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The Undisclosed "Rare Disease"
In the list of ongoing Anavex drug developments, once again, an undisclosed, "new rare disease" was mentioned:
What? Blarcamesine As An OTC Prophylactic?
Because BMS Wants to Own the Schizophrenia SOC
What does Dr. Sabbagh actually know?
Anavex 3-71 in New Schizophrenia Trials
Of course, blarcamesine against Alzheimer's is the major target for Anavex. But be aware of the company's new trials of Anavex 3-71 to safely and effectively treat patients with schizophrenia:
Should I Buy Some?
Right now, AVXL is up 7.27%. Any reason for this? I've got a few dollars sitting in my brokerage account. Should I buy a few more AVXLs? Why? Why not?
The experts in biotech investing are all here. Let everyone know your thoughts, please.
[Oops. That was a few minutes ago. Now, it's up 8.58% for the day. How come?]
Ensuing Anavex Failures—So Sad for the New Guy
If Australia, Then the World Right Away
The CNS Disease Semmelweis Factor
Anavex Has Valid Data
They are, actually, heroes.
How Come?
Schwab shows that AVXL closed at the high of the day, at $5.12. A for-the-day price gain of $0.28, 5.79%. Not typical.
Are some excited about the prospects of Anavex 3-71 being clinically tested in patients with schizophrenia?
If Approved, the Numbers Will Be Big
I just took those US schizophrenia numbers, and with more conservative numbers punched them into my spreadsheet.
Because some of the “real experts” have criticized me for previously posting tentative spreadsheet projections of Anavex financial data, for when the company is able to sell a drug to treat a disease, in due deference I’ll refrain from posting either my entries or their results.
But suffice it to say, even at very conservative numbers, should Anavex get approval to sell Anavex 3-71 to treat schizophrenia in the US, holders of AVXL positions of even the most moderate size will be well-rewarded.
Readers Beware --- Always, Everyone
When the Results Appear
Lots of Patients Are Available
I could not find the "N" for either part A or B.
I'm not aware of the number of schizophrenias patients that will be enrolled in the new clinical trial.
Of course, to attain statistical validity, a clinical study has to have an adequate number of patients being tested with the drug. And the trial participants have to have the properly-diagnosed disease being tested in the trial. For many CNS diseases, these can be complicating factors. As in Rett Syndrome. A rare disease, with a limited number of patients available at typical treatment centers. But in the case of schizophrenia, there are millions of Americans suffering from the disease. Every neurologist has dozens of such patients. Getting enough schizophrenia patients to adequately test Anavex 3-71 will not be a problem. When they find out about the drug's safety profile, from the Phase I study, patients will eagerly enroll,
The Anavex 3-71 Schizophrenia Study — Short and Strong
The Anavex 3-71 trial for the treatment of schizophrenia has officially begun. The first patient has entered the trial.
https://www.globenewswire.com/news-release/2024/03/18/2847701/29248/en/Anavex-Life-Sciences-Initiates-Placebo-Controlled-U-S-Phase-2-Clinical-Trial-of-ANAVEX-3-71-in-Schizophrenia.html
This trial will differ in several ways from any of Anavex’s previous human trials, in three significant ways.
First, this is the first Phase 2 trial of Anavex 3-71 in humans. To date, Anavex’s lead drug being tested and targeted at central nervous system (CNS) diseases has been Anavex 2-73, blarcamesine. Now, for the first time, it will be Anavex 3-71
In murine (lab animal) studies, Anavex 3-71 has been shown to be clinically functional at very small dosages, measured in micrograms, compared to the milligrams required for blarcamesine. (A microgram is one one-thousandth of a milligram; 1 microgram = 0.001 milligram.) With this, in both murine and Phase 2 safety and tolerability studies in humans, the drug has proven to be very safe, with no treatment-obviating adverse events (“side effects”). This contrasts with the very common and significant side effects of existing drugs used to treat schizophrenia.
Simply, the drug works safely and strongly.
Secondly, the two drug testing periods for the new trial, where Anavex 3-71 is being tested on patients with schizophrenia, are very short. In Part A, a proper statistically-valid double-blind, placebo-controlled, multiple ascending dose trial, will be used to select the exact dosage to be used in the main study, in Part B. The Part A study will take just 10 days for each enrolled patient.
The Part B study period, using the Part A-determined dosage, to see if Anavex 3-71 can yield safe, effective therapeutic outcomes for patients with schizophrenia, will require only 28 days of dosing and patient study.
Lastly, it appears that the biochemical anomalies that cause the symptoms of schizophrenia lend themselves to prompt, almost immediate amelioration by Anavex 3-71. Hence, the short study period of the trial.
And, as stated in the announcement, to determine treatment efficacy, “Standard clinical outcome measures, including the Positive and Negative Syndrome Scale (PANSS), and novel fluid and electrophysiological biomarkers will be assessed.”
At the end of the study, sometime soon, there should be no haziness or statistical questions about Anavex 3-71's ability to successfully treat schizophrenia. Millions of patients suffering from schizophrenia can have hope.
5.4X
For what it’s worth, Charles Schwab states that the 10-Day Average Volume of AVXL is 1,201,478.
The Schwab web page states that today’s volume was “6.5M, Above Avg.”
So, today, an AVXL trading volume increase of 5.4X over the 10-day average.
Is it one to ten?
Nothing Useful Can Be Posted
[Test}
Eventual Anavex Prophylaxis of Alzheimer’s
Anavex in the Insomnia Treatment Market
I just read today's Anavex U.S. patent application, where a patent for "A2-73 AS A THERAPEUTIC FOR INSOMNIA, ANXIETY AND AGITATION" is applied for.
Of course, insomnia, anxiety, and agitation are common complications of Alzheimer's disease, making things difficult for both people with Alzheimer's and their caregivers. Being able to therapeutically obviate these behavioral anomalies with Anavex's sigma-1 receptor molecules would be a major accomplishment.
The market for this use of Anavex 2-73 (blarcamesine) is gigantic. The vast majority of Alzheimer's patients suffer from insomnia, anxiety, and/or agitation.
But, of course, insomnia is not restricted to Alzheimer's patients. About one in ten Americans has chronic (long-term, persisting) insomnia, which pathologically disrupts normal living and mental and physical health.
https://cfah.org/sleep-statistics/
Presently, there are a number of prescription anti-insomnia drugs. But they have many deleterious side effects, which can be as bad or worse than the insomnia that is being treated.
In every human clinical trial of Anavex 2-73 (blarcamesine) adverse events ("side effects") have been either absent altogether, or involved only moderate to slight dizziness or headache that did not persist. The Anavex sigma-1 receptor agonists, such as Anavex 2-73 in every case have proven to be very safe and well-tolerated.
Consequently, as provided for in this patent application, there is the prospect that Anavex Life Sciences Corp can be a major producer of exceptional anti-insomnia drugs. The market for such will be billions of dollars.
Thank you for the suggested acronyms list edits.
I invite others to suggest edits they think would be helpful. In a few days, when everyone has had the chance to post their edits, I'll edit and re-post the list.
Here are the definitions of acronyms that appear on this message board:
AA — Alcoholics Anonymous — A voluntary organization that helps alcoholics recover.
Ad Comm — Advisory Committee of FDA — FDA (Food and Drug Administration) committee that advises on the approval of new drugs.
ADCS-ADL — Alzheimer Disease Assessment Scale - Activities of Daily Living — An analysis technique that quantifies the activities of daily living, in the presence of Alzheimer’s disease.
ADAS-Cog — Alzheimer Disease Assessment Scale- Cognition — An analysis technique that quantifies the status of cognition, in the presence of Alzheimer’s disease.
AGM — Annual General Meeting — An annual meeting of the shareholders of a corporation.
AVXL — The stock ticker symbol of the shares of Anavex Life Sciences Corp.
BIIB — The stock ticker symbol of Biogen Inc. The company owns, sells drugs for treating neurological and neurodegenerative diseases; a potential Anavex Life Sciences Corp competitor.
BLA — Biologics License Application — A formal request to introduce, or deliver for introduction, a biologic product into interstate commerce; after the drug has been approved by the FDA.
BTD — Breakthrough Therapy Designation — An FDA process to expedite the development and review of drugs intended to treat a serious condition, with preliminary clinical evidence that the drug may demonstrate clinically significant improvement over available drugs.
CC — Conference Call — A telephone call in which someone talks to several people at the same time. Often an event at annual meetings or technical conferences.
CEO — Chief Executive Officer — The individual holding the highest management position of a corporation.
CDR-SB — Clinical Dementia Rating Scale - Sum of Boxes — A statistics methods that yields global and Sum of Boxes (SOB) scores, with the global score regularly used in clinical and research settings to quantify dementia severity.
CGI-I — Clinical Global Impression - Improvement Scale — A scale where the clinician assesses how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention.
CNS — Central Nervous System — The tissues and organs in the brain and spinal cord.
CRO — Contract Research Organization — A company that provides clinical research services on a contract basis. CROs conduct clinical trials for pharmaceutical companies.
CRL — Complete Response Letter — Action by the Food and Drug Administration to a New Drug Application, indicating that the application will not be approved in its present form; but that it is approvable if certain studies are performed or more data is reported or manufacturing issues are cleared up.
CTAD — Clinical Trials on Alzheimer's Disease Conference — A periodic meeting focused on Alzheimer’s Disease Therapeutic Trials, with key leaders forming partnerships to speed the development of effective treatments to fight Alzheimer’s.
CTN — Clinical Trials Notification — This is the Australian equivalent to the FDA's IND or the EMA's Clinical Trials Application; where companies apply to be allowed to do clinical testing in their country or union.
CVR — Contingent Value Rights — In corporate finance, where certain rights are granted by an acquirer to a company’s shareholders, where some uncertainty is inherent.
DMT — Disease-modifying Therapy — A treatment that delays or slows the progression of a disease by targeting its underlying cause; as opposed to symptomatic treatments that merely treat the symptoms of a disease but do not address its underlying cause.
EHR — Electronic Health Record — A digital version of a patient’s paper chart; where real-time, patient-centered records make information available instantly and securely to authorized users.
EMA — European Medicines Agency — In the European Union the agency that evaluates and monitors human and veterinary medicines, and authorizes and approves the marketing of new medicines. Similar to the Food and Drug Administration in the U.S.
EU — European Union — The political union of the major countries in Europe.
FUD — Fear, Uncertainty, and Doubt — On this message board, a pejorative used to label posts or posters naysaying the viability or legitimacy of any Anavex topic.
GCP — Good Clinical Practice — An international quality standard, which governments can transpose into regulations for clinical trials involving human subjects.
HREC — Human Research Ethics Committee — Institutional committees that review research proposals involving human participants to ensure that they are ethically acceptable.
ICH — International Conference of Harmonization — A conference of international drug regulation bodies, such as the FDA and EMA that unifies technical requirements for the development and marketing of pharmaceutical products.
IND — Investigational New Drug — The FDA’s designation of a new drug for which a pharmaceutical company obtains permission to start human clinical trials and to ship an experimental drug across state lines before a marketing application for the drug has been approved.
IR — Investor Relations — At Anavex, on its website, Investor Relations is where shareholders or the public can ask questions about the company.
IRB — Institutional Review Board — A committee at an institution that mandates research ethics for proposed research done at that institution to ensure that the projects are ethical.
JPM — JP Morgan Chase healthcare conference — Sponsored by J.P. Morgan, this is an annual health care investment conference that focuses on emerging fast-growth companies, innovative technology creators, and members of the investment community.
LOI — Letter of Intent — A document outlining the understanding between two or more parties which they intend to formalize in a legally binding agreement.
LOL — Laughing Out Loud
LPLV — Last Participant Last Visit — Results submission to ClinicalTrials.gov is a deliverable required by law for most clinical trials, for which the deadline occurs one year from reaching the primary endpoint Last Patient Last Visit (LPLV).
M1 — muscarinic acetylcholine receptor M1
MM — Market Maker — A market maker or liquidity provider is a company or an individual that quotes both a buy and a sell price in a tradable asset held in inventory, hoping to make a profit on the bid–ask spread, or turn. The benefit to the firm is that it makes money from doing so; the benefit to the market is that this helps limit price variation by setting a limited trading price range for the assets being traded.
MAA — Marketing Authorization Application — An application submitted by a drug manufacturer seeking permission to bring a newly developed medicinal product to the market.
NDA — New Drug Application — The vehicle in the United States through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing.
NDA — Non-Disclosure Agreement — A legal contract between at least two parties that describes confidential material, knowledge, or information that the parties agree to disclose only to themselves.
MOA — Mechanism of Action — The specific biochemical interactions by which a drug produces its pharmacological effects.
NICE — National Institute for Health and Care Excellence — In the United Kingdom, an independent public body that provides national guidance and advice to improve health and social care.
NIH — National Institute of Health — The primary agency of the United States government responsible for biomedical and public health research.
MAA — Marketing Authorisation Application —In the United Kingdom and European Union, an application submitted by a drug manufacturer seeking marketing authorization, to bring a medicinal product (for example, a new medicine or generic medicine) to the market.
MACD — Moving Average Convergence/Divergence — A stock trading indicator used in the technical analysis of securities prices, designed to reveal changes in the strength, direction, momentum, and duration of a trend in a stock's price.
MBA — Master of Business Administration — A postgraduate degree centered on business administration.
MMSE — Mini-Mental State Examination — A 30-point questionnaire used extensively in clinical and research settings to measure cognitive impairment.
MRI — Magnetic Resonance Imaging — A medical imaging technique used in radiology to form pictures of the anatomy and the physiological processes of the body.
OLE — Open-Label Extension — Extended portion of a clinical study of a drug where the study is no longer blinded; where the patient, if desired, is allowed to know and take the actual drug being evaluated in the study.
OR — Odds Ratio — A statistic that quantifies the strength of the association between two events, A and B. The odds ratio is defined as the ratio of the odds of A in the presence of B and the odds of A in the absence of B, or equivalently (due to symmetry), the ratio of the odds of B in the presence of A and the odds of B in the absence of A.
P2b/3 — Phase 2b/3 Clinical Trials — Phase 2b trials specifically, rigorously test the efficacy of a drug in terms of how successful it is in treating, preventing, or diagnosing a disease. Phase 3 trials assess the effectiveness of a new intervention and, thereby, its value in clinical practice. Phase III studies are randomized controlled multicenter trials on large patient groups (300–3,000 or more depending upon the disease/medical condition studied) and are aimed at being the definitive assessment of how effective a new drug is, in comparison with current treatments.
PET — Positron Emission Tomography — An imaging technique that uses radioactive substances known as radiotracers to visualize and measure changes in metabolic processes, and in other physiological activities including blood flow, regional chemical composition, and absorption.
PD — Parkinson’s Disease
PD — PharmacoDynamics — PD describes how drugs act on target sites of action in the body.
PDD — Parkinson's Disease With Dementia
PK — PharmacoKinetics — PK describes the movement of drug into, through, and out of the body, involving its absorption, bioavailability, distribution, metabolism, and excretion.
PTA — Post Trial Access — The providing of continued access to study interventions (drugs, etc.) once a trial is completed, or after a participant’s involvement in a trial has ended
PR — Press Release — A message or information document released to news media.
RA — Regulatory Agency — A governmental body that regulates particular technologies or programs.
RSBQ — Rett Syndrome Behavior Questionnaire — A caregiver-completed rating scale assessing a wide range of neurobehavioral symptoms of Rett Syndrome.
RSI — Relative Strength Index — An equity price momentum indicator of speed and magnitude of a security's recent price changes.
RWE — Real World Evidence — Evidence obtained from real world data, obtained outside of randomized controlled trials or is generated during routine clinical practice.
SA — Scientific Advice — The provision of scientific advice for medicines and medical devices, in European Medicines Agency.
SAP — Statistical Analysis Plan — A document that gives comprehensive instructions on statistical programming and reporting of clinical trial outcomes.
SEC — Securities and Exchange Commission — The federal agency that regulates the U.S. securities markets, to protect investors.
SOC — Standard of Care — In relation to medical therapies, the drug or therapy most commonly used, preferred, and recognized as the most effective.
SP — Share Price
SPA — Special Protocol Assessment — A legal mechanism that enables the Food and Drug Administration (FDA) to provide input into the design of certain studies critical to marketing approval of medical devices or drugs prior to initiation.
Stat Sig — Statistical Significance – A complicated statistical datum that indicates the validity of a clinical result. Results with a P-value of 0.05 or lower are considered statistically significant; that the results are not merely by chance.
TA — Technical Analysis — The prediction of future stock prices by examining price movements, charts, trends, trading volume and other factors.
TGA — Therapeutic Goods Administration — Australia’s equivalent of the US FDA, which approves drugs.
TGD — The Good Doctor — A superficial reference to Dr. Christopher Missling, Anavex CEO.
TLD — Topline Data — A summary of demographic data, the data for the primary endpoint(s), the data for any secondary endpoint(s), and a summary of safety data, of a clinical trial.
TLR — Topline Results — No actual definition of this clinical results term can be found. There is a wide variety of definitions.
WGT — We Got This — A phrase applied to those who support or have exuberant perspectives on Anavex Life Sciences Corp.
XBI — An exchange-traded fund that tracks the performance of the biotechnology segment of the S&P Total Market Index.
Alzheimer’s MAB Drug Trails — All Practical Failures
As we all know, conventional medical wisdom arrogantly knows that the cause of Alzheimer's disease results from the accumulation of toxic proteins in the brain, beta-amyloids and tau plaques. Great effort has been extended to create monoclonal antibody (MAB) drugs that induce the immune system to attack and remove the waste proteins. With that, Alzheimer's would be cured.
And, of course, the MABs approved to treat Alzheimer's have all produced putatively favorable clinical results, allowing regulatory approval for their sales and therapeutic use.
But, in fact, did those clinical test results actually portend therapeutic safety and efficacy in subsequent patients not in a clinical trial? Here's what a meta-analysis of 19 studies on the matter determined:
Yes, "SOC."
What will be the new replacements?
Need to Know Names of Clinical Trials
Posters often mention the "EXCELLENCE" trial. A nice suggestive name. But what is it? What's it testing, in whom?
Can someone post a listing of the names of the Anavex trials? Just as important as the enigmatic acronyms.
CVR?
Joseph, thanks.
I've added those to the list, including the non-technical acronyms. Readers need to know what all of the acronyms mean. Otherwise, confusion.
Anyone else, post your list. I'll be able to post the list sooner.
I'm going to have to spend some time writing out clear, concise definitions of the acronyms; beyond just what the letters stand for. For example: RSBQ --- Rett Syndrome Behavior Questionnaire.
Good. But what IS the Rett Syndrome Behavior Questionnaire? What do readers need to know about it to make sense of its appearance in postings on this message board? Talk (or an acronym) is cheap. Useful understanding requires effort on the part of both the writer and reader.
Sage, thanks!
What you just posted, a list of acronyms that appear on this message board, is important. Thank you for compiling and posting those. I'll copy your list, get it on my word processor, and update it when I see other acronyms in messages. I'll also try to provide helpful, detailed definitions and information on the acronyms.
All stuff I did when I taught advanced placement biology to talented biology students headed for science and medical student scholarships. My students got big scholarships, and I got awards for my teaching.
This Message Board Needs an Acronym List
In good, acceptable technical writing, acronyms, sequences of otherwise meaningless letters, need to be clearly defined, to be understood by readers. Of course, there is very little acceptable technical writing on this message board. Certainly, a good number of new readers click on to see what's being presented. But postings are perfused with WTHITs --- What The Heck Is This acronyms and new readers click away and go off. Understandably.
So, in my idle moments in the next few days or weeks I'm going to try to assemble a list of the multitude of acronyms lazy message writers so inconveniently use in their postings. Inquiring minds will want to know what all those capital letters stand for. If anyone wants to assist, I'd be delighted. When convenient, post some of the acronyms that confuse readers. Collaboratively, we can bring a bit of clarity on this issue.
Comparison of Three FDA-approved Drugs for Rett Syndrome
This is Anavex 3-71's First Step
As Expected, An Anavex 3-71 Human Clinical Trial
I’m pleased to learn of Anavex’s new placebo-controlled Phase 2 trial of ANAVEX®3-71 for the treatment of schizophrenia. Very much as I expected.
Too early for me to lay out or describe what I believe will be the significance of this new trial. Anything I’d post on the matter would be summarily bashed, by all the usuals, with the usual arguments we’ve seen through the years here. Right now, I’ll keep to myself my understandings of the future of Anavex 3-71 for the company, its shareholders, and patients.
But this won’t be a lengthy trial. Anavex 3-71's abilities to safely and effectively treat schizophrenia will be promptly determined and illustrated.
If those Phase 2 results are positive, the naysaying Anavex experts will, of course, tell us why the required Phase 3 trial will then be a failure and Anavex Life Sciences Corp will fold. There will be some statistical errors or insufficiencies, of course. The game will be over. We’ve seen it all before. I’ll sit back and watch.
We Should Be Honored and Thankful
Ok, But Nature Has Embargo Policies
Now, Let's See the Paper Itself
The Article Will Get Posted Right Here