The debate around the U.S. 340B prescription drug discount program is once again heating up in court and in Congress. A day after the American Hospital Association called on the FTC and Department of Justice to investigate alleged antitrust issues with the rebate models a few drug companies have proposed, some members of Congress raised concerns Sept. 9 about how providers are abusing the program. Meanwhile, a U.S. appellate court heard arguments that same day on whether states can speak in the silence of the federal law that created the program more than 30 years ago.
Two months after starting the phase I/II Synrgy trial with its gene therapy, CAP-002, enrolling 12 pediatric patients with rare disease STXBP1 encephalopathy, Capsida Biotherapeutics paused the study following the death of the trial’s first patient.
LB Pharmaceuticals Inc. became the first traditional biopharma company to price an IPO in the U.S. since February, raising $285 million through the upsized sale of 19 million shares at $15 apiece. Shares, now listed on Nasdaq under the ticker symbol LBRX, rose 15% to close Sept. 11 at $17.30.
Maze Therapeutics Inc. CEO Jason Coloma said the latest phase I results with MZE-782, a prospect for phenylketonuria (PKU) as well as chronic kidney disease (CKD), bolster the firm’s “conviction to move into phase II” next year in both indications.
Positive results for Revolution Medicines Inc.’s phase I studies of its lead candidate, the RAS-blocker daraxonrasib for treating pancreatic ductal adenocarcinoma, prompted the company to say it’s time for a phase III study in the aggressive cancer. Revolution said it plans to begin a global, randomized and registrational trial in first-line metastatic disease sometime in the fourth quarter of 2025.
A tangle of DNA can look like a knotted ball in the cell nucleus. However, the genetic machinery has a complex and regulated structure. Its long repetitive sequences also seemed to have no function. They were called junk DNA, although they were not. The same happened with proteins and low-complexity domains, disordered chains of amino acids that were poorly understood. Nevertheless, that protein noise has turned into music for the 2025 Lasker Awards. These prizes have recognized the work of scientists who were able to see order in chaos.
RSS
