Also known as: RNAi
Arrowhead Pharmaceuticals is in focus after first-quarter results moved from a year-ago loss to profitability and the company shifted from the S&P 600 to the S&P 400. Investors are also watching progress in its RNAi pipeline, including two Phase 3 therapies, while valuation measures show a mixed picture.
Arrowhead Pharmaceuticals presented long-term data showing plozasiran achieved 83% median triglyceride reduction in severe hypertriglyceridemia patients with no acute pancreatitis events over two years. The RNA interference therapy demonstrated durable improvements in atherogenic lipoproteins and consistent safety profile. The company plans to complete Phase 3 studies by mid-2026 and submit for regulatory approval.
Pharmaceutical companies are developing innovative hair loss treatments that directly activate hair growth pathways rather than suppressing male hormones, with several candidates entering clinical trials and promising enhanced safety profiles.
Pharmaceutical companies are increasingly partnering with biotechs developing RNA-targeting small molecules, driven by advances in RNA structural biology and the success of drugs like Roche's Evrysdi. The approach aims to address "undruggable" targets while offering oral availability advantages.
Benitec Biopharma will present interim clinical study results for BB-301 gene therapy in oculopharyngeal muscular dystrophy patients at the Muscular Dystrophy Association Conference on March 9, 2026, including 12-month and 24-month follow-up data.
