Elizabeth Cairns' piece on $Novo is reminiscent of Bristol Myers' strategic fail against Merck and Keytruda. They were focused on the wrong things, made bad trial design decisions and got stuck in the mud of their own making. Everyone should read this. It's a cautionary tale for the industry.
“You can’t stand there after the fact and blame the open-label design,” he said. “You designed it. You designed a noninferiority margin. You picked the patients. And then the trial doesn’t work.” https://t.co/acS5i5s5NU

1/@TesseraTx has announced that the U.S. FDA has granted Fast Track and Orphan Drug designations to TSRA-196 - its leading In Vivo Gene Editing program which is being jointly developed with $REGN, for the treatment of patients with alpha-1 antitrypsin deficiency...
New: Generate caps a strong month for #biotech IPOs with $400M offering https://t.co/HzslZOPcwM by @gwendolynawu $GENB $AZN $AMGN #IPO
(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...
We'll be gearing up today's webinar on biotech venture capital at the top of the hour. Great guests, great topics. If this is a central issue in your life, tune in. Thousands have signed up already. https://t.co/c16y04mFsD
You've got to watch this Makary interview on CNBC. He attacks a drug, hits the Old FDA and circles all the wagons around Vinay Prasad, who "loves President Trump" and faces a fatwa (WSJ). Makary is getting major props for...

Direct-to-consumer gut microbiome assessments are unreliable, problematic: a review of 7 companies with significant methodologic issues https://t.co/atwmZUNjVE https://t.co/SqlMljuW0y
FDA Commissioner Marty Makary defends rare disease drug rejections, Vinay Prasad "Vinay Prasad is a genius,” Makary said. “He’s on loan from UCSF. He’s published over 500 scientific peer-reviewed articles, and some of the drugs they are criticizing him for...

Head-to-head trial of the oral GLP-1 drugs in people with T2D. Orforglipron was superior https://t.co/9B7RXIgdJY
$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...

We're making headway for off the shelf preventive cancer vaccines in people with hereditary forms of cancer, e.g. Lynch syndrome https://t.co/F3LqKS46BQ
$RVMD was collaborating with $TNGX in PRMT5, but now it's working with $BMY (remember that ex MRTX project?). Via @ByMadeleineA @ApexOnco -> https://t.co/mGZjKOiB7p (And $AMGN still dose optimising.)

The combination of GLP-1 drugs (such as Ozempic) and healthy lifestyle factors was associated with less major adverse cardiovascular events in ~100,000 people with T2D @TheLancetEndo https://t.co/phNzEBy7zY https://t.co/DDVi8uedRP
Psychedelic tape still trades on vibes > policy. Surgeon General nominee Casey Means says she “would not recommend” psilocybin to the American people today. Yes, she nods to “exciting work” in PTSD and veterans. But that is a far cry from...
An experimental therapy for a rare blood cancer was on the path toward approval by the Food and Drug Administration last year — with internal reviewers recommending it be cleared — before the agency rejected the drug last month, @adamfeuerstein...
Interesting read: the thread and the story. A drug that was approved in Europe in 2022, which looked like it was on track for #FDA approval, instead is rejected. What changed at FDA? 🤔
The Federal Register notice for the (now) March #ACIP meeting is now up & it's ... puzzling. Possible vote on Covid vaccine injury? ACIP can recommend changes to the way a vaccine is used, for safety reasons. But vaccine injuries aren't...
$LTRN is a live experiment in our thesis: AI doesn’t need to invent new drugs. It needs to improve probability of success in humans. If RADR consistently enriches responders, that’s durable value. If not, platform premium disappears. Listen to the podcast here: https://t.co/ZAg5btpcIK
interesting and different efficacy outcomes in ASyS and SSc autoimmune patients treated with blinatumomab (CD3/CD19) and teclistamab (CD3/BCMA), respectively, + Rituximab maintenance. Potential for targeting both BCMA and CD19, especially in ASys. https://t.co/Dyq7h9EeGo

One company is unlocking gene delivery to overcome the challenges that held back the promise of genetic medicine. If you want to learn more, they’ll be hosting an exclusive fireside chat at 9am Thursday, May 7th at SynBioBeta. Last year at...

A randomized trial of a stem cell therapy vs placebo for aging frailty showed durable and dose-dependent improvement in the 6-minute walk test @CellStemCell https://t.co/2jOYuIgUrY https://t.co/04mEV7OdLo
Online Patient Communities for the E-Patient “Betwixt & Between” a New Pt & an Expert [Aug 29, 2013] @mtmdphd #ASCOConnection https://t.co/IxinXTK715 #mmsm
Identification of Significant Barriers to Accrual (BtA) to @theNCI Sponsored Multiple Myeloma – Clinical Trials (MM-CT): A Step towards Improving Accrual to #ClinicalTrials - @mweissmdphd et al. #ASH12 Abstract 3165 https://t.co/i4zO7ktqHJ #mmsm #caxtx #ctsm
#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

.@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/uikUnnJd3n

Guidelines for determination of the number of prior lines of therapy in multiple myeloma [Aug 13, 2015] @VincentRK , Richardson, San Miguel @BloodJournal https://t.co/Tdmr2gTUi6 #mmsm #ctsm #ClinicalTrials https://t.co/ZOobout75Q

Comparative Effectiveness and Safety of PI-Rd Triplets in Relapsed/Refractory Multiple Myeloma: INSIGHT-MM Data Analysis [Jan 13, 2026] @NoemiPuigM et al. European Journal of Haematology https://t.co/Vf7USecLlR #NCT02761187 #mmsm #openaccess https://t.co/C5Ya6TTQfo

Assessment of biological age integrating electronic medical records and multi-omics increases accuracy for predicting outcomes @NatureAging https://t.co/7y60SSPTQf https://t.co/u02OlWMdQz

What gets missed in the hurly burly of a conference with many highlight reels? Here we explore emerging trends to watch out for when it comes to protein degradation/glues, & combination opportunities w/ CAR-T cells. Time for some...
The FT's report on GSK's $950M deal to buy 35Pharma and its pulmonary arterial hypertension drug includes an interesting unnamed insider's quote that new CEO Luke Miels is going after assets where "the science is relatively settled." Maybe leaning into...

Strong claim that needs independent replication: "Our findings provide compelling evidence that circulating smRNAs—especially piRNAs—are powerful predictors of survival in older adults and potential biomarkers of longevity" https://t.co/oi4220zxGY https://t.co/yk3MX4VdQj
With Vivtex deal, Novo gains a chance at better oral obesity drugs https://t.co/DhIelbC9jI by @gwendolynawu $NVO #obesity

Assets from China are ubiquitous but China is no longer the 'bargain basement' in terms of deals. Will be interesting to see how rising prices impact deal interest https://t.co/yM3P28QCDc $XBI $IBB $BBC https://t.co/W61XACPX7O
President Trump negotiated lower prices for some drugs on TrumpRx, but discounts only apply to cash paying patients not using their insurance. Many drugs already had big discounts through manufacturers or coupons, and some have generic equivalents that are already...
President Trump has called for his deals with drug companies to be enacted into legislation. It's hard to know what that means since the deals are not public.
Lot's of requests to discuss the Wedbush report on $ABVX today. It's a doozy. Obviously I'm a biased bull so sure, take my thoughts with a grain of salt, but this is truly, awful work from my perspective....
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.
$HELP adds former Pfizer CMO Dr. Freda Lewis-Hall to its board. Big pharma credibility bump. Now the pipeline has to earn it. https://t.co/CK7PsBE9B8
If FDA is serious about moving with urgency in rare disease, the key question is evidentiary standards. Speed matters. But clarity on what constitutes “adequate and well-controlled” in ultra-small populations matters more. Predictability > rhetoric https://t.co/XVXg71NmGq

There is one major barrier blocking AI from truly reshaping biology and no one is talking about it. Biology is not static. Most AI breakthroughs so far have treated proteins, cells, and systems as frozen objects. That abstraction worked to get...
Given 1mg/week of Zepound showed 0.7% reduction in A1c in trials, and so is actually an efficacious dose, this microdose would just be $30/month. (Would imply using for longer than recommended after opening).
The evolution to hepta-refractory myeloma involves sequential loss of CD38, BCMA and GPRC5D [Feb 17, 2026] Riedhammer et al. @LeukemiaJnl https://t.co/EyGBz7sEQz #mmsm #PrecisionMedicine #caxtx
Vir climbs on Astellas deal, study results for ‘masked’ T cell engager https://t.co/336c9FJdjt by @Lilah_Alvarado $VIR + 27% $JANX $ALPMY
Novo to cut GLP-1 drug prices; Palvella soars on study data https://t.co/UAs4IETgPX $NVO $PVLA+ 32% $GOSS $VNDA $PFE #biotech
Failing to find a buyer, BioMarin is voluntarily withdrawing Roctavian from the market. A fitting end for what was once one of the highest-profile gene therapies in development — and a reminder of the gap between promising science and a viable...
Novo, searching for a spark, spotlights new data for three-pronged obesity drug https://t.co/pPo9Oc1wlF $NVO $LLY #obesity
FDA Issues Plausible Mechanism Pathway Draft Guidance to Spur Innovation for Individualized Therapies https://t.co/OZW6Q27zMa via @Inside_PM
Immedica wins accelerated approval for once-rejected rare disease drug, plans to sell PRV - https://t.co/wU3AfqQzjw

Our perspectives piece on Agentic AI for biomedical research has been published in Nature Biotechnology https://t.co/PpFeGcvv0x #agenticAI #artificialintelligence #agents https://t.co/xoADUc7T7K