Trump’s Drug Pricing Deals Underdeliver on Promised Cuts
Trump celebrated closing the first round of 17 drug pricing deals, now looking ahead to many more. But the reality of the deals, so far, have fallen short of grand promises the president made. https://t.co/QmhvZNgNyI
FDA Approves First Free Gene Therapy Restoring Deaf Children's Hearing
JUST IN: FDA approves first ever gene therapy that restores hearing in children born deaf, with treatment available at no cost
AI Learns From Millions of Genomes, Not Text
What if you could train a ChatGPT-style AI not on text from the internet, but on millions of genomes across all of life? Samuel King has spent his @Stanford PhD building exactly that. He'll be on the "Genome as a Canvas" panel...
Regeneron Approves Free Gene Therapy, Sparks Industry Precedent Worries
$REGN wins FDA approval for its gene therapy to treat a very rare genetic form of hearing loss. The company is giving away the therapy for free. OTOF-related hearing loss affects approx. 50 newborns per year, so really rare, but......
Roche Bets on Cancer and Obesity Drugs Amid Biosimilar Pressure
Roche, facing biosimilar threats, puts faith in new cancer and obesity drugs https://t.co/IIt0INA9sI @ByJonGardner $RHHBY
New Trial Compares Dara‑Bor‑Dex vs Cy‑Bor
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/n2tSuJXS5b
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/VPIH8iphVP
Phase 3 Trial Tests Daratumumab Reg
.@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/xv1RlCv8gl
Backing Coherence Neuro's Real Solution for Glioblastoma
A great look at @coherenceneuro, a company we've been backing since 2024. Coherence is actually building something useful for a real market; the thousands of people who get diagnosed with glioblastoma - one of the deadliest brain cancers - each...
Sanofi Reports Strong Sales Amid Growing R&D Pressure
Sanofi posts upbeat sales as R&D pressure builds https://t.co/4WUccjXY5D by Kristin Jensen $SNY + 2% $REGN
Gene Therapy Cures Specific Deafness—A Modern Miracle
Another modern miracle: Gene therapy cures a form of deafness. Harvard to USA: You're welcome. https://t.co/mfEzVMGugp
Implant Keeps Drug‑Producing Cells Alive for a Month
A new bioelectronic implant can support populations of three different drug-producing cells for more than a month, keeping them supplied with the correct amounts of oxygen. These cells could produce drugs to treat various cancers, autoimmune diseases like arthritis, and...
Half of NMN Supplements Mislabelled, May Harm Users
David Sinclair's research helped turn NMN into a billion-dollar industry. Now he says roughly half the products don't contain what's on the label - and some contain ingredients that could harm you Firstly, NMN (nicotinamide mononucleotide) is a molecule that converts to...
Hims Secures GLP Deals, Positioning for Retatrutide Boom
This is massively good news for $HIMS members and $HIMS shareholders. $HIMS now selling $LLY GLPs which is Tirzepitide. $HIMS already selling the $NVO GLPs which is Semaglutide. This also means that $HIMS members should have access to Retatrutide...
Wrapping Up Molecules, Medicines & Markets Course
Final session of my "Molecules, Medicines, & Markets" class at Tufts Bio today. Here's the last slide (HT @dwarkesh_sp): https://t.co/1fkSW4BleX
ASCO Grants IDYA Late‑breaker Despite Extensive Prior Data
Given how much $IDYA has already released (ORR, PFS, stats, PFS curves) I'm a little surprised #ASCO26 is OK with giving it a late-breaker.
RG6735 Identified as New $RHHBY Phase‑1 Asset
Any idea what RG6735 is? Looks like a new $RHHBY code for sth that went into phase 1 earlier under a different name. https://t.co/7Cb5Ft2BWC
CEPI Shows Courage; Institutions Must Follow Suit
Many institutions are failing to meet the moment, but not all. @CEPIvaccines proves it has the courage & conviction to carry out essential vaccine work regardless of political pressure for complicity. I wish more institutions would follow their funder’s example.
OSK Reprogramming Restores Adult Heart Regeneration
NEW STUDY: OSK rewinds the clock and pushes adult heart cells into a regenerative state that improves heart repair Why is this such a big deal? Because adult heart cells do not meaningfully divide, which is why the heart heals with scar...
Crowdfunded Rapamycin‑Exercise Trial Shows Promising Aging Data
Five years ago, @BradStanfieldMD reached out with an idea: a crowdfunded clinical trial testing rapamycin combined with exercise in older adults. The results are now published — and Brad and I just sat down for a full 42-minute breakdown...
Shingles Vaccine Cuts Dementia Risk by Half.
Have you had your shingles shot? A major 2026 study of over 300,000 people age 65 and older found that the shingles vaccine reduces the risk of dementia by up to 50%. Remarkably, men, women, and folks across age and ethnic...
Fenebrutinib Shows Promise, Yet Safety Concerns Loom
Relevant to $TGTX was the Roche $RHHBY fenebrutinib trial results recently announced. Excellent efficacy, but two cases of Hy's Law. Both resolved, but a patient might not be so lucky next time. Hard for me to see this gaining widespread...
TGTX Shows Consistently Low Relapse Rates Across Trials
This very low relapse rate in this $TGTX Phase IV observational trial is consistent with that seen in their 6-year extension study: https://t.co/Shvb8h8VRr
Paxlovid Showed No Hospitalization Benefit in Vaccinated Seniors
In 2 randomized, open-label trials of Paxlovid there was lack of evidence of reduced hospitalizations among the participants, who were age 50+ with coexisting conditions, and who were vaccinated. The endpoint was very low (<1.2%) in the treatment and control...
Top Protein Degrader Session at AACR26 Highlights Await
Excellent protein degrader session today at #AACR26. Third speaker was all Do Not Post (sorry @HartungIngo !) so some highlights from the other three speakers coming up...
Alzheimer's Drugs Show Minimal Benefit, Review Reveals Deeper Issues
Alzheimer’s drugs offer little benefit, major review finds – and the reasons go deeper than the science https://t.co/cnMPAEQumY https://t.co/wtg8llquON
FDA Criticizes Replimune's Melanoma Trial Design
Some observers, including @US_FDA, have criticized @Replimune for the design of the trial that led to the decision not to grant accelerated approval of their melanoma treatment. I asked the company's Chairman about that view. Watch the full interview: https://t.co/XYVmSeodLv https://t.co/OO8eXTZOXh
Marijuana to Be Reclassified Alongside Ketamine, Steroids
"The new categorization would put it in line with ketamine and steroids." well it's a start
Pharma-Backed Petition Urges FDA to Overhaul Rejection Letters
New Pharma-backed petition calls on FDA to reform how it releases drug rejection letters, says current process is unlawful - https://t.co/XLwCxt1FY6
PK/PD Crucial for Next-Gen ADC Development
Wonderful tour de force on ADCs by @raffcolo highlighting the importance of PK/PD going forward with new formats https://t.co/nUpBNNoVil
Moderna Launches mRNA Bird Flu Trial After Funding Loss
Moderna, after losing US funding, rebounds to start mRNA bird flu vaccine trial https://t.co/cKyC9jfWAj by Kristin Jensen $MRNA
Prelude Tx Unveils Early-Stage KAT6A Degrader
Looking through my #aacr26 collection of posters from yesterday. Prelude Tx have a KAT6A degrader (PRT13722) in early development to compete with inhibitors: https://t.co/23zBtCX8Hs
Personalized CRISPR Poised to Become Standard Care
How individualized CRISPR genome editing can go from rare, expensive use to broader accessibility and a standard of care by @UrnovFyodor and Sadik Kassim @Nature https://t.co/ddc5ASPPAK https://t.co/GOFneIyuai
Merck Expands AI Push; Roche Reports MS Drug Data
Merck adds to pharma’s AI push; Roche details MS drug results https://t.co/VmpWyLhP9A $MRK $RHHBY $TGTX $RCUS $GILD #biotech
FDA Approval Could Ignite Peptide Boom and Doping Grey Zone
Peptide space is going to blowup if FDA approves it. Interesting grey area for sports doping.
Trump’s “Most‑favored‑nation” Deals Don’t Ensure Cheapest U.S. Drug
One lie worth refuting that @RobertKennedyJr keeps repeating across hours of testimony before Congress from the last week (final Senate Finance/HELP day today): The "most-favored nation" drug pricing deals that Trump crafted with 16 cos. DO NOT by any means guarantee...
Redefining ‘First‑in‑Class’ Amid Faster Competing Therapies
How do we define “first in class” when there are others further ahead? This example is from Atheron Therapeutics w/ a CCNE1 degrader #aacr26 https://t.co/s22biREUrt
CDC Vaccine Efficacy Report on Halved Winter Hospitalizations Shelved
The #CDC report that showed the #Covid vaccine halved the risk of a recipient needing emergency care or hospitalization this winter has been shelved by HHS, @bylenasun reports. https://t.co/tCNx8LSIKn
EMP‑01 Shows Strong Patient‑Reported Gains in Social Anxiety
What incredible news to wake up to! Some weeks ago, @ataibeckley reported positive topline results for EMP-01 (oral R-MDMA, a patent-protected version of #MDMA) for Social Anxiety Disorder(SAD). The data reported was the reduction in symptoms as reported by the treating...
AI‑Driven Discoveries Shift Medicine to Understanding the How
The Real Era of the Art of Medicine Begins with Artificial Intelligence. I mean, when AI discovers new treatments and runs in silico clinical trials that physicians, pharma companies, or medical innovators would never think of, our job will be understanding the...
Weight‑loss Meds Hit 8% of U.S. Prescriptions.
wow. "... the weight-loss drugs accounted for nearly 8% of all U.S. prescriptions in March" -- Axios Vitals
Insilico Tops AI Drug Discovery, Drives Oncology Breakthroughs
I had to skip this year's #AACR2026 but I am watching it from the distance. Our posters and team did a very good job - it looks like our clinical-stage oncology assets are finally being better understood by the pharma...
FDA Expands Tzield to Toddlers, Boosting Sanofi's Valuation
FDA expands Sanofi’s Tzield💉 to age 1 — expands TAM, re-rates Sanofi; catalyst vs limited pediatric data and commercialization risk; watch earnings for uptake. Insight: accumulate on pullbacks. — Viktor Kopylov, PhD, CFA More insights: t.me/si14Kopylov
Billionaires Drive Biotech's Race for Cancer Blockbusters
Just read "For Blood and Money: Billionaires, Biotech, and the Quest for a Blockbuster Drug" a good book about developing of a pair of cancer drugs. Great work by @nathanvardi https://t.co/rrTOe6GqIB
Blocking PGAM‑Chk1 Kills Senescent Cells, Eases Lung Fibrosis
Researchers found that senescent cells depend on a PGAM–Chk1 interaction to boost glycolysis and survive, and blocking this interaction selectively kills these cells and reduces disease effects like lung fibrosis. https://t.co/VTsHTkBcad
Durvalumab Plus FLOT Improves Resectable Gastric Cancer Outcomes
EFS in MATTERHORN: A Ph3 RCT of durvalumab plus 5-fluorouracil, leucovorin, oxaliplatin & docetaxel chemotherapy (FLOT) in resectable gastric/gastroesophageal junction cancer (GC/GEJC) - @YJanjigianMD et al. #ASCO25 LBA5 https://t.co/lW8KUtXdB3 #NCT04592913 #stcsm #esocsm
CtDNA‑guided Cam
Camizestrant + CDK4/6i for the Tx of emergent ESR1 mutations during 1L endocrine-based therapy & ahead of disease progression in Pts w/ HR+/HER2– advanced breast cancer (ABC): ...ctDNA-guided SERENA-6 trial - Turner et al. #ASCO25 LBA4 https://t.co/9jFfuGd1IS #NCT04964934 #bcsm
Rusfertide Shows Efficacy in Phase 3 PV Trial
Results from VERIFY, a phase 3, double-blind, placebo (PBO)-controlled study of rusfertide for treatment of polycythemia vera (PV) - @KuykendallMd et al. #ASCO25 LBA3 https://t.co/XID3tkj6Sk #NCT05210790 #mpnsm
Adjuvant Nivolumab Boosts Outcomes in High‑Risk HNSCC
NIVOPOSTOP (GORTEC 2018-01): Ph3 RCT of adjuvant nivolumab added to radio-chemotherapy in Pts w/ resected head & neck squamous cell carcinoma at high risk of relapse - Bourhis et al. #ASCO25 LBA2 https://t.co/Uxh4JhZyDE #NCT03576417 #hncsm #ImmunoOnc
Atezolizumab Boosts Adjuvant Therapy for dMMR Colon
RCT of standard chemotherapy +/- atezolizumab as adjuvant therapy for Pts w/ stage III deficient DNA mismatch repair (dMMR) colon cancer (@ALLIANCE_org A021502; ATOMIC) - @FASinicropeMD et al. #ASCO25 LBA1 https://t.co/cGalNPlxMe #NCT02912559 #crcsm