Results

Fevipiprant is in clinical development for the treatment of patients aged 12 years and older with uncontrolled asthma who remain symptomatic despite treatment with inhaled corticosteroids (ICS) with or without at least one additional controller. Whilst there is no cure for asthma, most patients are able to control their symptoms by taking daily preventative medication and additional controllers when required. However, a small subset of asthma patients are resistant to the current standard of care for asthma and are unable to control their symptoms. This can have severe implications on their quality of life as uncontrolled asthma can result in decreased physical fitness, decreased sleep quality and decreased productivity at work or school.

The triple fixed-dose combination (FDC), elexacaftor/tezacaftor/ivacaftor-FDC, is in clinical development for cystic fibrosis (CF) that is heterozygous for F508del mutation and a minimal function mutation for patients aged 6 to 11 years old. CF is the most common, life-limiting recessively inherited (a faulty gene inherited from both parents) disease in the UK. Genetic mutations affect the CF transmembrane conductance regulator (CFTR) gene, which is essential for the regulation of salt and water movements across cell membranes. These mutations mean that the CFTR protein is not processed and moved through the cells normally, resulting in little to no CFTR protein at the cell surface. This results in thickened secretions in organs with epithelial cell lining, mainly affecting the lungs and digestive system.

The triple fixed-dose combination (FDC), elexacaftor/tezacaftor/ivacaftor-FDC, is in clinical development for cystic fibrosis (CF) that is homozygous for F508del mutation for patients aged 6 to 11 years old. CF is the most common, life-limiting recessively inherited (a faulty gene inherited from both parents) disease in the UK. Genetic mutations affect the CF transmembrane conductance regulator (CFTR) gene, which is essential for the regulation of salt and water movements across cell membranes. These mutations mean that the CFTR protein is not processed and moved through the cells normally, resulting in little to no CFTR protein at the cell surface. This results in thickened secretions in organs with epithelial cell lining, mainly affecting the lungs and digestive system.

Dapagliflozin blocks the action of a protein in the kidneys called sodium-glucose co-transporter 2 (SGLT2). As blood is filtered by the kidneys, SGLT2 stops glucose in the bloodstream from being passed out into the urine. By blocking the action of SGLT2, dapagliflozin causes the kidney to pass out more glucose in the urine, thereby reducing the levels of glucose in the blood. Blood vessels can be damaged by the effects of high blood glucose levels and this can in turn cause damage to organs, such as the heart. Dapagliflozin may also increase the removal of fluid between tissue cells, contributing to reduced congestion with minimal impact on blood volume. If licensed, dapagliflozin may provide a treatment option for people with HFrEF who currently have limited therapies available.

Dapagliflozin blocks the action of a protein in the kidneys called sodium-glucose co-transporter 2 (SGLT2). As blood is filtered by the kidneys, SGLT2 stops glucose in the bloodstream from being passed out into the urine. By blocking the action of SGLT2, dapagliflozin causes the kidney to pass out more glucose in the urine, thereby reducing the levels of glucose in the blood. Blood vessels can be damaged by the effects of high blood glucose levels and this can in turn cause damage to organs, such as the heart. Dapagliflozin may also increase the removal of fluid between tissue cells, contributing to reduced congestion with minimal impact on blood volume. If licensed, dapagliflozin may provide a treatment option for people with HFpEF who currently have no effective therapies available.