UK-based Compass Pathways Plc has reported positive Phase 3 data for a synthetic psilocybin, and the first classic psychedelic for potential use in treatment-resistant depression. The small molecule drug, COMP360, achieved a clinically meaningful reduction in symptom severity compared with a placebo as measured on the Montgomery-Åsberg Depression Rating Scale, according to the company.
Liraglutide, a glucagon-like peptide-1 (GLP-1) receptor agonist approved to treat both obesity and type 2 diabetes, has shown effectiveness as a potential medicine for chronic migraine, according to a study presented at the European Academy of Neurology Congress on 21 June. Researchers at the University of Naples Federico II reported that liraglutide, administered to 26 adults with obesity and migraine, was able to significantly reduce episodes of migraine headache over a period of 12 weeks. At the same time, participants only registered slight decreases in body mass index (BMI).
The Philadelphia, US, based biotech company NodThera Ltd has started dosing patients in a trial of a small molecule drug for obesity that targets a protein in an inflammasome complex in the brain. The Phase 2 study will enrol 160 patients who are obese and may or may not also have type 2 diabetes. The trial will be carried out over 24 weeks with headline data expected in the second quarter of 2026. Although it is small, the trial is being positioned to test a relatively new concept: whether it is possible to change the course of obesity by correcting underlying metabolic dysfunction.
TILT Biotherapeutics Ltd has raised €22.6 million from the European Innovation Council and other investors to bring its oncolytic virus therapy for patients with ovarian cancer into Phase 2. The funding will also support earlier studies for the product, TILT-123, in other indications. TILT-123 is an oncolytic adenovirus encoding tumour necrosis factor alpha and interleukin 2. It is designed to work alongside T cell therapies and immune checkpoint inhibitors to target and kill cancer cells.
Arvinas Inc, a US biotech with a portfolio of protein degrader molecules, has reported positive data for a potential treatment for neurological diseases. The data were presented at the International Conference on Alzheimer’s and Parkinson’s Diseases in Vienna, Austria on 4 April, showing that the molecule was both safe and able to engage with the cellular target. The target is leucine-rich repeat kinase 2 (LRRK2), a protein that has been implicated in Parkinson’s disease and progressive supranuclear palsy. The Phase 1 study was conducted in healthy male volunteers.
A retrospective, observational study of semaglutide 2.4 mg (Wegovy) has shown that adults who are overweight with an established cardiovascular disease, but no diabetes, were able to reduce their risk of a major cardiovascular event over a period of years. Called SCORE, the trial compared outcomes for semaglutide users with non-users and established benefit for the drug taken as an adjunct to diet and exercise. The study started in 2018, enrolled 17,604 adults, and was conducted in 41 countries at more than 800 sites.
A new gene therapy for cystic fibrosis has entered clinical development in the UK with the aim of improving outcomes for people with cystic fibrosis regardless of their gene mutation. The trial specifically focuses on adults with cystic fibrosis who cannot benefit from the current modulators of the membrane protein – cystic fibrosis transmembrane conductance regulator (CFTR).
A radiopharmaceutical which is being developed for patients with neuroendocrine tumours, a rare form of cancer, has delivered positive data from a Phase 3 trial paving the way for a possible regulatory submission this year, according to the developer ITM Isotope Technologies Munich SE. The therapy, ITM-11, achieved statistically significant progression- free survival for patients compared with everolimus, a small molecule immunosuppressant that was first approved by the US Food and Drug Administration in 2009.
Shanghai, China-based YolTech Therapeutics Co Ltd has started clinical development of an in-vivo gene editing product for beta-thalassaemia, an inherited blood disorder that is estimated to involve one in every 100,000 patients worldwide. The disease affects the production of normal haemoglobin, a protein in red blood cells that carries oxygen to tissues in the body.
Zolgensma (onasemnogene abeparvovec), the gene therapy developed by Novartis for spinal muscular atrophy (SMA), has shown efficacy in a Phase 3 study of patients aged two to less than 18 years who are able to sit but have never walked independently. SMA is a genetic neuromuscular disease caused by the lack of a functional SMN1 gene that affects muscle functions and basic movement. Zolgensma was first approved in the US in 2019, and one year later in the EU, as a paediatric drug for children less than two years old and administered as a single, one-time infusion.