Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech financings
Spanish biotech Ona announced a $86.6 million Series B round, ranking among the largest venture financings 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 funds will expand Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical pipeline.
Promoters and Enhancers: Tool Catches Gene-Controlling DNA Sequences Doing Each Other's Jobs
Researchers at Cornell’s Weill Institute introduced QUASARR‑seq, a high‑throughput assay that measures promoter and enhancer activity simultaneously. The study found that most human regulatory elements can function as both promoters and enhancers, following a unified regulatory logic. A bidirectional feedback loop between promoters and enhancers was uncovered, showing mutual reinforcement. These insights could transform gene‑therapy design and reinterpret disease‑associated regulatory mutations.
Cybersecurity and AI in the Era of Home-Based Care Logistics
Kenco’s vice‑president of life sciences, Tim McClatchy, detailed how the firm is hardening cybersecurity across its manufacturer‑to‑home delivery network while deploying AI to streamline labor planning and route optimization. He explained the specific encryption and verification steps used at each...
IHE: U.S. Pharmaceuticals ETF With Excellent GARP Features
The iShares U.S. Pharmaceuticals ETF (IHE) is anchored by Johnson & Johnson and Eli Lilly, whose earnings per share have nearly doubled in the past year. IHE’s aggregate one‑year EPS growth estimate is an impressive 19.07% while trading at a modest...
STAT+: Trump Most-Favored Nation Drug Pricing Deals End After Three Years for some Companies
President Trump’s "most‑favored nation" (MFN) drug pricing agreements, touted as a safeguard against excessive prescription costs, have been revealed to run for three years for several participants. SEC filings show that 16 pharmaceutical firms have entered these deals, each with...
Size-Shifting Nanoparticles Successfully Deliver mRNA Medicine to the Pancreas
Researchers have engineered size‑shifting lipid nanoparticles that grow from ~100 nm to >300 nm after intraperitoneal injection, exploiting a capsule‑filter mechanism that blocks entry into the liver and spleen while allowing passage to the pancreas. The enlarged particles deliver mRNA payloads—including CRISPR‑Cas9...
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...
Lilly Launches LillyPod NVIDIA DGX SuperPOD for Genomics and Drug Discovery AI
Eli Lilly unveiled LillyPod, a DGX SuperPOD built with 1,016 NVIDIA Blackwell Ultra GPUs delivering more than 9,000 petaflops of AI performance. The system powers genomics, protein‑diffusion, small‑molecule graph neural networks and foundation models, allowing billions of in‑silico experiments. Constructed...
Pharmaceutical Executive Daily: FDA Approves Hernexeos
The FDA approved Hernexeos through the national priority voucher program, a mechanism that fast‑tracks drugs addressing unmet medical needs. Industry leaders are evaluating direct‑to‑consumer (DTC) models to broaden access to HIV prevention tools, leveraging digital and telehealth channels. Additionally, the...
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
A Tale of Three Bispecific Cities
Three bispecific antibodies targeting PD‑1 and CTLA‑4—lorigerlimab, volrustomig, and cadonilimab—illustrate divergent engineering strategies. Lorig erlimab relies on a knob‑into‑hole heterodimer, volrustomig adopts a common light chain format, and cadonilimab incorporates Fc‑silencing mutations. Their distinct designs have produced markedly different safety...
Drug Trials Snapshot: HYRNUO
Bayer's HYRNUO (sevabertinib) received accelerated FDA approval on November 19, 2025 for adult patients with HER2‑mutated, non‑squamous non‑small cell lung cancer who have progressed after prior therapy. The approval is based on the single‑arm SOHO‑01 trial, which enrolled 122 patients across 78...
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
Cellular Reprogramming: The Expert Roundup
A round‑up of leading experts highlights how cellular reprogramming has moved from a laboratory curiosity to a near‑clinical anti‑aging platform. Researchers describe partial, epigenetic‑focused approaches that can rejuvenate cells without erasing identity, and four biotech firms outline their distinct delivery...
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
FAQ: Inflation Reduction Act and Medicare Drug Pricing
The Inflation Reduction Act reshapes Medicare Part D by granting CMS authority to negotiate prices for high‑cost drugs and instituting a $2,100 annual out‑of‑pocket cap for beneficiaries starting this year. The first ten drugs, including Ozempic and Wegovy, will have negotiated...
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.
Pharma M&A in 2026: Can the Industry Keep Up the Momentum?
Dan Chancellor of Norstella explains that the looming patent cliff and persistent growth gaps are forcing pharma companies to rely heavily on mergers and acquisitions. 2025 saw a record $220 billion in deal value, driven by several mega‑transactions and numerous bolt‑on...
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...
AI for Scientists, FDA Reverses Course, and Advances in Cancer Therapeutics
The episode covers three main business stories: Tamarin Bio’s $13.6M Series A to build an AI model‑coordination platform that makes tools like AlphaFold accessible to wet‑lab biologists; the FDA’s reversal on Moderna’s mRNA‑1010 seasonal flu vaccine, spurring a modest stock...
Transduction-Ready Viral Particles
AMSBIO (Oxford, UK) now provides ready‑to‑use lentiviral particles with titers exceeding 1×10⁷ IFU / mL, eliminating the need for in‑house virus production. The third‑generation self‑inactivating vectors are supplied in research‑grade and GMP‑grade formats, requiring only BSL‑2 containment. High‑titer, purified particles enable efficient...
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
A New Antibody Treatment For Breast Cancer
A year-long trastuzumab emtansine (T‑DM1) regimen showed 98% three‑year invasive‑disease‑free survival in early‑stage HER2‑positive breast cancer, comparable to standard paclitaxel‑plus‑trastuzumab. The ATEMPT trial of nearly 500 patients found similar overall survival but markedly lower neuropathy and hair loss with T‑DM1,...
Boehringer Wins Speedy Lung Cancer Approval Under Commissioner’s Priority Program
The FDA granted accelerated approval to Boehringer Ingelheim’s oral HER2 kinase inhibitor Hernexeos six weeks after the company filed its application, marking the second approval under the new Commissioner’s National Priority Voucher (CNPV) program. The decision expands Hernexeos use to...
Promatix EGFR×EphA2 Bispecific ADC Exerts Antitumor Activity
Today’s brief highlights four pre‑clinical advances: Paratus Sciences unveiled PS‑1001, a pan‑inflammasome blocker targeting IL‑1β and IL‑18 for hidradenitis suppurativa; researchers identified the SCAN circuit as a core pathway disrupted in Parkinson’s disease; RX‑10616 demonstrated enhanced radiotherapy response in head‑and‑neck...
Prospect Therapeutics Identifies New JAK1 and TYK2 Inhibitors
Prospect Therapeutics announced the identification of a new series of small‑molecule inhibitors targeting Janus kinase 1 (JAK1) and tyrosine kinase 2 (TYK2). The lead compounds demonstrate nanomolar potency and selectivity in biochemical assays and effectively suppress cytokine signaling in cellular...
How PBM Reforms Could Push Drugmakers Into the Pricing Spotlight
The Consolidated Appropriations Act of 2026 bans pharmacy benefit managers (PBMs) from receiving compensation tied to drug list prices and mandates 100% rebate pass‑through to plan sponsors by August 2028. The law also allocates roughly $190 million to the Centers for...
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...
Drug Trials Snapshots
The FDA’s Drug Trials Snapshots program publishes concise, consumer‑focused summaries of the demographic makeup and outcomes of pivotal clinical trials for newly approved drugs. Launched in 2015, the tool covers only New Molecular Entities and original biologics approved after that...
Ionis’ Olezarsen sNDA Secures the US FDA Priority Review for Severe Hypertriglyceridemia
Ionis Pharmaceuticals received FDA acceptance of its supplemental NDA for olezarsen and a priority‑review designation, with a PDUFA action date of June 30, 2026. The decision is backed by two Phase III CORE studies—CORE (n=617) and CORE2 (n=446)—that evaluated once‑monthly subcutaneous...
Drug Trials Snapshots: EXDENSUR
GSK’s depemokimab, marketed as EXDENSUR, received FDA approval on December 16, 2025 as a six‑month subcutaneous add‑on for severe eosinophilic asthma in patients aged 12 and older. Approval was based on two 52‑week, double‑blind, placebo‑controlled trials (SWIFT‑1 and SWIFT‑2) that...
Polyploidy and Cellular Senescence Are Tangled Together
Researchers highlight that senescent cells arising from DNA replication errors that generate whole‑chromosome duplications—polyploidy‑induced senescence (PIS)—are biochemically distinct from senescence caused by other stresses. Existing literature often fails to separate polyploid senescent cells from diploid counterparts, obscuring their unique roles...
Bringing HIV Prevention DTC: Q&A with Ashley Gildea
Gilead’s U.S. HIV prevention VP Ashley Gildea unveiled a direct‑to‑consumer campaign for Yeztugo, the first twice‑yearly injectable PrEP. The campaign, anchored by a music‑driven “One2PrEP” spot, aims to lift awareness among the estimated 2.2 million Americans who could benefit from PrEP...
Podcast: Rare Disease Day: FDA Guidance Allowing Advancement
The FDA issued a suite of new guidance documents in early 2026 aimed at easing development of therapies for rare diseases, including the Rare Disease Evidence Pathway, Plausible Mechanism Pathway, and innovative trial designs for cellular and gene therapies. The...
Pharma Pulse: The Oral GLP-1 Battle and GSK’s Billion-Dollar Breath of Fresh Air
Eli Lilly’s oral GLP‑1 small‑molecule orforglipron demonstrated greater A1C reduction and weight loss than oral semaglutide, while offering flexible dosing without fasting. GSK announced a $950 million acquisition of 35Pharma, securing HS235, an activin‑signaling inhibitor aimed at pulmonary hypertension. The deal taps...
Quotient and Ipsen Extend Partnership for Ultra-Rare Disease Therapy
Quotient Sciences has extended its commercial partnership with Ipsen to manufacture Sohonos (Palovarotene), the approved therapy for fibrodysplasia ossificans progressiva (FOP), an ultra‑rare disease affecting fewer than 1,000 people worldwide. The deal includes Ipsen's investment in a pneumatic closed‑transfer system...
Lilly Reports ACHIEVE-3 Trial Outcomes for Type 2 Diabetes
Eli Lilly disclosed Phase III ACHIEVE‑3 results showing its oral GLP‑1 agonist orforglipron outperformed oral semaglutide in lowering A1C and inducing weight loss. The 52‑week, open‑label trial enrolled 1,698 patients across six countries and compared two doses of each drug. Orforglipron also...
Bayer Reports P-III (PEACE-3) Trial Data on Xofigo Combination for Metastatic Castration-Resistant Prostate Cancer (mCRPC) with Bone Metastases
Bayer announced that its phase‑III PEACE‑3 trial demonstrated a statistically significant overall‑survival benefit for the combination of Xofigo (radium‑223) and enzalutamide versus enzalutamide alone in patients with metastatic castration‑resistant prostate cancer (mCRPC) and bone metastases. Median overall survival extended to...
Mutant P53 Selective Reactivation Demonstrated in Advanced Solid Tumors
PMV Pharmaceuticals reported Phase I results of rezatapopt, a small‑molecule p53 reactivator, in 77 patients with advanced solid tumors carrying the TP53 Y220C mutation. The oral drug was generally well tolerated, with few dose‑limiting toxicities, allowing the selection of a...
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
ULTRA ADVANC3 and ULTRA ADVANC3 GOLD May Be Harmful Due to Hidden Drug Ingredients
The FDA has warned consumers against buying ULTRA ADVANC3 on Amazon and ULTRA ADVANC3 GOLD on NaturistaRex after laboratory tests revealed hidden prescription drugs. The supplements contain undeclared dexamethasone, diclofenac, and methocarbamol, each carrying serious health risks. The agency advises immediate discontinuation and...
Fulgent Genetics Inc (FLGT) Q4 2025 Earnings Call Transcript
Fulgent Genetics reported Q4 2025 revenue of $84.1 million, a sequential increase and strong year‑over‑year growth across its Precision Diagnostics and Biopharma segments. Non‑GAAP gross margin improved to 44.3% while operating expenses fell, delivering a $0.7 million adjusted EBITDA and a $4.5 million...
Johnson Floats ‘Right To Try 2.0’ As Makary Defends FDA’s Approval Standards
Senator Ron Johnson (R‑WI) is urging Congress to draft a “Right to Try 2.0” bill that would force the FDA to grant broader, faster access to experimental therapies for rare diseases. FDA Commissioner Martin Makary pushed back, defending the agency’s...
STAT+: Justice Department Backs AbbVie in Its Bid to Overturn a Colorado Law Guiding a Drug Discount Program
The U.S. Department of Justice filed an amicus brief supporting AbbVie in its effort to overturn a Colorado statute that prohibits pharmaceutical manufacturers from limiting discounts under the federal 340B drug pricing program when hospitals use contract pharmacies. The Colorado...
AI Designs Better Drug Candidates with Quantum Aid
Researchers from Japan Tobacco and D‑Wave have unveiled a hybrid framework that merges deep generative models with quantum annealing to design drug‑like molecules. The system introduces a Neural Hash Function that enables binarisation for quantum processing while preserving gradient flow....
FDA Grants Accelerated Approval to Zongertinib for HER2-Mutant NSCLC
The FDA granted accelerated approval to zongertinib (Hernexeos) for adults with unresectable or metastatic non‑squamous NSCLC that carry activating HER2 TKD mutations, extending its use to treatment‑naive patients. The decision rests on the Beamion LUNG‑1 trial, which reported a 76%...
How RNA Binding Selectivity Arises From Disordered Regions
RIKEN scientists have shown that an intrinsically disordered region (IDR) of the DEAD‑box helicase DDX3X confers selective binding to specific mRNA structures, a mechanism uncovered using solution NMR spectroscopy. The discovery clarifies how DDX3X distinguishes target transcripts, linking its specificity...
ARUP Creates Innovation Central Laboratory
ARUP Laboratories has unveiled the Innovation Central Laboratory, a dedicated hub for co‑development with pharmaceutical and biotech partners. The facility is designed to validate technologies and move next‑generation diagnostics from concept through to commercial readiness. It will host projects ranging...
Drug Trials Snapshots: KOMZIFTI
The FDA approved KOMZIFTI (ziftomenib) on November 13 2025 as an oral 600 mg capsule for adult patients with relapsed or refractory acute myeloid leukemia (AML) harboring an NPM1 mutation. Approval rests on the single‑arm KO‑MEN‑001 trial, which enrolled 112 participants across seven...
January 2026 Patent Highlights
The January 2026 Patent Highlights roundup spotlights a wave of new intellectual‑property activity across several cutting‑edge drug discovery areas. Notable filings include lysine‑directed covalent inhibitor chemotypes, strategies to balance potency with drug‑like properties, refined target‑selection frameworks, dozens of Polθ synthetic‑lethal patents...