Luspatercept for the treatment of adults with non-transfusion dependent beta (β)-thalassaemia
Luspatercept is under clinical development for the treatment of adult patients with beta-thalassaemia who don’t regularly require blood transfusion. Thalassaemia is a commonly inherited blood disorder resulting from an abnormality in one of the genes that affects the production of haemoglobin, a protein in red blood cells that carries oxygen throughout the body . Beta-thalassaemia is a subtype caused by a specific gene mutation. People with thalassaemia produce either little or no normal haemoglobin. Current treatment options for beta-thalassaemia are limited to blood transfusions with its associated risks and complications.
Dapagliflozin for chronic kidney disease
Dapagliflozin blocks the action of a protein in the kidneys called sodium-glucose cotransporter 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. SGLT2 inhibitors have exhibited beneficial effects on kidney function in those with diabetes and more recently has shown improvements in kidney function and all-cause mortality in those with CKD without a diabetes. If licensed, dapagliflozin may provide a treatment option specifically for people with CKD.
Osimertinib for EGFR-positive non-small cell lung cancer – adjuvant
Osimertinib is in clinical development as an adjuvant treatment for resectable, early stage non-small cell lung cancer (NSCLC). Lung cancer is one of the most common and serious types of cancer and NSCLC is the most common type of lung cancer. An adjuvant treatment is an additional cancer treatment given after the primary treatment, to lower the risk of the cancer returning. Resectable cancer means that the cancer can be removed by surgery, which means the cancer is normally in earlier stages. A proportion of patients have mutations to the protein epidermal growth factor receptor (EGFR) which controls cell growth. There are currently no recommended EGFR-targeted therapies for early stage NSCLC.
Durvalumab in combination with tremelimumab and chemotherapy for unresectable, locally advanced or metastatic urothelial cancer
Durvalumab and tremelimumab are immunotherapies, meaning they target the immune system. Durvalumab binds to a protein called PD-L1 to prevent it from binding its target (PD-1). In doing so, durvalumab allows immune cells, called T cells, to be activated so that they can destroy the cancer cells. Tremelimumab binds a protein called CTLA4 on T-cells, activating them and allowing them to kill cancer cells. Durvalumab and tremelimumab are administered intravenously. If licensed, durvalumab in combination with tremelimumab and chemotherapy will offer an additional treatment option for patients with unresectable, locally advanced or metastatic UC.
CC-486 for angioimmunoblastic T-cell lymphoma
CC-486 is in development for relapsed or refractory angioimmunoblastic T-cell lymphoma (AITL). AITL is a fast-growing type of T-cell Non-Hodgkin Lymphoma marked by enlarged lymph nodes and increased antibodies in the blood. Other symptoms may include a skin rash, fever, weight loss, or night sweats. AITL is more resistant to conventional chemotherapy than other forms of lymphoma and is generally associated with a poor outcome. In relapsed or refractory disease, survival durations are in the range of only a few months meaning there is unmet medical need in this patient population.
Lisocabtagene maraleucel for relapsed or refractory, aggressive B-cell non-Hodgkin Lymphoma – second line
Lisocabtagene maraleucel is an advanced immunotherapy administered intravenously that contains the patient’s own white blood cells (T-cells) that have been modified genetically in the laboratory so that they make a protein called chimeric antigen receptor (CAR). The CAR T-cells attach to a particular protein called CD19 on the surface of cancer cells resulting in the death of these cells. If licensed, will offer an additional treatment option for patients with relapsed/refractory, aggressive B-cell NHL who are not eligible for stem cell transplant.
BMS-986165 for moderate to severe plaque psoriasis
BMS-986165 is in clinical development for moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. Psoriasis is an inflammatory disease whereby the body’s immune system becomes over-active resulting in the life cycle of skin cells to drastically speed up. This causes a build-up of red, scaly, flaky and itchy patches of skin to appear that often involve the knees, elbows, scalp and lower back. Plaque psoriasis is thought to be caused by a combination of genetic susceptibility and triggers such as stress, smoking and hormonal changes. Treatment is determined by the area of skin affected and the severity of the plaque psoriasis and may include a combination of topical, phototherapy and systemic (oral or injected) therapies.
Semaglutide for overweight individuals
Semaglutide is in clinical development for the treatment of overweight and obese individuals. Excess weight can place stress on both mental and physical health, leading a number of complications such as depression, low self-esteem, and increased risk of heart disease, stroke and type 2 diabetes. Weight can be affected by a number of factors such as diet, physical activity, genetics and general health conditions, however diet and exercise are the two main contributing factors. Being overweight is reversible through lifestyle changes such as increased exercise, healthy diet and a net calorie deficit, along with help through counselling and medication. If weight is not able to be controlled, surgery may be required. Therefore, there is an unmet need for pharmacological therapies that target both weight and glucose control.
Ibrutinib in combination with venetoclax for chronic lymphocytic leukaemia or small lymphocytic lymphoma – first line
Ibrutinib in combination with venetoclax is being developed for elderly and at risk patients with chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL). CLL and SLL are types of cancer in which too many white blood cells are produced, in SLL cells build up in the lymph nodes, in CLL the cancer cells can be found in the blood and bone marrow. As these cells develop abnormally, they are unable to function, fight infection and reduce the production of healthy blood cells. These diseases are chronic and develop slowly. Treatment is complex and depends on a number of factors, including the extent of disease, previous treatment, patient’s age, symptoms and general state of health.