Section
Trials, approvals, and the drugs behind them.
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.
A pre-specified VESALIUS-CV analysis found evolocumab cut all-cause death by 20% in high-risk primary prevention, with the benefit appearing only after 1.5 years and a 1.8-point absolute gap at five years.
After encouraging early pancreatic data, demand for Alpha DaRT helped quadruple its IMPACT study from 12 patients to 48 as the company moves toward a pivotal trial.
The FDA's landmark approval of Revolution Medicines' daraxonrasib (Rasonque) introduces the first multi-selective RAS(ON) tri-complex inhibitor for metastatic pancreatic adenocarcinoma, doubling overall survival over standard chemotherapy.
MariTide delivered up to about 20% mean weight loss at 52 weeks without a plateau, then Amgen lost about $7 billion in market value. The remaining fight is GIP direction, first-dose gut burden, and conjugate manufacturing cost.
Suzetrigine beat placebo on SPID48 in both pivotal acute-pain trials, then failed the superiority test against hydrocodone/acetaminophen, including a statistically significant loss in bunionectomy.