Ianalumab Combined with Eltromb
Ianalumab [VAY736] plus Eltrombopag in Immune Thrombocytopenia [Dec 9, 2025] @CukerMd et al. for the VAYHIT2 Investigators @NEJM https://t.co/99urXh8agT #NCT05653219 #ITP https://t.co/qKKhwv4ofR
Duchenne Kids Show Dramatic Improvement After Microdystrophin Treatment
A couple of weeks ago when I visited Solid Bio, they showed me some videos of Duchenne kids pre and post microdystrophin treatment that I was amazed by - but they weren't sharable yet. Bo just presented them at Needham...
Anti‑amyloid Drugs Show Trivial, Non‑meaningful Cognitive Benefit
The effect of current anti-amyloid drugs vs Alzheimer's disease is "trivial" for cognitive function or dementia severity, not clinically meaningful, from a systematic @cochranecollab review of 17 trials, >20,000 patients https://t.co/9A6PeZOyAt https://t.co/c1PCOYZxws
MGTX Buys Back XLRP Gene Therapy for $25 M
$MGTX just announced that it has entered into an asset purchase agreement with $JNJ to acquire back its bota-vec Gene Therapy program for the treatment of X-linked retinitis pigmentosa (XLRP) 🧵👇 for an upfront cash payment of $25M & a...
FDA Approves Kidney Drug Despite No Functional Benefit
This week's Biotech Scorecard newsletter: $TVTX Travere Therapeutics' drug for a rare kidney disease doesn’t improve kidney function. The FDA approved it anyway. https://t.co/63hDsPfOcT
GLP‑1 Drugs Don’t Disproportionately Reduce Functional Muscle, DXA Misleads
1/2) New research suggests GLP-1 weight loss drugs probably do NOT cause disproportionate loss of functional lean muscle. The confusion has arisen from the fact that DXA scans capture and pool a lot of different organs and tissues into the category...
Roche Initiates New Elevidys Trial Targeting EU Approval
Roche to launch another Elevidys trial, with eyes on European approval https://t.co/eAANNiihpt via @DrewQJoseph $SRPT
Endovascular Therapy Shows Promise for Post‑Thrombotic Syndrome
Endovascular Therapy for Post-Thrombotic Syndrome — A Randomized Trial [Apr 13, 2026] Vedantham et al. for the C-TRACT [Chronic Venous Thrombosis: Relief with Adjunctive Catheter-Directed Therapy] Trial Investigators @NEJM https://t.co/Ayd4Fa0ARl #NCT03250247 #VTE https://t.co/6iD1NUQdgX
Patient‑Driven Platform Accelerates Multiple Myeloma Research
.@HealthtreeMM Hub: A Patient-Derived, Patient-Driven Clinical Cancer Information Platform Used to Overcome Hurdles & Accelerate Research in Multiple Myeloma [3/10/22] @NSweeneyPhd @AhlstromJenny @FaithEDavies1 @mtmdphd @JCOCCI_ASCO https://t.co/WOwLb9Eozv @myelomacrowd #mmsm https://t.co/6HieAzjSES
New Trial Compares Dara‑Bor‑Dex vs Cy‑Bor‑Dex in Myeloma‑Induced Kidney
EAA241 - Ph 2 RCT Dara-Bor-Dex vs Cy-Bor-Dex in the treatment of Newly Diagnosed Multiple Myeloma with Light Chain Cast Nephropathy (LCCN) [Study activated 8/11/25] @keruakous https://t.co/1NgvVZ3fTA #NCT07085728 #mmsm @eaonc https://t.co/KJfqVVcwEr
Daratumumab Boosts Revlimid Efficacy in Smoldering Myeloma
#EAonc EAA173 - Daratumumab to Enhance Therapeutic Effectiveness of Revlimid in Smoldering Myeloma (DETER-SMM) - PI: @nsc_natalie https://t.co/VtBMJUjI5X Activated: Apr 30, 2019 #mmsm @eaonc #NCT03937635 @VincentRK @mweissmdphd https://t.co/SKPUQiSaU4
Phase 3 Trial Tests Daratum
.@SWOG S2213 Ph3 RCT Dara-VC Induction Followed by ASCT or Dara-VCD Consolidation & Daratumumab Maintenance in Pts w/ Newly Diagnosed AL Amyloidosis [Activated: 12/1/23] https://t.co/OizUfJCc2c #mmsm #bmtsm https://t.co/Hq9VlRCTCI
Four Candidates Target Ultra‑Rare Polish PUS3 Syndrome
“We have four drug candidates for patients with an ultra-rare disease with Polish origins, the PUS3 syndrome; one of these candidates will be used in practice,” Lisowski said. https://t.co/GTJbPQSdXQ
When to Stop Myeloma Treatment: MRD Insights
MT @hhashmi87 #IMS25 Great Meet The Expert talk by @bdermanmd on MRD in Myeloma Risk vs benefit Who can stop treatment Active surveillance post discontinuation #mmMRD #mmsm https://t.co/FIDPrE7QRb
MRD‑negative Patients May Stop Myeloma Maintenance Therapy
Discontinuation of maintenance therapy in multiple myeloma guided by multimodal measurable residual disease negativity (MRD2STOP) - @bdermanmd et al. @ajjakubowiak #ASCO24 Abstract 106 https://t.co/FBTY7SnCxK #NCT04108624 #mmsm #mmMRD
ODAC Approves MRD as Early Endpoint for Myeloma Trials
A Historic Turning Point: ODAC Unanimously Votes [4/12/24] in Favor of MRD Testing as an Early Endpoint in Myeloma Clinical Trials to Support Accelerated Approvals of New Treatments [Apr 18, 2024] @IMFmyeloma https://t.co/eDOgIrpVeR #mmMRD #mmsm #ctsm @FDAOncology https://t.co/V4wrNgvTrG
Clinicians Show Mixed Adoption of Myeloma MRD Testing
Clinician attitudes and practices toward measurable residual disease in multiple myeloma [Jun 7, 2020] @bdermanmd @jagoda_jasielec @ajjakubowiak Brit J Haematol https://t.co/8zhxYfYwZQ #mmsm #mmMRD https://t.co/mPtRCjnB9f
Vaccines Vanishing Threatens Global Health Catastrophe
The Horrors That Could Lie Ahead if Vaccines Vanish — @propublica good article on the work of @NathanLo3579 @Stanford my former mentee and amazing colleague https://t.co/n9oUgkvOZ7
Glioblastoma's Immune Microenvironment Blocks Checkpoint Therapy
The glioblastoma tumor immune microenvironment (TIME) is an immunosuppressive barrier to therapy that encumbers glioblastoma responses to immune checkpoint inhibition (ICI). https://t.co/rgIKZ8N0Q4
Synthetic Biology Localizes Drug Production, Ending Fragile Supply Chains
Most pharmaceutical APIs still travel thousands of miles through fragile global supply chains before they reach a patient. COVID and the Russia-Ukraine war showed exactly what that fragility costs. Synthetic biology offers a different model: program cells to manufacture the molecules in...
Bergamottin Reduces Cancer‑related Cachexia via Multiple Pathways
A Novel Role of Bergamottin in Attenuating Cancer Associated Cachexia by Diverse Molecular Mechanisms https://t.co/pr3d4kg0hB #mdpicancers
Early GLP‑1 Rodent Studies Showed Weight Loss Before Diabetes Focus
I don't think this is correct from @nytimes op-ed on GLP-1s - wt loss known from early rodent studies involving injection into CSF as I recall. DM2 more tractable as initial pharma indication, esp at that time - many still...
New NEJM Review Links Hormone Therapy to Cardiovascular Risk
Sex hormones, clotting disorders, and cardiovascular risk An important new @NEJM review for MHT and testosterone latest data https://t.co/UNqXAwjsKM https://t.co/4ckJsc4YyE
FDA Reverses Ban on 12 Peptides for Review
So... @SecKennedy just announced that 12 peptides the Biden FDA shoved into "Category 2" — effectively banning them from regulated compounding pharmacies and driving people to the black market - are being pulled back for legitimate scientific review. Here's what each one...
Retatrutide Poised to Become History’s Biggest Drug
Not s hot take, but I think retatrutide could be the largest drug in history @TravisHoium makes a great case on why the best way to play the emerging peptide market is $HIMS
Engineered Brain Cells Erase Alzheimer’s Proteins in Mice
Enhanced brain cells clear away dementia-related proteins New cellular immunotherapy approach for Alzheimer’s disease shows promise in mice https://t.co/YBoqUZLvJo https://t.co/FjG699Etga
IPSC Tissues Supply Causal Data to Power Simulations
A general-purpose biological simulator, one that can predict how the human body responds to any intervention, isn't blocked by compute. It's blocked by data. Not data in general. Causal, human-relevant data. The kind where molecular interactions actually produce functional outcomes you...
AI-Driven Precision Oncology Breakthrough From Korean Researchers
One unexpected but very insightful paper in Nature BJC from a leading institution in Korea on precision oncology and AI-driven drug discovery. I read it with great pleasure and you should read it too. https://t.co/vpxofjt9Mn https://t.co/SXs4n72CcY
Elon Bets, GLP‑1 Relevance, and Consumer Sentiment Slump
🆓 Wednesday links: betting on Elon, why GLP-1 pills matter, and what's driving poor consumer sentiment. https://t.co/wCKQe3osVm image: https://t.co/QkWrhkJpqj https://t.co/aCwEbtyhzI
Promolytics: Targeting Cancer‑Promoting Clones to Halt Progression
A new review on tumor promotion and the evolution of cancer with the concept of "promolytics" —drugs that could prevent potentially dangerous clones and/or their progression https://t.co/IFah5gi8ZK @Nature
Former FDA Chief Urges Term Limits, Merit‑based Appointments
Former FDA cancer chief/CDER dir. Rick Pazdur told me earlier this week he's concerned w/political appointees injecting themselves into drug reviews - and that this might trickle down past this admin. He's calling for time limits on senior FDA positions...
Moderna's Cancer Therapy Rename May Mislead Physicians
Moderna mRNA cancer treatment used to be called a "cancer vaccine" but in 2023 they switched to "individualized neoantigen therapy." Some docs think the name change is misleading. https://t.co/UIn2djdDAv
AI Agents Power Future Biotech Labs and Organizations
Live in 15 minutes — I'm talking with Stanford's @james_y_zou on his latest research in building laboratories and biotech orgs made of hundreds or even thousands of AI agents Register now to watch live, for free: https://t.co/xSa5NXkX4A
Statins Don't Harm Muscle Health in Older Adults
Statin use does not impair muscle health in older adults: findings from the SCOPE study https://t.co/WKjk9Ov0rG https://t.co/xTo9mk3vbw
AI Designs Lab-Ready Antibodies From Text Prompts
Drug discovery has always meant finding. Screening libraries. Keeping what survives. Semiconductors don't work that way. Neither do aircraft. You design them computationally before anything gets built. @saakohl left @GoogleDeepMind after co-developing AlphaFold2 to do the same thing for biologics. @LatentLabs_ Latent-X2...
Chinese Biotechs Lead Licensing as M&A Surges
M&A is sizzling right along, with the Q1 numbers looking good for biotech. IPOs got started on the year with a spike in offerings and some mixed results on the Street. Dealmaking remains solid. And shall we just call it...
FDA Peptide Meeting Signals $100B Market Boom
I wish this FDA meeting to discuss peptides was happening a little sooner but I'm glad it's finally on the calendar so we don't have to keep guessing. Looks like they're going to start with 7 peptides (BPC-157, TB-500, KPV, Mots-C,...
Synthetic Biologists' Mirror‑image Microbe Dream Hits Complexity
Synthetic biologists were tantalized by the idea of making mirror images of microbes. Then things got complicated.
Two‑Month Dosing Likely Beats Monthly in PK Trial
As I commented before, they have Phase 2 PK data and they still took both 2 months and 3 months into Ph 3. The primary endpoint is purely a PK non-inferiority one (AUC through week 24). That means that 2...
FDA Panel to Consider Expanding Peptide Access
FDA panel will meet to discuss allowing broader access to certain peptides https://t.co/PDWKvmvvUH via @LizzyLaw_
WSJ Defends Replimune Drug Amid Mounting Criticism
@WSJ is not giving up the fight for Replimune's cancer drug. Second CRL draws another rebuke and a direct attack on @MartyMakary (Prasad gets another swipe as well, but he's about out the door now.) I often disagree with the...
AI-Driven Global Collaboration Needed for Longevity Research
Great speaking with @SpeakerPelosi at @semafor . In my opinion, drug discovery for cancer and age-related diseases should be a global effort. Nations need to strive to collaborate to extend healthy productive life. Please through longevity. It is the best...
Electromagnetic Gene Switch Extends Lifespan in Progeroid Mice
Wow, a technique that allows electromagnetic control of gene expression in vivo 🤯 And they tested their system with OSK partial reprogramming, showing it extends lifespan in progeroid mice. In normal mice, they report health improvements and a small reduction in mortality...
Liquid AI Launches Foundation Model for Drug Discovery
@semafor World Economy brought together most of the global CEOs to Washington, DC. And it is a pleasure and honor to meet our amazing partner @ramin_m_h of Liquid AI . We just published the Liquid Foundation Model capable...
AI Agents and Next‑Gen Alzheimer’s Drugs Beyond CRISPR
Endpoints' Drug Discovery Day is today — our own @RLCscienceboss will be talking about beyond CRISPR & future of Alzheimer's drugs I'm excited to talk with Stanford's @james_y_zou on his escalating research in building AI agents into co-scientists, labs, and now biotechs...
MRNA Nanoparticles Teach Beta Cells to Prevent Type 1 Diabetes
As a medical school professor, I can tell you: what we've been doing for type 1 diabetes is managing, not curing. University of Chicago scientists just changed the game. They developed mRNA-loaded nanoparticles that deliver genetic instructions directly to insulin-producing beta cells,...
Today's Teen Could Become First 150‑year‑old
David Sinclair says the first person to live to 150 is a teenager who's alive today. He's taken flak from colleagues for years over this prediction. He doesn't care. He still stands by it. "The first person to live to 150 has...
Advisory Committee Needed to Vet RP1/Replimmune Melanoma Data
I haven’t dug into the specifics of the RP1/Replimmune data in melanoma, but isn’t this an example of where a good old-fashioned Adcomm could help vet the data and overall risk-benefit? https://t.co/qAxtU9pUeF
New Drug Boosts Survival for Platinum‑Resistant Ovarian Cancer
Combining a new drug with #chemotherapy extends the survival of platinum-resistant ovarian #cancer patients in #clinicaltrial https://t.co/JE4r8nISUq
Lab Success Doesn't Guarantee Real Cancer Drug Efficacy
Just a reminder. Killing cancer cells in a lab is very easy. Almost anything will kill cells. Even water (because of osmotic damage). Inside our bodies killing cancer cells is extremely hard for many reasons so things that kill...