Clinical Research

Daprodustat, a candidate drug for the treatment of anaemia due to chronic kidney disease, has generated positive data in five Phase 3 trials – the first oral inhibitor of an enzyme linked to a protein complex to show efficacy without an increased cardiovascular risk. Data from two of the trials were published in The New England Journal of Medicine on 5 November 2021.

A candidate antiviral for the treatment of Covid-19 has delivered an 89% reduction in the risk of Covid-19 related hospitalisation or death compared with placebo in a Phase 2/3 clinical study, the developer Pfizer Inc, announced on 5 November. On the recommendation of the trial’s independent data monitoring committee, the study was stopped and data will be sent to the US Food and Drug Administration to support an emergency use authorisation.

An experimental breast cancer treatment for patients with estrogen receptor positive and HER2 negative breast cancer has met the primary endpoint in a Phase 3 trial paving the way for regulatory submissions in the US and Europe in 2022, the developers Menarini Group and Radius Health Inc, announced on 20 October.

An antibody combination therapy has shown a statistically significant reduction in severe Covid-19 or death among patients with mild to moderate disease, the developer AstraZeneca Plc reported on 11 October. This follows the successful trial of a small molecule antiviral agent produced by Merck & Co Inc and Ridgeback Biotherapeutics LP for a similar patient population which was announced on 1 October. Using different agents, the two trials both achieved efficacy rates of 50% compared with placebo.

An anti-viral medicine developed jointly by Merck & Co Inc and Ridgeback Biotherapeutics LP, has reduced the risk of hospitalisation or death in patients with Covid-19 by 50% compared with placebo, the two companies announced on 1 October. A Phase 3 trial of the drug, molnupiravir was stopped early and the developers plan to seek an emergency use authorisation from the US Food and Drug Administration in order to get the medicine onto the market quickly. Molnupiravir is a small molecule ribonucleoside analogue that inhibits the replication of the SARS-CoV-2 virus.

A Phase 3 study of Keytruda (pembrolizumab) in Asian patients with advanced hepatocellular carcinoma met is primary endpoint of overall survival, the developer Merck & Co Inc announced on 27 September. Patients in the trial had previously been treated with sorafenib, an approved treatment for kidney and liver cancers. Hepatocellular carcinoma is the most common type of primary liver cancer.

Lynparza (olaparib) has generated more positive data in prostate cancer, this time as a potential first-line treatment for men with metastatic castration-resistant prostate cancer with or without mutations in homologous recombination repair (HRR) genes. A Phase 3 trial comparing Lynparza against placebo when given in addition to the prostate cancer drug abiraterone showed a statistically significant improvement in radiographic progression-free survival, the developers AstraZeneca Plc and Merck & Co Inc, announced on 24 September.

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.