Clinical Research

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.

An antibody-drug conjugate drug for breast cancer, jointly developed by AstraZeneca Plc and Daiichi Sankyo Co Ltd, failed to show statistical significance in overall survival at Phase 3 versus chemotherapy despite having achieved progression free survival for the same patient group. Announcing the results on 23 September, AstraZeneca said that the survival results were likely to have been affected by changes in the standard of care for breast cancer during the drug’s development period.

ImmunOs Therapeutics AG, a Swiss biotech developing candidate therapies for cancer, has raised $11 million in a Series C financing round to advance its lead clinical programme targeting solid tumours. The product, IOS-1002, is a human leukocyte antigen (HLA)-based protein that block receptors in the innate immune system. The financing follows a Series B round that raised $74 million in 2022.

The latest financing was led by the existing investors Gimv, Pfizer Ventures, Mission BioCapital and BioMed Partners and supported by the new investor Double Point Ventures.

Data from an early clinical trial of a small molecule drug intended to treat solid tumours by modulating an enzyme that plays a key role in the presentation of antigen to the human immune system were positive, according to the developer Grey Wolf Therapeutics Ltd. Data from the Phase 1/2 study were presented at the 2024 American Society of Clinical Oncology annual meeting on 3 June. The drug, GRWD5769 was well-tolerated after repeat dosing. It also showed an ability to modulate the immunopeptidome.