Jason Assad

Former equities trader & small-cap investor. Strategic advisor / consultant to multiple private & public companies. Tweets include companies I work with. DYDD

[Jason Assad @AssadJason]

Great listen...

[Lou Basenese @LouBasenese]

What if Parkinson’s didn’t have to get worse?

$GANX reported updated data showing no decline in motor scores – something rarely seen in this disease.

I sat down with CEO Gene Mack again to break it all down 👇

https://t.co/HSIvSrjDRg

[joe iniowa @Microcapreturns]

$GANX Gain Therapeutics. Recent scientific article validates Gain's approach that increasing GCase can slow, stop and maybe reverse trajectory of Parkinson’s.

https://t.co/skvEDXOq7w

[Jason Assad @AssadJason]

@TreasuryEdge @HyperionDeFi That was a great listen!!! 👊

[Deep Isolation @DeepIsolation]

DOE-funded (@ENERGY) research studied how our Universal Canister System holds up deep underground, helping build understanding around deep borehole disposal as a long-term option.
🔗Full press release: https://t.co/7SQI30R86Z
#NuclearEnergy

[joe iniowa @Microcapreturns]

$GANX Gain Therapeutics in Parkinsons News Today

Early trial data suggest GT-02287 may aid Parkinson’s symptoms

https://t.co/iWiX9XxoKI

[hyunsu.hl @hyunsujung_]

My letter to the shareholders of @HyperionDeFi:

https://t.co/7nMGilcEz6

[joe iniowa @Microcapreturns]

$GANX Gain Therapeutics. Final update before January 6th KOL event. Signup available along with ability to ask questions.

Gain Therapeutics(GANX): Final Update before Phase 1b data at January 6th KOL Event

https://t.co/4Ny6AXnqj9

[MWB74 @MWB741]

Where We Are Now With Gain Therapeutics ($GANX) and GT-02287?

To say that the recent share price movement has been a roller coaster ride would be an understatement. My take on what happened is that the company underestimated the market’s expectations for the 1b topline PR and the average investor’s ability to grasp the implications of the landmark GluSph data, which provided strong proof that the drug is working as designed. Shorts took advantage of the uncertainty, and panic ensued. The share price dropped over 40% from the $4 level that same day. On the two days following (Friday and yesterday), the share price recovered by about 40% to $3.19 on big volume, suggesting institutional buy-in. In other words, retail investors panic sold, shorts cashed in on the immediate drop, and smart money accumulated over the last 2 days as they understood the implications of the GluSph data.

Conviction comes from setting emotions aside, assessing what is known, and then arriving at an investment thesis. To that end, here is my take (with the assistance of AI):

1) What the data says

Preclinical (core scientific foundation, including Neuroscience 2025)

- GT-02287 restores functional GCase activity by stabilizing folding and improving ER-to-lysosome trafficking.

- The Neuroscience 2025 poster extended this further, showing that restored GCase activity also improves mitochondrial health, restoring Complex I function, reduced oxidative stress, improved mitophagy, and enhanced neuronal survival.

- Across multiple models (GBA1 and idiopathic PD), GT-02287:

-Reduced toxic lipid accumulation (GlcCer / GlcSph)
- Reduced ER stress
- Improved lysosomal and mitochondrial pathology
- Reduced α-synuclein aggregation
- Reduced neuroinflammation
- Lowered neurofilament light chain (NfL), a marker of neurodegeneration

- Importantly, these cellular effects translated into functional rescue in animal models, including improvements in motor performance, gait, and higher-order behaviors (e.g., nesting), reinforcing that the biology is not isolated or cosmetic.

Brain penetration, target engagement, and safety (Phase 1a + Phase 1b)

Across both Phase 1a (healthy volunteers) and Phase 1b (Parkinson’s patients), GT-02287 has demonstrated a coherent and internally consistent profile:

- CNS penetration confirmed GT-02287 reaches the brain and cerebrospinal fluid (CSF) at exposures consistent with preclinical therapeutic levels, confirming it crosses the blood–brain barrier and is suitable for a CNS indication.

- Target engagement in humans Phase 1a showed a >50% increase in GCase activity at clinically relevant doses. Phase 1b extends this by demonstrating downstream biochemical effects in Parkinson’s patients, indicating that CNS exposure is not just theoretical but biologically active.

Large-magnitude, mechanism-predicted biomarker effect (Phase 1b)

In Parkinson’s patients with elevated baseline CSF glucosylsphingosine (GluSph), GT-02287 produced an approximately 75–95% reduction, bringing levels back toward the healthy range after 90 days. The implications of this cannot be understated:

- GluSph is not a cosmetic biomarker; it is a toxic lipid that accumulates when lysosomal GCase function fails

- A reduction of this magnitude strongly implies restoration of lysosomal degradative capacity, not partial or marginal engagement

- This level of reduction is consistent with true biological correction, not noise or placebo-driven fluctuation

- It provides direct human proof that GT-02287 is doing exactly what it was designed to do: restore GCase function and relieve lysosomal stress

Early clinical signal: MDS-UPDRS (Phase 1b interim)

- Interim Phase 1b data showed a ~4–5 point mean improvement in MDS-UPDRS total score at 90 days, driven primarily by motor (Part III) and activities of daily living (Part II) components. This matters because:

- Untreated Parkinson’s patients typically worsen ~3–5 points per year on Part III

- Symptomatic drugs (dopamine replacement) improve scores rapidly (days–weeks) and then plateau

- GT-02287’s improvement was delayed, emerging at ~90 days rather than immediately

Data summarized:

GT-02287 has now shown coherent biology from molecule → cell → animal → human CNS biomarkers, with early functional signals aligned to the mechanism. The open question is how durable these effects are over longer treatment periods.

2) What the company has said recently (PRs + interviews):

“I think we have a disease modifying drug for Parkinson’s, and every day I get more certain.” -CEO, Gene Mack

On additional data and disclosure limits

- Management has stated that additional clinical and biological data exist that could not yet be shared due to conference embargoes and active confidentiality agreements (CDAs) with potential partners, in addition to pending further analysis with top key opinion leaders in the world.

- In interviews with @LouBasenese , the CEO emphasized that non-motor functional improvements have been observed (e.g., sense of smell, balance, etc.), with current analysis focused on persistence over time, which is how placebo effects are separated from disease modification. Importantly, regaining sense of smell is not influenced by placebo-effect. We do not yet know the extent of these anecdotal reports other than there were multiple patients experiencing this.

On biomarker relevance

- The company has been explicit that GlcSph was not cherry-picked; it was chosen in advance with FDA and KOL input because it reflects core disease biology, not a peripheral signal.

- GlcSph reduction is linked with to downstream improvements in lysosomal, mitochondrial, and α-syn biology, consistent with the mechanistic framework reinforced by the Neuroscience 2025 poster.

On partnerships

- Gain has confirmed multiple active CDAs with large pharma, signaling ongoing diligence rather than a “wait until Phase 2” stance.

- In neurodegeneration, this typically precedes structured partnerships or option-to-buy arrangements when biology is compelling but durability is still being established.

On cash runway

- Through existing cash, ATM usage, and warrant exercises, management has stated they have sufficient runway to operate through 2026, covering the Phase 1b extension and next development steps without near-term financing pressure.

Upcoming KOL event

- On January 6th, along with two of the top experts in the world. the company will be discussing the findings on GluSph along with further data which supports disease-modifying potential in GBA-1 and idiopathic Parkinson’s.

Overall takeaway

“The data has never been as rich and robust, and the balance sheet never as strong” -CEO, Gene Mack

I don’t know what the price movement will be in the coming days, but I suspect that institutional buy-in, including life-science funds, will be significant in the coming weeks. We are past the question of “does GT-02287 reach the brain and hit the right target?”  The data now support that it does — and that it engages the broader mito-lysosomal disease axis, not just a single enzyme. Multiple biotech analysts were very bullish on the 1b news, and Roth increased their target from $6 to $10. This is after applying a standard biotech risk discount, which could be argued is less appropriate given the extent of derisking that the data supports.

We are now in the phase of “how durable and scalable is the effect?”  That is the type of remaining risk large pharma often chooses to share through partnerships, milestones, options, or CVRs, rather than waiting until all uncertainty is gone. We should know more on January 6th, and my take from company statements are that the remaining data is supportive of disease-modification, which would be a first for Parkinson’s disease, and would also be relevant for other neurodegenerative diseases such as Gaucher’s and Alzheimer’s. Not to mention, a success with GT-02287 would validate their Magellan drug-discovery platform and would greatly improve the likelihood that their back-up compounds hold real promise.

@kkernttb @RealAvidTrader @BiotechStockRsr
@odibro @yaireinhorn @thebiotechforum
@BiopharmIQ @BPharmCatalyst
@SupNovaTrading @StocksPursuit
@Microcapreturns @dixielee1969 @fundmyfund
@makedatbread88 @SheffStation @bwsm12702 @TopStockAlerts1

[Lou Basenese @LouBasenese]

🚀 The latest episode of The Upside is live!

ALS. Dementia. Alzheimer’s. Diseases that impact over 50 million people - with few treatment options.

But $COYA believes stopping progression is possible.

How? I sat down with @CoyaTx CEO Arun Swaminathan to explain it all... without the biotech jargon.

No spin. No gatekeepers. Just potential upside.

[TeraWulf @TeraWulfInc]

What do investors need to understand about $WULF's business model? 👇

Lou: This isn't just a straightforward real estate investment, right? It's not a pure technology one. It's construction. It's regulatory. It's technological... It's a lot more complicated than people think. -@LouBasenese

Paul: I think you got to know the sites. You got to understand the energy mix. You have to understand the transmission of the grid. And most of all, it's an execution story. You have to believe that guy knows how to build a facility and knows how to build a facility when he's had bumps in the road. -@PaulBPrager

[Lou Basenese @LouBasenese]

🚀 The Upside Episode #4 is live!

What’s the one factor that will separate AI data center winners from losers?

$WULF CEO @PaulBPrager lays it out – clearly and directly.

This matters for every name in the space $IREN $NBIS $CORZ $CIFR $CLSK $CRWV

Listen in 👇

[MWB74 @MWB741]

$GANX — The Most Asymmetric Setup I’ve Ever Seen (And the Clock Is Ticking)

This is a long post-- there's a TL;DR at the end.

I’ve made some posts already about the science of GT-02287 and Parkinson’s to the best of my ability, so I won’t bore people with another one (right now). And I don’t think it is necessary to understand the science behind it to recognize all of the signs of a very promising investment.

After watching Gain's CEO Gene Mack’s interview with @LouBasenese (link in comments), I was more convinced than ever that Gain Therapeutics ($GANX) could be sitting on something historic — likely the first truly disease-modifying drug for Parkinson’s. This isn’t hype. Every single data point with GT-02287 since day one has been consistent, strong, and conservative in how it’s been communicated. It's the totality of all of the pre-clinical and clinical work, the still-emerging science of Parkinson's, and company statements that make this such a compelling investment.

Touching on some of his quotes in the interview. Keep in mind that he had a good read on the data by the time of this interview.

1- “We’ve seen improvements in motor function, and other sensory types of things…reports of improvements in smell, improved balance…”

These are probably the most important of his quotes for obvious reasons. In 90 days, patients are showing improvements when they should be getting slightly worse. N is small, yes, but we should pay attention to what Gene is saying. He flew down to Australia to meet with the patients and the clinicians (more below).

2- “We think we have that.”

Gene said: “We have to look at…what happens inside these patients so that we can tie to why they may have improved, and WE THINK WE HAVE THAT, we think we’re gonna have that. And we’ll have it by the end of the year.”

That line speaks volumes. I believe they already had the data — they’re interpreting it. Parkinson’s has never been slowed or reversed before, so these biomarkers might actually represent new discoveries in neurology. Gene’s tone wasn’t speculative, IMO — it showed confidence.

3- “I think we have the first disease-modifying drug for Parkinson’s.”

You don’t casually say that on record unless you’ve seen something very convincing. The way he said it — calm, grounded, and matter-of-fact — tells me he’s very confident in what the data shows.

4- “This stock is a buy at $10, it’s a buy at $15, it’s a buy at $20…Over the next two months… that should be the switch.”

He’s almost laughing at how undervalued the stock price is at this level. And he should know-- for years, he was a senior analyst for multiple investment banks in the biotech and life sciences sector.

This is also Gene giving a not-so-subtle hint at timing and impact: data in hand, interpretation ongoing, and release coming in December (imminent). He’s mentioned before that it will be a switch, not a dial, referring to share price. He knows what’s coming — and he’s confident enough to predict a “switch” moment, meaning the share price will explode overnight.

For context, comparable sized biotechs with far less exciting data — like FULC and ANVS recently— went up over 70% overnight on their readouts. The asymmetry here is far greater. Also look at CAPR (6x overnight), PRAX (up 5X in a very short window), CADL (4x overnight in 2024), CGEN (3x overnight). The list goes on. Some of these are phase 3, but the point is that huge overnight gains happen often. And even though GT-02287 is only phase 2-ready, the historic potential here cannot be ignored.

5- “Patients were lamenting coming off the drug…”

Gene said when he visited with the patients in Australia, they didn’t want to stop taking GT-02287. Clinicians felt the same — so Gain set up a 9-month extension. That’s not normal for early Parkinson’s trials. When both patients and clinicians are begging to stay on a study drug, you know something real is happening. It’s also a win-win: more long-term data for Gain, and continued benefit for participants.

In response to being asked to come back after the data (and with a knowing glint in is eye): “Oh, we’re gonna have much to talk about. It’s gonna be great.”

Not sure how he can more clearly communicate that he is confident in what they have without risking regulatory violation.

The bigger picture: consistent wins at every step

Zooming out, every milestone so far — preclinical, Phase 1a, and interim 1b — has been a home run. No safety issues, target engagement proven, mechanistic validation, functional improvement shown.

Even the preclinical results (example linked in comments) were unlike anything seen before: restoring mitochondrial and lysosomal function, normalizing biomarkers, and improving motor and cognitive outcomes. The consistency across every stage has been unprecedented.

Gene’s conservative nature makes his recent tone all the more meaningful — he’s never overpromised. And yet, now he’s openly saying, “I think we have the first disease-modifying drug for Parkinson’s.”

This Isn't A Binary Event!

The upcoming phase 1b data readout isn’t pass/fail — safety is already confirmed. The only question left is how efficacious it is. We’ve already seen encouraging UPDRS improvements, and reports of smell and balance returning.

Even if the data isn’t as clean as bulls hope, the GBA1 subgroup alone (the mutation this drug was designed for) could justify a buyout at several times the current ~$250M market cap. There were three GBA1 patients in the trial, and we already saw the UPDRS scores for two of them, and they were great. The scores from one of them showed huge improvements. The drug working for the GBA1 group is my worst-case scenario at this point. There are about 100k GBA1 cases in the U.S. alone, and growing. To me, this is why the drug has been de-risked. Huge upside, little-to-no downside, IMO.

Realistically, anything showing disease-modifying biomarker trends could send this stock up 100%+ overnight.

And it’s not just 02287. Gain’s pipeline has other “Magellan” molecules that big pharma could develop immediately. Whoever partners or acquires them won’t be starting from scratch — they’ll be walking into a very promising platform. Gene said in the interview that some of their back-up compounds are even more potent in some cases. These include Alzheimer’s, Dementia with Lewy Bodies, Gaucher’s, Cancer, and metabolic diseases.

The timing

We’re now inside the likely data window — the next 6 days. Gain has had the data for weeks, Gene’s confidence has never been higher, and official company statements are telling. The company has likely been lining up interviews, media, and potential partnership/acquisition conversations.

When the switch flips, it will happen fast. And this will happen soon, IMO.

TL;DR

- Gene Mack is not a hype CEO — yet he’s publicly telegraphing that they have the supportive data. “I think we have the first disease-modifying drug for Parkinson’s.”

- Every step so far has been consistent and positive — no red flags anywhere.

- The company already has safety and mechanistic proof; the upcoming biomarker data could confirm true disease modification.

- $GANX is sitting at a ~ $250M market cap, with upside potential in the billions.

- Data readout expected within days.

This might be one of the most asymmetric setups we’ll see in biotech this decade.

@kkernttb @RealAvidTrader @BiotechStockRsr
@odibro @yaireinhorn @thebiotechforum
@BiopharmIQ @BPharmCatalyst @SupNovaTrading @StocksPursuit @Microcapreturns @dixielee1969 @fundmyfund
@makedatbread88 @SheffStation @bwsm12702 @TopStockAlerts1

[MWB74 @MWB741]

Inflammation: The Common Denominator in Parkinson's Disease ($GANX)

I made a short post the other day (https://t.co/xnfN0F4cIm) about the mitochondria-first model of Parkinson’s as an alternate intra-cellular explanation to the more established lysosome-first view. My take is that those are two of the three key players in PD’s self-amplifying doom loop. The third is inflammation. Parkinson’s is not believed to be one disease, but many: some cases start inside(cell)-out (e.g., GBA1 L444P, PRKN), others outside-in (chronic systemic inflammation, gut dysbiosis, or a leaky BBB). Whatever the trigger, once these three start interacting, the neuron begins circling the drain.

Here’s the loop: inflammatory cytokines and ROS/RNS push mitochondria into energy failure (Complex I, membrane potential, ATP). Energy collapse impairs lysosomal clearance and mitophagy, letting misfolded proteins and damaged organelles accumulate. Those spills release DAMPs that further activate microglia—more inflammation, more oxidative stress, and the doom loop accelerates.

Here’s an article about the cycle of inflammation in Parkinson’s which also features Joanne Taylor, SVP of research for Gain Therapeutics ($GANX): https://t.co/2CsDhJ3ASf

GT-02287 intervenes via Gcase at key points in this loop inside the neuron. It’s a true allosteric modulator of GCase that stabilizes the enzyme’s intermediate folding state. This is key and makes it stand out from the few other Gcase activators/stabilizers that are being tested because it restores proper trafficking and activity in both lysosomes and mitochondria. In lysosomes, that means better autophagic function and less α-syn/substrate buildup. In mitochondria, that means improved Complex I activity, healthier membrane potential, and fewer ROS. Fix the energy center and the waste-disposal system, and the inflammatory alarm quiets: fewer DAMPs, less microglial activation, and a de-escalation of inflammatory signaling.

This isn’t just theoretical. Company posters have shown:

• Phase 1a (HV): target engagement with surprising GCase activity increases and CNS exposure.

• Preclinical: restoration of Complex I/mitochondrial function, improved mitophagy, reduced α-syn pathology, and lower inflammatory readouts.

• Neuroscience (2024/2025) posters: mitochondrial and lysosomal rescue precede downstream α-syn improvements—exactly what you’d expect if you’re breaking the loop at its sources.

• Phase 1b interim: clean safety/tolerability and directional improvements on UPDRS over ~90 days (but not at 30 days, largely eliminating placebo-effect), aligning with a disease-modifying trajectory, not just symptomatic like the other drugs currently available. And I speculate that reports of patients experiencing improved sense of smell will at some point become the defining signals that the drug works.

Relevant posters can be found here: https://t.co/bNkE6PxBJ5

By the way, check out this part of an interview with CEO Gene Mack by @LouBasenese which was recorded after they had finished the phase 1a with healthy volunteers (but I believe before starting the phase 1b—the date stamp on the video is not correct). A listener asked him about inflammatory markers with the healthy volunteers in the study. Watch/listen to his response: https://t.co/YJmiOGB6cW

Bottom line is that whether PD begins in the gut/vasculature (outside-in) or with genetics inside the neuron (inside-out), inflammation is the universal amplifier. By repairing mitochondrial energy and lysosomal clearance, GT-02287 interrupts this doom loop and gives neurons room to recover—turning a downward spiral into a course-correction.

Data likely out in the next two weeks.

@kkernttb @RealAvidTrader @BiotechStockRsr @odibro @yaireinhorn @thebiotechforum @BiopharmIQ @BPharmCatalyst @SupNovaTrading @StocksPursuit @Microcapreturns @dixielee1969 @fundmyfund @makedatbread88 @SheffStation @bwsm12702 @TopStockAlerts1

[Roderick Watson @bwsm12702]

$GANX’s boost comes from GT-02287’s clinical success, strong analyst backing, and upcoming data. With a 62% gain this year, the stock may reach $6-$8 soon. Longer term, Phase 2 and platform growth will raise its value. 📈📷🔥

[MWB74 @MWB741]

Gain Therapeutics ($GANX) — quietly emerging as one of the most compelling Parkinson’s stories in biotech.

The set-up that Gain Therapeutics has right now is extremely rare, IMO. Largely de-risked, and is only a week or two away from releasing what is likely some of the most compelling evidence of a first, source-targeting, disease-modifying treatment in Parkinson's history, making it a prime target for partnership or acquisition.

- Preclinical: GT-02287 restored GCase activity, reversed lysosomal + mitochondrial dysfunction, and rescued motor / cognitive function in multiple PD models.

- Phase 1a: surprisingly strong — clean safety, clear CNS penetration, and a ~53 % increase in CSF GCase activity.

- Phase 1b interim: meaningful 90-day UPDRS II/III improvements with no safety issues — consistent with disease modification, not symptomatic relief.

- Human signal: patient reports of regained sense of smell — if more than just a couple, this would be an unprecedented indicator of neuronal repair.

- Scientific backdrop: recent studies highlight mitochondrial stress as a trigger for PD and lysosomal-gene variants (links in comments) as weak points in neuronal resilience — pathways GT-02287 directly stabilizes through its broad upstream mechanism.

- Preclinical evidence supports that GT-02287 should help in Alzheimer's, LBD, and Gaucher’s for the same reason it appears to be effective in Parkinson’s.

- Next catalyst: 90-day biomarker data expected very soon. Past data and company statements point to a high likelihood that they support the functional gains already observed.

- Strategic setup: first true disease-modifying small molecule for at least GBA1 IMO — and increasingly, idiopathic — Parkinson’s disease. Multiple pharmas are already in discussions as biotech M&A momentum accelerates.

If biomarkers validate the story, $GANX could become the next billion-dollar Parkinson’s acquisition.

BTW, this might be the dip that many people have been waiting for-- really great price right now IMO.

@LouBasenese @kkernttb @RealAvidTrader @BiotechStockRsr @odibro @yaireinhorn @thebiotechforum @BiopharmIQ @BPharmCatalyst @SupNovaTrading @StocksPursuit @Microcapreturns @dixielee1969 @fundmyfund @makedatbread88 @SheffStation

[MWB74 @MWB741]

Link to SA article: https://t.co/Sl4e6C8xwC

[MWB74 @MWB741]

My Swan Song: How 20 Years of Grinding Led Me to $GANX

I’ve spent the last 20 years building two small businesses. Most of my extra cash went right back into them — payroll, equipment, expansion, the next fire to put out. I dabbled in the market over the years, but I made every mistake in the book. I chased hype. I diamond-handed garbage. I watched “the next big thing” collapse on multiple occasions.

Every painful loss taught me something: how to separate noise from value. How to see through hype, and how rare real innovation actually is. I changed my approach a few years back with this new insight. I haven’t been right every time, but my batting average went way up. Most of the gains that I’ve had over the past couple of years were because I recognized the value first in bitcoin and bitcoin-related stocks, and then AI data centers. $IREN, $NBIS, $WULF to name a few. I sold the majority of my positions a couple of months back, but have since increased my positions again a fair amount after the sell-offs. I have a few different accounts, and they average about 300% gains over the past year, and over 1000% percent over the past three years. I only say this to illustrate that I’ve learned a lot (much from my mistakes).

I wouldn’t consider myself rich—and I’m totally fine with that-- but I’m doing very well. I don’t want to spend the rest of my life glued to screens and SEC filings. I’ve got a business to run and a life to live. But before I step back from active investing, I wanted to make one last move where the research, data, and science actually mean something.

$GANX – Gain Therapeutics, and it is by far the biggest position that I've ever held.

They’ve developed a molecule called GT-02287, which looks to be the first truly disease-modifying drug for Parkinson’s disease. Not my first rodeo in biotech, but by far the most exciting and promising.

GT-02287 looks like it’s not just slowing symptoms, but actually reversing the underlying pathology. And most of the world doesn’t yet know about it. Multiple catalysts coming up in the next few weeks, and is very likely a target of acquisition or partnership in the next couple of months. We know they are currently in discussions with multiple large pharmas. And plenty of cash to get there. A few basics:

- It restores lysosomal and mitochondrial function (the cellular engines that fail in PD).
- It clears alpha-synuclein clumps — the toxic protein driving neuron death.
- And it’s already showing early human results: improved motor scores, better daily function, and reports of patients regaining their sense of smell — something no Parkinson’s drug has ever done.

The company is tiny: ~$175M market cap, and full biomarker data due in the next few weeks. If those biomarkers confirm what the clinical improvements already suggest, this could go from obscure microcap to billion-dollar buyout territory fast.

If you want to understand the potential market value of a drug like this, you can read the article I'll link in the comments. Roche estimates that their much less promising drug, which is currently in clinical trials, will have peak sales of about $3.3 billion. Apply a multiple of 5 to that, and it’s worth $16.5 billion. Apply a multiple of 10 and it’s worth $33 billion.

This is my final high-conviction, highly-researched bet. Not because it’s hype, but because the science lines up.

Whatever happens next — up, down, or sideways — I’m proud of the due diligence I’ve put in. I’ve learned the hard way that real conviction isn’t blind faith in hype — it’s understanding why you believe. Know what you own and do your due diligence, of course.

@BiotechStockRsr @odibro @yaireinhorn @thebiotechforum @BiopharmIQ @SheffStation
@BPharmCatalyst @AviseAnalytics

[joe iniowa @Microcapreturns]

@mvcinvesting $GANX Gain Therapeutics. Their Parkinson’s drug has already returned smell to patients and full biomarker data coming in December. Market cap of $150m and ability to not only stop Progression of Parkinson’s but reverse it? That has to be worth much more than that.

[MWB74 @MWB741]

Gain Therapeutic’s ($GANX) drug GT-02287 improved motor function in Parkinson’s patients in 90 days, with reports of senses of smell and balance being recovered (more data and information on this in the coming weeks)

With the recent buzz around how Parkinson’s Disease can be triggered by damaged mitochondria (link below), there’s a drug that just finished a 90 day trial in Parkinson’s patients that interim data showed improved motor function and reports of recovery of senses of smell and balance in only 90 days, and a good portion of these improvements are likely due to recovery of mitochondrial function, and more likely the combination of healthier lysosome and mitochondria. Importantly, ~95% of PD patients lose their sense of smell, and it is very rare that it returns. No drug has previously been able to do this.

Gain Therapeutics just presented a new scientific poster on GT-02287 at Neuroscience 2025 last week on how it addresses mitochondrial and lysosomal dysfunction, and even though it looks technical, the takeaway is actually very simple: The drug is fixing the root problem inside brain cells — not just masking symptoms. Poster: https://t.co/Xw3ANNCObm

1. It restores energy production in sick Parkinson’s cells

Parkinson’s disease isn’t just about dopamine — the cells themselves run out of energy and start to malfunction.

The poster shows that GT-02287:

- Brings energy production back to normal levels
- Even boosts energy above normal in healthy cells
- Fixes the powerhouses (“mitochondria”) inside the cells

This is something no current Parkinson’s drug can do.

2. It reduces the “toxic stress” that kills neurons

The poster shows strong reductions in:

- Oxidative stress (the “rusting” of neurons)
- Toxic protein buildup
- Cell death

The drug makes dysfunctional neurons healthier again.

3. The results match the clinical improvements we’ve already seen

This poster connects the dots between:

- What happened in the cells, and
- What happened in the patients in Phase 1b

We’ve already seen early signs of Parkinson’s patients improving in 90 days:

- Better motor function
- Better daily movement scores
- Some early reports of smell/balance returning, which is extremely rare in PD

The new poster shows exactly why that’s happening on a cellular level.

4. It looks like true disease modification

Not symptom relief. Not dopamine boost. Not short-term improvement. But actually repairing the underlying machinery inside neurons — something no approved PD drug does today. This is why people and institutions are starting to take notice, not to mention big pharma's with whom Gain is talking right now. The question is when do they make their offers (if they haven’t already), and how much. This likely goes 5X-10X from the current price of ~$3, maybe more, IMO.

If the upcoming biomarker data confirms this (and it likely will based on company statements), this could be one of the most important breakthroughs in Parkinson’s drug development ever.

https://t.co/FmTJNMgzMe

@BPharmCatalyst @AviseAnalytics @TSTbiotech @BioStockAddict @HealthTrader

[MWB74 @MWB741]

$GANX The Holy Grail of Biotechs: Why December Will Likely Redefine the GT-02287 Story

(Summary at bottom)

With tech stocks (especially AI-related) struggling, and biotechs/pharmaceuticals finally waking up after a multi-year Winter, this could be one of the best short-term (and long-term) investments available, and will likely be insolated from whatever is happening in the broader market.

Gain Therapeutics ($GANX) is heading into what is almost surely to be a pivotal December. Multiple catalysts are stacking up — and together, they are likely to move the company from unknown micro-cap to best-in-class, validated disease-modifier in Parkinson’s. Importantly, there are currently no drugs available that address underlying disease progression-- there are only drugs that help with symptoms.

Here’s what’s likely coming and why it matters—note that recent company statements and enthusiasm (for example, watch the Oct. 31st @LouBasenese interview of CEO Gene Mack: https://t.co/H8Sl16E2tL), along with all of the pre-clinical and phase 1 data that we already have, make it highly likely that results will be positive, IMO:

(1) Expanded UPDRS Data (~16 Patients)

Additional MDS-UPDRS motor & daily-function data from the ongoing Phase 1b trial. Interim results already showed meaningful 90-day improvement (+3.8 pts Part III, +0.8 pts Part II) — suggesting disease-modifying benefit rather than symptomatic relief. Expanding to 16 evaluable patients adds statistical weight and strengthens confidence ahead of biomarker data.

Assuming that the scores for the additional 7 patients is consistent with what was seen in the first 9, this further reinforces the improvement trend and further de-risks the transition toward Phase 2. And while biomarkers are important, ultimately it is changes in clinical function that matter.

(2) 90-Day Biomarker Readouts

Comprehensive biomarker measurements across CSF, plasma, blood, and PBMCs, including GCase activity, Glucosylsphingosine (GlcSph) and Glucosylceramide (GlcCer), α-synuclein (aggregated, oligomeric, and total), inflammatory markers, ER and mitochondrial biomarkers. The inclusion of mitochondrial biomarkers shows that Gain is formally tracking the same mitochondrial restoration seen in their latest poster — including improved Complex I activity, reduced ROS, and normalized mitophagy.

Assuming these are positive (and it sounds like they are), this would validate GT-02287’s ability to repair the full lysosomal–ER–mitochondrial axis, addressing the cellular cascade that drives Parkinson’s. This would represent the strongest mechanistic proofs of disease modification ever seen in early-stage PD.

(3) Data Beyond 90 Days (Extension Cohort)

~80% of the patients have elected to participate in the 9-month extension phase. Gain announced the extension on Sept 18, and while dosing start wasn’t specified, the earliest participants likely resumed treatment by late September — meaning that by early December there could be up to two months of additional dosing data for some patients.

There could be some further UPDRS or biomarker assessments for some of these patients, along with varied washout gaps between 90-day completion and re-dosing. Any indication of continued or accelerating improvement would reinforce a sustained disease-modifying effect.

(4) IND Filing for U.S. Phase 2

Submission of the Investigational New Drug (IND) application to the FDA to begin U.S. trials. This marks the official regulatory transition from early proof-of-concept to full clinical development. This is a major credibility milestone — often followed by partnership momentum and valuation re-rating.

(5) The "Holy Grail Signal" — Recovery of Smell (Olfaction)

CEO Gene Mack mentioned “reports” of patients recovering their sense of smell within the 90-day period of dosing with GT-02287. This is potentially giant because sense of smell is one of the earliest and least reversible symptoms in Parkinson’s — and very rarely recovers spontaneously. The Gain executives are very conservative, never spreading hype. They would not throw out something like this if there were not something real going on here.

If multiple patients show measurable or reported improvement, it would serve as proof of disease modification tied directly to neuronal restoration in the olfactory bulb. This might become known as the Holy Grail Signal for Parkinson’s, and would instantly become the headline signal of the entire program — the “holy grail” indicator of functional reversal rather than just stabilization. Even a single line in a press release (e.g., “multiple patients reported improvement in smell”) could reframe GT-02287 as the first Parkinson’s drug with clear evidence of recovery of neuronal function.

(6) Partnership with or acquisition by a top pharmaceutical

The company has made it clear that they’ve been in discussions with multiple companies, including large ones. These companies have been waiting for the 90-day data, and now both Gain and the large pharma suitors have the data. I'd imagine that discussions are almost certainly heating up, and the questions are: (1) how much is the highest bidder willing to pay for what looks to be the first truly disease-modifying treatment for Parkinson’s disease?, (2) how quickly will they make a move?, and (3) will it be a full acquisition of the company or some sort of partnership or licensing? My guess is that there will be an partnership or acquisition announcement in Q1, but I would not be surprised to see this happen in late December. Either way, I think it happens before any need for a cash raise.

$1 billion would be a steal, IMO. That would be close to 10X from here (Gain's MC is ~$100m).

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If these align, December could mark GANX’s transformation from unknown to validated — with clinical, mechanistic, and regulatory proof converging at once.

Here’s the TL/DR:

- UPDRS improvements already demonstrated, and we likely see further reinforcement with the other 7 patients
- Biomarkers are likely to validate lysosomal + mitochondrial restoration, IMO
- Extension data may hint at long-term durability
- Olfaction recovery would be a “holy grail” functional signal
- IND filing opens the door to U.S. Phase 2 (and partnership)
- Partnership or acquisition is likely to happen very soon

Share price in the next few weeks: likely much higher

Share price in 3 months: good chance of 5-10X IMO

@BiotechStockRsr @odibro @yaireinhorn @thebiotechforum @BiopharmIQ