainties. · Our history of losses and no revenue raises a risk regarding our ability to continue as a going concern in the future; · We have a very limited relevant operating history upon which an evaluation of our performance and prospects can be made; · Our research and development plans will require substantial additional future funding; · We may be unable to raise additional capital when needed, which would force us to delay, reduce or eliminate our research and development activities; · The marketing approval process is burdensome and may not be successful; · If we or any companion diagnostic collaborator of ours are unable to timely develop and obtain regulatory approval for companion diagnostic tests for our drug candidates, we may not realize the commercial potential of our drug candidates; · The regulatory approval processes of the FDA, EMA and comparable foreign regulatory authorities are lengthy, time-consuming and inherently unpredictable, which could lead to our inability to generate product revenue; · Regulatory authorities may not accept data from our trials conducted outside the United States; · Fast Track designation or breakthrough therapy designation that we have received or may seek out may not actually lead to a faster FDA review and approval process; · We may be unable to maintain any benefits associated with orphan drug designation, including market exclusivity; · We may fail to demonstrate efficacy in our non-clinical studies and clinical trials; · If a particular product candidate causes undesirable side effects, then we may be unable to receive regulatory approval of or commercialize such product candidate; · Treatment of neurodegenerative and central nervous system, or CNS, disorders, is a field that has seen very limited success in product development; · The use of any of our products in clinical trials may expose us to liability claims, causing our business to suffer; · We are highly dependent on our key personnel and if we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy; · If we do not obtain the support of qualified scientific collaborators, our revenue, growth and profitability will likely be limited, which would have a material adverse effect on our business; · We may not be able to develop, market or generate sales of our products to the extent anticipated; · Our technologies and future products may be rendered undesirable or obsolete if our competitors succeed in developing products and technologies faster or that are more effective or with a better profile than our own, or if scientific developments change our understanding of the potential scope and utility of our potential products; · Our reliance on third parties may result in delays in completing, or a failure to complete, non-clinical testing or clinical trials if they fail to perform under our agreements with them or non-compliance with regulations; · If we fail to compete with respect to partnering, licensing, mergers, acquisitions, joint venture and other collaboration opportunities, our ability to research and develop our potential drug compounds may be limited; · Our business could be affected by litigation, government investigations and enforcement actions; · Loss of access to Australian government research and development income tax incentive refunds could have a negative effect on our future cash flows and the funding of future research and development projects; · Our ability to use our net operating loss carryforwards and tax credit carryforwards may be subject to limitation; · Healthcare laws and regulations could expose us to criminal sanctions, civil and administrative penalties, contractual damages, reputational harm and diminished profits and future earnings, among other penalties; · Failure to obtain or maintain adequate coverage and reimbursement for our product candidates, if approved, could limit our ability to market those products and decrease our ability to generate product revenue; · Issuing additional shares of common stock will result in the dilution of our existing stockholders and may cause our stock price to fall; · Our stock price has been volatile at times in the past and may be volatile in the future and our common stock may become the target of a “short squeeze”; · If we are unable to obtain and maintain sufficient intellectual property protection for our product candidates, our competitors could develop and commercialize product candidates similar or identical to ours, and our ability to successfully commercialize our product candidates may be impaired; · Intellectual property infringement claims may adversely affect our development and commercialization efforts; · We may be subject to claims that our employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties; · We may become involved in lawsuits to protect or enforce our patents or other intellectual property; · Obtaining and maintaining our patent protection depends on compliance with various requirements imposed by governmental patent agencies.
Since inception through September 30, 2025, we have accumulated a deficit of approximately $382 million.
We are a clinical stage pharmaceutical research and development company and may never be able to successfully develop marketable products or generate any revenue.
We have a very limited relevant operating history upon which an evaluation of our future performance and prospects can be made.
We are a clinical stage company and have not generated any revenues to date.
We are subject to all of the business risks associated with a pre-revenue company, including, but not limited to, risks of unforeseen capital requirements, failure of potential drug compounds either in non-clinical testing or in clinical trials, failure to establish business relationships and competitive disadvantages against larger and more established companies.
We may be unable to raise additional capital when needed, which would force us to delay, reduce or eliminate our research and development activities.
To date, we have funded our operations primarily through issuances of shares at-the-market sales agreements pursuant to which we offer and sell shares of common stock registered under an effective registration statement from time to time through a sales agent and, historically, also through a Purchase Agreement with Lincoln Park Capital Fund, LLC (“Lincoln Park”) pursuant to which the Company could direct Lincoln Park to purchase shares of common stock registered under an effective registration.
The Company will need to file a prospectus supplement in order to access funds under the 2023 Purchase Agreement and the 2023 Purchase Agreement will expire on February 3, 2026.
Until we can generate significant revenues, if ever, we expect to satisfy our future cash needs through equity or convertible debt financing.
A decline in the price of our common stock could affect our ability to raise further working capital and adversely impact our operations and would severely dilute existing or future investors if we were to raise funds at lower prices.
If our stock price declines, we may be forced to sell equity securities at such lower prices resulting in significant dilution to existing investors.
We believe the following factors could cause the market price of our common stock to continue to fluctuate widely and could cause our common stock to trade lower: ● actual or anticipated variations in our quarterly operating results; ● announcements of new services, products, acquisitions or strategic relationships by us or our competitors; ● the outcome of our clinical trials, which are inherently unpredictable; ● changes in accounting treatments or principles; ● changes in earnings estimates by securities analysts and in analyst recommendations; and ● general political, economic, regulatory and market conditions.
Risks Related to the Discovery and Development of Our Current and Future Product Candidates The marketing approval process for pharmaceutical products is a lengthy, complex and highly regulated process and we cannot predict the outcome of any interactions with the regulatory authorities or when we will receive marketing approval, if at all.
The regulatory approval processes of the EMA, the FDA, and other comparable foreign authorities are lengthy, time-consuming and inherently unpredictable, and the approval process can vary significantly depending on the regulatory authority.
Relevant health authorities may, at the time of the filing of the application for a marketing authorization, or later during their review, impose requirements that can evolve over time, including requiring additional clinical trials, and such authorities may delay or refuse to grant approval.
On November 14, 2025, we announced that the CHMP of the EMA has rendered a negative trend vote following an oral explanation of our MAA.
The CHMP is expected to adopt a formal opinion on the MAA at its December meeting.
We plan to seek a re-examination of the MAA upon its formal adoption.
However, we cannot predict the outcome of any interactions with the regulatory authorities and when we will receive a marketing approval, if at all.
In recent years, health authorities have become increasingly focused on product safety and on the risk/benefit profile of pharmaceutical products, which could lead to more burdensome and costly approval processes and negatively affect our ability to obtain regulatory approval for products under development.
For example, the FDA and the EMA have been implementing strict requirements for approval, particularly in terms of the volume of data needed to demonstrate a product’s efficacy and safety.
Obtaining and maintaining regulatory approval of blarcamesine or any future product candidates in one jurisdiction does not mean that we will be successful in obtaining regulatory approval of those product candidates in other jurisdictions.
Obtaining and maintaining regulatory approval of blarcamesine and any future product candidates in one jurisdiction does not guarantee that we will be able to obtain or maintain regulatory approval in any other jurisdiction, while a failure or delay in obtaining regulatory approval in one jurisdiction may have a negative effect on the regulatory approval process in others.
Approval procedures vary among jurisdictions and can involve requirements and administrative review periods different from each other, including additional preclinical studies or clinical trials, as clinical trials conducted in one jurisdiction may not be accepted by regulatory authorities in other jurisdictions.
In many jurisdictions outside the U.S., including Canada, and certain jurisdictions in the EU, a product candidate must be approved for reimbursement before it can be approved for sale in that jurisdiction.
In some cases, the price that we intend to charge for our products is also subject to approval.
Regulatory authorities in jurisdictions outside of the U.S. have requirements for approval of product candidates with which we must comply prior to marketing in those jurisdictions and such regulatory requirements can vary widely from country to country.
Obtaining other regulatory approvals and compliance with other regulatory requirements could result in significant delays, difficulties and costs for us and could require additional preclinical studies or clinical trials, which could be costly and time-consuming and could delay or prevent the introduction of our products in certain countries.
The foreign regulatory approval process involves all of the risks associated with FDA approval.
We do not have experience in obtaining regulatory approval in international markets or within the United States.
If we fail to comply with the regulatory requirements in international or domestic markets and/or obtain and maintain applicable marketing approvals, our target market will be reduced and our ability to realize the full market potential of blarcamesine or any future product candidates will be harmed.
Even if we are able to develop our potential drug compounds, we may not be able to receive regulatory approval, or if approved, we may not be able to generate significant revenues or successfully commercialize our products, which will adversely affect our financial results and financial condition and we will have to delay or terminate some or all of our research and development plans which may force us to cease operations.
These include: ● the possibility that non-clinical testing or clinical trials may show that our potential drug compounds are ineffective and/or cause harmful side effects; ● regulators may not authorize us to commence or continue a clinical trial or may impose a clinical hold or may limit the conduct of a clinical trial through the imposition of a clinical hold; ● the number of patients required for clinical trials for our drug candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, participants may drop out of these clinical trials at a higher rate than we anticipate or the duration of these clinical trials may be longer than we anticipate; ● our third-party contractors, including investigators, may fail to meet their contractual obligations to us in a timely manner, or at all, or may fail to comply with regulatory requirements; ● our potential drug compounds may prove to be too expensive to manufacture or administer to patients; ● our potential drug compounds may fail to receive necessary regulatory approvals from the United States Food and Drug Administration or foreign regulatory authorities in a timely manner, or at all; ● even if our potential drug compounds are approved, we may not be able to produce them in commercial quantities or at reasonable costs; ● even if our potential drug compounds are approved, they may not achieve commercial acceptance; ● regulatory or governmental authorities may apply restrictions to any of our potential drug compounds, which could adversely affect their commercial success; and ● the proprietary rights of other parties may prevent us or our potential collaborative partners from marketing our potential drug compounds.
It may take several years before we can develop potentially marketable products, if at all.
Any of these uncertain events can significantly change our cash requirements as they may involve one-time events such as the receipt or payment of major milestones and other payments.
Additional funds will be required to support our operations, including commercialization of our product candidates, and if we are unable to obtain them on favorable terms, we may be required to cease or reduce certain further research and development and commercialization programs of our drug product platform, sell some or all our intellectual property, merge with another entity or scale back operations.
Companion diagnostics are subject to regulation by the FDA, EMA and comparable foreign regulatory authorities as medical devices and will likely require separate regulatory approval prior to commercialization.
If we or third parties are unable to successfully develop companion diagnostics for our drug candidates, or experience delays in doing so: ● the development of these drug candidates may be delayed because it may be difficult to identify patients for enrollment in our clinical trials in a timely manner; ● these drug candidates may not receive marketing approval if their safe and effective use depends on a companion diagnostic; and ● we may not realize the full commercial potential of these drug candidates that receive marketing approval if, among other reasons, we are unable to appropriately identify patients targeted by these drug candidates.
Our drug candidates may be perceived negatively compared to alternative treatments that do not require the use of companion diagnostics, either due to the additional cost of the companion diagnostic or the need to complete additional [testing?] prior to administering our drug candidates.

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Gone since FY2024

We encourage you to carefully review the full risk factors contained in this report in their entirety for additional information regarding these risks and uncertainties. · Our history of losses and no revenue raises a risk regarding our ability to continue as a going concern in the future; · We have a very limited relevant operating history upon which an evaluation of our performance and prospects can be made.
We may never be able to successfully develop marketable products or generate any revenue.
If we cannot generate sufficient revenues, we may suspend or cease operations; · Our research and development plans will require substantial additional future funding.
We may be unable to raise capital when needed, which would force us to delay, reduce or eliminate our research and development activities; · We will need additional funding and may be unable to raise additional capital when needed, which would force us to delay, reduce or eliminate our research and development activities; · Even if our products are approved, we may not be able to generate significant revenues from or successfully commercialize them, which will adversely affect our financial results and financial condition and we will have to delay or terminate some or all of our research and development plans which may force us to cease operations; · Our research and development plans require substantial additional future funding which could impact our operations and financial condition; · If we or any companion diagnostic collaborator of ours are unable to timely develop and obtain regulatory approval for companion diagnostic tests for our drug candidates, we may not realize the commercial potential of our drug candidates; · The regulatory approval processes of the FDA and comparable foreign regulatory authorities are lengthy, time-consuming and inherently unpredictable, which could lead to our inability to generate product revenue; · Regulatory authorities may not accept data from our trials conducted outside the United States; · Fast Track designation or breakthrough therapy designation that we have received or may seek out may not actually lead to a faster FDA review and approval process; · We may be unable to maintain any benefits associated with orphan drug designation, including market exclusivity; · If we fail to demonstrate efficacy in our non-clinical studies and clinical trials our future business prospects, financial condition and operating results will be materially adversely affected; · If a particular product candidate causes undesirable side effects, then we may be unable to receive regulatory approval of or commercialize such product candidate; · Wea re highly dependent on our key personnel and if we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy; · If we do not obtain the support of qualified scientific collaborators, our revenue, growth and profitability will likely be limited, which would have a material adverse effect on our business; · We may not be able to develop, market or generate sales of our products to the extent anticipated.
Our business may fail and investors could lose all their investment in our Company; · None of our potential drug compounds may reach the commercial market and our business may fail; · Material modifications in the methods of product candidate manufacturing may result in additional costs or delay; · Our technologies and future products may be rendered undesirable or obsolete if our competitors succeed in developing products and technologies faster or that are more effective or with a better profile than our own, or if scientific developments change our understanding of the potential scope and utility of our potential products; · We have advanced our research and development efforts on the treatment of neurodegenerative and central nervous system, or CNS, disorders, a field that has seen very limited success in product development; · Our reliance on third parties may result in delays in completing, or a failure to complete, non-clinical testing or clinical trials if they fail to perform under our agreements with them or non-compliance with regulations; · If we fail to compete with respect to partnering, licensing, mergers, acquisitions, joint venture and other collaboration opportunities, our ability to research and develop our potential drug compounds may be limited; · The use of any of our products in clinical trials may expose us to liability claims, causing our business to suffer; · If we are unable to safeguard against security breaches with respect to our information systems, our business may be adversely affected; · Continuing regulatory obligations and ongoing regulatory review may result in additional expense.
Our compounds could be subject to restrictions on marketing or withdrawal from the market, and we may be subject to penalties when and if any of them are approved; · Changes in funding for the FDA, the SEC and other government agencies could prevent these agencies from performing normal functions on which the operations of our business may rely, which could negatively affect our business; · We receive Australian government research and development income tax incentive refunds.
Loss of access to such incentives could have a negative effect on our future cash flows and the funding of future research and development projects; · Operating our business internationally carries various risks which could materially adversely affect our business; · Our ability to use our net operating loss carryforwards and tax credit carryforwards may be subject to limitation; · Healthcare laws and regulations could expose us to criminal sanctions, civil and administrative penalties, contractual damages, reputational harm and diminished profits and future earnings, among other penalties; · We expect current and future legislation affecting the pharmaceutical industry, including drug pricing reform, to impact our business generally, which could adversely affect our business operations; · Failure to obtain or maintain adequate coverage and reimbursement for our product candidates, if approved, could limit our ability to market those products and decrease our ability to generate product revenue; · Issuing additional shares of common stock will result in the dilution of our existing stockholders and may cause our stock price to fall.
A decline in our stock price could affect our ability to raise further working capital and adversely affect our operations.
Raising funds at lower prices would severely dilute existing or future investors; · Our stock price has been volatile and may be volatile in the future and our common stock may become the target of a “short squeeze”; · If we fail to maintain an effective system of internal control over financial reporting, we may not be able to accurately report our financial results or prevent fraud.
Our disclosure controls and procedures may not prevent or detect all errors or acts of fraud.
As a result, stockholders could lose confidence in our financial and other public reporting, which would harm our business and the trading price of our common stock; · Patent terms may be inadequate to protect our competitive position on our product candidates.
If we are unable to obtain and maintain sufficient intellectual property protection for our product candidates, our competitors could develop and commercialize product candidates similar or identical to ours, and our ability to successfully commercialize our product candidates may be impaired; · If we fail to comply with our obligations in intellectual property licensing agreements or experience disruptions to our business relationships with our licensors, we could lose important intellectual property rights.
If we are unable to protect the confidentiality of our trade secrets, our business would be harmed; · Intellectual property infringement claims may adversely affect our development and commercialization efforts; · We may be subject to claims that our employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties or that our employees have wrongfully used or disclosed alleged trade secrets of their former employers; · We may become involved in lawsuits to protect or enforce our patents or other intellectual property; Obtaining and maintaining our patent protection depends on compliance with various requirements imposed by governmental patent agencies.
Changes in patent law could diminish the value of our patents and patent applications in general.
Since inception through September 30, 2024, we have accumulated a deficit of approximately $336 million.
We are an early clinical stage pharmaceutical research and development company and may never be able to successfully develop marketable products or generate any revenue.
We are an early clinical stage company and have not generated any revenues to date and have no operating history.
We have no relevant operating history upon which an evaluation of our performance and prospects can be made.
We are subject to all of the business risks associated with a new enterprise, including, but not limited to, risks of unforeseen capital requirements, failure of potential drug compounds either in non-clinical testing or in clinical trials, failure to establish business relationships and competitive disadvantages against larger and more established companies.
We will need additional funding and may be unable to raise additional capital when needed, which would force us to delay, reduce or eliminate our research and development activities.
To date, we have funded our operations primarily through private placement of our equity securities, through issuances of shares under the Purchase Agreement with Lincoln Park Capital Fund, LLC (“Lincoln Park”) pursuant to which the Company may direct Lincoln Park to purchase shares of common stock registered under an effective registration statement, or, historically, through draws under our “at-the-market offering” in connection with the Amended and Restated Sales Agreement with Cantor Fitzgerald & Co. and SVB Leerink LLC (the “Sales Agents”), pursuant to which we could offer and sell shares of common stock registered under an effective registration statement from time to time through the Sales Agents.
The Company terminated the Sales Agreement in July 2024.
Until we can generate significant revenues, if ever, we expect to satisfy our future cash needs through equity or debt financing.
Risks Related to our Business Even if we are able to develop our potential drug compounds, we may not be able to receive regulatory approval, or if approved, we may not be able to generate significant revenues or successfully commercialize our products, which will adversely affect our financial results and financial condition and we will have to delay or terminate some or all of our research and development plans which may force us to cease operations.
These include: ● the possibility that non-clinical testing or clinical trials may show that our potential drug compounds are ineffective and/or cause harmful side effects; ● regulators may not authorize us to commence or continue a clinical trial or may impose a clinical hold or may limit the conduct of a clinical trial through the imposition of a partial clinical hold; ● the number of patients required for clinical trials for our drug candidates may be larger than we anticipate, enrollment in these clinical trials may be slower than we anticipate, participants may drop out of these clinical trials at a higher rate than we anticipate or the duration of these clinical trials may be longer than we anticipate; ● our third-party contractors, including investigators, may fail to meet their contractual obligations to us in a timely manner, or at all, or may fail to comply with regulatory requirements; ● our potential drug compounds may prove to be too expensive to manufacture or administer to patients; ● our potential drug compounds may fail to receive necessary regulatory approvals from the United States Food and Drug Administration or foreign regulatory authorities in a timely manner, or at all; ● even if our potential drug compounds are approved, we may not be able to produce them in commercial quantities or at reasonable costs; ● even if our potential drug compounds are approved, they may not achieve commercial acceptance; ● regulatory or governmental authorities may apply restrictions to any of our potential drug compounds, which could adversely affect their commercial success; and ● the proprietary rights of other parties may prevent us or our potential collaborative partners from marketing our potential drug compounds.
It will take several years before we can develop potentially marketable products, if at all.
Any of these uncertain events can significantly change our cash requirements as they determine such one-time events as the receipt or payment of major milestones and other payments.
Additional funds may be required to support our operations and if we are unable to obtain them on favorable terms, we may be required to cease or reduce certain further research and development programs of our drug product platform, sell some or all our intellectual property, merge with another entity or scale back operations.
Companion diagnostics are subject to regulation by the FDA and comparable foreign regulatory authorities as medical devices and will likely require separate regulatory approval prior to commercialization.
If we or third parties are unable to successfully develop companion diagnostics for our drug candidates, or experience delays in doing so: ● the development of these drug candidates may be delayed because it may be difficult to identify patients for enrollment in our clinical trials in a timely manner; ● these drug candidates may not receive marketing approval if their safe and effective use depends on a companion diagnostic; and ● we may not realize the full commercial potential of these drug candidates that receive marketing approval if, among other reasons, we are unable to appropriately identify patients or types of tumors targeted by these drug candidates.
Our drug candidates may be perceived negatively compared to alternative treatments that do not require the use of companion diagnostics, either due to the additional cost of the companion diagnostic or the need to complete additional prior to administering our drug candidates.
The regulatory approval processes of the FDA and comparable foreign regulatory authorities are lengthy, time-consuming and inherently unpredictable, which could lead to our inability to generate product revenue.
The time required to obtain approval by the FDA and comparable foreign regulatory authorities is unpredictable, typically takes many years following the commencement of clinical trials and depends upon numerous factors, including the type, complexity and novelty of the product candidates involved.
Even if we eventually complete clinical testing and receive approval of any regulatory filing for our product candidates, the FDA and comparable foreign regulatory authorities may approve our product candidates for a more limited indication or a narrower patient population than we originally requested.
Applications for our product candidates could fail to receive regulatory approval for many reasons, including but not limited to the following: · the FDA or comparable international regulatory authorities may disagree with the design, implementation or results of our clinical trials; · the FDA or comparable international regulatory authorities may determine that our product candidates are not safe and effective, only moderately effective or have undesirable or unintended side effects, toxicities or other characteristics that preclude our obtaining marketing approval or prevent or limit commercial use; · the population studied in the clinical program may not be sufficiently broad or representative to assure efficacy and potency and safety in the full population for which we seek approval; · the FDA or comparable international regulatory authorities may disagree with our interpretation of data from preclinical studies or clinical trials; · the data collected from clinical trials of our product candidates may not be sufficient to support the submission of a Biologics License Application, New Drug Application or other submission or to obtain regulatory approval in the United States or elsewhere; · we may be unable to demonstrate to the FDA or comparable international regulatory authorities that a product candidate’s risk-benefit ratio for its proposed indication is acceptable; · the FDA or comparable international regulatory authorities may fail to approve the manufacturing processes, test procedures and specifications or facilities of third-party manufacturers with which we contract for clinical and commercial supplies; and · the approval policies or regulations of the FDA or international foreign regulatory authorities may significantly change in a manner rendering our clinical data insufficient for approval.
All but one of our clinical trials to date have been conducted outside the United States, and the FDA and other foreign regulatory authorities may not accept data from such trials.
There can be no assurance that the FDA or any other foreign regulatory authority will accept data from trials conducted outside of the United States or the applicable jurisdiction.
If the FDA or any comparable foreign regulatory authority does not accept such data, it would result in the need for additional trials, which would be costly and time-consuming and delay aspects of our business plan, and which may result in our product candidates not receiving approval or clearance for commercialization in the applicable jurisdiction.
Additionally, even if Priority Review is granted for one of our compounds, the FDA does not always meet its six-month PDUFA goal date for Priority Review and the review process is often extended by FDA requests for additional information or clarification.
We are highly dependent on our key personnel, and if we are not successful in attracting and retaining highly qualified personnel, we may not be able to successfully implement our business strategy.

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Sentence-level comparison of Item 1A in the two most recent 10-Ks (FY2024 ↗, FY2025 ↗). A reworded sentence counts as one dropped and one added, so heavy edits read as low “kept”. Headings and page furniture are stripped; nothing is summarised by a model.

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