Flare's GU26 Targets Undruggable PPAR‑γ, Yet Caveats Remain
Who's heard of Flare Therapeutics? #GU26 late-breaker shows promise in hitting "undruggable" PPAR-γ target, with a few major caveats. Via @ApexOnco -> https://t.co/1GepvywtL0
Europe Braces for Fallout of Trump's Drug Pricing Plan
“Nobody wants to feel cornered:" Europe braces for the fallout of Trump's drug pricing plan https://t.co/HZjZH0mdCU
Advocacy Bias Keeps Unproven DMD Therapy, Blocks HD Progress
The Duchenne muscular dystrophy patient & advocacy community keeps a gene therapy and multiple drugs on the US market, stopping the FDA from taking action, despite failed confirmatory clinical trials showing no efficacy and questionable safety. The Huntington's disease patient and...
FDA Limits NTLA Gene Therapy to Mild Cases, Undermining Demand
$NTLA drug is off hold but now being restricted by the FDA to the least severe/sick patients, which would logically be those least likely to want/need an experimental gene editing therapy. Existing options will dominate. Target/disease selection is a...
MRK's Ovarian Cancer Claim Rejected by FDA, EMA
$MRK claims a survival benefit in ovarian cancer irrespective of PD-L1 status, but FDA & EMA say BS. Via @ApexOnco -> https://t.co/Cld1O7Uuvp #ESGO26
FDA Demands Sham‑controlled Trial for uniQure’s AMT‑130
$QURE AMT-130 update: Still blocked The FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for...
NMN Restores NAD, Shields Heart via SIRT3
According to ITOA, NAD declines with age, causing epigenetic changes & disease susceptibility In 2010, we showed NAD-dependent enzyme, SIRT3, protects the heart 🐁 New study says NAD precursor NMN protects the heart via SIRT3 in mice fed a Western diet https://www.sciencedirect.com/science/article/pii/S0022316626000842
OpenADMET Releases Massive CYP Assay Datasets for Community AI
OK. Here it is. Over the last year, the @OctantBio and OpenADMET team have been hard at work developing scalable, quantitative, data-rich, and low-cost methods for assessing CYP reactivity and inhibition. The interplay between building a data engine and building...
Ginkgo's 2025 Revenue Far Misses $7.6B Promise
Ginkgo reported full year 2025 revenue of $170M at the end of last week. When it went public at a $15B valuation in 2021, they guided to investors that they’d be adding $7.6B in new business PER YEAR in 2025...
Welireg Poised to Broaden Reach per GU26 Data
$MRK Welireg could be about to expand its reach at last. #GU26 data via @ApexOnco with relevance to $RCUS -> https://t.co/k59284INdK
Miami Preps for Biotech Week; BiotechTV Covers Barclays Conference
Looks like Miami is getting ready for biotech week (the week after next). Note: BiotechTV will be broadcasting from the Barclays conference March 10 & 11. https://t.co/ToZH0EgOVz
IL11 Deficiency Causes Female Infertility, Low Weight, Osteoporosis
But note IL11 knockout mice are infertile (females) and have low body weight and some osteoporosis. Still interesting angle though.
Paper on Target Trial Emulation Overlooks Confounding by Indication
How is it that a paper that claims to show how to do target trial emulation does not address confounding by indication and its ramifications for data collection? https://t.co/dzGZZdpBoc #Statistics
Accelerating CAR Engineering Shows Promising Cancer Immunotherapy
Some very good news about engineering our cells vs cancer (accelerating the CAR). A short thread 1. For background, a new 5★ review on cancer immunotherapy @Cancer_Cell https://t.co/1Qz2rCs8I5 https://t.co/sJ6BwaXyjk
Single-Cell Atlas Links Marrow Immune Dysregulation to Myeloma Outcomes
A single-cell atlas characterizes dysregulation of the bone marrow immune microenvironment associated with outcomes in multiple myeloma https://t.co/5t0M2eX0fB https://t.co/p3kv8LEzrC
Generate Biomed IPO Marks AI‑Driven Protein Breakthrough
This morning @generate_biomed celebrated its IPO on the @NasdaqExchange. We founded Generate at @FlagshipPioneer in 2018, asking: What if we could generate novel protein therapeutics using generative AI tools – without relying on traditional trial-and-error discovery? Today’s IPO is a major...
Comparing Lorigerlimab, Volrustomig, Cadonilimab: Design Lessons
An intriguing tale of three cities. How do the PD1xCTLA4 bispecific agents lorigerlimab, volrustomig, and cadonilimab stack up when it comes to design and performance? Some useful and pointed lessons here for biotech execs: https://t.co/xaSW15Fvdl https://t.co/dhXbiI3N6v
Atrium Launches to Tackle Rare Heart Diseases with RNA
Atrium spins out of Avidity, aiming to target rare heart diseases with RNA https://t.co/xLp2oQDHGy $RNA $NVS #startups
De Novo Proteins Expected in Clinics Within Two Years
“The era of de novo proteins is coming a lot faster than probably people recognize,” Generate's CEO Mike Nally tells me. “I think within the next year or two you’ll start seeing de novo designs enter the clinic.” https://t.co/u3WQcDD3jL
AI Automation Threatens Life Sciences Lab Jobs
The Block layoff news was a big deal yesterday. I’m sorry to say that ML driven automated labs are going to do the same to lab jobs in life sciences that AI is starting to do to other industries.
Smartphone AI Beats Endocrinologists in Acromegaly Diagnosis
AI is helping to make the diagnosis of rare diseases. A new multicenter study in Japan showed smartphone AI diagnosis of acromegaly via hands was better than experienced endocrinologists https://t.co/qBiazetToV https://t.co/dSZpJ2us84
Novo's Missteps Echo Bristol Myers' Strategic Failure
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...
Trial Failures Stem From Flawed Design Choices
“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
FDA Grants Fast Track, Orphan Status to TSRA-196
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...
Biotech IPOs Surge with $400M Offering
New: Generate caps a strong month for #biotech IPOs with $400M offering https://t.co/HzslZOPcwM by @gwendolynawu $GENB $AZN $AMGN #IPO
Makary Misrepresents Unresolved Drug Applications, Conflates Therapies
(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...
Biotech VC Webinar Launches with Top Experts, Thousands Registered
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
Makary Slams FDA, Defends Rare Disease Drugs Amid Controversy
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...
DIY Gut Microbiome Tests Are Unreliable and Flawed
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 Chief Backs Rare Disease Rejections, Lauds Vinay Prasad
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...
Orforglipron Outperforms Other Oral GLP‑1s in T2D Trial
Head-to-head trial of the oral GLP-1 drugs in people with T2D. Orforglipron was superior https://t.co/9B7RXIgdJY
FDA Chief Warns Against Approving Risky, Ineffective Rare‑disease Drugs
$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...
Progress Toward Off‑the‑Shelf Vaccines for Hereditary Cancer
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 Switches PRMT5 Partner From T
$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.)
GLP‑1 Drugs Plus Healthy Habits Cut Heart Events
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 Stocks Overhyped as Nominee Warns Caution
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...
FDA Rejects Experimental Rare Blood Cancer Therapy
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...
FDA Rejects Europe‑approved Drug: What Shifted?
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? 🤔
ACIP Meeting Notice Raises Questions on Vaccine Injury Role
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...
AI Should Boost Human Response Rates, Not Invent New Drugs
$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
Dual BCMA‑CD19 Targeting Shows Promise in ASyS
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
AI‑Engineered AAV Capsid Makes Gene Delivery Programmable
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...
Stem Cell Therapy Boosts Frailty Walk Test Performance
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 Communities Bridge
Online Patient Communities for the E-Patient “Betwixt & Between” a New Pt & an Expert [Aug 29, 2013] @mtmdphd #ASCOConnection https://t.co/IxinXTK715 #mmsm
Identifying Key Barriers to Boost Myeloma Trial Enrollment
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
Quadruplet Therapy Effective without Early Transplant in NDMM
#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
Phase 3 Trial Tests Daratumumab Regimens for New AL Amylo
.@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
New Guidelines Define Prior Therapy Lines in Multiple Myeloma
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
PI‑Rd Triplets Show Superior Efficacy And
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
Combining EMR and Multi‑omics Sharpens Biological Age Predictions
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