A gene therapy intended as a one-time treatment for Huntington’s disease significantly slowed progression of the disorder in a pivotal Phase 1/2 trial, paving the way for a regulatory submission in the first quarter of 2026. The developer, uniQure NV, showed that it is possible to safely impact the disease by targeting both the disease-causing huntingtin protein and the normal protein using a vector-based gene therapy and a gene encoding a microRNA (miRNA).
The European Medicines Agency has issued a positive opinion for a new indication of an antibody therapeutic that targets B cells in order to treat immunoglobulin G4 related diseases. These are autoimmune disorders caused when the body’s own defence system attacks normal tissues. This can lead to fibrosis and inflammation in one or multiple organs of the body. The therapy, Uplizna (inebilizumab), has already been approved for the treatment of neuromyelitis optica spectrum disorder, an inflammatory disorder of the central nervous system.
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.