Belzutifan for von Hippel-Lindau disease-associated clear cell renal cell carcinoma – first line
Belzutifan is in clinical development for the treatment of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC). VHL is a hereditary disease that results in the body producing an increased amount of the protein hypoxia inducible factor (HIF) – 2α. This leads to an increased risk of tumours developing in various parts of the body including the kidney. Renal cell carcinoma (RCC) is the most common type of kidney cancer. ccRCC is named as such because when this type of tumour is viewed under a microscope the cells appear clear. Currently patients with VHL disease have limited treatment options which focus on surgically removing tumours once they have developed.
Pembrolizumab for recurrent or metastatic nasopharyngeal cancer
Pembrolizumab is an immunotherapy drug administered by intravenous (IV) infusion. It works by improving the activity of white blood cells (T-cells) by blocking a protein, PD-L1, thereby increasing the ability of the immune system to kill cancer cells. If licenced, pembrolizumab could provide an additional treatment option for patients with platinum pre-treated recurrent or metastatic nasopharyngeal cancer.
Mosunetuzumab for relapsed or refractory B-cell follicular lymphoma – third-line and greater
Mosunetuzumab is in clinical development for the treatment of patients with relapsed or refractory B-cell follicular lymphoma (FL). FL is a type of slow growing blood cancer that affects white blood cells. In FL, the affected white blood cells start to multiply in an abnormal way and begin to aggregate in certain parts of the body such as the lymph nodes. A recurrence of lymphoma after going into complete remission is known as a relapse. Lymphoma that is unresponsive to therapy it is called refractory lymphoma. The condition can become more difficult to treat if it is relapsed or refractory, as treatment options are limited.
Trastuzumab deruxtecan for unresectable or metastatic HER2-low (HR+/-) breast cancer – after chemotherapy
Trastuzumab deruxtecan is in clinical development for the treatment of adults with HER2-low, hormone receptor (HR) positive or HR negative (HR+/-), unresectable and/or metastatic breast cancer (BC) who have previously been treated with chemotherapy. Metastatic BC (stage IV) is when the cancer has spread beyond the breast and nearby lymph nodes, whilst unresectable refers to cancer that cannot be treated by surgery. HER2 receptors help control how a healthy breast cell grows, divides, and repairs itself. In the case of IHC 0 and 1+ results or IHC+ with a negative ISH assay, BC is considered HER2-low (or HER2-negative). Breast tumours are tested for both oestrogen receptors (ER) and progesterone receptors (PR). About 74 % of all BCs are both HR-positive (HR+) and HER2-negative. In England, adults with stage 4 breast cancer, that were diagnosed between 2012-2016 and followed up to 2017, had a 66% survival rate one year after diagnosis.
Durvalumab and bevacizumab in addition to transarterial chemoembolisation for locoregional hepatocellular carcinoma
Durvalumab and bevacizumab in addition to transarterial chemoembolisation (TACE) is in clinical development for treating patients with locoregional hepatocellular carcinoma (HCC). HCC is the most common type of primary liver cancer in adults and the most common cause of death in people with cirrhosis (scarring of the liver). It usually presents at an advanced stage and has a poor prognosis. The current standard of care can only slow the progression of the cancer and extend survival.
Lenvatinib in combination with pembrolizumab for advanced/unresectable hepatocellular carcinoma – first-line
Lenvatinib in combination with pembrolizumab is in clinical development for the treatment of hepatocellular carcinoma (HCC), which is cancer that originally develops in the liver and accounts for nine in ten primary liver cancer cases. Advanced or unresectable HCC occurs when the cancer has spread to lymph nodes or to other organs and cannot be treated by surgery alone. It is often diagnosed late in life and has a poor prognosis. It is a debilitating condition with many distressing symptoms, including pain, digestive problems and weight loss. There are currently limited treatment options, yet research has suggested combining different drugs may improve survival for HCC patients.
Trastuzumab deruxtecan for HER2-positive gastric or gastroesophageal junction adenocarcinoma – third-line
Trastuzumab deruxtecan consists of an anti-HER2 therapy (trastuzumab) combined with a chemotherapy agent (deruxtecan). Trastuzumab specifically binds to cancer cells that are HER2-positive, which provides a targeted delivery of the deruxtecan inside cancer cells to kill them. This reduces “healthy” cells exposure to the chemotherapy with the potential to reduce negative side effects. If licensed, trastuzumab deruxtecan will offer an additional treatment option for patients whose disease progressed despite previous treatment with other anti-HER2 therapies.
Burosumab for tumour-induced osteomalacia
Burosumab is in clinical development for the treatment of adults with tumour-induced osteomalacia (TIO). TIO is a rare disease where an overproduction of FGF23 leads to renal phosphate wasting and low levels of phosphate and vitamin D in the body. This causes weakening (or softening) of bones. Main symptoms include bone pain, muscle weakness/fatigue and fractures and without a timely diagnosis, TIO can lead to a significant decrease in quality of life, severe functional impairment and even prostration.