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Neurizon advances NUZ-001 into 250-patient pivotal ALS trial; FDA signals potential registrational pathFDA has indicated the trial could serve as single registrational study, accelerating path to approval for monepantel.
Editas Medicine's EDIT-401 gene-editing therapy cleared for first-in-human trial in Australia for familial hypercholesterolemiaStrive trial marks advancement of in vivo CRISPR approach for heterozygous FH, addressing cardiovascular risk in 1 in 250 individuals.
FDA review documents suggest Revolution's Rasonque has narrower efficacy profile than marketed; Zealand diabetes data disappointDisclosure raises questions about Rasonque's competitive positioning; shares tumbled on revised expectations.
Glaukos licenses Revopsis' RO-104 trispecific biologic for neovascular age-related macular degeneration and diabetic eye diseaseMulti-indication potential targets three major pathways implicated in retinal vascular disease; clinical timeline not disclosed.
FDA approves Roche's Tecentriq for deficient-mismatch-repair stage III colon cancerFirst immunotherapy approved specifically for dMMR colorectal cancer, which carries elevated recurrence risk post-surgery.
More: Pharmaceutical Executive
Life Biosciences reports three glaucoma patients dosed with cellular reprogramming drug; no serious adverse eventsSingle-injection approach marks early-stage milestone for regenerative medicine application in ophthalmology.
Iambic Therapeutics, backed by tech and pharma investors, files for $135M IPO focused on oncologyListing marks continued momentum in biotech capital markets after strong 2026 IPO activity.
- Mitorx Therapeutics advances MTRX-31, a mitochondrial-targeted compound addressing metabolic dysfunction in obesity
Program targets complementary obesity mechanism beyond GLP-1 agonists, addressing treatment resistance and real-world limitations.
Vinci Pharmaceuticals closes Series A at over $8 million to advance Epiplant ocular delivery platformFunding supports clinical development of proprietary technology designed to improve ocular drug bioavailability.
Ono Pharmaceutical licenses Biohaven's IgG degrader portfolio for Asia-Pacific for $100M upfrontDeal covers Japan, South Korea, Taiwan; expands Ono's immunology and inflammation pipeline with degrader technology.
HHS announces vaccine injury research and reporting initiative led by RFK Jr., including new NIH clinic and monitoring systemProgram will offer reimbursements to physicians reporting suspected adverse events, signaling policy shift on vaccine safety infrastructure.
MimiVax's SurVaxM cancer vaccine extends survival in Phase IIb glioblastoma trialSURVIVE trial marks first reportable results from fully enrolled study of personalized neoantigen vaccine in newly diagnosed patients.
Trex Bio prices $116.7M IPO to advance Treg cell-activating immunology drugs for atopic dermatitis and IBDEli Lilly is largest shareholder; company targeting immunological conditions via regulatory T cell activation.
- Simcere Pharmaceutical develops bicyclic GBA positive allosteric modulators for lysosomal storage and neurodegenerative diseases
Compounds enhance endogenous glucosylceramidase activity, potentially offering oral alternative to enzyme replacement therapy.
Marvel Biosciences reports MB-204 preclinical efficacy in Shank3 mouse model of Phelan-McDermid syndromeImprovements across multiple behavioral endpoints suggest potential for rare neurodevelopmental disorder with limited treatment options.
Ayass Bioscience characterizes AYA-1809002, a DNA aptamer with selective, reversible anticoagulation activity40-nucleotide aptamer targets human α-thrombin, potentially offering advantages over conventional anticoagulants.
Bio-Techne opens GMP small-molecule manufacturing facility in Bristol, UKLife science tools provider expands in-house production capacity for regulated small-molecule products.
- Ningbo Jiefu Biotechnology identifies novel EGFR inhibitors and protein degraders for cancer treatment
New compounds target both kinase activity and protein degradation pathways in EGFR-driven malignancies.
Phase I trial of first Bundibugyo ebolavirus vaccine begins in Uganda as DRC outbreak spreadsUganda Virus Research Institute enrolling participants as virus continues circulating in neighboring Democratic Republic of Congo.
Argenx terminates Phase 3 Sjögren's trial for Vyvgart after futility; posts positive celiac disease Phase 2 dataIndependent monitoring board recommended stopping the study; celiac data fails to offset investor disappointment over loss of blockbuster expansion opportunity.
More: BioWorld, BioPharma Dive
- NIH revives emerging disease surveillance network but imposes wildlife virus-hunting restrictions that conflict with program mission
New policy curbs research on novel pathogen discovery, drawing scientist criticism over pandemic preparedness implications.
- Terumo takes $610M charge after losing major US customer for plasma collection device
Japan-based medical device maker's revenue impact signals customer consolidation or competitive loss in blood plasma market.
- FDA approves label expansions for AbbVie's Camzyos and BeiGene's Jaypirca; denecimig review extended
Camzyos gains indication in obstructive hypertrophic cardiomyopathy; Jaypirca approval in marginal zone lymphoma expands BeiGene's hematology footprint.
Viatris to acquire Pacira BioSciences for $1.65B, gaining non-opioid pain drugs Exparel and Zilretta with combined $700M 2025 revenueDeal priced at $36.50/share; Pacira's two marketed assets face looming patent cliffs Viatris plans to address through IP strategy.
More: Pharmaceutical Technology, BioWorld, BioWorld, Pharmaceutical Executive
FDA clears seven novel drugs in August 2026; global approvals pace 6.4% below 2025 year-to-dateWorldwide approvals totaled 38 in August versus 39 last year; January-August cumulative of 337 remains fourth-highest on record.