Breaking: Ultragenyx just cratered on a Phase 3 failure
Everyone who shorted made bank
$RARE's drug "apazunersen" missed both primary and secondary goals in a Phase 3 trial for Angelman syndrome, a rare genetic disorder that causes severe developmental delays
It was the company's biggest remaining growth bet
However:
• Trial missed the primary cognition endpoint and the key secondary endpoint
• Second Phase 3 failure for Ultragenyx this year, following a miss back in January
• Company says it's now reviewing operations and cutting expenses
• CEO Emil Kakkis: "We are disappointed by the Aspire result"
Most puts were sitting under $2 yesterday.
• $25 Puts (9/18 exp): $1,000 -> $3,778
• $22.50 Puts (9/18 exp): $1,000 -> $4,588
• $20 Puts (9/18 exp): $1,000 -> $6,294
There's risk obviously, but that's where opportunity shows
• DefenCath's Medicare reimbursement (TDAPA) is transitioning off its temporary add-on pricing
• CorMedix depends heavily on a handful of large dialysis customers
• The stock is still down 43% from its 52-week high
But that's also why the setup is interesting
CorMedix might be one of the most contested plays in small-cap healthcare right now
Sitting on one of the highest short percentages
The company just finished signing all 5 of the top US dialysis providers to DefenCath supply deals, and posted $26,000,000 in net income last quarter
$CRMD is trading at $8.51
Here's the setup 🧵👇
Here's why the short interest matters:
• 77.94M shares outstanding
• Market cap: $663,269,400
• 17,149,908 shares sold short, 22% of shares outstanding
• ~$145,945,717 in short exposure at current prices
That much short exposure sitting on a stock that has every major dialysis operator in the country on contract is normally unseen
Breaking: Novartis crushed Sanofi in a multi-billion dollar showdown and Robinhood calls went vertical
$NVS's remibrutinib is a once-daily pill for relapsing multiple sclerosis and it blocks a protein called BTK that fuels the immune attacks on the brain and spinal cord
In two head-to-head Phase 3 trials against Sanofi's Aubagio, patients on remibrutinib had fewer relapses, fewer new brain lesions, and slower disability progression
Summary:
• Beat Aubagio on annualized relapse rate, the trial's main goal, in both trials
• Also beat on every secondary measure, including MRI lesion counts
• Analysts project $3B to $9B in peak annual sales for this one drug, inside a global MS market worth over $20B a year
Contracts spiked overnight:
• $170 Calls (9/18 exp): $1,000 -> $3,082
• $165 Calls (9/18 exp): $1,000 -> $2,818
• $155 Calls (9/18 exp): $1,000 -> $2,175
$NVS has added $17.7B in market cap today
Breaking: Capricor just got hit with a 9-3 negative FDA panel vote last month on its muscular dystrophy therapy
Yet $CAPR is up nearly 17% today
Here's why:
• The FDA agreed to review an amended application focused on the trial's strongest data, and extended its review by 3 months instead of rejecting it outright
• Cantor Fitzgerald just raised its price target from $3.50 to $28, an 8x jump
• Volume today is running 2.7x above normal
• The new FDA decision date is November 22nd, the next hard catalyst for the stock
Breaking: Moderna's stock has been broken for years, down nearly 90% from its 2021 highs on a shrinking COVID vaccine business
Yet $MRNA is up over 14% today
Here's why:
• Its Phase 3 cancer vaccine trial with Merck just hit both primary endpoints, the first successful late-stage readout ever for an mRNA cancer therapy
• 14% of the float is sold short, and it would take over 10 days of average volume to cover it
• Volume today is running 3.1x above normal as short sellers scramble to cover
• The vaccine targets melanoma recurrence, with expansion into lung and kidney cancer next
Breaking: Generate Biomedicines' 103 million share lock-up expires today after the close, the biggest supply test since its February IPO
Yet the stock is up nearly 10%
Here's why:
• On September 8th, data on its lead respiratory drug, GB-0895, will be presented at a major medical conference
• All 6 analysts covering the stock rate it a Buy, with price targets as high as $30
• 5.7% of the float is sold short, and it would take 15 days of average volume to cover it
• Volume today is running 4.4x above normal for $GENB
$RGEN keeps climbing as the mRNA vaccine race between $MRNA & $MRK heats up
Repligen supplies the bioprocessing equipment behind every mRNA therapy, so it wins as the pipeline grows without needing to own the winning drug
Breaking: Nancy Pelosi just filed up to ~13.5M worth of new stock trades
Including buying up to $12M of Bloom Energy $BE
Major Buys Include:
• Bought 15,000 shares of Bloom Energy $BE
• Bought 10,000 shares of Intel $INTC
She bought new call options:
• Bought up to $5M of $BE leaps
• Bought up to $500K of $INTC leaps
$HIMS keeps climbing as the $LLY & $NVO GLP-1 race heats up
Hims & Hers sells every FDA-approved GLP-1, so it wins as the obesity market grows without needing to own the winning drug
Breaking: Options traders who bet on $TEM overnight are sitting on some of the biggest paydays in the market this morning
A month ago, Tempus AI paid $1.5 billion for Personalis, a gene-sequencing company most people had never heard of and most of Wall Street said they overpaid
Then Moderna and Merck dropped Phase 3 results yesterday. Their personalized mRNA cancer vaccine, built on the exact sequencing tech Personalis owns, which is keeping melanoma from coming back. First time a personalized cancer vaccine has ever won a late-stage trial
The $1.5 billion "overpay" just became the smartest deal Tempus made all year
• TEM is up nearly 33% across two sessions, from $49 to $65+
• Over 20% of the float was sold short heading in, adding a squeeze on top of the news
• Tempus posted its first-ever quarterly profit in July and raised full-year revenue guidance to $1.6 billion
Contracts overnight exploded. 27,799 calls traded across three strikes this morning, most of them were sitting at pennies yesterday
$70 Calls (8/21 exp): $1,000 -> $3,500
$65 Calls (8/21 exp): $1,000 -> $3,772
$61 Calls (8/21 exp): $1,000 -> $2,189
Breaking: $MRNA is surging after Merck & Moderna's mRNA cancer vaccine hit its Phase 3 endpoint
This is Moderna's personalized cancer vaccine. It reads the mutations in a patient's own tumor and builds a custom shot that trains their immune system to attack it. Paired with Merck's Keytruda after surgery, patients stayed cancer free longer than patients who got Keytruda alone. This is the first time an individualized mRNA cancer treatment has ever succeeded in a late stage trial.
• First Phase 3 win ever for an mRNA cancer therapy
• Added $31B in market cap in a single day
• Roughly 15% of the float was short heading into this, some of the move is shorts getting run over
• Merck shares up too, they co-own this drug
Contracts overnight exploded and most of them were sitting at pennies yesterday
$85 Calls (8/21 exp): $1,000 → $6,200,000
$80 Calls (8/21 exp): $1,000 → $2,214,000
$75 Calls (8/21 exp): $1,000 → $594,167
Breaking: Moderna $MRNA is spiking after becoming one of the first companies ever to deliver a positive Phase 3 result for a personalized cancer vaccine
Treatment is built around each patient’s own tumor and when combined with KEYTRUDA it reduced the risk of melanoma returning or spreading
So Lilly isn’t just racing Novo Nordisk and the rest of pharma.
With retatrutide, Lilly is racing something much stranger:
the unauthorized market for its own drug.
And that market got a head start.
Andrew Huberman called retatrutide a potential “trillion dollar drug.”
Now Eli Lilly is suing people who are already selling it.
And the craziest part?
Reta is a 39-amino-acid peptide sitting right on one of the strangest regulatory lines in pharma. FDA has said you need 40-amino-acid to qualify for a patent .
This story is about way more than weight loss.
Thread 🧵👇
That might be the clearest sign yet that obesity drugs have entered a completely new era.
The molecule doesn’t need FDA approval to become culturally famous anymore.
The clinical trial itself can create demand.
And once millions of people know the name of the molecule…
the internet creates supply.