Carephar Pharmaceutical Co. Ltd. obtained marketing approval for keverprazan (KFP-H008) from China’s NMPA for use in duodenal ulcer and reflux esophagitis treatments, allowing it to tap an increasingly competitive market.
One of the most important transformations in the pharmaceutical industry over the next decade is the ability to modify genes or expression and go after any target, Cargene Biopharma Inc. CEO Kathy He told BioWorld, explaining that small molecules and large molecules can only go after 15% of the known targets. But the technology platform of short-interfering RNA (siRNA) opens a huge opportunity to make those inaccessible targets available, she said.
Hightide Therapeutics Inc. closed a $107 million series C round that will advance multiple global development programs for lead candidate HTD-1801, including a phase III trial in type 2 diabetes.
Phathom Pharmaceuticals Inc.’s shares sank 31% on news that the U.S. FDA will not take action on the company’s NDA for oral small-molecule potassium-competitive acid blocker vonoprazan by the Jan. 11 PDUFA date.
“Far superior to what almost anyone expected,” was how H.C. Wainwright analyst Ed Arce described the top-line readout for Madrigal Pharmaceuticals Inc.’s phase III study of resmetirom, which hit both of its dual endpoints in patients with nonalcoholic steatohepatitis (NASH) and is expected to form the basis of an accelerated approval application to the U.S. FDA in the first half of 2023.
Patients suffering in the long-stagnant therapeutic landscape of ulcerative colitis and Crohn’s disease gained encouragement with positive mid-stage results from Prometheus Biosciences Inc., which tested its tumor necrosis factor-like cytokine 1A-targeting PRA-023 against both forms of inflammatory bowel disease. San Diego-based Prometheus closed at $95.80, up $59.74, or 165.7% after the company made known data from the phase II study called Artemis-UC and the Apollo-CD phase IIa experiment. Prometheus intends to advance PRA-023 into phase III testing next year.
New and updated preclinical and clinical data presented by biopharma firms at the AASLD Liver Meeting, including: 89bio, Albireo, Ascentage, Gannex, Seal Rock, Vaccitech, Vir.
Modern molecular techniques have progressed to the point where sequencing can seem almost quaint. At the Basic Science Symposium of The Liver Meeting 2022, new techniques were on full display, with sessions devoted to epigenetics, microbiome analysis and spatial transcriptomics. But the first session was still on genetic variants in all their forms – rare variants, common variants and non-germline mutations.
New and updated preclinical and clinical data presented by biopharma firms at the AASLD Liver Meeting, including: Aligos, Asher, Assembly, Ocelot, Rivus, Terns.
Mirum Pharmaceuticals Inc. plans to “move rapidly toward regulatory filings” to expand use of IBAT inhibitor Livmarli (maralixibat) into progressive familial intrahepatic cholestasis (PFIC), President and CEO Chris Peetz told investors during a conference call Oct. 24 to discuss the positive top-line results from the phase III March study, which not only hit its primary endpoint in patients with PFIC2 but showed statistical improvements in pruritis across a range of PFIC subtypes.