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.
A prospective drug for schizophrenia failed to show efficacy in two Phase 2 trials leaving the developer, AbbVie Inc, without a molecule to challenge one of its biggest competitors in the field. The drug, emraclidine, is a small molecule positive allosteric modulator of the muscarinic M4 receptor designed to harness the psychosis in schizophrenia. Current antipsychotic drugs target a direct blockade of dopamine receptors which are overactive in schizophrenia.
Cambridge, US, based Wave Life Sciences Ltd reported positive proof of mechanism data on 16 October from a Phase 1b/2a study of an RNA editing oligonucleotide drug which restored levels of a critical protein in patients with alpha-1 antitrypsin deficiency, a genetic disease affecting the lung and liver. The estimated prevalence of the disease, with the mutation studied in the trial, is 200,000 in the US and Europe.
For the first time, a drug for diabetic macular oedema has been tested and shown to be effective in a cohort of individuals from minority groups who are disproportionately affected by the disease, the developer Roche announced on 18 October. Vabysmo (faricimab) is a bispecific antibody that was first approved in 2022 to treat age-related macular degeneration and diabetic macular oedema. Since that time it has also been authorised for retinal vein occlusion.
A retrospective analysis of biopsies taken from cancer patients who had undergone treatment with a checkpoint inhibitor and subsequently received an mRNA vaccine for coronavirus disease has shown an association between the vaccination and immune response to the therapies. Results of the analyses were presented at the 13 to 17 September meeting of the European Society for Medical Oncology. The lead author of the paper is Adam Grippin, a resident in radiation oncology at the University of Texas MD Anderson Cancer Center, US.
A Phase 2 trial of a small molecule drug for patients with mild dementia due to Alzheimer’s disease has been stopped due to lack of efficacy, the developer Sage Therapeutics Inc announced on 8 October. The drug, dalzanemdor (SAGE-718), did not show a statistically significant difference in patients with the disorder, compared with a placebo, on the basis of a widely-used intelligence test.
A consortium of universities and hospitals, with financial support from the UK government and industry, is to undertake a four-year project to collect data on patients who have received immunotherapy for cancer to establish the benefits and risks of these treatments. The treatments, which include checkpoint inhibitors, have extended the lives of many patients by enabling the body’s immune system to recognise and destroy cancer cells.