Today's Pharma Pulse
Global drugmakers accelerate U.S. manufacturing and R&D amid looming 100% tariff threat
Pharmaceutical giants are boosting U.S. production and research investments as the Trump administration threatens 100% tariffs on branded drugs unless prices are cut or production moves domestically. Companies including Pfizer, AstraZeneca, Eli Lilly, Johnson & Johnson and Roche have pledged commitments ranging from $3.5 billion to $70 billion to secure temporary tariff exemptions.
FDA Accepts Pharming's Joenja Resubmission, Paving Way for Pediatric APDS Treatment
The U.S. FDA has accepted Pharming Group's supplemental New Drug Application to expand Joenja (leniolisib) to children aged 4 to 11 with activated PI3K‑δ syndrome. The agency set a PDUFA target decision date for fall 2026, moving the therapy closer to the first pediatric label for this rare immunodeficiency.
Bio-Techne, Refeyn Partner on Workflow for Bispecific Antibody, Biosimilar Characterization
Bio‑Techne and Refeyn have launched an integrated workflow that pairs Bio‑Techne’s MauriceFlex icIEF fractionation system with Refeyn’s TwoMP mass‑photometry platform. The solution enables researchers to correlate charge heterogeneity with molecular weight and aggregation at single‑molecule resolution in about four hours...
IntraBio Announces Submission of Variation Application to the European Medicines Agency for AQNEURSA® for Ataxia-Telangiectasia
IntraBio Inc. submitted a variation application to the European Medicines Agency to expand AQNEURSA®’s label to include Ataxia‑Telangiectasia (A‑T). The filing marks the first EMA request for a therapy targeting this rare neurodegenerative disorder. In the United States, the company’s...
Study Data Convince Lundbeck to Push New Migraine Drug Forward
Denmark‑based Lundbeck reported Phase 2 data for its experimental migraine preventive, bocunebart, which targets the PACAP pathway. In the intravenous arm, patients experienced a 4.24‑day reduction in monthly migraine days versus a 2.86‑day drop with placebo, and the broader Phase 2 program...
Biogen's Salanersen Wins FDA Breakthrough Therapy Designation for SMA
Biogen announced that the FDA has granted Breakthrough Therapy Designation to its antisense drug Salanersen for spinal muscular atrophy (SMA). The designation follows Phase 1b data showing motor‑function gains in children who previously received gene therapy. The move accelerates Biotech’s Phase 3...
IMUNON Raises $10 Million to Fund Phase 3 Ovarian Cancer Trial, Bolstering B2B Pharma Pipeline
IMUNON, Inc. announced a $10 million cash financing package that includes preferred stock and secured promissory notes to fund its pivotal Phase 3 OVATION 3 ovarian cancer trial. The capital infusion is aimed at expanding enrollment and positioning the company for deeper B2B...
FDA Approves Ranluspec as Biosimilar to Lucentis
The U.S. Food and Drug Administration has granted approval for Ranluspec, an interchangeable biosimilar of Lucentis, to treat vision‑threatening retinal diseases. The drug is available in both vial and prefilled‑syringe formats and is offered in 0.3 mg and 0.5 mg strengths. Ranluspec...
Report: FDA Just Launched a Study on the Abortion Pill
The FDA has opened a six‑month safety review of mifepristone, the abortion pill approved since 2000, amid heightened political scrutiny from the Trump administration. Health experts emphasize that extensive research already confirms the drug’s safety, even when delivered via telehealth....
Mabwell Secures IND Clearance in China for 9MW5211 IBD Antibody
Mabwell announced that China’s National Medical Products Administration approved an IND for its antibody 9MW5211, allowing the first clinical trials of the drug in inflammatory bowel disease on the mainland. The clearance follows a U.S. FDA approval and positions the...
Quoin Secures Japan Orphan Drug Designation for QRX003 Targeting Netherton Syndrome
Quoin Pharmaceuticals announced that Japan's Ministry of Health, Labour and Welfare granted orphan drug designation to its QRX003 therapy for Netherton syndrome, promising up to a decade of market exclusivity. The move adds to QRX003’s U.S. pediatric rare‑disease and fast‑track...
Jade Biosciences Raises $150 Million in Equity Offering at $15 per Share
Jade Biosciences completed a $150 million public equity offering of 10 million shares at $15 each, with an additional 1.5 million‑share over‑allotment option. The proceeds will fund its Phase 2 trial of JADE101 and other R&D initiatives, underscoring strong investor demand for biotech capital.
Lupin Secures FDA Approval for Ranluspec, First Interchangeable Ranibizumab Biosimilar in U.S.
Lupin Limited announced that the U.S. FDA has approved Ranluspec™ (ranibizumab‑hkdz) as an interchangeable biosimilar to Lucentis®, making it the only such product available in both vial and pre‑filled syringe formats. The approval expands affordable treatment options for wet age‑related...
The Next FDA Commissioner Must Be a Champion for Gene Therapies
FDA Commissioner Marty Makary resigned on May 12, intensifying uncertainty for rare‑disease and gene‑therapy developers. Early 2026 data show orphan‑product rejection rates climbing to roughly 30%, up from about 10% in prior years. The volatility undermines investment decisions, prompting some...
GSK, Takeda and Others Notch Key Clinical Wins in Tough-to-Treat Diseases
Pharma giants reported breakthrough trial data across three hard‑to‑treat areas. GSK and Ionis’ antisense drug bepirovirsen achieved a 19% functional cure rate in a Phase 3 hepatitis B study, positioning it for fast‑track FDA approval and $2 billion‑plus sales. Takeda’s oral TYK2 inhibitor...
Leveraging Real-World Data for Proactive Protocol Design
Clinical trial protocols are becoming increasingly specialized, driving pressure for faster, more efficient development. Ashley Daigneau argues that real‑world data (RWD) should be embedded early in the design phase to stress‑test eligibility criteria, endpoint definitions, and enrollment forecasts. By combining...
Becoming a Cornerstone of Fujifilm’s CDMO Strategy in Denmark
Fujifilm is positioning its new contract development and manufacturing organization (CDMO) site in Denmark as a central pillar of its global strategy. The facility leverages early supply‑chain engagement and a continuous‑improvement model to lower risk, speed tech transfer, and shorten...
Innovation in Medicine Is Having a Breakthrough Moment
Decades of U.S. biopharmaceutical investment are bearing fruit as researchers unveil several high‑impact therapies. Revolution Medicines reported a late‑stage trial that doubled median survival for pancreatic cancer patients to 13.2 months, while Eli Lilly’s anti‑obesity candidate achieved weight‑loss results comparable...
Histamine Boost Improves Memory Retrieval and Decision-Making in Human Trial
Researchers published in Nature Communications reported that a single 36‑mg dose of the H3‑inverse agonist pitolisant significantly enhanced memory retrieval, decision‑making and reinforcement‑learning performance in a randomized, double‑blind trial of 58 healthy adults. The findings revive interest in histamine as...
![[Comment] Finerenone: Kidney Protection Beyond Type 2 Diabetes](https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/b396f6a91fcb1e7ba899cb118d2c2ff5.webp)
[Comment] Finerenone: Kidney Protection Beyond Type 2 Diabetes
Chronic kidney disease (CKD) kills roughly every 20 seconds and is projected to become the world’s fifth leading cause of death by 2040. While SGLT2 inhibitors and renin‑angiotensin system blockers have reduced CKD progression, a sizable residual risk remains. Non‑steroidal...
Amneal Secures FDA Approval for Romidepsin Injection, Gaining 180-Day Exclusivity
Amneal Pharmaceuticals announced FDA approval of a ready‑to‑use 27.5 mg/5.5 mL romidepsin injection for cutaneous T‑cell lymphoma. The designation as a Competitive Generic Therapy gives the company 180 days of market exclusivity, and shares rose 2.45% to $13.19 on the Nasdaq.
GH Therapeutics Hits Primary Endpoint in Phase 2a Postpartum Depression Trial
GH Research PLC announced that its inhaled candidate GH001 met the primary endpoint in a Phase 2a trial for postpartum depression, delivering a mean 35.4‑point reduction on the MADRS scale and 100% remission within two hours among ten patients. The...
ADC Therapeutics' Zynlonta Trial Shows 3‑Fold Higher Deaths, Shares Drop 52%
ADC Therapeutics reported that its phase 3 LOTIS‑5 trial of Zynlonta recorded 27 deaths (13.2%) in the treatment arm versus nine (4.6%) in the control arm. The safety signal erased a 52% plunge in the company’s stock and set up an...
Parabilis Eyes $475m IPO to Push Lead Asset Into Phase III
Parabilis Medicines is planning a Nasdaq IPO to raise up to $475 million, offering 25 million shares at $18 each. The biotech has secured a strategic investment from Regeneron, which will buy $75 million of stock at a 10% discount. Proceeds will fund...
ASCO26 Podcast: An Analyst Rundown of the Best Readouts and Key Trends
The GlobalData Healthcare podcast recapped the most compelling oncology data presented at ASCO 2026, featuring insights from analysts Jack Cuthbertson and Selena Yu. Their discussion highlighted breakthrough readouts across hematologic malignancies and solid tumors, with a spotlight on next‑generation modalities...
First Patient Dosed in Onchilles’ Phase I/II Trial of N17350
Onchilles Pharma has administered the first dose of its lead candidate N17350 to a patient in a combined Phase I/II trial targeting advanced solid tumours. The open‑label study evaluates intratumoral delivery of the ELANE‑pathway therapeutic, which is designed to induce...
Biogen’s Salanersen Gains FDA Breakthrough Therapy Designation for SMA
Biogen’s antisense oligonucleotide salanersen (BIIB115) received FDA breakthrough therapy designation for spinal muscular atrophy (SMA). The designation follows a Phase Ib trial where once‑yearly intrathecal dosing improved motor milestones and lowered neurofilament levels in children who had suboptimal responses to existing...
Biogen’s Salanersen Gains FDA Breakthrough Therapy Designation for SMA
Biogen’s antisense oligonucleotide salanersen (BIIB115) received FDA breakthrough therapy designation for spinal muscular atrophy (SMA). The designation follows a Phase Ib study in 24 children that showed once‑yearly dosing improved motor milestones and lowered neurofilament levels, a marker of neurodegeneration. Biotech’s...
Teva Launches Ahzantive (Biosimilar, Eylea) Across Key EU Markets
Teva Pharmaceutical has introduced Ahzantive, its aflibercept biosimilar to Eylea, across four major EU markets—France, Germany, Spain and the Netherlands—starting in May 2026. The product received European Commission approval in 2025 for wet age‑related macular degeneration, diabetic macular edema, myopic...
Azafaros Announces Publication of Phase 2 RAINBOW Study Data for Nizubaglustat in Molecular Genetics and Metabolism Journal
Azafaros announced that its Phase 2 RAINBOW study of the oral, brain‑penetrant azasugar nizubaglustat met its primary safety endpoint and demonstrated reductions in disease progression and seizure burden for patients with GM2 gangliosidosis and Niemann‑Pick type C disease. The full efficacy, safety,...
Otsuka Pharmaceutical Reports P-III (VISIONARY) Trial Data on Voyxact for IgA Nephropathy (IgAN)
Otsuka Pharmaceutical presented interim Phase 3 VISIONARY trial results for Voyxact (sibeprenlimab) in IgA nephropathy, enrolling 320 patients on drug versus 152 on placebo over a 24‑month period. At 12 months, Voyxact produced a mean eGFR change of +0.7 mL/min/1.73 m² compared with a...
Lundbeck Reports the P-IIb (PROCEED) Trial Data on Bocunebart for Migraine Prevention at AHS 2026
Lundbeck presented Phase IIb (PROCEED) trial results for bocunebart (Lu AG09222), an anti‑PACAP monoclonal antibody, as a preventive migraine therapy. In the intravenous cohort of 429 patients with one to four prior preventive treatment failures, bocunebart reduced monthly migraine days by...
Chitosan Nanoparticles Could Make Vaccines More Stable, Mucosal, and Needle-Free
A recent npj Vaccines review highlights chitosan‑based nanoparticles as a promising platform to stabilize fragile antigens, enable needle‑free administration, and boost mucosal immunity. By tweaking molecular weight, deacetylation and cross‑linking, researchers can tailor solubility, charge and degradation rates for optimal...
HHS Assures Experimental Ebola Treatment for High‑risk Americans
HHS confirms Americans with high-risk Ebola exposures will have access to experimental therapy https://t.co/69b364ZHlm via @statnews
UAE Approves Novo Nordisk’s Wegovy Pill, Calls for Careful Use
UAE health authorities have approved Novo Nordisk’s oral semaglutide pill, Wegovy, for prescription use, making it the first market outside the United States to receive the drug in tablet form. Novo Nordisk executives and local physicians are urging that the...
Iovance Wins Conditional Australian Approval for Amtagvi T‑Cell Therapy in Advanced Melanoma
Iovance Biotherapeutics announced that Australia’s Therapeutic Goods Administration granted conditional approval for Amtagvi (lifileucel) in adult patients with advanced melanoma who have failed anti‑PD‑1 and, when applicable, BRAF‑targeted therapy. The decision makes Amtagvi the first tumor‑derived autologous T‑cell therapy approved...
Breakthrough ‘Universal Vaccine’ Technology Promises Protection Against Future Virus Outbreaks
Researchers at the University of Cambridge and spin‑out DIOSynVax completed the first human Phase I trial of an AI‑designed universal vaccine targeting Sarbeco coronaviruses. The trial enrolled 39 healthy volunteers, showed no significant side effects, and generated robust T‑cell and antibody...
Fred Hutch’s Sylvain Simon Named ‘Rising Star’ at Grand Rounds U.S. in Seattle
Sylvain Simon’s team at Fred Hutchinson Cancer Research Center unveiled a synthetic chimeric T‑cell receptor (ChTCR) platform that outperforms traditional CAR T cells in antigen sensitivity and can be engineered for multispecific targeting. The technology, detailed in a March 2025 Nature...
How Regrowing Your Own Teeth Could Replace Dentures and Implants
About 178 million American adults have lost at least one tooth, and current solutions rely on metal implants or dentures that can cause pain and lack sensation. Over the past two decades, researchers in the UK, US, Japan and elsewhere have...
Nanoparticles Inspired by Lung Fluid Improve Therapies Targeting Respiratory System
Researchers at CIC biomaGUNE have created pulmonary surfactant nanoparticles that encapsulate antifibrotic drugs and can be delivered by inhalation. Using a microfluidic synthesis platform, the particles achieve uniform size, high drug loading and 90% retention in mouse lungs, dramatically lowering...
New Framework Enables Fully Autonomous AI Drug Discovery
https://t.co/3BWIq8mv2F A Framework for Autonomous AI-Driven Drug Discovery Douglas W. Selinger, Timothy R. Wall, Eleni Stylianou, Ehab M. Khalil, Jedidiah Gaetz, Oren Levy @plexresearch
Can MDMA Cure PTSD? A New Review of the Evidence Says It’s Too Early to Tell
A new meta‑analysis of eight randomized controlled trials involving 387 adults found that MDMA‑assisted therapy can reduce PTSD symptom severity, improve overall functioning, and lessen dissociative symptoms, though it showed no clear effect on depression. The authors rated the overall...
Three Years on Baricitinib Preserved Hair Regrowth in Severe Alopecia Areata
Baricitinib maintains hair regrowth over three years in severe alopecia areata. At week 152, 89.1% of patients on 4 mg and 83.6% on 2 mg who responded at week 52 kept SALT ≤ 20, with mean scores of 3.2 and 4.3 respectively. Deep regrowth...
STAT+: Drug Companies, Patient Groups Urge FDA to Pause Commissioner’s Voucher Program
The FDA convened a listening session on the Commissioner’s National Priority Voucher program, a fast‑track review initiative introduced by former commissioner Marty Makary. While a handful of speakers praised the accelerated approvals, most patient groups, pharmaceutical firms, and academic representatives...
Liminatus Pharma Secures $1.9 Million via Warrant Exercise, Issues New Warrants
Liminatus Pharma, Inc. closed a $1.9 million warrant exercise with existing accredited investors, converting 10.34 million outstanding warrants and issuing 20.688 million new warrants at a $0.18 exercise price. The cash infusion bolsters the small‑cap biotech’s runway as it seeks to advance its...
Alnylam Inks $2 B AI Drug Development Pact with Inceptive Ink
Alnylam Pharmaceuticals has signed a partnership with AI‑driven biotech Inceptive Ink valued at up to $2 billion, delivering a $30 million upfront payment. The deal merges Alnylam’s RNAi expertise with Inceptive’s foundation‑model AI to accelerate nucleic‑acid drug design under Alnylam’s 2030 strategy.
Brazilian Researchers Launch Nanoparticle Platform to Silence Genes Behind Psoriasis and Vitiligo
Researchers at the University of São Paulo's NanoGeneSkin lab unveiled a lipid‑nanoparticle platform that can deliver therapeutic RNA directly into skin cells, silencing the genes that drive psoriasis and vitiligo. The breakthrough, presented at FAPESP Week London, could replace systemic...
STAT+: Otsuka Kidney Drug Slowed Loss of Function, but Less than Expected, in Late-Stage Trial
Otsuka Pharmaceutical reported that its Phase 3 trial of Voyxact, an injectable therapy for IgA nephropathy, showed a slower decline in kidney function over one year. Patients receiving Voyxact experienced an annualized eGFR loss of 3 points versus 7.6 points in...
Experimental Adjuvant Could Strengthen Mucosal Immunity with Injectable Polio Vaccines
A MIT‑led study demonstrates that encapsulating the vitamin‑A derivative Am80 in lipid nanoparticles (LNPs) creates an injectable adjuvant that steers immune cells to the gut, generating mucosal immunity alongside the standard inactivated polio vaccine (IPV). In rat experiments, a single...
Stipple Bio and Lonza Agree to Focus on Advancing Oncology ADC Therapies
Stipple Bio and Lonza have signed a multi‑target licensing agreement to co‑develop precision oncology antibody‑drug conjugates (ADCs). Stipple will apply its Pointillist Platform to discover tumor‑specific cell‑surface epitopes and generate antibody binders, while Lonza will supply its GlycoConnect conjugation, HydraSpace...
A Q&A with Dr. Ganesh Bala on LC–MS and Multi-Attribute Methods for Bioconjugate CQA Monitoring
Agilent’s Dr. Ganesh Bala explained that multi‑attribute methods (MAMs) using LC‑MS peptide mapping have become a core analytical platform for monitoring critical quality attributes of antibody‑drug conjugates (ADCs) and other bioconjugates. He highlighted the growing heterogeneity of these molecules—variable conjugation sites,...