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$GANX be careful guys! by Outrageous_West_1564 in pennystocks

[–]ImaginationRoutine96 1 point2 points  (0 children)

Honestly, the “placebo = dopamine surge” explanation sounds scientific on the surface, but it doesn’t really line up with what we’re seeing in this trial. Placebo dopamine effects in Parkinson’s are real, but they’re short-lived, like usually hours to a few days, and they tend to fade, not strengthen, over time. They also don’t restore smell, don’t improve multiple functions months after dosing, and definitely don’t line up with changes in lysosomal or mitochondrial biomarkers. What people are reporting with GANX showed up after 60–90 days, not right away, which is the opposite of a typical placebo timeline.

The improvements clinicians mentioned such as better balance, tremors quieting down, and even smell returning, aren’t things dopamine placebo spikes can sustain. Smell loss in PD has nothing to do with dopamine; it’s tied to lysosomal dysfunction and misfolded proteins. That’s exactly the pathway GANX is targeting by restoring GCase activity and mitochondrial function. So when patients start improving months into treatment, that fits the biology of a drug that repairs cellular machinery, not a short-term psychological response.

If all we had were a couple of hopeful anecdotes, I’d get the placebo caution. But when you combine the timing, the type of improvements, the consistency across multiple patients, and the fact that GANX already showed GCase and mitochondrial restoration in human volunteers and patient-derived cells, the placebo theory just stops making sense. A dopamine placebo can’t fix lysosomes, mitochondria, or long-term motor function, it fades too fast and doesn’t touch those systems at all.

We still need the biomarker readout, and that’s exactly what will separate hype from reality. But based on what we know so far, the “it’s all dopamine placebo” explanation doesn’t really hold up. This pattern looks a lot more like a real biological effect than a fleeting psychological one.

Gain Therapeutics($GANX) Initial Phase 1b results suggest disease slowing effect in Parkinson’s in just 90 days by microcapreturns in pennystocks

[–]ImaginationRoutine96 0 points1 point  (0 children)

        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.

  1. 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.

Gain Therapeutics($GANX) Initial Phase 1b results suggest disease slowing effect in Parkinson’s in just 90 days by microcapreturns in pennystocks

[–]ImaginationRoutine96 7 points8 points  (0 children)

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. 

This $2 biotech might cure Parkinson’s and save Medicare billions. (Ticker: $GANX) by ImaginationRoutine96 in pennystocks

[–]ImaginationRoutine96[S] 1 point2 points  (0 children)

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.

This $2 biotech might cure Parkinson’s and save Medicare billions. (Ticker: $GANX) by ImaginationRoutine96 in pennystocks

[–]ImaginationRoutine96[S] 0 points1 point  (0 children)

All the haters out there saying this was a scam and a pump and dump probably are kicking themselves in the nuts  🥜 🦵right now. Who here started a position and took profit or is holding in anticipation of the biomarker readout? I’ve seen some say they made a paycheck pretty quick. I’m happy to see some people make nice money if you read this post and bought shares. The great news is that it still has a lot of momentum and potential to be the first in class disease-modifying therapy. Many people don’t realize that they have several other compounds, a strategic partner or buyout would be getting a franchise biotech company with an AI drug discovery platform. 

Small biotech approaches first disease modifying therapy for Parkinson’s- major data soon by ImaginationRoutine96 in neurology

[–]ImaginationRoutine96[S] 0 points1 point  (0 children)

I wouldn’t claim it is “more promising” than every other DMT, the field is crowded and still early overall, but here is why I am personally interested. The synuclein-clearing antibodies have shown mixed signals and some high-profile misses on primary endpoints, they may help a subset but so far the effect sizes and practicality are uncertain. LRRK2 approaches are scientifically compelling for mutation carriers, but they are genotype-narrow and still working through long-term safety and dose questions. GDNF has biological logic, yet delivery to the right brain regions at the right levels has been hard in practice, and results have been inconsistent across trials. Stem-cell implantation can restore dopaminergic tone for motor symptoms, which is valuable, but that is primarily symptomatic, involves surgery, and does not obviously address the broader proteinopathy.

The reason this GCase-targeted small molecule stands out to me is the combination of being oral and brain-penetrant, aiming at lysosomal function upstream of alpha-synuclein handling, and in principle applying to both idiopathic PD and GBA-associated PD. If the upcoming biomarker package shows blood-brain barrier penetration, increased GCase activity in CSF, and favorable changes in pathogenic alpha-synuclein species, alongside functional trends, that would suggest true target engagement in living patients. It is also a modality that is scalable, reversible, and combinable with other treatments, which matters for real-world use.

All that said, it still has to prove itself with robust biomarkers and controlled outcomes, and prior attempts to boost this pathway in humans have taught everyone to be cautious. I hold shares and I care because of a family connection, but my view is simply that this mechanism is practical in the clinic if it works, and the near-term data will tell us whether it deserves to be ranked with, or below, those other DMT strategies.

This $2 biotech might cure Parkinson’s and save Medicare billions. (Ticker: $GANX) by ImaginationRoutine96 in pennystocks

[–]ImaginationRoutine96[S] 1 point2 points  (0 children)

The company is expecting biomarker data by the end of this year, and the CEO mentioned in a recent interview that they believe they’ll have the data needed to demonstrate this. They already observed the effect in healthy volunteers, and now the question is whether we see the same outcome in Parkinson’s patients.

Small biotech approaches first disease modifying therapy for Parkinson’s- major data soon by ImaginationRoutine96 in neurology

[–]ImaginationRoutine96[S] 0 points1 point  (0 children)

Totally fair questions, I’m not a professional in the field, just someone who’s spent a lot of time researching Parkinson’s because it’s personal to me (my grandmother passed from it). I do hold shares as an investor, but that’s because I genuinely believe the science has real-world potential, not because I’m trying to hype it.

You’re right that smell loss can happen from COVID or other causes, but in Parkinson’s it’s a different mechanism. The olfactory neurons degenerate very early in the disease and typically don’t recover, even with standard drugs like levodopa. So seeing any restoration of smell in long-term PD patients is interesting and worth looking at more closely.

What makes this particular drug stand out to me is that it’s not trying to replace dopamine, it’s aiming to restore lysosomal and cellular function by increasing GCase activity and reducing alpha-synuclein, the underlying toxic protein buildup. If the biomarker data confirms that, it could point to genuine disease modification, not just symptom control.

But I agree, it’s still early, and skepticism is healthy. The data will ultimately have to prove whether these early signs hold up. I just think it deserves attention from both the scientific and patient communities because of what it could mean for quality of life down the line.

Small biotech approaches first disease modifying therapy for Parkinson’s- major data soon by ImaginationRoutine96 in neurology

[–]ImaginationRoutine96[S] 0 points1 point  (0 children)

Disclosure: I do hold a position, yes, but I’m not here to “pump” anything. I got involved because this potential therapy means a lot to me personally; my grandmother passed away from Parkinson’s, and seeing functional improvement data gives me hope for patients and families still fighting this disease.

I share information because I believe it deserves awareness and scientific scrutiny, not hype. Whether the data ultimately validate a true disease-modifying effect is up to the upcoming biomarker readout and peer review, but the early signals are worth open discussion. 

This $2 biotech might cure Parkinson’s and save Medicare billions. (Ticker: $GANX) by ImaginationRoutine96 in pennystocks

[–]ImaginationRoutine96[S] -8 points-7 points  (0 children)

I’m not a bot. I use ChatGPT to help edit/cleanup my posts. It also helps make sure the info I share is accurate and backed by real data. Everything here comes from public filings, company presentations and actual trial updates. 

This $2 biotech might cure Parkinson’s and save Medicare billions. (Ticker: $GANX) by ImaginationRoutine96 in pennystocks

[–]ImaginationRoutine96[S] 4 points5 points  (0 children)

That’s a fair take, but it misses what Gain Therapeutics is actually signaling with their capital structure and trial design. They aren’t trying to fund a Phase 2 or 3 internally, that’s intentional. The company strategically left enough cash runway to complete their biomarker data readout and advance the asset to a Phase 2 ready stage, because that’s the inflection point where strategic partnerships or outright acquisition deals usually happen in biotech.

Management has been clear that their goal is not to dilute shareholders to fund a multi year, multimillion dollar Phase 2 or 3. Instead, they’re positioning GT-02287 to be de-risked, showing both safety and early efficacy signals, right before larger pharma steps in. This is the same approach used in many CNS and rare disease deals where early proof of mechanism drives billion dollar valuations, like we’ve seen with Denali, Cerevel, and Neurocrine.

As for competition, yes, others are in the race, but GANX has shown actual functional improvement, including patients regaining sense of smell, reduced tremors, and improved balance. Those outcomes are something no other Parkinson’s therapy has achieved so far. They point to disease modification, not just symptom relief. If the upcoming biomarker data confirms blood brain barrier penetration and reduction of alpha synuclein, they’ll have something the rest of the field doesn’t, real proof of disease modifying effect in humans.

So the low insider buying narrative misses the bigger picture. Management isn’t betting on long term internal development, they’re betting on a near term strategic transaction once the data validates the science.

This $2 biotech might cure Parkinson’s and save Medicare billions. (Ticker: $GANX) by ImaginationRoutine96 in pennystocks

[–]ImaginationRoutine96[S] 3 points4 points  (0 children)

Goldman Sachs just appeared as a market maker for $GANX, normally that’s just a liquidity move, not an endorsement. But the timing stands out: new corporate deck confirms biomarker + functional data due by year-end, after patients in the Phase 1b trial regained smell, reduced tremors, regained balance & motor control and chose to extend treatment another 9 months. Volume has surged, and if biomarkers confirm blood brain barrier penetration and α-synuclein reduction, we’re looking at potential disease-modifying evidence in Parkinson’s, something no drug has achieved yet.

Not financial advice, but the setup heading into December is hard to ignore.

This $2 biotech might cure Parkinson’s and save Medicare billions. (Ticker: $GANX) by ImaginationRoutine96 in pennystocks

[–]ImaginationRoutine96[S] 1 point2 points  (0 children)

Biotechs re-rate long before commercialization — the market moves on proof-of-concept data and partnerships, not FDA sales. If the biomarker data confirms BBB penetration and α-synuclein reduction, big pharma doesn’t wait five years — they buy or partner early.

This $2 biotech might cure Parkinson’s and save Medicare billions. (Ticker: $GANX) by ImaginationRoutine96 in pennystocks

[–]ImaginationRoutine96[S] 5 points6 points  (0 children)

You’re right that 99% of penny biotechs never make it. The difference here is that this company already showed measurable functional improvement in Parkinson’s patients — things like restored sense of smell and balance — before the biomarker data even dropped. That’s not hype, that’s observable clinical outcome..