Clinical Research

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.

A gene therapy being investigated in a Phase 3 trial of boys with Duchenne muscular dystrophy (DMD) has failed to reach its primary endpoint of improvement in motor function, the developer Pfizer Inc announced on 12 June. The therapy, fordadistrogene movaparvovec, missed the primary endpoint at one year as well as key secondary endpoints compared with a placebo. The secondary endpoints included an improvement in velocity in a 10 metre run or walk. Participants were boys between the ages of four and seven who were on a daily regimen of glucocorticoids.

Eli Lilly and Co has reported positive data for tirzepatide, an approved drug for obesity and type 2 diabetes, which is now being investigated for metabolic dysfunction-associated steatohepatitis (MASH), a progressive liver disease. Formerly known as non-alcoholic steatohepatitis, MASH results from an excess of fat in the liver which can lead to liver-related complications and death. The global prevalence of the disease is increasing.

ITM Isotope Technologies Munich SE has raised €188 million from a group of investors to prepare for the commercial launch of its lead radiopharmaceutical product and increase its manufacturing capacity for radioisotopes. The equity financing was led by Temasek Holdings Ltd, a Singapore-based investment group, and supported by four other entities including the Qatar Investment Authority. ITM has a pipeline of candidate radiopharmaceuticals and diagnostics for cancer as well as manufacturing agreements to supply medical radioisotopes to others, including Novartis.