Once again, Arrowhead Pharmaceuticals Inc.’s Redemplo (plozasiran) is hot on the heels of Ionis Pharmaceuticals Inc.’s Tryngolza (olezarsen). Less than a month after the latter gained U.S. FDA approval for use in severe hypertriglyceridemia (sHTG), Arrowhead reported positive top-line results from its phase III studies in sHTG, with findings described by one analyst as “best-case scenario,” setting the stage for a supplemental NDA filing with the FDA before year-end.
Atrium Therapeutics Inc. has obtained IND clearance from the FDA for ATR-1072 for the treatment of protein kinase AMP-activated non-catalytic subunit γ2 (PRKAG2) syndrome.
Astrazeneca plc has returned to China’s CSPC Pharmaceutical Group Ltd. for another discovery collaboration, this time in a deal worth up to $1.77 billion to use CSPC’s siRNA drug discovery platform and extrahepatic targeted delivery technology to develop small nucleic acid drug candidates.