GANX Gain Therapeutics Inc

$GANX Khalid stepped out from behind the curtain yesterday. I said he would only do this if and when he knew he has it. This is a man who speaks with intent and purpose. Nobody has more integrity and his reputation is everything to him, next to helping the people who benefit from the drugs he has been involved with. This is a man whose pride possessions are the letters he has received over the years from both patients and their family members thanking him. Nobody knows rexacerat better and his post yesterday likely goes down as a timely tell. $XBI

$GANX This reminds me so much of PRVB, where all the pieces of the puzzle were publicly available but the market just didn’t bother putting two and two together. The company had sold off a single-digit percentage of royalty rights, and those were re-sold for about $200 mil. This imputed a value of the company itself of over $2 bil. Did we get repriced to $2 bil? Hell no. We also had Sanofi pay for right of first negotiation - so obvious BO interest. So PRVB was valued at like $600 mil right before they got bought out by their partner for $2.9 billion. Pretty obvious on both the buyer and price, but that’s just how stupid the market can be. There are fewer known specifics here but it’s clear the drug is safe, the data is consistently amazing in halting PD, and there is partner/BO involvement in major research decisions already. I feel pretty confident about what’s coming next.

$GANX If you are a Parkinson's patient, or your loved one is, realize that there is currently no other drug out there in development, that does what rexacerat does for them, nothing. Look high and low, it doesn't exist. The stock price while disappointing and absurd, is only further solidifying my opinion that the company likely has a new name on the door in the near future. Especially with thisnweeks conference, others and Jefferies coming up by year end. As I said before, shareholders are likely to play a key role with their votes determining if an offer is approved. The offer is likely to come with CVR's which we will have to factor into the equation. Some perspective, Vanqua is said to have a valuation of nearly 10X ours. They have a single drug that hails in comparison to rexacerat, let alone the fact we have another even possibly better drug right behind it that could be in the clinic in less than 2 years itself with rexacerat possibly being approved with a phase 2 being pivotal. In addition, they also have a handful of other compounds already likely laying in wait maling it a complete platform play with a large pipeline. This is literally the perfect opportunity for almost any major pharma due to the clear price disparity and pipeline expansion opportinity, the patent cliff approaching and #Neurodegeneration being completely underserved with huge unmet needs. It is inevitable in my mind...just like the recent Ferrari market which has exploded in the last year. What was an equitable and stable market can instantly change when scarcity value enters the equation. There is nothing like rexacerat, it is in a class by itself as the sole drug to ever show sustained disease modification in PD. Don't be surprised when you are asked to vote in the future. We have it and many will soon want it. I won't be surprised to soon see the shares better reflect this growing reality. Fine line between fear and greed and the pendulum tends to overswing...

$GANX Their full abstract for the international congress event says they will be presenting complete biomarker data, which they list as sphingolipids, neurofilament, inflammatory markers and alpha synuclein. I wasn't expecting any of this but maybe.

$GANX Looks like Roche has been following our results closely and is sold on GCase biology. Today they paid $100M upfront + $1.1B milestones for a pre-clinical asset also targeting GCase biology. https://www.reuters.com/legal/litigation/alector-soars-roche-licenses-parkinsons-therapy-up-127-billion-2026-10-05/ Not even tested in humans yet and won’t be orally administered. It’s an engineered GCase enzyme so it may have a potency advantage in terms of restoring GCase. Personally think their money would’ve been much better spent on buying Gain which is already derisked and ph2-ready... but I think overall it’s a positive signal for our prospects and value.

$GANX Never more...know what you own. It is about to finally get real imo. This is a billion dollar takeover target, with pharma already at the table and that price would be literally giving it away. rexaceract likely may end ends up in the company of insulin, aspirin, penicillin, the smallpox vaccine and morphine. Much like I said early on both $PRVB and $ABCL just recently, now up almost 3X and going higher. $COYA and Gain complete the trifecta.

$GANX I believe we can all agree, the only weakness of the company is the communication, at this point. We were under the impression the FULL dataset--(i.e--biomarker data), would be released at the Korea conf. & we still haven't been privy to. That doesn't concern me, if that wasn't expected. Just tell your shareholders that you're still validating the #'s or waiting for the last participant to finish the study. Simple. Say "we expect to release said data at the MJFF conference or another conference upcoming". Concrete information--i.e--release dates/information content with better communication, would go a long way to placate current shareholders/potential investors doubts. Otherwise, I'm not pessimistic at all. Patience pays here. Best to the longs....

$GANX Three M&A Fundamentals in Place The Patent Cliff: Over $300 billion in revenue (about 40% of total industry sales) is exposed to patent expirations over a five-year window, forcing companies to aggressively buy external pipelines. Spotty Internal Pipelines: Internal large pharma pipelines aren’t nearly strong enough to provide the growth drivers pharma needs to overcome upcoming patent cliffs. Over a Trillion Dollars of Buying Power: The pharma industry has approximately 1.2 trillion dollars in M&A buying power. Of course, only a fraction of this is going to get used but there is substantial capacity to buy the biotech market. This is not a theoretical topic. Our industry conversations aren’t telling us that M&A might pick up in the coming months. Those conversations tell us that M&A will pick up. And, with that, the biotech FOMO trade is likely to be right back.

$GANX Not sure how this Novartis deal got by me. Looks like 575m upfront for a phase 1 asset.

$GANX Great find. I’m way behind, as I’ve been traveling with limited ability to follow developments. of these particular biomarkers is very encouraging. In addition to durable UPDRS scores, if they are also able to show supporting biomarker data, especially evidence of flattening NfL, this would be ground-breaking. “Recent discussion with potential partners” as having influenced their decision to submit a new phase 2 protocol is the biggest sign yet that they are close to a deal. I’ve said this before over the years, but there has never been a better time to invest than now while the market is apparently sleeping on this new information, not to mention all of the data compliled over the past couple of years.

$GANX I feel like we are all missing something, or not being told something by mgmt. How can a drug with such great data, this far advanced, in such a huge potential mkt perform so badly!!! And how can such an experienced mgmt team be so bad at communicating the message conference after conference and generate no interest??! The the the frick is going on?!?!?’

$GANX Saw the comment about more data possible at next weeks conference. I went back and looked at the secondary biomarker endpoints. A couple of those would be real gamechangers. Would be nice if they held back more info that we see next week. Secondary and exploratory endpoints included pharmacokinetics, clinical scales (MDS-UPDRS, etc.), and plasma and cerebrospinal fluid (CSF) biomarkers (sphingolipids, neurofilament, inflammatory markers, LAMP-2, and α-synuclein) The LAMP-2 gene and its specific isoform LAMP-2A (lysosome-associated membrane protein 2A) play a critical role in Chaperone-Mediated Autophagy (CMA), a selective cellular clearance process that degrades toxic proteins like alpha-synuclein. [1, 2, 3] Key Connections to Parkinson’s Disease Reduced Levels in Parkinson's: Studies show that LAMP-2A protein and gene expression levels are significantly decreased in the substantia nigra and peripheral blood cells of patients with Parkinson's disease

$GANX At least 1 presentation at Neuroscience 2026 by Joanne Taylor on preclinical backup compound.

$GANX This is a great market analysis on Gain done by Ben Pieter back in May, longish read but a lot of interesting information.

$GANX an under powered 100 patients proof of concept type P2 would be a mistake that any potential partner would not sign off on. For me the P2 amendment is the most serious sign that they are in negotiations to run a P2/3 trial with a large partner. They can't fund that or do that alone so we just eliminated any other possibility

$GANX looks like they added smell tests for the preclinical mice tests after getting our human results.

$GANX A few other insights from the data: - 100% of patients on rexaceract remained stable, resisting the typical motor deterioration seen after 12 months. - These data strongly signal disease modification. As expected, dopaminergic neurons can be rescued, but not resurrected. - High and low-baseline GluSph patients showed near-identical stabilization. This will likely expand the TAM and target Phase 2 patient population. - The safety profile was exceptionally clean for a CNS drug. Other neurodegenerative treatments can cause brain bleeding or dyskinesia.

$GANX Its even clearer after todays release. As of August 31, 2026, 12 of the 13 evaluable patients have completed the full 12-month administration of rexaceract in the open-label extension, with one additional participant scheduled to complete dosing in November 2026. In the 12 evaluable participants, MDS-UPDRS motor scores were stable over the 12-month study period with rexaceract, demonstrating no clinically meaningful progression of motor symptoms on the MDS-UPDRS Parts IIand III.A clinically meaningfulchange in PD disease progression is generally accepted to be an increase in MDS-UPDRS Part II and III together of approximately 6 points over a year, which also is the mean annual rate of disease progression among individuals with early PD. The Company expects to submit a new Phase 2 study protocol to the U.S. Food and Drug Administration (the "FDA”) in the coming weeks, informed by insights provided by the Phase 1b study results and recent discussion with potential partners

$GANX Next catalysts: 1) Gain Therapeutics is presenting its poster at the International Congress of Parkinson's Disease and Movement Disorders (MDS Congress) on October 7, 2026. Presentation Details • Date: October 7, 2026 Perhaps we will get the Biomarker data and additional details that everyone is looking for (i.e…..improvement in smell, gate, balance, gate, etc). 2)FDA approved protocol amendment - looking forward to these details. Registration study would be a huge win for shareholders. 3) Any funding and/or partnership announcement related to who is running the phase 2 and/or phase 2/3 trial.

$GANX October is going to be a long month. Never thought Gene would have us staring down the barrel of a 52 week low after showing no progression at 360 days.