GT

MediumSinger8088

I am "hoping" (I hate that word, really; hope makes you believe things that just aren't true, but nonetheless) that GT-02287 keeps working. But here, objectively, where it stands: One expects MDS-UPDRS Part 3 (the semi- objective clinical evaluation) to increase by 2.5 points in on year for the patients in the Phase 1 study (assumptions: Early stage patients receiving drug from the levadopa "on" state). Divide that by 4, you get +0.63 points every 90 days. So expected +0.6 at 90 days, we've got -2.5, for a difference of -3.1. The minimum signal is about 3 points, some say 4 points. So time marches on and the ONLY question is this, in my opinion: Will the difference between the expected MDS-UPDRS Part 3 and the observed value grow, stay the same, or shrink as the continuation data trickles in? For those who want to play at home, here's the table to fill in. Numbers are rounded to the nearest tenth. KEEP IN MIND the scale is 132 points so a -3.1 is still only a 2.3% difference; minimum signal/noise. Repeated measures at equal time intervals can improve confidence because if the results of any one time point may be due to chance alone, but all-time intervals show the same effect, the probability of chance effect decreases multiplicatively. Time (days) Expected median change Observed median change Delta 0 0 0 0 90 0.6 -2.5 -3.1 180 1.3 ? ? 240 1.8 ? ? 360 2.5 ? ?

Sorry-Point-999

Already did...bought a Xiaomi phone last year (15 Ultra + camera kit) and ditched iPhone. Bought a Huawei watch recently (GT6 Pro). Both products are brilliant and far ahead of stagnant American tech. Will be putting my name down for one of the EVs that are heading here to Canada soon as well.

pmotiveforce

Sweet. Then I could beat you in a used 2024 Mustang Mach E GT that I bought for 30k.

OpeDefinitely

While we're throwing out dream cars, somebody buy me a 2013 Shelby Mustang GT500

ChuckOfTheIrish

Years late but ran back across this reflecting on the experience I had. Apple absolutely used toxic contracts to bankrupt the company, but company leadership for GTAT was either extremely incompetent or direct frauds. What was announced is sapphire glass would be used for the camera glass and potentially for the screens as it was wildly durable (significantly higher test ratings than gorilla glass), Apple investing was just what investors needed to believe it had selling power beyond just proof of concept. Turns out Apple included very high loans for the glass ovens used and added fine print for various penalty classes and that they could collect at any time, they decided to collect just after GTAT burned all this money on a mass production facility and ovens, bankrupting GT and allowing Apple to acquire on pennies to the dollar. Lawsuit was filed and a pretty decent payout was given to investors, I didn't get a ton, but wasn't that heavily invested (IIRC I got around 15% of my losses back which is good for class action payouts). While GTAT leadership clearly failed legal due diligence or were in on the scam, Apple and some other giants are very well known for intentionally bankrupting small partners with toxic contracts.

Correct_Proposal_409

Completely disconnected with the data, the science, the de-risking, and the potential. 7 analysts have an average target of $8/share, and this is after applying a standard risk discount of getting to FDA approval, which I believe does not take into account the extent to which GT-02287 has been de-risked. In other words, analysts will generally apply the same phase-based discount to to all drugs in phase 1, no matter how much more compelling the biology of one drug is vs another in the same phase. So the price targets do not fully capture this de-risking. I added yesterday, and will add more if it drops further. The silver lining here is that the most promising disease-modifying Parkinson's treatment being developed is at a giant discount.

pmotiveforce

I'm looking at used 2024 generally. Audi q8 (q6 seems to have issues). Got my eyes on a 24 Mach E GT Rally. Optiq and Lyriq other good options. A bit more but the 2024 BMW IX is fucking nice, if I can find one with drivers assistance pro and air suspension for under 50k id buy one.

Correct_Proposal_409

Theoretically, since GT-02287 is an oral drug which gets absorbed into the blood and then crosses the blood-brain-barrier (enabling it to address Gcase dysfunction the brain), it should also work for Gaucher’s, which mainly affects cells in the body. I’m sure Gain has blood plasma levels of GluSph. If they are anywhere close to what they showed in the brain, I’d expect that would make it relevant for Gaucher’s, and especially for the small percentage of cases that include neurodegeneration. Whether GT-02287 could be better than the current therapies remains to be seen, but a 81% reduction in 90 days, if similar in the peripheral cells, would be a bigger impact than current therapies remains. But there are other considerations, and they’d of course need to have proper clinical trials.

Outside_Reserve_2407

I think an example of Ford “coming full circle” would be when it sold exclusive hand-built cars like the GT40.

FUBOSOFI

Idk man everyone thinks they’re pretty lame. Unless it’s a Shelby GT

Correct_Proposal_409

No, and this is one of the things that is so remarkable. Of the 13 patients in the slide above, 7 turned out to have elevated levels of GluSph, and 4 of those 7 were idiopathic, 1 was GBA1, and 2 were "pending". In fact, only one of the patients who *didn't* have elevated levels of GluSph was confirmed to be idiopathic. There were 3 who were "pending" who did not have elevated baseline levels, so maybe there were more. Yes, this is a small sample, but it is completely fair to say that many idiopathic patients have elevated GluSph. This is not controversial. This a giant market. My understanding is that it is thought that GBA1 are more likely to have elevated GluSph than other genotypes, but it looks to be common in idiopathic cases as well. This also does not mean that the patients who did *not* have elevated levels at baseline will not develop elevated levels as their disease progresses. These patients who did not have elevated GluSph at baseline had what is considered to be mild baseline UPDRS pt. 3 scores, although most of the patients who had elevated baseline GluSph also fell into the mild category of pt. 3 UPDRS at baseline. They did not screen for patients who had elevated GluSph in order to enter the trial, so if we are to take this sample of patients as representative of Parkinson's populations, then about 50% of mostly mild cases have elevated GluSph levels. Maybe that number is higher if you include more advanced cases. There were 21 patients who entered the trial only 3 confirmed GBA1 patients. This jives with the estimate that 10-15% of Parkinson's cases are GBA1. This greatly de-risks phase 2 if they can screen for elevated GluSph, or if they can simply pre-specify a sub-group in the study of those with elevated GluSph. This is a huge number. And, importantly, it does NOT mean that GT-02287 cannot be helpful to patients who do not have elevated GluSph. Maybe it takes longer to show benefits, maybe it helps some patients in their mitochondria, and maybe it simply prevents levels of GluSph from elevating int he first place, and results in stable or slowed progression of UPDRS scores, which is disease-modification.

Correct_Proposal_409

You can find the patient demographics and baseline characteristics [here](https://gaintherapeutics.com/wp-content/uploads/2025/10/MDS-2025-Interim-Phase-1b-Data.pdf) for Gain's 1b. You are misinterpreting the Bial data. What they showed was an increase in Gcase activity, but it did not actually *improve* anything (in fact, GluCer *increased--* wrong direction*)*. Read MediumSinger's comment below. Peter Lansbury, who was one of the KOL's today in the Gain event, was involved in the development of Bial's drug, which did not show efficacy. This is why Lansbury was so impressed by the results for GT-02287. Here is a key distinction. It doesn't really matter if Gcase is increased 30% or 90% if it doesn't actually reduce lysosomal dysfunction (or mitochondrial function for that matter). Bial's activates Gcase already in the lysosome. But if there is limited Gcase in the lysosme, or if the lysosome is already dysfunctional, those limited "activated" Gcase may not be able to get the job done. That seems like it could be the issue there with Bial's drug. GT-02287 MOA is different in that it corrects folding in the ER and traffic functional Gcase to where it needs to go, including the lysosome and mitochondria. The GluSph reduction is proof that GT-02287 is working in the lysosome. BTW, GT-02287 also activates other Gcase that is already in the lysosome. A drug that increases Gcase by (just for example) 30%, but reduces GluSph by 80% is much more beneficial and efficient than a drug which activates Gcase by 100% (for example), but fails to decrease GluSph. ANd that's just the lysosome. Bial's drug theoretically should not be able to activate Gcase in the mitochondria, whereas GT-02287 has shown in patient-derived fibroblasts that it completely restored Complex I since it is able to traffic (and activate) Gace to the mitochondria. For the phase 1b, Gain did not screen for patients who had elevated levels of GluSph. Of the 13 patients in the slide above, 7 turned out to have elevated levels of GluSph, and 4 of those 7 were idiopathic. In fact, only one of the patients who *didn't* have elevated levels of GluSph was confirmed to be idiopathic. There were 3 who were "pending", so maybe there were more. Yes, this is a small sample, but it is completely fair to say that many idiopathic patients have elevated GluSph. This is not controversial. This a giant market.

Cautious_Room1910

Forgive me, can you tell me where you read in the publication that only 1 of the participants with a reduction in GluSph is a GBA1 case, 4 are idiopathic, and 2 are other cases? I can’t find this information. First of all, I would like to talk about BIAL: if I’m not mistaken, it is precisely in the PubMed article linked by the other person in the conversation that they show a 2x increase in GCase activity in patients with GBA1 Parkinson’s after 28 days, whereas, although different in the way and consequences, in the case of another drug and phase 1a, GCase activity increased by about 50% with GT. So in sick patients, the BIAL molecule reaches the right tissues and correctly activates GCase (I cannot speculate on whether this results in more or less effect than GT, despite the excellent numbers). This is an extremely promising data point regarding what the drug is precisely trying to achieve (i.e., not preclinical studies in animals that do not have a chronic disease course like in humans and cannot or should not influence proper financial decisions!). Regarding the concern raised by the other user about the transient increase in GluCer with BIAL’s drug, I would not worry, because, as also stated directly in the PDF published by Gain on January 6: page 20, "Reducing GluCer levels has no clinical effect in GBA-PD." In any case, just a few more months to see who will be right regarding the release of the data, noting that, being a private company in an already delicate sector, they are not absolutely required to release biomarker information to the public, and so far this has been the case. Furthermore, their phase 1 study was very short, so if this did not allow presenting useful data, we should at least keep the door open. Now, if it is true that Gain’s data show significant decreases in GluSph even in idiopathic Parkinson’s (I could not find this information), this could slightly change the company’s operational window. However, it is still incorrect to say, as you did, that there is a suggestion from these data that a large number of idiopathic Parkinson’s cases have lysosomal lipid degradation failure as a primary or co-factor… I see only 4 cases, not the majority of the 10+ million Parkinson’s cases recorded worldwide, and therefore, if you cite these as exceptional data, forgive me if I did the same with what I reported above. In conclusion, if GT does not show concrete potential for idiopathic Parkinson’s, it will then have to test its cards on GBA1. BIAL will present phase 2 data in about 3 months, allowing us to conclude the chain, connecting—or not—the increases in GCase functionality to improvement in lysosomal repair function. One cannot invest in BIAL, but are you sure you want to invest in a drug that in 3 months could have a competitor with the same functionalities but 2 years ahead?

Correct_Proposal_409

Well, I think they've gone a long ways already towards de-risking, but :de-risked" is never absolute. As more data comes in from the extension patients, assuming the scores remain stable or improve, this is further de-risking. I think the big question is at what point will a big pharma make a fair offer for buyout or partnership. I think Gain has already received offers, just not reasonable ones. Maybe there's one one the table or will be soon, or maybe potential partners are waiting for the 6-month UPDRS scores. I don't know. The only thing I can say with confidence is that with the GluSph reductions and what looks like a clear translation to UPDRS improvements, the GT-02287 alone is worth much more than what the current market cap. Not saying there are no risks, but I really like the risk/reward here. BTW, the more institutions wake up and are able to take positions here, the less volatile the share price becomes.

Correct_Proposal_409

A couple of things. First, what is interesting about the above slide is that only 1 of the 7 patients who showed a big drop in GluSph is GBA1. 4 of them were idiopathic, and 2 were "other". So this suggest that a large number of idiopathic cases feature lysosomal lipid stress as a primary or contributing factor. Second, GT-02287 is not simply “stabilizing GCase”. The human data now show functional lysosomal repair (large GluSph reductions) with patient-level concordance to clinical improvement. That’s very different from prior GCase approaches that increased protein or activity without correcting toxic lipid accumulation or showing a clear clinical correlation. Regarding BIAL’s program: it is restricted to GBA1-PD, targets a narrower downstream mechanism, and has not shown GluSph reduction or a biomarker-to-clinical linkage like this. Being further along in development doesn’t help if the biology isn’t hitting a disease-relevant bottleneck. Many PD programs have reached Phase 2+ without demonstrating disease modification. Maybe I'm missing this promising data, but from what I've seen, even pre-clinically, it has not shown improvement in mitochondrial function, reduction in a-syn aggregation, reduced neuroinflammation, reduced ER stress. All of these were shown pre-clinically by GT-02287, and now this reduction in GluSph, along with linking it to UPDRS improvements, goes a long way towards validating these pre-clinical findings. Do you have a link to this promising Bial data?

Cautious_Room1910

It has been observed numerous times in the scientific literature, as well as in the data presented at the most recent Gain event, that GCase activity can be relatively functional and GluSph levels relatively low, while a patient still fully suffers from idiopathic Parkinson’s disease. Of course, one could argue that if GT improves GCase stability, this would ultimately lead to a healthier cellular environment and more functional lysosomes, which would help prevent the formation of Lewy bodies. However, how much of Parkinson’s pathology truly derives from this additional GCase instability, rather than from mechanisms that are more intrinsic to idiopathic Parkinson’s disease itself? This is especially relevant if it is true that, in idiopathic Parkinson’s disease, the disease actually progresses at full speed despite stable GluSph levels and functional GCase-->the problem is somewhere else. GT appears to be a good molecule for cases of GBA1 mutation–associated Parkinson’s disease because, at the very least—if it performs as suggested by current data—it would slow the mutated disease course to regression timelines more similar to those of idiopathic Parkinson’s disease: this would partially eliminate the problem of a cellular environment with poorly active GCase, while leaving largely intact the fundamental pathological mechanisms of Parkinson’s disease. But In mutation cases, it could be possible that the factors driving idiopathic Parkinson’s disease act as a trigger that ignites alongside the problems caused by the GBA1 mutation, or alternatively that these are cases of full-blown idiopathic Parkinson’s disease that are exacerbated by the mutation; one scenario or the other will change the effect of GT for better or worse on the mutated disease, neutralizing a component that may or may not—depending on the aforementioned hypothesis—represent a fundamental bottleneck of the disease. In any case, why bet on Gain Therapeutics when the pharmaceutical company BIAL has a molecule with a similar mechanism of action to GT that exclusively targets Parkinson’s disease with GBA1 mutation, with extremely promising data and already in advanced Phase 2—thus 1–2 years ahead of GT?

Beneficial_Weight766

I dont know... They just had a webinar today to talk about the results and stuff. [Gain Therapeutics Highlights Biomarker Evidence Supporting Disease-Modifying Potential of GT-02287 | Markets Insider](https://markets.businessinsider.com/news/stocks/gain-therapeutics-highlights-biomarker-evidence-supporting-disease-modifying-potential-of-gt-02287-1035686118)

CEOofBeanz

I’ve been a racist since I was a child. I love Formula 1, IMSA, British GT, all kinds of racing.

TheVariantView

Thanks! I do agree it’s an improbable outcome, but it is a bullish one in the case where the administration does move into Venezuela. This post is just a special update for $GT on the macro events.

TheVariantView

Read the previous DD... Again, this is an update on a bullish potential outcome of the current events. If the US secures Venezuela and provides property rights whilst removing sanctions and bringing private companies back in (as they are doing with oil) to their respective plants, I can assure you these companies wont be vulnerable to attacks by gangs. The bullish case on the Venezuela capture is the above happening. The base case is cheaper oil for US $GT facilities, and the bear case is inexistent.

the_Q_spice

>Analyze outcomes Basically all of your “analysis” rests on the US securing Venezuela and basically giving handouts to private companies. You know *nothing* about Venezuelas government or society. It isn’t going to be a fast or clean transition and it’s increasingly clear by now that the US wasn’t invited. You give the *best possible outcome* for GT. The worst? They go through with this frankly hairbrained scheme, get attacked by [Colectivos](https://en.wikipedia.org/wiki/Colectivo_(Venezuela)), *actually* lose everything, and also get sanctioned in the process.

TheVariantView

This is an update on my previous DD, highlighting potential effects Venezuela has on $GT. US control vs Opposition’s control could lead to the same outcome with similar probabilities. I’m not buying $GT off the news, but saying it is bullish for the company given they can reclaim their Venezuelan factory & resume operations without sanctions.

dicktoballratio9000

Sub is full of liberals, what do you expect? Hope you get your GT3… just got my 4S a couple months and I also am on the road to a GT3 😂

microcapreturns

Gaucher was the original target and they did a lot of preclinical work. When they figured out it works for all Parkinsons patients they targeted the much larger market. GT-02287 could be a $50b a year Parkinsons and Alzheimer's preventative medicine.

HowLongCanIMakeACock

Lugano Blue GT3

Correct_Proposal_409

Phase 1 trials are to make sure that a drug is safe and shows target engagement. Phase 2 trials test efficacy in a controlled setting, and when phase 2 trials fail, it is usually because: * there is insufficient CNS exposure * the target turns out not to be central to the actual disease biology, * they don’t have the right patients. The drug may work for a particular subset of patients, but the trial has been diluted by too many patients who have a different disease biology. Parkinson’s Disease is often called Parkinson’s Disease**s** because there are multiple different points of initial failure which ultimately turns into Parkinson’s. Different genetic mutations, and some might start with lysosomal dysfunction, while others might start with mitochondrial dysfunction. Or maybe neuroinflammation. This is still being researched. * they are measuring the wrong endpoints, * they don’t have the timing right (i.e., trial not long enough to show efficacy, or the initial benefits don’t sustain for the length of the trial). * the biomarkers move, but they don’t translate to clinical benefits Gain’s 1b trial, along with the extension, gives them a lot of valuable information which goes a long way towards derisking the phase 2. Many phase 1 trials do not go nearly as far as Gain went since the 1b measured many biomarkers, along with clinical symptoms. This is unusual. The first major hurdle towards proving efficacy was reducing GluSph, since it is both a direct measure of lysosomal dysfunction and also a driver of further dysfunction and cellular stress. By reducing it in such a short time period (90 days) means not only that there was sufficient CNS exposure. It also strongly suggests that the lysosme has regained lost functionality and that the cellular stress burden has been reduced. And based on what we know about Gaucher’s disease, this GluSph reduction is a central marker for disease control, and the parallel lysosomal recovery is the key driver for disease-modification. So the most important biomarker for efficacy is already there. And the UPDRS score improvements that they’ve already released suddenly look more credible. We should know more about further scores and biomarkers shortly, but it follows that if GluSph was reduced so thoroughly in such a short time, other important biomarkers which take longer to affect should show signs of improvement even by 90 days, and if there is broad biomarker improvement, this reliably predicts clinical improvements since they are so well-correlated. The other big advantage of the data they know have is that they can use that to inform the phase 2 set-up. They’ve already narrowed the focus for their phase 2, tightening inclusionary and exclusionary criteria for enrolling patients, and they can further adjust as data comes in from the extension. Same with outcome measures. [Here is their current phase 2 registration](https://clinicaltrials.gov/study/NCT07280299?tab=table) (which can be updated). One inclusionary criteria that stands out is “Positive SAA in CSF at Baseline” and another exclusionary criteria is “PD-associated LRRK2 pathogenic variant or other PD-associated genetic mutations other than GBA1.”  They now know that GT-02287 is more likely to move the needle from a biomarker perspective by screening this way, so this increases the likelihood of success. So to answer your question, based on what we now know, I think the chances for success in phase 2 are high. And if we get more biomarkers (like Complex I, Miro1, aggregated a-Syn, NFL) that are beginning to show improvements, and if the UPDRS scores that are not worsening-- and I think Gain will show this data between the KOL and the AD/PD conference in March-- then I think the chances for phase 2 success will be extremely high.

HowLongCanIMakeACock

Just cold started the GT3

Brilliant_Goose7265

Curl the mostly heavily shorted. Tru and GT have more loyal followings to buy the dips.

Correct_Proposal_409

Yes. I'm not saying that there are no more risks to market. There are many risks from the beginning of a drug's development all the way through until approval by FDA. But GT-02287 has cleared the most significant of the risks. They first did extensive pre-clinical studies that showed that the drug *should* work in humans. They basically showed that GT-02287 reversed Parkinon's in animals. Then they passed a significant test in the phase 1a with healthy volunteers showing that the drug was safe, crossed the blood brain barrier, and achieved its target (Gcase) as predicted by the pre-clinical models. Many drugs fail in one or more of these steps. Most recently, in the phase 1b, safety was further established, and it took a big step towards showing efficacy by reducing GluSph by 75-95%, which had never been done in Parkinson's patients. The improvements in the UPDRS motor scores that showed in October suddenly look like they weren't just random increases. This reduction in GluSph also makes it likely that further downstream, slower-moving biomarkers should also show improvement,,, and clinical symptoms as well. So through the lens of likelihood of a successful phase 2 study, and also through the lens of large pharamas looking to partner, GT-02287 has been derisked to where the answer is "yes". I also think that the drug is even further derisked if you narrow the patient target to GBA1 Parkinson's (\~10% of Parkinson's cases), which is originally what they were targeting-- dysfunctional Gcase caused by a genetic mutation. They've since realized that improving functional Gcase is also important in idiopathic cases as well, which represent the vast majority of PD cases. This is why I think the asymmetry is so rare here. The risk is now low for failure, especially in GBA1 cases, but the upside is enormous.

Correct_Proposal_409

This assumes that GT-02287 doesn’t become known as the most promising disease modifying drug for Parkinson’s, and it also assumes that Gain doesn’t become one of the many companies with early phase treatments that gets acquired or partners with a large pharma that understands the potential and wants the drug in their pipeline. I’m confident that partnership or acquisition will happen by mid-2026, likely sooner. They’ve been in discussions with a handful for a long time now, and are currently under CDA’s with at least two!of them.

microcapreturns

GT-02287 boosts enzyme activity in Parkinson’s brain: Trial data Developer Gain Therapeutics to review results in January webinar https://parkinsonsnewstoday.com/news/gt-02287-boosts-enzyme-activity-parkinsons-brain-trial-data/

TickTockTaudit

Porsche Cayman GT4

No_Bus_9534

I’m trading away my c63 and was considering an m4 but just didn’t like it. About to purchase a GT C in few days. God Speed Brother

Correct_Proposal_409

Not sure I'm following you. I do believe they painted themselves into a corner in that they promised data by the end of the 4th quarter and also signed CDA's/embargos. But the GluSph biomarker was the big one all along, and they provided topline for that. You can find that this biomarker was [**pre-specified here**](https://gaintherapeutics.com/wp-content/uploads/2025/04/ADPD-2025-Phase-1b-Design.pdf). We also now know from the [**Roth report**](https://gaintherapeutics.wordpress.com/2025/12/19/gain-therapeuticsganx-roth-mkm-thought-on-phase-1b-data-raising-price-target-to-10/) that GluSph levels were reduced 75%-95% from baseline. That's a big deal. I think the company assumed that this alone would be big news and enough to lift the share price, but they obviously misread what this would mean for the average investor who has no idea what the implications of this biomarker means. In the PR, they did link the KOL event that had already been planned, and in which it states "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." In retrospect, they should have included this up top in the PR as one of the bullet points (instead of being relatively hidden in the link), something like "additional supporting data will be reviewed during our KOL event on Jan. 6th," To your statement on them raising-- they made it clear that they have enough cash to last through 2026, although this does not include phase 2. That's where partnership comes in, though. Lastly, yesterday's trading volume and price movement was very interesting. Nearly double the volume from the day before, which was the PR and big sell-off day, which was the biggest volume day in the company's history prior to yesterday. That's unusual. Usually it's the big sell-off day that is the high water mark for volume. Again, yesterday, where the share price recovered by finishing up 28%, the volume was nearly double from the day before (sell-off/news day). And there was also a record amount of after-marker volume. This tells me that there was some new buying coming in. Maybe life-science funds. And it would make sense. They are the ones who would understand the implications of what was shared in the PR from the day before.

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.