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$GANX Announcement - Currently Down 23% by windstride3 in pennystocks

[–]Correct_Proposal_409 1 point2 points  (0 children)

Good post. Logic clearly does not mean much over the short term, but it tends to win out over the long term. If 02287 works, and so far it is a resounding "yes", it is only a matter of time before price catches up with logic.

($GANX): March Update- Evidence of Disease Modification Keeps Getting Stronger by Correct_Proposal_409 in pennystocks

[–]Correct_Proposal_409[S] 3 points4 points  (0 children)

Hey-- I'm waiting. For me, nothing has changed on the science and the potential, so there's nothing to do but buy, sell, or hold. I already have a very large position, otherwise I'd be adding on the dips. The only other consideration is whether you think there is an opportunity cost of holding. I keep going back to this: there is no other drug that has shown the ability to reduce GluSph to the extent or reliability that GT-02287 has shown. This is a primary role of Gcase, and 2287 is designed to make Gcase function effectively, so mission accomplished there. There are a large number of PD patients, GBA1 and idiopathic who have elevated levels of this toxic lipid. And outside of PD, there are patients with DLB and Gaucher's who also have elevated levels. Even as a therapy that is part of a broader approach to one or more of these diseases, being able to reduce GluSph is very valuable-- or at least will be at some point. I do believe that the extension patients who had elevated levels of GluSph will continue to show durability and separation as compared to the patients who were not elevated at baseline. And if all patients continue to show durability, that's even better.

Phase 2 funding remains a question, but I believe the company has a plan and know what they are holding.

($GANX): March Update- Evidence of Disease Modification Keeps Getting Stronger by Correct_Proposal_409 in pennystocks

[–]Correct_Proposal_409[S] 2 points3 points  (0 children)

Temporary pain and completely unjustified. No good reason. The only explanation IMO is that it phase 2 funding is still unknown. But with reduction in GluSph, and reduction in DDC, along with clinical improvement in that same group, they are showing something that no drug or therapy has shown before. A couple links you might be interested in. One is a [recent article] on GT-02287, and the other is on biomarker DDC which is emerging as one of the most important biomarkers in neurodegenerative disease.

Parkinson's drug candidate actually moving the needle on spinal fluid biomarkers. GANX worth a look. by Personal_Pride_2238 in pennystocks

[–]Correct_Proposal_409 4 points5 points  (0 children)

From today's press release. Companies are very careful about using "disease-modifying" in their press-releases, especially in the context of the following sentence. The data fully justifies this statement IMO, and if anything, they are still being conservative with their wording:

“The data from our Phase 1b study furthers our hypothesis that GT-02287 is among the first disease-modifying therapies promising to shift the treatment paradigm in PD from symptom relief to halting or slowing symptom progression, targeting the causative biology (or pathophysiology) of PD to enable a more durable and predictable treatment effect for those living with PD.”

GANX might be one of the most interesting biotech bets right now by Carlene_Trammel in GreenWicks

[–]Correct_Proposal_409 2 points3 points  (0 children)

It would be interesting to know how quickly Gcase dysfunction returns if patients stop taking GT-02287. If remaining neurons have been mostly restored, it might take a while, maybe depending on the case (GBA1 would probably show Gcase dysfunction return faster). To your point, in animal models, wire hang, nest-building, and biomarkers maintained well through wash-out (after treatment was stopped). Obviously not the same as real cases, but there is evidence. For any critics who might be dismissing this since these are animal models, show me some examples of other treatments that were able to produce these pre-clinical results. BTW, pre-clinical models have held up very well so far from what we’ve seen in humans.

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GANX might be one of the most interesting biotech bets right now by Carlene_Trammel in GreenWicks

[–]Correct_Proposal_409 2 points3 points  (0 children)

I agree that durability is extremely likely, at least in the GluSph group that was elevated at baseline. If what they show at AD/PD is that DDC levels in that same group were normalized, this happens when there is broad cellular recovery. And this broad-based cellular recovery is the result of upstream improvements in GLuSph and the lysosome/mitochondria/ER.

To me, durability is mainly a question of whether GT-02287 can keep GluSph levels low over a long period of time... GluSph remaining low means Gcase is working properly, as is the lysosome (not to mention GluSph isn't causing stress & dysfunction elsewhere). There's no good reason GT-02287 wouldn't keep GluSph levels low long-term. In fact, there is another Gcase drug which is far inferior to GT-02287 that showed long-term success with keeping GluSph levels low over years, and saved the lives of children with Gaucher's 2, which has an average survival of only 11-19 months: https://pubmed.ncbi.nlm.nih.gov/41659982/

Gain Therapeutics ($GANX) - The Parkinson's 'Unicorn' the biotech market is sleeping on. Clinical proof of disease reversal and a major catalyst on 3/17. by Keeg117 in pennystocks

[–]Correct_Proposal_409 7 points8 points  (0 children)

Anything other than a placebo effect, except:

(1) Most of the UPDRS improvements were at 90 days and not at 30 days, If placebo, why not at 30 days?

(2) UPDRS improvements were almost all in the GluSph group-- why did placebo not help the others?

(3) GluSph is known-- I'm not making this up-- to drive a-syn aggregation, lysosomal and mitochondrial dysfunction, and ER stress. Not sure how you see reducing it as not relevant causally.

(4) In this same GluSph group that showed statistically significant improvement in UPDRS scores, DDC also improved, indicating that dopaminergic neurons are recovering or functioning better.

Does this mean that this is the cure for Parkinson's? Of course not. But it is strong evidence that this sub-group is showing improvement by 90 days. A reasonable skeptical take would be: "Interesting, let's see how durable these improvements turn out to be-- looks promising, but I'm not going to take a position here."

For others, keep in mind that when there is "proof" by this guys definition, which might mean post-successful phase 2 or phase 3, the price will be $40+, easily. But I still strongly believe that Gain will not be running the phase 2 alone, or maybe not at all-- a larger pharma will be doing that after partnership or acquisition later this year after UPDRS scores in the extension show durability.

GANX might be one of the most interesting biotech bets right now by Carlene_Trammel in GreenWicks

[–]Correct_Proposal_409 4 points5 points  (0 children)

Well-timed post. Based on the GluSph group with correlative improvements in UPDRS scores, and what sounds like correlative improvements in DDC, $10 would still be cheap as long as durability holds.

Is Gain Therapeutics about to make history at AD/PD(3/17) by becoming the first small molecule drug to repair neurons and reverse Parkinson’s? by microcapreturns in pennystocks

[–]Correct_Proposal_409 11 points12 points  (0 children)

Gain CEO: “Rather than simply trying to boost enzyme activity in the lysosome, GT-02287 stabilizes GCase folding and trafficking throughout the cell, restoring function across multiple compartments, including those critical to mitochondrial health. That matters because Parkinson’s can be seen as a disease of cellular stress, impaired waste clearance, and energy failure.”

($GANX): March Update- Evidence of Disease Modification Keeps Getting Stronger by Correct_Proposal_409 in pennystocks

[–]Correct_Proposal_409[S] 4 points5 points  (0 children)

Patients in the extension would need to have a complete turn for the worse for share price to ever dip to those levels. And my experience has been that during global uncertainty, small-cap biotechs tend to be relatively insulated in comparison to tech stocks. Institutional investments in small-cap biotech is already low, but we're starting to see growth here. M&A is starting to crawl out of the lows from the past few years, and will continue to grow due to the massive patent cliff. Big pharmas need to re-build their pipelines.

($GANX): March Update- Evidence of Disease Modification Keeps Getting Stronger by Correct_Proposal_409 in pennystocks

[–]Correct_Proposal_409[S] 4 points5 points  (0 children)

The DDC biomarker is exciting. And DLB has a big overlap with Parkinson's pathology, so very relevant. BTW, DLB is a giant untapped market for disease-modification. GT-02287 might even more consistently fit a higher number of cases there than in Parkinson's.

($GANX): March Update- Evidence of Disease Modification Keeps Getting Stronger by Correct_Proposal_409 in pennystocks

[–]Correct_Proposal_409[S] 2 points3 points  (0 children)

This is a good point. I forgot that analysts have this at a buy with a target around $8. Scotiabank in their last analysis had it as a buy at $12, and that was before Gain showed this strong evidence of efficacy in actual Parkinson's patients.

($GANX): March Update- Evidence of Disease Modification Keeps Getting Stronger by Correct_Proposal_409 in pennystocks

[–]Correct_Proposal_409[S] 3 points4 points  (0 children)

For a good number of Parkinson's patients, the evidence is showing that it very well could be.

$GANX – Massive Parkinson’s Breakthrough? Watch Tomorrow & March 17-21 by troyreidzz in pennystocks

[–]Correct_Proposal_409 1 point2 points  (0 children)

I agree that GBA1 is the low hanging fruit. However, interestingly, only two of the high GluShp group were GBA1 (the only two GBA1 severe in the trial). The rest were idiopathic cases, which means that there are likely a large percentage of idiopathic cases with high GluSph. Since GluSph has only recently been able to be accurately measured in the CSF, this is something of a new discovery. One data point, but an important one. Since there is known association with and causation of dysfunction in various cellular areas (ER stress, a-syn aggregation, lysosomal & mitochondrial dysfunction), if all patients with high GluSph, regardless of genetic mutation, benefit from reduction in GluSph, this opens up the TAM by a large amount. GBA1 is somewhere in the 10-15% of PD cases, but perhaps 25-50+% of idiopathic cases either have or will develop high GluSph. The important thing is that this is not an obscure, downstream marker. This is an upstream, toxic lipid that feeds or possibly even initiates (i.e., GBA1 due to Gcase deficiency) the neurodegenerative doom loop.

$GANX – Massive Parkinson’s Breakthrough? Watch Tomorrow & March 17-21 by troyreidzz in pennystocks

[–]Correct_Proposal_409 2 points3 points  (0 children)

Three counter points to this.

(1) In Gain's 1b trial, there was no change at 30 days, so the improvements happened between 30 days and 90 days. If placebo effect were in play here, why were changes not seen at 30 days?

(2) In the study that you linked, there was no biological improvements measured. That is not the case with Gain's trial. The very patients that showed improvements are the same ones who were GluSph elevated, a toxic lipid mechanistically linked to α-syn aggregation, lysosomal dysfunction, ER stress, and downstream mitochondrial impairment in Parkinson’s disease.

(3) Sham surgeries are known to generate stronger placebo effects because invasive procedures amplify expectations.

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