Anavex og CNS
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Anavex Ja så godt den store stigning i shorts.
Synes det virker lidt vildt, når der kan komme resultater hvornår det skal være.
Kommer der både positive resultater i PDD og RETT inden for de næste par uger, kan det sammen med den relative store andel af shorts blive lidt eksplosivt - hvis da markedet fatter/anerkender værdien af resultaterne og begynder at køber aktier i selskabet.
Hidtil har shorterne næsten haft frit spil! -
Anavex Udmærket hurtig gennemgang af casen.
Han glemmer dog at fortælle, at Anavex har to næsten identiske RETT forsøg kørende i + 18 år - en i US og en i AUS og ikke kun det ene han omtaler.
Han nævner dog RETT 5-18 år.De næste par uger med de samlede resultater fra Parkinson og fra RETT US bliver ekstrem vigtige!
Spændende om der er nyt her til investor CC på torsdag - ville da være et godt udgangspunkt til at få nye investorer ind i selskabet.
Historisk set har Anavex dog altid leveret data senere end forventet - så kan kun blive positiv overrasket.
Vi ved også, at der kommer en lovpligtig årsrapport indenfor de næste par uger - mon ikke Anavex arbejder hårdt på, at man senest her kan leverer de vigtige resultater? -
Anavex Nyt omfattende patent for 2-73!
Rett syndrome, autism spectrum disorder, Angelman syndrome, cerebral palsy and multiple sclerosis, among other indications.
Data suggests, that activation of the sigma-1 receptor is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.1Min. til 2037.
Man skulle næsten tro, at Anavex selv tror på eller måske endda ved at skidtet virker!
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Anavex Stigning ved patent.
Her er et eksempel på stigninger efter tildeling af et omfattende CNC patent i en anden aktie (ATHE), som blev nævnt af Makingmoney lidt længere oppe.
Lidt mindre kan også gøre - og når det nu er Anavex skal vi vel være glade for den ikke falder på en sådan nyhed.Alterity Therapeutics stock more than doubles on massive volume after new patent announcement
Tomi Kilgore
November 16, 2020
The U.S.-listed shares of Alterity Therapeutics Ltd. rocketed 129.6% on massive volume in morning trading Monday, enough to be the biggest gainer among those listed on major U.S. exchanges, after the Australia-based drug maker announced the allowance of a new patent for next generation compounds to treat neurodegenerative diseases.
https://finance.yahoo.com/m/756a5d3f-a7bd-31aa-9825-76ab79d8c1a5/alterity-therapeutics-stock.html -
@Tasso1
vedr. patent på indikation, så kræves der kun, såvidt jeg ved, at man redegør for virkningsmekanisme. Så det er vel ikke absolut nødvendigt for Anavex, at have data som viser en effekt af lægemidlet?
Ved du om det koster et større beløb at ansøge om patent i US? Er det dyrt at søge patentet, så er chancen for, at Anavex har positive resultater nok større.Ja, ellers falder Anavex da flot på nyheden.... "business as usual" fristes man til at sige.
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https://www.usa.gov/federal-agencies/u-s-patent-and-trademark-office
Og lige denne, hvis nogle leder efter nye emner
https://www.biopharmcatalyst.com/calendars/fda-calendar
Market falder generelt, så de normale 5% må vente
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Anavex Boersboe og patent.
Må siger jeg ikke er helt inde i, hvor meget data og hvor omfattende en redegørelse for virkemekanismen skal være.
Ved kun at der må ligge et omfattende arbejde bag en patentansøgning og at tingene skal være veldokumenteret for at man godkender og får et patent.
Et eller andet sted må der fra Anavexs side være en drivkraft og en viden, som de mener er arbejdet værd.
Siger ikke den skal stige 150 % som ATHE, idet Anavex er meget længere fremme i udviklingen og har andre omfattende patenter, men at et godkendt patent på områder, hvor der til dels ikke er nogen behandling, ligefrem skulle mindske værdien af selskabet, som vi nu ser med et kursfald - er efter min mening helt absurd og lugter langt væk af manipulation på samme måde da vi fik gode data i Parkinson. -
Anavex CC Evercore
Live webcast 3. dec. kl. 18.45 DK tid:
https://wsw.com/webcast/evercore11/avxl/2380047CCen var kort og måske lidt overfladisk.
Missling kom næsten ikke ind på PDD forsøget - ikke andet end de var rigtig gode.
Intet om hvornår resten af resultaterne kommer.
Understregede at PDD og Alzheimer forsøget fase 2/3 har meget tilfældes.
Understøtter MOA - virkemåden af 2-73.
Forventer fuld indrulning af Alzheimer fase 2/3 i starten af 2021 - resultater nok først start 2022.
Ikke behov for at kigge på midlertidig data i Alzheimer, da man ud fra PDD sandsynligvis ved hvad data ville blive i Alzheimer.RETT US er på lav dosis 2-73.
Rett AUS er på høj dosis
RETT international ( 5-18 år ) er på høj dosis.
Positivt at fraværet af bivirkninger tillader fuld dosis til børn.
FDA vil meget gerne se en dosis afhængig respons.Sammen udgør de sandsynligvis nok til ansøgning om godkendelse.
Data fra RETT US bekræftes inden udgangen af 2020!
Ellers var der ikke meget nyt - vi må bare afvente når de sidste data fra PDD og RETT US kommer indenfor de næste uger.
Der var til gengæld ikke noget negativt og slet ikke noget, som skulle give anledning til et kursfald til 5 $ området.
Spillet/manipulationen med kursen forsætter til vi har vandtætte resultater. -
Tak for resume Tasso1. Ja utroligt at den bliver banket under 5$ igen, på en omsætning under gennemsnitlig volume, kunne være dejligt hvis Missling denne gang tog fusen på markedet og smed et positivt RETT resultat afsted i morgen tidlig
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Jeg har lige lyttet til web cast og synes heller ikke at der er noget at værre bekymret for.
Ja skuffende at vi er under 5$ igen men tror det var investorer med kort tids horisont som havde håbet på "breaking news", der solgte ud efter CC.Jeg beholder alle mine aktier.
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Anavex. Udskrift fra CC Evercore
Selve webcastet udløber typisk efter en mdr.
Quote:
Everyboty, thak you for joining us. We are We were hosting Anavex and we have the pleasure of hosting CEO Christopher Missling.
So Chris, please introduce yourself and give us a quick overview of where the company is headed and what's in store.
Missling: Sure thanks again for inviting first of all and great that you are doing well so Anavex - myself I'm a CEO and president of Anavex Life Sciences. We focus on the diseases of CNS, those degenerative diseases as well as developmental diseases and we have several programs in our pipeline, among them the one which is near term is new developmental diseases a rare disease called Rett Syndrome with three ongoing studies, two adult studies and in two different doses. In one pediatric study. We expect reading out of the first study very soon. And then we have (a new developmental diseases program/corrected) in the degenerative diseases program in Alzheimer disease as well as Parkinson and Parkinson Dementia which we reported just recently with very exciting data.
Moderator: Great and excellent and before we dive into each of the various programs Anavex has perhaps you could provide a brief overview of blarcamesine and this overall Sigma one receptor pharmacology and its relevance to the various indications that Anavex is pursuing.
Missling: That's a very good question, so simply one is expressed in our body and is actually has no function, immediate function of day-to-day activities, for example if you knock it out the animals don't have any different life, unless they get very old and that's where we see that there is an accumulation of genetic dysfunction or just aging, and these considered disorganizations or hetero dysfunction will cause the system to start to cripple and what we have seen that the Sigma one is the (response of the jaw) endogenous response in the body to counter this homeostasis imbalances and since this is a GC coupled receptors Sigma one receptor we can activate it as it does it is also happens endogenously with external Sigma one small molecule agonists Anavex 2-73 or blarcamesine is one of them.
Moderator: Got i. t I mean it's, it's the whole mechanism is fascinating and I ask that question because it lies so upstream of various intracellular pathways and processes and at least preclinically has demonstrated evidence of neuro-protection so because it lies so upstream and has a lot of touchpoints into various biological processes, can can you be to selective for Sigma 1 receptor? Is there like a sweet spot or a optimal affinity that would be desired from that S1R agonist?
Missling: Yeah so an excellent question, I think that the whole area of Sigma 1 is really evolving and there's no week which passes by where we have new data confirming that and we constantly check also every time there's a publication about this intriguing protein and gene coming out if it's consistent with we have but we have observed, and so far it was always consistent and I think the way to look at that is that the upstream effect is the best analogy is a bit like immunostimulation oncology when you look at the history of oncology the really remarkable breakthrough came really with 99% survival rates not with the blocking the cancer directly but or blocking a pathway of the cancer but with the activation of the body on defense mechanism. In a way this (Sigma 1) activiation is a similar analogy to activate the body own defense mechanism.
Moderator: I see I see now that's nice background thank you Chris. Just now we keep moving and delve right into the programs. Perhaps one of Anavex's is most exciting programs is the Rett Syndrome program. Right now yeah, I know you have two phase two trials that are about to wrap up and possibly read out relatively quickly, and on a larger pediatric trial. So if you could just please just reaccquaint us with the the background on those trials and what we've seen so far.
Missling: Right so the three studies Alec, I wanna go through it in sequence of also read out. So the first study is the US study in adult Rett patients with a low dose. So we started with that because it was the first time we gave this to these Rett patients which are a very debilitating population of girls which have no cognitive features, very weak cognitive features, congnitive impaired, but on top of that are also have high level of anxiety, movement impaired they cannot walk, and have also respiration problems and other features of this function which are all caused by genetic dysfunction of one protein mecp2 which is a developmental gene in sort of since birth that is basically carried across their life. So that is the first study the adult Rett study with low dose Anavex 3-73 or blarcamesine that will read out this quarter and we are within the timelines of when we announced. The adult international study also called Avatar study is a very similar to the US Rett study in adult patients with the difference that the doses are higher so we call it the high dose study so of the ideas behind to have two studies placebo control and having one low and high dose to have the what the FDA often requests and likes to see a dose response if you select, and that's expected to read out in the first or second quarter of next year and then the pediatric study which is also a high dose study is will be reading out in the second half of next year.
Analyst: Chris, if I may step in, the the adult Rett study, one of them our understanding was wrapping up in October time frame, is that, has that completed is that data in house but being processed?
Missling: That is now in process in respect to announcement very soon once we have the data at hands. So we'll update and provide the top line data of that study, that's what I call it, this quarter. so it's given it basically.
Analyst: And when did that read out in mid October internally?
Missling: it didn't read out we just had the finish the trial and then you have to do the clean the data clean up so that it out now and it will be forthcoming.
Analyst: Aand what about the avatar? Sorry, Avatars recruiting and and then you have a second trial an adult also wrapping up which is a much bigger study. when is that?
Missling: Yeah so it's a bit like I think you mixed up quickly, the US study is not called Avatar U.S..... and the Avatar studt is the international guided study which results in first half of next year. (I got it) The pediatric study will read out second half of next year.
Moderator: And Chris, if I'm not mistaken, that larger pediatric study, that's not required for registration and that these two more near term adult studies even though they're early studies they could possibly be accepted ..or..or for registration is that true or how's the FDA thinking about that and what have you heard?
Missling: Yes so we have with the FDA a fast track designation for Rett with a Rett program and we have orphan designation and we also have pedriatic voucher ability but the two adults study you're correct if they have a consistent readout, we awfully, because there's no treatment available for this patient so far including the adult population rett syndrome is actually the potential depending on the data to discuss with the next steps. I also want to mention one thing which is important in developmental diseases it's often found that in your developmental diseases it's often hard to change the features of adult patients. So once they are basically above 18 something very difficult to change their trajectory or their phenotype so often you find studies not working out in adults of that same indication but when you take younger population they then have a very strong or strong signal and that shows what I'm trying to explain that often more advanced stage is impairment for positive readout. Having said that when we find signals in adult patients in this Rett Syndrome studies this will work be a very good you know encouraging signal for the younger study population.
Analyst: Chris sorry I I don't need to backtrack a little bit here but for the trial readout that's coming up any day could you remind us where are investors and where you guys most focused in terms of efficacy endpoints? Is it on the questionaire or on the CGI-I endpoint? And also if you could specify whether the base cases could possibly shorting??? the mutation subset knowing by the way that the sample size is small that status P values may not necessarily be something we should be focused on.
Missling: Yeah excellent point so the total patient size is 25 because six patients were already covered in the PK over label out of the 31 so the total cycles in patient is 10 placebo and 15 active arm so the total of the placebo cohort is 25 in total and the measures we're looking at is exactly what we have seen, what the the FDA is requesting, we are looking at the RSQ which is the major you know endpoint which is a more rigorous you know questionnaire of 45 questions and capturing very nice is entire spectrum of the Rett features which are as broad as anxiety to sleep to movement to seizures and reaction and so all these features are very nicely consistently captured in this questionnaire. But also you mentioned the CGI-I which is part of the study but also the atoms(?ADIS?) is measured which you might know the the anxiety and of news in an autism spectrum disorder measure. So I would think that into us the second question, I think what we're looking for in this first study cause also it's adult studies or older studies and what I just described before is most likely having a stronger signal in younger patients, I think that important notion will be the ability to demonstrate consistency between those measures because some of them overlap. For example there are certain scores in the atoms(??) domain, subdomains which overlap with the RSQ you so if you have their consistency that would be extremely encouraging and also if you have statistical significance in one of those endpoints that would be more than expected.
Analyst: Got it so you think the efficacy signal might be more pronounced in a younger population but your goal that you're looking for is consistency across those endpoints and what about the mutations do you expect efficacy broadly or perhaps only in a patient subset and how many patients is that, with mutations?
Missling: that's an excellent question here comes something which confirms the mechanism of action of our drug, which is of the extremely important, because that will carry all the other indications which we thinking of going after like fragile X, infantile spasm, and also Amgleman's syndrome. We have very very consistent and great preclinical data in but the key clinical intelligence out of the genetic separation multiple genetic analysis was deceitful one wild type is shown the strongest response in both the ultimate study as well as the PPD study in Parkinson's dementia study so now we will see in the Rett study that is where we also have pre specified so it's not a post hoc analysis, it is prespecified analysis of active against placebo of the common wild type Sigma 1 gene and if that also shows stronger than overall or the variant which is only a minority then we would have really shown clearly a manifestation at first of all the mechanism is truly confirmed because otherwise we would not be able to show that and Secondly we would be then able in the future to consider to maybe even focus on only this wild type population which the good news is that it actually represents the majority of the patients, you talk about 80-90 percent versus the minority which is very which is variant carriers.
Analyst: Got it but of the 25 patients so 15 or an active arm, ten on placebo how many of the 15 will have the mutation.
Missling: we don't know yet but we if we use published information it should be in the range of 10 to 15 to 20% so if it's
Analyst:10% of 15 so 1 1/2 patients
Missling: right yeah that's that's that's what the statistics, the public database discuss from information in the ....
Analyst: Si two to three patients and active arm might have a mutation.
Missling: we will see one thing is so interesting in the Optima study we had 25 out of the 20 which had the gene measured 5 have the mutation 15 didn't and in the PD study it was a similar ratio so we might see different here in the Rett study we will see.
Analyst: Got it... and Chris my last... one I'm sorry my God I wrecked your your change ??. My last one is Alzheimer's study, is there an interim analysis on 1st first subgroup of patients which only it has to go past certain thresholds? I understand that's the case with most Alzheimer's trials.
Missling: so you saw that what happened with Adu (BIIB drug?) with the interim analysis? You know? We decided because you also losing power and we had a very strong signal in the phase 2a, we don't, I think we had it in our protocol as a as a possibility, but we can still use it, but what we have thought about right now is the closest to interim analysis is actually intelligence out of the Parkinson's dementia study which the same doses as the Alzheimer's study and is very clear signal in one of the domains of the CDR system which is the episodic memory which has been published in 5000 patients to be consistent with 70% over consistency with the other score which is our primary endpoint in the Alzheimer's study which by the way is enrolling very nicely and we expect enrollment to be completed early next year so that is our closest to an interim analysis at this point. But we might still do interim analysis but right now we have not decided on that yet.
Analyst: Got it
Moderator:and just....just in the final minutes if we just kind of just stay on Alzheimer's and I know you had some promising phase two a study data where in the exploratory endpoint you set you saw some some significance on MSE an active daily living. Tat said given that ... your phase three trial is a mix of mild and prodromal patients it's only 48 weeks long, is that long enough? But for the patient mix?
Missling: That's an excellent question, so we have seen in the phase 2a way that patients in the baseline below 20 MMSE we found that we could get them not to decline over 48 weeks and the patients were above 20 MMSE were basically milder. We were able to show that some of those patients with the right concentration work which are the doses now tested in the trial, will be able to show them a net improvement which by the way we also saw in the PDD study that the high dose 50MG dose showed a net improvement in the measure of episodic memory which correlates 70% with the ??? so our expectation of the is that 48 week should be sufficient because we want to separate a strong as much as possible to consider noise (BREAK IN AUDIO HERE) Noise I still have a good gap two possible
Analyst: Got it
Moderator: Question to wrap up when can we expect this to end and get top line data so the the ultimate study will be enrolled early next year and then we have to add 48 weeks so early 2022 I guess is the right estimate for that reason see listening into the time I think we're up on the hour but thank you so much Christopher this has been very informative we really appreciate appreciate you speaking with the center conference and we hope to continue the discussion likewise thank you again
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https://piotrpeterblog.com/2020/12/07/rett-syndrome-data-already-out-anybody-pays-attention/ Rett Syndrome Data Already Out - Anybody Pays Attention?
Posted on December 7, 2020 by Piotr Pietrzkiewicz -
Han skriver i slutningen: Remember, I can tell you what Dr. Missling can not. I am looking into the future using clues from the present. I can be wrong but most likely I am only wrong in matters of details not the shape of things.
Nu håber jeg virkelig at vi snart ser nogle data. En prominent skribent på Stocktwists AVXL tråd har solgt alle hans AVXL aktier og skriver at der sker ikke noget med aktien før sidst i 2021 og det har han ikke tid til at vente på men regner med at gå ind igen på et tidspunkt. Han virker noget impulsiv synes jeg og er nok mere swing trader end langsigtet.
Jo det trækker ud men for mig ser det ud til at Missling opbygger hele puslespillet hvad Blarcamesine kan gøre mht RETT-PAR-ALL og det kan pludselig gå rigtig stærkt så jeg toer ikke selv sælge ud og vil i hvert fald vente indtil RETT er kommet ud og ta det derfra. -
Jeg har selv solgt 1/3 (også i damens potte) grundet Misslings håndteringer, samt udtalelsen om at det er mindre sandsynligt med gode resultater i "voksen RETT" end i børne versionen (giver egentlig god mening). Samtidig har RETT hele tiden været den indikation jeg tror mindst på. De manglende tal fra PDD (placebo gruppe mod modtagere) gjorde det ikke bedre. Når man går ud, skal man jo have et andet sted at gå ind, et sted man tror mere på. Det er jeg så ret sikker på jeg har fundet (i forhold til tidshorisont). Jeg har stadig mange, men har altså, hvis man skal være helt nøgterne, flyttet min risiko et andet sted hen SMILEY.
Pøj pøj til os
MM -
Mens vi venter på Missling!!
Heldigvis er der deadline på Q4 og Årsrapport d. 16. dec.
Får nyheder fra Rett Syndrome News, hvor der er en meget positiv omtale af Anavex:
https://rettsyndromenews.com/2020/12/04/anavex-2-73-treatment-rett-syndrome-other-neurodevelopmental-disorders-to-receive-new-us-patent/Igen stor tak til Tasso1 for at holde os informeret, sidst med den lange transkription af Missings oplæg.
Jeg synes stadig Anavex 3-73 lyder ekstremt lovende og forbliver lang i aktien selvom det tager sin tid at få de store professionelle investorer ind i casen og shorterne ud! -
Anavex Video gennemgang af RETT US forsøget.
I august 2020 havde Anavexs Walter Kaufmann ( Mr. RETT ) et indlæg vedr. resultaterne fra RETT US for de første 6 piger.
Det er dette forsøg vi jo afventer data fra for de resterende 25 piger ( 15 med 2-73 lav dosis og 10 placebo )
Han understreger, at der trods den relative lave dosis og korte behandlingstid ( 7 uger ) er opnået signifikante resultater i samtlige målepunkter.Anavex skal sådan set nu bare eftervise tilsvarende resultater for de sidste 15 piger.
Sammen holdt med det tilsvarende RETT forsøg med 30 piger i AUS og UK på høj dosis, der forventes afsluttet 1. eller 2. kvartal 2021, har man sandsynligvis tilstrækkelig data til at FDA kan tage stilling til evt. markedsgodkendelse for aldersgruppen + 18 år.
Både fra Parkinson og Alzheimer data, har vi set høj korrelation mellem dosis og respons.
Det internationale RETT forsøg med 69 patienter 5-18 år, 12 uger og høj dosis, der forventes afsluttet 2. halvdel i 2021, kan evt. så danne grundlag for en markedsgodkendelse i denne aldersgruppe.
Anavex har tidligt udtalt, at man vil se størst respons ved at komme tidlig i behandling.Så forventer følgende resultater fra de 3 RETT forsøg: God, bedre og bedst!
Uanset gode resultater i Parkinson og Alzheimer, er hurtigste vej til markedet nok via. RETT indikationen.
Til årsrapporten/CCen inden senest den 16. december 2020, er jeg overbevist om, at Anavex vil gøre alt for, at fremvise data på RETT US eller mere på Parkinson ( forudsat at de er gode ). Alzheimer fase 2 b forsøget med 21 patienter er også afslutte her i november.
Er meget spændt på om der figurerer en indtjening fra AUS, som følge af deres tildeling af SAS i Alzheimer tidligere på året og dermed i princippet oplyse aktiemarkedet, at Anavex ren faktisk er på markedet med 2-73 i Alzheimer!Vi kan kun afvente, men at aktiekursen nu er under det vi havde før de foreløbige super gode resultater fra Parkinson forsøget er efter min helt absurd.
Men med en daglig omsætning og handel på ca. 1 % af de udestående aktier, er der efter min mening frit spil til at manipulerer med kursen.
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