ANAVEX NY TRÅD JULI - PRÆVENTIV BEHANDLING AF ALZHEIMER!!!
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Lidt mere fra IHub
boi568 Tuesday, 08/10/21 02:34:22 PM
Re: Investor2014 post# 325174 0
Post # of 325246"Had only planned the PDD study as safety and PK information to decide whether a pivotal study would make sense, hence short 14 week study. Did not plan in advance to have 48 week OLE, but now very useful.
"He mentioned that the PDD ADS-COG 70% correlation to AD along with the SIGMAR1 biomarker results clearly stronger than the signal that got Aduhelm approved."
These comments reinforce further the force of the mRNA biomarker findings, something that Missling was not expecting to find (or he would have designed the PDD study differently).
I want to examine the PDD/AD 70 percent correlation remark in context. Missling is not going to be using a potential PDD NDA to promote the approval of the ongoing AD trial, obviously, although it does give a strong signal that the AD trial will also succeed. The fact that it may work in AD the same as PDD doesn't make the PDD case, alone, stronger. Instead, taken together the correlation and the validated biomarker strengthen his argument to the FDA that the mRNA biomarker, correlated for efficacy in both PDD and Rett 001, is very likely to do the same for Alzheimer's and all other CNS diseases that may involve the Sigma1 receptor. Missling is attempting to get the mRNA biomarker accepted as a CNS-wide biomarker that will ease his path in all future CNS NDAs.
Very useful report, Investor. Thanks for posting.
Det bliver godt nok spændene på torsdag, desværre sover jeg når Missling er på.
Kursen burde begynde at stige nu, det som de skriver er jo helt fantastisk og det vi håbede på.
Ja lad os håbe at Anavex allerede er i snak med en partner og de kan lave et samarbejde som begge parter er tilfredse med rent økonomisk.Kurs 18.5$ er latterlig lav efter min mening, vi burde værre over 40 men alle snakker om AD (Alz) og vores data derfra er med meget lavt n så ingen tar det alvorligt, endnu.
Som Boi skriver og Missling sagde ovenfor er PDD omkring 70% "beslægtet" med AD så gode data fra PDD lover rigtigt godt for AD og alle CNS sygdomme som involverer Sigma 1. -
Use Weakness in these 3 Alzheimer’s Disease Stocks as Opportunity
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InvestorPlace (investorplace.com)
Ian Cooper er en 5 stjernet finans blogger
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Det syntes rimeligt klart at man er i tæt dialog med FDA. Man har kompetencer med fortid i FDA, og det bestyrker mig i casen.
Der hvor jeg er mest søgende er på partnerdelen. Det er afgørende at få valgt en partner som har musklerne og viljen. Det vil være stærkt risikabelt at afvikle tre fase 3 studier uden en partner. Indikationer i den retning vil jeg lytte meget opmærksomt efter.
Begynder de dele at flade i hak, så er det komplet underordnet om prisen nu er 15$ eller 20$. Den skal nok komme op. Langsigtet i helt andre niveauer.
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Måske Takeda er mulighed, selv om de har andre samarbejdspartnere på området.
Men de havde vist tidligere noget med Zinfandel som de måtte lægge i graven.
CNDAP Partners with Takeda to Develop New Therapies for Alzheimer's Disease and Related Brain Disorders
/PRNewswire/ -- Cure Network Dolby Acceleration Partners LLC (CNDAP) and Takeda Pharmaceutical Company Limited ("Takeda") have entered into a research...
(www.prnewswire.com)
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Anavex Ny august præsentation.
Side 3:
ANAVEX Platform for Neurological Diseases.
(Dette er ikke en enkelt behandling, men en hel platform)Side 6:
ANAVEX
2-73 Establishes SIGMAR1 mRNA Predictive Biomarker of
Efficacy in Alzheimer's, Parkinson's and Rett Syndrome
(Fællesnævner på tværs af alle indikationer)Side 41:
OVERARCHING MESSAGE
A novel platform approach to address the
totality of CNS diseases
(Totality of CNS diseases!!! - store ord)Hvis man skal tror det udmeldte fra BTIG mødet, så er deres oversigt for pipelinen ikke opdateret endnu.
Dette kommer nok i forbindelse med Q3 på torsdag.Kørende og planlagte fase 3 forsøg med 2-73:
AD fase 2/3
AD præventiv fase 3
RETT fase 2/3 + 18år
RETT fase 2/3 5-18 år
PD fase 3
PDD fase 3
Fragil X fase 3
Ubenævnt sjælden indikation fase 3DVS HELE 7 forsøg i store og mindre indikationer, med en potentiel godkendelse i den sidste ende - og så i områder, hvor der ingen behandling er i forvejen.
- Alt dette formentlig afstemt i samarbejde med FDA!
(Der må godt nok være en varm tlf./mail linje mellem Anavex og FDA!)
Deres MOA og biomarkører har vist sig at hænge sammen på tværs af indikationerne - og vil i så fald bekræfte/afkræfte en effekt i rigtig mange CNS indikationer.
Hvad skal et sådan selskab prissættes til? - i hvert fald ikke tilde kun nuværende 1,38 mill. $. - og er det muligt for et så relativt lille selskab som Anavex at håndterer og financierer denne voldsomme udvikling, uden at have en stærk partner med inde over?
Shorterne har reduceret med ca. 1 million de sidste 2x14 dage og er nu gået fra ca. 8,7 til 6,7 millioner.
https://www.nasdaq.com/market-activity/stocks/avxl/short-interestNå - Spændende om Missling overrasker os til en afveksling og lukker op for posen i morgen torsdag kl. 13.00 og senere på selve webcastet kl. 22.30:
- Alt dette formentlig afstemt i samarbejde med FDA!
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Anavex. Q3 Rapport.
509 mod forventet 450 patienter blev indrullet i Alzheimer fase 2/3 forsøget - resultater 2. halvår 2022.
157,6 mill. $ på bogen - betydelig mere end forventet (ca. 40 mill. $ mere).
RETT fase2/3 Avatar + 18 år har også indrullet flere patienter end beregnet - resultater i 2021.
Ellers ikke meget nyt i denne rapport - håber på uddybning og gode spørgsmål fra analytikerne i aften.
Bl.a kunne det være spændende at høre, hvordan de ekstra penge skal bruges. -
Anavex Life Sciences Provides Business Update and Reports Fiscal 2021 Third Quarter Financial Results:
Højdepunkter:
Total of 509 Patients Enrolled in Confirmatory Late-stage Precision Medicine ANAVEX
2-73 (blarcamesine) Phase 2b/3 Clinical Trial in Patients with Alzheimer's DiseaseExceeding of Enrollment Target for the Precision Medicine ANAVEX
2-73 (blarcamesine) Phase 2/3 AVATAR Clinical Trial in Patients with Rett SyndromeCurrent Cash Position of $157.6 million
Okay så de har udvidet AD 2/3 forsøget til over 500 patienter. Det er nok for at værre mere sikker på godkendelse og mere nøjagtig data måling.
157.8 Mill$ i kassen, ser ud til at de har diluted lidt, nok flere forsøg i kikkerten.
Nok derfor at kursen er sivet noget over den sidste tid, men helt normalt i Anavex's situation.Flash Alert
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(flashalert.me)
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Supplerede yderligere igår, skulle nok have ventet til idag. Tænker den ryger 1-1,5 $ ned idag grundet den lidt fesne (i.f.t markedets altid store forventninger om nyheder) kvartals rapport.
Pøj pøj til os
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Anavex Webcast blev afbrudt og kom igen.
Alt ser godt ud!!
Igen rod med lyden i webcastet!
Der plejer at komme en udskrift fra Seeking Alpha inden for en dags tid.
Håber I andre kan formidle denne, da jeg skal ud at rejse.De vigtigste ting jeg nåede at fange:
2-73 har virket dosisafhængig i samtlige forsøg hidtil!!
Imaging forsøget betalt af MJFF bliver kørt!
Der kommer åbenbart endnu en Peer Review i forbindelse med opstart af Fragil X fase 3 forsøget. (Er dog ikke sikker på, om det ikke er den vi allerede er blevet stillet i udsigt for RETT?)
Man vil bruge Parkinson fase 2 forsøget til at gennemgå samtlige gener for RNA og MRNA, for at kunne vurdere, hvilke indikationer der er bedste egnet til nye forsøg med 2-73.
Missling er ud fra de ekstrem gode prækliniske forsøg overbevist om, at 2-73 vil kunne virke præventivt og før man udviser symptomer på Alzheimer!
Ja endda præventivt på stort set alle CNS indikationer!
(Dette er fuldstændig vandvittig, hvis dette bliver tilfældet - meget medicin bliver fremover overflødigt!!!)Endvidere har de også pengene på bogen til at agere og uden at skulle ud og lave større udvanding - dette hænger ellers altid som en skygge over disse mindre biotek selskaber.
Nå - der må følges op på tingene, når udskriften eller hele webcastet bliver tilgængelig.
Men igen - alt ser virkelig godt ud.
Tingene tager dog relativ lang tid, da Anavex er ekstrem grundig og vil have hele fundamentet på plads, så man kan designe kommende forsøg optimalt, både i de nuværende indikationer, men også i nye fremtidige indikationer. -
Anavex Komplet webcast og Q10 rapport.
Rigtig god webcast!!
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Anavex Fint resume fra IHUB af Q3.
MayoMobile:
Anavex 2021 Third Quarter CC
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509 patient AD 2b/3 top-line data expected in 2H 2022
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Restoration of complete housekeeping function in the body
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Anavex is continuing to pioneer approach of big data to leverage holistic genomic sequencing and biomarker. This increases chances of success in our trials.
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AVATAR enrollment was exceeded and expected top-line in 2H 2021.
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Enough cash for beyond 2025 (157 mil)
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Q: what do you think of the RS-001 Rett trial patient size, what about AVATAR size?
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A: U.S. study was a small trial with low dose, but we garbed large effect sizes. In AVATAR, dosed are higher with larger patient population. We are expecting a good dose response curve and expect good data from AVATAR.
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Q: is the pediatric (EXCELLENCE) study being slowed down?
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A: we are still enrolling and can't say for sure at this time how much impact new COVID restrictions will have in the long term. As of now, projections for trial completion are correct.
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Q: any further information IRT enrollment on AD phase 2b/3 into OLE?
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A: very impressed by very high rollover rate. Over 95% in fact. We have been informed that patients are reaching the end of the OLE, and are requesting additional time towards further extension.
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Q: is AVATAR study large enough? Any additional information?
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A: in dialogue with agency, have multiple FDA indications (orphan and fast track), also have voucher. Will update regulatory information when possible. Keeping exact high dose undisclosed at this time to prevent possible unblinking due to tight knit patient population.
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Q: baseline MMSE for AD 2b/3?
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A: patients above 20 MMSE did much better in the AD 2a. These patients are able to comply with trial regimen better, and efficacy is higher. Early Alzheimer's is the highest unmet need for cognition today, and this just so happens to address that patient group.
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Q: preclinical information on blarcamesine and its protective role? Which indication may be addressed best for protection?
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A: in preclinical animal models, blarcamesine was able to completely prevent cognitive impairment as compared to untreated rodents. Blarcamesine is upstream and can prevent the cellular stress and therefore these negative effects. Earlier phase patients would likely respond better towards preventative medicine if caught early. Potential to treat even before symptoms are present, much like a mini/aspirin for cardiovascular problems. We will one day be able to tackle that and we are planning such a study.
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Q: Rett trials and applicability to Fragile X?
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A: ADAMS is a secondary score in Rett and is normally primary in Fragile X trials. It was overwhelmingly positive in the RS-001 Rett trial. Peer review paper expected soon. This is a great indicator towards success in the Fragile X trial - Fragile X being the largest need in autism spectrum disorder. Symptoms are very similar between the two disorders.
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Q: phase 3 prevention study status?
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A: need to discuss further with regulatory agency. No details at this time. Seems to elude to the trial including broad cognitive indications such as Alzheimer's, Parkinson's, and other CND disorders in a single trial. Need to discuss with agency before providing more detail.
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Q: any more about post-PDD trial?
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A: the trial itself has finished but analysis is ongoing. Working analysis other than primary and secondary endpoints. Intel on RNA and whole genome which is being put together right now and will create an ultimate package for regulators which will totally fuel our 2b/3 PDD and PD trials.
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Q: is the MJFF imaging study going well, any other details?
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A: starting it this year, will detail blarcamesine in the brain of PD patients.
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Q: any information on the undisclosed indication? Any info on the 3-71 trial?
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A: several animal models have been positive towards this undisclosed indication. Ultra rare disease. Before we move forward we want to make sure we are choosing the right indication as there are multiple available to choose from. 3-71 is in phase 1 now and data is expected this year. First trial will be FTD or a different indication. The 3-71 is a safety trial. (Missling leaves it open to suggestion that there could be more than just safety data. Perhaps he is referring to genome data). Anavex has been efficacious and has dose response curve in all trials. All trials have also had very strong predictive biomarker response.
Overall a very positive conference call. To me the most important points are related to the ADAMS test for Rett/Fragile X indications. In regards to effect size, ADAMS scored highest on the RS-001 trial with 1.31 very large effect size. This seems to indicate Fragile X will be a home run. Also, Missling confirmed that for now (pending regulatory guidance) the preventative trial is being planned to address CNS-wide indications (Alzheimer's, PDD, etc.).
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Her er udskriften fra Seeking Alpha.
Jo jo det ser mægtigt godt ud synes jeg men det tager tid fordi Missling ikke overlader noget til tilfældigheder hvad der er fint, det er bedre at gøre det rigtigt i første omgang så der ikke kommer flere forsøg som vil trække det hele endnu længere ud!
En af dem der stiller spørgsmål; Tom Bishop er også meget positiv på Anavex's pipe-line/data:
Tom Bishop
Okay, well, you know, I've been very encouraged by the breadth of the positive readouts, you're getting on a variety of indications. And I think that helps support the idea that 2-73 really does something in the brain, which I think you have a little doubt of, and I do too, but it's just good to see so many different positive readouts coming. Thank you.
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Anavex Skidtet virker i alt hidtil!
Misslings afsluttende kommentar kan næsten ikke sige det mere tydligt!
" Christopher Missling
If I might add, it is really important to notice. And to highlight the fact that in all clinical trials we have performed so far, not only was ANAVEX 2-73 efficacious at the right doses, but it was also demonstrated a dose response curve, which is always a very clear indication of our effect. And thirdly, we have noticed that all the data, all the trials so far performed, had a very strong biomarker of response or predictive biomarker response, which was borne by the by the level of mRNA expression of the target of our drug itself. So there's really no better way of showing efficacy and confirming efficacy of a drug with these strong biomarker outcomes, which correlated with all the primary and secondary endpoints of the drugs we have performed."
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Anavex - MayoMobile med et interessant indlæg
Anavex Life Sciences Corp (AVXL): Where is the PDD Data? It is likely...
MayoMobile: Where is the PDD Data? It is likely Anavex anticipated full data to be finished by the end of 2nd quarter 2021; however, genomic sequencing probab...
InvestorsHub (investorshub.advfn.com)
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Anavex MayoMobiles indlæg stemmer meget godt med, at en på Ihub for noget siden, der påstod, at Peer Review artiklen i PDD var trukket tilbage under redaktionen for at gennemgå en større ændring.
Stemmer også med Misslings udsagn om, at de har videnskablig påvist Blarcamesines effekt gennem biomarkers.
Måske også en forklaring på at AD forsøget blev udvidet, idet der måske er fejldiagnosticerede pts indrulleret tidligere. Det fremgår, at der fejldiagnosticeres i AD, PDD og Lewi Body. Missling udtalte også at de gjorde sig umage med at få de rigtige pts med.
Spændende når vi hører nærmere her i efteråret.
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Anavex Udkast Peer Review for Fragile X!
Effects of the Sigma-1 Receptor Agonist Blarcamesine in a Murine Model of Fragile X Syndrome: Neurobehavioral Phenotypes and Receptor Occupancy
Fragile X syndrome (FXS), a disorder of synaptic development and function, is the most prevalent genetic form of intellectual disability and autism spectrum disorder. FXS mouse models display clinically-relevant phenotypes, such as increased anxiety and hyperactivity. Despite their availability, ...
(www.researchsquare.com)
Konklusion:
In conclusion, the present findings confirm the dose-dependent receptor occupancy of the S1R with blarcamesine and, combined with the therapeutic response observed at low doses in the tested preclinical model, emphasize the viability of S1R as a therapeutic target in FXS and the clinical potential of blarcamesine in FXS and other neurological disorders. Indeed, pre-clinical studies in a mouse model of Rett syndrome showed similar positive effects on multiple clinically relevant neurobehavioral phenotypes 20. Furthermore, clinical efficacy was demonstrated in a placebo-controlled Phase 2 study in Rettsyndrome (NCT03758924) and previously in a smaller PK cohort of patients with this neurodevelopmental disorder 36, as well as significant cognitive improvements in a Phase 2 trial in Parkinson's disease dementia (NCT03774459). Late-stage clinical studies of blarcamesine in adult and pediatric patients with Rett syndrome (NCT03941444, NCT04304482) and Alzheimer's disease (NCT02756858, NCT03790709) are currently ongoing. Continued findings from these clinical studies with blarcamesine, combined with the presented data strengthens the rationale for potentially a dependable and effective treatment strategy for FXS and other neurological disorders targeting the S1R with blarcamesine.
Denne foreløbige afhandling er fra marts 2021 og er stadig under evaluering af et større antal forsker, bl.a. fra Stanford Universitetet.
Ved sidste Q3-rapport varslede Missling, at den endelige Peer Review ville blive udgivet i tidsskriftet Natur.
Denne Peer Review skulle danne grundlaget for og bekræfte videnskaben for, at 2-73 kan gå direkte i fase 3 i Fragile X - men som det fremgår af konklusionen også potentielt i andre CNS indikationer.Som ved de prækliniske forsøg i RETT, blev disse positive resultater omsat til tilsvarende positive resultater i kliniske forsøg i RETT - ligesom det samme er tilfældet i Alzheimer og Parkinson.
Så skidtet virker!
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Hej Tasso!
Jeg har været på sidelinjen siden 2018 stykker. Jeg vil gerne sige tak for det store arbejde du ligger i alle dine indlæg.
Jeg har fået fingrene i denne rapport https://pubmed.ncbi.nlm.nih.gov/34383254/Er det noget du eller andre her på siden vil se. Jeg ved I kan tyde det bedre end mig!
Hilsen,
Michael
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