Disease or Syndrome
ACC25: Merck & Co.’s Winrevair reduces mortality in most severe PAH patients
Pulmonary arterial hypertension, Winrevair, Severe (severity modifier), Mortality Vital Statistics
Biotech Restructuring and European Regulatory Setbacks
LifeMine Therapeutics, layoffs, PTC Therapeutics, Translarna, Duchenne muscular dystrophy, European Commission, regulatory decision
Lilly’s Lepodisiran Shows Durable Lipoprotein(a) Reduction in Phase 2 Trial
lepodisiran, lipoprotein(a), Lp(a), cardiovascular disease, RNA interference, long-acting therapy
Beyond Black Box: How Data-Driven AI Is Transforming RNA Medicine Development
Aortic Valve Insufficiency, development aspects, RNA medicine, black box, Data-Driven AI, Transforming RNA Medicine Development, datasets, Drug Design, Clinical Trials
CHMP recommends seven label expansions and one new approval, rejects Lilly Alzheimer’s drug
CHMP, Labels (device), expansions, Approved
FDA Rejects Milestone Pharmaceuticals’ Etripamil Nasal Spray for PSVT
Etripamil, Cardamyst, PSVT, FDA rejection, Complete Response Letter, manufacturing issues, Milestone Pharmaceuticals
Novo inks second obesity deal of the week, with Lexicon oral drug partnership worth up to $1B
Obesity, LX9851, lexicon, Mechanism (attribute)
Equillium’s pivotal GvHD study of anti-CD6 antibody comes up short
United States Food and Drug Administration, itolizumab, In complete remission, day, Equillium
Nkarta lays off 3rd of staff, including CFO, to fund autoimmune CAR NK program
Nkarta, restructuring, Allogeneic CD19-OX40-CD3zeta-CAR-mbIL-15-expressing Natural Killer Cells NKX019, Cell Therapy
FDAs policy, communications staff likely to be among agencys 3,500 staff cuts
Policy, Communication, Staff, United States Food and Drug Administration, CHARGE Syndrome