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What changed in ANAVEX LIFE SCIENCES CORP.'s 10-K — 2024 vs 2025

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Paragraph-level year-over-year comparison of ANAVEX LIFE SCIENCES CORP.'s 2024 and 2025 10-K annual filings, covering the Business, Risk Factors, Legal Proceedings, Cybersecurity, MD&A and Market Risk sections. Every new, removed and edited paragraph is highlighted side-by-side so you can see exactly what management changed in the 2025 report.

+366 added−328 removedSource: 10-K (2025-11-25) vs 10-K (2024-12-23)

Top changes in ANAVEX LIFE SCIENCES CORP.'s 2025 10-K

366 paragraphs added · 328 removed · 246 edited across 7 sections

Item 1. Business

Business — how the company describes what it does

103 edited+42 added−38 removed248 unchanged
Biggest changeWhile the IND is active, progress reports summarizing the results of ongoing clinical trials and nonclinical studies performed since the last progress report must be submitted on at least an annual basis to the FDA, and written IND safety reports must be submitted to the FDA and investigators for serious and unexpected adverse events, findings from other studies suggesting a significant risk to humans exposed to the same or similar drugs, findings from animal or in vitro testing suggesting a significant risk to humans, and any clinically important, increased incidence of a serious adverse reaction compared to that listed in the protocol or investigator brochure. 22 There are also requirements governing the submission of certain clinical trials and completed trial results to public registries.
Biggest changeIn addition, appropriate packaging must be selected and tested, and stability studies must be conducted to demonstrate the effectiveness of the packaging and that the compound does not undergo unacceptable deterioration over its shelf life. 21 While the IND is active, progress reports summarizing the results of ongoing clinical trials and nonclinical studies performed since the last progress report must be submitted on at least an annual basis to the FDA, and written IND safety reports must be submitted to the FDA and investigators for serious and unexpected adverse events, findings from other studies suggesting a significant risk to humans exposed to the same or similar drugs, findings from animal or in vitro testing suggesting a significant risk to humans, and any clinically important, increased incidence of a serious adverse reaction compared to that listed in the protocol or investigator brochure.
The Phase 2 trial enrolled approximately 132 patients for 14 weeks, randomized 1:1:1 to two different ANAVEX ® 2-73 doses, 30 mg and 50 mg, or placebo. The ANAVEX ® 2-73 Phase 2 Parkinson’s disease dementia trial design incorporated genomic precision medicine biomarkers identified in the ANAVEX ® 2-73 Phase 2a Alzheimer’s disease trial.
The trial enrolled approximately 132 patients for 14 weeks, randomized 1:1:1 to two different ANAVEX ® 2-73 doses, 30 mg and 50 mg, or placebo. The ANAVEX ® 2-73 Phase 2 Parkinson’s disease dementia trial design incorporated genomic precision medicine biomarkers identified in the ANAVEX ® 2-73 Phase 2a Alzheimer’s disease trial.
Our Pipeline Our research and development pipeline includes ANAVEX ® 2-73 currently in three different clinical trial indications, and ANAVEX ® 3-71 currently in one clinical trial and several other compounds in different stages of clinical and pre-clinical development.
Our research and development pipeline includes ANAVEX ® 2-73 currently in three different clinical trial indications, and ANAVEX ® 3-71 currently in one clinical trial and several other compounds in different stages of clinical and pre-clinical development.
ANAVEX ® 3-71 is a CNS-penetrable potential disease modifying treatment for cognitive impairments. We believe it is effective in against the major Alzheimer’s hallmarks in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies, and also has beneficial effects on inflammation and mitochondrial dysfunctions.
ANAVEX ® 3-71 is a CNS-penetrable potential disease modifying treatment for cognitive impairments. We believe it is effective against the major Alzheimer’s hallmarks in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies, and also has beneficial effects on inflammation and mitochondrial dysfunctions.
Government regulation Government authorities in the United States, at the federal, state and local levels, and other countries extensively regulate, among other things, the research, development, testing, manufacture, quality control, approval, labeling, packaging, storage, record-keeping, promotion, advertising, distribution, marketing and export and import of products such as those we are developing.
Government regulation Government authorities in the United States, at the federal, state and local levels, and in other countries extensively regulate, among other things, the research, development, testing, manufacture, quality control, approval, labeling, packaging, storage, record-keeping, promotion, advertising, distribution, marketing and export and import of products such as those we are developing.
In addition to criminal penalties, violation of this statute may result in collateral administrative sanctions, including exclusion from participation in Medicare, Medicaid and other federal health care programs.
In addition to criminal penalties, violation of this statute may result in collateral administrative sanctions, including exclusion from participation in Medicare, Medicaid and other federal health care programs.
The Federal Anti-Kickback Statute, 42 U.S.C. § 1320a-7b(b), among other things, prohibits the knowing and willful offer, payment, solicitation or receipt of any form of remuneration, whether directly or indirectly and overtly or covertly in cash or in kind, in return for, or to induce the referral of an individual for the: ● furnishing or arranging for the furnishing of items or services reimbursable in whole or in part under Medicare, Medicaid or other federal healthcare programs; or ● purchase, lease, or order of, or the arrangement or recommendation of the purchasing, leasing, or ordering of any item or service reimbursable in whole or in part under Medicare, Medicaid or other federal healthcare programs.
The federal Anti-Kickback Statute, 42 U.S.C. § 1320a-7b(b), among other things, prohibits the knowing and willful offer, payment, solicitation or receipt of any form of remuneration, whether directly or indirectly and overtly or covertly in cash or in kind, in return for, or to induce the referral of an individual for the: 28 ● furnishing or arranging for the furnishing of items or services reimbursable in whole or in part under Medicare, Medicaid or other federal healthcare programs; or ● purchase, lease, or order of, or the arrangement or recommendation of the purchasing, leasing, or ordering of any item or service reimbursable in whole or in part under Medicare, Medicaid or other federal healthcare programs.
While current treatments can be effective in managing select symptoms, approximately 34% of people do not respond to therapy, with an additional 50-60% experiencing only a partial improvement in symptoms or unacceptable side effects. ● Fragile X – Fragile X syndrome (FXS) is the most prevalent genetic form of intellectual disability and autism spectrum disorder, primarily affecting boys.
While current treatments can be effective in managing select symptoms, approximately 34% of people do not respond to therapy, with an additional 50-60% experiencing only a partial improvement in symptoms or unacceptable side effects. 16 ● Fragile X – Fragile X syndrome (FXS) is the most prevalent genetic form of intellectual disability and autism spectrum disorder, primarily affecting boys.
Researchers do not know the exact number for how many Americans could have full mutation FXS. Studies estimate that the disease affects approximately 1:4,000 males and 1:6,000 females. Worldwide, more than 1,400,000 people could be affected by FXS. 17 ● Depression – Depression is a major cause of morbidity worldwide according to the World Health Organization.
Researchers do not know the exact number for how many Americans could have full mutation FXS. Studies estimate that the disease affects approximately 1:4,000 males and 1:6,000 females. Worldwide, more than 1,400,000 people could be affected by FXS. ● Depression – Depression is a major cause of morbidity worldwide according to the World Health Organization.
A person or entity need not have actual knowledge of this law or specific intent to commit a violation of this law. 29 18 U.S.C. § 1518 establishes criminal liability for whoever willfully prevents, obstructs, misleads, delays or attempts to prevent, obstruct, mislead, or delay the communication of information or records relating to a violation of a Federal health care offense to a criminal investigator.
A person or entity need not have actual knowledge of this law or specific intent to commit a violation of this law. 18 U.S.C. § 1518 establishes criminal liability for whoever willfully prevents, obstructs, misleads, delays or attempts to prevent, obstruct, mislead, or delay the communication of information or records relating to a violation of a Federal health care offense to a criminal investigator.
A determination of liability under such laws could result in fines, penalties, and exclusion, as well as restrictions on the ability to operate in these jurisdictions. 30 Corporate liability can be present as a result of the illegal activities of employees, representatives, contractors, collaborators, agents, subsidiaries, or affiliates, even if they were not explicitly authorized.
A determination of liability under such laws could result in fines, penalties, and exclusion, as well as restrictions on the ability to operate in these jurisdictions. Corporate liability can be present as a result of the illegal activities of employees, representatives, contractors, collaborators, agents, subsidiaries, or affiliates, even if they were not explicitly authorized.
In addition, 31 the Inflation Reduction Act (“IRA”) passed on August 16, 2022. The IRA, among other things, (1) directs HHS to negotiate the price of certain highly-utilized 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.
In addition, the Inflation Reduction Act (“IRA”) passed on August 16, 2022. The IRA, among other things, (1) directs HHS to negotiate the price of certain highly-utilized 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.
Our commercial opportunities could be reduced or eliminated if our competitors develop and commercialize products that are more effective, have fewer side effects, are more convenient or are less expensive than any products that we may develop. We believe our approach to the treatment of Alzheimer’s disease and other CNS diseases differs from our competitors.
Our commercial opportunities could be reduced or eliminated if our competitors develop and commercialize products that are more effective, have fewer side effects, are more convenient or are less expensive than any products that we may develop. 17 We believe our approach to the treatment of Alzheimer’s disease and other CNS diseases differs from our competitors.
While no agreement offers absolute protection, such agreements provide some form of recourse in the event of disclosure, or anticipated disclosure. 20 Our intellectual property position, like that of many biomedical companies, is uncertain and involves complex legal and technical questions for which important legal principles are unresolved.
While no agreement offers absolute protection, such agreements provide some form of recourse in the event of disclosure, or anticipated disclosure. Our intellectual property position, like that of many biomedical companies, is uncertain and involves complex legal and technical questions for which important legal principles are unresolved.
An approval letter authorizes commercial marketing of the drug with prescribing information for specific indications. The Pediatric Research Equity Act (“PREA”), requires IND sponsors to conduct pediatric clinical trials for most drugs, for a new active ingredient, new indication, new dosage form, new dosing regimen or new route of administration.
An approval letter authorizes commercial marketing of the drug with prescribing information for specific indications. 22 The Pediatric Research Equity Act (“PREA”), requires IND sponsors to conduct pediatric clinical trials for most drugs, for a new active ingredient, new indication, new dosage form, new dosing regimen or new route of administration.
Under the multiplicity control rule, a trial is successful in meeting the co-primary endpoints if the significance of each endpoint is P ® 2-73 and placebo groups for ADAS-Cog13 was significant at a level of P The comparison of individual dose groups vs placebo also supports blarcamesine’s efficacy.
Under the multiplicity control rule, a trial is successful in meeting the co-primary endpoints if the significance of each endpoint is P ® 2-73 and placebo groups for ADAS-Cog13 was significant at a level of P 8 The comparison of individual dose groups vs placebo also supports blarcamesine’s efficacy.
Two consecutive trial extensions for the Phase 2a trial have allowed participants who completed the 52-week Part B of the trial to continue taking ANAVEX ® 2-73, providing an opportunity to gather extended safety data for a cumulative time period of five years.
Two consecutive trial extensions for the Phase 2a trial have allowed participants who completed the 52-week Part B of the trial to continue taking ANAVEX ® 2-73, providing an opportunity to gather extended safety data for a cumulative period of five years.
Companion studies in rats demonstrated the lack of any effects on normal gastrointestinal transit with ANAVEX ® 1066 and a favorable safety profile in a battery of behavioral measures. ANAVEX ® 1037 ANAVEX ® 1037 is designed for the treatment of prostate and pancreatic cancer.
Companion studies in rats demonstrated the lack of any effects on normal gastrointestinal transit with ANAVEX ® 1066 and a favorable safety profile in a battery of behavioral measures. 15 ANAVEX ® 1037 ANAVEX ® 1037 is designed for the treatment of prostate and pancreatic cancer.
There are specific FDA regulations that govern this process. 27 U.S. coverage and reimbursement Significant uncertainty exists as to the coverage and reimbursement status of any compound for which we may seek regulatory approval.
There are specific FDA regulations that govern this process. U.S. coverage and reimbursement Significant uncertainty exists as to the coverage and reimbursement status of any compound for which we may seek regulatory approval.
Research and Development Expenses Historically, a significant portion of our operating expenses related to research and development. See our Consolidated Financial Statements contained elsewhere in this Annual Report for costs and expenses related to research and development, and other financial information for fiscal years 2024 and 2023.
Research and Development Expenses Historically, a significant portion of our operating expenses related to research and development. See our Consolidated Financial Statements contained elsewhere in this Annual Report for costs and expenses related to research and development, and other financial information for fiscal years 2025, 2024 and 2023.
No other clinical trials with ANAVEX ® 2-73 related to Rett syndrome have been conducted in the United States. 9 The second, international trial of ANAVEX ® 2-73 for the treatment of Rett syndrome, called the AVATAR trial, commenced in June 2019.
No other clinical trials with ANAVEX ® 2-73 related to Rett syndrome have been conducted in the United States. The second, international trial of ANAVEX ® 2-73 for the treatment of Rett syndrome, called the AVATAR trial, commenced in June 2019.
Orphan designation does not convey any advantage in or shorten the duration of the regulatory review and approval process. 25 If a product that has orphan designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same drug for the same indication for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan exclusivity or inability to manufacture the product in sufficient quantities.
Orphan designation does not convey any advantage in or shorten the duration of the regulatory review and approval process. 24 If a product that has orphan designation subsequently receives the first FDA approval for the disease or condition for which it has such designation, the product is entitled to orphan product exclusivity, which means that the FDA may not approve any other applications to market the same drug for the same indication for seven years, except in limited circumstances, such as a showing of clinical superiority to the product with orphan exclusivity or inability to manufacture the product in sufficient quantities.
The civil monetary penalties law, 42 U.S.C. § 1320a-7a, provides, in part, that the federal government may seek civil monetary penalties against any person who presents or causes to be presented claims to a Federal health care program that the person knows or should know is for an item or services that was not provided as claimed or is false or fraudulent, or the person has made a false statement or used a false record to get a claim paid.
The civil monetary penalties law, 42 U.S.C. § 1320a-7a, provides, in part, that the federal government may seek civil monetary penalties against any person who presents or causes to be presented claims to a Federal health care program that the person knows or should know is for an item or services that were not provided as claimed or is false or fraudulent, or the person has made a false statement or used a false record to get a claim paid.
All statistical analyses were performed by outside consultancy companies. 8 Furthermore, all pre-specified clinical endpoints were analyzed using a mixed model for repeated measures (MMRM).
All statistical analyses were performed by outside consultancy companies. Furthermore, all pre-specified clinical endpoints were analyzed using a mixed model for repeated measures (MMRM).
As discussed below, the ANDA or 505(b)(2) application also will not be approved until any applicable non-patent exclusivity listed in the Orange Book for the reference-listed drug has expired. 26 Market exclusivity provisions under the FDCA can delay the submission or approval of certain marketing applications.
As discussed below, the ANDA or 505(b)(2) application also will not be approved until any applicable non-patent exclusivity listed in the Orange Book for the reference-listed drug has expired. 25 Market exclusivity provisions under the FDCA can delay the submission or approval of certain marketing applications.
The Phase 2b/3 trial enrolled 508 patients, which were treated with a convenient once-daily oral formulation of ANAVEX ® 2-73 for 48 weeks, randomized 1:1:1 to two different ANAVEX ® 2-73 doses or placebo. The trial took place at 52 sites across North America, Europe and Australia.
The trial enrolled 508 patients, which were treated with a convenient once-daily oral formulation of ANAVEX ® 2-73 for 48 weeks, randomized 1:1:1 to two different ANAVEX ® 2-73 doses or placebo. The trial took place at 52 sites across North America, Europe and Australia.
The FDA must send a non-compliance letter to any sponsor that fails to submit the required assessment, keep a deferral current or fails to submit a request for approval of a pediatric formulation. 23 If a drug receives FDA approval, the approval may be limited to specific diseases and dosages, which could restrict the commercial value of the product.
The FDA must send a non-compliance letter to any sponsor that fails to submit the required assessment, keeps a deferral current or fails to submit a request for approval of a pediatric formulation. If a drug receives FDA approval, the approval may be limited to specific diseases and dosages, which could restrict the commercial value of the product.
In the United States, approximately 62,000 new cases of pancreatic cancer will be diagnosed this year and approximately 50,000 patients will die as a result of their cancer, according to the American Cancer Society. Sales predictions by Market Data Forecast predict that the market for the global pharmaceutical treatment of pancreatic cancer will increase to $3.7 billion by 2027.
In the United States, approximately 67,000 new cases of pancreatic cancer will be diagnosed this year and approximately 52,000 patients will die as a result of their cancer, according to the American Cancer Society. Sales predictions by Market Data Forecast predict that the market for the global pharmaceutical treatment of pancreatic cancer will increase to $3.7 billion by 2027.
Scientific Advisors We are advised by scientists and physicians with experience relevant to our Company and our product candidates. Our scientific advisors include clinicians and scientists who are affiliated with a number of highly regarded medical institutions. Employees We currently have approximately forty-two full-time employees, and we retain several independent contractors on a regular or as-needed basis.
Scientific Advisors We are advised by scientists and physicians with experience relevant to our Company and our product candidates. Our scientific advisors include clinicians and scientists who are affiliated with a number of highly regarded medical institutions. Employees We currently have approximately thirty-four full-time employees, and we retain several independent contractors on a regular or as-needed basis.
The second of these four patents claims pharmaceutical compositions containing a crystalline form of ANAVEX ® 2-73, and methods of treatment for Alzheimer’s disease using the compositions. This patent is expected to expire in June 2036, absent any patent term extension for regulatory delays.
This patent is expected to expire in July 2036, absent any patent term extension for regulatory delays. The second of these five patents claims pharmaceutical compositions containing a crystalline form of ANAVEX ® 2-73, and methods of treatment for Alzheimer’s disease using the compositions.
In the future, we may apply for restoration of patent term for our currently owned or licensed patents to add patent life beyond its current expiration date, depending on the expected length of the clinical trials and other factors involved in the filing of the relevant NDA.
In the future, we may apply for restoration of patent-term for our currently owned or licensed patents to add patent life beyond their current expiration dates, depending on the expected length of the clinical trials and other factors involved in the filing of the relevant NDA.
This patent is expected to expire in June 2034, absent any patent term extension for regulatory delays. We own one issued U.S. patent entitled “A2-73 crystalline polymorph compositions of matter and methods of use thereof”. It claims crystals of A2-73 freebase or its fumarate salt, dosage forms and pharmaceutical formulations.
We own another issued U.S. patent entitled “A2-73 crystalline polymorph compositions of matter and methods of use thereof”. It claims crystals of A2-73 freebase or its fumarate salt, dosage forms and pharmaceutical formulations. This patent is expected to expire in July 2039, absent any patent term extension for regulatory delays.
In November 2024, we announced the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for ANAVEX ® 2-73 for the treatment of Alzheimer's disease and, in December 2024, the EMA accepted the submission for scientific review.
In November 2024, we announced the submission of a Marketing Authorisation Application (“MAA”) to the European Medicines Agency (EMA), under the centralized procedure, for ANAVEX ® 2-73 for the treatment of Alzheimer’s disease and, in December 2024, the EMA accepted the submission for scientific review.
Further, we own one issued U.S. patent with claims directed to methods of treating cardiac dysfunction with ANAVEX ® 2-73. This patent is expected to expire in July 2038, absent any patent term extension for regulatory delays. Additionally, we own two issued U.S. patent for the treatment of insomnia, anxiety, or agitation.
Further, we own two issued U.S. patents with claims directed to methods of treating cardiac dysfunction with ANAVEX ® 2-73. These patents are expected to expire in July 2038, absent any patent term extension for regulatory delays. Additionally, we own three issued U.S. patents for the treatment of insomnia, anxiety, or agitation.
In our clinical trials, a full genomic analysis of Alzheimer’s disease patients treated with ANAVEX ® 2-73 (blarcamesine) has helped us identify actionable genetic variants.
A full genomic analysis of Alzheimer’s disease patients treated with ANAVEX ® 2-73 (blarcamesine) has helped us identify actionable genetic variants.
A preclinical study examined the response of ANAVEX ® 3-71 in aged transgenic animal models and showed a significant reduction in the rate of cognitive deficit, amyloid beta pathology and inflammation with the administration of ANAVEX ® 3-71. In April 2016, the FDA granted Orphan Drug Designation to ANAVEX ® 3-71 for the treatment of FTD.
A preclinical study examined the response of ANAVEX ® 3-71 in aged transgenic animal models and showed a significant reduction in the rate of cognitive deficit, amyloid beta pathology and inflammation with the administration of ANAVEX ® 3-71. The FDA has granted Orphan Drug Designation to ANAVEX ® 3-71 for the treatment of Frontotemporal Demetia (“FTD”).
Additionally, we own one issued U.S. patent with claims directed to pharmaceutical dosage forms of (-) enantiomer of ANAVEX ® 2-73. This patent is expected to expire in July 2036, absent any patent term extension for regulatory delays. We also own three (3) issued U.S. patents related to ANAVEX ® 1066.
This patent is expected to expire in July 2039, absent any patent term extension for regulatory delays. Additionally, we own one issued U.S. patent with claims directed to pharmaceutical dosage forms of the (-) enantiomer of ANAVEX ® 2-73. This patent is expected to expire in July 2036, absent any patent term extension for regulatory delays.
Additional preclinical data presented also indicates that in addition to reducing oxidative stress, ANAVEX ® 3-71 has the potential to demonstrate protective effects of mitochondrial enzyme complexes during pathological conditions, which, if impaired, are believed to play a role in the pathogenesis of neurodegenerative and neurodevelopmental diseases. 15 In July 2020, we commenced the first Phase 1 clinical trial of ANAVEX ® 3-71.
Additional preclinical data presented also indicates that in addition to reducing oxidative stress, ANAVEX ® 3-71 has the potential to demonstrate protective effects of mitochondrial enzyme complexes during pathological conditions, which, if impaired, are believed to play a role in the pathogenesis of neurodegenerative and neurodevelopmental diseases.
Sponsors of certain clinical trials of FDA-regulated products are required to register and disclose specified clinical trial registration and results information, which is made publicly available at www.clinicaltrials.gov. Failure to properly report clinical trial results can result in civil monetary penalties.
There are also requirements governing the submission of certain clinical trials and completed trial results to public registries. Sponsors of certain clinical trials of FDA-regulated products are required to register and disclose specified clinical trial registration and results information, which is made publicly available at www.clinicaltrials.gov. Failure to properly report clinical trial results can result in civil monetary penalties.
The third of these four patents claims pharmaceutical compositions containing a crystalline form of ANAVEX ® 2-73, and methods of treatment for Alzheimer’s disease using the compositions. This patent is expected to expire in June 2036, absent any patent term extension for regulatory delays. The fourth of these four patents claims method of making certain crystalline forms ANAVEX ® 2-73.
This patent is expected to expire in June 2036, absent any patent term extension for regulatory delays. The third of these five patents claims pharmaceutical compositions containing a crystalline form of ANAVEX ® 2-73, and methods of treating for Alzheimer’s disease using the compositions.
For additional discussion of the risks related to competition, see Item 1A “Risk Factors.” Patents, Trademarks and Intellectual Property We hold ownership or exclusive rights to twenty-five (25) U.S. patents, twenty-five (25) U.S. patent applications, and various PCT or ex-U.S. patent applications relating to our drug candidates, methods associated therewith, and to our research programs.
For additional discussion of the risks related to competition, see Item 1A “Risk Factors.” Patents, Trademarks and Intellectual Property We hold ownership or exclusive rights to thirty (30) issued U.S. patents, seventeen (17) pending U.S. patent applications, and numerous PCT and ex-U.S. patents and patent applications relating to our drug candidates, methods associated therewith, and to our research programs.
Accordingly, we attempt to optimize the value of intellectual property in developing our business strategy including the selective development, protection, and exploitation of our intellectual property rights.
We regard patents and other intellectual property rights as corporate assets. Accordingly, we attempt to optimize the value of intellectual property in developing our business strategy including the selective development, protection, and exploitation of our intellectual property rights.
According to the Centers for Disease Control and Prevention, in 2015 epilepsy affected 3.4 million Americans. Today, epilepsy is often controlled, but not cured, with medications that are categorized as older traditional anti-epileptic drugs and second-generation anti-epileptic drugs.
According to the Centers for Disease Control and Prevention, data from 2021 and 2022 suggest epilepsy affects roughly 3.4 million Americans. Today, epilepsy is often controlled, but not cured, with medications that are categorized as older traditional anti-epileptic drugs and second-generation anti-epileptic drugs.
Legislative Activities Aimed at Controlling Drug Costs In the United States, there have been, and continue to be proposed and enacted legislation at the federal and state levels designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the cost of drugs under Medicare, and reform government program reimbursement methodologies for drugs.
HIPAA does not create a private right of action for individuals, though individuals may submit complaints related to HIPAA to OCR. 31 Legislative Activities Aimed at Controlling Drug Costs In the United States, there have been, and continue to be proposed and enacted legislation at the federal and state levels designed to, among other things, bring more transparency to drug pricing, review the relationship between pricing and manufacturer patient programs, reduce the cost of drugs under Medicare, and reform government program reimbursement methodologies for drugs.
The first of the two patents claims methods of treating insomnia or anxiety with ANAVEX ® 2-73, ANAVEX ® 19-144, and/or ANAVEX ® 1-41. This patent is expected to expire in September 2038. The second of the two patents claims a dosage form comprising any of, or any combination of ANAVEX ® 2-73, ANAVEX ® 19-144, and/or ANAVEX ® 1-41.
The first of the three patents claims methods of treating insomnia or anxiety with ANAVEX ® 2-73, ANAVEX ® 19-144, and/or ANAVEX ® 1-41. This patent is expected to expire in September 2038.
FDA-cleared ANAVEX ® 3-71-SZ-001 clinical trial: a double-blind, placebo-controlled Phase 2 trial in schizophrenia. The trial consists of two parts to explore multiple ascending doses in individuals with schizophrenia followed by a 28-day treatment period in a larger cohort.
The first of these trials is being conducted in schizophrenia. 14 Schizophrenia In March 2024, we commenced the U.S. FDA-cleared ANAVEX ® 3-71-SZ-001 clinical trial: a double-blind, placebo-controlled Phase 2 trial in schizophrenia. The trial consists of two parts to explore multiple ascending doses in individuals with schizophrenia followed by a 28-day treatment period in a larger cohort.
This Phase 2b/3 trial design incorporates inclusion of genomic precision medicine biomarkers identified in the ANAVEX ® 2-73 Phase 2a trial. 14 We believe preclinical data from our studies also supports further research into the use of ANAVEX ® 2-73 as a potential platform drug for other neurodegenerative diseases beyond Alzheimer’s disease, Parkinson’s disease or Rett syndrome, more specifically, epilepsy, infantile spasms, Fragile X syndrome, Angelman syndrome, multiple sclerosis, and tuberous sclerosis complex (TSC).
Other indications We believe preclinical data from our studies also supports further research into the use of ANAVEX ® 2-73 as a potential platform drug for other neurodegenerative diseases beyond Alzheimer’s disease, Parkinson’s disease or Rett syndrome, more specifically, epilepsy, infantile spasms, Fragile X syndrome, Angelman syndrome, multiple sclerosis, and tuberous sclerosis complex (TSC).
The criminal health care fraud statute, 18 U.S.C. § 1347, establishes criminal liability for whoever knowingly and willfully executes, or attempts to execute, a scheme or artifice to defraud any health care benefit program, or to obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any health care benefit program, in connection with the delivery of or payment for health care benefits, items, or services.
In addition to criminal penalties, violation of this statute may result in collateral administrative sanctions, including exclusion from participation in Medicare, Medicaid and other federal health care programs. 29 The criminal health care fraud statute, 18 U.S.C. § 1347, establishes criminal liability for whoever knowingly and willfully executes, or attempts to execute, a scheme or artifice to defraud any health care benefit program, or to obtain, by means of false or fraudulent pretenses, representations, or promises, any of the money or property owned by, or under the custody or control of, any health care benefit program, in connection with the delivery of or payment for health care benefits, items, or services.
A new drug must be approved by the FDA through the NDA or ANDA process before it may be legally marketed in the United States. We are subject to various government regulations in connection with the development of our pipeline. U.S.
A new drug must be approved by the local regulatory authority through an NDA, MAA or other regulatory process before it may be legally marketed in each jurisdiction. We are subject to various government regulations in connection with the development of our pipeline. U.S.
All three patents are expected to expire in November 2036, absent any patent term extension for regulatory delays. For ANAVEX ® 2-73, ANAVEX ® 19-144, ANAVEX ® 1-41, and ANAVEX ® 1066, we also have granted or pending applications in Australia, Canada, China, Europe, Japan, and Hong Kong, which are expected to expire after 2035.
For ANAVEX ® 2-73, ANAVEX ® 19-144, ANAVEX ® 1-41, and ANAVEX ® 1066, we also have granted or pending applications in Australia, Canada, China, Europe, Japan, and Hong Kong, which are expected to expire after 2035.
With regard to a fast track product, the FDA may consider for review sections of the NDA on a rolling basis before the complete application is submitted, if the sponsor provides a schedule for the submission of the sections of the NDA, the FDA agrees to accept sections of the NDA and determines that the schedule is acceptable, and the sponsor pays any required user fees upon submission of the first section of the NDA. 24 Any product submitted to the FDA for approval, including a product with a fast track designation, may also be eligible for other types of FDA programs intended to expedite development and review, such as priority review and accelerated approval.
With regard to a fast track product, the FDA may consider for review sections of the NDA on a rolling basis before the complete application is submitted, if the sponsor provides a schedule for the submission of the sections of the NDA, the FDA agrees to accept sections of the NDA and determines that the schedule is acceptable, and the sponsor pays any required user fees upon submission of the first section of the NDA.
Our Target Indications We are developing compounds with potential application to two broad categories and several specific indications, including: Central Nervous System Diseases ● Alzheimer’s disease – In 2024, an estimated 6.9 million Americans are suffering from Alzheimer’s disease according to the Alzheimer’s Association ® .
Our Target Indications We are developing compounds with potential application to two broad categories and several specific indications, including: Central Nervous System Diseases ● Alzheimer’s disease – An estimated 7.2 million Americans aged 65 and older are living with Alzheimer’s dementia in 2025, according to the Alzheimer’s Association ® .
In November 2019, the FDA granted ANAVEX ® 2-73 the Rare Pediatric Disease (RPD) designation for the treatment of Rett syndrome. The RPD designation is intended to encourage the development of treatments for rare pediatric diseases. Further, in February 2020, the FDA granted Fast Track designation for the ANAVEX ® 2-73 clinical development program for the treatment of Rett syndrome.
The FDA has granted Orphan Drug Designation and the Rare Pediatric Disease (RPD) designation for the treatment of Rett syndrome. The RPD designation is intended to encourage the development of treatments for rare pediatric diseases. Additionally, the FDA has granted Fast Track designation for the ANAVEX ® 2-73 clinical development program for the treatment of Rett syndrome.
We own one issued U.S. patent entitled “ANAVEX ® 2-73 and certain anticholinesterase inhibitors composition and method for neuroprotection,” which claims a composition of matter of ANAVEX ® 2-73 directed to a novel and synergistic neuroprotective compound combined with donepezil and other cholinesterase inhibitors.
We own one issued U.S. patent entitled “ANAVEX ® 2-73 and certain anticholinesterase inhibitors composition and method for neuroprotection,” which claims a composition of matter of ANAVEX ® 2-73, a synergistic neuroprotective compound, combined with donepezil and other cholinesterase inhibitors. This patent is expected to expire in June 2034, absent any patent term extension for regulatory delays.
The analysis identified genetic variants that impacted response to ANAVEX ® 2-73, among them variants related to the SIGMAR1, the target for ANAVEX ® 2-73. Results showed that trial participants with the common SIGMAR1 wild type gene variant, which is estimated to be about 80% of the population worldwide, demonstrated improved cognitive (MMSE) and functional (ADCS-ADL) scores.
Results showed that trial participants with the common SIGMAR1 wild type gene variant, which is estimated to be about 80% of the population worldwide, demonstrated improved cognitive (MMSE) and functional (ADCS-ADL) scores.
We also face competition from academic institutions and government agencies, both in the United States and abroad. Our competitors may have significantly greater financial resources, an established presence in the market, expertise in research and development, manufacturing, preclinical and clinical testing, may be in the process of obtaining regulatory approvals and marketing of approved products.
Our competitors may have significantly greater financial resources, an established presence in the market, expertise in research and development, manufacturing, preclinical and clinical testing, or may be in the process of obtaining regulatory approvals and marketing of approved products.
The third of these three patents claims a dosage form for seizure reduction, comprising (i) ANAVEX ® 19-144, (ii) ANAVEX ® 2-73, or (iii) a combination of ANAVEX ® 19-144 and ANAVEX ® 2-73; and optionally further comprising a low-dose anti-epilepsy drug.
The third of these three patents claims a dosage form for seizure reduction, comprising (i) ANAVEX ® 19-144, (ii) ANAVEX ® 2-73, or (iii) a combination of ANAVEX ® 19-144 and ANAVEX ® 2-73; and optionally further comprising a low-dose anti-epilepsy drug. All three patents are expected to expire in October 2035, absent any patent term extension for regulatory delays.
In addition, through tumor cell membrane reorganization and interactions with ion channels, we believe our drug candidates may play an important role in inhibiting the processes of metastasis (spreading of cancer cells from the original site to other parts of the body), angiogenesis (the formation of new blood vessels) and tumor cell proliferation. 16 ANAVEX ® 1037 is currently in the pre-clinical and clinical testing stages of development, and there is no guarantee that the activity demonstrated in pre-clinical models will be shown in human testing.
In addition, through tumor cell membrane reorganization and interactions with ion channels, we believe our drug candidates may play an important role in inhibiting the processes of metastasis (spreading of cancer cells from the original site to other parts of the body), angiogenesis (the formation of new blood vessels) and tumor cell proliferation.
ANAVEX ® 1-41 ANAVEX ® 1-41 is a sigma-1 agonist. Pre-clinical tests revealed significant neuroprotective benefits (i.e., protects nerve cells from degeneration or death) through the modulation of endoplasmic reticulum, mitochondrial and oxidative stress, which damages and impairs cell viability.
This reduction in neuroinflammatory markers suggests a potential disease-modifying effect that may become more pronounced with longer treatment durations. ANAVEX ® 1-41 ANAVEX ® 1-41 is a sigma-1 agonist. Pre-clinical tests revealed significant neuroprotective benefits (i.e., protects nerve cells from degeneration or death) through the modulation of endoplasmic reticulum, mitochondrial and oxidative stress, which damages and impairs cell viability.
A high enrollment rate in the Open Label Extension (“OLE”) of over 91% and the high level of requests for the Compassionate Use Program (93%) provide solid numerical evidence for the reported positive Real World Evidence (RWE) from patients with Rett syndrome under Compassionate Use Authorization.
All patients who participated in the trial were eligible to receive ANAVEX ® 2-73 under a voluntary open label extension protocol, which was completed in June 2024. 12 A high enrollment rate in the Open Label Extension (“OLE”) of over 91% and the high level of requests for the Compassionate Use Program (93%) provide solid numerical evidence for the reported positive Real World Evidence (RWE) from patients with Rett syndrome under Compassionate Use Authorization.
The FDA sanctions could include refusal to approve pending applications, withdrawal of an approval, clinical holds on post-marketing clinical trials, enforcement letters, import refusals, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines, refusals of government contracts, mandated corrective advertising or communications with doctors, debarment, restitution, disgorgement of profits, or civil or criminal penalties.
The FDA sanctions could include refusal to approve pending applications, withdrawal of an approval, clinical holds on post-marketing clinical trials, enforcement letters, import refusals, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines, refusals of government contracts, mandated corrective advertising or communications with doctors, debarment, restitution, disgorgement of profits, or civil or criminal penalties. 23 Expedited development and review programs The FDA has a fast track designation program that is intended to expedite or facilitate the process for reviewing new drug products that meet certain criteria.
Regulatory authorities may determine that certain marketing, pricing, or other activities violate the Federal Anti-Kickback Statute or other applicable laws. Noncompliance with the Federal Anti-Kickback Statute can result in civil, administrative and/or criminal penalties, restrictions on the ability to operate in certain jurisdictions, and exclusion from participation in Medicare, Medicaid or other federal healthcare programs.
Noncompliance with the Federal Anti-Kickback Statute can result in civil, administrative and/or criminal penalties, restrictions on the ability to operate in certain jurisdictions, and exclusion from participation in Medicare, Medicaid or other federal healthcare programs. In addition, non-compliance can result in the need to curtail and/or restructure operations.
In August 2020, patients completing these Phase 2a trial extensions were granted continued access to treatment with ANAVEX ® 2-73 through the Australian Government Department of Health – Therapeutic Goods Administration’s compassionate use Special Access Scheme. A larger Phase 2b/3 double-blind, placebo-controlled trial of ANAVEX ® 2-73 in early Alzheimer’s disease commenced in August 2018.
In August 2020, patients completing these Phase 2a trial extensions were granted continued access to treatment with ANAVEX ® 2-73 through the Australian Government Department of Health – Therapeutic Goods Administration’s compassionate use Special Access Scheme. In July 2018, we presented the results of a genomic DNA and RNA evaluation of the participants in the Phase 2a clinical trial.
The preclinical data demonstrated that treatment with ANAVEX ® 2-73 significantly increased survival and reduced seizures in those mice. ANAVEX ® 3-71 ANAVEX ® 3-71 is an orally available clinical drug candidate with a novel mechanism of action via SIGMAR1 activation and M1 muscarinic allosteric modulation, which has been shown to enhance neuroprotection and cognition in Alzheimer’s disease models.
We may in the future utilize such an approach in certain indications in which ANAVEX ® 2-73 (blarcamesine) is being studied. 13 ANAVEX ® 3-71 ANAVEX ® 3-71 is an orally available clinical drug candidate with a novel mechanism of action via SIGMAR1 activation and M1 muscarinic allosteric modulation, which has been shown to enhance neuroprotection and cognition in Alzheimer’s disease models.
ANAVEX ® 2-73 treatment resulted in a sustained improvement in CGI-I response throughout the 7-week clinical trial, when compared to placebo in the ITT cohort (all participants, p = 0.014). Consistent with previous ANAVEX ® 2-73 clinical trials, patients carrying the common form of the SIGMAR1 gene treated with ANAVEX ® 2-73 experienced stronger improvements in the prespecified efficacy endpoints.
ANAVEX ® 2-73 treatment resulted in a sustained improvement in CGI-I response throughout the 7-week clinical trial, when compared to placebo in the ITT cohort (all participants, p = 0.014).
State Fraud and Abuse Provisions Many states have also adopted some form of anti-kickback and anti-referral laws and false claims acts and civil monetary penalties and other fraud and abuse provisions that apply regardless of payer, in addition to items and services reimbursed under Medicaid and other state programs.
Exclusion from federal health care programs—whether mandatory or permissive—may mean that our customers may not be able to get reimbursed by federal and/or state health care programs for use or dispensing of our products. 30 State Fraud and Abuse Provisions Many states have also adopted some form of anti-kickback and anti-referral laws and false claims acts and civil monetary penalties and other fraud and abuse provisions that apply regardless of payer, in addition to items and services reimbursed under Medicaid and other state programs.
We anticipate conducting further clinical trials of ANAVEX ® 2-73 in Parkinson’s disease dementia after submitting the results of the trial to regulatory authorities to obtain regulatory guidance.
We anticipate conducting further clinical trials of ANAVEX ® 2-73 in Parkinson’s disease dementia after submitting the results of the trial to regulatory authorities to obtain regulatory guidance. Also with respect to Parkinson’s disease, in January 2021, we were awarded a research grant of $1.0 million from The Michael J.
In March 2019, we commenced the first Phase 2 clinical trial in a planned Rett syndrome program of ANAVEX ® 2-73 for the treatment of Rett syndrome. The clinical trials were conducted in a range of patient age demographics and geographic regions, utilizing an oral liquid once-daily formulation of ANAVEX ® 2-73.
The study was funded by the International Rett Syndrome Foundation. Our Rett syndrome program includes several clinical trials that were conducted in a range of patient age demographics and geographic regions, utilizing an oral liquid once-daily formulation of ANAVEX ® 2-73.
The trial met its primary and secondary endpoints of safety, with no serious adverse events (“SAEs”) or dose-limiting toxicities observed. ANAVEX ® 3-71 was well tolerated in all cohorts receiving ANAVEX ® 3-71 in single doses ranging from 5 mg to 200 mg daily with no SAEs and no significant lab abnormalities in any subject.
ANAVEX ® 3-71 was well tolerated in all cohorts receiving ANAVEX ® 3-71 in single doses ranging from 5 mg to 200 mg daily with no SAEs and no significant lab abnormalities in any subject. In the trial, ANAVEX ® 3-71 exhibited linear PK. Its pharmacokinetics was also dose proportional for doses up to 160 mg.
The data demonstrated dose related significant improvements in an array of behavioral and gait paradigms in a mouse model with an MECP2-null mutation that causes neurological symptoms that mimic Rett syndrome. The study was funded by the International Rett Syndrome Foundation (“Rettsyndrome.org”).
Rett Syndrome In February 2016, we presented positive preclinical data for ANAVEX ® 2-73 in Rett syndrome, a rare neurodevelopmental disease. The data demonstrated dose related significant improvements in an array of behavioral and gait paradigms in a mouse model with an MECP2-null mutation that causes neurological symptoms that mimic Rett syndrome.
A violation of the Federal Anti-Kickback Statute can serve as a false or fraudulent claim for purposes of the civil False Claims Act and the civil monetary penalties statute.
Any penalties, damages, fines, exclusions, curtailment or restructuring of operations could adversely affect the ability to operate a business, financial condition, and results of operations. A violation of the Federal Anti-Kickback Statute can serve as a false or fraudulent claim for purposes of the civil False Claims Act and the civil monetary penalties statute.
The trial also met the secondary objective of characterizing the effect of ANAVEX ® 3-71 on electrocardiogram (“ECG”) parameters. There were no clinically significant ECG parameters throughout the trial. Participant QTcF measures were normal across all dose groups with no difference between ANAVEX ® 3-71 and placebo.
Gender had no effect on the PK of the drug and food had no effect on the bioavailability of ANAVEX ® 3-71. The trial also met the secondary objective of characterizing the effect of ANAVEX ® 3-71 on electrocardiogram (“ECG”) parameters. There were no clinically significant ECG parameters throughout the trial.
We also own exclusive rights to related patents or applications that are granted or pending in Australia, Canada, China, Europe, Japan, Korea, New Zealand, Russia, and South Africa, which are expected to expire in January 2030.
We also own exclusive rights to related patents or applications that are granted or pending in Australia, Canada, China, Europe, Japan, Korea, New Zealand, Russia, and South Africa, which are expected to expire in January 2030. 19 We also own other patent applications and certain granted foreign patents directed to enantiomers, crystals, formulations, uses, and patient selection methods that may provide additional protection for one or more of our product candidates.
Because of its role in maintaining neuronal homeostasis, we believe sigma receptors show significant promise as viable targets for therapeutic molecules in an effort to treat Alzheimer’s disease and other CNS diseases and disorders, including Parkinson’s disease and Rett syndrome, by restoring healthy gene expression. 18 At this time, our competitors are primarily other biomedical development companies that are aiming to discover and develop compounds to be used in the treatment of Alzheimer’s disease and other CNS diseases, and those companies already doing so.
Because of its role in maintaining neuronal homeostasis, we believe sigma receptors show significant promise as viable targets for therapeutic molecules in an effort to treat Alzheimer’s disease and other CNS diseases and disorders by restoring healthy gene expression.
Therapies that correct defects in cellular homeostasis might have the potential to halt or delay neurodevelopmental and neurodegenerative disease progression. 7 ANAVEX ® 2-73 (blarcamesine)-specific Biomarkers As part of some of our clinical trials, we have incorporated a genomic analysis to better understand potential populations for whom our clinical programs might benefit.
The preclinical data demonstrated that treatment with ANAVEX ® 2-73 significantly increased survival and reduced seizures in those mice. ANAVEX ® 2-73 (blarcamesine)-specific Biomarkers As part of some of our clinical trials, we have incorporated a genomic analysis to better understand potential populations for whom our clinical programs might benefit.
Our focus is on developing innovative treatments for Alzheimer’s disease, Parkinson’s disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders. We currently have two core programs and two seed programs. Our core programs are at various stages of clinical and preclinical development, in neurodegenerative and neurodevelopmental diseases.
The Company’s focus is on developing innovative treatments for Alzheimer’s disease, Parkinson’s disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders.
With the SIGMAR1 activation through SIGMAR1 agonists like ANAVEX ® 2-73 (blarcamesine), our approach is to restore cellular balance (i.e. homeostasis).
With the SIGMAR1 activation through SIGMAR1 agonists like ANAVEX ® 2-73 (blarcamesine), our approach is to restore cellular balance (i.e. homeostasis). Therapies that correct defects in cellular homeostasis might have the potential to halt or delay neurodevelopmental and neurodegenerative disease progression.
Clinical Trials Overview Alzheimer’s Disease In November 2016, we completed a Phase 2a clinical trial, consisting of Part A and Part B, which lasted a total of 57 weeks, for ANAVEX ® 2-73 in mild-to-moderate Alzheimer’s patients.
ANAVEX ® 2-73 is being developed as well as an oral once-daily capsule formulation for diseases such as Alzheimer’s disease and Parkinson’s disease, and in an oral liquid once-daily formulation for rare diseases such as Rett syndrome and Fragile X. 7 Alzheimer’s Disease In November 2016, we completed a Phase 2a clinical trial, consisting of Part A and Part B, which lasted a total of 57 weeks, for ANAVEX ® 2-73 in mild-to-moderate Alzheimer’s patients.
Families whose children were previously on drug or placebo in the placebo-controlled trial commented favorably on the improvement of their child’s daily life due to ANAVEX ® 2-73 treatment in the Compassionate Use Program. 10 Parkinson’s Disease In September 2016, we presented positive preclinical data for ANAVEX ® 2-73 in an animal model of Parkinson’s disease, which demonstrated significant improvements on behavioral, histopathological, and neuroinflammatory endpoints.
Families whose children were previously on drug or placebo in the placebo-controlled trial commented favorably on the improvement of their child’s daily life due to ANAVEX ® 2-73 treatment in the Compassionate Use Program.
An Institutional Review Board (“IRB”) at each institution participating in the clinical trial must review and approve each protocol before a clinical trial may commence at the institution and must also approve the information regarding the trial as well as the informed consent form that must be provided to each trial participant or his or her legal representative, monitor the study until completed and otherwise comply with all applicable IRB regulations.
An Institutional Review Board (“IRB”) at each institution participating in the clinical trial must review and approve each protocol before a clinical trial may commence at the institution and must also approve the information regarding the trial as well as the informed consent form that must be provided to each trial participant or his or her legal representative, monitor the study until completed and otherwise comply with all applicable IRB regulations. 20 Human clinical trials are typically conducted in three sequential phases that may overlap or be combined in certain cases: Phase 1: The compound is initially introduced into healthy human subjects and tested for safety, dosage tolerance, absorption, metabolism, distribution and excretion and, if possible, to gain an early indication of its effectiveness.

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Item 1A. Risk Factors

Risk Factors — what could go wrong, per management

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Biggest changeWe may be unable to raise capital when needed, which would force us to delay, reduce or eliminate our research and development activities; 32 · 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.
Biggest changeWe encourage you to carefully review the full risk factors contained in this report in their entirety for additional information regarding these risks and uncertainties. 32 · 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; 33 · 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; 34 · Obtaining and maintaining our patent protection depends on compliance with various requirements imposed by governmental patent agencies.
Many investors who have purchased shares in those companies at an inflated rate face the risk of losing a significant portion of their original investment as the price per share has declined steadily as interest in those stocks have abated.
Many investors who have purchased shares in those companies at an inflated rate face the risk of losing a significant portion of their original investment as the price per share has declined steadily as interest in those stocks has abated.
We seek to protect our proprietary position by filing patent applications in the United States and abroad related to our product candidates or by in-licensing intellectual property.
We seek to protect our proprietary position by filing patent applications in the United States and abroad related to our product candidates and/or by in-licensing intellectual property.
Accordingly, we are subject to risks related to operating in foreign countries, including: ● different standards of care in various countries that could complicate the evaluation of our product candidates; ● different United States and foreign drug import and export rules; ● reduced protection for intellectual property rights in certain countries; ● unexpected changes in tariffs, trade barriers and regulatory requirements; ● economic weakness, including inflation, or political instability in particular foreign economies and markets; ● compliance with tax, employment, immigration, and labor laws for employees living or traveling abroad; ● compliance with the FCPA and other anti-corruption and anti-bribery laws; ● foreign taxes, including withholding of payroll taxes; ● foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country; ● workforce uncertainty in countries where labor unrest is more common than in the United States; ● production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; ● different payor reimbursement regimes, governmental payors or patient self-pay systems and price controls; ● potential liability resulting from development work conducted by foreign partners; ● business interruptions resulting from natural disasters, outbreaks of contagious diseases, such as COVID-19, or geopolitical actions, including war and terrorism, or systems failure including cybersecurity breaches; and ● compliance with evolving and expansive foreign regulatory requirements, including data privacy laws (such as the GDPR). 49 Additionally, in connection with the ongoing conflict between Russia and Ukraine, the U.S. government and European Union countries have imposed enhanced export controls on certain products and sanctions on certain industry sectors and parties in Russia.
Accordingly, we are subject to risks related to operating in foreign countries, including: ● different standards of care in various countries that could complicate the evaluation of our product candidates; ● different United States and foreign drug import and export rules; ● reduced protection for intellectual property rights in certain countries; ● unexpected changes in tariffs, trade barriers and regulatory requirements; ● economic weakness, including inflation, or political instability in particular foreign economies and markets; ● compliance with tax, employment, immigration, and labor laws for employees living or traveling abroad; ● compliance with the FCPA and other anti-corruption and anti-bribery laws; ● foreign taxes, including withholding of payroll taxes; ● foreign currency fluctuations, which could result in increased operating expenses and reduced revenue, and other obligations incident to doing business in another country; ● workforce uncertainty in countries where labor unrest is more common than in the United States; ● production shortages resulting from any events affecting raw material supply or manufacturing capabilities abroad; ● different payor reimbursement regimes, governmental payors or patient self-pay systems and price controls; ● potential liability resulting from development work conducted by foreign partners; ● business interruptions resulting from natural disasters, outbreaks of contagious diseases, such as COVID-19, or geopolitical actions, including war and terrorism, or systems failure including cybersecurity breaches; and ● compliance with evolving and expansive foreign regulatory requirements, including data privacy laws (such as the GDPR). 51 Additionally, in connection with the ongoing conflict between Russia and Ukraine, the U.S. government and European Union countries have imposed enhanced export controls on certain products and sanctions on certain industry sectors and parties in Russia.
Restrictions under applicable U.S. federal, state and foreign healthcare laws and regulations include, but are not limited to, the following: ● the U.S. federal Anti-Kickback Statute, which prohibits, among other things, persons or entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, including any kickback, bribe or rebate, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase or lease, order or recommendation of, any item, good, facility or service, for which payment may be made under federal healthcare programs such as Medicare and Medicaid; 50 ● U.S. federal civil and criminal false claims laws and civil monetary penalties laws, including the civil False Claims Act, which impose criminal and civil penalties, including those from civil whistleblower or qui tam actions, against individuals or entities for, among other things, knowingly presenting, or causing to be presented, claims for payment that are false or fraudulent or making a false statement to avoid, decrease, or conceal an obligation to pay money to the federal government; ● The Stark Law prohibits a physician from making referrals for certain “designated health services” payable by Medicare to an entity in which the physician or an immediate family member of such physician has an ownership or investment interest or with which the physician has entered into a compensation arrangement, unless a statutory exception applies.
Restrictions under applicable U.S. federal, state and foreign healthcare laws and regulations include, but are not limited to, the following: ● the U.S. federal Anti-Kickback Statute, which prohibits, among other things, persons or entities from knowingly and willfully soliciting, offering, receiving or providing remuneration, including any kickback, bribe or rebate, directly or indirectly, in cash or in kind, to induce or reward, or in return for, either the referral of an individual for, or the purchase or lease, order or recommendation of, any item, good, facility or service, for which payment may be made under federal healthcare programs such as Medicare and Medicaid; ● U.S. federal civil and criminal false claims laws and civil monetary penalties laws, including the civil False Claims Act, which impose criminal and civil penalties, including those from civil whistleblower or qui tam actions, against individuals or entities for, among other things, knowingly presenting, or causing to be presented, claims for payment that are false or fraudulent or making a false statement to avoid, decrease, or conceal an obligation to pay money to the federal government; 52 ● The Stark Law prohibits a physician from making referrals for certain “designated health services” payable by Medicare to an entity in which the physician or an immediate family member of such physician has an ownership or investment interest or with which the physician has entered into a compensation arrangement, unless a statutory exception applies.
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 have a very limited relevant operating history upon which an evaluation of our performance and prospects can be made. There is no assurance that our future operations will result in profits.
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. There is no assurance that our future operations will result in profits.
Assuming that we are successful in developing our potential drug compounds and receiving regulatory clearances to market our products, our ability to successfully penetrate the market and generate sales of those products may be limited by a number of factors, including the following: ● If our competitors receive regulatory approvals for and begin marketing similar products in the United States, the European Union, Japan and other territories before we do, greater awareness of their products as compared to ours will cause our competitive position to suffer; 43 ● Information from our competitors or the academic community indicating that current products or new products are more effective or offer compelling other benefits than our future products could impede our market penetration or decrease our future market share; and ● The pricing and reimbursement environment for our future products, as well as pricing and reimbursement decisions by our competitors and by payers, may have an effect on our revenues.
Assuming that we are successful in developing our potential drug compounds and receiving regulatory clearances to market our products, our ability to successfully penetrate the market and generate sales of those products may be limited by a number of factors, including the following: 46 ● If our competitors receive regulatory approvals for and begin marketing similar products in the United States, the European Union, Japan and other territories before we do, greater awareness of their products as compared to ours will cause our competitive position to suffer; ● Information from our competitors or the academic community indicating that current products or new products are more effective or offer compelling other benefits than our future products could impede our market penetration or decrease our future market share; and ● The pricing and reimbursement environment for our future products, as well as pricing and reimbursement decisions by our competitors and by payers, may have an effect on our revenues.
Since patent applications in the United States and most other countries are confidential for a period of time after filing or until issuance, we cannot be certain that we or our licensors were the first to either (i) file any patent application related to our product candidates or (ii) invent any of the inventions claimed in our or our licensor’s patents or patent applications.
Since patent applications in the United States and most other countries are confidential for a period of time after filing or until issuance, 62 we cannot be certain that we or our licensors were the first to either (i) file any patent application related to our product candidates or (ii) invent any of the inventions claimed in our or our licensor’s patents or patent applications.
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. 39 In order to market any product candidates outside of the United States, we must establish and comply with numerous and varying regulatory requirements of other countries regarding safety and efficacy and potency and approval standards.
Applications for our product candidates could fail to receive regulatory approval for many reasons, including but not limited to the following: · the FDA, EMA or comparable international regulatory authorities may disagree with the design, implementation or results of our clinical trials; · the FDA, EMA 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, EMA 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, EMA 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, EMA or international foreign regulatory authorities may significantly change in a manner rendering our clinical data insufficient for approval. 40 In order to market any product candidates outside of the United States, we must establish and comply with numerous and varying regulatory requirements of other countries regarding safety and efficacy and potency and approval standards.
There is also a risk that legal disputes may arise as to the rights to technology or potential drug compounds developed in collaboration with other parties, all with attendant risk, distraction, expense, and lack of predictability. Third-party claims of intellectual property infringement may prevent or delay our development and commercialization efforts.
There is also a risk that legal disputes may arise as to the rights to technology or potential drug compounds developed in collaboration with other parties, all with attendant risk, distraction, expense, and lack of predictability. 59 Third-party claims of intellectual property infringement may prevent or delay our development and commercialization efforts.
In addition, if the FDA or a comparable foreign regulatory authority approves one or more of our compounds, the manufacturing processes, labeling, packaging, distribution, adverse event reporting, storage, advertising, promotion, import, export and recordkeeping for the approved drug will be subject to additional and potentially extensive ongoing regulatory requirements.
In addition, if the FDA, EMA or a comparable foreign regulatory authority approves one or more of our compounds, the manufacturing processes, labeling, packaging, distribution, adverse event reporting, storage, advertising, promotion, import, export and recordkeeping for the approved drug will be subject to additional and potentially extensive ongoing regulatory requirements.
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.
If the FDA , EMA 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.
Currently, the Company is outside of the binding three-year period with respect to OSF applicable to some of its programs being claimed in Australia. To the extent that some or all of our research and development expenditures are deemed to be “ineligible,” then our refunds may decrease or be eliminated.
Currently, the Company is outside of the binding three-year period with respect to OSF applicable to some of its programs being claimed in Australia. 50 To the extent that some or all of our research and development expenditures are deemed to be “ineligible,” then our refunds may decrease or be eliminated.
Therefore, we cannot know with certainty whether we were the first to make the inventions claimed in our patents or pending patent applications, or that we were the first to file for patent protection of such inventions. As a result, the issuance, scope, validity, enforceability and/or commercial value of our patent rights are highly uncertain.
Therefore, 56 we cannot know with certainty whether we were the first to make the inventions claimed in our patents or pending patent applications, or that we were the first to file for patent protection of such inventions. As a result, the issuance, scope, validity, enforceability and/or commercial value of our patent rights are highly uncertain.
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; 36 ● 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.
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; 37 ● 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.
The lack of patent protection in global markets for a specific end product or indication may inhibit our ability to advance our compounds and may make us less attractive to potential partners. 56 5. Defending a patent lawsuit takes significant time and can be very expensive. 6.
The lack of patent protection in global markets for a specific end product or indication may inhibit our ability to advance our compounds and may make us less attractive to potential partners. 5. Defending a patent lawsuit takes significant time and can be very expensive. 6.
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.
Even if we eventually complete clinical testing and receive approval of any regulatory filing for our product candidates, the FDA, EMA and comparable foreign regulatory authorities may approve our product candidates for a more limited indication or a narrower patient population than we originally requested.
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.
The time required to obtain approval by the FDA, EMA 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.
Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18 months after filing or, in some cases, not at all.
Publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18 months after filing or, in some cases, not at all until issuance.
Our research and development plans will require substantial additional future funding which could impact our operations and financial condition. It will take several years before we can develop potentially marketable products, if at all.
Our research and development plans will require substantial additional future funding which could impact our operations and financial condition. It may take several years before we can develop potentially marketable products, if at all.
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.
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.
In addition, the uncertainties associated with litigation could have a material adverse effect on our ability to raise the funds necessary to continue our clinical trials, continue our research programs, license necessary technology from third parties, or enter into development partnerships that would help us bring ANAVEX ® 2-73 or our other product candidates to market. 60 We may be subject to claims challenging the inventorship of our patents and other intellectual property.
In addition, the uncertainties associated with litigation could have a material adverse effect on our ability to raise the funds necessary to continue our clinical trials, continue our research programs, license necessary technology from third parties, or enter into development partnerships that would help us bring ANAVEX ® 2-73 or our other product candidates to market. 61 We may be subject to claims challenging the inventorship of our patents and other intellectual property.
For example, any of these parties may breach the agreements and disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for such breaches.
For example, 60 any of these parties may breach the agreements and disclose our proprietary information, including our trade secrets, and we may not be able to obtain adequate remedies for such breaches.
If we engage these organizations to help us with our non-clinical and clinical programs, many important aspects of this process have been and will be out of our direct control.
If we engage these organizations to help us with our non-clinical and clinical programs, 47 many important aspects of this process have been and will be out of our direct control.
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.
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.
Orphan drug designation neither shortens the development time or regulatory review time of a drug nor gives the product candidate any advantage in the regulatory review or approval process or entitles the product candidate to priority review. 41 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.
Orphan drug designation neither shortens the development time or regulatory review time of a drug nor gives the product candidate any advantage in the regulatory review or approval process or entitles the product candidate to priority review. 42 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.
Fast Track designation alone does not guarantee qualification for the FDA’s priority review procedures. 40 Under FDA policies, a compound is eligible for priority review, or review within a six-month time frame from the time a complete NDA is accepted for filing, if the compound provides a significant improvement compared to marketed drugs in the treatment, diagnosis or prevention of a disease.
Fast Track designation alone does not guarantee qualification for the FDA’s priority review procedures. 41 Under FDA policies, a compound is eligible for priority review, or review within a six-month time frame from the time a complete NDA is accepted for filing, if the compound provides a significant improvement compared to marketed drugs in the treatment, diagnosis or prevention of a disease.
The failure to demonstrate efficacy in our clinical trials would have a material adverse effect on our future business prospects, financial condition and operating results. 42 If a particular product candidate causes undesirable side effects, then we may be unable to receive regulatory approval of or commercialize such product candidate.
The failure to demonstrate efficacy in our clinical trials would have a material adverse effect on our future business prospects, financial condition and operating results. 43 If a particular product candidate causes undesirable side effects, then we may be unable to receive regulatory approval of or commercialize such product candidate.
The market price for our common stock may be influenced by many factors, including the following: 53 ● announcements of new data, clinical trial results or those of companies that are perceived to be similar to us; ● announcements related to any delays in any preclinical or clinical trials related to our products; ● announcements related to our products’ ability to demonstrate efficacy or an acceptable safety profile of our product candidates or similar announcements by companies that are perceived to be similar to us; ● our ability to meet or exceed expectations of analysts or investors; ● news that 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; ● actions taken by regulatory agencies with respect to our product candidates or the progress of our clinical trials, including with respect to any fast track or orphan drug designations; ● announcements of significant acquisitions, strategic partnerships, joint ventures or capital commitments by us, our strategic collaboration partners or our competitors; ● grants awarded to us or companies that are perceived to be similar to us from outside entities; ● variations in our financial results or those of companies that are perceived to be similar to us; ● trading volume of our common stock; ● developments concerning our collaborations or partners; ● the impact of the COVID-19 outbreak and its effect on us; ● the perception of the biotechnology or pharmaceutical industries by the public, legislatures, regulators and the investment community; ● developments or disputes concerning intellectual property rights; ● significant lawsuits, including patent or stockholder litigation; ● our ability or inability to raise additional capital and the terms on which we raise it; ● sales of our common stock by us or our stockholders; ● declines in the market prices of stocks generally or of companies that are perceived to be similar to us; and ● general economic, industry and market conditions.
The market price for our common stock may be influenced by many factors, including the following: ● announcements of new data, clinical trial results or those of companies that are perceived to be similar to us; ● announcements related to any delays in any preclinical or clinical trials related to our products; ● announcements related to our products’ ability to demonstrate efficacy or an acceptable safety profile of our product candidates or similar announcements by companies that are perceived to be similar to us; ● our ability to meet or exceed expectations of analysts or investors; ● news that 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; ● actions taken by regulatory agencies with respect to our product candidates or the progress of our clinical trials, including with respect to any fast track or orphan drug designations; ● announcements of significant acquisitions, strategic partnerships, joint ventures or capital commitments by us, our strategic collaboration partners or our competitors; ● grants awarded to us or companies that are perceived to be similar to us from outside entities; ● variations in our financial results or those of companies that are perceived to be similar to us; ● trading volume of our common stock; ● developments concerning our collaborations or partners; ● the perception of the biotechnology or pharmaceutical industries by the public, legislatures, regulators and the investment community; 55 ● developments or disputes concerning intellectual property rights; ● significant lawsuits, including patent or stockholder litigation; ● our ability or inability to raise additional capital and the terms on which we raise it; ● sales of our common stock by us or our stockholders; ● declines in the market prices of stocks generally or of companies that are perceived to be similar to us; and ● general economic, industry and market conditions.
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.
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.
During the year ended September 30, 2024 and 2023, we determined that there were no changes in ownership pursuant to Section 382. We are subject to healthcare laws and regulations which may require substantial compliance efforts and could expose us to criminal sanctions, civil and administrative penalties, contractual damages, reputational harm and diminished profits and future earnings, among other penalties.
During the year ended September 30, 2025, the Company determined that there were no changes in ownership pursuant to Section 382. We are subject to healthcare laws and regulations which may require substantial compliance efforts and could expose us to criminal sanctions, civil and administrative penalties, contractual damages, reputational harm and diminished profits and future earnings, among other penalties.
If we receive a patent of narrow scope, then it may be possible for competitors to design products that do not infringe our patent(s). 4. We are seeking patent protection for a number of indications, combination products and drug regimens.
If we receive a patent of narrow scope, then it may be possible for competitors to design products that do not infringe our patent(s). 4. We have patents covering, and are seeking patent protection for a number of indications, combination products and drug regimens.
Moreover, we cannot be certain that our research and development efforts will be successful or, if successful, that our potential drug compounds will ever be approved for sale to pharmaceutical companies or generate commercial revenues. We have no relevant operating history upon which an evaluation of our performance and prospects can be made.
Moreover, we cannot be certain that our research and development efforts will be successful or, if successful, that our potential drug compounds will ever be approved for sale to pharmaceutical companies or generate commercial revenues. We have a very limited relevant operating history upon which an evaluation of our performance and prospects can be made.
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 will need to establish relationships with leading scientists and research institutions.
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. While we have established relationships with leading scientists and research institutions, we will need to continue to establish these relationships.
Risks Related to our Company We have had a history of losses and no revenue, which raises a risk regarding our ability to continue as a going concern in the future. Since inception through September 30, 2024, we have accumulated a deficit of approximately $336 million.
Risks Related to our Company We have had a history of losses and no revenue, which raises a risk regarding our ability to continue as a going concern in the future. Since inception through September 30, 2025, we have accumulated a deficit of approximately $382 million.
While the FDA recently clarified that mere knowledge that a physician is prescribing an approved drug for off-label use is not sufficient to constitute unlawful off-label promotion, if we are found to have actively promoted such off-label uses, we may become subject to significant liability under the FDCA.
While the FDA recently clarified that mere knowledge that a physician is prescribing an approved drug for off-label use is not sufficient to constitute unlawful off-label promotion, if we are found to have actively promoted such off-label uses, we may become subject to significant liability under the Federal Food, Drug, and Cosmetic Act (FDCA).
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 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.
If we fail to become profitable, we may suspend or cease operations. 35 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.
If we fail to become profitable, we may suspend or cease operations. We may be unable to raise additional capital when needed, which would force us to delay, reduce or eliminate our research and development activities.
Our future operating and capital needs will depend on many factors, including: ● the pace of scientific progress in our research and development programs and the magnitude of these programs; ● the scope and results of pre-clinical testing and human clinical trials; ● the time and costs involved in obtaining regulatory approvals; ● the time and costs involved in preparing, filing, prosecuting, securing, maintaining and enforcing patents; ● competing technological and market developments; ● our ability to establish additional collaborations; ● changes in our existing collaborations; ● the cost of manufacturing scale-up; and ● the effectiveness of our commercialization activities.
Our future operating and capital needs will depend on many factors, including: ● the pace of scientific progress in our research and development programs and the magnitude of these programs; ● the scope and results of pre-clinical testing and human clinical trials; ● the time and costs involved in obtaining regulatory approvals; ● the time and costs involved in preparing, filing, prosecuting, securing, maintaining and enforcing patents; ● competing technological and market developments; ● our ability to establish additional collaborations; ● changes in our existing collaborations; ● the cost of manufacturing scale-up; and ● the effectiveness of our commercialization activities. 38 We base our outlook regarding the need for funds on many uncertain variables.
In addition, the IRA, among other things, (1) directs HHS to negotiate the price of certain 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.
In addition, the Inflation Reduction Act of 2022 (“IRA”), 53 among other things, (1) directs HHS to negotiate the price of certain 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.
Changes in patent law could impair our ability to protect our product candidates; and · We may fail to protect our intellectual property rights or the confidentiality of our trade secrets. Changes in patent law could diminish the value of our patents and patent applications in general.
Changes in patent law could impair our ability to protect our product candidates; and · We may fail to protect our intellectual property rights or the confidentiality of our trade secrets.
There can be no guarantee that our stock price will remain at current prices. 54 Our common stock may become the target of a “short squeeze.” Securities of certain companies have experienced significant and extreme volatility in stock price due to short sellers of shares of common stock, known as a “short squeeze.” These short squeezes have caused extreme volatility in those companies and in the market and have led to the price per share of those companies to trade at a significantly inflated rate that is disconnected from the underlying value of the company.
Our common stock may become the target of a “short squeeze.” Securities of certain companies have experienced significant and extreme volatility in stock price due to short sellers of shares of common stock, known as a “short squeeze.” These short squeezes have caused extreme volatility in those companies and in the market and have led to the price per share of those companies to trade at a significantly inflated rate that is disconnected from the underlying value of the company.
Until we can generate significant revenues, if ever, we expect to satisfy our future cash needs through equity or debt financing. We cannot be certain that additional funding will be available on acceptable terms, or at all.
We may not be able to generate significant revenues for several years, if at all. Until we can generate significant revenues, if ever, we expect to satisfy our future cash needs through equity or convertible debt financing. We cannot be certain that additional funding will be available on acceptable terms, or at all.
To receive an Advance Overseas Finding, the expenses must have been for eligible research and development activities, as determined by AusIndustry, and the expenditures must have a scientific link to the Australian activities, be unable to be conducted in Australia and the total actual and reasonably anticipated overseas costs must be expected to be less than the total actual and reasonably anticipated expenditures for activities conducted within Australia, as determined by AusIndustry at the time of application for an Advance Overseas Finding (“OSF”). 48 This OSF binds both AusIndustry and the Commissioner of Taxation for three income years.
To receive an Advance Overseas Finding, the expenses must have been for eligible research and development activities, as determined by AusIndustry, and the expenditures must have a scientific link to the Australian activities, be unable to be conducted in Australia and the total actual and reasonably anticipated overseas costs must be expected to be less than the total actual and reasonably anticipated expenditures for activities conducted within Australia, as determined by AusIndustry at the time of application for an Advance Overseas Finding (“OSF”).
However, for compliance purposes, specific issue guidance jointly issued by AusIndustry and the ATO in 2014 provides that an OSF can apply for the duration of the overseas activity provided the activities are not new or materially different than the activities described in the OSF.
This OSF binds both AusIndustry and the Commissioner of Taxation for three income years. However, for compliance purposes, specific issue guidance jointly issued by AusIndustry and the ATO in 2014 provides that an OSF can apply for the duration of the overseas activity provided the activities are not new or materially different than the activities described in the OSF.
We are highly dependent on our management, scientific and medical personnel. The loss of the services of any of our executive officers, other key employees and other scientific and medical advisors, and an inability to find suitable replacements could result in delays in product development and harm our business.
The loss of the services of any of our executive officers, other key employees and other scientific and medical advisors, and an inability to find suitable replacements could result in delays in product development and harm our business.
Therefore, the America Invents Act and its implementation could increase the uncertainties and costs surrounding the prosecution of our owned or in-licensed patent applications and the enforcement or defense of our owned or in-licensed issued patents, all of which could have a material adverse effect on our business, financial condition, results of operations, and prospects. 61 In addition, the patent positions of companies in the development and commercialization of pharmaceuticals are particularly uncertain.
Therefore, the America Invents Act and its implementation could increase the uncertainties and costs surrounding the prosecution of our owned or in-licensed patent applications and the enforcement or defense of our owned or in-licensed issued patents, all of which could have a material adverse effect on our business, financial condition, results of operations, and prospects.
Designation as a Breakthrough Therapy is largely within the discretion of the FDA. Accordingly, even if we believe that a compound meets the criteria for designation as a Breakthrough Therapy, the FDA may disagree and instead determine not to make such designation.
We may seek Breakthrough Therapy designation for one or more of our current or future compounds. Designation as a Breakthrough Therapy is largely within the discretion of the FDA. Accordingly, even if we believe that a compound meets the criteria for designation as a Breakthrough Therapy, the FDA may disagree and instead determine not to make such designation.
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 at a price below the price at which you purchase your shares of common stock: ● actual or anticipated variations in our quarterly operating results; ● announcements of new services, products, acquisitions or strategic relationships by us or our competitors; ● 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.
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.
Enforcing a claim that someone illegally obtained and is using our trade secrets, like patent litigation, is expensive and time consuming, and the outcome is unpredictable. In addition, courts outside the United States are sometimes less willing to protect trade secrets.
Enforcing a claim that someone illegally obtained and is using our trade secrets, like patent litigation, is expensive and time consuming, and the outcome is unpredictable. In addition, courts outside the United States are sometimes less willing to protect trade secrets. Our competitors may independently develop equivalent knowledge, methods and know-how.
Later discovery of previously unknown problems with our products, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things: ● restrictions on the marketing or manufacturing of our products, withdrawal of the product from the market or voluntary or mandatory product recalls; ● fines, restitutions, disgorgement of profits or revenues, warning letters, untitled letters or holds on clinical trials; ● restrictions on product distribution or use, or requirements to conduct post-marketing studies or clinical trials; ● product seizure or detention, or refusal to permit the import or export of our products; ● injunctions or the imposition of civil or criminal penalties; and ● refusal by the FDA to approve pending applications or supplements to approved applications filed by us or suspension or revocation of approvals.
Later discovery of previously unknown problems with our products, including adverse events of unanticipated severity or frequency, or with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in, among other things: ● restrictions on the marketing or manufacturing of our products, withdrawal of the product from the market or voluntary or mandatory product recalls; ● fines, restitutions, disgorgement of profits or revenues, warning letters, untitled letters or holds on clinical trials; ● restrictions on product distribution or use, or requirements to conduct post-marketing studies or clinical trials; ● product seizure or detention, or refusal to permit the import or export of our products; ● injunctions or the imposition of civil or criminal penalties; and ● refusal by the FDA to approve pending applications or supplements to approved applications filed by us or suspension or revocation of approvals. 44 The occurrence of any event or penalty described above may limit our ability to commercialize our compounds and generate revenue and could require us to expend significant time and resources in response or generate negative publicity.
If we cannot generate sufficient revenues, we may suspend or cease operations. We are an early clinical stage company and have not generated any revenues to date and have no operating history.
If we cannot generate sufficient revenues, we may suspend or cease operations. We are a clinical stage company and have not generated any revenues to date.
We have advanced our research and development efforts on addressing neurodegenerative, neurodevelopmental and CNS disorders. Collectively, efforts by pharmaceutical companies in the field of neurodegenerative, neurodevelopmental and CNS disorders have seen very limited successes in product development.
Collectively, efforts by pharmaceutical companies in the field of neurodegenerative, neurodevelopmental and CNS disorders have seen very limited successes in product development.
Our stock price has been volatile at certain times historically, and may be volatile in the future. We may incur rapid and substantial increases or decreases in our stock price in the foreseeable future that do not coincide in timing with the disclosure of news or developments by us.
We may incur rapid and substantial increases or decreases in our stock price in the foreseeable future that do not coincide in timing with the disclosure of news or developments by us.
Our ability to use our net operating loss (“NOL”) carryforwards and certain tax credit carryforwards may be subject to limitation. As of September 30, 2024, we had approximately $128.5 million of U.S. federal and $16.9 million of state and local NOL carryforwards. We had approximately $16.6 million of NOL carryforwards in Australia as of the same period.
Our ability to use our net operating loss (“NOL”) carryforwards and certain tax credit carryforwards may be subject to limitation. As of September 30, 2025, we had approximately $148.8 million of U.S. federal and $19.1 million of state and local NOL carryforwards. We had approximately $17.1 million of NOL carryforwards in Australia as of the same period.
The development of neurodegenerative and CNS therapies presents unique challenges, including an imperfect understanding of the biology, the presence of the blood brain barrier that can restrict the flow of drugs to the brain, a frequent lack of translatability of preclinical study results in subsequent clinical trials and dose selection, and the product candidate having an effect that may be too small to be detected using the outcome measures selected in clinical trials or if the outcomes measured do not reach statistical significance.
The development of neurodegenerative and CNS therapies presents unique challenges, including an imperfect understanding of the biology, the presence of the blood brain barrier that can restrict the flow of drugs to the brain, a frequent lack of translatability of preclinical study results in subsequent clinical trials and dose selection, and the product candidate having an effect that may be too small to be detected using the outcome measures selected in clinical trials or if the outcomes measured do not reach statistical significance. 45 The use of any of our products in clinical trials may expose us to liability claims, which may cost us significant amounts of money to defend against or pay out, causing our business to suffer.
The issuance of any such shares of common stock will result in a reduction of the book value or market price of the outstanding shares of our common stock. If we do issue any such additional shares of common stock, such issuance also will cause a reduction in the proportionate ownership and voting power of all other stockholders.
If we do issue any such additional shares of common stock, such issuance also will cause a reduction in the proportionate ownership and voting power of all other stockholders. Further, any such issuance may result in a change of control of our corporation.
Our competitors may independently develop equivalent knowledge, methods and know-how. 59 We seek to protect our confidential proprietary information, in part, by confidentiality agreements and invention assignment agreements with our employees, consultants, scientific advisors, contractors and collaborators. These agreements are designed to protect our proprietary information.
We seek to protect our confidential proprietary information, in part, by confidentiality agreements and invention assignment agreements with our employees, consultants, scientific advisors, contractors and collaborators. These agreements are designed to protect our proprietary information.
For example, over the last several years, the U.S. government has shut down several times and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical FDA, SEC and other government employees and stop critical activities.
For example, over the last several years, the U.S. government has shut down several times, including the longest government shutdown that began on October 1, 2025 and ended November 13, 2025, and certain regulatory agencies, such as the FDA and the SEC, have had to furlough critical FDA, SEC and other government employees and stop critical activities.
Further, any such issuance may result in a change of control of our corporation. In the event we do issue or sell additional shares of common or preferred stock, it may result in stockholder dilution and may cause our share price to fall. Our stock price has been volatile and may be volatile in the future.
In the event we do issue or sell additional shares of common or preferred stock, it may result in stockholder dilution and may cause our share price to fall. Our stock price has been volatile and may be volatile in the future. Our stock price has been volatile at certain times historically, and may be volatile in the future.
If a court or, in some circumstances, a board of a national patent authority, agrees, we would lose some or all of our patent protection. As a company, we have no meaningful experience with competitors interfering with our patents or patent applications. 2.
Competitors may also claim that we are infringing their patents and restrict our freedom to operate. If a court or, in some circumstances, a board of a national patent authority, agrees, we would lose some or all of our patent protection. As a company, we have no meaningful experience with competitors interfering with our patents or patent applications. 2.
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. The acceptance of study data from clinical trials conducted outside the United States by the FDA may be subject to certain conditions or may not be accepted at all.
All but one of our clinical trials to date have been conducted outside the United States, and the FDA, EMA and other foreign regulatory authorities may not accept data from such trials.
If these legislative or executive actions impose constraints on the FDA’s ability to engage in oversight and implementation activities in the normal course, our business may be negatively impacted.
If these legislative or executive actions impose constraints on the FDA’s ability to engage in oversight and implementation activities in the normal course, our business may be negatively impacted. Material modifications in the methods of product candidate manufacturing may result in additional costs or delay.
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 may seek Breakthrough Therapy designation for one or more of our current or future compounds.
Additionally, even if Priority Review is granted for one of our compounds, the FDA does not always meet its six-month Prescription Drug User Fee Act (PDUFA) goal date for Priority Review and the review process is often extended by FDA requests for additional information or clarification.
If we are not able to establish scientific relationships to assist in our research and development, we may not be able to successfully develop our potential drug compounds. If this happens, our business will be adversely affected. We may not be able to develop, market or generate sales of our products to the extent anticipated.
If we are not able to maintain our existing scientific relationships and establish new scientific relationships to assist in our research and development, we may not be able to successfully develop our potential drug compounds. If this happens, our business will be adversely affected.
If none of our potential drug compounds reach the commercial market, our business will likely fail and investors will lose all of their investment in our Company. If this happens, our business will be adversely affected. Material modifications in the methods of product candidate manufacturing may result in additional costs or delay.
If none of our potential drug compounds reach the commercial market, our business will likely fail and investors will lose all of their investment in our Company. If this happens, our business will be adversely affected.
If we are unable to obtain patent term extension or restoration, or the term of any such extension is less than we request, the period during which we will have the right to exclusively market our product may be shortened and our competitors may obtain approval of competing products following our patent expiration sooner, and our revenue could be reduced, possibly materially. 57 Also, there are detailed rules and requirements regarding the patents that may be submitted to the FDA for listing in the Approved Drug Products with Therapeutic Equivalence Evaluations, or the Orange Book.
If we are unable to obtain patent term extension or restoration, or the term of any such extension is less than we request, the period during which we will have the right to exclusively market our product may be shortened and our competitors may obtain approval of competing products following our patent expiration sooner, and our revenue could be reduced, possibly materially.
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.
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. 39 If any of these events were to occur, our business and growth prospects would be harmed materially.
If we receive marketing approval for a compound, physicians may nevertheless lawfully prescribe it to their patients in a manner that is inconsistent with the approved label.
In particular, a drug may not be promoted for uses that are not approved by the FDA as reflected in the drug’s approved labeling. If we receive marketing approval for a compound, physicians may nevertheless lawfully prescribe it to their patients in a manner that is inconsistent with the approved label.
If any of our products are subject to such negotiation, we may lose a significant amount of the revenues expected during the full life cycle of these products.
If any of our products are subject to such negotiation, we may lose a significant amount of the revenues expected during the full life cycle of these products. In addition, the current administration is pursuing other measures to reduce the cost of drugs in the United States.
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. Significant uncertainty exists as to the coverage and reimbursement status of any compound for which we may seek regulatory approval.
The coverage and reimbursement status of newly approved products is uncertain. 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.
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. Our ability to compete in the highly competitive biotechnology and pharmaceutical industries depends upon our ability to attract and retain highly qualified managerial, scientific and medical personnel.
Our ability to compete in the highly competitive biotechnology and pharmaceutical industries depends upon our ability to attract and retain highly qualified managerial, scientific and medical personnel. We are highly dependent on our management, scientific and medical personnel.
If we are unable to timely adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance post-marketing, we may lose any marketing approval that we may have obtained, and we may not achieve or sustain profitability. 47 Finally, we cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative or executive action, either in the United States or abroad.
If we are unable to timely adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance post-marketing, we may lose any marketing approval that we may have obtained, and we may not achieve or sustain profitability.
U.S. patents related to ANAVEX ® 2-73 are directed to ANAVEX®2-73 in its various optical or crystal forms, its therapeutic indications, and dosage forms comprising certain doses of ANAVEX ® 2-73 combined with another therapeutic agent.
U.S. patents related to ANAVEX ® 2-73 are directed to ANAVEX®2-73 in its various optical or crystal forms, its therapeutic indications, and dosage forms comprising certain doses of ANAVEX ® 2-73 combined with another therapeutic agent. We may not be able to obtain broader scope patent protection for ANAVEX ® 2-73 as a single drug or in other jurisdictions.
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.
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.
If any of these events were to occur, our business and growth prospects would be harmed materially. 38 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 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.
If one of our product candidates is approved and a patent covering that product candidate is not listed in the Orange Book, a manufacturer of generic drugs would not have to provide advance notice to us of any abbreviated new drug application filed with the FDA to obtain permission to sell a generic version of such product candidate.
If one of our product candidates is approved and a patent covering that product candidate is not listed in the Orange Book, a manufacturer of generic drugs would not have to provide advance notice to us of any abbreviated new drug application filed with the FDA to obtain permission to sell a generic version of such product candidate. 58 If we fail to comply with our obligations in the agreements under which we license intellectual property rights from third parties or otherwise experience disruptions to our business relationships with our licensors, we could lose intellectual property rights that are important to our business.
Our inability to promptly obtain coverage and adequate reimbursement from third-party payors for the product candidates, and for us or our collaborators to obtain coverage and adequate reimbursement for related companion diagnostic tests that may be developed, could have a material and adverse effect on our business, financial condition, results of operations and prospects. 52 Risks Related to our Common Stock 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.
Our inability to promptly obtain coverage and adequate reimbursement from third-party payors for the product candidates, and for us or our collaborators to obtain coverage and adequate reimbursement for related companion diagnostic tests that may be developed, could have a material and adverse effect on our business, financial condition, results of operations and prospects. 54 Risks Related to our Common Stock If we issue additional shares of common stock in the future, it will result in the dilution of our existing stockholders and may cause the share price of our common stock to fall.
In addition, the laws of foreign countries may not protect our rights to the same extent as the laws of the United States, or vice versa. Further, we may not be aware of all third-party intellectual property rights potentially relating to and/or interfering with our product candidates.
Further, we may not be aware of all third-party intellectual property rights potentially relating to and/or interfering with our product candidates.
Moreover, if disputes over intellectual property that we have licensed prevent or impair our ability to maintain our licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected product candidates, which could have a material adverse effect on our business, financial conditions, results of operations, and prospects. 58 If we do not obtain required intellectual property licenses or rights, we could encounter delays in our product development efforts while we attempt to design around other patents or even be prohibited from developing, manufacturing or selling potential drug compounds requiring these rights or licenses.
Moreover, if disputes over intellectual property that we have licensed prevent or impair our ability to maintain our licensing arrangements on commercially acceptable terms, we may be unable to successfully develop and commercialize the affected product candidates, which could have a material adverse effect on our business, financial conditions, results of operations, and prospects.

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Item 1C. Cybersecurity

Cybersecurity — threats and controls disclosure

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Biggest changeWe also use multi-factor authentication, maintain logical, physical and technical controls designed to deter, prevent, mitigate and respond to cybersecurity threats. Further, we provide periodical cybersecurity reminders to our employees to emphasize the importance of adherence to our security policies.
Biggest changeWe also use multi-factor authentication, maintain logical, physical and technical controls designed to deter, prevent, mitigate and respond to cybersecurity threats. Further, we provide periodical cybersecurity reminders to our employees and subscribe to a third-party cybersecurity training program required for all employees, to emphasize the importance of adherence to our security policies and awareness of evolving cybersecurity threats.
For certain vendors, these processes include a comprehensive vendor audit process. As of the date of this Annual Report, we do not believe that any past cybersecurity incidents that have been detected have materially affected, or are reasonably likely to materially affect, our business strategy, results of operations, or financial condition.
For certain vendors, these processes include a comprehensive vendor audit process. 63 As of the date of this Annual Report, we do not believe that any past cybersecurity incidents that have been detected have materially affected, or are reasonably likely to materially affect, our business strategy, results of operations, or financial condition.
See “ Risk Factors - Risks Related to Our Business and Operations ” for additional information about the risks to our business associated with cybersecurity or a breach or compromise to our information security systems. 62 Governance Our board of directors addresses the Company’s cybersecurity risk management as part of its general oversight function.
See “ Risk Factors - Risks Related to Our Business and Operations ” for additional information about the risks to our business associated with cybersecurity or a breach or compromise to our information security systems. Governance Our Board of Directors addresses the Company’s cybersecurity risk management as part of its general oversight function.
The audit committee of the board of directors is responsible for overseeing the Company’s cybersecurity risk management processes, including oversight of mitigation of risks from cybersecurity threats. The audit committee receives periodic reports from senior management concerning the Company’s significant cybersecurity threats and risk and the processes the Company has implemented to address them.
The audit committee of the Board of Directors is responsible for overseeing the Company’s cybersecurity risk management processes, including oversight of mitigation of risks from cybersecurity threats . The audit committee receives periodic updates as required from senior management concerning updates to the Company’s significant cybersecurity threats and risk and the processes the Company has implemented to address them.

Item 2. Properties

Properties — owned and leased real estate

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Biggest changeITEM 2. PROPERTIES We do not own any real property. We maintain a corporate head office at 630 5th Avenue, 20th Floor, New York, NY, USA. Our lease costs for this office are approximately $11,000 per month.
Biggest changeITEM 2. PROPERTIES We do not own any real property. We maintain a corporate head office at 630 5th Avenue, 20th Floor, New York, NY, USA. During the fiscal year 2025, our costs for our physical office were approximately $11,000 per month.

Item 3. Legal Proceedings

Legal Proceedings — active lawsuits and investigations

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Biggest changeITEM 3. LEGAL PROCEEDINGS The Company is subject to claims and legal proceedings that arise during the course of business. The Company is currently subject to the following lawsuits: On March 13, 2024, a shareholder class action complaint was filed in the United States District Court for the Southern District of New York. The complaint is captioned Blum v.
Biggest changeThe Company is currently subject to the following lawsuits: On March 13, 2024, a shareholder class action complaint was filed in the United States District Court for the Southern District of New York, and it named the Company and an officer of the Company as Defendants. The complaint was amended on July 12, 2024 (the “Initial Action”).
There are no other proceedings in which any of our directors, officers or affiliates, or any registered or beneficial stockholder holding more than 5% of our shares, or any associate of such persons, is an adverse party or has a material interest adverse to our or our subsidiaries’ interest. 63 ITEM 4. MINE SAFETY DISCLOSURES Not applicable. PART II
There are no other proceedings in which any of our directors, officers or affiliates, or any registered or beneficial stockholder holding more than 5% of our shares, or any associate of such persons, is an adverse party or has a material interest adverse to our or our subsidiaries’ interest. ITEM 4. MINE SAFETY DISCLOSURES Not applicable. PART II
The complaint asserts various common law claims (including breach of fiduciary duty) and violation of Section 14(a) of the Securities Exchange Act regarding the same or similar allegations at issue in the two purported class action lawsuits related to disclosures and statements made about certain clinical trials related to Rett Syndrome.
The complaint asserts various common law claims (including breach of fiduciary duty) and violation of Section 14(a)of the Securities Exchange Act regarding the same or similar allegations at issue in the purported class action lawsuit related to disclosures and statements made about certain clinical trials related to Rett syndrome.
Plaintiff Downing voluntarily dismissed his complaint subsequent to the filing of the motion to dismiss. On or about May 13, 2024, a derivative lawsuit was filed against the Company (as nominal defendant), Christopher Missling, and members of the Company’s Board of Directors in the U.S. District Court for the District of Nevada by another purported shareholder named Denise Deangelis.
On or about May 13, 2024, a derivative lawsuit was filed against the Company (as nominal defendant), an officer of the Company, and members of the Company’s Board of Directors in the U.S. District Court for the District of Nevada by another purported shareholder.
The motion to dismiss is fully-briefed and awaiting a decision by the Court. On May 8, 2024, a similar complaint was filed in the same court by Kenneth Downing (case no. 1:2024-cv-03529), a purported shareholder of the Company, against the same defendants as the March 2024 Complaint. The defendants filed a motion to dismiss the complaint.
On May 8, 2024, a similar complaint was filed in the same court by Kenneth Downing, a purported shareholder of the Company, against the same defendants. The Company believed that this lawsuit was without merit and filed a motion to dismiss the complaint. Plaintiff Downing voluntarily dismissed this complaint subsequent to the filing of the motion to dismiss.
Anavex Life Sciences, Corp. et al., case number 1:24-cv-01910, and it named the Company and Christopher Missling as Defendants. The complaint alleges violations of the Securities and Exchange Act of 1934 associated with disclosures and statements made with respect to certain clinical trials for ANAVEX ® 2-73 related to Rett syndrome (the “March 2024 Complaint”).
The complaint alleged violations of the Securities and Exchange Act of 1934 associated with disclosures and statements made with respect to certain clinical trials for ANAVEX ® 2-73 related to Rett syndrome. This lawsuit was dismissed by the United States District Court for the Southern District of New York on June 18, 2025.
Removed
At a hearing on or about June 13, 2024, the Court named another purported Company shareholder, Quintessa Huey, as lead plaintiff with respect to the March 2024 Complaint.
Added
ITEM 3. LEGAL PROCEEDINGS The Company is subject to claims and legal proceedings that arise during the course of business.
Removed
An Amended Complaint was filed by the appointed lead plaintiff on July 12, 2024, which asserts allegations related to purported violations of Section 10(b) of the Securities Exchange Act tied to disclosures associated with the same clinical trials related to Rett Syndrome, and which names the Company and Christopher Missling as defendants.
Added
The plaintiff filed a notice of appeal on July 17, 2025. Briefing on the appeal concluded October 30, 2025. No decision has been entered. No amount has been recorded in our consolidated financial statements for any loss contingencies associated with this lawsuit as the Company believes that it is not probable that any loss will occur.
Removed
The Amended Complaint seeks unspecified damages, as well as costs, including counsel and expert witness fees, on behalf of class of investors who purchased stock of the Company on the NASDAQ during the period February 1, 2022 through January 1, 2024. The defendants filed a motion to dismiss the complaint.
Added
On January 22, 2025, pursuant to a stipulation of the parties, the Court entered an order staying this purported derivative lawsuit until the motion to dismiss filed by defendants in the Initial Action is decided by the U.S. District Court for the Southern District of New York. The stay has been extended throughout the appeal.
Removed
The parties are currently due to file a proposed schedule for the anticipated motion to dismiss by the Company and Christopher Missling (the other named defendants have not been served with the complaint) on or before January 15, 2025.
Added
No amount has been recorded in our consolidated financial statements for any loss contingencies associated with this lawsuit as the Company believes that it is not probable that any loss will occur. 64 On February 14, 2025, another derivative lawsuit asserting state law breach of fiduciary duty and unjust enrichment claims based upon similar allegations was filed against the Company (as nominal defendant), an officer of the Company, and members of the Company’s Board of Directors in the Supreme Court for the State of New York, County of New York, by another purported shareholder.
Added
On August 18, 2025, pursuant to a stipulation of the parties, the Court entered an order staying this purported derivative lawsuit until the appeal in the Initial Action is resolved.
Added
No amount has been recorded in our consolidated financial statements for any loss contingencies associated with this lawsuit as the Company believes that it is not probable that any loss will occur.

Item 4. Mine Safety Disclosures

Mine Safety Disclosures — required of mining issuers

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Biggest changeCHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL MATTERS 71 ITEM 9A. CONTROLS AND PROCEDURES 71 ITEM 9B OTHER INFORMATION 71 ITEM 9C DISCLOSURE REGARDING FOREIGN JURISDICTIONS THAT PREVENT INSPECTIONS 71 PART III 72 ITEM 10 DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE 72 ITEM 11. EXECUTIVE COMPENSATION 77 ITEM 12.
Biggest changeCHANGES IN AND DISAGREEMENTS WITH ACCOUNTANTS ON ACCOUNTING AND FINANCIAL MATTERS 73 ITEM 9A. CONTROLS AND PROCEDURES 73 ITEM 9B OTHER INFORMATION 73 ITEM 9C DISCLOSURE REGARDING FOREIGN JURISDICTIONS THAT PREVENT INSPECTIONS 73 PART III 74 ITEM 10 DIRECTORS, EXECUTIVE OFFICERS AND CORPORATE GOVERNANCE 74 ITEM 11. EXECUTIVE COMPENSATION 78 ITEM 12.
ITEM 4. MINE SAFETY DISCLOSURES 64 PART II 64 ITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES 64 ITEM 7 MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATION 64 ITEM 7A QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK 70 ITEM 8. FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA F-1 ITEM 9.
ITEM 4. MINE SAFETY DISCLOSURES 65 PART II 65 ITEM 5. MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES 65 ITEM 7 MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATION 66 ITEM 7A QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK 72 ITEM 8. FINANCIAL STATEMENTS AND SUPPLEMENTARY DATA F-1 ITEM 9.
SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS. 83 ITEM 13. CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR INDEPENDENCE 86 ITEM 14. PRINCIPAL ACCOUNTING FEES AND SERVICES 87 PART IV 88 ITEM 15. EXHIBITS, FINANCIAL STATEMENT SCHEDULES 88
SECURITY OWNERSHIP OF CERTAIN BENEFICIAL OWNERS AND MANAGEMENT AND RELATED STOCKHOLDER MATTERS. 88 ITEM 13. CERTAIN RELATIONSHIPS AND RELATED TRANSACTIONS, AND DIRECTOR INDEPENDENCE 92 ITEM 14. PRINCIPAL ACCOUNTING FEES AND SERVICES 92 PART IV 94 ITEM 15. EXHIBITS, FINANCIAL STATEMENT SCHEDULES 94

Item 5. Market for Registrant's Common Equity

Market for Common Equity — stock, dividends, buybacks

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Biggest changeThese decreases were largely offset by the following increases in research and development expenditures over the comparable fiscal 2023 financial year: (i) an increase in personnel costs of $3.4 million related to the addition of new employees including new additions to the Company’s leadership team, and expenses related to the engagement of consultants to assist in the preparation of the submission of our Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA); (ii) an increase of approximately $3.6 million over the comparable period relating to manufacturing activities of ANAVEX ® 2-73 for potential commercial use, and to support the MAA; and (iii) an increase of $2.2 million over the comparable period relating to expenditures on the ANAVEX ® 3-71-SZ-001 clinical trial, which trial commenced in the second quarter of fiscal 2024. 65 The following table summarizes our research and development expenses for the years ended September 30, 2024, and 2023 (in thousands): 2024 2023 Costs of external service providers $ 21,974 $ 22,542 Personnel costs 13,676 10,264 Share-based compensation 5,813 10,812 Other common costs 375 99 Total research and development costs $ 41,838 $ 43,717 External service provider cost by product candidate was as follows (in thousands): 2024 2023 ANAVEX ® 2-73 $ 17,572 $ 19,540 ANAVEX ® 3-71 3,748 2,624 All other product candidates 150 6 Other external service provider costs 504 372 Total external service provider costs $ 21,974 $ 22,542 General and administrative expenses were $11.0 million for the fiscal 2024 financial year, as compared to $12.0 million in fiscal 2023.
Biggest changeThe following table summarizes our research and development expenses for the years ended September 30, 2025, 2024, and 2023 (in thousands): 2025 2024 2023 Costs of external service providers $ 16,348 $ 21,974 $ 22,542 Personnel costs 13,466 13,676 10,264 Share-based compensation 7,013 5,813 10,812 Other common costs 765 375 99 Total research and development costs $ 37,592 $ 41,838 $ 43,717 External service provider cost by product candidate was as follows (in thousands): 2025 2024 2023 ANAVEX ® 2-73 $ 10,292 $ 17,572 $ 19,540 ANAVEX ® 3-71 5.408 3,748 2,624 All other product candidates 297 150 6 Other external service provider costs 351 504 372 Total external service provider costs $ 16,348 $ 21,974 $ 22,542 During fiscal 2025, we experienced an overall decrease in total research and development expenses over the comparable fiscal 2024 financial year.
The fair value of all share purchase options and warrants are expensed over their contractual vesting period, or over the expected performance period for only the portion of awards expected to vest, in the case of milestone-based vesting, with a corresponding increase to additional paid-in capital. Compensation costs for share-based payments with graded vesting are recognized on a straight-line basis.
The fair value of all share purchase options and warrants are expensed over their contractual vesting period, or over the expected performance period for only the portion of awards expected to vest, in the case of milestone-based vesting, with a corresponding increase to additional paid-in capital. 71 Compensation costs for share-based payments with graded vesting are recognized on a straight-line basis.
Off-Balance Sheet Arrangements We have no off-balance sheet arrangements that have or are reasonably likely to have a current or future effect on our financial condition, changes in financial condition, revenues or expenses, results of operations, liquidity, capital expenditures or capital resources that are material to our stockholders. 68 Application of Critical Accounting Policies Our financial statements and accompanying notes are prepared in accordance with generally accepted accounting principles in the United States.
Off-Balance Sheet Arrangements We have no off-balance sheet arrangements that have or are reasonably likely to have a current or future effect on our financial condition, changes in financial condition, revenues or expenses, results of operations, liquidity, capital expenditures or capital resources that are material to our stockholders. 70 Application of Critical Accounting Policies Our financial statements and accompanying notes are prepared in accordance with generally accepted accounting principles in the United States.
Due to these risks and uncertainties, we expense the acquisition of patents and trademarks. 69 Share-based Compensation We account for all share-based payments and awards under the fair value-based method.
Due to these risks and uncertainties, we expense the acquisition of patents and trademarks. Share-based Compensation We account for all share-based payments and awards under the fair value-based method.
We do not anticipate earning any revenues until we can establish an alliance with other companies to develop, co-develop, license, acquire or market our products. 64 Our operating costs consist primarily of research and development activities including the cost of clinical studies and clinical supplies as well as clinical drug manufacturing and formulation.
We do not anticipate earning any revenues until we can establish an alliance with other companies to develop, co-develop, license, acquire or market our products. 66 Our operating costs consist primarily of research and development activities including the cost of clinical studies and clinical supplies as well as clinical drug manufacturing and formulation.
We estimate an implicit service period for achieving performance criteria for each award and recognizes the resulting fair value as expense over the implicit service period when we conclude that achieving the performance criteria is probable. We periodically review and update as appropriate our estimates of implicit service periods and conclusions on achieving the performance criteria.
We estimate an implicit service period for achieving performance criteria for each award and recognize the resulting fair value as expense over the implicit service period when we conclude that achieving the performance criteria is probable. We periodically review and update as appropriate our estimates of implicit service periods and conclusions on achieving the performance criteria.
In addition, if the Company has directed Lincoln Park to purchase the full amount of Common Stock available as a Regular Purchase on a given day, it may direct Lincoln Park to purchase additional amounts as “accelerated purchases” and “additional accelerated purchases,” each as set forth in the 2023 Purchase Agreement.
In addition, if we have directed Lincoln Park to purchase the full amount of Common Stock available as a Regular Purchase on a given day, we may direct Lincoln Park to purchase additional amounts as “accelerated purchases” and “additional accelerated purchases,” each as set forth in the 2023 Purchase Agreement.
During fiscal 2024, we recorded $2.3 million in research and development incentive income, consisting of the Australian research and development incentive credit administered through the ATO, in connection with fiscal 2024 eligible expenditures. In comparison, research and development incentive income for fiscal 2023 was $2.7 million in connection with fiscal 2023 eligible expenditures.
During fiscal 2025, we recorded $0.6 million in research and development incentive income, consisting of the Australian research and development incentive credit administered through the ATO, in connection with fiscal 2025 eligible expenditures. In comparison, research and development incentive income for fiscal 2024 was $2.3 million in connection with fiscal 2024 eligible expenditures.
Cash flow provided by financing activities Cash provided by financing activities in fiscal 2024 was $12.0 million, comprised of $11.3 attributable to cash received from the issuance of common shares at various market prices under the 2023 Purchase Agreement (as defined below) and $0.7 million received pursuant to the exercise of stock options.
Cash provided by financing activities in fiscal 2024 was $12.0 million, comprised of $11.3 million attributable to cash received from the issuance of common shares under the 2023 Purchase Agreement and $0.7 million received pursuant to the exercise of stock options.
MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market information Our common stock is quoted on the Nasdaq Global Select Stock Market (“Nasdaq”) under the symbol “AVXL.” Holders of Common Stock As of December 23, 2024, there were approximately 48 stockholders of record, and 84,815,517 shares of our common stock were issued and outstanding.
MARKET FOR REGISTRANT’S COMMON EQUITY, RELATED STOCKHOLDER MATTERS AND ISSUER PURCHASES OF EQUITY SECURITIES Market information Our common stock is quoted on the Nasdaq Global Select Stock Market (“Nasdaq”) under the symbol “AVXL.” Holders of Common Stock As of November 24, 2025, there were approximately 49 stockholders of record, and 89,348,107 shares of our common stock were issued and outstanding.
RECENT ACCOUNTING PRONOUNCEMENTS For a discussion of recent accounting pronouncements and their possible effect on our results, see Note 2 to our Consolidated Financial Statements found elsewhere in this Annual Report. ITEM 7A QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK Not required for smaller reporting companies. 70
RECENT ACCOUNTING PRONOUNCEMENTS For a discussion of recent accounting pronouncements and their possible effect on our results, see Note 2 to our Consolidated Financial Statements found elsewhere in this Annual Report. ITEM 7A QUANTITATIVE AND QUALITATIVE DISCLOSURES ABOUT MARKET RISK Interest Rate Risk We invest our excess cash in investment-grade, interest-bearing securities.
Past operating results are not necessarily indicative of results that may occur in future periods. This discussion contains forward-looking statements, which involve a number of risks and uncertainties. See Forward Looking Statements included elsewhere in this report. Financial Operations Overview We are in the development stage and have not earned any revenues since our inception in 2004.
Past operating results are not necessarily indicative of results that may occur in future periods. This discussion contains forward-looking statements, which involve a number of risks and uncertainties. See “Forward Looking Statements” included elsewhere in this report.
Cash Flows Following is a summary of sources of cash flows for the years ended September 30, 2024 and 2023 (in thousands) 2024 2023 Cash flows used in operating activities $ (30,812 ) $ (27,785 ) Cash flows provided by financing activities 11,975 29,651 (Decrease) increase in cash $ (18,837 ) $ 1,866 Cash flow used in operating activities There was an increase in cash used in operating activities of $3.0 million during fiscal 2024.
We intend to continue to use our capital resources to advance our clinical trials for ANAVEX ® 2-73 and ANAVEX ® 3-71, and to perform work necessary to prepare for future development of our pipeline compounds. 68 Cash Flows Following is a summary of sources of cash flows for the years ended September 30, 2025, 2024 and 2023 (in thousands): 2025 2024 2023 Cash flows used in operating activities $ (39,044 ) $ (30,812 ) $ (27,785 ) Cash flows provided by financing activities 9,434 11,975 29,651 (Decrease)/increase in cash $ (29,610 ) $ (18,837 ) $ 1,866 Cash flow used in operating activities There was an increase in cash used in operating activities of $8.2 million during fiscal 2025.
Other Financings 2023 Purchase Agreement On February 3, 2023, the Company entered into a $150,000,000 purchase agreement (the “2023 Purchase Agreement”) with Lincoln Park Capital Fund, LLC (“Lincoln Park”), pursuant to which the Company has the right to sell and issue to Lincoln Park, and Lincoln Park is obligated to purchase, up to $150.0 million in value of its shares of Common Stock from time to time over a three-year period until February 3, 2026. 67 On any business day and subject to certain customary conditions, the Company may direct Lincoln Park to purchase up to 200,000 shares of Common Stock (such purchases, “Regular Purchases”).
At September 30, 2025, there was an unused amount of $140.4 million under the Sales Agreement. 69 2023 Purchase Agreement On February 3, 2023, we entered into a $150,000,000 purchase agreement (the “2023 Purchase Agreement”) with Lincoln Park Capital Fund, LLC (“Lincoln Park”), pursuant to which we have the right to sell and issue to Lincoln Park, and Lincoln Park is obligated to purchase, up to $150.0 million in value of our shares of Common Stock from time to time over a three-year period until February 3, 2026.
We expect to continue to receive support from the Australian government for future clinical trials which we plan to conduct, in part, within Australia.
This income is driven by the clinical trial expenditures incurred in Australia, and the decrease year over year is a result of the completion of eligible R&D clinical trials in Australia. We expect to continue to receive support from the Australian government for future clinical trials which we plan to conduct, in part, within Australia.
Cash provided by financing activities in fiscal 2023 was $29.7 million, comprised of $27.9 million attributable to cash received from the issuance of common shares under the 2023 Purchase Agreement and $1.8 million received pursuant to the exercise of stock options.
Cash flow provided by financing activities Cash provided by financing activities in fiscal 2025 was $9.4 million, comprised primarily of net cash received of $9.2 million related to the issuance of common shares pursuant to the at-the-market offering and $2.9 million in cash from the exercise of stock options by our employees.
The decrease is attributable to a modest decrease in research and development expenses of $1.9 million (4.3%) to $41.8 million in fiscal 2024 as well as a small decrease in general and administrative expenses of $1.0 million (8.3%) to $11.0 million in fiscal 2024, as more fully described below.
Comparison of fiscal year 2025 to fiscal years 2024 Operating Expenses Our operating expenses for fiscal 2025 decreased to $51.4 million, from $52.9 million in fiscal 2024. The decrease is attributable to research and development expenses, as more fully described below.
Our future dividend policy will be determined from time to time by our Board of Directors.
Our future dividend policy will be determined from time to time by our Board of Directors. Performance Graph The following graph compares the performance of our Common Stock for the periods indicated with the performance of the NASDAQ Composite Index and the NASDAQ Biotechnology Index.
Net loss Net loss for fiscal 2024 was $43.0 million, or $0.52 per share, compared to a net loss of approximately $47.5 million, or $0.60 per share for fiscal 2023. 66 Liquidity and Capital Resources Working Capital (in thousands) 2024 2023 Current Assets $ 135,567 $ 154,386 Current Liabilities 15,304 12,534 Working Capital $ 120,263 $ 141,852 At September 30, 2024, we had $132.2 million in cash and cash equivalents, a decrease from $151.0 million at September 30, 2023.
Liquidity and Capital Resources Working Capital (in thousands) 2025 2024 Current Assets $ 103,815 $ 135,567 Current Liabilities 8,946 15,304 Working Capital $ 94,869 $ 120,263 At September 30, 2025, we had $102.6 million in cash and cash equivalents, a decrease from $132.2 million at September 30, 2024.
Other income (net) Net other income for the year ended September 30, 2024 was $9.9 million as compared to $8.3 million for fiscal 2023.
We expect to see our research and development expenditures increase from current levels as we continue to advance our pipeline compounds. Other income (net) Net other income for the year ended September 30, 2025 was $5.0 million as compared to $9.9 million for fiscal 2024.
On September 30, 2024, an amount of $110.8 million remained available under the 2023 Purchase Agreement.
On September 30, 2025, there was an unused amount of $110.8 million under the 2023 Purchase Agreement. The Company will need to file a prospectus supplement in order to access funds under the 2023 Purchase Agreement.
During the year ended September 30, 2023, the Company issued to Lincoln Park an aggregate of 3,288,943 shares of Common Stock under the 2023 Purchase Agreement, including 3,275,000 shares of Common Stock for an aggregate purchase price of $27.9 million and 13,943 commitment shares as well as the 75,000 initial commitment shares.
During the year ended September 30, 2025, the Company issued an aggregate of 927,910 shares of Common Stock under the Sales Agreement for net proceeds of $9.2 million, after deducting commissions and offering expenses.
Controlled Equity Offering Sales Agreement On May 1, 2020, we entered into an Amended and Restated Sales Agreement (the “2020 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 “At-the-Market Offering”).
Other Financings Sales Agreement On July 25, 2025, we entered into a Sales Agreement (the “Sales Agreement”) with TD Securities (USA) LLC (the “Sales Agent”). Pursuant to the Sales Agreement, the Company may offer and sell up to an aggregate offering price of $150 million (the “Offering”) in shares of common stock from time to time through the Sales Agent.
Recent Sales of Unregistered Securities Since the beginning of our fiscal year ended September 30, 2024, we have not sold any equity securities that were not registered under the Securities Act of 1933 that were not previously reported in a quarterly report on Form 10-Q or in a current report on Form 8-K.
This graph is not “soliciting material,” is not deemed “filed” with the SEC and is not to be incorporated by reference into any of our filings under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, whether made before or after the date hereof and irrespective of any general incorporation language in any such filing. 65 2020 2021 2022 2023 2024 2025 NASDAQ Biotechnology Index (NBI) 100.00 119.46 88.51 92.88 112.01 114.64 S&P Biotech ETF (XBI) 100.00 112.82 71.18 65.53 88.67 89.92 AVXL 100.00 394.51 226.81 143.96 124.84 195.60 Recent Sales of Unregistered Securities Since the beginning of our fiscal year ended September 30, 2025, we have not sold any equity securities that were not registered under the Securities Act of 1933 that were not previously reported in a quarterly report on Form 10-Q or in a current report on Form 8-K.
Removed
Comparison of year ended September 30, 2024 to year ended September 30, 2023 Operating Expenses Our operating expenses for fiscal 2024 decreased to $52.9 million, from $55.8 million in fiscal 2023.
Added
This graph assumes an investment of $100 after the market closed September 30, 2020 in each of our common stock, the NASDAQ Composite Index and the NASDAQ Biotechnology Index, and assumes reinvestment of dividends, if any. The stock price performance shown on the graph below is not necessarily indicative of future stock price performance.
Removed
During fiscal 2024, we experienced an overall decrease in total research and development expenses over the comparable fiscal 2023 financial year.
Added
For discussion and analysis pertaining to 2024 overview and highlights as compared to 2023, please refer to the Company’s Annual Report on Form 10-K, filed with the Securities and Exchange Commission (“SEC”) on December 23, 2024. Financial Operations Overview We are in the pre-revenue stage and have not earned any revenues since our inception in 2004.
Removed
The decreases were largely due to: (i) a decrease in share-based compensation expense of $5.0 million as a result of the vesting of previous option awards and a change in estimated vesting dates associated with performance-based option awards; (ii) a decrease of approximately $3.5 million relating to our Rett syndrome program as a result of the completion of the EXCELLENCE trial and the respective open label extension; and (iii) a decrease of approximately $1.2 million in expenditures over the comparable period relating to our Alzheimer’s program, as a result of the completion of the Phase 2b/3 clinical trial and its related open label extension.
Added
The main factors driving this decrease were as follows: (i) a decrease of approximately $3.6 million related to the Alzheimer’s program due to the completion of the ATTENTION-AD trial in the third quarter of fiscal 2024 and an overall decrease in related trial data analysis activities; (ii) a decrease of approximately $1.7 million associated with the Parkinson’s program related to planning activities for a future clinical trial in the comparable period; (iii) a decrease of approximately $1.5 million over the comparable period relating to manufacturing activities of ANAVEX ® 2-73 for potential commercial use, and to support the MAA; 67 (iv) a decrease of approximately $0.9 million related to the completion of a production run of ANAVEX ® 3-71 in fiscal 2024.
Removed
The primary reason for the decrease in general and administrative expenses was a reduction in share-based compensation charges of $1.9 million, as a result of the vesting of previous option awards and the extended timeline of milestone based vesting awards.
Added
(v) a decrease of approximately $0.8 million associated with our Rett syndrome program, due to the completion of the ANAVEX ® 2-73 EXCELLENCE OLE in the third quarter of fiscal 2024.
Removed
We expect to see our research and development expenditures increase from current levels as we advance our clinical programs, including continuation of ANAVEX ® 3-71 trial in Schizophrenia and subsequent advancements, planned advancement of ANAVEX ® 2-73 for Parkinson ’ s disease, planned initiation of an ANAVEX ® 2-73 for a Fragile X clinical trial, and as we continue to grow our staffing to manage and support these clinical initiatives.
Added
The above decreases were partially offset by an increase of approximately $3.0 million related to completion of Part B of the ANAVEX ® 3-71-SZ-001 trial during fiscal 2025, which was substantially larger in size than the preceding Part A during fiscal 2024.
Removed
The primary reason for the increase in other income was due to a one-time financing charge of $0.9 million recognized in the comparable year associated with entering into the 2023 Purchase Agreement (as described below), as well as an increase in interest income in fiscal 2024 earned on cash and cash equivalents, due to an increase in market wide interest rates year over year.
Added
General and administrative expenses were $13.8 million for the fiscal 2025 financial year, as compared to $11.0 million in fiscal 2024. The primary reason for the increase in general and administrative expenses was an increase in legal fees of $1.7 million, related to legal/regulatory matters, a new shelf registration statement, and various class action lawsuits.
Removed
This income is driven by the clinical trial expenditures incurred in Australia, and the decrease is a result of the completion of the EXCELLENCE trial in Rett Syndrome and the Phase 2b/3 clinical trial in Alzheimer ’ s disease, as well as related open label extension trials, which were completed during fiscal 2024.
Added
The primary reason for the decrease in other income was due to a decrease of $2.6 million in interest income as a result of withdrawals in principal balance applied to excess funds invested in a money market as well as a market wide decrease in interest rates.
Removed
We intend to continue to use our capital resources to advance our clinical trials for ANAVEX ® 2-73 and ANAVEX ® 3-71, and to perform work necessary to prepare for future development of our pipeline compounds.
Added
Net loss Net loss for fiscal 2025 was $46.4 million, or $0.54 per share, compared to a net loss of approximately $43.0 million, or $0.52 per share for fiscal 2024.
Removed
The principal reason for this is an increase in net cash expenses, after taking into account non-cash share-based compensation, over the comparable period of approximately $3.3 million.
Added
The principal reason for this is due to a large decrease in accounts payable during the year, as compared to a large increase in the comparable financial year, principally due to timing of payments for a large manufacturing campaign of ANAVEX ® 2-73.
Removed
No shares were sold during the years ended September 30, 2024 and 2023 under the 2020 Sales Agreement. The Company terminated the 2020 Sales Agreement on July 24, 2024.
Added
We utilized $2.7 million to satisfy tax withholding obligations associated with the net exercise of two expiring employee stock options to our CEO, in exchange for the withholding of shares.
Added
Upon delivery of a placement notice based on our instructions and subject to the terms and conditions of the Sales Agreement, the Sales Agent may sell shares of common stock by methods deemed to be an “at the market offering”, in negotiated transactions at market prices prevailing at the time of sale or at prices related to such prevailing market prices, or by any other method permitted by law, including negotiated transactions, subject to our prior written consent.
Added
We are not obligated to make any sales of shares under the Sales Agreement. We or the Sales Agent may suspend or terminate the Offering upon notice to the other party, subject to certain conditions.
Added
The Sales Agent will act as sales agent on a commercially reasonable efforts basis consistent with its normal trading and sales practices, applicable state and federal law, rules and regulations and the rules of Nasdaq.
Added
We have agreed to pay the Sales Agent commissions for its services of up to 3.0% of the gross proceeds from the sale of shares of Common Stock pursuant to the Sales Agreement. We have also agreed to provide the Sales Agent with customary indemnification and contribution rights.
Added
On any business day and subject to having an effective registration statement and subject to certain customary conditions, we may direct Lincoln Park to purchase up to 200,000 shares of Common Stock (such purchases, “Regular Purchases”).
Added
During the year ended September 30, 2025, the Company did not issue any shares of common stock under the 2023 Purchase Agreement.
Added
The primary objective of our investment policy is to preserve principal and liquidity. To achieve this objective, our investment policy allows for investments in domestic money market certificates, certificates of deposit, money market funds, bonds or commercial papers, and establishes diversification and credit quality requirements and limits investments by maturity and issuer.
Added
At September 30, 2025 and 2024, the majority of our excess cash was held in a JP Morgan Chase Prime Money Market Fund. The average amount invested at any given time throughout the year ended September 30, 2025 was $109.3 million (high: $127.0 million; low: $90.6 million) and the average rate of return was 4.28%.
Added
A hypothetical 100 basis point change in interest rates during any of the periods presented would not have a material impact on the fair market value of our cash and cash equivalents as of September 30, 2025 and 2024 and would impact our net loss by approximately $1.1 million.
Added
To date, we have not experienced a loss of principal on any of our investments and as of September 30, 2025, we did not have any allowance for credit losses from our cash and cash equivalents.
Added
Foreign Exchange Risk We face foreign exchange risk as a result of entering into transactions denominated in currencies other than U.S. dollars and as a result of the existence of sales and incentive tax receivables denominated in other than U.S. dollars. Due to the uncertain timing of expected payments in foreign currencies, we do not utilize any forward exchange contracts.
Added
All foreign transactions settle on the applicable spot exchange basis at the time such payments are made. Volatile market conditions and supply chain shortages may result in significant changes in exchange rates, and in particular a change in foreign currencies values relative to the U.S. dollar may affect our operating expenses as expressed in U.S. dollars.
Added
An adverse movement in foreign exchange rates could have a material effect on payments made to foreign suppliers. For the year ended September 30, 2025, a majority of our expenses were denominated in U.S. dollars.
Added
A hypothetical 10% change in foreign exchange rates applied to foreign currency transactions for the year ended September 30, 2025 would not have had a material impact on our consolidated financial statements. At September 30, 2025, we held net assets of $1.9 million (AUD $2.9 million) denominated in Australian dollars.
Added
A hypothetical 10% change in foreign exchange rates at September 30, 2025 would result in a change in reported net assets of +/- $0.19 million. Inflation Risk Inflation generally may affect us by increasing our cost of labor and clinical trial costs.
Added
We do not believe that inflation has had a material impact on our results of operations during the periods presented. 72

Other AVXL 10-K year-over-year comparisons