Biotech Company Finds High-Impact Alzheimer's Therapy in Europe

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Anavex Life Sciences Corp. (AVXL:NASDAQ) released positive results from a long-term extension study evaluating its investigational oral therapy, blarcamesine (ANAVEX2-73), in patients with early Alzheimer's disease. The data, presented at the AD/PDTM 2025 Conference, reflect up to four years of treatment from the ATTENTION-AD trial, the open-label extension of the earlier Phase IIb/III ANAVEX2-73-AD-004 study.

The delayed-start analysis conducted through Week 192 showed that patients who began continuous blarcamesine therapy earlier maintained greater cognitive and functional stability than those who initiated treatment later. Cognitive benefits were measured using the ADAS-Cog13 scale, where the least squares (LS) mean difference between early- and late-start groups was -3.83 (P = 0.0165). Functional improvements were noted using the ADCS-ADL scale, with an LS mean difference of +4.30 (P = 0.0206). According to a 2024 review in Alzheimer's & Dementia, changes greater than two points on ADAS-Cog13 are considered clinically meaningful.

The company also reported that continuous treatment without interruption further amplified clinical benefit. At Week 192, those who maintained consistent dosing showed an LS mean difference of -4.20 in ADAS-Cog13 (P = 0.0083) and +5.75 in ADCS-ADL (P = 0.0015) compared to those with interrupted treatment.

Blarcamesine was well-tolerated, with no treatment-related deaths or serious adverse events attributed to the drug. The most common side effect was dizziness, typically mild and transient during the initial titration phase. An adjusted titration schedule extended to 10 weeks in the ATTENTION-AD trial led to a reduced incidence of dizziness (9.6%), down from 25.2% in the preceding double-blind study. Other safety parameters, including electrocardiograms, lab tests, and vital signs, showed no concerning trends.

Compassionate use programs also continue to support the long-term use of blarcamesine. As of the latest data, 74 individuals remained on therapy, with some participants taking the drug for more than nine years. No severe or life-threatening events were reported in this cohort.

Dr. Timo Grimmer, a coordinating investigator for the study, stated that the results "demonstrate that diagnosing and treating people earlier and consistently in the continuum of Alzheimer's disease may lead to greater clinical benefit." Anavex President and CEO Dr. Christopher Missling added, "We believe the clinically meaningful study results combined with blarcamesine's convenient once-daily oral dosing supports long-term therapy."

Regulators and Investors Drive Early 2025 Activity in the CNS Therapeutics Market

The central nervous system (CNS) therapeutics sector continued to show notable momentum in early 2025, supported by market growth projections, regulatory activity, and increased investment in diagnostics.

According to a January 7 report from Research and Markets, the global CNS therapeutics market was projected to expand from US$144.3 million in 2024 to US$431.0 million by 2035, driven by a compound annual growth rate of 10.46%.

On April 7, H.C. Wainwright reiterated its Buy rating on Anavex Life Sciences and maintained a 12-month price target of US$42 per share.

The report attributed this growth to multiple factors, including "the rising prevalence of neurological disorders, ongoing advancements in drug development and approvals, and increased investments in research and development." The analysis also highlighted the expanding role of telemedicine and precision medicine in shaping future treatment approaches.

In the regulatory landscape, Reuters wrote on January 31 that the European Medicines Agency began a safety data review of Leqembi, a monoclonal antibody for Alzheimer's disease, following its initial recommendation for approval in late 2024. The review was initiated at the request of the European Commission to reassess newly available safety information. Eisai and Biogen, developers of the drug, stated that they "did not identify new safety signals" and believed that the submitted data would address the agency's concerns.

​The central nervous system (CNS) therapeutics sector has experienced notable advancements and projections in early 2025. According to a report from Blue Matter Consulting published on February 3, the CNS market is undergoing significant transformation, with innovations and therapies poised to drive growth throughout the year. ​

Analysts Endorse Long-Term Promise of Anavex's Alzheimer's Candidate

On April 7, H.C. Wainwright reiterated its Buy rating on Anavex Life Sciences and maintained a 12-month price target of US$42 per share. The firm highlighted the recently reported four-year open-label extension data from the ATTENTION-AD trial, stating that blarcamesine treatment demonstrated a "continued clinically and meaningful benefit for early Alzheimer's disease patients."

Analyst Raghuram Selvaraju noted that early and uninterrupted treatment led to statistically significant improvements in cognitive and functional endpoints, including a mean difference of -3.83 on the ADAS-Cog13 scale and +4.30 on the ADCS-ADL scale by Week 192.

D. Boral Capital published a positive update on Anavex, also assigning a Buy rating with a price target of US$46.

He further explained that these differences met thresholds for clinical significance. The report emphasized that the safety profile remained favorable, with no treatment-related deaths and a reduced rate of adverse events over time. Selvaraju concluded, "The safety profile and convenience (given the once-daily oral administration) of blarcamesine constitute key advantages of the drug within the context of the AD population."

That same day, D. Boral Capital published a positive update on Anavex, also assigning a Buy rating with a price target of US$46. Analyst Jason Kolbert wrote that blarcamesine "demonstrated a significant slowing of clinical decline," and that patients receiving continuous treatment showed notable improvements in cognition and daily function.

He noted that the favorable safety profile was maintained over the study duration, adding, "No severe or life-threatening adverse events were attributed to blarcamesine, and no deaths related to the study drug were reported."

Kolbert also highlighted Anavex's plans for regulatory engagement, including a submission to the European Medicines Agency and potential discussions with the U.S. FDA for accelerated approval. He valued the company's lead candidate using a model that assumes a 70% probability of success across multiple indications and projected that Anavex would maintain a four-year operating runway, supported by US$120.8 million in cash.

Anavex Targets Global Regulatory Pathways for Oral Alzheimer's Drug

According to the company's January 2025 investor presentation, Anavex is preparing to engage with regulatory authorities in the U.S., Europe, and Asia-Pacific to discuss the full Phase IIb/III data for blarcamesine. The company aims to explore potential approval pathways based on surrogate biomarker efficacy data. A peer-reviewed publication of the Phase IIb/III ANAVEX2-73-AD-004 trial is expected in the first quarter of 2025, with further analysis of RNA sequencing data from the study anticipated later in the year.

Anavex highlighted its proprietary SIGMAR1-targeting platform as a differentiated approach to addressing neurodegeneration. Unlike monoclonal antibody (mAb) therapies that require intravenous administration, blarcamesine is orally administered, potentially lowering treatment barriers and enhancing accessibility across diverse healthcare systems. The company also noted that its candidate may offer safety advantages compared to mAbs such as lecanemab and donanemab.

The company reported US$132.2 million in cash and cash equivalents as of September 30, 2024, and estimates it is funded for at least four years of operations and clinical programs. Anavex maintains no debt and attributes its sustainable runway to disciplined spending and support from non-dilutive sources, including the Michael J. Fox Foundation and the Australian Government.

Blarcamesine is part of a broader pipeline targeting central nervous system (CNS) disorders. Upcoming catalysts include top-line data from trials in schizophrenia (ANAVEX3-71) and Parkinson's disease, as well as new studies in Rett syndrome, Fragile X syndrome, and other rare diseases. The company also retains worldwide commercial rights for its candidates and has patent protection extending to at least 2030–2039.

Ownership and Share Structure

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Refinitiv lists Anavex Life Sciences Corp at 3.30% ownership by management and insiders. Of those, Christopher Missling holds 1.73% and Anthanasios Skarpelos holds 1.54%.

Institutions hold 35.22%, with BlackRock Institutional Trust Company at 7%, The Vanguard Group at 5.5%, State Street Global Advisors at 2.84%, Geode Capital Management LLC at 2.2%, and Two Sigma Investments LP at 1.49%. The rest is retail.

Anavex has 82.26 million free float shares, a market cap of US$696.69 million, and a 52-week range of US$3.25 to US$14.44