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Anyone who actually care to read the stats being refereed to, take a deep breath and think, will find that they are an amalgamation of treatment type, type of enabling technology and indications further marked by whether a new substance is involved (as apposed to a known substance approved already approved in another indication). A big pile of vary different circumstances.
Very few drugs is are in each indication and in CNS and specifically AD in 2022 there were none. Furthermore A2-73 is a new substance never approved for anything by EMA or elsewhere. All of this is the biggest reason that the silly idea of 85% chance of approval for A2-73 in AD, only because EMA has agreed to evaluate a complete and correctly completed MAA, is just complete fiction!
There is a chance of EMA approval for A2-73 in AD, but it won't be anywhere near 85%.
But but but it is a statistical fact that there is 85% chance I will get rich no later than 2025 cause the EMA said so and when I see 85% from EMA it means 85%! You just want to pee on my sugar sandwich.
Most of what you write is a good thesis, although I don't know we have information to conclude the reason why ADCS-ADL has been left out, other than it was most likely way off being stat sig.
Next I want to see the CHMP actually receive and validate an MAA, which we may or may not, but we likely will see PR'ed as it starts the clock ticking towards a material event.
Then I want know (but likely we won't be told) what objections/feedback the CHMP wants responses to from Anavex at the two process clock-stops.
Sometime late this year or early 2025 we will get to know the final verdict on the P2b/3 trial being pivotal. That is binary EMA process event that Anavex cannot escape making public.
I still maintain that anything like an 85% chance of approval is way beyond sensible at this point with what we currently know and don't know. Maybe the publication of the P2b/3 data analysis peer reviewed paper will cause me to reassess. Time will tell...
I computer generated this Transcript of Missling's JPM presentation. Have fun! It should answer some of the questions raised or at least clarify what was said.
00:00:02 Speaker 1 Forward-looking statement. Thank you. So Anavex is dedicated to creating scalable drug therapies that are both easy to administer and accessible. 00:00:14 Speaker 1 We are contributing to the sustainability of global healthcare as well. Our commitment is that cutting edge thinness treatments are. 00:00:25 Speaker 1 Available to a diverse patient population, ultimately enhancing the effectiveness in reach of healthcare systems, we believe that this focus on accessibility and sustainability represents an alternative and attractive opportunity in supporting innovative solutions for global healthcare. 00:00:47 Speaker 1 And with our scientific advancement, we have gotten in very interesting indications which have high unmet need which includes Alzheimer Disease, Parkinson disease, dementia, Parkinson disease and Red syndrome. Our precision medicine approach has developed drugs which. 00:01:07 Speaker 1 Activates the body, owns defence mechanism and makes it a small molecule, activating very more upstream than many therapeutic interventions developed so far, and we're aiming to bring this drug treatments to market across the globe. 00:01:26 Speaker 1 Let me explain a bit more about the mechanism. 00:01:29 Speaker 1 And when we are learned recently about GLP one agonist, we basically become intrigued by the fact that the another one of agonist in the metabolism. 00:01:45 Speaker 1 And and ultimately in the body on defence mechanism becomes a therapeutic intervention of choice with great success, which we know from GLP one agonists these days and Sigma one is also a target which we activate. So our drugs based on the Sigma one activation a protein. 00:02:06 Speaker 1 Which is required to restore homeostasis or reduce cellular stress in the body, which can be caused by either chronic cellular stress caused by genetic dysfunction like rat syndrome, fragile legs and others, or by the chronic accumulation of cellular stress by ageing, which is. 00:02:26 Speaker 1 The classical example of Alzheimer and Parkinson, and in both or all these cases, therapeutic indications. So far we've observed that every time we have this down relation of the Sigma one because of not enough countering arcanist endogenous are available in the body. We can provide them from the outside. 00:02:48 Speaker 1 To activate the Sigma one protein to increase the level of countering and engaging and restoring function and reducing cellular stress and ultimately benefiting the patients. We also have observed that the Sigma 1 binding is so. 00:03:08 Speaker 1 Target specific and has a binding affinity that is so specific that even when patients have a slight mutation or carrier of Sigma 1 variant that they're still are benefiting from the Sigma one activation. So in overall we have observed in all our trials so far. 00:03:29 Speaker 1 A benefit from all patients, regardless of their genotype, which is of course very important because that is comprehensively addressing the entire population, which can be treated with anavex 273 or black amazing. And with this approach. 00:03:47 Speaker 1 I will now also provide you a very important feature which is important in CNS, which is often lacking, which is a biomarker correlated outcome. 00:03:57 Speaker 1 So we have observed in all our clinical trials which we have completed so far and analysed with the expression level of the Sigma one, we have observed that every time there was a benefit, clinical benefit observed that the responding biomarker which we measured. 00:04:18 Speaker 1 By expression levels of mRNA in the blood was every time increased, so the level of Sigma one expression level correlates every time a benefit takes place. These are very strong pieces of evidence of mechanism of action and we have observed that in Alzheimer disease. 00:04:39 Speaker 1 In a study which was published as well as in Parkinson disease and Parkinson's dementia, and ultimately also in Rat syndrome, so every time a benefit was measured in the respective endpoints, the Sigma one activation in the active arm correlated with the benefit. 00:04:57 Speaker 1 And this was also confirmed with the Sigma 1 variant analysis, which is independent of the expression level of the Sigma 1. 00:05:08 Speaker 1 That ultimately leads us to be able to define the science of anavex as precision medicine because it increases likelihood of clinical success. Because we are able to observe the clinical benefit with a biomarker of efficacy. 00:05:28 Speaker 1 Now let me share with you the activities which are ahead of us, which are very exciting activities. 00:05:37 Speaker 1 One of the major activities are the full data of the ANAVEX 27304 Altima study phase 2/3 and we've been very cautious in the communication so far. We want to conserve this data to a a journal paper which requires. 00:05:57 Speaker 1 This procedure we also want to mention that we are in the process of starting a Parkinson's disease phase 2/3 study after we had successfully completed a Parkinson's dementia study and we measured mdsu Perez and showed improvement in this. 00:06:17 Speaker 1 Primary endpoint score, but we also want to proceed with our reduces franchise and we are planning starting a fragile X study of Phase 2/3 which is a very intriguing indication because it has overlapping features of Rett syndrome which will come in a minute or two. 00:06:37 Speaker 1 And the size of the fragile X market is 7 times the size of a red single market. So very attractive proposition is no drug yet approved for fragile acts. 00:06:48 Speaker 1 And then also, I'd like to point out that we have a pipeline of other drugs including UNIVEX 371. 00:06:55 Speaker 1 And among them. 00:06:57 Speaker 1 Finishing the solid phase one study which was very positive, we are now starting a schizophrenia study with ANAVEX 371 and what is very intriguing about this molecule ANAVEX 371 is that in addition to Sigma one activity, we also have with the univex 3 signal one. 00:07:18 Speaker 1 Molecule, a very strong and selective M1 activity and as you follow a little bit like the CNS landscape, you might have noticed that a company with an M1 agonist was recently bought for $14 billion in a buy out by Bristol-Myers. So there was. 00:07:38 Speaker 1 The company caruna with their M1 agonist for schizophrenia and our scientific founders always described to us that the 371 molecule, the anavex 371, might have very strong features for schizophrenia. 00:07:58 Speaker 1 Because of its potential synergy between Sigma one and M1 agonist, so we're looking forward to this trial and are excited to initiate that. We also have in the background which we have not disclosed yet, many more preclinical data with rare diseases which. 00:08:18 Speaker 1 Have shown positive outcome with our drug or drugs unobtrusive 3 and we are in the process of deciding which one of these indication should be selected as the next rare disease to also initiate a study in. But again this also contributes to the. 00:08:39 Speaker 1 Information that we really are sitting on a very strong pipeline and a platform pipeline, not only limited to 1 drug last night, but not least I also want to point out in addition to the therapeutic intervention of this upstream approach with this small molecule compensating for cellular stress which in the body. 00:09:00 Speaker 1 Is evolving in pathologies. We also observed the potential to prevent pathologies like Alzheimer disease in two different and independent animal models. For anavex 273 as well as Annex 371. So in both independent animal models we gave the drug to the animals. 00:09:21 Speaker 1 And then we made them sick with a better injection or other features of ulcer, partial Alzheimer pathology. And every time we have observed that the active arm did not become impaired cognitively, while the placebo or the control arm of these animals did so very nice to see also the path. 00:09:42 Speaker 1 Forward that this simple oral once daily molecule, which can be taken at breakfast if you like. One day could be something to think about down the road in the future. 00:09:54 Speaker 1 Is a administration for prevention of pathologies across the board. 00:10:00 Speaker 1 I'd like to now go more into details of the indications which we have so far explored with our Sigma One and Sigma receptor Discovery platform, which was established in the company since inception. So all the molecules are owned by the company. We have identified these molecules. 00:10:21 Speaker 1 In the lab, we then went to identify a biomarker of efficacy. I mentioned Sigma one expression level of mRNA in Sigma 1 variant levels with genomic precision and we're now went ahead and found indications which could benefit a patients. Among them are ultimate disease. 00:10:42 Speaker 1 Parkinson dementia and Parkinson disease itself, as well as rat syndrome. So we're now moving forward in schizophrenia frontal deployments area. We have also the plan to move forward with Fragile X on the right side and infantile spasm, angelmann syndrome and other indications I mentioned. 00:11:02 Speaker 1 And then all of this indication you see here, we have very strong fundamental preclinical data to support potentially entering with our molecules in the clinic. I also want to mention molecule on the right side under extent 66 which has been shown very encouraging data in a model of different models. 00:11:22 Speaker 1 Of pain, including visceral pain. 00:11:26 Speaker 1 So let me now go over in more details the different indications where we have clinical data we were able to get a complete 2 clinical data in Alzheimer's disease and the first one was a relatively. 00:11:41 Speaker 1 One study in ANAVEX 273 over lasted now over five years and patients on this study actually requested to still stay on study drug after five years. So we open up compassionate use programme for this patient with is still ongoing. We had an interim analysis done as 148 weeks of this study. 00:12:02 Speaker 1 It showed very nice resilience of for patients on the right dose to not decline in cognition and function. This data was published and the source is in this presentation. 00:12:14 Speaker 1 And then we went in on and did the larger study, the Phase 2/3, which I will describe a little bit like in the next slide, it's important to be aware this is the largest DNS indication of unit made with 35 million patients worldwide affected by that and 6.5 million in the US alone. And given that the clinical advancement. 00:12:34 Speaker 1 And medical improvement is so, so high to address every aspect of bodies impairments like replacing a kidney, replacing knee and replacing a hips. But the only area where we don't have yet the ability to enter and really do something fundamental difference really the brain. 00:12:55 Speaker 1 Because of its complexity. 00:12:57 Speaker 1 But we think we have something intriguing here because of our approach is more upstream and comprehensive and more comprehensive than downstream targeted therapies, which are also still in development. 00:13:11 Speaker 1 So the Parkinson disease franchise started with us with the background that Michael Fox Foundation. 00:13:17 Speaker 1 Fully funded us. 00:13:19 Speaker 1 To develop the confirmation of this molecule to be beneficial for Parkinson's disease, and this led to a discovery of that the drug was showing ability to. 00:13:33 Speaker 1 Benefit in a six. Oh, team six ohda model, which is a disease modifying model of Parkinson disease to reverse. Actually the pathology of parkinsonism and reduce inflammation and restore function for the animals. We then use that as a basis to. 00:13:52 Speaker 1 Because we had knowledge about the Altima features of the drug, so cognition benefit to go and test the hypothesis in Parkinson's dementia. So this way we could see if either or these two features which were confirmed would also benefit in this really difficult indication which has failed almost in all cases. 00:14:13 Speaker 1 In competitive landscapes, Parkinson dimension has failed in almost all cases in in other treatment regimens, and we successfully accomplished this phase two study in Parkland dementia and we're now planning to do a larger study to confirm that in a pivotal study. But the same time, because of the benefit. 00:14:33 Speaker 1 So in this dementia study with Parkinson, we noticed that a significant improvement of the motor impairment and quality of life measured by MBS Part 2 and part three was significantly improved and there was a huge demand from all foundations in the world including Michael Fox of Kyle Parkinson UK as well as the. 00:14:53 Speaker 1 So shake it up foundation in Australia to proceed with this molecule into a larger Parkinson's study. So we're planning now a larger Parkinson's study as well. 00:15:05 Speaker 1 So let me now move to the red. Is this franchise Red syndrome. We were able and again the same principle started here like with Parkinson disease, we gave the molecule to the rat foundation and they said we will send the molecule. We had no lab anymore to A to a lab. Which of that choice with the. 00:15:24 Speaker 1 Choice of a model of their choice, and lo and behold, they called us up and said what you got. And we said, well, it's our molecule. Well, they said that we have only one shot and we gave it to these animals and they improved better than any other model that that's really nice to hear. What shall we do? And they said, well, we should proceed in a clinical trial and they supported us. 00:15:45 Speaker 1 Also, in the first clinical trial, which went ahead and finished successfully in the US and then we went ahead in adults and then we went ahead and did a study same size in adults internationally, they called the other test study and after that we're actually not after that in parallel because we're so excited. We went ahead and did the studying. 00:16:05 Speaker 1 In in patriotic patients, the excellent study and this study was not fully powered. It was a Phase 2/3 and it was not that we were not happy about it, but we had observed a little bit too high over placebo effect which is not uncommon. Unfortunately in young patients and. 00:16:25 Speaker 1 The fact that we also randomised 2 to one did not help the fact, but we saw a very strong signal to move ahead with this programme and, as a matter of fact, after this study finished, after 12 week of treatment, the majority of patients requested to go into the open label extension of 48 weeks. 00:16:45 Speaker 1 And interesting enough, after 48 weeks, the majority of patients, 93%, requested to stay on studies drug. And so we opened up a campaign that use programme for these patients, which they're now on this programme for almost for almost four years in totality, including the Ole programme. 00:17:05 Speaker 1 And the prior other test study, which finished earlier than the excellent study, had even a higher rate of conversion into or request into the study of compassionate use. So very strong feature of confirmatory interest and benefit and you have to understand these patients are very impaired. 00:17:26 Speaker 1 And they're really are very sensitive. So if they would not have good safety observation and efficacy, they would drop it without any hesitation. We also see the voices from these patients being very encouraging by saying in one case we did a surprise once with her mobility. 00:17:46 Speaker 1 They're highly immobile. These patients are sitting in the wheelchair all the time. We heard a noise from our family room and next we looked and Madeline had climbed 12 steps upstairs to her bedroom by herself. And we were told she never, ever did that before. And another voice of her parent within a week of starting the Univex Open label. 00:18:06 Speaker 1 Extension. She only had one seizure and then she went three months without a seizure. You might be aware or not, but patients with Rett syndrome have a high features or or pathology of seizure. Up to 70% of patients have seizures. 00:18:23 Speaker 1 It's very intriguing evidence, and when you click on these links you will see more parents voices to that effect. 00:18:33 Speaker 1 Now let me go to. 00:18:35 Speaker 1 The next slide which is showing the overall pipeline and how we're going to move forward to address the unmet need. So really the image gives you a support and a feature of that. We really are sitting on a strong pipeline and also a platform of pipeline. 00:18:53 Speaker 1 Multiple drugs, which all have in common, more or less a affinity, different features with other targets of Sigma. One related activations, again Sigma. One activation is extremely intriguing. Novel approach of activating the body owned defence mechanism. 00:19:13 Speaker 1 Restore hemostasis and push back on cellular stress, which can be either caused by genetic dysfunctions, fragile eggs or rat syndrome, and others, or by by lifestyle and ageing like Alterman, Parkinson and and others. Eventually as well. 00:19:32 Speaker 1 So now let me move to a study which is the most advanced study in Alzheimer disease and probably the most important right now for the for the company. We did have 508 patient study in Alzheimer disease, early Alzheimer with the oral once daily treatment and we randomised to active. 00:19:52 Speaker 1 Arm and placebo and what is extremely important is to be aware that we have included in this study structural and functional MRI as well as all the biomarkers of relevance a better Tau and others and also what is very important in all our studies so far. 00:20:12 Speaker 1 We've always included full RNA and DNA expression levels, so we will always be able to later on analyse the extreme, interesting features of how the body reacts to the drug compared to placebo. 00:20:28 Speaker 1 And I want to mention quickly that we finished these RNA analysis expression levels for the red studies so far and for the Parkinson's dementia so far. And what we have observed is that every time we look at the pathology side of the patients, the placebo arm. 00:20:48 Speaker 1 We find the confirmation, which is published, that genes are overall downregulated in these pathologies in rat syndrome, as well as in Parkinson and also in Alzheimer. But when you look at the active ARM patients, their. 00:21:03 Speaker 1 Genes are suddenly completely opposite to the placebo arm upregulated, so it's very interesting to see in very broad features and clusters of genes which are countering apparently the pathology which is ongoing in their body. So extremely intriguing heat map, pictures of different colours. 00:21:24 Speaker 1 Between the active arm being upregulating genes, again towards healthy levels and the placebo arm showing mainly downregulated genes. 00:21:35 Speaker 1 So we had measured function and cognition in our key coprimary endpoints and we see there some of the boxes as a secondary endpoint and the data so far analysis I want to explain that to you because it's not trivial for regulatory purposes, you need both approval. 00:21:55 Speaker 1 For approval you need cognitive endpoints met as well as functional endpoints met the cognitive result as Coq. The functional is others Adcs ADL or CSML boxes. 00:22:07 Speaker 1 The pre specified clinical endpoints were all analysed in our study using the mmra M this method is the convention used for regulatory filings in early Alzheimer disease, the trial is successful. If the Co primary endpoints are met significantly for each endpoint at a P value. 00:22:27 Speaker 1 Lower than 0.05. However, there are also significant or if the significance is reached for one primary Co primary endpoint at a level lower than 0.025. In that case also the trial is successful. However, since we need for regulatory purposes. 00:22:47 Speaker 1 Also met meet the functional requirement of of the drug effect. The series from the boxes now comes the next in line and that needs to be analysed also at the P value less than 0.025 and when you can see here that in both cases the others called and the sellers on the boxes. 00:23:08 Speaker 1 The key endpoints are less than 0.0 to 5:00, so we meet the requirements of that. In addition to that, we also have measured specific biomarkers of the pathology and they were significant of a better reduction measuring the A better 4240 ratio, which is on the right side of the slide. 00:23:29 Speaker 1 Showing in studies before correlates very clearly with PET levels of the pathology of a better reduction in the brain, so can be used exactly for the same logic as a confirmation of the a beta reduction of the drug. However, what we are very intrigued about on top of it. 00:23:50 Speaker 1 And we believe we have not seen any drug showing that in Alzheimer disease is what is the most obvious pathologically signs which also allows Alzheimer has observed in his patients which is the shrinking of the brain and you see that on the right side of the slide. The healthy brain has a larger brain mass of white. 00:24:11 Speaker 1 In great matter, inside his shrinking feature, you see the whole space really building up the black holes in that brain, and that is what we have observed with our drug. We have observed that we can stop. 00:24:25 Speaker 1 Or delay the onset of this brain shrinking in many regions of the brain, which is fundamentally basically stopping the new degeneration in itself because of the data which we shared with regulatory authorities in Europe with the EMA's we have initiated based on their feedback the regulatory submission to the EMA. 00:24:46 Speaker 1 For glaucoma, azine for oral blackmachine for ultimate disease. 00:24:50 Speaker 1 We also have started. Now we started to explore potential commercial activities in examining innovative strategies to effectively engage patients, providers and payers and. 00:25:01 Speaker 1 It's also important. 00:25:02 Speaker 1 Which I mentioned before that we will expect the data full data, which again is not fully disclosed to come out in a peer reviewed journal. 00:25:11 Speaker 1 Also, we are want to make points aware that this study has completed the placebo control part. However is now has moved ahead with the Open label study of 96 weeks. In addition to the 48 weeks. So the in totality will be three years and as a matter of fact there will be already an extension. 00:25:32 Speaker 1 Of this 96 weeks to 144 weeks, because of the wrecks request of patients to stay on steady drug. So what we have observed previously again shows up also in this study a request from the patient to stay on study drug with this orally once daily dose where we also want to share with you. 00:25:52 Speaker 1 Briefly, is a bit of simple side-by-side analysis to do nabab, which has recently published their data of early Alzheimer and the others, Coq 13 was exactly also provided in this paper in a simple side by side analysis shows that our oral simple one daily drug has prior has superiority. 00:26:13 Speaker 1 With the non Bob by the fact that we are reaching delay of decline of minus point 1.783 much earlier than the banana map which has a -, 1.3 only and a much later time point which is 76 weeks. 00:26:34 Speaker 1 And this comes not at the cost of severe address events, which the antibodies carry, which is amyloid related imaging, abnormalities of edoema or a fusion observed with these antibodies. So we don't have that. And also with the much simpler. 00:26:54 Speaker 1 For once daily administration, the same is also the side by side analysis as simple comparison to Lacombe is similar feature. We are reaching A separation between active and placebo arm much at an earlier stage of time and which means that patients benefit much. 00:27:14 Speaker 1 Earlier in this case 24 weeks earlier, so very clear and exciting data of a a preference. We don't want to diminish the antibodies. They're very important that they started this industry, but it's also something to be aware of that there's never ever an overlap in the mechanism. So this could be also very good. 00:27:36 Speaker 1 Blacam is in a good drug to Co administration with the antibodies, if if that is of interest. 00:27:43 Speaker 1 So overall, the market of Alzheimer's starting to revamp even higher, we are now expecting in this projection that the growth of Alzheimer's will go over 150 hundred 30 million patients by 2050. And you can see from the slide, the ramp up is really scaling very highly. And again because of developed countries. 00:28:04 Speaker 1 Are the majority victims of this deliberative disease and deliberating disease? And because it's often combined also with lifestyle, of course. 00:28:15 Speaker 1 So that's why we're starting to explore potential commercial activities and engage in patients, providers and payers and the high demand for ultimate disease patients and families for easy access is in scalable treatment is really not to be underestimated and. 00:28:34 Speaker 1 And we of course want to reduce the need for this complex treatment, which are the antibodies, unfortunately right now are facing from what we hear from investigators in KLS, again, oral once daily blood comes in would be of course a convenient opportunity in comparison. If you look at the market size. 00:28:54 Speaker 1 It's really interesting to see that also that in the US alone 6.5 million, the antibody themselves can only potentially target a fraction of that amount because for the antibodies in their trial, they're required a almost a bottleneck or a enrichment of using patients only who had a several. 00:29:15 Speaker 1 Certain level of threshold of a better in the brain because the only mechanism of the antibodies is to reduce a better. So you need to have a better brain. Otherwise you can of course not administer the drug without benefit with benefit. So in our case our trial did not have that requirement. We measured a better in other. 00:29:36 Speaker 1 Marcus from baseline to end of trial, but we did not have that requirement because our mechanisms for the upstream and we knew from preclinical data that a beta is a part of our reduction of of the pathology including Tau and inflammation. So the physicians decided who will is eligible to the trial and for that reason we believe. 00:29:56 Speaker 1 The other existing 3 black comacine indication or drug has probably a chance to address the entire market of Alzheimer's disease because there's no limit in terms of diagnostic of the patient with Alzheimer disease. 00:30:11 Speaker 1 I want to also mention that we have strong patent protection and we own worldwide rights to our drugs and we also want to point out the separation between the oral once daily solution for the release franchise and the once daily liquid solution for the oil. 00:30:30 Speaker 1 For the Redis franchise and they are all solid administration for the Alzheimer pathology in Parkinson pathology. So you can very nicely separate those two franchises with different different doses as well as different branding. Again all the administered candidates have an immense potential. 00:30:51 Speaker 1 For clinical death benefit relative to costly and logistically challenges, biologically, antibodies and addressing also safety. 00:30:59 Speaker 1 So now let me start to closeout. We have now sufficient cash and we have over 150 million in cash as of last quarter. We also are benefiting room from non diluted funding from the Michael Fox Foundation from the Red Foundation and from the Australian Government. 00:31:19 Speaker 1 And so we have calculated that we have at least four years of runway as of today and the last year fiscal year 2023 required only a cash utilisation of 27.8 million. It might be a bit higher this year, but overall we have sustainable. 00:31:39 Speaker 1 Cache one way, due to disciplined operations and non dilutive cash sources. Last but not least all of this work hard work would not be possible without a dedicated team which we have would also like to specifically highlight our more recent joining our team which is kunjin who is very PEO of head of statistics. 00:32:01 Speaker 1 And he was 27 years at the new division at the FDA. Who is providing us with good insight and guidance on many features. Also, we have a very strong scientific Advisory Board, which consists of people you might recognise. 00:32:19 Speaker 1 And also I wanna with expertise and their guidance provided us where we are today and I'm very thankful for that. 00:32:27 Speaker 1 I also want. 00:32:28 Speaker 1 To closeout with the fact that we are sitting in our intriguing a platform which has the potential of transformation with this. With this precision medicine platform. 00:32:41 Speaker 1 We capitalised on significant market opportunities, we identified a precision medicine platform and noble Central Noble, central nervous system mechanism by improving the chance of success with these genetic genomic features with several different very promising therapeutic and very challenging. 00:33:01 Speaker 1 Areas of the CNS, again very complex diseases when you think about the ultimate pathology alone, I just read this morning another paper came out that a better in the patients is not equal to a better. They're different features of a better. So if you remove a better in some patients, it is more harmful than in others. 00:33:21 Speaker 1 So it's really very complex, this entire pathology and again, because our approach is more upstream, we think we can avoid this downstream micro management and approaches more macro upstream management. We have achieved multiple successful milestones so far and we're progressing also when we have sometimes a hiccup. 00:33:44 Speaker 1 With our robust data and commercial potential is now even highlighted with the progression into regulatory submission for unmixed 7 three black amazing in to the EMA in ultimate disease. And we also positioned very strong IP for the future and we own the commercial rights to our drugs. 00:34:05 Speaker 1 And glass note, but at least we have enough cash to move ahead. And that's again thank to the non dilutive funding which I want to thank at this point again today, Michael Fox Foundation, the International Red Foundation and Australian Government for supporting us so far, allowing us to have enough cash for the next foreseeable future. 00:34:26 Speaker 1 Including at least four years and last, not but not least, I want to closeout that anavex inherent advantage is this platform scalability, and this allows for equitable and accessible to diverse patient population and maintaining sustainability within global healthcare system. 00:34:47 Speaker 1 Which is something on a macro level. A big, big challenge. As we know, in All in all healthcare systems globally, not only the US but also Europe and as well as Asia as well with the lead. I'd like to thank for your attendance and thoughts Thursday morning, appreciate. 00:35:07 Speaker 1 Coming today, great crowd and thank you again and if you have more questions, feel free to reach out to us on www.anavex.com and stay in touch with us and we're looking forward to continuing the dialogue. Thank you very much. 00:35:27 Thank you very much and I. 00:35:28 Think with that, this concludes our. 00:35:30 Speaker 1 Presentation for this morning. Thank you all. Thank you.
Good to see the Odds Ratio nonsense that some fell for gone!
Going for a potential approval based on the totality of data showing responders with correlation to biomarkers has a chance. Which subgroup of responders and how large a chance of approval this board could endlessly debate, so we wait and see.
I think the longer we wait the sooner we get to know.
The Rett RWE videos are encouraging and anecdotal with no baseline or other references to relate them to. Also keep in mind that at least one of the Rett patients has a farther working closely with Anavex as the head of the Australian Rett Org. All good and is what it is.
Neither the P2b/3 or EXCELLENCE trial produced hard data pivotal results in the traditional to protocol manner - meeting all endpoints. Yet both could potentially based on subgroup analysis and less than gold standard outcomes be awarded some kind of approval.
Time will tell and Missling did a good job today of sounding enthusiastic and upbeat because he probably is.
Slide 6 on the JPM presention has subgroup analysis for ADL correlated with mRNA expression of SIGMAR1 p = 0.015 (no info on n) BUT IT IS FROM THE P2a PAPER!!!. Slide 7 still referencing Precision Medicine (by its classical definition). Nothing else of notice as I can see.
Of course yes the answer is a better balance between lifestyle improvements, both personal and systemic/environmental/social, and well targeted effective treatments for the rest with other causes that will always be there.
One question is then whether the A2-73 proposed MoA is upstream of those molecular difference in AD. Likely the factors of the five subtypes identified in the paper could be extracted from Anavex P2b/3 CFS samples and organised into factors for KEM analysis to perhaps further reveal/characterise subgroups of response to A2-73 .
Always new details of biological intricacy being worked out steadily increasing our ability to understand disease causes and ways of preventing and/or treating them. The tools available for research and analysis are getting ever smarter and capable of handling and navigating the enormous data relationship pool found in biology combined with external factors.
I personally think a focus on prevention would have by far the greatest pay-off across the so-called and clearly identifiable life-style diseases, but there are both personal and systemic challenges to resolve for that focus to pay-off. Meanwhile, drug upon drug appears to be the path of least resistance.