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Every morning
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Tuesday recap
GH Research’s inhaled mebufotenin (GH001) demonstrated ultra‑rapid, profound antidepressant effects in a Phase 2b trial for treatment‑resistant depression, with 73% of patients in remission after six months versus ~11% for standard oral therapies. Early‑stage studies in postpartum depression and bipolar disorder showed similarly swift remission, including a ten‑patient postpartum cohort where all achieved remission. GH001’s inhaled delivery yields onset within seconds and a brief 11‑minute psychoactive window, enabling same‑day discharge and potential integration into existing Spravato‑style clinic networks. The company is now preparing a pivotal Phase 3 program while remaining well‑capitalized and publicly traded.
Gilead Sciences agreed to acquire the remaining shares of Arcellx for an equity value of $7.8 billion, paying $115 per share—a 79.4% premium to the prior close. The deal secures full rights to Arcellx’s BCMA‑directed CAR‑T therapy anito‑cel, which is in...
Novo Nordisk’s experimental obesity drug CagriSema delivered a 23% average weight loss in the 84‑week REDEFINE 4 Phase 3 trial, but fell short of Eli Lilly’s tirzepatide (Zepbound) which achieved 25.5%. The head‑to‑head study failed the primary non‑inferiority endpoint, prompting a 15% drop...
Gossamer Bio announced that its Phase 3 trial of seralutinib for pulmonary arterial hypertension missed its primary endpoint. Patients on seralutinib walked 13 meters farther than placebo over six minutes, but the difference lacked statistical significance. The trial also reported cough...
Patient-Centered Care in Hematology: How Side Effect Severity Influences Decision-Making Autonomy in Relapsed Refractory Multiple Myeloma [Dec 8, 2024] Martinez et al. #ASH24 Abst 706 https://t.co/l7SHPBwbna #mmsm HT @Taxkourel
Lenalidomide + high-dose dexamethasone (RD) vs lenalidomide + low-dose dexamethasone (Rd) as initial therapy for newly diagnosed multiple myeloma: an open-label RCT [10/22/2009] @VincentRK et al. @TheLancetOncol https://t.co/hXxlFINejC #NCT00098475 #EAonc E4A03 #mmsm #caxtx #ctsm https://t.co/FjBnt6yIox
At the BIO Investor Growth (BIG) Summit, IBD Ventures’ associate director Nicole Schwerbrock discussed how venture philanthropy and patient advocacy are reshaping investment in inflammatory bowel disease (IBD) research. She explained that patient‑driven funding models complement traditional venture capital by...
#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/QdWETmSoiR
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/paGRlHV1Dv
Researchers discovered that age‑associated B cells (ABCs) actively impair immune function in older mice. Permanent genetic ablation of B cells reduced CD4 T‑cell aging, restored naive T‑cell pools, and prevented T‑cell receptor clonal restriction. The study identified B‑cell intrinsic insulin‑receptor...
Researchers identified oligodendrocyte myelin glycoprotein (OMG) in blood as a marker inversely associated with cortical amyloid‑β deposition and neurodegeneration. Large‑scale plasma proteomics across more than a dozen cohorts showed lower OMG levels in individuals with Alzheimer’s disease, other dementias, and...
Gilead Sciences announced a $7.8 billion acquisition of Arcellx, pricing the deal at $115 per share—a 79% premium to the prior close. The agreement includes an additional $5 per share contingent on future sales milestones. Central to the transaction is anito‑cel,...
Jay Bregman, CEO of Andel, says the FDA’s pending crackdown on non‑FDA‑approved GLP‑1 compounds is long overdue and will target the burgeoning market of compounded semaglutide. He estimates roughly 1.5 million patients currently rely on these unapproved products, a figure he...
Partner Therapeutics presented post‑hoc data from its Phase II eNRGy study of zenocutuzumab (Bizengri) in 27 patients with advanced NRG1‑positive non‑small cell lung cancer who received at least three doses beyond radiographic progression. Eight patients stayed on therapy for six months...
Novo Nordisk’s next‑generation obesity drug CagriSema failed to demonstrate non‑inferiority to Eli Lilly’s tirzepatide in the 84‑week REDEFINE 4 study, achieving 20.2% weight loss versus 23.6% for tirzepatide. The open‑label trial showed the two treatments were not statistically equivalent. Following the announcement,...
PRECISIS GmbH’s EASEE® epicranial neurostimulation system offers a minimally invasive alternative for drug‑resistant focal epilepsy. The implant, positioned under the scalp atop the skull, delivers targeted electrical pulses without opening the skull, achieving a median 68% seizure reduction over two...
Johnson & Johnson released long‑term QUASAR extension data for Tremfya (guselkumab) in ulcerative colitis, showing sustained efficacy through week 140. Clinical remission was achieved by 80.8% of patients, with 78.6% attaining histo‑endoscopic improvement and 53.6% reaching endoscopic remission. Approximately 89% of...
Researchers have demonstrated dissolving microneedle (DMN) arrays fabricated using resin‑based 3D‑printed master molds, enabling drug delivery through the inner cheek. The workflow pairs rapid SLA/DLP printing with polymer micromolding, allowing design changes from CAD to mold within hours. Mechanical, dissolution...
Vanda Pharmaceuticals announced that the U.S. FDA has approved Bysanti (milsaperidone), an active metabolite of iloperidone, as a first‑line therapy for acute manic or mixed episodes in bipolar I disorder and for schizophrenia. The approval is based on demonstrated bioequivalence...
In February 2024 Vertex and CRISPR Therapeutics launched Casgevy, and bluebird bio (now Genetix Biotherapeutics) launched Lyfgenia as the first FDA‑approved gene therapies for sickle cell disease. Two years later, uptake remains modest—only 64 patients received Casgevy and just over 100...
Madrigal’s Rezdiffra became the first approved therapy for metabolic dysfunction‑associated steatohepatitis (MASH), generating $817 million in revenue within six quarters and prompting a wave of big‑pharma acquisitions focused on FGF21 analogs. In 2025, GSK, Roche and Novo Nordisk spent nearly $10 billion acquiring...
CVRx reported fourth‑quarter 2025 revenue of $16 million, a modest 4% rise, while gross margin improved to 86% driven by higher selling prices and manufacturing efficiencies. The company added 13% more active implanting centers, reaching 252, and expanded U.S. sales territories...
GeneDx Holdings reported Q3 2025 revenue of $116.7 million, a 52% year‑over‑year increase, driven by a 66% jump in exome and genome revenue to $98.9 million and 25,702 tests performed. Adjusted gross margin rose to 74% as higher reimbursement rates and a...
Summit Therapeutics reported a strong cash position of $713 million with no debt at the end of 2025, while GAAP operating expenses fell to $225 million despite a rise in non‑GAAP R&D spend. The FDA accepted the Biologics License Application for ivonesumab...
Myriad Genetics reported Q4 2025 revenue of $209.8 million, essentially flat year‑over‑year but above its pre‑announced range, while test volume rose 2% to 382,000. Strong double‑digit growth was seen in MyRisk hereditary cancer (14% affected, 11% unaffected), Prolaris prostate cancer (12%...
Tarsus Pharmaceuticals reported record 2025 net product sales of $451.4 million, driven by XDEMVY adoption, and issued its first full‑year 2026 guidance forecasting $670‑$700 million, implying over 50% revenue growth. The company highlighted a 44% gross‑to‑net discount, a robust DTC campaign that...
Axsome Therapeutics reported a 66% revenue surge to $639 million in 2025, driven by strong growth in Auvelity, Sunosi and the newly launched Cymbravo. Net loss narrowed to $183 million, a 35% improvement year‑over‑year, while cash rose to $323 million. The company secured...
Recent analyses reveal a steady rise in medical stimulant prescriptions among U.S. adults, with a 30% increase between 2013 and 2022. The expansion of telehealth, especially after COVID‑19, contributed to roughly 40% of new adult ADHD initiations, while FDA alerts...
BioMarin Pharmaceutical raised its fiscal 2025 total revenue guidance, now starting at $3.15 billion, and reaffirmed Voxzogo revenue between $900 million and $935 million. The company lifted non‑GAAP operating margin guidance to 26‑27% and lifted diluted EPS guidance to $3.50‑$3.60. A $221 million IPR&D...
Artificial‑intelligence tools are now capable of designing genes that function in mammalian cells and have even generated a fully synthetic virus, marking a watershed for generative biology. In his new book, Adrian Woolfson outlines how computational models could evolve into...
Japan’s health ministry is set to grant conditional approval to two first‑of‑a‑kind regenerative medicines – Amchepry for Parkinson’s disease and ReHeart for severe heart failure – after tiny phase I/II trials involving seven and eight patients respectively. Both drugs are derived...
Researchers at Tokyo Metropolitan University have engineered a charge‑free polymer‑DNA complex using a thymine‑modified poly(ethylene glycol) (PEG) that binds plasmid DNA via annealing. In mouse models the formulation boosted cellular DNA uptake and gene expression up to 14‑fold compared with...
16-Hour Fasting: A Game-Changer for Cancer Immunotherapy 🧬⏱️ New clinical data in Cell Metabolism (Feb 2026) reveals that strategic fasting is more than just a metabolic trend—it's a powerful tool in oncology. The Key Findings: A 16-hour fasting window "reprograms" T cells,...
A new Nature Communications study reveals two neurobiologically distinct subtypes of adolescent major depressive disorder by analyzing information dynamics in sensory‑association cortices. One subtype shows heightened feedforward signaling, correlating with sensory hypersensitivity and anxiety, while the other exhibits reduced feedback...
I am deeply honored to be included in the inaugural Forbes 250: America’s Greatest Innovators list. Innovation is rarely a straight line, nor an easy journey. Whether it was democratizing education at Coursera or now redefining drug discovery at insitro...
Novavax (unlike the mRNA vaccines) generates antibodies to both S1 and S2 units of the spike protein - explaining why it provides somewhat broader protection. I've personally gotten Novavax every time it was available at vaccine time.
Scientists at McGill University and the Goodman Cancer Institute have engineered nanoparticles that deliver an existing immunotherapy directly to metastatic lymph nodes. The nanocomplex senses a molecule abundant in cancer‑laden nodes, activating the drug only at the disease site while...
The complexity of our immune system is daunting. But there's a path to deconvolute it and understand causal relationships. It's CRISPR. open-access @JExpMed https://t.co/QF6Cvkbfz2 https://t.co/OTL2z1fow0
The role of KRAB zinc-finger proteins in expanding the domestication potential of transposable elements https://t.co/4VtCDQomg6
Scientists at Columbia’s Zuckerman Institute and Cornell’s Maxson lab have linked a pulsed laser to a cryo‑electron tomography (cryo‑ET) microscope, creating a proof‑of‑concept that modulates electron phase to boost image contrast. The technique exploits laser‑induced lensing within a trillionth of...
1/ Sex chromosomes are bias magnets in WGS. Early large-scale genomes often showed weird copy-number or coverage patterns on X and Y that looked like biology, but were really mapping artifacts. https://t.co/YnApmtxHqm
Here is my favourite weekly 🧵- made by my dear friend @BiopharmIQ - which is a comprehensive recap of the past week in BioTech. This 🧵 gathers the best BioTech posts written by some of the best & brightest 𝕏...
Researchers have detailed how focused ultrasound safely and reversibly opens the blood‑brain barrier by transiently reorganizing tight‑junction proteins. The study shows that pulsed ultrasound creates a brief paracellular window that permits therapeutic agents to reach brain tissue without causing inflammation...
The key point here is that the proposed study would not have equipoise because almost certainly some babies in the control arm would get hepatitis from their infected mother.
Submitting an abstract for a late-stage large clinical trial for review at a major conference, where you're asking for an oral, but only having 2 authors should be disqualifying. Really, only *2* people acquired ALL the data for this? Disgraceful...
Researchers have identified the Ephrin‑A1/EphA2 signaling axis as a promising therapeutic target for preventing osteoporotic fractures. Preclinical studies demonstrated that blocking EphA2 activity enhances bone formation and improves microarchitecture in mouse models of age‑related bone loss. The findings suggest that...
By systematically assessing both DNA sequence variants and methylation, we're learning a lot more about risk of (and protection from) colon cancer @ScienceAdvances https://t.co/eA0Y0lrntr https://t.co/5iL4sg0rRC
1/ RNA‑seq batch effects are one of the easiest ways to fool yourself in genomics. They can create beautiful, completely wrong biology if you’re not careful. https://t.co/VBtySwZHVL
Teclistamab–Daratumumab in Relapsed or Refractory Multiple Myeloma [Feb 18, 2026] @End_myeloma et al. @NEJM https://t.co/PTSmdrV2zp #mmsm #tcellrx #ImmunoOnc QUICK TAKES: New phase 3 trial results are summarized in a short video.
Exposure–Response Analyses for Therapeutic Dose Selection of Belantamab Mafodotin in Patients with Relapsed/Refractory Multiple Myeloma [Sep 1, 2021] Ferron-Brady et al. Clinical Pharmacology & Therapeutics https://t.co/58kmDFKRwO #mmsm #oncopharm #caxtx
