Anavex Life Sciences at Needham Conference: Strategic Insights on Neurological Innovations

Published 07/04/2025, 22:02
Anavex Life Sciences at Needham Conference: Strategic Insights on Neurological Innovations

On Monday, 07 April 2025, Anavex Life Sciences (NASDAQ: AVXL) presented at the 24th Annual Needham Virtual Healthcare Conference. The company showcased its advancements in oral small molecules for neurological disorders, particularly Alzheimer's disease. The conference highlighted Anavex's regulatory progress, financial health, and strategic plans, balancing optimism with the challenges of entering competitive markets.

Key Takeaways

  • Anavex is progressing with regulatory submissions for blacamazine in Australia and Europe.
  • The company boasts a strong financial position with $120.8 million in cash and no debt.
  • Promising clinical trial data shows blacamazine's potential in reducing brain atrophy in Alzheimer's patients.
  • Upcoming milestones include Phase 2 trial data for ANAVEX3-71 in schizophrenia and new studies in Parkinson's disease.
  • Anavex aims to expand its CNS indications and explore disease prevention applications.

Financial Results

  • Cash Position: $120.8 million as of the last quarter
  • Debt: None
  • Operational Expenditures: $30.8 million last year
  • Cash Runway: Approximately four years based on current cash and expenditure
  • Funding Sources: Includes non-dilutive funding from the Michael J. Fox Foundation

Operational Updates

  • Regulatory Submissions: Blacamazine submissions accepted by the EMA, with further discussions planned for other regions.
  • Clinical Trials: Data from the Phase 2/3 Alzheimer's study and ANAVEX3-71 schizophrenia trial expected soon.
  • New Studies: Planned initiation of studies in Parkinson's disease, Fragile X syndrome, and another rare disease.
  • Rett Syndrome: Real-world evidence supports long-term benefits of blacamazine.

Future Outlook

  • Blacamazine Potential: May be used prophylactically due to its easy administration.
  • CNS Indication Expansion: Exploring new applications and disease prevention.
  • Publications: Additional research expected on blacamazine, ANAVEX2-73, and ANAVEX3-71.

Q&A Highlights

The conference did not include a Q&A session.

To explore the full details discussed during the conference, please refer to the complete transcript provided below.

Full transcript - 24th Annual Needham Virtual Healthcare Conference:

Poonakanan, Associate, Needham: Good afternoon, everyone. My name is Poonakanan, and I'm an associate at Needham. Thank you all for joining us this afternoon for Needham's twenty fourth annual health care conference. I'm very excited to have with us here today doctor Christopher Missling, CEO of Anavex Life Sciences, providing a corporate presentation. Doctor Missling, if you're ready, please take it away.

Christopher Missling, CEO, Anavex Life Sciences: Thank you very much for the very kind invitation and kind introduction. We will make forward looking statement, so I'd like you to read this. Thank you. So we'd like to share with you the story of Anavex, where we are. We're dedicated to pushing the boundaries of scientific discovery with novel oral small molecules that are tailored to potentially offer hope and relief.

More specifically, we're targeting one of the largest indication right now of unmet medical need, which is Alzheimer's disease and dementia, which is projected to grow over one hundred and fifty million by 02/1950. And in some countries or regions of the world, it's growing exponentially with 20,000,000,000,000 cumulative cost for Alzheimer and dementia care from 02/2015 to 02/1950. And one in three Medicare dollars today will be spent in the future on people living with Alzheimer and other dementias going forward, and eleven million are the numbers of of Americans providing unpaid care for people with Alzheimer and other dementias. So targeting these markets using a differentiated and transformative precision medicine platform is our goal. I'd like to give you an Anavex highlight.

We are in regulatory submission stage with our lead compound blacamazine in The AU with the EMA, which we filed last November and which was accepted a month later in December. We are now in the process of also meeting with other regulatory authorities in order to discuss to proceed with a submission for blatamazine with early Alzheimer or Alzheimer in other regions of the world as well. A quick note about our operations. We are able to fund, currently, that's an important subject, our operations, for at least four years without additionally, raising any cash, and we also have no debt. That is also based the projection on, past, utilization of our cash utilization rate.

We have, with our oral blacamazine drug, a advantage to currently approved drugs for Alzheimer disease, early Alzheimer's, Lykkembi and Kizunla, which are injectable drugs and get a required infusion regularly. While our oral drug is given once daily in a more patient friendly setting at home without the need for moving the patient to a facility or needing him require him to observe in a facility for safety updates or safety monitoring with MRIs, which are also inconvenient but also hard to schedule because of the lack of many centers to do that, especially in regions of the rural areas in the Midwest and in rural areas overall. We also have a patent protection for all our product candidates, which is solid. And in summary, blacamazine is a once daily oral convenient scalable potential treatment for Alzheimer's disease. We are not only, have Alzheimer's disease as an indication, and we also don't only have blacamazine as one drug in the company.

We also have another drug in the pipeline with clinical data reading out, relatively soon, which is ANAVEX three seventy one, which had been now tested in schizophrenia, and we're expecting the data the trial is still ongoing. The trader the data of that study to read out around mid of this year. That is a trial phase two in patients with schizophrenia, and the key endpoint is safety as well as biomarker of the EEG with additional clinical measures subsequent. We also have FONOVEX three seventy one, blacamazine, several formulations. The solid formulation is used for Alzheimer's disease, but we also have a liquid formulation which is planned for other indications, especially rare diseases where it's harder to swallow for patients who have a harder time to swallow a capsule or a pill, and it's easier to give them a liquid formulation.

So, again, orally administered candidates offer generally significant potential advantage for clinical benefit relative to costly and logistically challenging biological antibodies based drugs, which also, as we know, often present additional safety challenges. So we believe we are well, positioned for expanding this transformative market. And before I go into the details details, I'd like to share with you the mechanism, which is very important to understand because it's a differentiated mechanism. It's it's upstream of what probably targets you used to probably, know, in the ultimate pathology and other pathologies. We are restoring homeostasis by activating a protein called sigma one, which is impaired or has too low expression levels in pathological situations, and the endogenous agonists are not not sufficiently available so we can replenish them with the giving of blacamazine or our drugs.

And as a outcome, we expect and we see beneficial therapeutic effect for the patient. What's also noteworthy is that if you even have a patient with a mutated sigma one gene, that means the protein is not fully functionally, that still, works well for the patient to activate it with blacamazine. And in a nutshell, the effect is still powerful observed for all patients regardless of the genetic standing, but we did notice a better outcome for those patients with the sigma one wild type gene, which luckily represents the majority the vast majority with around seventy percent, of the population. But again, all patients benefit from the blacamazine activation of the receptor. And another important mechanism detail is relevant for Alzheimer's disease specifically where autophagy, which is a upstream compensatory therapeutic intervention in Alzheimer's, and it's demonstrated by the fact that in the brain, the survival of cells depend on homostasis, balance, restoring functionality, which is extremely complex, the entire brain function.

And so neurotoxins like a beta amyloid or tau or inflammation or mitochondrial dysfunction and others are basically together with the aging, basically pushing back on this homostasis balance which the body is tries to accomplish. And we're able to show that with upstream activation of the sigma one protein with blacamazine that can be countered. And to just to give you a point of reference, the approach of the antibodies, monoclonal antibodies through a infusion an injection infusion, through IV are trying to downstream, to reduce the epitope burden in the brain, while oral blacamazine is doing that more elegantly upstream and more comprehensively over restoring autophagy, activity and as a consequence, not only able to remove these proteins which are toxic, but also repairing and regenerate and build resilience of the, neural cells and the brain function overall. To now move to what are the activities ahead of us, we expect, one of the outcomes I mentioned, the schizophrenia top line data of the ANVEX three seventy one phase two study to expect it by the mid of this year. And we also expect additional data from the Alzheimer's study phase two slash three, and we did a whole genomic exome analysis of the RNA sequence.

And we like to see, if there is a pattern of certain genes being which are downregulated in the pathology being restored, closer to, normal healthy volunteers levels or other genes in the pathology of Alzheimer's disease are changed compared to placebo, we will have a very clear picture of that. And once we have that, we will, disclose that, of course. We also plan to initiate several other studies, and one of them is a Parkinson disease study, which will last more than six months, and to also, subsequently start with, two additional studies. One is a fragile X study and another study of a rare disease, which we have not disclosed yet. And we also expect additional publications, related to Blacar magazine, ANNOVEX two seventy three, and ANNOVEX three seventy one.

We're also expanding the CNS indication with this, platform, which compromises of blacamazine, ANNOVEX two seventy three, as well as ANNOVEX three seventy one, by also looking at potential disease prevention and, more about it later. So this is the overall population, indications we have so far, been able to accomplish, and it's very much driven by moving towards late stage, of trial. And, this gives you a good, overview of the platform potential of blacamazine and ANAVEX three seventy one, but also other drugs in the pipeline ANAVEX one forty one and ANAVEX ten sixty six, which are earlier stage. So Alzheimer's disease has seen the most trials for blacamazine with a phase one, a phase two, and a phase twothree and ongoing open label extension study. We have even Compression Duve program ongoing for over nine years.

And again, we have ongoing EMA review right now. We also see very nice data for Alzheimer preclinically with ANAVEX three seventy one, which was published, and we also finished a phase one study, with three seventy one, and that was also published. And I wanna point out also that the potential future one day is that blacamazine might be potentially used also prophylactically given that it's easy once daily administration and it's patient friendly and family friendly, and you have to transport the patient anywhere, and it can be used for that reason. We have pre clinical data with animal model of Alzheimer's disease where we gave the drug to the animals before they got sick through Alzheimer's pathology, and they never developed the symptoms of Alzheimer's disease dementia or cognitive impairment. And while they are counterparts, the placebo controlled did receive, not that benefit.

Something, interesting down the road and, would be an opportunity given it's an oral scalable therapeutic intervention. We are talking about Parkinson's and Parkinson's dementia. We did a Parkinson's dementia proof of concept study, and it was very intriguing because we saw both signals of proof of concept for the cognitive domain of the patients as well as the Parkinsonian domain. And we are now, you know, deciding how best to proceed with both indications which have unmet need while Parkinson is the larger population. The Parkinson dementia study is maybe has more unmet need.

So we will see how we proceed with both, but we're planning for both indications to move forward. Last but not least, the most advanced clinical trials for rare disease called Rett syndrome. We completed three trials, and we had also really nice, real world evidence from patients who have been now on the drug for over five years after finishing both placebo controlled studies as well as the open label studies. And they requested to continue to stay on study drug, and which the majority have, been able to, have requested and able to receive accordingly in three regions of the world, in North America, in Europe, as well as in Australia. So in totality, there are some patients with retardrome on blacamazine over five years.

That is combined open label extension and compassionate use as well. So now let's move to the early Alzheimer's disease trial, which was a forty eight week study in three arms, two doses, which were very similar turned out in one placebo arm. After forty eight weeks, the patient were offered a open label extension of up to a hundred forty four weeks that includes a totality of 192, of analysis, which we will show in a minute. What we found in this placebo controlled study is an extremely intriguing biomarker response, which is a classical feature of the Alzheimer pathology, which is shrinking of the brain. As you see on the left side, there's a healthy brain.

It's fully and it's has hardly any holes. On the right side, you see a brain with Ultima pathology. It has shrunk significantly and it demonstrates major holes in the middle and on the sides. So that is the hallmark of the Alzheimer brain losing mass of the brain, which, of course, translate also in cognitive impairment and functional impairment. And what we found in our trial was a drug dependent reduction of the atrophy of brain volume loss of almost all regions in the brain of the patients on study drug, which did not happen for patients on placebo.

And it was in the whole brain and the total gray matter in the parietal lobe, in the temporal lobe, in the limbic lobe, insular cortex, and frontal frontal lobe. So very broad countering countering the pathology of the Alzheimer pathology and significantly slowing the atrophy in broad brain regions after forty eight weeks of treatment. Now let me move to the open label study adding to the placebo controlled, the open label blacasm blacamisin treatment. And this actually allows for a nice observation or query of how do the patients who got the drug later after they got placebo, compare in their cognitive and factually outcome to those who got the drug earlier, which were the patient in the placebo controlled on blacamazine to begin with. So the group can be separated between continued blacamazine or also called early start group And in comparison to the patients which switched from placebo to blacamazine, also referred to as late start group.

And let's take a look what the difference was. So first, I'd like to start with the safety. There was a consistent safety profile with no new safety findings for over four years of treatment with blacamazine. We also were able to address the titration, which was too short in the placebo controlled study, and we expanded it longer in the open label study. And this titration adjustment demonstrated the manageable nature of what was the most frequent treatment emergent adverse event, TEAE, which was dizziness, which was reduced more than half, to less than ten percent in the open label study, demonstrating the manageability just by titration more slowly.

And also very importantly, we did see no deaths related to the study drug in the entire, trials that includes placebo controlled as well as the open label study. And regarding the efficacy, we saw that the patients who treated who treated earlier had a significant benefit compared to those patients who had a one year delay. And the difference was for ADAS COG over a hundred ninety two weeks up to, minus 3.83 points in the ADAS COG 13, which is an improvement for, ADAS COG 13, a negative number is respectively. And for the functional outcome ADCS ADL, the difference was 4.3 comparing the earlier start to the late start, which also is an improvement because in, activities of daily living, a positive score is an improvement. And in totality, the data indicates a disease modifying effect of oral blacamycin once daily.

In addition to that, the suggested earlier oral blacamycin treatment initiation may contribute to a continued long term beneficial therapeutic effect. And I also want to point out that a recent publication identifies what is a threshold for a clinically meaningful outcome measure for ADASCOG, and the publication refers to a two point or larger as a clinically meaningful improvement, which is demonstrated in this, data. So to look at the chart over time, you see here on the left side, the ADAS cog 13 and on the right side, the ADCS ADL. So left is the the cognition, on right side, the function, respectively. And you see nicely the separation between double blind study and open label extension study.

And it's interesting that because the trial, happened during COVID, there was not, like, a consistent administration or transition from patients who finished the placebo controlled study to the open label. In some cases, patients had to wait more than seventy five days, some even longer because they could not go to a hospital to be getting access to the to the open label extension part of the trial. So they had to wait, and they basically didn't get a drug in this period of time. And what we learned is that those patients who did not get the drug right away, which were previously on the active arm, they also did not do that well. And so we learned something in addition to the, long term benefit of dacamazine compared to a delayed start, is that there's also a benefit to not to interrupt the, the treatment of blacamazine once you start the treatment of blacamazine.

And, we look at the literature, there's actually a phenomenon which has been shared by other disease modifying drugs, which if you take the drug first and then interrupt it for too long, that it gets actually worse than if you just never had the drug before and you start the drug. So an interesting observation that leads to, again, to the conclusion, you want to take the drug as early as possible if you have Alzheimer's disease. And when you take it, you wanna take it continuously and not stopping it. And this is a lesson which we were able to learn from this phenomenon which happened during COVID. We also wanna point out that we published the majority of the study, the placebo controlled study, in a paper a few months ago in the Journal of Prevention of Alzheimer's Disease, and that was demonstrating in totality that the blatamycin advantage was in is the oral administration.

It's a novel target activating autophagy, which is upstream of a beta and tau, and for that reason might be more, closer to the core of the pathology itself and demonstrated promising clinical results. And you see on the picture, the actual capsules, how they look, which are of blacamesine. So now I'd like to move to, what we're doing in order to prepare for commercial activities. We are, very strongly now engaging with patient advocacy, with providers and payers to educate them about blacamazine in order to build also alliances down the road and, build also the case for the advantage of blacamazine over alternative treatment options. And, it's important to do that because, there is, first of all, an unmet need.

And the blacamazine features orally once daily versus challenges of biological antibodies, intravenous drugs, needs to be, of course, pointed out in this respect. When we look at the market size, we see that Alzheimer's is the largest market with six point nine million in The US, larger even in Europe with seven million patients. Asia, even larger, twenty three. So global, we're talking about thirty five million patients currently with this disease of Alzheimer pathology. Parkinson disease, is a bit smaller with one million in The US, One Point Four Million in Europe, and ten million global.

Schizophrenia has one point six million in US, Three Million in Europe, and, twenty four global. So we're addressing really very large markets, and you see also the numbers for the respective rare diseases. Wanna share briefly about our status on patents. We were very, adamant about protecting our composition of matter and other methods of use of our drugs, both ANAVEX two seventy three as well as ANAVEX three seventy one. So in total, we have good patent protection for both candidates up to 2,039.

And then that includes composition of matter of black caramazine, which is very important, with a crystallization, which is used and was used in all clinical studies of blacamazine in Alzheimer's disease specifically. We wanna now switch to the commercial and operational status. We believe we have a strong financial profile with at the last quarter of a hundred 20,800,000.0 in cash. We have no debt. And last year, the comp the operational expenditures were 30,800,000.0.

We expect it would be a bit higher this year. So we can calculate with current cash position that roughly four years of runway, and that is a sustainable cash runway, which is also due to a disciplined operations and utilizing non dilutive cash sources that includes cash from non non dilutive funding source, the Michael Fox Foundation, which we like to thank very much, for. I also like to quickly share the team, which is consist of senior managers with respective big pharma background. And, also, I like to point out that we have a strong scientific advisory board, which is headed by Marvin Sabag, which is the chairman of the scientific advisory board. And we continue to get us to with other candidates, which we might announce soon to add that list, which is already extremely rich and with high profiled physicians and key opinion leaders.

With that, I'd like to summarize where we are with Anavex. So I wanna again emphasize the key advantage of Anavex is the precision medicine platform and its scalability, which allows for equitable and accessible therapeutic intervention for diverse population and maintaining sustainability within the global health care system, which is a challenge these days because every global health care system is now looking to reduce cost and cut cost, and we would be able to be flexible with a small molecule to address this into with our interventions, upstream. With that, I'd like to, like to indirect direct you towards for more information on our website, www.anorex.com, for more information. And I I'm excited to keep you an update as we proceed with our programs. Thank you very much.

Appreciate it.

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