Neuregulin‑2 Predicts Venetoclax Sensitivity in Non
Identification of a Novel Drug Sensitivity Biomarker Neuregulin-2 for Venetoclax in Non-t(11;14) Multiple Myeloma [Dec 12, 2021] Dai et al. Abstract 459 #ASH21 https://t.co/mnXEThuKgy #mmsm #PrecisionMedicine https://t.co/zx7d29Jzpk
HHS Weighs Appeal of Controversial Vaccine Schedule Ruling
To appeal or not to appeal (yet): The potentially politically dicey situation facing HHS over a preliminary court ruling this week on #ACIP and the rewritten childhood vaccination schedule. By @ChelseaCirruzzo. https://t.co/3P1PW1lDFT
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/acq0EeyNFn
Phase 2 RCT Compares Dara‑
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/s8eqQJkQzK
Quadruplet Regimen Proves Effective in NDMM, Not Early
#EAOnc EQUATE EAA181 Effective Quadruplet Utilization After Tx Evaluation: Ph3 RCT NDMM Not Intended for Early ASCT [Activated: 10/27/20] PI= @myelomaMD https://t.co/qD26HMx3J8 #NCT04566328 #mmMRD #mmsm @eaonc @mweissmdphd @VincentRK @LynneWagnerPhD Wei Snyder Kostakoglu @mtmdphd
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/lI3MqKHIiY
FDA Finally Holds Public Hearing on Priority Review Vouchers
Oh, *now* the FDA has decided to have a public hearing on the national priority review voucher program. After four drugs have completed the process. A year after the program was announced. This must be that "radical transparency" that @DrMakaryFDA...
AI Simulates Cells to Accelerate Drug Discovery
AI didn’t just enter biology. It’s starting to rebuild it from first principles. @MichaBreakstone sold his last company for $575M. Then he went after a harder problem. What if you could model how cells signal, respond, and adapt… before you ever run an...
Companies Hide Device Risks, Harm Thousands
This is a recurrent fiasco. Health care companies that now the serious risk of their device or drug but keep selling it and do not acknowledge the hazard. Until so many people are harmed. Today's front page @nytimes by @katie_thomas @bostonsci...
Tumor Whole-Genome Sequencing Influences Care in 40%+ Patients
Why should tumor whole genome sequencing (WGS) be done for cancer? In real practice of medicine study of 888 patients with solid cancers, WGS directly led to clinical consequences in over 40% @NatureMedicine https://t.co/kFho0yuLS7
Biotech Giant Earendil Labs Secures $787M Funding
Giant biotech round that hit right after I sent Pro Rata: Earendil Labs raises $787M https://t.co/NWt2bBA9Lr
Prasad’s CBER Saw Frequent Overrules, Unlike Rest of FDA
caveat here is that it was routine when Prasad ran CBER (at least 4 overrules in Prasad's 10 mo. tenure) but it didn't/doesn't still seem to be happening elsewhere in FDA
AI Deciphers Life’s Language in New Nature Biotech Review
AI to understand the language of life. Our review @NatureBiotech just published https://t.co/i0WMaXBCHl Free access https://t.co/3LPulNxlD0 @VishRao5 @serena2z @BrianPlosky @pdhsu @BoWang87 @james_y_zou @marinkazitnik @pranavrajpurkar
Former FDA Chief Warns Political Pressure Plagued Trump Era
Political influence ‘a serious problem’ for FDA under Trump, former commissioner says https://t.co/52IR3LfECD via @statnews
Promising Therapeutics' Future at Risk Under Trump FDA
The future of promising therapeutics is on the line in the Trump administration's FDA, says the agency's former commissioner https://t.co/vyt49FkDsb
Rebuilding Vaccine Trust After Ideologically Flawed ACIP Recommendations
Good news for America’s children. ACIP was devoid of vaccine expertise and made harmful and ideologically driven recommendations that went against pediatric medical science. Hopefully we can rebuild pubic confidence in vaccines despite their efforts to undermine it, but it...
NVS
$NVS paying $2bn for Synnovation's SNV4818, after $LLY paid $1.5bn for Scorpion's STX-478. Mechanistically, seems like good news for $RLAY, bad for $OKUR & $COGT
ML and Quantum Computing Unite for Next‑Gen Drug Discovery
#compchem #machinelearning #quantumcomputing New preprint: "The Convergence Frontier: Integrating Machine Learning and High Performance Quantum Computing for Next-Generation Drug Discovery". @qubit_pharma https://t.co/4D23DV0uAk
Beyond AlphaFold: AI Generates Functional Proteins From Scratch
The AlphaFold moment was never the finish line. It was the starting gun. @saakohl helped build the system that cracked protein structure prediction… then left @GoogleDeepMind to go one step further. Design the molecules themselves. From scratch. In silico. Before a single experiment...
Mid‑session Unblinding Doesn’t Equal Procedural Equivalence
But one set of trials (TAD) were open label and other set of trials (psychedelic) included impt elements of experimental control, procedures like randomization and blinding. Does functional unblinding mid-session justify treating these trials as procedurally equivalent? No.
Head
Enjoyed thinking about the new study by @psybalazs & the critique below by @RCarhartHarris. Clearly Robin's SSRI vs psilocybin is the best & only study to compare head to head & I think psychedelics likely offer improved functioning, limited administration...
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
Genentech Exits SMA Program, Boosting SRRK’s Lead
Very positive development for $SRRK - Genentech ceases drug development to spinal muscular atrophy - puts SRRK clearly in the lead Cantor on it now https://t.co/IjCyBm2Kcv
Gene Therapy Delivers Real Results Amid Hype
Katrine Bosley: There's no question “hopes and aspirations” got ahead of the pace of any new science on gene editing #STATBreakthrough Seng Cheng: “The promise of gene therapy is correct. I think it has made that promise. and that's demonstrated by...
Early Experiments May Mislead; Models Need Validation
Jane Grogan from $BIIB again, on the fundamental challenge of drug discovery: do you believe what early experiments tell you? "We need to understand when [models] can be predictive and when they help at answering our hypothesis... “If you...
China Produces Triple U.S. PhDs, Boosting Biotech Innovation
$BIIB’s Jane Grogan on the impact of China on biotech innovation. “There's three times more PhDs that have been given last year in China than in the US. ... That's a lot of bright young things out there who are going...
Higher GLP‑1 Doses Show Cardiac Safety Signal
This definitely looks like a cardiac safety signal at the higher doses. The GLP-1's all slightly increase pulse rate as a starting point.
Breakthroughs Take Years, Then Appear Overnight
$BMY chief scientist Robert Plenge: says there is “a joke” drug developers often repeat. “It's an overnight sensation a decade in the making. These things can actually be going on for a very long time, and then suddenly the field catches...
FT Highlights CRUK's Aleta CAR‑T Trial in Lymphoma
The Financial Times covered recent initiatives at Cancer Research UK (CRUK) including a nice piece on the ongoing Aleta Biotherapeutics clinical trial in CD19-CAR-T treated B cell lymphoma patients. https://t.co/9sDZEItapu
Oral Ozempic Trials Fail to Show Alzheimer's Benefit
The negative oral Ozempic randomized trials (EVOKE, EVOKE+) for Alzheimer's disease have now been published @TheLancet https://t.co/Xx0YknTSC2
GLP‑1 Drugs May Protect Heart, Vessels, Kidneys in Type 1 Diabetes
GLP-1 drugs have established benefit for Type 2 diabetes. New data suggests that extends to Type 1 diabetes, with heart, vascular, and kidney protection https://t.co/nZDKHoFIRm https://t.co/lTHIgoj4ir
Prioritize Affinity, Specificity, Then Stability in Nanobody Design
Okay here's a game - you're making nanobody therapeutics. AI can help you optimise them, but you need to prioritise order of feature importance. What is your order? Affinity Specificity Expressibility Clinical toxicity In vivo stability Purified stability Patentability Other?
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...
Congruence Launches First Drug, Secures $40M Funding
Exclusive: Clarissa Desjardins' newest biotech has put its first drug in the clinic + raised $40M more. Congruence also expects to put 2 more molecules into clinic in early 2027, seeing its platform driving its R&D efficiency: https://t.co/pzxcnmKFBj
Implant vs Meds: Randomized Trial Questions LAA Closure Benefit
Questioning the benefit of an implant to close the left atrial appendage vs medical therapy in a randomized trial @NEJM https://t.co/N0hpdIfmLe https://t.co/8bkk8FzLuY
Stopping GLP‑1 Therapy Quickly Erodes Cardiovascular Protection
A new finding after stopping GLP-1 drugs in a large cohort with Type 2 diabetes: rapid erosion of CV benefit/protection New @bmj_latest by @zalaly @Biostayan https://t.co/JG6yB8wTJJ See thread: https://t.co/6JvfIIjVBw https://t.co/wUgWhvs9Yy
CBER Updates SOPs, Enables RMAT for Held Therapies
CBER changes its standard operating procedures to open the door to RMAT designations for therapies on clinical hold - https://t.co/bZmhga8xxC
In‑body Genome Editing Promises Cheaper, Practical CAR‑T Therapies
What if we could engineer T cells in the body, making CAR T vs cancer, autoimmune diseases, and other applications far more practical, much less expensive? A step forward today via genome editing T cells https://t.co/FjgQA8NUlu https://t.co/XXk43WL1ac https://t.co/rpC7eCAlJH
Science's AI Model Explosion Is Just
We're about to see an explosion of AI models for Science like Andre's LigandForge and I'm here for it!
Repeated Psilocybin Doses Yield 73% OCD Response
A randomized clinical trial of repeated doses of psilocybin for the treatment of obsessive–compulsive disorder 73.3% were responders (⩾35% reduction in YBOCS scores), with 40% in remission. These effects diminished but remained substantial at 6 months. https://t.co/tp2SPIKDrB
MRK Unveils PD-1/VEGF Bispecific at AACR26
$MRK's PD-1 x VEGF bispecific reveal, & other key #AACR26 presentations, via @APEXONCO -> https://t.co/ob1eswxCWF $RVMD $AMGN $GILD $BCYC $SDGR
RBC Flags Revolution, Xenon, Arrowhead as Top Biotech Takeover Targets
RBC: Revolution, Xenon, Arrowhead among top #biotech takeover targets https://t.co/h1toH9szrU by @realJacobBell $ARWR $XENE $RVMD $DYNE
5‑MeO‑DMT Shows Promise for Treatment‑Resistant Depression
Emerging evidence supports 5-MeO-DMT as a promising, ultra-short-acting psychedelic for treatment-resistant depression and other psychiatric conditions, warranting larger randomized controlled trials. https://t.co/6CcEOSat1F
Mitochondrial Transfer Shows Promise Against Parkinson’s in Animals
Potential of mitochondrial transfer to prevent or treat Parkinson' s disease, in mouse and monkey models @CellCellPress https://t.co/c0oqfagddX https://t.co/KyBc1zQttc
Comparing Psychedelic Trials to Antidepressants Skews Results
Does it stack the deck in favor of the standard antidepressants to pull data from placebo-controlled trials for one condition (the psychedelic) and compare it with single condition data for the standard antidepressants? Also...
New FDA Draft Bayesian Guidance Discussed with Dr. Gibson
Thrilled to be interviewed by imminent cardiologist and clinical trialist Mike Gibson yesterday about the new draft Bayesian guidance at FDA: https://t.co/I3uNISITKQ #Statistics #bayes #pharma #clinicaltrials #rct @CMichaelGibson
Europe's Biotech Funding Gap Spurs Urgent Investment Push
Europe's Biotech Investing Crisis: @OtelloVC Otello Stampacchia of Omega Funds discusses the drive to increase EU biotech investment on The Long Run. Sponsored by @AlphaSenseInc & Dash Bio https://t.co/ORIeN8IDEq
China’s CCP Stifles Cure Innovation, FDA Lag Exposes Gap
My @8VC partner Francisco is giving key testimony today about CCP crushing our ability to develop cures. China has legit innovators - but also a culture of stealing, and working around patents. Our legacy FDA is so slow that they can...
Key Strategies for Building Biotech: Science, Tech, Finance
It was a fun privilege to share a few thoughts on building biotechs with Nature Biotechnology for their 30th Anniversary issue - covering a broad range of topics including translating science into medicines, technology cycles, and financing biotech companies... https://t.co/LbnWMSJI9f
Excalipoint Secures $69M for Next‑Gen T‑Cell Engagers
Chinese biotech startup Excalipoint banks $69M for next-gen T cell engagers https://t.co/809RqRWJNL by @gwendolynawu #biotech #startups