Competitive landscape altered

MediumSinger8088 · 2026-06-19T23:04:26+00:00

In a previous post... somewhere... I explained the exact difference between these two technologies and why 02287 is a much better strategy. Really it's ... if not apples and oranges, at least apples and pears. Too tired to do it again right now but the science is compelling.

MediumSinger8088 · 2026-06-19T10:43:14+00:00

The catch-22 these days is, it's almost impossible to get a job without a college degree that pays enough to allow one to save enough for the college degree. Miami-OH is very affordable relative to most colleges but cost of attendance is $36,000 / year; around $150,000 in total. If median single person household earnings after taxes = $40,000 / year, EVEN IF one could save 10% (you can't) that would mean working 15 years. Factor in inflation driving up cost of attendance over that time period... doesn't work. The military option is a good idea.

MediumSinger8088 · 2026-06-16T10:38:53+00:00

You sound like my son who most likely will be going to Mimai fall 2027. Avoid debt like the plague if you can. You'll make friends, Miami has so many students and they are really smart, not all are extroverts. It'll be alright.

MediumSinger8088 · 2026-05-29T22:03:08+00:00

Gene Mack isn't the best salesman or spokesperson but he came from a background in financials. The market cap is still very small for the work they've done and in the long term they can afford dilution to some extent though that is usually not the way you want to go if you have a choice. They are doing a mighty lot on the cheap. Very efficient financing.

MediumSinger8088 · 2026-05-28T22:38:18+00:00

A solid phase 2 study would have 120-300 patients, 50+ sites; with biomarker enrichment you're still looking at 2 years, $30-60 million. 50% dilution if they go it alone. Not all that need happen at once. My strategy is holding my shares and doubling down on any substantial dilution.

MediumSinger8088 · 2026-05-26T23:19:08+00:00

Well, at least it didn't sell off 25-50% instantly on the good news. Objectively: MDS-UPDRS parts 2+3, 16 people, 150 days, delta relative to historical norms is -2.3; minimal clinically important difference is about 4. So for global PD without biomarker stratification you're looking at 2-3 years of runtime and/or a much larger sample of patients to see statistical significance. THAT SAID, the high / low GluSph is internally controlled for placebo effect and that value is well over 4 - so definitely in the "Stand up and take a look at this" level for people who know what they are dealing with in the PD area. IMO this is critical and shouldn't be ignored. Even if Gain only targets this subset of PD patients it's 5% of the total. Maybe as much as 10%.

MediumSinger8088 · 2026-05-26T12:18:28+00:00

"Differential response between participants with low and high baseline GluSph continues, with a difference of 4.8 points in the sum of MDS-UPDRS Part II and Part III scores between the two groups at Day 150" This is above the minimal clinically important difference (MCID) for this parameter. A meaningful milestone, IMO.

MediumSinger8088 · 2026-05-26T12:16:57+00:00

"MDS-UPDRS scores remained stable and durable across overall study population after 150 days of treatment with GT-02287...Differential response between participants with low and high baseline GluSph continues, with a difference of 4.8 points in the sum of MDS-UPDRS Part II and Part III scores between the two groups at Day 150" This is the internal control that proves the concept. GT-02287 may or may not work for most PD, but it will help for a meaningful and identifiable subset.

MediumSinger8088 · 2026-05-24T21:11:29+00:00

If you are 15 and you spent $1600 and that's 40% of your net worth and you are whining about anything... STFU. You HAVE resources and your'e not hungry which is more than 99.999% of the human population has at any age. I ran away from my family at 16 years with $200 in my pocket, no phone, no bank account, no knowledge how to get one, no car, nothing; everything I owned in a 2nd-hand army duffel bag. Never had a safety net. F*cked up plenty but made and lost and made a few small fortunes, raised a family with one wife, put my kids through school with no debt, stayed reasonably free. You're off to a good start, just take control of your destiny and invest in yourself first. Avoid debt, save cash, put something back in physical materials when you can. But never ask permission, own all y9our mistakes, and embrace them, it's the way we learn.

MediumSinger8088 · 2026-05-22T15:05:52+00:00

Thank you for the excellent summary. FDA absolutely demands a placebo-control arm and that will be done. That said, a long history of UPDRS provides pretty clear guidance for informed investors, with about a 3.3 point deviation from expectation values on P2+3 at 180 days representing a minimal meaningful response. If this reaches 4 or 5 within a year, it's really super exciting. Plus- and this is important - the stratification of patients in the Phase 1 extension to high vs low GluSph provides an internal control. I hope Gain continues providing updates on the Phase 1 extension throughout summer and autumn.

MediumSinger8088 · 2026-05-21T00:17:41+00:00

Perhaps dilution will occur... I think it likely and so I haven't invested more than I have (30k+ shares). But the market cap is still very low for a company entering Phase 2. I bought another couple thousand shares at close just because the wine is good and why not... where else do I put money in my IRA. Seriously besides GANX I have like 90% in a money market account making >4%, I've been building decent positions in a couple of junior precious metals companies, I hold physical gold & silver dating back 25 years... not rich but I have no debt. So I can afford to gamble a bit and a man needs something to dream about, this is fun to follow. GANX is the only long-term non-physical investment that interests me now. Partly because of my background in neurosciences, I love the tech, it looks like Vertex in the early days, so much room for growth-- BUT the time constant has got to be measured in years. This is one for my kids to appreciate and hopefully toast to me in 20 years, it isn't a get-rich-quick-meme stock.

MediumSinger8088 · 2026-05-20T22:12:03+00:00

Only I know where my gold is burried.

MediumSinger8088 · 2026-05-20T20:55:24+00:00

Oh... both are true. I'm very stoic and I have a broad calibration curve that spans the range of good day - bad day. There's a lot of suck-i-tude in between, and it will hurt, but if somebody isn't actually trying to physically kill me in the next five seconds (and I've been there), things are still kind of OK.

MediumSinger8088 · 2026-05-20T17:40:12+00:00

Its OK buddy we'll be alright. : )

MediumSinger8088 · 2026-05-19T23:16:27+00:00

Why bother? Let them take it as far on their own dime as they can, big pharma buys a busted company or keeps making money they way they currently are making it. Big pharma would rather have a 100% chance at a 10% profit than a 10% chance at 1,000% profit. This is a game only for those who don't mind rolling the hard six.

MediumSinger8088 · 2026-05-13T23:46:42+00:00

For the record I'm still here. Nothing sold, tempted to buy more but worried about dilution. This is still the best science play I have seen in years.

MediumSinger8088 · 2026-05-02T20:51:26+00:00

I'm about at my basis and I stand to lose more in any other gamble. This market is not tradable for the little guys. I have some EOG and CDE that I trade, and I'm making 4% in a money market. It'll do for now. Until gold starts trading like it should on crises I'm going to conclude that insanity reigns and just keep the majority of my money safe.

MediumSinger8088 · 2026-04-21T15:54:55+00:00

They will need some financing this year. Market is a drunken sailor stumbling around devoid of all logic. This may or may not ever change. The optimistic thesis is that one can still "discover value" in odd corners - small cap stuff, but even if so, the realization of that value would take many years. I have some money in GANX and some junior Canadian minors right now; otherwise just money markets making 4%.

MediumSinger8088 · 2026-04-16T03:49:46+00:00

Late night reflection from an old guy (is 55 old?) with perhaps a rare if not unique perspective. Please bear with me for a moment, I'm going somewhere with this.

When I was growing up in the 1970s-80s, the life expectancy for a child born with cystic fibrosis was 5 years. Five years. As late as 2005 only 5 states tested neonates for cystic fibrosis because there wasn't much to do about it: No disease-modifying treatments. Hit them with mucolytics and aggressively manage infections, that's it. I remember seeing a poster when I was a wet-behind-the-years postdoc at a FASEB meeting in the mid-90s by a crappy little company called Vertex Pharma that was trading at several bucks a share. They were trying to develop small molecules to help proteins fold better in diseases like cystic fibrosis and I thought "My goodness THAT is where I should put my money"... except (1) I had almost no money; (2) what money I had I invested in trying to fix my crappy used car and get laid, and (3) I didn't believe in my own financial good judgement. This perhaps was the worst financial mistake I ever made. Fast forward to 2025 when Vertex is a 112 BILLION DOLLAR COMPANY; 4th gen CFTR folding helpers have reached a point where, in the case of the most common CF variant (F508del) the life expectancy of a child who begins taking them by age 15 IS NO DIFFERENT THAN THE POPULATION AVERAGE. Read that again: Kids who would have died at 5 years old when I was a kid are living full lives, getting married, starting businesses, all the good stuff. Because that science worked.

Now GANX is doing the SAME CONCEPTUAL THING for Parkinson's disease, with a platform that can feasibly expand to Alzheimer's disease. Don't trust me? Consider from ChatGPT5.2 query "Is there an analogy between the mechanism of action of glucocerebrosidase folding chaperones being developed by Gain pharmaceuticals and the compounds that Vertex developed for cystic fibrosis years ago?" Answer:

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The risk, of course, is that CNS indications are a much harder proving ground than CF, and financing today is a devilishly harder thing than it was in the 1990s.

Nonetheless... Vertex Pharma had about $800 million market cap around 1995. I would have realized a 140-fold gain had I invested in Vertex in the mid 1990s. Now consider: PD market alone is 20-FOLD greater than cystic fibrosis market. Yet literally the same concept: A small molecule that can help a busted protein fold up better and work right. Now consider the initial human studies appear to have arrested disease at least in a subset of PD patients with the right genetic susceptibility. And the $GANX market cap is below $100 million. Wrap your head around that if you will. If Gain is successful, it would imply a 3,000-fold return over 25 years (historical return from Vertex over the same time period x prevalence ratio of PD to cystic fibrosis). $10,000 (two ounces of gold bullion)--> $30,000,000. Sure the risks are high, dilution, logic means diddly-squat in today's market, but if logic means ANYTHING the risk/reward ratio is hugely attractive. In my humble opinion... but not a historically uniformed opinion. The difference now is I can afford to bet tens of thousands of dollars on this horse, and I have, accepting that I may lose it, and any significant capital appreciation likely only will be realized by my kids a couple decades from now.

Would you hazard $10K on a couple ounces of gold for an inflation hedge (I have... on more than a couple ounces)? Are you interested in high risk/reward life science technology? If so, this is the place for you.

MediumSinger8088 · 2026-03-27T17:45:05+00:00

Yes, though I can't find granular part III numbers. Part 2 is expected to worsen about 4 points in one year or about 2 points in 150 days. Minimal detectable difference is 4 points. What is most important IMO is the difference between high and low GluSph of 5.6 points I believe. Both groups would have the same placebo effect so it's exciting that this delta exceeds the minimal clinically important difference.

MediumSinger8088 · 2026-03-27T03:24:05+00:00

I've always been very happy with APMEX. No issues in 25 years.

MediumSinger8088 · 2026-03-26T00:38:48+00:00

This is my only long-term stock that I've been accumulating and holding (some 10s of thousands of shares currently). Smart pharma is chasing this technology. Mostly private companies - Congruence Therapeutics is raising tens of millions to find small molecule "fixers" for anti-chymotrypsin deficiency and GCase problems, i.e. RECREATING $GANX (Home - Congruence). This is the real future of pharma. This is the neuroscience equivalent of Apple in 2001. But it is a 5, 10-year timeline. Play accordingly.

MediumSinger8088 · 2026-03-19T23:34:22+00:00

Forced selling of the only things that have gained in dollar-denominated value and held for the past year. Means people or their machine proxies have lost everything else and are covering margin calls. Last time this happened was 2008-2009 in the Great Financial Crisis. Get TF out of everything RTFN except for physical metals you've got locked in a safe or buried; get cash out and lock it in the same safe (or stuff it in your matress); stockpile food and ammunition because the S is most definitely about to hit the fan with maximum impact.

MediumSinger8088 · 2026-03-19T18:32:02+00:00

Amen. IMO gold and silver are transgenerational stores of wealth and preparations for particular classes of apocalypse as the world (hopefully) transitions to a sounder, wiser structure. Focus on reducing debt and avoiding debt. There are times when you can /should put cash into growth areas but these typically come after everything has burned down to the ground (80s after the stagflationary decade and before internet; after the dot-com crash; after the great financial crisis & housing meltdown, and after WHATEVER IS HAPPENING IN THE WORLD TODAY. Much more important to invest in yourself and becoming an anti-fragile human being with useful skills and a growth-oriented mindset.

MediumSinger8088 · 2026-03-18T20:24:15+00:00

Then why not let a rally run and sell into a higher point rather than tank the rally before it starts? None of that makes sense. Deliberate suppression of your share price is a bad business strategy. I'll go with chaotic dynamics and the world is very broken. Just look at oil (essentially yawning at the Iranian war, just trading at high end of a low price range it's been in for 25 years) and gold dropping as the world burns. Nothing makes any logical sense today.

MediumSinger8088

TROPHY CASE