FDA Accepts BridgeBio’s Application for Potential First Limb-Girdle Muscular Dystrophy Drug
BridgeBio’s oral therapy BBP‑418 has received FDA priority review, with a target action date of Nov. 27, 2025, positioning it for a potential launch in late 2026 or early 2027. The Phase 3 FORTIFY trial met all primary and secondary endpoints, showing functional, ambulatory, and pulmonary gains versus placebo. Analysts estimate peak global sales of about $1.1 billion and a 90%+ likelihood of approval. The move could mark the first disease‑modifying treatment for the rare LGMD2I/R9 subtype.
Congo Seeks US Monoclonal Antibody for Bundibugyo Ebola Strain
Democratic Republic of Congo officials have formally requested 500 doses of the experimental US‑developed monoclonal antibody MBP‑134 to treat patients infected with the Bundibugyo strain of Ebola. The antibody, which targets Zaire, Sudan and Bundibugyo variants, was previously used in...
Jade Biosciences Initiates First-in-Human Phase 1 Trial of BAFF-R Antibody JADE201
Jade Biosciences has dosed the first participant in a first‑in‑human Phase 1 trial of JADE201, an investigational half‑life‑extended afucosylated anti‑BAFF‑R monoclonal antibody. The randomized, placebo‑controlled study evaluates single ascending doses in rheumatoid arthritis patients, focusing on safety, tolerability, pharmacokinetics and pharmacodynamics....
STAT+: Kailera’s Own ‘Triple-G’ Drug Also Looks Very Powerful
The FDA postponed its decision on AstraZeneca’s experimental breast‑cancer therapy camizestrant after advisers criticized the SERENA‑6 trial design, giving the company extra time for additional analyses. Meanwhile, Blackstone Life Sciences pledged up to $1.3 billion to Apogee Therapeutics to fund Phase 3...
Chinese Scientists Use Supercomputer to Cut New Drug Screening Time From Years to Seconds
Chinese researchers have launched GalaxyVS, an AI‑driven drug‑discovery platform that leverages the Tianhe supercomputer to screen up to 100 billion chemical compounds in seconds. The system achieves a daily throughput of 16 trillion molecular dockings, a million‑fold speed increase over the previous...
Zuckerberg, Chan's Biohub Launches Protein 'World Model'
The Chan Zuckerberg Biohub has unveiled an AI‑driven "world model" for protein biology, built on an atlas of 6.8 billion proteins and 1.1 billion structures. The open‑source platform combines the ESMC language model with the ESMFold2 design engine to predict structures, design...
Lilly Just Bought Renowned Regulator Peter Marks a Pipeline to Play With
Eli Lilly has spent $3.8 billion acquiring Curevo, LimmaTech Biologics and Vaccine Company, giving the company a fledgling vaccine pipeline. The deals were driven in part by the hiring of former FDA CBER chief Peter Marks, who left the agency amid concerns over...
China’s Drug Regulator Clears Wave of Home-Grown Innovative Medicines Amid Biotech Boom
China’s National Medical Products Administration has approved 19 innovative drugs this year, with domestic firms accounting for 15 of them, highlighting a rapid biotech surge. The regulator recently overhauled its approval pathway to speed market entry for home‑grown medicines. Leading...
FDA Removes Boxed Warning About Risk of Leg and Foot Amputations for the Diabetes Medicine Canagliflozin (Invokana, Invokamet, Invokamet XR)
The FDA has eliminated the boxed warning on canagliflozin (Invokana, Invokamet, Invokamet XR) that highlighted a heightened risk of leg and foot amputations. The decision follows new data from three clinical trials showing the amputation risk is lower than originally...
Popular Anti-Aging Drug Combo Caused Severe Brain Damage in Mice
Researchers at the University of Connecticut discovered that the popular anti‑aging drug combo dasatinib plus quercetin (D+Q) causes severe myelin damage in mice, affecting both young and old subjects. The study, published in PNAS, showed dramatic loss of the protective...
Kura’s Encouraging Cancer Drug Data Could Unlock $2B Market Opportunity
Kura Oncology reported that its experimental farnesyltransferase inhibitor darlifarnib, combined with Bristol Myers Squibb’s KRAS inhibitor Krazati, shrank tumors in 77% of the 26‑patient FIT‑001 cohort. Objective response rates reached 67% in pancreatic cancer, 50% in non‑small cell lung cancer,...
D&D Pharmatech Reports P-II Trial Results on Zabopegdutide for Metabolic Dysfunction-Associated Steatohepatitis (MASH)
D&D Pharmatech disclosed topline Phase II data for its GLP‑1‑like agent zabopegdutide in 67 overweight or obese patients with metabolic dysfunction‑associated steatohepatitis (MASH). After a 2‑week titration to a 40 mg weekly maintenance dose, 48‑week histology showed a 50% fibrosis‑stage improvement versus...
Autobahn’s Elunetirom Receives FDA Fast Track Designation to Treat Adjunctive Bipolar
Autobahn Therapeutics received FDA fast‑track designation for elunetirom, an oral, brain‑penetrant thyroid hormone receptor agonist being developed as an adjunctive treatment for bipolar depression and major depressive disorder. The drug targets central nervous system thyroid receptors to boost neurobiological activity...
SK Bioscience and VECOL Partner on Colombia Vaccine Manufacturing Initiative
SK bioscience has signed a technology‑transfer and manufacturing agreement with Colombia’s state‑owned VECOL, joining the country’s $260 million vaccine localisation initiative. The partnership will initially produce the SKYVaricella varicella vaccine locally, with VECOL handling operations and SK providing expertise. SK will also...
One-Time Gene Editing Cuts LDL Cholesterol in Early Hypercholesterolemia Trial
A single intravenous infusion of VERVE‑102, an in‑vivo base‑editing therapy targeting the PCSK9 gene, produced dose‑dependent reductions in PCSK9 protein and LDL cholesterol in a phase 1 trial of 35 adults with heterozygous familial hypercholesterolemia or premature coronary disease. At the...
Retatrutide Reshapes Metabolism in Obesity and Type 2 Diabetes, Study Finds
A post‑hoc analysis of two phase‑2 trials shows that the triple‑receptor agonist retatrutide reshapes fatty‑acid oxidation and insulin‑resistance biomarkers in participants with obesity, with or without type 2 diabetes. Higher doses raised 3‑hydroxybutyrate by up to 198% and altered acyl‑carnitine ratios,...
Niowave, Fresh From Novartis Deal, Starts Building $75M Radioisotope Plant
Niowave announced the construction of a new $75 million actinium‑225 production facility in Lansing, Michigan, slated to begin operations in 2028. The plant will house proprietary superconducting linear accelerators and create about 70 high‑skill jobs in research, engineering, and quality assurance....
Corbus Pharmaceuticals Holdings, Inc. (CRBP) Discusses Clinical Data Update From Phase 1/2 Study of CRB-701 Presented at ASCO Transcript
Corbus Pharmaceuticals announced that it will present Phase 1/2 data for its oncology candidate CRB‑701 at the upcoming American Society of Clinical Oncology (ASCO) meeting. The data snapshot reflects an April 1 cut, and the company briefed investors via a conference call...
Thinning Revenues: Inside the $14bn Eliquis Patent Cliff
Eliquis, the $14.4 bn anticoagulant co‑developed by Bristol Myers Squibb and Pfizer, faces a staggered loss‑of‑exclusivity, with European patents expiring in 2026 and U.S. protection ending in 2028. Forecasts show sales plunging to $205 m by 2031, a 98.6% decline, as generics...
FDA Approves First Interchangeable Biosimilars to Simponi and Simponi Aria; Janssen Seeks a Preliminary Injunction to Block Their Launch
The FDA approved Accord BioPharma’s IMMGOLIS™ and IMMGOLIS INTRI™ as the first interchangeable biosimilars to Janssen’s Simponi® and Simponi ARIA® on May 15, 2026. Both products treat moderately to severely active rheumatoid arthritis, and IMMGOLIS also covers ulcerative colitis. Accord will commercialize the drugs...
New Drug Works Against Diseases Like Measles and Croup
Researchers at Georgia State University have identified GHP-88310, a new oral antiviral candidate that targets orthoparamyxoviruses such as measles and human parainfluenza virus type 3. The drug demonstrated potent, once‑daily efficacy and high tolerability in both rodent and non‑rodent animal models,...
Alaunos Reports Broad Metabolic Improvements with ALN1003 in Obese Mouse Studies
Alaunos Therapeutics reported that its oral small‑molecule candidate ALN1003 produced broad metabolic improvements in diet‑induced obese mice. Across two non‑GLP studies, treated animals showed up to a 13% reduction in body weight, significant fat loss, lower fasting insulin and HOMA‑IR,...
Lilly Gets Back Into the Vaccine Business With New Deals
Eli Lilly & Co. announced it will acquire three clinical‑stage vaccine developers for up to $3.8 billion, marking a major re‑entry into the infectious‑disease space. The deals are financed largely by cash generated from its blockbuster obesity drugs, such as Mounjaro and...
Next-Generation Platforms Poised to Redefine Crowded HAE Landscape
The hereditary angioedema (HAE) market has accelerated, with three FDA approvals in 2025 bringing the U.S. total to 11 marketed treatments. Nearly half of the 15 global pipeline candidates—46%—are now in Phase III, signaling a wave of imminent approvals. While most...
Merz Therapeutics and Kvvit Partner to Advance Inbrija in Mainland China, Hong Kong and Macao
Merz Therapeutics has licensed its inhaled levodopa product Inbrija to Chinese biotech Kvvit for exclusive commercialization in mainland China, Hong Kong and Macao. The agreement assigns Merz responsibility for drug supply, global quality oversight and holding the overseas registration certificate,...
Arthrosi Drug Trial in Gout Delivers for New Owner Sobi
Swedish biotech Sobi’s $1.5 billion acquisition of Arthrosi is yielding results as its gout drug pozdeutinurad met primary goals in the phase 3 REDUCE‑2 trial. The URAT1 inhibitor lowered serum uric acid to ≤6 mg/dl in 69.2% of patients at a 75 mg dose...
Nanoscale Drug Delivery Systems for Ovarian Cancer: Targeting Strategies, Theranostic Platforms, and Translational Challenges
A new review maps the evolution of nanoscale drug delivery systems (DDS) for ovarian cancer, shifting focus from blunt chemotherapy to precision nanomedicine. It outlines three core design strategies—active targeting, microenvironment‑responsive release, and theranostic integration—across carriers such as liposomes, polymeric...
Pierre Fabre’s Braftovi Gains CHMP Positive Opinion for mCRC
The European Medicines Agency’s CHMP issued a positive opinion for Pierre Fabre’s Braftovi (encorafenib) combined with cetuximab and Folfox as a first‑line treatment of BRAFV600E‑mutant metastatic colorectal cancer. The Phase III BREAKWATER trial showed a 51 % reduction in death risk, extending median...
Does Ceramide Lipid Metabolism Affect Response to Prostate Cancer Drugs?
Researchers discovered that ceramide lipid metabolism varies by genetic ancestry and influences response to androgen‑receptor pathway inhibitors in metastatic castration‑resistant prostate cancer. Black patients entered treatment with lower total ceramides but higher C24‑to‑C16 ratios, a pattern that flipped during therapy....
AstraZeneca Receives the CHMP Positive Opinion for Camizestrant to Treat Advanced Breast Cancer
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion on AstraZeneca’s oral selective estrogen receptor degrader camizestrant, to be used with a CDK4/6 inhibitor for ER‑positive, HER2‑negative advanced breast cancer harboring ESR1 mutations....
Amgen Drug Reviewed Amid Deaths
Amgen’s rare‑disease drug Tavneos (avacopan) resumed sales in Japan after a new warning label was added following 20 reported deaths linked to liver injury. The drug, acquired when Amgen bought ChemoCentryx in 2022, generated $459 million in 2025 sales. In the...
Follow the Money: AI Drug Design Engine, Treating Multiple Myeloma, Quantum Healthcare Platform
Isomorphic Labs closed a $2.1 billion Series B to scale its AI drug‑design engine, while CellCentric secured $220 million to push its oral myeloma candidate, inobrodib, into Phase 3 trials. NVision raised $55 million Series B to merge quantum computing with its POLARIS MRI platform, aiming...
4 Troubled Targets that Have Thwarted Biopharma
Biopharma’s pursuit of high‑profile targets—TIGIT, RIPK1, MYC, STING and alpha‑synuclein—has hit a series of setbacks. Gilead and Arcus dropped domvanalimab after a disappointing Phase 3 readout, while GSK, Sanofi, Eli Lilly and Roche have all abandoned RIPK1 programs. MYC remains structurally undruggable,...
REGENXBIO, Novartis, Dyne Near FDA Submissions in Muscular Dystrophies
A wave of near‑term regulatory activity is reshaping the muscular dystrophy landscape. REGENXBIO reported mixed Phase 3 data for its gene therapy RGX‑202, achieving 93% microdystrophin expression but raising safety flags, while targeting a 2027 launch. Novartis’ $12 billion acquisition of Avidity...
Noxopharm Studies Confirm Cancer-Fighting Potential of Sofra Platform
Australian biotech Noxopharm announced new data on its Sofra platform, a TLR8‑activating oligonucleotide technology that could enhance cancer immunotherapy. Preclinical studies demonstrated up to a 200‑fold boost in TLR8 activity in human skin biopsies and nearly three‑fold activation in animal...
NeuroScientific Hails 80% Clinical Response in Crohn’s Stem Cell Program
NeuroScientific Biopharmaceuticals reported that four of five patients with fistulising Crohn’s disease showed a clinical response to its StemSmart mesenchymal stem cell therapy, an 80% response rate. All participants experienced symptom improvement and no serious adverse events were recorded. The...
New Maternal RSV Vaccine Lowers Infant Hospitalization Rates, but Accessibility May Be Limited, Study Finds
A new maternal RSV vaccine introduced in fall 2023 reduces infant hospitalizations, with the Dallas study showing zero hospitalizations among vaccinated infants versus 3% among unvaccinated. Vaccination rates varied sharply by insurance type and race, with private‑insured mothers at 37%...
AstraZeneca, Daiichi Drug Approved as New First-Line Therapy for Tough Type of Breast Cancer
The FDA granted accelerated approval to Datroway (DS‑1062), an antibody‑drug conjugate co‑developed by Daiichi Sankyo and AstraZeneca, as a first‑line therapy for metastatic triple‑negative breast cancer (TNBC). Phase 3 data showed Datroway reduced the risk of disease progression or death by 43%...
Discovery of Novel 11 Beta-Hydroxysteroid Dehydrogenase Type 1 Inhibitor by Machine Learning Enabled Large-Scale Virtual Screening
Researchers applied a machine‑learning‑driven virtual screening workflow to the Mcule catalog of roughly 139.6 million compounds, ultimately identifying a single top candidate, MCULE‑6869845113, as a potent 11beta‑HSD1 inhibitor. The Gradient Boosting Classifier achieved an AUC of 0.88 and Matthews Correlation Coefficient...
When Psychedelics Work Therapeutically
Psychedelic compounds such as psilocybin and MDMA are re‑emerging in clinical trials, especially for PTSD, backed by substantial private funding and rigorous protocols. Researchers link their therapeutic effect to the brain's predictive‑coding system, proposing the REBUS model where psychedelics temporarily...
Clinical Operations Burnout Is Undermining Patient Enrollment
Clinical operations burnout is eroding patient enrollment and retention in trials, with nearly half of healthcare workers reporting burnout symptoms in 2022. Around 80% of studies miss their recruitment timelines, a problem tied to overburdened site teams handling growing protocol...
One-and-Done Heart Disease Prevention? Scientists Show It May Be Possible.
Scientists reported that a single infusion of an experimental gene‑editing drug lowered LDL cholesterol by up to 62 percent in an interim analysis of 35 patients. The reduction remained durable in a subgroup treated 18 months earlier, suggesting a potentially...
Pharming Reports the EC Approval of Joenja (Leniolisib) for Activated Phosphoinositide 3-Kinase Delta Syndrome (APDS)
The European Commission has granted marketing authorization for Pharming’s oral PI3Kδ inhibitor Joenja (leniolisib) to treat patients aged 12 and older with activated phosphoinositide 3‑kinase delta syndrome (APDS) across all 30 EEA states. Approval is based on a Phase II/III...
Caliway Secures FDA Clearance for CBL-514 Clinical Trial
Caliway Biopharmaceuticals received FDA clearance for its CBL‑0302 IND application, clearing the path for a pivotal global Phase III trial of CBL‑514 aimed at reducing abdominal subcutaneous fat. The double‑blind, placebo‑controlled study will enroll roughly 320 participants in the United States, Canada...
Gilead Reports the US FDA Accelerated Approval of Hepcludex to Treat Chronic Hepatitis Delta Virus (HDV) Infection
Gilead Sciences received accelerated approval from the U.S. Food and Drug Administration for Hepcludex (bulevirtide‑gmod), an 8.5 mg subcutaneous injection for adults with chronic hepatitis delta virus (HDV) infection. The decision is based on data from the Phase III MYR301 trial, which...
Ionis Pharmaceuticals’ SWOT Analysis: Stock Gains Momentum on FDA Approval
Ionis Pharmaceuticals (NASDAQ:IONS) secured FDA approval for REDEMPLO, the first siRNA therapy targeting familial chylomicronemia syndrome (FCS), in November 2025. The drug’s clean label, quarterly at‑home dosing and broad indication have positioned it for rapid uptake, with payer agreements covering...
Venus Remedies Gets Saudi FDA Approval for Speciality Oncology Therapy
Venus Remedies Limited announced that the Saudi Food and Drug Authority has granted marketing authorisation for its specialty oncology drug Plerixafor, a stem‑cell mobiliser used in autologous transplants. This marks the first global approval for the therapy, positioning Saudi Arabia...
Gene-Based Therapies Could Transform Future Pancreatitis Treatment
Pancreatitis remains a largely untreatable inflammatory disease, with current care limited to symptom management. Gene‑based therapies—spanning augmentation, inhibition, and editing—are emerging to address the genetic drivers across lipid‑metabolism, trypsin regulation, ductal secretion, and ER‑stress pathways. Recent preclinical work shows AAV‑mediated...
ASCO to Spotlight Pancreatic Cancer Innovation Beyond KRAS
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New Indicator for Response to Therapy in Pediatric Cancers Identified
Researchers at the University of Birmingham reported that a high aneuploidy score can predict which children with relapsed solid tumors respond to a combined low‑dose irinotecan and PARP‑inhibitor regimen. The Phase I/II eSMART arm enrolled 70 patients across the UK, France,...