Emily Schoenthaler is content producer for BioPharm International®.
Emerging sequential treatment study designs may simplify and accelerate IV-to-subcutaneous biologic conversions by reducing variability and shrinking clinical trial sizes. Charles Theuer discussed how streamlined pharmacokinetic (PK)-driven regulatory pathways and evolving EU initiatives are reshaping subcutaneous biologic development.
REGENXBIO announced positive topline Phase 3 AFFINITY DUCHENNE trial results showing that investigational gene therapy RGX-202 achieved significant microdystrophin expression and demonstrated a correlation with functional improvement in patients with Duchenne muscular dystrophy.
AstraZeneca reported that the Phase 3 VOLGA trial demonstrated statistically significant improvements in event-free survival and overall survival with perioperative durvalumab plus neoadjuvant enfortumab vedotin in cisplatin-ineligible patients with muscle-invasive bladder cancer.
Yelena Ionova, Pharm.D., Redica Systems, discusses how benchmarking inspection data, strengthening sterile manufacturing controls, and adopting continuous regulatory intelligence strategies can help cell and gene therapy manufacturers improve inspection readiness and quality system performance.
Yelena Ionova, Pharm.D., Redica Systems, described how FDA and EMA standards for cell and gene therapy chemistry, manufacturing, and controls are increasingly aligned, yet manufacturers continue to struggle with basic GMP elements such as quality oversight, sterile processing, and staff training. She emphasized that newer CGT facilities have a critical opportunity to design quality systems and data integrity controls correctly from the outset to avoid costly remediation later.
New analyses from Novo Nordisk’s Phase 3 OASIS 4 trial showed that adults taking oral Wegovy achieved substantial weight loss and improved physical mobility, reinforcing the potential of oral semaglutide in obesity management.
AstraZeneca reported positive Phase 3 CALYPSO data showing eneboparatide normalized serum calcium and reduced dependence on conventional supplements in adults with chronic hypoparathyroidism, supporting continued development of the investigational PTH receptor agonist.
Today’s BioPharm Brief covers a major gene therapy milestone, an FDA Breakthrough Therapy designation in rare cancer, and a multi-billion-dollar oncology partnership shaping global drug development pipelines.
AskBio has dosed the first participant in a Phase 1/2 clinical trial evaluating AB-1009, an investigational AAV-based gene therapy for late-onset Pompe disease.
Updated phase 1 KOMET-007 data suggest that adding ziftomenib to standard 7+3 induction therapy may improve remission and MRD-negativity rates in newly diagnosed AML with NPM1 mutations or KMT2A rearrangements.
Servier received FDA Breakthrough Therapy designation for emiltatug ledadotin, a B7-H4-targeting antibody-drug conjugate being developed for patients with advanced adenoid cystic carcinoma.
Zai Lab received FDA Fast Track designation for zocilurtatug pelitecan, a DLL3-targeting antibody-drug conjugate, advancing development of a potential new treatment option for extrapulmonary neuroendocrine carcinomas.
argenx secured expanded FDA approval for efgartigimod alfa-fcab and efgartigimod alfa and hyaluronidase-qvfc broadening access to all adult patients with generalized myasthenia gravis regardless of antibody status.
Today’s BioPharm Brief covers next-generation immunotherapy, scalable peptide manufacturing, and Johnson & Johnson’s dual-pathway approach to inflammatory bowel disease treatment.
Harbour BioMed has received FDA IND clearance to begin a Phase I trial of its B7H4xCD3 bispecific antibody HBM7004 for advanced solid tumors, advancing its pipeline of T cell–engaging cancer immunotherapies.
Johnson & Johnson reported Phase 2b results for its investigational dual-pathway co-antibody JNJ-4804, showing improved clinical remission and endoscopic outcomes in patients with refractory inflammatory bowel disease.
Merck scientists have published a new large-scale biocatalytic manufacturing approach for enlicitide decanoate, an investigational oral PCSK9 inhibitor, potentially advancing scalable production of macrocyclic peptide therapeutics for cardiovascular disease.
Today’s BioPharm Brief covers promising AML remission data from Aptevo, encouraging Duchenne muscular dystrophy trial results from Entrada Therapeutics, and a new GSK licensing deal expanding cardiometabolic RNA therapy development.
Aptevo Therapeutics reported updated Phase 1b data from its RAINIER AML study showing high remission and clinical benefit rates for mipletamig in combination with venetoclax and azacitidine in frontline acute myeloid leukemia patients.
Entrada Therapeutics reported positive topline Phase 1/2 data for ENTR-601-44 in Duchenne muscular dystrophy, showing favorable safety, increased dystrophin production, and statistically significant functional improvement in treated patients.
Kanvas Biosciences and LTZ Therapeutics raised a combined $86 million to advance novel cancer immunotherapy platforms targeting the microbiome and innate immune system, respectively.
Biopharma news today highlights precision MASH therapies, emerging immuno-oncology platforms, and major U.S. manufacturing investments reshaping drug development and supply chains.
Amgen and Eli Lilly and Company are expanding U.S. pharmaceutical manufacturing with multibillion-dollar investments aimed at strengthening domestic supply chains and supporting advanced therapies. The moves reflect a broader industry shift toward biologics scale-up, genetic medicine capabilities, and more resilient production infrastructure.
Madrigal licensed Arrowhead's ARO-PNPLA3 to target PNPLA3 I148M-associated metabolic dysfunction–associated steatohepatitis, expanding options beyond approved resmetirom.
Phase III data in thyroid eye disease, long-term ulcerative colitis outcomes, and Crohn disease advances highlight continued momentum in biologics and immunology innovation.
New long-term data reported by Eli Lilly and Company show Omvoh achieved sustained disease clearance through four years in ulcerative colitis, highlighting the potential durability of interleukin-23–targeted therapies.
Phase 3 FUZION data showed guselkumab improved fistula remission vs placebo in adults with perianal fistulizing Crohn disease.
FDA approvals, biotech acquisitions, and clinical trial updates are shaping today’s biopharma landscape. This episode of The BioPharm Brief covers the first approved PROTAC therapy for breast cancer, UCB’s planned acquisition of Candid Therapeutics, and new clinical data from Takeda Pharmaceutical Company in primary immunodeficiency.
The FDA has approved vepdegestrant, the first PROteolysis TArgeting Chimera protein degrader therapy, for estrogen receptor gene-mutated ER+/HER2- advanced breast cancer, offering a new option for patients with endocrine-resistant disease.
UCB’s planned $2.2 billion acquisition of Candid Therapeutics strengthens its immunology pipeline with bispecific T-cell engagers, led by cizutamig, a BCMA/CD3-targeting antibody in early-stage trials for autoimmune diseases.
