A gene therapy being developed by Beacon Therapeutics Holdings Ltd to treat X-linked retinitis pigmentosa has shown progress in two ongoing Phase 2 trials, SKYLINE and DAWN. The product, laru-zova, was generally well tolerated by participants evaluated in the 36-month SKYLINE trial and by participants in the nine months DAWN trial, showing improvements across key measures of visual function, including low luminance visual acuity and microperimetry.
A gene therapy being developed by Beacon Therapeutics Holdings Ltd to treat X-linked retinitis pigmentosa has shown progress in two ongoing Phase 2 trials, SKYLINE and DAWN. The product, laru-zova, was generally well tolerated by participants evaluated in the 36-month SKYLINE trial and by participants in the nine months DAWN trial, showing improvements across key measures of visual function, including low luminance visual acuity and microperimetry.
Switzerland-based Aphaia Pharma AG has started a second Phase 2 trial of a new oral candidate drug for obesity which aims to restore endogenous endocrine balance in individuals with the disorder. The drug is a coated formulation of glucose, which is included on the World Health Organization’s list of essential medicines. It is designed to be released in the small intestine to restore endogenous nutrient-sensing signalling pathways and stimulate the release of hormones that control multiple functions including appetite, hunger, satiety and glucose metabolism.
Eli Lilly and Co’s investigational obesity drug, orforglipron, achieved a weight loss of 10.5% at the highest dose in a third Phase 3 trial whose results were reported on 3 September. Orforglipron is small molecule glucagon-like peptide-1 (GLP-1) receptor agonist designed to be taken once per day without restrictions on food and water. It differs from Lilly’s first approved weight-loss drug Zepbound (tirzepatide) which is a polypeptide drug, administered subcutaneously and intended to be taken with a diet and exercise.
Ionis Pharmaceuticals Inc announced positive data on 2 September from two Phase 3 trials for its RNA-targeted medicine olezarsen (Tryngolza) in patients at risk of acute pancreatitis events. Tryngolza has already been approved in the US for the treatment of familial chylomicronaemia syndrome, a genetic disease that prevents the body from breaking down triglyceride fats. The company is now seeking to expand the drug’s use for people with severe hypertriglyceridaemia. This disorder occurs when the levels of triglycerides are significantly elevated, increasing the risk of acute pancreatitis.
Wegovy (semaglutide), the weight-loss drug developed and now marketed by Novo Nordisk A/S, is able to cut the risk of heart attack, stroke or death by 57% compared with tirzepatide (Zepbound), the competing drug developed by Eli Lilly and Co, according to the Danish pharma company. Novo presented the comparative data, derived from a real-world study of the two drugs’ benefits, at the annual European Society of Cardiology meeting in Madrid on 31 August.
Roche and Alnylam Pharmaceuticals Inc are to advance their jointly developed RNA interference therapy for hypertension into a Phase 3 trial following clinical data showing that the drug, zilebesiran, could have a significant impact on patients with uncontrolled hypertension. The data comes from three Phase 2 trials which tracked the performance of the drug in patients with hypertension and high cardiovascular risk.
AstraZeneca Plc disclosed the full results of its Phase 3 trial of baxdrostat, a drug for hypertension, on 30 August, confirming the therapy’s ability to reduce systolic blood pressure in patients with hard-to-control hypertension. In a presentation at the European Society of Cardiology Congress in Madrid, the company said that baxdrostat, a small molecule drug that inhibits aldosterone synthase, met all primary and secondary endpoints, delivering sustained blood pressure reductions.
Abivax SA reported a pooled clinical remission rate of 16.4% in two Phase 3 trials of its small molecule drug, obefazimod, for ulcerative colitis on 22 July, in one of the largest clinical development programmes for the disease ever conducted, The trials were eight-week induction studies and are being followed up by a maintenance trial which, if positive, will lead to a regulatory filing. Top-line results from the maintenance trial are expected in the second quarter of 2026, the company said.
Cosentyx (secukinumab), one of Novartis’ top-selling drugs, has failed to show efficacy in a Phase 3 trial of giant cell arteritis, an autoimmune disease affecting the blood vessels. The trial results were announced on 3 July and represent an unusual setback for a drug that has already been approved for seven immune-mediated inflammatory conditions. Cosentyx inhibits interleukin-17A and was first approved in the US in 2015 for plaque psoriasis. The name of the recent trial was GCAptAIN.