Workin' on it
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I don't think that's the reason Biostockclub stopped posting here. But you're right he was brighter than most.
It would be great to have him back posting whatever he wanted.
I'm learning to appreciate the Creative Light Above, Around and About that Shines Through Time Within and Without. And pay more attention when it shines.
I don't know? Maybe because Dr.'s Hagerman and Sabbagh are the most respected individuals in their fields, spend their lives learning, discovering new ways to solve the most difficult questions in medicine, trying to help the less fortunate CNS patients?
While the fudsters are of a different ilk.
Here are Dr. Hagerman's remarks from a 2020 presentation.
Hi I’m Dr. Randi Hagerman,
I’m a Clinician, I’m a developmental and behavioral Pediatrician and I have a great interest in the use of targeted treatments for a variety of neuro-developmental disorders. And again by targeted treatments, I mean medications that can modify the neurobiological changes that are made in the brain and individuals with neuro-developmental disorders. I participated in the Adult Rett Syndrome Study using Anavex2-73.
The results are being released now and these are very exciting results.
I was blinded during the control trial where individuals were randomized either to active drug, about 5mg a day vs. placebos so I didn’t know who was on what.
I saw a number of individuals who did very well on this medication and others who didn’t but also I have been involved now in the follow up where they go on the active medication so I can see actually the results now in individuals that I know are on medication.
I saw a variety of improvements and again as you heard in the last talk from Jeff, these individuals are very severely impaired.
They can’t talk or talk very little, often they’re in wheel chairs. They can’t use their hands well . They often times can have agitation, other types of problems like constipation and what we saw is that individuals were calmer, less agitated, they seemed to be happier. They were able to concentrate better. They made better eye contact and they seemed to understand questions or directions given to them and were able to follow through in many cases on some of the commands that their parents made.
There was less anxiety, better understanding and in general the parents felt they were calmer and less agitated and often we saw improvements in the gastrointestinal problems.
One patient that stands out that I just recently saw, was able to swallow her food better. She didn’t have any problems choking now that she’s on the active medication.
She was able then to eat better, happier. She had less bloating, less constipation and having better function GI wise I think can lead to less agitation.
The families were able to mark on the main outcome measures that were improved. The Rett Syndrome Behavior Questionnaire that the parents answered and also the Clinical Global Improvements Scale. The CGII that the Clinicians do. There were significant improvements with the Anavex2-73.
So this is very exciting.. So far the patients want to continue on the Open Label Extension on this medication.
This is a relatively unique medication that can actually improve the calcium dis-regulation, the mitochondrial function, the oxidative stress and the neural connections. These problems can be seen in variety of other neural developmental disorders and also neurodegenerative disorders.
They’ve carried out studies in Alzheimer’s and in Parkinson’s Disease looking at cognition in Parkinson’s disease. It improved cognition in both Alzheimer’s and Parkinson’s disease.
I do a lot of research also in Fragile X syndrome and this medication can improve the Fragile X symptoms in the mouse model of Fragile X so I’m very excited about this medication causing improvements not just in Rett Syndrome but in other neuro-developmental disorders and neurodegenerative disorders.
This medication can be a boon. I think in general for aging and certainly for aging syndromes like Parkinson’s Disease and Alzheimer’s and I can’t wait to start it on other neuro-developmental disorders like Fragile X Syndrome.
I am very excited about this medication and also as I age I would definitely like to take Anavex myself.
I’m excited that this is a break through medication and for not just Rett Syndrome but other disorders of aging and also neuro-degeneration.
We are open to answer questions that you may have.
This is an exciting new treatment.
When Clinicians performing trials are outspoken about the benefits of blarcamesine, that means more to me than paid hacks shorting a stock, endlessly finding fault with everything.
With Donnanemab screening out almost 90% of their participants they show a barely improved drop out rate than Anavex but the Fuds can't stop harping on it. The drop out rate has been discussed and is a non issue. The OLE will have more data when released.
Yes, Anavex takes longer analyzing data. You can fault that if you want but Anavex has better results and remains the best hope for CNS patients. Blarcamesine is far safer than the brain draining mabs, has improved efficacy, is orally available and as Dr. Hagerman attests after being a Clinician in a Rett trial, is a boon.
Boon; noun- something that is very helpful and improves the quality of life.
A Controlled Substance regulator, the EMA, will be evaluating blarcamesine in the near future then the World will see what Dr. Hagerman and Prof. Macfarland saw.
You forget how much smarter the experts on this board are than the peers that review drugs. 100 peer reviews isn't worth one sarcastic remark about the WGT group by an expert. But that shouldn't stop us from trying!
Here's something for them to discredit for now...
NEW YORK, June 14, 2021 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp.(AVXL) (“Anavex” or the “Company”) , 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, today reported that ANAVEX®2-73 (blarcamesine) and ANAVEX®3-71 (AF710B) are featured in a new peer-reviewed publication in the journal of Expert Opinion on Therapeutic Targets, titled “The emerging role of the sigma-1 receptor in autophagy: Hand-in-hand targets for the treatment of Alzheimer's”.1
Scientific Paper Highlights:
Sigma-1 receptor (SIGMAR1)’s expression increases with age, however in Alzheimer’s disease (AD) it decreases
The decrease in SIGMAR1 expression during AD coincides with an age-related decrease in autophagy
The SIGMAR1 may compensate for loss of receptors and autophagic machinery during healthy aging
SIGMAR1 is activated by ANAVEX-compounds
ANAVEX®2-73 has been shown to induce autophagy
Activation of the SIGMAR1 can induce cytoprotective autophagic pathways
The authors of the paper point out that studies using positron emission tomography (PET) have shown that in healthy aging, there is no loss of the SIGMAR1; in fact, there is a possible increase in SIGMAR1 expression2 that coincides with the age-related loss of the M1/M4 muscarinic receptors3, D1/D2 dopamine receptors4, and serotonin (5HT2A) receptors5. The increase in SIGMAR1 expression may be a compensatory mechanism for the loss of the other receptors6.
However, PET scans of patients with a recent AD diagnosis show a reduction of SIGMAR1 expression7. SIGMAR1 also promotes autophagy and results in the degradation of amyloid-beta precursor protein (APP) thereby inhibiting Aß production8.
The publication explains that AD is a multifactorial disease, where several pathways interlink with each other and cause cognitive impairments. The available drugs only tend to target a single pathway and mitigate the symptoms of AD without slowing the disease progression. Combinatorial therapy has been suggested as a treatment strategy; however, the existence of drug-drug interaction is a concern. Hence, there is a need for the development of drug molecules that can target multiple pathways to halt disease progression and improve the memory function.
SIGMAR1 has emerged as one of the prominent targets in treating neurodegeneration. It is involved in the modulation of glutamate levels, maintaining endoplasmic reticulum (ER) function, and calcium regulation, promoting neurogenesis, reducing reactive oxygen species (ROS) formation, suppressing neuroinflammation and ameliorating Aß toxicity9. Recent studies with ANAVEX®2-73 show that SIGMAR1 activation is also involved in autophagy, an intricate phenomenon that clears damaged cellular organelles and misfolded proteins10. SIGMAR1 agonists, including ANAVEX®2-73 and ANAVEX®3-71 have been reported to block toxic Aß, tau and neuroinflammation11.
Autophagy and the cellular machinery involved are essential to homeostasis and cell survival. Autophagy has been shown to be important for axonal health and homeostasis as autophagy inhibition leads to axonal wasting12.
During the early stages of AD, it has been noted that there is an accumulation of Aß and tau protein in the dystrophic or swollen neurites of AD patients’ brains. Furthermore, it is well known that autophagy plays a key role in the management of Aß and tau protein levels, and that some of the key proteins involved in the autophagy mechanism disappear with age, resulting in decreased autophagy in older brains13. At the same time the SIGMAR1 is upregulated, possibly compensating for the reduction in autophagy, and reduction in other receptors, such as muscarinic receptors14, dopamine receptors15, and serotonin receptors16, in an attempt to protect the neuron cells.
Since it has been observed that a number of SIGMAR1 agonists, including ANAVEX®2-73, is able to upregulate SIGMAR1 expression in the brain, it is possible that these drugs could help the cells to compensate for the loss of other receptors and autophagy machinery.
The authors conclude, that in the future it may be the case that SIGMAR1 ligands (or drug combinations) targeting the activation of autophagy, and other SIGMAR1 related neuroprotective pathways, are prescribed prophylactically, in much the same way as with statins for the prevention of heart disease today in an effort to prevent the loss of the SIGMAR1 receptor seen during AD.
“This independent paper highlights the understanding of the relevance of utilizing sigma-1 receptor activation as compensatory mechanism to chronic CNS diseases, currently tested in late-stage placebo-controlled ANAVEX®2-73 Phase 2b/3 clinical Alzheimer's disease study, which recently completed enrollment, as well as in Parkinson’s disease dementia (ANAVEX2-73-PDD-001) and ongoing Rett syndrome program (ANAVEX2-73-RS-001/002/003)”, said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex.
ANAVEX®2-73 activates the sigma-1 receptor (SIGMAR1). Data suggests that activation of SIGMAR1 results in the restoration of complete housekeeping function within the body and is pivotal to restoring neural cell homeostasis and promoting neuroplasticity.17
Anavex Life Sciences’ product portfolio platform includes small molecule drug lead candidate ANAVEX®2-73 for the treatment of Alzheimer’s disease, Parkinson’s disease and Rett syndrome and ANAVEX®3-71 for frontotemporal dementia.
I suspect the EMA submission will include the Anavex mitigation plans to reduce drop outs for tolerability issues regarding dizziness as spelled out during the recent earnings report. Plus the obvious positive fact that no one died or wasn't able to recover from titration by simply reducing or stopping dosing.
The latter understanding relieves the EMA from your overemphasized concerns and paves the way for blarcamesine approval. imo
If you own shares and follow Anavex you can find out more about what they are doing. You actually don't need to own shares. Do you own shares?
https://finance.yahoo.com/news/anavex-received-agreement-committee-medicinal-123000450.html
The Street has it's own agenda which, Jim Cramer will tell you, has nothing to do with stocks. This is a transcript from a youtube video Mr. Cramer made years ago. It might still be available to watch, I haven't looked recently.
“What’s important when your in that hedge fund mode is to not do anything that’s remotely truthful.
Because the truth is so against your view, that it’s important to create a new truth to develop a fiction. The fiction is developed by anybody who’s down 2% to up 6% here. You can’t take any chances, you can’t have the Market up any more than it is if you’re up 6. Because starting Gn 2 you’ll have all your money come out so what would you do if you’re in that situation? And you feel like you’re desperate? You would do these actions.
All they can do is one step at a time.
The difference is, when Anavex takes a step it moves them forward.
These other companies? Not so much.
What has management been doing?
Anavex has been producing the leading contender in the Alzheimer's Neurology space.
They are #1 with a bullet scheduled for approval in 2025. The Marketing Authorisation would allow direct market access throughout the European Union for oral blarcamesine for the treatment of Alzheimer’s disease.
They have data and safety which line up with the latest guidance from the FDA and the EMA has seen it. Having seen it, the EMA has requested Anavex submit for full approval for Alzheimer's Disease.
That's what Anavex has been doing.
All the other companies are fumbling about with drugs and trials unable to even considering applying for approval. The ones that have received FDA approval are very poor substitutes for a safe therapeutic drug.
Some strategies a prominent hedge fund manager recommended about 15 years ago. Initials are J.C.
“What’s important when your in that hedge fund mode is to not do anything that’s remotely truthful.
Because the truth is so against your view, that it’s important to create a new truth to develop a fiction. The fiction is developed by anybody who’s down 2% to up 6% here. You can’t take any chances, you can’t have the Market up any more than it is if you’re up 6. Because starting Gn 2 you’ll have all your money come out so what would you do if you’re in that situation? And you feel like you’re desperate? You would do these actions.
The great thing about the Market is, it has nothing to do with the actual stocks. Maybe 2 weeks from now, buyers will come to their senses and realize every thing they heard was a lie. Then again Fanny Mae lied about their earning for 6 billion dollars so it’s just fiction and fiction and fiction.
So the stock is manipulated all over the place, then the Company is forever blamed for any unfavorable price movements?
Oh! that's not enough you have the b*lls to call someone making a valid comment arrogant? And ignorant?
Really? Wow Pogu, that's a new low.
Thanks, I used to read the NYT, never picked up the London but they've given me hope they are legitimate.
In a golf tournament, you have to wait for everyone to finish before you award the trophy. Even if those who were expected to do well have fallen flat on their faces and stand no chance.
I think Anavex has a few holes to go and the score they have to beat is about 50 over par.
The London Times just did some serious hating on the mabs.
The paper said
Thanks catdaddy, I misread that.
Anavex can run trials and submit for approvals, that's the game.
Anavex has run trials but no submissions.
The Rett trial wasn't all bad but did miss an end point. Going to try again there, which makes sense, they were very close but they have to do that, no one is going to save them.
The Alzheimer's OLE trial not ending yet is a surprise, some were saying it's already finished. When it is, they have to submit for approval. No one is going to do that for them, they have to do it and should be more clear on when.
Would it be possible when applying for the Marketing Application for Alzheimer's Disease from the EMA, could Anavex also share the findings from the other trials conducted with Blarcamesine?
Could the EMA see the results of the several Parkinson's Disease Dementia trials and the Rett Syndrome trials as well?
That would increase the information available to further assess the safety of the drug, as well as show the positive effects it had on the patients in the Randomly Controlled Trials as well as the Open Label Extension and Compassionate use trials, as well.
I had some arthritis years ago and my doctor offered a sample drug. I took it not knowing what to expect. It made me a little dizzy and was affecting my sense of balance a little.
I didn't stay on it long enough to even notice whether it lessened my arthritis. Dizziness, that's it, I'm oughta here.
I told him about it and he had no comment.
I think when people who may have dropped out, hear of the experience of others, they might be willing to give it another try. But without that assurance that there was a benefit I can easily see how that simple awareness of an alteration would be enough to spook someone. Also, dosing at night might lessen the effect some.
Together, dosing at night and hearing about positive results of others might increase the number of patients willing to give Blarcamesine a try. I want that molecule in my body activating the S1-R and M1-4 receptors, letting them go to work in every organ in my body improving things.
Yes, the brain says, hey, somethings going on!
Yes, I know, let it go on.
Love it! Keep 'em coming.
Maybe we get to see some of that on Tuesday?
Speaking of dizzy and balance..
As I get older, trying to hang on to my sense of balance, each morning I test the longest distance neuro circuits I have from my brain, the ones in my feet.
I stand on one foot for 60 seconds, then the other. As I lose my balance, I correct some part of the structure, the foot, knee, hip, waist, head to regain being centered over the one foot.
It took a while to get to a minute and it wasn't easy but each day it requires less of a struggle as I relax and try to get my sense of balance to do the work, not muscling it so much. It's still kind of a pain and feels like work but might be better than the alternative.
Another geriatric chore but one that might be worth doing. Being conscious and aware doesn't come for free anymore.
Looks like we're on our own when it comes to taking care of our health. In the USA anyway.
No brain bleeds? No one died?
Dr. Missling told us years ago some people were getting dizzy, the dosage was adjusted and most made it through. That was one of the key ways to know if you were getting the drug.
When someone has a brain bleed or dies from a mab, did they tell them, Oh, that's ok, that way you know you're getting the drug? And you might even survive.
These are fragile old people with Alzheimer's disease, there's no reason to scare who are seeking relief from dementia, they are creating enough problems on their own.
As most everyone agrees, they'd rather take a pill, avoid the swelling, bleeding, MRI's with nasty contrasting drugs and 50k price tag.
Blarcamesine is safer than the mabs. It will very likely be approved by the EMA.
The main point is, Blarcamesine is safe. It doesn't have any SAE's. The Mabs do. The first, second, third and last thing Regulators do is look for safety.
The EMA found it in the Anavex drug along with clinically meaningful improvement in Alzheimer's Disease. The EMA did not find safety in the Mabs so far and said so, plainly and directly.
What don't you understand?
OK, you can't face facts or answer direct questions. Got it.
So your contention is, Anavex isn't as safe as the Mabs?
Possible but imo;
Sava is a distraction from Anavex;
Hey! Look over here! Look how the price goes up and down! Wow!
CEO indicted? No problem! We've got volatility high pps, always bouncing!
Keep some funds away from Anavex allowing someone to keep a lid on Anavex.
The main point is, Blarcamesine is safe. It doesn't have any SAE's. The Mabs do. The first, second, third and last thing Regulators do is look for safety.
They found it in the Anavex drug along with clinically meaningful improvement in Alzheimer's Disease.
You're a smart guy, Zig. But when YOU don't even believe what you're saying, it gets lost.
I'll admit you've been driven to delusion but in your case, it's a putt.
The light is fleeting, even when we're here for our very short time but I'll bet Ern and his family were glad for the extra light Ern saw.
And you'd have us believe it's the drugs fault.
Let Ern will show you the light..
No, with the high dropout rate in the placebo group it looks like this fragile group of participants had other things going on.
Covid was ruled out in some dropout cases but the drug doesn't automatically become suspect as further inspection reveals.
Alzheimer's patients have more challenges than most, for them to continue to take the drug while experiencing dizziness is a lot to ask.
But as Ern Heaven pointed out in his video, the experience of slipping into the netherworld of Alzheimer's wasn't pleasant either.
Does anyone have a link to the Ern Heaven video? I think it would be helpful to review that, it's been so long since we've seen it.
Then, after watching Ern a few times, I urge you to continue your crusade extolling the safety of Mab drugs.
Those drugs, Mabs, have a horrible safety record.
The safety record for Anavex Blarcamesine is unparalleled in Alzheimer's drugs. The oral administration is superior and it doesn't require extensive, expensive follow ups to try and protect patients.
2 drugs are being offered to people but no one wants them and no doctors want to prescribe them.
I'm just saying, ask the EMA if THEY WANT TO WAIT for the OLE extension. Which some have pointed out here isn't that critical, except for the safety factor which hasn't been a big deal.
No where near the risk associated with the APPROVED DRUGS.
Just wondering, is it forbidden to ask if a provisional approval is possible?
The EMA is aware of the OLE but remember Janet Woodcock talking about the great need for SOMETHING, anything for people demanding a drug? What was that, 3 years ago? Still no drug, to speak of?
Anavex could put something in front of the EMA, then let them decide if it is enough. Why should Dr. M's overly conservative, You'll understand why we did it this way..., timeline hold back relief for millions?
Let those whose decision it is at least have the option to say, OK, go ahead but understand if the OLE is a disaster then some adjustments will have to be made. If the OLE is OK, not great, just OK, then full speed ahead.
It's all about safety and I really don't see the great risk with compassionate use folks on it for 4 years
It surely wouldn't hurt.
Yes, how about Anavex keeps them apprised of the data as it becomes available and will submit as an appendum?
There is an urgency here.
Having seen the data the EMA is positive towards Anavex, requesting it ask for Full Approval for Alzheimer's Disease.
Without pushing it too far I wonder if Anavex needs to dot every i and cross every T? They have a hungry willing market begging for what they have. Would it be foolish to assume the EMA would like to have something in front of them they can approve? And won't be scrutinizing minutia with artificial intelligence?
Would it be foolish to assume some at the EMA would also like to start taking 30mg of Blarcamesine before year end?
I don't know how these things go but I don't think Anavex needs to ask for any favors to expect Blarcamesine to be approved.
Does Anavex need to include the OLE data?
Blarcamesine will be approved by the EMA.
The barrier that Lec, Adu, Doncorleonamab faced isn't the incremental reduction in decline, its the awful adverse side effects. Brain bleeding ARIA, swelling, death. Those are big turn offs.
Had approval been sought before the EMA had seen Anavex data, there might have been a greater chance for the Mab approval but why offer an inferior drug that can kill you to people when there is a better, safer alternative that doesn't require infusions, MRI's, etc..
--EF Hutton Initiates Anavex Life Sciences at Buy With $46 Price Target
That would make sense and agree, God Bless. Also, the EMA.