Results

Deferiprone is in clinical development for patients with sickle-cell disorder (SCD) and other anaemias that are suffering from iron overload due to frequent transfusions to increase their red blood cell count. SCD is a group of inherited disorders where the red blood cells become hard and sticky and look like a C-shaped farm tool called a “sickle”. The sickle red blood cells die early, and patients often require blood transfusions. Iron overload is an effect of frequent transfusions in SCD. Excess iron in the body can be toxic to major organs like the heart and liver.

Plinabulin in combination with G-CSF is being developed for the prevention of chemotherapy-induced neutropenia (CIN) in patients with solid tumours receiving myelosuppressive chemotherapy with docetaxel, doxorubicin, and cyclophosphamide (TAC). Neutropenia is a condition associated with low levels of neutrophils, a type of white blood cell, which is a common side effect of cancer treatment. Severe neutropenia can lead to life-threatening complications due to resulting infections, and cause reduction or delay of chemotherapy treatment, potentially compromising the benefit to cancer patients.

Imetelstat is in clinical development for the treatment of relapsed/refractory low or intermediate-1 risk myelodysplastic syndrome (MDS) in transfusion-dependent patients, following erythropoiesis-stimulating agent (ESA) treatment. MDS are a group of disorders in which red blood cells, white blood cells, and platelets produced by the bone marrow do not grow and mature normally. MDS are long-term debilitating and life-threatening diseases. MDS patients may require repeated blood transfusions and currently have few treatment options.

Mitapivat is currently in clinical development for the treatment of adult patients with pyruvate kinase deficiency (PKD) who have regular blood transfusions and those who do not have regular blood transfusions. PKD is a genetic blood disorder caused by low levels of the enzyme pyruvate kinase (PK). Low levels of PK result in a deficiency in energy and causes red blood cells to break down too early. This is known as haemolytic anaemia. Symptoms of PKD vary significantly with some patients requiring no treatment and some patients requiring blood transfusions, surgery to remove the spleen or haematopoietic stem cell transplant which are all associated with risks and complications. There are currently no disease modifying treatments approved for the treatment of PKD.

Maribavir, administered orally, is thought to block the action of an enzyme of the virus called UL97 kinase. By blocking the enzyme, the medicine is expected to prevent viruses from reaching maturity, so that no new infectious viruses can be produced. If licensed, maribavir would offer an alternative treatment option for patients with CMV infections that are clinically refractory and/or genetically resistant to GCV, VGCV, CDV or FOS after stem cell or solid organ transplantation.

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.

OTL-103 is administered intravenously. It is made up of immature bone marrow cells (called CD34+ cells) taken from the patient. It works by correcting cells using a modified virus that contains the correct gene for the WAS protein. When these corrected cells are transplanted back into the patient, they populate the bone marrow and produce healthy platelets and immune cells that produce the WAS protein, thereby relieving the symptoms of the disease. If licensed, OTL-103 will provide a treatment option for patients with WAS.

Tecarfarin, a vitamin K reductase, is given by oral administration and works by blocking the liver from using vitamin K to make clotting factors. Clotting factors work with blood cells called platelets that trigger the clotting process to form a blood clot. Stopping the activation of these vitamin K dependent clotting factors means the blood takes much longer to clot so there is a decreased risk of VTE. Tecarfarin works in the same way as warfarin, which is the current best treatment option for preventing VTE, but is expected to be safer and deliver more predictable clotting. If licensed, tecarfarin would offer an additional treatment option for preventing VTE in patients who require long-term anticoagulation therapy.

Avatrombopag is given as an oral tablet and works by mimicking the action of a hormone called thrombopoietin (TPO) which is responsible for causing pre-curser cells to mature into platelets. Avatrombopag binds to TPO receptors resulting in increased platelet production and increased platelet count. Results from clinical studies have demonstrated that avatrombopag is safe and efficacious. If licensed, avatrombopag could offer an additional treatment option for patients with chronic ITP who have had limited response to previous therapy.

Burosumab, administered by subcutaneous injection, is a monoclonal antibody (a type of protein) designed to recognise and attach to the FGF23 protein. By attaching to the FGF23 protein, the medicine blocks its activity, allowing the kidneys to reabsorb phosphate into the bloodstream and restore normal levels of phosphate in the blood. In studies of adults with XLH, burosumab is associated with normalisation of serum phosphorus, restoration of bone quality and improvements in pain, stiffness, fatigue, as well as healing of fractures compared to placebo.