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I'm looking forward to hearing the next Anavex update, hoping it will include;
FDA guidance on Rett.
When Anavex plans to submit a new drug application.
Progress on other indications;
Parkinson's, schizophrenia, Fragile X.
How long it will take to prepare the Alzheimer's OLE for top line data?
I'm glad to see the FDA guidance on Early Alzheimer's align with Anavex priorities, that is encouraging.
The FDA guidance was a surprise, if you want to give him credit for it.
Do you think they dreamed that up on their own?
Thank you for the simply stated truth and nice to see you on the MB tonight.
All of us long timers here are looking forward seeing Anavex vindicated from the smear campaign and taking its true place, safely helping CNS patients live their best lives.
Thanks boi568,
The Answer should be interesting.
And people got better safely so, they want to stay on blarcamesine.
It's accepted that Rett is a difficult condition to assess in a clinical trial.
As in the trial, the OLE population enjoyed improvements in their condition without suffering adverse events, understandably to most, they would like to continue to enjoy those benefits they see as improving their lives.
ANAVEX®2-73 (blarcamesine) Receives Compassionate Use Authorization for Pediatric Patients with Rett Syndrome
Health Canada Special Access Program (SAP) Authorizes ANAVEX®2-73 (blarcamesine) for patients after successful completion of Open-Label Extension (OLE) study
Granting of requests are also ongoing in United Kingdom and Australia – representing all regions of Rett Syndrome study participation
https://finance.yahoo.com/news/anavex-2-73-blarcamesine-receives-113000106.html
Lest we forget...
ANAVEX®2-73 (blarcamesine) Receives Compassionate Use Authorization for Pediatric Patients with Rett Syndrome
Health Canada Special Access Program (SAP) Authorizes ANAVEX®2-73 (blarcamesine) for patients after successful completion of Open-Label Extension (OLE) study
Granting of requests are also ongoing in United Kingdom and Australia – representing all regions of Rett Syndrome study participation
https://finance.yahoo.com/news/anavex-2-73-blarcamesine-receives-113000106.html
Fear not sage4, Ignatiusreilly35 assured me everything is under control.
We can trust the system, Thank God!
Is it possible this isn’t just normal trading but some type of PPS manipulation, Heaven forbid?
This signal would make a great T-Shirt.
Company LocationSouth San Francisco, CA
CERo Therapeutics Holdings, Inc., an immunotherapy company, focuses on advancing the development of engineered T cell therapeutics for the treatment of cancer. Its lead program in hematologic malignancies targets an Eat Me signal upregulated on B cell and myeloid tumors. The company is based in South San Francisco, California.
Stock up 700% this morning.
Important to always remember: Blarcamesine is safe to take from early childhood to really old.
The Excellence Trial was again a smashing success in reinforcing this fact.
And, in fact, everyone got better during the Trial.
A fair view of the Excellence Trial by Marisa Wexler, MS, Rett News
January 2, 2024
Treatment with Anavex 2-73 (blarcamesine) led to improvements in behavior among children with Rett syndrome in a Phase 2/3 clinical trial — but the extent of these gains was not significantly different from improvements seen in patients given a placebo.
Those are the top-line findings of the EXCELLENCE trial (NCT04304482) — the first to assess Anavex 2-73 in Rett children — conducted by the therapy’s developer Anavex Life Sciences. The trial failed to meet its goal.
“We believe that a high placebo response may have masked the therapeutic effect of this innovative orally available molecule,” Walter E. Kaufmann, MD, chief scientific officer at Anavex, said in a company press release.
Such “high placebo responses are well documented especially in pediatric clinical studies,” Kaufmann said, adding that Anavex plans to “further assess the collective results and discuss with the regulatory authorities next steps.”
Phase 2 extension trial shows sustained benefits of Anavex 2-73
EXCELLENCE is 1st trial to test Anavex 2-73 in children with Rett
Anavex 2-73 is an experimental oral therapy designed to help normalize brain signaling in Rett syndrome patients. It works by activating a brain protein called sigma-1 receptor (SIGMAR1).
Data from a Phase 2 clinical trial (NCT03758924) involving adults with Rett showed the therapy led to behavioral improvements — and that these benefits were sustained for at least a few months in an extension study.
Findings from a later Phase 3 study called AVATAR (NCT03941444) also indicated that Anavex 2-73 could help ease Rett syndrome severity in women with the disease. Anavex recently announced data indicating that the therapy corrected gene activity in that trial.
All of these studies were conducted in adults. To assess the treatment’s safety and efficacy in children with Rett, Anavex sponsored EXCELLENCE, which enrolled 92 girls with Rett syndrome, ages 5 to 17. Two-thirds of the girls were given Anavex 2-73 for 12 weeks, or about three months, while the rest received a placebo.
The study’s main goal was to assess the impact of treatment on scores on the Rett Syndrome Behaviour Questionnaire (RSBQ), a standardized assessment of the severity of behavioral abnormalities common in Rett syndrome.
The results showed that, among girls given Anavex 2-73, average RSBQ scores improved by 12.93 points after 12 weeks. For patients given the placebo, the average improvement was 8.32 points.
The difference between the Anavex 2-73 group and the placebo group was not statistically significant, which basically means there’s a non-negligible possibility that the difference could be due to random chance, rather than the effects of the medication.
However, looking at RSBQ scores after just the first four weeks of the study, there was a statistically significant greater improvement with Anavex 2-73 than the placebo — a mean 10.32 versus 5.67 points improvement.
FDA Grants Fast Track Designation to Anavex 2-73 for Treatment of Rett Syndrome
Trial’s failure may be due to ‘slight imbalance’ in a few factors, per Anavex
Another behavioral measure, the Anxiety, Depression, and Mood Scale, also tended to improve more with Anavex 2-73 than with the placebo. A clinician-rated measure, however, showed no meaningful difference between the two groups.
According to Anavex, the lack of statistical significance in the results may be due to some imbalances between the placebo and treatment groups prior to entering the study.
“Although data analysis is ongoing, the early conclusion is that the placebo rate could have been higher in the study due to a slight imbalance in disease severity at baseline, across the treatment arms [groups], and the 2 to 1 drug to placebo randomization ratio,” Kaufmann said.
Safety data from the EXCELLENCE study did not reveal any new concerns. Further, more than 90% of participants who completed the initial placebo-controlled study opted to continue into an open-label extension in which all will be treated with Anavex 2-73 for about a year.
Overall, the findings, which mirror data from earlier studies in adults, indicate that Rett families have generally had positive experiences with the experimental therapy, according to Anavex.
Although data analysis is ongoing, the early conclusion is that the placebo rate could have been higher in the study due to a slight imbalance in disease severity at baseline, across the treatment arms [groups], and the 2 to 1 drug to placebo randomization ratio.
“We believe that Anavex 2-73, as a new, potential convenient treatment option in the future, can contribute to patients and healthcare professionals by addressing unmet needs in the treatment of Rett syndrome,” said Christopher U. Missling, PhD, Anavex’s president and CEO.
“Based on these study results, we will continue to be committed to the Rett syndrome and rare disease community, given also the prior successful two placebo-controlled studies in adult patients with Rett syndrome,” Missling added.
To date, almost all of the pediatric patients who have completed the extension study have chosen to continue receiving Anavex 2-73 through a compassionate use program.
“We express our deep gratitude for the commitment of the study participants and their caregivers, whose dedication and generous participation in clinical trials made this research possible,” Missling said.
https://rettsyndromenews.com/news/anavex-2-73-shows-promise-rett-children-but-misses-trial-goal/
I guess potential buyers might conclude Dr. Missling would be looking for more than 14b for a buyout.
My question is; How much more?
Concerned about Anavex efficacy for Rett?
Randi J. Hagerman, M.D.
January 12, 2021 | 11:30-12:45p.m.
The need for treatments that improve mitochondrial function, such as Anavex 2-73 can make a difference for both neurodevelopmental disorders and neurodegenerative disorders including Rett syndrome, FXS, Parkinson Disease, Alzheimer Disease and FXTAS. Future new treatments for FXSD will be reviewed including genetic interventions.
https://health.ucdavis.edu/mindinstitute/videos/images/dls/hagerman-2021-dls.pdf
Did someone say 2-73? Here's a video many adore!
I guess there won't be any videos of Alz patients recovering after taking lecanemab.
Too bad. Biogen has such a good PR department, possibly thousands of desperate souls crying for help will only be disappointed and fleeced for their trouble.
Could the bar for approval be any lower?
Regarding the Red and Black article;
BMY paid 14b for a company offering a Muscarinic Receptor Agonist.
That receptor is in the Blarcamesine bag of tricks. How much is it worth when coupled with the S-1R receptor?
Anavex has the goods folks. Don't listen to the noise.
Alzheimer's
Rett Syndrome
Schizophrenia
Many more to follow.
Thanks for the effort Bas,
Anavex also has Health Canada in the loop and the support of the Rett Families can't be underestimated.
Blarcamesine, by any measure is the superior drug for Alzheimer’s and the rest of the CNS unmet need victims.
I agree with you.
tradeherpete
Re: None
Friday, December 22, 2023 7:24:38 AM
Post# of 447316
Someone just paid 14b for a company offering a Muscarinic Receptor Agonist.
That receptor is in the Blarcamesine bag of tricks. How much is it worth when coupled with the S-1R receptor?
All Content and Data provided by Third-Parties — Terms of Use
Bristol Myers Squibb Strengthens Neuroscience Portfolio with Acquisition of Karuna Therapeutics
BUSINESS WIRE 7:05 AM ET 12/22/2023
Symbol Last Price Change
BMY 51.26down 0 (0%)
KRTX 215.19down 0 (0%)
QUOTES AS OF 04:10:00 PM ET 12/21/2023
Transaction Delivers KarXT, a First-in-Class M1 / M4 Muscarinic Receptor Agonist, with Differentiated Efficacy and Safety
KarXT Is a Potential First-in-Class Treatment for Schizophrenia and as an Adjunctive Therapy, and First-in-Disease Treatment for Alzheimer’s Disease Psychosis, with Promise in Additional Neuropsychiatric and Neurodegenerative Indications
KarXT Is Expected to Launch in the U.S. for the Treatment of Schizophrenia in Adults with a Prescription Drug User Fee Act Date of September 26, 2024
Bristol Myers Squibb (BMY) to Host a Conference Call Today at 8:00 a.m. ET
PRINCETON, N.J. & BOSTON--(BUSINESS WIRE)-- Bristol Myers Squibb(BMY) and Karuna Therapeutics, Inc.(KRTX) (“Karuna”) today announced that they have entered into a definitive merger agreement under which Bristol Myers Squibb(BMY) has agreed to acquire Karuna for $330.00 per share in cash, for a total equity value of $14.0 billion, or $12.7 billion net of estimated cash acquired. The transaction was unanimously approved by both the Bristol Myers Squibb(BMY) and Karuna Boards of Directors.
Dr. Missling did a great job in pointing out the superiority of Blarcamesine over the current AD SOC in administration (oral vs infusion) and effectiveness, greater and faster improvement minus severe side effects.
Leqembi was approved in China for Alzheimer's. I'm not commenting, just informing. It was news to me.
EMA LOVES ANAVEX!
Wait for Blarcamesine or spend 40k a year for BadorWorseomab.
Classic Missling, the only sane voice in the Alzheimer’s conversation..
Ziggy, you haven’t been exactly fair in your allegations towards Anavex.
You have to earn credibility here before discussing trust. ;)
Thanks Steady,
You are right again.
The amount of noise the Board is making should reassure you they don't have enough fingers to plug up this dike.
Who was it talking about the 3 Gorges Dam letting go?
What I find most interesting in the specific genetic support for Rett is, this is just how Anavex perceives helping this indication.
This genetic supercharger is very likely also up-regulating every mitochondrial organ system in the body.
The mouse data suggests these other indications by way of the Patent. And Anavex didn't even mention the blood pressure improvement, sinus rhythm or ocular benefits.
The other thing is, Blarcamesine is encouraging the bodies own mechanisms to accomplish this. This isn't some Lab junkie floating another trial and error gene splice.
This PR supports the Rett Excellence Trial.
https://www.anavex.com/post/anavex-announces-first-entire-clinical-gene-pathway-data-of-anavex-2-73-from-avatar-study-in-patient
Please sell the last 10% of your holdings and shove off.
This article is a good place to start talking about the results of the Rett Excellence Trial.
https://csimarket.com/news/the-awakening-progress-on-rett-syndrome-an-unpacking-of-anavex-life-sciences-breakthrough2024-01-02130394
Thanks to georgejjl and isathrillhouse (?) from stocktwits.
The new Patent is the icing on the cake.
https://www.anavex.com/post/anavex-life-sciences-announces-grant-of-u-s-patent-covering-blarcamesine-anavex-2-73-for-treatmen
Aren't most trials powered to see infinitesimal positive changes, hoping for any morsel of improvement to repeat enough to be Stat Sig?
The Blarcamesine Excellence trial blew it out of the water. Everyone got better, the only problem is, the Control Group got better too.
Gee, that's a real problem! Now what are we going to do???
Someone can't see the forest for the trees.
People might take some solace in that the bar for Rett and Alzheimer's couldn't be any lower.
The poor Rett patients suffer so many complications in their minute to minute life, adding these side effects from the FDA Approved Trofinetide would seem cruel.
Anxiety
decreased appetite
diarrhea
seizures
weight loss
And more commonly seen:
Fever
headache
muscle aches
sore throat
stuffy or runny nose
unusual tiredness or weakness
So, everyone improved in the Rett trial? And the fact that the Control Group did better as well is the problem?
I think the FDA will be offering Rett Patients a drug that makes them better without furthering their distress.
https://www.mayoclinic.org/drugs-supplements/trofinetide-oral-route/side-effects/drg-20545955
I suspect a Rett household is a highly charged emotional roller coaster which most people can't imagine.
That could be a problem in assessing minute gains in patient improvements. How do you separate the effects of mood on the patients AND the parents.
I guess a longer trial would have done something to even things out more but that didn't happen.
The great part about Blarcamesine is still no serious adverse events, while showing improvements.
Thanks for the article georgejjl. It is a nice backdrop to begin discussing the advancements Anavex is producing.
I get it that the ones who benefited the most were the parents. Kind of a euphoria I suppose.
Your idea illustrates the result of a parent or caregiver giving the patient hope where there has never been any.
Never been any hope. Think about that. Then, one day there is hope. It doesn't get much more powerful than that.
These children are totally dependent on their parents and when a parent throws a lifeline to a drowning child you can believe that is a powerful drug.
That's what Blarcamesine was competing with.
Why I am I not surprised to see that from you?
"So, everyone improved in the Rett trial? "
And the fact that the Control Group did better as well is the problem?
Anavex could produce and market their placebo liquid that does almost double as well as trofinitide, have no drug related side effects at all and is cheap.
As far as I can tell by the data we have seen to date this idea should work well.
People might take some solace in that the bar for Rett and Alzheimer's couldn't be any lower.
The poor Rett patients suffer so many complications in their minute to minute life, adding these side effects from the FDA Approved Trofinetide would seem cruel.
Anxiety
decreased appetite
diarrhea
seizures
weight loss
And more commonly seen:
Fever
headache
muscle aches
sore throat
stuffy or runny nose
unusual tiredness or weakness
So, everyone improved in the Rett trial? And the fact that the Control Group did better as well is the problem?
I think the FDA will be offering Rett Patients a drug that makes them better without furthering their distress.
https://www.mayoclinic.org/drugs-supplements/trofinetide-oral-route/side-effects/drg-20545955
plexrec,
It's a great post worth repeating in all I can say.
Dr. Randi J. Hagerman of the UC Davis MIND Institute, will be featuring Anavex 2-73 (among other potential therapies she is investigating) in her January 12th talk, The Fragile X Spectrum and new Targeted Treatments for FXS and ASD. Dr. Hagerman was the 2-73 Rett Syndrome trial Investigator at the UC Davis trial site:
The need for treatments that improve mitochondrial function, such as Anavex 2-73 can make a difference for both neurodevelopmental disorders and neurodegenerative disorders including Rett syndrome, FXS, Parkinson Disease, Alzheimer Disease and FXTAS.
https://health.ucdavis.edu/mindinstitute/videos/images/dls/hagerman-2021-dls.pdf
Plexrec,
I haven't heard but Dr. Hagerman is an expert and her opinions would be welcome, I'm sure.
If you can post a link that would be good.
thanks,
Thanks Mike,
Golf course closed today?
I'm sure there's some golfers out their who need your opinion on their golf swings. Don't want to leave them hanging.. JK