Liposomal cytarabine-daunorubicin for treating relapsed or refractory acute myeloid leukaemia in paediatric patients.
Liposomal cytarabine-daunorubicin is made up of the chemotherapy drugs daunorubicin and cytarabine contained within fat-based particles called liposomes. Liposomal cytarabinedaunorubicin is delivered by intravenous infusion and provides controlled release of daunorubicin and cytarabine. Daunorubicin works by interrupting the copying of DNA which is necessary for cancer cell growth and cytarabine works by inhibiting DNA production to kill cancerous cells. If licenced, liposomal cytarabine-daunorubicin may offer an additional treatment option for paediatric patients with relapsed or refractory AML.
Bintrafusp alfa for locally advanced or metastatic biliary tract cancer – second-line
Bintrafusp alfa, delivered via intravenous injection, is a novel bi-functional fusion protein that is composed of an antibody fused with a receptor. Chemotherapy aims to kill cancer cells by directly attacking them, whereas bintrafusp alfa works twofold by preventing cancer from being resistant to a patient’s immune system and stopping cancer cells from growing.
Pralsetinib for RET-fusion positive metastatic non-small-cell lung cancer
Pralsetinib is an investigational, once-daily oral precision therapy designed to selectively target, and potentially inhibit, RET mutations, regardless of the tissue of origin. If licensed, pralsetinib will offer a treatment option for adult patients with RET-fusion positive advanced NSCLC, who currently have no highly selective therapies available.
Pevonedistat in addition to azacitidine for Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia and Low Blast Acute Myeloid Leukemia – first-line
Pevonedistat, administered by IV infusion, blocks the activity of an enzyme in the body called NEDD8-activating enzyme (NAE). NAE is involved in the growth and spread of cancer cells. By blocking NAE in patients with AML, pevonedistat is expected to prevent the degradation of certain proteins affecting key pathways including DNA repair, the cell cycle, and cell survival, thereby preventing the development and worsening of cancer. Chemotherapy drugs, such as azacitidine, work in different ways to stop the growth of cancer cells. If licensed, pevonedistat in addition to azacitidine would offer an additional first-line treatment option for adults with HR-MDS, CMML, or LB-AML.
Plinabulin in addition to docetaxel for advanced, metastatic non-small-cell lung cancer – second line or greater
Plinabulin is an intravenously administered drug that binds to and affects the function of the protein tubulin within cells of the body. In certain cells of the immune system called Dendritic Cells (DCs) tubulin targeting with plinabulin activates a protein named GEF-H1 which boosts the ability of DCs to activate T-cells, which go on to kill cancer cells. These effects are thought to contribute towards the anti-cancer benefits of plinabulin. If licensed, plinabulin in addition to docetaxel will provide a new regimen for advanced NSCLC.
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.