Anavex Life Sciences (NASDAQ:AVXL) executives used the company’s fiscal 2026 first-quarter earnings call to outline regulatory next steps for oral blarcamesine in early Alzheimer’s disease and to review a lower quarterly cash burn and operating expense base following the completion of prior-year manufacturing and certain clinical activities.
Regulatory and development update for blarcamesine
Chief Executive Officer Dr. Christopher Missling said Anavex remains focused on advancing its clinical pipeline, with oral blarcamesine positioned as the lead program in early Alzheimer’s disease. Missling said the company is “excited about the therapeutic potential” of the drug and intends to work with regulators in Europe and the U.S. to advance it as a potential treatment option.
Missling did not identify the new rapporteur and co-rapporteur, stating only that two countries among the 27 EU member states serve in those roles.
EMA re-examination: what Anavex said it will include
During the Q&A, Missling said the re-examination package will seek to address criteria related to conditional approval, including seriousness of the disease, unmet need, clinically meaningful effects, mechanistic rationale (including genetic variants), and translational support, as well as the sponsor’s commitment to confirmatory study execution.
He said Anavex is including multiple data components in the re-examination, including:
- Data from the Phase IIB/III ANAVEX2-73-AD-004 study
- Data from the open-label extension (described as an “open-label study”)
- Data related to the Aβ-Clear study population
- Correlation between clinical efficacy and reduced brain atrophy
Missling characterized the re-examination as, in part, a question of how to “repackage or re-articulate the strength of the package or of the data,” while also noting the company cannot guarantee an approval outcome.
FDA discussions and timing
In the U.S., Missling referenced feedback from a January FDA Type C meeting. He said the meeting covered potential pathways to support blarcamesine for Alzheimer’s disease and that “existing data” from the ANAVEX2-73-AD-004 program is expected to be submitted to the FDA as part of moving forward.
When asked about timing for a formal NDA submission, Missling said the company plans to advance its regulatory plan “once we are getting closer,” adding that the Type C meeting was “very productive.” He also said the FDA has meeting-request and scheduling requirements, and described the submission as being coordinated with a meeting request rather than simply sending data and receiving feedback.
Clinical interpretation and endpoints discussed on the call
Several questions focused on the CHMP’s stated rationale and Anavex’s view of the AD-004 results in genetic subgroups. Missling said the company would not criticize regulators, but reiterated Anavex’s interpretation that the trial met ADAS-Cog13, with greater significance in the SIGMAR1 wild-type population, and that CDR-SB was also superior in the wild-type group compared with the intent-to-treat population.
He said the ADCS-ADL endpoint was “the only one which was not significant,” though trending positively, and argued that the scale is not sensitive enough to detect changes in activities of daily living over 48 weeks in an early Alzheimer’s population. He also said that in the company’s described Aβ-Clear 3 population—referencing SIGMAR1 wild-type carriers with the COL24A1 wild-type gene—significance was reached “across the board” on ADAS-Cog13, ADCS-ADL, and CDR-SB, with what he called clinically meaningful effect sizes.
Missling added that Anavex requested involvement of the EMA’s Scientific Advisory Group (SAG) in the ongoing review process. He said the company will update the public once the process concludes and would not comment further while the re-examination is ongoing.
Pipeline activity and upcoming presentations
Missling highlighted Anavex’s participation as an industry partner in ACCESS-AD, a European Commission Innovative Health Initiative-funded program intended to accelerate adoption of diagnostic and therapeutic approaches for Alzheimer’s in real-world settings. He said blarcamesine will be evaluated in a placebo-controlled clinical prediction study within the program, including biomarker review (including autophagy signals) and efficacy assessments, and that Anavex plans to use the trial as part of its regulatory package to confirm efficacy in early Alzheimer’s disease. He indicated the target population is currently early Alzheimer’s disease, though it “could end up being a preventative also.” He confirmed the study corresponds to “AD006” on the company’s pipeline chart.
Regarding clinical activity underway, Missling said the only ongoing trial is a compassionate use program for Rett syndrome in Canada, the U.K., and Australia, along with compassionate use in Alzheimer’s disease. He said the company is planning studies in Parkinson’s disease, Fragile X syndrome, and another undisclosed indication, and also said it plans to continue a schizophrenia program. He clarified that a Parkinson’s disease trial has not yet started, distinguishing it from prior work in Parkinson’s disease dementia that he described as a basis for the planned Parkinson’s trial.
Missling also pointed to upcoming scientific communications, including an oral presentation at a March conference at Johns Hopkins University on findings that he said relate to biomarker relationships, correlations between clinical endpoints, and reduced brain-region atrophy with blarcamesine in early Alzheimer’s disease. He also cited planned publications on precision medicine patient populations from the AD-004 trial (including Aβ-Clear), a publication focused on the COL24A1 gene, and a Fragile X-related publication in a mouse model. In addition, he said ANAVEX3-71 is expected to be advanced toward pivotal clinical studies for schizophrenia-related disorders.
Financial results: cash runway and lower operating expenses
Principal Financial Officer Sandra Boenisch reported that Anavex ended the quarter with $131.7 million in cash at December 31 and no debt. The company used $7.1 million in cash and cash equivalents in operating activities during the quarter, after changes in non-cash working capital accounts. Boenisch said Anavex expects its cash runway to be “more than three years” at the current cash utilization rate.
Research and development expense was $4.7 million, down from $10.4 million in the comparable quarter of the prior year. General and administrative expense was $2.1 million, compared with $3.1 million a year earlier. Boenisch said the decrease in operating expenses was mainly due to the completion of a large blarcamesine manufacturing campaign in fiscal 2025 and lower clinical trial activity following completion of the ANAVEX3-71 Phase II study in schizophrenia.
Anavex reported a net loss of $5.7 million for the quarter, or $0.06 per share.
About Anavex Life Sciences (NASDAQ:AVXL)
Anavex Life Sciences Corp is a clinical‐stage biopharmaceutical company focused on the development of novel therapeutics for central nervous system (CNS) disorders. The company applies a proprietary drug discovery platform that targets sigma‐1 and muscarinic receptors to modulate cellular stress pathways and support neuronal function. Headquartered in New York City, Anavex is dedicated to advancing treatments for neurodegenerative and neurodevelopmental diseases with high unmet medical need.
The company’s lead product candidate, blarcamesine (ANAVEX2‐73), is a small‐molecule activator of the sigma‐1 receptor currently being evaluated in clinical trials for Alzheimer’s disease and Parkinson’s disease dementia.
