Clinical Research

The UK-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. 

A second candidate drug for Parkinson’s disease has failed to meet the primary endpoint in a clinical study of patients with early-stage disease. The Roche Group announced the results of its Phase 2b PADOVA study on 19 December, only days after UCB SA said its medication for early Parkinson’s disease had failed to show efficacy. Roche said it will continue to evaluate data from the main trial and an ongoing extension study in deciding what to do. The candidate drug, prasinezumab, is a monoclonal antibody designed to bind to alpha-synuclein protein and reduce neuronal toxicity.

A candidate drug for early-stage Parkinson’s disease has failed to show efficacy at Phase 2a despite having demonstrated promise in earlier studies, the co-developer UCB SA announced on 16 December. The drug, minzasolmin, is a small molecule compound designed to inhibit alpha-synuclein misfolding in the brain. Alpha-synuclein is a normal neuronal protein that regulates synaptic vesicle trafficking. However in Parkinson’s disease it misfolds and aggregates into toxic fibrils.