Myqorzo ACACIA Study Achieves Dual Primary Endpoint Success
$CYTK Here we go! Myqorzo ACACIA study results in nHCM --> BOTH co-primary endpoints hit with statistical significance. There may be debate about magnitude of improvements for Myqorzo via KCCQ-CSS and peak V02. But, if you were dreaming for a double stat sig win, you got it. Details in my story: https://t.co/ng3ytbZRNU
Patents Hide Life‑Saving Drug Clues Await Discovery
What if the next life-saving drug is buried in a patent diagram? In early drug discovery, progress can hinge on a single decision. The evidence exists, but some of the most valuable insights stay invisible to conventional search, especially inside...
Full Asset Acquisitions Avoid Margin Drag of Royalties
Seedy is not long enough $ABVX, however I’m old enough to remember when $ABBV spent $21B to buy half of ibrutinib.
New Trial Compares Dara‑Bor‑Dex vs Cy‑Bor‑Dex in MM with LCCN
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/Jrh4teOxUK
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/oQzEYsPoU4
Phase 3 Trial
.@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/zWMK1RfCFW
FDA Likely to Fast‑Track KRAS Drug for PR Boost
To try to offset this, I predict the FDA will approve the $RVMD KRAS drug in record time. Not just much quicker than usual. FDA needs a public relations win, and this will be their vehicle.
Gene Therapy Targets Childhood Blindness by Fixing Retinal Gene
Jean Bennett and Albert Maguire developed a gene therapy approach to correct a gene in the retina to treat childhood blindness. https://t.co/WJ99qvcUT1
Leroy Hood Unveils Dark Proteome Frontier at SynBioBeta 2026
“I am interested in deciphering human complexity in terms of wellness and disease. I guess you would describe me as a multi-disciplinary human biologist.” That is Dr. Lee Hood @ISBLeeHood in one quote. We're excited and honored to have Dr. Leroy Hood...
One Hepatic Failure Case Sparks LLY vs Novo Debate
$LLY $NVO So one case of hepatic failure reported with $LLY Foundayo. To put this in perspective, with the injectables in this class, the reported hepatic incidents have been well under 1 in 100,000. Other oral GLP-1 agonists have also...
Deviation Capital Launches $300M Fund, Seeks Bio‑tech Founders at SynBioBeta
Deviation Capital just launched with a $300M fund target, and founding partner Dusan Perovic will be at #SynBioBeta2026 this week in San Jose looking for the next generation of founders to back. Deviation spun out of @TwoSigmaVC with a clear thesis:...
Celcuity Bolsters ASCO‑highlighted Breast Cancer Drug Case
Celcuity strengthens case for ASCO-spotlighted breast cancer drug https://t.co/HdekunGXsu by @Lilah_Alvarado $CELC + 18% $NVS $AZN $RHHBY
Acarbose Protects Retina by Modulating Microglial Metabolism
Acarbose modulates microglial Pkm2 acetylation to reshape immunometabolism and preserve retinal neurons after ischemia-reperfusion 👉"Our findings support that ACA exerts retinal protection through the Sirt1-Pkm2-NAD axis, suggesting a metabolic checkpoint that integrates immune and mitochondrial regulation." https://t.co/V3WGPV26qk
German Lab Prints Tumors to Study Neutrophil Interactions
One of the cooler things I saw during my visit to Germany last week was this tumor printer. The company is using it to test how neutrophils affect the tumor microenvironment. https://t.co/jXzxDpj6nZ
Funding Cuts and Visa Limits Spark US Brain Drain
#NIH funding cuts & visa crackdowns are driving away talented foreign researchers who normally would have flocked to the US, a @statnews.com survey shows. "It is going to cause a long-term brain drain," one US based researcher told @DrewQJoseph. ...
BEAM Wins TIME 100, Delivers First Gene Base Editing Cure
Congrats to $BEAM on being named as one of @TIME’s 100 most influential & innovative companies that are shaping the world & our future. @beamtx’s leading Gene Editing platform - Base editing, has achieved a significant milestone when KJ Muldoon...
Top AI Researcher Departs Genentech, Rethinks Pharma Future
“It got a bit too distant for me." Some insights into big pharma & AI from @kchonyc, a top AI researcher who talked with me about leaving Roche's Genentech and what he's thinking about next: https://t.co/IvzWUjc9Qd
5-HT1A Blockade Amplifies DMT’s Subjective Experience
5-HT1A receptor blockade potentiates the subjective effects of DMT Zarmeen Zahid, Rick J. Strassman, Clifford R. Qualls, Sandeep M. Nayak, 2026 https://t.co/5tCpjw2Iaz
Pharma Embraces AI, Yet Breakthrough Drugs Remain Elusive
JUST IN: The pharmaceutical industry is adopting AI, but new drug discovery through it remains elusive.
Low‑dose Endoxifen Cuts Breast Density, Fewer Side Effects
Low-dose endoxifen reduces breast density by up to 26%, matching the effect of tamoxifen but with fewer side effects, suggesting potential for improved preventive strategies in breast cancer. breastcancer
Nature Study Turns Into Immediate
I wrote about this 6 weeks ago at Ground Truths when the 2 @Nature papers by @HugoAerts and colleagues came out. Today it's a "Health Alert"! https://t.co/CgxBU7IAk1 https://t.co/24PijmLhzR
GLP Success Sparked Unstoppable Peptide Boom
The (hard for some to accept) reality is that the popularity of GLPs, which of course have lots of RCTs to support them, are actually what opened the doors for the immense interest in all the other peptides. People are...
Wearables Transform Clinical Trials with Continuous Real‑World Data
Delighted to share details on an exciting forum that we are hosting here at the Querrey Simpson Institute for Bioelectronics at @NorthwesternU — Advanced Wearable Sensors and the Future of Clinical Trials — on June 24, 2026, at the...
Electronics‑Free Smart Lens Monitors Glaucoma and Releases Medication
Researchers have developed a prototype electronics-free smart contact lens that can track glaucoma in real time and deliver drugs in response. https://spectrum.ieee.org/smart-contact-lens-glaucoma-microfluidics
T‑cell Engagers Face Uncertain Future Amid Mixed Readouts
T cell engagers for I&I will have a lot of clinical readouts this year. This update (report from MS) from $AMGN on blinatumomab last week doesn't bode well for the class and it's unclear if next gen formats will...
Epigenetic Reprogramming Enters Human Trials for Vision
Aging biotech has made TIME's "New Frontiers" list. @lifebiosciences is using Yamanaka factors, and the FDA just cleared their first-in-human trial for optic neuropathies. Partial epigenetic reprogramming is no longer theoretical. It's in human trials. https://t.co/ZtVT5PxbHZ https://t.co/OhqLtNfqGT
Blood Factors Drive Aging or Rejuvenation; Therapies Emerging
Aging is increasingly understood as a system-wide process shaped by factors in the blood, which can actively drive either decline or rejuvenation rather than just reflect it. Emerging therapies that modify the circulatory environment show promise in reversing aspects of...
NMN Curbs Antibody Attacks, Eases Bleeding Disorder
NEW CLINICAL TRIAL RESULT: NMN suppresses harmful antibody-driven immune attacks to alleviate a common bleeding disorder. Authors suggest it may help other autoimmune diseases like lupus and rheumatoid arthritis 🙏🧵 https://t.co/eMvefTFxZo
AI's Measured Impact on Biopharma R&D Remains Limited
My 2c on this highly relevant @Loftus @WSJ on ai in biopharma R&D, an article that offers generally measured assessment of ai impact so far, and will not come as surprise to regular @timmermanreport readers…-> https://t.co/KPigSAw5RC
Patent Monopolies Fueled Opioid Crisis, Perdue Falls
With Perdue Pharma biting the dust, it might be a good time for a post-mortem that looks at the government-granted patent monopolies that provided the incentive for pushing opioids https://t.co/bWKnxlYaS2
Malaria Vaccine Team Faced Funding Delays at Every Stage
“The researchers who worked on this malaria vaccine struggled at every step of the process to get faster funding.” ~@salonium
ByteDance's AI-Designed Therapies Spotlight Global Progress
ByteDance’s drug unit presents AI-designed therapies at global conferences Yes this is reality of AI applications, but we are "winning" by slowng Chinese company progress on AI? https://t.co/G3lIeOAddU
KRAS G12D Drug Shrinks Tumors, Delays Progression
Setidegrasib, an investigational therapy targeting KRAS G12D, demonstrated tumor shrinkage and delayed disease progression in early trials for advanced lung and pancreatic cancers, offering a potential new approach for hard-to-treat mutations. oncology
R‑MDDMA Boosts Neuroplasticity and Ant
methylated MDMA analog in animals R-MDDMA "still promoted structural neuroplasticity in cortical neurons, facilitated fear extinction learning, and produced sustained antidepressant-like effects" did not directly activate 5-HT2B receptors https://t.co/w0ltV7VXQL
Boosting NAD+ May Restore Sleep in Dementia
NAD+‒circadian rhythm coupling in dementia "Pharmacological and lifestyle-based strategies targeting NAD+ restoration are outlined as potential approaches to improve sleep and circadian rhythm integrity..." https://t.co/qQXLHS8nou https://t.co/te8QGUftC4
AGEs Trigger SIRT1 Loss, Accelerating Osteoarthritis via RANKL
SIRT1 Downregulation by Advanced Glycation End Products Activates RANKL-Dependent Osteoclast Signaling and Drives Chondrocyte Senescence During Osteoarthritis Development "Targeting this mechanism may offer new therapeutic opportunities for delaying age-related OA progression." https://t.co/HB2nmyZ43u
Modern Meds Are Precise; Older Drugs Are Broad-Spectrum
Modern drugs tend to be very tightly targeted. Older drugs like metformin or Tylenol cut a wider swath.
Questioning High Drug Prices and Transparency of New Tech
Oh okay. Just like TrumpRx, which charges patients more for many common drugs. Let’s see some data on how well this technology works and how transparent it really is.
Allosteric Switch in TB Enzyme Opens New Drug Target
A newly identified allosteric switch in the ICL2 enzyme of Mycobacterium tuberculosis reveals a potential pathway for targeting drug-resistant TB, offering insight into protein regulation unique to the bacterium. structuralbiology
CNS Gene Therapies Showcase Tau-Targeted VY170
CNS Gene Therapies Featured in Multiple Presentations at ASGCT 2026, Including Late Breaker on Tau-Targeted VY1706 for Alzheimer’s Disease https://t.co/oQ5MCd5piS https://t.co/8KeWSTJ1q7
LQDA Repeats UTHR Play, Seizes 157% Market Growth
$LQDA "Meaning Yutrepia captured 157% of the incremental market (took share AND expanded the pie). This to me looks like a re-run of UTHR's old playbook."
FDA Approves Early, yet ARVN, PFE Lack Partner
FDA approves vepdegrestrant one month early. $ARVN and $PFE still haven't found (or disclosed) a third-party company to commercialize it. What a world...
ARCH Backs Mental‑health IPO, Celebrating Early Investment Success
Congrats to Daphne Zohar and team, and Steve Paul for an amazing IPO to fuel new drugs for mental health and brain disorders. ARCH invested early here because of the team, platform, and clinical need.
T‑cell Vesicles Deliver DNA, Converting Cold Tumors Hot
In cancer, activated T cells release abundant extracellular vesicles that transfer DNA to the nucleus of tumor and immune (dendritic) cells, turning tumors from cold to hot. An immunotherapy in the works. @Cancer_Cell https://t.co/mHcahijOJC https://t.co/oPT6d7lrFM https://t.co/I0egYxZold
Early Trial RNA‑seq Data: Exciting Yet Requires Caution
🧵 You just got your hands on early clinical trial RNA-seq data. Excited? You should also be cautious. Here's why. 👇 https://t.co/GjA8XIYcVQ
High‑dose Semaglutide Cuts Alcohol Use in RCT
Clinicians and patients saw big reductions in alcohol intake when starting GLP-1 medicine. This is the first randomized trial to look at the high dose of semaglutide. There was a study a couple years back looking at a lower dose....
Enzymatic DNA Synthesis Frees Synthetic Biology Design
“You should design for the biology you want to build, not for the process limitations of a supplier that’s been holding the field back.” That line from @Jason_Gammack , CEO of @AnsaBio , gets right to the heart of one...
FDA Approves First Non‑antipsychotic for Dementia Agitation
⚕️ FDA Approves First Non-Antipsychotic Drug to Treat Agitation Associated with Dementia 🔗https://t.co/cPNtkiSXBs 🌐 #DHPSP #Pharma #Healthcare https://t.co/KsBKc45ekk
Interim Data Hint Weaker PFS; Selloff Caps Losses
Leerink's Daina Graybosch on $SMMT "... In our base case scenario of the company’s likely statistical design choices and enrollment timing, not hitting statistical significance at this interim suggests a degradation in PFS Hazard Ratio (HR) of ~10 percentage points...
Semaglutide Cuts Heavy Drinking and Cravings in AUD
GLP-1 Drug Semaglutide (Ozempic, Wegovy) Reduced Heavy Drinking & Craving in Adults with Alcohol Use Disorder https://t.co/FYRkjWYhJQ https://t.co/cGtEajk9lp