Ionis Pharmaceuticals

Familial chylomicronemia syndrome (FCS) lacks approved therapies, but olezarsen and plozasiran show promise in phase 3 trials. Olezarsen, an antisense oligonucleotide, and plozasiran, a small interfering RNA, both demonstrated significant triglyceride reduction and reduced pancreatitis risk in FCS patients.

The FDA granted breakthrough therapy designation to Arrowhead Pharmaceuticals’ plozasiran for reducing triglycerides in familial chylomicronemia syndrome (FCS) patients, positioning it to compete with Ionis Pharmaceuticals’ olezarsen. Plozasiran, an RNA interference therapeutic, aims to lower triglycerides by inhibiting apolipoprotein C-III production. The decision was based on phase 3 PALISADE trial data showing an 80% triglyceride reduction and an 83% decrease in acute pancreatitis risk. Arrowhead plans to submit a new drug application to the FDA by year-end.

Intrinsic Medicine, led by CEO Alex Martinez, focuses on developing human milk oligosaccharides (HMOs) to treat chronic diseases like Parkinson’s and IBS, addressing the high cost of drug development and the shift towards orphan drugs. Their lead compound, 2’ Fucosyllactose (2’FL), has received approval in Australia for a phase 2 trial in Parkinson’s and IBS, leveraging the gut immune brain axis (GIBA) for disease treatment.

FDA approves Tryngolza (olezarsen) for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS), significantly lowering acute pancreatitis risk. Approved based on phase 3 trial data showing a 57% mean reduction in triglycerides over 12 months and a favorable safety profile. Ionis Pharmaceuticals granted approval.

GSK announced positive phase IIb trial results for bepirovirsen, showing potential for functional cure in chronic hepatitis B patients, with phase III trials starting H1 2023. Bepirovirsen demonstrated sustained clearance of HBsAg and HBV DNA, especially in patients with low baseline HBsAg levels.

ALS, a fatal neurodegenerative disease, has seen limited treatment options despite its discovery over a century ago. Recent scientific advancements offer hope, with new drugs like AMX0035 showing modest benefits in slowing disease progression and extending survival. However, challenges remain in understanding ALS's biology and developing effective treatments, especially for sporadic cases without known causes. The field is witnessing increased investment and research, aiming for breakthroughs that could significantly improve patients' lives.