The higher of two doses of Ionis Pharmaceuticals Inc.’s drug olezarsen for treating adults with familial chylomicronemia syndrome reduced fasting triglyceride levels by 44% from baseline at the six-month mark in a phase III study, but one case of pancreatitis in the trial paints a less attractive picture than top-line data the firm reported in September.
Metabolic dysfunction-associated steatotic liver disease (MASLD), previously known as nonalcoholic fatty liver disease, shows different occurrence between sexes, being less prevalent in premenopausal women than in men or postmenopausal women.
China’s Visen Pharmaceuticals (Shanghai) Co. Ltd. is targeting an IPO on the Hong Kong stock exchange with three rare endocrine disease therapies licensed-in from Denmark’s Ascendis Pharma A/S, including U.S. FDA-approved Skytrofa (lonapegsomatropin).
It has been previously demonstrated that insulin-reactive B cells act as antigen-presenting cells to promote type 1 diabetes by stimulating pathogenic T cells and leading to destruction of insulin-producing beta cells of pancreatic islets.
Building D&D Pharmatech Inc. has been a rollercoaster ride, according to CEO Seulki Lee. The U.S. and Korea-based biotech is on another ascent, having scored U.S. FDA fast track designation for its metabolic dysfunction-associated steatohepatitis (MASH) drug, ahead of its third attempt at a public listing.
Vertex Pharmaceuticals Inc. has described α1-antitrypsin (SERPINA1) (Z-mutant) polymerization inhibitors reported to be useful for the treatment of α1-antitrypsin (AAT) deficiency.
Repair Biotechnologies Inc. has reported new data showing that its lipid nanoparticle (LNP)/messenger RNA (mRNA) therapy rapidly reversed the progression of atherosclerosis in mouse models applicable to familial hypercholesterolemia as well as atherosclerosis in the broader population.
In a potential watershed moment for South Korea’s Hanmi Group, a hair-splitting vote at the 51st shareholder meeting favored the two sons of the late founder and Chairman Lim Sung-ki, effectively halting a merger between Hanmi and OCI Holdings Co. Ltd.
Trouble continues to dog Verve Therapeutics Inc.’s base editor of the PCSK9 gene, VERVE-101, so the company paused enrollment in a phase Ib study in cholesterol lowering to focus on the similarly designed VERVE-102.
Disc Medicine Inc. CEO John Quisel said that top-line phase II findings from the study called Aurora with bitopertin in erythropoietic protoporphyria are “hard for us to interpret. This package of data is something that we’re going to have to sort through,” and the Watertown, Mass.-based firm expects to talk with the U.S. FDA about next steps in the second half of this year.