ANAVEX LIFE SCIENCES CORP.
For the period ended Mar 31, 2026. Filed Aug 28, 2026.
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A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2026 · Filed Aug 28, 2026
AVXL$1.15+3.6%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-12-31; filed 2026-02-09.
0001731122-26-000200
ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONSCurrent report: 10-Q
Period ended 2026-03-31; filed 2026-08-28.
0001731122-26-001166
ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONSThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
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Added paragraphs (42)
Current report
ITEM 2. MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS.
Current report
We are a clinical stage biopharmaceutical company engaged in the development of novel therapeutics for the treatment of central nervous system (“CNS”) diseases with high unmet medical needs. Our primary focus is on advancing our lead compound ANAVEX 2-73 (blarcamesine) for the treatment of mild cognitive impairment (“MCI”) due to Alzheimer’s disease (“AD”) and mild AD (collectively known as “early AD”), and for Rett syndrome and Fragile X syndrome, both of which are neurodevelopmental rare diseases. Under new leadership, we are currently prioritizing engagement with the U.S. FDA to align on a clear, data-driven regulatory and clinical development strategy for ANAVEX 2-73 for the above indications.
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Clinical Development Pipeline
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Our lead compound is ANAVEX 2-73 (blarcamesine). Below is our clinical development pipeline for ANAVEX 2-73 (blarcamesine) across CNS indications:
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Progress bars show the most advanced phase per indication for ANAVEX 2-73 (blarcamesine), an investigational oral SIGMA-1 receptor agonist. Regulatory goals are planned and are subject to change, including as a result of ongoing and future discussions with the FDA.
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Following a review of our clinical development strategy under new leadership, we have de-prioritized our other assets, including ANAVEX®3-71, currently in the clinical stage, and ANAVEX®1-41 and ANAVEX®1066, currently in preclinical development. Development work on these de-prioritized assets has been paused, and future development work will be contingent on additional funding for such assets or the signing of a strategic partnership for such assets. It is also possible that we may license or sell one or more of our assets.
Current report
Following the previously disclosed termination of our former CEO in April 2026, management has determined that our disclosure controls and procedures and our internal controls over financial reporting were not effective as of September 30, 2025, December 31, 2025, March 31, 2026 and June 30, 2026, which is further described in Part II, Item 4 of this Quarterly Report on Form 10-Q. As we align with the FDA on a clear, data-driven regulatory and clinical development strategy for ANAVEX 2-73 for early AD, Rett syndrome and Fragile X syndrome, we will obtain guidance from the FDA on the extent to which the efficacy data from our past clinical trials can be applied to our clinical development strategy, and, to the extent such guidance is relevant to our ongoing development plans, we expect to provide information on how this efficacy data is viewed by the FDA. In light of the foregoing, investors should rely only on the descriptions of our clinical trials, including for our de-prioritized assets, contained in this Quarterly Report on Form 10-Q (including the description below of the CHMP’s Withdrawal Assessment Report on our submission to the EMA relating to ANAVEX 2-73 as an add-on therapy for early Alzheimer’s disease) and on any future updated information.
Current report
We plan to prioritize the advancement of our ANAVEX 2-73 clinical development pipeline in early AD, Rett syndrome and Fragile X syndrome in the U.S.
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EMA
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In April 2026, we requested scientific advice from the EMA regarding the design of our proposed pivotal Phase 3 clinical trial of blarcamesine in early AD, and the CHMP adopted its advice in June 2026. The advice addressed the overall design of the proposed Phase 3 trial, including matters such as study population, endpoint hierarchy, treatment duration, statistical framework and subgroup strategy. We intend to incorporate this feedback into our ongoing discussions with the FDA regarding future study design and the overall development program for blarcamesine.
Current report
In June 2026, the CHMP published a Withdrawal Assessment Report documenting the completion of its review of the MAA for ANAVEX 2-73, concluding that the overall benefit-risk balance was negative and recommending refusal of a conditional marketing authorization. The CHMP determined that our single pivotal Phase 2b/3 trial (ANAVEX2-73-AD-004) did not meet its co-primary endpoints. As reported by the CHMP, while the ADAS-Cog13 cognitive endpoint showed a nominally significant result under our primary analysis, the ADCS-ADL functional endpoint did not reach statistical significance, which the CHMP concluded rendered the trial formally negative. The CHMP further found that the statistical analysis underlying the nominally significant ADAS-Cog13 result was not the analysis pre-specified in the study’s protocol or statistical analysis plan, but reflected changes to the analysis model, choice of covariates, and covariance structure made after the study was unblinded. Therefore, the CHMP concluded that this modified analysis constituted a post-hoc analysis that could not render the failed study successful.
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FDA
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In March 2026, we submitted an Investigational New Drug (“IND”) application to the FDA for ANAVEX 2-73 for the treatment of early AD (the “AD IND”). The submission included cross-referenced information from an existing IND for Rett syndrome that we submitted to the FDA in October 2017 (the “Rett IND”). We are currently proceeding with nonclinical and two foundational clinical pharmacology studies under the AD IND to support and strengthen our regulatory strategy for ANAVEX 2-73 – an absorption, distribution, metabolism, and excretion (“ADME”) study and a drug-drug interaction (“DDI”) study. The first participant visit in the ADME study occurred in August 2026. Dosing in the DDI study is underway and the last participant completing dosing is targeted for the end of September 2026. These studies are not sequential requirements. Rather, these studies support the overall FDA regulatory requirements for ANAVEX 2-73 while we continue to advance our early AD program in parallel. Such studies are not indication-specific and would apply across all of our ANAVEX2-73 programs.
Current report
We have submitted clinical trial data under the AD IND from our Phase 2a clinical trial in mild-to-moderate AD (ANAVEX 2-73-002), the related open label extension trial (ANAVEX 2-73-003), the ANAVEX2-73-AD-004 trial and the related open-label extension trial (ANAVEX2-73-AD-EP-004). Data from these studies will form the basis of planned discussions with the FDA on a U.S. clinical development program and a Phase 3 protocol design.
Current report
The FDA has previously granted Orphan Drug Designation for ANAVEX 2-73 for the treatment of Rett syndrome and for the treatment of Fragile X syndrome. Additionally, the FDA has granted the Rare Pediatric Disease designation and Fast Track designation for ANAVEX 2-73 for the treatment of Rett syndrome. We are moving forward with initiating a Phase 3 clinical trial for Rett syndrome (ANAVEX2-73-RS-005) in adults while working with the FDA in parallel to align on inclusion of pediatric patients in this trial. We submitted a meeting request to the FDA in August 2026 to discuss adding pediatric patients to the protocol.
Current report
ANAVEX2-73-RS-005 study is a randomized, double-blind, placebo-controlled trial evaluating the safety, tolerability and efficacy of once-daily oral blarcamesine in approximately 170 participants with Rett syndrome. Following a 12-week double-blind treatment period and a 4-week safety follow-up, eligible participants may continue into an open-label extension.
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We plan to submit an IND for ANAVEX 2-73 for the treatment of Fragile X syndrome in September 2026.
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In summary, we plan to align with the FDA on our three prioritized clinical development programs, including obtaining guidance from the FDA on all of our past clinical trials for these programs and the extent to which they can support our clinical development plan. Specifically, we plan to align on three prioritized programs including (i) a clinical development strategy for the treatment of early AD, (ii) a Phase 3 trial protocol for the treatment of Rett syndrome that includes pediatrics and (iii) a clinical development strategy for the treatment of Fragile X syndrome. We plan to provide updates on the status of these programs in future filings as we receive guidance from the FDA.
Current report
We are party to an exclusive license agreement with Life Science Research Israel Ltd. (“LSRI”) pursuant to which we license certain intellectual property related to ANAVEX 3-71 in exchange for certain payments upon the accomplishment of certain regulatory milestones and a royalty applicable to net sales of any approved product using the intellectual property licensed from LSRI.
Current report
The FDA has granted Orphan Drug Designation to ANAVEX 3-71 for the treatment of Frontotemporal Dementia (“FTD”). Development work on ANAVEX 3-71 has been paused and further development work will be contingent on additional funding for ANAVEX 3-71 or the signing of a strategic partnership. It is also possible that we may sub-license ANAVEX 3-71.
Current report
ANAVEX 1066, a mixed sigma-1/sigma-2 ligand, is designed for the potential treatment of neuropathic and visceral pain. Development work on ANAVEX 1066 has been paused and further development work will be contingent on additional funding for ANAVEX 1066 or the signing of a strategic partnership. It is also possible that we may license or sell ANAVEX 1066.
Current report
Of the total, at least one (1) U.S. patent relates to crystalline forms of ANAVEX 2-73. This patent expires in 2036. At least seven (7) of the issued U.S. patents relate to ANAVEX 2-73 for the treatment of AD. These patents expire in 2034 as to one patent, 2036 as to one patent, 2037 as to four patents, and 2040 as to one patent. At least four (4) of the issued U.S. patents relate to ANAVEX 2-73 for the treatment of Rett syndrome. These patents expire in 2037 as to three patents, and 2040 as to one patent. Additionally, at least one (1) of the issued U.S. patents relates to ANAVEX 2-73 for the treatment of Fragile X syndrome and expires in 2040.
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Recent Developments
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Nasdaq Compliance
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As previously disclosed, on May 20, 2026, we received a deficiency notification letter from the Nasdaq Listing Qualifications Department (the “Nasdaq Staff”) indicating that, as a result of our inability to timely file our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2026, we are not in compliance with Nasdaq Listing Rule 5250(c)(1), which requires listed companies to timely file all required periodic financial reports with the SEC. On July 20, 2026, we timely submitted our plan (“Compliance Plan”) to the Nasdaq Staff to regain compliance with Nasdaq Listing Rule 5250(c)(1). The Compliance Plan is currently under review by the Nasdaq Staff.
Current report
We expect the filing of this Quarterly Report on Form 10-Q, and our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2026 to cure the deficiency. However, the Nasdaq Staff will consider multiple factors when reviewing our Compliance Plan, including our past compliance history, the reasons for the late filing, other corporate events that may occur within our review period, our overall financial condition and our public disclosures. If the Compliance Plan is not accepted by the Nasdaq Staff, the Company will have an opportunity to request a hearing on the Nasdaq Staff’s determination before an independent Hearings Panel.
Current report
Our research and development expenses for the three months ended March 31, 2026 were $4.2 million as compared to $9.9 million for the three months ended March 31, 2025.
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Comparison of the six months ended March 31, 2026 and 2025
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Operating Expenses
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Total operating expenses for the six months ended March 31, 2026 were $13.3 million, compared to $26.1 million for the six months ended March 31, 2025. The primary reason for the decrease in operating expenses is due to the decrease in research and development expenses, which is more fully described below.
Current report
Our research and development expenses for the six months ended March 31, 2026 were $8.8 million, as compared to $20.3 million for the six months ended March 31, 2025.
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Other income (net)
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Net other income for the six months ended March 31, 2026 was $2.3 million, as compared to $2.8 million for the six months ended March 31, 2025. The decrease is primarily related to a decrease in interest income of $0.5 million as a result of lower interest rates.
Current report
Net loss
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Net loss for the six months ended March 31, 2026, was $11.0 million, or $0.12 per share, as compared to $23.3 million, or $0.27 per share for the six months ended March 31, 2025. The decrease is primarily related to a decrease in research and development expenses, as more fully described above.
Current report
We had cash and cash equivalents of $127.4 million as of March 31, 2026, compared to $131.7 million as of December 31, 2025. We expect our cash balance at the end of the second quarter of 2026 to fund operations and planned development activities until mid to late fiscal 2028.
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Cash flows used in investing activities
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There were no cash flows from investing activities for the six-month periods ended March 31, 2026 and 2025.
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Financings
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Funding Requirements
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Previous report
Anavex Life Sciences Corp. is a clinical stage biopharmaceutical company engaged in the development of differentiated therapeutics by applying precision medicine to central nervous system (“CNS”) diseases with high unmet need. We analyze genomic data from clinical trials to identify biomarkers, which we use in the analysis of our clinical trials.
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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.
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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.
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The following table summarizes key information about our programs:
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** = Orphan Drug Designation by the FDA*
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Anavex has a portfolio of compounds varying in sigma-1 receptor (SIGMAR1) binding activities. Sigma receptors may be targets for therapeutics to combat many human diseases, both of neurodegenerative nature, including Alzheimer’s disease, as well as of neurodevelopmental nature, like Rett syndrome. When bound by the appropriate ligands, sigma receptors influence the functioning of multiple biochemical signals that are involved in the pathogenesis (origin or development) of disease. Multiple viruses including SARS-CoV-2 (COVID-19) induce cellular stress by intrinsic mitochondrial apoptosis and other related cellular processes, in order to ensure survival and replication. Hence, it is possible that SIGMAR1 could also play a role in modulating the cellular response to viral infection and ameliorate pathogenesis.
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Source: Reyes S et al., Sci Rep. 2021 Aug 25; 11(1):17150
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Cellular Homeostasis
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Many diseases are possibly directly caused by chronic homeostatic imbalances or cellular stress of brain cells. In pediatric diseases, such as Rett syndrome or infantile spasms, chronic cellular stress is possibly caused by the presence of a constant genetic mutation. In neurodegenerative diseases, such as Alzheimer’s and Parkinson’s diseases, chronic cellular stress is possibly caused by age-correlated buildup of cellular insult and hence chronic cellular stress. Specifically, defects in homeostasis of protein or ribonucleic acid (“RNA”) lead to the death of neurons and dysfunction of the nervous system. The spreading of protein aggregates resulting in a proteinopathy, a characteristic found in Alzheimer’s and Parkinson’s diseases that results from disorders of protein synthesis, trafficking, folding, processing or degradation in cells. The clearance of macromolecules in the brain is particularly susceptible to imbalances that result in aggregation and degeneration in nerve cells. For example, Alzheimer’s disease pathology is characterized by the presence of amyloid plaques, and neurofibrillary tangles, which are aggregates of hyperphosphorylated Tau protein that are a marker of other diseases known as tauopathies as well as inflammation of microglia. 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.
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Clinical Program Overview
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Alzheimer’s Disease
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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. This open-label randomized trial in Australia met both primary and secondary endpoints and was designed to assess the safety and exploratory efficacy of ANAVEX®2-73 in 32 patients. ANAVEX®2-73 targets sigma-1 and muscarinic receptors, which have been shown in preclinical studies to reduce stress levels in the brain believed to restore cellular homeostasis and to reverse the pathological hallmarks observed in Alzheimer’s disease. In October 2017, we presented positive pharmacokinetic (“PK”) and pharmacodynamic (“PD”) data from the Phase 2a clinical trial, which established a concentration-effect relationship between ANAVEX®2-73 and trial measurements. These measures obtained from all patients who participated in the entire 57 weeks include exploratory cognitive and functional scores as well as biomarker signals of brain activity. Additionally, the clinical trial appeared to show that ANAVEX®2-73 activity was enhanced by its active metabolite (ANAVEX19-144), which also targets the SIGMAR1 receptor and has a half-life approximately twice as long as the parent molecule.
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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. 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.
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In July 2018, we presented the results of a genomic DNA and RNA evaluation of the participants in the Phase 2a clinical trial. More than 33,000 genes were analyzed using unbiased, data driven, machine learning, artificial intelligence (“AI”) system for analyzing DNA and RNA data in patients treated with ANAVEX®2-73. 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. The results from this evaluation supported the continued evaluation of genomic information in subsequent clinical trials, since these signatures can now be applied to neurological indications tested in future clinical trials with ANAVEX®2-73 including Alzheimer’s disease, Parkinson’s disease dementia and Rett syndrome.
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ANAVEX®2-73 data met prerequisite information in order to progress into a Phase 2b/3 placebo-controlled trial. This larger Phase 2b/3 double-blind, placebo-controlled trial of ANAVEX®2-73 in early Alzheimer’s disease commenced in August 2018. 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. Primary and secondary endpoints to assess safety and both cognitive and functional efficacy, were measured through the Alzheimer’s Disease Assessment Scale – Cognitive Subscale test (“ADAS-Cog”), Alzheimer’s Disease Cooperative Study – Activities of Daily Living (“ADCS-ADL”) and Clinical Dementia Rating – Sum of Boxes for cognition and function (“CDR-SB”). In addition to these endpoints, the ANAVEX®2-73 Phase 2b/3 trial design incorporated pre-specified statistical analyses related to potential genomic precision medicine biomarkers previously identified in the ANAVEX®2-73 Phase 2a clinical trial. The trial was completed in mid-2022 and, in December 2022, the Company presented topline results from the Phase 2b/3 clinical trial. All statistical analyses were performed by outside consultancy companies.
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Furthermore, all pre-specified clinical endpoints were analyzed using a mixed model for repeated measures (MMRM). Under the multiplicity control rule, a trial is successful in meeting the co-primary endpoints if the significance of each endpoint is P < 0.05, or if the significance of only one co-primary endpoint is P < 0.025. If only one primary endpoint is significant at an α level of 0.025, then the secondary endpoint will be evaluated at the same level of 0.025. The trial was successful, the differences in the least-squares mean (LSM) change from baseline to 48 weeks between the ANAVEX®2-73 and placebo groups for ADAS-Cog13 was significant at a level of P < 0.025 and for CDR-SB was significant at a level of P < 0.025, in the patients with early Alzheimer’s disease.
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The comparison of individual dose groups vs placebo also supports blarcamesine’s efficacy. For the primary endpoint ADAS-Cog13, blarcamesine is significantly better than placebo (−2.027; P = 0.0079) as well as for both the 50 mg (−2.149; P = 0.021) and the 30 mg (−1.934; P = 0.026) blarcamesine dosage groups at Week 48, representing that blarcamesine slowed clinical progression at 48 weeks by 36.3% and by 38.5% and 34.6% in 50 mg and 30 mg groups vs. placebo, respectively. The functional co-primary endpoint, ADCS-ADL,
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was trending in a positive direction but did not reach significance at Week 48. The key secondary endpoint CDR-SB was significantly improved vs. placebo (−0.483, P = 0.0104) as well as in both 50 mg (−0.465; P = 0.045) and 30 mg (−0.502; P = 0.020) groups at Week 48. Clinical Global Impression – Improvement (“CGI-I”) was also significantly improved vs. placebo (−0.278, P = 0.004) as well as in both the 50 mg (−0.314; P = 0.008) and the 30 mg (−0.248; P = 0.024) groups at Week 48. The findings are supported by biomarkers, including plasma Aβ42/40-ratio and reduction of brain atrophy. Blarcamesine significantly slowed brain atrophy in key regions of interest, including the whole brain by 37.6%, total grey matter by 63.5%, and lateral ventricles by 25.1%.
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In the respective safety population, common treatment-emergent adverse events included dizziness, which was transient and mostly mild to moderate in severity, and occurred in 120 participants (35.8%) during titration and in 76 participants (25.2%) during maintenance with ANAVEX®2-73 and 10 (6.0%) during titration and 9 (5.6%) during maintenance with placebo.
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A long-term open label extension study of ANAVEX®2-73, referred to as the ATTENTION-AD trial, was initiated for patients who completed the 48-week Phase 2b/3 placebo-controlled trial referenced above. This trial extension for a duration of up to 96/144 additional weeks was completed in June 2024. The trial extension demonstrated that blarcamesine-treated patients continued to accrue benefit through up to 192 weeks, as measured by the clinical endpoints ADAS-Cog13 (-3.83; P = 0.0165) and ADCS-ADL (+4.30, P = 0.0206). No new safety findings were observed with continued blarcamesine treatment over three years, confirming good comparative safety profile and no associated neuroimaging adverse events. Delayed-start analysis of treatment with oral blarcamesine was significant, reflecting the importance of early treatment initiation.
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Additional precision medicine population 48-week data demonstrates cognitive stabilization in early Alzheimer’s disease. Cognitive outcomes observed in the oral blarcamesine 30 mg precision medicine cohort move toward normal aging profiles across validated clinical scales. For ADAS-Cog13, blarcamesine showed a 48-week change from baseline of 0.853 compared to ~1 point typical annual decline in prodromal (pre-dementia) aging adults. For CDR-SB, blarcamesine demonstrated a change from baseline of 0.465, aligning with the 0-0.5 point annual range seen in prodromal aging. These data are similar to referenced barely detectable prodromal Alzheimer’s disease decline, in spite of the more advanced stage of Alzheimer’s disease impairment at baseline of the blarcamesine population.
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Further posthoc analysis demonstrates continued long-term benefit from oral blarcamesine compared to decline observed in the Alzheimer’s Disease Neuroimaging Initiative (ADNI) control group. Externally matched control participants from the ADNI database were compared with participants over the 144-week period of ANAVEXÒ 2-73-AD-004 and its ATTENTION-AD (ANAVEXÒ 2-73-AD-EP-004) open-label extension (OLE) Phase IIb/III trial.[1] For ADAS-Cog13, total score ranges from 0 to 85 with higher scores indicating increased cognitive impairment.
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In the intent-to-treat (ITT) population, significantly less cognitive decline was observed for the blarcamesine participants compared to the ADNI control group at 48 weeks with a significant, and clinically meaningful difference in mean change from baseline ADAS-Cog13 total score of −2.68 points (p < 0.0001).[2]
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[1] Observed raw data was used. Scheduled visits were [OLE Week 0 = Combined Week 48], [OLE Week 48 = Combined Week 96], [OLE Week 96 = Combined Week 144]; Combined = DB (double-blind) + OLE (open-label-extension) trials. Up to 144 weeks ADNI control group data was available.
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[2] ADAS-Cog13 scores LS mean difference between the treatment groups being larger than 2 points are considered clinically meaningful improvements: Muir RT, Hill MD, Black SE, Smith EE. Minimal clinically important difference in Alzheimer's disease: Rapid review. Alzheimers Dement. 2024;20(5):3352-3363.
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Over the course of the open-label extension study at time point 96 weeks, these two groups diverged sharply, with statistically significant differences in mean change in ADAS-Cog13 total score at 96 weeks of −6.41 points (p < 0.0001). The difference between groups continues to increase at 144 weeks (ADAS-Cog13 total score difference of −12.78 points;p < 0.0001).
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The results provide evidence of the significant beneficial therapeutic effect of blarcamesine, which positively separates from the ADNI control group with duration of treatment.
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Parkinson’s Disease
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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. The study was funded by the Michael J. Fox Foundation. Additional data announced in October 2017 indicated that ANAVEX®2-73 induced robust neurorestoration in experimental Parkinsonism. We believe the encouraging results we have gathered in this preclinical model, coupled with the favorable profile of this product candidate in the Alzheimer’s disease trial, support the notion that ANAVEX®2-73 has the potential to treat Parkinson’s disease dementia.
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In October 2020, we completed a double-blind, randomized, placebo-controlled proof-of-concept Phase 2 trial with ANAVEX®2-73 in Parkinson’s disease dementia in Spain and Australia, to study the effect of the compound on both the cognitive and motor impairment of Parkinson’s disease. 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.
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The trial demonstrated that ANAVEX®2-73 was safe and well tolerated in oral doses up to 50 mg once daily. The results showed clinically meaningful, dose-dependent, and statistically significant improvements in the Cognitive Drug Research (“CDR”) computerized assessment system analysis. Treatment with ANAVEX®2-73 also resulted in clinically meaningful improvements as measured by the global composite score of Parkinson’s disease symptom severity, MDS-Unified Parkinson’s Disease Rating Scale (“MDS-UPDRS”) total score on top of standard of care including dopaminergic therapy, levodopa and other anti-PD medications after 14 weeks of treatment, suggesting ANAVEX®2-73’s potential capability of slowing and reversing symptoms that progress in Parkinson’s disease. In addition, the trial confirmed the precision medicine approach of targeting SIGMAR1 as a genetic biomarker in response to ANAVEX®2-73 may result in improved clinical outcomes.
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A 48-week OLE ANAVEX2-73-PDD-EP-001 Phase 2 trial was offered to participants after completion of the double-blind placebo-controlled ANAVEX2-73-PDD-001 Phase 2 trial discussed above. The OLE trial assessed safety, tolerability and efficacy, measuring among others, MDS-Unified Parkinson’s Disease Rating Scale Parts I, II, III, REM Sleep Behavior Disorder Screening Questionnaire (RBDSQ), CGI-I, as well as cognitive efficacy endpoint Montreal Cognitive Assessment (MoCA) over a 48-week period.
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In March 2023, we reported the preliminary ANAVEX2-73-PDD-EP-001 OLE trial data, which demonstrated longitudinal beneficial effects of ANAVEX®2-73 on the pre-specified primary and secondary objectives. Preliminary analysis reveals that ANAVEX®2-73 was found to be generally safe and well tolerated, and safety findings in this trial were consistent with the known safety profile of ANAVEX®2-73. In respect to efficacy, across all efficacy endpoints, patients performed better while on ANAVEX®2-73. While all patients were on drug holiday due to COVID-19 between the DB EOT and the OLE Baseline, the respective efficacy endpoints, including the MDS-UPDRS Part II + III and CGI-I, measured at the end of trial of the double-blind study (DB EOT) and the OLE Baseline, were worsening, as expected in a progressive disease like Parkinson’s. However, when patients resumed daily oral ANAVEX®2-73 treatment, a consistent improvement was observed during the extension phase from OLE Baseline through OLE Week 24, and OLE Week 48, respectively. These results are consistent with the pattern observed for all efficacy measures in the extension phase.
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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.
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Also with respect to Parkinson’s disease, in January 2021, we were awarded a research grant of $1.0 million from The Michael J. Fox Foundation for Parkinson’s Research to explore utilization of PET imaging biomarkers to enable measurement of target engagement and pathway activation of the SIGMAR1 with clinically relevant doses including in people with Parkinson’s disease. This study is currently in the planning stage.
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Rett Syndrome
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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. The study was funded by the International Rett Syndrome Foundation.
Previous report
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 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. The FDA Fast Track program is designed to facilitate and expedite the development and review of new drugs to address unmet medical needs in the treatment of serious and life-threatening conditions. An earlier application for a proposed Rett syndrome study in the United States resulted in the FDA requesting additional information. The resulting clinical hold and subsequent partial clinical hold have since been removed after the Company satisfactorily provided the additional information requested. The following is a summary of clinical trials conducted by the Company in Rett syndrome. The first Phase 2 trial, (ANAVEX®2-73-RS-001), took place in the United States, and was completed in December 2020. This trial was a randomized double-blind, placebo-controlled safety, tolerability, PK and efficacy trial of oral liquid ANAVEX®2-73 formulation in 25 adult female patients with Rett syndrome over a 7-week treatment period including ANAVEX®2-73-specific genomic precision medicine biomarkers. The primary endpoint of the trial was safety. The dosing of 5 mg ANAVEX®2-73 was well-tolerated and demonstrated dose-proportional PK. All secondary efficacy endpoints of the trial showed statistically significant and clinically meaningful response in the Rett Syndrome Behaviour Questionnaire (“RSBQ”) response, when compared to placebo, in the intent to treat (“ITT”) cohort (all participants, p = 0.011). 66.7% of ANAVEX®2-73 treated subjects showed a statistically significant improvement in RSBQ response as compared to 10% of the subjects on placebo in the ITT cohort (all participants, p = 0.011). 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.
Previous report
This clinical trial was funded, in part, by a financial grant from the International Rett Syndrome Foundation of $0.6 million. No other clinical trials with ANAVEX®2-73 related to Rett syndrome have been conducted in the United States.
Previous report
The second, international trial of ANAVEX®2-73 for the treatment of Rett syndrome, called the AVATAR trial, commenced in June 2019. This trial took place in Australia and the United Kingdom using a higher dose than the U.S. based Phase 2 trial for Rett syndrome. The trial was a Phase 3 randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of ANAVEX®2-73 in 33 adult patients over a 7-week treatment period including ANAVEX®2-73 specific precision medicine biomarkers. Based upon the input from the successful U.S. Phase 2 Rett syndrome trial (ANAVEX®2-73-RS-001), we updated the endpoints for the AVATAR trial (ANAVEX®2-73-RS-002) to appropriately assess the clinically meaningful outcome following International Conference on Harmonization (ICH) guidelines. These updates were approved by the respective regulatory authorities in the U.K. and in Australia, respectively, where the AVATAR trial was conducted.
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Reworded paragraphs (35)
Previous wording
We have based these forward-looking statements largely on our current expectations and projections about future events, including the responses we expect from the FDA, EMA and other regulatory authorities and financial trends that we believe may affect our financial condition, results of operations, business strategy, preclinical studies and clinical trials, and financial needs. These forward-looking statements are subject to a number of risks, uncertainties and assumptions including without limitation the risks described in “Risk Factors” in Part I, Item 1A of our Annual Report on Form 10-K filed with the Securities and Exchange Commission on November 25, 2025. These risks are not exhaustive. Other sections of this Quarterly Report on Form 10-Q include additional factors which could adversely impact our business and financial performance. Moreover, we operate in a very competitive and rapidly changing environment. New risk factors emerge from time to time, and it is not possible for our management to predict all risk factors, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. We cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable laws including the securities laws of the United States, we assume no obligation to update or supplement forward-looking statements.
Current wording
These risks are not exhaustive. Other sections of this Quarterly Report on Form 10-Q include additional factors which could adversely impact our business and financial performance. Moreover, we operate in a very competitive and rapidly changing environment. New risk factors emerge from time to time, and it is not possible for our management to predict all risk factors, nor can we assess the impact of all factors on our business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. We cannot assure you that the events and circumstances reflected in the forward-looking statements will be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable laws including the securities laws of the U.S., we assume no obligation to update or supplement forward-looking statements.
Previous wording
The SIGMAR1 gene encodes the SIGMAR1 protein, which is an intracellular chaperone protein with important roles in cellular communication. SIGMAR1 is also involved in transcriptional regulation at the nuclear envelope and restores homeostasis and stimulates recovery of cell function when activated. In order to validate the ability of our compounds to activate quantitatively the SIGMAR1, we performed, in collaboration with Stanford University, a quantitative Positron Emission Tomography (PET) imaging scan in mice, which demonstrated a dose-dependent ANAVEX®2-73 (blarcamesine) target engagement or receptor occupancy with SIGMAR1 in the brain.
Current wording
We do not have any products approved for commercial sale and have not generated any revenue to date. We have a portfolio of compounds in various stages of development targeting different sigma-1 receptor (“SIGMAR1”) binding activities. The SIGMAR1 gene encodes the SIGMAR1 protein, which is an intracellular chaperone protein with important roles in cellular communication. SIGMAR1 is also involved in transcriptional regulation at the nuclear envelope and restores homeostasis and stimulates recovery of cell function when activated. SIGMAR1 may be a target for therapeutics to combat many human diseases, both of a neurodegenerative nature, including AD, as well as of a neurodevelopmental nature, like Rett syndrome and Fragile X syndrome. When bound by the appropriate ligands, we believe SIGMAR1 influences the functioning of multiple biochemical signals that are involved in the pathogenesis (origin or development) of disease.
Previous wording
We continue to identify and initiate discussions with potential strategic and commercial partners to most effectively advance our programs and increase stockholder value. Further, we may acquire or develop new intellectual property and assign, license, or otherwise transfer our intellectual property to further our goals.
Current wording
We will seek to identify potential strategic and commercial partners to most effectively advance our programs and increase shareholder value. Further, we may acquire or develop new intellectual property and assign, license, or otherwise transfer our intellectual property to further our business strategy.
Previous wording
We believe ANAVEX®2-73 may offer a disease-modifying approach in neurodegenerative and neurodevelopmental diseases by activation of SIGMAR1. 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.
Current wording
We believe ANAVEX 2-73 may offer a disease-modifying approach in neurodegenerative and neurodevelopmental diseases by activation of SIGMAR1. ANAVEX 2-73 is being developed as an oral once-daily capsule formulation for early AD, and in an oral liquid formulation for Rett syndrome and Fragile X syndrome.
Previous wording
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. The MAA, if approved, would allow direct market access throughout the European Union for oral ANAVEX®2-73 (blarcamesine) for the treatment of Alzheimer’s disease. A company seeking to market a new pharmaceutical product through the centralized procedure must file safety data and efficacy data as part of the MAA. After the EMA evaluates the MAA, it provides a recommendation to the European Commission (“EC”) and the EC then approves or denies the MAA. On December 12, 2025, we announced that the Committee for Medicinal Products for Human Use (“CHMP”) of the EMA rendered a negative trend-vote following an oral examination of our MAA. On December 18, 2025 we announced that we have requested a re-examination of the MAA. The re-examination procedure is led by a different rapporteur and co-rapporteur, who will conduct a new evaluation of our MAA for blarcamesine. We have also requested that the EMA consult a Scientific Advisory Group as part of this process to provide an independent recommendation.
Current wording
In November 2024, we announced the submission of a Marketing Authorisation Application (“MAA”) to the EMA, under the centralized procedure, for ANAVEX 2-73 for the treatment of AD. In December 2025, we announced that the Committee for Medicinal Products for Human Use (“CHMP”) of the EMA rendered a negative opinion on the MAA and that we had requested a re-examination of the opinion. In March 2026, before the re-examination was complete, we announced that the MAA had been withdrawn following feedback from the CHMP indicating that the MAA was not currently approvable.
Previous wording
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®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. ANAVEX®3-71 indicates extensive therapeutic advantages in Alzheimer’s and other protein-aggregation-related diseases given its ability to enhance neuroprotection and cognition via SIGMAR1 activation and M1 muscarinic allosteric modulation.
Current wording
ANAVEX 3-71 is an orally administered clinical drug candidate with a novel mechanism of action through SIGMAR1 activation and M1 muscarinic allosteric modulation. ANAVEX 3-71 has been studied as a CNS-penetrable potential disease modifying treatment for cognitive impairments.
Previous wording
Preclinical data presented also indicates that ANAVEX®1-41 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.
Current wording
ANAVEX 1-41 is a sigma-1 agonist and a selective allosteric M1 muscarinic agonist believed to have 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 diseases. Development work on ANAVEX 1-41 has been paused and further development work will be contingent on additional funding for ANAVEX 1-41 or the signing of a strategic partnership. It is also possible that we may license or sell ANAVEX 1-41.
Previous wording
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.
Current wording
We hold ownership or exclusive rights to thirty-three (33) issued U.S. patents, seventeen (17) pending U.S. patent applications, and numerous Patent Cooperation Treaty (PCT) and ex-U.S. patents and patent applications relating to our drug candidates, methods associated therewith, and to our research programs.
Previous wording
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. For more information regarding our patents, patent applications, and challenges to our existing or future patents, see “Risk Factors” in Part I, Item 1A of our Annual Report on Form 10-K filed with the Securities and Exchange Commission on November 25, 2025.
Current wording
Our intellectual property position, like that of many biomedical companies, is uncertain and involves complex legal and technical questions. For more information regarding our patents, patent applications, and challenges to our existing or future patents, see “Risk Factors” in Part I, Item 1A of our Annual Report on Form 10-K filed with the SEC on November 25, 2025.
Previous wording
The following discussion should be read in conjunction with our condensed consolidated interim financial statements and related notes thereto contained elsewhere in this report. Past operating results are not necessarily indicative of results that may occur in future periods. The discussion contains forward-looking statements, which involve a number of risks and uncertainties. See “Forward Looking Statements” included elsewhere in this report.
Current wording
The following discussion should be read in conjunction with our condensed consolidated interim financial statements and related notes thereto contained elsewhere in this report. Past operating results are not necessarily indicative of results that may occur in future periods. The following discussion contains forward-looking statements, which involve a number of risks and uncertainties. See “Forward-Looking Statements” included elsewhere in this Quarterly Report on Form 10-Q.
Previous wording
We are in the development stage and have not earned any revenue since our inception in 2004. 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.
Current wording
We are in the development stage and have not earned any revenue since our inception. We do not anticipate earning any revenue until one or more of our product candidates is approved and commercial sales commence or we can establish one or more partnerships to develop, co-develop, license, acquire or market one or more product candidates, if approved.
Previous wording
General and administrative expenses consist of personnel costs, expenses for outside professional services and expenses associated with operating as a public company. Personnel costs consist of salaries and wages, benefits and share-based compensation for general and administrative personnel. Outside professional services and public company expenses include expenses related to compliance and reporting, additional insurance expenses, audit and SOX compliance, expenses associated with patent research, applications and filings, investor and stockholder relations activities and other administrative expenses and professional services.
Current wording
General and administrative expenses consist of personnel costs, expenses for outside professional services and expenses associated with operating as a public company. Personnel costs consist of salaries and wages, benefits and share-based compensation for general and administrative personnel. Outside professional services and public company expenses include expenses related to compliance and reporting, additional insurance expenses, audit and Sarbanes-Oxley Act of 2002 compliance, expenses associated with patent research, applications and filings, investor and shareholder relations activities and other administrative expenses and professional services. Subsequent to the end of the quarterly period ended March 31, 2026, we incurred increased legal and other professional expenses arising out of the review by the special committee (“Special Committee”) of our Board of Directors (“Board”), which resulted in the termination of our former CEO, and related matters, and we anticipate incurring higher legal and other professional expenses in future quarters in connection with these matters.
Previous wording
Total operating expenses for the three months ended December 31, 2025 were $6.8 million, compared to $13.6 million for the comparable three months ended December 31, 2024.
Current wording
Total operating expenses for the three months ended March 31, 2026 were $6.5 million, compared to $12.5 million for the comparable three months ended March 31, 2025. The primary reason for the decrease in operating expenses is due to the decrease in research and development expenses, which is more fully described below.
Previous wording
During the three-months ended December 31, 2025 and 2024, external service provider costs by product candidate was as follows (in thousands):
Current wording
During the three months ended March 31, 2026 and 2025, external service provider costs by indication and product candidate were as follows (in thousands):
Previous wording
Net other income for the three-months ended December 31, 2025 was $1.1 million, as compared to $1.5 million for the comparable three-months ended December 31, 2024. The decrease is primarily related to (i) a decrease in research and development incentive income of $0.4 million, as a result of the completion of our clinical trials in Australia and (ii) a decrease in interest income of $0.3 million as a result of decreased market wide interest rates. This was partially offset by a decrease in foreign exchange loss as a result of the stabilization of the Australian dollar against the US dollar, and the reduced incentive and tax receivable balance denominated in Australian dollars, as compared to the prior year period.
Current wording
Net other income for the three months ended March 31, 2026 was $1.1 million, as compared to $1.3 million for the three months ended March 31, 2025. The decrease is primarily related to a decrease in interest income of $0.2 million as a result of lower interest rates during the period.
Previous wording
Net loss for the three-months ended December 31, 2025, was $5.7 million, or $0.06 per share, as compared to $12.1 million, or $0.14 per share in the comparative quarter of fiscal 2024.
Current wording
Net loss for the three months ended March 31, 2026, was $5.3 million, or $0.06 per share, as compared to $11.2 million, or $0.13 per share, for the three months ended March 31, 2025. The decrease is primarily related to a decrease in research and development expenses, as more fully described above.
Previous wording
General and administrative expenses were $2.1 million for the three months ended December 31, 2025, as compared to $3.1 million for the same quarter of fiscal 2024. The decrease was primarily related to a decrease in legal and professional fees of approximately $0.3 million, as well as a decrease in personnel costs of $0.6 million, mostly related to a decrease in stock based compensation expense of $0.3 million as a result of the vesting of previously issued milestone options, and due to the forfeiture of options, and a reduction in accrued bonus compensation for existing staff of $0.3 million.
Current wording
General and administrative expenses were $4.4 million for the six months ended March 31, 2026, as compared to $5.8 million for the same period of fiscal 2025. The decrease was primarily related to a decrease in legal and professional fees of approximately $0.7 million, as well as a decrease in stock-based compensation expense of $0.4 million as a result of the vesting of previously issued milestone options, and due to the forfeiture of options by a deceased director, as well as $0.1 million related to a reduction in accrued cash bonus compensation for existing staff.
Previous wording
On December 31, 2025, we had net current assets of $126.6 million, an increase of approximately $31.2 million from our year ended September 30, 2025. The increase in net current assets is primarily related to cash received from the issuance of common shares pursuant to the 2025 Sales Agreement.
Current wording
On March 31, 2026, we had net current assets of $121.7 million, an increase of approximately $26.8 million from our fiscal year ended September 30, 2025. The increase in net current assets is primarily related to cash received from the issuance of common stock pursuant to the 2025 Sales Agreement (as defined below).
Previous wording
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 the work necessary to prepare for future development of our pipeline compounds.
Current wording
We intend to continue to use our capital resources to advance our clinical development of ANAVEX 2-73.
Previous wording
The following table summarizes cash flows during the three months ended December 31, 2025 and 2024 (in thousands):
Current wording
The following table summarizes cash flows during the six months ended March 31, 2026 and 2025 (in thousands):
Previous wording
Net cash used in operating activities for the three-months ended December 31, 2025 was $7.2 million, compared to $12.1 million during the comparable period ended December 31, 2024. The principal reason for this change is due to the decrease in net loss due to the decrease in operating expenditures, as described above.
Current wording
Net cash used in operating activities for the six months ended March 31, 2026 was $11.6 million, compared to $18.0 million during the comparable period ended March 31, 2025. The principal reason for this change is due to the decrease in net loss due to the decrease in operating expenditures, as described above.
Previous wording
Cash flows provided by financing activities for the three-month period ended December 31, 2025 was $36.3 million, compared to $0.7 million during the comparable three-month period ended December 31, 2024.
Current wording
Cash flows provided by financing activities for the six month period ended March 31, 2026, was $36.4 million, compared to $1.6 million during the comparable six-month period ended March 31, 2025.
Previous wording
During the three months ended December 31, 2025, cash provided by financing activities was related to cash received from the issuance of common shares pursuant to the 2025 Sales Agreement.
Current wording
During the six months ended March 31, 2026, cash provided by financing activities was related to cash received from the issuance of common stock pursuant to the 2025 Sales Agreement.
Previous wording
During the three months ended December 31, 2024, cash provided by financing activities was primarily attributable to cash received from the exercise of stock options by our employees.
Current wording
During the six months ended March 31, 2025, cash provided by financing activities was primarily attributable to cash received from the exercise of stock options by our employees.
Previous wording
On July 25, 2025, we entered into a Sales Agreement (the “2025 Sales Agreement”) with TD Securities (USA) LLC (the “Sales Agent”). Pursuant to the 2025 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.
Current wording
On July 25, 2025, we entered into a Sales Agreement (the “2025 Sales Agreement”) with TD Securities (USA) LLC (the “Sales Agent”). Pursuant to the 2025 Sales Agreement, we may offer and sell up to an aggregate offering price of $150 million (the “Offering”) in shares of our common stock (“Common Stock”) from time to time through the Sales Agent.
Previous wording
Upon delivery of a placement notice based on our instructions and subject to the terms and conditions of the 2025 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. We are not obligated to make any sales of shares under the 2025 Sales Agreement. We or the Sales Agent may suspend or terminate the Offering upon notice to the other party, subject to certain conditions. 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.
Current wording
Upon delivery of a placement notice based on our instructions and subject to the terms and conditions of the 2025 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. We are not obligated to make any sales of shares of Common Stock under the 2025 Sales Agreement. We or the Sales Agent may suspend or terminate the Offering upon notice to the other party, subject to certain conditions. 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.
Previous wording
During the three months ended December 31, 2025, the Company issued an aggregate of 6,003,237 shares of Common Stock under the 2025 Sales Agreement for net proceeds of $36.3 million, after deducting commissions.
Current wording
During the six months ended March 31, 2026, we issued an aggregate of 6,026,237 shares of Common Stock under the 2025 Sales Agreement for net proceeds of $37.2 million, after deducting commissions. We suspended sales under the 2025 Sales Agreement following the formation of the Special Committee described above under “—Financial Overview.” We will be unable to make sales under the 2025 Sales Agreement after we file our next Annual Report on Form 10-K due to our loss of Form S-3 eligibility for the untimely filing of our Quarterly Reports on Form 10-Q for the quarters ended March 31, 2026 and June 30, 2026 with the SEC. We will be unable to regain eligibility to use Form S-3 until we have timely filed all reports specified under Form S-3 for the preceding 12 calendar months.
Previous wording
At December 31, 2025, there was an unused amount of $103.3 million under the 2025 Sales Agreement.
Current wording
At March 31, 2026, there was an unused amount of $103.2 million under the 2025 Sales Agreement.
Previous wording
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 had the right to sell and issue to Lincoln Park, and Lincoln Park was 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.
Current wording
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 had the right to sell and issue to Lincoln Park, and Lincoln Park was obligated to purchase, up to $150.0 million in value of shares of our Common Stock from time to time over a three-year period.
Previous wording
In consideration for entering into the 2023 Purchase Agreement, the Company issued to Lincoln Park 75,000 shares of Common Stock as a commitment fee (the “initial commitment shares”) during the year ended September 30, 2023 and agreed to issue up to 75,000 shares pro rata (collectively with the initial commitment shares, the “commitment shares”), when and if, Lincoln Park purchased, at the Company’s discretion, the $150.0 million aggregate commitment.
Current wording
In consideration for entering into the 2023 Purchase Agreement, we issued to Lincoln Park 75,000 shares of Common Stock as a commitment fee during the fiscal year ended September 30, 2023 and agreed to issue up to 75,000 shares of Common Stock pro rata, when and if, Lincoln Park purchased, at the our discretion, the $150.0 million aggregate commitment.
Previous wording
During the three-month period ended December 31, 2025 and 2024, the Company did not issue any shares of common stock under the 2023 Purchase Agreement. On December 31, 2025, there was an unused amount of $110.8 million under the 2023 Purchase Agreement. The 2023 Purchase Agreement expired on February 3, 2026.
Current wording
During the six-month period ended March 31, 2026 and 2025, we did not issue any shares of Common Stock under the 2023 Purchase Agreement. The 2023 Purchase Agreement expired on February 3, 2026.
Previous wording
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.
Current wording
We have no off-balance sheet arrangements that have or are reasonably likely to have a current or future effect on our financial condition, revenues or expenses, results of operations, liquidity, capital expenditures or capital resources that are material to our stockholders.
Previous wording
We prepare our condensed consolidated interim financial statements in accordance with accounting principles generally accepted in the United States of America and make estimates and assumptions that affect our reported amounts of assets, liabilities, revenue and expenses, and the related disclosures of contingent liabilities. We base our estimates on historical experience and other assumptions that we believe are reasonable in the circumstances. Actual results may differ from these estimates.
Current wording
We prepare our condensed consolidated interim financial statements in accordance with accounting principles generally accepted in the U.S. and make estimates and assumptions that affect our reported amounts of assets, liabilities, revenue and expenses, and the related disclosures of contingent liabilities. We base our estimates on historical experience and other assumptions that we believe are reasonable in the circumstances. Actual results may differ from these estimates.
Previous wording
There have been no significant changes in the critical accounting policies and estimates described in our Annual Report on Form 10-K for the year ended September 30, 2025, as filed with the SEC on November 25, 2025.
Current wording
There have been no significant changes in the critical accounting policies and estimates described in our Annual Report on Form 10-K for the fiscal year ended September 30, 2025, as filed with the SEC on November 25, 2025.
Previous wording
Please refer to Note 2 “Recent Accounting Pronouncements” in notes to our Condensed Consolidated Interim Financial Statements included in this Form 10-Q.
Current wording
Please refer to Note 2 “Recent Accounting Pronouncements” in notes to our Condensed Consolidated Interim Financial Statements included in this Quarterly Report on Form 10-Q.
Rearranged current paragraphs (5)
Current report — wording carried from elsewhere
We have based these forward-looking statements largely on our current expectations and projections about future events, including the responses we expect from the FDA, the European Medicines Agency (“EMA”) and other regulatory authorities and financial trends that we believe may affect our financial condition, results of operations, business strategy, preclinical studies and clinical trials, and financial needs. These forward-looking statements are subject to a number of risks, uncertainties and assumptions including without limitation:
Current report — wording carried from elsewhere
General and administrative expenses were $2.3 million for the three months ended March 31, 2026 as compared to $2.6 million for the same quarter of fiscal 2025. The decrease was primarily related to a decrease in legal and professional fees of approximately $0.4 million.
Current report — wording carried from elsewhere
The following table summarizes our research and development expenses for the six months ended March 31, 2026 and 2025 (in thousands):
Current report — wording carried from elsewhere
During the six months ended March 31, 2026 and 2025, external service provider costs by product candidate were as follows (in thousands):
Current report — wording carried from elsewhere
The decrease in research and development expenses during the six-month period is primarily related to the following: