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"The scientific community has essentially finished with
amyloid - we've identified it, developed drugs, and can
clear it," Dr. Carmichael says. "The fact that clearing
amyloid doesn't substantially change the progression of
the disease means that other things are also going on in
the brains of Alzheimer's disease patients."
At UCLA, researchers are investigating, for example,
how inflammation may impact Alzheimer's disease
progression, as well as the role of the protein tau.
"There are a lot of interesting questions," Dr. Carmichael
says, "that now are even more important to answer."
"What's been surprising, and a bit disappointing, is that
these drugs didn't have as dramatic an effect on
cognitive recovery or cognitive decline,
Dr. Carmichael
says. "They had a very, very
- that's two 'verys'
modest effect on cognition."
https://www.uclahealth.org/news/fda-approval-brain-cancer-alzheimers#:~:text=Glioblastoma%20and%20Alzheimer's%20disease%20treatments%20advance%20through%20clinical%20trials.&text=A%20personalized%20vaccine%20developed%20by,aggressive%20form%20of%20brain%20cancer.
Lots of FUD being posted today on this holiday when most people are relaxing and having fun with friends and family!
I guess that is enjoyable for some!
From Michael Tavares on FB-
September 15th, Anavex CSO Dr. Walter E. Kaufmann will be delivering one of six keynote talks at the SSBP (The Society for the Study of Behavioral Phenotypes) 25th International Research Symposium. He will also participate in a live Q/A discussion alongside Dr. Randi Hagerman and others. https://ssbp.org.uk/
His keynote abstract, Development of Targeted Treatments for Rett Syndrome, can be viewed here: https://onlinelibrary.wiley.com/doi/pdf/10.1111/jir.13075 and below:
DEVELOPMENT OF TARGETED TREATMENTS FOR RETT
SYNDROME
W. E. Kaufmann1,2
1 Anavex Life Sciences Corp, New York, NY, USA
2 Emory University School of Medicine, Atlanta, GA, USA
Rett syndrome (RTT) is a severe neurodevelopmental disorder (NDD), characterised by multiple impairments, which affects predominantly females. Until March 2023, no drug treatments were available for RTT’s core symptoms. The recent approval of trofinetide by the FDA and the beginning of gene therapy trials represent major achievements with profound implications. They also represent a culmination of a process that began before the identification of mutations in the MECP2 gene as the main cause of RTT.
Knowledge on the pathology and neurochemistry of RTT and development of mouse models of Mecp2 deficiency has been instrumental in both identifying and validating new treatments. Availability of disorder’s natural history data has also been a major contributor. Efforts in the USA, Europe and Australia at characterising the range and evolution of neurobehavioral and systemic manifestations of RTT have allowed the most adequate selection of trial participants and the development of outcome measures.
The success of trofinetide’s studies, in combination with positive results of the mecasermin (recombinant human IGF-1) and blarcamesine (sigma-1 receptor agonist) drug development programmes, support strategies targeting multiple cell signalling pathways and endogenous homeostasis for ameliorating the numerous and greatly impairing symptoms of RTT, even in adults. These improvements have been observed despite short-duration treatments and suboptimal endpoints. Consequently, drug trials have facilitated the testing of therapies with even greater potential: attempts at correcting the genetic defect underlying RTT. Early information supports the safety of gene therapy in RTT; however, more time will be needed to determine their efficacy. The approval of a drug targeting core features of RTT has changed the stakeholders’ mindset, becoming not the end of a pathway but rather the first step in developing new treatments (even combining approaches). This and other learned lessons should be carefully examined by others in the NDD field.
Keywords: Drug trials, Gene therapy, Mouse models, Natural history, Neurodevelopmental disorders, Outcome measures, Rett syndrome
From Rett FB post
Annelise's Army • Follow
1d • G
Never give up trying, Annelise doesn't.
Annelise only learnt how to use a straw last
year, the past few days she has been learning
to hold her own cup!
Is Annelise still taking the medication
Anavex 2-73 !!! Does it appear to be helpful
if she is ?? Thank you very much!
Annelise's Army
yes Annelise is still
taking Anavex 2-73 we have found it
helpful for Annelise.
ENROLLMENT (ESTIMATED)
450
https://clinicaltrials.gov/study/NCT04314934?term=Anavex&rank=4
ANAVEX2-73-AD-EP-004
STUDY START (ACTUAL)
2019-10-10
PRIMARY COMPLETION (ESTIMATED)
2024-07-31
STUDY COMPLETION (ESTIMATED)
2024-07-31
https://clinicaltrials.gov/study/NCT04314934?term=Anavex&rank=4
https://www.mdpi.com/1422-0067/24/16/12980
Thanks 5RingsPatsRule from ST!
https://medicalxpress.com/news/2023-08-hypothesis-mitochondrial-dysregulation-contributor-schizophrenia.html?deviceType=desktop
Bodes well for upcoming A3-71 trial!
Thx Trainguy on ST
That could be really great news for Anavex!
Thanks sokol
Interesting info attila!
Have you been following the lawsuit against Cidadel from Northwest Bio?
Yes Bas! I agree with all those points.
First week of October
https://pubmed.ncbi.nlm.nih.gov/37429704/
I don’t remember if this was posted.
The response from IR to Trainguy was this often discussed topic-
“Communications with the
FDA are ongoing and it would be
impossible to constantly disclose
each and every correspondence. If
and when something is material, we
will issue a press release and/or file
an 8K.”
Question to IR fromTrainguy1 on ST-
If the FDA agrees to evaluate
Blarcamesine for accelerated
approval for Alzhiemers, has the FDA
indicated what parts of the standard
NDA need to be submitted to obtain
accelerated approval? Also, is
Anavex's response to 21 CFR 312.120
[Foreign Clinical Studies Not
Conducted Under An IND] required to
obtain accelerated approval?
>IR-We communicate with the FDA if
and when needed, as such, final
decisions are made based on
management, board of directors and
scientific advisors input, as well as
potentially lawyers and others that
are well-versed in the dealings with
the FDA. Communications with the
FDA are ongoing and it would be
impossible to constantly disclose
each and every correspondence. If
and when something is material, we
will issue a press release and/or file
an 8K.
Adam F out with a new AVXL hit piece-
https://www.statnews.com/2023/08/10/anavex-blarcamesine-rett-syndrome-clinical-trials/
Wow, really? I think it’s time to take a break from this message board!
Pathetic.
The global schizophrenia drugs market was valued at $7.16 billion in 2021, and is projected to reach $12.53 billion by 2031, growing at a CAGR of 5.8% from 2022 to 2031.
SAVXL “The collaboration in the last
paper on anavex 3-71 has been with
Clario ERT, a company that is
dedicated to managing data from
clinical trials. Anavex is doing a good
job of surrounding himself with the
best, or at least those who know
what works in their field. On the web
there is a success story about a
company that got accelerated
approval in AD in 2023... I'll leave it
here:) “
https://clario.com/resources/case-studies/clario-supports-large-pharmaceutical-company-with-a-complex-alzheimers-disease-study/case-study-cs-img-alzheimers/
https://pubmed.ncbi.nlm.nih.gov/37515316/
uploa….. Bullish
Thanks Monregi from ST!
https://pubmed.ncbi.nlm.nih.gov/37515316/
From Monregi on ST
I didn’t realize that that snippet wasn’t already on Jesse’s homepage.
Not sure about the embargoed part weeks after the conference.
Sorry, no link. He should be putting out his AAIC report soon.
From MayoMobile’s interview on July 18 at AAIC-
“Dr. Grimmer thinks they're
being approached/in-convo with big pharma for
purchase/partnering which is why we have not
seen data yet.”
SAD what has happened to this mb.
Alberto J Espay @Alberto... 4d
Donanemab in early symptomatic
Alzheimer's disease - Infographic
of the phase 3 trial data published
by JAMA earlier today.
Highlights:
• No improvement
• ~3-point less worsening in iADRS
(below MCID)
• 3.4% slower decline (not 35%!)
@alzforum @alzassociation
Alberto J Espay @Alberto... 4d
We are treating Alzheimer's as if it
were cancer. It isn't. The brain still
shrinks at any level of amyloid
detection, as @Kariem_Ezzat
reminds us. Amyloid is a normal
reaction to pathogens of various
kinds. By shooting the messenger,
we only create the illusion of
success.
Mayo had his interview with Dr Grimmer yesterday.
Interesting!
Be patient… there is a good reason that they are not releasing the data yet.
From Jesse’s interview with Dr Grimmer, it sounds like “Anavex has bigger fish to fry” in regards to AAIC and full data release, is his take on the situation also!
That is my opinion as well. Anavex has bigger fish to fry right now. Their focus is crunching the data for both AD and Rett. Meeting with the FDA, and putting together rock solid NDAs
I wouldn’t be surprised if they are also meeting with potential BP partners preparing for commercialization.
Presenting at AAIC at this stage would serve little purpose, in fact, might be viewed negatively as if to say “ Hey look at us! We have an AD drug too!!”
Exploring the Innovative Therapies of AVXL – Anavex Life Sciences Corp. Common Stock
July 12, 2023
Anavex Life Sciences Corp. Common Stock, also known as AVXL, is a pioneering biopharmaceutical company that is making significant strides in the development of innovative therapies for the treatment of neurodegenerative and neurodevelopmental disorders. The company’s groundbreaking research and development efforts are focused on addressing the unmet medical needs of patients suffering from debilitating conditions such as Alzheimer’s disease, Parkinson’s disease, and Rett syndrome.
AVXL’s primary product candidate, Anavex 2-73, is a novel sigma-1 receptor agonist that has shown promising results in early clinical trials. This drug candidate is designed to restore cellular health and function by targeting the root cause of neurodegenerative diseases, which is cellular stress. By modulating the sigma-1 receptor, Anavex 2-73 has the potential to halt or even reverse the progression of these devastating diseases.
The company’s innovative approach to drug development is based on the concept of precision medicine, which involves tailoring treatment to the individual patient based on their genetic, environmental, and lifestyle factors. This approach is expected to increase the efficacy of treatment and reduce side effects, thereby improving the quality of life for patients.
In recent years, AVXL has made significant progress in its clinical development programs. The company’s Phase 2b/3 clinical trial of Anavex 2-73 for the treatment of Alzheimer’s disease has shown encouraging results, with patients demonstrating improved cognitive function and reduced disease progression. Furthermore, the company’s Phase 2 trial of Anavex 2-73 for the treatment of Rett syndrome, a rare genetic neurological disorder, has also shown promising results, with patients experiencing significant improvements in their symptoms.
In addition to Anavex 2-73, AVXL is also developing a pipeline of other innovative drug candidates. These include Anavex 3-71, a sigma-1 receptor agonist that is being developed for the treatment of cognitive impairment associated with schizophrenia, and Anavex 1-41, a sigma-1 receptor agonist that is being developed for the treatment of depression and anxiety disorders.
The company’s commitment to innovation and patient care is reflected in its robust intellectual property portfolio, which includes patents covering its drug candidates and their use in the treatment of various neurological disorders. This strong intellectual property position provides the company with a competitive advantage and protects its innovative therapies from competition.
AVXL’s innovative therapies have the potential to transform the treatment landscape for neurodegenerative and neurodevelopmental disorders. The company’s focus on precision medicine and its robust pipeline of drug candidates position it well for future growth. Moreover, the company’s strong intellectual property position and its commitment to patient care underscore its potential to deliver significant value to its shareholders.
In conclusion, AVXL is at the forefront of developing innovative therapies for the treatment of neurodegenerative and neurodevelopmental disorders. The company’s groundbreaking research and development efforts, its focus on precision medicine, and its robust pipeline of drug candidates make it a compelling investment opportunity in the biopharmaceutical sector. As the company continues to advance its clinical development programs and bring its innovative therapies to market, it is poised to make a significant impact on the lives of patients suffering from these devastating diseases.
https://www.claytoncountyregister.com/uncategorized/exploring-the-innovative-therapies-of-avxl-anavex-life-sciences-corp-common-stock/12929/
From ST message bd-
100fold 18m
SAVXL The paper on small trials that
was linked here this morning was just
published in a peer review journal.
Note the 4 authors. 3 of them are
from Anavex, the 4th is an expert
statistician who worked for Vertex
Pharmaceuticals and developed
"modeling and simulation as the
cornerstone for drug development,
and his group supported all
development programs in the
company' I believe Vertex is a good
example of what to expect for future
valuations of AVXL. They are a
company that successfully developed
a drug development platform which I
believe is similar in concept to our
Sigmaceptor platform. That he is co-
authoring on this peer review article
with Anavex speaks volumes.
Another dot..
MayoMobile 39m
SAVXL Just finished reading the
unlocked: pubmed.ncbi.nlm.nih.gov/
374...
Extremely high effect sizes, especially
in S1R WT patients with longer
exposure to therapy.
It looks like Anavex has employed a
robust data analytics method to
increase strength of low-N trial
results. This will be helpful in future
rare disease trials as well.
It was good to see foreshadowing on
how the company will probably
display 2b/3 Alzheimer's data. Very
excited for data breakdowns on
dosage, S1R gene variants, and
probable metrics tying drug efficacy
to disease etiology (ex. Patients with
a primary mitochondrial cause
improve over baseline whereas
patients with a primary cholesterol
etiology decline but slower than
placebo, etc.).
I think we are about to be blown
away by cognitive and functional
improvements in a large number of
Alzheimer's patients. I'm also keen
for biomarker data, and charts
showing the longitudinal change
(slowing or halting) of disease
progression in most patients. Bullish
IMO, the reason that they have not yet moved on the Fragile X, PD,or rare disease trials is that they are focusing on the confirmation AD trial and of course, finishing up the RETT trial.