Argo Biopharma’s updated Phase 2 data show up to a 96% reduction in hereditary angioedema attacks with BW-20805, while plasma prekallikrein suppression remained substantial through Day 169.
The FDA has cleared Acepodia’s ACE723 IND, allowing the GPC3-targeted dual-payload antibody-drug conjugate to begin Phase 1 testing in unresectable or metastatic hepatocellular carcinoma.
AbbVie’s Phase 3 CERVINO trial met both primary endpoints as etentamig achieved a 74% response rate and reduced progression or death risk by 60% in heavily pretreated multiple myeloma.
FDA approves Etcamah after an ESR1 mutation is detected in blood before breast cancer progresses on scans, creating a first for ctDNA-guided treatment.
eGenesis says five patients have received EGEN-2784 pig kidneys under Expanded Access, with three maintaining kidney function beyond eight months and two later receiving human donor kidneys.
Stoke Therapeutics and Biogen say four-year zorevunersen data show durable seizure reductions plus continuing cognitive and behavioral improvements as Phase 3 EMPEROR advances.
The FDA has approved Ionis Pharmaceuticals’ ZANVASTRO for pediatric and adult Alexander disease after a 54-patient study showed significant stabilization of gait at 61 weeks.
Scholar Rock has started dosing in the 60-patient Phase 2 FORGE trial of apitegromab in facioscapulohumeral muscular dystrophy after receiving FDA Fast Track and Orphan Drug designations.
The FDA has accepted United Therapeutics’ supplemental Tyvaso application for idiopathic pulmonary fibrosis after combined TETON data showed a 111.8 mL advantage in forced vital capacity over placebo at 52 weeks.
uniQure has submitted AMT-130 for accelerated U.S. approval after three-year Phase 1/2 data showed a 75% slowing of Huntington’s disease progression against a matched natural-history control.