Inovio's Voceryq is the first plasmid DNA immunotherapy to win FDA approval. It cleared on a 27% complete response in a small open-label cohort, and a confirmatory trial is still ahead.
Top stories
Newsletter
Pipeline, markets, policy, and science. Once a week.
Its $1.275 billion upfront commitment to a Phase 1 program puts the spotlight on oral peptide developers, including Entera as it approaches Phase 3 in osteoporosis.
AstraZeneca's fifth Ultomiris indication arrives on a proteinuria surrogate, while the week-106 eGFR readout still decides whether the drug actually saves kidneys.
Bepirovirsen is the first therapy to offer people with chronic hepatitis B a shot at functional cure after a six-month course. It works in about one in five, and only in a narrow, already-treated population.
In depth
AHEAD-MERIT missed its progression-free survival endpoint. In this disease, pembrolizumab itself never won PFS either. The trial now turns to overall survival, the endpoint where this cancer actually rewards immunotherapy.
Read the storyArriVent's firmonertinib beat chemotherapy on investigator reads in a 398-patient Phase 3, but the blinded central review it chose returned p=0.0654, and first-line EGFR exon 20 lung cancer now belongs to combinations that keep chemotherapy in the regimen.
CSL is paying $355 million upfront for an antibody whose best evidence is a 26-patient interim readout. The deal prices claudin-1 biology, not proven data, across kidney and liver fibrosis.
The FDA granted accelerated approval to AstraZeneca’s camizestrant based on ctDNA resistance signals before radiographic progression, overruling a 6-3 advisory committee vote and betting on a new oncology paradigm.
Conjugating antisense oligonucleotides to transferrin receptor transport vehicles achieves fifty-fold deeper brain penetration, challenging the standard of invasive spinal punctures.
