Anavex og CNS
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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
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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. -
Takker Tasso1 for den gode (som altid) gennemgang af den nuværende situation.
Ja enormt frustrerende med kursen, den siver stille og roligt hver dag, typisk bio-tek men forhåbentlig er ventetiden snart forbi (hmm det har vi vidst sagt før).
Her er lige en sjov en jeg fandt paa Investershub som snakker om nøjagtig det samme:
Funny how some investors of VBI Vaccines have said the same things that are happening here with Anavex:
"Most manipulated stock ever"
"Cabal"
"Shorts"Seems like all pre-emerging biotech follow the same path with Wall Street. The pump and dump cycle repeats itself over and over until approval of the compound.
You are 100% correct about the "love the company, hate the stock". Its a common theme in biotech investing. With that being said, you go to be in it, to win it.
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Anavex Nyt fra IR - med forbehold.
I just talked to AVXL IR (Investors Relations).
My first question was "will the Rett trial and data be PRed this month". The answer was an unhesitating "yes".
I mentioned that Christmas week was not a good time to release a good PR and he said "we are well aware of that".
Only 7 trading days left before Christmas week, I'm feeling more confident that we will see the Rett PR pretty soon after my discussion with IR, we shall see.
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Anavex Lidt premarket aktivitet + 11 %.
Kun 1400 aktier, men fordelt på 7 handler og alle over 5 $.
Der er sikkert ikke noget i det, men man ser meget sjælden handel i Anavex før kl. 13.
Måske et lille håb for, at vi får nyheder i dag!https://www.nasdaq.com/market-activity/stocks/avxl/pre-market-trades
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Anavex Earnings Announcement Before Market Open
Dog ingen pr. fra Anavex endnu - men kommer nok senest før premarket i morgen.
Er som sagt meget spændt på, om Anavex har begyndende indtjening fra deres SAS i Alzheimer i AUS.
Det ville/ burde give et stort boost til kursen. -
Endnu ikke verdens længste tråd.....
http://sidelinien.dk/forums/showthread.php?5318-Verdens-længste-trådDer skal efterhånden scrolles lidt, før man kommer i bund i denne, men vi mangler lige et par 1000 flere indlæg...
Håber på en god julegave fra avxl, men mon ikke vi skal et godt stykke ind i 2021 før det sker....
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Anavex Eli Lilly køber PRVL for 1 milliard $
1.000.000.000 $ giver Eli Lilly for et biotek selskab med forsøg i fase 1 og lige påbegyndt fase 2 i Parkinson.
Så meget for, at et selskab skal have varer på hylden, for at have værdi!Anavex er i fase 2/3 i både Alzheimer og RETT - og snart nok også i fase 3 med Parkinson efter det afsluttede fase 2 forsøg.
TDT jeg scroller aldrig ned - jeg søger på tasso1 og trykker på sidste indlæg og vupti er jeg i bunden:)
Men starter nok en ny tråd, hvis Anavex når en afgørende milepæl i nær fremtid. -
Anavex Når både primære og sekundære endpoints i RETT - SÅDAN ANAVEX!!!!
Anavex Life Sciences Announces ANAVEX®2-73 (Blarcamesine) Meets Primary and Secondary Endpoints in Placebo-Controlled U.S. Phase 2 Clinical Trial for the Treatment of Adult Patients with Rett Syndrome
Primary safety, pharmacokinetics and secondary efficacy endpoints met, with consistent improvements in RSBQ Total scores and CGI-I Efficacy endpoints demonstrated statistically significant and clinically meaningful reductions in Rett syndrome symptoms and correlated with changes in biomarker (glutamate) of disease pathology Key milestone met to advance regulatory approval pathway for adult patients with Rett syndrome NEW YORK, Dec. 15, 2020 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company developing differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) disorders, today reported top-line results from a U.S. Phase 2 randomized, double-blind, placebo-controlled trial of ANAVEX®2-73 (blarcamesine) in adult female patients with Rett syndrome. The primary endpoint of the trial was safety. The convenient oral liquid once-daily dosing of 5 mg ANAVEX®2-73 was well-tolerated and demonstrated dose-proportional PK (pharmacokinetics). Adverse events related to study drug were similar between ANAVEX®2-73 (13.3%) and placebo (10%), with no reported serious adverse events (SAEs). The safety profile of ANAVEX®2-73 in this trial is consistent with prior clinical trial data. All secondary efficacy endpoints of the trial showed statistically significant and clinically meaningful sustained improvements for ANAVEX®2-73 compared to placebo, consisting of the Rett Syndrome Behaviour Questionnaire (RSBQ) (p = 0.048) and the Clinical Global Impression Improvement Scale (CGI-I) score (p = 0.014) in the intent-to-treat (ITT) population (n = 25). Statistically significant differences in patient symptoms between the active and placebo groups occurred as early as 4 weeks following the initiation of ANAVEX®2-73 administration. Improvements in RSBQ Total scores were correlated with parallel decreases (improvements) in glutamate plasma levels. ANAVEX®2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of the sigma-1 receptor (SIGMAR1) is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.1 Consistent with previous ANAVEX®2-73 clinical trials, patients carrying the common form of the SIGMAR1 gene treated with ANAVEX®2-73 experienced stronger improvements in the prespecified efficacy endpoints. All twenty-five patients in this randomized study elected to enter a 12-week ANAVEX®2-73 extension study. Anavex will be advancing its Expanded Access Policy in order to provide long-term therapy to current participants with Rett syndrome under an expanded access program for ANAVEX®2-73. “The outcome of this trial is very promising in terms of both safety and clinical improvement. Despite the challenges of the older age of the cohort (patients were over 18 years of age) and the relatively low dose (5 mg daily), ANAVEX®2-73 demonstrated clinically meaningful improvements in outcome measures evaluating multiple impairments,” commented Walter E Kaufmann, MD, Principal Investigator. Subsequent to his appointment as Principal Investigator of this Phase 2 ANAVEX®2-73 trial in adult Rett syndrome patients, Dr. Kaufmann joined Anavex as Chief Medical Officer. He also said, “Moreover, the convergent clinical evidence was supported by parallel changes in a key biomarker of disease. This strong body of data opens the possibility of successful treatment for both adults and children with Rett syndrome and early interventions for modifying the course of the disease.” Based on the results in this first of its kind U.S. Phase 2 (ANAVEX®2-73-RS-001)2 study in adult patients with Rett syndrome, Anavex is planning to meet with FDA to discuss the approval pathway. There are no FDA-approved drugs for Rett syndrome. ANAVEX®2-73 has Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation from the FDA for the treatment of Rett syndrome and may be considered for accelerated approval. ANAVEX®2-73 is currently being evaluated for Rett syndrome in two other ongoing placebo-controlled clinical studies: The Phase 2 AVATAR trial in adult Rett syndrome (ANAVEX®2-73-RS-002)3 and the EXCELLENCE Phase 2/3 pediatric Rett syndrome study (ANAVEX®2-73-RS-003)4. “These are strong and consistent data demonstrating tolerability and rapid and clinically meaningful improvements in key measures of Rett syndrome symptoms in the ANAVEX®2-73 treatment group compared to placebo,” said Christopher U Missling, PhD, President & Chief Executive Officer of Anavex. I would like to thank the patients and caregivers who participated in this trial, the Anavex team, trial clinics, and doctors who have worked tirelessly on this program. Special thanks go to the Rettsyndrome.org Foundation, which provided financial support for this trial; we look forward to continuing the journey together." Summary of Top-line ANAVEX®2-73 (blarcamesine) Placebo-Controlled Phase 2 U.S. Rett Syndrome Trial Results The study evaluated the safety, pharmacokinetics and efficacy of ANAVEX®2-73 in 25 adult female patients diagnosed with Rett syndrome (positive MECP2 gene mutation). Effect on Rett Syndrome Symptoms: ANAVEX®2-73 treatment yielded a statistically significant, drug exposure-dependent response in the RSBQ Total scores, when compared to placebo, in the ITT cohort (all participants, p = 0.048).66.7% of ANAVEX®2-73 treated subjects showed a statistically significant improvement in drug exposure-dependent RSBQ response as compared to 10% of the subjects on placebo in the ITT cohort (all participants, p = 0.011).Improvements in this adult population with Rett syndrome, assessed by RSBQ Total scores, are considered clinically meaningful according to published criteria applied to neurodevelopmental disorders.5ANAVEX®2-73 treatment resulted in a sustained improvement in CGI-I scores throughout the 7-week study, when compared to placebo in the ITT cohort (all participants, p = 0.014).86.7% of ANAVEX®2-73 treated subjects showed a statistically significant CGI-I response, defined as sustained improvement to treatment, as compared to 40% of the subjects on placebo in the ITT cohort (all participants, p = 0.014). Safety and Tolerability: ANAVEX®2-73 was found to be well tolerated with very good medication compliance during the trial.All 25 subjects completed the study. The overall incidence of patients who experienced adverse events related to study drug, which were mild, or moderate was 13.3% (2) for the ANAVEX®2-73 treatment group and 10% (1) for the placebo group.No serious treatment emergent adverse events were reported during the course of the trial.There were no clinically significant differences in vital signs, lab values and EKG parameters between the active drug and placebo groups.Collectively, the study results are consistent with the known safety profile of ANAVEX®2-73.There was no signal for increased risk for common disorder-related manifestations. About Rett Syndrome Rett syndrome is a devastating, non-inherited genetic post-natal progressive neurodevelopmental disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child’s life: their ability to speak, walk, eat and easily breathe. The hallmark of Rett syndrome is near constant repetitive hand movements while awake. The disease is characterized by normal early growth and development (6 to 18 months) followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, autistic features, slowed brain and head growth, ataxia, seizures and intellectual disability.Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups. The disease occurs worldwide in approximately one in every 10,000 to 15,000 live births. The population of patients with Rett syndrome is estimated to be between 6,000 to 9,000 patients in the U.S. There is currently no cure for Rett syndrome. About ANAVEX®2-73-RS-001 Clinical Study (NCT03758924) The Phase 2 trial is a randomized double-blind, placebo-controlled safety, tolerability, pharmacokinetic and efficacy study of oral liquid ANAVEX®2-73 to treat Rett syndrome in a total of 31 adult patients with Rett syndrome over a 7-weeks treatment period were evaluated incorporating precision medicine biomarkers. Preceding the placebo-controlled randomization of 25 patients (Part B), a 6-patient cohort (Part A) underwent a 7-weeks pharmacokinetic (PK) assessment with safety, tolerability, pharmacokinetic and efficacy evaluation of ANAVEX®2-73. All patients who participated in the study were eligible to receive ANAVEX®2-73 under an open label extension protocol. About Rettsyndrome.org Rettsyndrome.org is the most comprehensive nonprofit organization dedicated to accelerating research of treatments and a cure for Rett syndrome and related disorders while providing information and family empowerment. As the world’s leading private funder of Rett syndrome research, Rettsyndrome.org has funded over $40M in high-quality, peer-reviewed research grants and programs to date. The organization hosts the largest global gathering of Rett researchers and clinicians to establish research direction for the future. Rettsyndrome.org, a 501(c)(3) organization, has earned Charity Navigator’s most prestigious 4 star rating year after year. To learn more about our work and Rett syndrome, visit www.rettsyndrome.org or call (800) 818-7388 (RETT). About Anavex Life Sciences Corp. Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental disorders including Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other central nervous system (CNS) diseases, pain and various types of cancer. Anavex’s lead drug candidate, ANAVEX®2-73, recently completed successfully a Phase 2a clinical trials for Alzheimer’s disease and a Phase 2 proof-of-concept study in Parkinson’s disease dementia. ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer’s disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson’s Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson’s disease. ANAVEX®3-71, which targets sigma-1 and muscarinic receptors, is a promising clinical stage drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer’s disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial dysfunction and neuroinflammation. Further information is available at www.anavex.com. You can also connect with the company on Twitter, Facebook and LinkedIn. Forward-Looking Statements Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company’s most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof. For Further Information: Anavex Life Sciences Corp.Research & Business DevelopmentToll-free: 1-844-689-3939Email: [email protected] Investors:Clint TomlinsonEmail: [email protected] Media:Melyssa WeibleElixir Health Public [email protected] _______________________ 1 Advances in Experimental Medicine and Biology Volume 964 (2017) Sigma Receptors: Their Role in Disease and as Therapeutic Targets.2 ClinicalTrials.gov Identifier: NCT037589243 ClinicalTrials.gov Identifier: NCT039414444 ClinicalTrials.gov Identifier: NCT043044825 Chatham et al. Adaptive behavior in autism: Minimal clinically important differences on the Vineland-II. Autism Res. 2018;11(2):270-283. doi:10.1002/aur.1874; Luu et al. Response to Placebo in Fragile X Syndrome Clinical Trials: An Initial Analysis. Brain Sci. 2020 Sep 11;10(9):629. doi: 10.3390/brainsci10090629
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Er spændt på hvad det får af betydning for kursen... man tør jo ikke rigtig håbe længere. Umiddelbart tænker jeg, at den nok skal falde... ellers passer tingene jo ikke!
En ny tråd ved milepæl er måske ikke så tosset. Min browser bruger i hvert fald god tid på automatisk at sende mig til bunden af tråden.
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Rett Syndrome News formidler ny viden til personer, der er i berøring med Rett Syndromet.
Resultatet fra fase2 med Anavex omtales meget positivt, og de fortæller, hvor man kan henvende sig, hvis man ønsker at deltage i de næste forsøg.
Det må skabe en efterspørgsel for at få medicinen godkendt hurtigt.Fik du nyheder ud af Årsregnskabet for Anavex, Tasso1?
Der er sket lidt med kursen, men den burde jo stige som en raket!
det ikke så længe siden at vi lå og rodet rundt mellem 2,5-3 $ ... nu roder vi rundt mellem 5-5,5 $ og det ikke så ringe.
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