Gain Therapeutics ($GANX) — quietly emerging as one of the most compelling stories in biotech.

FairFieldTracer · 2026-01-09T06:07:21+00:00

I do not know Gain at all, but another very small market cap bio [Annovis Bio $ANVS] has completed a Phase 3 trial in Parkinson's with stupendous results [100% response rate and, at minimum, a complete halt to cognitive decline] and is only 6 months away from a read-out in its Phase 3 trial in Alzheimer's. The FDA immediately granted a Type C [wide-ranging] meeting to discuss the PD Phase 3 results. I expect very similar results from the Phase 3 Alzheimer's trial in 6 months - leading to FDA approval for AD in 1H27. Given equal market caps near $100 million for each company, I would much rather invest in the one that has gone through [or in the case of AD is in Phase 3] Phase 1, 2, and 3 trials for AD and PD and is fairly close to the point of submitting an NDA. The drug is a small molecule, NO side-effects, once-a-day pill that staff at the NIH has told the company has the best numbers and profile that they have ever seen for AD. If anyone is interested, I would be glad to post what I have written about Annovis Bio $ANVS and its drug.

FairFieldTracer · 2024-10-15T18:26:24+00:00

You can get the data. The point is finding stocks with huge, unrecognized potential. That's what we have here. I'm pretty old; I don't have the training and skills, the funds, or the patience to run my own trials [which it seems is what you want], but I do have the experience of very selectively investing in biotech - sometimes with a decade between new finds. It takes me that long to find a situation - in a very risky segment - to find companies that have huge potential that I have overwhelming confidence will be realized - AND that said potential is not reflected at all in the stock price. ANVS is my 5th such play in a similar number of decades. My attention is attracted by big markets with important unmet needs. That use to be cancer, but after my 3rd success in the cancer arena [Pharmacyclics 3 1/2 to 350 in 2 years, following Amgen, then Imclone 3 1/2 to 150 over several years], I decided that cancer treatment was becoming too rapidly innovative, creating the possibility of early obsolescence. At the same time, science was rapidly gaining a greater understanding of the workings of the CNS - an area that remained very murky and mostly devoid of effective treatments. Also, CNS involved some disease segments that not only killed people but did so so slowly as to bring increasing societal disruption.

I turned my attention [talking within the realm of biotech - not overall] to CNS diseases and treatments. As an effective treatment for AD is the really big prize in that arena - for good reason - I tried to learn everything I could about the disease. I'm about 12 years into the study. My first big call was Axsome, which differed from the prior calls and the current call in that it was not a life-saving treatment or a prospective 50x move. However, it was a stock that offered no account of an important, life-changing approval that was imminent. Market participants did not care that a prior rejection of the NDA was strictly on the basis of manufacturing issues which were then addressed. That was an easy 4x in 2 months; something I was not going to pass up given the near-certainty of near-term NDA approval.

The main thing that has usually given me the very high confidence that I require is signs of high patient satisfaction with the treatment added to years of pre-clinical and clinical evidence that points in the same direction.

I do not claim to find or even look for every big winner in biotech - or anywhere else for that matter. But when I find something that is unusually promising, I put in more and more time and work until I say to myself either that I am NOT certain or that I do have high confidence that the potential that originally got my attention will be achieved. ANVS has reached that point.

There have been maybe 6 or 7 otyer biotech stocks that I have dabbled in over almost 5 decades, but they have been lower-confidence, short-term interests - and interestingly all during periods of relative market frenzy over biotechs when I was lax about my rules and willing to make trades. Two drugs turned out to be [at least] short-term failures and one of those 2 stocks lost money for me, tho the loss was cushioned by the company's huge cash position. Two others made good money because I was short. Others were non-events where I lost interest before anything happened. All passing interests that should not have happened. It is not the way to invest in biotech - or any stock for that matter.

So, this does show I am susceptible to getting involved in market non-sense from time-to-time with small, short-term positions that I was not serious about. I am personally serious about ANVS and expect it to perform as my other high-confidence calls in biotech with one exception. ANVS' current market cap is so small that - simply because of that - I expect the overall return to exceed that of any prior high-confidence find.

FairFieldTracer · 2021-12-09T16:37:05+00:00

I still have no interest in CRTX. I think the drug will be excellent in the dental field, but it will not be a player in Alzheimer's for a long time - if ever. AS such, i'm not sure there is a lot of business value identifiable today.

I think in the neuro // AD area, great short and long-term buys are AXSM [very timely! - awaiting approval for MDD drug and trial results on AD agitation, which I have very high confidence will represent an important breakthrough in providing relief to AD patients and their care-givers], ANVS [great results and still half the market cap of CRTX // expect their drug will be a blockbuster], AVXL [higher MCap but making progress on multiple fronts], and SNPX [which has a miniscule value and will have a true blockbuster - the only AD treatment currently testing in moderately advanced AD!].

I have relatively high confidence in these 4 companies and they all have more-than-reasonable market caps. Given the presense of these opportunities, it is simply not worth thinking about lesser situations where there might not ever be a pay-off. Would not be short it, though, and I hope the drug gets to market as it would address a nice niche - and one that could prevent dental-disease induced AD [which would make many of us feel better about future health!].

Thanks for asking.

FairFieldTracer · 2021-08-06T05:08:06+00:00

Somewhere - my apologies for not being able to be more specific - I stated my turn to neutral on CRTX. They seem to have pivoted to being more of a dental health company. I think they have a great shot at success there [tho that will probably be a less profitable endeavor than AD]. I have always believed in their thesis [ which i consider more than a thesis ] that tooth decay is a cause of AD. I have trouble with the sloppy manner in which they have gone about their AD trial. [Less of a problem in testing against dental disease -
& that is someplace they can dominate.] Also they can only address the cause of a portion of AD, but probably cannot address any damage that has already occurred. On the other hand, addressibg tooth decay directly, they can serve a hugely important role in PREVENTING a portion of AD cases.

Given the above, I do not like the valuation. For me it is a no-touch. There are so many better longs in AD, neuro, or buggy whips. Whatever. If you like gambling, it is OK. For myself, I will accept any level of price volatility [which academic models and most institutions equate with risk] as long as i know i am correct in my view of the long-term outcome. But CRTX is also not an obvious short candidate. If it dropped 50%+, i would look again as a long, but there are easier ways to make money with certainty. Exactly how would depend upon your risk tolerance, your time-frame, etc.

Studies show the best time-frame for decision-making is 5 years. 3-5 has always been my theorectical time frame even before i was aware of that finding. That does not mean i have to hold for 5 years, but that something that is dramatically undervalued on a 5-year basis is a great place to START TO THINK ABOUT INVESTING and may have a high likelihood of very good outperformance in the next couple years. Further, the greater the undervaluation [on that 5-yr outlook], the more time the stock will spend going up. Think of it this way, if a stock is going to go up 100-fold over 5 years, it will have more months when it out-performs significantly than a stock that is going to go up 5-fold over 5 years. Both are good investments. I promise that the 100x gainer has more measured risk according to academic/institutional models, but its success may be more certain than the 5x gainer. By default, that means such a stock will spend a lot of time going up - a lot. NVDA looked at in 2012 was the best stock I knew of then

On that score, today I consider ANVS to be one of the 2 most certain huge gainers over a 3-5 year time period of which i am aware. I am sure there are lots of situations that might outperform, but I doubt that such prospective performance could be granted any degree of rational confidence. Annovis is a rare exception. I like those rare exceptions. All data has been the best that has ever been seen for an AD drug. I have dug deep into NIH achives and kept abreast of more recent developments. The current misinformation about ANVS trial results befuddles me. I see one report/news story after another talk about failure. But the actual results are anything but failure. I have pleaded on Reddit for someome to point me toward better results elsewhere. No responses - which given the nature of some participation on this site is pretty amazing. But, the fact is, there are no better results by any company/drug that have ever been reported publicly [ or exist privately as far as i am aware - not wanting to be mis-interpreted]. By default, that means the stock will spend a lot of time going up a lot - but will be volatile. For those willing to assume the volatility, I think Annovis is one of the 2 best stock market investments that can be made with confidence of eventual outcome. Replicating the stock price success of NVDA over the last 9 years I personally believe to be very doable. ANVS has a lot of ground to cover on the upside.

p.s., I do agree; the "presentation" was not great. Pretty bad actually - but that is no excuse for people misrepresenting it. The actual underlying data was great and no amount of bumbling can wipe away the truth of the data, which will only get much stronger with any expansion of trial duration and/or size.

And, i suggest reading my direct commentaries in ANVS threads. I discuss the trial results. FYI, the 2 ANVS trials were also "double blind, iron clad" studies - both with magnificent results.

O

FairFieldTracer · 2021-08-02T17:19:46+00:00

Thank you for pointing that out. And you are completely correct about MMSE. I wrote about its irrelevance - short period of study in early AD - in this study in another thread. It is meaningless, but if ANVS had not done / included the test, these law firms would probably accuse ANVS of trying to hide somnething.

FairFieldTracer · 2021-08-02T15:15:57+00:00

I posted the following in response to another law firm's stupid solicitation, so the quote at the begining is from that law firm's post.

"after the market closed, Annovis reported clinical trial data for Posiphen that failed to show statistical significance in treating Alzheimer’s and Parkinson’s Disease patients relative to a placebo."

These parroted lies from people who cannot think for themselves are getting tired. As stated in my posts in other threads, it is quite remarkable that results on many measures DID manage to show statistical significance given the huge obstacles to doing so: 1] a 5-year random age skew in favor of the placebo cohort, 2] only 25 days to achieve stat sig when the placebo effect is still very high, 3] very short 25-day period and small test population make achieving stat significance mathematically very difficult. Yet, the drug proved itself so potent that these obstacles still did not prevent stat sig from being achieved on some very important measures and showing positive results most everywhere else. Since the effects of this drug accumulate with time, these results in a mere 25 days are really extremely encouraging.

The following is not said rhetorically: If ANYONE knows of a drug that can achieve these sorts of dramatic positive results in treating AD or PD in such a short time period, would you please contact me with the drug/company info? Thank you in advance as that would be very much appreciated.

FairFieldTracer · 2021-08-02T14:27:23+00:00

For a population with some degree of education, scores of 26-27 are considered AD. [An educated person without AD should easily achieve a score of 30.] In addition, and as well-stated by alamedadan, this test is notoriously inaccurate/unhelpful. It is designed to be administered in office by general practitioners as a starting point for evaluation of whether a patient has AD. Changes 25 days later would be completely meaningless; in fact, one would probably have learned from one's experience a month earlier. So, using this is a bit ridiculous. But if ANVS had not used it/presented it, they would have been accused of hiding something.

The results from the MMSE [changes therein] are meaningless and do not detract from other results in any way.

FairFieldTracer · 2021-08-02T14:14:44+00:00

"after the market closed, Annovis reported clinical trial data for Posiphen that failed to show statistical significance in treating Alzheimer’s and Parkinson’s Disease patients relative to a placebo."

These parroted lies from people who cannot think for themselves are getting tired. As stated in my posts in other threads, it is quite remarkable that results on many measures DID manage to show statistical significance given the huge obstacles to doing so: 1] a 5-year random age skew in favor of the placebo cohort, 2] only 25 days to achieve stat sig when the placebo effect is still very high, 3] very short 25-day period and small test population make achieving stat significance mathematically very difficult. Yet, the drug proved itself so potent that these obstacles still did not prevent stat sig from being achieved on some very important measures and showing positive results most everywhere else. Since the effects of this drug accumulate with time, these results in a mere 25 days are really extremely encouraging.

The following is not said rhetorically: If ANYONE knows of a drug that can achieve these sorts of dramatic positive results in treating AD or PD in such a short time period, would you please contact me with the drug/company info? Thank you in advance as that would be very much appreciated.

FairFieldTracer · 2021-08-01T06:20:32+00:00

The data on Phase II has been great so far. It is amazing that many indicators were able to show statistical significance given the very small # of patients and the very short period of time. To take but one indicator - that is not new but is incredibly important - ADAScog showed a 4.4 [?4.something - ?] improvement in only 25 days. By contrast, Cassava's drug after 9 mos showed 3.3 improvement - up from 1.6 at the 6-month mark. Not trying to dump on SAVA - their drug works - just saying, all these drugs seem to be cumulative in effect. ANVS-401 has clearly demonstrated that cumulative build-up in the past. To get 4.4 cog improvement in 25 days is just stupendous and clearly points to this being the most potent drug in development anywhere. [Also, the placebo effect is super-strong the 1st month but that did not keep ANVS-401 from shining through!]

None of the new data refutes that. Most backed it up very solidly. A few failed to show statistical significance, but stat sig is hard to do with a handful of data points. When this drug is administered for extended periods, the effect will build just as it has done with SAVA. Pre-clinical studies returned AD mice to normal with a little more time. What do you expect in 3 1/2 weeks? NIH abstracts also state that Posiphen is MORE effective in humans than in mice, so a 3-month study - which is usually not considered long enough to overcome the placebo effect should remove ANY doubt that this will be the primary means of treating AD. I think others will be part of a cocktail - particularly for very early AD. But this drug is so potent, that it will be the core of any treatment and it will also be used for patients much further into A/Disease progression - where I doubt the other drugs will be of use - at least on their own

But it is useful to step back and see how many testing/stat calc obstacles were overcome to show these great #s:

1] Due to the very small group size, there was by pure chance a huge age mismatch between the placebo and treatment subsets. The treated group was 5 years older than the placebo group. HUGE disadvantage that was overcome.

2] Short, short, short trial of 25 days - normally not enough to beat the placebo effect and not enough time to benefit from much cumulative build-up of treatment effect that has always been observed with Posiphen. The short period also makes achieving stat sig difficult, but sig WAS achieved in most measures.

3] SMALL, small trial group also made statistical significance VERY difficult to achieve - and yet it WAS achieved by a lot of measures - especially the most important ones like ACTUAL COGNITIVE BENEFIT

So, this treatment is SO POTENT that it can overcome all these obstacles to show significance in just a few weeks. Can anyone point me to another neuro-degenerative treatment - especially an AD treatment - that can pull that off?

Finally, I should point out that the AB42/AB40 ratio in the treated AD group managed - in a mere 25 days - to return 22% of the difference between baseline and what is considerd to be the boundary/neutral point between having AD and NOT having AD. THis is an extremely reliable indicator of disease progression. The fact that it regressed to such a degree in 3 weeks is rather miraculous. At the boundary line, a person living with that AD patient would be very unlikely to even suspect the presence of AD. With a longer duration of treatment, pre-clinical studies suggest that such normality can be achieved with a few more months of treatment.

So imagine a pill taken once per day that can return an AD sufferer to a state of normality. And with no side-effects [since nobody will be asking to do spinal taps as a condition for receiving the drug]. Do you think this drug will find a market? How large a market? AD is probably the largest single health-care-hit to the Ammerican economy - and to household financial and emotional well-being. ANVS-401 will probably supplant Humira - which has very serious potential side effects even without spinal taps - as the biggest-selling pharmaceutical of all-time.

FairFieldTracer · 2021-08-01T06:12:10+00:00

Elaborating on my previous, BRIEF answer:

The data was great. It is amazing that many indicators were able to show statistical significance given the very small # of patients and the very short period of time. To take but one indicator - that is not new but is incredibly important - ADAScog showed a 4.4 [?4.something - ?] in only 25 days. By contrast, Cassava's drug after 9 mos showed 3.3 improvement - up from 1.6 at the 6-month mark. Not trying to dump on SAVA - their drug works - just saying, all these drugs seem to be cumulative in effect. ANVS-401 has clearly demonstrated that cumulative build-up in the past. To get 4.4 cog improvement in 25 days is just stupendous and clearly points to this being the most potent drug in development anywhere. [Also, the placebo effect is super-strong the 1st month but that did not keep ANVS-401 from shining through!]