GT

Correct_Proposal_409

Thanks for this. From what understand, Landsbury is very excited about GT-02287 and these results.

MediumSinger8088

Deep dive- Peter Landsbury, the KOL advising Gain, was the CSO of Lysosomal Therapeutics around 2018. That company had a compound LTI-291 that allosterically activates GCase, theoretically similar (but different from) GT-02287 which chaperones folding of the enzyme. Lysosomal and LTI-291 got acquired by BIAL, which is sponsoring a phase 2 trial of the drug now called BIA 28-6156 (pariceract) called the ACTIVATE study in PD. Last patient due to visit clinic in April 2026 and topline data expected Q2. Published data that LTI-291 (28 days, 40 patients) did not change GluCer in CSF or plasma but paradoxically increased the analyte in peripheral monocytes. No effect on MDS-UPDRS in this study. Take aways: IMO BIA 28-6156 is the closest competitor to GT-02287 but there are key differences and results from ACTIVATE may affect Gain price in ways that are illogical due to Mr. Market confusing the MOAs. Also GT-02287 seems to have done something unique in decreasing CSF GluSph. Note there is no data arguing glycosylsphingosine is a biomarker for PD progression but is a marker of target engagement which WILL bring some big pharma players into a conversation at least. There is some evidence that PD patients with elevated GluCer / SM are more prone to cognitive decline over 3 years, which indirectly speaks to cholinergic pathways involved in AD (there's that AD connection). It will take a long time to see this all play out. Years. Invest accordingly. And circling back: Listen to what Landsbury says in the KOL talk. He's probably the most informed guy in the world about this topic.

Correct_Proposal_409

Very unfortunate market reaction to positive news which goes a long ways towards confirming that the drug is working. The PR was to conservative, and they are holding back additional data (possibly due to CDA with a pharma partner, or the are holding data for the KOL event and the AD/PD conference, not sure). Regarding the KOL event, it sounds like they will be reviewing additional data: “The event will also review biomarker results from the Phase 1b clinical study of GT-02287, demonstrating disease modifying potential in Parkinson’s disease patients with or without a GBA1 mutation.” I just wish they had put that as one of the top bullet points for the PR. I’m holding for buyout/partnership, which I think is more likely now. BTW, the BTIG analyst just came out with this note, and he agrees: “Readout Suggests GT-02287 is Activating GCase in the CNS. Additional Steps to Confirm it a PD Drug, but the Gain Approach Works. WHAT YOU SHOULD KNOW: We see the findings today serving as definitive evidence supporting GT-02287's ability to activate GCase in the brain. As a result, we see validation for the Gain approach and increased likelihood others will agree and look to partner other novel applications where allosteric modulation of enzyme deficiencies might benefit patients. The readout today measured the levels of GCase substrate, glucosylsphingosine (GluSph) in the CSF. The observed GluSph reductions in every patient that had elevated levels of this GCase substrate at trial start make it clear: GT-02287 increases the activity of its target enzyme. After 90 days of GT-02287 treatment, all individuals with elevated CSF GluSph experienced significant decreases.”

Accomplished_Bath940

 Man made 50% profit when the market falling apart

OutlandishnessUsed24


Correct_Proposal_409

Okay. fair comment, since elevated liver enzymes are common with early oral small-molecule trials. But there was only one case of transient elevated liver enzymes in the 1b trial, and they withheld dosing, then reduced the dose and the enzymes normalized and stayed in normal range. And the only other case (of 77 people) was in the 1a trial and it was a mild increase: "One subject had a transient, mild increase in AST and ALT after receiving a single dose of GT-02287". No SAE's.

benjamin_noah

Sure. As I also said before, I am concerned that the trials have shown that GT-02287 causes elevated liver enzymes (ALT, AST, and alkaline phosphatase). Which, for what would be a long-term medication, could be problematic. But I believe there's also some evidence those levels could normalize over time. So, that feels like a normal biotech risk. I don't like that I've never seen either of you address that in your posts.

Ye_Olde_Basilisk

We bought a very expensive completely custom 2025 Mustang GT Premium last year. When I was talking to the sales guy, he said they had dozens of Lightnings they couldn’t sell. They can’t even get anyone to look at them. I was like, shit, my truck is 11 years old. Let’s see what kind of deal we can make. The dude said there is no wiggle room or negotiation on those prices. 

magicjohnson89


Correct_Proposal_409

Absolutely. If they prove GT-02287 in idiopathic Parkinson's cases, and early data says that this is likely, it opens up Alzheimer's and other neurodegenerative diseases. At the very least, I think this drug will work for GBA1 cases, so that's my base case, and that's multiples higher than current share price level.

strudel_investing

https://youtu.be/Rv7GT9HsJZc?si=SS5T-tDnRVYEVLSL

stupidber

Why can't I bring a GT Racer to the ski slopes?

Correct_Proposal_409

You are right about biotechs. But in my post, I addressed why this has been derisked and why I believe this is not a binary event. I also gave recent examples of where stocks have skyrocketed off of data, often on data that is much less compelling that what we already know about GT-02287. And the examples of these huge upside moves are endless.

Correct_Proposal_409

Anyone interested in looking a little closer into the science, here's a post that touches on GT-02287 and importance of mitochondria in Parkinson's: [https://www.reddit.com/r/pennystocks/comments/1pex0jz/gt02287\_addresses\_multiple\_potential\_sources\_of/?utm\_source=share&utm\_medium=web3x&utm\_name=web3xcss&utm\_term=1&utm\_content=share\_button](https://www.reddit.com/r/pennystocks/comments/1pex0jz/gt02287_addresses_multiple_potential_sources_of/?utm_source=share&utm_medium=web3x&utm_name=web3xcss&utm_term=1&utm_content=share_button)

ColdComplaint8

thanks - i'm acutely watching the MMM's predictions for $GT lately i'm honestly hoping for $9.25-$9.50 before they expire and I'll be cashing out a decent chunk of change

Beginning-Tap-95

GT too the moon let’s go! 

PhysicsViking0901

Interesting, I am actually a medical physicist working in radiation oncology. We have implanted several patients with what looks to be a competitor: GammaTile from GT medical. I'll have to look more into this.

_hiddenscout

Interesting write up.  However, you’re missing actual research into the company itself. I can look at their numbers in quickfs and tell it’s going to be a pretty bad investment in like 5 minutes.  https://quickfs.net/company/GT:US Just look at the QoQ numbers, it’s not a great story. Like over the past year, they are seeing negative revenue growth.  On top of they have terrible ROIC.  To each their own, just can’t picture this a good investment other than hoping for a buyout.  Elliot even tried to add activits to the board like two years ago and it’s still not working.  https://www.reuters.com/business/autos-transportation/goodyear-tire-add-three-directors-settle-with-elliott-2023-07-25/

Swearwolfofwallst

[How can Standard and Poors help you?](https://youtu.be/mwdo17GT6sg?t=154)

Correct_Proposal_409

The main point of the post is that it seems that there are two or more conditions that need to be present for Parkinson's disease, or at least particular cases of Parkinson's (and other neurodegenerative diseases), but that inflammation is a constant. I'm not claiming it is the cause in all cases or even most cases. Gene mutations for example could create a susceptibility for which systemic inflammation is the trigger. In others, it could be something else. The point is that we don't know, but neuroinflammation is a constant in all cases of Parkinson's. The second point of the post is to point out that GT-02287 has the key bases covered. It doesn't target neuroinflammation per se, since its primary role is to increase Gcase activity and effectiveness by stabilizing its intermediate folding state and restoring trafficking to the lysosome and mitochondria, which in turn addresses the dysfunction driving neuroinflammation.

microcapreturns

Yes the misfolding that Gains GT-02287 corrects is likely the start of the chain and makes it obvious why it works. Thanks for pointing that out.

microcapreturns

There was an article written by a short and they had not even looked at the data. Lol. Parkinson’s studies in the last 30 years that showed placebo affect showed it improving right after start but then heading back up very quickly. We are the opposite. GT-02287 is modifying the trajectory of Parkinson’s. Amazing. https://preview.redd.it/nbpgwlj6ys5g1.jpeg?width=1600&format=pjpg&auto=webp&s=abae53607209c13ec7af40d76e89932806884812

microcapreturns

This comment by the analyst on the three unique models that all show Gains GT-02287 works in Parkinson's is worth reading. Very important. Gain Therapeutics(GANX): H.C. Wainwright $8 PT 12/1/25 Three Month Data Imminent https://gaintherapeutics.wordpress.com/2025/12/01/gain-therapeuticsganx-h-c-wainwright-8-pt-12-1-25-three-month-data-imminent/ 

microcapreturns

Placebo affect goes out the door when patients show improvement after the first 30 days. If placebo affect was going to happen it happens right away. The data shows GT-02287 is working. That will be huge news for the world in the next few weeks. A $200m market cap and it should be $5b if it works.Huge change to their slide wording right before data release. https://preview.redd.it/smx8ovwyrs5g1.png?width=1052&format=png&auto=webp&s=8e49875b4fb1c018c6aad5a61dec3de9c0b37f00

Outrageous_West_1564

Yeah I know this interview. I heard it multiple times and my rigidy and fatigue got better the more I was reading about GT-02287. I feel better overall. Would I say my smell got better if someone got me a fructose pill at exactly the same time and told me "this maybe can cure Parkinsonism"? Probably.  And exactly that's the point for my initial Post. I know it first hand what hope and a good mood makes to people with Parkinson. An normal investor doesn't. You "just" see the data and compare it to other trials for other diseases and that's a big mistake when it comes to Parkinson trials. 

microcapreturns

I'm sure you listened to this CEO interview. He had just returned from visiting patients and clinicians in Australia. He is by far the most conservative CEO I have ever talked to. Its not just smell and if it wasn't a majority of the patients he would never had said it. I think it will be nightly news at some point. There is a reason Ken Marek is involved as a KOL and also running the M.J. Fox smell study. Leveraging AI: The Biotech Cracking the Code on Rare Diseases https://youtu.be/CJcH-n6iKVU?si=BWkW1cEYdv71FN9X CEO Gene Mack: This is a buy at $10, $15 or $20. Multibillion revenue opportunity per year. Alzheimers,  Gaucher and other neurological diseases also possible. Patients saw functional improvement in motor functions, tremors, balance and regained smell after only 90 days on GT-02287. Patients and clinicians requested extension of trial so they wouldnt lose the improvements they have seen.

microcapreturns

Can you link a placebo controlled trial that showed the placebo group getting worse for 30 days and then improve rapidly for the next 60? I have never seen that. The ones I saw had the patients improve from the start. Appreciate the discussion because it's hard to see every study. Did you find any studies where either the placebo control or active arm had people get smell back? From how the company explained why that happens on GT-02287 I can't see how that would happen on placebo?

optimalpooper

I don’t disagree that there should still be caution, cknowledging your point: everything important is still unproven • Many drugs in PD had great preclinical data and target engagement in humans (including venglustat, ambroxol, prasinezumab) and still failed to move clinical endpoints. • GT-02287 is only in Phase 1b; there is no randomized Phase 2/3 efficacy data yet. The field has a high failure rate, and mechanistic plausibility does not guarantee clinical success. Myself like many others is really bought into the science and not acknowledging some of the stark differences between what Gain is doing with their “mitochondria first” approach and the other mentioned is underrepresented in your analysis. No one in the current/previous Parkinson’s pipeline has really done exactly what Gain is doing (physics-heavy 3D platform tightly integrated with AI for discovery). I always have planned outs and have also been holding for quite some time. Anyone interested in the breakdown comparison can reference below. (Used AI to help simplify the summary): What’s potentially better about GT-02287’s science 1. Mechanism sits at a biologic “hub.” GCase deficiency links lipid metabolism, lysosomal stress, and α-syn accumulation. Fixing that one node could, in theory, influence multiple pathogenic processes at once. 2. Precision small-molecule design. GT-02287 isn’t a repurposed drug; it’s optimized for brain penetration, specific allosteric binding, and lysosomal function, based on 3D structural analysis of the protein (SEE-Tx®). 3. Broad applicability beyond GBA1-mutant PD. Because GCase activity is also reduced in idiopathic PD, Gain is deliberately studying with and without GBA1 mutations, which could widen the addressable patient pool if efficacy is shown. 4. Strong biomarker plan. The Phase 1b study is not just “does the drug look safe.” They’re explicitly measuring GCase modulation and substrate changes in CSF/plasma, which should give a clean read on whether the biology works in humans, even before larger efficacy trials. How GT-02287 is different from Venglustat: • Fixing the enzyme vs. turning down the faucet. • Venglustat: Tries to help by reducing substrate load but doesn’t correct misfolded GCase or restore its normal lysosomal function. • GT-02287: Directly stabilizes GCase and improves its trafficking and activity in lysosomes, aiming to normalize the enzyme itself. How GT-02287 is different from Ambroxol: • Rational design vs. repurposed drug. • Ambroxol was never designed for the brain or for PD; it has other pharmacology (e.g., effects on ion channels) and needs high doses to get robust CNS effects.  • GT-02287 was discovered with Gain’s SEE-Tx® computational platform to bind a specific allosteric pocket on GCase and is optimized for CNS penetration and GCase modulation.  • Allosteric STAR vs. mixed-mechanism chaperone. Ambroxol is essentially a “happy accident” GCase chaperone; GT-02287 is a Structurally Targeted Allosteric Regulator with a clearly mapped pocket and structure-activity relationship. • Program built around biomarkers from day 1. GT-02287’s Phase 1b trial is explicitly built around CSF and plasma biomarkers (GCase activity, lipid substrates, PD-adjacent markers) as key secondary endpoints, on top of safety.  How GT-02287 is different Prasinezumab (Roche/Prothena)/Cinpanemab (Biogen): • Upstream lysosome repair vs. downstream aggregate mopping. • mAbs are dealing with extracellular or interstitial α-syn; they do little for the intracellular lysosomal/ER stress and protein degradation defects that likely drive disease. • GT-02287 aims to repair lysosomal function at the enzyme level, indirectly reducing α-syn burden by making the cell’s own disposal system work better. 

microcapreturns

Well it was a lot more than smell and patients wouldn't say smell improved or returned if it stayed the same. The placebo affect is ruled out by the UPDRS data. After 30 days no improvement and after 90 days statistical improvement. Besides smell they had reduction in tremors, better balance and improved motor skills. These patients were being tested multiple times. Sure people have good and bad days but thats why its tested multiple times. Listen to the interview. Leveraging AI: The Biotech Cracking the Code on Rare Diseases https://youtu.be/CJcH-n6iKVU?si=BWkW1cEYdv71FN9X CEO Gene Mack: This is a buy at $10, $15 or $20. Multibillion revenue opportunity per year. Alzheimers,  Gaucher and other neurological diseases also possible. Patients saw functional improvement in motor functions, tremors, balance and regained smell after only 90 days on GT-02287. Patients and clinicians requested extension of trial so they wouldnt lose the improvements they have seen.

Correct_Proposal_409

For anyone interested, OP is talking about this post (which I made): [https://www.reddit.com/r/pennystocks/comments/1pex0jz/gt02287\_addresses\_multiple\_potential\_sources\_of/?utm\_source=share&utm\_medium=web3x&utm\_name=web3xcss&utm\_term=1&utm\_content=share\_button](https://www.reddit.com/r/pennystocks/comments/1pex0jz/gt02287_addresses_multiple_potential_sources_of/?utm_source=share&utm_medium=web3x&utm_name=web3xcss&utm_term=1&utm_content=share_button) The whole point of the post was to explore mitochondrial dysfunction as a potential root cause of Parkinson’s — something that recent research (which I linked) is starting to support. Gain’s Neuroscience 2025 poster ties directly into that idea, showing that GT-02287 restores both mitochondrial and lysosomal function, addressing multiple upstream sources of disease. The reference to the one PRKN patient was just to highlight how that case fits the same pattern — PRKN-related Parkinson’s is driven primarily by mitochondrial dysfunction, making it directly relevant to a mitochondria-first model of pathology. I was *not* saying that this one patient proves the mitochondrial-first. But that one patient did show improvement in UPDRS scores (4 point improvement on II/III) in the 90 days while treatment-naive, when the average Parkinson's patient would have worsened by a little more than 1 point in the same period. I find this both interesting and hopeful. Same as I do about the other patients who showed improvement during the same period. But the PRKN was relevant to the mitochondria-first model.

ImaginationRoutine96

        Yeah, I saw your other post and this comment and it reads like someone that’s trying to save their book, most likely because you have a short position and are getting wrecked. Some of the info is flat-out wrong and misleading.  1. Claim: “N=1 PRKN, 1–3 point UPDRS = noise” We’re not dealing with just one PRKN patient anymore. Gain’s December corporate deck shows 21 patients enrolled and 9 patients with 90-day MDS-UPDRS data, with a mean improvement of about –4.6 points on Part II+III at Day 90, and no acute dopaminergic bump at Day 30. Multiple PD studies treat roughly a 3–5 point improvement on MDS-UPDRS Part III as clinically meaningful. So saying “1–3 points is indistinguishable from noise” doesn’t align with actual PD clinical standards. 2. Claim: “Placebo effect makes this meaningless” True, PD has a strong placebo effect, but the numbers are being exaggerated. Yes, some trials have seen up to 20–30 percent placebo improvement, but that is the extreme end. A meta-analysis of blinded PD trials shows the average placebo improvement is closer to around 4 UPDRS motor points. Also, the GANX Phase 1b pattern is the opposite of a classic placebo spike. There was no benefit at Day 30 but a growing improvement by Day 90 in patients already on stable PD meds. Placebo responses are usually front-loaded, not delayed.  And Gain is not presenting Phase 1b as proof of efficacy; it’s safety plus biomarker plus functional signal to justify a randomized Phase 2. 3. Claim: “Venglustat failed, so this will too” Comparing venglustat to GT-02287 is scientifically inaccurate. They are entirely different mechanisms: Venglustat is a GCS inhibitor. It lowers substrate synthesis upstream. It reached target engagement but failed in the MOVES-PD trial, showing no benefit and possibly worsening progression. [Sources: MOVES-PD trial results] GT-02287 is an allosteric modulator of GCase itself. It stabilizes the misfolded enzyme, restores lysosomal function, and importantly, repairs mitochondrial pathways. The 2025 Neuroscience poster on Gain Therapeutics website shows GT-02287 increases mitochondrial GCase, restores complex I activity in severe L444P patient-derived cells, improves mitochondrial membrane potential, reduces ROS and cytochrome-c release, and protects dopaminergic neurons in MPP+ models. Independent research in Nature Communications also confirms that GCase is imported into mitochondria and is essential for complex I stability, reinforcing the mitochondrial mechanism that Gain is targeting. 4. Claim: “Other PD drugs like alpha-syn antibodies failed, so this will too” This comparison also doesn’t hold. Prasinezumab and cinpanemab were extracellular alpha-syn antibodies given to patients already far into disease progression. Targeting downstream aggregates didn’t slow disease enough in those studies. GT-02287 is targeting an upstream, genetically validated mechanism (GBA1) that affects lysosomal and mitochondrial failure. It has shown: • Target engagement in humans (53 percent increase in GCase in healthy volunteers by Day 14). • Preclinical rescue in both GBA1-mutant and idiopathic PD models. People should always be cautious with early-phase biotech, but framing GANX as if it’s nothing more than a placebo blip or “another venglustat” isn’t supported by the actual biology or the updated clinical data. If you want to argue valuation or risk profile, that’s fair but the scientific claims you made don’t match the real sources.

Correct_Proposal_409

I assume that when you’re referring to senescence in cells, you don’t mean the ability to divide — since neurons don’t divide to begin with — but rather a state of cellular dysfunction. There are multiple peer-reviewed studies showing that impaired GCase folding and trafficking lead to lysosomal and mitochondrial dysfunction, which are defining features of senescent-like neurons. The reason there aren’t yet any peer-reviewed papers showing the reverse — the impact of restoring properly folded and trafficked GCase on the function of senescent or senescent-mimicking neurons — is because no prior therapy has been able to achieve that (until GT-02287). Gain’s preclinical work shows that in senescent-mimicking cells, GT-02287 restores the key functional domains — lysosomal activity, mitochondrial performance, and mitophagy — which are the core issues driving the senescent-like state in Parkinson’s and related neurodegenerative diseases. Also, did you look at this? [https://gaintherapeutics.com/wp-content/uploads/2025/11/Poster-Neuroscience-2025.pdf](https://gaintherapeutics.com/wp-content/uploads/2025/11/Poster-Neuroscience-2025.pdf) The information is out there for anyone who wants to take an objective look.

Icy-Bullfrog1891

t’s not N=1…..there’s 21 patients, with multiple showing improvement (16 with 5 remaining patients to come). I believe theres one PKRN patient, who responded very well, but no one’s claiming it’s conclusive proof. It’s a useful data point when combined with signals in other participants (smell returning, etc), to help form a reasonable view to (I think) be excited about GT-02287. And regarding placebo, the delayed improvement pattern (Day 30 vs Day 90) is not characteristic of standard placebo response, suggesting a potential drug effect. Add to that the reports of smell returning, I’m thinking it’s very likely disease modifying. The phase 1b biomarker data is due any day/week, as well as the UPDRS for the other 5 patients, so let’s see. In my opinion, yes Phase 1 biotech is ALWAYS risky. But I see this stock as massively de-risked in the 2 years I’ve owned it and am itching to see the biomarker data.

ImaginationBroad2590

OP makes a fair point about the lack of placebo control. Allow me a rebuttal. This trial was not designed to assess the efficacy. It’s a Phase 1B assessing safety and tolerability. Phase 2 moving forward will certainly be designed with a control arm in order to achieve statistical significance on efficacy. That said, the UPDRS data reported from the first 9 patients, not 1, shows an increase in UPDRS scores after 30 days, followed by lower scores at 90 (increase = bad, decrease = good) The implied placebo effect should have lowered the scores after 30 days if that’s all that’s going on here, but it didn’t happen like that. What the observant bulls have pointed out is that this 30 day increase across 9 patients actually helps the GT-02287 case: there was no placebo effect occurring at 30 days nor had the drug had enough time to make an impact, so scores increased as the disease ran its course. Beyond 30 days the drug started to take effect and results were improved at 90 days. Not statistically significant efficacy results, but enough to show a lot of promise - especially if the additional 11 patient data to get to n=20 shows the same trend

MediumSinger8088

Thank you, I love the alternative read. I am enthusiastic but also urge caution and risk management. Respectfully in response to your excellent arguments: Gain is doing the studies right, and evidently plans to incorporate blinded placebo controlled studies rather than historical controls in Phase 2/3, as opposed to the historical controls being used in various rare disease indications. The non-parameteric P-values for MDS-UPDRS improvements reported on the recent poster, by my calculations from data in the table, was 0.14 - not formally significant (requires p<0.05) but odds of 14 against / 86 in favor are OK for me to lean positively into gambling some mad money and the trend is enough to suggest that, if the effect is significant, it is likely large enough that it could be elucidated in a feasible larger study. Regarding Venglustat, as I understand it (and I'm not an expert in this drug) the test agent targeted glucosylceramide synthase inhibitor initially for Fabry's disease. The idea is to prevent build-up of more complex downstream glycosylsphingolipids. GT-002287 GCase enhancer. This is a huge difference - Trying to target glucosylceramide synthesis (Venglustat) strikes me as a "WTH were you thinking???!!!!" strategy - That would create a metabolic block, increasing ceramide concentrations with potentially toxic effects. Excess ceramide can initiate metabolic cascades linked to neurodegeneration, inflammation, and synaptic dysfunction; notably inducing or exacerbating mitochondrial dysfunction. I could draw analogies to the development of cyclooxygenase-2 specific inhibitors that just backed up eicosanoid metabolites in toxic directions (e.g. celecoxib).... Anyway, enhancing GCase would remove a block, not create one. With respect to antibodies, these things have never worked for neurodegenerative diseases. Sure you can reduce amyloid burden or whatever but it's a crude mass-action effect, expensive, with immune complications and danger of freeing up toxic garbage that's safer where it is (think asbestos removal). The old amyloid and tau theories are half-baked biochemical equivalents of Ptolemaic astronomy anyway. If it hasn't translated in 3-4 decades it isn't gonna do it now. GT-02287 subtly engages mitophagy and metabolism in a way that nobody really understand yet but IMO that's better than engaging a faulty theory that "everybody knows is true" that nonetheless has never translated in the clinic using either antibody approaches or small molecules (e.g. Lily's Segamacestat back around 2013).

mjlamott

I was going to trade my GT350 in until I checked the Carvana price for it. Even with having to drive my ass six hours and buy a one way plane ticket home, I was ahead 10k.

microcapreturns

Leveraging AI: The Biotech Cracking the Code on Rare Diseases https://youtu.be/CJcH-n6iKVU?si=BWkW1cEYdv71FN9X CEO Gene Mack: This is a buy at $10, $15 or $20. Multibillion revenue opportunity per year. Alzheimers,  Gaucher and other neurological diseases also possible. Patients saw functional improvement in motor functions, tremors, balance and regained smell after only 90 days on GT-02287. Patients and clinicians requested extension of trial so they wouldnt lose the improvements they have seen.

Born2Vanderbilt

"Gain Therapeutics' experimental drug, GT-02287, has shown promising early results in human trials, including reports of reduced tremors and improved motor function in some Parkinson's patients, but it has not yet made tremors "gone" or "disappear" in humans. The treatment is still in clinical development. " Even weed can reduce tremors short-term Hahaha.

Correct_Proposal_409

I'd encourage you to spend a little more time looking into this. Reduced Gcase activity and misfolding is a key underlying issue in nearly all Parkinson’s cases, and is increasingly understood to be key player in cellular breakdown in other neurodegenerative diseases, and aging in general. Both lysosomal and mitochondrial function rely on Gcase especially. This is relevant far beyond the L444p GBA1 cases, although GT-02287 looks like it is especially powerful in reversing dysfunction in this sub-population. Stabilizing Gcase’s intermediate folding state is an important upstream target that is key in several parts of the “doom loop” featured in multiple neurodegenerative diseases, and likely addresses cellular stress and breakdown in healthy people who are merely aging. My base case for this drug is GBA1. Have you looked into what large pharma's are paying for rare diseases? Parkinson's is not a rare disease by any stretch, and it is growing quickly. There are 1 million cases in the U.S. alone. GBA1 represents 10% of Parkinson's cases, so 100,000 cases. That is multiples more than many rare diseases for which drugs are worth far more than Gain's current market cap. A trail for GBA1 in the U.S. would have no problem recruiting. But it will likely not come to that because of growing research that shows properly functioning Gcase as necessary far beyond GBA1.

ImaginationRoutine96

Anyone who starts a comment with ‘I’m a scientist’ instead of talking about actual data is usually not one. Real scientists discuss mechanism, biomarkers, trial design, and the published biology, none of which you addressed here. GT-02287 isn’t limited to mutation carriers, the trial includes idiopathic PD, and the mechanism is upstream of both genetic and non-genetic forms. Your points don’t line up with how GCase biology actually works. Also, if you look at his other posts he brags about shorting reddit posters stocks. He jumps across different threads pretending he’s an expert to potentially short the stocks. He even brags on one how he’s gone from 2k-20k. If he’s a scientist my dog is a neurosurgeon. 

DarthBuzzard

> And the industry is clearly not anywhere near where even the most optimistic claimed it would be... Yes, and this is a repeating cycle with many new hardware platforms. With PCs and consoles, people expected growth faster than what occurred, so at the time it sometimes looked pretty dire for both of those industries. > You say things like gta6 will be the end of the franchise because it's not vr which is hilariously insane. I never said that. I might have said that I think GTA 7 will be a VR game by the time it releases, can't remember if I said that or not, but I didn't say anything about GTA 6. > You told me given the choice everybody would choose VR over flat, but that just factually is not true. Most people prefer VR when they have the option. As evidenced by how most VR ports are received better in VR. Resident Evil, GT, Wipeout, Hellblade, No Man's Sky, Elite Dangerous etc. Fans and critics praise the VR version more. However those people happen to be early adopters so they are able to withstand the issues of current headsets. I've never made the claim that the general gaming population would prefer these in VR today, but I do think they will when VR has matured - when your average Joe can actually wear a headset for hours without issues where there is no setback in resolution or graphics. In other words if you took away the headset and just judge it based off the medium - what does VR as a medium add to the experience, to the game's design - then most people would prefer ports in VR.

Correct_Proposal_409

We may have already discussed this, but the L444P looks to be about 20-25% of the WT control, and GT-02287 increases it by about 360%, which should be very close to the healthy WT level. Pretty impressive. Is there an precedent that you know of that has achieved such a restoration?

Correct_Proposal_409

I'll answer to what I think could happen in the next three months. In the next week or two pre-data readout, the price could trade in this current range, but I could also see it running up to $5 or $6 in anticipation, especially if there are any leaks. Upon data readout (maybe as early as next Monday), assuming good data, I think the price jumps anywhere from 30% to 200% (look at CAPR today-- that's in phase 3, but the treatable patient population is 50X smaller than for Parkinson's). I really don't know. The market potential is huge, and there are no disease-modifying treatments available, but it is also only finishing phase 1. But I think in the days to follow, I think investors start turning their focus to partnership or buyout figures. So let's say that it settles around $9 in the days following data release. That's a $450M MC. I think $1B or more, either through partnership or acquisition, is likely by March. Many lesser drugs both in clinical trials and pre-clinically have purchased for big sums, so Gain should easily go for $1B in my opinion, especially since they have back-up compounds and a platform that discovered GT-02287 and the other compounds. $1B is \~$20 per share.

Correct_Proposal_409

We don’t know much about this at this point other than the CEO mentioning “reports” of patients saying their sense of smell retuned seemingly out of the blue (paraphrasing). This is an extremely conservative CEO, so I doubt he’d mention this publicly if there were only two reports. My speculation is at least 20% reported this, and from different sites. Even just two patients out of the 16 regaining this sense would be extremely unlikely. And everything we know about the mechanism of action of GT – 02287 would support this outcome.

Correct_Proposal_409

Lysosomal susceptibility due to genetic variants: [https://scienmag.com/tmem175-scarb2-ctsb-linked-to-parkinsons-risk-worldwide/](https://scienmag.com/tmem175-scarb2-ctsb-linked-to-parkinsons-risk-worldwide/) GT-02287 addresses this vulnerability.

Correct_Proposal_409

Here are two articles/papers that point to the mitochondria as sources of Parkinson's disease, and for which GT-02287 could address: [https://medicalxpress.com/news/2025-11-evidence-disrupted-mitochondria-parkinson.html](https://medicalxpress.com/news/2025-11-evidence-disrupted-mitochondria-parkinson.html) [https://pubmed.ncbi.nlm.nih.gov/28882997/](https://pubmed.ncbi.nlm.nih.gov/28882997/)

Correct_Proposal_409

For the first 18 patients, the drug was generally well tolerated, with no treatment-emergent serious adverse events observed. One discontinued due to panic attacks, nausea, and headaches, but it is unclear which (if any) of these had to do with the drug itself. The panic attacks are likely unrelated. Fairly common were reports of headaches & diarrhea, and less common were nausea and fatigue. Some of these could be drug-related, although these symptoms can be common in Parkinson's. Of the first 9 patients for whom we have UPDRS data, one clearly got worse. There are dozens of different gene mutations and other factors associated with individual cases of Parkinson's and it would be unrealistic to expect that GT-02287 could help with all of them. My base case is that it will help with the GBA1 sub-population, but there is strong evidence now that it can help with the majority of cases (idiopathic). The \~80% of patients continuing into the extension are signing up for at least one more lumbar puncture (invasive and unpleasant), blood tests, clinical assessments, and a daily drink that is not appetizing. This level of commitment strongly implies that these patients and their clinicians believe that the treatment is helping.

Correct_Proposal_409

There ate many reasons why it is not too early. And the company went public right as the biotech/pharma market went into free-fall. It’s just woken back up. GT-02287 has largely been derisked already… you just need to look the data. Summary is 90 day data coming up, patients already showing motor function improvement with reports of sense of smell returning (among other things). Very high likelihood pf partnership or buyout in the next few months at multiples of current share price. We know they are currently in discussions. Here’s one post I wrote that discusses some of this: https://www.reddit.com/r/pennystocks/s/qw9fRYQlMo

Correct_Proposal_409

Not sure what to tell you other than it is my story. But it isn't my story that is important, it's the story of Gain Therapeutics and GT-02287 that is important.

Correct_Proposal_409

Parkinson's, Lewy Doby Dementia, and Gaucher's Disease all share the common pathological thread of a-synuclein misfolding and aggregation in the lysosome-- this is what GT-02287 was designed to address. With Alzheimer's the same aggregation happens in the lysosome, but with with other proteins like tau, which corrected Gcase (which is what GT-02287 does) clears. I wrote a post about it 11 days ago (they won't let me send the link, but you can find under my post history-- it's called "Parkinson’s and Alzheimer’s common underlying link. Two birds with one stone?")

rameets

Cayenne Turbo GT! No brainer!!

Worldly-Buy-5874

I believe this will go up significantly before even it gets FDA approved. I think the company will be acquired very soon. If that happens, that will be a good signal since their flagship/primary drug is GT-02287. Another strong signal would be if this drug gets FDA fast tracked. But huge signal would be if they can proof that by providing biomarkers or showing more people got their smell back to show the drug actually restores. Very exciting future ahead with GT-02287 to be honest.

Correct_Proposal_409

Thanks for the great post. A couple things stand out. (1) Excellent point about early diagnosis and preventative care. These will be key strategies with growing focus (I'd hope). (2) More treatments that will work synergistically will become available in the coming years. You and I have chatted about the mitochondria as well, and how they seem to be a big part of the puzzle, alongside the lysosome. It's one of the reasons I am excited about what looks like a more holistic, upstream impact that GT-02287 has on the neuron. Looking forward to finding out what the biomarkers reveal, since they might shed more light on disease progression and pathology itself. Lot's of reasons to be optimistic about the ability to slow, stop, and reverse disease progression in the near future.

Worldly-Buy-5874

* \~11M diagnosed worldwide today; projected 25M by 2050 (fastest-growing neurodegenerative disease). * Diagnosis is still symptom-based; by onset, \~40% of dopamine neurons already lost → millions more undiagnosed. * New α-synuclein seeding assays (pathology-based) are gaining acceptance. Most likely, diagnosis will be faster even without showing symptoms. Preventative care will be super important. * No approved disease-modifying therapy yet; all current treatments are symptomatic only. We are still using levodopa since 1960. * First safe & effective disease-modifying drug will capture massive market share (tens of billions) and likely block recruitment for competing trials (patients won’t risk placebo/delay). Key competitors targeting misfolded α-synuclein which almost all people with PD has it: 1. GT-02287 (Gain Therapeutics): First-in-class oral GCase activator, restores lysosomal function → reduces toxic α-syn. Phase 1 completed. 2. Prasinezumab (Roche/Prothena): Monoclonal antibody directly binding misfolded α-syn; already in Phase 3. Far ahead. 3. HER-096 (Herantis): Intranasal CDNF-related molecule; early data suggest potential restoration, not just slowing. Mechanisms differ but complementary. Possible future combination use (Gain + Roche synergy plausible; acquisition interest likely). **Bottom line:** Positive GT-02287 data would be huge. Regulatory wind is strongly behind the first proven safe disease-modifying therapy, potentially fast-track/accelerated approval highly probable. First-to-market wins by a huge margin. I have PD and I follow every clinical trials/drugs/experiments religiously.

microcapreturns

Merck did a recent $3b deal for just using someone's drug discovery platform. We have an AI drug discovery platform that seems to work. Is that alone worth $60/share? GT-02287 you get for free? Talk about a switch going off and stock price going parabolic.

sleepyguy007

a g80 m3 that thing is hideous. I'd get the AMG GT just because its a coupe and looks like some who isn't blind designed it

amplaoumplasD

I'm a benz guy but BMWs are slowly growing on me. M3 is nice but AMG GT is on another level. Ofcourse depends on the use.

wilkonk

It was more the 8800GT that was crazy value IIRC.

ColdComplaint8

Tire bros are rolling towards fat greasy gains today $GT will be green in a sea of red

microcapreturns

I think it was in this interview and he said 80-90% wanted to continue on because they didn't want to lose their improvements. Leveraging AI: The Biotech Cracking the Code on Rare Diseases https://youtu.be/CJcH-n6iKVU?si=BWkW1cEYdv71FN9X CEO Gene Mack: This is a buy at $10, $15 or $20. Multibillion revenue opportunity per year. Alzheimers,  Gaucher and other neurological diseases also possible. Patients saw functional improvement in motor functions, tremors, balance and regained smell after only 90 days on GT-02287. Patients and clinicians requested extension of trial so they wouldnt lose the improvements they have seen.

Correct_Proposal_409

My base-case is that this at least works for the GBA1 subpopulation, which would be worth multiples of Gain's current market cap. But all signs point to GT-02287 working for a wide range of PD patients.

Correct_Proposal_409

One thing that the company has not done well IMO is to get out the word on what is outstanding pre-clinical work, and now clinical. So, yes, there aren't many of us who've done the homework to see what they have here. It's growing, though. You might be interested in some of the recent research on Parkinson's and the mitochondria. There is a Science Advances study that came out recently which has triggered a few articles. I wrote a post about it (linked below), which links one of the articles. In my post, I also included an abstract to a paper on GT-02287 and the mitochondria that coincidentally shows how it solves the the dysfunction that was explained in the Science Advances study. Gain also presented a poster at the recent Neuroscience 2025 conference, which can be accessed here: [https://gaintherapeutics.com/wp-content/uploads/2025/11/Poster-Neuroscience-2025.pdf](https://gaintherapeutics.com/wp-content/uploads/2025/11/Poster-Neuroscience-2025.pdf) The build-up of misfolded a-synuclein toxic substrates in the lysosome is a well-known "first domino" in the disease cascade, and is what GT-02287 was designed to address. But it turns out that the same Gcase enzyme that GT-02287 corrects and chaperones also plays a very important role in preventing mitochondrial dysfunction, which (at least in some cases) might be the genesis of Parkinson's disease cascade. The data is out there, but it is easy to understand why Gain is flying under the radar. But I think we are seeing the first signs of that changing. Here's the post: [https://www.reddit.com/r/pennystocks/comments/1p0d32f/mitochondrial\_dysfunction\_a\_prime\_suspect\_in/?utm\_source=share&utm\_medium=web3x&utm\_name=web3xcss&utm\_term=1&utm\_content=share\_button](https://www.reddit.com/r/pennystocks/comments/1p0d32f/mitochondrial_dysfunction_a_prime_suspect_in/?utm_source=share&utm_medium=web3x&utm_name=web3xcss&utm_term=1&utm_content=share_button)

Holiday-Career-9349

Completely unrelated here, but which car do you prefer? The Porsche 911 Turbo 964 from Bad Boys 1 or the Shelby GT500, Eleanor from Gone in 60 Seconds? Not that I can buy either, unless it's a scale model, secondhand, as my portfolio shows last month.

gravedigger997

Get a real car, GT4 or a GT500

Snoo23417

I would advise against buying a used GT63 for $45k until you get another 45 for the maintenance.

DonKeedic80

GT63 is for wankers.

1098duc_w_the_termi

Nah don’t listen to that bum. You could have bought a used GT4 and totaled and it still have been better off 🤣

AfraidAnalyst

GT shouldn’t be losing 28 to 0 against PITT into the second quarter, yet here we are

Correct_Proposal_409

That’s the big question. If the data is solid, and especially if a good number of patients are regaining, their sense of smell,$2-$3 billion is easily justified. Epecially if multiple pharmas start trying to outbid each other. $3 billion is nothing for a large pharma that wants to own a best in class, first disease-modifying drug for Parkinson’s, and possibly other neirodegenerative diseases. Not to mention the gain has a couple of assets that might even be better than GT–02287 for Parkinson’s and other indications. Anything under $1 billion would be a steal, although it’s a possibility. Chairman and founder Dr. Kalid Islam sold his last company for 1 billion, so this is not his first rodeo, and he’s never had a drug before with such promise. I think 1 billion is the ground floor. And depending on the data, I don’t think that $5 billion is out of the question if multiple large pharmas start trying to outbid each other.

Correct_Proposal_409

It sounds like Gain will fit this small list since, based on a previous statement, they will be measuring in the CNS: α-synuclein (total, aggregated, and phosphorylated). Regarding your last statement on wanting to see an increase in the CSF, it I'm reading that (paraphrased from AI) increased a-synuclein in the CSF happens with extracellular treatments like monoclonal antibodies, whereas GT-02287 restores function inside the lysosome where misfolded a-synuclein is degraded and not exported into the CSF, so there should be a reduction in "pathogenic or seed-competent a-synuclein species" in the CSF.

Immediate_College918

2020 Subaru Lagacy GT

InvestigatorWooden33

Quickly, year make and model of your car 2019 Kia Stinger GT

Correct_Proposal_409

I hope so too. I’m sorry to hear about your diagnosis. Exciting developments with GT-02287. 90 day data should be out in the next few weeks. Meanwhile, Gain Therapeutics presented new data, which came out today, which shows how GT – 02287 helps recover mitochondrial function. This is a key piece of the puzzle. It’s looking like this treatment checks all of the boxes. Gain and/or whatever pharmaceutical partners with or acquires them will be gearing up for phase 2 likely in the United States. If you are in the US, perhaps you could be part of this study. Hang in there.

ByahhByahh

If 🌽 falls much further we're going to see an influx of GT3RS hit the market

MediumSinger8088

[Gain Therapeutics Presents Preclinical GT-02287 Data at Neuroscience 2025 | Markets Insider](https://markets.businessinsider.com/news/stocks/gain-therapeutics-presents-preclinical-gt-02287-data-at-neuroscience-2025-1035581219)

Glam34

up 3k today GT CELH

ColdComplaint8

market finally waking up to good year($GT) I think we'll see $12 before mid dec

Correct_Proposal_409

Check out this article (linked below). The article explains how both Parkinson’s and Alzheimer’s begin with with misfolded proteins, a-synuclein and tau respectively, which causes a cascade of issues: p62 rises, autophagy stalls, lysosomes clog, and mitochondria start to fail. Once that cascade begins, neurons slowly lose function long before they die. GT-02287 seems to hit the first domino in that cascade. By stabilizing GCase before it reaches the lysosome, it restores proper enzyme trafficking, clears the backed-up protein waste (including α-synuclein and tau), lowers p62, reduces cellular stress, and should reboot autophagy and mitochondrial health. Btw, Gain has measured p62 before (see slide), and I wonder if it will be one of the biomarkers they’ll be measuring in the 1b. [https://newatlas.com/brain/same-mechanism-proteins-parkinsons-alzheimers/](https://newatlas.com/brain/same-mechanism-proteins-parkinsons-alzheimers/) And here’s the PubMed link: [https://pubmed.ncbi.nlm.nih.gov/41027737/?utm\_source=chatgpt.com](https://pubmed.ncbi.nlm.nih.gov/41027737/?utm_source=chatgpt.com) https://preview.redd.it/aek2tkwnoi1g1.jpeg?width=1241&format=pjpg&auto=webp&s=429bc654ee111a2dc475122e93a2652fab2c955a

Correct_Proposal_409

https://preview.redd.it/liydunxc5f1g1.jpeg?width=1284&format=pjpg&auto=webp&s=362b3c2f535995f3532c48efbe6ef92200e357a0 You’re right that dilution is common in biotech, and it in fact has been happening some via Gain’s ATM recently — but in Gain’s case it’s looking less and less likely that there will be a large, go-it-alone cash raise for P2. Gain has been in active discussions with a handful of mid- and large-cap pharmas for at least the last year, probably longer. Management has made it clear they do not want to run Phase 2 or Phase 3 alone, and these potential partners have already completed nearly all of their due diligence. At this point, the only missing piece is the biomarker data… and realistically, these companies either already have early access to it or will see it next week at Neuroscience behind closed doors. GT-02287 went into Phase 1b already mostly de-risked, with: • strong mechanistic clarity (ER → lysosome → mitochondrial rescue) • 53% GCase activity increase in healthy humans • full motor rescue in multiple animal models • dose-dependent reversal of α-syn and toxic lipids • safety and CNS penetration already proven The upcoming biomarker readout is essentially the final confirmation… no meaningful downside risk and enormous upside potential. Even (what I believe is) the base-case scenario (highly effective in GBA1 patients) is a multibillion-dollar market. Big pharma clearly does not necessarily wait for P2 anymore (see slide). If you’re Merck, AbbVie, Lilly, or Roche… why would you risk letting the first disease-modifying Parkinson’s drug in history slip to a competitor? Especially when: • 90%+ of PD patients have lysosomal / GCase pathway dysfunction • Early signals already include improved smell and balance — the rarest and strongest markers of disease modification • Gain’s market cap is \~$100M I this is exactly the point in development where large pharma steps in: after the biology is validated but before the price goes up 10×. Dilution is possible— but a partnership or acquisition is far more likely, and far more rational, given the competitive landscape and what’s at stake. Btw, they still have a significant amount of their ATM available, which they’ve been using strategically to add runway space.

Correct_Proposal_409

Forgot to address the UPDRS scores. Yes, their number is encouraging, but it was over 6 months I believe, not three months. So the scores are not better on a per month basis. With GT-02287, there were no changes at 30 days, but improvements at 90. This suggests no placebo effect. Not sure if there is data on that for Ambroxol. And it is unknown at what point the drug started improving motor symptoms. Was it at 30 days? at 60 days? It is thought that the improvements could be accelerating into 90 days and beyond, but this is unknown territory. Also, while anecdotal, patients reported a return of sense of smell, which can only point to a reversal of disease pathology.

Correct_Proposal_409

The increase of 35%, if I'm not mistaken, is an increase in protein concentration, not enzymatic activity, which I believe actually decreased. It is enzymatic activity that matters. GT-02287 increased enzymatic activity by 53% in healthy volunteers, which was surprising since it was thought that healthy volunteers would have near optimal levels and consequentially the drug wouldn't have much of an impact due to homeostasis. It is thought that GT-02287 will have an even bigger effect on actual Parkinson's patients, especially ones with GBA1 mutation.

yellowbreadletter

I think you should re-read the study data on Ambroxol a bit more carefully. They found a statistically significant increase of GCase levels in CSF of 35% which is quite a large increase. The decrease I believe you are referring to is the enzymatic activity of GCase in the CSF. Ambroxol is pH dependent. The pH of extracellular CSF is 7.4, due to its higher pH the GCase that is being tested in CSF does not allow the Ambroxol to detach from the GCase protein, therefore the protein can not function as an enzyme while bound to Ambroxol. Ambroxol is a weak base and binds to GCase at higher pH levels and detaches at lower pH levels, once reaching the lysosome where the pH is approximately 5.0 Ambroxol detaches from GCase allowing it to perform its enzymatic function. Study participants also showed an average of a 6.8 point reduction in MDS-UPDRS part III motor scale, which was a greater increase than was seen in the 1B trial for GT-02287. Certainly GT-02287 appears to be a better targeted treatment not requiring the patient to take 19 tablets per day, but if there is a generic that is "almost as good" then big pharma may be reluctant to make an acquisition here. I do look forward to reading the biomarker data on GT-02287 however.

Correct_Proposal_409

Yes, it has had underwhelming success to say the least. For whatever reason, it does not seam to increase Gcase activity consistently or impressively in the CNS (where it counts). In some trials, Gcase went down in the CNS. Preclinical work showed a little lysosomal clearing, but nothing like GT-02287, and no reduction of ER stress, improved mitochondrial function, reduced neuroinflammation like 2287 showed. Basically, not even considered competition.

Correct_Proposal_409

Thanks for your thoughts. That’s interesting about the neurotrophic response. I’ve heard the word thrown around before, but I didn’t know that this is what it meant. Sounds like this neurotrophic response is a part of neuroplasticity. I know that there are protocols to enhance autophagy, namely fasting, intermittent fasting, fast-mimicking diet. If/when the lysosome has recovered function (i.e., with GT-02287), autophagy should re-engage. Perhaps a fasting protocol would add synergy. May I ask if you are in a related field?

reddit_lemming

Bro, you gotta stop with the “bench” analogy. A top end GPU isn’t price inflated like a Rolls because of “exclusivity” and hand-stitched wool seats or whatever, it’s expensive because it’s using cutting edge tech to be fast as fuck on track, like a GT3 RS. You know what isn’t the fastest, but is still fast enough for most drivers to not notice the difference? A five year old previous gen GT3 RS. Rolls analogy is a straw man.

reddit_lemming

You’re right, they’re charging 911 GT3 RS prices. And you know what isn’t the FASTEST track car in the world, but is still pretty fucking fast? A previous gen 911 GT3 RS.

ImaginationRoutine96

You’re framing this like Gain is clueless about whether the drug gets into the brain, but that shows you haven’t actually looked at the data. They already measured CNS penetration in humans in the Phase 1 MAD cohort. It’s right there in the deck. Healthy volunteers showed clear CSF exposure of GT-02287 — 3.1 ng/mL on average, which matches the levels that worked in rodent models. The rodents actually had 2–8x higher drug levels in actual brain tissue than plasma, which is exactly what you want for a CNS drug. So no, they’re NOT “waiting to find out” if it crosses the BBB. They already know it does. The upcoming readout isn’t about basic penetration; it’s about confirming that long-term dosing in Parkinson’s patients produces consistent CNS levels and that those levels tie into: GCase activation, lysosomal + mitochondrial restoration, sphingolipid reduction and alpha-synuclein-related biomarkers On top of that, they’ve already shown functional improvement by Day 90 in the Phase 1b group. That alone sets them apart from pretty much every symptomatic PD treatment and most early “disease-modifying” attempts. And yes, other companies are working on Parkinson’s. That’s not the point. AskBio’s gene therapy is a totally different approach which is invasive, risky, and expensive. GT-02287 is an oral small molecule that restores GCase function across the entire disease cascade. If you actually compare mechanisms, they aren’t even in the same category. So the idea that Gain is behind or “doesn’t know” whether the drug hits the brain is just wrong. They already proved CNS exposure. What they’re about to share is the translation whether that CNS exposure, in actual PD patients, lines up with biomarkers and functional benefit. That’s the data big pharma waits for before writing a check.

Correct_Proposal_409

Absolutely correct. The other important point is that there doesn't need to be reversal of disease progression, or even stopping of disease progression. The first drug that SLOWS disease progression will be worth in the many billions. Yet GT-02287 is showing reversal to some degree.

Correct_Proposal_409

https://preview.redd.it/pwven8yh201g1.jpeg?width=1284&format=pjpg&auto=webp&s=56e0c4b9c277382b46e78a3cc95563f4cd1f5c17 Here are some recent early/ pre-clinical acquisitions just in the GBA1 drugs, all much lesser drugs with much smaller markets than GT – 02287. The upcoming data will prove the mechanism of action and effectively de-risk the drug. It would be foolish for large pharmas not to at least offer a partnership deal with upfront cash and milestones. It’ll get much more expensive for them to wait.

Correct_Proposal_409

While I expect that it will be fast-tracked since it has a good safety profile and since there is no current disease modifying treatment for Parkinson's, the company is not going to run the phase 2/3 study themselves. They've made it clear that they will partner with or be acquired by one of the handful of pharmas with whom they've been speaking. 90 day biomarker data along with UPDRS clinical assessment scores will be out in a few weeks. The CEO is confident that they will be consistent with the what they've already seen with the interim data and the patients who had already completed the 90 days. If and when this happens, talks with these "partners" will accelerate in earnest. I can't see them getting acquired for under $600 million. That would be a crime IMO. But I think it is more likely that it gets bid up. I doubt Merck wants one of their competition to own a drug that makes their treatments obsolete. The question is how much will they pay? Pfizer just did a deal for $10 billion for two weight loss drugs with a lower estimated peak yearly revenue (than a first and only disease modifying Parkinson's drug), and those two drugs might take longer to get to market than GT-02287.

microcapreturns

Patients had their ability to smell return. HUGE. Leveraging AI: The Biotech Cracking the Code on Rare Diseases https://youtu.be/CJcH-n6iKVU?si=BWkW1cEYdv71FN9X CEO Gene Mack: This is a buy at $10, $15 or $20. Multibillion revenue opportunity per year. Alzheimers,  Gaucher and other neurological diseases also possible. Patients saw functional improvement in motor functions, tremors, balance and regained smell after only 90 days on GT-02287. Patients and clinicians requested extension of trial so they wouldnt lose the improvements they have seen.

HartbrakeFL21

Hell yeah. I've got some old Mustang GT parts laying around that I'm re-gifting for Xmas.

Glam34

Im betting on GT. Reducing debt but got hit hard by rubber tariffs. Even if they dont get struck down, i think theyll be on the short list of "natural resources that cant be produced in u.s." that will get rid of them.

Correct_Proposal_409

Sounds like you have synesthesia. GT-02287 might help you with that.

TickTockTaudit

Absolute peak 90s is Einstein on the Beach by Counting Crows. Song fucking rips. Had a Fox body Mustang GT convertible, smoking Marlboro Lights, drinking Wawa iced tea. I’d go back in time if I could.

ColdComplaint8

any $GT tire bros out there?

Correct_Proposal_409

See my other response to you below, but this illustrates why dilution is zero concern for me. Pfizer just won a bidding war to acquire Metsera for $10 billion for their two GLP-1 (weight loss) drugs that may not get to market before Gain’s GT-02287. Those two drugs are estimated to have peak sales of $5 billion per year, whereas the first disease modifying drug for Parkinson’s would be somewhere in the $5 billion to $30 billion range. Due to the nature of Parkinson’s disease and they’re being no disease modifying treatment, GT-02287 could be expedited through the development process and available within 2 to 3 years, maybe less. https://www.reuters.com/business/healthcare-pharmaceuticals/pfizer-sweetens-offer-metsera-bidding-war-against-novo-bloomberg-news-reports-2025-11-08/

Correct_Proposal_409

This drug helps Parkinson’s patients, but it has been shown pre-clinically that the same enzyme, GCase — which the drug chaperones to the lysosome and beyond so it can do its job — is responsible for clearing out toxic substrates that build up inside neurons. In Alzheimer’s, those toxic substrates take the form of misfolded tau and amyloid proteins. By restoring GCase function, GT-02287 could help the brain clear these aggregates at their root cause, improving lysosomal and mitochondrial health — the same cellular systems that collapse in both Parkinson’s and Alzheimer’s.

Correct_Proposal_409

Very good point. GT-02287 clears the lysosome of tau (one of prime suspects for Alzheimer’s) just like it clears a-synuclein for Parkinson’s.

Correct_Proposal_409

Too early for what, buyout? Sanofi just acquired Vigil for $470 million for a phase 1 Alzheimer’s drug. Merck and Abbvie both bought pre-clinical GBA assets for over $600 million each ($100 million up front plus milestones). Merck also bought Calporta for their lysosomal treatment for $576 million. None showed disease modification like GT-02287, which it has shown both pre-clinically, and now in actual Parkinson’s patients.