Clinical Research

A novel form of insulin that is intended to accelerate absorption of the hormone by patients with Type 1 diabetes has met the primary endpoint of non-inferiority in a Phase 1 trial compared with NovoRapid, a standard of care. Arecor Therapeutics Plc, the developer, announced the results on 20 September. AT278 is an ultra-concentrated formulation that has been designed to speed absorption of insulin post injection even when delivered at a high concentration.

Astellas Pharma Inc has reported the death of a participant in a clinical trial of a gene therapy for the treatment X-linked myotubular myopathy, a rare neuromuscular disease caused by mutations in the MTM1 gene. The participant, who developed an adverse event during the trial, passed away on 9 September. “The cause of death is still pending,” the company announced on 14 September.

The US Food and Drug Administration has placed a clinical hold on a Phase 1/2 trial of a gene therapy for adults with phenylketonuria, a genetic disorder caused by the deficiency of an enzyme needed to break down an amino acid in certain foods. The clinical hold was based on findings from a preclinical study showing that six out of seven mice receiving the highest dose of the therapy developed liver tumours. One of the tumours was cancerous. BioMarin Pharmaceutical Inc, the developer, announced the clinical hold on 6 September.

A Phase 3 trial evaluating the Bruton’s tyrosine kinase (BTK) inhibitor rilzabrutinib in pemphigus, a rare autoimmune skin condition, did not meet its primary or key secondary endpoints, the developer Sanofi SA announced on 9 September. No new safety signals were identified.

Two Phase 3 trials of bumetanide, which was being investigated in children and adolescents with autism spectrum disorder, have been discontinued due to a lack of efficacy, the sponsors Les Laboratoires Servier SAS and Neurochlore SAS announced on 7 September.

A Phase 3 trial of Kymriah (tisagenlecleucel), the pioneering chimeric antigen receptor (CAR) T cell therapy, did not meet its primary endpoint of event-free survival in patients with aggressive B cell non-Hodgkin lymphoma, Novartis announced on 24 August.

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.