Allogene's Interim Data Hints at Off‑Shelf CAR‑T Breakthrough
This week's Biotech Scorecard: Allogene $ALLO: A preview of its April interim analysis. Very interesting (to me, at least) There just might be a path forward for off-the-shelf CAR-T therapy in B-cell lymphoma. Frontline consolidation. Read all about it: https://t.co/tcPFelUH3C
Analysts Bullish on MAZE, yet Pre‑market Price Drops
$MAZE - Analysts pitching tent poles over the AMKD data, yet stock is down pre market. What's the disconnect?
AVXL Withdraws Alzheimer's Drug From EMA After Rejection
$AVXL pulled its blarcamesine Alzheimer's application from the EMA after regulators there said there was no fking way it was going to approve a drug that does not work.
Patented Chemo Reform Boosted Profits, Not Patient Benefits
Please. This is nonsense. You figured out a way to swap castor oil for albumin making it easier to administer paclitaxel, a standard chemotherapy. Same side effects. Look at the label. The best thing you did, for you, was secure...
Peaky Blinders Fans Will Love This Fitting Tommy Shelby Finale
If you’re a Peaky Blinders fan, you’ll like the movie a lot. A fitting end for Tommy Shelby.
Lilly’s Biz Dev Handles 10 Weekly Deals, Public Offers Stalled
Some interesting comments from Jacob Van Naarden, $LLY head of business development, at STAT's Breakthrough Summit East event yesterday: On deal volume: Jake, himself, is seeing approx 10 deals per week, ie deals that are important enough, or diligenced enough,...
Biotech Godfather Stelios Papadopolous Joins Garde on Podcast
This week's Readout LOUD podcast is now available on all your favorite podcast outlets -- Stelios Papadopolous, the ‘godfather’ of biotech, in conversation with @damiangarde https://t.co/RHh5Qv9OyT via @statnews
FDA’s Post‑Prasad Path: Six Drugmakers, Six Months
This week's Biotech Scorecard newsletter: Six drugmakers, six months: Charting the FDA’s course in the post-Prasad era $REPL Pierre Fabre $ATRA $QURE $CAPR $RGNX https://t.co/sGa7G6YTDn
FDA Lets Sarepta Push Failed Drugs, Blocks UniQure
Let's look at how the FDA is treating $SRPT and $QURE differently. The FDA grants accelerated approval to Amondys and Vyondys to treat Duchenne muscular dystrophy. Sarepta conducts a post-marketing confirmatory study that fails to show a benefit for either...
Accountability Reporting Secures Prison for Fraudulent Drug CEO
Playing a role in the securities fraud conviction of a drug company CEO is rewarding, especially when the CEO, CytoDyn's Nader Pourhassan, endangered the lives of patients while he was putting shareholder cash in his pocket. I had lost...
White House Claims Vaccine Rollout Complete; MAHA Pushes Back
White House says it’s ‘done’ with vaccines. MAHA begs to differ https://t.co/atqj3ymMqe via @_daniel_payne @ChelseaCirruzzo
Jefferies Paints WVE Data as Win‑win‑win; Bear Sees Combo Role
Doing some reading ahead of the next wave of $WVE INHBE "body composition" 🙄 data, smirking at the Jefferies analyst set up, which basically frames 6-month data as a win-win-win for the stock price. His "bear case" "Bear case would still...
Pierre Fabre, ATRA to Meet FDA on Ebvallo CRL Soon
Pierre Fabre and $ATRA said FDA meeting to discuss the Ebvallo CRL has been scheduled. (Likely occuring within the next month.)
FDA Cracks Down on Misleading GLP‑1 Telehealth Marketing
The FDA is targeting telehealth marketing of GLP-1 drugs. Who’s prescribing them? Medical groups face ‘tricky’ questions when partners make misleading claims via @KatieMPalmer https://t.co/pf4Fwa7W7r
New Nonprofit Secures $500M to Upgrade Science for AI
New nonprofit launches with at least $500 million to modernize scientific process for AI era https://t.co/yuypno6rVY via @ADeAngelis_bio
BBIO, ALNY Surge on Pfizer Tafamidis Patent Win
$BBIO $ALNY moving higher on apparently good news re: Pfizer's tafamidis patent defense. If you have access to Umer Raffat's research, he has all the details.
FDA Accepts CAPR's Deramiocel Resubmission, Decision Aug 22
$CAPR resubmission of the deramiocel application accepted by the FDA. New decision date: Aug. 22. https://t.co/mWSyyXg8di
Vertex Drug Cuts Kidney Disease Marker in Late-Stage Trial
Vertex $VRTX says its drug successfully reduced marker of kidney disease in late-stage trial https://t.co/TbEkcHVYBB via @Jasonmmast
FDA Leaders Makary and Pazdur Meet Investors in Miami
Re: Prasad, from this morning's @statnews Readout newsletter: FDA Commissioner Marty Makary is in Miami this week speaking to health care investors attending a slew of broker conferences, noted Mizuho health care strategist Jared Holz. That should make for some...
Five Key Lessons From Vinay Prasad’s FDA Tenure
. @matthewherper is so damn reasonable. Smart, too. 5 lessons from Vinay Prasad’s turbulent tenure at the FDA https://t.co/R65vPj5ep5
RLMD's Strong Bladder Cancer Data Hits IBRX Valuation
The $RLMD bladder cancer data (phase 2) reported this morning look very good. Another competitive blow to $IBRX particularly at its bloated valuation. https://t.co/wYThfwIpG8
Vinay Prasad Mocked for Hosting Controversial HHS/FDA Call
Too funny. @emilyakopp (below) outs Vinay Prasad as the host of Thursday’s HHS/FDA media call to discuss $QURE.
FDA's New Attack on UniQure Sparks Controversy
New from me on $QURE and FDA. Free, no paywall. The FDA, urged to avoid controversy, creates a new headache with attack against UniQure Anonymous diatribe from a senior official plunges agency back into headlines https://t.co/ceDE0TXjdI
FDA's Extremist Shifts Harm Rare Disease Stakeholders
In this week's Biotech Scorecard newsletter: The extremism of the FDA’s Peter Marks and Vinay Prasad has come with costs Two regulators, two extreme regulatory philosophies, one replacing the other. The rare disease community is suffering whiplash. Drugmakers ( $QURE...
Prime Medicine Pursues Approval After Two-Patient Gene Editing Trial
Prime Medicine $PRME to seek approval for gene editing treatment after two-patient trial https://t.co/nBnrUzqSCN via @Jasonmmast
FDA Doubts QURE Gene Therapy Benefit, Leaks to Media
New reporting from me taking you inside the $QURE - FDA Huntington's gene therapy imbroglio. I spoke to a senior FDA official late yesterday, who told me agency reviewers "are not convinced there’s any therapeutic benefit of the product. If...
Advocacy Bias Keeps Unproven DMD Therapy, Blocks HD Progress
The Duchenne muscular dystrophy patient & advocacy community keeps a gene therapy and multiple drugs on the US market, stopping the FDA from taking action, despite failed confirmatory clinical trials showing no efficacy and questionable safety. The Huntington's disease patient and...
FDA Demands Sham‑controlled Trial for uniQure’s AMT‑130
$QURE AMT-130 update: Still blocked The FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for...
Makary Misrepresents Unresolved Drug Applications, Conflates Therapies
(Hopefully) Final word today on the Makary $QURE (or was it $RGNX) thing: Bottom line: Makary shouldn't be talking publicly about unresolved drug applications. Uniqure doesn't even have an application submitted; and RegenxBio's CRL letter isn't posted to the FDA's...
FDA Chief Warns Against Approving Risky, Ineffective Rare‑disease Drugs
$QURE down this morning on comments made by FDA Commissioner Marty Makary to CNBC's Becky Quick. In a discussion about rare-disease drug approvals and Vinay Prasad, Makary said this: "I think there has been a bit of an effort to...
FDA Says Data, Not Numbers, Prove IBRX Benefit
Th FDA is absolutely correct, and confirms what I’ve said all along. This $IBRX @DrPatrick “lymphopenia” push means nothing until or unless they come up with real data showing patient benefit. The “number” doesn’t matter.
Biotech Embraces Data‑Driven Mornings, Gains Afternoons
I love that biotech has returned to the "data in the morning, raise in the afternoon" way of living its best life. $VIR $PVLA
Lymphopenia Reversal Won’t Secure Anktiva FDA Approval
Reversal of lymphopenia is not an acceptable endpoint for Anktiva FDA approval. $IBRX could file but if it did, the application would be rejected.
Gossamer Lung Trial and ProMis Alzheimer Therapy Spotlight
This week's Biotech Scorecard newsletter: -- What to expect from $GOSS Gossamer Bio’s late-stage lung disease study -- A better, safer Alzheimer’s treatment? ProMis $PMN takes its shot https://t.co/YaA8kvUR4Z
INSM Projects 2026 Brinsupri Sales Minimum $1B
$INSM guides to 2026 Brinsupri sales of "at least $1B" in line with consensus. https://t.co/o7JhPKU3m1
FDA's Moderna U‑turn Highlights Agency Chaos
The FDA’s Moderna $MRNA pirouette is one more sign of chaos at the agency https://t.co/zThJqhT2BS via @matthewherper
Moderna's Flu Vaccine Review Set for August 5
$MRNA says FDA will now review its flu vaccine. PDUFA date is Aug. 5. https://t.co/npZzeEa5nk
Saudi‑US Biotech Tie Is Just a Pay‑for‑promotion Scheme
This is not a Saudi-USA Biotech Alliance, it’s a Saudi @DrPatrick $IBRX “you pay we pay” marketing campaign. Anktiva is stalled in the US because he can’t generate the clinical data to move the drug forward, so he goes to...
FDA Rejects IRON Disc’s Rare Disease Therapy, CNPV Stumbles
FDA rejects rare disease therapy from $IRON Disc Medicine, early recipient of commissioner’s voucher CNPV program falters out of the starting gate... https://t.co/w4ptB2ecRS via @LizzyLaw_ and me
FDA Approved Pembrolizumab Using Single‑Arm Trials Across Tumors
It would be highly instructive to read the pembrolizumab paper highlighted by @DrPatrick. Of course, he doesn't expect anyone to do that. This is what it says IN THE FIRST PARAGRAPH: The FDA approved pembrolizumab on May 23, 2017, for...
FDA’s Prasad‑Era: Rules Shift, Rare‑Disease Drugs Stalled
This week's Biotech Scorecard newsletter: The old Vinay Prasad never left. He just changed jobs Submissions to the FDA are handled by teams of reviewers, of course. But when I look across all of these recent cases, I see a...
Corvus Pill Yields 75% EASI‑75 Response in Eczema Trial
$CRVS Corvus pill shows promise in early-stage eczema trial 75% of participants treated with soquelitinib achieved EASI 75 after 8 weeks compared to 20% of placebo participants offered a placebo. https://t.co/XlOmX0GUZf
IBRX Quilt-3.092 Study Mandates Pre‑
The $IBRX Quilt-3.092 CAR-NK study also requires patients to undergo lymphodepleting chemotherapy prior to cell therapy infusion. https://t.co/fPMR7Jhwht
JPM Week 2026 Highlights: Why It Was Fantastic
My latest Biotech Scorecard newsletter, post #JPM26 edition. JPM Week 2026 is over. It was fantastic. Here’s why https://t.co/uwgz7J4WRk
Approved CD19 CAR‑T Cures Lymphoma; NK Therapies Lag
Currently approved CD19 CAR-T therapies extend lymphoma patient survival for years. These treatments are basically curative. Available right now for patients. On the other hand, experimental NK cell therapies have never worked because the cells aren’t persistent. @DrPatrick acts like...
IBRX’s Dilutive ATM Funding Fuels Pump‑and‑Dump Tactics
$IBRX Anktiva q/q sales growth slowed, cash balance decreased. How much dilutive cash did the company raise from its adjusted ATM in January? https://t.co/UTKcVOAs4x Classic: Issue lots of press releases, pump stock higher, sell stock.
Possible Coordinated Fake Biotech Tips Manipulate OCUL Stock
Is there a single person feeding La Lettre fake biotech takeover tips, or is this a group effort? The stock manipulation is brazen. First, we learn all the $ABVX $LLY French Ministry speculation was fake, now today it's $SNY $OCUL...
Project Prometheus Expands VC Bob Nelsen Beyond Healthcare
Secretive Project Prometheus takes VC Bob Nelsen beyond just health care https://t.co/iRuv6MP3bW via @ADeAngelis_bio #JPM26
Novo CEO Targets Obesity Drug Acquisition to Boost Leadership
$NVO CEO Mike Doustdar was our guest on this week's live taping of the Readout LOUD. And yes, he spoke fairly plainly about his ambition to acquire another developer of obesity drugs. $5B - $20B doesn't matter, it's more about...
Saudi FDA Approves IBRX Despite Flawed, Inconclusive Data
The Saudi FDA approved $IBRX Anktiva in lung cancer based on a discontinued randomized study with inconclusive post-hoc data + a non-randomized study with a made-up nonsense responder analysis. These are the data that @DrPatrick and $IBRX tried to submit...
