Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech rounds
Spanish biotech firm Ona announced an $86.6 million Series B financing, ranking among the largest venture rounds in Spain’s life‑science sector this year. The round was led by Seventure Partners with participation from existing backers and new strategic investors. The funding will be used to scale Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical programs.
Earendil Labs to Scale AI-Driven Biologics Platform with $787 Million Funding
Earendil Labs announced a $787 million financing round, the largest recent capital raise for AI‑enabled drug development. The funding, led by Sanofi and the Biotech Development Fund, will be used to scale its AI‑driven biologics discovery platform, expand R&D teams, and push more than 40 antibody programs toward clinical trials, including Phase II candidate HXN‑1001. The company markets its platform as a continuous production engine that integrates computational modeling with experimental validation. Strategic collaborations with Sanofi broaden the reach into autoimmune and oncology indications.
The New Chapter Added to My Vaccine Book
Dr. Gator’s latest update adds a new chapter to *Between a Shot and a Hard Place*, reflecting a year of rapid shifts in U.S. vaccine policy, legal battles, and scientific debate. The chapter details CDC schedule changes that now mirror...
Imatinib
Imatinib (Gleevec®/Glivec®) is an oral ATP‑competitive inhibitor of the BCR‑ABL fusion tyrosine kinase, approved by the FDA in 2001 for Philadelphia chromosome‑positive chronic myeloid leukemia and other malignancies. The drug emerged from high‑throughput screening, structure‑activity relationship optimization, and structure‑based drug...
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...
UK’s NICE Revisits Lilly, Eisai Alzheimer's Drugs Under New Pricing Threshold
The UK’s National Institute for Health and Care Excellence (NICE) has reopened its evaluation of Eli Lilly’s donanemab and Eisai’s lecanemab after initially rejecting them on cost grounds. A newly‑introduced cost‑effectiveness threshold of roughly £20,000 per quality‑adjusted life year (QALY)...
Pharma M&A Roundup: Novartis to Acquire Pan-Mutant-Selective PI3Kα Inhibitor From Synnovation Therapeutics, Collegium to Acquire Azstarys From Corium Therapeutics
Novartis announced a deal worth up to $3 billion to acquire SNV4818, a pan‑mutant‑selective PI3Kα inhibitor aimed at hormone‑receptor‑positive, HER2‑negative breast cancer. The transaction includes $2 billion upfront and potential milestones exceeding $1 billion, with closing expected in early 2026. Collegium Pharmaceutical agreed...
How to Get Specialty Drug Launches Right From Pre-Launch to Patient Access
ConnectiveRx VP Kristine McGaughey emphasizes that an 18‑to‑24‑month pre‑launch window is critical for specialty drug rollouts. Early alignment of market access, patient services, pricing, and hub teams prevents day‑one friction and enables rapid patient access. She contrasts emerging biopharma, which...
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
AI Model Uses 3D Lipid Structures to Improve mRNA Nanoparticle Delivery
Researchers at China’s National Center for Nanoscience and Technology have developed an AI‑driven platform that screens ionizable lipids based on their three‑dimensional conformations. The model identified a novel lipid, P1, which delivers mRNA 14.8 times more efficiently than the clinically...
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
The Ron Lanton Report: When Policy Becomes a Capital Event
The Ron Lanton Report highlights a fundamental shift where regulatory signals now drive company valuations and strategic choices before formal rules are enacted. Investors are pricing policy risk early, treating anticipated regulations as a capital‑allocation factor. The discussion spans FDA...
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
Pharma Pulse: FDA Greenlights Higher-Dose Wegovy with Pricing Commitments, and the State of the US Generics Market
The FDA granted a 54‑day fast‑track approval for Wegovy HD, a 7.2 mg semaglutide injection that delivered a 20.7% average weight loss in trials, under the National Priority Voucher program. In return, the maker committed to specific Medicare and Medicaid pricing...
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
Orthogonal Tri‐Modular Coiled‐Coil Assembly for Programmable Multi‐Cargo Display on Escherichia Coli Nissle 1917
Researchers have engineered a tri‑specific scaffold cell (TriSC) platform in the probiotic strain *Escherichia coli* Nissle 1917 that uses three orthogonal coiled‑coil motifs to create a programmable, reversible surface. The system enables simultaneous, selective attachment of multiple nanobody cargos and...
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...
Roche Ends Run for Muscular Atrophy Drug, Leaving Door Open for Competitors
Roche announced it will discontinue development of its anti‑myostatin antibody emugrobart for spinal muscular atrophy (SMA) after the Phase 2/3 MANATEE trial failed to demonstrate consistent muscle‑growth or motor‑function benefits. The study enrolled 259 patients and compared emugrobart plus Evrysdi against...
Rhythm Obesity Drug Wins Broader Use From FDA
The FDA approved Rhythm Pharmaceuticals' once‑daily injection Imcivree for acquired hypothalamic obesity in adults and children aged four and up. Clinical trials showed an 18‑percentage‑point weight‑loss advantage over placebo, making it the first therapy for this rare, brain‑injury‑driven condition. Rhythm...
Genentech Culls Muscle-Preserving Drug in Genetic Diseases, Raising Questions About Obesity Trial
Roche’s Genentech has halted the Phase III development of its muscle‑preserving therapy for spinal muscular atrophy (SMA) and facioscapulohumeral muscular dystrophy (FSHD). The decision follows disappointing efficacy signals and safety concerns that emerged in late‑stage trials. The program also included an...
Appeal Finds NICE Must Reconsider Alzheimer's Drugs
NICE has agreed to revisit its June 2025 rejection of Eli Lilly’s Kisunla (donanemab) and Eisai’s Leqembi (lecanemab) for NHS reimbursement. The appeal will send the dossiers back to the appraisal committee to re‑examine clinical benefits, long‑term data, infusion costs and unpaid...
Glioblastoma Hijacks Sugar Metabolism to Evade Immune Attack
Northwestern researchers discovered that microglia within glioblastoma uniquely express the fructose transporter GLUT5 and metabolize fructose to suppress immune activity. In mouse models, genetic deletion of GLUT5 halted tumor growth and provoked a strong CD8⁺ T‑cell response. The study, published...
Shionogi Enrols First Patients in Esprit Trial for Pompe Disease
Shionogi announced the first patient enrollment in its global Phase II Esprit trial, evaluating the oral substrate‑reduction therapy S‑606001 in adults with late‑onset Pompe disease. The 52‑week, double‑blind, placebo‑controlled study will run across the EU, the UK and the United States,...
First-of-Its-Kind Implant Could Transform Tissue Loss Treatment
Researchers at Technion’s Levenberg Laboratory have created a first‑of‑its‑kind three‑dimensional implant that merges muscle, fat, a hierarchical blood vessel network and, uniquely, a lymphatic system. The construct is printed with a custom extracellular‑matrix bio‑ink and matured in a flow‑controlled bioreactor....
Mind-Altering Substances Are (Still) Falling Short in Clinical Trials
Psychedelic research has surged, but recent psilocybin trials reveal modest benefits that fail to outpace placebo. A German study with 144 treatment‑resistant depression patients found no statistically significant advantage for high‑dose psilocybin. An open‑label review of 24 trials concluded psychedelics...
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
LivaNova’s Aura6000 System Secures the US FDA Premarket Approval to Treat Obstructive Sleep Apnea (OSA)
LivaNova received FDA Premarket Approval for its aura6000 neurostimulation system, targeting adults with moderate‑to‑severe obstructive sleep apnea (OSA) who cannot use or have failed first‑line therapies. The approval is based on the OSPREY randomized controlled trial, which showed a 65%...
STAT+: Novartis Picks up Experimental Breast Cancer Therapy for $2B
Novartis announced the acquisition of experimental breast‑cancer drug SNV4818 from Synnovation Therapeutics for a $2 billion upfront payment, with up to $1 billion in milestone‑based earn‑outs. The oral agent is in a Phase 1/2 study targeting mutant PI3Kα in HR‑positive, HER2‑negative tumors while...
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
Modifying T Cell Receptor Improves Targeted Cancer Therapy
Researchers from UCLA, Stanford, Utah, and Columbia have engineered T cell receptors to strengthen catch‑bond interactions with prostate cancer antigens, improving cytotoxic function. By altering just one or two amino acids in the TCR, the modified cells exhibit longer bond...
Lab-Grown Oesophagus Restores Pigs’ Ability to Swallow
Scientists at University College London have engineered bio‑grown oesophageal segments using patient‑derived stem cells and implanted them into minipigs, restoring normal swallowing. The grafts were seeded onto decellularized scaffolds, covered with a biodegradable mesh, and integrated functional muscle, nerves, and...
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...
Magnetic Fields Guide Lab-Grown Blood Vessels Into Precise Patterns for Drug Testing
Researchers at the Institute of Physical Chemistry (IPC PAS) and the University of Warsaw have created a magnetic‑field‑driven system that arranges endothelial‑cell‑coated microparticles into predefined lattices, prompting the growth of microvascular networks with precise architecture. By using super‑paramagnetic beads and micromagnets,...
Recursion’s Khan: AI Will Be Judged by the Medicines It Makes
Recursion Pharmaceuticals CEO Najat Khan says AI’s worth will be judged by the medicines it helps create, not by the sophistication of the tools. She emphasizes deploying AI selectively in chemistry, trial execution, and out‑of‑domain predictions to generate clinical proof...
STAT+: Trump’s Medicare Director Seeks to Rein in Expectations for TrumpRx
Chris Klomp, the HHS Medicare director, warned that President Trump’s TrumpRx drug‑discount platform is not intended for the majority of Americans with health insurance. He clarified that the program targets cash‑pay patients, leaving the 170 million commercially insured and 68 million Medicare...
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...
BIOTECanada Welcomes the Announcement of the Pharmaceutical and Life Sciences Sector Task Force
BIOTECanada has praised the federal government’s launch of the Pharmaceutical and Life Sciences Sector Task Force, signaling a strategic push to strengthen Canada’s biotech and pharma ecosystem. The task force is tasked with reviewing investment incentives, regulatory frameworks, and market...
First Surrogate Endpoint in Osteoporosis Clinical Trials with FNIH’s Dr. Tania Kamphaus — Episode 247
On December 2025 the FDA officially qualified dual‑energy X‑ray absorptiometry (DXA) bone density scans as the first surrogate endpoint for fracture outcomes in osteoporosis trials involving post‑menopausal women. The qualification, achieved through a request from the Foundation for the National...
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...
Could Data From 100 Million Species Help Cure Disease? One Startup Is Betting on It
Basecamp Research announced the launch of its Trillion Gene Atlas, a project to collect and model genomic data from over 100 million species, expanding known genetic diversity a hundred‑fold. Backed by $85 million in venture funding, the initiative partners with Anthropic, Ultima...
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...
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...
FDA Drug Safety Communication: Updated Information on 32 Mg Intravenous Ondansetron (Zofran) Dose and Pre-Mixed Ondansetron Products
The FDA announced that the 32 mg intravenous dose of ondansetron (Zofran) will be withdrawn from the market due to its association with QT‑interval prolongation and the risk of Torsades de Pointes. The agency is coordinating a voluntary recall of all...
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.
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
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.
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