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As amyloid-targeting therapies for Alzheimer's fail, where might success lie?


tupungato/iStock via Getty Images

Mid- and late-stage data readouts over the last few years for Alzheimer's therapies have had a similar theme: Failure.

Almost all these candidates have one thing in common: They target reducing beta-amyloid plaque in the brain

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Rod Raynovich profile picture
excellent charts and overview!
Darp Research profile picture
This article missed NervGen NVG-291, which may be the most promising AZ drug.

Vancouver, British Columbia--(Newsfile Corp. - August 2, 2022) - NervGen Pharma Corp., (TSXV: NGEN) (OTCQX: NGENF), a clinical stage biotech company dedicated to developing a first-in-class neuroreparative drug to treat nervous system damage, will present the study design for its upcoming Phase 1b/2a Alzheimer's disease clinical trial at the 2022 Alzheimer's Association International Conference (AAIC) on August 3, 2022. NervGen's Chief Medical Officer, Dr. Daniel Mikol, will present a poster summarizing unblinded data from the single ascending dose (SAD) cohort of the study and interim blinded data from the multiple ascending dose (MAD) portion of the study, and for the first time will introduce the study design for the upcoming Phase 1b/2a trial of NervGen's lead drug candidate, NVG-291, in subjects with mild cognitive impairment or mild dementia due to Alzheimer's disease.

Dr. Mikol stated, "I am very excited about sharing our trial design at AAIC. Importantly, it was developed with substantial input from our distinguished Alzheimer's Disease Clinical Advisory Board which is comprised of leading physicians and researchers in Alzheimer's disease."

George Perry, PhD, Founding Editor-in-Chief of the Journal of Alzheimer's Disease and Semmes Distinguished University Chair in Neurobiology at the University of Texas, San Antonio, and member of NervGen's Alzheimer's Disease Clinical Advisory Board, stated, "I believe NVG-291's novel mechanism of action and the demonstration of function improvement in several different animal models of nervous system damage is of significant interest to the Alzheimer's disease field. I'm excited about playing a part in testing this drug in patients so we can determine if the repair mechanisms such as increased plasticity, axonal regeneration and remyelination results in favorable effects in Alzheimer's disease patients."

Paul Brennan, NervGen's President & CEO, stated, "NVG-291 has significant potential to repair the damage from this relentlessly progressive disease. Given the unique mechanism of NVG-291, we believe we have an opportunity to see changes in imaging biomarkers in this trial, which has been designed to give us the best opportunity to detect efficacy in Alzheimer's patients."

It has already as of last week shown more promise for Stroke than any drug before.

7-28-2022, peer reviewed study shows NVG-291 repairs stroke damage 7 days after a stroke, truly a breakthrough as no drug has ever done anything like this before. finance.yahoo.com/...

NervGen $NGENF up 40% in 3 Days on news of 65% repair of damage in tests on Stroke Damage with NVG-291, but is this an underreaction?
This is an animal study, but results like these have never been shown before in any tests. No repair of stroke damage has ever been shown. And 75% of the time the results for humans are 50-100% as good as for animals. Even at 50% as good, it would be the best human stroke drug ever.

NervGen has already regenerated human neurons under the microscope and neurons in live rats with severed spinal cords. The rats regained most of the function in paralyzed legs.

The market cap of NervGen is just over $100 million. It is currently in Phase 1 FDA trials in humans and is about to go into Phase 2.

NervGen has exclusive rights to the technology from Case Western University. It is backed by top experts in the field and staffed by very high-level pharma executives.

It has no competitors that can repair damage to the nervous system. If it works 50% as well in humans as in animals, a $15 billion market cap is feasible.
Anthony Cataldo profile picture
No mention by SA of the SAVA patent...example:

Ocuphire gets new U.S. patent for its oral drug candidate APX3330 for use in diabetics
SA NewsWed, Jun. 29
Desert M profile picture
@Anthony Cataldo Outrageous selective reporting.
Anthony Cataldo profile picture
Would be nice to get a 9A EST PR on the Wang patent. By the way, Wang is a "star" at work. Patents are a big deal at universities. Stay well.
@Anthony Cataldo This patent seems just reveal the SAVADX trade secret. From a trade secret to a patent. From presentations it seems current SAVADX is not good enough to diagnose AD. The company is trying a new approach for SAVADX.
Anthony Cataldo profile picture
Guessing SA will note new patent for SAVA.
Anthony Cataldo profile picture
@Anthony Cataldo For all practical purposes, this patent news is not yet out.
skeptic67 profile picture
@Anthony Cataldo I really wish they would say something. And please, NO momentum-killing trading halts, Remi!
Anthony Cataldo profile picture
@skeptic67 This is your opportunity to buy before the news hits.
Anthony Cataldo profile picture
I am guessing 9A PR on new patent.
Anthony Cataldo profile picture
Pain Therapeutics, Inc. (Now Cassava Sciences) Granted U.S. Patent #11,385,221 Alzheimer's disease assay in a living patient
BENZINGA 6:28 AM ET 7/12/2022
Desert M profile picture
@Anthony Cataldo This sounds like very good news, applied for 3 years ago, but not being a superior brain worker, I am cannot tell anyone the practical import of this patent.

My sense is that it is a source, with other developments, for the extreme confidence SAVA staff have in the drug.

Here are some details from the link:

Alzheimer's disease assay in a living patient

An assay for Alzheimer's disease (AD) pathology in a living patient is disclosed wherein an amount of .alpha.7nAChR or TLR4 in a FLNA-captured protein complex or .alpha.7nAChR in an A.beta.-captured protein complex or .alpha.7nAChR-FLNA, or TLR4-FLNA and/or .alpha.7nAChR-A.beta..sub.42 complex present as a protein-protein complex in a sample is compared to the amount in a standard sample from a person free of AD pathology. An amount greater than in the standard sample indicates AD pathology. Also disclosed is an assay predictive of prognosis for treatment with a medicament in which the amount of an above protein or protein complex is compared to an amount in the presence of a medicament that binds to a FLNA pentapeptide and contains at least four pharmacophores of FIGS. 7-12. An amount of protein or protein complex determined in the presence of medicament that is less than the first amount indicates a favorable treatment prognosis.

Inventors: Wang; Hoau-Yan (Philadelphia, PA), Burns Barbier; Lindsay (Austin, TX)
Name City State Country Type

Pain Therapeutics, Inc.
Assignee: Pain Therapeutics, Inc. (Austin, TX)
Family ID: 1000006427191
Appl. No.: 16/289,215
Filed: February 28, 2019
Anthony Cataldo profile picture
@Desert M Really looking forward to seeing how FUDsters deal with this one. You can forward the link to those writing negative articles over the past year or so. I guess Bik could criticize the U.S. Patent office.
Desert M profile picture
@Anthony Cataldo So am I. The fuds are beginning to look like a lobster who has entered a lobster trap....the dead fish looks tempting but there is no way back out after it is consumed.....then the lobster is our dinner!
Anthony Cataldo profile picture
Another day of biotech mentioned favorably on CNBC. Sector may be good. Inflation kills the sector, and if overall inflation has peaked, this is good for the sector. Wage inflation will; however, persist IMHO. AJ
Anthony Cataldo profile picture
Yesterday, after close, CNBC technicals guy said biotech sector hit bottom and turning. This, after they said biotechs up about 25% since mid-June. Also said about 850-860 biotechs. Recall some big deals, recently. Every stock is different, but I would expect some fund flows into biotech sector. Stay well.
Anthony Cataldo profile picture
Good: Guy on CNBC says shorts may be more reluctant to short biotechs. He is on right now.
Darp Research profile picture
@Anthony Cataldo I tend to agree. Chart wise biotech looks decent here.
Anthony Cataldo profile picture
Would love to hear a deal for SAVA like this....

5:19 am ET
Merck Is Now in Advanced Talks to Buy Cancer Biotech Seagen for $40 Billion: Report -- Barrons.com
Dow Jones
@Anthony Cataldo I am not sure if a full buy out would be the best option, maybe.
Anthony Cataldo profile picture
@Totenpole $40B is about $1K per share. Partnership is good, also, and what we anticipate.
pretty good summary and interesting discussion !
@valtr rather, not so good a summary, and a mostly corrective discussion
In general people pay more attention to the louder voices (The squeaky wheel gets the oil), but forget the quality of the information.
When the company presents the clinical cognitive scores, the biomarkers from the clinical trials, these are generated by highly regulated healthcare industry, they are presented as the FACTs in these specific trial settings.

Let me repeat, these are presented and treated as FACTs, if the company is found to have fudged any of them, there is virtually no way for them to escape punishments from law.

While the FUD campaign is merely expressing "concerns", these are opinions. They can hold these same "concerns"/opinions even after SAVA's drug simulilam is approved and reaches $100B annual sales without breaking any law.

It is your own choice which camp you like to join.
@Sam_101 Even two out of the four anecdotal accounts have to be true: SAVA sponsored Dr. Gonzalez's story, and Dr. Nikolov was talking about the patients under his care(clinical trial site).

These are all verifiable facts.

Otherwise, all of them can be in big trouble.

“In general people pay more attention to the louder voices (The squeaky wheel gets the oil), but forget the quality of the information.”

Kind of like how cheerleader Remi Barbier boasted wildly about SAVA and that noise resulted in a hyped run up, and subsequent fall.
@brad_scat I don't realize how despicable you are.
Why do you hate SAVA so much?
Falconetti profile picture
Regarding Simifulam:

Desert M profile picture
@Falconetti This is a very good excerpt. No where did it state that PH3 had to be completed to arrive at efficacy.

If a sizeable number of PH3 patients show improvement at about 1 year (such that it makes sense to unblind) perhaps approval comes early.

If 120 enrolled in PH3 as of May 5,2022 (if true) then there could be adequate evidence by June 30 2023 for PH 3 to potentially unblind.

This would be a nightmare for shorts and foundation could be laid in advance of this with solid OL 200 results, and solid CMS results, which number many as 165.
Clearly, science has not discovered the cause of AD. They are taking shots in the dark because the rewards of a hit are so great.

Sometimes we miss the elephant in the room, because we are so focused on expensive tech. How much of this is caused by lifestyle? I see a large percent of the population running around on 10 or more prescription drugs, drinking large amounts of alcohol, getting high on various legal and illegal chemicals. And probably a large cause, sugar in very high doses. No exercise, sleep deprived and on drugs to stay somewhat alert.

Walking young zombies grow up to be walking old zombies.
@mkkby The incidence of Alzheimers in India is 25% of the US diet or genetics?
coffeebreak profile picture
@maybewiser it is mainly the US diet, the food quality and the food quantity, plus the eating habits. it is important what you eat every day and how you eat it too.
David/David profile picture
@mkkby Alcohol consumption in the US is down, not up. Obesity, on the other hand, is way up. As for dementia, the highest rates in the US, maybe the world, are on Guam. This was observed before the westernizing diet appeared on that island.

It would help if the powers that be at the NIH/NIA started funding more non-amyloid work. Maybe amyloid really is the consequence and not the cause??
Clearly the science hasn't figured out what actually causes Alzheimer's disease. The indication is that plaques are a symptom rather than a cause. The best choice is to wait for better science rather than buying into the snake oil.
Desert M profile picture
@hound1965 if you had AD would you wait?

The SAVA scientists have been researching ad for years... they have published results...they have done early trials...they are now doing later trials... why do you think this is not potentially better science based on the results thus far?
@hound1965 @mkkby
Actually Dale Bredesen, MD, has done an excellent job explaining the causes of Alzheimer's disease. Trouble is, there is not one single cause, as with other, simpler diseases, but rather a constellation of several dozen possible causes which present in various combinations and permutations. Dr. Bredesen has developed protocols so that symptomatic individuals can determine which of the many possible causes are pertinent in their situation. Then various remedies can be applied. It's a complex disease with an array of causes and a number of remedies; each case must be dealt with individually. It's all explained in his book, "The End of Alzheimers" (2017) which you can probably find at your local library or purchase at Amazon or elsewhere for around $15. I had symptoms of cognitive impairment as a result of some heavy chemotherapy treatments. It was like a preview of Alzheimer's. For me, Bredesen's book was a real page turner. I've followed a number of his suggestions pertinent to my situation, and I'm pleased with the results.
Please read the book! It was life-changing for me.
(note: Bredesen has published several books. Be sure to start with his FIRST book, "The End of Alzheimer's" published in 2017. The other books have similar titles but they are follow-ups.)

@hound1965 Waiting for better science?
SAVA and others are advancing the science.
Falconetti profile picture
Let's just ask the question why 200 patients take the alleged fraud drug Simifulam for a year during OL and then about 165 of them decide to go into the CMS study for 6 more months, along with the risk of being in the placebo group. And now there is also a 92 week extension study following this. What patient or relative will be found if the drug doesn't help? Who wastes so much time with a fatal disease without getting any benefit from the drug?
Desert M profile picture
@Falconetti yes this is exactly the point that no one can answer....anyone who has ad knows that it is a race against the clock....yet people stay on the drug... it will come to the point that certain people will have stayed on the drug so long that they should have been dead by the time the latest trial (a total of 4 years) ends... Yet Sava is not given credit and many people do not seem to believe that the drug is providing a real benefits... To extend a trial an additional 92 weeks for a drug that provides no benefits is inconceivable... And it's not like the trial is being extended for 10 or 15 or 20 people....now as many as 165 are invited who have been on the drug already two years to participate in almost two more years... Someone who has AD declines in 2 years and two years after that at least in the case of my late father they will be confined to a wheelchair and unable to care for themselves... If this drug provided no benefit whatsoever after the first two years no one would go on it for two more years and that I think is both your point and my point.
@Desert M Well I agree with this, but it should be pointed out that the drug is free to them, but still, why even take it for free if it doesn;t seem to help?
Desert M profile picture
@Stockdoc96 that's exactly my point... this is a wasting disease...you can't afford to be on a drug that doesn't work if you have ad... And no one would know better than if the drug worked then the caregivers... sons daughters, brothers sisters, etc.
Jonathon, with all due respect, I think it's too premature to make a call SAVA. While I am not an expert in anything, I do use plain old common sense to help me analyze and understand many of my investments. Hence, I ask three questions:

1 - Has any malfeasance been proven? No, there has been nothing to date that conclusively indicates any malfeasance or efficacy issues. Large Pharmaco's and Biotech's, with an army of researchers, lawyers, and allies, have had testing and reporting challenges in past New Drug Approvals (NDA). Can you imagine the difficulty for a small firm of less than 150 people. Subpar research and clinical trials reports are not acceptable, but do not mean that there was any wrongdoing.

2 - What's In It for Me (WIFM)? A cadre of investors and legal firms have been either shorting or threatening litigation. All clearly have a vested interest in raising the heat on SAVA's and its eventual demise.

3 - How is the internal team reacting? SAVA's management team is under huge pressure. If you look at their credentials, they could work anywhere. The easy thing to do would be to bail, especially for those who just joined in the past 2-4 years. Yet, has anyone them bailed? NO. Not to my knowledge. If that doesn't say something about this company and drug, I don't know what will.

Let's hold off on making any prognostications until we have the FACTS. "The truth will set you free". For better or worse, the results of the 3rd clinical trails will do that for all of us.
if fraud existed at SAVA it would have been detected by now, given the large number of people with a vested interest in finding it
skeptic67 profile picture
@LongviewInvestor Excellent points, all. Articles like this are both sides-ism at its worst. The only thing controversial about SAVA is the endless stream of baseless accusations made against it.
Desert M profile picture
@LongviewInvestor very good points... we should hear them more often!
MickeyD77 profile picture
I have been watching Anavex (AVXL) for a number of years and I think their primary product Anavex 2-73 may end up being the earliest treatment approved for multiple diseases (Rett syndrome, AZ, Parkinson's). Data should be out in next 6 months that may get this drug to market in short order. Some of you may be familiar with a drug that is approved for Alzheimer's disease called Aricept. This drug does work to stabilize AZ but only for a short period of time (6 months-2 years) and then AZ progresses quickly in a downhill manner. I think that Anavex 2-73 may work in a similar manner (stabilizing the disease) but it has data that shows that it stabilizes the progression for much longer (they have data showing 4 years of stabilization).
It is important to note that Anavex 2-73 does NOT cure AZ but stabilizing it for a long period would be a miracle. There are probably other drugs coming that may "cure" the disease but Anavex 2-73 may be the quickest to market.
Having dealt with a family member with AZ, stabilization of the disease at an early time would have been a godsend.
04 Jul. 2022
@MickeyD77 Anavex 2-73 purports to mediate it's effect via Sigma1 receptor activation. It's funny you mention Aricept as one of it's actions is
Sigma1 receptor activation with receptor binding that is even stronger for Aricept (not that that has to be the full story). AVXL has never put forward a credible reason for 2-73 to do the Sigma1 job better than Aricept much less data that would support such a claim. Add to that some dubious treatment of the their AD data and I think the prospects for success are much lower than the many proponents here expect.
MickeyD77 profile picture
@REM I have not looked at clinical trial data for Aricept for a long time but it appears that Aricept does not stabilize for a long period of time but Anavex 2-73 has data up to 4 years and continuing. Anyone know how long Aricept works in the real world?
Also, Anavex 2-73 has data supporting efficacy in PD and Rett. Not sure if Aricept has been looked at in these areas.
If indeed, they find that Anavex 2-73 can stabilize AZ for long periods of time, this will fill a huge societal need.
@MickeyD77 just a basic point the disease progression of Rett, ALZ and PD are very different.
Therefore it may be better to say that Anavex are targeting the neurocognitive features of late stage PD, rather than PD in its entirety. If Anavex do not put 2-73 vs Aricept there will be lots of questions.
And as cited many times the causes of these diseases are not well understood and the likely fix will be a cocktail of drugs.
Tridentine profile picture
The best trade I ever made was going long BIIB when no one thought Aduhelm wouldn’t be approved then dumping out day later when it was,then never looking at an Alzheimer’s drug company again.
mpcascio profile picture
@Tridentine Good for you. I too was going to take the plunge but then I backed off. I really never thought it was going to get approved.

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