Anavex Ny tråd december 2025
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Anavex Paratokset mellem den faglige ekspertise og den regulatoriske arm (EMA) i EU udstilles i min optik med udvælgelsen af netop Blarcamesine, som den kandidat med største potentiale til at bidrage og ændre den nuværende behandling inden for Alzheimer i EU. (UK er også repræsenteret i ACCESS-AD programmet)
Må antage - iflg listen i linket, at dette delvis EU finansieret program (37 millioner euro over 5 år), består af nogle af de ypperste eksperter/institutter inden for Alzheimer/neurologi.
At man så udvælger netop Blarcamesine som den medicin man ønsker at bruge krudtet på, må betro på en nøje forudgående evaluering af potentialet af mekanismen og effekten.
https://www.eurekalert.org/news-releases/1112308Paratokset ligger i, at de medlemmer i CHMP komiteen som ikke havde en neurologisk baggrund, var dem som skulle afgøre om Blarcamesine kunne godkendes i EU - i første omgang!
Håber at en ny sammensætning med mere kompetente medlemmer og inddragelsen af N-SAG kan ændre udgangen ved den kommende re-examination.
Tænker også det må være svært at finde 27 nye CHMP medlemmer og medlemmer af N-SAG gruppen, som enten ikke direkte eller indirekte har en relation/kendskab til de eksperter, som skal evaluerer Blarcamesine i ACCESS-AD regi?Som Solsen skriver, så må EMA blive sat i et dilemma - ja i yderste konsekvens kan der faktisk komme til at sidde medlemmer i den nye CHMP komitee, som sidder med i både CHMP og ACCESS-AD programmet!
EMA ser ikke ud til at være gearet til noget så sjældent som en præcisionsbehandling på pilleform i en så kompleks indikation som Alzheimer.
Og spørgsmålet er om EMA reglementet på nuværende faktisk kan begrænse inddragelsen af præcisionstilgangen og OLE data mm., at der trods for en åbenlys effekt i en defineret subgruppe, ikke kan godkende Blarcamesine i EU?
Deltagelsen i ACCESS-AD programmet kan dog ikke undlade at påvirke beslutningsprocessen, selvom EMA er så regulatorisk konservativt!Helt anderledes er vejen meget mere innovativ og åben hos FDA, for netop det Anavex/Blarcamesine repræsenterer.
Inddragelse af og vægtning af:
Subgruppe data
Biomarkører
OLE data
RWE - Real World Experience
Et enkelfase 3 forsøgDvs en samlet vurdering af hele datagrundlaget.
Derudover er FDA også meget mere åben over for acc. godkendelse, betinget godkendelse i subgrupper og betinget godkendelser, hvor man sideløbende efter en evt. godkendelse skal køre et kontrolforsøg.Tidsmæssigt kan vejen via. FDA være både hurtigere og faktisk mere værdifuldt på kort sigt - vil mene at en godkendelse i US vil vægte tungere i aktiemarkedet end en godkendelse i EU.
Mulige partneraftaler og værdien på samme vil uanset hvor Anavex opnår en godkendelse stige betragtelig.
Er overbevist om, at Anavex også i et eller andet omfang har modtaget tilbud igennem årene, men at Anavex ikke har anset at tilbuddene som værende tilstrækkelig attraktive.JP Morgan er det største udstillingsvindue for et biotekselskab, hvor man kan præsenterer sig for potentielle investorer og evt. kommende partner/købere.
Anavex har i nat torsdag den 15. januar kl. 1.30 DK-tid en 40 min. præsentation i konferencens sidste præsentationsblok.
Ved ikke hvad prisskiltet på Anavex skal være, men deltagelsen i ACCESS-AD programmet må have øget værdien. -
Fine indlæg fra Solsen og Tasso1
Uddrag:
Blarcamesine som den medicin man ønsker at bruge krudtet på, må betro på en nøje forudgående evaluering af potentialet af mekanismen og effekten.Når jeg læser hvad der står i presse meddelsen 13. januar.
så skal der nye studier/forsøg ??Uddrag af PM 13. januar
Denne infrastruktur vil indsamle data fra den virkelige verden fra mere end 500 patienter på tværs af en række sundhedssystemer og registrere sikkerhed, klinisk udvikling og behandlingsresultater under rutinemæssige kliniske forhold. Registret vil også understøtte et pilotstudie, der tester muligheden for at kombinere DMT'er med en flerkomponent livsstilsintervention og avancerede kosttilskud fra Nestlé, samt et klinisk prædiktionsstudie af den orale behandling blarcamesine, der tages én gang dagligt.Det er vel ikke resultater ret rundt hjørnet vi skal se fra ACCESS-AD.
Der er da relativt kort tid fra evt. godkendelse af Re-exm. til vi kan forvente en afgørelse med det nye hold EMA'er
Taler vi afgørelse maj/juni.??Stiller mig tvivlende overfor om samme mennesker kan sidde i begge udvalg som Tasso1 er inde på --- tænker inhabilitet her.
Kan det blive en udfordring for EMA at finde nok "Kvalificeret" medlemmer hvis de aller dygtigste hoveder fra EMA sidder i ACCESS-AD udvalget ?
Og hvor længe har ACCESS-AD været i live ?
Bare nogle tanker
Søren
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Nok for Nørder dette
APOE4-genet er en indikator for at udvikle Alzheimers syndrom.
Søren
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"Den tålmodige investor."
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Anavex Udskrift fra JP Morgan den 14. januar 2026.
Udmærket præsentation.
Hovedpunkter - ikke meget nyt:
Re-examination bekræftet til 1. halvdel 2026.
Opstart fase 3 forsøg i Parkinson i 2026.
Opstart fase 2 i Fragile X i 2026.
Bekræfter deltagelse i nyt EU konsortie.
Præsentation udlader ADL - erstattet med CDR-SB.
Forsat understregning af vigtighed af tidlig behandling - evt. præventivt på sigt.
Patentbeskyttelse helt frem til 2040.
Åben for partneraftaler pga. størrelsen af AD og PD.- synes de manglede udsigten til komplette data fra fase 2 forsøget med 3-71 i Skizofreni. (måske betragtet som sekundært på nuværende tidspunkt med øget fokus på Blarcamesine pipeline EMA, FDA og EU-konsortiet?)
For de med manglende eller ingen kendskab til Anavex/Blarcamesine, må data have gjort et stort indtryk - især ift. de alternative behandlinger eller mangel på samme på Alzheimer området.
JP Morgan, 14jan2026, Missing transcript per SA...
I learned from a successful oncology research, which I was involved before prior to Anavex that one important lesson. The power of the body to fight cancer by activating the body on defense mechanism. That led to the question, why wouldn't that be possible with CNS diseases, which consist of even more complex pathologies. Here at Anavex, we like to change things. We like to move science forward. We are dedicated to therapeutics, discovery and development of targeted central nervous systems treatment.
Instead of trying to fix already broken things happening downstream within the neuron, we try to fix wrong things at the start, at the upstream, at the reaction cascade or processes. The way to address complex diseases especially in Alzheimer's disease, is transforming brain health to patient-oriented personalized medicine. And the company's lead asset, blarcamesine is a once-daily oral small molecule that enhances our autophagy through sigma-1 activation and restore cellular homeostasis.
Impaired autophagy processes are upstream of both a beta and tau and therefore anticipate the neurodegenerative process in Alzheimer's disease. We believe the way that we are well positioned to expand on the transformative precision medicine platform and capitalize on significant market opportunities.
So let me share this market opportunity with you. The aging is correlated Alzheimer's, and the worldwide dementia cases are projected to reach 130 million people by 2050. This data was recently received by Alzheimer's Association and is addressing a growing burden with convenient oral once-daily blarcamesine or commercially scalable potential medicine like blarcamesine might be the way to address this growing market.
I'd like to share with you the data which indicated, which was published recently that blarcamesine in its Phase II/III study demonstrated significant slowing of patient relevant cognitive decline in the Phase IIb/III trial. We demonstrated in 48 weeks a 36.3% and benefit and in the prespecified patient population was up to 49.8% at 48 weeks with the primary cognitive endpoint ADAS-Cog13.
We also demonstrated a very solid safety profile with no associated neuroimaging adverse events, and we observed no deaths in the trial, and that extended also to the open-label extension study. In addition to that, 2 very strong biomarkers of the pathology, one is the brain volume loss, which is the correlation of the disease or also called atrophy was significantly slowed with the active arm blarcamesine. In addition to that, the classical biomarker of the pathology measured by a beta 42 plasma ratio was significant as well.
When we did a survey and it was also a published survey we looked up, it sounds intuitive, but there's a very high preference from patients and caregivers to request an oral dosage form for disease-modifying drugs for treating Alzheimer's disease. And also, the explanation for that, these patients are impaired and have a high propensity of not wanting to leave their house or their home, their family environment because they already are in a situation of impairment and it will make it even worse.
We also hear a lot of patients telling us that one of the key features why they appreciate much more an oral solution for Alzheimer's treatment is because every moment when they can stay with the families together or with their cared -- with their loved one is so much more value compared to being taken out to a hospital where they have to undergo a PET scan or an MRI review.
In summary, I'd like to point out that blarcamesine offers this opportunity as a convenient once-daily oral treatment. And we have demonstrated in our programs a significant improvement over comparable currently available clinical outcomes both from a safety as well as efficacy point of view. We also have a financial comfortable situation of having over 3 years of cash without debt on our balance sheet. And we have a very solid IP protection reaching for our market product candidates up to 2040. But more importantly, we are in currently discussions with regulatory agency in key markets in progress with the objective to determine the potential pathways to obtain marketing authorization for blarcamesine.
We are not only sitting on blarcamesine for Alzheimer's disease. We also have a broader portfolio, encompassing other indications with high unmet medical need. One of them is, in addition to Alzheimer's, Parkinson's disease. We already completed a Phase II study in Parkinson's disease dementia, and we are proceeding now this year with a larger Parkinson's disease trial.
We also have expanded blarcamesine's potential in a rare disease setting, specifically Rett syndrome, which we will continue with. But also, we're expanding this year a study in other rare disease, which is much larger than Rett syndrome, which is Fragile X syndrome, which is the biggest part of the autism spectrum disorder. And we're planning a study this year, more about later. But we also have other indications infantile spasm and Angelman syndrome, where we have very solid preclinical data and we could advance it as well.
But last but not least, I also want to point out, we have an expanded portfolio with another drug called ANAVEX 3-71, which is also an orally medication -- potential medication. And we just completed recently a Phase II study in schizophrenia with solid data in Phase II, and we're planning to advance that indication in this program as well. And we have also orphan designation for frontotemporal dementia and very solid preclinical data with ANAVEX 3-71 in Alzheimer's disease.
Again, in summary, an oral convenient treatment options have a strong potential to be more cost efficient, especially in this DNA of looking for reducing costs for the entire Medicare system and a global health care system while biologics have not only the higher cost burden but also potential safety concerns.
We believe we are well positioned to access this economic value with our progressing pipeline and multiple milestones are in reach towards potential commercialization. More about this in due course.
One of the key features of the mechanism of blarcamesine as well as ANAVEX 3-71 is the restoration of autophagy. And autophagy is an important feature, which is an upstream tangential degenerative approach in the biology of nature, which does not start with a pathology itself but degrades over time. So while we age in this procedure of health path, we are seeing a degeneration or a decline of autophagy activity.
But this autophagy is essential for CNS because CNS molecules neurons cannot get rid of their trash or their -- what have the -- it has to be discarded. It has to be recycled, and that process is called autophagy. And this cleanup mechanism diminishes with aging. And we have the fortunate situation, we're able to confirm in vivo and in animals that all blarcamesine acts at a clear reactivator of this neural impaired autophagy. And that gives us a chance to also point out, in addition to that, that the cellular stress, which is also increasing while autophagy is decreasing, that one of the key features of blarcamesine or our drugs is to reduce cellular stress. So it's really 2 converging features of the pathway, which are countering the aging process in the pathological process correlated with aging.
I'd like to now share at this point a bit more about our pathway to advancing autophagy platform. I mentioned we have regulatory interaction. The most advanced is right now with EMA, and we are in the process of getting reexamination of the blarcamesine for Alzheimer's disease in Europe through the EMA, and this will take place and will start in the first quarter of this year. It's starting sooner than in the first quarter, but it will take place in this period of time.
We also have been fortunate enough to be part of a larger European EU-funded consortium, which includes blarcamesine in a predictive study of once daily oral blarcamesine in Alzheimer's disease.
I mentioned we are planning a Parkinson's disease study, a larger study, which will be larger than 6 months. And we're also planning to study -- start a study in Fragile X, the largest indication of autism spectrum disorder of genetically identified autism spectrum disorder, where we found a very strong biomarker of EG, which has been now submitted for publication, which correlates with the pathology in humans and in animals. So this paper describes the EG biomarker in animals, which, however, is also confirmed in humans. So this is a very important part of the clinical study to add a strong biomarker of pathology to the clinical trial.
And last but not least, we're also expecting several publications. Among them, the precision medicine, ABCLEAR publication for automate disease with blarcamesine, which was submitted as well as a publication of Collagen 24A1, which is demonstrated to be instrumental also the wild-type gene for response of blarcamesine in Alzheimer's patients and more about that soon.
So let me now share with you more about the Phase IIb/III, and let me start with the mechanism of blarcamesine. One of the key features of autophagy of the Alzheimer's pathology is to identify the true causality, which probably is closer to the true origination of this complex disease. And we hear often that a beta or tau's [indiscernible] start or defined pathology. That's actually not entirely the case because impaired autophagy has been now identified precedes these beta and tau aggregations, which then lead to neurodegeneration.
And it's really important to differentiate between the quality of a biomarker of pathology and the potential target of a disease. So we are completely in sync and understand and believe that very good biomarkers are concerning a beta and tau are very valuable, and we integrate them and have integrated them in our clinical trials and will continue to do so. But we believe that the closer to the origin of the pathology is more likely the autophagy impairment, which precedes that. In order to confirm that we were able to identify with the help of third-party scientists in in vivo and in vitro, the exact pathway blarcamesine reactivates impaired autophagy. And that is very important to appreciate because it's relatively complex. I want to run you through this slide.
So when autophagy is impaired, that means that circle, that half circle does not close out in a full circle to build the lysosome. And that's often because of either lack of certain key autophagy proteins like LC3 or other reasons, they cannot form this. We are able to demonstrate that sigma-1 activation through blarcamesine in combination with another protein of autophagy called GABARAP are forming a synergistic aggregate, which then is able to move forward the autophazone into a functional and fully bioactive lysosome. And that has been demonstrated in this publication quoted on the slide below. So it's really important to appreciate that we exactly understand how blarcamesine is able to restore and switch on the autophagy, which is impaired by bringing in or sending in sigma-1 activation through blarcamesine presence.
And another very important feature of pathology is the observation that the brain shrinks with the progression of the disease. And not only does the brain shrink, but also the holes in the brain called lateral ventricles, they are enlarging, which you can see nicely in this picture. And we demonstrated very nicely dose dependently also that in our Phase IIb/III study, blarcamesine was able to reduce or retain the brain volume, reduce the brain volume loss or retain the brain volume consistent with the normal brain function. And that was demonstrated in the whole brain in total gray matter in parietal lobe, in temporal lobe, limbic lobe, insular cortex and frontal lobe, very important regions of the brain correlated with function and cognition of a patient.
So a significant correlation not only with the downstream pathology or some scientists refer to this as the N in the ATN spectrum. A standing for a beta, T for tau, N for neurodegeneration. So the shrinking of the brain is -- represents neurodegeneration. So we're able to show that we're able to stop and halt the neurodegeneration of a patient with blarcamesine.
And if I now switch over to the clinical data of blarcamesine, you will appreciate what you see in a minute that since we activate with blarcamesine, sigma-1 proteins, which is underexpressed in the pathology compared to healthy brains. So study in healthy brain show a high -- very high expression of sigma-1 protein, while patients with Alzheimer show a very low expression with a PET scan of sigma-1.
We're able to demonstrate that in our trial, the patients with carried the sigma-1 wild-type gene, which is represented by around about 70%, 7-0, of the population also are able to demonstrate a stronger signal of response of clinical response to the drug to blarcamesine compared to their respective genetic mutations, which are simple missense variant, which does not indicate by the way, to be more likely getting Alzheimer or being impaired in their life or getting any other diseases. So it's not like ApoE4, which is a risk gene. The 1-point mutation sigma-1 is a complete random mutation, which has no impact on quality of life or such. It's just an ability to demonstrate the fidelity of the signal of blarcamesine in the clinical trial. So if you like, you can point out this is another biomarker of showing the fidelity of the signal.
But it's important to notice that the signal for the clinical outcomes are very clinically meaningful, both in the ITT and in the prespecified sigma-1 wild-type population, both for clinical endpoints, ADAS-Cog13 and CDR sum of the boxes. And it's important also to point out that this consistent safety profile was confirmed also in the long-term extension study over 4 years. And the titration was able to reduce one of the major adverse events observed in this trial, which was dizziness and demonstrating that this adverse event is a manageable adverse event.
And we also want to point out regarding safety, we did not observe any associated neuroimaging events and no deaths were observed in the trial duration caused by the study drug. So again, summarizing that the clinically meaningfulness of this study was confirmed with precision medicine and validating the mechanism of action.
In the long-term extension study, which I was referring to, we demonstrated in a comparison to a natural history study, which has been used quite often recently also by other companies, the ADNI control group, and we demonstrate over a period of 144 weeks, a very resilient, strong holding up and not declining as compared to standard of care as demonstrated in the ADNI study with clinically meaningful outcomes over time. And this data allows you to calculate the time saved of a patient. And that's what is a measure with patients appreciate much more because they don't understand what ADAS-Cog13 boxes is, but they understand very well the expression time saved. How long will you stay with your families or with your loved ones without any decline of your function or capabilities.
This patient relevant outcome was identified or calculated to be -- to reach up to 18 months so that is the period of time which a patient will be provided sustained patient benefit by maintaining functionality and independence based on this ADNI comparison -- ADNI study. And this in totality, of course, is relevant for a patient and extends the dignity of aging in these patients.
I described the impact of autophagy decline over time. And the question was what would we -- what would happen if we interact earlier in the pathology of the disease before the pathology even starts. So what if we do a test of pretreating the -- in this case, we did animals first before doing this with patients. If we pretreat with blarcamesine animals, which are healthy and then make them sick with a beta injection or tau or other features, and we then observe what happens to these animals in a cognitive and functional state.
And we observed that animals who received blarcamesine preventative for 7 to 10 days never developed cognitive impairment measured by the water maze, while those who did not get the treatment but got placebo instead, they developed the expected cognitive impairment in the water maze, and this was published recently. So it confirms that this continuum of autophagy impairment does not have to start when the pathology is already present, but you can also think of this as a prevention of prophylactic effect of blarcamesine potentially going forward. Of course, it has to be confirmed in humans to claim that in humans as well.
Now let me switch gears and share with you what we refer to moving now into precision medicine in Alzheimer's disease. And this is important because it is also the way the future of clinical treatment has been demonstrated, taking the playbook from oncology, which I mentioned at the beginning, where precision medicine or patient-oriented treatments are today the standard of care.
And our ambition is to find new precision medicine approaches, which approximate the normal cognitive decline as observed in healthy aging adults, which also demonstrate a decline, by the way, nobody stays cognitively perfect over time while they're aging. And we have to really factor in the reason for looking into this is heterogenesity of the disease, the complexity requires to look more into detail how is the -- these patients constituted in order to find better response to patients than what we have found today.
And we identify a strong effect with the wild-type sigma-1 population already, the 70%, which I showed you with you and which also is consistent the mechanism of action and specific to blarcamesine to restore functionality and cognition.
But now I will share with you, in addition to the sigma-1, another gene, in combination, which gives us the following data. And the data you see here is a chart of the blue line of blarcamesine treatment from our Phase II/III trial in combination of the wild-type sigma-1 with the Collagen 24A1 wild-type cohort. So 2 wild-type cohorts, which both individually represents around about 70%, 7-0, of the entire population, both when you look at gene databases as well as in our trial, consistent with our trial. And the orange line is a comparison of a -- publication comparison of barely detectable prodromal Alzheimer's patients decline. So patients who are not cognitively impaired. And you see they are matching the trajectory while the gray line is the placebo standard of care arm of our study Phase IIb/III.
And what you see that in this cohort, the 30-milligram cohort, which was the most homogeneous cohort of the trial shows a similar referenced to the outcome to the reference barely detectable prodromal decline. And that is even strong -- to be considered to be more impactful, the Alzheimer effect because the baseline, which is not shown here, is much -- the Alzheimer progressed baseline of the blarcamesine cohort was more advanced the prodromal patient cohort, non-patient but healthy cohort.
And if you calculate now the difference between the effect of the drug blarcamesine to the placebo, it results in 84.7% clinical benefit for patients compared to placebo. And that is really moving the needle towards normal aging profiles. And that's extremely powerful point to make to a physician and to a family that if you take this drug, you potentially would become able to retain your function for much longer and an extremely high level.
And the unprecedented blarcamesine effect is demonstrated in this chart. And you can see now the green box pointing out in this cohort, which is again between 50% to 70%. And the strongest was a homogeneous cohort of 30 milligram in this arm, reaching significant effects for ADAS-Cog13 of minus 4.7, highly significant and the 84.7% improvement as well as the CDR sum of the boxes, it was minus 1.4 points and also highly significant, reaching 75.2% in difference to placebo. So extremely meaningful and also real impactful effect sizes for patients with an oral once-daily treatment potential.
I also want to point out one of the key features we have heard also in regulatory review discussion was what is the benefit to the patient? And one scale we covered in our Phase II/III trial was actually the quality of life AD scale, which is the patient-oriented benefit scale, which measures the self-assessed quality of life of the individual Alzheimer's patient.
And in this measure, you go through a question list what the patient will be asked, physical health, overall physical well-being. Energy, a level of energy and vitality. Mood, emotional state of feelings. Living situation, satisfaction with where the person lives. Memory, cognitive function and memory abilities. Family, quality of relationship with family members. Marriage, significant other, satisfaction with the relationship or partner. Friends, quality of social relationship with friends. Self, ability to do chores, ability to do things with fun, enjoyment and leisure activities. Money, financial well-being. Life as a whole. So really very detailed measures of quality of life.
And in the next slide, you will see the outcome of this scale in totality. You see already a trending in the ITT population as in the active [indiscernible] versus placebo. But in the quality of life measures for the respective genetic groups I defined ABCLEAR3, especially, you see a significant improvement over a placebo, but not only that, but it was above baseline when they tried -- when they start the trial. So the quality of life for these patients is not only sustained, but it's actually net improved with blarcamesine after treatment period of 48 weeks. And that's what really measure -- is a measure relevant for patients.
So going into what is relevant for patients, I want to touch upon the convenience for the patients, families and also for the physicians. So let me share with you that one of the key advantages of blarcamesine is that it allows direct access for the patient to a new oral treatment because he just goes to the doctor and gets the prescription. So the focus stays on the individual patient. He's not being pulled into all directions and has to go to MRI centers or PET centers, which, as I mentioned before we heard, he doesn't like to do.
The advantage for the family is also there's less stress for the caregivers and the families. There's no financial strain involved. They don't have to take off time to ship them or send them or drive them to a PET center or an MRI center or infusion center, as a matter of fact. That means there's no impact on the own workforce schedule for the family.
And last but not least, also the physician benefits from that because he keeps his streamlined workflow. He doesn't have to become a project manager for orchestrating PET scans, MRI in another place and so whatnot. So the simplified patient access or care with all therapy means no PET, no MRI needed, no lumbar punctured really what patients and families and physicians are looking for.
And in order to complete the picture of the large patient-oriented identified accessible opportunities, I want to mention with you that -- to you that we have been in close contact now with advocacy groups, with patients and around the globe, and they all tell us the same thing. There's such a demand for a simplified solution with an oral effective drug, which is safe, which can be given, which can be shipped to the Midwest, which can be given without limitations to areas of limited access and knowledge. So all of this is an ongoing education, which we will also continue in order to help our community to drive and build resilience and health ability to have -- offer potential actionable solutions.
I'd like to also mention the addressable market, which we are covering with our portfolio is not limited to Alzheimer's. We talked about Alzheimer's a lot today, but there's also a large unmet need in Parkinson's disease and schizophrenia, I mentioned, which I mentioned, and also Fragile X, which is 6x the size of Rett syndrome. And as I mentioned, we're progressing that as well. And as a reminder, we have good IP protection in the global markets of relevance up to 2040.
I also want to touch upon briefly on the financial background. We have right now in the last reported cash position around about $120 million. With our current cash utilization, we're expecting to be comfortable with more than 3 years with that. And we also have, in addition to that, no debt, which we have to service.
And we also like to point out this fiscal responsible proceeding is also thanks to nondilutive funding, which I'd like to thank for the International Rett Syndrome Foundation, especially also the Michael Fox Foundation. So we continue also to keep this conservative approach fiscal responsibly going forward.
And in summary, I'd like to again summarize, we focus on patient-oriented brain medicine. We address large-stage Alzheimer's program, a huge significant unmet need with a scalable commercial opportunity. And the emerging body of clinical data with blarcamesine suggests a very high efficacy in genetically defined large Alzheimer's population through precision medicine. And autophagy platform, which we represent in our portfolio offers an opportunity through expansion into broader CNS space as well. We have a strong financial position, and we believe we have long-term IP protection secured. And we also expected key near-term milestone.
With that, I'd like to thank you, and we're open to questions. Thank you.
Question-and-Answer Session
Colette van Buchem
Thank you very much for the very informative presentation, Christopher. We'll open the floor for any questions that the audience may have. Otherwise, I have a few to ask. Could you briefly explain and outline the sigma-1 activation mechanism underlying blarcamesine and explain why it plays such a central role in your therapeutic approach?
Christopher Missling
President, CEO, Secretary & DirectorVery good question. So blarcamesine mode of action was confirmed in several peer-reviewed publications. Blarcamesine is an activator of in vivo agonist and the confirmation of that was established in a PET study. So it was also published, demonstrating dose-dependent sigma-1 receptor engagement. Sigma-1 is an integral membrane protein involved in restoration of cellular homeostasis. It activates an upstream compensatory process and autophagy through sigma-1 activation.
Sigma-1 receptors are significantly lower expressed in patients where the brain is not functional anymore compared to healthy normal brains. And hence, activation with a selective sigma-1 agonist helps like blarcamesine could restore the compensatory upstream process of autophagy through sigma-1 activation.
Colette van Buchem
Great. I have another question. How does Anavex plan to position blarcamesine from a commercial standpoint?
Christopher Missling
President, CEO, Secretary & DirectorSo a recent high-profile Alzheimer's disease trial failure of the GLP-1 agonist demonstrated that Alzheimer's disease is a very complex disease. In addition to that, we also saw a failure of a -- failure in an anti-tau program recently by J&J, for example. And it shows that while these are downstream approaches, we are thinking that more upstream restoring autophagy through sigma-1 receptor might become or more related and closer to the pathology of Alzheimer's disease, which is very complex. And this gives the aging population potentially a solution which they're still demanding and waiting for potentially safe and accessible approach, which is given the unmet need and the safe accessible medicine would be something which we would make sure we don't delay too long for having patients access to such a potential solution.
Colette van Buchem
Great. Any other -- great -- the microphone will come to you.
Unknown Analyst
I just had a question I want to understand. So does this mean that you have to treat presymptomatic patients?
Christopher Missling
President, CEO, Secretary & DirectorNo. We trialed in our Phase IIb/III trial early Alzheimer patients. Early Alzheimer's is defined by MMSE baseline score from 20 to 28. It's also the same population the 2 antibodies were treated. And we also preceded the Phase IIb/III study with a Phase IIa study, which had mild-to-moderate patients. So we have been demonstrated to be able to address or give patients benefit in the mild to moderate and the stronger data, of course, with the larger trial in early Alzheimer's. And that's also the defined population in the regulatory discussion.
Colette van Buchem
Any other questions from the audience? Then I want to thank you once again, Christopher, for the presentation.
Christopher Missling
President, CEO, Secretary & DirectorThank you very much.
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Ny artikel der baseres på gennemgang af studier af 450.000 cases viser betydningen af de forskellige APOE gen kombinationer, se
https://www.eurekalert.org/news-releases/1111832?utm_source=perplexityPubliceret 9. Januar 2026
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Anavex Udmærket referat fra JP Morgan.
Bemærk at Anavex/Missling helt undlader at nævne ADL.
Som tidligere nævnt kan CDR-SB erstatte ADL, hvis den samlede p-værdi for ADAS-COG og CDR-SB er under 0,0165, hvilket Anavex kan opfylde fra det eksisterende fase 2/3 forsøg i hele patientgruppen (som jo var den ansøgte gruppe og i særdeleshed i ABCLEAR subgrupperne, som udgjorde ca. 70 %)
Spørgsmålet er om EMA vil accepterer denne ændring i re-examination.
Dette er også inden for kriterierne, som FDA normalt også ville kunne accepterer, hvilket burde stille Anavex i en god position når ansøgningen til FDA skal udformes.Anavex Life Sciences Highlights Oral Alzheimer’s Drug Blarcamesine, Precision Strategy at JPM Conference
Anavex Life Sciences (NASDAQ:AVXL) outlined its strategy to develop oral, precision-medicine therapies for central nervous system (CNS) diseases during a presentation at the J.P. Morgan Healthcare Conference, with President and CEO Christopher Missling emphasizing the company’s focus on upstream dis
Yahoo Finance (finance.yahoo.com)
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Tasso1 -- flot referat fra JPMorgan conferencen.
Tak for detHar tygget mig igennem referatet med Goggle --- min første tanke var, hvordan hulen holder Missling rede på alle data, hjernefunktion, og alle mulige sygdomme. -- Flot
Helt sikkert Tumbs up til Missling.Ting er på gang
https://firstwordpharma.com/story/7064734Harald har været ansat i Anavex eller som konsulent, kalde det hvad i vil !!!! --- ser vi en kommende aftale med Bristol Meyers Squibb ??
Og ikke mindst: Dr. Juan Carlos Lopez-Talavera, er den nuværende chef for forskning og udvikling i Anavex Han var tidligere en tidligere højtstående direktør hos Bristol Myers Squibb -- tænker forbundne kar herBMS har misset kliniske forsøg -- så kanske Anavex kan være nøglen til susses !!!
Spørgsmål -- har BMS penge på bogen
Kanske på Vildspor
Spændende tider
Søren
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Lidt til Weekenden !!!
https://www.youtube.com/watch?v=DBlOJpT9Y1Y
Og fra vores store nabo i syd
Abgelehnte und zurückgezogene Zulassungsanträge bei der EMA
Jeden Monat gibt die Europäische Arzneimittel-Agentur ihre Empfehlung ab für die Zulassung oder Nicht-Zulassung neuer Medikamente. Im Dezember-Meeting konnte ein neues Alzheimer-Präparat nicht überzeugen.
Laborjournal (www.laborjournal.de)
Søren
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Anavex Har hørt hele inteweiwet - og må sige, at det er noget af det mest positive Trump har gjort, ved at udpege Marty Makary, som ny FDA direktør.
Marty Markary beskriver nødvendigheden i at reformerer hele FDA processen, hvor det ikke giver mening at bruge 8-10 år for at få ny medicin godkendt, når man med nutidens AI værktøjer mm. meget tidligere kan eftervise en god effekt og bivirkningsprofil - og hvor man i dag har AI værktøj/programmer til at monterer bivirkningsprofilen løbende, efter en markedsgodkendelse.
Der er heller ikke behov for enorme, omfattende, dyre og langvarige forsøg, fordi man af samme årsag med AI langt bedre får troværdige data.
I praksis har FDA jo allerede taget Voucher programmet i brug, hvor FDA-processen afkortes til 1-2 mdr.
Tillader anvendelse af mindre og færre forsøg.
Anvendelsen af biomarkører i et større omfang.
Samt vægtning af RWE i en større grad.Dette passer helt perfekt i den tilgang Anavex har kørt, hvor anvendelsen af AI og præcisionstilgang har været integreret helt fra starten og løbende tilpasset, som et naturligt værktøj.
Har en god fornemmelse af FDA processen med den nye ledelse og de nye innovative rammer for FDA, som synes at være skræddersyet til den tilgang Anavex står for.
Som Marty Makary udtrykker det, "så anvender vi ikke stentavler mere!"
Tror EMA kunne lære en del af FDA!
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Tasso1 "Anavex Har hørt hele inteweiwet"
Tak for den
for os der er bedre i Vendelbomålet end engelsk og ud i det medicinske er det super du deler på denne måde
Skønt der er nogen der får fat i det hele !!!!I forhold til mit skriv i går omkring BMS kan jeg se de har en enorm pipeline og penge på bog !!
Ligger link fra Tasso1 tidligere om Pharma selskabers muskler
BMS er klar -- hvis !!!
https://www.biospace.com/business/the-top-big-pharmas-have-1-2t-in-stretch-m-a-firepower-availableSøren
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Har ikke hørt interviewet, men AI får ikke træerne til at gro ind i himlen. Dit output bliver ikke bedre af at smide det gennem AI. Et godt forskningsmotto er "lort ind, lort ud".
Jeg synes optimismen tager overhånd - igen. AI ændrer ikke på kvaliteten af data. Det kan måske styrke analysen, HVIS man har gjort de rette antagelser og "fodret" AI korrekt. AI kan nok også se sammenhænge i data, der for mennesker kan være svære at gennemskue. Jeg arbejder selv i sundhedsbranchen og beskæftiger mig med forskning; herunder inden for neuro. AI har uden tvivl en plads, men det er altså ikke den hellige gral, der lige fikser alle fuck ups i et snuptag.
I øvrigt synes jeg, at antagelsen om at EMA skulle have haft et "amatørudvalg" på Anavex' case virker langt ude. Neurovidenskab er komplekst, og der er mange af de cellulære processer, som vi ikke forstår. Det kommer ikke til at ændre sig med et ekspert-udvalg. De har måske bedre styr på neuroteorien, men jeg har en stærk formodning om og tro på, at det ikke er rene amatører, som EMA har ansat til at vurdere kvaliteten af et studie og dertilhørende data. Jeg ved ikke om og hvordan EMA vurderer en case i re-evalueringen med det ekstra data som måtte komme.
Sigma-1 agonister kan måske noget. Jeg har ikke mistet håbet om, at Anavex måske har fat i noget. Men jeg ville være forsigtig med at tro på en godkendelse i april/maj.
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Det er et langt men meget interessant interview med Makary under JPMorgan uge.
Det politiske vil jeg ikke berører idet den orange djævel synger på sidste vers (håber jeg).
Makary beskriver hans første år som leder i fda. Omtaler korruption, fyringer af gamle stivnakker og omorganisering af processerne i fda.
Fokus er nu på udvikling af lægemidler hurtigt og lade en tvivl komme patienterne tilgode. Altså hurtigere godkendelser på mindre bagvedliggende forsøgsvirksomhed. Men opfølgning på sikkerhed og effekt med monitorering af pts i og efter behandling (her blev AI nævnt som oplagt redskab til at indsamle data og holde styr på det)
Han nævner eksempler på gennemprøvede stoffer der senere viste sig medførte mange dødsfald og blev stoppet alt for sent.
Måske en fremtid med rolling trials, hvor der indtages pts løbende så fase 1-2-3 er et sammenhængende forløb. Monitorering live vil med nye og nu godkendte bayesian analyser (statistisk metode) kunne sige, hvornår der er et statistisk sikkert signal på at stoffet er effektivt og sikkert. Herefter kan godkendelsesproces påbegyndes.
Det koncept kan uden tvivl reducere tiden fra tanke om nye stoffer til brug i praksis. Hvis jeg husker rigtigt vil det reducere omkostningerne for biotech og pharma med 2-$300 mln i hele forløbet.
Mange andre spændende oplysninger. Bl.a. slut med monkey forsøg forud for antistofforsøg. Kun krav om et fase 3 forsøg (fra tidl. to), inddragelse af Rwd data, fda som aktiv imod firmaer, der har lovende ting i pipelinen etc.
Sidstnævnte kunne være Anavex. Makary sagde, at fda læste peer review artikler med stor interesse og nævnte et par eksempler på, hvordan de efter info herfra har inviteret firmaet til et møde med fda, hvorefter der er tildelt en voucher med accelerated review til følge.
Der blev også langt vægt på unmet need.
Spændende tider forude for fda og pts, men også afsløringer der viser gennemgribende korruption i fda, som man kan håbe forsvinder.
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Helt enig @Boerboe i at EMA' vurderingsgrupper selvfølgelig er eksperter på at vurdere studier. Problemet ligger i de små biotek selskabers kompetencer i at design og gennemføre studier professionelt.
Det sagt ser behandlinger baseret på SigmaR1 så lovende/spændende ud at jeg tror blarcamesine og måske Anavex kommer videre og vi måske får vores investeringer igen.
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Det er godt med lidt skepsis, er helt enig med dig take omkring AI, det er ikke bare plug and play.
Tror heller ikke CHMP er amatører, men jeg syntes CM kommer godt omkring mange af de kritikpunkter der blev rejst af CHMP - og jeg syntes den logik som CM lægger på bordet er meget overbevisende.
Der er et "hul" som jeg stadigt ikke helt kan finde hoved og hale i, og det er kritikpunktet omkring selve studie designet. Så jeg holder på min nuværende (måske lidt for store) position i stedet for at supplere op, men har en bedre mavefornemmelse nu end for 1 måned siden hvor det så rigtigt træls ud.
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