Clinical Research

Valneva SE has started submitting regulatory data in support of its Covid-19 vaccine candidate to the UK Medicines and Healthcare products Regulatory Agency (MHRA) with a view to receiving an initial approval before the end of the year. The vaccine, VLA2001, is currently being studied in separate Phase 3 trials in the UK and New Zealand. Top-line results from the UK study are expected early in the fourth quarter.

Interim data from an open-label extension study of bimekizumab, a treatment for plaque psoriasis, showed that a majority of patients who responded to the therapy at Phase 3, continued to benefit from the drug after two years. Bimekizumab is currently under review by the US Food and Drug Administration for the treatment of moderate-to-severe plaque psoriasis, a chronic inflammatory skin disease for which there is no cure. In June, it was given a positive opinion by the European Medicines Agency.

AstraZeneca Plc is discontinuing a Phase 3 trial of Ultomiris (ravulizumab), a newly acquired antibody therapy being investigated in amyotrophic lateral sclerosis, due to lack of efficacy. The decision was taken following a recommendation of the trial’s independent data monitoring committee. The trial did not throw up any new safety findings, the company announced on 20 August.

A new version of a Covid-19 vaccine being developed by CureVac NV and GlaxoSmithKline Plc has shown an improved immune response and protection in a preclinical study, compared with an earlier version of the vaccine, the two companies announced on 16 August.

A single dose vaccine in development to protect against the chikungunya virus has been shown to be effective with no safety concerns, the sponsor Valneva SE announced on 5 August. The vaccine VLA1553 met its primary endpoint in a Phase 3 pivotal trial achieving a seroprotection rate of 98.5% - well above the 70% threshold set by the US Food and Drug Administration.

A Phase 3 trial of the checkpoint inhibitor Libtayo (cemiplimab) combined with chemotherapy for patients with advanced non-small cell lung cancer was stopped early for efficacy, the sponsors Sanofi SA and Regeneron Pharmaceuticals Inc announced on 5 August. The combination therapy was found to reduce the risk of death by 29% compared with chemotherapy alone in a trial of 466 patients with locally advanced and metastatic disease.

CureVac NV’s candidate messenger RNA (mRNA) vaccine against Covid-19 has shown an overall efficacy rate of 48% in a large Phase 2b/3 trial during which multiple new strains of the SARS-CoV-2 virus were in circulation. The efficacy rate was achieved against Covid-19 disease of any severity, including single non-respiratory mild symptoms, the developer announced on 30 June.

A genome editing therapy designed to inactivate a mutated gene in the liver has reported positive Phase 1 data – the first therapy of its kind to achieve an effect inside the human body. The drug, NTLA-2001, is intended as a one-time treatment for people living with transthyretin (ATTR) amyloidosis, a rare disease caused by misfolded proteins. The study results were reported on 26 June by Intellia Therapeutics Inc and its partner Regeneron Pharmaceuticals Inc, and simultaneously published in The New England Journal of Medicine.

A Phase 3 study of an experimental gene therapy for choroideremia failed to meet its primary endpoint, and also did not show efficacy on key secondary endpoints, Biogen Inc announced on 14 June. Choroideremia is a rare, inherited retinal disease resulting in progressive vision loss, ultimately leading to blindness.

After interim data failed to meet the statistical criteria for success, CureVac NV is pressing ahead to complete development of its first Covid-19 vaccine while making plans to start studies of a second generation compound later this year. On 16 June, CureVac announced that its messenger RNA vaccine, CVnCoV, demonstrated an interim efficacy rate of 47% in a Phase 2b/3 trial. The outcome was weaker than expected.