Results

Empagliflozin is given by mouth in the form of a tablet and works by inhibiting the sodium transporter NHE1. This prevents salt from being re-absorbed so there is increased excretion of salt from the body and a reduced volume of fluid in the blood vessels. It’s thought that these changes in sugar, salt and water metabolism in the body may contribute to the reductions in cardiovascular death. If licensed, empagliflozin may provide an additional treatment option for people with HFrEF who currently have limited therapies available.

Elexacaftor and tezacaftor are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The triple therapy of elexacaftor/tezacaftor/ivacaftor-FDC may result in an effective therapeutic option for people with CF heterozygous for F508del mutations, who currently have limited options.

Gefapixant is an oral drug that blocks receptors in the sensory cells in the airways and lungs, potentially reducing cough frequency in patients with refractory or unexplained chronic cough. Preliminary results from early studies have demonstrated that gefapixant is efficacious and safe. If licenced, gefapixant could provide a treatment option for patients with refractory or unexplained cough who have no licensed therapies available and few effective therapies available.

Vericiguat is a medicinal product in clinical development for the treatment of heart failure (HF) with reduced ejection fraction. HF is a complex clinical syndrome of symptoms and signs that suggest the efficiency of the heart as a pump is impaired. Symptoms include breathlessness and fatigue, and signs of the condition include swollen ankles and crackling sounds in the lungs. More than half of people with HF have a reduced ejection fraction (HFrEF), also referred to as systolic heart failure, where the heart muscle does not contract effectively, and therefore less oxygen-rich blood is pumped out to the body. There remains a large unmet need for new therapies in the treatment of HFrEF.

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.