Clinical Research

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.

A combination antibody treatment for Covid-19 has met its primary endpoint of improving  survival in patients hospitalised with the disease, the developer Regeneron Pharmaceuticals Inc announced on 16 June. The treatment, REGEN-COV, is a combination of the monoclonal antibodies casirivimab and imdevimab, which are both investigational compounds. The US Food and Drug Administration has given the combination treatment an emergency use authorisation.

The Swedish biotech Modus Therapeutics Holding AB has entered into a partnership with a team at Imperial College London, UK to investigate the compound sevuparin in patients with severe malaria. Modus is currently developing sevuparin for sepsis and septic shock, life-threatening conditions caused by the body’s reaction to an infection. The company has now extended its research to do more work in malaria.

Aspirin has been eliminated as a possible treatment for patients hospitalised with Covid-19. This follows a large randomised clinical trial in the UK that assessed the effects of the compound on the survival of Covid-19 patients and their risk of developing a rare type of blood clot. Aspirin is widely used to reduce blood clotting in other diseases.

argenx SE is to take back rights to the antibody therapeutic cusatuzumab after its partner Cilag GmbH International, one of the Janssen Pharmaceutical Companies of Johnson & Johnson Inc, announced plans to quit the three-year-old collaboration. Cusatuzumab is in a Phase 1b study in newly-diagnosed, elderly patients with acute myeloid leukaemia (AML). It is being tested with venetoclax and the chemotherapy azacitidine.

The scientific journal Nature Communication has published data from a Phase 1/2 study of an autologous faecal microbiota transfer treatment in patients with acute myeloid leukaemia (AML). Top-line data from the study were first presented at the American Society of Hematology Annual Meeting in December 2018. With further outcome data now available, the treatment has been shown to have had a positive impact on survival after two years.

NOXXON Pharma NV has reported more positive data from a study of its RNA oligonucleotide drug, plus radiotherapy, in patients with newly diagnosed brain cancer. The drug, NOX-A12, targets a chemokine protein in the tumour microenvironment with the goal of breaking tumour protection against the immune system.

Sanofi SA has stopped a clinical programme in autosomal dominant polycystic kidney disease after its small molecule drug venglustat failed to show a meaningful reduction in the total kidney volume growth rate. The programme was halted during the first stage of a pivotal Phase 2/3 study. Venglustat was targeting glycosphingolipids (GSL), which modulate membrane-to-protein functions and contribute to cell-to-cell communication.