Two-year ORIGIN 3 data show weekly atacicept flattened eGFR decline to 0.6 mL/min per year and cut kidney-progression events by 76 percent, with no dialysis or transplants on drug.
Definium's Phase 3 Panorama study met its primary endpoint in generalized anxiety disorder with a 5.1-point HAM-A gap, testing whether an active 50 µg control and zero psychotherapy can clear the FDA hurdle that broke Lykos.
The FDA has granted accelerated approval to Bayer's Hyrnuo as a frontline therapy in HER2-mutant lung cancer, matching rival zongertinib's response rates while carrying twice-daily meal requirements and steeper gastrointestinal toxicity.
FDA approval of Isembyld gives spinal muscular atrophy its first muscle-targeted therapy, moving motor function in patients plateaued on Spinraza or Evrysdi while introducing a 9% fracture warning and a double-biologic payer burden.
Solstice Oncology launched with a $225 million Series A led by RA Capital to advance Harbour BioMed-licensed porustobart into neoadjuvant MSS colon cancer.
Pharvaris reported Phase 3 CHAPTER-3 data for deucrictibant extended-release 40 mg, delivering an 83% attack reduction in hereditary angioedema and matching the efficacy of injectable biologics.
KRAS sat on the undruggable list for decades. Silexion's SIL204 is built to silence the mutation earlier in the cell, and the Phase 2/3 program is now moving into patients.
Del-desiran missed its primary endpoint in myotonic dystrophy type 1, wiping 13% off Novartis, pulling rival Dyne down 22%, and casting doubt over TfR1 RNA delivery.
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.
Novartis said pelacarsen lowered Lp(a) in patients already on guideline-directed care, but Lp(a)HORIZON missed its cardiovascular composite versus placebo. The first large outcomes test of the inherited risk factor now hangs over later trials from Amgen and Lilly.
AbbVie's etentamig beat older salvage on response and progression-free survival in Phase 3 CERVINO. The harder question is whether a single step-up and monthly intravenous schedule can loosen the REMS-and-hospitalization architecture that still gates approved BCMA T-cell engagers.
uniQure asked FDA for accelerated approval of AMT-130 on a 12-patient, 36-month high-dose dataset, after the agency twice reversed itself on external-control evidence.