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.
Denifanstat Posts Positive Phase III Results for Acne Treatment
Ascletis Pharma announced that its oral fatty‑acid‑synthase inhibitor denifanstat (ASC40) achieved positive Phase III results in moderate‑to‑severe acne vulgaris. In a 480‑patient double‑blind trial, 33% of participants reached clear or almost‑clear skin versus 15% on placebo, and an open‑label safety study of 240 patients showed no grade 3‑4 adverse events. The China NMPA has accepted Ascletis’s New Drug Application, positioning the drug for imminent regulatory review. Sagimet Biosciences, the global licensor, is extending development beyond Greater China.
Investors Overvalue Cagrisema Despite
Who are the people who thought cagrisema was worth this much for $NVO? I want to find and study them. The cross trial comparison data that we already had clearly showed Cagrisema was worse than $LLY by essentially the...
Accelerating Next Generation Medicine with New Drug Delivery Platform
Scientists at the University of Nottingham have unveiled a modular materials platform that self‑assembles with RNA to form nanoscale delivery particles. The system uses reversible host‑guest linkages, allowing rapid tuning of stability and behavior for diverse therapeutic needs. In pre‑clinical...
Prime-Edited Therapy Restores Immune Function in Teen
Below is the story of the first patient treated with a prime-edited therapeutic, developed by @PrimeMedicine in a trial led by Dr. Élie Haddad and his team at CHU Sainte-Justine. This teenager suffered from chronic granulomatous disease (CGD), an immunodeficiency,...
Merck’s Oncology Spin-Out: Organizational Design as Patent Cliff Strategy
Merck has created a standalone oncology business unit to confront the concentration risk posed by Keytruda, which now generates over half of its pharmaceutical sales and faces patent expiry around 2028. The restructuring treats organizational design as a strategic lever...
STAT+: Bayer Sues J&J over ‘False and Misleading Claims’ About Competing Prostate Cancer Treatments
Pharmaceutical giant Bayer has filed a lawsuit against Johnson & Johnson, accusing the latter of false advertising around its prostate‑cancer drug Erleada. Bayer claims J&J’s February 2 press release and website slides used flawed data to portray Erleada as more effective...
Gilead Deal Validates FDA One‑Trial Path, Boosts IMMX
$GILD $ACLX $7.8 billion acquisition 68% premium, Ph3 just starting. Validates new FDA policy for a single pivotal trial validated by BLA acceptance. Large pharma moving faster, de-risked. $IMMX thesis and data grows stronger, 95% (CR), same parallel strategy, and clear...
How a Sirtuin Protects Against Brain Diseases
Researchers in Aging Cell reveal that the nuclear sirtuin SIRT6 safeguards brain health by preserving nucleolar integrity and curbing excess protein synthesis. Loss of SIRT6 triggers nucleolar enlargement, heightened rRNA production, and uncontrolled protein translation, leading to protein aggregation and...
Merck Reorganizes Human Health Work Into Two New Units
Merck is reorganizing its Human Health division into two distinct units—Oncology and Specialty, Pharma & Infectious Diseases—to sharpen focus as its flagship immuno‑oncology drug Keytruda approaches patent expiry. Jannie Oosthuizen, formerly head of U.S. Human Health, will lead the Oncology...
The IPO Buzz: Generate Biomedicines (GENB Proposed) Launches $400 Million IPO
Generate Biomedicines, Inc. (GENB) launched a $400 million IPO on Feb 23, 2026, offering 25 million shares at $15‑$17 each on NASDAQ. At the midpoint price of $16, the company would be valued at roughly $2.04 billion. The proceeds are earmarked for two Phase 3 trials...
FDA Unveils Pathway for Ultra-Rare Disease Therapies
The FDA released draft guidance introducing the Plausible Mechanism Framework, a new approval pathway for individualized therapies targeting ultra‑rare diseases. The framework permits sponsors to seek clearance for gene‑editing and RNA‑based treatments when traditional randomized trials are infeasible due to...
Bringing Quantum Ideas to the Messy World of Disordered Proteins
Intrinsically disordered proteins (IDRs) make up roughly 79 % of human cancer‑associated proteins and defy traditional structure‑based drug design. Akshay Uttarkar’s team introduced QuPepFold, a Python package that translates short peptide sequences onto a tetrahedral lattice and uses a CVaR‑optimized...
Key Differences Between Anti‑cel and Cilta‑cel Explain GILD Deal
By chance I was writing about anito-cel vs. cilta-cel on Friday, not knowing $GILD acquisition was coming down the pike. There are some crucial differences between the two products to take note of, which may explain why they were...
FDA Issues Guidance on New Individualized Therapy Pathway
Building off the Baby KJ success story: FDA unveils long-awaited guidance on new pathway for individualized therapies - https://t.co/Uetp1cGFq2
What the FDA’s Priority Voucher Decision Means for Psychedelic Drug Development
The FDA’s Commissioner’s National Priority Review Voucher (CNPV) pilot compresses review timelines to one‑to‑two months for qualifying drugs, but its criteria favor applications with mature data packages. Compass Pathways’ synthetic psilocybin (COMP360) was listed as eligible yet did not receive...
How to Achieve Superior BCMA Response Rates without the Liability of Delayed MNTs
Recent analyses of BCMA CAR‑T therapies reveal that superior response rates can be achieved without the historically accepted trade‑off of delayed movement and neurocognitive toxicities (MNTs). Emerging data pinpoint specific construct features—particularly signaling domains and hinge designs—as the primary drivers...
Karma-Karma-Karma Chameleon
Balancing potency with oral bioavailability remains a core hurdle as drug candidates grow larger and more complex. Researchers now focus on "chameleonicity"—the ability of a molecule to toggle between polar and lipophilic conformations—to reconcile solubility and permeability. The article outlines...
Collaboration at Its Core: Launching Spain’s First Fully Integrated Spatial Omics Platform
IRB Barcelona has unveiled Spain’s first fully integrated spatial omics platform, uniting spatial transcriptomics, proteomics, histopathology, advanced microscopy and bioinformatics under one workflow. The facility draws on five core units to deliver an end‑to‑end pipeline from sample preparation through computational...
SGLT2s Linked to Lower Cardiorenal, Hepatic Risks in Type 2 Diabetes
A Taiwanese retrospective cohort of 24,259 adults with type 2 diabetes and liver cirrhosis found that initiating sodium‑glucose cotransporter‑2 inhibitors (SGLT2is) markedly reduced the risk of end‑stage kidney disease, acute kidney injury, major adverse cardiovascular events, all‑cause mortality, and hepatic decompensation...
BCAX's Phase 3 Ficera Dose Raises Critical Concerns
Why is $BCAX testing a ficera dose in phase 3 that it now says isn't optimal (and other uncomfortable questions)? Via @ApexOnco -> https://t.co/mLWKP6AAZ9 $JNJ $GMAB
Silencing Ghrelin The Hunger Hormone
Recent research reveals that several snake lineages have completely lost the hunger hormone ghrelin and its activating enzyme, yet maintain normal energy balance. Modern anti‑obesity drugs, such as GLP‑1 receptor agonists, achieve weight loss by amplifying satiety signals rather than...
EU Pledges €225m to Develop Next Generation of Flu Vaccines
The European Commission has pledged €225 million through a pre‑commercial procurement model to accelerate the development of next‑generation influenza vaccines. Ten entities, including Sanofi and Bavarian Nordic, will receive eight‑year contracts that cover clinical testing through market authorisation. The funding targets...
Rival Drug Wins, Costs Us Marketing Gold
That feeling you get when you run a head-to-head study and the rival drug beats you out. Spending a fortune to hand your competitor a marketing message. Sometimes it feels like $NOVO is snake bit. $LLY https://t.co/gGUjl6iqPg
Module 2, Section 1: Target Selection Strategy
The module outlines a five‑dimensional framework for target selection, linking biological relevance, drugability, disease impact, competitive landscape, and development risk. It contrasts first‑in‑class and best‑in‑class strategies, highlighting how pioneering mechanisms can command premium market positions. The content identifies oncology as...
Six RNAi Companies to Keep an Eye on in 2026
RNA interference (RNAi) has moved from a scientific curiosity to a fast‑growing therapeutic platform, with the global market projected to reach $1.97 billion by 2032. Six biotech firms—Aerska, ADARx, Alnylam, Arrowhead, City Therapeutics and SanegeneBio—are leading the next wave of innovation...
Bayesian Designs Decide Faster than Frequentist Group Sequential Methods
Goal-Driven Flexible Bayesian Design presentation updated w/comparison of performance of frequentist group sequential designs: https://t.co/QCZ6DQrm07 . Frequentist approach takes far too long to make a decision by controlling something that is NOT an error prob. #Statistics #rct https://t.co/X2knbmzTuX
Trump's Drug Pricing Deals Promise Lower Consumer Costs
My @CNBC segment on how President Trump's drug pricing deals will achieve many of the initial aims set out by tariff proposals, with respect to reshoring drug manufacturing, and how the biggest consumer impact in terms of lower drug costs...
A New Era in Neuropsychiatry: Dr. Velichka Valcheva on GH Research’s Rapid-Acting Breakthrough, GH001
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...
Gilead Shows Belief in Its Partner’s Cancer Treatment with $7.8 Billion Buyout
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’s CagriSema Falls to Lilly’s Zepbound in Daring Head-to-Head Test
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...
STAT+: Gossamer Lung Disease Drug Fails Late-Stage Study, but Company Will Still Seek FDA Approval
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...
Doctor-in-the-Loop AI Enh
Evaluating a doctor-in-the-loop AI-agent for multiple myeloma chart reviews [Nov 3, 2025] Karla Mariana Castro Bórquez et al. @HealthTree #ASH25 abs25-15262 PID 4424 https://t.co/MvBeHqOxCg #mmsm #AI #EMR #RWD https://t.co/onY817NfVr
Histopathology Genomics Predicts Myeloma Risk in 1,429 Patients
Individualized treatment risk stratification using histopathology-based genomics prediction in multiple myeloma: A multicenter Study in 1429 participants [Nov 3, 2025] Rajanna et al. @DrOlaLandgren #ASH25 abs25-11635 PID 4008 https://t.co/gOprMCjfgh #mmsm #AI #oncopath https://t.co/pjikEsgfHC
The BIG Summit Meets at the Intersection of Patient Advocacy and Venture Investment
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...
Race‑agnostic AI Accurately Predicts M‑protein in Myeloma
Race agnostic models maintain accuracy in predicting M-protein levels in multiple myeloma [Nov 3, 2025] @rahul2u et al. #ASH25 abs25-11347 PID 2810 https://t.co/A27ozBoW8h #mmsm #AI #CancerDisparities https://t.co/ubJ6NGMFUE
Unstructured Serum Data Hinders AI in Myeloma Care
Unstructured serum electrophoresis data limits real world AI models in multiple myeloma: Evaluation of 384 institutions across United States [Nov 3, 2025] @MalekEhsanMD et al. #ASH25 abs25-11278 PID 2809 https://t.co/mAC8l7xnaG #mmsm #cancerdisparities #hpeonc https://t.co/veJxBV5S0N
OMG as a Marker of Resiliency to Neurodegenerative Processes
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...
Patients Favor Step‑Up Dosing for Teclistamab, Talquetamab
Patient perspectives & preferences for step-up dosing and treatment w/ teclistamab & talquetamab: Insights from a patient survey [Nov 3, 2025] @JayHydren et al. @RahulBanerjeeMD abs25-8335 PID 6354 https://t.co/pRlflaSb2I #mmsm #Tcellrx @HealthTree https://t.co/3I9nwsxZMx
Patient‑Centered Registry Accelerates Multiple Myeloma Care
The HealthTree Cure Hub registry: A patient-centered, multicenter approach to advancing multiple myeloma care - Jorge Arturo Hurtado Martínez et al. @HealthTree #ASCO25 Abst e19569 https://t.co/X2pdaIDYth #mmsm
STAT+: Gilead to Buy Arcellx in Nearly $8B Deal
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,...
Most Myeloma Patients Prefer Shared Decision‑Making
Patient Preferences on Clinical Decision-Making in Myeloma: A 747-Patient Survey [Dec 7, 2024] @ManniMD1 et al. - @HealthTree https://t.co/jATJRKEfB2 #ASH24 Abst 2281 #mmsm HT @Taxkourel
Side Effect Severity Shapes Patient Autonomy in Myeloma Care
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
How FDA's Action Against Non-FDA-Approved GLP-1 Products Affects the Competitive Landscape
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...
Strong Care Team Ties Shape Myeloma Treatment Choices
The effect of care team-patient relationship on decision-making in relapsed/refractory multiple myeloma - @MyelomaTeacher et al. @HealthTree #ASCO24 Abstract e19530 https://t.co/Ss5LZHH0Oj #mmsm
Patients Reveal Hopeful Views on Myeloma Cure
A survey on the patient perspective on cure in multiple myeloma [Oct 9, 2022] @ManniMD1 et al. @HealthtreeMM @TheLancetHaem https://t.co/1XhKT7AWGi #mmsm
Patient‑Driven Platform Accelerates Multiple Myeloma Research
.@HealthtreeMM Hub: A Patient-Derived, Patient-Driven Clinical Cancer Information Platform Used to Overcome Hurdles & Accelerate Research in Multiple Myeloma [3/10/22] @NSweeneyPhd @AhlstromJenny @FaithEDavies1 @mtmdphd @JCOCCI_ASCO https://t.co/WOwLb9Eozv @myelomacrowd #mmsm https://t.co/dZzhU3N32Q
Patient Portal Streamlines Myeloma Imaging and Pathology Reporting
. @HealthtreeMM Patient Portal mediated myeloma patient reported diagnostic imaging and pathology testing - @NSweeneyPhd @AhlstromJenny @mtmdphd J Clin Oncol 38: 2020 (suppl; abstr e20565) #ASCO20 https://t.co/CRLXETpeMm #mmsm #oncorad #oncopath https://t.co/m1UGDimLWm
Low‑dose Dexamethasone Improves Survival in Newly Diagnosed Myeloma
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
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/QdWETmSoiR
New Trial Compares Dara‑Bor‑Dex vs Cy‑Bor‑Dex for Myeloma‑Induced Kidney Damage
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