Anavex Life Sciences at H.C. Wainwright: Strategic Developments in Alzheimer’s

Anavex Life Sciences Corp. (NASDAQ:AVXL) presented at the H.C. Wainwright 27th Annual Global Investment Conference on Tuesday, 09 September 2025, highlighting significant advancements in their neurological disorder treatments. Led by President and CEO Dr. Christopher U. Missling, the company showcased its robust pipeline and financial health, while navigating regulatory reviews and potential commercialization strategies.

Key Takeaways

• Anavex has completed a Phase 2/3 study for Alzheimer’s and is under EMA regulatory review.
• The company boasts over three years of cash runway and no debt.
• Blarcamesine, their leading drug, offers superior safety and efficacy with convenient oral administration.
• Anavex’s pipeline includes treatments for various neurological disorders, with new trials for Parkinson’s and Fragile X syndrome commencing soon.
• The company is exploring both partnership and independent commercialization paths.

Financial Results

• Cash Position: Anavex has secured a cash runway extending over three years, ensuring financial stability for ongoing and future operations.
• Debt Status: The company operates with no debt, enhancing its financial flexibility.
• Foundation Support: Acknowledged support from organizations such as the Michael J. Fox Foundation.
• Intellectual Property: Patent protection extends up to 2039, safeguarding their innovations.

Operational Updates

• Alzheimer’s Progress: The Phase 2/3 study is complete, with ongoing EMA regulatory review.
• Pipeline Expansion: Includes treatments for Parkinson’s, Parkinson’s dementia, Fragile X syndrome, infantile spasm, Angelman syndrome, Rett syndrome, and schizophrenia.
• New Trials: Initiation of trials for Parkinson’s and Fragile X syndrome is on the horizon.
• Commercialization Strategy: Anavex is considering both partnerships and independent commercialization, ensuring flexibility in market entry.

Future Outlook

• Regulatory Milestones: Expectation of EMA review results early next year.
• Data Readouts: Upcoming results from additional genetic data analysis.
• Trial Launches: New studies for Parkinson’s and Fragile X syndrome are set to begin.
• Strategic Decisions: Determining the path for commercialization, whether through partnerships or independently.

Conclusion

Readers interested in detailed insights are encouraged to refer to the full transcript for comprehensive coverage of Anavex’s strategic direction and scientific advancements.

Full transcript - H.C. Wainwright 27th Annual Global Investment Conference:

Daniel Smith, Equity Research Associate in Biotechnology, H.C. Wainwright: Good afternoon, everyone, and thank you for joining Day 2 of the H.C. Wainwright 27th Annual Global Investment Conference 2025. My name is Daniel Smith, and I’m an H.C. Wainwright Equity Research Associate in Biotechnology. With that said, let me introduce our presenter for the session. I’d like to welcome Dr. Christopher U. Missling, President and CEO of Anavex Life Sciences Corp., who are developing sigma-1 receptor activators for neurological disorders. Anavex Life Sciences Corp. is traded on the NASDAQ under the ticker AVXL. Christopher, the floor is yours.

Christopher U. Missling, President and CEO, Anavex Life Sciences Corp.: Appreciate it. Thank you for the very kind invitation and introduction. I’d like to share with you Anavex Life Sciences. We are a public company. I’d like you to read this. We are dedicated to advancing new therapeutic investigations of the discovery of small molecules, which have a wide reach of potential relief for patients. One of the major indications we’re covering is Alzheimer’s disease, which is still a huge unmet need. There are over 20 trillion people who are in need. One in three can get the disease, and it’s still not yet addressed because of the aging population. You will see more patients getting older and are able to get a replacement of the hip or a heart replacement, but not the brain. That’s what makes it an exponentially rising indication, a growing indication.

We focus on Alzheimer’s disease, and we completed our Phase 2/3 study in Alzheimer’s patients. We are now in the middle of a regulatory review with the European Medicines Agency (EMA), and we also are planning to engage with other regulators. We have currently a runabout cash lasting over three years, and we have no debt. Our drug has been shown objectively superior safety and efficacy than currently approved drugs, including the antibodies Kisunla and Leqembi. The oral once-daily convenient scalable potential treatment is certainly welcomed. We have also other indications in the pipeline, not only Alzheimer’s, but also Parkinson’s and Parkinson’s dementia, and also rare diseases, including Fragile X syndrome, infantile spasm, Angelman syndrome, and Rett syndrome. We also have another drug, ANAVEX®3-71, which is right now in a schizophrenia trial, a Phase 2 safety study. We expect this to be reading out as well soon.

Orally administration of drugs, which is convenient and has a high propensity of being welcomed by also by the patient. I’d like to share with you the mechanism of action, which is upstream. Think of the a-beta and target and accumulation of tau being downstream in the pathology, and think of what precedes the accumulation of a-beta and tau is actually autophagy impairment. That’s what we are addressing with our orally once-daily approach. We’re able to restore the impaired autophagy before the a-beta and tau and other inflammation and other mitochondrial dysfunctions can take place in this disease. As an ultimate outcome, we’re able to be more elegantly able to remove, repair, regenerate, and build resilience in the body and in the brain of the patients. The next upcoming milestones are reading out of additional gene data, which we started this morning to announce a bit more about it later.

We are starting several trials, among them a Parkinson’s study and a Fragile X study. We have the readout, what I mentioned before, of the Phase 2 schizophrenia safety study, which is a focus on safety. The pipeline is relatively rich, we believe, with potential for progressing towards commercialization. We also have a pain drug in the pipeline, which eventually also will move forward. One of the things which we have noticed, and it was recently published, is that the Alzheimer’s therapeutic indication is extremely valuable, but also the prevention is a very important aspect of that. We demonstrated it in a recent study, which was done outside of Anavex Life Sciences Corp., that the drug blarcamesine also is able to prevent the onset of cognitive impairment in animals.

That’s extremely advantageous when you think about that if you’re able to identify in the family members who are at risk of this disease one day to prevent it. This is for the future to be considered with an orally once-daily treatment option. The trial of the Phase 2/3 was placebo-controlled in three different arms: placebo, active arm towards 30 milligrams, towards 50 milligrams, and on top of it, an open-label extension up to over 144 weeks. In totality, it was four years. We are intrigued about it, that we found a very good dose, and this was published also recently at JPET, where we showed oral administration, convenient, with a novel target, and impacts neurodegeneration, which we demonstrated that the atrophy, the brain shrinking, which is commensurated with the pathology, was able to be delayed by the drug compared to placebo. Overall, very promising clinical results.

One point I mentioned was the feature of the degeneration. The brain shrinks, as you can see very nicely on the right side of the slide, compared to normal brains, which do not shrink. We were able to show statistically significant delay of the shrinking, sometimes even halting of the shrinking process in almost all regions of the brain. This is whole brain. This is gray matter, parietal lobe, temporal lobe, limbic lobe, and others. It is very important to be aware that this pathological feature of Alzheimer’s disease, which is a bit the analogy in oncology, the growing of the cancer is a shrinking of the brain in Alzheimer’s. It was able to significantly demonstrate it with the drug to be stopped or delayed.

I mentioned the open-label study, which allows for a very elegant, what’s called delayed start analysis, because you’re able now to keep patients on the drug which had been previously in the placebo-controlled study on the drug, and you then compare that with the patients who got the drug later in the second half of the design of the open-label then. You want to see if those patients who got the drug earlier, if they are benefiting more than those who got the drug later. That’s what we were able to demonstrate. First of all, the safety was consistent with the Phase 2B/3 placebo control, but also that the titration adjustment allowed for a significant reduction of the most often observed adverse event, which was dizziness, which was manageable and lasted sometimes between 7 to 11 days, was reduced almost to similar to placebo levels.

We also didn’t observe any deaths in the study, which is also extremely advantageous compared to currently approved drugs like antibodies, which are known that they cause death due to bleeding and brain swelling and have a black box warning. I also want to point out that the efficacy was favorable, that those patients who received the drug earlier, they also had a benefit in the overall period of the open-label study, and that was demonstrated with a significant P-value. All of this, in summary, suggests that oral blarcamesine could be a long-term benefit effect and could be also disease-modifying. There’s also a threshold what is considered clinically meaningful, and that is a 2-point scale on the ADAS-Cog 13. We certainly have demonstrated that reaching that, which is almost 4 points in the ADAS-Cog 13 after four years. This is the visualization of this.

You can see both on the left side, ADAS-Cog 13, as well as the ADL. On the right side, they show significance the longer the treatment lasts, and it’s consistent with the description provided before. This is now something new. I want to elaborate a bit more. In the placebo-controlled study, we also analyzed the gene of all patients, and we identified, it was pre-specified, that the sigma-1 gene benefits patients because it’s the target of the drug. If you have a wild type, a fully functional gene, if you like, you have a better outcome. If you have a mutation of that gene, you have also a good outcome, but it’s not as good. We noticed that, and it was published in a previous trial, this description. What we did when we analyzed all genes, we found another gene called COL24A1, which is a gene in the extracellular matrix.

It’s intriguing because it’s where these neurons and astrocytes are residing, and it matters in what environment they’re sitting in. What turned out to be an analogy to the sigma-1, we found that if you have a COL24A1 gene or this extracellular matrix wild type, so, very good, all perfect genes, you have a better response than if you have a situation where you have a mutation of this one gene in your extracellular matrix, and the response is not as strong. The good news is that the majority of the patients, as in the public databases, have a wild type sigma-1 gene and a wild type COL24A1 gene. That’s really the good news. For up to 70%, this is beneficial.

The data you see here is that measuring the time from baseline to end of trial of the active arm and demonstrating that the decline in the active arm is comparable to barely detectable prodromal Alzheimer’s decline. It’s pre-dementia patients, if you like. That’s extremely intriguing because you can definitely then identify the trajectory of a patient who already has Alzheimer’s disease, as you can see from this chart, from this table, where the advanced stage is manifested in the higher scores of the ADAS-Cog baseline and the CDR-SB baseline. You have more advanced patients, already Alzheimer’s pathology, and you bring them back and make them basically flatlining, not getting worse as you would expect and you would see in the placebo arm. As a matter of fact, this score of blarcamesine’s 48-week score of 0.8 in the ADAS-Cog was compared to placebo 4.7 better than placebo, minus 4.7.

This is a significant effect. If you put it in another word, in percentages, to the placebo effect, it’s 84.7%. That’s what I refer to, or we refer to, almost resembling or matching a very flat cognitive decline. It’s very intriguing because you can identify these patients with a simple swab test like saliva or with a simple prick test, similar to an AbbVie IV test. I want to now move forward to the overall feedback we received from physicians and from advocacy members. We found that there’s a lot of inbound interest for appreciation for an orally once-daily treatment option because it’s a consistent, proven protocol, like you treat today Alzheimer’s patients with donepezil or rivastigmine, and it allows to be given or identified the patient’s pathology very quickly. It continues also to put the focus on the patient and not on other imaging or other ancillary requirements.

What is also very important for the family, they have less stress. They don’t have to run around and make time and appointments for an MRI test, for a PET study, and for other activities. They are not paid when they do that for their family members. There’s no impact on their own work schedule, and it is less financial strain as well. This is really what we refer to as family and patient-centric care, which can be then supplemented, of course, with diet and other activities. For the physician, the big advantage is that this is like a help timely without delays. They are set up in a way they have always been. There’s really a hard process to engage in another routine, and it’s a streamlined workflow. There’s no logistical barriers at all, and they don’t need to do any PET scan. They don’t need any lumbar puncture.

They don’t need any repeated MRIs. It is just really a convenient approach with the benefit of a drug which is orally available and with better safety profile, which I indicated before. To put this in perspective, we are asked to put this in perspective. I’ll show you in the next slide the comparison to the approved drug Kisunla and Leqembi. It’s both data from publications of all the patients ITT, intent to treat, or mITT, and you can see visually very clearly that blarcamesine demonstrates numerical superiority at an earlier time point already than Kisunla or donanemab, which lasted longer in the trial but had a much smaller effect in the ITT population. The same picture is actually with the CDR-SB boxes which were used in the analysis of Leqembi or lecanemab.

You see the same picture here that the delta, the effect, the strength of the improvement over placebo was numerically superior to the effect of Leqembi. The effect of blarcamesine was superior to the effect numerically to Leqembi as well. Very consistent data. Now, if you put on top of it the genetic profile, the new genetic population data, this, of course, is a much stronger picture than already this ITT. Just the ITT alone indicates a stronger effect of the drug compared to the two antibodies. We have started in parallel to engage with activities of target engagement with advocacy groups, with KOLs. We started also engaging with commercial entities. We have the choice to move forward commercially in a partnership or in a direction of more independence. We will come to that conclusion when it’s time to make that decision.

We will get closer to the regulatory review, which we expect early next year for the European Medicines Agency (EMA). The size of the indications we cover are all very large, therapeutically very unmet need and significant large indications. Alzheimer’s is 7 million in the U.S., similar in Europe, in Asia even more, and similar to Parkinson’s, which is also the second largest indication which we cover in our portfolio. I want to also quickly mention the picture of the IP. We have a really good, strong IP protection composed of meta. We also filed very actively. Every new data was covered before presented, like the gene data you just saw has been also, in the meantime, filed. The patent portfolio protection coverage for all our portfolio is up to 1,039. It’s a solid coverage protection without any extension.

Last but not least, I also want to mention that we have a solid cash position with over three years of cash. We try to be mindful of that going forward, as we always did in the past. We also like to thank foundations which helped us to retain this cash efficiency, among them the Michael J. Fox Foundation and those other foundations. We have a strong team with team members which have been gone through this before to a regulatory review in marketing drugs and the regulatory side of it as well. A very comprehensive, solid team in Anavex Life Sciences Corp., which I’m very proud of. We also have a distinguished scientific advisory board with members in each respective area of expertise. I would like to especially highlight Marvin Sabbagh, who is extremely supportive as the Chairman of the Scientific Advisory Board.

Last but not least, I want to leave you with the Anavex advantage, which is the platform scalability. It is not only for one indication. It’s multiple indications. Also, because of its administration route, it is equitable, accessible for a diverse population. That maintains the sustainability within a global healthcare system. As you know, everyone wants to shrink the budget for healthcare. This would fit very much in that need, and we would be able to accommodate that. With that, I’d like to leave you with the information for further interest on anavex.com, or feel free to reach out to us. Thank you very much for your attention.

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