TheFranchute

The edge is in the process. The freedom is in the Exit. 🇫🇷
Biotech & tech | Ex-Investment Banker

[TheFranchute @the_franchute]

Runway is the right question. On the cash first: the $90m is the 30 June position. Two quarters later you're closer to $78m, so you're paying around $10m for the pipeline. The free option closed in June, but $10m for this asset is still a very low bar.

Burn ran $18m a quarter in H1, so roughly 4.3 quarters mechanically. Company guides into Q4 2027 and has pushed that out three times this year, which points the right way.

And this is where it gets interesting: the readout lands well before they're forced to raise. Reading out with four quarters of cash is a completely different negotiation from reading out with two. A good interim and the raise happens at $7 instead of $3, which changes everything for the holder.

The ATM is the real thing to watch, more or less $92.7m still available against an $88m cap. But that's exactly why the readout counts twice: it settles the clinical question and sets the price at which the dilution happens.

[TheFranchute @the_franchute]

$ACRV: 52% response in serous vs 22% non-serous. The confidence intervals overlap, so everyone says the separation is unproven.

Overlapping intervals aren't a test of a difference between proportions. Fisher exact: p=0.024.

Full note below. https://t.co/nmzED2djj6

[TheFranchute @the_franchute]

$EVAX

Merck $MRK and Moderna $MRNA just hit on INTerpath-001. First positive Phase 3 ever for an individualized neoantigen therapy. The modality is no longer the question.

The question is target selection. Intismeran encodes up to 34 neoantigens per patient and brute-forces it. That's the hard part of this field, and nobody talks about it because it's not a headline.

$EVAX reports 86% of the neoantigens its AI picked generated potent T-cell responses in EVX-01. Phase 2 advanced melanoma: 75% ORR, 4 complete responses in 16, 92% still responding at two years, tumor reduction in 15 of 16.

Now look at who's on the cap table. MSD's venture arm is Evaxion's largest shareholder at just under 20%. Same Merck that just won the Phase 3. They also licensed EVX-B3 off the platform last year, $7.5m upfront, up to $592m in milestones.

And the physician presenting EVX-01's three-year data at ESMO on Oct 24 is Adnan Khattak, the same investigator who authored the KEYNOTE-942 five-year update for intismeran.

Three-year data drops in Madrid in two months. Includes EVX-01 as monotherapy, no anti-PD-1. That's the readout nobody is positioned for.

Microcap. ~$18m cash last reported, runway into H2 2027. Trading like the platform is worth nothing.

n=16, single arm, advanced setting not adjuvant. I'm not pretending it's a Phase 3. But Merck already told you what it thinks of the platform with its own balance sheet.

Long $EVAX.

[TheFranchute @the_franchute]

$ALKAL
Kalray just published its H1 2026 revenue after market close. This is what a thesis confirmation looks like.

1. Revenue from the semiconductor business hit €8.5M in H1 2026, up 58% on a comparable basis versus H1 2025. The guidance was double-digit growth. We are running well ahead of it. More importantly, EBITDA is expected to be significantly positive in the first half alone, not just for the full year as originally guided. Eighteen months ago this company was burning €5M of cash per quarter. The model has fundamentally changed.

2. The contract that was announced in April as "in the process of being finalized" with a major AI and HPC infrastructure player has been signed. €2.2M was already recognized in H1 revenue, representing roughly 26% of total semiconductor revenue for the period. The client has acquired a non-exclusive license to integrate Kalray's MPPA technology into its next generation of high-performance chips. That is not a pilot. That is a design win with a tier-one industrial player, generating real revenue on day one.

3- The Openchip angle delivered an unexpected twist that actually strengthens the thesis rather than weakening it. Openchip has announced its intention to exercise its 2.88 million BSAs before July 31, which will give it approximately 15% of Kalray's diluted capital. The dilution is real at -15.75% for existing shareholders. But the context matters entirely. Openchip has simultaneously announced that the Spanish government intends to invest up to €116M into its capital, subject to completion of the complementary private financing currently underway. A financially distressed opportunist capturing value at a discount is one story. A heavily capitalized strategic partner anchoring a formal equity stake in its core technology supplier is a completely different one. When your largest client becomes a 15% shareholder backed by sovereign capital, the probability of contract renewal, pipeline expansion, and long-term collaboration structurally increases.

4. The Openchip partnership extension for at least 12 additional months through July 2027 was also confirmed, consistent with the April communiqué.

Full year 2026 guidance maintained: double-digit revenue growth and further EBITDA improvement. With H1 already at +58%, the trajectory is clear.
Three months ago the market was pricing Kalray at roughly 12x revenues with zero credit for the royalty model and zero credit for the second contract. Both are now confirmed. The re-rating has started. It is not finished.

Position declared. Not investment advice.

[TheFranchute @the_franchute]

That antibody threshold point is the most important thing said in this thread and I should have raised it myself. You're right that comparing programs dosing at 1:400 versus 1:20 is genuinely problematic. Elevidys was treating patients that Solid and REGENXBIO were screening out. Some of the hepatic signal probably reflects that broader inclusion rather than the capsid alone. Fair correction and I'll carry it forward.
On functional convergence, I think you're probably right for the standard endpoints. TTR and NSAA in ambulatory kids with preserved baseline function at 12 to 18 months may just not separate programs that are all achieving robust fiber coverage. The place I'd still expect nNOS to matter is on sustained exercise rather than short bursts/ post-exertional recovery, fatigue curves. Brenman and Sander showed the ischemia happens specifically during contraction, not at rest, and a 10-meter walk doesn't stress that mechanism the way prolonged activity does. My honest suspicion is that signal won't show up in Phase 3 and will only emerge in real-world data, if at all.
The Endeavor point is well taken too. 1,500 patients will teach things no trial can, and if sirolimus protocols substantially clean up Elevidys in ambulatory patients the competitive gap narrows regardless of what SGT-003 shows.
So yes, more cautious on the magnitude of the differentiation case after this conversation. But two things haven't changed. Forty-six kids without a liver event on a capsid engineered to avoid one is still a real signal, even partially confounded. And Perceptive, Bain and RA Capital put $240M in on an oversubscribed basis in March after seeing the 40-patient data. They had access to exactly the questions you're raising. They wrote the checks anyway.
But the functional data and the FDA decision are still what this comes down to...

[TheFranchute @the_franchute]

$SLDB

Elevidys killed two patients from acute liver failure. The FDA pulled its non-ambulatory label. Sarepta $SRPT lost 85%.

Solid Biosciences saw this coming in 2022 and built a different capsid.

AAV-SLB101 was engineered to target muscle integrin receptors rather than the liver receptors that AAVrh74 hits. The consequence across 46 kids: zero drug-induced liver injury. Zero myocarditis. Zero serious adverse events of any kind. Steroid-only immunosuppression. The safety record isn't luck : it's the molecular consequence of a design decision made before the crisis existed.

The efficacy picture is cleaner than any competitor. 110% of normal dystrophin at Day 90. Highest expression ever published in DMD gene therapy. Elevidys came in lower. REGENXBIO's RGX-202 was described by BMO as "in line with Elevidys, not superior." Then there's something no competitor has touched: the nNOS domains that restore blood flow to working muscle during exercise. Every DMD gene therapy addresses the structural problem. SGT-003 is the only one addressing the vascular problem simultaneously.

In March, Perceptive, Bain Capital Life Sciences, RA Capital, Deep Track, and Janus Henderson put $240M in at $5.61. Oversubscribed. They had 40-patient data. They wrote the check anyway. They're up 82% and haven't sold.

Now the Friedreich's ataxia program. First patient in FALCON: 40% dentate nucleus coverage. The company expected 10-20%. H.C. Wainwright raised to $25 on two patients. The market started pricing a second franchise for the first time.

FDA accelerated approval decision is weeks away. The precedent is Elevidys, approved in 2023 on microdystrophin expression data that was lower than what SGT-003 is producing on a safer capsid in more patients.

14 analysts. All Buy. Median target $17. Cash $ 381M. Clean structure.

The answer hasn't come yet.
NFA

https://t.co/8qf4DmI7tT

[TheFranchute @the_franchute]

https://t.co/prsjeQ8hPZ

[TheFranchute @the_franchute]

That's a fair challenge and one worth sitting with rather than explaining away.

Honestly, I don't know, and I think anyone claiming certainty here is overreaching. Two readings are possible. In my view, Solid may be holding functional data for the FDA accelerated approval submission rather than releasing it piecemeal — this is a documented pattern. Sarepta withheld PROMOVI functional data while pursuing Elevidys accelerated approval on microdystrophin expression, and those data only surfaced after approval. When your surrogate endpoint is doing the regulatory work, releasing ambiguous functional data early creates a public narrative problem before the FDA has seen the full package. The other reading is that the functional data isn't as clean as the biomarker story and they're being selective. I can't rule that out either.

What I'd add is that biomarker correction precedes functional improvement in a slowly progressive disease, sometimes by a long time. Kids at 7 to 11 with preserved baseline function may not show dramatic timed-test improvements early even with robust molecular correction at the fiber level.

But you're right. Superior biomarkers don't equal superior outcomes until the functional data confirms it. That story isn't finished yet...

[TheFranchute @the_franchute]

Fair points from both of you, and I'd rather engage them seriously than move on.

$SRPT took the first real risk here. Getting Elevidys approved meant running a trial with no gene therapy precedent in this disease, convincing a divided FDA committee, and building manufacturing from nothing. That's a genuine contribution and I don't think anyone serious is dismissing it.

Where I'd push back is on the idea that they learned from the liver toxicity and adapted. The response was to restrict the label and add a black box warning, which I think was the right regulatory call. But AAVrh74 still hits the liver the same way it always did. What changed is who's allowed to receive the drug, not how the capsid behaves.

What I find compelling about SGT-003 is that the liver-sparing design predates the Elevidys deaths by three years. Solid made that engineering decision in 2022 because the hepatic tropism risk was always in the biology of systemic AAV delivery. Zero liver injury across 46 kids isn't them reacting to what happened at Sarepta. It's the outcome of a call they made before anyone got hurt.

On the "nothing has done better" point, I'd say that's true if you're looking at approved products. But you find the signals before the approvals, not after them. The published data shows 110% of normal dystrophin on a capsid that hasn't produced a single liver event in 46 patients. Will that hold at Phase 3 scale? That's always the question at this stage. It's also exactly what the FDA conversation happening right now is there to answer

[TheFranchute @the_franchute]

$EVAX $MRK

People focus on EVX-01 and the ESMO catalyst in September. Fair enough, that is the main event.
But Evaxion just announced a poster presentation for EVX-V1, their cytomegalovirus vaccine candidate, at the International Herpesvirus Workshop in Montreal on July 12.

CMV infects 60 to 70% of adults in developed countries and close to 100% in developing economies. One in 200 babies is born with congenital CMV infection. Despite decades of research, no approved CMV vaccine exists. The market was valued at $474 million in 2023 and is growing at 6.6% annually.

What makes this interesting is not the CMV program specifically. It is what it says about PIONEER. The same AI platform that selected the targets behind EVX-01's 75% response rate in metastatic melanoma is now designing antigens for a completely different pathogen using completely different biology. And it is finding novel CMV antigens that are entirely new to the vaccine research field, ones that inhibit viral infection and reduce cell-to-cell spread.

That is three independent disease areas now. Melanoma. AML. Cytomegalovirus. One platform. Same underlying engine.
Merck paid $7.5 million cash for a preclinical license on an infectious disease program from this same platform. They know what they are looking at.
Enterprise value today is under $10 million.
ESMO is in September.

Not financial advice.

https://t.co/3JW89OZ45u

[TheFranchute @the_franchute]

$TSHA

One week ago the stock was at $6.95. Then came a $200 million offering at $6.00. Every biotech investor knows what that does to a stock in the short term.

The offering closed Thursday. 32.5 million new shares at $6.00. Goldman Sachs, Jefferies, Piper Sandler and Cantor on the book. The institutions that got allocations are already up 10% because the stock is now at $6.65.

That is not a coincidence. It means the book was oversubscribed. More institutional demand than shares available at $6.00. The ones who did not get their full allocation are now buying on the open market above the offering price. That is what a well received deal looks like.

The dilution picture is also cleaner than people expected. Only 833,000 pre-funded warrants were issued alongside the 32.5 million shares. This was essentially a clean equity raise with minimal warrant overhang. Total fully diluted shares come to around 400 million post offering.

Cash is now roughly $470 million. Nearly three years of runway at current burn. No ATM hanging over the stock. No emergency raise risk before the BLA. An acquirer looking at Taysha today sees a program with a 100% response rate, deepening gains at 12 months, 17 patients in the pivotal trial against a 33% success threshold, a manufacturing process already FDA aligned, and enough cash to reach approval without touching markets again.

BMO raised to $14. Citizens raised to $11. BofA raised to $10. Jefferies is still Buy. Median analyst target across the coverage is $12.

Pivotal 6 month interim in December. The offering is done. The overhang is gone. The only thing left is the biology.

Still holding. Not financial advice.

[TheFranchute @the_franchute]

https://t.co/prsjeQ8hPZ

[TheFranchute @the_franchute]

People keep calling the $SLS rally a meme stock move. The chronology tells a different story.

Q1 2026, stock still in single digits: State Street triples their position, adding 6.2 million shares for $26M. BlackRock adds 4.7 million shares, up 61.8%. Jones Financial nearly quadruples. 120 institutions build positions. Nobody talks about it.

May 12: SELLAS confirms 78 of 80 events in REGAL. CEO says the readout "will be an important milestone." Stock gaps 25% that day alone.

May 20: CEO at Stifel Oncology Forum says survival is "unexpectedly long," some patients still on treatment more than three years after enrollment, and there is "a very good chance" GPS beats Phase 2. Stock up 7% premarket.

Late May: Jim Cramer gives SLS "two thumbs up" on Mad Money. CEO participates in TD Cowen Oncology Innovation Summit, described as the final major conference before the REGAL readout.

June 11 to June 24: Stock grinds from $7 to $10 on sustained volume. Institutions continuing to accumulate on the back of three consecutive bullish CEO appearances.

June 25: 8-K on executive change of control. Volume nearly doubles to 15 million shares versus the 65-day average of 7.6 million. Short interest confirmed at 63.36 million shares, 32.65% of float, days to cover 9.3.

June 26 to 29: Plus 14%, plus 18%, plus 16%. WallStreetBets picks up the name. Not the cause. The accelerant on a move already built on institutional accumulation and serial CEO signaling.

Since then the stock has pulled back about 15% from the $15.88 peak and sits around $13. Normal consolidation after a parabolic extension. The clinical catalyst has not changed. The 80th event has not been announced. The short base has not moved.

This is not a meme stock. It is an institutional accumulation story that retail amplified. The data is still coming.

[TheFranchute @the_franchute]

What I know for sure is short interest climbing from 25% of float in April to 33% by June 15, building steadily through the entire rally. Delta hedging doesn't accumulate like that, it moves daily with options flow. Tudor and Verition are confirmed directional. Susquehanna shows up on the long side in 13F filings which suggests market making but their short book is private.
Curious what's driving your read on the retail vs institutional split though. Are you seeing something in the options flow or borrow rates that points to hedging?

[TheFranchute @the_franchute]

$SLS short interest at 33% of float as of June 15, before the stock added another 50% on top of that move.

Think about what that means. The shorts who built that position are now sitting on massive losses on a stock that is 7 days green, with executive change of control agreements just restructured and a Phase 3 readout one event away. They did not cover into the rally. They added 33% short float going into a binary where the clinical data points to 75% POS.

The squeeze was always part of this thesis. The shorts just handed it to us on a plate. $SLS at $11.80 right now!

https://t.co/xA0tqEATH2

[TheFranchute @the_franchute]

https://t.co/f448DX00Q0

[TheFranchute @the_franchute]

Most $SLS holders are focused on REGAL, and for good reason. The clinical results are exceptional. But the platform behind GPS is what makes this a generational acquisition target, and almost nobody is talking about it.

The clinical results we all already know :
AML CR1: median OS 31 months versus 14 to 16 months historical. Two patients still alive at 54 and 78 months in a population where long-term survival essentially does not exist.
AML CR2: median OS 16.3 months versus 5.4 months historical, p equal to 0.0175. These numbers are not incremental improvements. They suggest a fundamentally different biological outcome.

The mechanism behind those results :
GPS activates both CD4+ helper T-cells and CD8+ cytotoxic T-cells simultaneously against WT1. That dual engagement is why the immune response rate hits 80% and why those responses appear durable years out. This is genuine T-cell memory, which means the body keeps hunting WT1-expressing cells long after the last injection. And because WT1 is overexpressed in more than 20 tumor types while remaining essentially silent in healthy adult tissue, the mechanism does not stop at AML.

The platform beyond AML :
Mesothelioma: WT1 overexpressed in 95% of cases, Phase 2 data already exists, Fast Track designation from the FDA.
Ovarian cancer: combination with nivolumab at ASCO showed strong immunogenicity in a biomarker-selected population.
Multiple myeloma: preliminary Phase 1/2 data.
The IP on the most immunogenic WT1 peptides is licensed from Memorial Sloan Kettering and protected past 2033. Nobody else is in a late-stage randomized trial on this target.

Potential Acquirers
1. $BMY BMS owns CC-486, approved only in CR1. GPS gives them the complete AML maintenance continuum and a platform they can take into solid tumors.
2. $MRK Merck already ran a GPS combination study with Keytruda. A validated REGAL readout hands them a proven combination backbone across multiple indications past the patent cliff.
3. $ABBV AbbVie built its oncology franchise on venetoclax. SLS009 addresses venetoclax resistance directly, and GPS maintains the remissions SLS009 creates.
4. $AZN AstraZeneca faces the same checkpoint inhibitor combination logic as Merck and the same strategic rationale applies.

A positive REGAL readout does not just approve one drug in one orphan AML indication. It validates a WT1 platform with clinical data across four tumor types, an active Fast Track in mesothelioma, a proven checkpoint inhibitor signal, and IP protection through 2033. The executive Change of Control agreements restructured this week tell you the board has already thought carefully about what comes next.

[TheFranchute @the_franchute]

A cancer with no approved targeted therapy. Standard of care fails 85% of the time and $ACRV just posted 67% confirmed response rate in that setting. Market cap is $69M. They have $105M in cash.

The pipeline is priced at negative value. Here is what the market is missing and why I see it as an opportunity.

RA Capital manages $12 billion. 70+ PhD scientists on staff whose only job is evaluating biotech. Their board observer Derek DiRocco had been sitting in internal management meetings and receiving live Arm 3 trial updates for months before they bought 3.89 million shares at $1.80. Not in a PIPE. In the open market. At market price. With the preliminary data already in hand.
I bought today at $1.58. Below their entry.

The clinical picture. ACR-368 produced 67% confirmed ORR in biomarker-positive serous endometrial cancer. The standard of care here is below 15%. Immunotherapy fails because these tumors are microsatellite stable. PARPs fail because BRCA mutations are uncommon. Elacestrant, the best approved oral SERD in an adjacent setting, produces 17% ORR. Nothing is approved beyond first line in this population. The FDA granted Breakthrough Device Designation to the selection test itself, not the drug. That distinction matters. The agency is saying the diagnostic tool alone is worth accelerating.

Two days ago a new board member accepted options at $1.52, below current price, multi-year vesting. Director Baum holds options expiring 2036. These are decade-long commitments.

RA Capital 28.8%. Perceptive 17%. 46% of the float between two of the best healthcare funds alive.

Arm 3 initial data in weeks.

Full breakdown below.

#ACRV #biotech #oncology

https://t.co/4BoJFGvOOI

[TheFranchute @the_franchute]

$EVAX
The stock is at $3.15. Enterprise value is $7.9 million. The cash on the balance sheet alone is $18.4 million. So the market is pricing the entire pipeline at negative ten million dollars.

Here is what that pipeline actually looks like, and why I see it like a real opportunity.

EVX-01 delivered a 75% objective response rate in Phase 2 metastatic melanoma. For context, pembrolizumab alone in the same setting delivers around 33%. The gap is 42 percentage points. But the number that really matters is the deepening rate. 54% of evaluable patients saw their response improve between month 12 and month 24. Partial responses converting to complete. Stable disease converting to partial. That pattern is statistically incompatible with a checkpoint inhibitor effect alone because pembrolizumab works the same way at month 6 as it does at month 18. It does not get better over time. The vaccine does. Three year monotherapy data is coming at ESMO in September. That readout will show whether the T cells created by the vaccine persist and keep working without pembrolizumab at all.

$MRNA Moderna is running a similar concept in adjuvant melanoma and they are valued at 43 billion dollars for it. Evaxion is valued at 26 million.

The platform behind this is called PIONEER. It selects vaccine targets at 86% accuracy meaning 86 out of every 100 antigens it identifies actually trigger a measurable immune response in patients. Competing platforms convert 50 to 65%. That gap compounds across 7 to 10 targets per patient. You end up with a meaningfully broader and more durable immune response and the data reflects exactly that. The Prix Galien UK committee of 12 scientific leaders, evaluating clinical application and real world impact, just gave PIONEER their Best Digital Health Solution award. That is the second Galien recognition in under a year. These committees look at clinical outcomes, not pitch decks.

EVX-04 is the off the shelf version of the same platform applied to acute myeloid leukemia. The challenge with off the shelf cancer vaccines is that human immune systems vary enormously across patients. What triggers a response in one person may do nothing in another. The EHA data showed that EVX-04 drives specific immune responses across different human immune profiles meaning the AI selected targets that work regardless of the patient's immune genetics. It also showed that the immune cells these targets activate actually kill AML tumor cells in functional assays. That is not just immunogenicity. That is efficacy. Clinical trial filing is coming in the second half of this year.

$MRK Merck owns 29% of this company. They paid $7.5 million cash upfront to license EVX-B3 at the preclinical stage with up to $592 million in milestones. They then reinvested at $2.71 during the NASDAQ delisting scare with full access to non public data on everything in the pipeline. They have not sold a share.

ESMO is in September. Eleven weeks.

Still holding. Not financial advice.

#Biotech #CancerVaccine #ESMO2026

[TheFranchute @the_franchute]

$SLS touching $10 today after SELLAS quietly updated Change of Control agreements for CEO, CFO and CDO.
This matters.

Change of Control clauses define executive compensation in an acquisition scenario. You don't renegotiate those terms two events before a Phase 3 OS readout unless the board is actively preparing for what comes after a positive result.

13 employees. No sales force. An orphan AML asset with no approved competitor in CR2. The exit was always M&A. The contract update tells you the timeline is now.

Full REGAL breakdown: https://t.co/xA0tqEATH2
$SLS

[TheFranchute @the_franchute]

https://t.co/f448DX00Q0

[TheFranchute @the_franchute]

What still holds is the delivery route difference. NGNE is ICV, neurosurgical access. TSHA-102 is IT lumbar, standard 20min procedure at any neurology center. Taysha's own market research documents explicit clinician and caregiver preference for IT-L. That gap matters commercially regardless of which BTD came first.
And then there's the Nov 2024 fatality at the high dose via ICV. That event is why NGNE is capped at the lower dose only. TSHA-102 has zero SAEs across 29 patients at both dose levels. Different routes, different doses, so not a clean comparison, but the safety record speaks for itself.
The deepening data is the part that genuinely excites me on TSHA and I haven't seen anything from NGNE's interim that matches the 94% figure for patients past 12 months. Curious what their mid-2026 Phase 1/2 readout shows on that front

[TheFranchute @the_franchute]

$TSHA
Two days after delivering exceptional clinical data, Taysha just launched a 200 million dollar underwritten offering. Goldman Sachs, Jefferies, Piper Sandler and Cantor on the book. Stock dropped about 10% in after hours, which is the standard mechanics of an overnight deal pricing.

Here's the concrete dilution picture :

- There are now roughly 428 million fully diluted shares outstanding, including around 91 million pre-funded warrants at a 0.001 dollar exercise price.

- About 79 million of those warrants come from the May 2025 offering at 2.75 and the June 2024 offering at 2.25. These holders entered between 100 and 170% below today's price and have not sold once despite the stock touching its 52-week high of 7.30. They are not sellers at $6. They are waiting for what comes after December.

- The new holders from tonight's deal will enter via Goldman Sachs. They ran a full diligence process on a program with a 100% response rate, accelerating deepening and a 33% pivotal threshold. They are not here for a 10% trade either.

- What tonight actually does is remove all financing uncertainty through the BLA approval and potential acquisition. Cash goes to roughly 470 million dollars. Nearly three years of runway at current burn. No more ATM overhang. No more emergency raise risk. An acquirer looking at Taysha now sees a fully funded program with exceptional clinical data and a manufacturing process already aligned with the FDA.

Pivotal interim in December.

https://t.co/neHX0GD596

[TheFranchute @the_franchute]

$TSHA
Just to remember why I'm so convinced on this one. Let me explain what the data actually says.

Rett syndrome patients aged 6 and above in developmental plateau have essentially zero chance of gaining developmental milestones on their own. That's not my opinion, that's the FDA-validated natural history data that Taysha used to design the trial.

TSHA-102 just delivered a 100% response rate in 12 patients. Every single one gained or regained milestones that the disease had taken from them.

But the number that really matters isn't the response rate. It's the deepening. Milestone gains went up 69% from month 6 to month 12, and 94% for patients past the 12 month mark. The effect isn't plateauing. It's accelerating. That has never been documented before in CNS gene therapy.

Zero serious adverse events across 29 patients dosed. The same capsid used in Zolgensma. The same team that built and sold AveXis for 8.7 billion dollars. The same regulatory playbook, executed by the same people who wrote it the first time.

The only real competitor is Neurogene. Their high dose program had a patient fatality in November 2024 and was permanently discontinued. They continue at the lower dose only, without Breakthrough Therapy Designation, using a neurosurgical delivery route that clinicians and caregivers explicitly say they don't prefer. TSHA-102 has BTD, zero SAEs at any dose, and is delivered via a standard 20 minute lumbar puncture at any neurology center. That gap is not small.

The market is 15,000 to 20,000 patients across the US, EU and UK. No approved treatment exists today for the genetic root cause of this disease. At pricing consistent with other orphan gene therapies, you're looking at peak revenues north of a billion dollars from the US alone. AveXis sold for 8.7 billion before Zolgensma was even approved. The same team is now running the same playbook in a different rare pediatric CNS disease.

The pivotal interim in December needs a 33% response rate to pass. Part A just delivered 100%.