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We need to see/do more of these RWE-RWD proofs.
Does Blarcamesine do what Anvex says it does?
georgejji
Expect HUGE NEWS in less than
Nasdaq Futures
15,178.50
+78.25(+0.52%
ATTITUDE MATTERS...
tredenwater2
Many, who are still waiting for a missed endpoint will soon imo see the vast long term efficiency being created right before our eyes and right before a plethora of rare disease trial approvals.
GREAT THOUGHT TRADEHERPETE...LETS REWARD-RECOGNIZE EXCELLENT RWD-RWE when we see examples...excellent support from Canada and Australia as it happens...IMO, we could not have any stronger or more capable support than them.
The rest of the Rett population should be putting pen to paper to request access asap. Remember this is a safe drug that could be relieving significant suffering for children.
Perhaps a contingent of physicians joining forces could influence the FDA to move things along?
We know Alzheimer's patients from the P2a trial in Australia are also enjoying continued access, again, with a safe drug.
Yesterday was FUDsters' "trash day",
crescencentmotor, this is an excellent example of post with fact based , informational, value added posting...We need more of same when appropriate. IMO....
thx
It is very important to note that the improvement in symptomatic and disease modifying effects was statistically significant even when comparing the original treatment group to the placebo group, which also showed those symptomatic and disease modifying effects after being put on Blarcamesine during the OLE period. These outstanding trial results have never been seen before in Rett Syndrome. I cannot wait to see the juvenile results.
WE DO NOT CARE>>>>
hnbadger
I was much more interested in the news today.
“Study Demonstrates Disease Modifying Effects of ANAVEX®2-73 “
The lift off counter has been started...standby for AVXL LIFTOFF ... momentarily... BLESS RSD KIDS AND FAMILIES
Sad example of what some people will do for money...pathetic.
JoeBear906
The transition from unaffected to disease modifying improvement in the placebo track study participants in the OLE seals it for me.
plexrec
the 2 sites I saw PR on including Anavex web site say "June 28,2023"---
tredenwater
I wonder how the relationship with Ariana is embraced with today’s announcement?
bas2020
A biotech doesn't form these types of strategic partnerships if it doesn't believe it'll be marketing its product soon.
As discussed here on AVXL board, it looks like the AVXL TRIFECTA is a good candidate for demonstrating how this process will work. Start with AVXL DB -trial data and results to date...quickly publish strong trial results to date (lead w/ PD-OLE) , followed then by RSD and AD.
BTW, this could be published any time SOON. IMO. This is A (THE) plan , followed by YUGE explosion in AVXL SP. ...BYE BYE BP.
NEW YORK, June 27, 2023 (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) diseases, and Partex N.V. Group (“Partex”), the first Data-to-Drugs digital pharma platform, today announced a strategic partnership with the overall ambition to reshape the future of the biopharma business model.
By combining Anavex's innovative small molecule precision medicine drug development platform and Partex's disruptive approach of Artificial Intelligence (AI) enabled drug development and healthcare sales marketing, this collaboration is intended to drive efficiency, effectiveness, and innovation across the value chain with patient-centric focus at every step.
TOLD YA...https://investorshub.advfn.com/boards/read_msg.aspx?message_id=172215312
IMO, the old school model of BP controlling the CNS universe just got blown up. Big money has some new science galaxies to model and digest. In the meantime, the model for how CNS medical revenue will be sourced/controlled (direct product sales or WW diagnostics for CNS diseases defies BP existing science models). BP labs and clinics better circle the wagons b/c they will soon be obsolete. This technology change will change most existing BP models. Transition period in phases where phase 1 will take 0-5 years.
The transition from vacuum tubes to solid state (semiconductor ) physics comes to mind as an example of how 80%+ of the established revenue base(products) became obsolete overnight. First though, BP has to now understand what just happened. . They will be in denial for a while. AVXL is the new Texas Instruments/Motorola/Fairchild startup master technical CNS wave, bye-bye BP, you are obsolete but do not yet know it. IMO, transitions will begin with CNS diseases (highest complexities) and go on from there.
BTW, we are all about to realize that the CNS controls the ENTIRE BALL OF HUMAN DISEASES STRING.
tredenwater2 link
Whole blood transcriptomics analysis (RNAseq) was performed for the PDD study at two timepoints: baseline and Week 14. After quality control filtering, 14,150 genes were retained for analysis. Methods: Weighted gene correlation network analysis (WGCNA) was used to identify clusters of genes that show a correlated expression change across patients and timepoints. Thus, a data-driven gene network was generated. Associations between the identified clusters and treatment arms were explored in order to select clusters for which gene expression is significantly associated with the high blarcamesine dose. These associations were assessed using linear mixed effect models with three covariates: dose, patient and timepoint. Significance was assessed with Dunett’s test. Results:
Mikesc
Think Basket!!!!!
Plex...happy to help...I could have missed it...BR.
Plex..."nidan-".did not find , "nearing"----Thanks !!"
Plex, this is as close as it gets...IMO...https://www.yahoo.com/video/edited-transcript-avxl-oq-earnings-133000371.html...did not find , "nearing"
Plexrec
"nearing commercialization"---have seen this quote (in the recent past)--I believe TGD said this--can someone please provide a link where this phrase was used ????? TIA
Great insight-
Irish--" one pre-market morning when I see our stock price up $40 or better! "--got my approval for this to happen--release complete TLD from ADTrial-with very positive results would definitely help !!!!! (on the horizon) !
What are examples of chronic problems?
Common chronic conditions are arthritis, Alzheimer's disease, diabetes, heart disease, high blood pressure, and chronic kidney disease. Unlike acute conditions, chronic health conditions cannot be cured—only controlled.Mar 9, 2020
plexrec
power--"FYI - Corinne Lasmézas, PhD"--good find also--hopefully this Dr. in close communication with our GD---guiding and helping on our road to approval !!!!!
https://alz-journals.onlinelibrary.wiley.com/doi/full/10.1002/trc2.12013
A precision medicine framework using artificial intelligence for the identification and confirmation of genomic biomarkers of response to an Alzheimer's disease therapy: Analysis of the blarcamesine (ANAVEX2-73) Phase 2a clinical study
Harald Hampel, Coralie Williams, Adrien Etcheto, Federico Goodsaid, Frédéric Parmentier, Jean Sallantin, Walter E. Kaufmann, Christopher U. Missling, Mohammad Afshar
First published: 19 April 2020 https://doi.org/10.1002/trc2.12013Citations: 13
Abstract
Introduction
The search for drugs to treat Alzheimer's disease (AD) has failed to yield effective therapies. Here we report the first genome-wide search for biomarkers associated with therapeutic response in AD. Blarcamesine (ANAVEX2-73), a selective sigma-1 receptor (SIGMAR1) agonist, was studied in a 57-week Phase 2a trial (NCT02244541). The study was extended for a further 208 weeks (NCT02756858) after meeting its primary safety endpoint.
Methods
Safety, clinical features, pharmacokinetic, and efficacy, measured by changes in the Mini-Mental State Examination (MMSE) and the Alzheimer's Disease Cooperative Study-Activities of Daily Living scale (ADCS-ADL), were recorded. Whole exome and transcriptome sequences were obtained for 21 patients. The relationship between all available patient data and efficacy outcome measures was analyzed with unsupervised formal concept analysis (FCA), integrated in the Knowledge Extraction and Management (KEM) environment.
Results
Biomarkers with a significant impact on clinical outcomes were identified at week 57: mean plasma concentration of blarcamesine (slope MMSE:P < .041), genomic variants SIGMAR1 p.Gln2Pro (?MMSE:P < .039; ?ADCS-ADL:P < .063) and COMT p.Leu146fs (?MMSE:P < .039; ?ADCS-ADL:P < .063), and baseline MMSE score (slope MMSE:P < .015). Their combined impact on drug response was confirmed at week 148 with linear mixed effect models.
Discussion
Confirmatory Phase 2b/3 clinical studies of these patient selection markers are ongoing. This FCA/KEM analysis is a template for the identification of patient selection markers in early therapeutic development for neurologic disorders.
Abstract
Introduction
The search for drugs to treat Alzheimer's disease (AD) has failed to yield effective therapies. Here we report the first genome-wide search for biomarkers associated with therapeutic response in AD. Blarcamesine (ANAVEX2-73), a selective sigma-1 receptor (SIGMAR1) agonist, was studied in a 57-week Phase 2a trial (NCT02244541). The study was extended for a further 208 weeks (NCT02756858) after meeting its primary safety endpoint.
Methods
Safety, clinical features, pharmacokinetic, and efficacy, measured by changes in the Mini-Mental State Examination (MMSE) and the Alzheimer's Disease Cooperative Study-Activities of Daily Living scale (ADCS-ADL), were recorded. Whole exome and transcriptome sequences were obtained for 21 patients. The relationship between all available patient data and efficacy outcome measures was analyzed with unsupervised formal concept analysis (FCA), integrated in the Knowledge Extraction and Management (KEM) environment.
Results
Biomarkers with a significant impact on clinical outcomes were identified at week 57: mean plasma concentration of blarcamesine (slope MMSE:P < .041), genomic variants SIGMAR1 p.Gln2Pro (?MMSE:P < .039; ?ADCS-ADL:P < .063) and COMT p.Leu146fs (?MMSE:P < .039; ?ADCS-ADL:P < .063), and baseline MMSE score (slope MMSE:P < .015). Their combined impact on drug response was confirmed at week 148 with linear mixed effect models.
Does this meet PEER Reviewed Paper criteria? Looks like it does. Dr.M.is one of credited people contributors. First Published 16 June 2023 (last Friday) .
https://doi.org/10.1002/alz.059024
WHAT COLOR IS THIS RED PIECE OF PAPER???[color=red][/color]
[color=red][/color]
https://alz-journals.onlinelibrary.wiley.com/toc/15525279/2023/19/S7
Study of the mechanism of action of Blarcamesine (ANAVEX®2-73): Whole blood transcriptomics analysis identifies treatment impact on compensatory pathways by restoring key neurodegenerative pathways functionality, including Alzheimer’s disease pathway
Coralie Williams, Martin Kindermans, Frédéric Parmentier, Federico Goodsaid, Edward Hammond, Christopher U Missling, Mohammad Afshar
First published: 16 June 2023 https://doi.org/10.1002/alz.059024
[img][/img]
Jimmy_Mcy...looks like they got some dates crossed...note 2023...???
https://alz-journals.onlinelibrary.wiley.com/doi/10.1002/alz.059024
Study of the mechanism of action of Blarcamesine (ANAVEX®2-73): Whole blood transcriptomics analysis identifies treatment impact on compensatory pathways by restoring key neurodegenerative pathways functionality, including Alzheimer’s disease pathway
Coralie Williams, Martin Kindermans, Frédéric Parmentier, Federico Goodsaid, Edward Hammond, Christopher U Missling, Mohammad Afshar
First published: 16 June 2023 https://doi.org/10.1002/alz.059024
Results
Blarcamesine significantly restores functionality in key pathways of Alzheimer’s disease, Parkinson’s disease and Prion diseases. The analysis identified two clusters of genes that are significantly differentially expressed in treated patients and represent compensatory pathways to genes dysregulations induced by neurodegenerative diseases. In both clusters, majority of genes were confirmed to interact (STRING database). Amongst interacting genes, neurodegenerative pathways were identified as overrepresented (p < 0.01). Down-regulation of genes involved with neurodegeneration was observed in the placebo arm, however, was compensated in the blarcamesine arm.
Conclusions
This analysis identified a gene network that is differentially expressed in patients treated with blarcamesine after 14 weeks of treatment, compared to placebo. The biological relevance of this gene network was assessed. Pathway analysis confirmed the impact of the treatment on pathways involved in neurodegenerative diseases The identification of a gene network as the blarcamesine response pathway lays the foundation to better understand the mechanism of action at the molecular level of blarcamesine, thus unlocking characterization of responders based on molecular profiling, as well as identification of new indications in the area of neurodegenerative and other disorders.
Has FDA painted itself into a corner on the Amyloid thesis? If the AVXL alternative shows evidence of no risk and efficacy using a daily pill then the hill is even steeper for Amyloid , unless BP shows strong evidence of efficacy (for which no evidence exists) . If you were calling the shots at FDA...become a hero and take no risks w/new treatment or......??....it seems like a no brainer(not intended as humor).
Folks at FDA must be getting tired by now. They are in the spotlight and they know it.
falconer66a:
Well, the first sentence in the abstract summarizes the entire matter:/
WGT
Excellent assessment falconer66a , as usual , thanks. My sense staff AVXL are positioning to , "Take the BP CNS diseases Hill ". IMO, AD has been a difficult clinical opponent, inclusive of a long list of likely associated other CNS indications such as Dementia, PD, RSD, and many, many more. Some of us would like to see a kind of AVXL CNS disease DOMINO effect . We'll just have to hang on a little longer, but our day is coming.
Thanks again for your efforts here. BR...NIDAN
a reasonable comparison of results shows?....WGT
AVXL:
Conclusions
This analysis identified a gene network that is differentially expressed in patients treated with blarcamesine after 14 weeks of treatment, compared to placebo. The biological relevance of this gene network was assessed. Pathway analysis confirmed the impact of the treatment on pathways involved in neurodegenerative diseases The identification of a gene network as the blarcamesine response pathway lays the foundation to better understand the mechanism of action at the molecular level of blarcamesine, thus unlocking characterization of responders based on molecular profiling, as well as identification of new indications in the area of neurodegenerative and other disorders.
Suggest you re-read...https://alz-journals.onlinelibrary.wiley.com/doi/10.1002/alz.059024
It establishes what steps, methods and process sequence are used to do the work and to measure the results.
Definitions 21 CFR 820.3 (z)(1) Process Validation means establishing by objective evidence that a process consistently produces a result or product meeting its predetermined specifications.Sep 30, 2015
Quality System Regulation Process Validation - FDA
This language reads as a V&V (verification and Validation) statement written in FDA speak. In other words, it may be read as a statement of "Validation" for the methods used. Very positive.
https://alz-journals.onlinelibrary.wiley.com/doi/10.1002/alz.059024
This language reads as a V&V (verification and Validation) statement written in FDA speak. In other words, it may be read as a statement of "Validation" for the methods used. Very positive.
https://alz-journals.onlinelibrary.wiley.com/doi/10.1002/alz.059024
hnnbadger
s this considered a “peer reviewed “article ?
https://alz-journals.onlinelibrary.wiley.com/doi/10.1002/alz.059024
Coming from the Alzheimer’s Association is a big step forward IMO!
Redshoulder.
MOA study from the Alzheimer Association. New Article, Date June 16th"
Very powerful words here --take note shorties !!!---While the data already released was superior to lecanemab, once the 50 mg alone cohort data comes out the results should be considerably better than for the combined group. So I think the data so far was encouraging, but the best news is yet to come — and the stock remains a favorite for 2023.
plexrec
Very, very positive - smoke free assessment ...happy to see a clear message.
[quoteAnavex (AVXL), is a biopharmaceutical company dedicated to the development of novel drug candidates to treat central nervous system diseases with very encouraging results so far in Alzheimer’s, Parkinson’s and Rett Syndrome, suggests Tom Bishop, editor of BI Research.
The big news for Anavex was the release of the preliminary data from the 508 patient, placebo controlled, double-blind, 48-week, Phase 2b/3 ANAVEX 2-73 (blarcamesine or A2-73) Alzheimer’s trial which tested 3 equal cohorts with placebo, 30 mg or 50 mg oral doses.
First I need to point out two very important things:
1) There was a delay in getting the data from one of the trial sites that resulted in the company only getting the data from the third party statistical analysis company just 1 day before it was on the schedule to present at the CTAD Alzheimer’s conference. This therefore limited what the company could accurately report at that time.
2) Some of the best results from an earlier trial were for the 50 mg. cohort, while the 30 mg barely worked. However, ALL of the analysis presented recently was for the combined 30+50 mg patients as a single cohort, diluting the efficacy results.
Therefore, results yet to come should be even better once the company reports on the 50 mg cohort by itself. The primary endpoints were the reduction in the decline of cognition (ADAS-COG) and activities of daily living (ADCS-ADL) at 48 weeks.
On this the company said, “The Anavex 2-73 (blarcamesine) study met the primary and key secondary endpoints showing statistically significant reduction of clinical decline in global cognitive and functional scales in a clinical study of patients with early Alzheimer’s disease.” (MMSE baseline scores 20-28).
And more to the point: “Treatment with ANAVEX®2-73 statistically significantly reduced cognitive decline, measured with ADAS-Cog, compared to placebo at end of treatment by 45% (p=0.033).”
This is the most significant and directly on point outcome released so far for this trial given this was considerably better than Biogen/Eisai’s 27% slowing of cognitive decline recently reported for lecanemab.
Nonetheless lecanemab's meager result caused those two companies to increase $20 billion in market cap overnight. Most observers believe this Phase 3 result paves the way for likely FDA approval. However, Anavex’s drug beat lecanemab by a wide margin (a 45% slowing vs. 27%) in its Phase2b/3 trial.
But here’s the thing … Biogen’s drug requires IV infusion therapy and periodic MRIs to check for brain swelling and bleeding (two nasty side effects of lecanemab), while A2-73 is a pill and doesn’t require periodic MRI’s, a huge advantage even ignoring that A2-73 worked better.
While the data already released was superior to lecanemab, once the 50 mg alone cohort data comes out the results should be considerably better than for the combined group. So I think the data so far was encouraging, but the best news is yet to come — and the stock remains a favorite for 2023.
Subscribe to BI Research here…]
tschuss...Yes---Tom Bishop--BI Research----Tom has been a fan of Anavex for quite a long time and has it as one of his top picks for 2023--- https://www.moneyshow.com/expert/cb494a88b95e450b95e7564f10d4a10f/
McM
When will anavex start trying to save human beings from dementia death?
At some point they are willing accomplices to a corrupt and wicked system.